Immune Pharmaceuticals to expand clinical development plan for Bertilimumab

Immune Pharmaceuticals Inc. announced today that it intends to expand its planned bullous pemphigoid (BP) clinical development for bertilimumab, a fully human monoclonal antibody targeting eotaxin-1. Bullous Pemphigoid is an orphan auto-immune blistering disease of the skin affecting approximately 60,000 patients in the US and Europe. Immune expects to start enrolling patients in a Phase II clinical trial in Israel to be treated during the second quarter of 2015. Additionally, following an International Medical Advisory Board meeting held in San Francisco immediately prior to the American Academy of Dermatology annual meeting, Immune has decided to expand its clinical program to include US centers in the Phase II BP development program. The Company also intends to initiate studies to further investigate the relationship between eotaxin-1 levels and the Bullous Pemphigoid Disease Area Activity Index (BPDAI) and to assess the burden of illness from a medical and economic standpoint. Immune has formed an International Medical Advisory Board for the development of Bertilimumab in BP, with Key Opinion Leaders including Professor Neil Korman (Case Western, Cleveland, USA), Professor Russell Hall (Duke University, USA), Professor Pascal Joly (Rouen, France) and Professor Dedee Murrell (Sydney, Australia).

"Immune Pharmaceuticals is committed to better understanding this orphan disease and improving clinical outcomes and quality of life for patients with Bullous Pemphigoid and other auto-immune diseases," said Dr. Daniel Teper, CEO of Immune Pharmaceuticals.

Immune Pharmaceuticals Inc. applies a personalized approach to treatment, developing novel, highly-targeted antibody therapeutics to improve the lives of patients with inflammatory diseases and cancer.

British researchers discover Anglo-Saxon remedy that kills Superbug MRSA

A 1,200-year-old Anglo-Saxon remedy called Bald’s Eye Salve has proven “astonishingly” effective in battling the MRSA superbug, which kills more than 5,000 people a year in the US.
The potion, composed of garlic, onion or leeks, wine, and ox bile, kills up to 90 per cent of antibiotic-resistant Staphylococcus aureus bacteria in mice, according to scientists at the University of Nottingham.
The Mediaeval treatment was rediscovered by Christina Lee, an associate professor who specialises in disease and disability in the Anglo-Saxon and Viking eras, who translated it from old English.
The recipe, including detailed instructions on how long to chill the ingredients (nine days at 4C), was found in Bald’s Leechbook, a leather-bound medical textbook from the 9^th Century held in the British Library. Microbiologists recreated Bald’s Eye Salve as faithfully as possible, even using a wine from a historic vineyard near Glastonbury, and tested it both in vitro and on wounds in mice.
“We thought that Bald’s eye salve might show a small amount of antibiotic activity, because each of the ingredients has been shown by other researchers to have some effect on bacteria in the lab,” said microbiologist Freya Harrison. “We were absolutely blown away by just how effective the combination of ingredients was.“We tested it in difficult conditions too,” Dr Harrison stated. “We let our artificial ‘infections’ grow into dense, mature populations called ‘biofilms’, where the individual cells bunch together and make a sticky coating that makes it hard for antibiotics to reach them. But unlike many modern antibiotics, Bald’s eye salve has the power to breach these defences.”
A global hunt for new weapons against antibiotic-resistant infections was launched last year, spearheaded by British Prime Minister David Cameron.

New Cancer drugs sales vastly overestimated

A hot new class of drugs designed to help the body's own immune system fend off cancer by blocking a protein called PD-1 is going to be big, but not nearly as big as investors think, according to a new analysis. Former top Nomura analyst Amit Roy, who now runs independent research firm Foveal, thinks sales of drugs inhibiting Programmed Death receptor (PD-1), or a related target PD-L1, will sell much less than expected. "We conclude that the global anti PD-1/PD-L1 market is worth an un-risk-adjusted $10 billion a year, materially less than the optimistic (in our view) $20-30 billion forecasts in the market today," Roy said in a report on Tuesday.
PD-1 is used by tumors to evade disease-fighting cells and the ability to block the protein provides a new weapon in the fight against cancer, prompting multiple drug development programs at leading drugmakersBut PD-1 inhibitors only work for certain groups of cancer patients and several layers of selection are likely to be needed for patients with cancers such as breast and colorectal.The new drugs are currently prescribed until a patient's cancer progresses. But there are a growing number of cases of very long responses, suggesting that shorter therapy may be sufficient.
Reducing the duration of therapy to eight to 16 weeks could shrink the value of the PD-1 market to just $2.9 billion to $5.8 billion, Roy said. Rising sales forecasts for such medicines have been an important driver of share prices in companies in the immuno-oncology space in the last two years.

New data helps doctors with Breast Cancer risks,Treatments

Triple negative is one of four subtypes of breast cancer, and a new report emphasizes how important it is for doctors to identify the risks and treatments for each. For example, triple negative cancers do not respond to certain hormonal therapies that can help other women.
The nationwide data, published in the Journal of the National Cancer Institute and co-authored by the American Association of Central Cancer Registries, the American Cancer Society, the Centers for Disease Control and Prevention, and the National Cancer Institute at the National Institutes of Health  may help doctors identify which patients are at most risk for each type of breast cancer and which treatments may be most effective.
The authors of the paper looked at the impact of a number of genetic, environmental and social factors including ethnicity, race, age and poverty level that may contribute to breast cancer risk.
When a woman is diagnosed with breast cancer, her doctor must identify the subtype in order to provide the best and most effective treatment to help her beat the disease. The four subtypes are classified by their hormone receptor (HR) status and expression of the HER2 gene. They are known as Luminal A (HR+/HER2-), Luminal B (HR+/HER2+), HER2-enriched (HR-/HER2+) or triple negative (HR-/HER2-) breast cancer.
Doctors are now reporting their patients' breast cancer subtypes to cancer registries nationwide, which could help develop further investigations about why certain demographics of women are more likely to develop one subtype or another.

U.S. Cancer incidence and mortality are mostly stable or decreasing

The rate of people being diagnosed or killed by cancer in the U.S. is stable or decreasing for men and women, according to a new report. "For the main cancers, it's really pretty much good news, incidence and mortality is decreasing," said Recinda Sherman, an author of the new report from the North American Association of Central Cancer Registries (NAACCR) in Springfield, Illinois.
A highlight of the report is that for the first time it breaks breast cancer into specific groups based on how it responds to hormones, said Ahmedin Jemal, vice president of surveillance and health service research at the American Cancer Society (ACS). Triple-negative breast cancer, which is the most aggressive type, represents about 13 percent of breast cancers for all women, but represents about 23 percent of breast cancers among black women. Because triple-negative breast cancer is less likely to be caught by mammography, Sherman said there may be opportunities to target black women with messaging about that specific type of breast cancer. The analysis, published in the Journal of the National Cancer Institute, is the latest in a series of annual reports on cancer compiled since 1998 by the NAACCR, the ACS, the Centers for Disease Control and Prevention and the National Cancer Institute. The organizations found decreases over the past 20 years in the number of new cases for several of the major cancers diagnosed among men, including prostate, lung, colon, stomach, brain and throat cancers. There were also decreases among colon, ovary, cervix, oral and stomach cancers among women.
Overall, cancer diagnoses decreased by 1.8 percent each year between 2007 and 2011 among men, and remained stable among women.

Canadian researchers discover breakthrough in Ovarian Cancer war

Queen's University cancer researcher Madhuri Koti has discovered a biomarker that will help lead to better predictions of the success of chemotherapy in ovarian cancer patients. This discovery could lead to better treatment options in the fight against ovarian cancer. Biomarkers are an indicator of a biological state or condition. "Recent successes in harnessing the immune system to combat cancer are evidence for the significant roles of a cancer patient's immune responses in fighting cancer," explains Dr. Koti (Biomedical and Molecular Sciences). "Many of these success are based on boosting anti-cancer immunity via different therapies. Such therapies would prove to be most effective when coupled with markers predicting a patient's eventual response to a specific therapy."
Dr. Koti conducted the study in retrospective cohorts of over 200 ovarian cancer patients.
The study utilized a combination of recent cutting-edge and more established detection technologies for identifying such markers. Initial discovery of these markers was made in frozen tumor tissues accrued from tumor banks such as the Ontario Tumor Bank and the Ottawa Health Research Institute and Gynecology-Oncology and Pathology services of the CHUMHospital Notre-Dame, Montreal.
Phase II validations are currently under way in retrospective cohorts of over 500 ovarian cancer patient tumors accrued from the Terry Fox Research Institute-Ovarian Cancer Canada partnered, Canadian Ovarian Experimental Unified Resource.
A major impact of this discovery is that these novel markers, when used at the time of treatment initiation in the specific type of ovarian cancer patient, will help gynecologic oncologists make decisions on additional treatment needed in these patients, thus increasing the potential for patient survival.
Ovarian cancer leads to approximately 152,000 deaths among women worldwide each year, making it a leading cause of gynecological cancer related deaths in women.

Munich developed new targeted alpha therapy for Bladder Cancer

A new treatment for bladder carcinoma is being developed by the JRC and Munich’s Technical University hospital Klinikum rechts der Isar. Recently, the first in-human tests have been conducted showing positive results.
Bladder cancer is the most common malignancy of the urinary tract. Carcinoma in situ (CIS) is an aggressive form of the tumour located at the surface of the bladder and represents about 10% of all cases. The standard therapy for CIS consists of surgical resection of the main tumour mass followed by intravesical immunotherapy.
Targeted alpha therapy uses an anti-epidermal growth factor receptor (EGFR) antibody that specifically binds to bladder carcinoma cells but not to healthy cells. The antibody is labelled with the short lived alpha emitter (an isotope of bismuth - 213Bi) and instilled in the bladder for 90 minutes. The high energy and very short range (less than 0.1 mm) of the emitted alpha radiation leads to a very effective and selective killing of the tumour cells while sparing surrounding healthy bladder tissue.
Within the last 12 months, six patients that had failed all standard treatments were treated with this new therapy. In three patients the tumor was eradicated following a single treatment, and the patients' bladders were preserved. No signs of toxicity or side effects were observed. In the other three cases, residual tumor was still detectable after the treatment. Increasing the applied dose or number of treatments is expected to further improve the therapeutic success rate. Patient recruitment for further tests is currently ongoing.

Dr Fisher and Dr Slamon, two key Warriors in Breast Cancer war

Dr. Fisher caused international controversy by suggesting that cancer cells metastasized throughout the body rather than spread like a stain. Based on that idea, he recommended local lumpectomies to remove breast tumors rather than disfiguring radical mastectomies, the traditional treatment of the time that involved removal of the breast, large areas of muscle and various lymph nodes.
Accused of endangering women’s lives, Dr. Fisher became “the most hated doctor in the history of mankind,” but his ideas won the day when studies proved that lumpectomies had survival rates equal to radical mastectomies. Metastasis now explains how cancers spread.
Dr. Slamon, chief of the Division of Hematology/​Oncology and executive vice chairman for research for the University of California, Los Angeles’ Department of Medicine, would help develop Herceptin, the common immunotherapeutic drug to treat HERS2-positive breast cancer. He pressured Genentech Inc. to conduct human clinical trials to test the drug with HER2 representing 20 percent of all breast cancers. This pioneer aided in the push in immunotherapeutic cancer drugs, he played an aggressive role in pressing to translate breakthroughs into treatments.

Duke researchers discover Polio virus cure for Cancer

An experimental program at Duke University is using a surprising new weapon in the war on cancer, infusions of the polio virus that have successfully been used to treat patients with inoperable brain tumors. While the doctors and researchers spearheading the effort are reluctant to use the words “cancer cure” to describe the work, the early success of the innovative effort is at least the next best thing. If ongoing clinical trials of the technique continue to prove promising, it could add a new way to treat cancer, alongside surgery, radiation, and chemotherapy. “The idea of targeting cancer with viruses has been around for at least 100 years,” notes Matthias Gromeier, M.D., one of the lead investigators heading up the new anti-cancer research at the Preston Robert Tisch Brain Tumor Center at Duke.
“However, valid strategies of using ‘oncolytic’ (cancer-fighting) viruses emerged only recently. This is mostly due to technological advances in genetic engineering of viruses.”
The Duke project involves injecting a genetically engineered polio-virus, known as PVS-RIPO into deadly brain tumors. Early testing involving primates and human patients has found PVS-RIPO homes in on cancer cells and destroys them, without harming healthy tissues.
Dr. Gromeier explains that the Duke team essentially disarmed the virus through genetic manipulation, so it cannot cause polio, while maintaining its ability to infect, target, and kill certain cells, specifically brain tumor cells.

UK Cancer survival rates trail 10 years behind other European countries

The Concord-2 global study looked at survival rates in 67 countries for patients diagnosed with lung, breast, colon and stomach cancers in 1995 to 1999, compared with levels in 2005 to 2009.
It acknowledged there had been improvements in rates in the UK, where cancer survival has doubled in the last 40 years, but not enough to catch up with levels achieved in many European countries a decade earlier. Macmillan found that by comparison UK cancer survival rates were “stuck in the 1990s”. One of the most stark examples was lung cancer, which only 7% of patients survived in the 1990s in the UK. The rate improved to 10% a decade later but this was still behind a 14% survival rate achieved in Austria in the 1990s. By the 2000s 18% of patients diagnosed with lung cancer in Austria survived, almost twice the rate in the UK. Five other European countries (Finland, Germany, Italy, the Netherlands and Norway) also recorded better survival rates for lung cancer in the 1990s than Britain in the 2000s. A similar pattern emerged for breast cancer. In the past decade the survival rate was 81% in the UK, a level exceeded 10 years before in Sweden, France and Italy. For colon cancer six European countries (Finland, France, Italy, the Netherlands, Norway and Sweden) had better survival rates in the 1990s than Britain achieved 10 years later. In the 2000s 19% of British patients diagnosed with stomach cancer survived. Better survival rates were recorded a decade earlier in Austria, Germany, Italy, Norway and Sweden. Macmillan said the figures showed that much better survival rates were achievable in the UK. Its chief executive, Lynda Thomas, said: “Because UK cancer survival rates are lagging so far behind the rest of Europe, people are dying needlessly. Frankly, this is shameful. If countries like Sweden, France, Finland and Austria can achieve these rates, then the UK can and should, bridge the gap.”

Duke researchers use Polio virus to cure Cancer

Like many original ideas, Matthias Gromeier's notion that polio might kill cancer tumors was met with disdain. But two decades later, the use of the virus known for crippling and killing millions is showing promise against one of the most virulent forms of cancer, glioblastoma brain tumors. Two patients Scott Pelley meets in the first clinical trial for the treatment have been declared cancer free by doctors. Pelley's report, in which 60 Minutes cameras spent 10 months capturing patients receiving the therapy and learning of its effects, will be broadcast on Sunday, March 29 at 7 p.m. ET/PT.
"I got a range of responses, from crazy to you're lying, most people just thought it was too dangerous," says Gromeier, a molecular biologist, when he started pushing his idea to attack tumors with the polio virus. One of those naysayers was Dr. Henry Friedman, a neuro-oncologist who is the deputy director of the Brain Tumor Center at Duke University.
"I thought he was nuts," Friedman tells Pelley. "I really thought he was using a weapon that produced paralysis." That was 15 years ago. Today, after research, animal trials and now this human clinical trial, he is more than optimistic. "This, to me, is the most promising therapy I have seen in my career, period." Friedman has been researching a cure for glioblastoma for more than 30 years.
Gromeier's research yielded a genetically modified polio virus that could be used safely in animals and now, it seems, in humans. He explains how it works. "All human cancers, they develop...protective measures that make them invisible to the immune system and this is precisely what we try to reverse with our virus," he says. "We are actually removing this protective shield, enabling the immune system to come in and attack."

Oncolytics' Cancer drug Reolysin gets orphan status in EU

Reolysin, has been granted orphan drug status by the EMA for the treatment of ovarian, fallopian tube and primary peritoneal cancers. Oncolytics Biotech Inc. is also seeking orphan drug status for the candidate for the treatment of pancreatic cancer in the EU. According to the company, ovarian cancer affects approximately 150,000 women at any given time in the EU and has a prevalence of approximately three women per 10,000. The EMA usually grants orphan drug status to drugs that are being developed for the treatment of life threatening and debilitating diseases affecting no more than five in 10,000 people in the EU, for which no satisfactory method of treatment, prevention or diagnosis exists. This designation allows the product to enjoy a 10-year period of marketing exclusivity in the EU, upon approval. Incentives that come with orphan drug status include protocol assistance and potential fee reductions. Reolysin already has orphan drug status for four indications (primary peritoneal cancer, fallopian tube cancer, ovarian cancer and pancreatic cancer) in the U.S. Oncolytics is also seeking orphan drug designation for Reolysin in the U.S. for high-grade gliomas in pediatric patients. Meanwhile, Reolysin is currently in several studies for a wide range of cancers including advanced or metastatic colorectal cancer and recurrent or metastatic pancreatic cancer.

Drug Xtandi positive results in Prostate Cancer study

Earlier this week, Medivation, Inc. and Astellas Pharma Inc. jointly announced new data from a randomized, double-blind phase II study (TERRAIN) on Xtandi. The study compared the efficacy and safety of Xtandi to AstraZeneca’s  Casodex for the treatment of metastatic castration-resistant prostate cancer (CRPC) in patients whose disease progressed following a luteinizing hormone-releasing hormone (LHRH) analogue therapy or after surgical castration. In the TERRAIN study, Xtandi showed an improvement over the standard practice of the adding Casodex to a LHRH therapy. The study successfully met its primary objective of a statistically significant increase in progression-free survival (PFS) under treatment with Xtandi compared to Casodex. The median PFS in the Xtandi arm (15.7 months) was 9.9 months longer than that in the Casodex arm (5.8 months). Median time on treatment was longer for Xtandi (11.7 months) compared to Casodex (5.8 months). However, a higher proportion of patients (31.1%) in the Xtandi arm reported serious adverse events compared to those in the Casodex arm (23.3%). Moreover, common adverse events were reported by a higher percentage of Xtandi-treated patients versus patients administered with Casodex.Meanwhile, Medivation also provided results from the final overall survival (OS) analysis of a placebo-controlled phase III study (PREVAIL) on Xtandi for the treatment of chemotherapy-naive metastatic CRPC patients whose disease progressed on androgen deprivation therapy. The co-primary endpoints of the study were OS and radiographic PFS. The analysis showed that Xtandi led to a statistically significant OS benefit with a 23% reduction in risk of death and a 4-month improvement in median survival over placebo. The above data were presented at the European Association of Urology Congress held at Madrid, Spain.

Theranostic Nanoparticles for Cancer Treatment

Theranostic medicine is based on the concept of delivering both therapeutic and imaging agents to the same targeted location in the body, using a single delivery platform enabled by nanotechnology. These nanosystems can improve drug delivery and diagnosis, and also monitor therapeutic responses to medication. These capabilities will play an important role in the progress of personalized medicine.
As part of a partnership with the National Cancer Institute's Alliance for Nanotechnology in Cancer, researchers at Emory University are attempting to develop a multifunctional theranostic nanoparticle platform that combines the receptor specificity and imaging capability of the nanoparticles with innovative drug delivery systems targeted at specific tumors.
This drug delivery platform leverages the unique pharmokinetic properties and surface functions of magnetic iron oxide nanoparticles, and offers a solution for overcoming intrinsic and physical barriers that cause drug resistance in pancreatic cancer.
Doctors Mao and Yang led the research team, which aims to develop the magnetic iron oxide nanoparticle (IONP) based nanoconstructs targeted to cellular receptors, such as the urokinase plasminogen activator receptor (uPAR). These nanoconstructs enable penetration of drug-carrying nanoparticles into the endothelial cell layer of the tumor, destruction of tumor stromal fibroblasts, and also enhance intracellular drug delivery by receptor-mediated endocytosis.
The research team are also working on methods and strategies for controlled release and loading of multiple or single therapeutic agents such as small molecules, chemotherapy drugs and siRNA-expressing DNA cassettes, into the pancreatic cancer cells.

Personalized gene therapy to fight against Cancer

Gene therapies have already found clinical successes in the treatment of several non-cancer diseases, though the oncolytic or immune system-assisted destruction of tumors relies on specific mechanisms not necessarily transferrable from other gene therapies. "Gene therapies for cancer are ideal and potentially much more effective for certain cancer types when using a targeted approach (cancer cell specificity) and recruiting the patient's own immune system," said Bruce Carlson, Publisher of Kalorama Information. "Advanced forms of cancer are able to progress due to the immune system's inability to recognize the threat."
The following is a partial review of prospective clinical gene therapies for cancer that are in Phase III trials:

• Advantagene's Gene Mediated Cytotoxic Immunotherapy (GMCI) gene therapy technique is featured in the Phase III study begun 2011 for newly diagnosed prostate cancer. The GMCI ProstAtak therapy uses an adenovirus vector to deliver (mediate) the delivery of a herpes simplex virus thymidine kinase (tk) gene to tumor cells at the site of the injection. The tk gene works as a 'suicide gene' that allows for the enzymatic conversion of a non-toxic, antiviral drug Valacyclovir into a cytotoxic drug that causes tumor cell death during radiotherapy.
• VBL Therapeutics plans to begin Phase III trials for its VB-111 lead product for the treatment of recurrent glioblastoma multiforme in mid-2015. The gene therapy targets a highly malignant type of brain tumor that generates vasculature tissue in a process known as angiogenesis. Angiogenic tumors are the target of VBL's Vascular Targeting System (VTS). VB-111 is intended for combination with chemotherapy and radiotherapy.
• Amgen demonstrated in its recent Phase III trial that its talimogene laherparepvec (T-Vec) gene therapy resulted in tumor shrinkage and remission for metastatic melanoma patients. Amgen is seeking to improve its T-Vec therapy in clinical trials for melanoma by pairing it with oncology drugs.
• Cold Genesys is advancing to Phase III trials for its adenovirus-mediated oncolytic gene therapy targeting invasive bladder cancer. Similar to T-Vec, Cold Genesys' CG0070 modified virus contains a cancer-specific promoter sequence and GM-CSF-encoding sequence that serves to selectively lyse cancer cells and release GM-CSF antigen to train the immune system. Cold Genesys underwent a round of financing in mid-2014 with the goal of funding CG0070's Phase III trials.

Novogen's experimental Cancer Drugs are making headlines

Novogen Limited, an Australian and U.S. biotechnology company focused on new treatments for cancer, is making headlines recently as its pre-clinical family of drugs is demonstrating the ability to attack and kill cancer stem cells in vitro, thus allowing for the possibility of a much more effective and directed chemotherapy that isn't any more toxic to non-cancer cells in the body. Cancer stem cells are a key driver of the disease's spread even after treatment as they can self-renew and regenerate a tumor after it has been treated by existing chemotherapy drugs. NVGN believes that its drugs will be able to demonstrate successful cancer remission once it conducts clinical trials, thus making its chemotherapies the ideal compliment or replacement to treatments for all types of cancers.
Novogen has four novel cancer treatments in its pipeline. The first for ovarian cancer is backed by Yale studies; Phase 1 is projected to start Q3 of 2015. 

Fighting Cancer and Ebola with Nanoparticles

Nanotechnologies, the manipulation of matter at a molecular and even atomic scale to penetrate living cells, shows promise of opening a new front against deadly conditions from cancer to Ebola.

According to Dr Thomas Webster, the chair of chemical engineering at Northeastern University in Boston, research into medical nanotechnology is gaining pace and the medical establishment is starting to sit up and pay attention.

At the core of the technology is the ability to attach drugs, and in some cases metals and minerals, to nanoparticles that would then bind themselves to life threatening cancer cells or viruses.

Dr Webster's team is developing methods to attach gold nanoparticles to cancer cells.

Infrared light would then heat up the nanoparticles, killing the cancer cells with heat but leaving the healthy cells alive to do their job.

"This technology has been studied for the better part of a decade, but we're looking at ways of making it better," Dr Webster stated. "One that we've created in the lab we've called 'nanostars.'

"A star shape has a lot more surface area, so they can kill cancer cells faster than a nanosphere because they heat up faster. "Even if it's carrying a drug, a star has a lot more surface area on which to attach it, it's got a different morphology."

 Dr Webster said that research into nanoparticle selenium -- an essential trace mineral in the human die, as a nano-scaled tool in the fight against cancer was the latest avenue of study.

"Selenium is a natural part of our diet, but we've made nanoparticles of selenium that we're seeing in the same way as we've seen with gold and infrared, having the ability to kill cancer cells and kill bacteria and at the same time not have any toxicity problems you might see with anti-bacterials like silver."

New treatments extend lives of patients with Lung Cancer

New and experimental drugs are extending the lives of people with the deadliest forms of cancer. At the University of Colorado Cancer Center in Aurora, Dr. Ross Camidge leads clinical trials for lung cancer, which kills more people each year than breast cancer, colon cancer and pancreatic cancer combined. Camidge calls them “niche-busters”, targeted therapies that dig deep into the profiles of each individual cancer. Researchers have discovered that just as individual patients have different genetic make-ups, so do their tumors. The revolution in the past decade has been in diagnosing and treating cancer at the molecular level, says Camidge.

“It’s not one miracle drug,” says Camidge. “It’s lots of different miracles for lots of different cancers.” The clinical trial uses a combination of dabrafenib and trametinib. British pharmaceutical company GlaxoSmithKline won licenses for the two drugs in 2013 to treat melanoma for patients who exhibited a mutation in a gene called BRAF.

The mutation is common in melanoma but rare among patients with lung cancer. There are only around 4,000 people nationwide who have the mutation. Fewer than three dozen people around the country are enrolled in a trial to see if the drugs can also slow tumor growth in lung cancer as they do for melanoma.The University of Colorado Hospital is among the top-ranked cancer treatment centers in the nation, according to U.S. News and World Report. Outcomes for its cancer patients routinely top state and national rates. The Cancer Center is one of 68 institutions given a special designation by the National Cancer Institute to conduct cancer research, the only such facility in the state.

Australian Prostate Cancer discovery offers new treatment

Researchers in Sydney have opened the door to a new treatment for prostate cancer. They've identified three specific nutrients that prostate cancer cells need to grow. Every year in Australia, more than 3,000 men die from prostate cancer and more than 20,000 men are told they have the disease. There may be a new treatment available, thanks to a breakthrough in research conducted by a team led by Associate Professor Jeff Holst at Sydney's Centenary Institute.
Jeff Holst states that what was discovered this time around is that prostate cancer cells increase one of the pumps that bring a nutrient called glutamine into the cells. If we can block the pumps that bring glutamine into the cells, then we can actually starve the cancer cells and stop them from growing.
If there's an increase in number of these pumps, that then gives us the ability to try and target those pumps and block them selectively in the cancer cells compared to the normal cells around them. At the moment there's a lot of good therapies but no really good therapies for late stage prostate cancer. And that's where these sort of compounds come in, if they can starve the cancer cells, they will stop them from growing, a lot of men will die of old age rather than dying of prostate cancer. The next step involves identifying a drug to switch off the nutrient pump. They, together with some researchers at the University of Sydney, have the first such compounds that they are currently testing, first of all in the cell models, but also they're moving into what are called tumor explants where they take a piece of a patient's prostate, grow it in a dish and put these compounds on and see if it's able to stop the cells growing in that context.

Nanotechnology shows promise for treating Pancreatic Cancer

Scientists at UCLA's California NanoSystems Institute and Jonsson Comprehensive Cancer Center have combined their nanotechnology expertise to create a new treatment that may solve some of the problems of using chemotherapy to treat pancreatic cancer.
The study describes successful experiments to combine two drugs within a specially designed mesoporous silica nanoparticle that looks like a glass bubble. The drugs work together to shrink human pancreas tumors in mice as successfully as the current standard treatment, but at one twelfth the dosage. This lower dosage could reduce both the cost of treatment and the side effects that people suffer from the current method. The study was led by Dr. Huan Meng, assistant adjunct professor of medicine, and Dr. Andre Nel, distinguished professor of medicine, both at the Jonsson Cancer Center.
Recent research has found that combining Gemcitabine with another drug called Paclitaxel can improve the overall treatment effect. In the current method, Abraxane, a nano complex containing Paclitaxel, and Gemcitabine are given separately, which works to a degree, but because the drugs may stay in the body for different lengths of time, the combined beneficial effect is not fully synchronized.
"The beauty of the silica nanoparticle technology is that Gemcitabine and Paclitaxel are placed together in one special lipid-coated nanoparticle at the exact ratio that makes them synergistic with one another when co-delivered at the cancer site, giving us the best possible outcome by using a single drug carrier," Meng said. "This enables us to reduce the dose and maintain the combinatorial effect."

Japanese researchers find heart drug reduces risk of Cancer Spreading

Most people who die from cancer do so because their tumour has spread, or metastasised. Yet most of today's cancer drugs don't stop metastasis, they just kill any cancer cells they come into contact with.

The hope is that the compound could be part of a new class of drugs designed to block tumor spread. "This could be a very important advance," says Andrew Reynolds of the Institute of Cancer Research in London. Cancers are much easier to treat if they have not yet spread.

A few years ago, a team led by Takashi Nojiri of Osaka University in Japan was exploring whether giving a drug called Atrial Natriuretic Peptide (ANP) to patients before lung cancer surgery could reduce subsequent heart problems. ANP is a signalling molecule found in the heart and has been used as a treatment for heart failure in Japan for 20 years.

This approach worked, and it also had another benefit. Two years later, 91 per cent of people treated with ANP were free from secondary tumors, compared with 75 per cent of a control group.

Because ANP affects the blood vessels rather than the cancer cells, it could be used for all kinds of tumors, says Nojiri, who is working with the Japanese drug company Shionogi to turn ANP into a cancer drug.

As in Nojiri's study, if given before surgery, it could be used to reduce the chance of the operation in helping the cancer tumors spread elsewhere in the body. It is thought that cutting into the tumor sometimes lets cancer cells escape. ANP could also be used as a general anti-metastasis drug, given whether or not people need cancer surgery.

UK researchers find new potential for personalized treatments in Bowel Cancer

Cancer Research UK researchers at the University of Birmingham have found that certain genetic flaws in bowel cancer are more likely to trigger an immune response at the site of tumors, meaning that treatments to boost this immune response further could potentially be helpful for these patients.
Finding out what’s happening in a cancer patient’s immune system can be difficult and takes time.  These findings suggest that genetic profiles of patients’ tumors could be used as an easy and fast way of diagnosing whether they are suitable for immunotherapy treatments, and if so which ones.
Cancer Research UK’s FOCUS4 trial is already using the genetics of bowel cancer to offer patients stratified medicine and this study suggests that this could further expand the work to include immunotherapies.
Gary Middleton, Professor of Medical Oncology at the School of Cancer Sciences at the University of Birmingham, said: “The field of immunotherapy is gaining lots of momentum and this study shows a new finding for bowel cancer.  We are already using genetic profiling for stratified medicine in bowel cancer in the FOCUS4 trial. But this research indicates that we could marry immunotherapy with the work we are already doing to personalize treatment even more.”
Researchers used The Cancer Genomic Atlas, a large database, to study this relationship.  From this research, scientists can now start looking at what causes a weak immune response and in the future, could target drugs to switch off the immune suppression associated with certain genetic mutations.

Saccharin to be used to treat Aggressive Cancers

Saccharin is utilized as a sweetener in many sugar-free products, and now researchers are proposing that it could be used as a key ingredient in new drugs for treating aggressive cancers with fewer side effects. "It never ceases to amaze me how a simple molecule, such as saccharin, something many people put in their coffee every day, may have untapped uses, including as a possible lead compound to target aggressive cancers," says study author Robert McKenna, PhD, from the University of Florida. Saccharin's potential use in the development of new anticancer drugs is due to the way in which it binds to and deactivates a protein called carbonic anhydrase IX. This protein is found in some aggressive cancers and plays a role in the proliferation of these cancers in the brain, breast, kidneys, liver, lungs and pancreas.
Due to the way in which saccharin interacts with carbonic Anhydrase IX, the researchers want to use it as a base for drugs that could restrict the growth of cancer and make cancerous cells potentially more vulnerable to other therapies such as chemotherapy and radiation treatment.
Carbonic Anhydrase IX regulates the pH of cancer cells and their surroundings, maintaining levels of acidity that are optimum for the growth and spread of cancer to other parts of the body. As carbonic anhydrase IX is not typically found in most healthy human cells, McKenna states that it is a prime target for anticancer drugs.

Eli Lilly & Innovent Biologics of China announce collaboration of Cancer Treatments

Eli Lilly and Company and Innovent Biologics, Inc. announced the companies will collaborate to support the development and potential commercialization of at least three cancer treatments over the next decade. Innovent is a leading biopharmaceutical company in China focused on the development and manufacturing of complex, high-end biologics for both the Asian and global markets that meet EMA and FDA/cGMP standards. Innovent will lead the development and manufacturing for the China market, while Lilly will be responsible for commercialization of the three potential medicines. Innovent also has co-promotion rights. Innovent will receive a total upfront payment of $56 million. Lilly could also issue future payments exceeding $400 million for the pre-clinical immuno-oncology molecule if the product reaches certain development, regulatory and sales milestones.
Lilly will contribute its cMet monoclonal antibody gene for possible treatment of non-small cell lung cancer. Innovent will contribute its monoclonal antibody targeting protein CD-20 for investigation in hematologic malignancies. Innovent will contribute a pre-clinical immuno-oncology molecule for development in China. The companies have agreed that Lilly will be responsible for development, manufacturing and commercialization of this molecule outside of China.
Lilly will also receive rights to develop and commercialize up to three pre-clinical bispecific immuno-oncology molecules outside of China.

New Zealand researchers hail 'pivotal breakthrough' in Cancer treatment

Researchers are hailing a "pivotal milestone" in the treatment of deadly types of Stomach and Breast Cancer that they hope will offer an alternative to a main treatment involving the removal of a patient's entire stomach.
University of Otago researchers established a genetic connection in early stage diffused stomach cancer and lobular breast cancer, and have spent nearly 20 years hunting for better ways to fight the disease. "What we're trying to do and we think we've finally broken the back of the problem is identify this class of drugs that can be used to prevent the cancer developing in the first place," Professor Parry Guilford said.
The genetic mutation is hereditary, with around 20 families in New Zealand known to have the mutated gene.
Worldwide there's around 400 cases, with that number on the rise as more are discovered.
The drug development has the potential to treat hundreds of thousands of more common forms of cancer, meaning it could be up to 10 years before a new treatment is released.

Novel therapy for Hereditary Breast/Ovarian Cancer

European scientists recently discovered a novel therapy to treat a subgroup of patients with hereditary Breast/Ovarian Cancer. Both the EU and the US have approved an accelerated procedure to market this promising new treatment with few side effects. They have now also developed a special technique to keep tumor tissue alive outside the human body. They can use this to identify patients that are likely to respond to the new therapy. The first results suggest that more than a thousand patients in the Netherlands alone may benefit from the new treatment every year.
Most patients with hereditary Breast Cancer have so-called BRCA1 or BRCA2 mutations. These strongly increase their risk of Breast Cancer and Ovarian Cancer. However, the mutations are also the weakness of the tumor cells. The novel treatment exploits this weakness to kill cancer cells and leave healthy cells unharmed. BRCA proteins are involved in repairing breaks in the DNA double helix. Such breaks may cause cell death when left un-repaired. Healthy cells have two methods of repairing these breaks.Tumors with a BRCA defect have lost one of these methods, leaving them with only one.
Researchers at Erasmus MC (Rotterdam, the Netherlands) and LUMC (Leiden, the Netherlands) developed a technique to identify patients that may benefit from the treatment. The new technique enables scientists to keep a biopsy from the tumor alive outside the patient's body. As a result, it is possible to test the treatment on the patient's own tissue. This effectivity test is an important step towards personalized medicine.

Scientists discover how to change Leukemia cells into Immune cells

A team at Stanford's School of Medicine has reportedly uncovered a potent new treatment method for combating one of leukemia's most aggressive forms. While survival rates for B-cell acute lymphoblastic leukemia, a particularly nasty form of white blood cell cancer, have risen to about 85 percent over the past decade thanks to the advent of stem cell therapies, the prognosis for this disease in the presence of a Philadelphia chromosome mutation remains quite poor. Due to a chance observation by Dr. Scott McClellan, the Stanford team believes it's figured out way to neutralize the disease using its own cancerous cells against it.
Dr. McClellan noticed that a number of leukemic cells had transformed from cancerous cells into "mature" macrophages. These are a type of immune cell tasked with not only directly consuming cellular debris, pathogens and cancers but also with recruiting other immune cells for the fight. "B-cell leukemia cells are in many ways progenitor cells that are forced to stay in an immature state," Dr. Ravi Majeti, an assistant professor of medicine at Stanford, said in a statement. But with the addition of various transcription factors, proteins capable of latching onto and activating specific DNA sequences, Majeti and McClellan essentially forced these deadly cancer cells to "grow up" into fully formed macrophages.
What's more, the researchers discovered that this process didn't just neutralize that specific cancer cell. It also directed the newly made macrophage to begin digesting the cancerous plague and fire-up the rest of the immune system to fight the mutation. "Because the macrophage cells came from the cancer cells, they will already carry with them the chemical signals that will identify the cancer cells, making an immune attack against the cancer more likely," Majeti said.

Startup uses Artificial Intelligence to find Cancer drugs

It’s called BPM 31510. It’s one of many cancer drugs being developed in Massachusetts, but it’s different. BPM 31510 may be the first drug candidate that did not come from a scientist running experiments based on a hypothesis. Instead, it came from artificial intelligence.

They’re processing tissue, urine and blood samples, both from thousands of cancer patients and from thousands of healthy patients, many samples that were taken over long periods of time. Narain says the machines are churning out huge amounts of valuable raw data. What makes Berg different is that it built an artificial intelligence system. The complex computational platform processes trillions of data points to identify molecules that may be effective drugs.

“So we’re not dismissing the scientific process,” Narain said. “We’re just kind of putting one step ahead of it. Instead of the human brain deciphering or proposing those hypotheses, we’re allowing the data from the patient biology to generate those hypotheses.”

At least so far, trying something new seems to be working. Berg’s computational tools have identified drug candidates for different cancers and for Alzheimer’s and Parkinson’s diseases.They’ve done so in a relatively short amount of time.“You have all this genomic data plus all these other molecular measurements,” Butte said. “All this computational work to figure out what’s causing what. And if that works, they’re going to be the first to come up with the first data-driven drugs.”

Cancer therapy 'tumor sanctuaries' the breeding ground of drug resistance

Tumors acquiring resistance is one of the major barriers to successful cancer therapy. Feng Fu, Sebastian Bonhoeffer (ETH Zurich) and their collaborator Martin Nowak (Harvard) use mathematical models to characterize how important aspects of tumor micro-environment can impair the efficacy of targeted cancer therapies.Failure of cancer therapy is commonly attributed to pre-existing resistant mutants already present prior to treatment. The research highlights the important role of tumor sanctuaries in the rapid acquisition of resistance.
The authors offer an in silico model for predicting treatment outcomes that depend on the tumor micro-environment within a solid tumor or across metastases. The results show that resistance in non-sanctuary sites is likely originated from sanctuaries with little drug exposure.
There is increasing evidence that the tumor microenvironment influences cell sensitivity to drugs mediating the evolution of drug resistance. The results provide new insights into understanding why cancers tend to quickly become resistant, and that cell migration and the presence of sanctuary sites with little drug exposure are essential to this end.
"In order to improve our ability to fight against cancer, not only we should search for more effective therapies that sufficiently target tumor genetic heterogeneity, but also such efforts will have to be paralleled with finding novel delivery approaches aimed at eliminating potential tumor sanctuaries."

A drug Gleevec for Brain Cancer

The drug Gleevec (imatinib mesylate) is well known not only for its effectiveness against chronic myeloid leukemia (CML) and acute lymphoblastic leukemia, but also for the story behind its development. The drug was specifically designed to target an abnormal molecule, a fusion of two normal cell proteins, that fueled a tumor’s growth.
A similar drug might be able to tame some brain cancers, new research from Columbia University Medical Center has shown. A team led by Antonio Iavarone, MD, professor of neurology and of pathology and cell biology, Institute for Cancer Genetics, previously discovered that a fusion of two proteins (present only in cancer cells and different from the two in CML) drives some cases of glioma, a common form of brain cancer.
The team’s most recent study looked closely at two patients affected by recurrent Glioblastoma with the fused proteins, in a first in-human trial of a drug that targets half of the fusion protein. Those patients, the researchers found, responded particularly well to the drug, with clinical improvement and radiological tumor reduction. The responses lasted 115 and 134 days, respectively.
“This suggests that if we developed a drug that hits the fused protein more precisely, while leaving normal cells alone, we may get even better results,” said Dr. Iavarone. “The real test of that will have to wait for the development of such a drug and the clinical trials.”
The study also found the fused protein in a significant fraction of the 795 Glioma cases they examined, indicating that a smart drug that targets the fused proteins could have a meaningful impact.

Rare African bush may help Kidney Cancer treatment

New University of Leeds research has shown why a bush that is only found in some African countries could hold a key to killing renal (kidney) cancer cells.
Phyllanthus engleri, also known as spurred phyllanthus, is only found in Tanzania, Zambia, Malawi, Zimbabwe and Mozambique. A research team led by Professor David Beech, of the School of Medicine at the University of Leeds, has discovered that Englerin A in very small amounts activates a particular protein, TRPC4, and its close relative TRPC5. This triggers changes in the renal cancer cell which kills it. About 10,000 new cases of renal cancer are diagnosed each year in the UK. Half of those survive at least 10 years beyond diagnosis.
Professor Beech said: “This unexpected discovery is exciting because it means we could develop new cancer drugs towards these particular proteins, TRPC4 and TRPC5. Englerin A is particularly interesting because it is selective, it only kills renal cancer cells and a few other types of cancer cell. Other cell types are resistant to it, so we think Englerin A has a great deal of potential."

Senate,slams big Pharmas for pushing costly Cancer drugs with limited benefits

In an explosive submission to a Senate inquiry, the medical brains who decide which drugs get government subsidies have lashed out at some of the big pharmaceutical company's strategies.
The Pharmaceutical Benefits Advisory Committee (PBAC) typically deliberates behind closed doors but has taken the unusual step of making a public submission.
In the submission it complained that some companies push costly cancer drugs which add little to a patient's life expectancy. It also accused some companies of using the timing of their submissions to the PBAC as a wedge to maximise their chances of approval by, for example, submitting an application for a government subsidy before the regulator has approved it for use.
The submission stated that some new cancer drugs would on average only add about two months to a patient's life, but can cost up to $100,000. "It is important to note that almost all new cancer drugs are not dramatically more effective than existing treatments," it stated. "PBAC is required to consider the adverse effects of new drugs compared to existing treatments, as well as the effect on quality of life.
"In the case Regorafenib (a bowel cancer drug) for example, the PBAC found that the very small gain in life expectancy was outweighed by the significant increase in toxicity as well as substantially higher costs." The committee said it was strongly opposed to suggestions that companies be allowed to bypass the PBAC to fast-track access to new cancer drugs.
It said this would greatly increase the costs to taxpayers and lead to "justified resentment" among patients with other diseases.
The committee said it was worried patients were being misled about the potential benefits of some medications.

Tetanus Vaccine boosts Cancer Vaccine In fighting Brain Tumor

In a small trial involving 12 patients, researchers recently used the tetanus vaccine to “prime” the immune systems of patients with brain tumors. Then they administered immunotherapy using a new kind of cellular cancer vaccine that trains the immune system to fight the tumor without affecting nearby healthy cells. Using this one-two punch of a highly effective traditional vaccine and an innovative cancer vaccine, patients with glioblastoma, lived from 4 to 8 more years after their treatment, compared to 11 more months in the patients who received the immunotherapy with a placebo injection instead of the tetanus booster. One patient is still alive eight years later. Although the trial is small, the dramatic difference in additional survival time among those who first received the tetanus vaccine implies the strategy worked.
The use of dendritic cell vaccines is not new to cancer immunotherapy. A great deal of research, especially with advanced melanoma, has shown their effectiveness in killing cancer cells when trained what to target. But using dendritic cells to target an actual virus instead of cellular mutations is new. By targeting cytomegalovirus, present only in the tumor cells, healthy cells don’t get caught in the crossfire. Using the tetanus vaccine like a warning siren to increase the dendritic cells’ success is new as well and might have applications well beyond glioblastoma treatment.
“It certainly has implications to be brought into other very aggressive cancers where you have cancer-specific targets because tetanus is so good at creating an inflammatory response and waking up the immune system.”

Light therapy now treats even the deepest Cancer

Light therapy is a safe, easy way to kill cancer and treat other diseases, but it's normally limited by its nature to illnesses that are skin-deep. Washington University researchers have developed a phototherapy method that brings light directly to tumor cells, no matter how deep they are. The technique has you ingest sugar combined with radioactive fluorine and light-sensitive, cancer-fighting nanoparticles. When you go through a PET scan, the sugar lights up and promptly kicks the nanoparticles into high gear. Effectively, this is the Trojan horse, since tumors eagerly absorb sugar, they're sowing the seeds of their own demise.
The procedure should be safe. The researchers note that the light and material are only targeted at tumors, so you shouldn't get sick because of some stray nanoparticles floating around your body. There are far fewer drugs involved than in chemotherapy, too. You may not have to wait long to find out how well this in-body light therapy works in practice, Washington University is already planning a small-scale trial for its approach. If it proves successful and leads to official approval, you may have a shot at recovering from particularly sinister forms of cancer without facing terrible side effects.

New possibilities for the treatment of Breast Cancer

A means of reprogramming a flawed immune response into an efficient anti-tumoral one was brought to light by the results of a translational trial relating to breast cancer. Thanks to the innovative combination of mathematical modelisation and experimentation, only 20 tests were necessary, whereas traditional experimentation would have required 596 tests to obtain the same results.
The study was jointly conducted by Doctor Marie-Agnès Doucey (Experimental oncology, Centre Ludwig de l'UNIL pour la recherche sur le cancer), Professor Ioannis Xenarios (UNIL, SIB, Vital-IT) and Professor Jean-François Delaloye (Breast care center-CHUV, UNIL). Beyond demonstrating the continued collaboration between three of Switzerland's leading scientific institutions, the trial is noteworthy for its combination of experimental oncology and modelisation. Indeed, it is the first such trial to exploit modelisation to identify efficient therapeutic approaches to be used on cells of breast cancer patients. The funding awarded by the Fond National Suisse pour la Recherche Scientifique and the publication of its results in the scientific review Plos Comp Biologie further give weight to both the validity and to the potential of its findings. Monocytes are immune cells present in the blood. They are consequently also found in tumors. Monocytes are known to promote the development of the tumoral blood vessels (referred to as their angiogenic action) and to suppress the immune response directed at the tumor (referred to as their immunosuppressive action). The trial revealed that, in patients suffering from breast cancer, once blood monocytes are present in the tumor, they considerably increase their angiogenic and immunosuppressive activity. This observation indicates that the tumor has the ability to shape monocyte activities to its advantage.The key objective of the trial was consequently to block the tumoral monocytes' angiogenic and immunosuppressive capacities. This implied identifying the molecular mechanisms behind this activity.
The trial led to a further trail of discovery. The tumoral monocytes are highly adaptable; as a result of the double treatment they transform themselves into cells capable of giving rise to an immune response directed against the tumor. The results underline that tumoral monocytes represent a new treatment target and suggest that this double treatment could contribute to an immunotherapy approach in the treatment of Breast Cancer.

Ukraine, the most corrupt nation in Europe,even the National Cancer Institute

Ukraine’s National Cancer Institute occupies three smoke-grey, six‑story blocks in a residential district on the edge of Kiev. The external walls are tiled, when Soviet workmen completed the facade, they built the date,“1968” into it. Since then, maintenance appears to have been erratic.Half of Ukraine’s men, and a fifth of its women, smoke; the national diet is heavy with animal fat; the national drink is vodka. Radiation from the Chernobyl disaster spread thyroid cancers throughout the 1980s generation, increasing the incidence among children tenfold. There are few family doctors, which means that breast, prostate and bowel tumors often go undetected for months. Survival rates for these cancers are among the worst in Europe. Professor Igor Shchepotin took charge of the Cancer Institute, which is both the country’s leading cancer hospital and its premier research institution, and was granted extensive powers to mend Ukraine’s health, including a budget independent of the health ministry, so that he could buy his own medicines and equipment. In Britain, he would be known as the “Cancer Tsar”, in Ukraine, he is called the “chief oncologist”.
It feels like a place where patients can come knowing that the goal is to get them well again. But three surgeons working here, a former health minister, patients and anti-corruption activists all claim that this is not the whole truth. They claim that the hospital, like government bodies all over Ukraine, appears to have been infected by corruption. “Presumably there is money,” said Konstantin Sidorenko, a consultant anaesthetist at the institute. “But for some reason that money doesn’t reach the most important places, like intensive care. So it means we have to earn everything ourselves.”
He explained that almost all of his doctors collect the money from patients, then pass it on to him. He uses it to maintain the machines that keep his patients alive. These are the realities of being a doctor in Ukraine. Shchepotin, the head of the institute, refused to comment on the specific allegations made by Sidorenko that such practices were taking place at the institute.

New Colon Cancer culprit found, a protein

Now, a team of researchers from the University of Pennsylvania has found evidence of a new culprit in the disease, a protein called MSI2. Their findings provide a new target for potential therapeutic intervention in colorectal cancer and enhance our understanding of the complexities of cancer initiation and progression. Further studies of MSI2 may even help explain how the disease can return after lying dormant for years.
In earlier studies, Lengner and Kharas had found that an RNA binding protein called MSI2 played a role in supporting the potency of hematopoietic stem cells. This same protein was also found to be highly active in blood cancers. Yet unlike other well-established genes that, when mutated, result in increased tumor formation, the MSI2 gene itself is not directly mutated in tumors. Rather, the normal, intact gene becomes highly activated as cancer progresses.
When MSI2 is active, the protein promotes cancer not by changing the expression of genes but by altering the ability of RNA to make proteins. Until now, the contribution of MSI2 went undetected by traditional research techniques that are largely aimed at identifying mutations in DNA sequence and alterations in gene expression patterns.
Instead, in the current work, the Penn-led team performed an analysis to look for RNA transcripts that were highly expressed in cancerous tissues but not in normal tissue. They found over-expression of MSI2 was a common characteristic of colon cancer tumors.
Next, they used colorectal cancer cell lines to experimentally block MSI2 activity and found the growth of the tumors was strongly inhibited, another sign that MSI2 promotes cancer growth.

Scripps Florida scientists confirm key targets of new Anti-Cancer Drug

Scientists from the Florida campus of The Scripps Research Institute (TSRI) have confirmed the ribosome assembly process as a potentially fertile new target for anti-cancer drugs by detailing the essential function of a key component in the assembly process.
"This study confirms that ribosome assembly is a good therapeutic target in cancer," said Katrin Karbstein, a TSRI associate professor who led the study. "Whether or not we have pinpointed the best molecule remains to be shown, but this is a vindication of our basic research. There should be effort devoted to exploring this pathway."
Understanding ribosome assembly, which involves about 200 essential proteins known as "assembly factors" in addition to the four RNA molecules and 78 ribosomal proteins that are part of the mature ribosome--has become a fruitful area of research in recent years because of the importance of ribosome assembly for cell growth.
The new study highlights the molecules Casein kinase 1δ (CK1δ) and CK1ε, which are essential for human ribosome assembly. The expression of CK1δ is elevated in several tumor types, as well as Alzheimer's and Parkinson's disease--and CK1δ inhibitors have shown promise in some pre-clinical animal studies.
In the new study, Karbstein and her group, working closely with three labs across the state of Florida, including the laboratory of William Roush at Scripps Florida, used Hrr25, the yeast equivalent of Casein kinase 1δ (CK1δ) and CK1ε, as a research model.
In biochemical experiments, the team showed that Hrr25 is necessary for ribosome assembly and that the molecule normally adds a phosphate group to an assembly factor called "Ltv1," allowing it to separate from other subunits and mature. If Hrr25 is inactivated or a mutation blocks the release of Ltv1, the assembly process is doomed.
"Inhibiting Hrr25 and the subsequent release of Ltv1 blocks the formation of other subunits that are required for maturation and the subsequent production of proteins,"

Australian researchers develope genome-editing technology to help treat Blood Cancers

Melbourne researchers have developed a new genome editing technology that can target and kill blood cancer cells with high accuracy.
Using the technology, researchers from the Walter and Eliza Hall Institute were able to kill human lymphoma cells by locating and deleting an essential gene for cancer cell survival.
The research provides a 'proof of concept' for using the technology as a direct treatment for human diseases arising from genetic 'errors'.
Dr Brandon Aubrey, Dr Gemma Kelly and Dr Marco Herold adapted the technology, called CRISPR, to specifically mimic and study blood cancers. The Walter and Eliza Hall Institute has one of the most advanced CRISPR laboratories in Australia, established and led by Dr Herold.
Dr Aubrey, who is also a haematologist at The Royal Melbourne Hospital, said the team used the CRISPR technology to target and directly manipulate genes in blood cancer cells.
"Using preclinical models, we were able to kill human Burkitt lymphoma cells by deleting MCL-1, a gene that has been shown to keep cancer cells alive," he said. "Our study showed that the CRISPR technology can directly kill cancer cells by targeting factors that are essential for their survival and growth. As a clinician, it is very exciting to see the prospect of new technology that could in the future provide new treatment options for cancer patients."
The CRISPR/Cas9 system works by efficiently locating and targeting particular genes of interest in the whole genome. It can either target the gene to introduce mutations that make the gene non-functional, or introduce changes that make mutated genes function normally again.
Dr Herold said pharmaceutical companies around the world were already investing millions of dollars to develop CRISPR as a tool for treating genetic diseases such as cancer.
"There is a lot of excitement and a significant amount of resources being invested worldwide to use CRISPR technology for treating patients," Dr Herold said. "The technology can directly target any gene in the person's genome, therefore overcoming many common drug development problems."

Cancer drug promotes regeneration of damaged neurons in Spinal Cord

Damage to the spinal cord rarely heals because the injured nerve cells fail to regenerate. The regrowth of their long nerve fibers is hindered by scar tissue and molecular processes inside the nerves. An international team of researchers led by DZNE scientists in Bonn now report that help might be on the way from an unexpected quarter: in animal studies, the cancer drug Epothilone reduced the formation of scar tissue in injuries to the spinal cord and stimulated growth in damaged nerve cells. Both promoted neuronal regeneration and improved the animals' motor skills.
Experiments have shown Epothilone works on several levels. Epothilone reduces the growth of scar tissue by inhibiting the formation of micro-tubules in the cells that form the scar tissue. Therefore they cannot migrate to the spinal cord lesion and cause wound scarring. At the same time, Epothilone promotes growth and regeneration in the nerve cells by causing micro-tubules to grow into the damaged axon tips.

Companies selling Cannabis-infused products warned by FDA on health claims

The FDA sent letters over the past two weeks to nearly a dozen companies, including Washington-state based Canna Companion, which markets a supplement infused with hemp to dog and cat owners.

"This product is an unapproved new animal drug and your marketing of it violates the FD&C Act," the letter reads. The act gives the FDA authority to oversee safety and benefit claims of food, drugs and cosmetics.

Warning letters have also been sent to Seattle-based Canna-Pet, LLC, which makes pet treats and supplements infused with CBD, an active cannabinoid, and to California-based Hemp Oil Care, which sells cannabis-infused "products for therapeutic healthcare purposes" marketed to humans.

"The reason they got warning letters is not what is or isn't in the product," said FDA spokesman Jeff Ventura. "It's because they made therapeutic claims."

The letters require each company to remove claims on its website, packaging and marketing material that the products can improve health or prevent disease and illness.

The FDA, which says it supports research into cannabis, said scientists tested all the cannabis-laced products to ensure they did not pose a risk to public safety.

In some cases, products that claimed to owe their benefits to cannabis contained less of the compound than advertised or none at all, Ventura said.

Guidelines on Stem Cell technology to be released in China

The China Food and Drug Administration recently held a meeting with stem cell experts and announced that the formation of guidelines on the application of stem cells will be concluded during the first half of this year at the earliest, according to the report.
One of the experts said that developing the guidelines should not be difficult because China could refer to those established by the International Society for Stem Cell Research.
An industry insider said China is at the forefront of stem cell research, citing experiments conducted in the 1980s by Wu Zuze, a former head of the Military Academy of Medical Sciences who successfully transplanted fetal liver stem cells in 11 patients. Nine of the patients are still alive today, the newspaper said. But the development of stem cell technology in China has been hampered by a lack of regulation, said the report.
Li Dexing, a research fellow at the Chinese Center of Disease Control and Prevention, suggested, for example, that the Chinese government adopt the "compassionate use exemption" used in the United States in over 300 cases for treatments involving stem cells.
The commercialization of stem cell technology remains in its early stages in China.
According to Zhong Hua, a professor at the Chinese Academy of Medical Science, the sector is currently involved in the collection and storage of stem cells, the development of stem cell technology, and transplants and treatment.

Regulatory uncertainty hits drug trials in India

An avalanche of reforms to rules governing the conduct of clinical trials in India has led to an exodus of drug companies and research organizations. Given the uncertain regime, drug companies and CROs are cutting back on clinical trials in India. The Indian Pharmaceutical Alliance (IPA), which represents leading domestic pharmaceutical companies, says that the increasing reluctance of India’s drugs and devices regulator to grant approvals for new drugs, even if they are approved in developed countries, and to allow clinical trials or bio-studies for export purposes, has severely hampered the industry. The effects of the regulatory uncertainty are already being felt. Max India, for example, has announced that it will sell its clinical research business for US$1.5m to JSS Medical Research, a Canadian CRO. The company says the regulatory challenges have made it difficult to scale up the business.
Other Indian pharmaceutical companies, such as Biocon, Alembic, Zydus Cadila, Torrent and Lupin, have already moved trials out of the country. At Biocon, the research and development (R&D) spend is 134% higher in the second quarter of 2015 compared with the same period in 2014. The company’s chairman, Kiran Mazumdar-Shaw, said at an analyst briefing that the high R&D spend is a result of ongoing global clinical trials that require large investment. For example, Biocon’s global phase III trial for biosimilar trastuzumab is being conducted in Europe.
Indian CROs, such as Veeda Clinical Research and Lambda Therapeutics, have also moved out to other Asian countries, including Malaysia and Thailand. An official at Veeda says the high degree of uncertainty in India has discouraged clinical trials.

China drug approval is backlog, up by a third from last year

China had more than 18,500 drugs waiting for approval at the end of 2014, up by a third from a year before, the official Center for Drug Evaluation said on Friday, reflecting industry concern that it is getting harder to get medicines approved in the China market.
Drug company executives say China has toughened the approval process, with companies forced to go through six to eight-year wait times in the world's second biggest pharmaceutical market, where spending is set to hit as much as $185 billion by 2018, according to IMS Health.
China's fast-growing healthcare market is a magnet for drug makers, medical device firms and hospital operators, with a broader healthcare bill set to hit $1 trillion by 2020. However, the sector is riddled with issues from rampant bribery to huge divides between urban and rural care.

China's drug trial center received 8,868 drug applications in 2014, up from 7,610 the year before, according to the annual report Center for Drug Evaluation, overseen by the country's food and drug regulator, and released on Friday.
The organization said it had increased the number of reviews it completed last year, but it was outflanked by an increase in new drugs to review. 

New research shows possibility of cure for HPV positive Throat Cancer patients

Nice, France: Patients with cancer of the throat caused by the Human Papilloma virus (HPV+) have a better prognosis than those who are negative for the virus (HPV-). Now, for the first time, researchers have shown with convincing evidence that a group of patients with HPV+ cancer of the oropharynx (the part of the throat located behind the mouth, that makes up the region of the tonsils and the back part of the tongue where it connects to the swallowing part of the throat), can be cured in some cases even after disease has spread to distant organs in the body, like the lungs.
"This research has shown that metastatic HPV+ OPC patients who receive active treatment can survive considerably longer than those who did not receive treatment. One of the reasons patients with metastatic disease do not receive aggressive treatment is due to the physician and patient's perception that this is an incurable state. We hope that these results will motivate researchers to optimize management strategies for these patients. This will not only help to produce a better quality of life and a return to work for them, but also reduce the cost to healthcare systems," Dr Huang will say.
"We also hope that our study may trigger research to explore cost-effective methods for the early detection of metastases. Optimizing and tailoring surveillance strategies for HPV+ patients are also important. For example, our research has shown that the surveillance period should be longer than the traditional two-year window, due to the possibility of later onset of metastases."

UK study shows promise for Epithelioid Mesothelioma Patients

More than 30 percent of patients with Epithelioid Mesothelioma, the most common cellular strain of this cancer, lived for five years or more after a multidisciplinary treatment approach in England, raising hopes and expectations everywhere for this rare and deadly disease.
The startling success rate stems from a retrospective study of 102 mesothelioma patients who underwent aggressive lung-saving, pleurectomy/decortication (P/D) procedure performed by U.K. thoracic surgeon Dr. Loic Lang-Lazdunski. All patients also were treated with chemotherapy and post-surgery radiotherapy, along with the controversial hyperthermic povidone-iodine wash of the chest cavity before surgical closure. "This treatment plan represents an alternative to the classical trimodality approach," wrote Lang-Lazdunski "Because of the multiple therapies being used in this study, the exact impact of povidone-iodine lavage on outcomes and long-term survival is not possible to precisely know. However, we believe this is a cheap, safe and possibly useful adjunct after P/D."
Povidone-iodine is a chemical complex popular since the 1950s as a traditional antiseptic — often marketed as Betadine, and used by doctors as a pre-surgery scrub. Recent laboratory work shows it can suppress and destroy mesothelioma tumor cells.
Although it is part of several clinical trials involving other cancers, it has not been clinically studied specifically with mesothelioma patients. Some surgeons, including Lang-Lazdunski, are using it as an alternative to a chemotherapy wash of the chest cavity after surgery. They believe it could prevent tumoral seeding following surgical resection and slow any future metastasis.
Yet even in his report, Lang-Lazdunski did not fully endorse the treatment without a trial designed to prove its effectiveness.

KEYTRUDA is available to patients through U.K. Early Access to Medicines Scheme

“This news will be greatly received by the melanoma community in the U.K. At the moment, there are limited options for advanced melanoma patients, the majority of whom are in difficult positions which simply means they cannot afford to play any kind of waiting game,” said Gillian Nuttall, Melanoma UK. “We welcome early access to this treatment for advanced patients made possible through this new scheme. There is clearly an unmet need and we are delighted such progress is being made.”
“We welcome the Government’s proactive approach in facilitating early access to medicines for critically ill patients and are delighted that pembrolizumab will be the first innovative medicine to be available to patients through the Early Access to Medicines Scheme,” said Mike Nally, managing director for MSD UK and Ireland.
To date, more than 3,500 patients in over 40 countries have received early access to pembrolizumab for the treatment of advanced melanoma through the company’s global expanded access program.
The U.K. EAMS aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. Under the scheme, the MHRA will give a scientific opinion on the benefit/risk balance of the medicine, based on the data available when the EAMS submission was made. The opinion lasts for a year and can be renewed. 

Zykadia by Novartis recommended for EU approval to treat ALK+ NSC Lung Cancer

Zykadia from Novartis was recommended for EU approval to address treatment for adult patients suffering from anapestic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC), also known as ALK+NSCLC, which has been treated previously with crizotinib. If approved, this would be the first treatment option for patients in Europe.
“Patients with advanced ALK+ NSCLC have few options when their cancer does not respond to currently approved therapy. As a leader in the development of precision oncology medicines Novartis is committed to developing and bringing to market new treatments for patients with ALK+ NSCLC. This positive CHMP opinion for Zykadia brings us one step closer to providing the lung cancer community with new hope in the fight against this terrible disease,” stated Alessandro Riva, a Novartis representative.
Two studies support Zykadia’s recommendation by the EMA, including open-label, global multi center and single-arm studies.

Study A evaluated 246 ALK+NSCLC patients addressed with Zykadia: 163 individuals received previous treatment with an ALK inhibitor and 83 were ALK inhibitor-naïve. Study B was a Phase II study designed to assess the safety and efficacy of the drug in individuals with locally advanced or metastatic ALK+ NSCLC. 140 patients were previously treated with 1 to 3 lines of chemotherapy and further treatment with crizotinib and who had experienced cancer progression while on crizotinib.

The studies’ outcomes show that brain metastases at baseline were found in 60.1 percent and 71.4 percent of the patients that had had previous treatment with an ALK inhibitor. The results for patients with brain metastases at baseline were very similar to those reported for the overall population.
Zykadia is currently approved in the United States, Chile, Mexico, Guatemala, South Korea and Ecuador.

FDA proposal would allow drug-makers to contradict safety warnings

The Food and Drug Administration is proposing to allow pharmaceutical companies to contradict official safety warnings in sales presentations to customers.
While an FDA warning about a drug’s dangers can scare off buyers, the new proposal would allow the companies to present customers with information that undermines official warnings as long as it comes from a peer-reviewed journal article.
The proposal is supported by pharmaceutical manufacturers, who argue that the policy would allow them to give doctors and hospitals the benefits of the latest research.
But the proposal is drawing an avalanche of criticism from public health advocates who argue that because individual studies can differ widely in their results, a drug company could easily mislead customers, and possibly endanger patients by presenting only a selection of new research.
The proposal “seriously undermines FDA authority,” Sidney Wolfe, founder of Public Citizen’s Health Research Group wrote Wednesday to the agency. “Its main supporters are drug companies and their associations, all of which would benefit from being allowed and encouraged to sell more drugs by making them seem safer than FDA has judged them to be.”

Read more here: http://www.heraldonline.com/news/nation-world/national/article13432754.html#storylink=cpy

Targeted Lung Cancer therapies are replacing conventional treatments

As the market for lung cancer therapeutics continues to grow, drug manufacturers are competing to find their own niches. Approximately 130 drugs to address small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC) are currently in several stages of advancement and are expected to enter the market until 2020.
While lung cancer is the 3rd most prevalent type of cancer in the world, there are only 18 drugs available in the market to address the disease. Popular drugs such as Avastin and Almita are close to having their patents expire in 2017 and 2015, respectively.
According to the new Frost & Sullivan’s assessment “A Product and Pipeline Analysis of the Lung Cancer Therapeutics Market,” many drug manufacturers are committed to developing and researching new targeted therapies that might replace conventional medicine in the next 10 years. The European Medicines Agency, United States Food and Drug Administration (FDA), FDA Japan, National Institute for Health and Care Excellence are highly encouraging investments in targeted therapies. The market for lung cancer therapeutics is marked with strong competition, especially strong among some of the world’s top firms such as AstraZeneca, Bristol-Myers Squibb, Eli Lilly, Merck and Roche. AstraZeneca, Bristol-Myers Squibb, Merck and Roche are in particularly close competition and are developing drugs that target the PD-L1 pathway and anapestic lymphoma kinase (ALK). Bristol-Myers Squibb recently became the first company to launch a PD-L1 specific candidate in Japan, Opdivo (Nivolumab).
Treating late-stage cancer is about 43 percent more expensive that treating the disease at early-stage phases. “For instance, the first-line treatment for NSCLC is typically chemotherapy. This might change with the introduction of Necitumumab by Eli Lilly, at present in the third phase of clinical trials in the US. In combination with chemotherapy, Necitumumab has the potential to be the first targeted therapy used for cost-effective first-line treatment,” 

F.D.A. approves Zarxio, the first Biosimilar drug of the FDA

The FDA has approved the first so-called biosimilar drug for use in the United States, paving the way for less expensive alternatives to an entire class of complex and costly drugs.

The drug, called Zarxio, produced by Sandoz, is used to help prevent infections in cancer patients receiving chemotherapy. It is a close copy of an existing medication called Neupogen, made by Amgen. It was approved in Europe in 2009 as Zarzio but has not been used in the United States, in part because no regulatory pathway existed to bring biosimilars, approximate copies of drugs in a class known as biologics to market.

“Biosimilars will provide access to important therapies for patients who need them,” Dr. Margaret A. Hamburg, the commissioner of the F.D.A., said in a statement.

The approval is significant because it opens the door to a new class of potentially cheaper lifesaving drugs for millions of Americans. It involves biologic drugs, which are made using living cells and not synthesized from chemicals like typical drugs. Some popular biologic drugs are Remicade and Enbrel for autoimmune diseases, and Herceptin and Avastin for cancer. Some of the world’s most expensive medications are biologics.

Express Scripts estimates that Zarxio’s introduction in the United States could save $5.7 billion in drug costs over the next 10 years. It estimated that $250 billion in drug costs could be saved over the next decade if 11 biosimilars in development were approved.

Coffee shows protective effects on Cancer and Diabetes

The recently released 2015 Scientific Report of the Dietary Guidelines Advisory Committee, available online, includes 209 references to coffee, most of them favorable, particularly for those who don’t add cream and sugar and limit daily consumption to three to five cups and no more than 400 milligrams of caffeine.
It’s the first time the committee has addressed the health effects of coffee and caffeine. Every five years, the USDA uses the report to establish science-based dietary guidelines.
“Strong and consistent evidence shows that consumption of coffee within the moderate range, is not associated with increased risk of major chronic diseases, such as cardiovascular disease and cancer and premature death in health adults,” the report states in awarding coffee a “strong” grade.
As it turns out, the bean-like seeds inside the coffee plant’s red and purple berries can reduce the risk of type 2 diabetes by 36 percent with protective effects against liver and endometrial cancers. Slight benefits were noted for other cancers the report didn’t identify.
“Moreover, moderate evidence shows a protective association between coffee/caffeine intake and risk of Parkinson’s disease,” the report states.
Overall, research found reductions in mortality among regular coffee drinkers. Studies not cited in the report showed benefits in preventing depression and Alzheimer’s disease, among other neurodegenerative conditions.

TWi Biotechnology receives Rare Disease Drug Designation for AC-203 in Taiwan

TWi Pharmaceuticals, Inc. today announced that its fully owned subsidiary, TWi Biotechnology, Inc., has received the designation of Rare Disease Drug by Taiwan FDA for use of AC-203 to treat Epidermolysis Bullosa Simplex (EBS), and is eligible for applying for coverage under National Health Insurance Administration (NHIA).  TWi Biotechnology has developed proprietary formulations to be used topically for the indication of EBS and will plan for further development and product launch according to the designation.

"We are very pleased with the TFDA's approval of the rare disease drug designation for treating epidermolysis bullosa simplex.  This is a major milestone of AC-203, which has obtained the orphan drug designation from U.S. FDA just several months ago.  In addition, this fulfills the mission of our new drug business for developing proprietary formulations and meeting unmet medical needs," said Dr. Calvin C. Chen, President of TWi Biotechnology.  "Epidermolysis Bullosa, or EB, is a very rare genetic connective tissue disorder that affects several hundred of children in Taiwan suffering from this disease; patients with EB are known as 'Bubble Dragon Children' in Taiwan or 'Butterfly Children' in the United States due to their extremely fragile skin that blisters and tears from friction or trauma. We hope this rare disease drug designation can not only help the patients in Taiwan but also speed the development of topical AC-203 in other places of the world.  Current treatments for EBS focus on relief of symptoms such as itching and pain, and wound care to prevent infection, which include expensive treatments such as artificial skin and may require frequent visits to hospitals.  In contrast, the topical formulation of AC-203 is developed to prevent or reduce the blisters that, if successful, may alleviate the suffering of the patients and their families from this deliberating disease."

Irish research center wins EU funding for Cancer study

Researchers at Ireland's Waterford Institute of Technology have been awarded nearly $1 million to test and develop transdermal medications for conditions such as non-melanoma skin cancer.
The drugs will be of high potency, meaning a quantity of the candidate weighing less than a grain of sugar could be efficacious. That's why the manufacturing facilities of Ireland's Eirgen Pharma are also being deployed in the drug delivery initiative.
"Currently, many drugs are taken orally or injected directly into the bloodstream where they travel around the body. This is very inefficient as most of the drug taken does not reach the target organ that you wish to treat. In addition, many of the drugs used today are highly toxic and delivery of these drugs around the body can result in debilitating side effects. If we can deliver these drugs directly to the target across the skin, for example in skin cancer treatment, we can maximize the therapeutic effect and minimize the unpleasant side effects," said WIT's Dr. Peter McLoughlin.

Kaiser Vallejo involved in new clinical trials

Kaiser Permanente is enrolling lung cancer patients in two new clinical trials, which profile tumors for genetic markers and then direct patients to the experimental medications that are most likely to be effective. The LUNG-MAP and ALCHEMIST clinical trials are available to all qualified members at every medical center in Kaiser Permanente's Northern California region, officials said. "These studies have the potential to fundamentally transform how doctors identify the best experimental treatments for cancer patients," Kaiser Permanente's Vallejo Medical Center hematology oncologist and the studies' co-investigator J. Marie Suga said. "Both trials test cancer tissue to find the malignant genetic changes, in order to identify the best treatment for the patient." Clinical trials are studies that test experimental medications and treatments for safety and efficacy. Funded by the National Cancer Institute, LUNG-MAP (Lung Cancer Master Protocol) targets patients with advanced squamous-cell carcinoma, about a quarter of lung-cancer patients, who have already completed one line of treatment for metastasized cancer. ALCHEMIST (Adjuvant Lung Cancer Enrichment Marker Identification and Sequencing Trial) targets patients with nonsquamous lung cancer that has been surgically removed.  Kaiser Permanente is using its integrated electronic medical records to identify patients who may qualify for these clinical trials, officials said.

Suga is spearheading the new treatment protocols across the Northern California region. Kaiser Permanente's Southern California, Northwest, and Hawaii regions are also participating officials said.
Since Kaiser Permanente Clinical Oncology Trials began enrolling patients in cancer treatment clinical trials more than 25 years ago, the program has grown into one of the nation's largest, they said. In 2014, Kaiser Permanente became an NCI--designated Community Oncology Research Program