PROVENGE shows sustained immune response Two Years after Treatment

"It is very encouraging to observe that PROVENGE provides an immune response in men with biochemical-recurrent prostate cancer long after the course of androgen deprivation therapy has ended," said Neal Shore, M.D., medical director at the Carolina Urologic Research Center. "This study may also provide guidance on the optimal sequencing of immunotherapy and ADT in biochemical-recurrent prostate cancer."
PROVENGE, the first personalized immunotherapy, stimulates a patient's own immune system to fight cancer. It is approved in the U.S. and the European Union as a treatment for asymptomatic or minimally symptomatic metastatic castrate-resistant prostate cancer.
"These are very encouraging preliminary data and the longest duration of immune responses observed following PROVENGE completion in men with this particular type of prostate cancer," said Andrew S. Sandler, M.D., chief medical officer at Dendreon. "Dendreon and Valeant remains committed to exploring the use of PROVENGE in different prostate cancer treatment settings to provide important clinical information to practicing oncologists."

Research opens new avenue for Bile Duct Cancer Treatment Trials

Researchers in Edinburgh have discovered  how a molecular mechanism drives the growth of bile duct cancer, a rare form of the disease for which there are few treatment options.
The mechanism, known as the ‘Wnt’ pathway, is well-known to cancer experts. It consists of a network of proteins that carry signals inside cells. Experimental drugs that block it are already being tested in patients with other cancers. But the latest study, led by the University of Edinburgh, gives the first indication that bile duct cancer may be particularly susceptible to these drugs.
“Identifying the signals that control bile duct cancer’s growth will allow us to design better treatments that are urgently needed,” said Dr Luke Boulter, of the MRC Human Genetics Unit at the University of Edinburgh, who led the research.
Plans are now in the pipeline to find out whether the drugs will be effective in patients.
“These drugs are being tested in clinical trials for other types of cancer. The next step will be to find out if this treatment would benefit patients with bile duct cancer. There are very few treatment options for this disease, so research into new therapies is essential.”
Bile duct cancer, also known as cholangiocarcinoma, is a rare but aggressive type of cancer that affects the network of tubes that carries bile from the liver to the digestive system. It is often diagnosed at an advanced stage, meaning it is very difficult to treat with surgery.
Around 2,100 people are diagnosed with bile duct cancer each year in the UK.

New approach to assessing effectiveness of Anti-Cancer drugs

Scientists have a developed a new method to monitor the effect of anti-cancer drugs on very rare leukemia stem cells. The approach potentially allows doctors to screen patients and personalize their treatment. The recent development of novel agents has improved outcomes for patients with chronic myeloid leukemia (CML). These so-called tyrosine kinase inhibitors (TKIs) target abnormal proteins caused by commonly found genetic mutations in CML patients. However, the existence of treatment-resistant cancer stem cells, cells that are able to repeatedly renew the leukemia cell population, is one way that many patients experience disease recurrence when treatment stops. Any new drug must therefore be tested on such stem cells, but unfortunately they are only found in very low numbers and are identified by certain cell surface markers. Now researchers at The University of Manchester, part of the Manchester Cancer Research Center, have tested a way to monitor the effect of drugs on small samples of cells.
The research team looked at an antibody-based approach to detect structural changes in certain proteins, in order to track the effectiveness of the TKI drugs. The instrument used fixes proteins in place and holds them, there allowing for a better signal to be generated from less material. With this approach they found that they could record changes in samples of only a few thousand critically important but rare stem cells.
"This new approach will enable us to test drugs on cells taken from patients, either at presentation or in a clinical trial setting. It has great potential to allow us to implement precision medicine, where patients receive the most appropriate treatment to target their individual tumor," added Professor Whetton.

NEW Urine test leads to better Treatment of Bladder Cancer

Researchers at the University of Birmingham believe that a simple urine test could help to guide clinicians in the treatment of bladder cancer patients. Being able to reliably identify those patients with the most aggressive cancers early via urine tests, and expediting aggressive therapeutic strategies, may significantly improve outcomes. The scientists believe that the validation of two urinary biomarkers could spell a new way of tailoring treatment.
Patient management has changed little over the last three decades, so it is hoped that this research, published in British Journal of Cancer, will prove to be a step forward for the field with a view to providing improved care for each patient.
Dr Douglas Ward, from the University of Birmingham, explained, "There is an urgent need for prognostic bio-markers that could guide patient management. If such a test could be delivered, in a non-invasive way, it could make treatment much more efficient and that can only be a good thing."
Although a number of prognostic markers have been reported for bladder cancer, they are not currently used in the clinic. These markers are either based on nucleic acids (such as gene expression and mutation profiles) or protein expression levels; and as such require expensive and invasive analysis of tumor tissue via complex laboratory tests.
This paper identified two prognostic urinary biomarkers, epidermal growth factor receptor (EGFR) and a protein, epithelial cell adhesion molecule (EpCAM), and validated them in over 400 clinical samples. Both urinary EGFR and EpCAM were found to be independent predictors of bladder cancer-specific survival and have prognostic value over and above that provided by standard clinical and pathological observations. Higher levels of the biomarkers correlated with more aggressive cases of cancer and those with poor survival. Measuring the biomarkers could therefore represent a simple and useful approach for fast-tracking the investigation and treatment of patients with the most aggressive bladder cancers.
Such tests would be useful in both newly-diagnosed patients and existing patients who receive a cystoscopic diagnosis of recurrence during surveillance.

A new FDA approved way to try many drugs on many Cancers

Chemotherapy and radiation failed to thwart Erika Hurwitz’s rare cancer of white blood cells. So her doctors offered her another option, a drug for melanoma. The result was astonishing.

Within four weeks, a red rash covering her body, so painful she had required a narcotic patch and the painkiller OxyContin, had vanished. Her cancer was undetectable.“It has been a miracle drug,” said Mrs. Hurwitz, 78, of Westchester County. She is part of a new national effort to try to treat cancer based not on what organ it started in, but on what mutations drive its growth.

Cancers often tend to be fueled by changes in genes, or mutations, that make cells grow and spread to other parts of the body. There are now an increasing number of drugs that block mutations in cancer genes and can halt a tumor’s growth.

While such an approach has worked in a few isolated cases, those cases cannot reveal whether other patients with the same mutation would have a similar experience.

Now, medical facilities like Memorial Sloan Kettering Cancer Center in New York, where Mrs. Hurwitz is a patient, are starting coordinated efforts to find answers. And this spring, a federally funded national program will start to screen tumors in thousands of patients to see which might be attacked by any of at least a dozen new drugs. Those whose tumors have mutations that can be attacked will be given the drugs.The studies of this new method, called basket studies because they lump together different kinds of cancer, are revolutionary, much smaller than the usual studies, and without control groups of patients who for comparison’s sake receive standard treatment.

Researchers and drug companies asked the Food and Drug Administration for its opinion, realizing that if the F.D.A. did not accept the studies, no drugs would ever be approved on the basis of them. But the F.D.A. said it sanctioned them and could approve drugs with basket study data alone.

These are the sorts of studies many seriously ill patients have been craving, a guarantee that if they enter a study they will get a promising new drug.The studies move fast; it does not take years to see a big effect if there is one at all.

WHO VOTED HER IN? Nevada lawmaker says Cancer is a Fungus,Simply just Wash It Out

Nevada Assemblywoman Michele Fiore (R) wants to reform the rules of end-of-life medical care so that more cancer patients can simply flush out their disease using baking soda.
Fiore, who is also CEO of a healthcare company, told listeners to her weekly radio show on Saturday, that she will soon introduce a “terminally ill bill,” to allow more non-FDA-approved treatments for those diagnosed as having terminal illnesses.
 Fiore told listeners: “If you have cancer, which I believe is a fungus, and we can put a pic line into your body and we’re flushing, let’s say, salt water, sodium carbonate , through that line, and flushing out the fungus… These are some procedures that are not FDA-approved in America that are very inexpensive, cost-effective.” The American Cancer Society warns that while cancer patients whose immune systems are weakened by high doses of chemotherapy can sometimes contract fungal infections, “there is no evidence that anti-fungal treatment causes the patients’ tumors to shrink.” Cancer Research UK dismisses the claim that sodium bicarbonate (baking soda) can cure cancer as a debunked “persistent cancer myth.”

Fiore added that Nevada is already “the capital of entertainment” and this bill could help “make it the medical capital of the world as well.”
Weeks after being removed from her position as Republican Majority Leader over allegations of more than $1 million in tax liens, Fiore made news last Wednesday for her assertion that “young, hot little girls on campus” need to be armed with guns to prevent themselves from being raped, saying that every citizen should “have the right to defend him or herself from sexual assault.”
In 2012, she proposed arming school officials and college students as a way of combating school shootings.
Although Fiore’s views on cancer are particularly fringe, the bill she is backing is gaining traction in a number of states. At least five states have now passed similar legislation that allows patients to use drugs not cleared by the FDA, dubbed so-called “right to try” bills. The campaign to pass these bills has been led by the libertarian Goldwater Institute.

EC approved REVLIMID for the treatment of adult patients with untreated Multiple Myeloma

Celgene International Sàrl, a wholly owned subsidiary of Celgene Corporation (NASDAQ: CELG),  announced that the European Commission (EC) has approved REVLIMID (lenalidomide) for the treatment of adult patients with previously untreated multiple myeloma who are not eligible for transplant. The REVLIMID Marketing Authorisation has been updated to include this new indication in multiple myeloma, building upon the already approved indication of REVLIMID in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least one prior therapy. Multiple myeloma is a persistent and life-threatening blood cancer that is characterised by tumour proliferation and suppression of the immune system. It is a rare but deadly disease: around 38,900 people were newly diagnosed with multiple myeloma in Europe in 2012, and 24,300 people died from the disease in the same year. On average, multiple myeloma is diagnosed in people 65-74 years of age, and the majority of newly diagnosed patients may not be eligible for more aggressive treatment options such as high-dose chemotherapy with stem cell transplant.
Professor Thierry Facon, Services des Maladies du Sang, Hôpital Claude Huriez, and CHRU Lille, France, says: "Having a new treatment option now available for patients newly diagnosed with multiple myeloma is a real step forward. Treating patients continuously until disease progression is supported by several clinical studies, and will have an important impact on how we manage the disease over the long-term."
In the United States, REVLIMID is approved in combination with dexamethasone for the treatment of patients with multiple myeloma. In the European Union, REVLIMID is approved for the treatment of adult patients with previously untreated multiple myeloma who are not eligible for transplant. REVLIMID is approved in combination with dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy in nearly 70 countries, encompassing Europe, the Americas, the Middle-East and Asia, and in combination with dexamethasone for the treatment of patients whose disease has progressed after one therapy in Australia and New Zealand.
REVLIMID is also approved in the United States, Canada, Switzerland, Australia, New Zealand and several Latin American countries, as well as Malaysia and Israel, for transfusion-dependent anemia due to low- or intermediate-1-risk MDS associated with a deletion 5q cytogenetic abnormality with or without additional cyto-genetic abnormalities and in Europe for the treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with an isolated deletion 5q cyto-genetic abnormality when other therapeutic options are insufficient or inadequate.
In addition, REVLIMID is approved in the United States for the treatment of patients with mantle cell lymphoma (MCL) whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib. In Switzerland, REVLIMID is indicated for the treatment of patients with relapsed or refractory MCL after prior therapy that included bortezomib and chemotherapy/rituximab.

E U Doctoral students of 'Magicbullet' network to develop 'gentle' Cancer Treatment

Medication that specifically targets cancer cells and delivers its active agent without harming healthy cells, this is what doctoral students of the 'Magicbullet' network will be working on from mid-2015. Bielefeld University is coordinating the program for the development of "gentle" cancer treatment. The European Union is supporting it with around €4 million in funding.
Current cancer therapies are usually accompanied by severe side effects. "The reason for this is that active agents are used which are designed to poison and kill the cancer cells. These active agents also damage healthy cells," says Professor Dr. Norbert Sewald. He is the coordinator of the new programme, a European Training Network for young researchers in the frame of the Marie Sklodowska-Curie actions.
The development of the targeted, gentle cancer medication involves the researchers of Magicbullet attaching the toxic active agent (payload) to a peptide (a small protein molecule,delivery vector). These delivery vectors recognize molecules that are typical for tumor cells, bind to them and deliver the payload. "The delivery vectors are a kind of address label containing information as to where the anti-tumor payloads are to be delivered," says Sewald. According to the scientist, such new treatments could fulfill the earlier vision of Nobel laureate Paul Ehrlich (1854-1915), who coined the term "magic bullets" for such tumor-targeting conjugates.
"Drugs based on this principle are already on the market," explains Sewald. "These contain antibodies, though, that is to say large protein molecules produced by means of biotechnology, which have to be purified elaborately and are, therefore, very expensive. But we want to develop small protein molecules, peptides, as the transporters of the active agent. The advantage is that the chemical processes to produce them are simpler and quicker than those for antibodies." For instance, during the production process it is easier to remove impurities. "Peptides can take a higher load of active agent and easily penetrate tissue. That's what makes them so special," says Sewald. Peptides are roughly one hundred times smaller than antibodies. For Sewald it is also important that they can be produced in a highly purified form. The new program is all about fundamental research. "Our drugs will be prototypes. But we will be creating the scientific basis for anti-tumor medicines which could come on the market in perhaps ten or twenty years' time," explains Sewald.

Half a million cancer patients in EU use targeted drugs

French healthcare information firm Cegedim looked at the top five European Union countries (in terms of population): UK, Germany, France, Italy and Spain, and their experience in treating the disease. It found that between 500,000 and 600,000 patients are currently treated with targeted therapies in these countries, all of which have a total population of around 320m.

In the five countries studied, Cegedim's analysis found targeted therapies represent an average of 32% of all cancer drugs used, the remaining 68% being cheaper chemotherapy drugs and hormonal treatments, whose data the study did not collect. The report was conducted by anonymised patient information collected through electronic case report forms (e-CRF) from a quarterly panel of 1,400 specialists.The analysis is based on 110,000 patient cases (ie, raw data) and covers a 12-month period in 2014 using projected data. The report also found that Germany had the highest rate of target therapy use (36%) compared to the other countries.

Breast cancer is the most commonly treated, accounting for around 16% of all tumors across all five countries. Non-small cell lung cancer (NSCLC) was a close second, although the rates of targeted drug use for the disease varied greatly among the individual countries.

In France the use was 15% while it was 12.3% in Italy and Germany, but 11% in Spain and only 7.1% in the UK. In fact the UK had the lowest level of cancer patients using these new types of treatments, with 67.9%. Spain had the highest level at 71.9%, followed closely by Italy with 71.6%. 

Cegedim says that these differences are influenced by the launch dates of the drugs, as well as the health authority and market access policies in each country.

Patients with pancreatic cancer, for which there are few personalized drugs licensed in Europe, made the least use of personalized medicines, with only around 2.5% of patients across the five countries receiving new treatments for the disease.

Vargatef(nintedanib) approved in the EU for Lung Cancer patients

Boehringer Ingelheim announced that the European Commission has granted EU marketing authorisation for Vargatef® (nintedanib*), valid for the 28 countries within the EU. Vargatef® in combination with docetaxel is indicated for use in adult patients with locally advanced, metastatic or locally recurrent non-small cell lung cancer (NSCLC) of adenocarcinoma tumour histology, after first-line chemotherapy.
“The approval of nintedanib offers a much needed new treatment option for adult lung cancer patients with advanced adenocarcinoma in the second-line setting,” commented PD. Dr Martin Reck, Head of Department of Thoracic Oncology, Lung Clinic Grosshansdorf, Germany and lead investigator of the LUME-Lung 1 trial. “The clinical data has shown that patients receiving nintedanib plus docetaxel experienced over one year overall survival with no further compromise to their quality of life, compared to docetaxel alone.”

Adenocarcinoma is the most common type of lung cancer and the majority of patients are diagnosed in an advanced stage. Most patients will experience disease progression during or after first-line chemotherapy and there is a significant unmet need for new, effective second-line treatments.

“We are delighted by the European Commission’s decision to approve Vargatef® in the EU and feel extremely proud that our long standing commitment to oncology research and development has brought a new option to lung cancer patients with this specific type of disease,” said Professor Klaus Dugi, Chief Medical Officer, Boehringer Ingelheim. “The approval of Vargatef® expands our oncology portfolio, following last year’s approval of GIOTRIF® (afatinib) for another specific type of lung cancer. In the idiopathic pulmonary fibrosis indication, nintedanib has recently been approved by the U.S. FDA.”

New compound may lead to development of cheaper Anti-Cancer Drugs

Professor Bernie Kraatz, chair of the Department of Physical and Environmental Sciences at U of Toronto Scarborough, has developed a new compound that can be used to monitor the biochemical processes involved with a group of enzymes called protein kinases. The prevailing form of monitoring kinase activity involves the use of radioactive isotopes, which is costly because the isotopes are expensive to use and have a short shelf life. They are also difficult to work with because of the regulations associated with their handling and disposal. "We wanted to find a way to get around the hurdles involved with using radioactive isotopes," says Kraatz. "Having a compound that can be used in live cells is important because it can help clinicians better monitor kinase activity and to determine if a drug they're administering is effective in terms of regulating kinase activity."
Protein kinases play an essential role in energy transfer within all living things including controlling cell division, growth and death while also regulating metabolic pathways and even cell communication. If protein kinase activity is not regulated properly it can lead to certain diseases, including cancer.
Kraatz's team was able to develop a way to monitor the activity of protein kinase, in this case a sarcoma-related kinase, by using a redox label rather than a radioactive label. A redox label is a compound that provides an electrical signal that can be detected very accurately. The result is a safer, simpler and cheaper alternative to monitoring the efficiency of drugs and kinase activity.
"Chemists often come up with wonderful models and reactions but translating those into a biomedical environment can be very difficult," says Kraatz. "We were able to take a fantastic label that can be used perfectly well in a biomedical environment."

Experimental Cancer Treatment teaches body to fight Melanoma Cancer Cells

Many lives are cut short by Melanoma because advanced treatments don't always work.
Researchers here in Tampa are trying to change that with a new experimental therapy that they say is showing promising results. In cancer patients, the cancer cells trick the body. Their immune system sees the cells as normal, and allows them to multiply, out of control. Chemotherapy and radiation kill cancer cells, but in the process, they also kill fast-growing normal cells, too. In some cases chemo and radiation are curative. But for many patients, the cancer eventually comes back.
This new generation therapy works differently by teaching your own white blood cells to seek out and destroy cancer cells. It starts by removing some of the patient's tumor and white blood cells, called T cells, or TILs. (Tumor Infiltrating Lymphocytes.) In the lab, the two are combined. The cells are stimulated to help them recognizing the cancer as foreign. It's a process called activation.
The TILs are then allowed to multiply, a process that takes seven weeks on average.
"Our goal is to generate 50 billion," Dr. Sarnaik said. "We get a response in about half the patients, but for the home run that we're looking for, which is a complete clinical response meaning that all of the measurable tumor has gone, that happened about a fifth of the time," Sarnaik said.

Optimized Cancer Therapy with Nanomedicine Drug combinations, Nanodiamonds

Currently, doctors use people's genetic information to identify the best possible combination therapies, which can make treatment difficult or impossible when the genes in the cancer cells mutate. The new technique does not rely on genetic information, which makes it possible to quickly modify treatments when mutations arise: the drug that no longer functions can be replaced, and FSC.II can immediately recommend a new combination.
"Drug combinations are conventionally designed using dose escalation," said Dean Ho, a co-corresponding author of the study and the co-director of the Jane and Jerry Weintraub Center for Reconstructive Biotechnology at the School of Dentistry. "Until now, there hasn't been a systematic way to even know where the optimal drug combination could be found, and the possible drug-dose combinations are nearly infinite. FSC.II (the tool, Feedback System Control.II, or FSC.II, considers drug efficacy tests and analyzes the physical traits of cells) circumvents all of these issues and identifies the best treatment strategy."
The researchers demonstrated that combinations identified by FSC.II could treat multiple lines of breast cancer that had varying levels of drug resistance. They evaluated the commonly used cancer drugs doxorubicin, mitoxantrone, bleomycin and paclitaxel, all of which can be rendered ineffective when cancer cells eject them before they have had a chance to function.
The researchers also studied the use of Nanodiamonds to make combination treatments even more effective. Nanodiamonds, by-products of conventional mining and refining operations, have versatile characteristics that allow drugs to be tightly bound to their surface, making it much harder for cancer cells to eliminate them and allowing toxic drugs to be administered over a longer period of time.
The use of nanodiamonds to treat cancer was pioneered by Dean Ho, a professor of bioengineering and member of the UCLA Jonsson Comprehensive Cancer Center and the California NanoSystems Institute.
"This study has the capacity to turn drug development, nano or non-nano, upside-down," he said. "Even though FSC.II now enables us to rapidly identify optimized drug combinations, it's not just about the speed of discovering new combinations. It's the systematic way that we can control and optimize different therapeutic outcomes to design the most effective medicines possible."
The study found that FSC.II-optimized drug combinations that used nanodiamonds were safer and more effective than optimized drug-only combinations. Optimized nanodrug combinations also outperformed randomly designed nanodrug combinations.
"This optimized nanodrug combination approach can be used for virtually every type of disease model and is certainly not limited to cancer," said Chih-Ming Ho, who also holds UCLA's Ben Rich Lockheed Martin Advanced Aerospace Tech Endowed Chair. "Additionally, this study shows that we can design optimized combinations for virtually every type of drug and any type of nanotherapy."

London, scientists find Cancer weak spots for New Targeted Drugs

A major computational analysis by scientists at the University of Sussex and The Institute of Cancer Research, London, has found a number of potential targets for drugs that exploit the inherent weaknesses of cancer cells.The findings could lead to personalised medicine that 'reads' a cancer patient's DNA and only attacks defective cells, in contrast to the scattergun approach of conventional chemotherapy, which attacks all dividing cells, including healthy ones.
The study is published today (Tuesday 24 February 2015) in the journal Nature Reviews Cancer.
Scientists from the University of Sussex and The Institute of Cancer Research (ICR) analysed the patterns of mutations found in the DNA sequences of tumors from more than 5,000 cancer patients.
The team, jointly led by Dr Frances Pearl (Sussex) and Dr Bissan Al-Lazikani (ICR), focused on the 'DNA repair' systems that protect the genetic information of the cell, and are mutated in almost all cancers. Breaking these systems for DNA repair allows cancer cells to divide uncontrollably and generate even more mutations, helping them become resistant to chemotherapy and radiation treatments.
"Knowing which DNA repair processes are defective in an individual tumor allows us to target new drugs that are only toxic to cells with a particular pattern of mutations, cancer cells," said Dr Pearl, who heads the Bioinformatics Research Group at Sussex.
One class of drug called PARP inhibitors already target DNA repair systems. They are being used in clinical trials to treat women with breast or ovarian cancers that have mutations in BRCA genes, and one of the class, Olaparib, has recently been licensed for women with ovarian cancer in Europe and the US.

Novartis's, Jakavi superior to standard therapy in rare blood Cancer Polycythemia Vera

Novartis announced that The New England Journal of Medicine (NEJM) published results from the pivotal Phase III clinical trial demonstrating Jakavi^® (ruxolitinib) significantly improved hematocrit control without the need for phlebotomy (a procedure to remove blood from the body to reduce the concentration of red blood cells) and reduced spleen size in patients with polycythemia vera (PV) who had an inadequate response to or unacceptable side effects from hydroxyurea as defined according to the modified European LeukemiaNet (ELN) criteria. In PV, hematocrit control and spleen size reduction are key measures of a patient's response to therapy.

PV is a chronic, incurable blood cancer associated with an overproduction of blood cells that can cause serious cardiovascular complications, such as stroke and heart attack. Approximately 25% of patients with PV develop resistance to or intolerance of hydroxyurea and are considered to have uncontrolled disease, which is typically defined as hematocrit levels greater than 45%, elevated white blood cell count and/or platelet count, and may be accompanied by debilitating symptoms and/or enlarged spleen. Elevated white blood cell count and hematocrit are also associated with an increased risk of blood clots.

"A key challenge in treating patients with PV is the development of resistance or intolerance to currently available therapies such as hydroxyurea, which leaves us with very limited alternative treatment options to effectively manage the disease," said Dr. Alessandro M. Vannucchi, Azienda Ospedaliera Universitaria Careggi, University of Florence, Italy and lead study author. "This study indicates that ruxolitinib may represent an important advance for this population of patients with PV, a disease that can lead to serious complications and difficult daily symptoms."

Wisdom teeth Stem Cells can transform into cells that could treat corneal scarring

Corneal blindness, which affects millions of people worldwide, is typically treated with transplants of donor corneas, said senior investigator James Funderburgh, Ph.D., professor of ophthalmology at Pitt and associate director of the Louis J. Fox Center for Vision Restoration of UPMC and the University of Pittsburgh, a joint program of UPMC Eye Center and the McGowan Institute for Regenerative Medicine.
"Shortages of donor corneas and rejection of donor tissue do occur, which can result in permanent vision loss," Dr. Funderburgh said. "Our work is promising because using the patient's own cells for treatment could help us avoid these problems."
Experiments conducted by lead author Fatima Syed-Picard, Ph.D., also of Pitt's Department of Ophthalmology, and the team showed that stem cells of the dental pulp, obtained from routine human third molar, or wisdom tooth, extractions performed at Pitt's School of Dental Medicine, could be turned into corneal stromal cells called keratocytes, which have the same embryonic origin.
The team injected the engineered keratocytes into the corneas of healthy mice, where they integrated without signs of rejection. They also used the cells to develop constructs of corneal stroma akin to natural tissue.
"Other research has shown that dental pulp stem cells can be used to make neural, bone and other cells," Dr. Syed-Picard noted. "They have great potential for use in regenerative therapies."

Chiesi gains a pioneering marketing OK for Stem Cell Therapy in Euro-Nations

The European Commission has waved through the continent's first stem cell therapy, providing conditional marketing approval for what is essentially a new lens that can be fashioned out of a patient's own cells and used to restore sight in people whose eyes have been severely damaged.
Italy's Chiesi now can begin the process of selling the treatment, dubbed Holoclar, 7 long years after gaining an orphan drug designation and more than 15 years after establishing a proof of concept for the treatment method. Holoclar will be made by a company called Holostem, a spinout from the University of Modena which Chiesi backed to develop a qualified manufacturing operation that could win over regulators to the complex technology.
Thousands of people in Europe may benefit from this treatment, which will be closely monitored by health officials under the conditional OK that was granted.
Eyes damaged by burns or chemicals can lose their ability to regenerate the corneal surface of the eye, a process that naturally occurs in undamaged eyes every few months. With Holoclar, physicians can harvest some of the remaining cells required for this regeneration and construct a patch that can be used to repair the damage. And because these are autologous cells (from the patient), Chiesi can do this without spurring a rejection.

NEW Chemotherapy Combo Regimen can lengthen Breast Cancer patients' survival

A two-year long clinical trial conducted around the world shows a drug and chemotherapy combination can lengthen the survival for patients with HER2-positive metastatic breast cancer. The author of the CLEOPATRA study says the findings give patients with an aggressive disease hope to live a longer life.
Dr. Sandra Swain of MedStar Washington Hospital Center is the study's author. She says that this could be the start of a very promising future for these patients.
"It really is dramatic results. To have a 16-month improvement in survival, we've never seen that before. It really is unprecedented," says Dr. Swain.
HER2-positive tumors are aggressive and spread quickly. In this study, patients received two cancer drugs, Perjeta and Herceptin, that together target the HER2 gene along with chemotherapy.
"They block any kind of signaling or talking that these proteins have to the cell to make it grow," according to Dr. Swain.
Another plus: the tumor shrinks in nearly 80 percent of the patients who received this combo. Dr. Swain added, "And those patients who have their tumor shrink it stays small for eight months longer than if they just gotten Herceptin."
Dr. Swain says if you are fighting HER2-positive breast cancer, now is the time to ask your doctor about this regimen. The combination is currently being tested in patients who have tumors at an early stage.
"I see that as a potential cure if it actually works well in the early stage as it has in the advanced stage," she told us.
She's not using the term cure lightly.

"I've spent my life trying to find a cure, so I really believe that. I really believe that these drugs have the potential for doing that," Dr. Swain told us.

Foundation Medicine, H3 Biomedicine partner to develop Cancer Drugs

A partnership announced today between two Cambridge companies, Foundation Medicine and H3 Biomedicine, is further evidence of the role precision medicine is playing in fueling the growth of diagnostics. That is the same idea, which is behind today's announcement of the multi-year partnership between Foundation Medicine and H3, which is intended to help find new genetic causes of cancers as well as drug to address them. Under the agreement, H3 will pay Foundation
Medicine for access to its genetic data as well as various milestones and royalties for any drugs developed out of that partnership. While the dollar amounts and exact time frames are not being disclosed, the deal affords the Cambridge maker of two cancer diagnostics; FoundationOne and FoundationOne Heme, yet another source of revenue as the company works to get the tests covered by insurance companies.
"Large-scale genomic data sets are the key to identifying actionable targets and addressing the multitude of difficult-to-treat cancers," Warmuth said in a statement. "We believe Foundation Medicine offers one of the most comprehensive cancer genomics knowledge bases available. This collaborative effort will further empower our state-of-the-art discovery engines to deliver novel first-in-class medicines and improve our clinical development strategies by accessing a wealth of clinically annotated, genomic information at least in part derived from later-stage and metastatic cancers."
H3, the wholly-owned subsidiary of the Japanese drug company, Eisai, has a 10-year, $200 million commitment which has allowed it to build its own 48,000 square-foot lab and hire 75 full-time employees to date. Warmuth said the company is now "looking to expand the investor base" and is also seeking partners in the bio-pharmaceutical industry. H3 has two lead drug programs, one in blood cancer and one in liver cancer, for which it hopes to apply this year for approval to begin clinical trials.

Bristol-Myers adds to Cancer-Treatment push with acquisition

Bristol-Myers Squibb is using an acquisition that could be worth more than $1 billion and a separate collaboration agreement to continue its push into developing cancer treatments that use a patient's immune system to attack the disease.
The New York drug maker said Monday that it will buy the privately held biotechnology company Flexus for $800 million up front and another $450 million in possible milestone payments. That deal gives Bristol-Myers access to a portfolio of potential treatments that aim to help the body's immune system attack tumors more effectively. These treatments reduce the production of Kynurenine, which comes from tumor-generated enzymes and can hamper the immune system's ability to identify and destroy certain types of tumors.
The boards of directors of both companies and Flexus shareholders have approved the deal, which is expected to close later in the first quarter.
Bristol-Myers Squibb Co. has been making a long-term transformation from producing pills for the masses to creating complex, expensive drugs for cancer and rare disorders. It's been investing significantly in immuno-oncology treatments, or drugs that take the brakes off the immune system so it can better recognize and attack cancer cells.
The company also said Monday that it also will start a collaboration with Rigel Pharmaceuticals Inc. to develop and sell so-called immuno-therapies from Rigel's portfolio of potential treatments.

New Ovarian Cancer drug 'Lynparza' one step closer

Ovarian cancer patients throughout Europe and the United States could soon be treated with a new drug discovered through pioneering research at the University of Sheffield funded by Yorkshire Cancer Research. Lynparza has become the first of a new class of drugs called PARP inhibitors to be granted approval by the European Commission and the US Food and Drug Administration (FDA). The National Institute for Health and Care Excellence (NICE) will now assess the drug to see whether it should become widely available on the NHS, with a decision expected in September 2015.
In 2005, Professor Thomas Helleday and his team of researchers at the University of Sheffield demonstrated how PARP inhibitors could be used as a tailored treatment for patients with BRCA2 mutations, which are seen in both hereditary ovarian and breast cancer.
The discovery was patent protected and licensed to pharmaceutical company Astra-Zeneca who continued the development process and undertook successful clinical trials.
Professor Helleday said: "I am delighted that after all the hard work carried out since 2005 when we made the original discovery, Lynparza has now been licenced by the European Commission and the FDA. 150,000 women in the European Union suffer from ovarian cancer. Last year, 21,980 women were diagnosed with ovarian cancer in the United States. Many of these patients will benefit from this new therapy.
PARP inhibitors work by blocking the cancer cell's ability to repair damage. Healthy cells reproduce by dividing DNA into two strands and copying each strand. Before they do this, they repair damage in the DNA using the PARP protein.
If PARP is suppressed, normal cells use a second mechanism for DNA repair. Some cancers cannot undergo this second process because they have a mutation in a BRCA gene. These cancer cells rely completely on PARP to fix the damage and so when PARP is suppressed the BRCA mutated cell is unable to grow and eventually dies. Normal cells of the patient with a functioning BRCA are not affected.The novel element of the treatment is the fact that it is the mutation causing cancer that is exploited to specifically kill the cancer; this reduces the side effects often experienced with traditional anti-cancer treatments.

Lynparza has become the first of a new class of drugs called PARP inhibitors to be granted approval by the European Commission and the US Food and Drug AdminisThe National Institute for Health and Care Excellence (NICE) will now assess the drug to see whether it should become widely available on the NHS, with a decision expected in September 2015In 2005, Professor Thomas Helleday and his team of researchers at the University of Sheffield demonstrated how PARP inhibitors could be used as a tailored treatment for patients with BRCA2 mutations, which are seen in both hereditary ovarian and breast cancer.

Study shows Gazyva offers strong benefits as Lymphoma Cancer drug

Swiss drugmaker Roche said on Wednesday a late-stage study involving non-Hodgkin's lymphoma patients showed they lived longer without the disease worsening when treated with its Gazyva drug, giving a boost to its line-up of cancer treatments.
Gazyva - marketed as Gazyvaro in the European Union and Switzerland - is being positioned as an alternative follow-on medicine to Roche's Rituxan, or MabThera, which generated 6.9 billion Swiss francs ($7.46 billion) in sales last year.
Roche is hoping to switch as many patients as possible to the newer product before Rituxan faces competition from cheaper copies when its patent protection expires.
The study showed that patients with relapsed slow-growing, or indolent non-Hodgkin's lymphoma lived significantly longer without their disease worsening when treated with Gazyva and bendamustine followed by Gazyva alone, the company said in a statement.

Data from the study will be submitted to the U.S. Food and Drug Administration, European Medicines Agency and other health authorities for consideration for approval, the company said.
The performance of Gazyva against Rituxan in two ongoing head-to-head late-stage trials will be another important indicator, analysts said.
The trials involve patients with large B-cell lymphoma and indolent non-Hodgkin's lymphoma.

Karcinolys receives U.S. and EU orphan designations for the treatment of Pancreatic Cancer

Paris, 2015: Karcinolys SAS, a privately-held biotechnology company, today announced that orphan designations have been granted by the U.S. Food and Drug Administration (FDA) and by the European Commission for its oncolytic virus Myb34.5 for the treatment of pancreatic cancer. The U.S. Orphan Drug Act (ODA) provides for granting a special status to a drug or biological product intended to treat a rare disease or condition. An orphan designation qualifies the sponsor of the drug for various development incentives and seven years of U.S. market exclusivity after marketing approval. European orphan medicinal product status confers up to ten years of marketing exclusivity in all European Union (EU) member countries following marketing approval, and a range of other benefits including EU-funded research, protocol assistance, and fee reductions. “We are committed to developing a therapy for one of the most insidious and lethal cancers, particularly as only standard treatments relying on a few approved palliative chemotherapies and targeted therapies are currently available,” said Jean-Luc Béjot, MD, MBA, Chief Executive Officer of Karcinolys. “We are pleased that the FDA and the European Commission have granted Orphan Designation to Myb34.5 as a potential new therapy for Pancreatic Cancer, a disease with great unmet medical need.”

One-two punch catches Cancer Cells in vulnerable state

Timing may be decisive when it comes to overcoming cancer's ability to evade treatment. 
By hitting breast cancer cells with a targeted therapeutic immediately after chemotherapy, researchers from Brigham and Women's Hospital (BWH) were able to target cancer cells during a transitional stage when they were most vulnerable, killing cells and shrinking tumors in the lab and in pre-clinical models.
"We were studying the fundamentals of how resistance develops and looking to understand what drives relapse. What we found is a new paradigm for thinking about chemotherapy," said senior author Shiladitya Sengupta, PhD, associate bioengineer at BWH.
Previous studies have examined cancer stem cells (CSCs), small populations of cells within a tumor that are resistant to chemotherapy.
Sengupta and his colleagues took breast cancer cells that did not have the markings of CSCs and exposed them to docetaxel, a common chemotherapy drug.
The team found that after exposure to chemotherapy, the cells began developing physical markings usually seen in CSCs, including receptors on the cell surface to which specific proteins can bind.
These "markers of stemness" suggested that the cells were transitioning into a different state, during which time they might be vulnerable to other cancer drugs.
To test this, the researchers treated the cells with a variety of targeted therapeutics immediately after chemotherapy.
The researchers observed that two drugs each killed a large fraction of the cells that had begun transitioning: dasatinib, a drug that targets the Src Family Kinase (SFK) and RK20449, a new drug in pre-clinical testing that specifically targets one of the SFK proteins called Hck.
The researchers theorize that the cancer cells go through a temporary transition state, which means that administering the drugs in a very specific time-frame and sequence is important.
"By treating with chemotherapy, we're driving cells through a transition state and creating vulnerabilities," said first author Aaron Goldman, PhD, a postdoctoral fellow in biomedical engineering at BWH.
"This opens up the door: we can then try out different combinations and regimens to find the most effective way to kill the cells and inhibit tumor growth."

Stem Cell Therapy fixes post-surgical airway abnormality

"This is another interesting new therapeutic approach for stem cells," said lead researcher Dr. Francesco Petrella, deputy director of thoracic surgery at the European Institute of Oncology in Milan, Italy.
The patient, a 42-year-old firefighter, developed the fistula after surgeons removed a lung as part of treatment for mesothelioma cancer. A fistula is abnormal tissue connecting an organ, blood vessel or intestine to another structure. In this case, the fistula developed between the lower airway and the tissue that surrounds the lungs.
"Our clinical experience supports the idea that stem cells could be effectively used to close some tissue defects developing after very complex surgical procedures, thus restoring a functioning airway," Petrella said.
A fistula that develops after chest surgery is serious and even deadly, Petrella said. Current treatments involve removing ribs and taking medications for months or years, he explained.
"Less invasive approaches like endoscopic glue injections have only poor results, so our proposed techniques could improve quality of life in these patients," Petrella said.
Sixty days after stem cell therapy, the firefighter's fistula was healed, the researchers said. The hole seen before stem cell therapy was no longer visible, having been replaced by new tissue created by the stem cell implant, they explained.
Some people are born with a fistula. Other causes of fistulas include complications from surgery, injury, infection and diseases, such as Crohn's disease or ulcerative colitis.
Petrella believes that this same stem cell technique could be used to treat fistulas that develop elsewhere in the body.
"For example, I think it could be used to treat fistulas in the esophagus as well as in the stomach or colon, and fistulas that connect the lower portion of the large intestine with the rectum and vagina," he said.

Eight cancer drugs to be denied on NHS England

Thousands of cancer patients will be denied NHS drugs following a decision to withdraw funding for at least eight life-extending treatments.

Medication which offers a last chance to patients with breast, prostate and bowel disease will no longer be funded by the NHS, under plans to cut spending.

Ahead of the last election, the Conservatives pledged to introduce a Cancer Drugs Fund so that cancer patients were no longer denied drugs on the grounds of cost.

But the £200m fund, introduced in 2011, has overspent its budget, despite the fact it was increased to £280m, leading to a review of 25 drugs which are used in 42 treatments.
The treatments include six drugs for breast cancer – Halaven, Avastin, Kadcyla, Afinitor, Tyverb and Perjeta. They also include Jevtana - the only treatment for hormone resistant advanced prostate cancer, when disease has progressed in spite of chemotherapy – and Zaltrap, a drug used for bowel cancer drug that can extend life after a tumour has spread. The two latter drugs have been requested for NHS funding for 2,000 patients in the last 18 months, while the breast cancer drugs have been funded around 3,000 times in the last year. Danni Manzi, head of policy at Breast Cancer Care charity, said: “This re-evaluation process is a warning sign that the entire Fund is unravelling.
“We are extremely concerned that breast cancer drugs could be removed.
“It would be soul destroying for many secondary breast cancer patients if they were unable to access these drugs simply due to cost.
"We want to get more drugs to people more quickly and in the UK today there are some people – thousands of people. who want a certain cancer drug, whose doctors tell them they should have a certain cancer drug, who don't get it."
At the same time as reassessing current drugs, the NHS is also evaluating 12 new therapies.
Any patient currently having a drug paid for by the fund will not be affected by any changes which come into effect in March 2015.
Professor Peter Clark, an oncologist and chairman of the Cancer Drugs Fund, said: "We need to get maximum value for every pound we spend".
"We can no longer sustain a position where we are funding drugs that don't offer sufficient clinical benefit when drugs that will do more for patients are coming on stream.

Europe's Cancer Drugs shortage is hurting patients

Hospital pharmacies across Europe are reporting difficulties getting hold of a range of commonly used cancer drugs, including 5-fluorouracil, carboplatin, cisplatin, doxo-rubicin, etoposide, melphalan, methotrexate, oxaliplatin and vincristine. Medicines used for pain relief including morphine are also reported to be in short supply in some countries. The problem has only recently come to light thanks to a survey carried out by the European Association of Hospital Pharmacists (EAHP), which was sent to 600 hospital pharmacies across 36 countries. Although there were significant differences between European countries in the frequency and nature of shortages experienced, none were spared this problem. Denmark, Iceland, Malta, Romania and the UK all reported that medicines shortages are a daily occurrence. Only 14% of respondents said they never had trouble getting hold of vital medicines, while 66% reported this as a daily or weekly problem. Cancer drugs were ranked as the second most commonly affected area for shortages (55% of respondents) after anti-microbials (57%). Difficulties and delays in getting hold of the right cancer drugs can seriously damage patient care. Medicines shortages lead to delayed or interrupted treatment, or dose reductions, which can have life-threatening consequences. The use of an alternative medicine increases the likelihood of a medication error and can result in the patient experiencing unnecessary side-effects. There is also a cost associated with the time hospital pharmacists have to spend in sourcing and procuring an alternative medicine, which is often more expensive than the one originally prescribed. The EAHP is now calling for a more reliable cataloguing of medicines in short supply across Europe, comparable to the list recently established by the EMA’s American counterpart, the FDA, which details the reasons for, and possible duration of, drug shortages, as well as suggesting potential alternatives. Such a list would provide health professionals with the information necessary to anticipate and manage the problem.

Development of personalized cellular therapy for Brain Cancer

The new preclinical study, conducted in collaboration with Hideho Okada, MD, PhD and his colleagues at the University of Pittsburgh, details the design and use of T cells engineered to express a chimeric antigen receptor (CAR) that targets a mutation in the epidermal growth factor receptor protein called EGFRvIII, which is found on about 30 percent of glioblastoma patients' tumor cells. More than 22,000 Americans are diagnosed with glioblastoma each year. Patients whose tumors express the EGFRvIII mutation tend to have more aggressive glioblastomas. Their tumors are less likely to respond favorably to standard therapies and more likely to recur following those treatments.
"Patients with this type of brain cancer have a very poor prognosis. Many survive less than 18 months following their diagnosis," said M. Sean Grady, MD, the Charles Harrison Frazier Professor and chair of the department of Neurosurgery. "We've brought together experts in an array of fields to develop an innovative personalized immunotherapy for certain brain cancers."
The new trial is led by Donald M. O'Rourke, MD, an associate professor of Neurosurgery, who oversees an interdisciplinary collaboration of neurosurgeons, neuro-oncologists, neuropathologists, immunologists, and transfusion medicine experts.
First, the team developed and tested multiple antibodies, or what immunologists call single-chain variable fragments (scFv), which bind to cells expressing EGFRvIII on their surface. The scFvs recognizing the mutated EGFRvIII protein must be rigorously tested to confirm that they do not also bind to normal, non-mutated EGFR proteins, which are widely expressed on cells in the human body.
The researchers then generated a panel of humanized scFvs and tested their specificity and function in CAR modified T cells. (Humanized scFvs are molecularly changed from their origins in non-human species to increase their similarity to human antibodies.) Out of the panel of humanized scFvs that were tested, the researchers selected one scFv to explore further based on its binding selectivity for EGFRvIII over normal non-mutated EGFR. They also evaluated the EGFRvIII CAR T cells in an assay utilizing normal EGFR-expressing skin cells in mice grafted with human skin. They found that the engineered EGFRvIII CAR T cells did not attack cells with normal EGFR in this model. The lead scFv was then tested for its anti-cancer efficacy. Using human tumor cells, the scientific team determined that the EGFRvIII CAR T cells could multiply and secrete cytokines in response to tumor cells bearing the EGFRvIII protein. Importantly, the researchers found that the EGFRvIII CAR T cells controlled tumor growth in several mouse models of glioblastoma, as measured by magnetic resonance imaging (MRI) and luminescence of tumors in the mouse brains. In the mouse model, the EGFRvIII CAR T cells caused tumor shrinkage when measured by MRI and were also effective in eliminating tumors when administered in combination with temozolomide chemotherapy that is used to treat patients with glioblastoma.
The new trial will enroll 12 adult patients whose tumors express EGFRvIII, in two groups: One arm of 6 patients whose cancers have returned after receiving other therapies, and one arm of 6 patients who are newly diagnosed with the disease and still have 1 cm or more of tumor tissue remaining after undergoing surgery to remove it.

Liver cells from Stem Cells can be infected with Malaria and used to test potential drugs

In 2008, the World Health Organization announced a global effort to eradicate malaria, which kills about 800,000 people every year. As part of that goal, scientists are trying to develop new drugs that target the malaria parasite during the stage when it infects the human liver, which is crucial because some strains of malaria can lie dormant in the liver for several years before flaring up.
A new advance by MIT engineers could aid in those efforts: The researchers have discovered a way to grow liver-like cells from induced pluripotent stem cells. These cells can be infected with several strains of the malaria parasite and respond to existing drugs the same way that mature liver cells taken from human donors do.
Such cells offer a plentiful source for testing potential malaria drugs because they can be made from skin cells. New drugs are badly needed, since some forms of the malaria parasite have become resistant to existing treatments, says Sangeeta Bhatia, the John and Dorothy Wilson Professor of Health Sciences and Technology (HST) and Electrical Engineering and Computer Science at MIT.
“Drug resistance is emerging that we are continually chasing. The thinking behind the call to eradication is that we can’t be chasing resistance and distributing bed nets to protect from mosquitoes forever. Ideally, we would rid ourselves of the pathogen entirely,” says Bhatia, who is also a member of MIT’s Koch Institute for Integrative Cancer Research and Institute for Medical Engineering and Science (IMES).
Until now, malaria researchers have not had many reliable ways to test new drugs in liver tissue. “What’s historically been done is people have tried to make do with the systems that were available,” Bhatia says. Those systems include testing drugs in cancerous liver cells or in mice infected with a rodent-specific version of the malaria parasite. However, cancerous cells divide much more frequently than normal adult liver cells, and are missing some of the genes required for drug metabolism. The mouse model is not ideal because the rodent version of malaria is different from the human one, so drugs that are successful in mice don’t always work in humans.
“This study is a major breakthrough,” says Dyann Wirth, chair of the Department of Immunology and Infectious Diseases at the Harvard School of Public Health, who was not part of the research team. “This technique may prove not only a useful tool for finding drugs that will target the liver stage of the parasite, but it could also contribute to our fundamental understanding of the parasite.”

The practice of private Stem Cell clinics in Australia

Even though stem cell science is at a relatively early stage and there are limited mature clinical data, adult stem cell therapies are already being provided directly to patients in private clinics across Australia. The study identified 17 private clinics and three stem cell companies that currently provide autologous adult stem cell therapies. These clinics offer autologous stem cell therapies for a wide range of diseases outside of clinical trials, including OA and musculoskeletal pain; neurodegenerative disorders such as muscular dystrophy and MS; stroke; retinal neuropathy; spinal cord injury; headache and migraine; asthma; autism; ‘facial rejuvenation’; and anti-aging. The medical professionals who provide stem cell therapies in these private clinics in Australia include cosmetic surgeons, sports medicine physicians, orthopedic surgeons, and general practitioners, and specialists in the diseases being treated in these clinics, such as neurologists, generally are not involved in the provision of care. Most of these clinics have websites with detailed information regarding treatments available, media coverage of the clinic, health professional profiles, and the cost of treatment.
Seven of these clinics claim to participate in research regarding stem cell therapies. However, with one exception, the health professionals at these clinics appear not to have published their findings in peer-reviewed journals. On their websites and in media interviews, both the clinics and the health professionals who provide stem cell therapies frequently make claims about efficacy and safety that cannot be supported by published evidence.
In Australia, the regulation of therapeutic goods, including prescription, non-prescription, and complementary medicines as well as medical devices, is overseen by the Therapeutic Goods Administration (TGA). The TGA regulates cell products, including blood products, vaccines, and hematopoietic stem cells, used in allogeneic transplantation. However, the TGA has specifically excluded from its regulatory jurisdiction human cells that are collected from a patient who is under the clinical care and treatment of a registered medical practitioner if the cells are manufactured by that medical practitioner for therapeutic application in a single treatment. Consequently, any registered medical practitioners in Australia can offer autologous stem cell therapy to patients for a single treatment or disease, such as OA, completely outside of any form of regulation by the TGA. 

Gold nanotubes launch a three-pronged attack on Cancer Cells

Scientists have shown that gold nanotubes have many applications in fighting cancer: internal nanoprobes for high-resolution imaging; drug delivery vehicles; and agents for destroying cancer cells. Study lead author Dr Sunjie Ye, who is based in both the School of Physics and Astronomy and the Leeds Institute for Biomedical and Clinical Sciences
at the University of Leeds, said:  “High recurrence rates of tumors after surgical removal remain a formidable challenge in cancer therapy. Chemo- or radiotherapy is often given following surgery to prevent this, but these treatments cause serious side effects.

Gold nanotubes, that is, gold nanoparticles with tubular structures that resemble tiny drinking straws  have the potential to enhance the efficacy of these conventional treatments by integrating diagnosis and therapy in one single system.”

“When the gold nanotubes travel through the body, if light of the right frequency is shone on them they absorb the light. This light energy is converted to heat, rather like the warmth generated by the Sun on skin. Using a pulsed laser beam, we were able to rapidly raise the temperature in the vicinity of the nanotubes so that it was high enough to destroy cancer cells.” In cell-based studies, by adjusting the brightness of the laser pulse, the researchers say they were able to control whether the gold nanotubes were in cancer-destruction mode, or ready to image tumors.

“The nanotubes can be tumor-targeted and have a central ‘hollow’ core that can be loaded with a therapeutic payload. This combination of targeting and localized release of a therapeutic agent could, in this age of personalized medicine, be used to identify and treat cancer with minimal toxicity to patients.” 

The use of gold nanotubes in imaging and other biomedical applications is currently progressing through trial stages towards early clinical studies. 

New study reveals how to improve chemotherapy use in Prostate Cancer

Prostate cancer is the second leading cause of cancer for men in the United States. Only one class of chemotherapy called taxanes is effective against the disease. A study published online in Clinical Cancer Research, researchers have found that a newer member of the taxane family called cabazitaxel, an FDA approved drug, has properties that could make it more effective for some patients -- a hypothesis currently being tested in clinical trials. Researchers also found a genomic marker that could help physicians identify which patients might benefit most from cabazitaxel.
"It was surprising to find that cabazitaxel functions differently than docetaxel in killing cancer cells, even though they're both taxanes," says senior author Karen Knudsen, Ph.D., Interim Director of the Sidney Kimmel Cancer Center and a professor of cancer biology at the Sidney Kimmel Medical College at Thomas Jefferson University. "It shows that we may not be taking full advantage of this next generation taxane in the clinic."
For years, docetaxel has been the only effective chemotherapy for men whose cancer was no longer responding to hormone treatments. The next generation drug in the taxane family, cabazitaxel, was approved in 2010, but only for patients whose cancer no longer responded to hormone therapy or docetaxel treatment.
Drs. Kelly and Knudsen are testing their hypothesis in a phase II clinical trial (ABICABAZI NCT02218606), currently recruiting patients. The study is funded by Sanofi and conducted as a collaboration between Sidney Kimmel Cancer Center and Memorial-Sloan Kettering Cancer Center. Patients with metastatic prostate cancer who have not yet been treated with chemotherapy will be given either the second-line hormone therapy abiraterone, or abiraterone in combination with cabazitaxel. In addition, researchers will scan the tumors for their RB gene expression to test whether low levels of RB correlate with strong responses to cabazitaxel.
"These results from our laboratory research give us a strong reason to believe that this drug could be more useful to some men earlier in their course of treatment," says Dr. Knudsen. "The ABICABAZI trial puts these ideas to the test in humans, and if we are correct, has the capacity for the first time to tell us what patients might most benefit from chemotherapy."

Changing stem cell structure may help fight Obesity, Cancer

The research, conducted at Queen Mary University of London (QMUL), found that a slight regulation in the length of primary cilia, small hair-like projections found on most cells, prevented the production of fat cells from human stem cells taken from adult bone marrow.

Part of the process by which calories are turned into fat involves adipogenesis, the differentiation of stem cells into fat cells. The researchers showed that during this process of adipogenesis, the length of primary cilia increases associated with movement of specific proteins onto the cilia. Furthermore, by genetically restricting this cilia elongation in stem cells the researchers were able to stop the formation of new fat cells. Recent research has found that many conditions including kidney disease, blindness, problems with bones and obesity can be caused by defects in primary cilia. Melis Dalbay, co-author of the research from the School of Engineering and Materials Science at QMUL, said: “This is the first time that it has been shown that subtle changes in primary cilia structure can influence the differentiation of stem cell into fat. Since primary cilia length can be influenced by various factors including pharmaceuticals, inflammation and even mechanical forces, this study provides new insight into the regulation of fat cell formation and obesity.” Professor Martin Knight, a bioengineer and lead author of the research, said: “This research points towards a new type of treatment known as ‘cilia-therapy’ where manipulation of primary cilia may be used in future to treat a growing range of conditions including obesity, cancer, inflammation and arthritis.”

NovoTTF-100A: The 'Anti-Cancer Hat' giving hope to brain tumor patients

NovoTTF-100A,the device, known among researchers as the “anti-cancer hat”, may soon prove a lifeline to patients with brain tumors. Early trials of the device are reported to increase patients’ chances of surviving two years by up to 50 percent.

A white skull-cap wired to a battery pack, the device, which has been in development for more than 14 years, has been hailed as a new, effective treatment for cancer that does not require doses of debilitating chemicals, radiation, or the surgeon’s knife.
Its manufacturer, Novocure, says it can help to rid you of cancer as you pack the shopping or do the washing up. That may or may not prove correct, but early evidence suggests that the treatment is highly effective.
The cap works by preventing cancer cells from dividing by emitting a wave-like electrical field into the brain. Developed exclusively to treat the most common form of adult brain cancer, Glioblastoma, it is made for a disease with a very poor prognosis: the average life expectancy is only 14 months from diagnosis, even with chemotherapy and radiotherapy.
Of the 315 Glioblastoma patients who entered a recent trial, all of whom were also receiving chemotherapy, 43 percent wearing the device were alive two years later, compared with 29 per cent who were not. Removed Glioblastoma tumors also took longer to regrow: 7.1 months compared with four months.
Ariane, 35, who lives in Germany, is among those early patients to wear the cap. She was diagnosed almost two years ago while pregnant. Her baby survived, but Ariane’s Glioblastoma returned, despite her having undergone surgery, chemotherapy and radiotherapy. She now wears the device everywhere and is happy not to suffer the side effects of chemotherapy.
“I do everything like I did it before,” she said. “I continue to play with my one-year-old son. I go walking and shopping and I just put the battery pack in the storage compartment of the buggy.”

Keck Stem Cell researchers, garner $4.3 million in funding

Three scientists from Keck Medicine of USC have won grants exceeding $4.3 million from the California Institute for Regenerative Medicine (CIRM) for research that includes creating a temporary liver for transplant patients, finding novel ways to treat immune disorders and blood diseases, and developing a new animal model for exploring diseases such as heart failure, diabetes and neurodegenerative diseases.
The grants were received by USC Stem Cell Principal Investigators Paula Cannon, PhD; Toshio Miki, MD, PhD; and Qi-Long Ying, PhD. The funds are part of the CIRM Tools and Technologies initiative, which supports projects addressing the challenges of translating stem cell discoveries into cures. The winners were chosen from 212 proposals to create, design and test the key technologies needed to usher in the era of regenerative medicine.
“Sometimes even the most promising therapy can be derailed by a tiny problem,” said Jonathan Thomas, JD, PhD, chair of CIRM’s governing board. “These awards are designed to help find ways to overcome those problems, to bridge the gaps in our knowledge and to ensure that the best research is able to keep progressing and move out of the lab and into clinical trials in patients.”
Miki’s team at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC plans to develop what he calls an “extracorporeal liver support system (ELS)” for patients with liver failure. The ELS will house a collection of human liver cells, produced from stem cells, in a device outside of the patient’s body but connected to the patient’s circulation. The ELS will therefore be able to function as a temporary liver: removing toxins, preventing irreversible brain damage, and giving the patient’s own liver a chance to recover and regenerate.
If successful, the device will allow patients to recuperate without undergoing liver transplantation.

Cannon plans to improve the precision and safety of “targeted nucleases,” which she describes as “scissors” used to edit specific genes in hematopoietic or blood-forming stem cells. Cannon hopes to develop the next generation of targeted nucleases to treat severe immune deficiencies and blood diseases, such as sickle cell disease.
Cannon and her colleagues have already developed a targeted nuclease that could potentially cure HIV/AIDS by introducing a mutation in a gene called CCR5 that confers natural immunity to HIV.

Ying, who is also at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC, plans to use stem cell-based technology to create genetically modified laboratory rats for research into new therapies for heart failure, diabetes and neurodegenerative diseases.
Transgenic rats will provide an even more powerful tool than transgenic mice, which for nearly 25 years have allowed scientists to study and model a wide range of diseases that also occur in humans. The rat is widely accepted as more similar to the human in its physiology, which is essential for cardiac, metabolic and neurological studies.

Human Neural Stem Cells alleviate `chemobrain` post chemotherapy

Scientists have explained how human neural stem cells repair cognitive function after cancer treatments. According to UC Irvine researchers, stem cell treatments are showing promise for reversing learning and memory deficits after chemotherapy. In preclinical studies using rodents, they found that stem cells transplanted one week after the completion of a series of chemotherapy sessions restored a range of cognitive functions, as measured one month later using a comprehensive platform of behavioral testing. In contrast, rats not treated with stem cells showed significant learning and memory impairment.

The frequent use of chemotherapy to combat multiple cancers could produce severe cognitive dysfunction, often referred to as "chemobrain," which can persist and manifest in many ways long after the end of treatments in as many as 75 percent of survivors, a problem of particular concern with pediatric patients.

Charles Limoli, a UCI professor of radiation oncology, said that the findings provide the first solid evidence that transplantation of human neural stem cells can be used to reverse chemotherapeutic-induced damage of healthy tissue in the brain.

For the study, adult neural stem cells were transplanted into the brains of rats after chemotherapy. They migrated throughout the hippocampus, where they survived and differentiated into multiple neural cell types. Additionally, these cells triggered the secretion of neurotrophic growth factors that helped rebuild wounded neurons.

Importantly, Limoli and his colleagues found that engrafted cells protected the host neurons, thereby preventing the loss or promoting the repair of damaged neurons and their finer structural elements, referred to as dendritic spines.

The study suggests that stem cell therapies may one day be implemented in the clinic to provide relief to patients suffering from cognitive impairments incurred as a result of their cancer treatments, Limoli said.

Stem Cells from Placenta show promise for treating Heart Failure

Stem cells collected from placenta, which is generally discarded after childbirth, show promise as a treatment for heart failure. Found in the latest issue of STEM CELLS Translational Medicine, a new study using mice determined that human-derived adherent cells (PDAC® cells) significantly improved cardiac function when injected into the heart muscle.
Currently, about 6 million people in the United States alone suffer from heart failure, which is when the heart’s pumping power is weaker than normal. Despite intensive medical care, almost 80 percent of people die within eight years of diagnosis, making it the world’s leading cause of death. Heart failure can be the result of coronary artery disease, heart attack and other conditions such as high blood pressure and valve disease.
Cell therapies for cardiac repair have generated considerable interest in recent years. While earlier studies using autologous bone marrow transplantation (stem cells collected from the patient’s own bone marrow) helped improve cardiac function after myocardial infarction (MI); more recent studies showed no benefit in the early stages after MI. This has led researchers to question whether mesenchymal stem cells from sources other than bone marrow, such as cord blood and placenta tissue, might yield better results.
“In this animal model of progressive heart injury, stem cells isolated from placenta showed promise as an off-the-shelf therapy for cardiac repair, warranting the need for testing in additional models, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

Newly discovered protein may increase the regenerative potential of Stem Cells

Scientists at the University of California in Los Angeles have identified a protein that regulates the ability of stem cells to renew themselves. The results may lead to better methods to grow stem cells, such as those derived from cord blood, and increase both the number and potential of stem cells in life-saving cell therapy treatments.
Unlike most other cells in the body, stem cells have the exceptional ability to renew and grow into more stem cells, and change into the mature functional cells of the body such as blood, muscle, and nerve cells. An ongoing challenge of cell therapy is the development of methods to grow valuable stem cells once they are removed from the body so that more cells are available for use, resulting in improved transplantation outcomes. A further challenge is the growth of stem cells after they have been transplanted into a recipient.
The current study shows that a newly identified protein, known as protein tyrosine phosphatase sigma (PTPσ), regulates the ability of stem cells to proliferate upon transplantation. Researchers Dr. John Chute and colleagues showed that stem cells from PTPσ-deficient mice had an improved transplantation outcome compared to normal stem cells. The scientists then selected human umbilical cord stem cells that express low levels of PTPσ, and discovered that these cells also had a better regeneration potential following transplantation. Future therapies may make use of this discovery to target the PTPσ protein in order to increase the therapeutic potential of cord blood stem cells.

Trial in Australia: Mesenchymal Stromal Cells for Acute Graft Versus Host Disease

This trial is investigating the possible beneficial effect of mesenchymal stem cell transplantation in acute graft versus host reaction following bone marrow transplantation or donor lymphocyte cell therapy. The study is being undertaken at the Royal Perth Hospital in Western Australia where patients with newly diagnosed, untreated grade 2-4, acute graft versus host disease will be randomly assigned to control and treatment arms of the study. Those in the control group will receive standard treatment (methylprednisolone intravenously). Those in the treatment group will receive standard treatment plus donated mesenchymal stem cells intravenously. Participants will be assessed for complications and any changes in clinical condition. The trial plans to enrol 66 patients by December 2015 and to be completed by December 2016.z Link to Clinical Registry

Allogeneic Stem Cell Transplants associated with risk for Diabetes and Hypertension

An allogeneic stem cell transplant is a common treatment for some types of cancers, particularly leukemia and lymphoma. The process of an allogeneic stem cell transplant typically includes the use of high-dose chemotherapy or total body irradiation. These treatment approaches tend to kill more cancer cells than conventional doses; however, they are also associated with more severe side effects.
One common and potentially life-threatening side effect of these high-dose therapies is severely low levels of blood cells. Fortunately, hematopoeitic stem cells (immature blood cells) from a donor can be infused into the patient following high-dose therapy. These stem cells will then mature into functioning blood cells and can reduce serious complications associated with low levels of blood cells. Another approach to stem cell transplant is an autologous stem cell transplant, in which the patient’s own stem cells are collected prior therapy and reinfused following therapy.
Because patients who undergo allogeneic stem cell transplants can achieve long-term survival, researchers have become more focused on identifying long-term side effects that may be associated with this treatment approach.
Researchers affiliated with the Bone Marrow Transplant Survivor Study recently conducted a study involving 1,089 survivors who had undergone allogeneic or autologous stem cell transplants. The average follow-up was 8.6 years. The main findings of this study included the following:

• A 3.65-fold increase in diabetes was observed in allogeneic transplant recipients.
• A 2.06-fold increase in hypertension (high blood pressure) was observed in allogeneic transplant recipients.
• Total body irradiation was associated with an increased risk of diabetes.
• Autologous stem cell transplants were not associated with an increased risk of diabetes or hypertension.

These authors suggested that allogeneic transplant recipients may be at increased risk of future side effects including diabetes and hypertension. Patients who are recipients of allogeneic stem cell transplants may benefit from screening and monitoring for diabetes or hypertension so that these conditions may be identified and treated early.

A few antiviral cells might prevent Bone Marrow Transplant infections

Bone marrow transplantation is a life-saving therapy for patients with blood cancers like leukemia or lymphoma. However, the depletion of the patient’s immune system prior to transplantation can put patients at risk of an infection by a virus called cytomegalovirus (CMV) that can be life-threatening in these immune-compromised individuals.
Now, researchers have found that a very small subset of antiviral immune cells transplanted along with a donor’s blood stem cells could be enough to fight and even prevent the disease caused by CMV.
Between 50% and 80% of adults in the United States are infected with CMV, although the virus is kept under control by a healthy immune system. In patients with weakened immune systems, however, CMV can become reactivated and cause life-threatening pneumonia, among other symptoms. Current treatment includes antiviral medications, but these are not always well tolerated by patients and they also harm the very cells that bone marrow transplantation aims to replenish.
“We know that re-establishment of antiviral immunity in these patients is critical to fully control cytomegalovirus in bone marrow transplant recipients,” said the study’s senior author, Christopher Snyder, Ph.D., an assistant professor of microbiology and immunology at Thomas Jefferson University. “Our study suggests that, in addition to infusing stem cells that restore the bone marrow, lifelong anti-CMV immunity may be rapidly restored by also infusing a subset of antiviral immune cells that have stem cell-like properties.”
Currently, investigators around the world are experimenting with restoring the immune cells responsible for keeping CMV in check by transplanting those specific antiviral cells from healthy donors, a type of immunotherapy. “The problem,” said Dr. Snyder, “is that current methods for selecting antiviral immune cells may inadvertently limit the ability of those cells to restore lifelong immunity.”
Researchers have focused on developing anti-CMV immunotherapy around the “fighter” cells, called CD8 T effector cells, that attack and kill virally infected host cells. These cells are selected and expanded in the lab to increase their numbers, but this process may limit their lifespan and ability to divide.
The infused memory cells became major contributors to the recipient antiviral immune response, persisting for at least 3 months of time and producing the “fighter” cells at a steady stream.
“This may be a valuable approach to keep the disease from emerging in people.”
“Our data argue for developing new clinical trials focused specifically on using these T memory cells, in order to determine if it would indeed be better than current therapeutic options,” said Dr. Snyder.

Stem Cell Transplants may work better than existing drug for severe multiple sclerosis

Stem cell transplants may be more effective than the drug mitoxantrone for people with severe cases of multiple sclerosis (MS), according to a new study published in the February 11, 2015, online issue of Neurology, the medical journal of the American Academy of Neurology.

The study involved 21 people whose disability due to MS had increased during the previous year even though they were taking conventional medications (also known as first-line treatments). The participants, who were an average age of 36, were at an average disability level where a cane or crutch was needed to walk.

In MS, the body's immune system attacks its own central nervous system. In this phase II study, all of the participants received medications to suppress immune system activity. Then 12 of the participants received the MS drug mitoxantrone, which reduces immune system activity. For the other nine participants, stem cells were harvested from their bone marrow. After the immune system was suppressed, the stem cells were reintroduced through a vein. Over time, the cells migrate to the bone marrow and produce new cells that become immune cells. The participants were followed for up to four years.
"This process appears to reset the immune system," said study author Giovanni Mancardi, MD, of the University of Genova in Italy. "With these results, we can speculate that stem cell treatment may profoundly affect the course of the disease."
"More research is needed with larger numbers of patients who are randomized to receive either the stem cell transplant or an approved therapy, but it's very exciting to see that this treatment may be so superior to a current treatment for people with severe MS that is not responding well to standard treatments," Mancardi said.

Breakthrough Therapy Designation for Genentech’s Investigational Cancer Immunotherapy in Non-Small Cell Lung Cancer

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced that it has received a second Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its investigational cancer immunotherapy MPDL3280A (anti-PDL1).
"Lung cancer is the leading cause of cancer death globally, and we are pleased the FDA has granted breakthrough designation for MPDL3280A in non-small cell lung cancer," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "We are committed to personalized healthcare, developing medicines like MPDL3280A with companion tests that may help us identify those who may be appropriate candidates for our medicines."
Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious diseases and to help ensure patients have access to them through FDA approval as soon as possible. 

New Cancer Drug to bring in $1 Billion a year by 2020

Eisai Co. said it expects its new drug for thyroid and other cancers to bring in more than $1 billion a year by 2020.
Ivan Cheung, an Eisai executive leading the rollout of the drug called lenvatinib, said in an interview Thursday that approval from the U.S. Food and Drug Administration for thyroid cancer was “imminent.”
An Eisai-funded study published Thursday in the New England Journal of Medicine said 65% of thyroid-cancer patients taking Lenvatinib showed a response to the drug, compared with 1.5% on placebo. The study involved thyroid-cancer patients whose disease didn’t respond to initial treatments such as radioactive iodine.
Some patients experienced serious side effects in the study. Six of the 261 patients on the drug died for reasons that study investigators judged were treatment-related.
Lenvatinib, a pill taken once daily, works by inhibiting the creation of new blood vessels. Eisai says the drug, created in its laboratories in 1999, inhibits more substances that foster blood vessels, making it harder for cancer cells to resist.
“You block the front door and block the back door,” said Mr. Cheung.
He declined to say how much the drug would cost, but such cancer drugs generally cost tens of thousands of dollars a year.

Prostate Cancer patients who smoke fare worse

Smoking doubles the chances that a prostate cancer patient will see his disease spread and that he will eventually die from his illness, a new study finds.
"Basically we found that people who smoke had a higher risk of their tumor coming back, of it spreading and, ultimately, even dying of prostate cancer," said study co-author Dr. Michael Zelefsky. He is vice chair of clinical research in the department of radiation oncology at Memorial Sloan Kettering Cancer Center in New York City.
"Interestingly, this applied only to current smokers who were smoking around the time they received external beam therapy," Zelefsky added, referring to the standard form of radiation treatment for prostate cancer. "Former smokers did not have the increased risk for disease spread and recurrence that current smokers did," he said.
"However, we also looked at how smoking affected treatment side effects, from the radiation treatment, which can include rectal bleeding and/or frequent and urgent urination", he noted. "We saw that both patients who smoked and former smokers seemed to have a higher risk of urinary-related side effects after therapy."

MRI improves Prostate Cancer biopsy accuracy

Prostate biopsies that combine MRI technology with ultrasound appear to give men better information regarding the seriousness of their cancer, a new study suggests.
The new technology which uses MRI scans to help doctors biopsy very specific portions of the prostate diagnosed 30 percent more high-risk cancers than standard prostate biopsies in men suspected of prostate cancer, researchers reported.
These MRI-targeted biopsies also were better at weeding out low-risk prostate cancers that would not lead to a man's death, diagnosing 17 percent fewer low-grade tumors than standard biopsy, said senior author Dr. Peter Pinto. He is head of the prostate cancer section at the U.S. National Cancer Institute's Center for Cancer Research in Bethesda, Md.
These results indicate that MRI-targeted biopsy is "a better way of biopsy that finds the aggressive tumors that need to be treated but also not finding those small microscopic low-grade tumors that are not clinically important but lead to over treatment," Pinto said.