In a development that could lead to a new
generation of drugs to precisely treat a range of diseases, scientists
from the Florida campus of The Scripps Research Institute (TSRI) have
for the first time designed a drug candidate that decreases the growth
of tumor cells in animal models in one of the hardest to treat cancers, triple negative breast cancer.
"This is the first example of taking a genetic sequence and designing
a drug candidate that works effectively in an animal model against
triple negative breast cancer," said TSRI Professor Matthew Disney. "The
study represents a clear breakthrough in precision medicine, as this
molecule only kills the cancer cells that express the cancer-causing
gene, not healthy cells. These studies may transform the way the lead
drugs are identified, by using the genetic makeup of a disease." The lab's compound, known as Targaprimir-96, triggers breast
cancer cells to kill themselves via programmed cell death by precisely
targeting a specific RNA that ignites the cancer.
The drug candidate was tested in animal models over a 21-day course of
treatment. Results showed decreased production of microRNA-96 and
increased programmed cell death, significantly reducing tumor growth.
Since targaprimir-96 was highly selective in its targeting, healthy
cells were unaffected.
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