After decades of unfulfilled promises and setbacks, the field of gene therapy broke through with three FDA-approved products last year, ushering in what is likely to be a rapid escalation of new treatments for some of the rarest and most debilitating diseases.
“Part of the idea of the Human Genome project was that once we had the identity of all the genes, it would be important and straight forward for us to development more therapeutic options for people with serious inherited diseases, said Katherine High, M.D., president and director of research and development for Philadelphia-based Spark Therapeutics. “But it turned out this took a little longer to do than the Human Genome project.” Continue reading
One of the largest analyses of stroke factors ever conducted is providing scientists with new clues to identify stroke mechanisms and potential treatments. Researchers have identified 22 new genetic risk factors, tripling the number of gene regions known to affect stroke risk.
By mining an enormous trove of data, a team of international researchers obtained critical new insights into the specific genes, molecular pathways, and cell and tissue types through which the new genetic risk factors cause a stroke. Continue reading
In December 2016, Charles Piller (@cpiller), the west coast editor for Stat, reported that a genetic test to identify patients who could be prone to addiction lacked a firm scientific basis.
With an eye-opening headline, “Called ‘hogwash,’ a gene test for addiction risk exploits opioid fears,” the article raised important questions about the Proove Opioid Risk test from Proove Biosciences in Irvine, Calif. Continue reading
The placebo effect presents quite the conundrum to researchers attempting to discern whether a particular intervention truly offers clinical benefit for patients. Those most susceptible to the effect can feel so much better after a placebo that their “improvement” exceeds that of others taking a chemically active drug.
This problem may slow down the pipeline for new effective drugs, Erik Vance suggests in his recent Washington Post article, “People susceptible to the placebo effect may be keeping us from getting new drugs.” Continue reading
The nation’s clinical laboratories have pushed back hard against a proposal by U.S. Food and Drug Administration in October 2014 that would regulate laboratory developed tests. More than a year later, the disagreement became more heated when the Association of Molecular Pathology (AMP) accused the FDA of fabricating examples in a November report on LDTs.
In the report, “The Public Health Evidence for FDA Oversight of Laboratory Developed Tests: 20 Case Studies,” the agency cited 20 examples of problematic LDTs, saying, “…these products may have caused or have caused actual harm to patients.” We covered this report in a blog post earlier this month.
But that’s just the beginning of the story, as Allison Proffitt, editorial director of BioITWorld, found. Continue reading