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Welcome back from the summer holidays; now off to the salt mines!
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FDA approves landmark CAR-T drug
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In what is a historic day more than a decade in the making, the FDA last week has approved the first ever gene therapy – CAR-T – for a rare form of leukemia. First conceived by Carl June and colleagues at UPenn and then developed by Novartis, Kymriah is made of re-engineered T cells from a patient that are injected back in to attack the cancer, and has a price tag to match its amazing technology: $475,000. In a clinical trial, 83% of participants were cancer-free after 3 months, which led FDA panelist Dr. Tim Cripe to state: “I think this is most exciting thing I’ve seen in my lifetime,”. Novartis said it will work on a pay for performance deal with CMS such that that the government only pay for the therapy if the patient responds within a month of treatment. Others pharma’s are developing their own CAR-T therapies as well, including the recently acquired Kite Pharma (by Gilead Sciences for $11.9B). Kite’s CAR-T, called Axi-Cel, is being reviewed by the FDA and will be decided by November of this year. Stay tuned for personalized cancer treatments made especially for/from you.
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Research use antibodies play an important role in research – as we all know – and you may have heard that you should validate them upon receipt. You also may have heard about our antibody validation initiative that includes both manufacturers and academics to define validation guidelines and a scoring system. But sometimes we don’t always see why validating upon receipt matters. Our friends at CiteAB recently blogged about a study in Nature Communications highlighting how not validating antibodies has delayed breast cancer research over the last 20 years. The study authors note that the discovery of estrogen receptor β (ERβ), was a potential landmark discovery 20 years ago that was touted to be a biomarker to breast cancer, and yet, there is still no agreement as to where the protein is actually expressed. Performing rigorous validation on 13 anti- ERβ antibodies, the team found that only one – a rarely-used monoclonal – specifically targets ERβ in immunohistochemistry assays, and is expressed in testis, ovary, and other cells. With numerous publications and 20 years, we don’t know how many millions of dollars could have been saved with rigorous validation – but this could serve as a call to action to coalesce the research community to adopt rigorous antibody validation standards.
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In what is good news to this writer as a life-long asthma sufferer, it looks like the genes linked to asthma, high cholesterol and Alzheimer’s are being “weeded out” by natural selection. The study, published in PLOS Biology, suggests that negative traits for adapting to our modern lifestyle are being slowly eliminated. Also of interest, a one-year delay in child-bearing lowered infant death rate by up to 6%.
Transparency of clinical trial data is an issue near and dear to British celebrity physician Ben Goldacre, and he published a study in BMJ identifying the major pharma’s policies on publishing trial data. In this audit of pharma policies, he found a lot of variability despite the many laws in the UK requiring data sharing. While 23 of the top 25 pharma’s have committed to sharing, policies were incomplete and most commonly lacked timeline requirements. More information on this can be found here.
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And of course, we cannot forget the solar eclipse. For those who drove to see the total eclipse portion, the pictures are spectacular. Hopefully all of you wore glasses to watch, and Mashable pulled together a list of 10 glasses that “will protect absolutely no one during the solar eclipse” and is totally worth seeing. Have a great weekend!
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