It’s a big challenge to get a cell therapy approved by the FDA and if you look at my list of FDA-approved stem cell therapies, it’s not as long as we might hope.
FDA OK on cell therapy from Gamida Cell
For this reason, it was excellent news to see that the FDA approved a new cell therapy. Here’s some coverage from MedPage Today: FDA OKs Cell Therapy to Lower Infection Risk After Stem Cell Transplant.
That article highlights the key positive clinical outcome, “Omidubicel reduced infections in blood cancer patients from 60% to 39% at 100 days posttransplant.” I wrote about Gamida Cell and its drug Omidubicel a while back prior to approval. In that piece, I discuss the mechanism of action of these umbilical cord cells via enhanced homing and engraftment. Gameda Cell stock is up more than 100% since the news broke.
Here’s the Phase III trial paper in Blood on Omidubical.
Chalk one up on the regenerative medicine win column for patients.
Recommended reads
Judicial interference with mifepristone, Science. In this opinion piece, former Commissioner Margaret Hamburger and former Principal Deputy Commissioner of the FDA Joshua Sharfstein make some good points about judicial interference in FDA matters. Of course, courts do sometimes rule on FDA decisions, but this case seems so politically motivated. This part of the piece caught my eye as a stem cell biologist, “There is already political pressure against vaccines, antidepressants and other psychotropic medication, and certain cell-derived therapies. If judges begin to dictate the terms of medication access, then others will seek to use ideology and influence to advance their agendas.” I’m guessing that when referencing cells they are referring to human embryonic stem cell-based therapy research.
Some of you may not know that years ago a judge made human embryonic stem cell research funding by the NIH unlawful for a time. It could happen again, although public opinion seems even stronger now in support of such research.
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice, Nat Bio Engin.
Duke Has Quietly Discontinued a Costly, Unproven Autism Treatment, Vice. I talked to author Anna Merlan for her story. Here’s my take on this situation: Why did Duke autism team halt its troubling pay-for-play program?