Today an opinion piece that I wrote about CIRM was published in the San Francisco Chronicle. The unusual element here is that the article is critical of CIRM. More specifically I raised concerns about a recent political trend at CIRM under its new President Randy Mills lobbying for dramatically weaker stem cell regulatory oversight. The Chronicle piece was first posted online on Thursday on the paper’s website. I encourage you to read it when you get a chance.
For a decade I have been one of the most consistent voices advocating for CIRM, its future, and all the great things that it does so it wasn’t an easy decision to publicly criticize the agency at this point. I’m still a big backer of CIRM, which made this even harder.
For me the tipping point to action was Mills’ recent opinion piece on Fox News with former Republican Senator Bill Frist that I believe was anti-FDA in tone and utilized hype (e.g. words like “miracles”, “gift”, and “beautiful” medicine) to promote a deregulatory agenda. This extreme plan includes conditional approval of unproven stem cell therapies and elimination of the requirement for Phase III clinical trials in certain cases, which would be very risky to patients and the stem cell field.
Mills also recently gave a speech at a conservative group (the self-styled “Bipartisan” Policy Center or BPC at which Frist is a leader) that counterproductively characterized the FDA as the one big problem for the field. Note that BPC also wants to charge patients to be in clinical trials to get experimental therapies. Historically if anything changed hands in such situations it was patients getting something in return for being so brave to be in clinical trials.
As I pointed out in my opinion piece in the Chronicle, the stem cell status quo is not getting the job done, but this is only in part due to the FDA and the changes that BPC and CIRM are advocating for are too radical. These proposed changes also fit with the REGROW Act, a bill that would codify many of them. REGROW would force the FDA to conditionally approve stem cell therapies that frankly are just not ready for primetime, putting patients at great risk. Note that the big changes being lobbied for in FDA regulations would only apply to adult stem cells. I favor more careful changes at the FDA such as using Breakthrough designation for stem cells, something the FDA has not yet done. They should.
The fact that REGROW is floating out there also raises the stakes on CIRM’s lobbying as well. While CIRM officially takes no position on REGROW, their leader’s words and even those on the CIRM blog when talking about the huge changes they want at the FDA fit nicely with REGROW priorities.
REGROW has little support in the stem cell community overall. Leading stem cell organizations ISSCR and ARM as well as 10 top patient advocacy groups also oppose REGROW. Super stem cell advocate Don C. Reed also opposes the bill.
It would be wonderful if we could start developing stem cell therapies, do early phase clinical trials, and then just give the experimental treatments right away to patients with a compelling expectation of safety and efficacy, but that is more a hopeful dream than a data-based reality. History would argue that rushing stem cell therapies to patients would be incredibly risky to them and likely do much harm. It’s just not that simple to say “step on the gas” without more data than early studies provide and without considering risks. Not only would many patients almost certainly be harmed, but also there would be big risks to the stem cell field overall too from the likely bad press of patients being harmed. The fact that BPC wants to charge patients for this would probably just add a financial insult to injury for some patients.
If you read Mills’ Fox News article and listen to his speech, there is no mention of risks of what he is proposing. He, BPC, and those favoring REGROW never mention the very real and common scientific and medical challenges to stem cell therapies. Without context, from their words you might assume that there are loads of stem cell therapies waiting in the wings that would absolutely be safe and effective for patients if only the big bad FDA system would get out of the way, but that’s just not reality. We should resist the temptation to turn the current FDA regulatory system into a political punching bag.
Another level of context here is that the dubious stem cell clinic industry is pushing against the FDA too. While CIRM has always opposed the clinics and I’m 100% sure it still does, I’m concerned that CIRM’s lobbying against the FDA could by accident help the clinics.
Prior to the Chronicle piece, I talked with Mills on the phone about the key issues that were on my mind. I greatly appreciate him taking the time to talk and I found it valuable, but it just reinforced my impression that the agency is committed to lobbying for weaker stem cell oversight. They truly believe this is the right thing to do. When I asked Mills about the aspirational language such as “miracles” in his Fox News piece, I got the sense that he feels passionately about the potential of adult stem cell therapies. There are a lot of strong feelings about stem cells, but plans should be built on a foundation of data.
Together all of this went into my decision to publicly raise concerns about the now more politically active CIRM in its new incarnation “CIRM 2.0” that is lobbying so hard against the FDA phased clinical trial system. Most of the stem cell scientists that I have talked to (admittedly with a few notable exceptions that include some long time stem cell advocates and scholars) are opposed to CIRM’s lobbying on this front, but are uncomfortable speaking out.
Today the question of what constitutes appropriate regulatory oversight is the biggest debate in the stem cell arena. It needs more discussion and debate out in the open.
Note that David Jensen’s California Stem Cell Report on Friday published an anonymous piece that was highly critical of the new CIRM for being too corporate. I didn’t write that piece as I always sign my name to what I write. Importantly, also I was unaware of that piece until after mine was published the day before.