The Niche top posts of 2016

stem cell fireworksWhat were the top posts here on The Niche for the past year? I’ve listed some of them below along with some posts from 2015 that remain highly read.

Some top 2016 posts

2015 and older posts that remain highly read every day

Perspectives on Cures Act stem cell provisions possible benefits & risks

The 21st Century Cures Act has some important provisions that would impact the stem cell and regenerative medicine field.  What is driving these efforts is a widespread sense that the status quo of regenerative medicine oversight isn’t working very well.

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I’m talking with key stakeholders and still trying to sort out my thoughts on the stem cell provisions in Cures. What would it mean? Cures’ language on stem cells is nuanced and its provisions also are not so black and white as REGROW was in my view.

You can see my interview with ARM’s Executive Director Michael Werner on Cures here. ARM supports Cures. ISSCR issued a statement this morning on Cures, which is upbeat but stresses the importance of data. The statement is not a clear thumbs up or down. Earlier this year, both ARM and ISSCR opposed REGROW.

Overall, there are both potential risks and benefits to Cures’ stem cell provisions, which I discuss below. Note that this post is focused primarily on the stem cell-related provisions of Cures. There is ambiguity in terms of how Cures will affect NIH overall or have broader outcomes for the life sciences.

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Trump Has FDA in Crosshairs: Impact on Stem Cell Oversight?

fda-trump-healthcareWill Trump trump the FDA?

Our President-elect recently released his health care policy agenda and it looks like the FDA is a target in terms of reducing oversight. Over at the Washington Post, Carolyn Johnson has a nice article on this development.

One point in the agenda in particular has caught people’s attention as potentially the key indicator of what is to come for regulatory oversight of biomedical products:

  • “Reform the Food and Drug Administration, to put greater focus on the need of patients for new and innovative medical products”

This one sentence says a great deal in terms of signaling that Trump wants dramatically less oversight or at least that’s how “reform” is being interpreted.

For those of us in the stem cell field this sentence from Trump sounds very reminiscent of language from proponents of reduced regulation and even stem cell clinics, who say they are just in it for the patients but keep keeping racking up profits.

This signal from Trump does not bode well for stem cell and regenerative medicine oversight in the coming years, but we’ll have to stay tuned to see how this develops.

REGROW Act to do reappearing act?

One of the biggest stem cell news stories of 2016 has been the REGROW Act, a bill intended to accelerate investigational stem cell and regenerative medicine therapies to patients by lessening FDA oversight, but in the last couple months REGROW has mostly disappeared from the news.

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Will REGROW do a reappearing act like some kind of magician?

REGROW is in fact still out there so it is not gone by any means. Republican senators have been discussing including REGROW in a large medical spending bill that could include funding for NIH.

It could come up after the presidential election during the lame duck session. Would it pass? If so, would Obama sign it? It seems like the odds are still very long for REGROW becoming law, but it’s not impossible.

Another possibility is that REGROW could come up in its current or a new form in the more distant future. There is definitely political will in some quarters in the long haul for reduced FDA oversight.

Readers of this blog will know that I have been opposed to REGROW in its various forms as it has evolved this year because I believe it would pose unacceptable risks to patients and to the stem cell field by promoting transplantation of stem cell-based products into patients based on less data than is prudent.

There are some very accomplished people who disagree with me and with the others (e.g. ARM and ISSCR) who oppose REGROW, so the debate has and will continue.

The main reason that I have taken the risk of publicly opposing REGROW and other talk of strongly weakening the FDA’s oversight in this area is the dangers this would pose to patients and the field. Some have attacked me for my outspoken opposition to REGROW and to extreme criticism of the FDA.

REGROW’s earliest incarnation proposed a highly risky conditional approval system that would have taken a shortcut around Phase III clinical trials, spurring me to speak out. There was even talk amongst REGROW supporters of charging patients for receiving still unproven stem cell therapies, going against the longstanding and I think very wise tradition of not charging patients for unproven therapies, with only the rarest exceptions that the FDA can grant in certain cases.

REGROW has evolved to be substantially less extreme, but it would still be too risky. The current FDA system is not ideal and some changes could be helpful, but a major challenge is to find a way to identify the “sweet spot” of regulation that is efficient, but rigorous and doesn’t put so many risks on patients.

Unproven stem cell and regenerative medicine therapies are not somehow magically special in the sense of having a dramatically better chance of being ultimately proven to be both safe and effective as compared to other kinds of investigational drugs (e.g. drugs taken as a pill). As a result of this reality, history tells us that the vast majority of cellular therapies will not ultimately be proven both safe and effective, which means that while they are still in an unproven state, rushing cell therapies to patients will only do these people more harm and in turn hurt the field.

8 key arguments, take-homes on day 1 of FDA stem cell meeting

Today was the first part of the FDA’s 2-day meeting on stem cell draft guidances. It was a great meeting I thought.

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Screenshot from video of FDA stem cell meeting of CIRM President Randy Mills speaking.

A few consistent themes and notable things emerged as raised by participants.

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