Stem cell wish list for 2017

stem-cell-wish-list

Last week I posted my list of 2017 predictions for the stem cell field. Today a couple of days into 2017 I’m more focused on hope than realism. What would I wish for in the stem cell and regenerative medicine arena in the coming year?stem-cell-wish-list

More stem cell clinical trial data posted and published. There are few things as exciting as stem cell and regenerative medicine clinical trials across the full spectrum of stem cell types including adult, embryonic, and IPSC. But we need to have actual trial data be peer reviewed and published or at least posted. Clinical trial updates only by press release are not helpful to patients or the field.

Clinicaltrials.gov adapts. This vital resource of trial listings adjusts to new realities. It either filters its listings to screen out for-profit entries that aren’t real trials or it provides more practically useful information such as at a minimum clear indications of whether a listing has an IND (this doesn’t need to violate any confidentiality rules) and whether the listing requires payment as an inclusion criteria. See my interview with the leader of Clinicaltrials.gov.

FDA speaks clearly. Whatever the FDA does or does not do in terms of actual stem cell & regenerative medicine-related actions in 2017, it is clear about it. This year I hope the FDA provides concrete, consistent explanations in the public domain that don’t require an FDA-ese jargon dictionary to try to understand.

FDA and its CBER center are consistent with good & bad citizens of the stem cell arena. The FDA has a tough job overall and its CBER branch focusing on biologics including stem cells has its own specific challenging task set. However, for years CBER has held different players in the stem cell arena to different rules and expectations. Paradoxically, essentially the better a citizen you are, the more the FDA expects from you. On the flip side, if you are for instance a stem cell clinic with no intention of following the rules (no BLA, no IND, no pre-IND, no expertise in stem cells, no data, etc.) CBER has historically generally left you alone.

What the heck? That’s got to change.

Of course the FDA and CBER are not police, but by analogy imagine if police devoted fewer resources and tended to take less action proportionately as people behaved worse and worse, and were conversely strictest with the most law-abiding citizens? It makes no sense.

FDA accelerated review or fast track, etc. of stem cell product. We can all hope that there is a strong enough stem cell/regenerative medicine product and that the FDA considers it expeditiously enough (e.g. under Cures Act provisions) that we can see in 2017 a stem cell product get into the fast track somehow.

Less hype on all sides. If one can get away with hype and perhaps most likely only pay a price like a correction or retraction at most just years down the road on the academic side or no price at all in the direct-to-consumer arena of clinics, some people are going to continue hyping up a storm. However, hype is bad all around and is selfish. I’d wish to see less of it in the stem cell world in 2017. Scientists and the media can talk about new, outstanding findings (see below in next section) without invoking miracles or overly aspirational language.

Loads of exciting papers. You can have your great published science without hype. For this year I expect we’ll see dozens of very cool stem cell papers that are at the same time not hyped. As a scientist, for me reading new, interesting data in papers is a real joy.

13 Comments


  1. “Observational” trials are something to look out for on clinicaltrials.gov. These are not treatments themselves, but rather just plans to follow patients that receive whatever treatment that the clinic is doing. This is pretty common for clinics doing unregulated stem cell transplants. To me, it looks like a pure marketing strategy. The clinics are trying to get credit while still charging people for treatments that have no basis in scientific evidence and may be harmful.

    I’m making no accusations of wrongdoing, but do take a look at the listing for Stemgenex for Parkinson’s disease: “https://clinicaltrials.gov/ct2/show/NCT02184546?term=stemgenex&rank=1


    • Hi Jeanne,
      Agreed on observational trials.
      But without changes at Clinicaltrials.gov, patients don’t easily know the difference.
      For instance, sometimes patients email me referring to a specific listing as a “clinical trial” and I recognize the entity listed there as a stem cell clinic that as far as I know has no IND, etc.
      A small change at Clinicaltrials.gov of including a required notation in all the listings that says (IND: YES or NO) would be useful. Another change could be requiring a “Yes” or “No” answer to whether patient payment is an inclusion criteria. That could be helpful.
      I’m not holding my breath.
      It may be a long shot that the NIH folks doing the website and the FDA, for instance, will work together any time soon to make Clinicaltrials.gov a more useful tool in this regard, but it can be a goal.
      Paul


      • Just look at “Health Authority”. If it doesn’t say “United States: Food and Drug Administration” then there is no IND, right? Take Stemgenex for example. Their Parkinson trial says “United States: Institutional Review Board”. Also, what about trials in other countries? These would say “IND:NO” but still could be government regulated. For example, in the EU, a trial could be under the EMA and completely “legitimate” but they would still have to say “IND:NO”.


        • I know that you aren’t confused, Bill, but the I__ designations can confuse people.

          IRB is Institutional Review Board- that’s a local group of people who judge protocols involving human subjects. It’s important to keep 2 things in mind: the IRBs are local, so if a protocol is approved by my local IRB, it doesn’t mean that your IRB would approve it. Second, there are professional IRBs…so you can get what you pay for.

          IND is Investigational New Drug- that’s an FDA designation. FDA approves INDs, which allow clinical trials to move forward.

          So saying that a procedure has IRB approval does NOT mean that the FDA even knows about it.


        • Well, it sounds like they have a IND, but an IND is not mentioned in the Press Release. It says this is an FDA-approved trial and that they are working with the FDA. Hopeful that is not simply meaning the trial is listed on Clinicaltrials.gov and instead means that they have an IND.


  2. “…imagine if police devoted fewer resources and tended to take less action proportionately as people behaved worse and worse, and were conversely strictest with the most law-abiding citizens?” That’s called “gun control in Chicago”


  3. Those are all good suggestions!

    Are you going to post anything on the Bayer AG Bluerock venture for remuscularization with iPSC after MI? In conjunction with Memorial Sloan Kettering, Dr. Lorenz Studer, McEwen Centre, Dr. Gordon Keller and CCRM in Toronto for GMP manufacturing.
    If you were to forecast this one, how long do you think it will take to attain approval?

    http://www.prnewswire.com/news-releases/bayer-and-versant-ventures-join-forces-to-launch-stem-cell-therapy-company-bluerock-therapeutics-with-usd-225-million-series-a-financing-300376367.html


  4. I asked this in a previous post but no one replied. I spoke with the head of one of the major stem cell companies on the east coast and they said that in order for the FDA regulations to become law that congress would have to vote on it. Is that true? If it is, isn’t all your pleading with the FDA for nothing? Shouldn’t you be addressing congress instead to take the regulations and make them law?


    • @Dan,
      The head of that stem cell company is incorrect.
      There is a difference between regulations/guidances and laws when it comes to the FDA.
      There are already laws passed giving the FDA clear authority to regulate biological products and based on those laws the FDA makes regulations/guidances that have the weight of law behind them so there is no need for a new law.
      But the current situation isn’t working.
      What the FDA needs to do a better job with is issuing finalized guidances that are timely, crystal clear and that serve as unambiguous regulations for this industry, and then it needs to enforce those consistently with actions or the words become operationally of little meaning in the real world.
      For a variety of reasons (including potentially budgetary and politics) the last few years the CBER branch of the FDA, which regulates stem cells, hasn’t effectively managed the stem cell clinic industry and there are serious consequences to that. I hope that in 2017 things will turn around, but the wheels turn slowly and there are obstacles.

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