What happens next with FDA & stem cells: lower your expectations?

FDA stem cell meetingIn the total the FDA has held 3 days of public meetings on stem cells in the last few weeks ranging from science to policy and hearing from many parties including patients and even stem cell clinics so the big question now is, “what happens next?”

The FDA has 4 draft guidances pertaining to investigational stem cell clinical procedures and these were the subject of the second meeting that last a couple of days. Soon the FDA’s online commenting period related to these issues will close (you can still make comments here, but don’t expect the comment to appear quickly as I submitted mine 4-5 days ago and it’s still not up).

Once that is done, can we expect the FDA to finalize those guidances? And if it does, will the guidances change at all due to the meetings or online comments on the docket? My sense is that the FDA will take at a minimum months to consider the comments at the meeting and those submitted online so it’ll be 2017 most likely before they are even close to taking a next step at all.

I do not expect the guidances to change substantially. As a stem cell biologist, I didn’t hear anything at the meetings in terms of science or data that provided a compelling rationale to change the guidances. The FDA should base its decisions on the biomedical science.

There were a variety of ideas presented at the meeting for changes or new elements to the stem cell clinical arena including a “middle ground” that would get stem cell therapies to patients more quickly, but the trade-off there is that there would be far less data to support the potential safety and effectiveness of those stem cell products. On the whole this would simply put more patients at risk in my view.

Several speakers mentioned starting a registry for stem cell clinics, but I don’t see that as having a major positive impact as there are too many unknowns and complexities. Who would run the registry and pay for it? Who would oversee it (the FDA?) and what would happen if commercial entities were noncompliant with participating in the registry? What about the already existing clinicaltrials.gov?

A big meme at the FDA meeting was the idea of very widespread functional homology of fat stem cells throughout the body via paracrine signaling. Yes, the heterogeneous cells within “fat stem cell” products do make many growth factors that could act in a paracrine manner, but I don’t buy the idea that this makes them homologous to many diverse tissues in the body and I don’t expect the FDA will either.

The future

The FDA could never finalize the guidances, resulting in either the status quo continuing of thorough oversight of the good citizens of the field and little-to-no action on the stem cell clinics. Or even without finalizing the guidances, the FDA could step up its oversight of stem cell clinics, which would be helpful given that there are around 600 of them in the U.S. operating without FDA approval and the FDA has been issuing <=1 warning letter per year lately.

If the guidances remain (finalized or not) and are the basis for the FDA’s current thinking moving forward, then most adipose stem cell clinics should be subject to FDA action given that they are using more than minimally manipulated cells and hence drugs in an unapproved manner and they are almost always using the cells in a non-homologous manner.

Perhaps in the short run the most likely thing is that nothing will happen. As my state’s governor Jerry Brown once famously said to voters, “lower your expectations” and perhaps that can be applied to the FDA on stem cells. However, I’m not going to do it. I’m going to keep my expectations high on this.

Top 10 Google Stem Cell News Stories: Perspectives

What does Google think (if Google does indeed think) are top 10 stem cell news stories right now?

I took a screen shot below.

Here are some thoughts on those stories.

top-stem-cell-headlines

First, lung organoids are neat, but they have been grown before by several groups. Why is that the top story? I’d have to ask Google. Better PR? Still looks interesting and could have real impact for lung disease in the future.

The second story is on the transplantation of allogeneic IPS cells into monkeys without immunosuppression.This is an important finding with clinical impact from Dr. Takahashi’s group.

That third story seems odd to me. Seems like an over the top claim.

The fourth one with its “for the first time” I’m not so sure about and number five seems to be on the same story. I have doubts about that trial given the lack of detail and the potential for harm to patients. It sounds premature.

Then we have cancer stem cell stories at number six and another at number eight.

Number seven and ten both refer to the experience of one patient in the Asterias stem cells for spinal cord injury trial. Number ten’s headline is dubious from a scientific perspective with its “as a result” claiming the stem cells made the man better for sure. I really hope that’s true, but we don’t know yet although more recent data on more patients is encouraging. Controls are needed in the long run to iron things out.

Number nine is about stem cell clinics. It seems to be the only one mentioning the historic FDA stem cell meeting this week.

Nominations open for Stem Cell Person of the Year 2016 Award

Nominations are open starting today for the Stem Cell Person of the Year Award for 2016. Please email me your nominations: knoepflerATucdavisDOTedu.stem-cell-person-of-the-year-award

This is a unique award as it is given to an individual who has taken risks to help others within the stem cell field and they based their actions on outside-the-box thinking.

Another unusual aspect is that anyone is eligible for the prize whether you are a scientist, physician, patient, writer, student, etc. There are also no geographic restrictions.

The winner receives recognition as a positive leader in this arena and a $2,000 cash prize that I award myself out of pocket.

Nominations will close one month from today on October 15th.

The nominations I receive will then be subject to an Internet vote and the top 50% will be the finalists, from which I will choose the winner. While I alone choose the winner, I often get feedback from leaders around the globe in the stem cell and regenerative medicine field.

Previous winners include these stellar stem cell leaders:

Who will win the Stem Cell Person of the Year Award for 2016? Send me your nominations.

Early, but encouraging data from Asterias on stem cells for spinal cord injury

asteriasAs I posted recently, Asterias Biotherapeutics has had good early safety results so far in its historic stem cell trial for spinal cord injury and now the company presented some early, encouraging hints at efficacy.  However, a caveat here is that this is not an RCT (randomized controlled trial).

Ed Wirth, CMO of Asterias, presented the early data at the 55th Annual Scientific Meeting of the International Spinal Cord Society. You can take a look at the very interesting talk slides here. Asterias is a subsidiary of BioTime.

According to the press release (PR) from the company, while only 4 of 5 patients are 90 days or more after treatment, all patients have shown some improvement in motor function and a subset have a substantial improvement, meeting the target at this point. This trial is funded in part by CIRM so kudos to our state’s stem cell agency on this.

MIT Tech Review is on the story to for more background. I’ve also posted from Dr. Wirth’s PowerPoint the study design image below.asterias-sci-stem-cell-study-design

Bottom line. Overall, I think this development is encouraging. While still early in the clinical trial and drug testing process, the fact that there is a good safety profile and early indications of possible efficacy yield real hope. Still, it is important to be cautious in interpreting early data. We’ll all need to follow this trial and then look for future RCT results to clarify how promising this drug is overall in the long haul.

I’ve posted the key bullet points from the Asterias PR below the fold.

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Report on day 2 of FDA stem cell meeting: patients, researchers, & more

The FDA’s stem cell meeting wrapped up today on day two with a diverse group of individual speakers. A series of patient testimonials today in favor of clinics was one thing that stood out. You can read my take of day one and the account of Jeanne Loring who was at the meeting. I’ve noted that on average about 350 people were watching the webcast of the meeting both days.

fda-stem-cell-meeting-day-2

Screen shot from FDA stem cell meeting webcast

The patients gave powerful, often emotional testimonials today on their experiences at various stem cell clinics including Celltex and most prominently Stemgenex. I think there were 7 of its patients who spoke. I have no doubt of the patients’ sincerity in their belief that they’ve been helped.

Various stem cell clinic doctors also testified.

As with yesterday, a common statement was that there are “no side effects” of stem cell treatments, which is concerning as all medical therapies have some risk of side effects.

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