Meeting review of unique stem cell ethics symposium at UC Davis School of Medicine

On Thursday here at UC Davis School of Medicine we held our second annual Stem Cell Ethics Symposium. The symposium was organized by Drs. Mark Yarborough and Nanette Joyce as well as me.

In this post I report on key take homes from the meeting and summarize the specific talks.

As far as I know, this is one of the few events in the world where there is such a wide diversity of stakeholders present both as speakers and in the audience to specifically discuss stem cell ethics for a full day. The participants included numerous patients and patient advocates, bioethicists, stem cell researchers, physicians, attorneys, institutional compliance officers, and more.

One of the things that I most appreciated about this meeting is that diverse views were welcomed and we dealt head on with some of the most challenging ethical issues related to stem cells.

I also got to meet in person some people I had only previously known on-line including keynote speaker Tim Caulfied, patient advocate Ted Harada, and advocate and frequent commenter on this blog, Cathy Danielson, which was fantastic. My colleague Leigh Turner was also present. Old friend and Huntington’s Disease advocate, Judy Roberson, was there as well. You can see the picture above of Ted, Judy, and me.

After a great introduction by our new Dean of the School of Medicine, Dr. Julie Freischlag, Nanette Joyce started things off with a very powerful talk about her experiences with her ALS patients. Her patients are facing incredibly intense challenges as they deal with all that ALS brings. Both in her medical specialty and amongst her patients, she noted, there is growing interest in stem cell treatments. Dr. Joyce expressed her concern over the lack of evidence of safety and efficacy, and how elements of hype have entered into the arena of ALS and stem cells. ALS is a very heterogeneous disease with ups and downs as well in individual patients, but life expectancy on average is very short after diagnosis.

One of Dr. Joyce’s patients, Michael, then gave a talk about his experiences with ALS. I found it really striking. Literally every day he goes on-line and checks out resources to search for opportunities for ALS patients such as clinical trials. Michael indicated that as far as he knows he’s not eligible for any trials so far. This was a theme that several people mentioned during the day: just because there are trials out there does not mean that patients with ALS or other conditions can be part of them. Patients are seeking other opportunities.

Next up was Ted Harada, who spoke passionately about his experiences with ALS, the ups and the downs, what is really like to get a stem cell treatment and be in a clinical trial. His talked about his willingness to take risks. He said he’d literally rather die trying something than not try to do something about his ALS. Ted has had two rounds of treatment for ALS as part of the Neuralstem clinical trial and he reported that it has really helped him. One of Ted’s concerns is that he may not be eligible to get a future third treatment from Neuralstem for various technical rules of the clinical trial, which is one reason he is such a strong advocate for Right To Try laws. However, Ted said he feels that most likely few or no patients will be directly treated as a result of Right to Try, but rather that it will open up a dialogue and create other positive outcomes that lead to more patient opportunities.

From both Michael’s and Ted’s talks, i also learned that it can take a very long time (potentially a few years) to definitively diagnosis ALS. Patients facing ALS are likely to have a much higher level of risk tolerance than others might imagine. Understandably they have a strong drive to take action.

I spoke next and focused on the growing challenge that the FDA and the stem cell field face of chains of stem cell clinics that appear to be selling unapproved stem cell drugs without FDA licensing. I talked a bit about the growth in providers offering unapproved stem cell “treatments” at Cell Surgical Network as an example. More broadly, the clinics argue that what they do is beyond the scope of the FDA’s regulatory authority. I discussed new FDA guidances that bear on this issue and which would seem to directly contradict the views of many of the clinics, squarely placing them and their products in the FDA sphere from my perspective. The clinics of course disagree. I also emphasized how the FDA currently is asking for comments on these guidances for the next couple weeks.

Tim Caulfield spoke next, giving a wonderful talk. In the stem cell world, hype is not just limited to dubious stem cell clinics. Tim focused on hype in the stem cell field and in particular hype involving scientific publications. He even talked about hype about hype. In the current environment there are strong pressures for scientists to hype their work, including in particular in abstracts. What is the relationship between hype in science articles and in the media? He discussed how widespread pressure to commercialize biomedical research including stem cell efforts sometimes has negative consequences and can contribute to hype.

Alison Sorkin, Deputy General Counsel for University of Colorado Health, talked about Right To Try from her experiences in the trenches. With the passage of the Right To Try Law in Colorado, Sorkin was there for the intense repercussions. The next working day after the law was passed, patients starting contacting the University of Colorado seeking treatment specifically under the law. Sorkin talked about strict the Colorado law actually is, making it very limited in scope in reality. She also discussed problematic issues with the specifics of the law such as that patients would be responsible for paying for all of their own healthcare for 6 months after treatment under Right To Try as insurers would be exempt from having to provide ANY coverage. There seems to be a growing sense that Right To Try in Colorado may not actually lead to any patients getting non-FDA approved drugs. However, the FDA is paying close attention to Right To Try and one potential consequence of the various Right To Try laws passed in states is that the FDA could be influenced in terms of policies on expanded access (compassionate use).

Leigh Turner gave a very timely, excellent talk on the FDA, stem cell clinics that do not have FDA licensing or approval, and the potential consequences of this current dynamic. Leigh went into helpful detail on the clinic claims, the types of stem cells they use, and how these relate to the new FDA guidances. He discussed Cell Surgical Network and Stemgenex. It is notable that Leigh gave specific examples and names clinics by name, which I agree is vital to the discussion of the regulatory sphere and the place of stem cell clinics.

Gerhard Bauer, my colleague here at UC Davis and one of my favorite stem cell scientists, gave a wonderful talk on his experiences and perspectives over the decades as a pioneering stem cell and gene therapy clinical researcher as well as earlier as a regulator at the FDA of Austria. Gerhard’s discussion ranged from his experiences in these roles to also his own person experiences as someone who has dealt with doctors and also lost many friends to disease including HIV. I also appreciate how he provided his views from his real world experiences wending his own clinical trials through the processes and how much work it takes just to get an IND. How great it feels to get to that point!

A few additional notes

Mary Ann Chirba, a voice for increased patient autonomy in terms of their cells, was going to be a speaker, but got snowed in at Boston. Richard Garr, Neuralstem CEO, was also scheduled originally to speak, but couldn’t make it. They were missed, but still we had a diverse, dynamic group of perhaps 70-80 attendees.

A big thanks to Julie Bechtel who helped to arrange the event.