ISSCR 2014 Day 2: Navigating Balanced Stem Cell Path to Clinic

I’ve really enjoyed day 2 of ISSCR 2014.

So far this day has already packed in a slew of great talks both in the lecture halls and outside one-on-one with old friends and new ones. How great is that?

I’ll focus on just a few highlights to keep it concise.

Dubious stem cell clinics and the FDA: a match not made in heaven?

The ISSCR conference day started off a session entitled “Therapies in the Clinic” and my colleague Leigh Turner kicked things off with a great talk on regulatory oversight of stem cell interventions and dubious clinics.

Leigh rightly pointed out how dubious stem cell clinics are exploding right here in the US. He used the stem cell clinic network, Cell Surgical Network (CSN), as a case study. For background, you can read my blog interviews with the leaders of CSN here and here as well as my specific concerns about how they are operating here. The leaders of CSN were remarkably frank about their views in these interviews. CSN seems to make the case in a general sense that they are not subject to any FDA regulation, but you can see for yourself based on their comments in the interview pieces.

Leigh pointed out that CSN is a growing network of dozens of clinics operating without any apparent licensing or approval from the FDA, they use devices not specifically approved for production of specific stem cell products for human use, and they offer to treat a host of conditions.  For the first time, Leigh also publicly revealed today that he sent a letter of concern about CSN to the FDA way back 18 months ago. It seems that the FDA has not specifically had any reaction to CSN. Who knows, maybe it never will.

In fact more broadly I don’t see any evidence of FDA action regarding the more than 100 total such clinics throughout the US. What is going on and why no apparent FDA action? I believe the FDA is on course to address the growing issue of dubious stem cell clinics, but is moving far too slowly…kind of like a glacier.

Meanwhile the number of such clinics and as a result from my view the population of patients being put at risk is rapidly rising. My message to the FDA would be that you need to speed up and make clear whether you are (or are not) going to regulate such clinics or it will result in default deregulation. To the members of ISSCR and other stem cell researchers I would say these clinics are not only putting patients at risk, but they are also a serious danger to reputation of the whole stem cell field with the public.

Stem cell translation to the clinic for Type I Diabetes and Parkinson’s Disease.

Then came two great talks on clinical translation of stem cell-related therapies for two major illnesses: Type I Diabetes and Parkinson’s Disease (PD).

James Shapiro of the University of Alberta gave an intriguing talk on the history and future of stem cell treatments for Diabetes. Islet cell transplantation conducted in the past has yielded clinically very significant outcomes in about 15% of patients. It was exciting to hear that more recent adaptations of such transplants are giving about half of treated patients insulin-independence over a period of a few years of follow up. Adverse outcomes only rarely occur and include infection and immune responses. The new protocol includes the key added feature of incorporating alemtuzumab treatment. He also mentioned alternatives to islet transplantation including stem cell-based approaches such as those being done by Viacyte. Very exciting.

Roger Barker of the Cambridge Center for Brain Repair talked about fetal stem cell transplants for PD. One thing that surprised me was the stat he quoted that 1 in 800 people get PD, which means a huge number of people are impacted. Dr. Barker presented a very concise, clear overview of how hard it is to develop a therapy for PD that would be considered a success. Nonetheless his talk gave reason for optimism. Past fetal grafts resulted in some degree of benefits to recipients who had PD, but there was also a risk of dyskinesias. There will be a new trial (called something like EuroTrial?) based on this approach starting in a few weeks for which there is hope. hESC and iPS cells are additional alternative bases for treating PD.

Patient Advocacy

We also heard from patient advocate Jennifer Molson who gave an inspiring talk about her experience with Multiple Sclerosis (MS). Molson had a devastating form of MS that derailed her life at a very young age. A hematopoietic stem cell transplant in 2002 led to a dramatic recovery for her to a point of regaining her life. She is celebrating her 12-year anniversary of treatment. My favorite quote from her talk: “I am living proof that stem cell research can change lives!” Patients such as Molson are the reason why many of us work on stem cells.