It is nice to be back home, but I really enjoyed the World Stem Cell Summit.
It was an energizing, inspiring meeting.
There was a huge amount of information presented including new ideas and challenges to the existing paradigms.
I’ve condensed it down for you. Below I list and briefly discuss what I thought were the top 6 critical take home messages from the meeting.
1) We are a great team, but we need to be more aligned. Our opponents on the other side are uber aligned and formidable. We need to be more integrated and have coordinated plans. The WSCS is awesome and it sure helps align us, but we need to interconnect on a more than yearly basis.
2) We need a strategic plan and improved message discipline. We are getting a little better at working with the press to advance our cause, but too often our message is overly complex and diluted. I think our diversity gives us strength, but we should have some key messages/talking points.
3) We need to rapidly evolve a new partnership with the FDA that includes promoting our priorities of balancing risk in a way that is more context-dependent and accelerating stem cell drug development (think Andy Grove’s talk here).
4) We need scientists to be advocates. Of course there are already great scientist advocates and many were at the WSCS, but they are probably <1% of stem cell scientists. We need scientists to go outside their traditional comfort zones and advocate using their authority in public. This is a woefully underutilized strength. Some ideas include workshops by CIRM and WSCS that directly teach and encourage scientists to do this. I’d be happy to be involved in making this happen. It is crucial.
5) We need to be realistic and forward thinking about funding. Bob Klein’s talk about funding was outstanding. There is a looming black cloud over funding for stem cell research because the same kind of ominous cloud is over science and research more generally in the U.S. We need to continue the battle within the U.S., but his idea of an international basis for research funding is intriguing.
6) We need to be wary in a smart way of so-called “stem cell tourism”. Many at the Summit don’t like the term “stem cell tourism” and I agree it has its problems, but I think when we say that phrase people know what we are talking about and it resonates. Regardless of terminology, clinics or even individual physicians offering non-FDA approved stem cell-related treatments have the potential to not only seriously hurt or kill people, but also hurt our field by association. All you have to do is imagine a scenario where we have a death such as the one that occurred at the German clinic and imagine the roaring headlines of something like “stem cell treatment kills”…and we could be in the same boat as gene therapy was, which as a field has taken more than a decade to recover. It doesn’t matter if the clinic involved in such a hypothetical scenario was not legitimate to us; there will be guilt by association and the public won’t distinguish.
7) We need to be conscious of the cost issue. No matter how incredibly stem cell treatments will be for a variety of diseases, realistically we have to be aware of how much they will cost. For many diseases it seems hard to imagine putting a limit on what would be a reasonable cost. For example, I as a prostate cancer survivor would think that a cure for prostate cancer would be worth a very high cost. The same goes for other life-threatening, life-changing conditions. But as a society, cost is an enormously important issue. On the other hand the cost of caring for patients exceeds a trillion a year. Stem cell treatments, even expensive ones, may be money savers in the long run. Still, we need to keep this issue in mind and be as realistic as possible.
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Dr. Knoepfler,
Thank you for sharing your experience at the WSCS. Many of your key points resonate with me. I have the good fortune of being part of a stem cell study for ALS and have been fortunate to receive incredible benefits from the million stem cells injected into my spinal cord.
As a patient with ALS as well as someone who is a “research subject” of a stem cell study and a verified recipient of benefits from stem cells this is my perspective based on my extremely limited knowledge. I will go in numerical order of your points.
1) No matter what business or industry you are in this makes sense. A great example of this may be in a post you made a few months ago in regards to genotype. In collaboration with China you seem to have some technolgy which provides the means to map your genotype. I am not sure if this is viable now or still progressing. Certainly as one of the responses pointed out if it is an option we should be matching stem cells up with patients. Perhaps since I have seen so much more progress then anyone else see if I match up more closely to the fetal stem cells being utilized. As I said I am just a laymen so perhaps this makes no sense and if so I apologize for my naivete.
2) There is so much talk about ESC’s, FSC’s, ASC’s, IPSC’s but there is not a clear cut message as to what is the best and why. There is both accurate and misinformation flooding the internet and trying to decipher it as a non – scientist without a clear cut focused message is difficult. Personally, despite the fact I received fetal stem cells, in order to avoid controversy and ethical concerns I hope that IPSC’s (which I believe are adult stem cells) become the ultimate viable option. Of course there is so many mixed messages that are difficult to decipher. Who is the leader and the spokesmen? Seems to me too many companies / scientists with conflicting messages that ultimately reflects only their best interest but not the best interest of the industry or of patients.
3) The FDA situation is a difficult but vital situation that needs to be resolved. For example despite the safety results that the trial I am in has produced they have not yet received permission from the FDA to move ahead to patients 13 – 18 where the stem cells will be injected into the cervical area of the spine. Think about it, 12 patients completed, no adverse side effects from either the surgery nor the stem cells. The two patients in the study that have died were not due to anything with the study. One was due to a undiagnosed heart condition and one due to respiratory issues as a result of the advancement of his ALS condition. The autopsy of their spinal cords showed that there was not even scarring on the spinal cord. So despite all of the positive safety data being submitted to the FDA back in June the FDA has not allowed the progression of the trial. The surgery for patient 13 was scheduled for the end of August but has been forced to be delayed while they wait for the FDA to make a decision.
4) I could be mistaken but from my outside and again limited perspective 3 & 4 are related. Of course most of my exposure is to practicing Dr’s who are also researchers. I believe that while they are willing to speak out and be advocates they are also extremely cautious as to what they say due to the FDA and fears of ramifications.It appears the FDA holds too much power and is the defacto judge, jury and executioner and both Dr’s and researchers do not want to be on the wrong side of the FDA.
There also needs to be a lot more factual education and advocation on behalf of stem cell research and it’s potential. Not only could this help the image of the research here, which could prompt more funding, your next topic, but it could also reduce stem cell tourism which is topic #6.
5) The econmy both here in the U.S. and globally is what it is. So everybody is fighting for dollars. There could possibly be some stem cell fatigue among investors as stem cells have been positioned as the next big thing for over a decade now. In a economic climate where we judge our investments every 90 days there is little patience and therefore little money leftover for longterm investing and results. Again your points 2,3 and 4 tie directly into 5. I am sure if the study I am in could showcase more openly funding would drastically increase. As it is now the eceedinly and frustrating slow pace in which the FDA forces trials to run makes investments in companies doing this research unappealing. A united message withing leading researchers and scientists being advocates and out in fromt of the public would serve to not only educate the general public but the investment community as well.
6)Stem cell tourism is a legitimate threat to stem cell research and science. I belong to a few ALS forums and it is painful to see people spend their money and their precious time investing in these frauds. 60 minutes and a couple of other media sources has done some stories on the dangers of this fradulent clinics yet people with no hope continue to turn to them. Again lets review the previous topics. No one out in front communicating on behalf of stem cell education to the public, FDA making trials go excrutiatingly slow with little communication on results, little funding it is no wonder people turn to these unscrupulous clinics they have little information to know better and no alternatives. Another side effect I see from stem cell tourism on the ALS forums is that some people sour on stem cells all together. They have been hearing about the possibilities of stem cells for more then 10 years and the only results they see are the lack of progress and the fradulent clinics. So they end up throwing the baby out with the batwater and losing interest in stem cells altogether.
7) Why cost and fiduciary concerns are always important, what is the value of a human life? I have seen great imptovement from my stemcell treatment. I no longer go to the ALS clinic, only my research appointments. This is a cost savings right there. Long term costs of treating ALS patients are estimated as high as $250k annually. If they can duplicate my results in other patients it is not only a win for humanity but a cost win as well.
I have referred to my results and the study I am in a few times. To be fair I must point out that the results are not a cure. It is a small pool of patients being studied thus far and there is no placebo group. The results are still early and a lot yet to be learned. That being said their is empirical data that provides reason for hope and to be cautiously optimistic. This story is not about me it is about shining the light on ALS itself, the need for advancing and funding this and similar types of research and providing hope to the ALS community which is a commodity that is sorely lacking among ALS patients and their families.