Diverse Stem Cell Person of the Year 2014 Award Finalists

Stem Cell Award Poll 2014With more than 4,142 votes cast, the readers of this blog have chosen the top 12 finalists for the Stem Cell Person of the Year Award for 2014 from the 27 nominees.

You can see the final vote tallies at left. The votes came from more than 50 countries with some interesting geographic patterns (I may do a post on that as a follow up).

I’ve pasted the brief bios of the twelve finalists below at the end of this post.

Now comes the tough task for me to pick a single winner from this amazing group. I will announce the Stem Cell Person of the Year 2014 within 1-2 weeks.

The finalists are a diverse group. They include scientists from academia and industry, patient advocates, a blogger, and the Pope. We have six male and six female finalists who live all around the world including in the US, Japan, Sweden, Canada, and Vatican City.

I’m happy to see both some familiar faces from nominees and finalists from past years and new ones too.

Who would you pick as the one winner and why? Post in the comments.

Finalists Bios (including in bold quotes from nominators)

Chris Fasano. A principal investigator at the Neural Stem Cell Institute where he uses stem cells to study early nervous system development. “Chris stands out for his energy, enthusiasm, dedication to the field, creativity and accomplishments.”

Don C. Reed. Long-time stem cell research advocate who played a key role in the success of Prop 71 and the creation of CIRM. “A tireless stem cell advocate always there to make a positive difference.”

Janet Rossant. Professor, University of Toronto. Stem Cell Researcher and Past President, ISSCR. “She works tirelessly to create new opportunities and collaborations…globally respected for her work in early development and embryonic stem cells”

Judy Roberson. Long-time Huntington’s Disease patient advocate. “She makes concrete positive developments happen such as millions of dollars in research funding for HD.”

JuuichiJigen. Japanese blogger who investigates scientific misconduct and played a key role in revealing the STAP scandal. He was the first to investigate and bring to the public of problems with STAP papers. His investigations demonstrated the role of social media and post-publication peer review in rapid self-correction of science.”

Malin Parmar. Associate Professor, Developmental and Regenerative Neurobiology, Lund University. Top neural regeneration scientist. “Young, hard worker who is doing very well”.

Masayo Takahashi. Stem cell researcher leading the team that is doing the first ever clinical study based on human iPS cells. “Creative and courageous clinical stem cell researcher.”

Pope Francis. Leader of Worldwide Catholic Church. “Strong supporter of adult stem cell biotechs and research”.

Robert Lanza. CSO of Advanced Cell Technology, which has multiple ES cell-based clinical trials ongoing. “Visionary and practical so makes the impossible possible with stem cells”.

Susan Solomon. Co-Founder and CEO of The New York Stem Cell Foundation (NYSCF). Remarkably effective advocate for stem cell research. “Not many leaders have created their own research laboratories and raised $100 million plus. Seriously, what an accomplishment!”

Ted Harada. Leading stem cell research advocate and very effective ALS patient advocate. “An Energizer Bunny for the ALS community and stem cell advocate”

Tory Williams. Stem cell advocate and author of the 2014 book, Inevitable Collision. Co-Founder and Executive Director of the Alabama Institute of Medicine (AIM). “A true hero who inspires and makes real things happen like AIM”.

Encouraging New Paper on ACT Stem Cell-Based Trial for Macular Degeneration

The stem cell biotech Advanced Cell Technology (ACT) reported new, positive data in a paper in Lancet from their clinical trials using retinal pigmented epithelial cells (RPEs) made from human embryonic stem cells (hESC) for treatment of different forms of macular degeneration (MD).

The paper was entitled “Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt’s macular dystrophy: follow-up of two open-label phase 1/2 studies” with first author Steven D. Schwartz and senior author Robert Lanza, CSO of ACT.

These two trials (one each for Stargardt’s MD and age-related MD (AMD) with 9 treated patients each) are combined prospective phase 1/2 studies. The primary goal of these trials is to assess drug safety. Importantly so far no major adverse outcomes were reported, but some adverse side effects appeared related to the procedure itself and to immunosuppression so those must be kept in mind. As to the latter, in theory an autologous induced pluripotent stem cell (IPSC)-based therapy could be superior in terms of likely not needing immunosupression, but there may be practical advantages to an hESC-based therapy in other ways (e.g. lower cost).

A potential major bonus here in the ACT report today is that despite the fact that the trial used relatively low doses of cells and despite the primary measure here being safety, ACT reported in this publication that a substantial number of the patients also had measurable improvements in their vision:

Vision-related quality-of-life measures increased for general and peripheral vision, and near and distance activities, improving by 16–25 points 3–12 months after transplantation in patients with atrophic age-related macular degeneration and 8–20 points in patients with Stargardt’s macular dystrophy.

This is a very positive, even if somewhat surprising development in terms of potential efficacy. The main cause of vision impairment in MD is not thought to be loss of RPEs, but rather photoreceptor cells. So how could transplanted RPEs (and relatively low cellular doses for the most part at that) potentially improve vision? The working theory seems to be that the RPEs might help remaining photoreceptors stay alive, healthier, and perhaps more properly functional.

ACT FIgure 1

I thought it was notable that 72% of the transplant recipients had measurable increases in subretinal pigmentation and pigmentation gradually increased over time, indicative of a high-rate of stable engraftment of the RPEs (see image above from Figure 1).

The authors summarized their interpretation of their results in this way:

Our study provides the first evidence of the medium-term to long-term safety, survival, and possible biological activity of pluripotent stem cell progeny after transplantation in people with any disease. The results suggest that human-embryonic-stem-cell-derived cells could provide a potentially safe new source of cells for the treatment of various medical disorders that require tissue repair or replacement.

I’ll be very curious to see the future results as ACT likely begins to treat patients with higher doses of cells and patients with relatively earlier (potential more treatable) stages of MD.

In the wider scheme of things, ACT’s results are also encouraging for other stem cell biotechs and other similar kinds of studies. For example, it will be interesting to see how the IPSC-based RPE clinical study in Japan for MD proceeds and how the BioTime subsidiaries (1) Asterias’ hESC-based trial for spinal cord injury and (2) Cell Cure’s hESC-based trial for AMD proceed. There can perhaps be greater hope of safety for these other vision-related pluripotent stem cell-based trials as well now and also for other studies such as ViaCyte’s hESC-based trial for Diabetes, which may start very soon.

Still, it’s relatively early days and these kinds of endeavors are risky marathons rather than sprints, so quite a lot of caution is in order.

Disclosure. The author has a small, long-term stock position in ACT. This post is not intended to be financial or health advice. Consult your financial advisor and doctor (not blogs) for making those kinds of important decisions.

ACT’s Bob Lanza Does the #IceBucketChallenge

Before I did the #icebucketchallenge, I challenged the leader of Advanced Cell Technology (ACT), Dr. Bob Lanza, to do the Ice Bucket Challenge.

He did it and leading up to it he provided a quite articulate message for context (see video below). Bob is one very cool guy even without ice water.

Bob nominated ACT scientists Irina Klimanskaya, Shi-Jiang (John) Lu, and Erin Kimbrel to go next.

Where I Do The Ice Bucket Challenge & Who Did I Challenge?

Paul  ice bucket challengeI was challenged to do the Ice Bucket Challenge by Roman Reed.

I did it yesterday and can see it was mighty cold! Total shock, but fun. It sure wakes you up. Yeah, we have a drought but I did the big splash with tons of ice.

I did the challenge in honor of patients with ALS and the ALS Foundation, patients with Spinal Muscle Atrophy (SMA) and the Gwendolyn Strong Foundation, and the St. Baldrick’s Foundation for children’s cancer research. I’ve already given to St. Baldrick’s and shaved my head this year for the third year in a row. I’ll be giving donations to the ALS and Gwendolyn Strong Foundations. Please consider giving as well.

In my video (below) I challenge Robert Lanza of Advanced Cell Technology (ACT) to do the challenge too. What do you say, Bob?

Top 10 questions for stem cell field bouncing around on a Friday morning

top ten listWhat are the most interesting questions for the stem cell field in 2014 right now?

What’s on your mind?

I’m not going to give this deep thought, but rather just list those questions bouncing around at the moment in no particular order. You can see a lot of translational/clinical things are on my mind. I’ve put possible answers, but these are highly speculative and/or based on hope in some cases.

  • What’s next for ACT’s hESC-based clinical trials and Takahashi’s iPS cell-based clinical study for macular degeneration? I’m optimistic on safety for ACT and hopeful on efficacy, while it’s harder to predict for the iPS cell study.
  • How will ViaCyte’s new early hESC-based trial for Diabetes go? I’m cautiously optimistic.
  • In a year, where will the iPS cell IP/patent arena stand and who is BioGatekeeper? I expect no more clarity in a year overall, but BioGatekeeper will be outed by then or much sooner.
  • Will the trend of for-profit networks of stem cell clinics exploding across America continue? Unfortunately, yes.
  • Is the FDA planning to get back into the ballgame of regulating such stem cell clinics or has it punted due to (fill in the blank: budget problems at the agency, other priorities, a change of philosophy, etc.) Who knows? It’s been a disappointing year for the agency in this area.
  • Will the FDA apply Fast Track, Accelerated or Priority Review or Breakthrough Therapy Designation to any emerging stem cell products? It’s about time. They should.
  • What will the impact of the likely growing number of state Right To Try laws be? I expect it will increase both risk and innovation, but unfortunately much more of the former than the latter.
  • Was the flood of very high profile stem cell paper retractions so far in 2014 an aberration or will it painfully continue? I hope it does continue, but it’s a worry as the field needs public trust and respect.
  • Will a big pharma acquire a small biotech like Athersys, ACT, StemCells, Inc., Neostem, in the coming year? More money is sure flowing from big to small (witness ViaCyte’s new influx of money) and we saw this with happen California Stem Cell. I think we’ll see this trend of money flow or even acquisitions accelerate although there are structural obstacles to being acquired for certain companies.

What questions are on your mind right at the moment related to stem cells and regenerative medicine?

Note this post is simply informational and is not financial advice. Only make financial decisions after consulting with a certified financial planner, which I am definite not. I hold no stake in any of the companies mentioned.