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.

Stem cell treatment cost 2.0: legit therapy

stem cells costWe hear so much about exciting potential stem cell therapies. Some of these are rigorously evaluated ones in the FDA clinical trial pipeline and others are available right now mainly through predatory stem cell clinics. Earlier this year I posted about the cost of the offerings of dubious stem cell clinics.

In this post, I address the cost of a future, legitimate, FDA-approved stem cell therapy. How high will that be?

This is a critical question because if many patients cannot afford a stem cell therapy then the impact of that therapy is reduced. Cost is inversely related to access. On the other hand, stem cell biotechs must make some profit or they will go out of business. Investors, who are often enthusiastic boosters of the stem cell field, will lose large sums of money and confidence in the field too in that scenario if stem cell treatments are priced “too low”. What is the “right” price?

There is likely to be increasing pressure on biologics drug prices as well from the federal government. Witness Hillary Clinton’s recent tweet on this topic below that sent people into a tizzy.

At the state level, such as here in California, the question of stem cell treatment cost is also becoming more pressing including for our state stem cell agency, CIRM. As CIRM-funded clinical trials advance, which is a wonderful thing, at the same time we get closer to where someone will have to decide on stem cell price tags.

We can look at what other cellular drugs have cost as guidance for the price tag range for stem cell treatments. For instance, Prochymal (its old name under Osiris) that is now rebranded as TEMCELL from Mesoblast/JCR likely will cost about $200K for a full treatment for GVHD (HT to Alexey). Provenge, the cellular prostate cancer drug from the controversial biotech Dendreon had (has?) a price tag of $93K. The most expensive drug in history, the gene therapy med Glybera will cost around $1.5 million per patient.

Realistically, a typical legit stem cell therapy could easily be $100K per patient. A personalized cellular medicine such as an autologous stem cell-based therapy could easily run into the hundreds of thousands per patient. Some therapies could go as high as $500,000 (see this helpful piece by David Jensen) or even into the millions.

Irv Arons

Irv Arons (@iarons) has come up with a great table of cost estimates focused in the area of vision therapy (free registration required). Thanks to Irv for permission to use it here (above).

How will patients afford such expensive therapies?

Will such therapies be covered by governmental agencies or insurance companies? They should.

We should also be keeping in mind the current costs of treating today’s patients with major and sometimes chronic diseases. These costs run into the hundreds of billions or above a trillion dollars each year in the US alone. That’s important context and rightly indicates that the costs of stem cell therapies to society may be appropriate even if at an individual level they seem high.

How does this compare to stem cell treatments at predatory clinics?

Such “treatments” range from $5,000-$20,000 each and most patients with whom I have talked either received or were pitched at least two such treatments, amplifying the total cost. The cost to the clinic of the treatment itself can be as low as $500-$1,000. Some clinics claim to have treated thousands of patients suggesting they are making millions in profits.

Why are stem cell clinic offerings typically relatively cheaper than legit treatments? Frankly, it is because they don’t follow the rules or do the necessary studies to prove safety and efficacy. Ten thousand dollars is still a lot to pay for something that doesn’t work and could even be harmful.

Even so some consumers may perceive dubious stem cell treatments as the way to go because of the lower cost, particularly if the legitimate stem cell field fails to do a good job at educational outreach and the FDA continues to effectively do nothing about the stem cell clinic problem.

The bottom line remains a question. Where’s the stem cell price sweet spot where we can help the most patients, but also generate a needed profit for the biotechs?

We need to find an answer to this question soon.

Stem Cell Person of the Year 2014: Masayo Takahashi (高橋 政代)

Masayo Takahashi

Dr. Masayo Takahashi,  Asahi photo

Congratulations to Masayo Takahashi (高橋 政代), MD, PhD, the winner of the 2014 Stem Cell Person of the Year Award.

Dr. Takahashi received this award including the $2,000 prize for her exceptional achievements in stem cell research in 2014. She was selected as the winner from a stellar group of top 12 finalists this year.

Takahashi leads a team doing high-risk, high reward research that is conducting the first induced pluripotent stem cell (IPSC) clinical study in humans ever. I interviewed Takahashi at the beginning of this year and you can learn more about her research and vision for the future from reading that interviewMonkey stem cell RPEs

The Takahashi team clinical study is intended to examine the safety of a human retinal pigmented epithelial cell (RPE) product made from each patients’ own IPSCs. You can see at right RPEs produced by her team from monkey pluripotent stem cells.

In an astonishing feat of speedy clinical translation, Takahashi’s team transplanted its first macular degeneration patient recently on September 12, only 7 years after human IPSCs were first ever published. The usual timeline for such translation would be 20 years. In that regard, in a recent interview I did with him, Nobel Laureate Shinya Yamanaka had this to say of Takahashi and her work:

I was surprised that after the announcement of human iPSCs in 2007, Dr. Takahashi told me that she would bring iPSC to the bedside within five years. I thought it possible technically speaking, but doubted it could be done so soon, since we needed to improve the technology and get government approval. It took 7 years, which is remarkable considering the work required. Both the accomplishment and the speed at which it was achieved are testaments to Dr. Takahashi’s leadership and her strong team.

Her achievements extend beyond this year to an outstanding long-term track record in vision research including a very impressive track record of highly-cited publications. Takahashi is physician scientist, who is a faculty member and Project Leader at the Laboratory of Retinal Regeneration at the CDB at RIKEN. Some of her nominators for the Stem Cell Person of the Year Award described her as a “transformative” and “courageous” stem cell scientist. Below you can see a TEDx talk from just a few months ago by Takahashi explaining her work.

Takahashi joins previous Stem Cell Person of the Year Award recipients Roman Reed and Elena Cattaneo as outside-the-box thinkers who to take risks to make outstanding new developments in the arena of stem cell research with the goal of helping others.

More about the Stem Cell Person of the Year Award. I fund this prize myself as a way of giving back to the stem cell community and recognizing transformative people who take risks to help others. It is to my knowledge the only annual, international science-related prize personally funded by a professor.

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.

Great 2nd Place ISSCR Essay Related to Vision and Stem Cells

Hikita2smallerISSCR and I held a short essay contest for a free registration to ISSCR 2014 in Vancouver. The winning piece was written by student Mohamed Gatie, but there was a great 2nd place essay by Sherry Hikita that deserved acknowledgment. I’m publishing that essay below and Sherry will be receiving a free signed copy of my book and a free stem cell t-shirt. Sherry was formerly at UCSB and is now a scientist at the very cool stem cell biotech, Asterias.

Here is Sherry’s essay:

It was pitch black.  My eyes were wide open but I could not see anything.  I could, however, smell something delicious.  I gingerly felt where the silverware was on the table, but without being able to see, I realized it would be incredibly difficult to use these utensils.  Dining with my hands was better.  I could at least feel where different foods were on my plate and could feed myself, though identification of the meal was another challenge.  This might be chicken.  Or pork?  There may have been a carrot but the other objects were a mystery.  My “Dining in the Dark” meal (hosted by Foundation Fighting Blindness to raise awareness about retinal degenerative diseases) lasted only 30 minutes, but this period of being sightless was a truly profound experience.  My motivation to investigate and develop a stem-cell based therapy for macular degeneration has been fueled by the intellectual challenge, the potential of stem cells and the worthwhile endeavor of restoring vision to those who cannot see.  But this particular experience turned into a much higher octane fuel.  I knew, with great relief, the lights would be turned on soon and I would continue living, with vision.  But for those who are blind and vision impaired, there is no such relief.  Our servers were vision-impaired people who shared their stories and challenges, while graciously providing advice for the unexpectedly difficult task of navigating a meal in the dark.  I did not see their faces but I heard their voices and felt their hopes for research that might enable restoration of sight.  These people are my jet fuel and motivate me to keep learning and striving as a stem cell scientist.  And the ISSCR and its annual meetings are critical for helping me continuously develop my craft.