The stem cell biotech Advanced Cell Technology (ACT) reported new, positive data in a paper in Lancetfrom 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.
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 trialfor 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.
Nominations have closed and we have more than two dozen nominations for Stem Cell Person of the Year 2014. It’s an exciting, diverse group including some news faces as well as nominees from years past as well.
You can now vote for your choice for the top finalists in the poll below. One vote per IP address is allowed per day. Voting will end on October 22 at midnight. The winner of the voting may not be the overall winner of the 2014 Stem Cell Person of the Year Award, but more votes sure cannot hurt. This voting will pick the top 12 finalists from whom I will choose the winner.
A short bio of each nominee is included below the poll with the nominees listed in alphabetical order by first name. Their names have links to pages about them. In addition, I have included a quote from the nominator(s) in green. I encourage you to learn more about these amazing people.
Bernard Siegel.Bernie is a long-time stem cell advocate who runs the yearly World Stem Cell Summit. “Has any one single person done more for the stem cell field?”
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.”
Diana DeGette.Rep. DeGette is a politician who has been working to pass important stem cell legislation. “Long time supporter of legislation to support stem cell research and regenerative medicine.”
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.”
Emmanuel (Ed) Baetge. Head of Nestlé Institute of Health Sciences at Nestlé Health Science and formerly CSO of Novocell/ViaCyte. “…Novocell (Viacyte’s) diabetes program using hESCs has huge potential and Ed deserves the credit for his leadership and development of the technology as well as the company.”
Ellen Feigal. Senior Vice President, Research and Development at the California Institute for Regenerative Medicine (CIRM). “At CIRM Dr. Feigal makes the Development Program happen.”
Ian McNiece.Professor, Department of Stem Cell Transplantation, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center.
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”
Jeanne Loring. Stem cell researcher and scholar, leading iPS cell clinical work in the pipeline for Parkinson’s Disease, and patient advocate. “Supportive to the advocacy community. Courage in supporting the challenge to WARF patents. Excellent scientist engaged in policy discussions.”
John Sinden. The co-founder of ReNeuron, a biotech company based in UK. “Thanks to his determination and drive, ReNeuron has a clinical approved stem cell product currently tested in two clinical trials.”
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.”
Leigh Turner. Associate Professor, Center for Bioethics, University of Minnesota. Outspoken advocate of evidence-based medicine in the stem cell field. “Gave an inspiringly frank talk about for-profit stem cell clinics that do not FDA regulations at ISSCR 2014.”
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.”
Mike West.CEO of BioTime. He also founded Geron and was CSO of Advanced Cell Technology. “He has been a leader in our regen field for many years but he made a bold decision to resurrect Geron’s cell therapy for spinal cord injury this year and hopefully confirm the promise of hESC technology clinically.”
Patricia Olson.Executive Director of Scientific Activities at CIRM and active in CIRM scientific leadership from day 1. “A driving force in the stem cell field. CIRM could not function half as well without her.“
Peter Zandstra. Professor at University of Toronto. Stem cell researcher. Canada Research Chair of Stem Cell Bioengineering. “Peter is a recognized pioneer and respected world leader at bringing concepts of scale-up, regulation, and industrialization to stem cell-derived cell therapy technologies.”
Pope Francis.Leader of Worldwide Catholic Church. “Strong supporter of adult stem cell biotechs and research”.
Richard M. Cohen. An MS patient in a clinical trial who has chronicled his experiences. An author and Meredith Vieira’s husband, father of three children. “Mr. Cohen is an inspiration to me and others because he’s very honest and doesn’t sugar-coat his struggles, loses, frustrations, anger, embarrassments, and he also shares his gains.”
Richard Garr. CEO of Neuralstem, conducting work on using stem cells for ALS and advocate of Right To Try laws. “Richard does two excellent things at once: making a treatment for ALS and connecting with ALS patients.”
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”.
Shoukhrat Mitalipov.Stem cell researcher who first successfully made nuclear transfer human ES cells by therapeutic cloning and developing oocyte transfer-based therapies for mitochondrial disorders. “Gutsy pioneer of new, game changing technologies.”
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!”
Takaho Endo. Senior Researcher at the Riken Center for Integrative Medical Sciences in Yokohama who published key genetic workon possible origin of STAP cells. “Courage to publish the genetic data challenging origin of STAP while also being a scientist at RIKEN.”
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”.
Some notes. I do not endorse any specific nominee and just because someone is a nominee does not mean I support all of their specific efforts. I also do not necessarily agree with all the language used to describe the candidates by the nominators. Voters who repeatedly do not follow the rules my have their votes deleted and be blocked from voting again.
Stem cell biotech BioTime announced the news today that its subsidiary Cell Cure Neurosciences (Cell Cure) has filed an IND with the FDA for an embryonic stem (ES) cell-based therapy for Dry age-related macular degeneration (AMD). The product to be tested is OpRegen, which the company indicates is “the first IND for an ES cell-based therapy developed in Israel.”
Cell Cure in its PR on this IND also listed three other key bullet points:
“IND filed for Phase I/IIa dose escalation trial in patients with dry-AMD
No approved therapy exists for dry-AMD, the leading cause of visual impairment in the aging population
OpRegen®will be the first preparation of xeno-free RPE cells to be evaluated clinically for dry-AMD”
I’m very curious to see how OpRegen performs in the trial.
This is only the third IND for an ES cell-based therapy with the other two coming from Advanced Cell Technology (ACT) and Geron.
I asked Dr. Mike West, CEO of BioTime, about the trial and he had this to say:
“Age-related degenerative diseases are really the frontier of medicine in our time. The aging of 76 million baby boomers and the consequent tsunami of healthcare costs associated with palliative as opposed to truly therapeutic care is a top national priority. I believe that AMD is only one of numerous examples where the stable engraftment of young healthy cells may provide important new therapeutics for these long-term and expensive problems of aging.”