Mini-bios on some top finalists for Stem Cell Person of the Year

Heather_MainBy Heather Main

I was a little ashamed of myself that I didn’t know who half of the finalists were in Paul Knoepfler’s Stem Cell Person of the Year Award. I decided to look into it and find out what (or who) I have missed…..Interestingly, 3 of the 7 listed here are not scientists but instead are patients and/or patient advocates, showing the major impact these groups have on progression of our field. I have listed my write-ups of the finalists here alphabetically.

Bill Moss – FSHD sufferer, FSHD global foundation founder and chairman

Bill was diagnosed with FSHD (the most common form of muscular dystrophy) at 28 years old. The youngest child of a gardener, Bill became a masterful businessman and spent 23 years as a senior executive and Executive Director of a pre-eminent Australian investment bank before leaving at 52 years of age to pursue philanthropic initiatives. He has self-funded programs for industry development in Australian aboriginal communities, advocated for disability rights and established the FSHD Global Research Foundation. This foundation funded derivation of the first FSHD affected hESC and successful highthroughput modeling of the disease through development of an efficient protocol to differentiated them to skeletal muscle. 

Jeanne Loring – Academic researcher

Personally I think PluriTest (the bioinformatic test for assessing pluripotent status) alone earns Jeanne a spot in this ‘hall of fame’. Her lab also leads fields including the ‘Frozen Zoo’ (iPSCs from the endangered species), translational projects in arthritis, Alzheimer disease, Parkinson’s disease, autism, and addiction as well as creating collections of iPSC lines for disease modeling and ethnically diverse toxicity testing. Jeanne is also recognized for her public work in ethics, legal issues, clinical use, cell generation and clinical use, the legal implications of patents and stem cell tourism. In December she will be awarded the 2015 World Stem Cell Summit ‘Stem Cell Action Award for advocacy’.

Judy Roberson – Huntington’s Disease patient advocate

Judy is a registered nurse, who now works as a full time volunteer patient advocate for Huntington’s disease. This includes supporting the UC Davis Huntington’s Disease Clinic, the UC Davis Institute for Regenerative Cures, and CIRM (California’s Stem Cell Agency), including legislative advocacy. She was appointed as the first voting patient representative to the FDA Huntington’s Disease Advisory Committee and is past president of the northern California chapter of the Huntington’s Disease Society of America. Judy advocates for the role patient advocates play in driving stem cell sciences towards making the goals a reality.

Michele de Luca and Graziella Pellegrini – academic researchers/clinicians

Professor De Luca, and his principal collaborator Graziella Pellegrini, have worked together over 20 years, towards the use of human epidermal stem cell cultures for life saving burns treatments as well as treatment of repigmentation and piebaldism. The team has also established human limbal stem cell culture for sight restoring corneal regeneration. Michele De Luca is coordinating the first (successful) gene therapy clinical trial for junctional epidermolysis bullosa and studies the stem cell behaviours of epithelial stem cells. Professor Graziella Pellegrini is based at the Centre for Regenerative Medicine “Stefano Ferrari” at the University of Modena and Reggio Emilia and one of the principal scientists on the corneal repair system Holoclar ®.  Together they have achieved Europe’s first approved advanced therapy medicinal product (ATMP) to be integrated into health services for patients across Europe in the future. 

Robert Lanza – ‘child science star’, CSO of Ocata Therapeutics

Reading about Robert Lanza is like reading about a rock star. In TIME magazine’s 2014 100 most influential, in FORTUNE magazine and even suggested to be played by Ben Affleck if his work leads to restoring eyesight to millions. Son of a professional gambler and growing up in Boston, Massachusetts, the weirdest find is that at the age of 14 (1969), he partially induced melanin synthesis in chicken to change the colour of its feathers for a science fair project. While it is hard to find the details of this first study, at the age of 19, with the assistance of Stephen Kuffler and Joshua Sanes at Harvard Medical School, Robert published a complete change in pigmentation in Nature using nucleoprotein from the Harco chicken and a standard cross between the Barred Plymouth Rock hen and the Rhode Island Red rooster. Since this early fascination of genetics, Robert developed methods for deriving hESC lines from single blastomeres in an attempt to avert the ethical discussions of the destruction of human embryos and is now running clinical trials for macular degeneration with hESC derived retinal cells.  Robert is currently Chief Scientific Officer at Ocata Therapeutics (formerly Advanced Cell Technology).

Shoukhrat Mitalipov – academic researcher

Shoukhrat was the first to derive hESC through somatic cell nuclear transfer, publishing in Nature 2013. He is also the ‘father’ of 3-parent IVF where by ‘spindle transfer’ the genetic material of a donor oocyte is swapped out for the DNA of a mother who carries a mitochondrial disorder. This creates a new egg with ‘normal mitochondria’ and nuclear genetic information from the mother, which is then fertilised with the sperm of the father. The technique is successful in rhesus macaques but has not yet been tested in humans. Shoukhrat’s company Mitogenome Therapeutics appears to be pursuing 3-parent IVF in China, because it is not currently allowed by the FDA in the US.

Ted Harada – ALS sufferer and unproven stem cell treatment recipient

Another patient advocate, Ted has received two doses of fetal neural cells in his spinal cord from Emory University in Atlanta as an experimental treatment to slow progression of his ALS. While Ted showed improvement in limb movement, strength and dexterity from the first transplantation his abilities have slowly been regressing, leading to this second treatment. Ted is a strong advocate for “Right To Try” that patients’ choice to receive experimental treatments should be respected and will speed up innovation.  He challenges the idea that patients who are out of options may need to settle for ‘silver standards’ with novel FDA approved treatments taking 10 – 15 years and sometimes nearly a billion dollars. He feels that these patients do not have that time to wait. Refreshingly, he is not a fan of stem cell tourism and the Right-To-Try movement stipulates the treatment has to have completed an FDA phase I portion of a trial and been approved for phase II.

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.

Neuralstem flirting with stem cell noncompliance in Colorado via Right To Try Law?

Colorado is an independent-minded state. For example, it is one of only a few states including Washington State with legalized marijuana use.

More recently, Colorado passed a new law supported by the ultra-conservative Goldwater Institute, the so-called Right To Try Law. This law allows patients to try experimental, unapproved medical interventions that have completed a Phase 1 clinical trial. Most Phase 1 drugs ultimately fail to be proven safe and effective and Phase 1 only demonstrates a minimum baseline level of safety.Dallas_Buyers_Club_poster

Interestingly, Colorado is also a hot bed for experimental stem cell-related interventions. Putting these two realities together, when the Right-To-Try Law passed it seemed likely that we’d be seeing Colorado ending up as a magnet for patients wanting to try experimental, non-FDA approved stem cell therapies and that stem cell companies, including some that previously had records of compliance, would eventually begin federally non-compliant or at least unapproved operations in Colorado.

It didn’t take long for these predictions to be realized as stem cell biotech Neuralstem announced on June 5 that it would be offering its experimental, non-FDA approved human neural stem cell therapy for ALS in Colorado. And it might do it in a for-profit manner. Further, Neuralstem CEO Richard Garr has indicated that the company will not work with the FDA for the human experiments in Colorado. It is worth pointing out that CEO Garr is also Chair of the Goldwater Institute’s Right To Try National Advisory Council.

One of the big concerns about Right To Try-enabled operations of this kind is they could well end up being conducted in a manner that is noncompliant with federal law such as by exposing relatively large numbers of patients to risks.

Apparently the Colorado Right-To-Try Law was inspired by the movie, The Dallas Buyer’s Club, at least in a quote attributed to Colorado State Sen. Irene Aguilar. In his column on the law, David Kroll in turn quoted a column by bioethicist Art Caplan criticizing the law:

“Passing bills full of empty promises based on Hollywood rather than the real world of today is not compassionate but, actually, flat-out cruel.”

As much as I support expanded compassionate use for patients with a fatal illness such as ALS or terminal cancer, Right To Try is not likely to be the solution. It is not the same thing as carefully administered compassionate use, it could put patients at risk, and it could falsely raise hopes of patients. I can understand how some patients and their families would support Right To Try though.

There is a not so easily resolved tension between the completely understandable desire of terminally ill patients to be allowed to try experimental drugs and the concerns of those who advocate for appropriate regulatory oversight as well as evidence-based medicine. This is made all the more complicated by the current clinical trial system that is slow, expensive, and clunky, but absolutely something that should not be thrown out entirely by any means.

Until you are facing a potential terminal illness you don’t know what it is like to be in those shoes. When I was diagnosed with a very serious form of prostate cancer in late 2009, I thought for a while it might ultimately be a terminal cancer for me given what my doctors were telling me. Now four and a half years after surgery I’m doing great (knock on wood), but the cancer could come back. I know that and in part because of my own experience I respect patients greatly.

Still there are definitely some complexities and potential problems that are going to pop up in Colorado due to this new law. Patients are likely going to be put at risk and potentially end up spending tens of thousands of dollars for interventions that don’t work, but as a company Neuralstem is also potentially putting itself in jeopardy. Given that federal law trumps state law, it is unclear to me that Neuralstem can simply ignore the FDA.

It is also likely that the Right To Try Law will embolden other for-profit stem cell entities in Colorado to push the limits of what is permissible in terms of experiments on patients and may well lead companies from other states to do some stem cell interventions in Colorado thinking they are shielded by the state law. They might not be. To protect patients at least financially, I propose that Right To Try laws require that companies that engage in these activities be required to do so only in a not-for-profit manner. It would also be ideal if such companies consult with bioethicists in advance of beginning such operations.

Given that other states have already passed Right To Try laws including Louisiana and Missouri that are awaiting their respective governor’s signatures and that Arizona voters will see a similar bill on the ballot in November, will the FDA feel it needs to take swift action to avoid things spiraling out of control?

Importantly, PBS reports that a federal law has been proposed that is similar to Right To Try. The so-called the Compassionate Freedom Choice Act of 2014, if passed, would in my view introduce chaos into the federal system of careful medical oversight. A key part of this bill reads that it would:

“Amends the Federal Food, Drug, and Cosmetic Act to declare that nothing in it or in the Public Health Service Act shall prevent or restrict, and the Food and Drug Administration shall not implement or enforce any law to prevent or restrict, the manufacture, importation, distribution, or sale of investigational drugs or devices for terminally ill patient.”

In a sense, the Colorado Right-To-Try Law as it pertains to stem cells is itself a big human experiment and it will be probably end up being fascinating and perhaps disturbing to see how this all plays out even as there are concerns over risks it brings with it. I worry it might do more harm than good. What do you think?

For other takes on Right To Try laws, see this very thorough piece by Orac and a piece supporting these kinds of laws from the Goldwater Institute.