Top 10 stem cell stories of the year 2015

liver organoidWhat were the top 10 stem cell stories of the year?

What were the biggest news and science headlines in the stem cell field in 2015, whether good or bad?

Below are mine. What are your top 10 stem cell stories of the past year? Let us know in the comments.

  • Organoids explode. Stem cell-based mini-organs (organoids) have exploded in terms of impact in the last couple years. 2015 had many examples of these super cool human organs in a dish made from stem cells. Organoid technology has huge potential for teaching us about human organ development and function as well as disease pathogenesis and treatment. I recommend this interview by Alexey with organoid pioneer Madeline Lancaster for more on this exciting trend.
  • Clinical trials pile up. More good news. The number of real, promising stem cell clinical trials continues to build. This is fantastic and shows the continuing maturation of the stem cell/regeneration medicine/cell therapy fields. Think about where things were 5-10 years ago with stem cell clinical trials and you see how much progress has been made.
  • Sale of CDI and Ocata to Japanese pharma. Japan continues to show leadership in commercializing stem cells and regenerative medicine, and this in part manifested in 2015 with the big-time purchases of two American stem cell biotechs by Japanese pharma companies. Does the nation of a biotech company matter in this day and age? Are pharma acquisitions of small stem cell biotech good news or bad or some combo of the two? We can expect more corporate acquisitions in the stem cell arena in the coming year. Stay tuned for my top predictions for the stem cell field in 2016.
  • Stopping of IPS cell clinical study in Japan with broader regulatory switch there to allogeneic IPS cell focus. The pioneering macular degeneration clinical study using IPS cells in Japan was stopped this year and now a second IPS cell study (Parkinson’s Disease) there has been delayed. Regulatory changes now mandate that IPS cell clinical work be done with allogeneic cells. This change still remains somewhat of a mystery given the big potential of IPS cells on the patient-specific front using an autologous approach. We’ll hear a lot more about this in 2016. Another regulatory change in Japan is the move to allow charging of patients for clinical trial participation. I’m not a fan of this change to put it mildly.
  • CRISPR. Of course CRISPR is not specific to stem cells, but it opens new doors to understanding stem cell biology and development, particularly in the human research sphere. The genetic research on human stem cells that has opened up because of CRISPR is very exciting.
  • Human genetic modification on the table. Related to the above, gene editing of human pluripotent stem and germ cells has big ethical and social implications. The potential future production of genetically modified human beings via primordial germ cell or pluripotent stem cell editing raises big, thorny questions. Whether we are talking about genetic modification of somatic cells for therapy or production of actual designer babies, this topic was a hot one in 2015. For more, check out my new book, GMO Sapiens.
  • Stem cell clinics continued to bloom. From Internet searches to patient contact, the data out there are consistent with a continued rapid growth of stem cell clinics pitching at best unapproved stem cell interventions to at worst stem cell snake oil.
  • FDA action absent still on predatory clinics. For two years the FDA has practically speaking done nothing to address the exploding problem of stem cell clinics (see above) selling unapproved stem cell “treatments” to tens of thousands of patients in the US. We cannot forget about stem cell tourism, but the stem cell clinic problem in the US is massive and growing, and only made worse by an FDA that appears to either have mixed feelings on it or simply be moving in slow motion. Something’s got to give on this and I thought that would happen in 2015. Maybe it will in 2016.
  • Celebrities as advertising for stem cell clinics. Also in the dubious sphere we saw in 2015 an acceleration of celebs being the top drivers of patient traffic to stem cell clinics. Gordie Howe made the biggest news, but then there was Bart Starr and many others. For every famous person who puts themselves at risk or who is put at risk by their families looking for hope from stem cells, how many ordinary people follow suit and how many people will lose precious savings and potentially be hurt?
  • CIRM 2.0 picks up pace. Ending with some good news, the new incarnation of The California Institute for Regenerative Medicine (CIRM) as CIRM 2.0 (a name I admittedly enjoy saying as I coined the term) continued to build momentum in 2015. CIRM 2.0 has a distinctly clinical focus. The agency is picking up the pace on issuing RFAs and new funding. 

Brief quote from Bob Lanza on Ocata acquisition by Astellas

Robert-LanzaI asked Ocata CSO, Bob Lanza, for a comment on the big news of the acquisition of the company by Astellas.

Here’s what he had to say:

“I can say that this is an exciting time for regenerative medicine.  The merger should greatly accelerate the pace of discovery, and, I hope, lead to treatments for a long list of diseases.”

The day after hearing the news, I myself feel a bit more optimistic that Astellas will continue this important work and help patients with vision impairment as well as other diseases.

Ocata bought by Astellas of Japan: initial perspectives on end of an era

AstellasBombshell news in the stem cell field as Ocata Therapeutics (OCAT; formerly Advanced Cell Technology or ACT) is reportedly to be acquired by Astellas Pharma, Inc.

The offer for purchase of OCAT will be $8.50/share or almost $380 million. For more details see this detailed PDF from Astellas.

This quote on the deal form Ocata:

“Paul Wotton, Ph.D., President and CEO, Ocata said, “I am impressed by the vision and commitment of Astellas and believe that with their global resources behind our regenerative platform, patients suffering from debilitating diseases like AMD and SMD will soon benefit from having access to regenerative medicine.”

What will this mean for the scientists at Ocata?

What role might Dr. Robert Lanza, CSO of Ocata, play after the deal?

Ocata was founded 21 years ago as ACT and despite the recent name change to Ocata, it has largely been the same company. With the purchase by Astellas, that era is over and so begins a new one.

My initial reaction is that this isn’t reflective of the full value of Ocata. It’s disappointing and creates uncertainty about the future of Ocata’s pipeline.

At the same time I’ve had this feeling for quite some time that Ocata would be acquired in coming years at some point. That time is now and a bit sooner than I had thought.

Over at Investor Stem Cell, which heavily covers Ocata, the reaction has been negative to the deal.

Note: I have a very small, long-term position in OCAT.

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.

New UK trial & Ocata give reason for stem cell hope on vision impairment

Peter Coffey Lyndon Da Cruz

Image from Catapult

We’ve been hearing a lot this week about an important new clinical study for macular degeneration in the UK. This team is using retinal pigmented epithelial cells (RPEs) made from embryonic stem cells (ESC). They are now testing safety.

The new work in the UK is Moorfields Eye Hospital is a great addition to the growing list of stem cell clinical studies and provides real hope for those dealing with macular degeneration.

In their PR on the study, part of the  London Project to Cure Blindness this is rightly described as a “major milestone” for the project. The co-leaders, Peter Coffey and surgeon Lyndon Da Cruz (pictured above), will guide this work.Pfizer is also part of the team for this study. I’m curious to see how it progresses and hopeful on the outcome. 

There’s real reason for excitement in this area more broadly and that context is important to include, but many newspaper articles haven’t.

For instance, Ocata Therapeutics is doing very similar clinical work and is already years into its FDA-approved clinical trials for macular degeneration using RPEs made from ESC. So far that work has proceeded really well without safety concerns and with hints of efficacy. Also the clinical study in Japan for macular degeneration using IPS cells, while at present on hold, will likely start up again in a new iteration later this year or more likely in early 2016.

Disclosure: I have a very small, long-term position in Ocata stock.