10 finalists for Stem Cell Person of the Year 2016

scpoy-2016-finalistsThe voting on the 20 nominees for Stem Cell Person of the Year finished at midnight last night.

After more than 1,000 votes, we have the top 10 finalists.

These ten are some of the most remarkable people in the world of stem cells today. They have all had great impact, but in very diverse ways.

They run the spectrum from patient advocates to the President of CIRM to a host of top stem cell researchers. Patient advocates Ted Harada and Judy Roberson have the top two spots in terms of votes.

Now I have the tough task of picking just one winner as the Stem Cell Person of the Year. The awardee wins international recognition as the top outside the box thinker and positive impactor of the year and a $2,000 prize. You can read more about all the 20 people who were nominated here.

Vote now for your pick for Stem Cell Person of the Year 2016

Vote on your pick for the top stem cell outside the box thinker and positive impactor in 2016 from the 20 choices below. The top 10 vote getters will be finalists from which I will have the tough task of picking the one winner as Stem Cell Person of the Year along with the $2,000 prize and recognition.

You can vote once per day. The voting closes in 10 days on December 15th at 11:59pm Pacific Time. Read more about the 20 nominees here.

20 Nominees for Stem Cell Person of the Year 2016 Award

stem-cell-person-of-the-year-awardI received a score of great nominations for the Stem Cell Person of the Year 2016 Award and have briefly described the twenty nominees below. The point of the award is to honor the top positive stem cell leader who specifically thinks outside the box and takes risks.

I’ve started an on-line vote where you can vote once per day for your favorite nominee(s) for Stem Cell Person of the Year. The top half or so of nominees getting the most votes will be the finalists from which I will choose the final winner, who receives the $2,000 prize and international recognition as a global leader in the stem cell and regenerative medicine field.

Past winners of the Stem Cell Person of the Year Award include the following:

  • Top stem cell scientist Jeanne Loring in 2015.
  • Pioneering vision and pluripotent stem cell clinical researcher, Masayo Takahashi in 2014.
  • Neural stem cell scientist and very effective Italian politician Elena Cattaneo in 2013.
  • Stem cell patient advocate Roman Reed in 2012.

Here are the 2016 nominees in alphabetical order by first name with some description of who they are and a bit of the words from the person(s) who nominated them in some cases. Where I could find a link to websites describing their accomplishments, I have provided those.

Amy Wagers, Professor at Harvard. She has a long track record of cutting edge research on stem cells including recently very provocative work on the role of stem cells in human aging and approaches to reversing aging.

Arnold Caplan, Professor at Case Western Reserve. He is often considered the “father” of the mesenchymal stem/stromal cell (medicinal signaling cell) field and has done important research on MSCs over many years.

Connie Eaves, Distinguished Investigator at Terry Fox Laboratory at UBC. She has a remarkable track record of innovative research on stem cells including both normal and cancer stem cells and a reputation as a fantastic mentor and leader in the field more generally. “Brilliant scientist with unmatched piercing view of science”.

Hiroshi Nagashima, Professor at Meiji University, Tokyo. “A true translational scientist (with a wicked sense of humor!)” He works in part on cloning technology and could revolutionize organ transplantation approaches leading to huge impact.

Jim Gass. Jim is a patient who suffered a stroke and then sought stem cell treatments to try to reverse some of the damage. Somewhere along the lines, one or more of the unproven stem cell therapies he received caused him to develop a spinal tumor. He had the courage to go public with his story and describe his experiences, potentially risking litigation. “A gutsy man who has prevented others from getting injured.”

John Pimanda, Associate Professor of Medicine and Stem Cell Biology, UNSW Australia. He researches transcriptional regulation of adult stem cells and now the use of fat stem cells for spine injury.

Judy Roberson. She is a tireless Huntington’s Disease (HD) advocate, always working to make a positive difference. “She is a straight shooter who will tell you what she thinks and work to make it a reality.”

Jun Takahashi. He is a Professor at CiRA and pluripotent stem cell biology researcher. Jun has done pioneering IPSC research and is working to start a very exciting Parkinson’s Disease clinical trial using IPSC in Japan.

Margaret Goodell, Professor at Baylor College of Medicine. She is an internationally respected scholar in the stem cell field. She conducts cool, innovative research on transcriptional and epigenetic regulation of hematopoietic stem cells and how this goes awry in leukemias.

Mike West. Often mentioned as one of the founders of the regenerative medicine field, he is the leader of BioTime and is a thought leader in the field. “Mike knows all about taking risks in regenerative medicine leading to big, positive leaps forward.”

Nissim Benvenisty, Professor of Genetics at the Hebrew University of Jerusalem. He is a super-prolific, long-time stem cell researcher. His latest work this year was on revolutionary production of haploid ES cells.

Oliver Brustle, Professor and Director of the Institute of Reconstructive Neurobiology and Professor of Reconstructive Neurobiology at the University of Bonn Medical Center. He conducts innovative neural stem cell research and is a globally respected stem cell leader.

Randy Mills, President and CEO of CIRM. He has been a leader in stem cell biotech for years and has shaken things up at the helm of CIRM with a much more translational emphasis. “Randy has CIRM on track to meaningful clinical outcomes in a way that I cannot imagine another leader could have achieved. The outcome will change the world.”

Richard Ambinder, Johns Hopkins Hospital. Professor Ambinder has done pioneering work in the area of stem cells and viruses, including HIV, as well as stem cells for patients with hematopoietic malignancies. A scientist with a prodigious publication record of high-impact papers.

Robert Lanza. He has been a regenerative medicine leader for, what, decades? Long time scientific leader behind ACT and then its new incarnation as Ocata, which was purchased by Astellas and he leads global regenerative medicine at Astellas.”We expect something new and big from Bob at every turn”.

Sally Temple, Scientific Director, Co-Founder, and Principle Investigator at the Neural Stem Cell Institute. She is also the President of ISSCR. Scholar and innovative researcher in the stem cell field with a focus on stem cells in the brain. Past MacArthur Fellow. “One of the brightest developmental biologists in the world and a natural leader.”

Sheng Ding, Senior Investigator, Gladstone Institute of Cardiovascular Disease. Dr. Ding has done some of the most creative and impactful research in the stem cell field to date, and continues to crank out new discoveries in particular related to chemical reprogramming. He also has co-founded a number of exciting biotechs including Fate Therapeutics. “He has been a positive leader in the stem cell field, and his outside-of-the-box thinking has greatly enhanced our collective efforts to advance the field.”

Shoukhrat Mitalipov, Professor at ONPRC and OHSU. Shoukhrat is a top researcher in the stem and germ cell arenas of research including cloning and mitochondrial transfer, with cutting edge high impact papers published every year. “Fearless and one of the premier innovators in the field”.

Ted Harada (posthumous). Ted was one of the most prominent patients participating in a stem cell clinical trial ever. He fought for patients and efforts such as right to try every step along the way, and brought people together in the field. You can see his obituary and tributes here.

Theresa Liao. Powerful advocate for the use of stem cells to treat recessive dystrophic epidermolysis bullosa (RDEB). Through relentless advocacy she has made a profound difference in this area of clinical research.  “A parent and visionary patient advocate.”

New Mitalipov paper on stem cell mitochondria: challenge for IPS cell field?

A new paper from Shoukhrat Mitalipov’s lab on stem cell mitochondria points to a pattern whereby induced pluripotent stem (IPS) cells tend to have more problems if they are from older patients.

What does this paper mean for the stem cell field and could it impact more specifically the clinical applications of IPS cells?

Mitalipov mitochondrial mutations

Graphical Abstract, Kang, et al. 2016

The new paper Kang, et al is entitled “Age-Related Accumulation of Somatic Mitochondrial DNA Mutations in Adult-Derived Human iPSCs”.

This paper reminds us of the very important realities that mitochondria are key players in stem cell function and that mitochondria have their own genomes that impact that function. A lot of us don’t think about mitochondria and their genome as often as we should.

The paper came to three major scientific conclusions (this from the Highlights section of the paper and also see the graphical abstract for a visual sense of the results overall):

  • Human iPSC clones derived from elderly adults show accumulation of mtDNA mutations
  • Fewer mtDNA mutations are present in ESCs and iPSCs derived from younger adults
  • Accumulated mtDNA mutations can impact metabolic function in iPSCs

Importantly the team looked at IPS cells derived from both blood and skin cells and found that the former were less likely to have mitochondrial mutations.

This study suggests that those teams producing or working with human IPS cells (hIPSCs) should be screening the different lines for mitochondrial mutations. This excellent piece from Sara Reardon on the Mitalipov paper quotes IPS cell expert Jeanne Loring on this very point:

“It’s one of those things most of us don’t think about,” says Jeanne Loring, a stem-cell biologist at the Scripps Research Institute in La Jolla, California. Her lab is working towards using iPS cells to treat Parkinson’s disease, and Loring now plans to go back and examine the mitochondria in her cell lines. She suspects that it will be fairly easy for researchers to screen cells for use in therapies.”

Mitalipov goes further and suggests that his team’s new findings could support the use of human embryonic stem cells (hESC) derived by somatic cell nuclear transfer (SCNT) which would be expected to have mitochondria with fewer mutations. However, as Loring points out in the Reardon article, SCNT is really difficult to successfully perform and only a few labs in the world can do it at present. In that context, working with hIPSC and adding on the additional layer of mitochondrial DNA mutation screening could be more practical.

New York stem cell researcher Dieter Egli, however, is quoted that hIPSC have other differences with hESC as well such as epigenetic differences and he’s quoted in the Reardon piece, “It’s going to be very hard to find a cell line that’s perfect.”

One might reasonably ask both Egli and oneself, “What is a perfect cell line?”

In the end the best approach for use of human pluripotent stem cells of any kind is going to involve a balance between practicality of production and the potentially positive or negative traits of those cells as determined by rigorous validation screening.

With this new paper we’ve just learned more about another layer of screening that is needed. An interesting question is whether adult stem cells such as mesenchymal stromal/stem cells (MSC) also should be screened for mitochondrial mutations. They are often produced from patients who are getting up there in years. I hope that someone will publish on that too.

As to pluripotent cells, I expect that sometimes the best lines, meaning those most perfect for a given clinical application, will be hIPSC (autologous or allogeneic in some instances) and in other cases they may be hESC made from leftover IVF embryos. If SCNT-derived hESC can be more widely produced in an affordable manner and they pass validation as well then those (sometimes called NT-hESC) may also come into play clinically. So far that hasn’t happened for the SCNT cells, but it may over time.

The more types of pluripotent cells and indeed stem cells more generally including adult stem cells that we have in our clinical arsenal to help patients the better as long as the cells and any derivatives made from them have gone through vigorous validation in each case and there’s hard data to support their use in patients with a reasonable expectation of both safety and efficacy going into any particular clinical study.

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.