Takahashi team IPS cell vision paper marks major stem cell milestone

Ring the bell for a stem cell milestone.

There’s been a whole lot of commotion about the NEJM article yesterday documenting the experiences of three women with macular degeneration who were blinded by non-FDA approved stem cell eye injections of fat stem cells at a business in Florida, but in the same issue of the journal there also was some encouraging stem cell news that came in the form of essentially a mirror image of the bad news paper. We can call it the “stem cell good news-bad news” issue of NEJM.

Takahashi IPS transplant

Mandai, et al. NEJM 2017 Figure 1C

The good news was the publication of the first paper on clinical use of IPS cell-derivatives in a human patient. A big milestone. This groundbreaking manuscript comes from the pioneering team in Japan led by stem cell scholar Dr. Masayo Takahashi. I’ve written extensively in the past about the work of Takahashi and her team with IPS cells, and she received my Stem Cell Person of the Year Award back in 2014.

In the new paper they detail their data from the clinical study using sheets of retinal pigmented epithelial cells (RPEs) made from IPS cells in this case derived from the patient herself for autologous use. Remarkably in Figure 1C (above) you can see the actual transplanted RPE sheet in the eye of the patient (see dark area indicated by white arrow). The most encouraging part of this study was that the patient’s vision remained stable (rather than declining as expected) following the treatment. Was that due to the transplant? We can’t be sure.

Also, this is just a beginning as it is just one patient, but it is very exciting and represents a big milestone for the IPS cell and broader stem cell field, providing real hope for patients with vision loss along with parallel ESC-based clinical trial work as well.

This paper contrasts so much with the report from the other one in the same issue on the terrible outcomes from the stem cell clinic’s use of fat stem cells in the eye. While the use of fat stem cells themselves is highly questionable in my view for this application, the biggest differences between the two approaches is that the Takahashi team work was extremely rigorous, careful, based on extensive preclinical studies, had governmental approval, and was in essence science-based clinical medicine.

For instance, the Takahashi team was appropriately cautious with Patient 2 since the cells exhibited some genomic changes. At least in part for that reason, moving forward this clinical work will primarily focus on allogeneic use of IPS cells via an IPS cell bank being developed by Shinya Yamanaka.

We can also look to other future IPS cell-based trials coming on-line including for Parkinson’s Disease and other conditions, which are likely to be allogeneic as well in Japan, but probably autologous here in the U.S.

I love a good stem cell milestone!

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.”

Multiple filters for stem cell research at Canadian stem cell conference

By Samantha Yammine, PhD Candidate in Derek van der Kooy’s lab at the University of Toronto. See tweets live from #TMM2016 via @SamanthaZY here.

Whistler, BC, Canada.

The location of this year’s annual TMM in Whistler, BC, Canada.

Last week, 430 Canadian scientists, trainees, industry professionals, science communicators and international guests gathered in the picturesque ski town of Whistler, British Columbia for the annual Till & McCulloch meeting (TMM). This is Canada’s premier conference for stem cell research, which is co-hosted by the Centre for Commercialization of Regenerative Medicine, the Stem Cell Network, and the Ontario Institute for Regenerative Medicine.

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Cynata approval for 1st ever allo trial of IPSC-derived MSCs for GVHD

cynataCynata Therapeutics Limited has received approval from UK regulators to start a first-of-its-kind allogeneic IPSC-based trial of MSCs for graft versus host disease (GVHD). Cynata also had some big news a couple weeks back with a deal with Fujifilm.

The company is aiming to recruit 16 patients to test whether the MSCs (a type of adult stem cell) made from pluripotent stem cells created in the lab is safe and eventually whether it can aid patients facing GVHD, a potentially life threatening consequence of bone marrow/hematopoietic stem cell transplantation. There are 4 key bullet points the company released on the study:

  • “UK regulatory authority MHRA approves Phase 1 trial with Cymerus(TM) MSCs
  • World first clinical trial with allogeneic iPSC-derived product
  • Major milestone for stem cell therapeutics and regenerative medicine
  • Cements Cynata’s global leadership in second generation MSC therapeutics”

Another allogeneic IPSC study, this one in Japan and led by Masayo Takahashi, appears to be on the cusp of beginning using IPSC-derived retinal pigmented epithelial cells (RPEs) to treat macular degeneration. An earlier related autologous clinical study began with one patient receiving autologous IPSC-derived RPEs, but was halted due to regulatory changes in Japan. Also, IPSC-derived RPEs from a different patient were found to have a few mutations, which I’m still unclear as to whether had any significance.

Takahashi’s team just published a couple important papers on the allogeneic therapy reporting encouraging pre-clinical data in non-human primates where there wasn’t rejection. My sense is that their human clinical study is likely to start early in 2017.

I expect between these trials and other coming new ones we could see a half-dozen or so IPSC-based trials in the works by the end of 2017. Exciting times in the pluripotent stem cell-based clinical translation arena.

One question here with Cynata’s approach that first comes to mind relates to the question or autologous versus allogeneic therapies.

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