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Non-Commercial Organizations Developing iPSC Therapies

iPSC therapies academic and government

There are diverse and ever-growing number of commercial entities supporting the development of induced pluripotent stem cell (iPSC) therapies. However, there are also government and academic entities who are pursuing therapeutic progress with the cell type.

The most notable institutions working toward this goal are:

  1. RIKEN Center in Kobe, Japan
  2. Kyoto University in Kyoto, Japan
  3. California Institute for Regenerative Medicine (CIRM) in California, USA

Each of these important institutions and their contributions to the development of new iPSC therapies is presented below.

RIKEN 

In 2013, Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe, Japan, initiated the first ever clinical research trial involving transplant of iPSCs into humans. Dr. Takahashi and her team are investigating the safety of iPSC-derived cell sheets in patients with wet-type age-related macular degeneration.  While the trial was initiated in 2013, and production of iPSCs from patients began at that time, it was not until August of 2014 that the first patient, a Japanese woman, was implanted with retinal tissue generated using iPSCs derived from her own skin cells.  A team of three eye specialists, led by Yasuo Kurimoto of the Kobe City Medical Center General Hospital, implanted a 1.3 by 3.0mm sheet of iPSC-derived retinal pigment epithelium cells into the patient’s retina.[1]

Preliminary results are indicating positive results for the participants in this iPSC clinical trial.  As the first site to ever initiate an iPSC trial in humans, the RIKEN Center deserves recognition.

Kyoto University

As mentioned previously, research and experimentation using mouse cells by Shinya Yamanaka’s lab at Kyoto University in Japan was the first instance in which there was successful generation of iPSCs.

Current research underway at Kyoto University includes differentiation of iPSCs into dopamine-releasing neurons for transplantation into patients who are afflicted with Parkinson’s disease, a trial which may begin as early as 2016.  University researchers are also working on generating a formulation of platelets that will assist with blood clotting.  Dr. Shinya Yamanaka, who is credited with discovering iPSCs in 2006, and who shared the 2012 Nobel Prize in Medicine for the discovery, leads the existing iPSC research center at Kyoto University.

Furthermore, Dr. Shinya Yamanaka, one of the founders of iPSC technology, will be heading an iPSC therapy center scheduled to open at the Kyoto University Hospital in Kyoto, Japan in 2019.[2]  As such, Kyoto University is doing a great deal to push forward progress in the area of iPSC therapy development.

California Institute for Regenerative Medicine

Well known in the stem cell sector, the California Institute for Regenerative Medicine (CIRM), an organization tasked with deploying $3 billion dollars in California tax money to support the translation of stem cell research into clinical therapies, has increasingly been favorable toward funding iPSC research projects with a clinical (“translational”) focus.

In one example, the Parkinson’s Institute was awarded $6.5 million to support four separate research projects focusing on development of patient-specific iPSCs from individuals with Parkinson’s disease.[3]  Within a brief period of time, CIRM awarded $3 million to the Cedar-Sinai Medical Center for derivation of iPSCs from patients with inherited nerve disease and for research into the feasibility of transplanting these cells back into patients after genetic corrections have been applied.[4]  $1.3 million was next awarded to Stanford University to allow for creation of cardiomyocytes from iPSCs that can be used to explore causes of cardiovascular disease.[5]  Clearly, CIRM’s favorability toward funding iPSC research is gathering momentum.

In another example, the Coriell Institute for Medical Research and Cellular Dynamics International (CDI) announced receiving two multi-million dollar grant awards from CIRM in on March of 2013.   Specifically, CIRM awarded CDI $16 million for the purpose of creating three induced pluripotent stem cell lines derived from 3,000 healthy and diseased donors using the episomal (“footprint-free”) reprogramming method first developed by CDI.[6]  Within this grant funded research, tissue samples will be taken from patients with Alzheimer’s disease, autism spectrum disorders, liver diseases, cardiovascular diseases, neurodevelopmental disabilities such as cerebral palsy and infantile epilepsy, diseases of the eye, and respiratory diseases, to allow for further research into these conditions.[7]

Furthermore, CIRM awarded the Coriell Institute nearly $10 million to set up and biobank the iPSC lines, of which CDI will be the primary subcontractor.  Under the grant terms, Coriell Institute will establish a biorepository with proven methods for managing sample collection and tracking, safe storage, and capabilities for worldwide distribution of iPSCs generated by CDI.”[8]

Grant Funding by CIRM for iPSC Production & Biobanking

Grant Recipient Amount of Grant Funding by CIRM
Cellular Dynamics International (Now Owned by Fujifilm Holdings) $16 Million
Coriell Institute for Medical Research $10 Million
TOTAL = $26 Million

Induced pluripotent stem cell lines can be generated from any living being and reprogrammed into any cell type, making these cells a powerful tool for studying genetic variation between individuals, understanding disease, and personalizing medicine.

Within the context of this CIRM-funded grant, patient samples will be primarily acquired through doctor’s office blood draws, as Cellular Dynamics International (now owned by Fujifilm Holdings) pioneered the method to create iPSCs from blood.  The importance of this CIRM-funded grant is that it will create the world’s largest human iPSC bank.

To learn more about expanding opportunities for iPSCs, view the “Global Induced Pluripotent Stem Cell Industry Report.”

Footnotes
[1]  Cyranoski, David. ‘Japanese Woman Is First Recipient Of Next-Generation Stem Cells’. Nature (2014): n. pag. Web. 6 Mar. 2015.
[2] The Japan Times,. ‘Kyoto University Hospital To Open Ips Cell Therapy Center In 2019 | The Japan Times’. N.p., 2015. Web. 7 Mar. 2015.
[3] Cirm.ca.gov,. ‘The Parkinson’s Institute | California’s Stem Cell Agency’. N.p., 2015. Web. 7 Mar. 2015.
[4] Cirm.ca.gov,. ‘Human Ipsc Modeling And Therapeutics For Degenerative Peripheral Nerve Disease | California’s Stem Cell Agency’. N.p., 2015. Web. 7 Mar. 2015.
[5] Cirm.ca.gov,. ‘Tissue Collection For Accelerating Ipsc Research In Cardiovascular Diseases | California’s Stem Cell Agency’. N.p., 2015. Web. 7 Mar. 2015.
[6] Cellular Dynamics International (2013). Cellular Dynamics and Coriell Institute for Medical Research Awarded Muli-Million Dollar Grants from California Institue of Regenerative Medicine to Manufacture and Bank Stem Cell Lines [Press Release]. March 21, 2013.
[7] Ibid.
[8] Coriell Institute for Medical Research (2013). Coriell Awarded $10M Induced Pluripotent Stem Cell Grant [Press Release]. March 21, 2013.

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