On May 26, 2022, SDEE hosted an event on stem cell therapy at the Hera Hub in Sorrento Valley.  A big thank you to the sponsors that made the event and the reception possible: Felena Hanson, founder of Hera Hub, Alexandria, Morrison and Foerster, Yowa Kirin, Takeda, BDO, CBRE, Longfellow, Marsh McLennan, MUFG, Duane Morris, and Wagenknecht Law Group.

After Marilyn Ferrari, SDEE’s president, introduced the upcoming events and workshops organized by SDEE, Rajesh Ambasudhan of Allele Biotech, Thorsten Gorba of Aspen Neuroscience, and Alexey Terskikh of Stemson Therapeutics spoke about their work on stem cell therapies.

Technology is influencing all aspects of our lives. New technologies encompass everything from smart phone and speakers to manipulation of genes involved in cholesterol metabolism, personalized medicine, or regeneration of damaged cells. Cell and tissue regeneration, the topic of the event, can be accomplished with the use of stem cells.

Different kinds of stem cells have been described: embryonic, fetal, adult, and induced pluripotent stem cells (iPSC). iPSC derived from somatic cells that are harvested before damage occurs. Upon treatment with specific factors, cells convert to stem cells. They can then be then programmed to generate any type of human cell for therapeutic purposes, including Parkinson’s disease, cancer, spinal cord injury, etc. as evidenced by the fact that there are currently 20 trials using iPSC. The science is exciting not only for scientists, but also for investors. Twenty-three billion dollars have been raised in 2021 on iPSC technology, and BlueRock Therapeutics, for example, was recently acquired for over a billion dollars.

iPSC have the potential to treat any disease that requires cell replacement. Ideally, replaced cells should last for life without the need of immunosuppression, and artificial intelligence-based genomics can be used to characterize cells and predict which cell type has the highest likelihood to be functional once introduced in the patient. However, challenges include: procurement of starting material, differentiation process, manufacturing, regulatory, and reimbursement by payors. Manufacturing is a major hurdle because one needs standards, infrastructure for scaling up operations, and automation to produce cells with high yield, purity, and viability. 

Allele Biotech focuses on neurological and pancreatic diseases and plans to start clinical studies next year. Aspen Neurosciences has pioneered a personalized medicine approach using autologous iPSC to treat Parkinson’s disease. Use of iPSC for PD holds great potential, as the disease affects a large number of patients, symptoms are debilitating, yet the chronicity of the disease affords the necessary time to harvest and differentiate the few million cells that are needed. Benefits of iPSC injection in the brain should last for decades, however some trials have not been successful, possibly because cell purity was suboptimal. Stemson Therapeutics is working on hair loss and alopecia, which is devastating for many patients. iPSC can be derived from cells in the hair follicles and used to regenerate lost hair.

In addition to the three companies featured at the event, Fate Therapeutics and other companies in San Diego are working on stem cells, making San Diego a leader in this new therapeutic space.