Year 3/NCE

The FDA is overwhelmed with applications for approval of “regenerative medicine” therapies.  The failure of all clinical trials using fibroblasts called “mesenchymal stem cells” for heart disease therapy is particularly disappointing to regulators  One of the disadvantages of using cells like MSCs is that the cells do not engraft, and their effects, if any, are transitory.  This is also an advantage: because the cells do not remain in the body, the possibility of them become tumors is minimal.  Another approach, durable engraftment of functional stem cell-derived cell types that are intended to replace degenerated or damaged cells in the body, is likely to be far more effective, but also raises concerns because the cells will remain in the recipient for years or even decades.  Because the cells remain in the body, it is critical that the bar be raised for both safety and efficacy.  

Raising the bar for FDA approval of stem cell therapies has been our goal for the last 12 years.  

With CIRM and other funding, we have applied our expertise in genomics specifically to the problem of ensuring the safety and efficacy of durable cell therapies.  This award enabled us to develop genomic tools to be certain that the cells engrafted are the right cells for treating the disease and that every effort has been made to make sure they will not form tumors.  These genomic tools have enabled us to progress in development of an autologous dopamine neuron replacement therapy for Parkinson’s disease, using patients’ own induced pluripotent stem cells (iPSCs) for making neurons that will be effective and will be matched to the patient so they won’t be rejected.  Unfortunately, CIRM did not offer support for our clinical work, so we have obtained investment capital to move through the FDA approval process for this therapy.