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Harnessing the potential of stem cells: Adult cells made to become induced pluripotent stem cells (iPSCs) may be used to treat brain disorders

In 2010, the NIH Common Fund launched the NIH Center for Regenerative Medicine (NIH CRM) to serve as a national resource for stem cell science to accelerate the development of new medical applications and cell-based therapies. NIH CRM focuses on induced pluripotent stem cells (iPSCs), which are generated by coaxing adult cells into reverting back to an embryonic stem cell-like state, which then can generate a multitude of different cell types for use in therapies or screening. These cells offer the potential of therapeutic strategies using a patient’s own cells, thereby avoiding immune rejection complications typically associated with transplanted cells from other sources. From Fiscal Year 2010 to Fiscal Year 2013, the NIH CRM supported pilot projects within the NIH Intramural Research Program to bring unique resources at the NIH to bear to help translate iPSC research into the clinic. In addition, the NIH CRM has developed multiple stem cell lines, set up contracts for stem cell services and storage, developed standard consent forms, compiled the supporting protocols and standard operating procedures used to derive, culture, and differentiate stem cells into different cell types, and developed training courses. In 2014, the NIH CRM awarded a Therapeutic Challenge Award to Dr. Kapil Bharti (NEI) to move his work beyond the pilot stage toward the clinic. Dr. Bharti’s research focuses on age-related macular degeneration (AMD), a leading cause of blindness in the elderly. Dr. Bharti’s Therapeutic Challenge project will use iPSC derived retinal pigment epithelium (RPE) cells, the type of cell damaged in AMD, in preclinical efficacy and safety studies. While Dr. Barti will work towards the goal of a Phase 1 clinical trial using patient-derived cells, the methodological and regulatory challenges that must be overcome will be relevant to the broader community.


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