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Program Snapshot

The goal of the Regenerative Medicine Program (RMP) is to serve as a national resource for stem cell science to accelerate the development of new medical applications and cell-based therapies. The RMP is pursuing this goal through two major initiatives; a Therapeutic Challenge Award to Dr. Kapil Bharti at the National Eye Institute (NEI) and a recently established Stem Cell Translation Laboratory (SCTL) within the National Center for Advancing Translational Sciences (NCATS).

Dr. Bharti’s research focuses on developing a stem-cell based therapy to treat age-related macular degeneration, a leading cause of blindness in the elderly; and the methodological and regulatory challenges that must be overcome to move his research beyond the pilot stage toward the clinic is relevant to the scientific and clinical communities. The SCTL is designed to tackle top scientific and technological problems that currently impede therapeutic use of induced pluripotent stem cells (iPSCs) — adult cells reprogrammed to behave similarly to embryonic stem cells — and to rapidly deliver the resulting protocols, standards, data, tools and molecules to the broader scientific community.

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Program Highlights

Two-year update on RMP Therapeutic Challenge Award to Dr. Kapil Bharti
On September 18, 2015 Dr. Kapil Bharti of the National Eye Institute and his research team presented an update to his Therapeutic Challenge award to develop a stem-cell based therapy to treat age-related macular degeneration (AMD). In AMD patients Retinal Pigment Epithelium (RPE) cells become damaged and lose function. Replacement of damaged RPE cells with functional stem-cell-derived RPE cells is a viable treatment believed to restore or improve vision. Inducible pluripotent stem cells (iPSCs) are stem cells that have been reprogramed from adult cells and can develop into any cell type in the body. For clinical applications iPSCs are to be manufactured using strict guidelines called current good manufacturing practice (cGMP) to ensure quality, purity, and safety of cells. Dr. Bharti’s team has developed a streamlined manufacturing process to generate transplant-ready cGMP-grade RPE cells from iPSCs and the entire manufacturing process takes slightly under 150 days. His team is currently performing pre-clinical animal toxicity, efficacy, and transplantation device compatibility studies in preparation for submitting a phase I Investigational New Drug (IND) application with the Food and Drug Administration in 2017. Learn more about Dr. Bharti’s research.

Research articles from Dr. Bharti’s Therapeutic Challenge Award

  1. Hotaling, N.A., Bharti, K., Kriel, H., and Simon, C.G. (2015) Validated open source nanofiber diameter measurement tool. Biomaterials. 61: 327-338.
  2. Hotaling, N.A., Bharti, K., Kriel, H., and Simon, C.G. (2015) Dataset for the validation and use of DiameterJ an open source nanofiber diameter measurement tool. Data In Brief. 5: 13-22.

Updates from the RMP!

The Regenerative Medicine Program (RMP) recently launched the Stem Cell Translation Laboratory (SCTL) at NCATS and hired Dr. Ilyas Singec as its new Director – read an interview with Dr. Singec in the NIH Catalyst! More information on how intramural and extramural researchers can apply to become collaborators with the SCTL will be available in the future.

Definitions

Stem cell – Stem cells are distinguished from other cell types by two important characteristics: 1) They are unspecialized cells capable of continually renewing themselves through cell division and 2) they have the potential to develop into many different cell types of the body. Given their regenerative potential, stem cells offer new opportunities for treating diseases.
 
Adult stem cell – An unspecialized cell found among specialized cells in a tissue or organ. Adult stem cells can renew themselves and they are multipotent, meaning they have the potential to develop into a limited number of cells in the body (some or all of the specialized cell types of the tissue or organ from which they were derived).
 
Embryonic stem cell – An unspecialized cell type derived from early-stage embryos. Embryonic stem cells can renew themselves and they are pluripotent, meaning they have the potential to develop into any cell type of the body.
 
Induced pluripotent stem cell (iPSC) – An unspecialized, embryonic stem cell-like cell that has been derived from an adult cell through epigenetic reprogramming. (Epigenetics relates to cellular changes caused by external or environmental factors that switch genes on and off and affect how cells read genes instead of being caused by changes in the DNA sequence.) Thus, just like embryonic stem cells, iPSCs can also renew themselves and they are also pluripotent.

 
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