The Regenerative Medicine Program (RMP) started in 2010 and is developing resources to catalyze therapeutic use of a special type of stem cells called induced pluripotent stem cells (iPSCs). RMP consists of two initiatives: (1) the Stem Cell Translation Laboratory (SCTL), headed by Dr. Ilyas Singec, within the National Center for Advancing Translational Sciences (NCATS); and (2) a Therapeutic Challenge Award to Dr. Kapil Bharti at the National Eye Institute (NEI). RMP also supports distribution of induced pluripotent stem cell lines developed by the program for laboratory and clinical research. Despite the potential benefits of iPSC technology, there are some limitations. These include the complicated process required to generate patient-specific, iPSCs safe for human use. The Regenerative Medicine Program is working to accelerate breakthroughs in the development of stem cell-based therapies for complex diseases.
RMP’s Stem Cell Translation Laboratory (SCTL) at the National Center for Advancing Translational Sciences (NCATS)
Induced pluripotent stem cells (iPSCs) are cells derived from patient tissues and transformed into a stem cell-like state where they can become any cell type in the body. The development of iPSC technology opened unique opportunities in regenerative medicine, disease modeling, and drug discovery. Despite the potential benefits of iPSC technology, there are some limitations. These include the complicated process required to generate patient-specific, iPSCs safe for human use. To help overcome this limitation, the NIH Common Fund supported establishment of the Stem Cell Translation Laboratory (SCTL) within the National Center for Advancing Translational Sciences (NCATS) in 2015. Currently, SCTL is working with collaborators to address challenges that prevent translation of regenerative medicine applications from the lab to the clinic.
The SCTL is pursing the following goals to overcome iPSC technical issues that prevent translation:
1. Establish detailed quality control (QC) standards to define human pluripotency and differentiated cell types
2. Develop methods to assess heterogeneity in cultured cells derived from iPSCs
3. Develop standardized methods to produce mature cells meeting the QC standards above
4. Discover, validate, and disseminate small molecule reagents to replace expensive recombinant proteins, xenogenic material, and undefined media components in cell differentiation protocols
Take a virtual tour of the SCTL
RMP’s Therapeutic Challenge Award
In 2014, Dr. Kapil Bharti at the National Eye Institute (NEI) received a four-year Therapeutic Challenge Award to move his research beyond the pilot stage toward the clinical use. Dr. Bharti is developing a stem-cell based therapy to treat age-related macular degeneration (AMD), a leading cause of blindness in the elderly. Since receiving the award, Dr. Bharti and his research team developed a novel iPSC-based therapy to prevent blindness in animal models of retinal degeneration. In 2019, Dr. Bharti is expected to submit an Investigational New Drug (IND) application with the U.S. Food and Drug Administration to test the safety of the novel iPSC-based therapy in a Phase I clinical trial for AMD.
Watch Dr. Bharti explain his research: https://youtu.be/k6DTj597Gv8
RMP-Generated Induced Pluripotent Stem Cell (iPSC) Lines
Early in the program, RMP developed a clinical-grade iPSC line that meets current good manufacturing practices (cGMPs) and 14 research-grade iPSC lines to help translate iPSC-based approaches into clinical applications. These resources are available to the scientific community through RUCDR Infinite Biologics at Rutgers University. View the catalogue of RMP-generated iPSC lines at https://commonfund.nih.gov/stemcells/lines.
This page last reviewed on July 31, 2019