The Regenerative Medicine Program efforts aimed to accelerate breakthroughs in the development of stem cell-based therapies for complex diseases. The overall goal of the program was to develop resources to catalyze therapeutic use of a special type of stem cells called induced pluripotent stem cells (iPSCs). RMP consisted 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 supported distribution of induced pluripotent stem cell lines developed by the program for laboratory and clinical research.
RMP has transitioned from Common Fund support. Common Fund programs are strategic investments that achieve a set of high-impact goals within a 5-10 year timeframe. At the conclusion of each program, deliverables will transition to other sources of support or use within the scientific community.
RMP was supported by the Common Fund from 2010 to 2020.Currently, the Stem Cell Translation Laboratory (SCTL) is being supported by the National Center for Advancing Translational Sciences (NCATS). Dr. Kapil Bharti and his team at the NIH National Eye Institute are conducting the first clinical trial to test the safety of a new stem cell therapy for vision loss among dry age-related macular degeneration (AMD) patients. RMP-generated induced pluripotent stem cell lines are being managed by NINDS and distributed through RUCDR Infinite Biologics at Rutgers University.
Highlights of the Regenerative Medicine program’s major accomplishments are:
- Developed a clinical-grade, current Good Manufacturing Practice (cGMP) iPSC line and 14 research-grade iPSC lines
- Dr. Bharti and his team conducted pre-clinical research to develop a novel iPSC-based therapy to prevent blindness in animal models of retinal degeneration. Findings from this study informed the first-in-human clinical trial to treat AMD that may lead to new treatments that will prevent blindness in older individuals.
- The SCTL established a new resource that can help bring induced pluripotent stem cell technology closer to clinical applications
Please note that since the Regenerative Medicine Program is no longer supported by the Common Fund, the program website is being maintained as an archive and will not be updated on a regular basis.
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 established the Stem Cell Translation Laboratory (SCTL) within the National Center for Advancing Translational Sciences (NCATS) in 2015. SCTL worked with collaborators to address challenges that prevent translation of regenerative medicine applications from the lab to the clinic.
The SCTL pursued 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 (link is external)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. Bhart developed 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 2020, Dr. Bharti launched Phase I clinical trial to test the safety of the novel iPSC-based therapy for AMD.
Watch Dr. Bharti explain his research: https://youtu.be/k6DTj597Gv8 (link is external)
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 (link is external). View the catalogue of RMP-generated iPSC lines at https://commonfund.nih.gov/stemcells/lines.
New Approaches in Stem Cell Survival
Dr. Ilyas Singec and his team at the Stem Cell Translation Laboratory (SCTL), within the NIH National Center for Advancing Translational Sciences (NCATS), developed a small molecule cocktail to protect induced pluripotent stem cells from cell death. In preventing cell death, the cocktail has the potential to help treatment for diabetes, Parkinson’s disease, and spinal cord injury. This work was supported by RMP. Read more: https://go.usa.gov/xHmk8.
Stem Cell Therapy Gets Closer to Clinic
Dr. Kapil Bharti and his team at the National Eye Institute launches the first clinical trial to test the safety of a new stem cell therapy for vison loss among dry age-related macular degeneration (AMD) patients. The clinical trial may lead to new treatments that will prevent blindness in older individuals. This work is funded in part by our Regenerative Medicine program.