Stem-cell therapies or stem-cell-targeted treatments are being used in clinics across the country to treat a variety of diseases and conditions. In many cases, the Food and Drug Administration (FDA) has not been able to provide the oversight to prove the effectiveness or efficacy of these treatments. Fortunately, the 21st Century Cures Act, which was enacted in December2016, includes the Regenerative Medicine Initiative which will help to address this issue. Research results will help us to better understand how clinicians may use stem cells safely and effectively in therapy.
For this initiative, NIH is collaborating with the FDA to solicit applications for clinical research involving human adult stem cells. The research may include autologous and non-autologous use of stem cells, including cells derived from eligible induced pluripotent stem cells (iPSCs) to generate or supplement the evidence needed for clinical applications, including:
- submission of a pre-Investigational New Drug (IND)
- pre-Investigational New Device Exemption (IDE)
- submission of an IND/IDE application
- support of research with authorized INDs or IDEs.
NIA is particularly interested in this research area because a significant percentage of older people may potentially benefit from stem cell therapies. And, a larger number of people will benefit from knowing that well-conducted research studies support the use of stem cells in clinics.
Important things to keep in mind
◊ Studies using non-human stem cell transplantations or human embryonic or fetal stem cells are not eligible for this funding. Human adult stem cells and human iPSCs not derived from embryonic or fetal sources may be used.
◊ Research may include biologics such as growth factors and cytokines, OR biomaterials such as extracellular matrices and scaffolds that stimulate host adult stem cell growth, differentiation, and function, or otherwise directly act upon host adult stems cells may be used.
◊ Funds may also be used to help advance research to clinical trials including chemistry, manufacturing, and controls to support the production using good manufacturing practices.
◊ Set-aside funds for this initiative in the first fiscal year are limited, but will be more generous in the following three fiscal years. So, FY 2017 funding will be allotted through revision awards (also known as competing supplements) to PIs who have current NIH support through one of these funding mechanisms: R41/42, R43/44, U01, U54, UM1, UM2, UC4, U24, R01, R24, and P50. Projects can have support from NIA or other participating NIH Institutes. While only revision awards are sought for FY 2017, new applications will be accepted each year in FYs 2018, 2019, and 2020.
◊ Awards will require non-federal matching funds in at least a one-to-one ratio for the amount requested. You must show evidence of the commitment to provide these funds in your application. Matching funds can come from state and local funding not originating from federal funds, as well as investments and donations from private-sector businesses and non-profit organizations. Matching support can include cash and third-party in-kind resources such as equipment, facilities, and personnel.
Where to find the details
Check out the 11 Notices of Intent to Publish Funding Opportunity Announcements for the FY 2017 research supplements. They range from NOT-HL-17-496 to NOT-HL-17-506 and were published in the NIH Guide in the first week of April. NIA will support the following funding mechanisms under this initiative:
- R01: NOT-HL-17-504
- R41/R42: NOT-HL-17-499
- R43/R44: NOT-HL-17-497
- U01: NOT-HL-17-496
- U54: NOT-HL-17-502
The FOAs themselves will be published in the next few weeks. The deadline for applications is projected for this June, with review expected in August. We’ll also publish some Q&As about this unique initiative.
Please feel free to post your questions or comments here so we can make sure we add them to our Q&A list. In the meantime, keep an eye out for the FOAs, coming soon!