Regenerative medicine conference points to new research opportunities
January 31, 2018
Health Scientist Administrator,
Division of Aging Biology (DAB).
Among the initiatives launched under the 21st Century Cures Act, which brought us the Cancer Moonshot, the BRAIN initiative, and the All of Us precision medicine program, is the Regenerative Medicine Innovation Project (RMIP). NIH, in coordination with the Food and Drug Administration (FDA), is taking the lead on this exciting new venture, which seeks to accelerate clinical research on adult stem cells while promoting the highest standards for conducting research and protecting patient safety. RMIP offers exciting possibilities for aging research, which we are beginning to explore.
A gathering of minds
Just a few weeks ago, more than 400 experts from academia, industry, and private foundations met with staff from multiple NIH institutes and the FDA in Bethesda, Maryland, to discuss the future of adult stem cell therapies. Cochaired by National Heart, Lung, and Blood Institute Director Dr. Gary Gibbons and FDA’s Dr. Peter Marks, the gathering assessed the state of the science and the basis for developing safe and effective regenerative products targeting adult stem cells.
Importantly, the meeting resulted in plans to publish Funding Opportunity Announcements (FOAs) later this year. Similar to supplements funded last year, applicants must obtain dollar-for-dollar non-federal matching funds for these applications. The workshop agenda (PDF, 284K), speaker biographies (PDF, 2.6M), and videos of each session are available online. Based on a set of key questions, the workshop was designed to identify scientific, technical, and operational challenges and highlight strategies for enabling major advances and development of clinical applications using adult stem cells.
A focus on aging
Many cutting-edge findings relevant to the aging community were presented and discussed. For example:
Dr. Joshua Hare focused on treating frailty in older people with mesenchymal stem cell transplantation, reporting results of seven promising Phase II clinical trials.
Dr. Robert Mays reported safety and efficacy results of a randomized, double blind, placebo-controlled Phase II trial which used multipotent adult progenitor cells to treat ischemic stroke in middle aged and older adults.
Dr. Lorenz Studor described phase I trials using stem cell-based treatments for Parkinson’s disease.
Dr. Sean Savitz spoke on the importance of matching the appropriate cell types and states of maturation to particular diseases. He also mentioned the need to understand the mechanisms involved in more complex diseases such as Alzheimer’s.
Upcoming funding opportunities
The workshop recommendations have led to several planned initiatives in FY 2018. As many as 12 NIH Institutes and the FDA are supporting this effort. Three initiatives will focus on clinical trial research for nonprofit organizations and small businesses, while three others will invite applications for research that does not require a clinical trial (U01, UT1/UT2, U43/U44). The Notices of Intent to Publish are now available at:
UG3/UH3 – clinical trial required
U01 – clinical trial not allowed
U43/U44 – clinical trial required
U43/U44 – clinical trial not allowed
UT1/UT2 – clinical trial required
UT1/UT2 – clinical trial not allowed
To help would-be applicants, a list of frequently asked questionsis available on the Regenerative Medicine Innovation Project website. Please feel free to post your comments or additional questions so we can make sure we add them to the list. In the meantime, keep an eye out for these initiatives, coming soon!
Longeveron is researching treatments based on its Mesenchymal Stem Cells for a variety of aging-related diseases. In 2017, Longeveron published positive Phase I and Phase 2 clinical studies in the Journals of Gerontology that evaluated the safety and efficacy of its Mesenchymal Stem Cells in patients with Aging Frailty, a serious geriatric syndrome that can lead to other severe health conditions, such as heart disease. The company is now recruiting for an expanded Phase 2b Aging Frailty study.
Longeveron is also recruiting for a Phase 1 Alzheimer’s trial, and Phase 1 and 2 trials to evaluate the safety and efficacy of its stem cells for improving flu vaccine immune response in Aging Frailty patients. Longeveron’s MSC product is derived from the bone marrow of young, healthy adult donors.
Longeveron (www.longeveron.com) is a regenerative medicine therapy company founded in 2014. Longeveron’s goal is to provide the first of its kind biological solution for aging-related diseases, and is dedicated to developing safe cell-based therapeutics to revolutionize the aging process and improve quality of life. The company’s research focus areas Aging Frailty, the Metabolic Syndrome and Alzheimer’s Disease and gratefully acknowledges the generous support of the National Institutes of Health, the Alzheimer’s Association and Maryland Stem Cell Research Fund. Longeveron is also conducting a Phase 1 trial to study Hypoplastic Left Heart Syndrome, a rare indication that affects infants, supported through a grant from the Maryland Stem Cell Research Fund.
For more information about the clinical trials
Longeveron is sponsoring, visit ClinicalTrials.gov
or Longeveron’s website www.longeveron.com
SOURCE Longeveron LLC