Dr. Jae Lee, Professor, Department of Neurological Surgery and The Miami Project received outstanding news that his RM1 Interdisciplinary Team Science Grant will be funded by the National Institute of Neurological Disorders and Stroke (NINDS) which is a part of the U.S. National Institutes of Health (NIH). The proposal entitled “Targeting cell-type specific disease phenotypes to promote CNS repair” is a collaboration between Drs. Nagi Ayad at Georgetown (cancer biology), KiBum Lee at Rutgers (nanobiotechnology), and Jae Lee at UM (spinal cord injury). The project will develop a novel drug discovery platform that uses single cell RNAseq datasets to identify compounds that can potentially reverse cell-type specific disease signatures and use advanced drug delivery systems to target these specific cell types at the spinal cord injury site.
Despite decades of intensive research, there are currently no disease-modifying therapies to treat spinal cord injury. One major reason for this dire unmet need is the heterogeneity of the cells that comprise the injury site. The cell types and their cellular states vary widely depending on their location around the injury site as well as the time after injury. Therapeutic molecules that target one cell type may be contraindicated for another cell type, thereby masking any potential beneficial effects. Current treatment strategies largely ignore this problem. Dr. Lee is tackling this important issue through this recently awarded Interdisciplinary Team Science grant. This collaboration will use a novel drug discovery platform that analyzes the genetic signature of every cell type known to exist at the spinal cord injury site to identify compounds that are predicted to reverse the disease signature of specific cell types.
“Using advanced sequencing technologies, we have been able to obtain the gene expression profiles of every cell type that exists at the spinal cord injury site. Now using a novel bioinformatic platform developed by Dr. Nagi Ayad, we have the opportunity to leverage this large dataset for drug development purposes,” said Dr. Jae Lee. He continued, “Everyone recognizes that treating spinal cord injury is going to take a combinatorial approach, so we are going to combine the expertise of three laboratories that span three completely different fields to take a novel approach to developing therapeutics for spinal cord injury”.
Identifying a cell-type specific drug is only part of the solution, another challenge is delivering that drug to the intended cell target, and this is where Dr. KiBum Lee will use his decades of experience to develop an advanced drug delivery system capable of highly efficient cell-type targeted delivery with stimuli-responsive drug release at the spinal cord injury site. This RM1 Interdisciplinary Team Science Grant, awarded by the NINDS, will be for $6 million over 5 years.