Molecular Therapeutics Program (MTP) is a statewide drug discovery consortium supporting early-stage drug discovery research and facilitating collaborative translational research partnerships. The program helps researchers identify complementary, supporting and external partnerships for their work, as well as funding opportunities for early-stage drug discovery projects.
The recipients of 2019 Molecular Therapeutics Program awards are:
- Kevin Vaughan, PhD, University of Notre Dame
- Proposal: In Vivo Screen of Oxysterol Toxicity in a Mouse Model. The Vaughan laboratory has discovered a previously-unknown cellular mechanism of cholesterol transport that is impaired in Niemann Pick Type C (NPC) disease. Developed a cell-based screen and identified individual oxysterols as potent signaling molecules that are deficient in NPC disease.
- Gayle Gordillo, MD, Indiana University
- Proposal: The development of a Nutraceutical Product as a Treatment for Hemangiomas. Hemangiomas are endothelial cell tumors and are the most common soft tissue tumors of infancy, affecting up to 10% of all live births. The product now has FDA IND approval for first in human testing in infants with hemangiomas, supported by the National Institutes of Health.
- Ruchi Pandey, PhD, Indiana University
- Proposal: The pharmacokinetic profiling of novel and potent small molecules for inhibition of a frequently mutated oncogenic receptor tyrosine kinase. Acute myeloid leukemia (AML) is the second most common leukemia with survival rates under 30%. Pandey’s laboratory has optimized and validated two new chemical entities using in vitro assays. Plans to run in vivo efficacy studies using clinically relevant models of AML present in the lab.
- Jaipal Singh, PhD, Indiana University
- Proposal: The formulation and PK study of a novel compound for pulmonary arterial hypertension. Singh’s laboratory has validated a small modulator that metabolizes asymmetric dimethylarginine that contributes to the pathogenesis of pulmonary arterial hypertension. The modulator has shown efficacy in a rat model. Further studies on the development of oral formulation and pharmacokinetic studies are underway.