Therapeutic Discovery and Translation Grant Awardees

The McKnight Brain Institute is pleased to congratulate our 2014 Therapeutic Discovery and Translation Grant Awardees

The MBI Therapeutic Discovery and Translation Grants were established to support translational studies in Neuromedicine.  Two winners were selected and will each receive $75,000 for their study for one year.

“Screening for Alpha-Synuclein & Tau Aggregation Inhibitors”

Dr. Benoit Giasson, Department of Neuroscience, CTRND

Dr. Kevin Felsenstein, Department of  Neuroscience, CTRND

Neurodegenerative disorders such as, Parkinson’s disease (PD), characterized by extrapyramidal motor dysfunction, and, Alzheimer’s disease (AD), defined by dementia manifest pathological proteinaceous aggregates of α‐synuclein as Lewy bodies in PD, and microtubule-associated protein tau in the form of neurofibrillary tangles in AD. These represent the major pathological hallmarks in PD and AD, respectively. The amyloidogenic formation of both α‐synuclein and tau appear to be toxic for neuronal cells, and therefore represent an appropriate target for the identification and design of molecules that may control morphological and functional alterations leading to impairment.  The initial goals of this program are to identify compounds that may inhibit or prevent the toxic accumulation of these proteins. Longer term goals include transitioning the program from screening to a ‘hit’ to lead medicinal chemistry effort to identify new analogs with improved potency, reduced off‐target activities, and physiochemical/metabolic properties suggestive of potentially reasonable in vivo pharmacokinetics for animal testing, and eventual investigational new drug enabling studies.


“Novel Discovery Vehicles for Brain Tumors”

Dr. Ana Tari Ashizawa, Department of Neuroscience

Dr. Dennis Steindler, Department of Neurosurgery

The blood brain barrier is a major hurdle in treating malignant brain tumors like glioblastoma (GBM). To combat GBM more effectively, novel drug transport technology is needed so that enhanced concentration of therapeutic molecules can cross the blood brain barrier and exert their activity against GBM. We have engineered an innovative liposome formulation that targets multiple proteins essential to the endocytosis and transcytosis processes of brain microvascular endothelial cells. The innovative liposome was also designed to carry a high payload of nucleic acids and be internalized by GBM. In this application, we aim to demonstrate as proof-of-concept that the liposomes can safely and effectively deliver microRNA (drug cargo) to GBM. We envision developing these prototype liposomes into a pipeline technology for the delivery of other types of drug molecules to GBM, and to modify their composition for drug delivery to other types of brain tumors.