Miller School Research Team Wins First Prize in Genetic Medicine Grand Challenge
The $100,000 grant recognized the team’s therapeutic strategy to treat brain disorders, including Alzheimer’s disease and Dravet syndrome.
A University of Miami Miller School of Medicine research team won a $100,000 grant in the recent Eli Lilly and Company Genetic Medicine Grand Challenge for their novel therapeutic strategy to treat brain disorders, including Alzheimer’s disease and Dravet syndrome.
The Miller School team, one of 10 academic or university-associated startups selected from an initial pool of 300 teams to compete for the grant, was represented by graduate student Jack Stahl and Claes Wahlestedt, M.D., Ph.D., a professor of psychiatry and behavioral sciences and director of the Center for Therapeutic Innovation (CTI) at the Miller School.
Dr. Wahlestedt and Stahl presented their therapy, a novel, University of Miami-based oligonucleotide — a short, single- or double-stranded DNA or RNA molecule — and RNA technology that stands as a potential treatment for a variety brain disorders. The team focused on Alzheimer’s disease, taking into account extracellular amyloid-β (Aβ) peptide deposits and the accumulation of hyper-phosphorylated tau protein as neurofibrillary tangles. Their approach targets both amyloid and tau pathology simultaneously.
“Most common human disorders are polygenic or caused by more than one gene,” Stahl said. “We have invented a novel way to create single molecules that have the activity of two or more oligonucleotide-like drugs to simultaneously target two or more genes with just one molecule”.
The team took aim at Dravet syndrome, a severe form of childhood epilepsy caused by mutation in the SCN1A gene that leads to loss of function in the voltage-gated sodium channel subunit α Nav1, as well. But the technology may ultimately have considerably more applications for other disorders.
“While we are currently developing therapeutics for Alzheimer’s disease and Dravet syndrome, this technology can be applied to any disease, even outside the central nervous system,” Stahl said. “We are excited to further explore the therapeutic potential of this new class of molecules with the help of Eli Lilly.”
“The prize will be well used to further the new technology,” said Dr. Wahlestedt, “but the prospect of working closely with a wide range of Eli Lilly scientists is of even greater value to us.”
Tags: Alzheimer's disease, Dr. Claes Wahlestedt, grant, medical students, Psychiatry and Behavioral Sciences, student research