Trained as a mathematician, Peter Park, AB ’94, SM ’94, SM ’00, PhD (pictured), a professor of biomedical informatics in the Blavatnik Institute at Harvard Medical School, is developing analytical methods to interpret the human genome. He and his lab colleagues recently discovered an interesting drug target, but they had neither the resources nor the expertise to pursue it. Enter the Blavatnik Therapeutics Challenge Awards (BTCA).
The Blavatnik Family Foundation provides $1 million over two years to each BTCA recipient with the goal of advancing translational therapeutics research projects that are within two years of a commercial exit. Park says his lab’s immediate goal is to develop a therapy for frontotemporal dementia, a devastating brain disorder that affects thousands of people and has no treatment.
“Our long—term goal is to develop an efficient platform to identify any genetic mutation in an individual by sequencing the person’s genome and to design a molecule to counter the effect of the mutation—to enable personalized therapeutics,” says Park.
Park says the BTCA program offers far more than funding. “The award allows us to take advantage of the expertise and resources of many people on campus and beyond.” He said his lab has benefited tremendously, for example, from its interactions with Ifat Rubin-Bejerano, PhD, the director of translational research programs at HMS, and with members of the Business Development team in the Harvard Office of Technology Development.
Explaining his BTCA project, Park says that in some patients with frontotemporal dementia and other neurodegenerative diseases, an important protein called progranulin is not produced in sufficient quantities due to a mutation in the DNA. Modulating the level of this protein in the brain has been particularly difficult because it is hard to deliver a drug to the brain.
“With bioinformatic analysis of genomic data, we found a way to increase the amount of protein by targeting the specific genetic defect with a molecule that we designed. Called ‘antisense oligonucleotide,’ this relatively new class of molecules can be delivered to the brain effectively. Our laboratory experiments have validated our computational predictions, and we are now ready to perform animal studies,” Park says.
The inaugural cycle of the BTCA program was completed successfully in 2020, and a request for proposals was sent out earlier this year for the 2021 cycle. All faculty at the rank of assistant, associate, or full professor based at HMS or its affiliated hospitals and research institutions are eligible to apply as lead principal investigator for the awards. Funding for the 2021 projects is expected to start in late summer, and additional program cycles are anticipated in 2022 and 2023.
The award allows us to take advantage of the expertise and resources of many people on campus and beyond.
“Our hope and expectation is that these awards will accelerate the translation of scientific discoveries into therapies to improve health and alleviate suffering,” says Len Blavatnik, MBA ’89, head of the Blavatnik Family Foundation and a member of the HMS Board of Fellows. “I look forward to following the continued progress of Dr. Park and the other inaugural award winners as they address a diverse range of medical conditions—frontotemporal dementia, Type 1 diabetes, asthma, cancer-associated blood clots, and a rare congenital immune disease.”
Blavatnik Therapeutics Challenge Awards
The BTCA program is part of the Blavatnik Family Foundation’s $200 million commitment to HMS, which has bolstered new cross-disciplinary collaborations, research programs, and essential infrastructure in support of the School’s mission to transform human health through the translation of scientific discoveries into treatments and cures. Administered by HMS, the BTCA program aims to accelerate therapeutics research across the School and its affiliated hospitals, help investigators navigate the intricacies of intellectual property development and licensing, and spur the creation of new companies. The five inaugural BTCA projects listed below are led by principal investigators spanning four institutions, demonstrating the broad reach of the program across the Harvard medical community.