Stepping on the gas: accelerating promising life science technologies
With news that might be the envy of NASCAR drivers, Chenghua Gu and her colleagues have an opportunity to turbocharge their efforts to deliver small molecules past the blood-brain barrier. That opportunity came in the form of a Development Grant from Harvard’s Blavatnik Biomedical Accelerator, one of 10 awards made in 2014 to researchers across the University whose work holds promise for new therapies.
Gu, an associate professor in the Department of Neurobiology at HMS, has been focused on the molecular mechanisms that determine how neural and vascular networks interact. Gu said that because “neurovascular biology is a relatively young field and very little is currently known. Characterizing these interactions could provide important tools with far-reaching applications in the understanding of diseases of the brain and their potential treatment.”
And, as things turned out, at least one big surprise of immense potential. In studying the blood-brain barrier, Gu and her colleagues have identified a specific transmembrane protein, Mfsd2a, which plays a major role in regulating what can and what cannot cross the barrier. “So far,” Gu said, “this is the first molecule found to specifically suppress trancytosis in brain endothelial cells.”
I knew that corporate leaders and private investors would be very interested in finding pathways across the barrier, but I didn’t know how to get their attention. Michal and Curtis convinced me that our discovery of Mfsd2a is exactly the kind of research that the Blavatnik Biomedical Accelerator grants are designed for.
Associate Professor of Neurobiology, HMS
In studies using mice with Mfsda2 genetically knocked out, Gu and her colleagues observed leaks in the blood-brain barrier. That observation led in turn to a great many questions about the fundamental constituents of the blood-brain barrier and the need for better technology to study it.
By blocking this protein and thereby allowing therapeutic agents to cross the barrier, Gu believes there is tremendous potential for effective treatments for a range of central nervous system diseases and disorders, from Alzheimer’s disease to brain tumors. Michal Preminger, Executive Director of the Medical School branch of Harvard’s Office of Technology Development (OTD) and Curtis Keith, Chief Scientific Officer of the Blavantik Biomedical Accelerator, believe it too.
“I knew that corporate leaders and private investors would be very interested in finding pathways across the barrier,” Gu said, “but I didn’t know how to get their attention. Michal and Curtis convinced me that our discovery of Mfsd2a is exactly the kind of research that the Blavatnik Biomedical Accelerator grants are designed for,” Gu said, “and we were excited by the prospect of moving forward on these studies.”
A Blavatnik Accelerator grant can make a huge difference [when] the potential is clear but the technology is too early and additional resources and capabilities outside the university are needed to advance it to a partnerable stage.
OTD Chief Scientific Officer of the Blavantik Biomedical Accelerator
These grants are not, however, just about the funding. The OTD team brings a different kind of expertise to research, their business experience and their industry connections. “Candid feedback from our contacts in relevant industries are critical at this stage of research,” Preminger said. “As we started interacting with industry contacts and potential investors, it became clear that further work is required in order to attract commercial partners.”
Keith describes Gu’s research as “the perfect setting where a Blavatnik Accelerator grant can make a huge difference. The potential is clear but the technology is too early and additional resources and capabilities outside the university are needed to advance it to a partnerable stage. We were convinced that this would be a good investment.”
The nine members of the Accelerator Advisory Committee were convinced as well. Earlier this year, they awarded Gu with a two-year Development Grant to do just that. This level of grant, between $200,000 and $300,000 over one to two years, is intended to support efforts that may reasonably be expected to result in partnerable technology within two years.
The accelerator concept is intended to actively search for scientists across the University who have these early-stage discoveries, to provide them with funding and to match them with advisors and mentors who have been through the whole process themselves.
Senior Associate Provost, OTD Chief Technology Development Officer
Isaac Kohlberg, Senior Associate Provost and OTD’s Chief Technology Development Officer, said that “many scientific discoveries are at too early a stage to be considered viable as a business venture. I call it the ‘development gap’ and our mandate is to find the almost-but-not-quite-ready innovations where we can bridge that gap.”
To that end, Kohlberg says, “the accelerator concept is intended to actively search for scientists across the University who have these early-stage discoveries, to provide them with funding and to match them with advisors and mentors who have been through the whole process themselves.”
Harvard’s biomedical accelerator was launched in 2007 with $10,000,000 in philanthropic support. Half of that amount came from the Blavatnik Family Foundation, headed by HBS alumnus Len Blavatnik. With an engineering background and an entrepreneurial spirit, Blavatnik liked what he saw in the results of the early accelerator grants. In 2013, the Blavatnik Family Foundation made a new commitment of $35,000,000 to the Accelerator.
In addition to the Development Grants, the Blavatnik Biomedical Accelerator also makes Pilot Grants, awards of up to $100,000, intended to support proof-of-concept activities that (if successful) would establish a basis for a subsequent Development Grant proposal. David Sinclair, Professor of Genetics, is one recipient of a 2014 Pilot Grants.
Our Blavatnik Accelerator grant will allow us to validate which of these genes have therapeutic relevance. We anticipate finding leads for development of peptides that can be used to treat metabolic, neurological and orphan diseases.
Professor of Genetics, HMS
Sinclair’s research has looked at some of the technical challenges to peptide hormone drug research. In response to those challenges, his team developed new hardware and software – including mini-supercomputers based on high-end graphics processors used in the gaming industry – that has allowed his group to identify hundreds of new peptide-coding genes in the human genome.
“Our Blavatnik Accelerator grant will allow us to validate which of these genes have therapeutic relevance,” Sinclair said. “We anticipate finding leads for development of peptides that can be used to treat metabolic, neurological and orphan diseases.”
Thanks to Blavatnik Foundation support, grants awarded this year total $1.6 million. The Blavatnik gift was not, however, made to establish an endowed fund. The Blavatnik Biomedical Accelerator’s long-term sustainability will rely on a share of revenue realized when Accelerator-funded research leads to commercial success.
“It’s a good bet, “ Kohlberg said. “Of the projects funded between 2008 and 2014, half of them are already in partnerships with industry. We want to keep our foot on the gas.”