In a letter to all faculty, Jeffrey S. Flier, dean of Harvard Medical School, announced the formal launch of the Harvard Program in Therapeutic Science, or HiTS, a collaborative endeavor aimed at enhancing and eventually leading efforts to reinvent how we discover and evaluate therapeutic drugs and devices. Heading this effort is Peter Sorger, the Otto Krayer Professor of Systems Pharmacology.
Sorger has been a leader in the field of systems biology. His lab focuses on pathways that control life and death decisions in human cancers by incorporating both experimental and computational biology. Over the years he has pioneered approaches to modeling signal transduction at the single-cell level. In addition, Sorger cofounded the Cambridge-based systems biology biotech company Merrimack Pharmaceuticals and also Glencoe Software. In 2011, he authored a definitive report on systems pharmacology for the National Institutes of Health.
“Therapeutics has been at the forefront of my priorities as dean,” Flier said in the letter. “Over the last few years I have been keenly aware of the crisis in the pharmaceutical pipeline, how the vast investments required by traditional pharmaceutical development are not yielding needed therapies and how a deep solution to this problem will require that new concepts and models be developed within the academy. HMS is extremely well positioned to convene key entities, imagine new solutions and marshal the necessary talent and resources to address this issue—one of biomedicine’s greatest challenges.”
“We need a comprehensive approach to rethinking how we go about the most fundamental processes of drug discovery,” Sorger said. “The truth is, we simply don’t understand how most drugs work, particularly in terms of patient-to-patient variability and the emergence of resistance. In industry, timelines are necessarily too tight and programs too focused to delve deeply into some of the most intriguing therapeutic questions. In contrast, academia is equipped to study basic, open-ended questions and to partner with industry when the ideas are ready for development in actual drugs.”
The program will include both new research and education programs involving collaborations among HMS and other Harvard schools, HMS-affiliated hospitals and research institutes, the US Food and Drug Administration, the pharmaceutical industry and other Boston-area universities.
There will be four primary components of HiTS:
· The Laboratory of Systems Pharmacology is a new research facility in the Armenise building partly made possible by a recent $5 million capital grant from the Massachusetts Life Sciences Center. First announced as an idea in 2011, the lab is a multidisciplinary program exploring how drugs work in complex systems that will open this year with Sorger as director. Tim Mitchison, the Hasib Sabbagh Professor of Systems Biology at HMS, will be deputy director.
· The Therapeutics Technology Cluster will enhance existing core facilities and build new ones needed to create and test next-generation therapeutic agents, including molecules and materials designed for function rather than structure. Stephen Blacklow, head of the Department of Biological Chemistry and Molecular Pharmacology at HMS, will direct this effort. Caroline Shamu, director of the HMS Institute of Chemistry and Cell Biology, will act as assistant director.
· The Program in Regulatory Science is an education and research program focusing on the science needed to more effectively test and evaluate new medicines. The program aims to develop tools for predicting drug efficacy and toxicity in preclinical experiments and clinical trials. It will also develop new curricula for regulatory science to be available to multiple constituencies. A director will be named.
· A Therapeutics Graduate Program will award a certificate in therapeutics, initially to doctoral students in biological science within the Harvard Graduate School of Arts and Sciences and Division of Medical Sciences. Students in the program undertake internships either in industry, the clinic or a regulatory agency under the leadership of David Golan, HMS dean for graduate education. This program will combine with the extensive educational efforts in therapeutic science under Harvard Catalyst leadership.
“The systematic analysis of human genetics has identified precise molecular lesions associated with many human diseases, potentially identifying numerous new therapeutic targets,” said Blacklow. “The goal of the therapeutics technology cluster is to develop and deploy the methods needed to analyze these targets and to modulate them with proof-of-concept agents.”
Joining the leadership team is Laura Maliszewski, executive director of HiTS. Marc Kirschner, chair of the HMS Department of Systems Biology and the John Franklin Enders University Professor of Systems Biology, will serve as senior scientific advisor.
“Industry is increasingly concerned about the reliability of the academic data on new drug targets and disease mechanisms,” said Maliszewski. “In HiTS, we are addressing this by applying quantitative, statistically rigorous, computational methods to such studies. We will also attempt to improve our ability to validate therapeutic hypotheses in humans, and not simply to provide industry with partially validated ideas.”
According to Flier, “This is among our most ambitious efforts, and I believe that HMS is one of the few places in the world with the broad cross-disciplinary expertise to succeed in this area.”