This innovative approach will leverage data that already has been collected on these molecular compounds as well as the collective power of researchers across the nation.
“Such visionary thinking is imperative if we want to take bold action to speed up drug development,” Collins said.
Some compounds do not prove effective for the specific use for which they were developed. However, if additional research is conducted, some of these existing compounds may succeed for a different therapeutic use. A prime example is the drug azidothymidine (AZT), which failed to show efficacy against cancer, but was later found to be the first drug effective against HIV, the virus that causes AIDS.
HHS, NIH and pharmaceutical partner leadership respond to journalists’ questions about NCATS’ new therapeutic discoveries program. They are (from l) Sebelius; NIH director Dr. Francis Collins; Michael Manganiello, patient representative; Rod Mackenzie, Pfizer Inc.; Don Frail, AstraZeneca; Jan Lundberg, Lilly; and NCATS acting deputy director Dr. Kathy Hudson.
Photos: Ernie Branson
The repurposing of AZT gave patient advocate Michael Manganiello a fighting chance after he was diagnosed with HIV. “A drug rescued from obscurity 25 years ago is the reason I can say that AIDS-related complications will most likely not be the cause of my death,” he said. “What failed in one purpose may hold great promise for another.”
The compounds for this new NIH program, called Discovering New Therapeutic Uses for Existing Molecules, have undergone significant research and development by industry, including safety testing in humans, making them prime candidates for further development. Using compounds that have previously been tested in humans provides a strong starting point for scientists and permits the process to move more rapidly.
“It’s a win-win-win,” Collins said. “It’s a win for investigators who gain access to these compounds, for companies whose compounds get a new chance and most of all for patients, whose hopes for the discovery of new treatments are significantly advanced through this initiative.”
In fiscal year 2013, NCATS will provide up to $20 million to fund 2- to 3-year cooperative research grants for pre-clinical and clinical feasibility studies to test more than 20 compounds from industry partners for their effectiveness against a variety of diseases and conditions. For their part, the companies will provide successful applicants with access to the compounds and related data.
“We are committed to these compounds and we’re encouraging the investigators to publish their work, which was part of the agreements. Our involvement is a reconfirmation of our commitment to open innovation strategies,” said Don Frail, vice president of science within the new opportunities innovative medicines unit at AstraZeneca.
The pilot program incorporates innovative template agreements designed to streamline the legal and administrative process for partnering across multiple organizations. These template agreements reduce time and effort as well as enable greater participation than traditional partnerships.
“We got involved in this effort quickly in part because we wanted to set an example that we have to do things faster and not get caught up in endless discussions about legal agreements,” said Jan Lundberg, executive vice president for science and technology and president of Lilly Research Laboratories. “We are here to serve patients, and in the end, we are all patients.”
The template agreements also provide a roadmap for handling intellectual property used in or developed through the program. Participating industry partners will retain the ownership of their compounds, while biomedical research partners will own any intellectual property they discover through the research project with the right to publish the results of their work.
“This initiative gives us a chance to access, on a grand scale, the wonderful minds that we have out in the academic community,” said Rod MacKenzie, group senior vice president and head of Pfizer PharmaTherapeutics research and development. “This kind of public-private partnership is exactly what we need as we create a new R&D environment.”
“This program is just the first of what we hope will be many more NCATS-led efforts to pick up the pace of translational research,” said NCATS acting director Dr. Thomas Insel.
For more information, visit http://ncats.nih.gov.