The National Institutes of Health (NIH) has launched collaborative programme that will match researchers with a selection of pharmaceutical industry compounds to help scientists explore new treatments for patients. NIH's new National Centre for Advancing Translational Sciences (NCATS) has partnered initially with Pfizer, AstraZeneca, and Eli Lilly and Company which have agreed to make dozens of their compounds available for this initiative's pilot phase.

“Americans are eagerly awaiting the next generation of cures and treatments to help them live longer and healthier lives. To accelerate our nation’s therapeutic development process, it is essential that we forge strong, innovative, and strategic partnerships across government, academia, and industry,” said Health and Human Services Secretary Kathleen Sebelius.

In recent years, researchers have succeeded in identifying the causes of more than 4,500 diseases. But it has proven difficult to turn such knowledge into new therapies; effective treatments exist for only about 250 of these conditions. NCATS was established last year to help address this gap. It supports rigorous scientific research designed to reengineer elements of the development pipeline to move basic research findings into new treatments for patients.

Some compounds do not prove effective for the specific use for which they were developed; however, if additional research is conducted, they may succeed for a different therapeutic use. A prime example of a compound that did not prove effective for its initial use but succeeded for a different use is azidothymidine (AZT), which failed to show efficacy against cancer, but was later found to be the first medicine effective against HIV, the virus that causes AIDS.

“Clearly, we need to speed the pace at which we are turning discoveries into better health outcomes,” said NIH director Francis S Collins, MD, PhD. “NIH looks forward to working with our partners in industry and academia to tackle an urgent need that is beyond the scope of any one organization or sector.”

The initiative, Discovering New Therapeutic Uses for Existing Molecules, will direct researchers’ attention to a part of the therapeutic pipeline that traditionally has been difficult for them to access: compounds that already have cleared several key steps in the development process, including safety testing in humans. The President’s fiscal year 2013 budget proposed $575 million for NCATS, of which approximately $20 million total will be provided to support research grants of up to three years duration for pre-clinical and clinical feasibility studies.  These studies will test more than 20 compounds from industry partners for their effectiveness against a variety of diseases and conditions. The companies will provide the researchers with access to the compounds and related data.

The pilot programme incorporates innovative template agreements designed to streamline the legal and administrative process for participation by multiple organizations. These template agreements reduce time, cost, and effort, as well as allow greater participation than traditional partnerships.

The templates also provide a roadmap for handling intellectual property used in or developed through the programme. Participating industry partners will retain the ownership of their compounds, while academic research partners will own any intellectual property they discover through the research project with the right to publish the results of their work.

For this pilot programme, NIH is excited to have three participating companies. Additional companies may join and contribute their compounds for further investigation.

“Pfizer has a rich history of partnering with the academic and public sectors to discover and develop innovative medicines,” said Rod MacKenzie, Ph.D., group senior vice president, head of Pfizer PharmaTherapeutics Research and Development. “We believe that participating in this unique program, and making well researched molecules available to NIH's broad network of researchers, is another important step in advancing a vibrant biomedical ecosystem.”

“AstraZeneca strives to realize the full potential of its project portfolio by collaborating with the best clinical and pre-clinical research experts worldwide, leaving no stone unturned in our search for new and effective medicines,” said Menelas Pangalos, Ph.D., executive vice president, Innovative Medicines, AstraZeneca. “Partnering across government, academia andindustry is a critical way to spur additional scientific innovation and the delivery of new treatments for people who desperately need them.”

“We are pleased to see the significant progress made to date by the newly established NCATS under Dr. Collins’ leadership, especially given that we share a similar mission of speeding the delivering of innovative medicines to the patients who are waiting,” said Jan Lundberg, Ph.D., executive vice president, science and technology, and president, Lilly Research Laboratories. “Discovering and developing new medicines, regardless of the therapeutic area being studied, is a complex and expensive process and we look forward to collaborating with the NIH and academia for the benefit of the patients we all serve.”

The National Centre for Advancing Translational Sciences (NCATS) aims to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.

NIH, the nation's medical research agency, and a component of the US Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.