Last week, the National Institutes of Health unveiled a collaborative program that will match researchers with a selection of pharmaceutical industry compounds to help scientists explore new treatments for patients. NIH’s new National Center 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. Additional companies may join and contribute their compounds for further investigation, according to NIH.
“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. Some of these challenges were identified at an NIH-Industry Roundtable in April 2011, which included a group of senior leaders and experts from the pharmaceutical industry, government, academia, and the non-profit sector to explore opportunities to foster new NIH-industry partnerships that facilitate drug rescue and repurposing.
Stakeholders pointed to: resource implications (the time and resources for a pharmaceutical company to maintain, update, and organize their compound libraries for drug rescue and repurposing); patent considerations (off-patent compounds or compounds whose patents are close to expiring, may not be attractive to industry because the financial return and market incentives for the product may be limited); and transactional hurdles related to developing, negotiating and implementing appropriate legal agreements among the parties, including addressing such concerns as intellectual property rights and liability.
NCATS was established last year to help address this gap. 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. 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, M.D., Ph.D. “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 program 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 program. 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.
“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.”
Another industry collaboration recently announced was between GlaxoSmithKline and Yale University. The partnership will concentrate on creating a possible new class of drugs. Under the discovery agreement, GSK’s scientists will work alongside Yale researchers to build on Yale’s pioneering work with so-called proteolysis targeting chimeric molecules (PROTACs).
PROTAC technology assists the cellular destruction of disease-causing proteins by sending them to a cell’s “garbage disposal” for culling. Mutant or unusually high levels of these proteins can be connected with disease progression in a number of areas, such as oncology and inflammation, but currently cannot be targetted by traditional ways of making medicines, GlaxoSmithKline notes.
The terms of the partnership provide GSK with the right to use PROTAC technology for multiple disease-causing proteins across all therapy areas. In return, for each protein-degrading drug discovered and developed, Yale University will receive milestone and royalty payments. The partners have already started research as part of their collaboration and GlaxoSmithKline said proof of principle data is anticipated to be accessible by the end of the year.
GlaxoSmithKline has already created such risk-sharing type or arrangements with academic institutions in the UK, but this is the first one in the US, and it is also important due to its scope around a possible new class of medicines.
Kris Famm, head of GSK’s Protein Degradation effort, who will be leading the programm with Yale University’s Professor Craig Crews, commented that “this partnership is exploring a new way for promising, but unproven therapeutic approaches to jump from the academic lab more quickly into the early stage pharmaceutical pipeline.”
“The relationship between the pharmaceutical industry and academia is changing,” Yale University’s Professor Craig Crew noted, adding that the teamwork “offers a new paradigm for how pharma companies and academic researchers can benefit from working more closely together.”
Conclusion
NIH should once again be commended for this recent initiative. We previously noted that NIH was collaborating with Eli Lilly on a similar initiative. What is important about these collaborations is the ongoing recognition of the federal government that medicine and science cannot progress without industry collaboration. As the Request For Information (RFI) acknowledges, “Partnerships with industry have been used throughout government and the biomedical research community to leverage each sector’s expertise to speed scientific research and corresponding commercial development.”
Yale and GSK should also be applauded for their recent initiative, especially in light of the fact that several top medical research universities have begun to turn away research grants and funding from industry. Clearly, these two organizations recognize the tremendous potential and benefit that science and medicine can gain by working together.
Ultimately, NIH is moving away from the trend of some agencies and scientific institutions who believe it is better to operate in separate silos from industry, instead of open and transparent communication and collaboration. NIH should continue adding more partners to this new endeavor and industry should be lining up to show their support. It is through partnerships such as these that new breakthroughs in cancer, heart disease, diabetes, and obesity will come, which will reduce America’s health expenditures and help our healthcare system control its spiraling costs.