The National Institutes of Health today announced an agreement with two non-profit organizations to accelerate the development of potential clinical therapies for rare blood cancers.
The cooperative research and development agreement has been established as a shared commitment to move therapies for rare blood cancers into clinical proof-of-concept studies so that promising treatments can eventually be commercialized. The agreement is among the University of Kansas Medical Center, Kansas City, The Leukemia & Lymphoma Society (LLS), the NIH Therapeutics for Rare and Neglected Diseases (TRND) program and the Hematology Branch within the National Heart, Lung and Blood Institute.
"The goal of this collaboration, called The Learning Collaborative (TLC) is to bridge the gap in time and resources that often exists between basic research and human testing of potential new treatments and accelerate and make more efficient the delivery of improved therapies to patients with rare blood cancers," said Christopher P. Austin, M.D., director of the TRND program, currently administered by the National Human Genome Research Institute, a part of NIH. "As a result of our work, as the TLC name implies, we plan to learn more about and improve the drug development process for all diseases."
"This agreement recognizes that academic, philanthropic and patient organizations have a role – and a responsibility – to be active leaders in the drug discovery process," added Roy A. Jensen, M.D., director, The University of Kansas Cancer Center, Kansas City. "I hope this agreement can serve as a model to other academic and patient communities."
The collaboration's first project is a pilot effort under the TRND program that focuses on further development of an existing small molecule drug used to treat arthritis called auranofin. Auranofin will be evaluated as a treatment for relapsed chronic lymphocytic leukemia (CLL) – one of the four major types of leukemia and one that typically affects older people. The goal is to accelerate the development of this drug and complete preclinical through clinical trial studies within two years, at which time an industry partner will be engaged.
Approximately 15,000 people in the United States receive a diagnosis of CLL each year. CLL is currently treated with various chemotherapies, but patients eventually reach a stage where they become resistant to this treatment and can die as a result. In addition, chemotherapy can be quite toxic, whereas auranofin has received regulatory approval and was demonstrated to be reasonably safe and effective in the treatment of arthritis.
"Chronic lymphocytic leukemia is a complex disease that, while treatable, cannot be cured with chemotherapy," said Adrian Wiestner, M.D., Ph.D., an investigator in the NHLBI's Hematology Branch. "There is an especially urgent need to develop novel therapies for chemotherapy-resistant disease, and through this agreement the Hematology Branch can bolster its clinical and translational research program in this disease as well as other hematologic conditions with unmet clinical needs."
"Development of new therapies for diseases like the blood cancers poses both scientific and economic challenges," said Dr. Louis J. DeGennaro, Ph.D., executive vice president and chief mission officer, LLS. "Partnerships are the key to addressing this significant unmet medical need and LLS is proud to add our expertise to this collaboration."