November 01, 2011
By KUMC News
|Scott Weir, PharmD, PhD, is director of KU's Institute for Advancing Medical Innovation|
As part of an aggressive effort to speed delivery of treatments to patients by finding new uses for approved drugs, researchers at the University of Kansas Medical Center have begun a clinical trial targeting the most common form of adult leukemia with a drug first approved to treat arthritis more than 25 years ago.
Earlier this month, KU researchers treated the first trial participant, a Kansas City-area patient suffering from chronic lymphocytic leukemia or CLL, with the drug auranofin, which has long been used to treat patients with arthritis.
The trial is one key piece of a larger collaboration between KU, The Leukemia & Lymphoma Society (LLS) and the National Institutes of Health (NIH) to accelerate discovery and development of safe, effective and affordable cancer treatments. Over the last two years, the group discovered that auranofin kills CLL cells in test tubes, and received approval to test the drug in CLL patients.
"Today's process of discovering and developing new drugs for patients takes too much time and costs too much money," said Louis J. DeGennaro, Ph.D., executive vice president and chief mission officer, LLS. "The collaboration between KU, LLS and NIH is committed to giving new hope to patients by reducing sharply the time and costs associated with developing new therapies. Auranofin is a great example of what is possible through an effective public-private partnership."
"Spending more than $1 billion and taking more than a decade to deliver new therapies to patients is simply not sustainable," said Scott Weir, PharmD, PhD, director of KU's Institute for Advancing Medical Innovation. "Our group moved this new discovery into a clinical trial in just two years and for about $1 million, representing significant time and cost savings from business as usual."
In the coming weeks, additional patients both in Kansas City and at partnering sites including the NIH in Bethesda and the James Comprehensive Cancer Center at The Ohio State University will be enrolled in the trial.
CLL is currently treated with drugs that are initially effective. But patients often reach a stage where they become resistant to treatment, including chemotherapy, leading to death absent other treatments. And while chemotherapy can be quite toxic, auranofin has been demonstrated to be reasonably safe and effective in the treatment of arthritis.
The development of new uses for approved and abandoned drugs is termed drug repurposing and is a major focus of the KU-LLS-NIH collaboration. In addition to auranofin, researchers are currently exploring other existing drugs that may offer similar hope for patients with leukemia and other blood cancers.
"The face of modern medicine may rapidly change if accelerated drug development, as demonstrated in the repurposing of auranofin for CLL, is successful," said Christopher P. Austin, M.D., director of the NIH Center for Translational Therapeutics, currently administered by the National Human Genome Research Institute, a part of NIH. "Perhaps most exciting is that this model of science and collaboration is scalable and generally applicable to other cancers and diseases, and thus has enormous potential as a new paradigm for therapeutics development."