NCATS Matches Researchers with Pharmaceutical Industry Assets to Test Ideas for New Therapies

For every drug that is approved by the U.S. Food and Drug Administration, many others are not due to lack of effectiveness in treating the disease they were designed to target. NCATS leads initiatives to repurpose some of these partially developed investigational drugs (also known as assets) to treat new disease indications and get more treatments to more patients more quickly.

Through its Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program, NCATS recently awarded nearly $3 million to support four academic research groups that will test a selection of pharmaceutical industry assets for new therapeutic uses. The awarded projects are aimed at finding therapies for:

  • Type 2 diabetes
  • Glioblastoma (one of the most aggressive brain tumors in adults)
  • Acute myeloid leukemia (an aggressive blood cancer)
  • Chagas disease (a neglected tropical disease that causes heart, digestive and neurological problems)

Each award recipient will test a selected asset for its effectiveness against a previously unexplored disease or condition. The industry partners for these projects are AstraZeneca and Sanofi.

“The New Therapeutic Uses program helps re-engineer the research pipeline by crowdsourcing pharmaceutical company assets that already have cleared several key steps in the development process, including initial safety testing in humans,” said Christine M. Colvis, Ph.D., NCATS director of drug development partnership programs. “This approach enables scientists nationwide to contribute their expertise to accelerate the pace at which discoveries are turned into treatments and cures for patients.”

“Too often academic, industrial and community partners operate in separate silos,” said Craig Wegner, Ph.D., head of the Boston Emerging Innovations Unit, Scientific Partnering & Alliances within AstraZeneca's Innovative Medicines and Early Development Biotech Unit. “The crowdsourcing aspect of the New Therapeutic Uses initiative enables these parties — who might otherwise not have connected ― to combine their knowledge and resources to push the boundaries of medical science.”

The 2015 projects are:

“There is a longstanding need to develop new treatments for Chagas disease, the most common parasitic disease in the Americas,” said Elias Zerhouni, M.D., president of Sanofi Global R&D. “Through collaborations facilitated by the New Therapeutic Uses initiative, we have an opportunity to address this neglected public health issue and deliver real value to patients with unmet medical needs in developing regions across North and South America.”

Nick Giannoukakis, Ph.D., explains his research project on type 2 diabetes. (Allegheny Health Network Research Institute Video)

The 2015 awards will fund projects for researchers to conduct preclinical validation and additional safety and tolerability studies as needed. If specific milestones are met, investigators can begin clinical feasibility studies or proof-of-concept clinical trials to explore whether the selected assets may be effective as treatments for other diseases. The projects will be supported for up to three years.

The pilot phase of the New Therapeutic Uses program tested the utility of template agreements established with each industry partner that proved successful in facilitating negotiations. The agreements reduced the time required to establish collaborations between industry and academia to about three months from the more typical nine months to one year.

NCATS announced another early program success in March 2015: New Therapeutic Uses support to a Yale research team led to an expedited clinical trial to test an experimental cancer drug as an Alzheimer’s treatment.

NCATS leads the New Therapeutic Uses program with additional scientific expertise provided by the National Cancer Institute; National Heart, Lung, and Blood Institute; National Institute of Allergy and Infectious Diseases; National Institute of Diabetes and Digestive and Kidney Diseases; National Institute of Mental Health; National Institute of Neurological Disorders and Stroke; National Institute on Aging; National Institute on Alcohol Abuse and Alcoholism; and National Institute on Drug Abuse.

For more information, visit NCATS’ New Therapeutic Uses Web page.


Posted July 2015