New NIH program launched to develop treatments for rare and neglected diseases

A $24 million grant from Congress will kick start the National Institutes of Health’s new Therapeutics for Rare and Neglected Diseases program, an initiative aimed to overcome many of the road blocks associated with developing new drugs for the approximate 7,000 illnesses that fit into these categories and affect millions of Americans each year.

“While Congress has previously taken important steps to help these patients such as providing incentives for drug companies under the Orphan Drug Act, this is the first time the NIH is providing support for specific, preclinical research and product development known to be major barriers preventing potential therapies from entering into clinical trials for rare or neglected disorders,” Stephen C. Groft, PharmD, director of the NIH Office of Rare Diseases Research, said during a press briefing.

Therapeutics for Rare and Neglected Diseases (TRND) will be a collaborative effort between senior scientists from 27 different NIH centers including the Office of Rare Diseases Research, the National Human Genome Research Institute and the Chemical Genomics Center and an external expert panel made up of members from academia, industry and disease advocacy groups.

Many molecular targets or chemicals with therapeutic potential never make it into the drug development process because academic researchers often do not have the resources to conduct the multitude of studies needed to develop a new drug, Groft said.

Furthermore many drug candidates that do receive funding for development never make it out of the preclinical phase, which many in the industry dub the “Valley of Death” due to the 80% to 90% project failure rate that occurs during this stage.

Christopher P. Austin, MD, director of the NIH Chemical Genomics Center likened the preclinical process to solving a Rubik’s cube. He said that drug candidates must fulfill about 20 different characteristics to make it to the clinical trial phase. “You can have a compound that has good potency, but you will also need it to have good blood solubility,” he said. In the process of improving one characteristic, several others that were already achieved may disappear, he explained.

This process can be long and expensive — taking about two to four years and costing about $10 million on average. And pharmaceutical companies are hesitant to invest highly in drug candidates which may yield low returns.

“TRND will develop promising treatments for rare diseases to the point that they are sufficiently “de-risked” for pharmaceutical companies, disease-oriented foundations or others, to undertake the necessary clinical trials,” Alan E. Guttmacher, MD, acting director of the NIH National Human Genome Research Institute, said during the briefing.

Another main goal of the program will be to pinpoint reasons behind high preclinical failure rates by openly sharing information. Austin called attention to the fact that journals only publish data from successful trials and not those that fail, which he said “leads to stagnation in drug development.” He said TRND plans to publish failures as well, so everyone in the scientific community can learn from the mistakes.

Although NIH officials are not sure exactly which specific diseases the program will home in on, they emphasized that the focus will be on disorders that are not currently well funded, such as schistosomiasis and other tropical diseases. Currently, NIH is working to establish criteria to determine which projects will be pursued. – by Nicole Blazek