The U.S. Food and Drug Administration (FDA) has awarded a $2 million, five-year grant to Dr. David Lynch of the Children’s Hospital of Philadelphia to study the natural history of Friedrich’s ataxia (FA) and five other rare diseases.
The award is part of the FDA’s Orphan Products Grant Program.
“We believe these important studies will provide key information about how these rare diseases develop and progress and can ultimately help in the development of models of disease behavior that can make the development process more efficient,” FDA Commissioner Scott Gottlieb said in a press release. “We’ve been working overtime to develop models that can simulate the behavior of placebo arms in the setting of very rare diseases, where recruiting for clinical trials can be especially hard.”
The natural history of a disease is the history of its course in the absence of any treatment. It begins before symptoms appear, through to the stage in which symptoms and signs of the disease start, and follows clinical progression to the final outcome.
Moreover, the natural history could potentially be used to make a model of a rare disease. This model could serve as a control in a clinical trial, instead of a separate group of patients who do not receive drug being tested, to compare with the test group. In rare diseases like FA, recruiting enough patients for a study is always a challenge.
The European Friedreich Ataxia Consortium for Translational Studies has also set up a FA registry to better understand its natural history.
“One of the challenges we encounter developing therapies for rare diseases is the lack of natural history data to guide the design of successful clinical trials,” said Dr. Nora Yang, director of portfolio management and strategic operations at the National Center for Advancing Translational Sciences (NCATS), a unit of the National Institutes of Health. “We are pleased that we can collaborate with the FDA to fund two natural history studies this year that will help NCATS develop novel treatments for patients who suffer from these devastating diseases.”
Friedreich’s Ataxia News recently interviewed Lynch, one of the principal investigators conducting the MOXIe Trial ((NCT02255435) testing omaveloxolone in FA. Friedreich’s ataxia is a genetic disease characterized by progressive degeneration of the nervous system and difficulties with movement. Symptoms first become evident during childhood or adolescence, or in early adulthood.