I+A for Rare Diseases
The Program’s interest in I+A for rare diseases represents the flip side of the coin to neglected diseases endemic in low- and middle-income countries. While for neglected tropical diseases, the patients may number in the millions, but the affordability of treatment may be very low. For many rare diseases, the number of patients are small, but the unit costs for treatment can be very high. As a result, both rare and neglected diseases face challenges in bringing new treatments to market.
To this end, over the past several years, Josh Sommer served as a Fellow in Strategic Philanthropy and Health in the GHTA Program. Diagnosed with a rare form of cancer as a freshman at Duke, Josh Sommer decided that the best way to beat the odds of his disease was to accelerate the search for a cure. Josh co-founded the Chordoma Foundation, and went on to win a prestigious Echoing Green Fellowship for social entrepreneurship. The Echoing Green Fellowship allowed Josh to develop the Chordoma Foundation’s efforts. The Chordoma Foundation has now taken on Josh as its full-time Executive Director. At the end of September 2011, Josh made the transition from the GHTA Program to that role.
The Chordoma Foundation under Josh’s stewardship has made significant strides in its work:
- Chordoma Foundation-funded research at the Sanger Institute in the United Kingdom led to the discovery of an entirely new way that cancer can form—through shattering of a chromosome. The results were published in the prestigious journal Cell and reported in the New York Times.
- The Chordoma Foundation pioneered the first use of an Innocentive prize for the creation of quality cell lines for chordoma.
- Hosting the Third International Chordoma Research Workshop, the Foundation brought 85 physicians and scientists together from 9 countries and heard 32 presentations on new, unpublished data.
- The Foundation awarded four $25,000 seed grants to researchers at the Massachusetts General Hospital, the University of Pittsburgh, and two labs at Johns Hopkins aimed at developing new treatments for chordoma.
Dr. So contributed to the Institute of Medicine Forum on Drug Discovery, Development, and Translation’s workshop and monograph (see chapter on “Strategies for Navigating Intellectual Property” on “Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies,” published in February 2009.
He also served on the Institute of Medicine Committee that produced the report, Accelerating Rare Diseases Research and Orphan Product Development, released in October 2010.
He also spoke on “Providing Public Sector Incentives and Ensuring Fair Returns for Drug Rescue and Repurposing” at the NIH-Industry Roundtable on “Exploring New Uses for Abandoned and Approved Therapeutics” in April 2011.