A Crusade Against Huntington’s Disease

  • Medicine & Health
  • 1997

Huntington’s disease is a genetic disorder that destroys portions of the brain involved in movement, thinking, and emotions, progressing over decades with ultimately fatal results. It strikes perhaps 30,000 persons in the U.S., and if one of your parents had it, you have a 50-50 chance of developing the disorder yourself. For years it was one of those afflictions that attracted relatively little research funding or attention outside of the families plagued by it. That changed in the mid-1990s, when a single donor began to pour large resources into a broad campaign against the disease.

Andrew Shechtel entered Johns Hopkins University at the precocious age of 16 and left three years later with degrees in math and political economy, then spent time at Harvard Business School and Wall Street before entering an investment firm focused on abstruse quantitative financial trades. He became a multibillionaire, and continues in the field, while also giving away several billion dollars.

His favorite cause is battling Huntington’s Disease, to which he has donated at least $732 million directly since 2000, plus more indirectly via grants to allied organizations. In recent years, his annual spending of more than $100 million for an HD cure has exceeded the total efforts in this area of the massive National Institutes of Health (which devoted $55 million to Huntington’s in 2013). With the charitable trust established by Shechtel containing $5 billion as of 2013, a good deal more spending can be expected.

The donor’s gifts are primarily channeled through his CHDI Foundation, whose mission is to “bridge the translational gap that often exists between academic and industrial research” and “develop drugs that will slow the progression of Huntington’s disease and provide meaningful clinical benefit to patients as quickly as possible.” The organization’s description of itself is businesslike: “Essentially, CDHI is a science-management organization…. Our science directors and project managers work closely with a network of more than 600 researchers…in the pursuit of novel therapies, providing strategic scientific direction and management. [Our] overall strategy is to de-risk therapeutic approaches [so] pharmaceutical company partners will view them as a good investment…. Our bottom line is ensuring the shortest possible time to getting effective therapeutics to HD patients.”

Part of this involves encouraging scientists to take up work on Huntington’s, by providing background knowledge, chemicals and supplies, animal models, and working protocols that can speed research. The nonprofit also has created its own large registry and observational study of families experiencing Huntington’s disease. And it collaborates with a network of other advocacy organizations to support patients, family members, and clinicians.