Sickle Cell Disease Legislation Passes House, Introduced in Senate

The House of Representatives last week passed HR 2410, the Sickle Cell Disease Research, Surveillance, Prevention, and Treatment Act. Among other provisions, this legislation would enhance research and surveillance efforts, which would improve the current understanding of this life-long disease and potentially identify more curative options. Treatments for this disease are limited and often focus on relieving symptoms, reducing pain, and avoiding crises and complications. At this time, hematopoietic stem cell transplantation is the only cure. Several gene therapy trials are now underway for sickle cell disease (SCD), with promising early results. No explicit funding for SCD has been provided since 2009.

Also last week, Senator Cory Booker (D-NJ) and Senator Tim Scott (R-SC) introduced the companion bill yesterday (S 2465), which was referred to the Senate Health, Education, Labor and Pensions Committee. ASGCT signed on to a letter of support, along with 66 other organizations, to Senators Scott and Booker. In a round table discussion yesterday, Senator Booker provided more information about the bill and spoke with patients, a parent, and physicians about its significance.

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