Leukodystrophy Resource & Research Organisation

ALD Hearing on 8.27.2015

join posterThe ALD patient and physician community has advocated for years to get ALD added to the Recommended Uniform Screening Panel (RUSP). RUSP is the list of diseases that the federal government (through the Secretary of Health) recommend that all states test newborns for. The latest nomination to add ALD to the RUSP was September 2012 when the community was told that there was not enough evidence for ALD newborn screening (NBS). The meeting on Thursday, August 27th in Rockville, MD, was the culmination of years of follow-up work to gather expert opinion and unpublished data to give the case for the clear benefit and feasibility of newborn screening for ALD in the US.

Some of the most active groups have been ALD ConnectStop ALDBrian’s HopeThe Aidan Jack Seeger FoundationFight ALD, and The Ethan Zakes Foundationbluebird bio was honored to help support the community’s efforts to raise awareness and gather the necessary information.

At Thursday’s meeting, parents and family members gave heart-wrenching public testimony during the public comment period on why NBS and early detection can save lives and impact families. Amber Salzman, Elisa Seeger, Kathleen Kelley, Janis Sherwood, and Marty Luczak brought tears to the eyes of everyone in the room. But the conversation and debate were still touch-and-go…because the disease is so rare and there are so little data available. The data that bluebird bio, Florian Eichler, and NY state provided were the basis for understanding how many lives can be saved by early diagnosis.

When the time for the vote came, we all sat together, holding hands, not knowing how it would go. There was one abstention and one no…but the rest all voted yes, not only to recommend ALD’s addition to the RUSP but to also have funding provided so that the states can implement and track outcomes, something none of the other diseases were given.

California and NJ will start testing for ALD as soon as the Secretary of Health finalizes the additional of ALD to the RUSP, and Connecticut has a meeting next week where the RUSP decision will have a huge impact on the final decision. (Tennessee, Illinois, and Massachusetts have also passed the required votes/legislation required, but they have not yet implemented.)

At the celebration after the vote yesterday, there was already an action plan in place for how to get this implemented in all states.

Thank you to all of our supporters!