Statement from the ALD Connect Board of Directors

The ALD Connect Board of Directors released the following statement on August 10, 2021:

We would like to acknowledge the recent press release from bluebird bio. bluebird bio has sponsored our ALD Connect programs, projects, and Annual Meetings for many years.

Yesterday, bluebird bio announced that they received a report of myelodysplastic syndrome (MDS) in a patient treated with elivaldogene autotemcel (eli-cel, or Lenti-D™), their investigational gene therapy for early cerebral adrenoleukodystrophy (cALD). The U.S. Food and Drug Administration (FDA) has put the clinical studies on ALD on hold in order to independently evaluate the benefit/risk of Lenti-D™. We will continue to assess this situation as more information becomes available.

bluebird bio also announced that they have decided to concentrate their severe genetic disease focus on the United States and will wind down their independent operations in Europe due to multiple contributing factors.

This news is concerning for many patients, families, and other members of our ALD community. Families around the world have made personal sacrifices to participate in the clinical trials. Academia and industry have collaborated for years to advance this treatment option. With the current news much remains unknown, and this uncertainty is difficult. We hope that the continued commitment across our community will bring about answers and progress in the days to come.

ALD Connect does not recommend or endorse any specific physicians, treatments, procedures, or products. If you have specific questions or concerns about your child or Lenti-D™, please reach out to your child’s physician directly.