Jörn-Sven Kühl, MD, Department of Pediatric Oncology, Hematology and Hemostaseology at University Hospital Leipzig, discusses long-term results from the Phase 2/3 Starbeam study and data from the Phase 3 ALD-104 study.

Bluebird bio, Inc. recently presented data on the Starbeam study and ALD-104 study at EBMT 2020. 

The Starbeam study is a phase 2/3 clinical trial evaluating elivaldogene autotemcel (eli-cel) gene therapy for cerebral adrenoleukodystrophy (CALD). 

CALD is a progressive neurodegenerative disease that involves the breakdown of myelin. Symptoms of CALD usually occur in early childhood and, if untreated, rapidly leads to severe loss of neurologic function and eventual death in most patients. CALD is associated with six major functional disabilities (MFDs), which severely compromise a patient’s ability to function independently: loss of communication, cortical blindness, need for tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement. Nearly half of boys with CALD who do not receive treatment will die within five years of symptom onset.

As Dr. Kühl states, the primary endpoint of the Starbeam study was the proportion of patients who were alive and free of MFDs after 2 years. Data from the study showed that 20 out of 23 participants in the study were free of MFDs after 24 months following eli-cel infusion. The primary safety endpoint of the Starbeam study was the proportion of patients who experienced acute or chronic Graft versus Host Disease (GvHD) by month 24. No events of acute or chronic GvHD were reported post-eli-cel treatment. Additionally, there were no reports of graft failure or graft rejection.

The ALD-104 study is a Phase 3 study designed to assess the efficacy and safety of eli-cel in patients with CALD using a different chemotherapy conditioning regimen than used in the Starbeam study. 

The primary efficacy endpoint of the ALD-104 study is the proportion of patients who are alive and free of MFDs at Month 24, and the primary safety endpoint is the proportion of patients with neutrophil engraftment after eli-cel infusion.

For more information about CALD and other rare neurological disorders, visit checkrare.com/neurology