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THURSDAY, Oct.10, 2024Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, two new reports find. The 77 boys were treated for cerebral adrenoleukodystrophy (CALD), a formerly incurable and progressive genetic brain disease that typically led to a loss of neurological function and early death. However, most patients treated with “eli-cel” gene therapy six years ago are still showing no declines in brain function and remain free of disability, researchers reported Oct. 9 in two studies published in the New England Journal of Medicine. There was a disturbing finding from one of the two studies, however: Six of 35 patients who received one form of the gene therapy have developed blood cancers that seemed tied to the gene therapy. However, the overall news is positive for children who otherwise faced a death sentence, the researchers said. “Cerebral adrenoleukodystrophy is a devastating brain disease that strikes children in the prime of their childhood and development,” explained Dr. Florian Eichler, director of the Leukodystrophy Clinic in the Department of Neurology at Massachusetts General Hospital and lead author of one of the two studies. “When I initially began treating patients with CALD, 80 percent came into our clinic on death’s door, and now the ratio has flipped.” “We cautiously celebrate that we have been able to stabilize this… read on > read on >