New Muscular Dystrophy Treatment Offers Hope
Study Shows Patients With Duchenne's Muscular Dystrophy Are Walking Better With PRO051 Treatment
Three of the patients improved their walking distance by 213 feet or more. That may not seem like much. But in just the six to 15 months between the first and second phases of the study, these patients' six-minute walking ability had declined by 121 feet.
"This is exciting, but it is not going to be for everyone with DMD," Kula says. "But the exciting thing is, if the technology works out, you can generate drugs for different DMD mutations. That is the wave of the future."
Indeed, van Deutekom says that is exactly what her company is doing. PRO051 should work for patients with DMD mutations on or very near exon 51. Now in the works are similar drugs targeting exons 44, 45, 52, 53, and 55.
PRO051 is not the only drug candidate in clinical trials. A U.S. company, AVI BioPharma, is testing a similar exon-51-skipping antisense oligonucleotide called AVI-4658. Promising findings -- but not clinical improvement -- was reported last October from early clinical trials of AVI-4658.
Van Deutekom and colleagues report their findings in an early online release of the New England Journal of Medicine.