Thomas Crawford, MD, on Confirming Long-Term Efficacy of Nusinersen for SMA

“The NURTURE study was one of the major 3 studies from Biogen that were used to establish the benefit of nusinersen in the treatment of spinal muscular atrophy. The first 2 were in babies and children and they are the ones that showed the extraordinary benefit. Babies were rescued from death. My previous career was basically as a hospice doc, and I went into caring for kids with SMA here at Hopkins and personally held 72 babies that died of this disease. And with the approval of nusinersen, basically, that's not happening anymore, those babies are being rescued.”

Updated analyses from the phase 2 NURTURE study (NCT02386553) have demonstrated that children who received nusinersen (Spinraza; Biogen) treatment before the onset of clinical spinal muscular atrophy (SMA) symptoms continued to maintain and make gains in motor function over 5 years. The updated data showed that 23 of 25 children are walking independentlyand alsosuggest that early markers of disease activity may be predictors of motor function outcomes including respiratory function, swallowing, and feeding.

CGTLive spoke with investigator Thomas Crawford, MD, to learn more about the updated analyses and its implications. He touched on how it compared to the other studies supporting nusinersen’s approval for treating SMA in that it included older patients which are still seeing benefits from the therapy today. He also stressed the transformative nature of the approval as well as the approvals of onasemnogene abeparvovec (Zolgensma; Novartis) and risdiplam (Evrysdi; Genentech) that followed.

REFERENCE

Crawford TO, Swoboda KJ, De Vivo DC, et al. Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study. Muscle Nerve. 2023; 68(2): 157- 170. doi:10.1002/mus.27853