Health
First prenatal treatment for spinal muscle atrophy shows promising results
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder that continues to move before birth. The first was led by scientists at St. Jude Children's Research Hospital Inside the uterusTreatment of SMA with oral administered drug Lisdipuram. No identifiable features of SMA have been observed since more than two years after the child was born. This study demonstrates the potential to treat SMA before birth and supports further investigation of the approach. The survey results today New England Journal of Medicine.
Our main objectives were feasibility, safety and tolerability. That's why we are very pleased that our parents and children are doing well. The results suggest that it is worth continuing to investigate the use of prenatal interventions in SMA. ”
Richard Finkel, Maryland, The corresponding author of the study, St. Jude Center for Experimental Neurotherapeutics Director and Member of the Faculty of Pediatrics
SMA is caused by a lack of survival motor neuronal proteins and occurs at about 1 per 11,000 births in the US. If untreated, the most common and severe form of SMA type 1 (SMA-1), leads to progressive muscle weakness that leads to death. Currently, treatment for SMA-1 demonstrates improved survival and motor function in infants. This is not a treatment, especially if it is administered immediately after giving birth, just before symptoms begin.
N-of-1: Prenatal Lisdipuram case study
Survival motor neuronal proteins are most needed during the third stage of fetal development and the first three months after birth. Therefore, symptoms severity is closely related to the time of intervention. Because of this clinical need, St. Jude researchers have launched a unique clinical protocol for studying Lisdipuram in a single patient as part of the Pediatric Translation Neuroscience Initiative. The goal was to determine the likelihood of starting fetal treatment with SMA-1 Inside the uterus.
The parents in this case were both known carriers of SMA genetic variants and had previous infants born with SMA-1 before current treatment became available. Genetic tests performed by amniocentesis confirmed that the fetus does not have a copy of the survival motor neuron gene. Risdiplam was administered to pregnant mothers during the last six weeks of pregnancy.
Safe and promising results will promote future research
Immediately after birth, the infant was diagnosed with three developmental abnormalities: ventricular septal defect (resolution), optic hypoplasia, brainstem asymmetry has delayed visual and overall development. These abnormalities are thought to have occurred early in fetal development before exposure to lisdipuram.
The two and a half year old is currently being monitored regularly at St. Jude. “During the course of the assessment, we really didn't see any signs of SMA,” Finkel said. This study demonstrates the safety and feasibility of the approach and strengthens the case for a more comprehensive study.
Authors and Funding
Another author of this study is Julieann Parker of Obgyn Partners in Augusta. Heidemarie Kletzl of Lutz Mueller and F. Hoffmann-La Roche Ltd; Saint Jude of Samuel Hughes, Matthew Civitello, Alfonso Labado and Heather Mefford;
The drugs used in this study were provided by F. Hoffman La Roche, and the study was supported by funding from the American Lebanon-Syrian Charities (ALSAC), a fundraising and awareness organization for St. Jude. Ta.
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Journal Reference:
Finkel, rs, et al. (2025). Lisdipuram for prenatal therapy for spinal muscular atrophy. New England Journal of Medicine. doi.org/10.1056/NEJMC2300802.
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