The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants

By Cure SMA | Published On December 7, 2016

Today, Lancet published results from the Phase 2, open-label trial of nusinersen (Spinraza) in infants. This trial initiated in 2013, and patients continue to receive the drug. 

The results show that infants as young as five weeks old with the most severe form of spinal muscular atrophy (SMA) - a leading genetic cause of infant mortality - can be treated safely with nusinersen. This investigational treatment slowed progression of the disease, improved survival, and in some cases demonstrated remarkable improvement in muscle function, according to the research published online by The Lancet

"With nusinersen, these infants are not only living longer, but they're living better", said Richard S. Finkel, MD, lead author of the study and chief of neurology at Nemours Children's Hospital in Orlando, Fla. "SMA is no longer a death sentence for infants. This treatment is by no means a cure, but it is more than we've ever been able to offer these families before." 

The multi-site, phase 2, open-label trial of patients with infant-onset SMA targeted the SMN2 gene with a tiny fragment of DNA called an anti-sense oligonucleotide (ASO), injected directly into the spinal fluid of 20 participating infants. This ASO gets absorbed into nerve cells of the spinal cord and brain, and promotes increased production of a critical protein that is deficient in babies with SMA. Not only was the series of nusinersen treatments delivered safely to these fragile babies, but in the majority of patients it was found to halt progression of the disease and in many cases improve motor function, sometimes enabling children to gain skills not seen in SMA Type 1 - sitting, rolling over and standing - as well as improving survival without dependence upon the continuous use of a ventilator. 

Cure SMA Provides Seed Funding for Nusinersen 

Starting in 2003, Cure SMA provided the seed funding needed to begin investigation into this therapeutic approach. The intellectual property generated with our funding was then licensed to Ionis Pharmaceuticals to create nusinersen. 

Cure SMA would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals. We specifically thank Dr. Adrian Krainer and his colleagues at CSHL for years of dedication to and hard work on the preclinical development of nusinersen for SMA, and Drs. Ravindra Singh and Elliot Androphy for their work funded by Cure SMA in originally identifying the ISSN1 gene sequence, which is the sequence targeted in nusinersen. In addition, Drs. Krainer and Androphy are long standing members of the Cure SMA Scientific Advisory Board.

Topics: Clinical Trials, Research, Front Page News

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