According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of two and five indicates that the treatment could have significant benefits for patients in this age range. The drug, which was approved earlier this year, is currently approved for patients that are younger than two years.
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy is a type of neuromuscular disorder in which the motor neurons are destroyed, leading to muscle wasting. Without prompt treatment, the disease is lethal in many cases. This disorder is linked to genetic defects of the SMN1 gene. This gene encodes a protein called SMN, and when not present in certain amounts, neurons are unable to function. There are different kinds of spinal muscular atrophy that are categorized by when symptoms first appear. These symptoms may include loss of reflexes, muscle weakness and poor muscle tone, problems with feeding and swallowing, developmental delays, respiratory muscle weakness, tongue twitching, and a bell shaped torso. The most effective treatment currently available for the disease is called Zolgensma. To learn more about spinal muscular atrophy, click here.
The clinical trial, which has so far enrolled 31 patients with the type 2 form of the disorder, utilized the Hammersmith motor function scale-expanded in order to measure changes in motor function following the administration of the gene therapy. Patients saw a median score increase of 5.9 points after being treated.
While the impact of the treatment may be difficult to understand based on results from a measurement scale, the difference could allow patients to stand and walk that would not have been able to without treatment. While the final results of the study upon its completion will be more meaningful as to the future of spinal muscular atrophy treatment, it can’t be denied that these interim findings are encouraging. Positive final outcomes could ultimately lead to the expanded approval of Zolgensma for older patients, increasing the number of people that can potentially benefit.
Gene therapies like Zolgensma have a drawn a lot of attention because of their potential to revolutionize the treatment of many genetic disorders. With data like this coming out, it’s difficult to ignore the capabilities that these kinds of treatments can have.