John Brandsema, MD, on Balancing Risks and Rewards With Muscular Dystrophy Gene Therapy
The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed how Sarepta’s Elevidys has affected the landscape of care for DMD.
“While the label is broad and is for everyone we don't necessarily have the data to back that up yet and that makes the conversation more nuanced. We also aren't sure about the durability of transgene expression. Muscle is a high turnover tissue, and therefore whether this is going to be a lifelong effect is still up in the air; although it does seem, from the early boys treated that there is a measurable difference in their trajectory years after the dose, up to 5 or 6 years at this point now.”
Over the past 5 years or so, the treatment landscape for Duchenne muscular dystrophy (DMD) has rapidly evolved with the arrival of a number of new disease-modifying therapies that have been approved by the FDA. Notably, just last year, Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus (AAV) vector-based gene therapy, was approved by the FDA, adding to other options available to patients such as exon-skipping therapies. As such, the decision-making process for treatment for DMD for patients, families, and doctors is now more complex than ever.
In the lead up to World Duchenne Awareness Day, which was held on September 7, CGTLive® reached out to John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, to get his view on the newly emerging landscape of care for DMD and the real-world experience gained with Elevidys in the time since it’s approval a bit more than a year ago. Brandsema briefly discussed the other treatment options for DMD that have come onto the scene in the past 5 years, and then brought the focus to Elevidys, and spoke about some of the distinct advantages and unique risks that it carries as the only AAV vector-based gene therapy currently approved for DMD. He emphasized the need for more long-term data, especially for older patients, as many patients treated thus far have been on the younger end of the spectrum.
The Challenges of Gene Therapy Approaches in Advanced Muscular Dystrophy
September 13th 2024Kevin Campbell, PhD, a Howard Hughes Investigator at the University of Iowa, discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.
