BOSTON — A drug commonly prescribed to reduce the risk of severe side effects in gene therapy may inadvertently weaken the therapy’s effectiveness, according to a new study.
The trial, conducted by the biotech startup Encoded Therapeutics, evaluated a gene therapy designed to treat Dravet syndrome, a severe and often debilitating form of genetic epilepsy. A major challenge in gene therapy is the potential for patients to develop an immune response against the engineered viruses used to deliver therapeutic genes into the brain.
In the study, Encoded Therapeutics assigned most of the 21 participating children to receive steroids, the standard immune suppressant used to mitigate such reactions. However, a subset of patients—including those receiving the highest dose of gene therapy—were also given sirolimus (also known as rapamycin), a drug typically prescribed to prevent organ transplant rejection.
The findings suggest that while sirolimus may help control immune responses, it could also interfere with the gene therapy’s ability to deliver its intended therapeutic effect.
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