A clinical trial for NGN-401, a gene therapy designed to treat Rett syndrome, recently faced a setback when a young participant experienced a severe immune reaction after receiving a high dose of the treatment. This reaction, called systemic hyperinflammatory syndrome, is a rare and dangerous immune response, causing her condition to become critical. As a result, high-dose treatments have been paused.
Rett syndrome is a genetic disorder that primarily affects girls, leading to severe developmental delays, loss of motor skills, and difficulties with communication. Life expectancy for individuals with Rett syndrome is often reduced, with many living into their 40s or 50s, but this can vary depending on the severity of the condition and the presence of complications.
Learn more about Rett Syndrome through the story of Kerrigan, a patient with Rett syndrome at St. Louis Children's Hospital.
Media source: https://www.youtube.com/watch?v=FFIAlkQ2Vq8
Rett syndrome occurs due to mutations in the MECP2 gene, which disrupts the normal development of the brain. NGN-401 works by delivering a healthy version of the MECP2 gene directly into the brain using a modified virus (AAV). The trial showed promise when low doses were used, with participants showing improvements in skills like swallowing, sleep, and constipation.
Despite this setback, Neurogene, the company behind NGN-401, continues the trial with a lower dose after consulting with the FDA. They have expressed deep concern for the affected family and emphasized the importance of patient safety as they move forward with the trial.
This case highlights the challenges faced in gene therapy, where promising treatments must be balanced with ensuring the safety of patients, especially in vulnerable populations like those with Rett syndrome.
Created: Nov 22 th, 2024
Citations:
Bryson, S. (2024, November 20). Rett girl has severe immune reaction to high dose of NGN-401. Rett Syndrome News.
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