The New England Journal of Medicine has shared early results from a phase 1 trial of a gene therapy approach that appears to help some patients with progressive multiple sclerosis move more freely.
A video posted by the New England Journal of Medicine (@NEJM) shows a patient with progressive multiple sclerosis (MS) — a form of the condition where disability worsens steadily over time — attempting to walk before and after receiving an experimental treatment called JY231. The difference between the two clips is visible.
JY231 is described by the journal as an off-the-shelf lentiviral vector — in plain terms, a modified virus used to deliver genetic instructions — designed to engineer the patient’s own immune cells inside the body, without removing them first. The goal is to produce what are known as CAR T cells that target CD19, a protein found on certain immune cells thought to drive MS damage. This approach, called direct in vivo (inside the body) engineering, differs from existing CAR T cell therapies, which require cells to be extracted, altered in a laboratory, and reinfused.
These are preliminary results from a phase 1 trial, meaning the study is at an early stage focused primarily on safety rather than proving the treatment works at scale. The findings have been published in the New England Journal of Medicine as a phase 1 study of 16 patients, with researchers noting that larger-scale trials will be needed to confirm safety and effectiveness.
According to the NHS, over 150,000 people in the UK live with MS (a figure that reflects more recent NHS-linked and MS charity estimates, superseding an earlier NHS figure of around 130,000), and progressive forms of the condition currently have limited treatment options compared with relapsing MS. Any new approach that could slow or reverse disability would be of considerable interest to patients and neurologists alike — but researchers and clinicians routinely caution that phase 1 results, however striking on video, do not confirm a treatment is safe or effective for wider use.
Kent residents with concerns about MS symptoms or existing diagnoses should contact their GP or call NHS 111 for guidance.
Source: @NEJM