Gene therapy prevents, even reverses, MS in animal model – new research

Multiple sclerosis can be inhibited, or even reversed, according to University of Florida researchers. They used a novel gene therapy technique that, they claim, stops the disease’s immune response in mouse models.

Obviously, if that can be reproduced in humans, it has the potential to be wonderful news.

The researchers paper is “Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis”. Molecular Therapy published it last week.

gene therapyBy combining a brain-protein gene and an existing medication, researchers were able to prevent the mouse version of multiple sclerosis. Likewise, the treatments produced near-complete remission in the animal models.

Researchers said that their findings have significant potential for treating MS and other autoimmune disorders.

The paper says researchers used an adeno-associated virus, to deliver a brain protein gene into the livers of mouse models. The virus sparked production of so-called Tregs (regulatory T cells), which suppress the immune system attack that defines multiple sclerosis. The gene targeted the liver because it has the ability to induce immune tolerance.

Brad E Hoffman PhD is an assistant professor in pediatrics and neuroscience at the University of Florida College of Medicine. He said: “Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis.”

Encouraged by longevity of gene therapy

Hoffman says he Is encouraged by the longevity of the gene therapy. Mouse models treated with gene therapy showed no signs of disease after seen months. He compared It with a group of untreated mouse models that had neurological problems after 14 days.

The protein was combined with rapamycin — a drug used to coat heart stents and prevent organ transplant rejection — and researchers found its effectiveness was further improved.

We chose this drug because it allows helpful regulatory T-cells to proliferate while blocking undesirable effector T-cells, Hoffman said.

Further research is needed before tests of the therapy in humans are carried out in a clinical trial, Hoffman said. Researchers also need to target the full suite of proteins that are implicated in multiple sclerosis, he added.

Still, Hoffman said he is extremely optimistic that the gene therapy can be effective in humans.

“If we can provide long-term remission for people and a long-term quality of life, that is a very promising outcome,” he said.

The research was funded by grants from the National Multiple Sclerosis Society, the National Institutes of Health and the Children’s Miracle Network.

My view is that while it is still early days, the results so far seem very encouraging. I look forward to learning about further developments as time progresses.

* * * * *

Affiliate disclaimer: This affiliate disclosure details the affiliate relationships of MS, Health & Disability at with other companies and products. Read more.

* * * * * is the personal website of Ian Franks, a Clinical Writer with Healthline, the fastest growing health information site. He enjoyed a successful career as a journalist, from reporter to editor in the print media. He gained a Journalist of the Year award in his native UK. Ian received a diagnosis of MS in 2002 and now lives in the south of Spain. He uses a wheelchair and advocates on mobility and accessibility issues.


Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s