Multiple sclerosis can be inhibited or reversed using a novel gene therapy technique that stops the disease’s immune response in mouse models, University of Florida Health researchers have found.
By combining a brain-protein gene and an existing medication, the researchers were able to prevent the mouse version of multiple sclerosis. Likewise, the treatments produced near-complete remission in the animal models. The findings, which researchers said have significant potential for treating multiple sclerosis and other autoimmune disorders, are published today (Sept. 21) in the journal Molecular Therapy.
Multiple sclerosis affects about 2.3 million people worldwide and is the most common neurological disease in young adults. The incurable disorder starts when the immune system attacks the myelin sheath surrounding nerve fibers, making them misfire and leading to problems with muscle weakness, vision, speech and muscle coordination.
The researchers used a harmless virus, known as an adeno-associated virus, to deliver a gene responsible for a brain protein into the livers of the mouse models. The virus sparked production of so-called regulatory T cells, which suppress the immune system attack that defines multiple sclerosis. The gene was targeted to the liver because it has the ability to induce immune tolerance.
“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,” said Brad E. Hoffman, Ph.D., an assistant professor in the departments of pediatrics and neuroscience at the University of Florida College of Medicine.
The protein, myelin oligodendrocyte glycoprotein, was found to be effective in preventing and reversing muscular dystrophy on its own. A group of five mouse models that received the gene therapy did not develop experimental autoimmune encephalomyelitis, which is the mouse equivalent of multiple sclerosis in humans. In another experiment, all but one mouse model showed a significant reversal of the disease eight days after a single gene therapy treatment.
Hoffman said he was also encouraged by the treatment’s longevity. After seven months, the mouse models that were treated with gene therapy showed no signs of disease, compared with a group of untreated mouse models that had neurological problems after 14 days.
When the protein was combined with rapamycin — a drug used to coat heart stents and prevent organ transplant rejection — its effectiveness was further improved, the researchers found. The drug was chosen because it allows helpful regulatory T-cells to proliferate while blocking undesirable effector T-cells, Hoffman said.
Among the mouse models that were given rapamycin and the gene therapy, 71 percent and 80 percent went into near-complete remission after having hind-limb paralysis. That, Hoffman said, shows the combination can be especially effective at stopping rapidly progressing paralysis.
While researchers have established how gene therapy stimulates regulatory T cells in the liver, Hoffman said little else is known about the detailed mechanics of how that process works.
Before the therapy can be tested in humans during a clinical trial, further research involving other preclinical models will be needed, 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 Latest on: Multiple Sclerosis
- GoFundMe page to get multiple sclerosis sufferer Richard Johns lifesaving back-up generator on February 6, 2019 at 4:32 pm
A GoFundMe page has been created to raise money for a potentially life-saving back-up generator for Adelaide multiple sclerosis sufferer Richard Johns. As revealed on Advertiser.com.au yesterday, Mr J... […]
- Six factors that drive a multiple sclerosis patient's choice of disease-modifying therapy on February 6, 2019 at 3:12 am
MS advocate Trishna Bharadia explains the numerous factors that lead patients to choose a disease-modifying therapy, including the important considerations healthcare professionals and pharma often fo... […]
- Lyfebulb and Celgene Announce Call for Innovation Challenge Applications to Address Unmet Needs in Multiple Sclerosis on February 5, 2019 at 4:58 am
NEW YORK, Feb. 5, 2019 /PRNewswire/ -- Lyfebulb, a chronic disease-focused, patient-empowerment platform that connects patients with industry and investors to support user-driven innovation, and Celge... […]
- Bike rally marks Multiple Sclerosis Day in India on February 5, 2019 at 4:09 am
A bike rally was organised by the Delhi Chapter of Multiple Sclerosis Society of India to mark the 'India MS Day'. It was flagged off by the Disability Commissioner Mr Daniyal at Pitampura. […]
- EPA-Registered Herbicide Found to Trigger Inflammation Linked to Onset of Multiple Sclerosis on February 4, 2019 at 8:04 pm
(Beyond Pesticides, February 5, 2019) Linuron, an herbicide registered for use by the U.S. Environmental Protection Agency (EPA), yet recently banned in Europe due to health concerns, appears to trigg... […]
- Secondary osteoporosis, rheumatoid arthritis improve FRAX accuracy in multiple sclerosis on February 4, 2019 at 6:29 am
“Our study extends the range of clinical conditions where FRAX has been shown to be of value for assessing fracture risk, but it may underestimate that risk in people with multiple sclerosis (MS),” Wi... […]
- Global Multiple Sclerosis Dynamic Market Forecast to 2026: Two Additional Anti-CD20 mAbs will Compete Against Blockbuster Ocrevus on February 4, 2019 at 6:20 am
Dublin, Feb. 04, 2019 (GLOBE NEWSWIRE) -- The "Multiple Sclerosis: Dynamic Market Forecast to 2026" report has been added to ResearchAndMarkets.com's offering. Multiple Sclerosis (MS) is a rapidly ... […]
- Woman with multiple sclerosis speaks out after dog feces smeared across car over handicap placard on January 30, 2019 at 1:24 pm
A woman believes she was targeted for her invisible disability. Shellie Nichol Chandar was diagnosed with multiple sclerosis in 2012. She told ABC7 News her symptoms have recently grown worse. […]
- This May Be a Good Time to Have Multiple Sclerosis on January 30, 2019 at 7:42 am
I am fortunate to be a part of this time in history. After all, who knows? A cure for multiple sclerosis (MS) might be around the corner. As the world changes — with new generations, new technology, a... […]
- Biogen beats estimates on multiple sclerosis drugs sales on January 29, 2019 at 9:10 pm
(Reuters) - Biogen Inc beat analysts’ estimates for fourth-quarter profit and revenue on Tuesday, buoyed by higher sales of its top-selling multiple sclerosis drugs, and forecast full-year profit ahea... […]
via Google News and Bing News