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New gene therapy pumps the brakes on MS

New gene therapy pumps the brakes on MS Gene therapy and a T cell-blocking medication could be the key to reversing MS.

University of Florida researchers have found a way to stop mouse-specific multiple sclerosis in its tracks. They believe the treatment could help humans if their solution makes it to clinical trials.

Using a gene therapy method in conjunction with existing medication, the researchers were able to stop or even reverse MS among the study mice. They also saw near-complete remission in the subjects.

How researchers made their findings
Although the cause of MS is unclear, current literature cites the condition as an autoimmune disease, meaning the immune system mistakenly targets healthy parts of the body. With MS specifically, the attack affects the myelin sheath surrounding nerve fiber and the actual fibers.

"Researchers saw a prevention and reversal of muscular dystrophy."

To combat this action, the UF team gave the mice a nonthreatening adeno-associated virus. This virus then delivered a brain protein, myelin oligodendrocyte glycoprotein, to the subjects' livers, which in turn activated T cell production to slow the immune response.

Certain mice also received rapamycin. This drug was suitable for the study because it helps regulate T cells to allow only regulatory ones to work and inhibits the effector variation. Typically, rapamycin aids in organ transplants and heart stent procedures.

Through their method, the researchers discovered the following:

  • During a seven-month observation period, mice treated with the gene therapy appeared to be disease-free.
  • With only gene therapy, the UF team saw a prevention and reversal of muscular dystrophy. 
  • Among one study group, a mouse required only eight days following a single treatment to see symptom reversal.
  • Control group mice showed neurological symptoms in as little as two weeks.
  • With the combination of rapamycin and gene therapy, the majority of mice who had hind-limb paralysis reached near-complete remission.

How the study can help humans
Hundreds of thousands of Americans have MS, with 10,000 new cases each year. According to the UF team, the global figure of diagnosed individuals is 2.3 million. Given the symptoms associated with the condition, including fatigue, limb numbness and pain, the combination of the studied gene therapy and rapamycin could present a life-changing treatment for countless MS patients.

"If we can provide long-term remission for people and a long-term quality of life, that is a very promising outcome," said Brad E. Hoffman, University of Florida College of Medicine assistant professor in the departments of pediatrics and neuroscience.

The researchers have a few steps before they can start clinical trials with humans. Their future preclinical examinations need to first examine all of the proteins involved in MS. Additionally, they don't have all the details about how the gene therapy affects liver T cells beyond the stimulation aspect.

Currently, MS treatment options include prescription medication, lifestyle adjustments and therapies, and some patients have symptoms that require these intervention techniques on a frequent basis. If the UF researchers are successful, all MS sufferers could return to a normal quality of life.

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