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Gene Therapy Found To Prevent MS ,Reverse Resulting Paralysis In Mice

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system that disrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS.

Now, in a similar pursuit, researchers at the University of Florida, took a novel approach to turn off immune attacks in mice with an MS-like disease. They have reportedly managed to prevent a mouse version of MS by combining a brain-protein gene. The treatment produced near-complete remission in the animal models.

Delivering regulatory T cells, or Tregs, to an MS patients is an attractive way to rein in the self-destructive nature of the effector T cells involved in the ailment, but this method is not particularly long-lasting. Therefore, the team of researchers used an adeno-associated virus to transfer the protein myelin oligodendrocyte glycoprotein to the liver in mouse models of MS.

This treatment stimulated Tregs to curb self-reactive effector T cells, thereby protecting myelin and preventing mice from

developing MS symptoms for seven months. It also reversed mild and moderate neurological symptoms, and restored mobility in mice with hind-leg paralysis.

Mice that were treated with the therapy were seen to not develop the disease. They also had higher numbers of Tregs in their blood. This supported the notion that the therapy works by harnessing the tolerogenic mechanisms that prevent an immune response.

Much more importantly, the therapy could reverse disease, the researchers discovered. The treatment even improved walking ability in mice with severe paralysis of their hind legs. Tests in earlier disease stages showed equally good results.

The effects were also durable, researchers said. Treated animals lived for seven months without showing signs of disease, while all the control mice had neurological problems after two weeks.

Another round of tests found that the therapy was even more effective if the protein was combined with a drug called rapamycin. Often used to prevent patients’ bodies from rejecting transplanted organs, rapamycin works by increasing the numbers of regulatory T cells and block other aggressive types of T cells.

The researchers tested the combination of the gene therapy and rapamycin on two groups of mice that had had their hind legs paralyzed by the disease. Sure enough, 71 percent of the mice in one group and 80 percent in the other were almost completely healed.

While researchers have established how gene therapy stimulates regulatory T cells in the liver, Brad E. Hoffman, co-author of the study 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.

We have demonstrated that stable immune tolerance can be re-established and that active disease can be stopped and clinical symptoms reversed using our gene immunotherapy, especially during early onset of disease,” Hoffman said. “Even though these studies were performed in a less complex mouse model, the data suggest this may be a potential therapy in humans with additional optimization.

Hoffman expects clinical trials to start in three to five years and if everything goes well, the treatment could go to market in 10 years.

If we can provide a long-term treatment for these patients that have very little side effects and they have a higher quality of life, that would be a successful result for me,” Hoffman said.

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