Advancing Prevention and Treatment of Multiple Sclerosis

“It is remarkable that a protein that unwinds RNA is a central player in
how we recognize our cells as our own, not to be confused with
invading pathogens,” Garcia-Blanco said.

He noted that the new understanding could help lead to better, more
targeted treatments: “While there are effective treatments for
multiple sclerosis and other autoimmune diseases, most of these lead
to general suppression of the immune system and makes patients
susceptible to infections or incapable of responding well to
vaccines.”

Understanding Multiple Sclerosis

Multiple sclerosis is a potentially disabling autoimmune disorder in which the
immune system begins to attack the sheath-like coverings that protect
our nerves. The damage interrupts the nerves’ ability to transmit
communications through the body.

This leads to symptoms such as muscle weakness and stiffness,
spasms, fatigue, numbness, and difficulty moving. The
disease is estimated to affect nearly a million Americans and almost
3 million people worldwide.

The new work from Garcia-Blanco and his collaborators sheds important
light on how our immune systems are calibrated to prevent MS and
identify several key places where things might go wrong.

For example, the researchers conclude that the master gene they
identified, DDX39B, is an “important guardian of immune tolerance.”

Game-Changing Finding

This means that it helps keeps the body’s immune response working at the
appropriate levels so that the immune system doesn’t begin to
attack the body’s own cells – as is the case in MS and other
autoimmune diseases.

This master gene, the researchers found, directs the activity of
another gene critical in the production of important immune cells
called T regulatory cells (Tregs) previously linked to MS.
This second gene, FOXP3, is already known to play a critical role in
autoimmune disorders.

These new insights into how the immune system functions, or should
function, help doctors and scientists better understand the
underlying causes of multiple sclerosis and give them attractive
targets in their efforts to develop new treatments and preventive
measures.

Advancing Multiple Sclerosis Care

“In cases of autoimmune diseases, we would want to activate DDX39B with
small-molecule agonists, for which there is strong pre-clinical
precedent,” said Chloe Nagasawa, a graduate student with
Garcia-Blanco and second author of the new scientific paper outlining
the findings.

“Multiple sclerosis takes a massive toll on patients and society, affecting
disproportionately young women, and to date, there is no cure. We
believe that a basic understanding of molecular mechanisms
underpinning immune tolerance will open paths to truly targeted
therapy.”

Reference :

1. The RNA helicase DDX39B activates FOXP3 RNA splicing to control T
   regulatory cell fate – (https://elifesciences.org/articles/76927)

Source: Eurekalert