Tiny molecule slows progression of Lou Gehrig's disease in mice

Dec 10, 2009

Researchers at UT Southwestern Medical Center have found that a molecule produced naturally by muscles in response to nerve damage can reduce symptoms and prolong life in a mouse model of amyotrophic lateral sclerosis (ALS).

"We believe we can apply this research toward drug development," said Dr. Eric Olson, chairman of molecular biology at UT Southwestern and senior author of the study, which appears in the Dec. 11 issue of Science.

ALS, also known as , damages motor nerve cells that control muscles, leading to , paralysis and death. There is no treatment that can slow it, and no cure.

As ALS kills nerves, the muscles they control begin to wither.

The damaged muscles, however, can "re-innervate" themselves by prompting healthy nerves to send new branches their way, like limbs in a damaged hedge filling in a gap.

Dr. Olson said skeletal muscles produce a molecule called microRNA-206 (miR-206) to serve as a chemical signal to steer the new and maintain their interactions with muscles. But the research suggests that miR-206 can only work for so long. As nerves continue to die, there comes a point where the surviving nerves can no longer carry the load, and symptoms like muscle weakness appear.

"While miR-206 initially prompts nearby surviving nerves to send new branches to the muscles, it only delays the inevitable," Dr. Olson said. "Our findings correlate with the observation in ALS patients that the disease is nearly asymptomatic until a large fraction of has died, at which point the few remaining ones can't compensate sufficiently. These results provide a new perspective on the mechanisms of ALS," he said. "MiR-206 seems to sense nerve injury and promote regeneration.

"Because miR-206 only exists in , a drug based on it might not affect other tissues. That limits its risk of side effects and is a key part of its appeal as a potential therapy."

In collaboration with a company he co-founded, called miRagen Therapeutics, Dr. Olson is developing potential drugs based on miR-206.

Provided by UT Southwestern Medical Center (news : web)

Explore further: Early detection and transplantation provide best outcomes for 'bubble boy' disease

add to favorites email to friend print save as pdf

Related Stories

Research suggests new direction for ALS treatment

Nov 28, 2007

A research team from Wake Forest University School of Medicine is the first to show that injections of a protein normally found in human cells can increase lifespan and delay the onset of symptoms in mice with ALS (amyotrophic ...

Finding clues for nerve cell repair

Jun 03, 2008

A new study at the Montreal Neurological Institute at McGill University identifies a key mechanism for the normal development of motor nerve cells (motor neurons) - cells that control muscles. This finding is crucial to understanding ...

Recommended for you

Key to aging immune system is discovered

5 minutes ago

There's a good reason people over 60 are not donor candidates for bone marrow transplantation. The immune system ages and weakens with time, making the elderly prone to life-threatening infection and other ...

Putting a number on pain

25 minutes ago

"How much pain are you in?" It's a harder question than many people think. Tools for assessing patients' pain—be they children or adults—rely on perception: a subjective measure that eludes quantification ...

New infections cause dormant viruses to reactivate

35 minutes ago

The famous slogan is "A diamond is forever," but that phrase might be better suited to herpes: Unlike most viruses, which succumb to the immune system's attack, herpes remains in the body forever, lying in wait, sometimes ...

User comments : 0