麻豆传媒

Spinal muscular atrophy is a debilitating disease that causes weakness and wasting of the muscles. The disease ranges in severity with patients experiencing different symptoms, from the inability to sit up and stand, to trouble walking. In its severest form, the disease results in difficulty breathing and leads to death.

鈥淲e鈥檝e identified the enzyme JNK3 as a therapeutic target to treat the symptoms of spinal muscular atrophy,鈥� says , Ph.D., an associate professor in the Center of Emphasis in Neurosciences at (TTUHSC El Paso) who led the research.

In a recent published in the December 15th issue of Human Molecular Genetics, Dr. Gangwani and his team of researchers at TTUHSC El Paso describe how mice with spinal muscular atrophy saw great improvement when the JNK3 enzyme was genetically inhibited to eliminate its activity. The finding suggests that patients with spinal muscular atrophy could also see improvement if treated with a drug that suppresses the activity of JNK3.

鈥淲e saw less muscle degeneration, more muscle growth and better muscle strength, and improvement in overall movement,鈥� says Gangwani. 鈥淲hat鈥檚 more striking was a four-fold reduction in initial mortality period and a two-fold increase in total lifespan.鈥�

Spinal muscular atrophy is caused by a genetic mutation that triggers the loss of spinal cord motor neurons 鈥� neurons that help control the body鈥檚 movements. As the neurons deteriorate, so does one鈥檚 ability to control their own movement.

This same study, however, found that inhibiting JNK3 in mice prevented the loss of motor neurons, which is particularly important to prevent progression of disease, says Gangwani.

鈥淪o far, spinal muscular atrophy research has focused on targeting the genetic mutation to prevent degeneration of spinal motor neurons, but it hasn鈥檛 been successful because of challenges associated with gene therapy,鈥� he says. 鈥淭his is the first study done that identifies a target, JNK3, that鈥檚 independent of the genetic mutation of spinal muscular atrophy for novel therapeutic development.鈥�

The TTUHSC El Paso research team now plans to test pharmacological compounds that can inhibit JNK3 in mice to identify drug compounds that will slow the progression of the disease and reduce the overall burden of this illness in patients. They鈥檝e also decided to their idea of using a JNK3 inhibitor to treat spinal muscular atrophy.

Gangwani says, 鈥淛NK3 represents a promising new avenue of research for clinical advances in developing a treatment.鈥�