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New ALS gene therapy could slow progress of ALS

July 26, 2006

Researchers at the Center for Neurologic Study in Cleveland say that a buildup of toxic mutant proteins is what causes many degenerative neurologic diseases such as motor neuron disease (MND) and amyotrophic lateral sclerosis (ALS).

"Limiting mutant damage to microglia robustly slowed the disease's course, even when all motor neurons were expressing high levels of an SOD1 mutant," the researchers wrote in the June 2nd issue of the journal Science.

"Our research suggests that what starts ALS and what keeps it going are two separate phases. It also suggests that, with the right therapy, ALS could become a manageable, chronic disease."

The damaged proteins in inherited ALS is the protien SOD1, and the researchers have developed a molecule called an "antisense" oligonucleotide and is delivered through the cerebrospinal fluid and silences the gene that codes for the protein, turning off SOD1 production; thus helping to manage ALS and MND.

In ALS, SOD1 kills the motor neurons that run from the brain to the muscles, the mechanisms that create/facilitate movement.

The gene therapy used in the study works through the entire nervous system, but the team discovered that the parts that needed it most were not the motor neurons, but the neuron helper cells in the spinal cord called microglia, which travel through nerve tissue and nourish the nerve tissue and remove debris.

The gene theraphy hopes to reach ALS patients for phase one in 2007.