Amyotrophic Lateral Sclerosis (cont.)
What research is being done?
The National Institute of Neurological Disorders and
Stroke, part of the National Institutes of Health, is the Federal Government's leading supporter of
biomedical research on ALS. The goals of this research are to find the cause or
causes of ALS, understand the mechanisms involved in the progression of the
disease, and develop effective treatment.
Scientists are seeking to understand the mechanisms that
trigger selective motor neurons to degenerate in ALS and to find effective
approaches to halt the processes leading to cell death. This work includes studies in animals to
identify the means by which SOD1 mutations lead to the destruction of neurons.
The excessive accumulation of free radicals, which has been implicated in a
number of neurodegenerative diseases including ALS, is also being closely
studied. In addition, researchers are examining how the loss of neurotrophic
factors may be involved in ALS. Neurotrophic factors are chemicals found in the
brain and spinal cord that play a vital role in the development, specification,
maintenance, and protection of neurons. Studying how these factors may be lost
and how such a loss may contribute to motor neuron degeneration may lead to a
greater understanding of ALS and the development of neuroprotective strategies.
By exploring these and other possible factors, researchers hope to find the
cause or causes of motor neuron degeneration in ALS and develop therapies to
slow the progression of the disease.
Researchers are also conducting investigations to
increase their understanding of the role of programmed cell death or apoptosis
in ALS. In normal physiological processes, apoptosis acts as a means to rid the
body of cells that are no longer needed by prompting the cells to commit "cell
suicide." The critical balance between necessary cell death and the maintenance
of essential cells is thought to be controlled by trophic factors. In addition
to ALS, apoptosis is pervasive in other chronic neurodegenerative conditions
such as Parkinson's disease and Alzheimer's disease and is thought to be a major
cause of the secondary brain damage seen after stroke and trauma. Discovering
what triggers apoptosis may eventually lead to therapeutic interventions for ALS
and other neurological diseases.
Scientists have not yet identified a reliable biological
marker for ALS—a biochemical abnormality shared by all patients with the
disease. Once such a biomarker is discovered and tests are developed to detect the marker in
patients, allowing early detection and diagnosis of ALS, physicians will have a
valuable tool to help them follow the effects of new therapies and monitor
disease progression.
NINDS-supported researchers are studying families with
ALS who lack the SOD1 mutation to locate additional genes that cause the
disease. Identification of additional ALS genes will allow genetic testing
useful for diagnostic confirmation of ALS and prenatal screening for the
disease. This work with familial ALS could lead to a greater understanding of
sporadic ALS as well.
Because familial ALS is virtually indistinguishable from sporadic ALS
clinically, some researchers believe that familial ALS genes may also be
involved in the manifestations of the more common sporadic form of ALS.
Scientists also hope to identify genetic risk factors that predispose people to
sporadic ALS.
Potential therapies for ALS are being investigated in animal models. Some of
this work involves experimental treatments with normal SOD1 and other
antioxidants. In addition, neurotrophic factors are being studied for their
potential to protect motor neurons from pathological degeneration. Investigators
are optimistic that these and other basic research studies will eventually lead
to treatments for ALS.
Results of an NINDS-sponsored phase III randomized, placebo-controlled trial
of the drug minocycline to treat ALS were reported in 2007. This study showed
that people with ALS who received minocycline had a 25 percent greater rate of
decline than those who received the placebo, according to the ALS functional
rating scale (ALSFRS-R).
Next: How Can I Help Research on ALS? »
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