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Assessing ALS treatments

assessing_als_treatment.pngWhen testing a new treatment, researchers must assess whether it is improving symptoms. Taking advantage of the capabilities within the Harvard biomedical research community, Harvard NeuroDiscovery Center members developed a technology to detect small changes in disease progression in patients with amyotrophic lateral sclerosis (ALS).

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ALS strikes motor neurons, nerve cells that reach from the brain... to the spinal cord... and to muscles throughout the body. As motor neurons degenerate, they send fewer and fewer impulses directing muscles to move.  

Until now, scientists could quantify the number of remaining motor neurons in the muscles of ALS patients, but not how well they were functioning. With support from the Harvard NeuroDiscovery Center, Lisa Krivickas and colleagues at Spaulding Rehabilitation Hospital developed a method to detect impulses from these motor neurons and translate the signals into a measurement of function. After further refinement, the technology will provide a more accurate measure of ALS severity. It may also be used to assess whether emerging therapies are helping patients.

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The challenge of neurodegenerative diseases

Neurodegenerative diseases occur when nervous system cells (neurons) in the brain and spinal cord begin to deteriorate. Changes in these cells cause them to function abnormally and eventually result in the cells' demise. As neurons deteriorate, an individual may first experience relatively mild symptoms — problems with coordination or remembering names. But as huge numbers of neurons die, symptoms progressively worsen. In some cases, patients lose the ability to walk independently, think clearly, or generally function in the world. Ultimately, many of these diseases are fatal.

Today, 5 million Americans suffer from Alzheimer's disease; 1 million from Parkinson's; 400,000 from multiple sclerosis (MS); 30,000 from amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease), and 30,000 from Huntington's disease. Because neurodegenerative diseases strike primarily in mid- to late-life, the incidence is expected to soar as the population ages. (By 2030, as many as 1 in 5 Americans will be over the age of 65.) If left unchecked 30 years from now, more than 12 million Americans will suffer from neurodegenerative diseases.  Finding treatments and cures for neurodegenerative diseases is a goal of increasing urgency.

In recent years, scientists of the Harvard NeuroDiscovery Center and many others have made great strides in understanding the underlying causes of neurodegenerative diseases. As we uncover more details of these diseases, we are confident that in time we will be able to treat and prevent them. But as for most major endeavors, the greater the resources we bring to bear, the faster the progress toward our goal.  If — as a community — we want to avoid the looming and dramatic impact of these diseases on our increasingly elderly population, we must all redouble our efforts, now.