Research &
Development

Science

Diseases

Neurodegenerative diseases as a whole represent a relatively scarcely touched frontier in medicine. Unfortunately, because they principally involve the brain and spinal cord, the neurodegenerative disorders are not as well understood as many other diseases, such as the various forms of cancer, infectious diseases, and many others. The primary reason for this situation is that the central nervous system is not as easily accessed as every other part of the body. Of paramount importance is the fact that biopsies, which are commonly taken from most areas of the body, are taken from the brain and spinal cord only rarely. In fact, human samples of these organs are most often taken after death. Unfortunately, biopsy specimens provide significant opportunities for research, including the elucidation of information about physiological and pathological processes.

For example, as many as 30,000 people have ALS in this country, and many more outside the United States. Average survival in ALS is 3 – 5 years from date of diagnosis. Loss of function and resulting debilitation can occur rapidly, resulting in rapid loss of quality of life. People with ALS lose their ability to walk, use their hands, eat and speak, and eventually become dependent on respirators. The situation puts considerable strain on patients and their families and friends, and a treatment for the disease is urgently needed.

Additionally, treatment options are extremely limited in ALS. Rilutek, the sole FDA-approved product for this disease, is of marginal benefit, and many ALS patients do not take it, due to expense, side-effects, and limited, if any, benefits. The vast majority of therapeutic options for people with ALS are supportive, such as tools for assisted ventilation and physical therapy. The situation is even worse in Huntington’s disease, for which there are no therapeutic options at all.