ALS (Amyotrohic Lateral Sclerosis) -- also know as Lou Gehrig's disease -- represents one of the most challenging disorders for neurologists. Despite the efforts of the best minds in medical research, there is still no cure for ALS. In many cases, the disease is rapidly progressive. In fact, once the diagnosis is made, only half of ALS patients will survive for two years.
There is one FDA-approved treatment, riluzole, but as any treating physician will admit, it has little effect on the disease progression and is often associated with serious side effects. Unfortunately, riluzole is about all most patients will ever hear about. But there are other ideas out there that may play a powerful role in treating ALS, and in my opinion, it's time to get the word out.
As it turns out, more than a decade ago a neurologist, Dr. Benjamin Brooks, at the University of Wisconsin in Madison happened to be treating a woman with ALS who, in addition, was being treated for breast cancer with the drug tamoxifen. Dr. Brooks observed that the patient's ALS appeared to be taking an extremely slow course.
Thereafter, based upon Dr. Brooks' observations, several researchers demonstrated that tamoxifen was clearly extending the survival in laboratory mice that expressed the disease. Human trials soon followed, conducted by Brooks and other researchers, and the results clearly demonstrated the prolongation of survival with only minimal side effects -- the most common being hot flashes.
The mechanism by which tamoxifen helps ALS patients has not been fully elucidated as of yet, but it is known that the drug is a neuroprotectant -- as has been demonstrated in animal models of stroke and oxygen deprivation. But despite the fact the scientists haven't fully worked out exactly how tamoxifen works in ALS, a number of institutions, including the Massachusetts General Hospital, have been so impressed by tamoxifen's potential that they are now moving ahead with large-scale studies of even higher doses of the medication than those used in the original research.
Unfortunately, the results of these studies may not be available for several years; way too long to wait for patients now suffering from the disease.
Those of us treating ALS patients frequently, have the experience of having patients look at us with desperate, pleading eyes and asking: "Doc, is there anything out there that you think could help me?"
In the humble opinion of this writer, a neurologist treating ALS patients -- based on the available evidence -- it is reasonable and compassionate to offer tamoxifen therapy to our ALS patients right now with a full explanation that the current level of research isn't totally conclusive. Criticism could be raised by saying that the use of tamoxifen for ALS "isn't FDA approved," but tamoxifen is indeed an FDA approved medication, and FDA approved drugs are widely employed in "off label" applications by many if not most physicians.