Sunday, August 8, 2010

Thomas Edison said ...

When discussing the light bulb, "I have not failed. I have successfully discovered ten-thousand ways that won't work."

He also said, "I am not discouraged, because every wrong attempt discarded is another step forward."

In my interviews with young researchers last year, I asked what keeps them going when some potential treatment they have been working on for months, or even years, does not work in humans. Even though their answers were not the same as Edison's quote above, they were all similar. They had discovered something that does not work, so they can rule that out and get back to the drawing board.

Last week the clinical trial (either Phase III or IV) results for "leuprorelin" were published. The trial was 48-weeks in length and was designed to determine the efficacy (capacity or power to produce a desired effect) and safety of leuprorelin on patients with Spinal Bulbar Muscular Atrophy (Kennedy's Disease). In an earlier trial using this same drug, there appeared to be some swallowing benefits from using the drug.

199 patients were involved in this double-blind placebo-controlled trial with 100 taking leuprorelin. The results were "treatment with leuprorelin did not show significant effects on swallowing function in patients with spinal and bulbar muscular atrophy, although it was well tolerated. Disease duration might influence the efficacy of leuprorelin and thus further clinical trials with sensitive outcome measures should be done in subpopulations of patients."

I guess this is why I never became a scientist or researcher. I want to fix something, but I do not want to go through a hundred, let alone thousands of iterations to find the solution. If you have read my articles, then you know that I am very respectful of the work researchers do. Patience, yes one of my favorite words, is a virtue and these researchers continue to amaze me with the abundance of patience they exhibit.

It seems like whenever I push for a potential date when the latest wonder drug will be ready for a clinical trial, the answer is something similar to "we hope soon, but most likely we are still one-to-two years out." For those of us living with Kennedy's Disease, one or two years more seems like an eternity. For the researcher, however, it is just another phase in a long process that might end up proving to be another drug that does not work. Yet, they continue on still hoping for that breakthrough.

Once again I want to say "Thank You" to all those men and women out there working to find a treatment or cure for Kennedy's Disease. They are our hope for a future where we are free from this disease.

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