Tuesday, June 15, 2010

Because Someone Has To

A few years ago a report came out claiming some fantastic results involving Lithium's efficacy in ALS. The paper was published in a respectable journal. However, reaction was surprisingly cool. Because Lithium has been used for years in much higher doses for maintenance of bipolar disorder and was cheap and easy to get, many PALS started their own off-label "trial" which collected valuable data (unfortunately to the contrary of the results of the original study). This first in history patient-driven trial forced multiple professional clinics to stage real, placebo controlled, clinical trials (with results which matched the patient-driven trial). Recently there has been interest in a natural bile acid which has anti-apoptotic effects, specifically in the mitochondria. There are two companies already in clinical trials with proprietary substances intended for mitochondrial support. Ursodeoxycholic Acid (UDCA) is found to be anti-apoptotic, easily crosses the blood-brain barrier, has an excellent safety profile, and is inexpensive and easy to obtain. A Phase 1 trial was already done which showed excellent safety for ALS patients. A few scattered PALS have been safely using UDCA and some anecdotal reports from S. Korea (using a version with a starch binder) have indicated efficacy. Unfortunately no clinic is interested in performing the trials necessary to fully investigate UDCA. So I have created the second patient-driven clinical trial intended to provide some good data on the efficacy of UDCA as a treatment for ALS. While not a true clinical trial it should be a decent indicator. I did this because the risk is low and the potential benefit high. I did this because doing nothing gets you nothing. I did this because someone has to.

1 comment:

  1. I was one of the early participants in the patient-driven Lithium trials and stayed on it for well over a year and the results of the clinical trials were announced. This new patient-driven trial also strikes me since I am excluded from most of the clinical trials. Thanks, Eric, for taking this bull by the horns.

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