FAST-initiated Clinical Trial
I want to thank all those parents who applied for enrollment in the minocycline clinical trial. The clinical trial began on schedule April 16, 2012. We are now at a point where half of the patients have received the drug for eight weeks and are beginning to be seen for their follow up appointments. As we pass this critical point of the clinical trial, an update
and some reflection is in order. Parents and patients have found their way to Tampa, Florida, and, with the help of Joe Grieco, have gone through the initial evaluation without any significant problems. As expected, we have seen little adverse effects of the minocycline and are as anxious as the rest of the community to evaluate the results and make these results public. If the study proceeds as proposed, it will end in the middle of March 2013. This may seem like an eternity, but it is actually an incredibly truncated timeline in comparison to other clinical trials for Angelman syndrome. The entire team here at the University of South Florida will work to culminate, analyze, statistically test, and report our findings as quickly as possible after the conclusion of the study.
Many parents, clinicians, and other investigators have asked, “What are the actions of minocycline and what do you think it is doing in the brain?” This is an exceedingly difficult question to answer for the very reason that its action beyond its use as an antibiotic is not well characterized. Of all the tetracycline antibiotics, minocycline is the most lipid soluble. This means minocycline has the ability to deeply penetrate the brain. However, its actions at a molecular level may take a considerable amount of time to determine. It is important here to note that there is a precedent for the use of drugs without a clear knowledge of their exact mode of action. For example, lithium is a common chemical that has been used to treat schizophrenia and other neuropsychiatric disorders for decades without knowledge of exactly how this simple molecule works in the brain. Only recently has research begun to unravel the possible molecular mechanisms for lithium.
This brings up the question of fast-tracking research to identify and bring to clinical trial therapeutics for Angelman Syndrome. This strategy was used to identify minocycline. We are currently trying to understand the molecular mechanisms of minocycline and our results will be published in time. It is important to know that any clinical trial, despite whether it involves an FDA-approved drug or an experimental compound, must undergo a rigorous and comprehensive review by the FDA, a scientific review committee, departmental review (an institutional requirement), and an institutional review board (IRB). The minocycline clinical trial has undergone review from all of the above-mentioned regulatory bodies as well as peer review by physician scientists outside of the University of South Florida. While there may be some controversy involved in deciding how much information should be known prior to conducting a clinical trial, you can rest assured the minocycline clinical trial has undergone significant review to ensure the highest safety level for the children involved. The mechanism of the drug and its use as a therapeutic are not mutually exclusive. We will continue to fast-track research for any potential therapeutic that will lead to possible treatment for Angelman Syndrome. I also want to thank everyone for their support and the support of the Foundation for Angelman Syndrome Therapeutics.