They work, we know they work, and they work on their intended target as they were designed, tested, and approved for use by the FDA after years of clinical trials and untold expense to pharmaceutical companies. As such, pharmaceutical companies continue their quest to develop, test, and provide drugs to improve the health-related quality of life of people living with Parkinson’s Disease.
With repurposed drugs there is nothing new, yet there is something new when smaller trials are designed as a proof of concept (for a certain medical condition or disease) to see if a certain drug/compound alters disease progression. A common question asked is, What is the mechanism of action? Along with this, some would ask: How does it work? What part of the brain does it impact? Is the impact measurable? Is the target affected by slowing, reversing, or stopping the disease progression? These are some of the key questions that repurposed drug trials aim to answer. So much is known, yet as much as is known with FDA approved drugs, the gold standard for clinical trials continues to be the randomized double blind placebo controlled trial. In essence, there has to be evidence that what the drug/compound is being tested for is what the results show. (Being that these are usually smaller phase two trials, if no evidence shows disease modification, then financial losses are kept to a minimum and other prospective drugs can be taken to trials).
Having mentioned this, we are fully aware that not all of the answers are what we would expect, yet using the “at all levels, from all angles” approach can give us hope in finding relief from progressive neurodegenerative diseases such as Parkinson’s Disease.
So, as the results of the Exenatide clinical trial make their rounds and discussed from every perspective possible, we must remind ourselves that although Exenatide is available by prescription, specific end points or results are being sought in these trials for Parkinson’s Disease and may not have shown dramatic improvement, promise still holds out that this and/or other FDA approved drugs could impact the millions living with Parkinson’s Disease by making their health-related quality of life the best that it can be. By way of caution, there is no evidence yet that would recommend a change in medication regimen and researchers/clinicians are urging this caution as well.
In the meantime, there is more work to be done in preparing for a phase three trial, other clinical trials to develop and recruit participants for, promotion of these trials, helping to secure funding as well, and most importantly for all of us, doing what we can, to the extent that we can, to stay active and involved in the research process.