Resource • Article
Parkinson’s Disease, Part 1: The Importance of Successful Clinical Trials
- Losing total control of physical and mental motor skills
- Taking simple things for granted such as holding a pen, a hair brush or tying a shoe
- Knowing life with these symptoms may never end
There are an estimated 7-10 million people in the world today living with Parkinson’s disease and the reality of Losing, Taking and Knowing the dire impacts of this nasty disease. Parkinson’s is a neurodegenerative brain disorder whereby a person’s brain stops producing a neurotransmitter called dopamine. As the amount of dopamine decreases, a person has less and less ability to regulate body movements, and emotions. It is a debilitating, but a non-fatal disease with no known cure.
Parkinson’s disease is a complicated indication possibly comprised of several diseases with unique genetic and treatable characteristics thus sweeping curative approaches are not necessarily the best method. Researchers have discovered the molecular targets of investigational products and are evolving their understanding of the basic science behind Parkinson’s, beyond the loss of dopaminergic receptors. As a result, Parkinson’s drugs focus primarily on treating the symptoms of the disease (with the 4 main ones being: tremor, bradykinesia, rigidity, and postural instability). Patients may live a long, fulfilling life, with adequate and effective treatment for symptom management.
Naturally, development of these crucial drugs depends largely in part on successful clinical trials. Biorasi has built a remarkable knowledge base formed from study experience and team expertise. The clinical trial process starts at the very beginning, with optimal designs. The selected clinical endpoints must represent a true measure of the underlying disease and mitigate confounders due to symptomatic effects of study treatment. The successful execution of these trials depends heavily on understanding the placebo response rate. This is often an underestimated, yet major hurdle in demonstrating clinical significance. By focusing on these two major factors in the beginning, Biorasi sets up Parkinson’s disease clinical trials for study success and drug approval.