Within the medical realm, clinical trials play a crucial role in understanding disease progression, medication safety and efficacy, and patient outcomes. Recently, PTC Therapeutics began a Phase 1 clinical trial to test the safety of their investigational therapy PTC857. As of today, the first patient received a dose of PTC857, reports Parkinson’s News Today.
Evaluating PTC857
During this clinical trial, researchers will test PTC857 on healthy volunteers. They aim to understand how the therapy impacts the body, as well as how it is processed. If researchers determine that the therapy is safe, they will then hold additional studies for patients with Parkinson’s disease.
Trial participants will either receive:
- One single dose of PTC857
- Multiple doses of PTC857, increasing over time
This investigational therapy stems from PTC Therapeutics’ Bio-e platform. Through this novel platform, which targets enzymes called oxidoreductases, PTC Therapeutics focuses on treating central nervous system disorders. In this case, the therapy treats patients with GBA gene mutations.
Some researchers believe that neurological disorders result from inflammation. By targeting 15-lipoxygenase, PTC857 directly reduces neuronal inflammation and oxidative stress.
We will better understand the therapy’s potential benefits once the study yields data.
Parkinson’s Disease
Parkinson’s disease is a progressive nervous system disorder which alters and hinders movement. It occurs when dopaminergic (dopamine-producing) neurons die, interrupting communication between the brain and the body. While many patients are older (50+), there are cases of Parkinson’s disease appearing in younger individuals. For example, Michael J. Fox was only 29 when he received his diagnosis!
Generally, Parkinson’s disease occurs in 5 stages. In the first, mild tremors appear on one side of the body. These extend to both sides by Stage 2, with added muscle rigidity. Stage 3 is characterized by slowed movement and issues with balance. Throughout the final 2 stages, patients lose their abilities to live independently, stand, and walk.
Symptoms of Parkinson’s disease include, but are not limited to:
- Tremors
- Dementia or hallucinations
- Muscle stiffness and rigidity
- Inability to blink, smile, walk, or stand
- Slurring or stuttering
Learn more about Parkinson’s disease here.
