Recently, the Michael J. Fox Foundation (MJFF) awarded $1.4 million in funding for Modag, a drug developer whose solutions treat patients with neurodegenerative diseases. In fact, says Parkinson’s News Today, this funding will be used to hold the first human trial to test the efficacy of anle138b for Parkinson’s disease. Previously, the molecule was shown to be safe and well-tolerated in healthy volunteers.
According to Modag, anle138b targets “disease-specific pathological oligomers.” In this case, the therapy targets fibrous structures with repeated patterns formed by alpha-synuclein (a-synuclein) clumps. For those of you who know about Parkinson’s disease or multiple system atrophy (MSA), you’ve probably heard of these a-synuclein clumps, or Lewy bodies. When these accumulate in the brain, they contribute to cell death and neurodegeneration. Modag explains:
Certain proteins including α-synuclein can form disease-specific fibrillar amyloid aggregates. The misfolding/aggregation pathway resulting in amyloid fibrils involves characteristic oligomer species which have been shown to be the most relevant toxic protein species.
Because the formation and structure of these oligomers is so unnatural, anle138b is able to easily target, remove, and prevent them.
In a 3-part Phase 1 clinical trial, researchers examined the safety, tolerability, and pharmacokinetics of anle138b in 68 healthy volunteers. First, patients received either a placebo or anle138b. The first segment of the trial saw only a single dose, while the second saw a daily dose taken for 1 week.
Next, researchers tested the intersection of food and anle138b. In both fasting and high-fat breakfasts, researchers saw no side effects or adverse reactions. Additionally, no adverse reactions occurred even up to the highest dose of 300mg. Researchers note that 100mg anle138b provides a therapeutic dose.
Parkinson’s disease is a progressive neurodegenerative disorder which impacts the central nervous system. As a result, patients gradually lose movement abilities. Generally, Parkinson’s disease results from dopaminergic nerve death. However, doctors are not sure what causes this nerve death. It is typically believed to be a mix of environmental and genetic factors, as well as the development of Lewy bodies.
Parkinson’s disease occurs in five stages. By stage one, patients only experience light tremors. However, by the third stage, patients experience difficulty balancing. In the final stage, patients are unable to live, walk, or stand independently. Usually, Parkinson’s disease affects people over the age of 50.
- Muscle pain and rigidity
- Slowed movement
- Poor balance or posture
- Inability to laugh, smile, or blink
- Slurring or stuttering speech
- Tremors, particularly in the hands
Learn more about Parkinson’s disease here.