Friday, November 30, 2012

New drug promising for treatment of Parkinson's

Parkinson's disease affects the part of the brain that takes care of muscle control. Because the brain part responsible for these functions is dying in patients with Parkinson's, the symptoms slowly progress from slight tremors to all kinds of problems that also affect cognition and behaviour. Our ever-increasing knowledge of the brain and its pathology has resulted in several interesting and novel treatments, such as electrical stimulation. In addition, scientists have recently uncovered how vitamins, diet and genes affect symptoms in Parkinson's. However, there is still no proper cure, but a novel drug that is currently being tested has shown very promising results.

While we continue to learn more about why Parkinson's occurs in certain people, the supply of new treatments to the clinic is slow. Because we are currently unable to find a cure, most new drugs are targeted towards slowing down the progression of the disease, and possibly even alleviating some of the symptoms. In the end, patients still progress in the disease, which imposes a large effect on their quality of life A novel drug, called GM1, underwent clinical phase testing with patients and has shown beneficial results, especially because it is able to counter the progression.
The structure of GM1.
In a clinical setting with 77 patients, the drug was compared with conventional treatment during a period of approximately two years. What the investigators found is that GM1 not only slows down the progression of the disease, but also results in better function of the muscles compared to the situation before treatment. When compared to the treatment that Parkinson's patients normally receive, GM1 was also found to perform better.

The brain
Although the exact mechanism in human patients is not known, the scientists suspect that GM1 protects the brain cells that are at risk in Parkinson's disease. This was found in pre-clinical research aimed to investigate the mechanism by which the drug acts. This research was performed in mice, which means we still need to properly investigate whether the same thing happens in human beings. Because patients with Parkinson's decline due to ever-increasing brain cell death, protection is an important feature of any drug attempting to treat the disease.

Currently, GM1 is being tested in a clinical trial setting. That means it is still experimental, although it has already reached the human phase. Because extensive testing is necessary to ensure safety and efficacy, it will still take years before GM1 can reach the market, provided that follow-up studies do not reveal adverse effects. But so far these results look very promising. It is unclear which studies the investigators are planning next.

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