A Stepping Stone to Treating an Incurable Disease

Basal ganglia in Parkinson's disease
Circuits of the basal ganglia in Parkinson’s disease
Image from Wikimedia Commons

Parkinson’s disease is a serious degenerative nerve condition that causes tremors a lack of movement, slowness of movement and problems walking. The disease becomes increasingly disabling with the symptoms growing more severe the longer one has it. The disease is caused by degeneration of a small part of the brain and as these cells die the brain becomes deprived of the chemical dopamine. This disease effects over half a million Americans with 50,000 new cases diagnosed every year usually symptoms are not visible until 60 but there are more people being diagnosed young every year. Mutations in the gene LRRK2 are known high risk factors for the disease but now there may be a way to neutralize this risk factor.

A group of UK researchers recently found that certain drugs could restore the movement ability of a fly that had the Parkinson’s mutation. The drugs work because they deacetylase the inhibitors they then target the transport system and reverse the effects of the mutated gene in the nerve cell. One of the leading researchers of the study Dr. De Vos said “The study provides compelling evidence that there is a direct link between defective transport within nerve cells and movement problems in flies caused by the mutation.” This study also shows that the negative effects from these gene mutations could be reversible. By targeting the transport protein with the drugs flies with the defect were able to fully restore movement and flight capability.

The gene affects the cell by producing a protein that affects many of the processes in the cell. They bind to microtubules which causes a defect in the transport system contributing to the symptoms of Parkinson’s disease. Microtubules are the organelles in the cell that act like the skeletal system allowing the organelles to move around in the cytosol. Preventing the association of the mutant protein with the microtubule transport system returns it to its normal function. The drugs successfully increased the acetylated form of a-tubulin within the microtubules which does not associate with the mutated protein. This change has a direct impact on the cells ability to move and perform motor functions.

This is important in the field of medicine because it is a stepping stone to developing a new treatment system for Parkinson’s which to this point has no cure or effective treatment. In most cases the treatment for the disease starts out effective with drugs but overtime its effect usually weakens to the point where it no longer has much of an effect against the symptoms of the disease. This is a positive step for research on Parkinson’s disease but there is more testing needed to show that this correlation and treatment for the defective is effective in other organism besides the fruit flies. If this is proven then more tests must be done to demonstrate that there are no harmful side effects involved with the treatment method for the mutated gene. This study gives thousands of Americans hope that there is possibly an effective treatment option for a condition that has so far been incurable, but it will be years before a treatment will be available to people and that is only if it is possible to treat Parkinson’s symptoms in humans with these drugs.

About Mr. Mohn

Biology Teacher

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