Gene Therapy for Cholesterol

The Facts:

In this age where diet and fitness are becoming more and more important to people, it has become apparent that high levels of cholesterol can lead to heart disease. Unfortunately for some people, even a well planned diet and exercise regime does not guarantee them a low level of blood cholesterol. This is the case for people who suffer from familial hypercholesterolemia, a genetic disease results in a lack of receptors on the liver which would normally dispose of any harmful cholesterol.

Familial Hypercholsterolemia affects 7 in 1000 people Affected men typically experience heart attacks in their 40s and 50s 85% of affected men experience heart attacks by the age of 60 The main treatments include diet, exercise and cholesterol lowering drugs

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The Role of Gene Therapy:

While it has been possible to reduce cholesterol levels using combinations of diet, exercise and drugs in people suffering from familial hypercholesterolemia, the incidence of heart attacks remains quite high. In attempts to find a long term cure, familial hypercholesterolemia became one of the first diseases to be studied for gene therapy treatment. Early results were mixed, with poor efficiency of treatment, but much progress has since been made.

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Recently, a gene therapy trial on a 29 year old woman attempted to insert the normal receptor genes into the liver, thus reducing the patient's levels of cholesterol. Before this treatment, the patient had a cholesterol level of above 500 mg/dl, though this was subsequently reduced by around 30% over a period of six months. This is a significant reduction in cholesterol and is far in advance of any existing treatments.

There is now also speculation that gene therapy may make it possible to defend against blood clots, which would normally lead to heart attacks. The idea is that a gene could be inserted which produces a protein capable of breaking down any such clots. Another focus lies with the genes that initiate the formation of blood vessels, via a process known as angiogenesis. By injecting tissue with these genes, it should ensure that even if blood clots do occur, there will be the capability to form new arteries and veins, meaning that the clot poses no real threat.

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Conclusions:

Familial Hypercholesterolemia is one of the major causes of heart disease. There are also many other causes of heart disease, which is the biggest killer in the United Kingdom. However, gene therapy appears to bring about endless prospects in the treatment of such problems, perhaps one day providing a significant breakthrough in the fight against heart failure.