New hope for Huntington's sufferers

Aug 22, 2007

A major breakthrough in the understanding and potential treatment of Huntington's disease has been made by scientists at the University of Leeds.

Researchers in the University’s Faculty of Biological Sciences have discovered that one of the body's naturally occurring proteins is preventing 57 genes from operating normally in the brains of Huntington's sufferers. In addition, the destructive nature of this protein could potentially be halted using drugs that are already being used to help cancer patients.

“This is a really exciting breakthrough,” says researcher Dr Lezanne Ooi. “It's early days, but we believe our research could lead to radical changes in treatment for Huntington's sufferers. The fact that these cancer drugs have already been through the clinical trials process should speed up the time it takes for this research to impact directly on patients.”

Huntington's is an inherited degenerative neurological disease that affects between 6500 and 8000 people in the UK and up to 8 people out of every 100,000 in Western countries. Any person whose parent has Huntington's has a 50-50 chance of inheriting the faulty gene that causes it and everyone with the defective gene will, at some point, develop the disease.

It is characterised by a loss of neurons in certain regions of the brain and progressively affects a sufferer’s cognition, personality and motor skills. In its later stages, sufferers almost certainly require continual nursing care. Secondary diseases, such as pneumonia are the actual cause of death, rather than the disease itself.

Dr Ooi's research has identified the effects of one of the body's proteins on the neurons of Huntington's sufferers. Neurons are usually protected by the protein BDNF (brain derived neurotrophic factor), whose many functions also include encouraging the growth and differentiation of new neurons and synapses. However, in Huntington’s sufferers, the repressor protein known as REST - which is usually found only in certain regions of the brain – enters the nucleus of the neuron and decreases the expression of BDNF.

She has also been studying some of the enzymes which assist the function of this protein. It is these enzymes that provide the mechanism for the protein to wreak havoc in the brains of Huntington's sufferers, and that are already being targeted in certain cancer drugs.

Currently, the symptoms of Huntington's can be managed through medication to help with loss of motor control and speech therapy but there is no definitive treatment. This research provides a first step in developing a treatment regime that may halt the onset of the disease.

“Huntington’s is a devastating illness that affects whole families. Those who know they’ve inherited the faulty gene live in a shadow of uncertainty over how long their symptoms start to develop. It can also be particularly cruel since every child born to a parent that has the HD gene is at 50% risk of having inherited the gene,” says Cath Stanley, Head of Care Services at the Huntington’s Disease Association.

“As such, any developments in the understanding of this disease are welcome, but this breakthrough is particularly exciting as it opens up an avenue for researching a possible treatment using drugs that are already available, rather than starting from scratch.”

Source: University of Leeds

Explore further: Nigeria death shows Ebola can spread by air travel

add to favorites email to friend print save as pdf

Related Stories

Sick Alaska seal shows possible spread of disease

Mar 08, 2012

(AP) -- Federal scientists said Wednesday that a nearly bald, lethargic seal recovered from the southeast Alaska coast showed the same symptoms of a disease that sickened ringed seals and Pacific walrus on ...

Solving the puzzle of Henry VIII

Mar 03, 2011

Blood group incompatibility between Henry VIII and his wives could have driven the Tudor king's reproductive woes, and a genetic condition related to his suspected blood group could also explain Henry's dramatic ...

Recommended for you

S.Korea detects second foot-and-mouth case

16 minutes ago

South Korea on Monday reported its second case of foot-and-mouth disease in less than a week, triggering fearful memories of a devastating 2011 outbreak that forced the culling of millions of livestock.

Ebola kills Liberian doctor, 2 Americans infected

46 minutes ago

(AP)—One of Liberia's most high-profile doctors has died of Ebola, officials said Sunday, and an American physician was being treated for the deadly virus, highlighting the risks facing health workers trying ...

Hepatitis C virus genotype 1 is most prevalent worldwide

1 hour ago

In one of the largest prevalence studies to date, researchers from the U.K. provide national, regional, and global genotype prevalence estimates for the hepatitis C virus (HCV). Findings published in Hepatology, a journa ...

1 in 3000 blood donors in England infected with hepatitis E

1 hour ago

The first systematic analysis of hepatitis E virus (HEV) transmission by blood components indicates that about 1 in 3000 donors in England have HEV in their plasma. The findings, published in The Lancet, suggest that around ...

Biologic treatments for RA carry similar infection risk

1 hour ago

(HealthDay)—The risk of hospitalized bacterial infections in older rheumatoid arthritis (RA) patients is similar for rituximab or abatacept compared to etanercept, although it is higher for infliximab, ...

New oral drug regimens cure hardest-to-treat hepatitis C

1 hour ago

Two new pill-only antiviral drug regimens could provide shorter, more effective treatment options with fewer side effects for the majority of patients infected with hepatitis C, even those most difficult to treat, according ...

User comments : 0