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Huntington’s disease: The latest research


There have been a number of groundbreaking discoveries in recent years. Here we outline research that may provide treatments for the future.


Embryonic brain cell transplants

Embryonic brains contain a multitude of stem cells. A recent study has showed that transplanting cells from the striatum of an aborted human embryo into the brain of someone with HD holds promise.


Five patients with mild to moderate Huntington's disease had the grafts. After 2 years, 3 of the 5 patients had improved motor and cognitive functions as compared to those who had not had a graft. Two of the 5 showed decline in line with other people with HD.


High throughput screening

Scientists know that the faulty huntingtin protein causes HD, but how it kills cells is not fully known. By screening hundreds of compounds, scientists hope to find one that prevents the aggregation or cleavage of the mutant huntingtin protein – and thereby stops the cells being killed.


If successful, they will try this in tissue culture, then examine the effects on animals carrying the faulty HD gene. If the results of both these are successful, the compound can be safety tested in preparation for human trials.


Reducing the toxic huntingtin protein

Scientists at the Department of Medical Genetics in Cambridge University delayed the onset of HD in mice by giving them rapamycin. This drug works by making cells break down proteins more quickly, and reduces the levels of the Huntingtin protein.

Interfering with the genes

In a ground-breaking study, scientists were able to prevent mice carrying the HD gene, from actually getting HD.


This amazing research was led by Beverly Davidson of the University of Iowa, who used a genetic technique known as RNA interference (RNAi). This revolutionary technique worked in the mice by ‘silencing’ their faulty HD gene before it made a protein.


Much more research is needed, but if safe, she hopes clinical trials might begin in 2010.



Some people with Huntington's disease say they find self-medication with cannabis helpful.


To explore this as a treatment option, doctors at the Queen Elizabeth Psychiatric Hospital in Birmingham, England are about to start a clinical trial with nabilone.


Nabilone, swallowed as capsules, binds to the same receptors as the active ingredient in cannabis. Many of these cannibinoid receptors are found on the output neurons of the striatum.


Can fish oils help people with HD?

A recent Canadian clinical trial with ethyl-EPA, an omega 3 fatty acid, showed minor results. Whilst overall there were no significant improvements in motor symptoms, it seems people who are earlier in the course of the disease might have a benefit. A new trial will explore this possibility. It is likely that fish oils protect neurons in a non-specific way.


Keeping brain cells alive

If you have HD, your brain cells become over-stimulated and literally excite themselves to death. One of the neurotransmitters responsible for this is glutamate.


A drug called Riluzole reduces the release of glutamate in the brain and may help people with HD. There is currently a major European trial with this drug.


Cells love CNTF

During your development, CNTF was the main growth factor for brain cells in your striatum. It is these cells that are damaged in HD. So could CNTF revive damaged brain cells in HD? A small-scale trial in France infused CNTF into the brains of people with HD. The results showed no improvements, but it is thought infusing CNTF still holds promise for HD.


For more information and the latest research, click here