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How can we stop these diseases?

March 23, 2013

Huntington's Disease Youth Organization

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How can we stop these diseases?

Q. how can we stop these various diseases?

Nandhini, 11, India

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A. Dear Nandhini,

This is a very big question. I will just talk about genetic disorders of the brain. HD is a good example of these, but there are lots of others where the same issues apply.

The first key step was identification of the faulty or mutated gene that caused HD. This allowed the fundamental cause of HD to be identified and allowed research into why the brain was damaged.

The second key step was developing animal models of HD. That is, finding a way to put the human HD gene into experimental animals such as mice so that how a mutation in the gene caused the disease could be studied in detail

The third step is to use that knowledge to identify key steps in the pathway from the faulty gene to brain damage.

The fourth step is to use that knowledge to invent treatments, such a medicines, to prevent brain damage and then to test these treatments in animals with HD.

At the present time we are tackling steps 3 and 4. This is likely to take a few more years. The time taken to tackle steps 3 & 4 will always be too long for families dealing with these conditions, but the science is very complicated. Taking extra time to make sure that mistakes aren’t made is upsetting to people dealing with HD, but is essential if the best job is to be done.

The next step, number 5, which hasn’t started yet, will be to test the benefits & also the side effects in humans with HD of treatments that helped animals with the HD gene. This is likely to take a few years. It ’s possible that some treatments that worked in animals with HD will not work or will cause unacceptable side effects in humans. Only careful drug trials will allow us to get the best treatments.

Anther important issue is being able to detect the benefits of any useful treatments of HD. This is a very difficult thing for doctors to do. At present there are still gaps in our knowledge of how HD affects individuals.These gaps make it difficult to use any new treatments to the maximum benefit of HD patients. To fill these gaps a very important study of people with HD or at risk of HD has been started. This study is called Enroll-HD. It will be world wide, including in India, and will allow us to scientifically study HD in many thousands of people. It is necessary to study in HD in many thousands of people to get the sort of detailed information that we need to get the best treatments.

Enroll-HD has been started in North America and is about to be launched in Australia, New Zealand, parts of Asia, Europe & South America. It will generate huge amounts of data that will allow us to better design drug trials and to test new treatments. Again, it will take us several years to get the information from Enroll-HD and then use it to help people with HD. So watch this space.

If you come from a HD family then adult members of your family might be asked to participate in Enroll-HD.

I am very hopeful that over the next few years step 5 will be commenced and that Enroll-HD will give us the information that we need to develop new treatments. That is, I am very hopeful that over the next 10 years medical science will yield effective new treatments for HD.

I hope this very long answer explains the very big issues which scientists & doctors face in trying to find useful treatments of HD,