Research Updates for the Russian HD Families:
A Moving Forward webinar

Written by the Moving Forward Team on September 6th 2023 

The Moving Forward team has conducted several surveys that proved that information about research and clinical trials is always a priority for HD families all over the world, who are looking for an effective treatment to stop the disease. 

Therefore, the team organized a webinar for the Russian-speaking HD community on the latest developments in Huntington’s disease research.

The webinar was held in early July and hosted by Astri Arnesen, the President of the European Huntington Association. The Russian HD families had the chance to learn about all the ongoing clinical trials happening worldwide. A simultaneous translation service was  available during the webinar so that everyone could learn about the latest news without the language barrier and in a family-friendly language.

27 family members took part in the webinar and were able to share their questions and comments with the Moving Forward team. The participants were grateful for this webinar, since they could learn about the latest HD research in an informal space without any language, geographical and political barriers and were able to see each other again. Watch the recording of the webinar in Russian here

The attendees were able to ask questions during the Webinar. Since the Moving Forward team believes that the answers to these questions provided by Astri Arnesen are interesting to the wider HD community, we publish them here.

Will the experimental drugs of the clinical trials covered by this webinar be fitted to individuals at any HD stage or to individuals at specific HD stages?

The majority of clinical trials recruit patients in an early or moderate HD stage.  During a trial, when a drug is proved to be clinically effective, it is effective for the specific stage involved in that trial. Then, the researchers will check if the drug can help people with other stages of HD. More and more pharmaceutical companies tend to start clinical trials in people at a very early stage of HD or even in premanifest HD individuals in order to start treating the disease as earlier as possible, before the onset of the disease. It is difficult to tell for what HD stage pridopidine will be approved. 

My wife's HD started 6 years ago. Is it possible for us to get involved in a any clinical trial abroad? If yes, how can it be done?

In general, it is very difficult. It is extremely important for a doctor to know the patient and to follow up his/her condition during and after the end of the trial.  This can be very complicated with foreign participants. Moreover, it is essential to speak fluently the language of the country where the clinical trial takes place, so that the patient fully understand the informed consent process, the purposes of the trial and can do the trial assessments. There are a lot of countries where clinical trials on HD are never conducted,  like for example, Norway. But once the drug proves to be efficient for HD treatment, the national associations will do their best to make the drug available in their own country. 

When a cure for HD is available, how much will it cost? Will it be very expensive?

There is no definite answer to this question. Usually, when breakthrough therapies appear on the market, they are extremely expensive. But we, as patients’ representatives, fight to establish reasonable prices. The drug must be available to the patient! It is very-very important! Otherwise, there is no sense in all clinical trials if the patient has no access to the drugs he/she needs. Different countries have different measures to provide drugs to patients and it really depends. In the European Union there is a lot of work to ensure equal access to different medicines. 

What pharmaceutical company is the most promising in finding the cure or slowing down the progression of HD? And what is the timeline for these inventions?

It is a difficult question. For example, the PROOF-HD trial is now completed, and the company is deciding right now if they should go on with testing the drug on a larger group of people or approve the drug for a specific cohort of HD patients. This also depends on the budget of the clinical trial and on how many people can be recruited to the trial. But I feel that we will have something new in a few years, because we have many companies and investigators working to find an effective treatment for HD.

Will these experimental drugs tested in the clinical trials be able to stop progression of HD or even restore some lost functions in patients with symptoms?

Many experimental drugs are being tested to stop the progression of the disease and development of HD symptoms. That is why researchers include people in early disease stages in the trials. That is why some companies include presymptomatic individuals in clinical trials to try to prevent the onset of HD symptoms in this group. And there are things that improve the quality of life of HD families, such as sufficient sleep, physical activity, healthy lifestyle- There are studies proving the efficacy of these non-medical approaches. 

It is difficult to motivate a person with HD also because of the HD treatment effects.

This is true, that is why doctors should follow patients closely to adapt the drug dosage so that it has the best effect on the patients’ life. 

Can the European Huntington Association help families in Belarus in getting access to tetrabenazine?

Actually no, but the families in Belarus can unite to claim for their rights and for access to the needed drug.

My daughter has severe problems with spelling the words. Can you recommend any exercises with speech therapy available on the net, so that we could practice these exercises at home?

I am sure there are. Just need to find it and send it to you. But I would recommend your daughter singing as a method of training her breath and vocal cords.