HD research news - medical research into treatment & prevention

The HDA Research Fund

January 2005

"This article aims to explain the process by which money is allocated"

One of the objectives of the HDA is to promote research into Huntington's disease. Unfortunately, only a relatively small budget can be allocated to support research projects. This article aims to explain the process by which money is allocated to a specific project and to explain how the HDA has responded to the initiative such as the UK HD Network and more recently the Euro HD Network. The decision to give money to a particular research project is taken by the Executive Council (EC) but the EC takes advice from its Medical Advisory Panel. Traditionally, researchers in the

UK are invited to submit proposals for funding; these are then sent to the Medical Advisory Panel. The remit of the Medical Advisory Panel is to ensure that each proposal is independently reviewed and to present the EC with the projects in an order of priority for funding.

At the moment we are supporting 2 projects one in Cardiff and another in Glasgow. The Cardiff project is a PhD studentship. The student is working with Prof. Ann Rosser and Dr M. Taylor. The Cardiff team have been involved in the neural cell transplant programme for some years but the availability of these cells is limited. This project is trying to understand how stem cells differentiate into the nerve cells which are affected by HD and, in the future, this could lead to the development of a new source of cells for transplantation The Glasgow project is helping Dr. Peggy Shelbourne employ a researcher to work on a project related to a mouse model of HD. Dr. Shelbourne has written an article about her project for this newsletter which is included below.

This year has seen a departure from the traditional approach of funding specific because the HDA has decided to use the research fund to support the development of the Euro HD Network. The background to this development demonstrates the value of our patients' organisation having strong links with researchers and professionals who have a special interest in HD.

If a new treatment is proposed for HD we need to know whether or not it is safe and effective. Work in the laboratory can only go so far. In the end, the treatment has to be tried on a large number of patients in a scientific process which is called a clinical trial. At the end of such a trial we can all judge whether or not the proposed treatment is safe and effective. In the past, many studies have been limited to just a few patients and it is difficult to come to any conclusions. As large numbers of patients are required for such a study, it is important to have a network of doctors and patients who are willing to record details about HD in a standard way. Such a network exists in the United States and recently they have been able to test one or two treatments on a large number of patients relatively quickly. Approximately 3 years ago, the HDA was approached by Professors Peter Harper, Ann Rosser and Steve Dunnett from Cardiff for help in developing a similar network in the UK. The idea was to build on the project they had been doing in which detailed observations had been made on about 60 patients. The research team were aware that if we were going to do studies which assessed the value of particular treatments there would be a continuous need to recruit more than 60 patients. The Executive Council of the HDA was supportive of this and was also aware that treatments are currently being tried in animal models of HD. The EC was aware that none of these has reached the stage of needing a clinical trial but we all hope this will be the case and therefore we wanted to support a process which will allow clinical trials to be more easily conducted in the future. The request for funding from the Cardiff team was more than the HDA could manage; however, we responded by agreeing that this was a good idea and that the HDA would facilitate obtaining money from another charity. The HDA was able to approach the Dunhill Medical Trust and support Prof. Rosser and her colleagues obtain funds to establish a network. Prof. Rosser's application was successful and doctors from a number of clinics agreed to be part of the network in the UK.

About the same time, similar events were taking place in Europe. The population of Europe is larger than that of the United States. We, in Europe, should be able to undertake similar studies; there is a particular problem that we all speak different languages so the forms which allow us to ask questions about HD in a standard way and record the answers all have to be translated into different languages. Such a study has been taking place and a network formed but as in the UK the European researchers wanted to build on this for the future. A large research foundation called High Q has funded a 10 year project to develop this sort of network in Europe. At first sight a research foundation called High Q makes no sense but you need to remember that the gene which causes HD is larger than normal. Genes make proteins and the scientific shorthand for the part of the protein which is enlarged is called "Q".

One of the first tasks of the European network was to develop a secure computer program so we could all communicate with each other and record the information about patients in a way which is secure and no-one except the doctor treating the patient can know the name of the patient. The person leading this European Development is Prof. Bernhard Landwehrmeyer. who came to the first meeting of the UK HD network and explained how the system would work. It was immediately obvious that some of the issues about recording information safely had been solved. The UK network agreed to be part of Euro-HD. Some of you will know that Prof. Landwehrmeyer was visiting Manchester and Sheffield just before our 2003 AGM in Derbyshire. Prof. Landwehrmeyer agreed to rearrange his travel plans for the Saturday and talk at the AGM. The idea of developing the network was greeted with enthusiasm. The team in Cardiff became the lead centre for the UK and Ireland.

The members of the Medical Advisory Panel met and realised that if the UK was to respond properly to this initiative then other clinics participating in the Network would need extra resources. The Panel advised the EC to spend the research fund money on this project. As always, there is not enough money to allow us to give extra resources to every clinic so a new independent panel was formed to assess applications from clinics who felt able and willing to participate actively in the Euro-HD project. The clinics chosen were in Manchester, Sheffield, Birmingham and two in London. We would have liked to have supported more centres but that will require extra fundraising in addition to that which is done to maintain our Regional Care Advisory Service.

Your medical data is confidential. Doctors cannot decide it is a good idea to ask you if we can change your name to a number and then send your clinical information to Euro-HD using a computer. The whole process has to be checked by an ethics committee who will want to know that the information is held securely and managed properly. The ethics committee will also want to know that Doctors explain that anyone participating understands what is happening to their information and that they are free to agree to participate, or not, and that they can withdraw from participating in the future if they wish. At the time of writing, the team in Cardiff have submitted an application to an Ethics Committee requesting permission for Doctors in the UK who are part of the network to ask patients if their information can be sent to the network. Once the ethics committee is satisfied that all the arrangements are satisfactory, the HDA will release the research money to the clinics. We are hoping that this will be done by next January.

At the moment the network is still in a stage of development but the investment of resource in the European Network by a charitable foundation has meant that the whole process can and has developed much faster than would otherwise be the case. The funding for Euro-HD allows researchers and members of the HDA to attend meetings. The first meeting of the Euro-HD network has been held in Spain. Two Regional Care Advisers attended along with researchers, neurologists, genetic counsellors and clinical geneticists from the UK. The first step is to develop the network so that we are able to get details on a large number of patients in Europe all collected in the same way. At the moment no treatments are being tried; we need to walk before we can run but already a number of working groups have been formed so that we can use the network to obtain better information about particular aspects of HD and build on projects and ideas which are already in place in Europe. You may be interested to know that the work our own HDA has done on juvenile HD has been recognised and we have been given an opportunity to use the resources of the European network to develop this further.  There are lots of other examples but it would take a separate report to describe each of them.

Whilst at the meeting one of the speakers said something which I think is especially important. I cannot remember the exact words but the general message was as follows. In other conditions the first treatments only made a small difference to patients. We need to start with a treatment which makes a real difference to patients. It does not matter if that real difference is only small because we can develop from there. The HDA is supporting the development of the Euro-HD network. By itself, the network will not lead to treatments which make a real difference to HD but it is an essential pre-requisite for this and means that we can progress faster but still safely towards that goal.

• Oliver Quarrell
  Member EC of HDA and member of Euro-HD.