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Have you ever wondered exactly what all those research terms mean, like human trials, animal models, or blindtesting? Explaining how research works was a major topic at Ataxia UK’s conference in October, with a wellattended breakout session on the progress of typical research projects.
The first step in research is getting the idea. This might arise from observing that something seems to have caused the patient’s condition to improve, or a new biological discovery relevant to the condition. Next the idea will be tested to see if it’s viable and could potentially lead to a drug that might be beneficial. The next step in developing a particular drug or treatment would be to test it in cells, usually patient blood cells, or commercially available nerve cells. If this looks promising, a treatment will next be tested in animal models, to measure its effect on the body. Examples of animals that are often used are mice, which function in a similar way to humans, and simpler organisms like worms, fruitflies, or even yeast. These are fast, cheap to use, and can be easily manipulated in a lab. Ataxia UK has funded research to develop models of mice with with genes for two types of ataxia, which can then be used to test potential treatments.
Next, the drug is usually tested on animals to see if it is safe for use and will not be toxic to the body. After this testing stage has been completed, and if the new drug or treatment still looks promising, it moves on to human trials. In some cases the process can move faster, and for example move straight from the idea to human trials, eg if the drug is already approved for other conditions. One new possible group of drugs, HDAC inhibitors, is currently moving to the human trial stage, an exciting development. Human trials work in three stages. The first is to test it in healthy volunteers to ensure it is safe for use in humans. Next is to test the treatment in people who actually have the condition. This stage is usually a relatively small study, testing to see if the treatment is safe and appears to be effective for the condition. The third stage usually takes longer and involves a larger group, with a control group (some are given an ineffective placebo and some given the actual treatment, to accurately test if the treatment makes a difference). If a trial is described as ‘blinded’, this means that people do not know if they are taking the placebo or the actual drug, in order to give unbiased results. Finally, if all the stages have gone well, approval is needed from regulatory bodies like the MHRA or EMEA (Medicines and Healthcare Products Regulatory Agency, European Medicines Agency). Once obtained, the drug or treatment can be given to patients.
There is currently no cure for the ataxias but there are treatments to help control some of the symptoms, such as tremor and spasticity. Physiotherapy and occupational therapy can also be helpful to help cope with some of the symptoms.
See here for more details on stem cell research.
There are various factors to consider when reading research findings to evaluate whether the new discovery is useful or not. For example, look at what stage the research is at and whether it was testing a new treatment in people with similar conditions to you or for something different. Other things to consider include: Who funded or carried out the study, e.g. has it been carried out by a company which produces the treatment being talked about? Have there been other studies in this area or is it a brand new finding? Sometimes even well-designed studies can have misleading results if they are small studies in a new area and it can take years of further research to come up with definitive conclusions.
For a fact-sheet from the National Institutes of Health on understanding medical research stories in the media visit NIH
Ataxia UK sometimes helps researchers to recruit people with ataxia to take part in trials or contribute to other research. Occasionally ‘normal’ volunteers who are not affected by ataxia are also required. Details of research projects in which you may be able to take part are posted here.
One of the ways in which you could potentially help is by becoming a Friend of Ataxia UK and letting us know exactly what kind of ataxia you have been diagnosed with. This enables us to build up a database of people around the country and to potentially help researchers to put together a group of people with a particular condition for future research projects. Any personal information given to Ataxia UK will be kept in strict confidence in accordance with the 1998 Data Protection Act.
For more information about what it is like to take part in a clinical trial, see the clinical trials - a patient perspective website.