Research
FAQs: getting involved in research
People with MND and their carers can play an important role in research.
What can I do to contribute to research?
People with MND and their carers play an essential role in clinical trials of potential new drugs (see below); without their support and commitment these trials couldn't be carried out. People with MND can also contribute to the advancement of research in other ways:The MND Association DNA Bank and National Database is an exciting project set up by the MND Association. It aims to build a collection of at least 5,000 DNA samples, taken from a simple blood test, from people with MND, their carers and families. This will provide a valuable resource for researchers all over the world investigating the genetic causes of MND. More information on the DNA Bank and how to participate is available from Research Information Sheet I -participating in the DNA bank .
People with MND can also make a vital contribution towards finding the cause and developing treatments for the disease by donating their brain and spinal cord tissue for use in research after their death. More information on tissue donation is given in Research Information Sheet L -Helping MND research through tissue donation .
From time to time, researchers carrying out healthcare studies require the help of people with MND and their carers. Participation in these studies could involve activities such as completing a series of questionnaires or interviews about aspects of life with MND. It is usually necessary for participants to live relatively near to the university or hospital where the study is based. For more information contact research@mndassociation.org
Where can I learn about clinical trials in the UK?
As soon as the MND Association learns of a new trial underway in the UK, we will post information on the Treatment Trials page of our website. We also work with the co-ordinators of the trial (often pharmaceutical companies) to produce information sheets for people with MND. Information on clinical trials is also included in our quarterly magazine Thumb Print. The centres recruiting participants for the trial will also notify eligible people. Two clinical trials involving UK patients are currently underway; recruitment for both has now closed.
More information on the objectives and results of treatment trials carried out around the world is available on our treatment trials page . You can also read more about clinical trials taking place overseas on the American ALS Association website and on a dedicated American government website.
Why is a placebo necessary?
Placebos (chemically inert substances, or dummy drugs) help to prevent unsuspected biases invalidating the results of clinical trials. A placebo group is particularly important in ensuring that any apparent benefit (or harm) is not due to factors unrelated to the drug on trial. How does the placebo control work?
The patients are placed in random groupings and coded, with some patients receiving the active substance and others a placebo. No one knows (except the central pharmacist), not even the investigator, who is taking the active substance and in what dose, or who is taking the placebo.At the end of the trial the codes are broken and results analysed. By comparing the data, it is hoped that there will be a clear difference between groups of patients.
The difference may be minimal which is why there are a large number of tests carried out during the course of the trial, to ensure even minor differences are picked up.
It is only by making comparisons between groups of patients on the placebo and on the active substance that the pharmaceutical companies can prove whether a substance is effective.
