MIROCALS (Modifying Immune Response and OutComes in ALS) is recruiting for participants with Amyotrophic Lateral Sclerosis (ALS) - the most common form of Motor Neurone Disease - if they meet a list of criteria.
The study focuses on some types of immune cells in the blood which are capable of influencing the speed at which ALS progresses. This is because these immune cells, called Regulatory T Cells, are believed to play a part in protecting the motor neurones.
A substance which is naturally produced in the body, called Interleukin-2, can increase the production of these special immune cells in the blood.
As a result, the trial tests the safety and effectiveness of introducing low doses of Interleukin-2 in people with ALS. The purpose of this is to stimulate the production of the immune cells and to explore whether increased levels of these can slow down the progression of the disease.
Interleukin-2 is already used, at higher levels, to treat some forms of cancer. However, due to some of its undesirable side effects on the immune system, only small doses are recommended for non-cancerous diseases.
Recruitment for the MIROCALS clinical trial in Scotland is now closed, however recruitment is still ongoing at other centres in the UK and France. There are entry requirements for the trial which include the fact that patients who wish to participate cannot currently be taking Riluzole.
To test the effectiveness of the treatment, researchers will measure its success based on rate of progression, changes in day-to-day activities and quality of life. In addition, any side effects that people may experience when taking this drug will be closely monitored.
As a secondary goal, the researchers will search for biomarkers, unique biological signatures, in the blood and cerebrospinal fluid (CSF). Finding biomarkers for ALS would allow researchers to determine whether a drug is working. The discovery of these biomarkers would also mean that clinicians are able to diagnose ALS faster and to better monitor the progression of the disease.
A clinical trial is a scientifically controlled study exploring both the effectiveness and safety of a particular therapeutic treatment, such as a drug.
In most clinical trials, including MIROCALS, the impact of a treatment is determined by comparing groups of patients who do receive treatment (treatment or experimental group) and do not receive the treatment (control or placebo group).
The approach means that participating in a clinical trial does not guarantee that an experimental treatment will be received.
Any therapy approaching the first small-scale trials in humans will already have undergone a lengthy period of development and testing in the laboratory.
The involvement of human patients only occurs at the last stage of testing before a drug is licensed for general use.
Facts about clinical trials:
Lawrence Cowan | Chair of MND Scotland