Clinical trials and treatment development

There is a wide range of research and clinical trials taking place around the world, trying to find and improve treatments for MS.

It is difficult to scientifically compare people's experiences of MS. Extensive placebo-controlled clinical trials are required to research disease patterns and the effectiveness of new treatments.

What is a clinical trial?

Clinical trials are research studies that test the effects of “health interventions” on people. Such health interventions are most commonly new drugs or treatments, though clinical trials may also examine the effects of other health interventions such as diagnostic methods, surgeries or procedures, technological devices and educational methods.

Clinical trials usually only begin once pre-clinical laboratory studies have already shown promising results. These initial studies typically involve human cells or animals. 

The most reliable way of showing whether a new intervention is safe, effective or better than what is already available is through clinical trials.

What are the clinical trial phases?

If the pre-clinical laboratory based studies have shown promising results, the health intervention may go to trial stage. The trials themselves have to progress through a set sequence of four "phases" to ensure the data collected is reliable and all those taking part are protected. Phase I must be successfully completed prior to moving on to Phase II and so on for all remaining phases. Testing can be stopped at any time in any phase in order to ensure the safety of participants.

The trials at each phase have a different purpose and help scientists answer different questions:

In Phase I trials, researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.

In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits and optimal use. 

What future treatments are in development?

Researchers are working hard to find new and better treatments for people with MS. There are currently three oral drugs for relapsing-remitting MS available; Fingolimod (in US, EU and other countries), Cladribine (Russia and Australia) and Teriflunomide (US and Australia), with several more oral therapies undergoing phase II and III clinical trials and expected to come on the market during 2013.

Stem cell therapies are another exciting area of research in treatments for MS. There are currently several trials investigating the potential of various types of stem cells for different forms of MS. They include mesenchymal stem cell (MSCs), haematopoietic stem cells (HSCs) and neural stem cells (NSCs). MSCs are currently being trialled for MS. It is thought they may have a positive effect through ‘immunomodulation’ and might also promote the nervous system’s own repair mechanisms to repair damaged myelin. Watch a film about recent research into stem cells in MS and read more about stem cells in MS in focus - stem cells and regeneration in MS.

Neural stem cells are responsible for repairing myelin in the brain and are expected to be trialled for MS soon. HSCs already used to treat leukaemia, lymphoma and several inherited blood disorders. HSCs are being trialled in highly active forms of MS, where it is thought they may help prevent damage to myelin by altering how the immune system functions.

The International Progressive MS Alliance, formed in 2012, is looking specifically at researching and developing more treatment options for people with the progressive forms of MS.