Providing Insight into Multiple Sclerosis and the Evolving Treatment Landscape
Associate Professor Megan Munsie, Dr Julia Morahan, Associate Professor John Moore and Dr Jennifer Massey describe the evolving treatment landscape for Multiple Sclerosis, and explore how to navigate treatment options.
Over 25,600 people in Australia and more than two million across the globe live with multiple sclerosis. New treatments are improving lives for some, but there remains no cure. A recent SBS Insight program explored what it is like to live with this condition and how the hugely variable nature of multiple sclerosis (MS) means that navigating treatment options can be daunting. Understanding more about the cause of the condition and how treatments work, who they will work for, is an essential first step for anyone contemplating their options.
What is MS?
Multiple sclerosis (MS) is a disease that typically affects young people. The average age of diagnosis is 30, with the condition disproportionately affecting women. Of every four Australians diagnosed with MS, three are women.
In MS a person’s own immune cells attack and damage the myelin sheath, the protective insulating layer around the nerve fibre in the brain and spinal cord. Damage to myelin results in a slowing of the electrical signals between nerves cells and other parts of the body. Depending on the region of the brain and spinal cord that is attacked, symptoms can range from issues with vision, difficulty with walking, coordination or balance, altered sensations such as numbness or tingling, debilitating fatigue or pain. Every case is different. Not everyone with MS will experience these symptoms, and symptoms can vary in severity over time.
Although there is no known single cause of MS, many genetic and environmental factors have been shown to contribute to its development.
Different types of MS
People living with MS are usually diagnosed as having one of three particular types of MS. Relapsing remitting MS is the most common form and is characterised by flare-ups where the body’s immune system attacks the brain, spinal cord and/or optic nerve resulting in symptoms. These attacks are also referred to as relapses and are followed by periods of recovery or remission as the body is able to naturally repair this damage. However, the repair is often incomplete. After repeated episodes of relapse, the affected nerves may die.
Once this happens, people enter the progressive phase of MS, where symptoms no longer come and go but get progressively worse over time. This is known as secondary progressive MS. More rarely people can be diagnosed with progressive MS right from the beginning of their symptoms, these people are given a diagnosis of primary progressive MS. In this form, symptoms are due to the damage to the underlying nerve cells and not primarily through the immune system.
The MS treatment landscape
The MS landscape has been comprehensively transformed over the last few years by progress in research and therapeutics. There are now 12 disease modifying therapies available in Australia for people with relapsing remitting MS, the most common form of the disease. These treatments are immunomodulatory, in that they work by having an effect on the immune system. MS medications can be tablets or capsules, injections or infusions but all are based on dampening down the effect of the immune system, in order to attempt to suppress further attacks on the brain, optic nerve or spinal cord. These medications usually target particular types of immune cells in the body thought to be involved in MS attacks. Almost all are provided in Australia under the Pharmaceutical Benefits Scheme (PBS). Over the last 15 years, people with MS are being diagnosed earlier, and the long term outcomes for people with MS have improved significantly, with certain disability milestones being reached almost 8 years later on average.
For progressive MS, however, there are limited treatment options. As the immune system is not as actively involved in this phase, the therapies that are available for relapsing and remitting MS are not appropriate for these patients. Immunomodulatory therapies are unable to repair damage to the nerve fibres. Australian researchers are working with colleagues across the globe to better understand this form of the condition and accelerate the discovery of new treatments.
Autologous Haematopoietic Stem Cell Transplantation (AHSCT) for MS
Doctors and scientists around the globe are also exploring another way to suppress the immune system in relapsing remitting MS. One possible treatment being explored is Autologous Haematopoietic Stem Cell Transplantation (AHSCT) where the aim is to reset the patient’s entire immune system. Haematopoietic stem cells reside in your bone marrow and are responsible for making red blood cells, platelets and the immune cells of the body. In this approach, the patient’s own haematopoietic stem cells are collected and stored before they are given chemotherapy to remove most or all of their immune cells. The patient’s own haematopoietic stem cells are then reinfused and over time the stem cells will rebuild the immune system as well as red blood cells and platelets. While the blood and immune systems are being re-established, the patient is at risk of developing infections and bleeding and patients need to be carefully monitored. AHSCT has been routinely used for the treatment of blood cancers for many years.
Since AHSCT targets the immune system, it is most likely to benefit people who have active relapsing forms of the disease where the immune system is the predominant driver of the disease.
Some people with relapsing remitting MS have had good results with AHSCT, including those from the recent AHSCT observational trial in Sydney however it is not without high risks and the results show that it is not effective or suitable for everyone with MS. Additionally, the long term effects of AHSCT on MS remains unknown.
International studies have suggested that some people with MS may experience some reversal of disability following AHSCT, however, this is thought to be primarily as a result of normal repair of myelin around intact nerves that can occur once the inflammatory attack in the brain and spinal cord is suppressed.
Access to AHSCT in Australia
In Australia, AHSCT is available to those who are most likely to benefit and those who have the highest likelihood of success. Research shows that these people are younger people with MS, with highly active MS, where other approved therapies have failed. More information can be found in MS Research Australia’s position statement. In these cases, AHSCT is available and carried out under an observational clinical trial setting. A referral from a neurologist is required and patients need to meet the criteria for each site.
Why are people with MS going overseas?
As mentioned on the Insight program, some Australians have travelled overseas for AHSCT for their MS as they are not currently eligible for the treatment in Australia. This is often at considerable personal cost. The decision for people to do this is complex and outcomes for these individuals, as with everyone with MS, can be variable. While people with MS will naturally wish to explore all potential treatment avenues, the potential risks and likelihood of success of AHSCT needs to be considered for each individual.
Risks can include infections such as pneumonia, excessive loss of blood and even death. Such decisions should be made in consultation with an Australian neurologist who is fully aware of their condition and what options are available at home.
Newer generation treatments for MS are highly effective in the majority of people with MS, but do not work for everyone. AHSCT does have a role to play in the range of treatments available to treat MS, however, it is very unlikely to be right for everyone. While the notion of nothing ventured, nothing gained is an understandable driver, the costs can be more than financial.
More information
MS Research Australia has more information about AHSCT for MS, outcomes of clinical trials and a position statement. Visit msra.org.au/ahsct for details.
Information about the Authors
Associate Professor Megan Munsie is from the Centre for Stem Cell Systems, Dr Julia Morahan from MS Research Australia and clinical colleagues Associate Professor John Moore and Dr Jennifer Massey from University of NSW and St Vincent’s in Sydney.