CAN BLOOD STEM CELLS REBOOT THE IMMUNE SYSTEM?

There is a great deal of scientific and community interest in the role that stem cells might play in treating MS. Stem cell therapy is a treatment that uses or targets stem cells; usually to help replace or repair damaged cells or tissue. However, the treatment currently receiving significant interest in the MS community is something called Autologous Haematopoietic Stem Cell Transplantation (AHSCT).

This treatment is first and foremost an immune-suppressing chemotherapy treatment that almost completely removes a person’s immune system – it is a type of chemotherapy normally used for people with particular types of blood cancers. The use of stem cells in this case, is to help restore the blood and immune system after the chemotherapy. There is no evidence that they contribute directly to repair of the nervous system.

The idea of this treatment for MS, is that it essentially resets, or reboots the immune system so that it is less likely to attack the brain and spinal cord.

Like the available disease modifying therapies currently registered for use, this treatment is not a cure for MS, but some scientific reports published over the last few years reveal encouraging clinical findings suggesting that the treatment can prevent or reduce relapses for quite some years following the procedure. The research to date suggests that people who have very active relapsing forms of MS and are younger (40 or younger) can do well after the treatment, although, people with progressive forms of MS are less likely to get a benefit from the treatment.

The treatment does however, come with significant risks and given the number and range of approved therapies on the market it is generally only recommended by doctors for people who have not responded well to other types of MS treatments. The risks are mainly due to the extreme suppression of the immune system in the few weeks following chemotherapy that can leave people very vulnerable to life-threatening infections that need intensive care treatment. Longer term risks associated with the chemotherapy are also known to occur, including effects on fertility and longer-term cancer risks. The global rate of mortality (people dying as a result of the procedure) has reduced in recent years from about 5% to approximately 1.3%.

MS Research Australia has provided a comprehensive review of what is involved in AHSCT on their website with information about what the research has shown to date. msra.org.au/AHSCT

Availability of the treatment in Australia

MS Research Australia, MS Australia and MS Queensland deeply understand and fully acknowledge that people with MS will wish to explore all potential treatment avenues. AHSCTdoes have a role to play in the range of treatments available to treat MS, however, it is very unlikely to be right for everyone. Its potential risks and benefits must be carefully considered in relation to each individual’s personal circumstances in consultation with their health care providers. Currently in Australia it is provided through two observational clinical trials and by a limited number of centres on a case by case basis. In all cases, the hospitals require a referral from a neurologist and in the majority of cases, hospitals will only accept patients who have failed to respond to other available MS therapies. More information about these clinical studies can be found on MS Research Australia’s website here msra.org.au/AHSCT and on their MS Trials website mstrials.org.au.

What research and advocacy is happening for AHSCT in Australia?

For hospitals and clinicians to be able to provide a particular therapy for patients they must have a strong body of evidence available to understand and weigh-up all of the potential benefits and risks of the treatment and understand for which patient population the treatment is most appropriate. This type of evidence usually comes from large scale clinical trials and long-term studies.

The international evidence for the use of AHSCT to treat MS has been growing gradually over the last several years. However, the results of gold-standard, randomised controlled trials, have only recently begun to emerge. This together with large observational studies conducted in Europe and elsewhere, is leading to a much greater understanding of the treatment.

To contribute to this global accumulation of knowledge MS Research Australia, together with a steering committee of haematologists and neurologists, established the Australian MS AHSCTRegistry in 2011, to collect clinical data on Australian patients who have undergone the procedure, either in Australia or overseas. The work to collect and analyse long-term follow-up data is ongoing. In the interim, MS Research Australia and the steering committee developed a position statement, with reference to the current data published in the international peer-reviewed scientific literature, on the use of AHSCT for MS with guidance on its possible place within the range of treatment options for MS. Visit msra.org.au/AHSCT for the AHSCT Position Statement.

MS Research Australia is also funding a research study to examine the effect of AHSCT on the immune system. This study will help determine how the treatment works and who is most likely to benefit. This will help better identify individuals for whom the potential benefits of the procedure far outweigh the significant risks.

Globally, amongst the MS research community, there is also recognition that a large gold-standard clinical trial is still needed to help understand how the treatment measures up in terms of the duration of its effect and its safety in comparison to some of the other highly-effective MS therapies currently available.

MS Research Australia is continuing to monitor the outcomes of clinical trials overseas, and is actively consulting on a global scale for opportunities to work with other countries to further develop the evidence. They have also worked to provide as much evidence-based information, including videos and easy-to follow infographics, so that people with MS can make informed choices about their treatments in collaboration with their neurologists.

MS Australia and MS Research Australia have also been working together with haematologist and neurologist groups and the state and federal governments to raise awareness on the role that AHSCT may play in the treatment of MS and discuss appropriate access to the treatment in Australia.

Jesse Shares his Story

Jesse Frick from Adelaide was diagnosed with relapsing remitting MS in 2005 when he was just 18 years old and by the time he was 22 he used a wheelchair. After exhausting all of his treatment options, his neurologist suggested that AHSCT could be of benefit.

He says, “I remember him telling me that the most he hoped for was that the treatment halted the progression of disability and anything else was a bonus. Of course I was apprehensive as although the mortality rate was low at 2-5% (at the time), it was there and something I had to consider.”

Jesse was the first person to undergo this treatment at a clinic in Adelaide. He says, “The treatment itself was very invasive and the whole experience was very isolating. I was under no illusion that it would work miracles for me but at the time the benefits outweighed the risks. However I was given the appropriate care before and after the treatment to reduce the risks and increase the chances of it working for me.”

Four years on and Jesse hasn’t experienced any major setbacks and his latest MRI showed no real disability progression. He says, “There’s no doubt it was a worthwhile treatment for me to have as I was out of all other options but it hasn’t been the miraculous experience that the media seem insistent to report on. My disability hasn’t been reversed; I still use a wheelchair every day.” He adds, “I don’t like to read much into media stories anyway and I’m pleased I don’t because I would only have been disappointed with the outcome. I’m proof this treatment is not the miracle cure they’re reporting it is. They are always going to sensationalise and I think it’s irresponsible playing on the vulnerability of people living with this unpredictable condition. All I can really say is talk to your neurologist about it to see if it’s an appropriate option for you. I’m proof that it isn’t a miracle cure treatment.”

An important aspect of Jesse’s treatment journey in Australia was his pre-treatment assessment and the provision of appropriate care before and after treatment. This level of assessment and care is not always available at clinics overseas.

Sian Shares her Story

Four short years ago I was diagnosed with MS. I had tried three disease modifying drugs with no success and my MS continued to aggressively progress. Mainly through media reports, I became aware of the great successes HSCT was having for people diagnosed with MS, in Moscow. This was an option, but not a country I had ever dreamt of visiting! I went to Moscow in Summer – the weather was very similar to Queensland in Spring; it was lovely and green; flowers everywhere. Definitely not what I expected! The 30 days of treatment and care received as a patient in the National Pirogov Medical Centre was exceptional! I took each day as it came and as a result, the time during the treatment flew!  The friendships formed with other patients and carers from Australia have been priceless with positive updates being shared often since the treatment. The recovery can be like a rollercoaster but for me, the long term benefits outweighed the probability of a continued decline. My MS has been halted and that is the main hopeful outcome when undertaking the treatment. My fatigue/fogginess vanished and speech is back to normal. I had fairly significant right side weakness before I went. Although this remains, I am working hard (with my extra energy!) to re-strengthen the muscles with slow, steady success. The treatment is not cheap and associated travel costs need to be considered. I am so grateful I had the opportunity to stop my MS in its tracks!

Article courtesy of MS Research Australia www.msra.org.au