New stem cell and drug treatments offer hope for the MS community
Multiple sclerosis (MS) is a neurological disease caused by inflammation of the myelin sheath, which protects nerve fibres in the body. In later stages this can lead to paralysis, loss of motor function, and even death.
Since 1993 drugs have been available that can treat relapsing remitting, for most patients the first stage of the disease, but the search goes on for drugs that can treat progressive MS, as well as for for a cure.
One of the biggest breakthroughs in MS treatment has been the development of stem cell therapy, using a bone marrow stem cell transplant previously used only for cancer patients.
This ‘radical treatment for an incurable disease’ first came to public attention after a BBC Panorama documentary in January, which focused on MS sufferers involved in a Sheffield based trial for the stem cell treatment. Two of the patients showcased have since been able to walk or cycle again after the treatment. One of the patients, Louise Barrett, said: “To live without that burden [of disability] is going to be the biggest freedom for me. You can’t get any better than that.”
This development in MS treatment is far from perfect. The process involves intensive chemotherapy and the administering of growth hormones, after which the patients own bone marrow is used to rebuild an obliterated immune system from the ground up. While a trial in Ottawa showed promising results for MS sufferers, one the twenty-four trialled patients one died from a heptic necrosis brought about by the chemotherapy.
Harold Atkins, a stem cell transplant physician at the Ottawa hospital, said: “It is important to note that this therapy can have serious side-effects and risks, and would only be appropriate for a small proportion of people with very active MS.”
While this stem cell treatment can ease the symptoms of MS by returning the immune system to a point before the MS developed, in addition to any adverse reactions to the treatment, the multiple sclerosis may still return over time.
Paul Friend, who has had relapsing-remitting MS since 2003, said: “There’s no point in repairing the myelin if you’re not going to switch off the process that’s causing the damage.” As undeniably ground breaking as stem cell treatment is, without discovering the exact cause of multiple sclerosis it can only be a partial solution.
An innovative and alternative route to stem cell therapy is currently in its early stages at Johns Hopkins university in Baltimore. Using a method developed in Japan of creating stem cells from small skin donations, researchers at Hopkins have been able to create cell clusters, referred to as ‘mini-brains’. The research facility hopes to use these clusters to create a disease modifying drug that can not only reduces MS symptoms but can also actively repair damage to the myelin sheath, which protect axons (nerve fibres) in the body.
Dr. Thomas Hartung, a researcher at Johns Hopkins, said: “Generating the mini-brains, we succeeded in obtaining a substantial fraction of glial cells. About 10% of all glial cells are oligodendrocytes, which add myelin to the axons by wrapping themselves around them. This opens up to study the myelination process and its disturbance, e.g. creating models to treat MS. Whether this is suitable for drug screening the future will show.”
Currently trials of stem cell transplants are aimed at treating sufferers of the relapsing remitting form of MS. Disease modifying drugs are also used to treat relapsing remitting, but recent developments suggest there may now also be potential for treatments that can slow the effects of progressive MS (65% of those with relapsing remitting will transition to secondary progressive).
Dr. Victoria Singh-Curry, from the Institute of Cognitive Neuroscience, said: “A lot of the drugs used to treat relapsing-remitting have been trialled on a progressive form of the disease, but the results haven’t been that promising, even quite negative.
“However this is changing. There’s a drug going through trials at the moment called Ozoralizumab and there is some exciting data suggesting that it has a beneficial effect on progressive as well as relapsing remitting MS. That would be the first drug that has evidence of helping to reduce progression.”
With the potential of a drug to treat progressive MS comes the potential to shift the approach towards MS sufferers. Currently the majority of care and resources is directed at the eight in ten MS sufferers who have relapsing remitting MS, accepting that those with progressive can be cared for but not medically treated. With the development of treatment with proven results it may ensure a capacity to reduce demyelination and MS symptoms for the entire lifespan of someone diagnosed with MS.
While disease modifying drugs have transformed the treatment of MS, and the development of Ozoralizumab is an achievement that offers hope for those with the progressive form of the disease, the search for an outright cure for remains elusive.
Stem cell presents the most exciting possibility for MS treatment in modern medical history, and appears to offer something of an outright cure for a number of patients. It is also by no means an option available to all patients, or completely effective for all those who receive the treatment.
Lynn Hurst, manager of the Harrow MS centre, said: “When I started to work here over twenty years ago I was told there would definitely be a cure within ten years. I’ve found that to be a golden age, that the cure for MS is always ten years away.”
Even with the promising research at Johns Hopkins and across the world, the day when MS can begin to be eradicated completely still belongs in the not too distant future.