The most effective treatment for multiple sclerosis is hardly used
In many patients with multiple sclerosis, quality of life is so low that HSCT treatment appears to be the best option – a one-off treatment with medium/low dose (myeloablative/non-myeloablative) chemotherapy, which eradicates autoreactive lymphocytes. This gives the patient a good chance of escaping chronic health problems due to disease activity, functional decline, medication side effects and the transition to secondary progressive multiple sclerosis (chronic neurodegeneration) (3).
Disease-modifying drugs have the same therapeutic window as HSCT, but have only an immunomodulatory/immunosuppressive effect on autoreactive lymphocytes and can neither halt the disease nor prevent the transition to secondary progressive multiple sclerosis (3). Side effects significantly reduce quality of life in many patients, and the most effective disease-modifying drugs have serious side effects that limit their duration of use. Health problems often persist because of continued disease activity.
Internationally, however, most neurologists still consider chemotherapy with autologous stem cell support to be an experimental treatment, even though it has yielded good results for more than ten years (7). Many countries require that such treatment be performed within prospective randomised controlled trials, in which the control group receives disease-modifying drugs and the intervention group HSCT treatment. Neurologists expect it takes several years before there are sufficient data to judge whether the treatment can be offered outside such studies.
The inclusion criteria for these studies are very stringent – they are based upon the type of multiple sclerosis, age, annual relapse rate, contrast-enhancing lesions on MRI and unsatisfactory effect of at least two disease-modifying drugs. Phase 2 studies from university hospitals in Sweden, Italy, Israel and Russia, among others, suggest that HCST treatment is also effective in progressive multiple sclerosis, in elderly patients, in those who do not have annual relapses and in those without contrast-enhancing lesions (8). Today, these patients have few if any places to which they can turn for HSCT treatment.
For me, it is incomprehensible that one of the inclusion criteria is that patients must have used two disease-modifying drugs. For many, this means that they no longer fulfil the criteria regarding age and secondary progressive multiple sclerosis, that they suffer increasing irreversible nerve damage, and that the treatment comes too late.