Summary: This post summarises the current scientific perspective of HSCT and attempts to put it as a treatment for MS into perspective.
Like most modern treatments that get adopted prematurely in MS, i.e. before the necessary science is done to convince the wider community, they are driven by social media. We have a lot of experience on this blog with CCSVI; in fact, CCSVI is one of the main reasons why I started this blog.
The question I now find myself asking has HSCT become the new CCSVI? Could the current support for HSCT as a treatment for MS getting ahead of the curve? Is the HSCT movement in MS becoming an anti-science movement, i.e. adopted against the predominant scientific opinion of the time? The wider MS community, pwMS, neurologists, scientists, charities, healthcare systems, etc. have agreed that there is a place for HSCT in the treatment of MS, but until we have better evidence of its relative benefits and risks compared to licensed DMTs we should be limiting HSCT to patients with more active MS who have failed at least one (high-efficacy), or two, established DMTs. In direct opposition, there are a group of HSCT-supporters who disagree and want everyone with MS to have the option of being treated with HSCT first-line. The latter recommendation is not supported by the science/data.
- The ‘dissident scientists’ who lends credibility to the theory.
- The ‘cultropreneurs’ who peddle the therapy.
- The ‘living icons’ and ‘miraculous responders’ who have been treated.
- The ‘praise-singers’, ‘journalists’ and ‘politicians’ who promote the theory
I agree that HSCT looks like a very effective treatment for MS. Does it cure MS? No, we can’t make that claim yet. Cohorts of HSCT-treated pwMS have not been followed long enough to justify this claim. Is it better than established or licensed DMTs? Possibly, indirect comparisons suggest it might be, but until we have head-2-head studies we can’t make that claim. Is it safer than established DMTs? It depends on what you are comparing it with and the specific outcome. For example, when it comes to the risk of neutropenic sepsis it is riskier than licensed therapies. Similarly, it has a much higher risk of ovarian toxicity (premature menopause) than established treatments. Safety, or relative safety, is another reason why we need large head-2-head studies to define the risks better in well-controlled randomised trials of sufficient size.
It is, however, reassuring to note that in the UK the costs of HSCT under the NHS appear very reasonable compared to licensed treatments. This could potentially make it a very cost-effective option for treating MS in the future. There are well established local and regional guidelines for HSCT and NICE is trying to establish national guidelines.
1. London MS-AHSCT Collaborative Group eligibility criteria
2. The Bristol guidance document for pwMS considering HSCT abroad
3. NICE draft technology appraisal for HSCT as a treatment for MS in England.