Rituximab: a possible treatment for SPMS?

Rommer et al. Rituximab for secondary progressive multiple sclerosis: a case series. CNS Drugs. 2011 Jul 1;25(7):607-13.

Treatment options are very limited for MS'ers with SPMS. In this observational study the investigators present clinical and spinal fluid findings in 3 patients with SPMS who were treated with rituximab, a drug that target the B-cell or the cell that produces antibodies, for at least 15 months. During the observation period, no severe adverse effects occurred and the disability score stabilised in all subjects after a dramatic increase over the previous years. Conclusion: The investigators concluded that Rituximab seems to be effective in active SPMS. 

"What is wrong with this study? Firstly, it is very small; only 3 MS'ers. Secondly, it may be biased by the design, i.e. it is an open study. Thirdly, the EDSS is a poor outcome measure and too insensitive for change over such a short period of time. Do you think this paper should have been published?"

"What is right with this study? The investigators should be complemented for trying Rituximab in SPMS. In the past this is how therapies evolved; you started with an idea or scientific rationale and you then test the drug in an individual with the disease. If it appeared to work in the individual you then test it in a few more cases; this is called a case series. If the results then look promising you do a trial. Unfortunately, the regulatory, ethical and financial constraints in the NHS means that this classical route of investigator-led drug development is now virtually non-existent. It is becoming increasingly difficult to get PCTs or Commissioners to pay for off-license use of drugs like Rituximab. The usual response to an individual funding request to a PCT is "NO"; particularly if it is the off-license use of an expensive drug. Ultimately, this will hamper medical discovery and staff in the NHS will drop down the league tables of innovation. It also means that drug discovery will become almost the exclusive reserve of the Pharma Industry; this is something we should try and avoid as the needs of Pharma are often at odds with the unmet needs of MS'ers (please see previous posts on oral cladribine). Any thoughts on this?"

CoI: Multiple

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