As part of the global efforts to combat multiple sclerosis (MS), the Charcot Foundation will be launching a major initiative by conducting a clinical study (PIXAMS) whose results might represent a significant step forward in treating the disease.

One of the six standard treatments used to combat MS - namely the drug mitoxantrone - was developed in Belgium.  Highly effective, mitoxantrone nevertheless has a major drawback: its cardiac toxicity. As an "emergency treatment" which is used to block the progression of the disease temporarily, it can only be prescribed once, for a period not exceeding three years and then only to patients at risk of rapid deterioration leading to serious disability. Given that MS is a chronic disease, relapses will unfortunately be inevitable in the years following treatment with mitoxantrone.

However, research never stops and - with continuing support from the Foundation - a new molecule, similar to mitoxantrone, has been developed. The new drug, called pixantrone, has the same immunosuppressive properties as mitoxantrone but with a much lower level of cardiac toxicity. Immunosuppressors are in fact anticarcinogenic substances whose toxic properties are used to control the blood cells responsible for immune responses in order to "reduce" immunity in certain autoimmune disorders, including MS.

Thus the Charcot Foundation Charcot decided to conduct a phase I/II clinical study on 20 patients. Named PIXAMS, this project will be implemented - starting early in 2008 - in some specialised centres: The National Centre for MS (in Melsbroek), the Neurology Department of the Catholic University of Louvain (UCL, Brussels) and the Neurology Department of the University of Rennes(France).

The results of the PIXAMS project - which should enable researchers to confirm the lower cardiac toxicity and equal (or even higher) therapeutic efficacy of pixantrone in comparison with mitoxantrone - are eagerly awaited by the scientific community and patients alike. If the results are positive, pixantrone could be prescribed to a larger proportion of MS patients, repeatedly, if necessary. It would then be possible to delay the onset of handicaps by five to ten years in younger patients and to block the progression of the disease for longer periods in patients who already have some degree of disability. If the efficacy and good tolerance of pixantrone are confirmed, this will constitute a major step forward in the treatment of MS and pixantrone will eventually replace mitoxantrone.

It is hoped that the PIXAMS study and the use of pixantrone will make it possible to improve the quality of life of MS patients very significantly.
The results of the tests will be known by the end of 2011.

Since 2003, the Charcot Foundation has been financing and coordinating Phase III of the  ASIIMS clinical study. This is a therapeutic association of interferons, which are inflammation inhibitors, and an antioxidant (inosine), which protects the nervous system. It is hoped that patients will not only experience fewer relapses thanks to the interferons, but will also evolve more slowly into the progressive stage of the disease through the action of inosine.

The study is being conducted jointly in 12 Belgian neurological centres as well as a hospital in Luxembourg (*). It involves 160 patients and will not produce results before 2008. The total budget for this type of study is in excess of 1.5 million euros.

Inosine is a natural, non-patentable substance. Therefore, as it cannot become a source of major financial profits, no pharmaceutical company is interested in developing it as a medicine despite the promising preliminary tests (Phase II). Financing its activities through its own funds and donations from private and public sources, the Charcot Foundation is in a position to initiate and organise this kind of study exclusively for the benefit of patients.

(*) AZ Middelheim, AZ Sint Jan, AZ VUB, Cliniques Universitaires St Luc, CHU Charleroi,
LUC Biomedisch Onderzoekinstituut, UZ Antwerpen, Centre neurologique Fraiture, Hôpital de la Citadelle, Nationaal MS Centrum, Clinique St Pierre Ottignies, Elisabeth Ziekenhuis and Centre Hospitalier de Luxembourg.