In 2002, a placebo controlled trial of patients with either secondary progressive multiple sclerosis (MS) or worsening multiple sclerosis demonstrated that intermittent infusions of Novantrone® or Mitoxantrone, a previously used cancer chemotherapeutic agent, would slow the progression in MS for at least two years. As a whole, the disability of the treated group actually decreased by a small amount. Only 5 patients out of 60 had worsening of their MS by more than 1 point of the EDSS (Extended Disability Severity Scale) at the end of the two year trial. A whole host of secondary endpoints ranging from better looking MRIs to decreased hospitilizations were all better in the treated group. Needless to say, this is considered a very positive trial.

The downside to Novantrone is that it is a chemotherapeutic agent and there is known cumulative toxicity for the heart from this medication. This limits its use to about 2 3/4 years. In the trial, the patients were stopped after two years and it has been reported that they have done well after the third year as well. The best method of dosing this medication is not known and its best method of use awaits further trial but for those patients who are progressing despite the use of standard immunomodulating agents, it is a great step forward. Some neurologists attempt to combine the medication with immunomodulating agents but there can be increased lowering of the white count with the interferons combined with this agent. There is some theoretic interference of this medication with copaxone and therefore some neurologists will discontinue copaxone as well.

Another unanswered question is what do to when the patients hit their lifetime acceptable dose. There are no clear answers but certainly if the patient only received one type of immunomodulating therapy, copaxone or interferon, then placing the patient on the other type would seem to make the most sense. Hopefully, new agents like antegren will become available before patients finish their use of novantrone® giving neurologists and their patients a new option.