A miracle drug carries some serious risks.

The latest medical studies explained.
Oct. 31 2008 7:10 AM

The Good News and Bad News About MS

A miracle drug carries some serious risks.

Problem: Multiple sclerosis is a quite common and often terrible disease that most frequently attacks young adults—especially young women. There are two phases to MS—an early one and a late one. The early phase, in which the disease waxes and wanes, is caused when the body becomes allergic to its own tissues —specifically, white matter located in the brain and spinal cord. The inflammation caused by this allergy (which attacks the cells that form a protective layer surrounding the long, cablelike structures in nerve cells responsible for carrying electrical signals) causes the early symptoms (like visual disturbances or unsteadiness in walking) and primes the body for the second phase, in which irreversible damage is done to nerve cells, causing marked weakness, fatigue, loss of balance and coordination, bladder and bowel problems, and even changes in thinking and depression. There is a lot of evidence that if the early phase is managed in ways that decrease symptoms, the late phase, during which most of the irreversible damage happens, can be delayed and perhaps even prevented.

An obvious approach, then, to treating early MS would make use of medications that alter the body's immune response to decrease auto-allergy. Indeed, some immunosuppressive medicines already play an important role in the treatment of MS, but so far, the results have not been as good as we'd like.

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New research: An important new study gives us reason to be optimistic about this approach. The University of Cambridge researchers tested Campath-1H, an artificially produced human antibody that was developed for use in a specialized cancer treatment. Campath-1H inactivates some cells important for the immune response in patients with early-phase MS. The study compared this medication with another drug already in use to treat MS, and the results were pretty striking. The benefits of the experimental treatment compared with conventional treatment were very, very impressive: After three years, the disease progressed much more slowly and the disability associated with MS actually decreased in patients who received the experimental treatment, while patients getting the conventional medication became increasingly disabled. Because the new treatment lessened early-phase inflammation and symptoms, it would be reasonable to expect to have considerable long-term benefits, since it might delay the development of the late phase of the disease, in which nerve cells are progressively damaged. (The medication being tested has no effect at all once a patient reaches this phase.)

Caveats: This drug, though both powerful and effective, isn't likely to become part of mainstream treatment for MS. Campath-1H can lead to very serious side effects. For example, 3 percent (six of 223) of the patients on whom it was tested developed ITP, an illness in which blood leaks through the walls of damaged blood vessels. Some others developed auto-allergic thyroid problems. There was one death among the patients who developed ITP. Because of these risks, the study was closed early, and it is unlikely that the drug (at least in its present form) will be promoted for use in MS. But since it is already licensed for use in cancer treatment, a few doctors are using it "off-label" for patients with MS, despite the risks.

Conclusion: The mix of good news and bad news raises agonizing personal decisions for patients and agonizing professional decisions for doctors. How do we go about balancing risks and benefits? I suspect that each of us would have a different answer to this question. For some, the prospect of advancing disability seems so terrible that they will risk the dangers of serious and perhaps lethal side effects in the hope for an improved outcome. Others might choose to avoid the immediate possible dangers of this treatment in favor of a longer-term risk of disability (and I should say that some patients with MS do very, very well), with the hope that future developments, perhaps drawing on this research study, will lead to more effective but safer, newer treatments. How would you choose?

Sydney Spiesel is a pediatrician in Woodbridge, Conn., and clinical professor of pediatrics at Yale University's School of Medicine.