Guarded Optimism for Experimental MS Drug
Alemtuzumab Appears to Repair Damage to Brain in Patients With MS
Response 'Unprecedented' continued...
But Cole says the fact that the experimental treatment actually appeared to reverse damage to brain tissue caused by MS is the most exciting finding from the study.
"That is unprecedented and very big news," he says. "Another important part of the strategy involves treating patients very early in the course of the disease with the most effective agents we have."
The study was funded by the drug companies Genzyme and Bayer Schering Pharma AG, which own the marketing rights to alemtuzumab.
In a Wednesday morning news conference, Genzyme Medical Director Susan Moran, MD, addressed the death that occurred during the study.
The patient died from an autoimmune-mediated blood condition known as idiopathic thrombocytopenic purpura (ITP). Moran said the death could have been avoided if ITP had been recognized as an adverse effect of the treatment.
"Unfortunately, the patient had symptoms of ITP but did not seek medical attention prior to diagnosis because this was not recognized as an adverse event," she says.
Once the risk was known, patients in the study were monitored closely for ITP. Five additional cases were identified, and all were managed with treatment.
Close Monitoring Essential
Moran says all the patients enrolled in the phase III trial and all patients who end up taking the drug if it is approved for MS will have to be monitored closely for this adverse effect.
Researchers are also working on ways to identify patients who are most likely to develop ITP before treatment and to identify the MS patients who are most likely to benefit from early, aggressive therapy.
In an editorial published with the study, neurologist and longtime MS researcher Stephen L. Hauser, MD, writes that it is not yet clear if alemtuzumab will prove to be an acceptable first-line treatment for early MS.
Hauser is chief of neurology at the University of California, San Francisco Medical Center.
"Taken together, the toxic effects associated with alemtuzumab considerably dampen any enthusiasm for its routine use in patients with multiple sclerosis until more is known about its long-term safety and sustained efficacy," he writes.
John Richert, MD, of the National Multiple Sclerosis Society (NMSS), tells WebMD that it is increasingly clear that aggressive treatment early in the course of disease is a better strategy than waiting until MS progresses.
He agrees that alemtuzumab's role in MS treatment remains to be determined.
Richert is vice president for research and clinical programs for NMSS.
"This may be the breakthrough drug we are looking for, but we won't know that until the phase III study is done," he says.