November 1, 2012
New Treatment For Multiple Sclerosis Offers Promising Results
Lawrence LeBlond for redOrbit.com - Your Universe Online
A new drug to fight multiple sclerosis (MS) is showing promise as one of the “most effective” treatment options ever produced for the debilitating disease, according to UK researchers.
The promising results of two phase III clinical trials on the new treatment were published today in the journal The Lancet.
The researchers, from the University of Cambridge, said this could have significant implications for the some 100,000 people known to have MS in the United Kingdom. Most people diagnosed with MS have the relapsing-remitting version of the disease, in which symptoms can seem to disappear for a time, before suddenly returning.
Now, they believe the drug Alemtuzumab, which is used for the treatment of leukemia, may be a beneficial treatment for MS.
In leukemia, the drug controls the excess production of white blood cells. In MS patients, the drug has been found to eliminate these cells entirely, forcing a new immune system to be built from scratch which should not attack the nerves.
“Although other MS drugs have emerged over the last year, which is certainly good news for patients, none has shown superior effects on disability when compared to interferon except Alemtuzumab,” said Dr. Alasdair Coles, of Cambridge. “No other treatment has led to improvements in disability.”
“It is certainly the most effective MS drug, based on these clinical trials, but this is definitely not a cure,” Coles told the BBC´s James Gallagher.
The new studies, sponsored by Genzyme (a Sanofi company) and Bayer Schering Pharma, have shown that Alemtuzumab significantly reduces the number of relapses experienced by people with MS compared to interferon beta-1a (Rebif).
In the first of the two studies, Cambridge researchers followed 563 previously untreated patients. These patients were broken into two groups, one group took Alemtuzumab and the other received Rebif. After two years, 22 percent of the Alemtuzumab group had relapsed, compared to 40 percent of those on Rebif.
In the second study, which included 840 patients whose MS symptoms were not being controlled with other treatment methods, Alemtuzumab was administered. These treatments resulted in 35 percent of patients relapsing over two years, compared to a 51 percent relapse rate among those treated with Rebif. Moreover, patients in this study who took Alemtuzumab were less likely to have additional MS-related disabilities after the trial ended.
“Our research shows the transformative effect that alemtuzumab can have for people with MS. Patients who continue to show disease activity while on their initial therapy are especially difficult to treat. Now, we have shown that alemtuzumab works where first-line drugs have already failed. It not only reduces the chances of disability associated with MS but may even result in long-term clinical improvements,” said Professor Alastair Compston of Cambridge, principal investigator on both studies and Chair of the Steering Committee which oversaw these and earlier clinical trials.
However, the drug does not come without side-effects, such as the risk of infection from a depleted immune system which means the drug would not be suitable for everyone, warned Compston.
“Although alemtuzumab causes potentially serious side-effects, these can be identified and treated provided a monitoring schedule is carefully followed. Additionally, we think that we can identify which patients are at risk of autoimmune disease after alemtuzumab, and we are currently recruiting for a clinical trial which will explore whether we can use a drug to reduce the risk of autoimmunity in those at highest risk,” said Coles.
The drug would likely be most useful for patients where standard treatment had already failed and in a small number of patients as a first-choice drug, he noted.
Compston said that Alemtuzumab would likely be less effective when relapsing-remitting MS becomes progressive MS.
Another downside of the drug could be cost.
The drug has already been withdrawn from European and US markets as the manufacturer, Genzyme, intends to re-license it as a treatment for MS, and will most likely bump up the price tag.
“There is concern that with a license for multiple sclerosis, the cost of alemtuzumab could rise and might become too expensive for many patients and health systems,” said an editorial note in The Lancet. “Finding promising treatments such as alemtuzumab is important. But so is keeping alemtuzumab accessible and affordable.”
“Alemtuzumab has been found to be an effective treatment for people with MS - but it's only useful to them if it's available on the NHS,” Dr. Doug Brown, head of biomedical research at the MS Society, told the BBC. “We urge Genzyme to price the treatment responsibly so that if it's licensed, it's deemed cost-effective on the NHS [National Health Service].”
Genzyme said it would not come up with a price for the drug “until it is approved by regulatory authorities” and that it would “engage constructively” with the National Institute for Health and Clinical Excellence, which evaluates the cost-effectiveness of drugs for use in the NHS.
Drug licensing decisions for alemtuzumab (known commercially as Lemtrada) by both the European and US regulatory authorities are expected in 2013.
In the US, multiple sclerosis affects 400,000 people.