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Mary Filipi, UNMC nurse practitioner, evaluates Andrea Kunkel during a neurological exam. Kunkel, who has lived with multiple sclerosis for four years, currently suffers from fatigue and episodes of pain in her arms and legs. She receives weekly injections for her disease. |
“It’s a very promising medication that appears to slow down the disease more than any of the other medications we have now,” said Eliad Culcea, M.D., UNMC assistant professor of neurology, who was part of the UNMC team. “It showed a significant decrease in the reduction of new lesions in the brain, which means patients can be functional a longer period of time without disease symptoms.”
Results of the phase II, six-month preliminary study were published in the Jan. 2 issue of the New England Journal of Medicine. UNMC enrolled and evaluated some of its multiple sclerosis patients for the study, according to Kathy Healey, UNMC nurse practitioner and coordinator of the study. UNMC health professionals see 800 to 900 MS patients in its University Medical Associates clinic.
What is multiple sclerosis?
Multiple sclerosis is a disease of the white matter of the brain and spinal cord that affects the central nervous system. Symptoms of the disease, such as weakness, tremors, loss of vision, cognitive changes, depression and motor problems are episodic, meaning they come and go.
The frequency of symptoms varies from patient-to-patient from one short episode a year to those lasting six months. With each flare of symptoms, the number of brain lesions increase and the disease advances, according to Mary Filipi, UNMC nurse practitioner who was involved in the study. If left untreated, half of MS patients become wheelchair dependent within 15 years of disease onset.
Medication manages symptoms and acute attacks. However, the use of current medications being prescribed slow the disease by only 30 to 60 percent, according to one of the UNMC study group members.
Preliminary study results
Preliminary results of the study showed the new drug dramatically reduced the number of new brain lesions in patients with MS and cut the number of relapses in half. In Crohn’s patients, the study drug increased the rate of remission and improved the patients’ quality of life.
In the randomized, double-blind trial, researchers studied 213 patients with what’s called relapsing-remitting or relapsing secondary progressive multiple sclerosis. At every 28 days over a six-month period, about one-third of the patients received 3 mg of intravenous natalizumab per kilogram of body weight, another third received 6 mg per kilogram and about a third received placebo every 28 days for 6 months.
Researchers found marked reductions in the number of new lesions in both groups receiving the drug: 9.6 per patient in the placebo group, as compared with 0.7 in the group given 3 mg of the drug and 1.1 in the group given 6 mg of the drug. Twenty-seven of 71 patients in the placebo group had relapses, as compared with 13 in the group given 3 mg of natalizumab and 14 in the group given 6 mg of natalizumab.
A new treatment hope
The drug, a monoclonal antibody, works by blocking the adhesion of immune cells to blood vessels and can inhibit movement of immune cells from the bloodstream into the brain, according to the published paper. According to researchers, the drug is a new class of medication that prevents inflammation by interrupting the process of inflammation.
Currently, MS patients may receive injections anywhere from once a week to every day. “If this drug turns out to be effective, it would be very, very helpful for patients to only receive one injection every 28 days,” said Ekaterini Markopoulou, M.D., Ph.D., assistant professor of neurology at UNMC. “If it turns out that this drug can decrease the disease burden and resulting disability in the long-term, it would be an important development in the treatment of MS.”
MS is a difficult disease for patients to handle, Filipi said. “You don’t know when episodes will hit you, what part of the body it will affect, how hard it will hit, how long it will stay and how much deficit you will have when it goes away. A teenaged athlete ended up having to periodically use crutches because of the debilitating effects of MS. He told me he’d rather have had a stroke because he would then know how he would be from day-to-day. The uncertainty he felt was heart-wrenching.”
Plans to seek approval for the drug
The drug companies that funded the study, Elan Pharmaceuticals and Biogen, expect to seek approval for the drug in the United States and Europe at the end of 2003.
Number of cases, life expectancy
In Nebraska, about 110 to 140 cases per 100,000 people will occur, according to the National Multiple Sclerosis Society. An estimated 1,600 to 1,800 Nebraskans have MS.
Life expectancy for people with MS has steadily improved. In 1890, the disease was treated with herbs and bed rest, and life expectancy was five years, according to the NMSS. By 1970, life expectancy rose to about 32 years, and today, people have a normal life expectancy.
Nationwide, it is estimated 250,000 to 350,000 people have multiple sclerosis, which is often diagnosed between age 20 and 40. It is more common among Caucasians, particularly those of northern European ancestry, and is more common in women than in men. Some of the potential causes of the disease are believed to be viruses, as well as environmental, genetic, and immune system factors.
More extensive studies underway
Researchers say although the study found no major side effects to the drug, a more extensive phase III study is now underway to study it effectiveness.
UNMC investigators involved in the study
“Our research team should be congratulated for contributing to such a high quality international study that brings a potentially unique, well-tolerated and very effective therapy for multiple sclerosis,” said Pierre Fayad, M.D., Reynolds Centennial Professor and chairman of the UNMC department of neurological sciences.
Besides Drs. Culcea and Markopoulou, Healey and Filipi, other members of the UNMC research team were: Agapito Lorenzo, M.D., clinical associate professor, and Sandy Roberts, nurse. The late Jinan Al-Omaishi, M.D., was principle investigator of the study at UNMC.