June 16, 2003
By Kristi Nelson
For 18 years, Jan Barton has had a disease for which there's no cure. For three years, she's been treating its symptoms.
But Campath, a new drug currently on trial in Knoxville and 19 other cities worldwide, may give her new options - and new hope.
Barton, 37, has multiple sclerosis, a chronic and debilitating disease of the central nervous system. Her disease hasn't progressed to the point where she's lost mobility, and although she regularly battles numbness in her legs, she's still able to work full-time as an office manager for an obstetrician/gynecologist.
Fatigue is her biggest problem; "I really have to guard my energy and time," she said. "I know if I push (myself), I'll regret it."
But Barton knows that in time her disease could progress to the point where she would daily deal with loss of balance and muscle coordination, slurred speech, tremors, vision problems, trouble swallowing, bladder or bowel problems, or even attacks on her memory. MS destroys the myelin sheath - a protective coating over nerve fibers in the brain and spinal cord - before attacking the bare nerves themselves, and it can affect any body function related to the brain or spinal cord.
So, every other day, Barton sticks a huge needle into her stomach, thigh, arm or hip, injecting Betaseron (interferon beta-1b), one of five drugs approved by the U.S. Food and Drug Administration to "modify" MS.
Betaseron and three of the other drugs (Avonex, Copaxone and Rebif) are injected under the skin to modulate the immune system, hopefully keeping it from attacking the person's own body. The fifth, Novantrone (mitoxantrone), is given intravenously to suppress the immune system.
All the drugs have shown potential to limit the development of lesions on the brain, slowing the disease's progression - in about 30 percent of the people who use them. Barton hopes she's in that number but said it's hard to tell.
"Have I not had as many problems because of (Betaseron), or would I not have had them anyway?" Barton asked. "That's the frustrating thing with MS; unless you're in the middle of (a flare-up), it's hard to tell if (treatment) is doing anything or not."
She's also been undergoing chemotherapy with Novantrone every three months for a year, hoping to increase her odds, though she said, "It gets harder to recover from every time - that wiped out feeling."
Still, it's all there is for Barton. "I figured it was worth a try," she said.
But if preliminary studies pan out, Barton and those like her may have more options in a few years. In pilot studies spanning 10 years, Campath (alemtuzumab) - a drug approved by the FDA to treat B-cell chronic lymphocytic leukemia - suppressed MS activity for prolonged periods, and patients' relapse rates decreased with no increase in disability. Too, Campath is given intravenously for five days once a year - no injections.
Knoxville Neurology Specialists is one of 20 sites worldwide participating in a Phase II clinical trial of Campath. Neurologist Dr. Sybil Wray is principal investigator of the randomized, open-label study, which compares lose-dose Campath versus high-dose Campath versus high-dose Rebif (interferon beta-1a). Rebif is substituted for a placebo arm of the study, since it has been shown to have some effect on MS patients.
"That actually raises the bar for (Campath)," Wray said, "because they have to do better than a good drug."
Campath targets rogue cells in a patient's immune system that attack nerves in the brain. Unlike standard forms of chemotherapy, it destroys specific "bad" cells without also destroying "good" cells.
Side effects common during the first week of treatment have included fever, rash, nausea, vomiting and headache. About one-third of patients develop Graves disease, a serious but treatable thyroid condition. Some also developed an acute flare-up of MS symptoms that had to be treated with steroids.
To qualify for the study, patients must have been diagnosed within the last three years with active MS (verified by an MRI scan of the brain) and must not have already been treated with any of the interferons or Copaxone (glatiamer acetate).
While those specifications could make it more difficult to find patients, since most people now begin treatment soon after diagnosis, Wray - who practices in both Baptist's and Covenant's health systems and once oversaw Knoxville's MS Clinic - said, "I see a surprising amount of MS" in the East Tennessee area.
More than 350,000 Americans have MS. Most were diagnosed between the ages of 30 and 50 and have relapsing/remitting MS, with symptoms ranging from mild to severe that "come and go." About 200 more are diagnosed each week. Most people with MS are able to manage their symptoms long-term and lead productive lives.
"It doesn't have to be a completely debilitating disease," Wray said.
Though researchers have yet to define what causes MS, much has been learned about the disease in the past five years, Wray said.
"There are more drugs, for one thing," she said. "I think the treatment for MS is more aggressive now to stop the disease progression. There are so many trials ongoing, it's very exciting. I think there are going to more and more treatments for MS - and appropriately so, because this is a disease that affects younger people. We really need to find out how to stop this."
Campath (alemtuzumab) clinical trial
Who: Patients definitely diagnosed with relapsing/ remitting multiple sclerosis within last three years who haven't been treated with interferon drugs or Copaxone.
Info: Carla Jackson, 865-541-1121, or ask doctor for referral.
Copyright © 2003, Knoxville News-Sentinel Co.