First oral MS drug helps mom get back to a more normal life

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Denise Malchano was diagnosed with multiple sclerosis, a disease that attacks the central nervous system, on Jan. 16, 1996.

"I lost my whole left side," recalled Ms. Malchano, 40, of Pine, a social worker and mother of a 10-year-old boy. "My face drooped; I lost my gag reflex. I lost everything on that left side."

At the time, she was able to get around with the help of a cane, but she has spent time in a wheelchair, too.

In MS, which affects some 400,000 Americans, certain white blood cells damage the protective insulation around the nerves of the central nervous system and may also damage the nerves themselves. As a result, message-carrying nerve impulses from the brain and spinal cord may short-circuit, causing reduced or lost bodily function.

Ms. Malchano has relapsing-remitting MS, the most common form of the disorder, affecting some 80 percent of all patients. In that kind of MS, patients experience symptoms for a period of time and then go into remission or a lessening of symptoms for another period of time before the cycle starts all over. She averaged one relapse per year, usually on the left side.

In primary-progressive MS, which affects most of the other 20 percent of patients, there is a more steady progression of the disease. There are also some other less common types of MS.

MS typically strikes people between the ages of 20 and 50 and affects women at least twice as often as men depending on the type.

"There are many factors in why people get MS," said neurologist Rock Heyman, director of the Multiple Sclerosis Center at UPMC. They include genetics and environmental factors. For example, Dr. Heyman said, "cloudy parts of the world have more MS." And, said neurologist Thomas Scott, director of the Allegheny Multiple Sclerosis Treatment Center, the disease "is more common in the North than the South."

The Pittsburgh area has a higher incidence of MS than others -- about one in 750, Dr. Heyman said. But it is not the highest by any means. "The highest I know is Ottawa, Canada, one in 400," he said.

There is no cure for MS. But, Dr. Heyman said, the Food and Drug Administration has approved eight drugs to help slow its damage since it OK'd the first, an interferon called Betaseron, in 1993.

Ms. Malchano said she tried them all without success but with bad allergic reactions -- until she participated in a trial at the Allegheny Multiple Sclerosis Treatment Center for a drug approved by the FDA last September.

That drug is Gilenya (generic name fingolimod) and it is the first drug approved for MS that is taken by mouth rather than by injection or IV infusion.

"The approval of Gilenya was a very exciting step because it's the first oral drug, and it appears to be quite an effective drug," said neurologist Aaron Miller, chief medical officer of the National Multiple Sclerosis Society and medical director of the Corinne Goldsmith Dickinson Center for MS at the Mount Sinai School of Medicine in New York City.

"If the person has an issue with giving themselves an injection or receiving injections, it's the only oral therapy," said Dr. Heyman, whose center also participated in the Gilenya trials. "That's probably its strongest point. ...

"We're getting to the point in MS care, it's like high blood pressure where you used a medication, you pick a medication and if it's not working, [you] look at what the other options are."

Gilenya works by preventing "the white blood cells called lymphocytes from developing in the lymph nodes and getting out into circulation so they can go attack the brain," Dr. Heyman said.

Taking a cautious approach, he, Dr. Scott and Dr. Miller have prescribed Gilenya for a limited number of patients.

"I'm being conservative about using it because of limited experience with the drug," Dr. Miller said.

"The safety we learn in studies and over time we learn does it stay safe," Dr. Heyman said.

"I think it's too soon to talk about my personal experience with the drug. I haven't been using it long enough," said Dr. Scott, who is Ms. Malchano's neurologist. "But I'm very encouraged with the results of the trials we've completed."

Gilenya is the first drug in its class, and in the only head-to-head trial so far it performed better than an older drug called Avonex. Still, it is not a wonder drug, simply another weapon to use in the war on MS.

It has, however, done wonders for Ms. Malchano.

Over the first year of the two years that she has been taking the drug she experienced steady improvement, and she has had no relapses.

"It was slowly over a year," she said of her comeback. "My balance was better -- it wasn't perfect, but it was so bad before. [Then it was] I don't need a wheelchair anymore. I can go to the grocery store. I can use a cane. It was just gradual; it wasn't all of a sudden I'm all better."

But late last summer, when she was able to start walking two miles four times a week with 10-pound weights on each leg, she knew she had found the right drug for her. Now, she said, "for the first time in a minimum of seven, maybe seven to 10 years, I'm able to balance standing on my left foot."

Those benefits outweigh the facts that the drug caused her blood pressure to go up (it's controlled by a low dose of medication) and fails to address two of her MS symptoms, fatigue and heat intolerance.

Novartis, maker of Gilenya, warns of other potential and more serious side effects that Ms. Malchano doesn't have: slow heart rate, especially after the first dose; a risk of serious infections; macular edema, a swelling or thickening of the part of the retina responsible for central vision; shortness of breath; and liver problems. Novartis also cautions that Gilenya may harm an unborn baby.

It says as well that patients may have to be vaccinated for chicken pox and wait one month before starting Gilenya. "If they get that infection it could be severe or could be fatal," Dr. Heyman said.

Because there were several cases of skin cancer reported during the trials, Dr. Heyman said the UPMC center also has patients screened for skin cancer before starting a regimen of Gilenya.

Ms. Malchano went through a "strict regimen of screenings [that] changed as we went on in the study" and expects them to continue. "They're still going to keep an eye on all that stuff for taking the medicine."

But in the meantime she's making up for lost time, working and enjoying her son, Brandon.

"We now go hiking. I can play with him. We jump on the trampoline together. I play miniature golf with him. We go camping together -- all the things that caused difficulties before, we're now doing and that really is the biggest blessing of this Gilenya," she said.

"What I always tell people [is] even if Gilenya isn't right for you don't give up," she said. "There is a medicine out there that will help you. I've been battling this for such a long time and finally found what works for me."

Correction/Clarification: (Published July 20, 2011) Denise Malchano of Pine, a social worker, mother of a 10-year-old son and a multiple sclerosis patient doing well on a new drug called Gilenya, is 40. Her age was incorrect in a story in Monday's editions.

Pohla Smith: or 412-263-1228. First Published July 18, 2011 4:00 AM


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