While there's no cure, new drugs have made the feared disease recede

Zach Nielson at age 11 couldn't quite put his finger on the two words his doctors were avoiding using around him. But his mother certainly could. Deb Nielson knew people in wheelchairs, knew what it meant that Zach was waking up numb, knew how scary it was when he staggered stiffly down the hallway like a young drunk. But just over two years later, a new generation of powerful drugs has drained the power of the words "multiple sclerosis" for Zach and thousands of patients like him. The drugs have erased symptoms and reversed nerve scarring in the largest subgroup of MS sufferers, transforming for the first time victims' experience with the disease. Far from a life sentenced to relentless disability, Zach Nielson is among a growing group who feel as if their MS never happened.

"When they first told me, I was worried it would get worse and worse, and I wouldn't be able to have my dream job," said Zach, who just turned 14. "Now I know I can be a pilot."

The new class of drugs "gave me my active son back," Deb Nielson said. "I'm convinced of that now."

Eight hundred MS patients are on Zach's miracle drug, Tysabri, through the Rocky Mountain MS Center and the University of Colorado Denver's Anschutz Medical Campus. Hundreds more are on Gilenya, the first approved oral treatment for MS, and other new drugs introduced in recent years.

Anschutz MS expert Dr. Tim Vollmer calls it a "rich tool set of many drugs," and said it's "not unusual for those patients to come in and say I don't feel like I have MS anymore."

"People with MS have a reason to be optimistic," said Dr. Timothy Coetzee, chief researcher at the National Multiple Sclerosis Society in New York.

Elissa Berlinger, 25, now feels that kind of hope as she leaves Colorado for graduate school that she only recently thought might be impossible.

The MS education for Berlinger began five years ago, when she was 20 and energetic, and about to leave on a dream student-travel trip to Europe. She got strep throat and mono just before she left, then felt weakness in her hips. While traveling, she collapsed on the way to a hotel bathroom.

Her descent into the mystery of the disease followed classic lines: flare-ups every few months, numbness from hip to toe, amateur diagnosis of a slipped disc or lingering mono. X-rays for a tumor were negative, and the next step was an MRI.

"I always said the unknown is scarier than anything else," Berlinger said.

Before she got the MRI results, a colleague with MS tried to prepare her for bad news with supportive advice. Berlinger turned 24, got an MS diagnosis the next day, and promptly returned to Europe and the land of denial.

Eventually, her stateside doctors gave her a book on "the big four drugs" at the time and told her to go home and decide which one to try. They all sounded terrible, painful shots with uncertain results. "The choices were which is the lesser of the evils," said Berlinger, who works in student affairs at the University of Colorado at Boulder.

The older set of MS-fighting drugs were interferon-based, with many possible side-effects. They stopped relapses only about a third of the time for patients in the most common category, relapsing-remitting MS.

Berlinger, aided by parents more aggressive than her in researching the illness, chose to go on a clinical trial of a newer treatment. CU Anschutz, a national leader in MS research, has 30 clinical trials at a given time on measures to attack the disease.

But Berlinger's trial didn't work out; she had three relapses in 12 months, and her lesions were still growing.

In the meantime, the Food and Drug Administration had begun approving the new generation of measures that targeted MS on the molecular level, rather than the cruder blocking of immune system problems. Gilenya, the first oral drug, was effective more than half the time.

Tysabri was a bigger breakthrough, using IV treatment and stopping relapses 70 percent of the time, while removing lingering traces of symptoms. The new agents are powerful and dangerous, though, and the FDA briefly yanked Tysabri from the market when some patients with a common background virus developed brain infections.

In a rare move, the FDA allowed Tysabri back on the market with strict protocols. Patients are tested for the virus, must come in for the IV treatment every four weeks and must check in with their neurologist every three months.

Berlinger went on Tysabri in January 2011. She has had no flare-up of symptoms since then. An MRI in December showed there had been no new progression of the lesions.

Like the other 800 Tysabri patients at Anschutz, Berlinger just has to wile away two in-chair hours a month. She brings her iPad and noodles around on it while she's hooked up to the drip.

"Tysabri is a paradigm changer," Vollmer said. "We're no longer just trying to slow the disease. Now we're reversing it."

"I no longer worry about waking up numb," Berlinger said. She's leaving soon for grad school at Smith College in Massachusetts.

Success with the new drugs has dimmed the power of long-held assumptions about MS.

Deb Nielson said that between Zach's MS diagnosis and learning of the new class of highly effective drugs, "I spent many nights crying without the kids knowing."

At his worst, Zach had been in the hospital four times in six months with crippling flare-ups. Now what the Nielsons see is Zach spending weekends winter camping and snowmobiling with his Boy Scout troop, and ready to launch on his final Eagle Scout project.

What the Anschutz researchers want to do now is start combining the new class of drugs to see whether layering the chemicals can solve ongoing MS mysteries.

The new drugs are still considered treatments, not cures. In some patients, symptoms linger even when their worst relapses are blocked.

And then there are the causes of MS, which despite all the advances are still the core puzzle of the disease. The nerve-sheath damage may affect more than 400,000 people in the U.S., yet why it starts in those patients remains the thin section of medical manuals.

Scientists believe MS begins somewhere in a combination of genetic vulnerability, infections and environmental triggers. Vitamin D deficiency is one factor, explaining why Colorado, Minnesota and other states far from the equator's full sunshine report more cases. Researchers at Anschutz have likened studying MS in Colorado to studying malaria in Africa.

Without the precise triggers established in MS, the ultimate goal of a vaccine proves daunting. Yet Anschutz and other researchers are avidly pursuing one. Anschutz has launched a Translational Research Laboratory at the Rocky Mountain MS Center to push basic petri dish findings into clinics.

Vollmer is interested in a type of regulatory cell that goes haywire in the MS process. He'd like to grow a replacement cell that blocks reactivation or flare-up of the MS. If that worked, it might at least be a vaccine for family members of MS patients — they are up to 50 times more likely than the average person to also contract MS, and those cells could block them from starting the disease.

None of the science comes cheap. Tysabri and other new drugs can cost $50,000 a year for each patient. Insurance companies may pay if other treatments have failed, but patients can be overwhelmed negotiating that lab
yrinth.

"We're very fortunate. I have very good insurance," said Deb Nielson. Still, Nielson had to make extra effort because Zach's Tysabri had not yet been approved for children.

She feels relief every time she looks at Zach's face. Or through the distorted lens of his past sixth-grade graduation photo in which he was puffed up by cruder steroid treatments.

Zach's main preoccupation with MS these days is how to turn his personal knowledge into a good Eagle Scout project.

"It feels manageable now," he said.

Michael Booth: 303-954-1686 or mbooth@denverpost.com; Twitter: @MboothDP

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While there's no cure, new drugs have made the feared disease recede