Christy Sloan rises at 5:30 a.m. weekdays, in time to bathe and dress herself, despite the failing arm muscles that make her fingers feel like 10-pound weights.

She allows two hours to get ready, before she'll take her desk at First Union Bank by 8 a.m. On a good day, the 32-year-old Charlotte woman might sleep an extra half-hour -- a tradeoff for boldly asking a neighbor at her condo to help button a blouse or zip her zipper.

"It took a long time for me to swallow my pride to do that,'' admitted Christy, an Asheville native and one of 30,000 Americans battling the incurable neuromuscular disease amyotrophic lateral sclerosis, or ALS. "The toughest thing is giving up your independence and taking help when it's offered; but it pays off."

Christy can't afford to waste a half-hour these days on wardrobe minutiae. A star field hockey player who once went head-to-head with four older brothers in skiing, basketball or football, she now pours her declining strength into defying the three- to five-year life expectancy most ALS patients face. Starting with a hometown fund-raising concert in Asheville Saturday, she and her family hope to raise millions of dollars to speed new treatments for ALS, including drug "cocktails" and stem-cell transplants. "It's a race, because you've got this sand in the hourglass going out by the day,'' said Steve Sloan, a brother in Riceville who is hosting Saturday's 7:30 p.m. benefit at Diana Wortham Theatre. "A pessimist would say, 'Your efforts are futile. It's not going to help your sister.' We just want to feel like we're doing absolutely everything we can do -- not only for her but for others."

Seeking cures for an 'orphan disease'

Also known as Lou Gehrig's disease, ALS affects one in 10,000 people in most parts of the world, according to the Muscular Dystrophy Association, with 5,000 patients diagnosed each year. Yet their relatively small numbers -- compared to millions threatened by AIDS or cancer -- make it a low priority for government grants and drug makers, researchers argue.

"ALS is an orphan disease," said Dr. Jeffrey Rothstein, a Johns Hopkins University neurologist who is at the forefront of ALS research. "And it takes time to translate work in the lab to a patient -- often a long time."

The disease progressively destroys nerve cells in the brain and spinal cord that control muscles used for swallowing and breathing, plus major muscles in the arms, legs, back and neck. The lone approved drug for ALS, riluzole, prolongs life by only a matter of months.

But experiments with stem cells -- the specialized cells through which researchers aim to heal nerves damaged by ALS, Parkinson's, Alzheimer's and other diseases -- hold promise for scientists including Rothstein, who last fall teamed with Johns Hopkins colleagues to restore movement in paralyzed mice through stem-cell transplants.

"When a cell dies in the brain, it's gone for good. So even the best drug we could develop today would not give (ALS) patients back their strength,'' Rothstein said. "Only stem cells could give the patient back something ... if we're lucky enough to get stem cells to work."

While Rothstein credits private donors with helping to accelerate research with stem cells drawn from human embryos, Christy Sloan supports the federal funding limits President Bush announced earlier this month on that branch of stem-cell experiments.

"There are ways to make tremendous strides in stem-cell research ... that would not encourage production of what may or may not be a viable life solely for research,'' she said. "I have a lot of faith in drug screening with a variety of FDA-approved drugs that are already on the market, testing their efficacy with ALS."

'Sloan's disease'?

Much of Christy's faith rests on Dr. Jeffrey Rosenfeld, a Charlotte neurologist who has cared for her for two years. She met him after her first symptoms surfaced at age 3o -- a decade or more before the disease generally strikes.

Shaping up for an annual ski trip to Vail with her girlfriends, Christy had spent January 1999 working out hard at the YMCA. But a muscle spasm that developed in her left biceps -- and sudden difficulties lifting cups and pens -- led her to a doctor the next month.

"I thought I had a pinched nerve, and so did my doctor,'' recalled Christy, whose physician soon referred her to a specialist when the problems persisted.

Neurological tests that spring pointed to early-stage ALS, a diagnosis she received at Winston-Salem's Bowman Gray in May 1999. When she sought a second opinion from Rothstein at Johns Hopkins, he confirmed the tentative diagnosis.

But because ALS more commonly affects men, and because of Christy's age and limited symptoms, the Sloans waited hopefully while Rosenfeld ruled out other disorders that can mimic ALS. Hesitating initially to label Christy's case ALS, Rosenfeld nicknamed the condition "Sloan's disease" and monitored her closely.

By November 2000, Rosenfeld was ready to include Christy in a clinical trial studying ALS patients' response to Oxandralone, a steroid that can counteract weight loss that may result from difficulties chewing and swallowing. His move coincided with a turning point in Christy's illness, when the weakness that had been confined to her arms spread to her legs and voice. Her strides gradually slowed to a shuffle --which Christy likens to walking in ski boots -- and a slur crept into her speech.

Curious observers noticed her symptoms, and Christy and the people closest to her shifted into crisis-intervention mode: If it would take a miracle to save her, they would do all they could to coax along a cure.

The Sloans drew inspiration from high-profile patients in their 30s whose stories echoed Christy's: New York theater and film producer Jenifer Estess and suburban Boston's Stephen Heywood.

Refusing to accept a death sentence at age 35, Estess and her sisters spearheaded the non-profit Project ALS, parlaying their Hollywood and Broadway ties into more than $6 million in donations to ALS research (including Rothstein's work). In Newton, Mass., engineer-turned-biotech-upstart Jamie Heywood launched the non-profit ALS Therapy Development Foundation, aiming to expedite new therapies or drugs that could prolong his brother Stephen's life.

"It was like a light bulb came on,'' Christy said, recalling their exposure to the Estess and Heywood families, as well as a memorable sermon from her minister in Charlotte. "He said, 'God will give you the strength, but don't wait on the answer. Be the answer.'"

Charity would aid Charlotte clinic

At Rosenfeld's Carolinas Neuromuscular/ALS-MDA Center, Christy is among the outpatients who crowd into a Charlotte waiting room quarterly for a marathon day of specialized care. Traveling here from three countries and 22 U.S. states, these ALS patients come for respiratory, speech and physical therapy, nutrition counseling, neurological check-ups and to get equipped with the latest technologies to adapt to their disabilities.

"I want my patients to see themselves as healthy people with a disability, rather than as disabled people who can do a few healthy things,'' said Rosenfeld, who exhorts patients to "live with the disease" rather than dying with it.

The waiting room is abuzz with camaraderie and optimism, despite patients' challenges. Some bite down on paper towels to control involuntary drooling. Respirators or abdominal feeding tubes distinguish others. Some maneuver the halls in wheelchairs, carrying laptop computers to help them communicate. Others wear neck or ankle braces, or simply pass notes to each other on spiral notebooks.

During Friday's clinic, Christy's second, she meets a newcomer. Tristan Hagerman, 26, of Kannapolis has been diagnosed with a motor neuron disorder that may, likewise, turn out to be ALS. He exchanges information with Christy about Saturday's concert, praising the ALS Race for Research charity the Sloans have organized.

"It's fantastic,'' Hagerman said. "But it seems like a lot of trouble when you'd think there would be easier avenues of support."

Christy and her backers are quick to credit the Estess and Heywood families for their originality and leadership, saying they merely want to pursue new, untapped funding streams.

"I want this money to be spent immediately,'' Christy explains. "I want the ability to direct the funds to whoever is leading the pack."

Those leaders will include Rosenfeld, who plans to study whether drug or dietary supplement combinations can more aggressively fight ALS, similar to the potent mixes that have helped HIV patients live longer.

"We're working here to launch a multidrug cocktail," he said, for example. "We're trying to cull the best, most-promising of those to see if we can determine any synergy."

Rosenfeld predicts a ripple effect from the Sloans' crusade.

"It would mean a great deal to other people: It could add momentum to a new or existing research program. It may mean a new clinical trial,'' the neurologist added. "Or it may mean investing in an area of research that has been too speculative to pursue."

Tirelessly promoting the fledgling charity, Joanna Sloan of Asheville is fond of quoting her only daughter: "Doing nothing is not an option."

Oldest brother Jay Sloan, a volunteer at Rosenfeld's clinic, describes Christy's attitude in competitive terms.

"She's a Sloan,'' he said, smiling. "She's not ready to wave the white flag yet."

UNDERSTANDING AMYOTROPHIC LATERAL SCLEROSIS

Although a French neurologist identified the disease in 1869, amyotrophic lateral sclerosis came to worldwide attention in 1939 when it ended the Hall of Fame baseball career of New York Yankees first baseman Lou Gehrig. He died of the illness in 1941, at age 37.

The disease causes specialized nerve cells in the brain and spinal cord, called motor neurons, to gradually disintegrate. Most people who develop it are between ages 40 and 70.

When motor neurons die, patients lose the ability to initiate and control muscle movements, leading to total paralysis in the late stages. Involuntary muscles, cognition, the senses and the voluntary muscles that move the eyes generally aren't affected.

No cure or treatment halts or reverses ALS. Death usually results from paralysis of respiratory muscles, and typically follows within five years of diagnosis for up to 70 percent of patients. Up to 10 percent survive more than 10 years.

The past decade has unlocked a wealth of new understanding about the disease, which researchers hope will expedite new therapies.

Existing drug treatments can control involuntary muscle twitching, cramping and excessive salivating associated with ALS. Health care focuses largely on extending survival by maintaining patients' mobility and independence, often through the use of adaptive technologies.

SOURCES: Muscular Dystrophy Association; ALS Association

ABOUT THE CHARITY

For information about the non-profit fund the Sloan family has established, contact:

ALS Race for Research
Attention: Christy Sloan
3708-4 Selwyn Farms Lane
Charlotte, NC 28209
www.als-rr.org

Limited seating is available for the charity's kickoff event, a 7:30 p.m. concert Saturday at Diana Wortham Theatre featuring Irish musicians Paddy Keenan and Tommy O'Sullivan. Call 258-8744 for information.

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