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Doctor studying effects of antibiotic on patients with Lou Gehrig's' disease

FRESNO, Calif. — About 600 patients nationwide could be among the first to know if an antibiotic holds the key to slowing the progression of an incurable, degenerative nerve disease that most know as Lou Gehrig's disease.

A doctor in Fresno, Calif., is among those enrolling patients with ALS (amyotrophic lateral sclerosis) in a national clinical trial to study the benefits of ceftriaxone, an antibiotic given intravenously to fight bacterial infections.

The disease affects motor neurons in both the brain and spinal cord that control muscles. As the disease progresses, the nerves die and paralysis occurs.

Research already has shown that ceftriaxone, a member of a class of antibiotics called cephalosporins, appears to protect motor-nerve cells from damage, said Dr. Jeffrey Rosenfeld, chief of neurology at the University of California, San Francisco-Fresno medical education program. The drug could be a way to "slow or stop the progressive degeneration of the nerve cells," he said.

The first phase of the study, which began in 2006, found the drug was safe for patients, Rosenfeld said. The second phase found the optimal dose. Now, the third phase of the trial will see if the drug improves muscle strength, respiratory capacity and length of survival, he said.

Rosenfeld wants to enroll 15 patients from his area in the study. Nationwide, researchers need 600 enrollees in the double-blind trial — patients randomly are chosen to either get the drug or to get a placebo. Study participants receive intravenous injections twice daily.

"The trial ends when the last patient enrolled is on the drug for a year," Rosenfeld said. So far, he's enrolled two patients.

Rosenfeld said he doesn't know how many ALS patients there are in his region, but he's been busy diagnosing and treating them since coming here in 2008, he said.

About 5,600 people in the United States are diagnosed with the disease each year, according to the ALS Association Web site. And about 30,000 Americans may have the disease at any one time. The disease is often referred to as Lou Gehrig's because Gehrig, a Hall of Fame baseball player, was diagnosed in the 1930s.

The disease progresses at different rates, but early symptoms often include problems with speech, and muscle weakness in the hands and arms. There is no cure. Life expectancy is from two to five years, the association says. But Rosenfeld said with aggressive treatment and clinical trials of new drugs, survival and function improve.

Doctors don't know the exact cause of the nerve deaths — but they have theories, Rosenfeld said.

Researchers believe an excessive amount of glutamate, a substance that excites nerve cells, is involved. Excitement stresses nerve cells and promotes their death, he said. Glutamate, which normally is released and then disappears, stays around longer in ALS patients, Rosenfeld said.

Laboratory studies have shown that ceftriaxone increases the level of a protein the body uses to get rid of glutamate, he said.

So far, only one drug — riluzole — has been approved by the federal Food and Drug Administration to slow progression of the disease. That drug showed some positive benefit on survival, Rosenfeld said. Patients in the ceftriaxone study will be allowed to be on riluzole, he said.

Jacquelyn McNeal, 40, of Bakersfield, Calif., joined the drug trial about a month ago. Her husband, Patrick McNeal, gives her the twice-daily injections of ceftriaxone.

Her first symptom was a persistent hoarse voice that started about a year and a half ago. Tests revealed one of her vocal cords was paralyzed. She also noticed cramping in her fingers and hands before she began losing muscle strength in them, Patrick McNeal said.

The couple hopes the drug will slow the progression of the disease. Since being diagnosed last December, Jacquelyn McNeal has lost the use of her hands and arms and has difficulty speaking. "Hopefully, it will rebuild some of my strength," she said.

Jacquelyn McNeal was working as an office teacher in Bakersfield until she had to leave the job when the paralysis expanded to her hands and arms, Patrick McNeal said. "I can't believe how fast it's progressed in just a year," he said.

The McNeals understand the ceftriaxone study holds hope — but no promise.

"There's so little out there for this disease," Jacquelyn McNeal said. It's worth it to be a part of the study, she said. "Even if I'm on the placebo — the information will help other people."