September 8, 1997

Media Contacts: Sarah Goodwin, 404/727-3366 - sgoodwi@emory.edu
Kathi Ovnic, 404/727-9371 - covnic@emory.edu

Patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, are being enrolled by Emory University neurology researchers into the first international clinical trial designed to study the effects of a new drug which may stimulate production of growth factors within nerve cells. The trial also represents the first large study where the an interaction between the experimental drug and the only currently approved medication for ALS will be examined.

ALS is the most common degenerative motor neuron disease. Motor nerve cells in the brain and spinal cord control motor functions such as breathing and movement. The outpatient, Phase III clinical trial will evaluate the effect of an experimental drug, 57746A on survival, breathing function and strength in patients with ALS. In addition, the drug will be evaluated in patients concurrently taking Riluzole (rilutek), which has been the only approved treatment for ALS.

The study drug binds to a serotonin receptor in the brain and subsequently promotes the action of growth factors essential for nerve cell growth and survival. Alternatively, the drug itself may act as a growth factor to preserve the life of motor neurons, says principal investigator of the Emory study, neurologist Jeffrey Rosenfeld, M.D., Ph.D. Emory has also been recently recognized by Muscular Dystrophy Association as an ALS Center to promote further treatment and research in ALS; Dr. Rosenfeld is the director of the ALS Center.

Qualified participants will take one pill a day for the 18 month duration of the study. Participation will involve approximately 12-14 visits to The Emory Clinic, Building A, at 1365 Clifton Rd., N.E., Atlanta.

The trial is sponsored by Sanofi Researche, a French pharmaceutical company. About 2,000 ALS patients will be enrolled through more than 40 medical centers worldwide, including centers in Europe, Canada and the United States. Dr. Rosenfeld plans to enroll between 70 and 100 patients in the Emory Center.

In addition to this newest trial, Dr. Rosenfeld has been involved in a Phase I study of a different experimental agent, glial cell-derived neurotrophic factor (GDNF) injected directly into the brain. That study involves surgical placement of a catheter within the brain through which GDNF is administered monthly.

For information on ALS treatment and research, call 404-778-3754 to speak Dr. Rosenfeld or study coordinators Meraida Polak, RN or Tom Pianta.


(Amyotrophic Lateral Sclerosis)


  • The worldwide frequency of affected individuals is estimated to be five to 10 persons per 100,000.

  • During ALS, motor nerve cells in the brain and spinal cord which control motor functions such as walking, talking and breathing, selectively and progressively deteriorate. They do not regenerate.

  • The condition is fatal and has no cure. Riluzole is the only drug approved for ALS treatment by the Food and Drug Administration. It has been shown to have only a modest effect, prolonging by about three months the time patients have to breathe without assistance before requiring a ventilator.

  • The disease can be inherited (in about 10 percent of patients) or acquired, and the mean survival is between two to five years; few patients (up to about 10 percent) survive longer.

  • ALS became known as Lou Gehrig's disease after the major league baseball player by that name died of the disease. Lou Gehrig is said to have had a "brilliant major league career" as a first baseman for the New York Yankees before becoming affected by the disease. He tallied 493 home runs, 23 career grand slams and played in a record 2,130 consecutive games (14 seasons). He stopped playing just two years before his death on June 2, 1941.

  • THE ALS FOCUS AT EMORY: Each year Emory neurologists treat about 275 ALS patients from across the Southeast. In addition to treatment with Riluzole (rilutek), doctors also offer pulmonary evaluation and treatment, nutritional evaluation and treatment, access to occupational and physical therapy, and psychological services to assist the patient and family in coping with the disease. Emory neurologists have participated in several major studies of ALS agents, including the later development stages of Riluzole, clinical trials of Ciliary Neurotrophic Factor (CNTF), and Brain-Derived Neurotrophic Factor (BDNF) and Glial cell line derived neurotrophic factor (GDNF). In addition to participation in the Amgen GDNF trial, the researchers expect to begin investigating another novel growth-promoting factor in 1997-98. "Emory has been and continues to be at the forefront of the treatment of this devastating disease," says ALS expert Jeffrey Rosenfeld, M.D., Ph.D., assistant professor of Neurology, Emory University School of Medicine.

For more general information on The Robert W. Woodruff Health Sciences Center, call Health Sciences News and Information at 404-727-5686, or send e-mail to hsnews@emory.edu.

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