January 23, 1997

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Sarah Goodwin, 404/727-3366 -
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A new means of delivering drugs to the brain which bypasses the blood-brain barrier is being tested at Emory University and five other sites nationally.

Joey Dixon, 44, of Greenville, S.C., is the first patient with Lou Gehrig's disease at Emory University and the third in the world to receive Glial Cell-Derived Neurotrophic Factor (GDNF), an investigational therapy for Lou Gehrig's disease, via the new device.

Lou Gehrig's disease is the familiar name for the most common degenerative motor neuron disease: amyotrophic lateral sclerosis (ALS).

On Jan. 9, neurosurgeons implanted a permanent device into Mr. Dixon's brain which will serve as the port through which GDNF may be periodically delivered. The Phase I clinical trial in which the patient is enrolled will evaluate both the tolerability of GDNF, developed by Amgen, and the effectiveness of the implanted device, which was developed by Medtronics Inc.

"This delivery system is the most exciting thing to happen in ALS research in quite some time," says Jeffrey Rosenfeld, M.D., Ph.D., principal investigator of the Emory component of Amgen's six-center trial. Dr. Rosenfeld is assistant professor of neurology at the Emory University School of Medicine. He specializes in ALS treatment and research. "GDNF is aimed at delaying or halting the degeneration of motor nerve cells which characterizes ALS."

GDNF is a growth factor that was first characterized in 1993 as having significant therapeutic potential in ALS and Parkinson's disease. It is a protein isolated from humans and produced in E. coli bacteria cells containing the DNA that expresses the human protein. The recombinant protein injected into patients has an identical amino acid sequence to the native human GDNF protein. The Medtronic device is surgically implanted into the region of the brain known as the right lateral ventricle. The catheter is placed so that a port lies just underneath the scalp which may be accessed for injections. During the trial the first injection is administered on an inpatient basis; patients receive subsequent injections as outpatients. If successful, the system might also be used to deliver drugs for other neurological conditions such as Parkinson's disease and Alzheimer's disease.

Although he has been involved in the investigation of other ALS agents, Dr. Rosenfeld said he is particularly optimistic about the current trial for two reasons: first, GDNF promoted the survival of motor nerve cells which otherwise would have died when tested in animals, and secondly, the Medtronic delivery system allows for the injection of the drug directly into the brain's cerebrospinal fluid, thus circumventing the blood-brain barrier.


The Emory team will be enrolling five ALS patients into this Phase I part of the study; 24 will participate nationwide.

Each year Emory neurologists treat about 275 ALS patients from across the Southeast. In addition to treatment with Riluzol (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 Riluzol, and clinical trials of Ciliary Neurotrophic Factor (CNTF) and Brain-Derived Neurotrophic Factor (BDNF). 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," Dr. Rosenfeld says.


(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. Riluzol 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.

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