November 16, 2003
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Neuroprotective drugs may hold hope for optic neuropathies, surgeon says

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ANAHEIM, Calif. — Surgeons managing patients with incurable optic neuropathies should consider enrolling these patients in current clinical trials of neuroprotective drugs, a speaker here suggested. Another option is to seek a compassionate-use exemption for a potentially neuroprotective drug.

“These are our options because most optic neuropathies do not have effective treatments,” said Leonard A. Levin, MD, PhD, here at the American Academy of Ophthalmology meeting. At present, glaucoma and compressive optic neuropathy are the only treatable optic neuropathies, he said.

According to Dr. Levin, the pathophysiology behind conditions such as ischemic optic neuropathy, Leber’s hereditary optic neuropathy and optic neuritis is minimally understood, and therefore these conditions are difficult to treat. At present, there are no Food and Drug Administration-approved neuroprotective drugs that can effectively prevent the retinal ganglion cell death that occurs during these optic neuropathies, he said.

“We must recognize that we have no information from a randomized clinical trial to guide us in our decision making,” Dr. Levin said.

Neuroprotectors such as beta-1 antagonists, NMDA antagonists and anti-apoptotic agents have been studied in animals and more recently in humans for Alzheimer’s disease, spinal cord trauma and other nonocular conditions. Clinical trials of potentially neuroprotective drugs are now in progress for some ocular diseases. The drugs being tested include brimonidine for ischemic optic neuropathy and normal-tension glaucoma and memantine for glaucoma, Dr. Levin said.

He encouraged surgeons to enroll their patients in these trials, when possible, or treat patients through “compassionate use” of a potentially neuroprotective drug. This should be done only with the highly informed consent of the patient, he noted.

“Even if the treatment given is ineffective, we can have hope for the future that stem cell replacement of ganglion cells may one day be achieved,” Dr. Levin said.