ROANOKE TIMES
Copyright (c) 1997, Roanoke Times
DATE: Friday, January 17, 1997 TAG: 9701170063
SECTION: NATIONAL/INTERNATIONAL PAGE: A-10 EDITION: METRO
DATELINE: MIAMI
SOURCE: Associated Press
AN EXPERIMENTAL treatment may prolong life for those with Lou Gehrig's disease, an incurable neurological disorder.
Kenneth Sibley let surgeons drill a pinhole in his skull and insert a tiny flexible tube deep in his brain in hopes it will buy him a few more years of life.
It is the first time doctors have used an implanted catheter to regularly administer medicine they hope will slow Lou Gehrig's disease, an incurable neurological disorder that slowly destroys the body.
Sibley, a 39-year-old commercial real estate agent who doctors say has only three years to live, said he is willing to be a human guinea pig despite the risk that the treatment itself could cause bleeding that could kill him.
``A surgical implant of a device into my brain is a scary proposition, to say the least, but I am determined to fight this disease as aggressively as possible for the sake of my family,'' Sibley said, using a tiny keyboard to communicate.
The Birmingham, Ala., man, who is married with three children, received the implant on Dec. 4 and was in Miami this week for his second treatment when doctors at the University of Miami announced the experiment.
Surgeons there inserted the catheter through the front of Sibley's skull just under his scalp, across the top of the brain and then 3 inches down into the frontal lobe. The catheter is virtually invisible underneath the scalp.
The drug GDNF, or Glial Derived Neurotrophic Factor, or a placebo is injected via the catheter directly into Sibley's brain once a month.
He is the first to take part in the experiment, which is expected to last at least 1 1/2 years. He eventually will be joined by 24 others nationwide with Lou Gehrig's disease.
The disease is also known as ALS, or amyotrophic lateral sclerosis. Symptoms include muscle weakness, spasms and eventual respiratory failure. Less than 20 percent of patients live five years or more with the disease.
Researchers hope GDNF will slow the disease as much as 50percent and double Sibley's life expectancy to six years, said Dr. Walter Bradley, who heads the university's drug trial program for the pharmaceutical companies Amgen and Medtronic.
The medicine has had good success in lab experiments on animals and in tissue cultures, Bradley said, but it may do nothing for ALS sufferers; even worse, it could cause a fatal hemorrhage.
GDNF is manufactured by Thousand Oaks, Calif.-based Amgen. Tests of another Amgen drug, BDNF, were canceled after it was deemed ineffective. BDNF was injected into patients under the skin.
With GDNF, the catheter - developed by Medtronic of Minneapolis - is being used to get the drug directly into the brain. Unlike other organs, the brain is surrounded by a ``blood-brain barrier'' that would prevent the medicine from entering were it injected or taken in pill form.
``There is around every blood vessel a barrier that blocks out foreign proteins,'' Bradley said. ``It allows us to survive, but that is a problem when you have a drug that you just can't get into the brain. This device allows one to circumvent the blood-brain barrier.''
GDNF is not a cure for Lou Gehrig's disease. Nor will it stop its progress or help rebuild what it has already destroyed.
``The hope is that we can make nerve cells generate, but that is still in the science-fiction realm,'' Bradley said.
Sibley was diagnosed with Lou Gehrig's disease just over a year ago while on a ski trip in Colorado with his family. He was having trouble speaking. It was a devastating diagnosis for the otherwise healthy man, who still works about four hours a day with the help of an assistant.
``Even at this early stage of treatment, I believe that I may be experiencing some improvement in my swallowing,'' Sibley said. ``It may be a dream, but I can see myself skiing with my kids again in a year or two.''
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