WASHINGTON -- The Food and Drug Administration announced yesterday that it will make a controversial, experimental Alzheimer's disease drug widely accessible to patients while it is still under study.
Tacrine, or THA, will be the first drug made available to treat the progressive degenerative disease, which is the fourth-leading cause of death among adults in the United States.
The drug is not a cure for the ultimately fatal disease, which causes gradual mental deterioration, including loss of memory. But it has helped some patients improve their ability to think and function, according to its manufacturer, Warner-Lambert Co. of Morris Plains, N.J..
The drug also has been shown to cause liver damage.
Alzheimer's disease afflicts about 4 million Americans and kills 100,000 annually. It strikes more than 10 percent of the population over age 65 and nearly 50 percent of those 85 or older.
The FDA acted on the advice of a key agency advisory committee that recommended in July, with reservations, that THA be offered to Alzheimer's patients if carefully controlled clinical trials of the drug continued at the same time.
"Caution needs to be the watchword," said Dr. David A. Kessler, commissioner of the FDA. "Patients on the drug have demonstrated a small improvement on psychological tests. It still remains to be seen whether that translates into a meaningful clinical benefit."
There has been anecdotal evidence of dramatic gains in some patients, but Dr. Kessler said confirmation by studies is needed.
"There is a reason to believe the drug may work," Dr. Kessler said. "The definitive evidence is still not there. However . . . when there are no alternative therapies, it's not fair to ask patients to wait."
The new access will be permitted simultaneously with placebo-controlled clinical trials using much higher doses than have previously been studied, the FDA said.
Both the access program and the trials will use progressively higher doses of the drug and will exclude patients who begin to experience liver problems as the doses increase.
Eventually, Alzheimer's patients cannot care for themselves and require constant supervision. Over the course of the disease, which can often take years, it typically takes a heavy emotional and financial toll on the victim's families and friends.
The Alzheimer's Association, a national voluntary organization, estimates the annual cost of caring for the nation's Alzheimer's patients at $80 billion to $90 billion.
"I remember vividly a patient's daughter saying to me that the death of the mind is the worst death of all," Dr. Kessler said. "The cruel and relentless nature of Alzheimer's underscores the urgency of expediting important new therapies."