Thalidomide shows that it can heal, too From deformer of babies to force for good

April 02, 1995|By Jonathan Bor | Jonathan Bor,Sun Staff Writer

Nobody imagined that a drug blamed for 8,000 deformed babies -- limbless newborns with flippers and stumps at their shoulders and hips -- would ever become a force for good.

The medication left a trail of sorrow that stretched around the world, touching 46 countries before disgraced pharmaceutical companies yanked it from the market in 1962. Even as the drug receded into history, the name thalidomide remained a ghastly reminder of medicine gone bad.

But in an atmosphere of caution and hope, thalidomide has re-emerged.

Physicians at the Johns Hopkins Oncology Center have discovered its surprising power to cure an often fatal condition that strikes cancer patients in the aftermath of a bone marrow transplant. Today, having treated about 90 people, the doctors consider thalidomide their most effective weapon.

Elsewhere, scientists are studying its potential to arrest two leading eye diseases before they rob people of sight. A clinical trial began this year. And, because thalidomide seems to tame the body's immune system, scientists believe the drug might combat lupus and rheumatoid arthritis and reverse the wasting effects of cancer, tuberculosis and AIDS.

Thalidomide's rebirth shows how medical progress is sometimes driven by odd combinations of tragedy, happenstance and piercing insights of scientists working on seemingly unrelated problems. Still, no drug has rebounded quite like this.

"It's pretty remarkable, pretty exciting," said Dr. Gary Gordon, a pharmacologist who proposed thalidomide's use against graft-vs.-host disease while a junior resident at Johns Hopkins Hospital. "The drug has redeemed itself, really."

Although it may be years before researchers gauge thalidomide's true worth, they are braced for the difficult challenge awaiting doctors and regulators if the drug finds a large market that includes women of childbearing age. No matter how promising its comeback, it remains the most powerful engine of birth defects in the pharmaceutical world.

Thalidomide was peddled as the perfect sedative by a German company, Grunenthal, in the late 1950s and early 1960s. Unlike other sleeping pills, it was suicide proof. No amount could cause an overdose. And, it eased nausea, making it seem the ideal antidote to morning sickness.

After doctors gave it to pregnant women, the resulting birth defects so horrified some British hospital workers that they delayed presenting the infants until their unsuspecting mothers were ready to go home.

After doctors gave it to pregnant women, the resulting birth defects so horrified some British hospital workers that they delayed presenting the infants until their unsuspecting mothers were ready to go home.

The characteristic deformities -- children without arms, without legs or completely without limbs -- didn't immediately implicate thalidomide because they occur randomly, although rarely, in nature. And, until the tragedy mounted, the births were scattered across Western Europe, Australia and the Third World.

In the United States, a company had amassed the raw material to produce 15 million pills. But Dr. Frances Kelsey, a South Dakota physician who was reviewing one of her first cases for the Food and Drug Administration, had misgivings about the drug's safety even before the limb deformities came to light.

Putting the brakes on market approval, Dr. Kelsey said she was not satisfied that the company had explained a side effect -- numb or painful extremities -- that were being described in European journals.

"This raised the question: What would happen if a fetus were exposed to it for nine months or portions thereof?" recalled Dr. Kelsey, who, at 80, still works for the FDA. "Nerve problems produced in an adult might be serious for the child."

Although U.S. women who took thalidomide in experiments gave birth to nearly 20 deformed babies, Dr. Kelsey's caution spared the nation a public health disaster.

In Germany and Australia, two doctors who refused to accept the birth defects as random uncovered a unifying fact. All the mothers had taken thalidomide in the first trimester of their pregnancies, precisely at the stage when arms and legs emerge from fetal "limb buds."

Testing for drug toxicity was a relatively primitive science. Grunenthal insisted that the drug had proved safe for rats and mice before it was given to humans. But when independent scientists tested it on pregnant rabbits and monkeys, many of the offspring lacked arms and legs.

Leaving shattered families as its legacy, thalidomide disappeared. Or so it seemed.

Just as the public was becoming alarmed about thalidomide, an Israeli dermatologist made a curious observation.

Dr. Jacob Sheskin had been giving the drug to leprosy victims to ease pain and sleeplessness. They were suffering from a hellish syndrome that occurs after patients take antibiotics and their immune systems besiege dying bacteria.

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