Thalidomide Makes A Comeback

Diagnosed with brain cancer, Elizabeth Narcessian did not hesitate when her doctor prescribed thalidomide, the drug banned a generation ago after it caused thousands of deformities in newborn babies.

"When you have a life-threatening illness and you are with a doctor you trust, you do anything," said Narcessian, a 40-year-old physician from Ridgewood, N.J., who credits the drug with helping to shrink her tumor.

Thalidomide, approved by regulators in July to treat a complication from leprosy, is making a comeback that extends well beyond that rare disease.

Narcessian's doctor is one of a growing number prescribing it for some of the deadliest cancers even though tests have yet to definitively prove it works.

Thalidomide, originally intended for morning sickness and as a sedative for pregnant women, was banned worldwide in the early 1960s after 12,000 babies were born with no limbs or flipperlike arms and legs, serious facial deformities, and defective organs.

The pill had been sold in 48 countries, though not in the United States. A scientist with the Food and Drug Administration who was not satisfied with the safety assurances from the manufacturer blocked the approval.

In the early 1990s, scientists discovered thalidomide could be a potent treatment. In addition to cancer, testing is now under way in patients with AIDS, lupus, and other diseases.

Wall Street seems to have already decided what it thinks about the drug's comeback. Shares in Celgene Corp., the only U.S. seller of thalidomide, have more than doubled in the past month to about $16 after the company bought marketing rights to the drug from its closest rival.

Analysts predict that thalidomide will post $45 million in sales this year, $70 million in 2000, and possibly $300 million within five years.

About 200 thalidomide prescriptions, which cost $7.50 to $30 per day depending on the dosage, are being written a week, according to IMS Health, a market research firm. Nearly two-thirds were written for cancer patients.

Thalidomide, which Celgene sells under the brand name Thalomid, works by cutting off the blood supply to tumors. When combined with chemotherapy or radiation therapy, the cancer cells, in theory, die of starvation.

Celgene allows prescriptions to be dispensed only by doctors and pharmacists that the company trains about thalidomide's dangers. So far, more than 2,000 physicians and 2,000 pharmacists have signed up.

The company has strict rules to make sure pregnant women don't take it. All women must undergo repeated pregnancy tests, and men and women must sign statements acknowledging they were instructed to use effective birth control.

Not everyone is convinced about its effectiveness for cancer treatment.

"Thalidomide does not yet appear to be a shining star," said Dr. Ajrmon Eyre, executive vice president for research at the American Cancer Society.

Eyre said the drug hasn't always workd and he prefers to see final test results before reaching a conclusion on its effectiveness.

Even doctors who prescribe it do so only after other less dangerous drugs fail.

Narcessian, a doctor who specializes in rehabilitation medicine, said taking the drug makes her feel as if she is actively fighting the cancer even after completing six weeks of radiation therapy. She said she has experienced no side effects.

Will patients take a drug with such a notorious past?

"You are talking about people on death's door," said Dr. Michael Gruber, director of neuro-oncology at New York University Medical Center.

He said about two-thirds of the approximately 100 brain cancer patients he has treated with thalidomide have responded positively; one-third have seen their cancer disappear.

Narcessian said she hadn't planned to have any more children, and knows she made the right decision.

"I have an 8 year-old-daughter," she said. "And last week she said, 'Mommy, you don't look sick anymore'."

Reported By Phil Galewitz