WEDNESDAY, Oct. 17, 2001 (HealthDayNews) -- Thalidomide, a drug with a notorious past that has been rehabilitated in recent years as a booster for anti-cancer medications, may work all by itself to fight a deadly skin cancer.
Doctors at St. Louis University say they've used thalidomide by itself to eliminate a highly malignant form of skin cancer. Their research, described in a letter in the upcoming issue of The New England Journal of Medicine, is unusual because until now doctors thought thalidomide worked best with other chemotherapy. Thalidomide, a former anti-nausea drug, was banned decades ago because it causes birth defects.
The St. Louis doctors treated a 63-year-old man with malignant and recurrent melanoma on his scalp. After surgery to remove the first tumor, two other sores appeared nearby.
When an immune-stimulating vaccine didn't shrink the new tumors, the man began taking 200 milligrams a day of thalidomide. Six weeks later the lesions were much smaller, and within six months they had vanished, the researchers say.
"We used thalidomide as almost a last-ditch effort to see if it might benefit him in any way, and surprisingly it did," says Dr. Ganesh Kudva, a fellow in hematology and oncology and lead author of the research letter.
Dr. Wen-Jen Hwu, a melanoma expert at Memorial Sloan-Kettering Cancer Center, in New York City, and a pioneer in using thalidomide to treat tumors, had a mixed reaction to the report.
"From this case, it looked like the thalidomide also had direct anti-tumor activity, and other studies didn't look at thalidomide alone," says Hwu (pronounced "hoo"). On the other hand, she says the location of the patient's melanoma on his head and the fact that it hadn't spread to other organs suggests that his disease was not especially aggressive. More virulent tumors might have been less responsive to the drug, she says.
In 1999, Hwu became the first researcher to show that a combination of thalidomide and another cancer drug, temozolomide, could treat melanoma that had spread to the brain. She tested that regimen on a woman with the disease, who is still alive and free of cancer.
Another watershed trial found that thalidomide doubled the five-year survival rate in patients with recurring myeloma, an incurable blood cancer, from 29 percent to 58 percent. The drug was hailed as a great advance for patients with the deadly disease, which strikes nearly 14,000 Americans and kills 11,000 each year. Thalidomide, which starves tumors of blood and other nutrients, also works well against colon cancer.
Although thalidomide hasn't been approved by the Food and Drug Administration for use in cancer patients, clinical trials with the drug are underway nationwide. In addition to being the most effective new treatment for myeloma, the drug also is popular among patients because it can be taken as a pill, not an injection like other potent chemotherapy.
Malignant melanoma, the most deadly form of skin cancer, strikes an estimated 51,000 Americans a year, killing some 7,800. Unlike other skin tumors, which tend to expand across skin, melanoma has a nasty propensity to burrow deep into the tissue until it reaches lymph glands and blood, giving itself entree to the entire body. Once melanoma penetrates into the brain, patients typically live only a few months.
Thalidomide, while far from the standard of care, is increasingly impressing cancer specialists with its ability to stop the spread of melanoma. These tumors have an insatiable need for blood to fuel their growth, and even build channels out of their own cells to draw nutrients into themselves.
Thalidomide cracks down on this vessel building, a process called angiogenesis, depriving tumor cells of the blood they need to survive.
Nearly a year after treatment with thalidomide, Kudva's patient still has no skin tumors and after a brief bout of severe constipation that forced him to halve his dose of the medication, he's back up to 200 mg of thalidomide a day. The patient will take the drug "indefinitely," since Kudva says he and his colleagues aren't sure what might happen if they stop the treatment.
Thalidomide is costly, however, running about $500 a month, and it has side effects. Kudva says more limited courses of the drug would be preferable, but only additional research will tell if that's possible.
Meanwhile, Kudva, like Hwu, says stand-alone thalidomide therapy has relevance only to patients with melanoma that hasn't yet spread beyond the skin. "In skin-only it may have some promise by itself. That's as best as I can say."
What To Do
For more on melanoma, visit the Melanoma Patients' Information Page, or the American Cancer Society.
Read what the U.S. Food and Drug Administration has to say about thalidomide.
Thalidomide's dark past and brighter future potential are discussed at the Mayo Clinic.
