NEW YORK -- Thalidomide, the morning sickness drug that produced flipper limbs in thousands of babies in the 1950s and 1960s, is making a comeback as a tumor fighter, helping to more than quadruple the share price of its manufacturer Celgene Corp. since the year began.
Warren, N.J.-based Celgene is testing thalidomide in more than 60 clinical trials to treat a wide range of cancers as well as other diseases, such as rheumatoid arthritis and Crohn's disease, a chronic inflammation of the bowels.
The drug was banned in the early 1960s after causing severe birth defects in more than 10,000 babies, mostly in Europe and Canada. But the drug continued to intrigue researchers because of its suspected special properties, including the ability to tame inflammation and block formation of blood vessels that feed tumors.
Thalidomide is a drug "with significant activity in a wide range of diseases," John Jackson, Celgene's chief executive, said in an interview with Reuters.
Since early January, shares of Celgene have rocketed to a 52-week high of 67-11/16 from 15-3/8 on the Nasdaq in anticipation of continuing positive clinical data in the months ahead. Early Monday afternoon, Celgene was up 1/4 at 57-3/8.
Celgene stock got a boost in mid-November after release of a study in the New England Journal of Medicine, which reported that thalidomide reduced, or in some cases, eliminated, tumor activity in patients with advanced multiple myeloma, an incurable blood cancer.
Thalidomide is part of a class of promising cancer fighters called angiogenesis inhibitors, which disrupt growth of blood vessels by attacking the cells that form their walls. Without such vessels, tumors are starved of nutrients and cannot grow.
In 1998, Celgene was granted approval to market thalidomide, sold under the brand name Thalomid, for treating the inflammatory complications of leprosy.
The return of the one-time pariah has allowed doctors to legally prescribe thalidomide for cancer on an "off-label" basis -- for a condition other than the one for which it was approved by US regulators.
However, Celgene and the US Food and Drug Administration have developed severe restrictions for using Thalomid to ensure that it is not taken by women who are -- or might become -- pregnant.
Of 43 angiogenesis inhibitors in clinical trials, analysts said Thalomid is the only one available on the market, although complete safety and efficacy data in the related cancer trials have not yet come in.
As the first marketed angiogenesis inhibitor, Thalomid has at least a three-year head start on potential competitors in this emerging field,'' said Peter Ginsberg, analyst at US Bancorp Piper Jaffray.
Potential rival products are being tested by Warner-Lambert Co. and Pharmacia & Upjohn.
About 76 percent of the 25,000 prescriptions written for Thalomid since its October 1998 launch have been for cancer, according to Jackson.
The three cancer targets farthest along in Celgene's 60 clinical trials are multiple myeloma, prostate cancer, and an aggressive form of brain cancer called glioblastoma.
Jackson said that according to Phase II trial results for Thalomid, 30 percent of patients with multiple myeloma have seen a reduction in tumor size of more than 50 percent. Encouraging results also have been seen in the prostate cancer and glioblastoma trials.
Ginsberg said he anticipates Thalomid's sales for cancer to expand sharply over the next six to 12 months, spurred by new data.
Just one indicator of the medical world's renewed interest in Thalomid is the American Society of Hematology meeting scheduled for Friday through next Wednesday, Dec. 8, for which more than a dozen abstracts of research on the drug have been accepted.
Michael Wood, an analyst at Warburg Dillon Read, said Thalomid has had US sales of about $16 million for the year to date. He predicted sales would rise to $51 million in 2000.
Thalomid also has been shown to have a second mechanism of action -- an ability to block the overproduction of tumor necrosis factor (TNF), a naturally occurring inflammatory protein implicated in a number of diseases, including Crohn's, rheumatoid arthritis, and diabetes.
In preliminary Phase II results for Crohn's disease, more than 60 percent of patients taking Thalomid who did not respond to standard therapy showed improvement, according to Jackson.
Celgene's near-term product-development pipeline also has received a boost from Attenade, an altered form of Novartis AG's drug Ritalin for treating attention deficit disorder. Jackson said Celgene will seek marketing approval for the drug during the first half of 2000.
In addition, Celgene is studying the next generation of anti-inflammatory and anti-angiogenesis drugs, called SelCIDs and IMiDs, that it says may ultimately supersede Thalomid. The company said they have shown higher potency and fewer side effects than thalidomide in early human trials. Celgene said the earliest it expects to file for marketing approval for those compounds is in 2002.
Copyright* 1999 Reuters Limited.*
