Pioneer in biotech offers view of future

October 17, 1995|By MARK GUIDERA | MARK GUIDERA,SUN STAFF

Imagine a day, says M. James Barrett, when a simple injection of genetic material could help people suffering from painful cancers effectively fight the diseases with few, if any, side effects.

No more nausea, diarrhea or other debilitating effects from chemotherapy or radiation. The scary picture of going into surgery eliminated.

"This is where the next generation of medicine is headed," says Mr. Barrett, the chairman and co-founder of Genetic Therapy, which yesterday was honored by the Greater Baltimore Committee for its huge revenue growth -- 9,643 percent -- during the past five years.

Founded in 1986, Genetic Therapy was the fastest growing company in Maryland between 1990 and 1995, according to the GBC, which yesterday honored the company and 39 others for their performance during its first Fast 40 Technology Awards event, held in Baltimore.

The company's promise for making gene therapy, which involves using pieces of DNA to treat disease, helped rocket the biotechnology company's revenues to $17.2 million in 1994, up from just $177,000 in 1990.

Genetic Therapy by no means had the highest revenues of any of the companies honored yesterday. Integrated Health Services, an Owings Mills company providing post-acute care services, had the highest revenues of the top 40 -- $682 million. Fourteen other firms also had larger revenues than Genetic Therapy.

But the prospect of the company's reaping revenues in hundreds of millions of dollars annually is so strong that it attracted deep-pocket investors.

Among them was Swiss pharmaceutical giant Sandoz Ltd., whose payments for research accounted for a "vast majority" of Genetic Therapy's revenue growth in the past five years, said Marc Schneenbaum, Genetic Therapy's chief financial officer. Sandoz bought the company in August, paying $295 million or $21 a share for the company, a tidy return for those that bought the stock when it went public at $9 a share.

The message to biotechnology companies, said Mr. Barrett: "Your most important asset is your intellectual property."

The Gaithersburg-based company is developing ways to treat at least 11 diseases with gene therapy. Among the potentially profitable areas the company is conducting research: leukemia, malignant melanoma, AIDS and Gaucher disease, which causes weak bones and enlarged organs.

Mr. Barrett, whom some have called the father of the biotechnology industry in Maryland, remains pragmatic.

"Gene Therapy could either be a huge success or a huge flop. There is still a lot of research to be done before we can say," said Mr. Barrett. "But the trials so far look very promising. Hopefully this will be the front-line therapy one day. That would be a great thing. It would be a vastly different experience for the patient than chemo or radiation."

His cautious outlook is apparently well-founded.

For one, there are years and millions more to be spent before Genetic Therapy and its new parent, Sandoz, before the company could see Food and Drug Administration approvals to commercialize a product.

And last year the New England Journal of Medicine reported that gene therapy failed to deliver benefits to all people afflicted with cystic fibrosis and muscular dystrophy in two separate studies.

But Mr. Schneebaum notes that the cystic fibrosis study at least did show that the genes given to participants did transfer to the body, a finding he argues shows that the therapy can work if there is more research.

"Gene therapy is alive and well," he said. "What we are doing holds the potential to change the face of medicine."

For now, Genetic Therapy remains like most of the other other 1,300 U.S. biotechnology companies: in the red.

Last year the company had a net loss of $5.5 million. Huge losses like that are the result of the high costs of research yet to be balanced with approved products for the marketplace, although some industry analysts say that day could be near for several biotechnology companies.

Mr. Barrett says the pressure is on for his company to get that coveted FDA approval to market one of its genetically engineered products.

Mr. Barrett is particularly hopeful about the company's strongest candidate thus far: a gene therapy for inoperable and operable brain cancer.

That therapy is scheduled to go into final trials early next year, he said. One of the trial sites will be at the Johns Hopkins Hospital.

Mr. Barrett also is upbeat about the company's newest field of inquiry: removing cells from the body, altering them genetically, and placing them back into the body so they correct a disease. Scientists working for the company are attempting to determine how such therapy could be used.

That therapy holds the potential to treat genetic disorders such as cystic fibrosis, a deadly clotting of the lungs, and hemophilia, a debilitating blood disorder.

"To me, it's this kind of research that is more exciting than what our revenues might happen to be," said Mr. Barrett.

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