Banking on genetic keys

Genomics: A Rockville corporation is among the companies preparing to take advantage of a wonder of science: a map of human genes.

August 01, 1999|By Mark Guidera | Mark Guidera,SUN STAFF

For two men who get paid to tinker daily with DNA, the building block of life itself, David Hilbert and Paul Moore seem about as ordinary as they come.

Hilbert, 41, a cell biologist, Ivy League graduate and snowboarding fan, likes to spend spare time at home with his wife and two young children.

Moore, 33, a molecular biologist from Scotland and recently married, is an avid golfer.

But their work is spent at the frontiers of medicine -- and they know it.

"This isn't all just theory, we're looking at practical applications like new therapeutics that might make a real difference in people's lives," said Hilbert.

Said Moore, "We're pushing the edges of research and medicine."

Both men work for Human Genome Sciences Inc. in Rockville, one of the early entries into the genomics industry. Genomics is a burgeoning field focused, in part, on understanding the specific role of human genes, as well as the proteins they trigger.

Now, after years of anticipation -- and frustration for investors -- the toil is beginning to pay off.

Two weeks ago, Hilbert and Moore emerged from the obscurity of the laboratory when Human Genome announced that a team led by the two men had discovered the protein responsible for triggering production of B cells, key immune system cells that help fight infections.

The elusive protein -- long sought by scientists -- could have a wide range of applications, from treating some cancers to immune system disorders, say company executives.

Human Genome and similar companies -- about a dozen public and as many private -- hope to make millions, if not billions of dollars in the next century as they uncover the genetic keys to controlling and diagnosing diseases, and to offer a way to efficiently develop new drugs by trimming the high costs of the inevitable failures.

Human Genome expects to move a protein-based drug based on the discovery into human trials as early as next year.

The drug would be the third experimental treatment the company has produced from its in-house genetics research. It has a fourth in development in a joint venture with Vascular Genetics Inc.

Chairman and Chief Executive Officer William A. Haseltine says the company has a significant bank of gene data and should be able to churn out new drug candidates for years.

It took company scientists about six months to isolate the B cell protein, B lymphocyte stimulant, or BLyS (pronounced "bliss").

The speed with which the protein was found and its potential application for a line of new drugs is considered breathtaking by medical experts like Dr. Anthony Fauci of the National Institutes of Health. To Fauci, the BLyS discovery is a testament to the emerging power of genomics research.

"I can't think of any major drug company that does not have some kind of research deal or alliance with a genomics company," said Elizabeth A. Silverman, an independent New York-based genomics and biotechnology industry analyst formerly with BancBoston Robertson Stephens.

"The pharmaceutical companies are aware of just how enormously important genomics will be to medicine. It will be the foundation for most drug development in the future," said Silverman.

Johnson & Johnson has research deals with Incyte Pharmaceuticals of Palo Alto, Calif., which provides information on active genes and their potential medical usefulness, and with Gene Logic Inc. of Rockville, which uses a proprietary technology to determine which genes are being turned on and off during different stages of diseases.

"The advantage to genomics is sort of the same as being in a helicopter over the freeway," said Silverman. "You get a view of an entire system, rather than frittering away looking at one exit at a time. The amount of genetic information that will be available soon will be vast."

A turning point on the horizon, say genomics experts, is the mapping of the entire human genome -- a blueprint of the estimated 100,000 to 250,000 human genes.

There are three entities with major efforts to be the first to do this within the next two to three years: the federal government's $3 billion Human Genome Project; Rockville-based Celera Genomics, a unit of the big gene sequencing equipment company PE Corp. and a consortium of European drug companies headed by powerhouse Novartis AG.

PE Corp is spending more than $300 million to equip Celera with super-fast gene analyzers and to hire 200 top technicians and scientists. The European consortium plans to spend $45 million on a two-year effort.

With the human genome map on the horizon, and much of it to be placed in the public domain, companies like Human Genome and Cambridge, Mass.-based Millenium Pharmaceuticals Inc. are racing to patent gene findings so that they can lock up rights -- and a share of profits -- to potential new treatments and diagnostics.

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