In a breakthrough that could have dramatic implications for treating immune-system disorders and other diseases, scientists at a Maryland biotechnolgy company have discovered a natural trigger for producing one of the body's most important warriors against infection and disease.
Scientists at Human Genome Sciences Inc. in Rockville said yesterday they are proceeding with development of an experimental drug based on the breakthrough and hope to see it tested in humans this year.
Believing it might have a financial blockbuster on its hands, the company plans to make the drug's development a priority.
As word of publication of the discovery leaked out late yesterday, shares in Human Genome rocketed, closing at $55.50, up $14.625, or 35.8 percent.
Human Genome scientists claim to have discovered the human protein responsible for triggering production of B cells, a type of white blood cell. These immune-system cells generate the important antibodies that attack bacteria, viruses, fungi and other invaders.
Company executives said the newly identified protein, B lymphocyte stimulant, or BLyS (pronounced "bliss"), might have a wide range of uses, including boosting immune systems weakened by chemotherapy or organ-transplant drugs, and treating deadly immune diseases such as AIDS, leukemia and lymphomas.
The scientific team's findings are published today in Science, a publication of the National Association for the Advancement of Science.
One of the country's leading immune-system experts, Dr. Anthony S. Fauci, director of the National Institute of Allergy and Infectious Disease, hailed the discovery as a stunning example of the growing use of genomic information to find the keys to how the body works and how to manipulate those functions to fight diseases.
"I look on this as being important because it demonstrates how incredibly powerful some of these biotech companies are becoming in doing things that would have seemed impossible a few years ago," Fauci said.
He added: "Bill Haseltine [co-founder and chairman of Human Genome] has been saying for years that this could be done, and he's suffered many skeptics. It seems now he has the last laugh."
At least a dozen biotechnology companies -- three based in Maryland -- are attempting to decipher the growing library of information about the body's 80,000 to 100,000 genes to identify those that help trigger and prevent diseases and disorders.
Scientists around the globe have searched in vain for more than a decade for a way to stimulate the growth of B cells without triggering the growth of other cells or causing unwanted side effects.
Human Genome Science's team determined which protein stimulates B cell production within a year of embarking on the task, said Kate De Santis, a spokeswoman for the company.
The company accomplished its task so quickly by using superfast machines and computers to systematically sift through thousands of full and partial genetic codes -- a sequence of chemicals that establish each gene's duties -- which the company has compiled in a vast genomic database. Scientists then sorted out those that seemed to be active in generating proteins that stimulate immune-system functions.
The scientists next developed laboratory tests to pinpoint which proteins were responsible for production of immune-system cells and the B cells specifically.
The company has not decided on which disease or disorder it will seek Food and Drug Administration clearance to test an experimental B cell stimulant. But it hopes to decide shortly so it can begin human studies late this year or early next year, said Dr. Craig Rosen, Human Genome's senior vice president for research and development.
"This presents a lot of openings for us. There are no treatments available now for stimulating B cell production, so we see this discovery as potentially meeting several unmet medical needs," Rosen said.
"There are immune-system cells you might be able to live without, but without B cells you are essentially the boy in the bubble," he added.
The company hopes to develop treatments that not only boost production of B cells, but also others that would retard production in cases where B cells are proliferating rampantly, as with some lymphatic cancers.
Among the other options the company is assessing, said Rosen, are BLyS as an additive to vaccines against malaria and other diseases that are enormously difficult to develop vaccines for, and as an immunity booster for people fighting bacteria resistant to antibiotics, an emerging threat.
It normally takes five to eight years of human testing before the FDA will consider approving an experimental drug.
Developing drugs based on stimulants -- also known as "growth factors" -- is not a new concept. About a dozen growth factor- and growth hormone-based drugs are commercially available, according to Pharmaceutical Research and Manufacturers of America, a trade group.