Posted on 07/08/2004 11:27:15 PM PDT by neverdem
Amgen's anemia drug, the best-selling product developed so far by the biotechnology industry, might have broad new uses, recent studies have found.
Laboratory and animal studies have shown that in addition to bolstering the body's red blood cells, the drug, EPO, is present in the central nervous system and acts to protect cells and tissues from damage and death. That could make it useful as a treatment for strokes, spinal cord injuries, multiple sclerosis and many other ailments.
Testing in humans is in very early stages. A small study by academic scientists in Germany found that EPO, when given within eight hours of a stroke, helped protect the brain from damage and improve patient recovery. A larger trial is now under way there. Another early-stage trial in Germany is testing EPO as a treatment for schizophrenia, and in the United States, academic scientists are planning trials for AIDS-related dementia and for a nerve disease similar to multiple sclerosis.
EPO, short for erythropoietin, is sold by Amgen as Epogen and, in a newer form, as Aranesp. Johnson & Johnson, under license from Amgen, sells it under the names Procrit and Eprex. The versions had combined sales of $8 billion last year.
A paper being published in the journal Science today discusses a new development that could pave the way for broader use. Scientists associated with Warren Pharmaceuticals, a small, privately held biotechnology company in Ossining, N.Y., say they have developed a chemically modified version of EPO that does not increase red blood cell levels but, in animals, still protects tissues from damage.
Such a modified form could avoid a major risk of using EPO to treat people who are not anemic. The risk is that red blood cell levels might be increased to dangerously high levels, causing the blood to thicken and posing a risk that life-threatening clots would form.
Anthony Cerami, the chief executive of Warren and an author of the Science paper, said that with the company's modified EPO, "we can give tremendous amounts of it" to animals "and have no effect on their hemoglobin levels, on their red cell levels."
Most of the work involved in discovering the other roles that EPO plays in the body was done mainly by academic scientists, not by the companies, although the companies might be the ultimate beneficiaries.
"They missed the boat on this," Dr. Cerami said of Amgen.
But the companies do seem to be working on these new aspects of EPO quietly. Amgen has done experiments in the laboratory, said Kelly Stoddard, a spokeswoman. She had no comment on the paper in Science other than that it was "interesting."
Mark Wolfe, a spokesman for Johnson & Johnson, said the company did not discuss its research beyond what was on its Web site, which lists nothing about new uses of Procrit. Some employees, however, were co-authors of a paper about the stroke clinical trial.
Stuart A. Lipton, scientific director of the center for neuroscience and aging at the Burnham Institute in San Diego, said the modified form of EPO developed by Warren was potentially promising but would require a complete set of clinical trials because it would be considered a new drug by regulators.
He said it would be faster to use EPO itself, as it is already on the market and has been proved basically safe.
Dr. Lipton said he was working on ways to deliver EPO to the brain in a way that would minimize its effects on red blood cells.
In a paper published last month in the Proceedings of the National Academy of Sciences he showed that combining EPO with another drug, insulinlike growth factor-1, would allow less EPO to be used.
Dr. Lipton has formed a company, NeuroMolecular Inc., and said he was trying to start a clinical trial using EPO to treat the dementia that sometimes accompanies H.I.V. infection. People infected with that virus tend to be anemic, so any increase in their red cell counts would not be a problem, he said.
Sanjay Keswani, assistant professor of neurology at Johns Hopkins, said he hoped to begin a trial this year using EPO to treat transverse myelitis, an inflammatory disease of the spinal cord that resembles multiple sclerosis. He said that he also wanted to try the drug to treat nerve damage in the limbs.
"We've been very impressed with the neuro-protective properties of erythropoietin," Dr. Keswani said.
A question remains about whether other companies will be able to sell EPO or modified forms of EPO without infringing Amgen's patents. Ms. Stoddard of Amgen said it was too early to comment.
Dr. Cerami said his company's drug did not increase red cells, so it was not really EPO and should not infringe the patents.
Warren is seeking its own patent on what it calls carbamylated EPO, or CEPO, after the chemical process used to modify the protein.
H. Lundbeck, the Danish drug company, has licensed the rights to use CEPO and other modified EPO's developed by Warren for treating central nervous system disorders. Dr. Cerami said Warren was talking to other companies about the rights for other diseases. One of those is Amgen, he said.
Warren had some help in getting in the door of Amgen. One of its director is George B. Rathmann, the original chief executive at Amgen, who said yesterday that he saw no conflict between the companies.
"Erythropoietin is going to serve another important medical need," Dr. Rathmann said. "It's really a remarkable molecule."
PING
BTTT
This week has had a LOT of great news!
Carolyn
Disclaimer: Opinions posted on Free Republic are those of the individual posters and do not necessarily represent the opinion of Free Republic or its management. All materials posted herein are protected by copyright law and the exemption for fair use of copyrighted works.