Basic research conducted at the University of California, San Francisco (UCSF) into botulism has translated into a novel antitoxin to protect against bioterrorism and the first clinical trials to assess the resulting vaccine’s safety may launch soon.
The human therapy targets the deadly Clostridium botulinum neurotoxin and could treat poisoning and protect against it for six months to a year, according to James Marks, PhD, a UCSF professor and chief of anesthesia and professor of pharmaceutical chemistry at the UCSF-affiliated San Francisco General Hospital.The first approach to treating the toxin in nearly a century, the disease afflicts small numbers of people—fewer than 200 per year in the United States.
“Botulism is considered an orphan disease and is frequently found in places where home canning is common, such as Eastern Europe,” Marks said. “But it is also one of six Category A bioterrorism agents, along with smallpox and anthrax. The current treatment uses antitoxin made in horses, which cannot be given to prevent disease.”The antitoxin emerged from nearly two decades of research in Marks’ laboratory at UCSF, during which he and colleagues at the U.S. Army Institute of Infectious Diseases identified three lead antibody candidates that, when combined, were highly effective in neutralizing the toxin, even at very low doses.
“We’re working with one of the most dangerous substances known to man,” Marks said. “The only way to neutralize it is to have a very potent antibody. And in order to have a realistic dose, we needed to combine three antibodies into one antitoxin.”
Release Date: May 5, 2011
Source: The University of California, San Francisco (UCSF)
Filed Under: Drug Discovery