NIH awards LIAI major grant to test safety of new smallpox treatment

Treatment could be the nation's first line of defense against terrorist smallpox outbreak

SAN DIEGO – (May 8, 2008) The La Jolla Institute for Allergy & Immunology (LIAI) has received a $7.1 million grant from the National Institutes of Health (NIH) to fund safety and effectiveness testing of an antibody treatment that quickly fights the smallpox virus. The treatment could be the nation’s first line of defense in protecting against a terrorist-originated smallpox outbreak and may eventually be stockpiled nationwide alongside the smallpox vaccine.

“This work is particularly important because those younger than 36 years old in the U.S. population have not been vaccinated against smallpox, which makes the need for a strong and fast-acting treatment all the more vital should we ever face a smallpox outbreak,” said Mitchell Kronenberg, Ph.D., LIAI president & scientific director.

LIAI scientist Shane Crotty, Ph.D., who developed the antibody treatment, said the NIH grant will fund pre-clinical testing of the work he and his research team started three years ago. In 2005, the researchers, studying blood samples from people who had received the smallpox vaccine, were able to isolate the anti-H3 antibody as an extremely potent fighter against smallpox. The scientists then proved its effectiveness by testing in mice.

The NIH was intrigued by the work and asked Dr. Crotty to further develop the antibody treatment for use in humans. “Now we’ve made fully human antibodies to fight the smallpox virus,” said Dr. Crotty. The collaborative research team led by Dr. Crotty developed the human antibodies using technology which allows human antibodies to be developed from special genetically engineered mice.

“You have to have fully human antibodies to use this treatment as a human therapeutic,” explained Dr. Crotty. If further testing shows the treatment to be safe and effective in humans, Dr. Crotty said the NIH has indicated interest in stockpiling the antibody nationwide. “Hopefully we will never need to use it, but if we do, the treatment will be there.”

Dr. Crotty said further testing of the human antibody for safety and efficacy will be conducted using highly sophisticated animal models. “We can’t actually do these experiments in humans because it would be extremely dangerous and absurd to purposefully expose people to the smallpox virus,” he said, noting that no live smallpox virus will be used in any of the studies. “We will use experimental animal systems that can show that this antibody would work in humans without actually testing it on infected humans.” Dr. Crotty said the anti-H3 human antibody has already proven to be effective in animal testing protocols that the FDA used to license the only currently existing smallpox treatment.