Monday, June 18, 2007

Major Breakthrough Speeds Therapies to the Brain

BOSTON, June 17, 2007-New technology promises to open new avenues for treatment of viral infections, Alzheimer’s and Parkinson’s disease, and many other neurological illnesses

Researchers at the Immune Disease Institute—formerly the CBR Institute for Biomedical Research—have overcome a major hurdle in the delivery of therapeutics to the brain: getting past the blood-brain-barrier (BBB) which excludes most large and small molecule drugs. Their breakthrough research is published in the June 17, 2007 issue of Nature magazine, with Manjunath Swamy, M.D., as principal investigator and Priti Kumar, Ph.D., as first author on the paper, with collaboration from scientists at the University of Iowa and Hanyang University in South Korea.

The BBB—comprised of the endothelial walls of 100 billion capillaries in the brain—is a superfine filter that prevents transport of harmful pathogens and beneficial drugs alike. To overcome the BBB in situations of disease, IDI researchers in the Swamy lab employed a modified Rabies virus glycoprotein peptide name CORVUS that slipped past the BBB and, in mice, delivered small interfering RNAs as a therapy to neuronal cells in the brain. The siRNAs effected specific gene silencing in the brain, without side effects.

A visionary discovery, this new technology from the Swamy lab provides a non-invasive, intravenous means of delivering, throughout the brain, the powerful therapy known as RNA interference (which suppresses disease-causing genes) as well as, potentially, DNA for gene therapy. The CORVUS technology also promises to be a brain delivery system for a wide slate of conventional drugs in the form of antibodies, proteins, and other compounds.

Many late-stage clinical trials have failed when candidate drugs are frustrated at the BBB; this new ability to send therapies selectively to the brain may revolutionize the treatment of diseases including Alzheimer’s, Parkinson’s, multiple sclerosis, psychiatric illnesses such as schizophrenia, fatal infections including encephalitis and meningitis, and central nervous system traumas, among other illnesses.

Previously, Swamy, Priti Kumar, and colleagues had used RNAi to defeat brain infection caused by Japanese encephalitis and West Nile virus. With the breakthrough CORVUS technology, they will be able to prevent such deadly infections in mice by administering the same siRNAs intravenously.

In addition, there is a great need for new therapeutics for Alzheimer’s and other “neuronal” diseases associated with aging, as the U.S. population becomes older. Effective delivery of therapies to the brain is a vital component for making progress against these diseases.


Founded in 1953, the Immune Disease Institute—formerly known as The CBR Institute for Biomedical Research—is an independent, nonprofit biomedical research institute in Boston, Mass., affiliated with Harvard Medical School. Its world-class investigators conduct breakthrough research on the immune system and inflammation—work leading to new therapies for millions of patients suffering from illnesses such as cancer, heart disease, HIV/AIDS, lupus, Alzheimer's disease, and immune deficiencies. Visit to learn more.

The CORVUS technology (CBRI ID 06-001) is a patent pending, novel drug delivery and neuronal cell transfection method utilizing a small peptide to selectively deliver drug payloads across the BBB and spread the drug evenly throughout the brain. IDI is currently evaluating collaborative research and licensing opportunities with industry. For more information and licensing terms, please contact Ryan Dietz, at 617-278-3463 or

Manjunath Swamy, M.D., 617-278-3240 or
Priti Kumar, Ph.D., 617-278-6623 or
Hal LaCroix, Public Affairs, 617-278-3321 or
Ryan Dietz, Office of Tech. Development, 617-278-3463 or