Researcher Makes Gains In Finding Treatments

Armen Hareyan's picture
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Leading RSV investigator Dr. John DeVincenzo has taken another step toward finding effective treatment for the disease that causes bronchiolitis and pneumonia according to the Centers for Disease Control. RSV, or respiratory syncytial virus -- is the leading cause of hospitalization of infants in the US and one of the leading causes of viral death in this age group.

DeVincenzo is using the process of RNAi to target RSV. Clinical trials of ALN-RSV01, a treatment that applies new RNA processing discoveries, have demonstrated that the drug was safe and had statistically significant anti-viral activity in experimentally infected adults. The treatment showed a reduced infection rate from approximately 70 percent to 40 percent and doubled the number of uninfected subjects.

"ALN-RSV01 offers a potential new therapeutic approach for the treatment of RSV infection, a serious respiratory viral disease that leads to the hospitalization of nearly 300,000 pediatric and adult patients annually in the U.S. alone," said DeVincenzo.

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DeVincenzo, an investigator at the Children's Foundation Research Center located at Le Bonheur Children's Medical Center and the University of Tennessee, is attempting to develop the first human therapeutic that uses the 2006 Nobel Prize winning discovery of RNA interference, or RNAi. "Harnessing RNA interference could be used to conceptually treat many diseases," DeVincenzo said. "RNA interference can theoretically block the production of any disease-causing protein."

Thus, its potential application includes blocking oncogenic proteins that cause cancer and applications to other diseases as wide-ranging as genetic diseases such as cystic fibrosis and other common diseases such as cardiovascular disease.

DeVincenzo's RSV study is the first definitive proof that RNA-interference-based therapeutics can work in humans. He is working with Alnylam Pharmaceuticals to develop the treatment and presented the complete results of the study at the International Symposium on Respiratory Viral Infections meeting in Singapore in early March.

The clinical trial, named GEMINI, was a double-blind, placebo-controlled, randomized study of ALN-RSV01 in 88 healthy adult subjects experimentally infected with a wild-type clinical strain of RSV developed and manufactured from one of DeVincenzo's study patients at Le Bonheur. DeVincenzo's GEMINI study's results and their potential impact on human health were reviewed in the Wall Street Journal in early March.

The next stage in developing the treatment will be to conduct Phase II trials in naturally infected adult patients. Subsequent studies involving children are being planned.

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