The Albert and Mary Lasker Foundation today named seven scientists as winners of its annual awards, which honor basic medical research, clinical research and special achievement. Each category will receive $250,000.
The Lasker-DeBakey Clinical Medical Research Award went to two virologists — Ralf F. W. Bartenschlager of the University of Heidelberg in Germany and Charles M. Rice of Rockefeller University in New York — for creating a system to replicate the hepatitis C virus in the laboratory, and to Michael Sofia, an industry scientist, for using the system to develop the hepatitis C drug, now sold as Sovaldi.
Scientists first sequenced the genome of the hepatitis C virus in 1989. Researchers then believed they could insert that newly sequenced RNA into cultured cells and watch it replicate — but this approach was not so simple.
In the 1990s, Bartenschlager’s and Rice’s labs discovered a method to replicate fragments of the hepatitis C virus’s RNA in lab-grown cells. The researchers’ discoveries, which were published in journal articles in 1999 and 2000, happened after many failed attempts, since hepatitis C virus is well-known for being difficult to replicate in a lab setting.
Afterward, pharmaceutical researchers targeting hepatitis C used Bartenschlager’s and Rice’s replication method in the drug discovery process. In 2005, Sofia joined a small biotech company called Pharmasset, where he developed a chemical that blocked hepatitis C replication. His work led to the development of a new oral drug, sofosbuvir, marketed as Sovaldi. Sovaldi was approved by the FDA in 2013 to treat hepatitis C virus, which affects about 3.5 million Americans, and as many as 170 million people worldwide.
Sovaldi can cure patients, without severe side effects. Since Sovaldi was approved, other hepatitis C treatments have come on the market, including Gilead’s Harvoni, which has cure rates of 94 percent to 99 percent in eight to 12 weeks of therapy. Despite these successes, hepatitis drugs are high in price, with some between $54,000 and $94,000 for a full course of treatment.
A team of researchers from Harvard University, Johns Hopkins University, and the Francis Crick Institute were also honored for their discovery of the pathway by which human cells sense and adapt to changes in oxygen availability. Their work led to the discovery of a protein, HIF-1, that emerged when oxygen was scarce and then activated a large network of genes. Their discovery has had implications in treating heart disease and cancer.
Filed Under: Drug Discovery