New treatment shuts off oncogene - using anti-sense molecules to treat leukemia - Brief Article

Science News, June 6, 1992

A gene known as p53 plays a role in the development of many cancers, including a severe type of leukemia -- cancer of blood and bone marrow cells. This gene is turned off in most immature bone marrow cells. However, scientists believe that cancer arises when something turns p53 on at an inappropriate time, causing blood and bone marrow cells to proliferate.

On the basis of this hypothesis, Jorge Spinolo of the University of Nebraska Medical Center in Omaha and his colleagues devised a treatment strategy, using so-called anti-sense molecules (SN: 6/10/89, p.360), for acute myelogenous leukemia (AML). The technique blocks the activity of the p53 gene. In laboratory studies, the new treatment has slowed the growth of malignant cells obtained from people suffering from AML. That in vitro finding raises the hope that a similar strategy might help combat leukemic cells in the human body, not just in a petri dish.

Before Spinolo's group could test the treatment's efficacy, the researchers had to demonstrate its safety. They gave the new drug to seven monkeys. Several weeks later, the team sacrificed four of the monkeys and found no evidence of drug-related toxicity.

The remaining three monkeys are alive and well, Spinolo reported May 22 at the American Association for Cancer Research meeting in San Diego.

Spinolo wants to treat 10 to 15 AML patients with the drug and plans to take such a research proposal to the U.S. Food and Drug Administration this month. If the team gains FDA approval, that preliminary human trial would begin sometime this summer, Spinolo says.