Health : Firm Seeks to Test Anti-Leukemia Drug
San Diego-based Genta filed for permission Tuesday to begin testing the first therapeutic drug that incorporates molecules that some scientists say could hold the key to treating heretofore incurable diseases.
Genta filed for an investigational new drug permit with the Food and Drug Administration to begin testing an “antisense oligonucleotide” compound to treat chronic myelogenous leukemia, a cancer that strikes 4,000 Americans a year. Only about 15% of those stricken are successfully treated.
If approval is granted, testing would begin in three or four months on 10 leukemia patients at University of Texas M. D. Anderson Cancer Center in Houston. A sample of the patients’ bone marrow would be extracted, treated with the Genta drug to rid it of diseased cells and then reintroduced in the patients’ bodies.
Albert Deisseroth, chairman of protocol at the clinic, said laboratory tests have indicated that Genta’s product “has the capacity to silence the abnormal signal within the leukemia cell that drives it to an abnormal pattern of growth” without harming the normal cells in the bone marrow.
Antisense oligonucleotides are molecules that can prevent the production of disease-causing proteins by binding specifically to messenger RNA molecules that are used to produce those proteins. At least a dozen drug companies are pursuing the technology.
Brandon Fradd, a biotechnology analyst with Montgomery Securities of San Francisco, said the theoretical appeal of antisense oligonucleotides is that they are very disease-specific and cause a minimum of harmful side effects to normal cells.
