Weill Cornell’s Dr. Melnick also discusses why turning science into medicine is happening much more rapidly.
In a significant breakthrough published recently in Nature, researchers at Weill Cornell Medical College and the University of California, San Francisco, have been able to overcome resistance of a form of leukemia to targeted therapy, demonstrating complete eradication of the cancer in cell and animal studies.
The study shows that an investigational drug, RI-BPI, developed at Weill Cornell, in combination with the drug Gleevec shut down stem cells responsi ble for about one-third of acute lymphoblastic leukemia (ALL), a cancer of white blood cells that affects young children as well as older adults. This form of ALL has the so-called Philadelphia chromosome, which is also found in chronic myelogenous leukemia (CML). But while Gleevec has greatly improved survival in CML, it has had a less dramatic effect in ALL, and most patients still die within a relatively short time frame.
That prognosis may change given these results, says co-senior investigator Dr. Ari Melnick, associate professor of medicine and director of the Raymond and Beverly Sackler Center for Biomedical and Physical Sciences at Weill Cornell Medical College, and a hematologist-oncologist at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. Dr. Melnick and his colleagues developed RI-BPI, and they have shown its potent effects in non-Hodgkin’s lymphoma (NHL) with no toxicity to normal cells. The drug targets the transcription factor BCL6, a master regulator of hundreds of genes that provides strong growth signals to NHL cells.
“I am surprised, and extremely glad, to see that RI-BPI has such strong activity in a leukemia. This opens up the possibility that the agent will have similar beneficial effects in other tumor types,” says Dr. Melnick.
Background on the development of RI-BPI at Weill Cornell Medical Center: Diffuse Large B ell lymphoma (DLBCL) is a common and aggressive subtype of lymphoma that is frequently associated with deregulation of the oncogene BCL6 (oncogene is a genetic material that carries the ability to induce cancer). Deregulated BCL6 activity keeps B cells in a rapidly proliferating (reproducing) state. The high levels of BCL6 expression in DLBCL coupled with the low or nil expression of BCL6 in normal cells have made BCL6 an attractive candidate for anti-cancer drug development.
Personalized lymphoma medicine offers the hope that by identifying lymphoma-causing mutations in critical regulatory genes, we can target these mutant proteins to cure lymphoma while limiting the side effects. Continue reading “Weill Cornell Investigational Drug Shuts Down Aggressive Form Of Leukemia That Affects Children”