Dr. John Leonard Comments on CAR T-Cell Therapy Outlook

Dr. John Leonard at State of the Science SummitChimeric antigen receptor (CAR) T-cell therapy is an emerging form of immunotherapy that leverages the strength of a patient’s own immune system to fight cancer.

Immune cells called T-cells are extracted from the patient’s blood and modified in the laboratory to produce chimeric antigen receptors, surface-level proteins that enable the T-cells to recognize and fight targeted antigenic tumor cells. The newly engineered T-cells are then cultivated in a lab before infusion back into the patient’s body, where they further multiply and go to work attacking cells that possess the antigen that they were programmed to destroy.

At the OncLive State of the Science Summit on Treatment of Hematologic Malignancies, Dr. John Leonard, who served as co-chair for the May 4 event, expressed promise in the use of CAR T-cell therapy for patients with acute lymphoblastic leukemia (ALL), in particular.

Dr. Leonard said that in a small group of clinical trial recipients with ALL, the immunotherapy has produced excellent, seemingly durable responses, and more data on CAR T-cells for patients with hard-to-treat lymphomas, like resistant forms of diffuse large B-cell lymphoma (DLBCL), are forthcoming.

While patient selection is a crucial part of interpreting the data and planning for the future, Dr. Leonard believes that the main challenges in the development of CAR T-cell therapy relate to factors of patient selection such age, comorbidities, and aggressive cancers with prohibitive wait times for engineered cells, which can take as long as several weeks depending on the specific CAR product being used.

 “I think there’s no doubt that some patients benefit, but at least in the near-term, it’s going to be a relatively small number of patients that will get CAR T-cells for lymphoma,” he said.

Check out what else Dr. Leonard had to say about CAR T-cells in this video from OncLive:

 

Weill Cornell Investigational Drug Shuts Down Aggressive Form Of Leukemia That Affects Children

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.

Click here to read the press article describing the study results. Click here to read the published research paper.

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”