Dr. Gail J. Roboz is the principal investigator for a new immunotherapy clinical trial at Weill Cornell Medicine and NewYork-Presbyterian for people with acute myeloid leukemia (AML). The clinical trial is evaluating engineered CAR-T cells, UCART123, that have been specially engineered in the laboratory to be able to target a molecule on the surface of leukemia cells called CD123. It is hoped that the T cells will be able to destroy the CD123 positive leukemia cells and lead to remission in selected patients with AML.
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In the summer of 2015, Weill Cornell Medical College and Cellectis, a French pharmaceutical company, announced a translational research collaboration for a new allogeneic chimeric antigen receptor T cell therapy called UCART123. This “off-the-shelf” product is a directed immunotherapy for patients with acute myeloid leukemia (AML), an aggressive blood cancer. T cells are harvested from healthy donors and engineered to target the CD123 antigen, which is found on AML blast and stem cells, as well as tumor cells in another aggressive malignancy, blastic plasmacytoid dendritic cell neoplasm (BPDCN).
The clinical trial of UCART123 in AML is being led by principal investigator Dr. Gail J. Roboz, Professor of Medicine and Director of the Leukemia Program at Weill Cornell Medicine and NewYork-Presbyterian. Translational scientific studies for the trial are being conducted in the laboratory of Dr. Monica Guzman, Associate Professor of Pharmacology in Medicine, also at Weill Cornell. Clinical research on UCART123 in BPDCN will be led by Dr. Naveen Pemmaraju and Dr. Hagop Kantarjian at the MD Anderson Cancer Center in Houston, Texas.
Stay tuned for more updates regarding the new phase 1 clinical trial for UCART123 to be initiated here at the Leukemia Program in the coming months.
Dr. Ellen Ritchie recently participated in an OncLive discussion on the latest modifications to the World Health Organization (WHO) classification of Myelodysplastic Syndromes (MDS). WHO classification is the standard diagnostic system utilized by medical institutions worldwide, including here at Weill Cornell Medicine. Recent advances in our understanding of the biological course of MDS have warranted revision to its WHO classification, which was last updated in 2008. In particular, mutational and cytogenetic analyses have to led to refinement of diagnostic terms for MDS. These modifications include a distinction between single versus multilineage dysplasia and elimination of the term “cytopenia.”
The OncLive discussion centered on implications of the new classification on the prognosis and treatment of MDS. While the WHO classification is just one of many factors to consider when evaluating the prognosis of the disorder, the panelists agree that the new modifications will make it easier to determine an appropriate course of treatment for their patients. To learn more, click here or watch the video below.
December is an exciting month here at the Leukemia Program, as each year, our doctors and researchers are invited to attend and present their work at the annual meeting of the American Society of Hematology (ASH). This important meeting provides the opportunity to network with thousands of hematology specialists from all over the world.
This year, the 58th ASH Annual Meeting & Exposition is being held December 3-6 in San Diego, California. We are very proud to play an integral role in research that is changing the way leukemia is diagnosed, tracked and treated. The below abstracts are being presented in oral or poster sessions by the Leukemia Program’s physicians, researchers, and collaborators.
#226. A Randomized Phase II Study of Low-Dose Decitabine Versus Azacitidine in Patients with Low- or Intermediate-1-Risk Myelodysplastic Syndromes: A Report on Behalf of the MDS Clinical Research Consortium
#902. Analysis of Efficacy By Age for Patients Aged 60–75 with Untreated Secondary Acute Myeloid Leukemia (AML) Treated with CPX-351 Liposome Injection Versus Conventional Cytarabine and Daunorubicin in a Phase III Trial Clinically Relevant Abstract
#904. Long Term Survival and Clinical Complete Responses of Various Prognostic Subgroups in 103 Relapsed/Refractory Acute Myeloid Leukemia (r/r AML) Patients Treated with Guadecitabine (SGI-110) in Phase 2 Studies
#906. Survival Following Allogeneic Hematopoietic Cell Transplantation in Older High-Risk Acute Myeloid Leukemia Patients Initially Treated with CPX-351 Liposome Injection Versus Standard Cytarabine and Daunorubicin: Subgroup Analysis of a Large Phase III Trial
#1063. The Use of Hypomethylating Agents (HMAs) in Patients with Relapsed and Refractory Acute Myeloid Leukemia (RR-AML): Clinical Outcomes and Their Predictors in a Large International Patient Cohort
#1070. Determination of IDH1 Mutational Burden and Clearance Via Next-Generation Sequencing in Patients with IDH1 Mutation-Positive Hematologic Malignancies Receiving AG-120, a First-in-Class Inhibitor of Mutant IDH1
#2816. Thioguanine Combined with Decitabine Can Overcome Resistance to Hypomethylating Agents: Final Results of a Phase I Trial of a Pharmacodynamically-Conceived Thioguanine/Decitabine Combination in Patients with Advanced Myeloid Malignancies
#3548. Current Diagnosis Patterns for Acute Myeloid Leukemia (AML) in Clinical Practice Compared with World Health Organization (WHO) 2008 Recommendations: Outcomes from the CONNECT® Myelodysplastic Syndromes (MDS) and AML Disease Registry
#1629. A Pediatric-Inspired Regimen Containing Multiple Doses of Intravenous Pegylated Asparaginase Appears Safe and Effective in Newly Diagnosed Adult Patients with Ph-Negative Acute Lymphoblastic Leukemia in Adults up to Age 60: Results of a Multi-Center Phase II Clinical Trial
#3090. ENESTgoal Treatment-Free Remission Study: Updated Preliminary Results and Digital Polymerase Chain Reaction Analysis in Patients with Chronic Myeloid Leukemia in Chronic Phase Who Switched from Imatinib to Nilotinib
#479. Interim Analysis of the Myeloproliferative Disorders Research Consortium (MPD-RC) 112 Global Phase III Trial of Front Line Pegylated Interferon Alpha-2a Vs. Hydroxyurea in High Risk Polycythemia Vera and Essential Thrombocythemia
#4271. Impact on MPN Symptoms and Quality of Life of Front Line Pegylated Interferon Alpha-2a Vs. Hydroxyurea in High Risk Polycythemia Vera and Essential Thrombocythemia: Interim Analysis Results of Myeloproliferative Disorders Research Consortium (MPD-RC) 112 Global Phase III Trial
#112. Frequency and Prognostic Significance of Cytogenetic Abnormalities in 1269 Patients with Therapy-Related Myelodysplastic Syndrome – a Study of the International Working Group (IWG-PM) for Myelodysplastic Syndromes
Earlier this week the U.S. News and World Report released their annual survey of “Best Hospitals”. NewYork-Presbyterian one of the country’s largest and most comprehensive hospitals was ranked New York’s No. 1 hospital for the 16th year in a row, and No. 6 ranked hospital in all of the United States. Dr. Augustine M.K. Choi, interim dean of Weill Cornell Medicine commented,
“Our esteemed physicians and scientists at Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medicine always put patients first, providing them with the finest, most comprehensive care so that they can live their healthiest lives. Together we create one of the top academic medical centers in the United States, motivated by a shared commitment: to drive excellence in healthcare and truly make a difference in New York and beyond.”
This commitment is shared by the physicians, researchers, and staff in the Leukemia Program.
Gail Roboz, MD from Weill Cornell Medicine discusses minimal residual disease (MRD) found in acute myeloid leukemia (AML) patients. According to Dr Roboz the biology of the remaining leukemia cells may not be similar to the bulk disease that was eliminated with initial therapy. Currently there are efforts to characterize and quantify the remaining cells, with the hopes to determine whether existing or novel treatments can be used to lower their number to below the threshold level required for stem cell transplants. Furthermore, stem cell transplants are dramatically less effective if there is minimal residual disease detected so any therapy to reduce these cells may confer an advantage. Recorded at the 2016 Annual Meeting of the British Society of Haematology (BSH) and International Society of Hematology (ISH), in Glasgow, Scotland.
Original story posted to Video Journal of Hematological Oncology [go]