Weill Cornell Leukemia Program Abstracts @ ASH 2016

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.

AML

#98. Results of a Clinical Study of Pevonedistat (Pev), a First-in-Class NEDD8-Activating Enzyme (NAE) Inhibitor, Combined with Azacitidine (Aza) in Older Patients (Pts) with Acute Myeloid Leukemia

#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

#433. Cooperative Epigenetic Remodeling By TET2 Loss and NRAS Mutation Drives Myeloid Transformation and MEK Inhibitor Sensitivity

#438. BCL6 Is Critical to Overcome Oncogene-Induced Senescence in RAS-Mediated B Cell Transformation

#582. Acute Myeloid Leukemia Cells Resist Chemotherapy through a Reversible Senescence-like State Maintaining Repopulation Potential

#599. Changes of the Mutational Landscape in Relapsed Acute Myeloid Leukemia

#765. Allogeneic Tcrα/β Deficient CAR T-Cells Targeting CD123 Prolong Overall Survival of AML Patient-Derived Xenografts

#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

#903. Durable Overall Survival Benefit in Patients ≥ 60 Years with Relapsed or Refractory AML Treated with Vosaroxin/Cytarabine Vs Placebo/Cytarabine: Updated Results from the Valor Trial

#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

#1048. Cooperative Gene Repression By DNA Methylation and LSD1-Mediated Enhancer Inactivation in Acute Myeloid Leukemia

#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

#1069. Final Results of the Chrysalis Trial: A First-in-Human Phase 1/2 Dose-Escalation, Dose-Expansion Study of Gilteritinib (ASP2215) in Patients with Relapsed/Refractory Acute Myeloid Leukemia

#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

#1077. CD97 Is a Critical Regulator of Acute Myeloid Leukemia Stem Cell Function

#1553. PI3 Kinase p110 Delta Is Required for Leukemic Cell Survival and Self-Renewal in t(8;21) Acute Myeloid Leukemia

#1680. Genetic Determinants of Response to Guadecitabine (SGI-110) in AML

#1693. Selection and Characterization of Antibody Clones Are Critical for Accurate Flow Cytometry-Based Monitoring of CD123 in Acute Myeloid Leukemia

#2011. Comprehensive Analysis of Safety: Rigosertib in 557 Patients with Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia

#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

#2888. Minimal Residual Disease Assessment of Common and Rare NPM1 Mutations Using a Single Massively Multiplex Digital PCR Assay

#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

#4039. Pre-Clinical Studies of Anti-CD123 CAR-T Cells for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

ALL

#279. The Pro-Tumorigenic Vascular Niche Sustains the T-Cell Acute Lymphoblastic Leukemia Phenotype and Fosters Resistance to Therapy

#907. Oncogenic Feedback Activation Between BCL6 and MLL Promotes Malignant Transformation in MLL-Rearranged Acute Lymphoblastic Leukemia

#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

#4088. CD25 Enables Oncogenic BCR Signaling and Represents a Therapeutic Target in Refractory B Cell Malignancies

CML

#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

MPN

#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

MDS

#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

#297. Is Serial Monitoring of Myeloid Mutations Clinically Relevant in Myelodysplastic Syndromes (MDS): A Report on Behalf of the MDS Clinical Research Consortium (CRC)

#343. Enasidenib (AG-221), a Potent Oral Inhibitor of Mutant Isocitrate Dehydrogenase 2 (IDH2) Enzyme, Induces Hematologic Responses in Patients with Myelodysplastic Syndromes

#964. Runx1 Deficiency and MDS-Associated U2af1 Mutation Cooperate for Leukemia Development in a New Mouse Model

#2011. Comprehensive Analysis of Safety: Rigosertib in 557 Patients with Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)

#4321. Ring Sideroblasts and SF3B1 Mutations in Myelodysplastic Syndromes (MDS): Are They Two Faces of the Same Coin? a Study on Behalf of the MDS Clinical Research Consortium (MDS CRC)

#4322. Optimal Treatment Order of Lenalidomide and Hypomethylating Agents for Lower-Risk Myelodysplastic Syndromes: A Report on Behalf of the MDS Clinical Research Consortium

#4332. Importance of Complete Remission on Predicting Overall Survival in Patients with Lower-Risk Myelodysplastic Syndromes


Weill Cornell Medical College and Cellectis Announce Research Alliance Advancing Drug Discovery and the Translation of Novel Immunotherapies in Leukemia

Collaboration Will Focus on Improving Patient Outcomes in AML Using Targeted Cellular Therapy Developed by Cellectis

June 02, 2015 05:00 PM Eastern Daylight Time

NEW YORK–(BUSINESS WIRE)–Regulatory News:

“Cellectis has interesting preclinical data on UCART123 and our alliance will seek to build on these findings to better understand the clinical potential of this therapy. Our patients are anxiously awaiting the start of clinical trials.”

Weill Cornell Medical College and Cellectis have entered into a strategic translational research alliance to accelerate the development of a targeted immunotherapy for patients with acute myelogenous leukemia (AML), a deadly blood cancer. The alliance will foster the development of Cellectis’ lead product candidate in AML, called UCART123.

The collaboration combines Weill Cornell’s broad expertise and resources in translational stem cell science and developmental therapeutics with Cellectis’ work in development and manufacturing of gene edited CAR-T cell product candidates, a special kind of immune cell that includes an antibody-derived receptor. The research will be led by co-principal investigators Dr. Gail J. Roboz, director of the leukemia program and an associate professor of medicine at Weill Cornell, and Dr. Monica Guzman, an assistant professor of pharmacology in medicine at Weill Cornell. Dr. Roboz is an internationally recognized leader in the field of acute leukemia and will design and implement clinical testing of UCART123 in patients with AML. Dr. Guzman is a renowned leukemia stem cell biologist who specializes in preclinical and early-stage testing to optimize the development of stem cell-targeted cancer drugs.

The alliance will seek to accelerate the development of Cellectis’ UCART123 in AML. Cellectis’ proprietary allogeneic CAR T-cell platform utilizes T-cells (immune cells) from healthy donors. The T-cells are engineered with a Chimeric Antigen Receptor (CAR), which enables them to detect specific proteins (antigens) expressed on malignant tumors. Large numbers of allogeneic CAR-modified T-cells are grown in the laboratory and then infused into a patient. The enhanced cells are designed to recognize and attack stem cells harboring the CD123 antigen, which is present on AML blast and stem cells. To enhance safety and minimize toxicity for patients, the company’s gene-editing process features customized control properties that seek to prevent the T cells from inappropriately attacking healthy tissues. Cellectis hopes to develop a cost-effective, “off-the-shelf” allogeneic CAR T-cell product, designed for efficient storage and distribution to patients around the globe.

Cellectis in April opened a new research and development facility in New York City, located in close proximity to the Weill Cornell campus.

“We are pleased to collaborate with Cellectis to develop and advance next-generation treatments for patients with this devastating form of leukemia,” said Dr. Laurie H. Glimcher, the Stephen and Suzanne Weiss Dean of Weill Cornell Medical College. “Cellectis’ proficiency in genome engineering and our complementary expertise in translational research will help us realize our common goal of improving human health in New York and around the globe.”

“CAR-T cells have shown remarkable promise in the treatment of acute lymphoblastic leukemia,” Dr. Roboz said. “Cellectis has interesting preclinical data on UCART123 and our alliance will seek to build on these findings to better understand the clinical potential of this therapy. Our patients are anxiously awaiting the start of clinical trials.”

“Weill Cornell offers unsurpassed expertise in translational research, with a wealth of leading-edge technologies and resources to help advance our pipeline of unique CAR-T product candidates,” said Dr. Mathieu Simon, executive vice president and chief operating officer at Cellectis. “We are excited by the prospect of working with Dr. Roboz, Dr. Guzman and other premier investigators in leukemia stem cell research.”

Weill Cornell’s Office of BioPharma Alliances and Research Collaborations negotiated the three-year alliance. In the program’s pre-clinical phase, Weill Cornell researchers will perform multiple analyses, including data mining of primary AML samples, immune profiling of AML patients and in vitro evaluation of allogeneically derived anti-CD123 CAR-T cells. In the alliance’s second phase, Weill Cornell and Cellectis will jointly develop protocols to facilitate early-phase testing, including phase 1 clinical trials.

“Cellectis believes the CAR-T platform has the potential to transform the way cancer patients are treated. We are confident that our broad, cross-discipline collaboration with Weill Cornell will foster creativity and speed in drug development for the benefit of clinicians and patients living with AML,” said Dr. André Choulika, chief executive officer of Cellectis.

The mission of Weill Cornell’s Office of BioPharma Alliances and Research Collaborations is to proactively generate, structure and market translational research alliances with industry in order to advance promising research projects that have commercial potential. For more information, contact Larry Schlossman at las2041@med.cornell.edu or at 212-746-6909.

About Weill Cornell Medical College

Weill Cornell Medical College, Cornell University’s medical school located in New York City, is committed to excellence in research, teaching, patient care and the advancement of the art and science of medicine, locally, nationally and globally. Physicians and scientists of Weill Cornell Medical College are engaged in cutting-edge research from bench to bedside aimed at unlocking mysteries of the human body in health and sickness and toward developing new treatments and prevention strategies. In its commitment to global health and education, Weill Cornell has a strong presence in places such as Qatar, Tanzania, Haiti, Brazil, Austria and Turkey. Through the historic Weill Cornell Medical College in Qatar, the Medical College is the first in the U.S. to offer its M.D. degree overseas. Weill Cornell is the birthplace of many medical advances—including the development of the Pap test for cervical cancer, the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial of gene therapy for Parkinson’s disease, and most recently, the world’s first successful use of deep brain stimulation to treat a minimally conscious brain-injured patient. Weill Cornell Medical College is affiliated with NewYork-Presbyterian Hospital, where its faculty provides comprehensive patient care at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. The Medical College is also affiliated with Houston Methodist. For more information, visit weill.cornell.edu.

About Cellectis

Cellectis is a preclinical stage biopharmaceutical company focused on developing immunotherapies based on gene edited engineered CAR-T cells (UCART). The company’s mission is to develop a new generation of cancer therapies based on engineered T-cells. Cellectis capitalizes on its 15 years of expertise in genome engineering – based on its flagship TALEN® products and meganucleases and pioneering electroporation PulseAgile technology – to create a new generation of immunotherapies. CAR technologies are designed to target surface antigens expressed on cells. Using its life-science-focused, pioneering genome-engineering technologies, Cellectis’ goal is to create innovative products in multiple fields and with various target markets. Cellectis S.A. is listed on the Nasdaq Global Market (ticker: CLLS) and on the NYSE Alternext market (ticker: ALCLS). To find out more about us, visit our website: www.cellectis.com

Contacts

Media contacts
Weill Cornell Medical College
Sarah Smith, Director of Media Relations
Phone: 646-317-7401
email: sas2072@med.cornell.edu
or
Cellectis
Jennifer Moore, Director of Communications
Phone: 917-580-1088
email: media@cellectis.com
or
BMC Communications
Brad Miles
Phone: 646 513-3125
email: bmiles@bmccommunications.com
or
IR contact
Cellectis
Simon Harnest, VP Finance and Investor Relations
Phone: 646-385-9008
email: simon.harnest@cellectis.com


Dr. Gail Roboz reviews existing and evolving approaches to the treatment of patients with AML for Medscape Education

Medscape AML Presentation  To view the entire presentation and slideshow, click here.


Targeted Healthcare interviews Dr. Gail Roboz on improving the standard treatment of AML

To view the video, click here.

Targeted Healthdare Interview


New Clinical Trial: Combination Chemotherapy and Dasatinib in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

The Weill Cornell Leukemia Program is now recruiting patients for a new study, “CALGB 10801: A Phase II Study of Induction (Daunorubicin/Cytarabine) and Consolidation (High-Dose Cytarabine) Chemotherapy Plus Dasatinib and Continuation Therapy with Dasatinib Alone in Newly Diagnosed Patients with Core Binding Factor Acute Myeloid Leukemia (AML).”

The physician leading the study at Weill Cornell is Gail Roboz, MD. For more information or to see if you are eligible for the study, please contact Tania Curcio, RN at (212) 746-2571 or email Tania at tjc9003@med.cornell.edu.

Study details:

This is a clinical trial for patients with acute myeloid leukemia (AML) that possesses an abnormal molecular feature (a gene mutation). The purpose of this study is to test the safety and effectiveness of adding the drug dasatinib to a treatment regimen in patients with AML and to determine how well the leukemia responds to the treatment. The study is being done because currently available treatment is not effective in curing patients with this type of leukemia.

There are three parts to the treatment in this study. The first part of the therapy will test the safety and effectiveness of adding dasatinib to the standard combination of chemotherapy drugs used to treat AML that include daunorubicin and cytarabine. The second part of the therapy will test the safety and effectiveness of combining dasatinib with another chemotherapy treatment, consolidation therapy with high-dose cytarabine. Finally, the third part of the therapy will test the effectiveness of the use of dasatinib alone for 12 months during continuation therapy.

Patients will receive therapy for about 18 months on study. After you are finished with the therapy, you will be asked to visit the office for follow-up at least every 2 months for 2 years, then every 3 months for 2 years, then yearly for a maximum of 10 years from when you entered the study.

Key Eligibility
  • Men and women age 18 and older
  • Acute myeloid leukemia (AML with Core Binding Factor (CBF) abnormality
  • No prior chemotherapy for leukemia or myelodysplasia
  • Detailed eligibility reviewed when you contact the study team

Dr. Roboz Speaks on Genomics in AML

Dr. Gail Roboz spoke with ecancertv at ASH 2011 in San Diego about the major genomic research on acute myeloid leukaemia.  There has been a lot of recent success on identifying mutations and abnormalities in AML; however, Prof Roboz believes that the discovery period with genomic research is coming to an end and a move towards clinical trials and targeted therapies need to be developed.  The largest development has been the role of stems cell in research and how to target the cells that are left over after chemotherapy.

Roboz ecancer.tv


Leukemia Team Presents at ASCO Annual Meeting

The work of Drs. Ritchie, Roboz, Scandura, Gergis, and Feldman, and nurse Tania Curcio was presented at the 2009 American Society of Clinical Oncology Annual Meeting.  The presentation focused on a clinical trial treating elderly AML and high-grade MDS patients.  To view the presentation, click here.