On August 30, 2017, the United States Food and Drug Administration (FDA) approved the cell-based gene therapy Kymriah for treatment of children and young adults with a certain form of acute lymphoblastic leukemia (ALL), the most common childhood cancer in America. The approval greenlights the first gene therapy to be made available in the United States.
Each dose of Kymriah is customized to the individual patient by way of an emerging form of immunotherapy called chimeric antigen receptor (CAR) T-cell therapy. T-cells are extracted from the patient’s blood and genetically modified in the laboratory to produce chimeric antigen receptors, surface-level proteins that enable the T-cells to recognize and fight leukemia cells that possess the antigen CD19. The newly engineered T-cells are then infused back into the patient’s body. The goal of Kymriah and other forms of immunotherapy is to target and attack the cancer cells that they are programmed to destroy.
This historic approval follows clinical trials demonstrating durable safety and efficacy in children and young adults up to age 25 with relapsed or refractory B-cell precursor ALL.
Weill Cornell Medicine and NewYork-Presbyterian Hosptial has ongoing clinical trials evaluating CAR T-cell therapy in adults with certain forms of leukemia. To learn more, visit: https://jcto.weill.cornell.edu/.
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
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]
This clinical study is aimed at men and women with a diagnosis of: Chronic Neutrophilic Leukemia (CNL), Chronic Myelomonocytic Leuekmia (CMML), atypical Chronic Myeloid Leukemia (aCML), Juvenile Myelomonocytic Leukemia (JMML), and Myelodysplastic & Myeloproliferative Neoplasm Unclassifiable (MDS/MPN-U). Click here to learn more or see if you are eligible to participate.
This special week corresponds with the National Organization for Rare Disorders (NORD)’s Rare Disease Day, which is held on February 29.
For more information please visit AAMDS
Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of Azacitidine with or without Birinapant for subjects with Higher Risk Myelodysplastic Syndrome or Chronic Myelomonocytic LeukemiaPosted: January 22, 2015
The Weill Cornell Leukemia Program has recently opened a new clinical trial for men and women who have been diagnosed with Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL) . The study sponsor is TetraLogics Pharmaceuticals and the principal investigator at Weill Cornell is Dr. Ellen Ritchie. For more information about the study, please call Katherine Hassfurter, RN at (212) 746-4882 or e-mail firstname.lastname@example.org.
- Men and women age 18 and older
- Subjects that have not been treated with hypomethylating agents for MDS or CMMoL
- Histologically confirmed diagnosis of the following conditions:
– Myelodysplastic Syndrome (MDS)
– Chronic Myelomonocytic Leukemia (CMMoL)
- Life expectancy of at least 3 months
- ECOG score of 0 or 1
- Detailed eligibility reviewed when you contact the study team
This is a randomized, double-blind, clinical trial for men and women with high risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL). The study is evaluating an experimental drug called Birinapant.
Birinapant is being studied as a potential new treatment for MDS or CML. Birinapant removes certain chemicals (proteins) in a cancer cell which leads to the death of cancer cells. From laboratory and animal studies, birinapant is more likely to cause the death of cancer cells than normal cells. Studies combining birinapant with chemotherapy in the human cancer cell laboratory models showed that the addition of birinapant to chemotherapy can result in further blocking of cancer growth, or overcoming cancer resistance to chemotherapy. In previous research studies, birinapant has shown to be well-tolerated when given alone or in combination with other chemotherapy drugs.
The purpose of this study is to to compare the safety and efficacy (how well it works) of azacitidine plus an investigational drug (birinapant) versus azacitidine plus a placebo (an inactive substance), in patients with higher-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL).
Dose Escalation and Cohort Expansion Study of TEN-010 in Patients with Acute Myeloid Leukemia and Myelodysplastic SyndromePosted: November 17, 2014
The Weill Cornell Leukemia Program has recently opened a new clinical trial for men and women who have been diagnosed with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). The study sponsor is Tensha Therapeutics, Inc. and the principal investigator at Weill Cornell is Dr. Gail Roboz. For more information about the study, please call Tania Curcio, RN at (212) 746-2571 or e-mail email@example.com.
- Men and women age 18 and older with a confirmed diagnosis of AML or MDS
- Previously treated with at least one prior therapy
- Subjects with a history of allogeneic (from another person) stem cell transplant are eligible for study participation
- Life expectancy of at least 2 months
- Detailed eligibility reviewed when you contact the study team
This is a Phase 1, non-randomized, open-label, multi-center study that utilizes the investigational study drug TEN-010. TEN-010 belongs to a group of drugs called bromodomain inhibitors. Bromodomains are found in cancer cells and bromodomain inhibitors may have promise as a therapy for patients who have cancer. Currently, there are no bromodomain inhibitors approved by the FDA for humans. Research in the laboratory has shown that TEN 010 kills cancer cells in different types of both blood cancers.
The study is conducted in two parts; Part A and Part B. In Part A, escalating doses of TEN-010 will be administered to patients to evaluate safety and side effects that may limit the amount of TEN-010 given to patients. One of the goals of Part A is to establish the maximum tolerated dose (MTD) of TEN-010. Part B is an expansion study in which patients are treated at the MTD of TEN-010 to identify safety, tolerability, and how well the disease responds to treatment with TEN-010.
All subjects participating in this study will receive the study drug TEN-010 once daily. Subjects will be assigned to one of three different dose levels ranging from 0.06 mg/kg to 0.24 mg/kg .