FDA Approves CAR T-Cell Therapy for Kids and Young Adults with Acute Lymphoblastic LeukemiaPosted: September 1, 2017 Filed under: Leukemia News, Uncategorized | Tags: Acute lymphoblastic leukemia, ALL, CAR T-Cell Therapy, CD19, Childhood cancer, Clinical Trials, FDA Approval, Gene therapy, Immunotherapy, Kymriah, Leukemia, Young adult cancer Comments Off on FDA Approves CAR T-Cell Therapy for Kids and Young Adults with Acute Lymphoblastic Leukemia
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/.
FDA Approves Two Treatments for AMLPosted: August 4, 2017 Filed under: Clinical Trials, Leukemia News, Patient Education, Uncategorized | Tags: Acute Myeloid Leukemia, AML, CPX-351, FDA Approval, IDH-2, Idhifa, leukemia clinical trials, Vyxeos Comments Off on FDA Approves Two Treatments for AML
The United States Food and Drug Administration (FDA) has approved CPX-351, a combination of chemotherapy drugs daunorubicin and cytarabine also known as Vyxeos, for treatment of two types of high-risk acute myeloid leukemia (AML).
Clinical trial participants with newly diagnosed therapy-related AML (t-AML) and those with AML accompanied by myelodysplasia-related changes (AML-MRC) demonstrated increased life expectancy when treated with CPX-351, as compared to those treated with separate administrations of daunorubicin and cytarabine.
The Weill Cornell Medicine and NewYork-Presbyterian Leukemia Program, in collaboration with our Joint Clinical Trials Office, participated in the expanded access protocol for CPX-351, and we continue to use the drug across our various studies.
We were also among the sites for the clinical trial that led to another of this week’s FDA approvals: Idhifa, a targeted therapy for relapsed or refractory AML patients with the genetic mutation isocitrate dehydrogenase-2 (IDH2). After treatment with Idhifa, 34 percent of the 157 trial participants who required blood or platelet transfusions at the start of the study no longer required transfusions.
We are proud to be among the first medical centers offering novel treatment options like CPX-351 and Idhifa to our patients and look forward to continued prompt delivery of therapies that may improve life expectancy and quality of life for those affected by leukemia.
Weill Cornell Leukemia Program Abstracts @ ASH 2016Posted: December 5, 2016 Filed under: Leukemia News, Uncategorized | Tags: acute lymphocytic leukemia, Acute Myeloid Leukemia, ALL, AML, Blood Disorders, bone marrow, cancer, cancer treatment, chemotherapy, CML, hematology, Leukemia, Leukemia News, MDS, Oncology, Research Comments Off on 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.
#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)
#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
#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
#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
Cancer Care: The Next GenerationPosted: September 20, 2016 Filed under: Clinical Trials, Leukemia News, Patient Education, Uncategorized | Tags: Blood Disorders, cancer treatment, Gail Roboz, hematology, Leukemia, Leukemia Treatment, MDS, Myelodysplastic Syndrome, New York Presbyterian, Weill Cornell, Weill Cornell Leukemia Program Comments Off on Cancer Care: The Next Generation
Dr. Gail Roboz on ABC talks about “taking a chance on something new.” [go]
NewYork-Presbyterian Hospital Ranked as New York Best HospitalPosted: August 4, 2016 Filed under: Leukemia News, Uncategorized | Tags: Leukemia News, New York Presbyterian, Weill Cornell Comments Off on NewYork-Presbyterian Hospital Ranked as New York Best Hospital
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.
Treatment of minimal residual disease in AML patientsPosted: July 15, 2016 Filed under: Leukemia News, Patient Education, Physician Presentations, Uncategorized | Tags: Acute Myeloid Leukemia, Blood Disorders, cancer, Gail Roboz, hematology, Leukemia, Weill Cornell, Weill Cornell Leukemia Program Comments Off on Treatment of minimal residual disease in AML patients
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]
Not just a shot in the darkPosted: July 1, 2016 Filed under: Clinical Trials, Laboratory Research, Leukemia News, Patient Education, Physician Presentations, Uncategorized | Tags: Blood Disorders, cancer awareness, Gail Roboz MD, leukemia clinical trials, Leukemia Treatment, Moonshot, Weill Cornell Comments Off on Not just a shot in the dark
In many ways, it’s a science success story: 8-year-old boy with a rare form of brain cancer is treated by one of the world’s leading experts in the disease, who collaborates with a pioneering precision medicine institute to sequence his cancer and create a first-of-its-kind tumor model replica in the lab, allowing for further analysis and treatment testing without risk of harm to the child.
Upon analysis, the physician-scientist discovers a mutation previously not known to be linked to that type of cancer — and it happens to be in the protein that his colleague has spent a career studying. He contacts the colleague to ask if there is a drug to target the protein, and it arrives the next day. Applied to the tumor model, the drug effectively kills 80-90 percent of the diseased cells.
If only the story ended there.
Unfortunately, although the drug has been approved by the FDA, it cannot be used on the young patient because it has never been tested in children, and the pharmaceutical company controlling the drug is not willing to take the risk.
“We now start the gymnastics of trying to get permission from the FDA based on compassionate use,” said Jeffrey Greenfield, M.D., Ph.D. “We’ve done it before, and it takes anywhere from 3-6 months. This boy doesn’t have 3-6 months.”
Greenfield, a neurosurgeon at Weill Cornell Medicine and NewYork-Presbyterian, shared the anecdote at a special event held at Weill Cornell Medicine on June 29, one of 270 across the United States convened by Vice President Joe Biden in tandem with a national summit at Howard University in Washington, DC
Biden invited regional participants to discuss the goals of the “Cancer Moonshot” mission, announced in January by President Barack Obama as a way to accelerate cancer research, foster data sharing and collaboration, and improve patient access to care — all on a five-year timeline.
Greenfield said his story summed up some of the challenges the nation will face in trying to achieve such an ambitious goal.
“The promise of precision medicine, which is enormous and which we have all bought into, doesn’t deliver in this case,” Greenfield said. “We’ve done all the work that we’ve promised to do, and we still have hurdles. The science is great, the medicine is great, but we’ve got to figure out a way to bridge the chasm between academia, pharma and clinic.”
The future is now
Greenfield was joined at the event by more than a dozen other distinguished researchers and physicians, as well as a standing-room only crowd of around 100.
Participants heard that in many ways, the future of medicine is already here. Silvia Formenti, M.D., discussed how she uses radiation therapy to turn patients’ own tumors into internal “vaccines,” and Ching Tung, Ph.D., director of the Molecular Imaging Innovations Institute described new ways of “seeing” cancer.
Neurosurgeon Mark Souweidane, M.D., spoke about the importance of developing new forms of drug delivery and working with industry to be able to integrate research and technology into the operating room. His colleagues Susan Pannullo, M.D., and Michael Kaplitt, M.D., Ph.D., explained stereotactic radiosurgery and the use of ultrasound technology to poke holes in the blood-brain barrier.
“These are ways we can use novel non-invasive technologies that will put us as surgeons out of business, unfortunately, but will help heal the world,” Kaplitt said.
Gail Roboz, M.D., director of the Weill Cornell Leukemia Program, described immunotherapy, and in particular the use of CAR-T cells as an emerging therapy.
“The idea isn’t new, “Roboz said. “What’s new is that we can actually do it, we are able to finally do things that were Jetsons level before.”
“In 2016, we are at an amazing inflection point in cancer therapy,” added neurosurgeon Rohan Ramakrishna, M.D. “It’s one thing to say you want to accomplish big change in five years, it’s another to be able to do that.”
But he added that the time it takes to get discoveries from bench to bedside is still too long. We need to innovate, Ramakrishna said, and we need to incentivize high-risk research.
To read the full story [go]
Some Pediatric Regimens Can Be Successful in Adults With ALLPosted: March 17, 2016 Filed under: Leukemia News, Patient Education, Uncategorized | Tags: acute lymphocytic leukemia, ALL, Blood Disorders, Gail Roboz, hematology, Weill Cornell Comments Off on Some Pediatric Regimens Can Be Successful in Adults With ALL
When Jody Winsick-Soluri was diagnosed with acute lymphoblastic leukemia (ALL), she found out she had a chromosomal abnormality, the Philadelphia chromosome, which made her prospects bleak.
“I was bleeding out; they said I might only have 24 hours to live,” Winsick-Soluri remembers. Now, after many rounds of chemotherapy, total body irradiation, two bone marrow transplants and seven years, Winsick Soluri takes a targeted drug — Sprycel (dasatinib) — that blocks a protein leukemia cells need to proliferate. “Now, I’m four-and-a-half years out from the last transplant,” the New Jersey mother of four says. “More people with ALL are staying alive a lot longer.”
See the original article posted on March 16, 2016
Phase 3 Trial Shows CPX-351 (Vyxeos) Boosts Overall Survival in AMLPosted: March 16, 2016 Filed under: Clinical Trials, Leukemia News, Uncategorized | Tags: Acute Myeloid Leukemia, Blood Disorders, Gail Roboz, hematology, Leukemia, New York Presbyterian, Weill Cornell Comments Off on Phase 3 Trial Shows CPX-351 (Vyxeos) Boosts Overall Survival in AML
What to Expect When You’re Expecting MDSPosted: March 10, 2016 Filed under: CRUSH!!MDS, Leukemia News, Patient Education, Uncategorized | Tags: Blood Disorders, cancer, Ellen Ritchie, hematology, Myelodysplastic Syndrome, Weill Cornell, Weill Cornell Leukemia Program Comments Off on What to Expect When You’re Expecting MDS
Dr. Ritchie provides and in-depth look at what it takes to diagnose MDS. For more information visit crushdmds.org.