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


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 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 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 dark

Friday, July 1, 2016

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

Rohan Ramakrishna, M.D., and Gail Roboz, M.D., at the NYC regional Cancer Moonshot Summit at Weill Cornell Medicine
Rohan Ramakrishna, M.D., and Gail Roboz, M.D.. Photo: Ira Fox

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]


Dr. Gail Roboz discusses the latest research, clinical trials and breakthroughs to be presented at the upcoming American Society of Clinical Oncology (ASCO) annual meeting

Dr. Gail J. Roboz appeared as a guest on Fox 24’s News Talk Central to discuss the latest research, clinical trials and breakthroughs to be presented at the upcoming American Society of Clinical Oncology (ASCO) annual meeting. Please view the WGXA-FOX 24 segment

“One of the most important things coming out of this meeting is that there is more collaboration between people and there’s more collaboration between pharmaceutical companies to bring new drugs and new technologies together,” notes Dr. Roboz.

To follow ASCO’s annual meeting
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The broad spectrum of myelodysplastic syndrome

Ellen Ritchie, M.D., assistant professor of clinical medicine, joins OncLive in a discussion on the current best practices in the management of myelodysplastic syndrome and how the latest research is likely to impact the field.

“I think it’s very important when we talk to patients about MDS that we talk about the broad spectrum of disease and that we don’t label it as cancer necessarily. Because I think that it can almost be as detrimental to their quality of life with the disease afterward, by labeling it as such, than the treatments and supportive care that we have to give them.” – Dr. Ellen Ritchie

To learn more about our CRUSH!!MDS program and to participate in a clinical trial [go]

See original post on the Meyer Cancer Center site May 12, 2016