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Kidney Cancer
Lung Cancer
Myelodysplastic Syndromes
Solid Tumors
Skin Cancer
Lymphoma
Pancreatic Cancer
Breast Cancer
Immunotherapy

FDA Pipeline: Designations in Kidney and Lung Cancers, Myelodysplastic Syndromes, and More

Over the past few weeks, the U.S. Food and Drug Administration (FDA) has issued designations and accepted applications for novel agents, as well as approved companion diagnostics. We summarize these regulatory movements below. Breakthrough Therapy Designation for MK-6482 in von Hippel-Lindau...

Myelodysplastic Syndromes
Genomics/Genetics

Role of TP53 Mutations on Disease Severity in Myelodysplastic Syndromes

Considered the “guardian of the genome,” TP53 is the most commonly mutated gene in patients with cancer. TP53's normal function is to detect DNA damage and prevent cells from passing this damage on to daughter cells. When TP53 is mutated, the protein made from this gene, called p53, can no longer...

Myelodysplastic Syndromes

Pevonedistat Plus Azacitidine May Be of Benefit in Higher-Risk Myelodysplastic Syndromes

In higher-risk myelodysplastic syndromes (MDS), a global phase II open-label study found that the combination of pevonedistat plus azacitidine was associated with a trend toward improved event-free survival and numerically longer overall survival, as compared with azacitidine alone, according to...

Myelodysplastic Syndromes

FDA Approves Oral Combination of Decitabine and Cedazuridine for Adult Patients With Myelodysplastic Syndromes

On July 7, the U.S. Food and Drug Administration (FDA) approved an oral combination of decitabine and cedazuridine (Inqovi) for adult patients with myelodysplastic syndromes (MDS), including the following: Previously treated and untreated de novo and secondary MDS with the following...

Leukemia
Lymphoma
Myelodysplastic Syndromes
Symptom Management

EHA25 Virtual: Roundup of Findings in Leukemia, Lymphoma, and More

Advances in the treatment of hematologic malignancies and some of their associated symptoms were presented during EHA25 Virtual, an ongoing online conference from the European Hematology Association (EHA). Advances in the Treatment of High-Risk CLL: CLL2-GIVe Results In the CLL2-GIVe trial, the...

leukemia
myelodysplastic syndromes

Mikkael A. Sekeres, MD, on MDS, CMML, or AML: Pevonedistat and Azacitidine

Mikkael A. Sekeres, MD, of the Cleveland Clinic, discusses data from a phase II study of pevonedistat plus azacitidine vs azacitidine alone in patients with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, or low-blast acute myeloid leukemia (Abstract 7506).

Myelodysplastic Syndromes

Luspatercept-aamt for Anemia in Adults With Myelodysplastic Syndromes

On April 3, 2020, luspatercept-aamt was approved in the treatment of anemia failing to respond to an erythropoiesis-stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks.1,2 The treatment is geared toward adult patients with very low– to intermediate-risk myelodysplastic ...

Hematologic Malignancies
Leukemia
Lymphoma
Multiple Myeloma
Myelodysplastic Syndromes
Immunotherapy

Highlights From ASH 2019 Included New Data in Leukemia, Lymphoma, Myeloma, and Myelodysplastic Syndromes

The 2019 American Society of Hematology (ASH) Annual Meeting & Exposition featured a cornucopia of sessions. It was impossible to attend all the lectures, symposia, oral presentations, poster presentations, and special events because many were concurrent. Below, we have selected some...

Myelodysplastic Syndromes

Distinct New MDS Subtype Proposed Based on Presence of Genetic Mutation

In a special report published by Malcovati et al in the journal Blood, an international working group of experts in myelodysplastic syndromes (MDS) has proposed the recognition of a distinct subtype of MDS based on the presence of a nonheritable genetic mutation that causes the disease. The...

Myelodysplastic Syndromes
Symptom Management

FDA Approves Luspatercept-aamt for Second-Line Treatment of Anemia in Adult Patients With Myelodysplastic Syndromes

On April 3, the U.S. Food and Drug Administration (FDA) approved luspatercept-aamt (Reblozyl) for the treatment of anemia that fails to respond to an erythropoiesis-stimulating agent and requires two or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk...

Lung Cancer
Thyroid Cancer
Myelodysplastic Syndromes

FDA Pipeline: Priority Review Granted for Treatment in Lung and Thyroid Cancers

This week, the U.S. Food and Drug Administration (FDA) granted Priority Review to a treatment for lung and thyroid cancers with a RET fusion or mutation; gave Breakthrough Therapy designation to a doublet therapy for TP53-mutated myelodysplastic syndromes; and issued an update to their...

Myelodysplastic Syndromes

Luspatercept in Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes

In the phase III MEDALIST trial reported in The New England Journal of Medicine, Fenaux et al found that luspatercept significantly reduced the severity of anemia vs placebo in red blood cell transfusion–dependent patients with very low- to intermediate-risk myelodysplastic syndromes with ring...

Lung Cancer
Bladder Cancer
Colorectal Cancer
Hematologic Malignancies
Myelodysplastic Syndromes
Breast Cancer
Head and Neck Cancer
Immunotherapy

FDA Pipeline: Priority Reviews in SCLC, Bladder Cancer, Colorectal Cancer

Recently, the U.S. Food and Drug Administration (FDA) granted Priority Reviews for treatments in extensive-stage small cell lung cancer (SCLC), non–muscle invasive bladder cancer, and BRAF V600E–mutant colorectal cancer. The Agency also recently issued multiple Breakthrough Therapy designations and ...

leukemia
myelodysplastic syndromes
genomics/genetics

Ilaria Iacobucci, PhD, on AML and MDS: Moving Beyond Gene Panel–Based Classifications

Ilaria Iacobucci, PhD, of St. Jude Children’s Research Hospital, discusses her work to more accurately define mutation subtypes in acute myeloid leukemia and myelodysplastic syndromes, as well as the implications for diagnosis, prognosis, and treatment (Abstract LBA-4 ).

Leukemia
Myelodysplastic Syndromes

ASH 2019: New Genomic Mutational and Expressional Data in AML, MDS

Researchers have completed the first comprehensive analysis combining full genomic sequencing and gene-expression profiles of more than 1,300 patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The research was presented by Iacobucci et al during the 2019 American Society...

myelodysplastic syndromes
leukemia

Mikkael A. Sekeres, MD, on Newly Diagnosed Hematologic Malignancies: Early Trial Findings on Glasdegib Plus Azacitidine

Mikkael A. Sekeres, MD, of the Cleveland Clinic, discusses results of a phase Ib study of glasdegib in combination with azacitidine, which showed activity in patients with untreated myelodysplastic syndromes, acute myeloid leukemia, and chronic myelomonocytic leukemia who are ineligible for intensive chemotherapy (Abstract 177).

Myelodysplastic Syndromes

Personalized Treatment Approaches on the Horizon for Myelodysplastic Syndromes

Despite the spate of recent drug approvals in blood cancer, it’s been more than 13 years since the U.S. Food and Drug Administration (FDA) has approved a treatment for myelodysplastic syndromes (MDS), a bone marrow disorder characterized by ineffective hematopoiesis. Nevertheless, data from a...

myelodysplastic syndromes

Rafael Bejar, MD, PhD, on Myelodysplastic Syndromes: Advances in Personalized Therapeutic Approaches

Rafael Bejar, MD, PhD, of the University of California, San Diego Moores Cancer Center, discusses the tailored nature of MDS treatment, including somatic mutations that could alter treatment decisions, and novel therapies with predictive biomarkers.

Myelodysplastic Syndromes
Leukemia
Lung Cancer
Solid Tumors
Colorectal Cancer
Hepatobiliary Cancer

FDA Pipeline: Fast Track Designation in Myelodysplastic Syndrome and AML, Plus Multiple Breakthrough Designations

Recently, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to magrolimab in myelodysplastic syndrome and acute myeloid leukemia (AML). The agency also granted Breakthrough Therapy designations in lung cancer and desmoid tumors, as well as Breakthrough Device designations...

colorectal cancer
hepatobiliary cancer
myelodysplastic syndromes
immunotherapy

FDA Pipeline: Designations and Reviews in Myelofibrosis, Myelodysplastic Syndromes, Biliary Tract Cancer, and T-Cell Receptor Therapy

Recently, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for momelotinib in myelofibrosis, granted Priority Review to a biologics license application for luspatercept in myelodyslastic syndromes and beta-thalassemia, granted Orphan Drug designation for a new chemical...

Myelodysplastic Syndromes

Early Studies of Two Regimens Show Benefit in High-Risk Smoldering Multiple Myeloma

MORE DATA are in to support early intervention for high-risk smoldering multiple myeloma—an early, asymptomatic entity lacking the presence of CRAB criteria (elevated calcium, renal failure, anemia, bone lesions). The latest come from two phase II studies presented at the 2018 American Society of...

Myelodysplastic Syndromes

Expert Point of View: Joseph Mikhael, MD

PRESS BRIEFING moderator Joseph Mikhael, MD, Professor of Applied Cancer Research and Drug Discovery, Translational Genomics Research Institute, City of Hope Cancer Center in Phoenix, noted that traditional models are based on simplicity, and the scoring system contains few variables. “In an era...

Myelodysplastic Syndromes

New Model May Improve Prediction of Risk in Myelodysplastic Syndromes

A PERSONALIZED risk-prediction model for myelodysplastic syndromes (MDS) has been developed through the use of a machine-learning approach that analyzes genomic and clinical data for an individual patient. According to lead investigator Aziz Nazha, MD, of the Cleveland Clinic, the model provides...

breast cancer
leukemia
myelodysplastic syndromes

FDA Pipeline: Designations for Myelodysplastic Syndromes, Triple-Negative Breast Cancer, AML, and EBV-Associated Cancers

Over the past week, the U.S. Food and Drug Administration (FDA) granted several Fast Track and Orphan Drug designations to treatments for myelodysplastic syndromes, triple-negative breast cancer, acute myeloid leukemia (AML), and Epstein-Barr virus (EBV)-associated cancers. Fast Track Designation...

skin cancer
myelodysplastic syndromes
symptom management

FDA Pipeline: Designation in Uveal Melanoma, Application for MDS Side-Effect Management

Over the past week, the U.S. Food and Drug Administration (FDA) granted a Fast Track designation to a treatment for metastatic uveal melanoma and received a biologics application for an agent to control anemia. Fast Track Designation for Tebentafusp in Metastatic Uveal Melanoma The FDA granted...

breast cancer
leukemia
myelodysplastic syndromes

FDA Pipeline: Mammography Policies, Designations for Leukemias and Myelodysplastic Syndrome

This week, the U.S. Food and Drug Administration (FDA) announced policy changes to modernize mammography policies and issued a Breakthrough Therapy designation, an Orphan Drug designation, and an investigational new drug application. FDA Advances Policy Changes to Modernize Mammography Services...

Leukemia
Myelodysplastic Syndromes
Lymphoma
Multiple Myeloma
Immunotherapy

Highlights From the 2018 ASH Annual Meeting & Exposition

TO ADD to our ongoing coverage of the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition, we bring readers of The ASCO Post these summaries of an assortment of interesting studies. They focus on novel therapies under investigation in the treatment of acute lymphoblastic...

Myelodysplastic Syndromes

Novel Treatments of Myelodysplastic Syndromes

AS PART of The ASCO Post’s continued coverage of the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition, here is an update on seven different studies on new therapeutics in myelodysplastic syndromes (MDS). Among the treatments highlighted here are the erythroid maturation...

leukemia
myelodysplastic syndromes

Combination Azacitidine and Lenalidomide as Salvage Therapy for Relapsed AML After Allogeneic Stem Cell Transplantation

In a phase I trial reported in the Journal of Clinical Oncology, Craddock et al found evidence that the sequential combination of azacitidine and lenalidomide may be an effective salvage therapy in patients with acute myeloid leukemia (AML) relapsing after allogeneic stem cell transplantation...

leukemia
myelodysplastic syndromes

Study Finds Elevated Risk of MDS and AML After Chemotherapy for Most Solid Tumors

Findings from a new study by researchers at the National Cancer Institute (NCI) show that patients treated with chemotherapy for most solid tumors from 2000 to 2014 experienced an increased risk of therapy-related myelodysplastic syndrome/acute myeloid leukemia (MDS/AML). The study, which used U.S. ...

myelodysplastic syndromes

Mikkael A. Sekeres, MD, on MDS: Results From the Medalist Trial

Mikkael A. Sekeres, MD, of the Cleveland Clinic, discusses phase III study findings on luspatercept to treat anemia in patients with very low-, low-, or intermediate-risk myelodysplastic syndromes with ring sideroblasts who require red blood cell transfusions (Abstract 1).

palliative care
survivorship
myelodysplastic syndromes
leukemia

Suleika Jaouad on Making the Most of a Life Interrupted: A Young Adult Perspective on Cancer

Suleika Jaouad, an Emmy Award–winning writer, advocate, and cancer survivor who was diagnosed at age 22 with myelodysplastic syndrome and acute myeloid leukemia, discusses what she has learned about coping with cancer, learning from it, and growing beyond it.

Hematologic Malignancies
Multiple Myeloma
Lymphoma
Leukemia
Myelodysplastic Syndromes

Selected Abstracts on Novel Therapies for Hematologic Malignancies

THE 22ND CONGRESS of the European Hematology Association (EHA) was held in June in Madrid, drawing hematologists and allied professionals from every subspecialty of hematology from around the world. Among the extensive educational and scientific program, the EHA Congress provides a forum for...

Myelodysplastic Syndromes

High-Risk Mutations Predict Poor Outcomes for Patients With Myelodysplastic Syndromes Undergoing Transplantation

Although several treatment options are available for patients with myelodysplastic syndromes (MDS), hematopoietic stem cell transplantation (HSCT) remains the only curative therapy.1 The risks of complications and death from transplantation can be substantial. Determining which patients may...

Myelodysplastic Syndromes

Mutations in Myelodysplastic Syndrome Associated With Poorer Outcome After Stem Cell Transplantation

In a study reported in The New England Journal of Medicine, R. Coleman Lindsley, MD, PhD, of Dana-Farber Cancer Institute, and colleagues found that a number of mutations present in patients with myelodysplastic syndromes (MDS) were associated with poorer clinical outcome after allogeneic...

hematologic malignancies
leukemia
lymphoma
myelodysplastic syndromes

FDA Allows Marketing of Test to Aid in the Detection of Certain Leukemias and Lymphomas

On June 29, the U.S. Food and Drug Administration (FDA) allowed marketing of ClearLLab Reagents (T1, T2, B1, B2, M), the first agency-authorized test for use with flow cytometry to aid in the detection of several leukemias and lymphomas, including chronic leukemia, acute leukemia, non-Hodgkin...

myelodysplastic syndromes

Reduced-Intensity vs Standard Conditioning Followed by ASCT in Myelodysplastic Syndrome

In the phase III RICMAC trial, use of a dose-reduced vs a standard myeloablative conditioning regimen prior to allogeneic stem cell transplantation (ASCT) did not appear to reduce 2-year relapse-free or overall survival in patients with myelodysplastic syndrome. These findings were reported by...

Myelodysplastic Syndromes

Expert Point of View: Steven D. Gore, MD, and Rami Komrokji, MD

Steven D. Gore, MD, Director of Hematologic Malignancies at Yale School of Medicine, New Haven, Connecticut, applauded Dr. Stein for “accruing a terrible patient population, mutation-wise” and called the preliminary findings for the benefit of enasidenib in patients with ASXL1 mutations...

Myelodysplastic Syndromes

Early Success Reported With Two New Agents for High-Risk Myelodysplastic Syndromes

At the 2016 American Society for Hematology (ASH) Annual Meeting & Exposition, researchers reported early success with two new experimental agents for high-risk myelodysplastic syndromes—enasidenib (also known as AG-221), a potent oral inhibitor of the isocitrate dehydrogenase 2 (IDH2) enzyme,...

hematologic malignancies
myelodysplastic syndromes

Prognostic Scoring System for Patients Undergoing Allogeneic Hematopoietic Cell Transplantation for Myelodysplastic Syndrome

As reported in the Journal of Clinical Oncology, Shaffer et al have developed a prognostic scoring system for patients undergoing allogeneic hematopoietic cell transplantation for myelodysplastic syndrome. Study Details The study involved data from 2,133 patients with myelodysplastic syndrome in...

myelodysplastic syndromes

No Significant Survival Advantage for Rigosertib in High-Risk Myelodysplastic Syndromes, but Subgroups May Benefit

As reported in The Lancet Oncology by Garcia-Manero et al, the phase III ONTIME trial showed that treatment with the Ras mimetic rigosertib did not significantly improve overall survival vs best supportive care in patients with high-risk myelodysplastic syndromes after failure of hypomethylating...

Myelodysplastic Syndromes

Selected Abstracts From 2015 ASH Annual Meeting: Part 2

Here are several more abstracts selected from the proceedings of the 2015 American Society of Hematology (ASH) Annual Meeting and Exposition, highlighting therapeutics in acute leukemias and myelodysplastic syndromes. For five other top abstracts on therapies for acute leukemias and myelodysplastic ...

myelodysplastic syndromes
leukemia

Alan F. List, MD, on Sotatercept in Lower-Risk Myelodysplastic Syndromes

Alan F. List, MD, of Moffitt Cancer Center, offers his thoughts on abstract 3251, “An Open-Label, Phase II, Dose-Finding Study of Sotatercept (ACE-011) in Patients With Low or Intermediate-1–Risk Myelodysplastic Syndromes or Nonproliferative Chronic Myelomonocytic Leukemia and Anemia Requiring Transfusion,” presented by Rami S. Komrokji, MD.

lymphoma
myelodysplastic syndromes

Richard M. Stone, MD, on the SWOG S1117 Study

Richard M. Stone, MD, of Dana-Farber Cancer Institute, offers his thoughts on abstract LBA-5, “A Randomized Phase II Study of Azacitidine Combined With Lenalidomide or With Vorinostat vs Azacitidine Monotherapy in Higher-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia: North American Intergroup Study SWOG S1117,” presented by Mikkael A. Sekeres, MD, MS.

myelodysplastic syndromes

Alan F. List, MD, on the CC-5013-MDS-005 Study

Alan F. List, MD, of Moffitt Cancer Center, offers his thoughts on abstract 409, “Efficacy and Safety of Lenalidomide vs Placebo in RBC Transfusion–Dependent Patients With IPSS Low-/Intermediate-Risk Myelodysplastic Syndromes Without Del(5q) and Unresponsive or Refractory to Erythropoiesis-Stimulating Agents: Results From a Randomized Phase III Study (CC-5013-MDS-005),” presented by Valeria Santini, MD.

myelodysplastic syndromes

Alan F. List, MD, on Rigosertib in Relapsed/Refractory Higher-Risk Myelodysplastic Syndrome

Alan F. List, MD, of Moffitt Cancer Center, offers his thoughts on abstract 163, “Overall Survival and Subgroup Analysis From a Randomized Phase III Study of Intravenous Rigosertib vs Best Supportive Care in Patients With Higher-Risk Myelodysplastic Syndrome After Failure of Hypomethylating Agents,” presented by Guillermo Garcia-Manero, MD. Time: 1:54

myelodysplastic syndromes

Alan F. List, MD, and James O. Armitage, MD, FACP, FRCP, on Advances in Myelodysplastic Syndrome

Alan F. List, MD, of Moffitt Cancer Center and the University of South Florida, and James O. Armitage, MD, FACP, FRCP, of the University of Nebraska Medical Center, discuss the biology and diagnostic criteria for myelodysplastic syndrome, as well as advances in the disease presented at the 56th ASH Annual Meeting.

leukemia
myelodysplastic syndromes

James O. Armitage, MD, FACP, FRCP, and Richard M. Stone, MD, on Clinical Trial Results for AML, MDS, and CMML Treatment

James O. Armitage, MD, FACP, FRCP, of the University of Nebraska Medical Center, and Richard M. Stone, MD of the Dana-Farber Cancer Institute, discuss three clinical trials: different doses of daunorubicin for AML; comparing azacitidine plus lenolidomide to vorinostat vs azacitidine monotherapy in MDS and CMML; and sorafenib vs placebo in addition to standard treatment for AML.

Myelodysplastic Syndromes
Leukemia
Hematologic Malignancies

Myeloablative Conditioning for Stem Cell Transplantation Remains Standard of Care in Patients With MDS and AML

A randomized trial from the Bone and Marrow Transplant Clinical Trials Network was halted early after concluding that allogeneic stem cell transplantation after a reduced-intensity conditioning regimen resulted in higher relapse rates compared to myeloablative conditioning. The phase III randomized ...

Myelodysplastic Syndromes

Ezatiostat Gets Orphan Designation for Treatment of Myelodysplastic Syndrome

Telik, Inc, announced that its product candidate, ezatiostat hydrochloride (Telintra), has been granted orphan drug designation by the FDA for the treatment of myelodysplastic syndromes (MDS). Ezatiostat is an investigational agent in development for the treatment of MDS and idiopathic chronic...

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