Newsstand | Page 12 | Aplastic Anemia & MDS International Foundation

Here's where you'll find a regularly updated, broad range of articles written by the AAMDSIF team, allied health organizations and news organizations. By staying well-informed, patients and families are practicing a form of self-support that will help them be more effective self-advocates when engaging with health care providers.


FDA approves new drug for relapsed or refractory AML

Originally Published: 08/30/2017
On August 1, 2017, the U.S. Food and Drug Administration granted regular approval to enasidenib (IDHIFA, Celgene Corp.) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test. This is the first FDA approval for relapsed or refractory AML specifically with an IDH2 mutation. The FDA concurrently approved a companion diagnostic, the RealTime IDH2 Assay, used to detect the IDH2 mutation. The enasidenib approval was based on Study AG221-C-001 (NCT01915498), an open-label, single-...

New treatment approved for certain high-risk AML

Originally Published: 08/30/2017
The U.S. Food and Drug Administration today approved Vyxeos for the treatment of adults with two types of acute myeloid leukemia (AML): newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Vyxeos is a fixed-combination of chemotherapy drugs daunorubicin and cytarabine. “This is the first approved treatment specifically for patients with certain types of high-risk AML,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug...

Marrow protein may be key to SCT success

Originally Published: 08/29/2017
Bone marrow contains hematopoetic stem cells, the precursors to every blood cell type. These cells spring into action following bone marrow transplants, bone marrow injury and during systemic infection, creating new blood cells, including immune cells, in a process known as hematopoiesis. A new study led by University of Pennsylvania and Technical University of Dresden scientists has identified an important regulator of this process, a protein called Del-1. Targeting it, the researchers noted, could be an effective way to improve stem cell transplants for both donors and recipients. There...

MDS Update - August 2017

Originally Published: 08/29/2017
MDS Update - August 2017

FDA approves first drug to treat GVHD

Originally Published: 08/24/2017
A drug currently used to treat several forms of blood cancer, ibrutinib (Imbruvica®), has been approved by the Food and Drug Administration (FDA) for the treatment of chronic graft-versus-host disease (cGVHD). The agency’s decision, announced on August 2, makes ibrutinib the first approved therapy for this potentially fatal and common side effect of cancer-related stem cell transplants. The approval covers the use of ibrutinib in patients with cGVHD that is not responding to other standard treatments, namely corticosteroids. Ibrutinib is already approved by FDA for the treatment of several...

Top honor goes to AAMDSIF Medical Advisor

Originally Published: 08/24/2017
The American Society of Hematology (ASH) will present the 2017 William Dameshek Prize to Benjamin L. Ebert, MD, PhD, of Harvard Medical School, Brigham and Women’s Hospital, and Dana-Farber Cancer Institute for his seminal discoveries in multiple areas of non-malignant and malignant hematology. The William Dameshek Prize is awarded to an early- or mid-career hematologist who has made a recent outstanding contribution to the field of hematology. This prize is named after the late William Dameshek, MD, a past president of ASH and the original editor of Blood. ASH President Kenneth C. Anderson...

New study on SCT therapy for SAA, MDS patients

Originally Published: 08/21/2017
Gamida Cell announced today that an investigator-initiated study testing their candidate CordIn, to treat severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS) has begun, and the first patient was given the drug. Severe AA is a condition in which the bone marrow does not produce enough blood cells for the body, and hypoplastic MDS is characterized by decreased marrow cellularity. Cordin is a variety of stem cell transplantation that uses blood from an umbilical cord. It is a curative treatment in development for an array of rare diseases, including sickle cell disease,...

Making Progress for Myelodysplastic Syndromes

Originally Published: 07/26/2017
Myelodysplastic syndromes (MDS) are rare, which can make them difficult to study. The biggest challenge is finding enough patients to enroll in clinical trials that could help to advance treatment. But the Aplastic Anemia and Myelodysplastic Syndrome International Foundation (AAMDSIF), a nonprofit organization, wants to change this by providing patients and patient data to support research. One of its efforts in this area is the MDS Clinical Research Consortium. In an interview with CURE, Rami Komrokji, M.D., a hematologist at Moffitt Cancer Center in Tampa, Florida, discusses the advances...

Promising news for older AML patients

Originally Published: 07/19/2017
A combination of the BCL-2 inhibitor venetoclax with a hypomethylating agent may produce high overall response rates among older patients with newly diagnosed acute myeloid leukemia (AML), early data showed. Among 100 patients age 65 years and older with previously untreated AML, the combination of venetoclax with either decitabine or azacitidine was associated with a 69% overall responses rate, reported Keith W. Pratz, MD, of Johns Hopkins University in Baltimore. “Venetoclax, when combined with decitabine or azacitidine, has a tolerable safety profile in treatment-naive elderly patients...

Filling Unmet Need in MDS Treatment

Originally Published: 07/10/2017
Rami S. Komrokji, M.D., clinical director of Hematologic Malignancies at Moffitt Cancer Center, discusses an unmet need in the treatment of myelodysplastic syndrome (MDS). While some agents have made progress in the treatment of MDS, stem cell transplant is still the only curative method, and patients need more options. But now that researchers have spent the last few years better understanding the biology of the disease, there are some promising trials and treatments in the pipeline, Komrokji says.