Newsstand | Page 3 | Aplastic Anemia and 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.

More promising news on CAR-T

Originally Published: 10/12/2017
Learn how our own immune system cells can be engineered to fight some cancers. 

New AML drugs to accelerate genetic testing

Originally Published: 09/25/2017
In this final issue of our brief 3-part tour of new clinical trial findings and treatment strategies in hematologic cancers, we visit Dr David Steensma of the Dana-Farber Cancer Institute for his take on some of the most important recent data sets in myeloid cancers. While my discussion with Dr Steensma focused on a number of topics, his review of research in acute myeloid leukemia (AML) is perhaps most relevant because after many years of disappointment, clinical development and new drug approvals in this disease are now exploding.

Senate floats another red-herring health bill

Originally Published: 09/19/2017
The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement opposing the “Graham-Cassidy” plan to repeal and replace the Affordable Care Act (ACA): “The Senate is currently considering a proposal put forward by Senators Graham, Cassidy, Heller, and Johnson that would repeal and replace the Affordable Care Act (ACA). If passed, this plan (known as ‘Graham-Cassidy’) has the potential to jeopardize access to care for millions of individuals with rare diseases. Once...

Foundation Update - September 2017

Originally Published: 09/12/2017
Foundation Update Newsletter - September 2017 New Drug Approved to Treat cGVHD Last month the Federal Drug Administration (FDA) approved an existing drug, ibrutinib (Imbruvica®) for treatment for chronic graft vs. host disease (cGVHD), a serious complication that can occur with cancer-related stem cell transplants. Ibrutinib may now be used in patients who fail to respond to established treatments for cGVHD. Already approved for use in certain types of lymphomas and one type of leukemia, ibrutinib's approval came after a randomized clinical trial in which...

Explaining SCT vs. Cellular Therapies

Originally Published: 09/08/2017
Donor stem cell transplants and other cellular therapies are treatment approaches that harness the immune system to fight cancer using cells from the patient or from healthy donors. Stem cell transplants are used to treat blood-related cancers such as leukemia, lymphoma, and multiple myeloma, as well as certain non-cancerous blood disorders. Patients first receive chemotherapy and/or radiation therapy to erase or reduce the number of cells in their bone marrow, home to the hematopoietic (blood system) stem cells that give rise to all other types of blood cells. They then receive an infusion...

Promising trial results for lower-risk MDS drug

Originally Published: 09/06/2017
Myelodysplastic syndromes are characterised by ineffective erythropoiesis. Luspatercept (ACE-536) is a novel fusion protein that blocks transforming growth factor beta (TGF β) superfamily inhibitors of erythropoiesis, giving rise to a promising new investigative therapy. We aimed to assess the safety and efficacy of luspatercept in patients with anaemia due to lower-risk myelodysplastic syndromes.

New study on how red blood cells mature

Originally Published: 09/05/2017
Every cell in the body, whether skin or muscle or brain, starts out as a generic cell that acquires its unique characteristics after undergoing a process of specialization. Nowhere is this process more dramatic than it is in red blood cells. In order to make as much room as possible for the oxygen-carrying protein hemoglobin, pretty much everything else inside these precursor red blood cells—nucleus, mitochondria, ribosomes and more—gets purged. Jam-packing red blood cells with hemoglobin is essential. Doing so ensures that all the body’s tissues and organs are well nourished with oxygen to...

Study suggests heart drugs may boost chemotherapy in AML

Originally Published: 09/05/2017
Scientists may have identified a way to boost the effect of chemotherapy against one of the most common forms of leukemia in adults: acute myeloid leukemia. In a new study, researchers found that acute myeloid leukemia (AML) triggers blood vessel leakage in bone marrow, which prevents chemotherapy drugs from being effectively delivered to leukemia cells. However, by using drugs normally used to treat heart and blood vessel disease, the researchers found that they were able to prevent this blood leakage and increase the efficacy of chemotherapy. First study author Diana Passaro, of the...

Allogeneic transplant: does age still matter?

Originally Published: 08/31/2017
In this issue of Blood, on behalf of the Center for International Blood and Marrow Transplant Research (CIBMTR), Muffly et al report on the incidence and outcome of allogeneic stem cell transplantations (alloSCTs) performed between 2000 and 2013 in 1106 patients aged ≥70 years. The incidence of alloSCT in this age group accounted for 0.1% of all stem cell transplantations (SCTs) reported to the CIBMTR in 2001, and it rose to 3.85% by 2013. Comparison of 2 time periods of performing SCTs, 2000 to 2007 and 2008 to 2013, revealed significant improvement in overall survival (OS) and progression-...

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-...