Newsstand | Page 8 | 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.


What the Future May Hold

Originally Published: 10/24/2017
Dr. Jason Butler and his team discovered that aged blood vessels strongly influence the functional characteristics of blood stem cells.  Transplantation of young blood vessel cells into older mice makes their aged stem cells take on the characteristics of young stem cells. This image shows a blood-vessel defect that occurs as a result of aging: the increased presence of reactive oxygen species (magenta) in aged bone marrow-derived blood vessels (depicted in red and green with blue nuclei). The excess buildup of reactive oxygen species can interfere with the ability of blood vessels to...

Repurposing older drugs to help prevent GVHD

Originally Published: 10/18/2017
The oral histone deacetylase (HDAC) inhibitor (vorinostat) is safe and results in low incidence of acute graft-versus-host disease (GVHD) after reduced-intensity conditioning, related donor hematopoietic cell transplantation (HCT). However, its safety and efficacy in preventing acute GVHD in settings of heightened clinical risk that use myeloablative conditioning, unrelated donor (URD), and methotrexate are not known. We conducted a prospective, phase 2 study in this higher-risk setting. We enrolled 37 patients to provide 80% power to detect a significant difference in grade 2 to 4 acute...

Foundation Update - October 2017

Originally Published: 10/12/2017
Foundation Update Newsletter - October 2017 Confronting Challenges in Finding Matches for Multiracial Patients 34-year-old Krissy Kobata is a passionate patient and advocate/activist. Diagnosed in 2008 with MDS, she’s the epitome of resilience as she has waited for years to find a matched donor. She has devoted this time to helping other patients by enlarging the Be The Match® bone marrow registry. She’s directed numerous bone marrow drives and fundraisers, bringing thousands of new DNA donors to the registry. However, her own path to finding a donor has been challenging and frustrating....

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...