http://www.raredr.com/conferences/nord2017/aamds
Aplastic Anemia & MDS International Foundation Provides Funds for Young Researchers

Barbara Hozler

Aplastic anemia and myelodysplastic syndrome (MDS) are rare disorders related to the bone marrow’s inability to properly produce blood cells.

At the National Organization for Rare Disorders (NORD) Rare Disease & Breakthrough Summit in Washington D.C., Rare Disease Report spoke with Barbara Holzer, Communications Director of the Aplastic Anemia & MDS International Foundation about the foundation and how it helps sponsor research to eventually find treatments for these blood disorders. 



Rare Disease Report: How does the Aplastic Anemia & MDS International Foundation help patients?

Barbara Holzer: We serve the community of patients who are suffering with bone marrow failure disease. When your bone marrow stops creating cells—white blood cells, red blood cells, or platelets—your body doesn't get the blood it needs to support a strong and healthy life. While people do live with bone marrow failure disease it is a tough one to conquer. There is no cure, per se, and often bone marrow failure requires the transplant. And finding a transplant donor can be hard very hard to find for certain people, especially people with multiracial backgrounds.

Also, people of a certain age generally aren't strong enough for a transplant and it comes with its own variety of risks. So, it's a tough disease and no one's immune to it. There's no profile for someone who gets a bone marrow failure disease and it's by and large, it is an acquired diseae so it's not as if you inherit it. You might be exposed to pesticides or benzene or some chemical that shouldn't be around you and that might have triggered it, but science hasn't figured it out yet.

RDR: What is the current state of the research?

Holzer: The problem with rare diseases in general, and with bone marrow failure in particular, is that fewer people have these diseases so it's extremely hard to get federal funding for the research that's necessary to improve treatments, work towards a cure, and eventually make the kind of incremental progress that one day will turn out to be a cure, The diseases should be manageable one day like other diseases that have been conquered but it's not yet.

One of the things we do at our foundation is support research by giving grants every year to young researchers who are working on certain as-yet unstudied aspects of bone marrow failure. And to that end, we also have a scientific symposium for an international audience of hematologist and oncologist who are expert in this area. We're constantly trying to bring together the great minds in science to pool their resources to learn from each other and come up with the kind of solution that will lead to an overarching cure and a manageable disease.

RDR: How can researchers learn more about those grants?

Holzer: Young scientists who are interested in bone marrow failure have the opportunity to apply for a grant at the foundation. We have a large Medical Advisory Board comprised of experts in the fields of hematology and oncology. They review these applications and pick the most unique among them from the highest ranking students and we provide two-year grants so they can work on their particular project.  I'm so proud to say that oftentimes a couple of years down the road after these grantees have done their work, they're showing up at our scientific symposium and presenting at it because they've developed a niche of their own and they have something to provide to the greater scientific community.

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