Issue: # 38 
May 2014 
In This Issue
Message from DBAC
DBAF Funds New Research
Mother's Day to Father's Day Celebration
Journal Club
Attn: Amazon Shoppers
Like Us and Follow Us

A Message From DBAC


Hello Families,
We would love for all of you to be a part of the first edition, DBA  Family Photography book. This will be a 
10 x 8 coffee table book filled with creative, beautiful, amateur photography. 


Grab your cameras and show us what you've got! We are looking for any tasteful photographs that you have taken and want to share. We are asking for your best shot, including photos of landscapes, skylines, flowers, children, animals, nature,etc. that you have captured and are willing to share in a professionally printed book.


Along with your high quality, high resolution photo, please send  a couple of paragraphs about your DBA journey and a small picture of you or your DBA child to be included in the book. 
We are also accepting submissions from family members, friends, caregivers, researchers/doctors of DBA patients and families. Along with these pictures, photographers are encouraged to include one line with words of encouragement for the DBA patient and family. 


Once completed, this book will be a compilation of all of our work. It will be a beautiful book to look at, as well as telling our DBA stories and sentiments offering encouraging words. 
The books will go on sale and proceeds will benefit supporting DBA research.


For more information, please email
Janet Pereira
Executive Director
DBA Canada 



Upcoming Events

Cleveland HUGS Fundraiser for DBA

August 16, 2014

Wesley Memorial United Methodist Church

Cleveland, TN


Robert Lee


Friends of DBA Golf Outing  September 15, 2014
Fox Meadow Golf Club
Medina, OH 
Jim & Carol Mancuso


Friends of DBA 5K / 1 mile Walk 
October 4, 2014
Fox Meadow Golf Club
Medina, OH 
Jim & Carol Mancuso

Ongoing Fundraisers
Family Letter Writing Campaign  
Pre-printed letters and envelopes have been created for you to send to your contacts! Call or email for more information.
Dawn Baumgardner


Wristbands Available 
Twila Edwards





Tribute Cards Available
(3 Styles)
In honor of...
In memory of...
Holiday giving...
Dawn Baumgardner 
  donation donation
5" x 5" Decals Available
Dawn Baumgardner 
  window sticker

7" x 5" Decals Available
David Voltz
Cure DBA decal_Voltz.  
Cookbooks Available  
Betty Lightner  
To order online, visit:
cookbook cover  


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DBAF Funds New Research Initiatives 
Award Given to Dr. Daniel Finley

The Diamond Blackfan Anemia Foundation (DBAF) is pleased to announce the funding of another exciting research project. A grant for $35,000 was awarded to Daniel Finley, Ph.D. for his project entitled, "A new mode of ribosome regulation specific to reticulocytes."

Dr. Finley, Anthony Nguyen, Dr. Mark Fleming

Dr. Daniel Finley, a well-established investigator and a professor at Harvard University, is new to the DBA field.  He will be collaborating with Mark Fleming, MD, DPhil of Boston Children's Hospital. Dr. Finley works on a system by which proteins are targeted for destruction within cells.  His interest in DBA stems from recent work in his lab where he identified a gene that targets ribosomal proteins for destruction in maturing reticulocytes.  This is a highly original project that is very likely to provide important insight into the regulation of ribosome stability in erythroid precursors, which will be of major interest for DBA research. In addition, it could potentially unravel a new therapeutic avenue for treating DBA.

Dr. Finley, Dr. Fleming, and Anthony Nguyen stated, We wish to thank the DBAF for its support of our work on red blood cell differentiation. Over the years, we have had the opportunity to study a number of genetic mutant strains of mice with anemia. Unexpectedly, one of these anemic mouse strains carried a mutation in a gene that marks proteins to be degraded. We found that these animals have exceptionally high ribosome levels due to a failure of ribosome elimination, which normally occurs in red blood cells as they mature. This is the opposite of DBA, which is most often due to a defect in ribosome production. An unusual mutant like this can allow us to think about the problem of DBA in a new way, and has the potential to provide significant insights into the disease. With funding from the DBAF, we will be able to test whether inhibiting this protein, and thus preserving ribosomes, could alleviate the anemia seen in DBA."

Mother's Day to Father's Day Celebration
Check Out Our Progress
Once you read our Research Director's article this month, you will understand our need to encourage families to donate, fundraise, and help spread the word.  In an article below, Steve Ellis, PhD, explains a recent research proposal that we have committed to funding. In addition to this very important $95,000 project, the DBAF has also committed to a $36,000 project that you will read about next month, and there are three more projects out for scientific review. We NEED your help!

There are many ways to donate! Right now, we are trying to meet a goal for a Mother's Day to Father's Day Celebration Campaign.  You can follow our progress and add your name to the list of donors willing to support DBA research by visiting our online fundraising page

While we are proud of our accomplishments, we realize we must continue to work diligently to fulfill our mission. We need your help. 
Please consider partnering with us to help find a cure for DBA. Thank you for your careful consideration. 
Journal Club


First there is a mountain

then there is no mountain

then there is


Steve Ellis
Steven R. Ellis, PhD
DBAF Research Director

Anyone exiting the elevator on the 5th floor of the research tower at the University of Louisville Health Science Center is likely to be greeted by music wafting down the hall from my office. Day, night, weekend, if I am here, the music is on and invariably that music is late 60's early 70's, music from my formative years. Not classic rock, mind you, as listening to top 40 classic rock day in and day out is my vision of purgatory. No, instead, Deep Tracks from Sirius/XM. I'll leave for you to wonder what it says about me that I consider underplayed psychedelic/acid rock the underpinning of my development, but for now I would like to turn your attention back to Donovan's musings.


First there is a mountain,

then there is no mountain,

then there is


What could these lyrics possibly have to do with Diamond Blackfan Anemia?  It's just that I can't help but think of these lyrics whenever I am confronted with what has become known to the Board of Directors of the DBA Foundation as, "The Lodish situation."


First Dr. Lodish was funded to perform research bearing on finding more effective treatments for DBA,

then he wasn't,

now he is


See what I mean?


Let us turn to an investigator spotlight. Dr. Harvey Lodish, a professor at the Whitehead Institute at the Massachusetts Institute of Technology. A man considered by this reporter (and many others I suspect) a superstar in the field of biomedical sciences. Dr. Lodish has had a long and illustrious career performing seminal research in many different areas including erythropoiesis.  He was among the founders of a multibillion-dollar biotech company that has brought orphan drugs to market for the treatment of rare diseases and co-authored a highly regarded textbook, Molecular and Cellular Biology


A few years back, Marie Arturi recruited Dr. Lodish to the DBA field by inviting him to one of the DBA ICC meetings hosted by the Daniella Maria Arturi Foundation. In addition to his work on erythropoiesis, I felt Dr. Lodish was ideally suited to bring new insights to the DBA field based on a paper he published back in the 70's suggesting how reduced amounts of something as critical to protein synthesis as a ribosomal subunit could alter the amounts and types of proteins within a cell. As the effects of ribosomal protein haploinsufficiency on the translational output of a developing red blood cell was, and still is, a viable model for the clinical features of DBA, his insights into disease pathophysiology could be invaluable. 


First Dr. Lodish was funded to perform research bearing on finding more effective treatments for DBA


Dr. Lodish has remained in the field since this original meeting and in the last year or two has begun a screen for new drugs that can be used for treating DBA. He began this screen by looking for drugs that stimulated erythropoiesis at or near the point that steroids have their effect in stimulating red cell production. The rationale here being that such drugs may work through a mechanism similar to steroids without their side effects.  Alternatively, these drugs could work along with steroids increasing their effectiveness at lower doses, potentially reducing steroid toxicities. It is reasonable to ask whether the drugs that come out of this screen will only work with patients who respond to steroids. In fact, Dr. Lodish was asked this question when presenting his research at the 2014 ICC conference.  His view was that while the design of the screen appears directed at steroid responsiveness, there is no reason to believe that these drugs couldn't also work for patients who are transfusion-dependent (I realize this sentence includes a double negative, so read: drugs coming from this screen could also possibly work for patients who are transfusion dependent).  


Dr. Lodish's studies were supported by the U.S. Army Medical Research and Material Command's Telemedicine & Advanced Technology Research Center. As noted in the April DBAF enewsletter, because of difficulties in the Department of Defense budget, the DBA Foundation needed to step in to provide $21,281 in stopgap funding so the Lodish laboratory could complete the first of three years of funding through the DOD program. This stopgap funding was considered a temporary fix until the DOD worked through its budgetary issues and resumed funding.


then he wasn't


Dr. Lodish has recently been notified that the Telemedicine & Advanced Technology Research Center program has been reorganized and discontinued due to lack of available funding from Congress, leaving him with no funds to continue this important research. Without these funds research would halt at a critical juncture where the Lodish group has identified a set of drugs that appear to work synergistically with steroids in stimulating erythropoiesis in mice. Importantly, these drugs are already used for other purposes in humans. Thus, the toxicities and side effects of these drugs have been well studied, which may streamline the process of devising and initiating clinical trials for DBA patients. A caveat to this upbeat assessment is that Dr. Lodish's work has so far been in mice and before transitioning to human trials these studies need to be repeated in human cellular models of DBA, which is where their studies were going in year two and three of the DOD grant. With the DOD program reorganized and funding stopped, this research was jeopardy of being halted.      


now he is


At Dr. Lodish's request, the Board of Directors of the DBA Foundation unanimously voted to step in again for the Department of Defense and provide continued funding for this important research. The one-year award is for $95,000, somewhat less that the grant awarded by the Department of Defense, but it fully covers personnel and animal costs allowing the research to continue. 


The decision to support this research was not made easily. The amount of the award is approximately 3 to 4 times the average award made in recent years by the DBA Foundation. Other proposals currently under consideration by the DBAF were either declined for funding or placed on hold to make room for this large outlay. The Board realizes that it is taking a significant risk in providing such a large outlay in the current fiscal environment, but does so in the hopes of high rewards in terms of improved care for DBA patients.


and back to Donovan...


"Oh Juanita, Oh Juanita, I call your name"


It is important that the lay DBA community realize that the paralysis in Washington has led to substantial cuts in all areas of research with many researchers in the DBA field finding it extremely difficult to find Federal support for their work.  This situation has caused at least one promising young investigator to leave the field due to funding restrictions.  The dearth of funding at the Federal level has created a vacuum that the DBAF is being increasing asked to fill.  Your efforts are essential for keeping research on DBA moving forward during these difficult times and they are sincerely appreciated by the research community.  

Amazon Shoppers... This One's For You!
Donate while you shop
The Diamond Blackfan Anemia Foundation 

is participating in the AmazonSmile program. AmazonSmile offers the same wide selection of products, low prices, and convenient shopping features as The important difference is that when customers shop through AmazonSmile (, the AmazonSmile Foundation donates 0.5% of the price of eligible purchases to the charity of your choice!
It simple to do and will not add to the price of your purchase. Visit, select Diamond Blackfan Anemia Foundation as your charity, and let the shopping begin! You can use your existing Amazon account on AmazonSmile. Once you select the Diamond Blackfan Anemia Foundation as your charity, all your shopping visits will benefit the DBAF! It's free and easy. Start your Amazon shopping on AmazonSmile today!  

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