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 Issue: # 36 
March 2014 
In This Issue

We NEED and appreciate YOUR monthly Commitment to a Cure

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Over the past two years, the DBAF has reached out to our families asking for their support. Thank you to the families that have responded with their personal donations, and thank you to the families that have made a monthly pledge.  We are so pleased that more and more families are making a monthly commitment to the DBAF. From $20/month to $200/month, these monthly donations are easy to set up through your bank or through our website using a credit card or PayPal. Of course, a monthly check can also be mailed to the DBAF. Please join the many other families that have made this commitment.

 

Your generous support is appreciated and will help all DBA families. Please visit our website for donation information. 

http://dbafoundation.org/donate/

Since 1994, your donations and fundraising efforts have allowed the Diamond Blackfan Anemia Foundation (DBAF) to provide funding for major research projects that have resulted in the establishment of the DBA National Registry (DBAR), the discovery of defective "DBA genes," a better understanding of DBA on both the molecular and genetic levels, advances in creating animal models, novel approaches to less toxic treatments, and promising advances towards successful therapeutic cures. We have sponsored, supported and cosponsored international scientific meetings, have sponsored national family conferences and retreats, and have provided our families with informational materials and support. 

 

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. I am happy to answer any questions you may have regarding fundraising efforts or the DBAF.  

 

Thank you for your careful consideration. Please commit to find a cure!

Sincerely,
Dawn Baumgardner
Executive Director
716.674.2818 

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Upcoming Events

  

DBA Bowling Bash
April 5, 2014
Benton, AR
Contact:
Rachel Ruff
ruff22282j@gmail.com  

 

Annual Strike Out DBA 
Bowling Fundraiser
April 26, 2014
Grand Haven, MI
Contact:
Tammi Lanore
dbafundraiser@gmail.com

 

Cleveland HUGS Fundraiser for DBA

August 16, 2014

Wesley Memorial United Methodist Church

Cleveland, TN

Contact:

Robert Lee
dinklee1@yahoo.com

  

Friends of DBA Golf Outing  September 15, 2014
Fox Meadow Golf Club
Medina, OH 
Contact: 
Jim & Carol Mancuso 
c-mancuso@sbcglobal.net

 

Friends of DBA 5K / 1 mile Walk 
October 4, 2014
Fox Meadow Golf Club
Medina, OH 
Contact: 
Jim & Carol Mancuso 
c-mancuso@sbcglobal.net

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.
Contact: 
Dawn Baumgardner
dbaumgardner@dbafoundation.org
716.674.2818  
  

 

Wristbands Available 
Contact:  
Twila Edwards  
twilak@cox.net
 
 
 

 

 

 

 

Tribute Cards Available
(3 Styles)
In honor of...
In memory of...
Holiday giving...
Contact:
Dawn Baumgardner
dbaumgardner@dbafoundation.org 
716.674.2818
  donation donation
 
 
5" x 5" Decals Available
Contact:  
Dawn Baumgardner  
dbaumgardner@dbafoundation.org 
716.674.2818
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7" x 5" Decals Available
Contact:  
David Voltz  
dcvol@yahoo.com
Cure DBA decal_Voltz.  
 
Cookbooks Available  
Contact:
Betty Lightner  
betty.lightner@gmail.com  
To order online, visit:
cookbook cover  

 

AmazonSmile Program
You Shop... Amazon Gives!
If you shop on Amazon, please log in using smile.amazon.com, select Diamond Blackfan Anemia Foundation as your charity, and shop as usual. Amazon will donate .05% of your purchase to the DBAF.
 
 
 
Good Search/Good Shop  
Raise money for DBAF 
just by searching the web and shopping online!   
  

 

Quick Links
The Diamond Blackfan Anemia Foundation (DBAF) is committed to keeping you updated and connected to the entire DBA community. The DBA Foundation is YOUR Foundation!  We encourage you to share your ideas, photos, and stories for our website and upcoming newsletters.  Contact us at DBAFoundation@juno.com.
A Huge Success!
DBAF warmly welcomed DBA researchers and doctors from all over the world.

The Diamond Blackfan Anemia Foundation (DBAF)  was pleased to host the 13th DBA International Consensus Conference (ICC). We are proud and honored to continue this important conference founded and previously hosted by the Daniella Maria Arturi Foundation. The goals of the ICC are to facilitate international exchange and to encourage cooperation in research investigations for DBA. Click to view the meeting agenda.

 

Summary of the ICC provided by the DBAF's Research Director, Steven R. Ellis, Ph.D.

The 13th Diamond Blackfan Anemia International Consensus Conference was held March 8th through the 10th at the Ritz-Carlton Hotel in downtown Atlanta, Georgia.  This year's conference saw a record number of abstracts accepted for presentation as either short talks or posters.  This increased number of abstracts  submitted relative to previous years in part reflects an increasing number of investigators entering the DBA field.  This, in turn, is a reflection of the interest in DBA generated on many fronts by the remarkable progress in the field over the past 10-15 years.

  

The desire to give as many investigators as possible the opportunity to present their work within a very short time frame made for a very jam-packed agenda.  Fortunately, various session moderators and facilitators kept a tight reign over their different sessions and the meeting progressed without a hitch.  While I may seem a bit obsessed with the meeting's logistical issues, efficiently packing all this content into a day and a half was a major factor the organizers had to contend with in setting up the meeting's agenda.  As a way of limiting redundancies in meeting presentations, Jeff Lipton (New York) started the meeting off by giving an overview of DBA from both a clinical and scientific perspective.

  

Dr. Lipton's talk emphasized the limitations of current therapies for DBA (transfusions, steroids, and bone marrow transplants) and the need for improved therapies with fewer complications.  This emphasis tied in nicely with Dawn Baumgardner's (Buffalo) opening remarks stating that an overriding goal of this and past ICC meetings has been to identify therapies and strategies to improve the quality of life for patients and families with DBA. 

  

Current Clinical Trials (New Mechanistic Insights)

In this light, it is worth mentioning the 3 different clinical trials (Leucine, Lenalidomide, and Sotatercept) currently underway in DBA patients.  One of the more interesting aspects of this year's meeting concerns the drug Sotatercept.  This drug was initially developed by Acceleron Pharma as a potential treatment for osteoporosis, but in clinical trials for this condition, it was found that individuals receiving the drug showed a significant increase in red blood cell production.  This increase could be considered an adverse side effect in individuals with osteoporosis, but as a consequence of this serendipitous finding, Sotatercept is now being tested as a treatment for different types of anemia, including DBA.  A talk by Jingping Ge (Philadelphia) provided a potential mechanistic rationale for why this drug may be efficacious in DBA.  Her work on induced pluripotent stem cells (IPSC) from DBA patients revealed that the same signaling pathway targeted by Sotatercept appears to be dysregulated in DBA patients.  This observation provides a more solid scientific basis for the Sotatercept trail (in terms of full disclosure, I have no financial ties to either Acceleron Pharma or Celgene which is supporting the trial).  The Sotatercept-DBA relationship was further reinforced by studies from Shuo Lin (Los Angeles) showing that the generic form of this drug rescues the DBA-like phenotype in zebrafish.  While the Sotatercept clinical trial is still in early stages, these results certainly generated guarded optimism for a positive outcome.

  

The New Drug Pipeline (Turning the Spigot On)

In addition to the drugs currently in clinical trials for DBA, one of the most remarkable features of this year's meeting was the number of drugs entering the pipeline as potential therapies for DBA. Talks by Hsiang-Ying Lee and Harvey Lodish (working together in Boston) described screens for drugs that

CONTINUE READING ARTICLE HERE
THANK YOU ICC SPONSORS!!!
The Diamond Blackfan Anemia Foundation is grateful to all those that supported the 13th Diamond Blackfan Anemia International Consensus Conference. These organizations and family sponsorships contributed to the success of the conference. Thank you!!

 

Premier Sponsor   

Diamond Blackfan Anemia Canada  

Diamond Sponsor   

Daniella Maria Arturi Foundation  

Conference Sponsors  

Captain Courageous (Australia)

DBA-UK

 

Contributing Sponsors:

Meeting Session Sponsor

Josephine & Shiufun Cheung

Event Collateral

Good Dog, Good Works by Mopdog

AV equipment

D'Amico & Sinatra Families  

Meeting booklets and supplies

Mike & Tina Singhas & family  

Young DBA investigator Sponsors

Dennis Beck & Elaine Marchese 

Kinny's Kause

Peyton's Pals, Inc.

Barbara Shore

Varughese Family (3)

Andreas Wagner Family

Coffee breaks

Marilyn & Ernest Carnicelli

Brad & Bonnie Koch

Anthony & Liz Marchese

Welcome Bags

Devere & Jane Fair

Internet

Peyton's Pals, Inc.

Travel scholarships

Falivene - Maso Family

Lori & Vance Franks

Dana & Cipriana Harrison

Rich and Sherry Kofron in honor of our daughter, Kendra (2)

Awards

James & Lindsay Garrett    

Registration

Peter & Dawn Baumgardner & family

Name tags

Casey Leonard in memory of Lakshmi Desai

Incidentals & Supplies

Tim & Kathi Vroman & family

Kick-off Dinner Plate Sponsors

Todd D. Cavalcanto
Jeff & Donna Childs
Michael & Amy Davieau
Mom Mom & Pop Pop Fontana in honor of Olivia Rose & Sophia Grace (2)
Friends of DBA (2)

Together We Can Find a Cure... Russ and Dot Grady
Richard & Madeleine Lapierre in memory of Mark Lapierre 10/18/70 - 3/14/13
Martineck Family
Messenger Family (UK)
Pilat Family in honor of the Baumgardner Family
John and Sheila Reiner
Anita Shier Bruton

 Show Us Your Logo

Our friends at Team Irelynn are hard at work raising funds to support DBA research. Please take a moment to check out this awesome t-shirt and all the exciting events they have planned! The t-shirt (with both our logos) and a chance to win an oceanfront vacation in Fort Walton Beach, FL are just a few examples of what they are working on. Thanks Nick & Melissa Rose for supporting the DBA Foundation! 


Here's the challenge:
 

We would like to see how many places we can show off our logo!

logoSnap a picture sporting our logo and send us your story. Draw it, print it out, wear it, wave it, tattoo it, carve it, stick it... be creative! Take us to school, on vacation, to the hospital, on a plane, to the game, in your home... anywhere!  Show us your logo!  Send your photos and stories to DBAFoundation@juno.com.

 

IMPORTANT STUDY - DON'T MISS THIS OPPORTUNITY

 

The DBAR in collaboration with the Pediatric and Adult Endocrinology at Cohen Children's Medical Center and North Shore LIJ Health System, respectively, did a review of 12 patients who had an endocrine evaluation when they visited our institution. Most of these patients were adults and most were on chronic blood transfusion therapy. Importantly, over half were found to have endocrine issues including low male/female hormones, low thyroid function, Vitamin D deficiency, diabetes and growth hormone deficiency. Two of these patients were diagnosed with their endocrine issues before age 18. For this reason, the DBAR has started the study described below. Please participate. If you have already spoken to Dr. Lahoti, please get the blood work done and send the results to the DBAR or to him. If you want to participate, please call the numbers below. If you don't want to participate in the study, please get the bloods done anyway and have your doctor look for the endocrine issues - get on top of these NOW. Don't wait until a problem happens!! It is always best to prevent than to treat!! This study will close to new patients by March and all results will need to be in by April so they can be analyzed. Dr. Lahoti will be leaving in June as his training will be completed.

 

The Diamond Blackfan Anemia Registry (DBAR) has noted that these and other patients with DBA have issues that may be due to the hormone-producing gland (endocrine) system abnormalities, including short stature, thyroid dysfunction, delayed puberty in both boys and girls, and diabetes. We are interested in determining if these problems are due to long-term continuous steroid use, iron overload from transfusion therapy or from DBA itself. We are therefore recommending an endocrine evaluation for all DBA patients, those with endocrine issues and those without. We are hoping to assist those with endocrine abnormalities as well as document what they are. We are also gathering information on patients without endocrine problems to see if we can screen for them, predict them in advance, and possibly treat them before they occur.

CONTINUE READING HERE
Journal Club

ICC Review Highlight Article in this e-newsletter

Steve Ellis
Steven R. Ellis, PhD
DBAF Research Director

 

Many many thanks to Steve Ellis for his continued dedication to Diamond Blackfan Anemia research, the DBA Foundation, and our patients and families.  We couldn't do what we do without his hard work, guidance, and constant support. 

 

The ICC summary provided by Steve in this e-newsletter will replace this month's Journal Club article. Watch this spot in the next edition of our e-newsletter for Steve's journal article review!

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