[Congressional Record (Bound Edition), Volume 153 (2007), Part 19]
[House]
[Pages 27155-27157]
[From the U.S. Government Publishing Office, www.gpo.gov]




 EXPRESSING THE SENSE OF THE HOUSE OF REPRESENTATIVES WITH RESPECT TO 
                        DIAMOND-BLACKFAN ANEMIA

  Ms. BALDWIN. Madam Speaker, I move to suspend the rules and agree to 
the resolution (H. Res. 524) expressing the sense of the House of 
Representatives with respect to Diamond-Blackfan Anemia, as amended.
  The Clerk read the title of the resolution.
  The text of the resolution is as follows:

                              H. Res. 524

       Whereas Diamond-Blackfan Anemia (``DBA'') is a rare genetic 
     bone marrow failure disorder affecting children and adults, 
     90 percent of whom are younger than 1 year of age when they 
     are diagnosed, and results in severe anemia due to failure to 
     produce red blood cells;
       Whereas individuals and families suffering with rare 
     diseases such as DBA not only face the challenges of their 
     debilitating and life-threatening diseases, but must also 
     confront the consequences of their rare disease status;
       Whereas individuals suffering from rare diseases need 
     access to treatment options and the potential for a cure;
       Whereas research is proving the study of complex, rare 
     diseases such as DBA yield tremendous advancements in other, 
     larger disease areas that affect millions of Americans;
       Whereas the children living with DBA have an increased risk 
     of leukemia, solid tumors, and complete bone marrow failure, 
     and 50 percent of patients with DBA are born with birth 
     defects including abnormalities to the face, head, upper arm 
     and hand, genitourinary, and heart with 21 percent of 
     affected patients having more than 1 defect;
       Whereas the study of DBA will yield the true incidence of 
     aplastic anemia, myelodysplastic syndrome, leukemia, and the 
     predisposition to cancer in DBA and will serve as an 
     important model for understanding the genetics of birth 
     defects;
       Whereas treatments for DBA, including the use of steroids 
     (such as prednisone) and blood transfusions, have potential 
     long-term side effects, including osteoporosis, impaired 
     growth because of the steroids, diabetes, and iron overload 
     because of the transfusions;
       Whereas the only cure for DBA is a bone marrow transplant, 
     a procedure that carries serious risks and, since most 
     patients lack an acceptable donor, is an option available for 
     only about 25 percent of patients;
       Whereas rare diseases, such as DBA, benefit greatly from 
     well-established comprehensive care centers such as the DBA 
     Comprehensive Clinical Care Center at Schneider Children's 
     Hospital in New Hyde Park, New York (the ``Center''), which 
     has become the multidimensional hub for the care and 
     treatment of DBA patients across the country, as well as the 
     home of the DBA Patient Registry which has become a valuable 
     national resource for investigators utilizing the Center to 
     accomplish research in a multitude of areas not specific only 
     to DBA;
       Whereas the successful establishment of the Center became a 
     model for how to diagnose, treat, and improve the lives of 
     patients with rare diseases, while learning from the disorder 
     to yield advancements in other areas of disease research;
       Whereas the success of the initial Center prompted the 
     Centers for Disease Control and Prevention's DBA Public 
     Health Outreach and Surveillance Program to establish 3 
     additional DBA Centers in Texas, California, and 
     Massachusetts to further patient access to information, 
     treatment, and care by DBA experts, which has resulted in a 
     doubling of patient care visits for DBA care and surveillance 
     since their establishment;
       Whereas the DBA Public Health Outreach and Surveillance 
     Program at the Centers for Disease Control and Prevention 
     (``CDC'') has resulted in the completion of the first CDC 
     brochure for the DBA patient population, the introduction of 
     a DBA hotline and dedicated DBA nurse, and has resulted in a 
     25-percent increase of enrollment of DBA patients into the 
     DBA Patient Registry in the first 2 years of the program;
       Whereas the collaboration between the National Institutes 
     of Health and the Centers for Disease Control and Prevention 
     and their close collaboration with the Daniella Maria Arturi 
     Foundation and the DBA Foundation have driven the many recent 
     successes in the DBA field and serve as a model for 
     addressing rare disease research efforts through close public 
     and private collaboration to achieve the highest levels of 
     success in the areas of improved patient care and disease 
     research;
       Whereas the interagency collaboration achieved within the 
     National Institutes of Health between the National Heart, 
     Lung, and Blood Institute, the National Institute of Diabetes 
     and Digestive and Kidney Diseases, the National Cancer 
     Institute, and the Office of Rare Diseases to advance the 
     research and understanding of DBA has resulted in significant 
     advancements not only in the DBA scientific arena, but in 
     understanding its many links to more prevalent disorders; and

[[Page 27156]]

       Whereas the DBA research initiatives have already yielded 
     tremendous success including the discovery of 2 ribosomal 
     protein (``RP'') genes and the identification that DBA is the 
     first human disease linked to a ribosomal protein problem 
     which, as a fundamental unit of cellular function, has been 
     implicated in a wide range of human disorders including 
     cancer, making this discovery a profound example of the 
     additional benefits that may result from the study of DBA: 
     Now, therefore, be it
       Resolved, That the House of Representatives--
       (1) recognizes that the identification of Diamond-Blackfan 
     Anemia (``DBA'') may advance the understanding of DBA, 
     identify implications of cancer predisposition, and serve as 
     an important model for understanding human development and 
     the molecular basis for certain birth defects;
       (2) recognizes the importance of comprehensive care centers 
     in providing complete care and treatment for each patient, 
     leading to an increase in correct and early diagnosis;
       (3) commends Schneider Children's Hospital for providing 
     the first DBA Comprehensive Clinical Care Center for patients 
     across the country, for developing the DBA Patient Registry 
     which has proven a robust surveillance tool to understand the 
     epidemiology, biology, and treatment of DBA, and for proving 
     a valuable resource for investigators at a national level, 
     working to understand DBA's link to more prevalent disorders 
     facing Americans;
       (4) commends the Daniella Maria Arturi Foundation and the 
     Diamond-Blackfan Anemia Foundation for their efforts to 
     facilitate the successful collaboration among the National 
     Institutes of Health and the Centers for Disease Control and 
     Prevention to achieve a successful multidisciplinary approach 
     between clinical and scientific DBA efforts with the goal of 
     shortening the life cycle of success realized between the 
     laboratory and applied patient care; and
       (5) encourages research efforts to further understand 
     ribosomal protein deficiencies in rare inherited diseases and 
     to advance the treatment options available to those with DBA.

  The SPEAKER pro tempore. Pursuant to the rule, the gentlewoman from 
Wisconsin (Ms. Baldwin) and the gentleman from New York (Mr. Fossella) 
each will control 20 minutes.
  The Chair recognizes the gentlewoman from Wisconsin.


                             General Leave

  Ms. BALDWIN. Madam Speaker, I ask unanimous consent that all Members 
have 5 legislative days to revise and extend their remarks and include 
extraneous material on the resolution under consideration.
  The SPEAKER pro tempore. Is there objection to the request of the 
gentlewoman from Wisconsin?
  There was no objection.
  Ms. BALDWIN. Madam Speaker, I yield myself such time as may consume.
  I rise in support of H. Res. 524, expressing the sense of the House 
with respect to Diamond-Blackfan anemia, DBA.
  DBA is a rare genetic bone marrow disorder affecting children and 
adults, 90 percent of whom are younger than 1 year of age when they are 
diagnosed. DBA results in severe anemia due to the failure to produce 
red blood cells. The symptoms may vary greatly, from very mild to 
severe and life-threatening. Unfortunately, because DBA is a rare 
disease, there is limited research being done, and treatment options 
are not optimal.
  The resolution before us today as amended expresses the sense of the 
House of Representatives that we should encourage further efforts to 
clarify the natural history of DBA, continue efforts to raise awareness 
and ease access of information about DBA, encourage research efforts 
that will advance treatment options and seek a cure and encourage 
cross-institutional research initiatives to study the intricacies 
involved in this rare inherited disease.
  This is an important piece of legislation, and I would like to 
acknowledge and thank my colleague Representative Carolyn McCarthy for 
her hard work and dedication on this issue. I urge all of my colleagues 
to join me in support of this legislation.
  Madam Speaker, I reserve the balance of my time.
  Mr. FOSSELLA. Madam Speaker, I yield myself such time as I may 
consume.
  Let me join my colleague from Wisconsin in supporting H. Res. 524 and 
also acknowledging again at the outset the work and efforts of my 
colleague from New York (Mrs. McCarthy).
  As was mentioned, the resolution recognizes the elements of the 
Diamond-Blackfan anemia and the research being done on the disease.
  DBA is a blood condition, as mentioned, present at birth which is 
characterized by failure of the bone marrow to produce red blood cells, 
and unlike other types of anemia, DBA relates to a bone marrow failure. 
It's been the result of a genetic mutation and has generally been 
diagnosed at birth.
  The purpose of this resolution is to bring awareness to this disease 
and the research and education surrounding Diamond-Blackfan anemia. As 
is always the case, although the word is rare and operative, the point 
is if somebody is suffering from DBA they're suffering, and just 
because there may not be tens of thousands a year, the fact is that 
suffering doesn't go away.
  So I would urge the adoption.
  Madam Speaker, I reserve the balance of my time.
  Ms. BALDWIN. Madam Speaker, I am now proud to yield 5 minutes to my 
colleague, the author of this resolution, the gentlelady from New York 
(Mrs. McCarthy).
  Mrs. McCARTHY of New York. Madam Speaker, I'd like to thank my 
colleague Ms. Baldwin for her support, and I'd also like to thank my 
colleague from New York, Vito Fossella, for taking a strong stance on 
this issue.
  I also want to say that this bill would not have made it to the floor 
without the help of my good friend and colleague, Congressman Pallone 
from New Jersey, for his support in bringing it up.
  It was mentioned that Diamond-Blackfan anemia, or DBA, is a rare 
genetic bone marrow failure disorder that affects children and adults, 
stopping the body's ability to produce red blood cells.
  A lot of our colleagues might remember, every year I go around and 
ask all of my colleagues to sign a book so that I can have the 
opportunity to teach my colleagues about DBA, so as we go down the road 
mostly hopefully to get more research money.
  Ninety percent of those suffering this disease were younger than 1 
year old when they were diagnosed. Children living with DBA have an 
increased risk of leukemia, solid tumors, and complete bone marrow 
failure. Fifty percent of patients with DBA are also born with birth 
defects, including abnormalities to the face, head, upper arm and hand, 
and heart. Twenty-one percent of affected patients suffer from more 
than one defect.
  The individuals and families suffering from rare diseases such as DBA 
not only face the challenges of their life-threatening diseases, but 
they must also confront the limited treatment and the research options.
  Researchers believe that the study of DBA will yield clues to several 
other widespread diseases, providing valuable insights into the biology 
of blood disorders, blood cell formation, leukemia, and serve as an 
important model for understanding the genetics of birth defects.
  Unfortunately, many of the long-term treatments for DBA have the 
potential for serious side effects, including impaired growth, 
diabetes, and iron overload.
  The only cure for DBA is a bone marrow transplant, a procedure that 
carries serious risks. And since most patients lack an acceptable 
donor, it's an option available for only about 25 percent of the 
patients.

                              {time}  1715

  Rare diseases, such as DBA, where there are no regional or ethnic 
trends and a small number of patients, make progress in treatment and 
research difficult. Thankfully, there are centers across the Nation 
that devote countless hours into understanding this disease. One such 
center is based out of my district on Long Island. The DBA 
Comprehensive Clinical Care Center at Schneider Children's Hospital in 
New Hyde Park, New York, has become the hub for the care and treatment 
of DBA patients across the country. The facility is also home of the 
DBA Patient Registry, which has become a valuable national resource for 
families and the researchers.

[[Page 27157]]

  The success made at Schneider's Children's Hospital have prompted the 
Centers for Disease Control and Prevention's DBA Public Health Outreach 
and Surveillance Program to establish three additional DBA centers in 
Texas, California, and Massachusetts to further patient access, 
information, treatment, and care by DBA experts. This has resulted in a 
doubling of patient care visits for DBA since their establishment.
  The effects are also felt on a national level. The CDC has dedicated 
resources and manpower to the study of DBA as well as patient outreach. 
Because of these efforts, we have seen a 25 percent increase of 
enrollment of DBA patients into the DBA Patient Registry in the first 2 
years of the program. The collaboration achieved through Federal 
programs such as NIH and the CDC and private groups such as the Daniela 
Maria Arturi Foundation and the DBA Foundation have driven the many 
recent successes in the DBA field. This partnership should serve as a 
model for addressing rare disease research efforts through close public 
and private manners. I have been working with the Arturi family for 
many years. Their daughter Daniela was affected by this rare disease, 
and they have been the vocal voices for increased funding for research 
and treatment. Today, we in Congress will give them and all families 
suffering from this rare disease a chance of hope.
  Let me say that we hear constantly of these very rare diseases, and 
the families sometimes feel they have no hope. I would encourage them 
to reach out on the Internet to find the information they need to. The 
foundation that was started 10 years ago has come such a long way where 
researchers from across the world now come in for a conference every 
year to find out more and what work has been done. And even though the 
cure for DBA has not happened yet, the other research has helped many, 
many other families. So, please, join me in supporting this resolution 
and telling the families and the children with DBA that they are not 
alone. I ask my colleagues to support H. Res. 524.
  Mr. FOSSELLA. Madam Speaker, I reserve the balance of my time.
  Ms. BALDWIN. Madam Speaker, I am pleased to yield 2 minutes to my 
colleague from New York, Congressman Bishop.
  Mr. BISHOP of New York. Madam Speaker, I rise in strong support and 
as a proud cosponsor of this resolution, recognizing the importance of 
the Federal Government's continued support for research into the rare 
bone marrow failure disorder for which there is no known cure known as 
Diamond-Blackfan anemia.
  I am very proud to represent Manny and Maria Arturi of Remsenberg, 
NY, located in my district. After the loss of their daughter Daniela 
Maria nearly 12 years ago, the foundation they created and that bears 
her name continues making great strides toward the ultimate goal of 
finding a cure.
  When a tragic disorder like this strikes infants within the first 
year of their lives, it is all the more important for Congress to go on 
record voicing our unwavering support to raise awareness and broaden 
support for funding rare disease research. Accordingly, this resolution 
demonstrates we support giving experienced doctors the resources for 
the most complete care for those patients. And by encouraging the 
National Institutes of Health and Centers for Disease Control to 
coordinate a multidisciplinary approach toward a cure, this legislation 
brings hope that other parents will be spared from the kind of 
devastation felt by the Arturis once they learned of their child's 
diagnosis.
  Therefore, Madam Speaker, I encourage my colleagues to support this 
resolution as well as other measures that will ultimately bring about a 
cure for Diamond-Blackfan anemia.
  Mr. FOSSELLA. Madam Speaker, let me again congratulate Mrs. McCarthy 
for bringing this to the floor, and constantly, not just here but 
constantly bringing attention to DBA.
  Whenever a parent gets bad news on a child and an illness, you know 
it takes to the heart. And there are so many innovative, wonderful, 
compassionate health care professionals who try to bring a level of 
comfort to those families, and I know that here in Congress we do the 
same and try to bring awareness. And although rare, or rarer than many 
illnesses, nevertheless, the pain and suffering remains the same.
  I yield back the balance of my time.
  Ms. BALDWIN. Madam Speaker, Diamond-Blackfan anemia is such a serious 
condition; and because it is such a rare disease, there is a real need 
for increased awareness and research. I commend my colleague Mrs. 
McCarthy for her advocacy on this issue, and I urge my colleagues to 
support the resolution.
  I yield back the balance of my time.
  The SPEAKER pro tempore. The question is on the motion offered by the 
gentlewoman from Wisconsin (Ms. Baldwin) that the House suspend the 
rules and agree to the resolution, H. Res. 524, as amended.
  The question was taken; and (two-thirds being in the affirmative) the 
rules were suspended and the resolution, as amended, was agreed to.
  A motion to reconsider was laid on the table.

                          ____________________