[Congressional Bills 108th Congress]
[From the U.S. Government Publishing Office]
[H.R. 894 Introduced in House (IH)]







108th CONGRESS
  1st Session
                                H. R. 894

 To amend the Public Health Service Act to require the Director of the 
    National Institutes of Health to expand and intensify research 
                   regarding Diamond-Blackfan Anemia.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                           February 25, 2003

  Mrs. McCarthy of New York introduced the following bill; which was 
            referred to the Committee on Energy and Commerce

_______________________________________________________________________

                                 A BILL


 
 To amend the Public Health Service Act to require the Director of the 
    National Institutes of Health to expand and intensify research 
                   regarding Diamond-Blackfan Anemia.

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Diamond-Blackfan Anemia Act''.

SEC. 2. FINDINGS.

    The Congress finds as follows:
            (1) Diamond-Blackfan Anemia (in this section referred to as 
        ``DBA'') is a rare genetic bone marrow failure disorder, which 
        develops in infancy and results in severe anemia due to failure 
        to make red blood cells.
            (2) 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, and iron overload because of the transfusions. 
        DBA patients have an increased risk of leukemia, solid tumors, 
        and complete bone marrow failure.
            (3) 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 for only about 25 
        percent of patients.
            (4) Although there are a few studies focusing on other bone 
        marrow failure syndromes and one intramural study investigating 
        the efficacy of stem cell treatment for DBA patients, there are 
        almost no studies conducted at or with extramural funding from 
        the National Institutes of Health that are specifically aimed 
        at the identification, treatment, and cure of DBA.

SEC. 3. DIAMOND-BLACKFAN ANEMIA.

    The Public Health Service Act is amended by inserting after section 
424B (42 U.S.C. 285b-7b) the following:

                       ``diamond-blackfan anemia

    ``Sec. 424C. (a) In General.--The Director of the Institute, in 
coordination with the National Heart, Lung, and Blood Institute and the 
Office of Rare Diseases, shall expand and intensify research and 
related activities of the Institute with regard to Diamond-Blackfan 
Anemia.
    ``(b) Programs.--In carrying out subsection (a):
            ``(1) Scientific workshop.--The Director of the Institute 
        shall convene a scientific workshop for the following:
                    ``(A) Evaluation of the state of research on 
                Diamond-Blackfan Anemia.
                    ``(B) Determination of areas of importance for such 
                research (which may include red cell differentiation, 
                ribosomal protein function, cancer predisposition, and 
                gene discovery).
                    ``(C) Development of a comprehensive plan for 
                conducting and supporting such research.
            ``(2) Research.--In accordance with the comprehensive plan 
        developed under subsection (b)(1)(C), the Director of the 
        Institute shall conduct or support research on the following:
                    ``(A) The pathophysiology of Diamond-Blackfan 
                Anemia.
                    ``(B) The relationship between Diamond-Blackfan 
                Anemia and predisposition to cancer.
                    ``(C) Developing better treatments and ultimately a 
                cure for Diamond-Blackfan Anemia.
            ``(3) Database.--The Director of the Institute shall 
        facilitate the expansion and maintenance of, as well as foster 
        the use of, the Diamond-Blackfan Anemia National Registry.''.
                                 <all>