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.

February 25, 2003

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

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.'.

END