When the federal government is handing out money, it’s all too common for rare diseases to be overlooked since, by definition, they affect far fewer people. But December was a good month for our community. The U.S. Senate and House of Representatives passed bipartisan legislation, along with a spending bill, which both will benefit patients with bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… disease.
On Dec. 16, Congress finalized the reauthorization of a bill on stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… sponsored by U.S. Rep. Chris Smith (R-NJ), who also introduced the original legislation in 2005. The Stem Cell Therapeutic and Research Reauthorization Act (H.R. 2820) will continue to fund two key donor networks that support treatment and therapies using adult stem cell lines.
For the next five years, the C.W.
Bill Young Cell Transplantation Program will receive $30 million annually, and
its companion program, the National Cord Blood Inventory, will receive $23
million per year, enabling more patients to have access to life-saving
treatments.
And on
Dec. 18, Congress passed the annual omnibus
spending bill that appropriates funds to run the government
for another year. The bill included a $2 billion boost to fund NIH research programs
and an additional $132
million for the FDA, including new money to increase its grants program for
orphan drugs, the kind our patients need to treat their rare conditions.