(Novemer 8, 2001) - WASHINGTON - Senator Edward M. Kennedy has introduced legislation that would dramatically increase federal spending on rare diseases, including the establishment of regional ''centers of excellence'' where research on the 6,000 rare disorders would be conducted. Boston is among the possible sites for such a center.
While many rare diseases are little known and affect only a small number of people each, the overall impact is large, with an estimated 25 million affected.
In the bill, Kennedy cited several examples of rare diseases, including cystinosis, a metabolic disease that causes havoc in kidneys and other organs and affects about 600 people in the United States. The Globe on Sunday profiled an 11-year-old Burlington boy, Shea Hammond, who has cystinosis and whose mother, Colleen Hammond, has been leading a fight for more funding for rare disease research.
Advocates for more funding have noted that research on rare diseases has the potential for discoveries that can help uncover information on common diseases. Medical specialists said some of the most important discoveries about heart disease and stroke have come from the study of rare diseases, but funding of research on the lesser-known disorders has been minuscule compared with the amounts devoted to the most widespread diseases.
Kennedy called for doubling the funding for the Office of Rare Diseases at the National Institutes of Health to $4 million annually; increasing drug research grants for rare diseases from $12 million to $25 million; and setting aside $20 million to create the centers of excellence, where clinical trials could be conducted.
The location of the centers would be determined by independent specialists, with Boston among the possible sites, a Kennedy aide said. A companion bill would accelerate tax credits for ''orphan drugs,'' which are developed for rare diseases, a measure that may attract the most interest from biotechnology companies.
Kennedy, who filed the bill on Friday, said in a statement yesterday that he hoped the increased funding would ''greatly enhance the prospects for developing new treatments and diagnostics, and even cures for literally thousands of rare diseases and disorders.''
