This week, Lauran Neergaard of the Associated Press examined the difficulty in treating rare diseases. But, she notes, there is hope:
“The National Institutes of Health this fall will open a center to speed genetic discoveries into usable therapies, doing some of the riskiest early-stage research in hopes companies then will step in.
“A new International Rare Diseases Research Consortium is pushing for at least 200 more treatments by 2020, in part by pooling the work of far-flung scientists and families.
“Rather than starting from scratch, the Food and Drug Administration is pointing the way for manufacturers to “repurpose” old drugs for new use against rare diseases, publishing a list of those deemed particularly promising.
“And bipartisan legislation recently introduced in the Senate, called the Creating Hope Act, would offer drug makers another financial incentive – a voucher promising fast FDA evaluation of their next blockbuster drug in return for developing a therapy for a rare or neglected disease that disproportionately affects children.”
In addition to helping treat rare diseases (a rare disease is one which affects fewer than 200,000 individuals), Neergard quotes Dr. Francis Collins of the NIH as saying that the search for a treatment for a rare disease may lead scientists towards a cure for a more common ailment.
To learn more about rare diseases and NIH’s involvement, read Neergaard’s story.