A drug has shown promise for “slowing the progression of a rare, painful genetic condition that causes excessive calcium buildup in the arteries, known as arterial calcification due to deficiency of CD73 (ACDC).” The study with the results was published in the journal Vascular Medicine.
ACDC has no known cure. It can make walking “painful and difficult” and, in severe cases, can eventually lead to “potential limb loss.” The disease is extremely rare, affecting “only about 20 people worldwide” with an estimated prevalence of less than 1 in 1 million. The drug that showed promise is an existing one called etidronate.
Researchers were able to evaluate the safety and effectiveness of the drug in the legs of seven people, who “collectively represent about one-third of all the known cases in the world.” The researchers measured calcium deposits and tested blood flow over a three-year period during which the patients were taking the oral drug daily for 14 days every three months.
No adverse side effects were reported and “the drug appeared to slow the progression of new calcium deposits in the blood vessels of the legs as well as slow the progression of blood flow inhibition. However, the drug did not reverse calcium deposits that were already present in the affected blood vessels and joints, nor did it show clear improvement in blood flow. Questionnaires administered to the patients suggested that symptoms, such as pain and motion impairment, were improved.”
NIH’s website has the news release.
