FTA:

    They found cyclodextrin and initially tried using it in
    oral doses, which is known to be safe. However, the 
    chemical couldn’t effectively reach the brain that way.
    The couple made headlines with their tireless efforts to
    get drug companies, the FDA, and doctors to let them try
    out intravenous treatments of cyclodextrin for their
    twins—and they won. Regular treatments gradually
    improved—although didn’t cure—the twins’ conditions.
    Cyclodextrin is now in clinical trials to treat other
    kids with NPC.