Hopes of family, firm collide on unproven drug

Jack Fowler, 6, has a severe form of Hunter syndrome, a rare metabolic disease that causes physical and mental impairments and affects an estimated 2,000 patients worldwide.
Jack Fowler, 6, has a severe form of Hunter syndrome, a rare metabolic disease that causes physical and mental impairments and affects an estimated 2,000 patients worldwide.John Zich for The Boston Globe

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Jamie and Jason Fowler arrived early at a Chicago airport hotel on a recent Saturday morning, anxious to meet the pharmaceutical executive from Massachusetts they hoped would help save the life of their 6-year-old son, Jack. So began a quest that provides a window into one of the most heart-wrenching dilemmas in modern medicine: should an experimental drug be given to a dying patient if it is unproven and might unravel a carefully designed clinical trial? For the Fowlers, it would be compassionate indeed if Shire, an Irish-based drug company that has a rare diseases division in Lexington, Mass., agreed to provide the drug that potentially could save their child. But for the company, the risks of providing a drug in the early stage of testing can be high.

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