A clinical trial for Huntington’s opens the door to hope for some patients. Only so many can get in
PERRY, N.Y. — Reminders of Huntington’s disease populate Tammy Stewart’s apartment. They are also glimpses into her future.
On the table beside the couch sit framed pictures of her dad, Roy, and one of her sisters, Debbie, who both died from the disease. The apartment’s second bedroom belongs to Stewart’s brother, Mike, who at 60 years old is in the late stages of the disease. He spends most of the day in either a hospital bed or a reclining chair. He struggles to speak, his hands shake and ball up, and he relies on Stewart as a full-time caretaker.
Stewart, 46, also has the genetic mutation that causes Huntington’s. Tremors have started to trouble her hands and feet, and she has bouts of depression and flashes of rage. She sometimes loses track of what she is saying.
“If I’m at the beginning stages of it now, my quality of life with stress and stuff will go down fast,” she said. “And I don’t want that. I don’t want to live like that. I don’t.”
Stewart hopes she now has the opportunity to avoid that fate, or at least postpone it. She is one of about 800 patients around the world enrolled in a final-stage clinical trial of a drug designed to slow — maybe even stop — the progression of the disease. Though it wouldn’t be a cure, it’s the first time a therapy aimed at the actual root of Huntington’s has reached this far in its development odyssey. And it is one of a wave of therapies to rely on advances in genetic medicine to combat previously untreatable inherited diseases.
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