ALS is a rare degenerative disease that progressively weakens the body until the person no longer has the ability to breathe. There is no cure. But a new treatment that is in human trials is sparking hope.
“I owe you a yo-yo today.” That’s the mantra Patty Haberstroh repeats as she goes through her monthly exercises at her home in Westport, Conn. Haberstroh lives with ALS.
She gestures for me to sit on her walker next to her glowing Christmas tree. She adjusts the electric recliner that with the push of a button, can lift her up so it’s easier for her to stand. She has a brace over her right leg and foot. Her speech is slurred, which happens when she tires. In a couple of hours, she’ll be mingling at a Christmas party, so whenever she speaks, her bell necklace and earrings jingle. Haberstroh now relies on a walker but may soon be bound to a wheelchair.
“In the beginning it was really just my toes would drag,” she said. “It’s called foot drop. And I could lift my foot, but oftentimes my big toe would curl under and that would cause you to fall oftentimes.”
It took nine months for her to be diagnosed with ALS – going from doctor to doctor to rule out everything else since, she says, there is no definitive test for ALS. The average lifespan from diagnosis is two to five years, but she hopes she can be like the late astrophysicist Stephen Hawking, who lived with ALS for 55 years. But as of now, only two treatments are FDA-approved. And neither bring back muscle function that has been lost.
But the ALS Therapy Development Institute, or ALS TDI, has been developing a new treatment called AT-1501. Phase I of human trials began in late November with 32 patients. CEO Steve Perrin anticipates good results.
“So we’re really excited about AT-1501 because, at least in animals, it was one of the first drugs we’ve ever tested in animal models of ALS that slowed down disease progression and improved survival and it improved muscle function,” Perrin said.
It took $17 million to bring the drug to human trials. And Haberstroh contributed over $600,000. She is the face behind the viral video, The Pepper Challenge. She and her family challenged the world to eat a, usually hot, pepper or donate to ALS research. Their goal is now to raise $1 million.
Many organizations and clinics are also anticipating FDA approval of AT-1501, but remain cautious.
“This is the first time, in human [trials] this type of treatment has been evaluated,” said Dr. Rahman Pourmand who treats ALS patients at the Neuromuscular Disease & ALS Center in Stony Brook, Long Island. “But it’s starting early stages now. Only a few patients are enrolled there to have a study done.”
Drugs can take years to go from trial to FDA approval so Haberstroh is unsure if she will ever benefit from the new treatment herself. But her goal all along was to raise awareness for ALS – and hopefully recruit advocates who do not live with the disease.
“There are no survivors. We die. Cancer patients have advocates who are still here and can fight. We can’t. Cause we’re not here anymore.”
While Haberstroh is still here, she keeps very busy. She is family-oriented, works at the town’s Social Services Department and is excited for the holidays.