CurePSP Hosts Landmark FDA Meeting to Put PSP Community on the Record

Kelly’s wife Miranda loved to cook. But as PSP progressed, focusing her eyes downward on a knife became nearly impossible — until one day, she looked down to chop vegetables, lost her balance and fell into a cabinet door. It broke off and stabbed her in the arm. “This can't be happening,” Kelly remembered thinking. “I don't understand what is going on.” That was the moment he realized Miranda would never cook again.

On February 6, Kelly was one of more than 20 PSP community members who shared stories like this directly with FDA officials, researchers and drug developers during CurePSP's Patient-Focused Drug Development (PFDD) meeting. For a disease with no approved treatments and no shortage of moments like Miranda's, it was long overdue. Those testimonies will now be compiled into an official Voice of the Patient report, published on the FDA’s website and used by the staff who make drug approval decisions. The report will also be publicly available through CurePSP and serve as a critical resource for researchers and drug developers working to advance treatments for PSP.

The meeting ran all day across two sessions, with the morning focused on symptoms and daily impact and the afternoon on current and future treatments. More than 200 community members joined virtually, and dozens spoke directly by phone or video. CurePSP CEO Kristophe Diaz set the tone in his opening remarks: the goal, he said, was to put the lived experience of people with PSP and their care partners “on the record, clearly, directly, and in a way that can inform decision making.” That framing means something to a community that has long felt left out of rooms where these decisions get made.

"These powerful testimonies are now part of the official record," said Dr. Diaz, "and they will help guide regulators, researchers and industry toward treatments that reflect the real needs of the people and families living with this disease."

Multiple people who spoke used the same metaphor independently: a tsunami. One wave builds, and then before you've fully adjusted, another is already forming behind it. “You reach a new normal and you think, ‘well, this is not so bad,’” said Steve, who cared for his wife Carol until she passed away in 2023. “But every time you do, it worsens.” For Carol, it started as a balance issue. Then Steve was walking alongside her. Then holding her up. Then she was in a wheelchair. By the time several waves are building at once, he said, independence is gone.

For many, the waves start with falls. Letty, from El Paso, watched her husband Javier go from occasional stumbles to completely bedbound over the course of a few years. The man she described as “strong, active, funny and brave” — someone who loved camping, road trips and volunteering at church — now needs help with everything. “We no longer have independence, a social life,” she said, “and my husband has lost a sense of dignity and self-worth.”

Communication goes next, and with it, connection. Kelly described how Miranda's speech has narrowed to only what is strictly necessary. Single words: Bathroom. Dinner. Neck. Pillow. The small moments and inside jokes that once came easily have become harder to reach. Their two teenagers have had to grow up faster than most, but Kelly said they've handled it with a kind of quiet grace. His son will come into the kitchen unprompted and start unloading the dishwasher. His daughter does the same. “It's an unspoken road,” Kelly said, “but it's there. They know what I'm going through and I know what they're going through.” Miranda passed away from PSP after the February 6 meeting. Kelly's voice that day was also hers, and a reminder of the urgency of finding treatments for this disease.

Tony described how even going out to a restaurant, one of the last things he and his wife Phyllis could still do together, is slowly becoming impossible as her swallowing grows more difficult. Fran, whose husband Stuart called in from South Carolina, has stopped going to church because she can no longer hold a conversation with anyone there. “She's the more social member of the family,” Stuart said, “but she really can't talk to anybody.” Ashley, whose father Peter passed away from PSP last year, found a note he had written sometime before he died. It said: “I just want someone to talk to.”

The community's resilience has far outpaced the treatments available to them. Physical therapy, speech therapy and occupational therapy remain the primary tools people with PSP rely on to manage symptoms. Some people find modest relief from Parkinson's medications, though most report limited benefit. Nothing currently approved slows or stops the disease. Jack, the board chair of CurePSP, described watching his wife Linda participate in a clinical trial that produced three extraordinary months: she was walking, talking and engaging again in ways she hadn't been able to for some time. “It was like a gift,” he said. Then it wore off. She passed away that August. “I just tell you, it was such a special time.”

Short of a cure, the overwhelming priority for future treatments was simply to slow or stop disease progression, to give people more time. Geraldine, who is in the earlier stages of PSP, put it plainly: “I don't want to fall anymore. I've fallen enough.”

These voices are now on record. The Voice of the Patient report will compile everything heard on February 6 — panel testimonies, phone calls, polling results and written comments — and will be published on the FDA's website. Written comments are still being accepted through March 9. If you have a story to add, submit it here.