Timing is Key in Parkinson’s Clinical Trials
A Florida scientist is urging sponsors and care providers to do a better job of getting early-stage Parkinson’s sufferers into clinical trials before they start treatment.
Robert A. Hauser, director of the Parkinson’s & Movement Disorder Center at the University of South Florida and author of one of the most widely used patient diaries for Parkinson’s trials, says researchers need to recruit patients at the first sign of trouble — before they start taking meds so they have time to assess experimental therapies against placebos.
The critical time to enroll patients is during their so-called Golden Year — that is, as soon as they’re diagnosed with “mild classic motor features until they truly require symptomatic therapy,” Hauser said.
This gives both researchers and patients the best shot at coming up with a treatment that may stave off symptoms and prevent disease progression, he tells CenterWatch.
Hauser says his clinic is part of at least four early-stage Parkinson’s trials, two privately sponsored and two funded by a combination of grants from private foundations and the National Institutes of Health. There are about 20 participants in total in those studies but Hauser says he’s lost count of the number of patients who came to see him long after their symptoms started and it was too late to enroll them.
“My experience is a lot of patients come to us and say, ‘I’m really here because my symptoms are bothering me.’ Boy, if only this patient had come a year ago,” Hauser says.
He notes that patients, care providers and especially sponsors can play a critical role in early trial recruiting.
Parkinson’s develops subtly — a tremor here, slight difficulty moving there, says Sofija Jovic, business transformation advisor at MedAvante-Prophase. Typically, patients have about six months to a year after displaying very early signs before requiring systemic therapies to address underlying symptoms.
Parkinson’s disease, and most other neuro-degenerative disorders, can hide for a long time because human brains are adaptable. That’s good news for stroke patients because the brain will dig new neural pathways. But it’s bad news for Parkinson’s sufferers because it generally means that by the time symptoms present their brain has literally run out of room to adapt.
“You have sort of a window during which there are enough symptoms for us to be sure that the person has this disease but it’s not so far gone that we’re not likely to see any improvement,” Jovic says.
As it stands, less than 3 percent of Parkinson’s patients enroll in clinical trials, Jovic says. Patients are a lot like the frog in the boiling water — they don’t realize there’s trouble until it’s already too late, she adds.
Recent discoveries that Parkinson’s appears to be related to the buildup of misfolded alpha-synuclein proteins — and that those protein clusters may respond to treatments — has given researchers renewed hope that they’re on a path toward effective treatments and even cures, Hauser says.
But he stresses that won’t happen without enough patients to recruit for clinical trials at the earliest, pre-med stages of the disease.
He admits that the subtlety of early-stage Parkinson’s creates “a Catch-22” of sorts. “How do we get this message to patients who don’t know there’s this message to be received?” he says.
The answer, he tells CenterWatch: Sponsors have to do a better job of getting the word out to doctors that they shouldn’t rush to prescribe systemic meds prematurely — and should instead refer patients to early clinical trials.
Jovic agrees that sponsors have to do a better job establishing relationships with care providers. But she says they also need to develop technology that enables virtual trials because neuro-degenerative disorders don’t lend themselves to site visits.
“You have a patient who’s been up half the night, and you’re going to ask his caregiver to drive him two hours to a site for four hours of tests?” Jovic says. “That’s just not realistic.”