Quantifying disease impact and overcoming practical treatment barriers for primary progressive aphasia

Imagine gradually losing the ability to express yourself — not because you've forgotten the words, but because they simply won't come out. This is the reality for individuals living with primary progressive aphasia (PPA), a rare form of dementia that usually begins in middle age and increasingly impairs language abilities over time.

Researchers at the University of Chicago Medicine are working to illuminate the struggles of those living with this condition and pioneer accessible treatment models. They recently published new studies that measure PPA’s significant impact on quality of life and demonstrate the feasibility of international telemedicine interventions — research that could reshape care delivery and inform policy decisions.

Understanding PPA

PPA is a unique neurological condition that primarily affects language skills, setting it apart from more common and well-known forms of dementia like Alzheimer's dementia that primarily impacts memory in the earliest stages.

“PPA is a relatively rare dementia,” said Emily Rogalski, PhD, the Rosalind Franklin PhD Professor of Neurology at UChicago and a leading researcher in the field. “It's often overlooked in the literature because it can be difficult to assemble large groups of people to survey lived experiences.”

She said it’s frequently overlooked by medical professionals as well, going undiagnosed particularly among those with lower socioeconomic status.

“Lack of diagnosis can be such a barrier to care for anyone not living next to a specialized medical center,” she said.

A feature that makes PPA especially challenging is its early onset.

“These individuals are at a different life stage from late-onset Alzheimer’s dementia patients,” Rogalski said. “They're often still in the prime of their working career; they may have young children in the home.”

This can mean PPA not only affects the patients but also has significant implications for their families, family relationships and economic stability.

Measuring PPA’s impact on quality of life

To better understand how PPA affects daily living, Rogalski and her collaborators conducted a study using the Health Utilities Index (HUI), a standardized tool that measures various aspects of well-being, including physical abilities, emotional health and cognitive functions.

Unsurprisingly to those familiar with the disease, the results showed that PPA has a moderate to severe negative impact on patients’ health-related quality of life. The results also showed that greater language impairment in individuals with PPA was linked to a lower quality of life, particularly affecting domains such as hearing, sensation, cognition and speech.

“It was important to confirm that the HUI, a commonly used health measure across diseases, was capturing the essence of these patients’ primary impairment,” Rogalski said.

Thomas Hopkins, PharmD, MS, the study’s first author, explained the study’s twofold purpose: obtaining detailed information about quality of life for individuals with PPA, and enabling direct, objective comparisons between the impact of PPA and that of other diseases. Since the Health Utilities Index is a generic measurement, it can be applied to any health condition — even those dissimilar to dementia, such as cardiovascular disease or cancer.

“When it comes to policy-making and government allocation of resources, it really is important to have these broadly applicable measures so better decisions can be made,” Hopkins said.

This quality-of-life data can inform crucial decisions including research funding and prioritization, insurance regulation, disability coverage and more. Now armed with concrete evidence that PPA can impact the lives of middle-aged adults just as dramatically as many other disruptive diseases, researchers, patients and families can advocate for more resources and support.

Expanding access to care with telemedicine

Even while working to establish objective measurements of PPA’s impact, Rogalski and her colleagues were simultaneously enrolling patients in a clinical trial exploring the feasibility of delivering speech-language therapy for PPA via telemedicine. They recently reported the successful enrollment of 95 participant pairs — each comprising a PPA patient and their primary caregiver — from four countries, demonstrating that remote recruitment and video chat intervention are viable options for overcoming geographic and socioeconomic barriers to treatment.

“We found a way to deliver care that creates a little bit more of an equal playing field,” Rogalski explained. “Persons with PPA don't have to be living next to a major academic medical center or specialty center to get in touch with an expert and receive treatment.”

This success also paves the way for future research and interventions — and not just for PPA.

“We see our approach as a potential model,” Rogalski said. “We think the framework we have can be adapted and used to support advocacy and interventions for multiple different dementia syndromes and conditions.”

Offering hope even in the absence of a cure

Rogalski points out that care partners play a crucial role in studies like these, providing essential insights into daily challenges and diverse needs that span family dynamics and life situations.

“We're giving families a voice to share their lived experiences,” she said.

Despite these research advancements, challenges remain in diagnosing and treating PPA, and no drug or therapy that can cure the disease has been developed yet. But broadening understanding of PPA’s profound impact on quality of life and demonstrating the effectiveness of telemedicine interventions are meaningful steps that can encourage patients and families.

“Providing hope and practical support for these families is really important,” Rogalski said. “Too many people have lived experiences in which, even when they find a specialist, that specialist may say, ‘There's nothing we can do. It's a terminal diagnosis.’ But finding a cure isn't the only way we can help people. Maximizing independence, emotional well-being, confidence — all of those things can have a lot of practical aspects on one's daily life. If we can ramp those up as much as possible while we're simultaneously looking for pharmacological solutions, that's a winning combination.”

To learn more about ongoing PPA studies and related research, visit the Healthy Aging & Alzheimer's Research Care (HAARC) Center website.

“Assessment of Disease Impact through Health-Related Quality of Life Measurement in Primary Progressive Aphasia” was published in Alzheimer's & Dementia: Translational Research & Clinical Interventions in November 2024. Authors include Thomas Hopkins, Eunbi Kwon, Allison Lapins, Nathan Gill, Angela Roberts and Emily Rogalski.

“Communication Bridge 2 Randomized Controlled Trial: Recruitment and Baseline Features” was published in Alzheimer's & Dementia: The Journal of the Alzheimer's Association in November 2024. Authors include Emily Rogalski, Matthew Bona, Marissa Esparza, Aimee Mooney, Melanie Fried-Oken, Alfred Rademaker and Angela Roberts.