Parkinson’s disease in women: Research gaps, treatment challenges, and new hope through GEM-PD

May is Women’s Health Month and in observation, Bio.News sat down with Ragasudha Botta, MBBS, PhD, MMSc, Senior Scientific Director, Critical Path for Parkinson’s (CPP) and Critical Path Institute (C-Path) to discuss how women’s lived experiences with Parkinson’s disease have too often been overlooked in clinical research and treatment development—and have even resulted in later diagnosis. From biological differences and disparities in care access to the promise of precision medicine initiatives like GEM-PD, we explore how researchers and advocates are working to ensure women with Parkinson’s are not just data points, but are active voices when it comes to drug development and patient care.

What is Parkinson’s disease, and what does the current treatment landscape look like?

Parkinson’s disease (PD) is a progressive neurodegenerative disorder that affects movement, but it also involves many non-motor symptoms that can shape a person’s daily life. Biologically, PD is characterized by loss of dopaminergic neurons and accumulation of misfolded alpha-synuclein pathology. Clinically, diagnosis still relies largely on motor features, particularly bradykinesia together with rigidity and/or resting tremor.

The current treatment landscape has improved considerably, but it remains largely focused on symptom control rather than stopping the disease itself. Levodopa remains the most effective treatment for bradykinesia and other core motor symptoms, and dopaminergic therapies remain central to PD treatment. Other commonly used symptomatic treatments can improve symptoms and quality of life, but long-term treatment is often complicated by wearing-off, dyskinesias, or other adverse effects depending on the medication used. PD research is moving toward therapies that target disease biology, including alpha-synuclein aggregation, mitochondrial dysfunction, LRRK2 and GBA1-related pathways, gene therapy, and cell replacement strategies. 

Despite this progress, no currently available treatment has been shown to definitively halt PD progression or prevent neurodegeneration. So, the current landscape is both encouraging and incomplete. We have increasingly effective ways to manage symptoms, but the field still urgently needs therapies that change the long-term course of the disease.

How do biological differences and other gender-related factors significantly shape the lived experience of women with Parkinson’s disease?

Sex and gender influence how women experience PD, but these differences are often overlooked. From a biological standpoint, women may have a different PD profile than men. Epidemiologically, men are diagnosed more often, but this does not mean Parkinson’s is less important in women. Women may face delays in diagnosis, reduced access to movement disorder specialists, and less informal caregiving support. In women, symptoms may present differently, with a greater burden of non-motor symptoms.

Women may also respond differently to dopaminergic therapies. Some studies suggest women may experience more levodopa-induced dyskinesia, while access to device-aided therapies such as deep brain stimulation may be lower for women. This means that the same disease can translate into a different therapeutic journey for women, with different side effects, and care needs. 

Biological sex differences strongly influence the lived experience of women with Parkinson’s. Many women are also caregivers themselves to spouses, children, grandchildren, or aging parents and their own symptoms may be minimized or accommodated quietly until the disease has already affected independence, employment, relationships, and emotional well-being.

The key point is that women with Parkinson’s are not simply a “smaller group with PD.” Their biology, symptoms, treatment responses, social roles, caregiving realities, and access to care intersect. If research and clinical care do not capture these differences, we risk designing trials, endpoints, digital measures, and treatment pathways that do not fully reflect women’s real lives.

So, improving outcomes for women with Parkinson’s requires a sex- and gender-informed approach like enrolling enough women in studies, analyzing outcomes by sex, capturing hormonal and reproductive history where relevant, prioritizing patient-reported outcomes, and listening carefully to women’s voices. Precision medicine in PD will remain incomplete unless it includes the lived experience of women as central evidence, not as an afterthought

As a young investigator and clinician researcher, what attracted you to join C-Path and contribute as Senior Scientific Director of the CPP global consortium?

What attracted me to C-Path and the CPP global consortium was the opportunity to connect my clinical background, research training, and personal motivation with work that can translate into accelerating PD drug development. My journey into medicine and neurology is deeply personal. I lost my mother to meningitis when I was in middle school, after symptoms such as neck stiffness, headache, and vomiting were initially dismissed as vitamin deficiency. That experience shaped my decision to become a doctor and drew me toward neurology.

Over time, I developed an interest in neurodegenerative disorders. As a clinician, I often heard PD patients ask, “How long do I need to take these medicines?” and “Is there a cure?” Those questions stayed with me and pushed me toward Parkinson’s research, clinical trials, and drug development, ultimately leading me to pursue a Master of Medical Sciences at Harvard Medical School focused on clinical trials and drug development.

During my PhD, my clinical research work at Centre for Brain Research, and later my work at Massachusetts General Hospital, I came to appreciate the power of high-quality data. My own research work benefited from CPP’s Integrated Parkinson’s database, including work now published in npj PD. That experience made CPP’s mission very real to me, as better data can help us understand which patients are at risk, design better trials, and move closer to therapies that are targeted to the right patients. This work also introduced me to Dr. Stephenson, Vice President at CPP, who has since been an important mentor and source of motivation in my journey.

I joined C-Path and CPP because I wanted to contribute to that translational bridge, turning rigorous science, patient-level data, and global collaboration into evidence that can help answer the questions patients are still waiting on, i.e. “Can we do better, can we move faster, will there ever be a cure and can we bring real hope to people living with PD?”

What is C-Path doing to accelerate drug development for Parkinson’s disease, and why was the GEM-PD initiative launched? What is GEM-PD?

C-Path is helping accelerate Parkinson’s drug development by creating a neutral, precompetitive space where industry, academia, patient organizations, data scientists, and regulators can work together on the shared barriers that slow clinical trials. Through the CPP consortium, C-Path focuses on building drug development tools that can make Parkinson’s trials more efficient and informative. That includes integrated patient-level databases, disease progression models, clinical trial simulation tools, biomarkers, digital health technologies, and more meaningful clinical outcome measures. The goal is to help drug developers design better trials, identify the right patients, choose better endpoints, and generate evidence that is useful for regulatory decision-making.

A major part of this work is the CPP Integrated Parkinson’s Database, which brings together anonymized patient-level data from observational studies and clinical trials. This includes data from 27 studies, more than 15,000 participants. This kind of data infrastructure is important because Parkinson’s is heterogeneous, and no single study can answer all the questions needed to improve trial design.

The regulatory milestones of CPP include FDA and EMA support letters for dopamine transporter imaging as an enrichment biomarker, and an FDA Letter of Support related to α-synuclein seed amplification assay as an enrichment biomarker for trials in synuclein-related disorders. C-Path is not only generating science but is helping translate science into tools that regulators and drug developers can use.

GEM-PD stands for Global Evidence in Medicine for Parkinson’s Disease. It aims to accelerate more personalized treatments by using diverse data, artificial intelligence, and digital health technologies, with the goal of improving detection, disease management, and therapies for women affected by Parkinson’s. GEM-PD launched in March 2025 and connects it with the broader momentum around sex-informed clinical evaluation and sex- and gender-informed Parkinson’s care. 

GEM-PD is not simply a “women’s Parkinson’s project.” It is a precision medicine initiative. It asks: what are we missing when women are not fully represented in the data, in trial design, in symptom measurement, and in regulatory conversations? By answering those questions, GEM-PD can help ensure that future Parkinson’s therapies are developed for the real diversity of people living with the disease, not just for an average patient who may not represent everyone.

Why is giving women with Parkinson’s disease representation and a voice so crucial?

Giving women with Parkinson’s disease representation and a voice is crucial because many of their most burdensome symptoms are still not routinely brought into the clinical conversation. Many non-motor symptoms remain under-discussed in routine care. Because some of these symptoms feel private or embarrassing, patients may not raise them unless the clinician opens the door. So, the symptom remains invisible; not because it is unimportant, but because no one asked. This is a global issue. Women’s experiences of PD may vary across countries, cultures, and healthcare systems, in relation to access to care, stigma, and willingness to discuss symptoms. Because our database also includes patients outside the U.S., GEM-PD can help ensure that we are not defining women’s needs from a single healthcare context but are listening to a broader and more diverse global PD community.

This is why “voice” matters as much as representation. Representation ensures that women are present in the data. Voice ensures that their actual experiences shape the questions we ask. It reminds us that Parkinson’s care should not depend only on what clinicians observe, but also on what patients may be quietly living with.

Ultimately, giving women with Parkinson’s a voice is crucial because it changes what becomes visible. And once these experiences become visible, they can be measured, treated, and prioritized. Without listening to women directly, we risk defining Parkinson’s disease too narrowly and missing the symptoms that most affect their daily lives.

What is the future for patients with Parkinson’s disease, and what are your hopes specifically for women affected by the disease?

Our hope at C-Path, through GEM-PD, is to help advance a future in which women with Parkinson’s Disease are more equally represented in clinical trials, and where the scope of drug development includes new therapies that focus on women’s needs. Our hope is that the future of Parkinson’s disease will move toward care that is not only more biologically precise, but also more attentive to the realities patients live with every day. With FDA’s guidance increasingly emphasizing the importance of sex differences, this is not just a dream, it is becoming a necessary direction for more inclusive Parkinson’s research, care, and drug development.

Recent reviews continue to emphasize that women may have distinct clinical profiles, including greater vulnerability to disabling motor complications and non-motor fluctuations, while also facing disparities in care. But the future should not stop at identifying biological differences. We also need to change what we routinely capture in clinical care and research. For women, our hope is that these experiences become part of the standard Parkinson’s conversation. When these symptoms and experiences are recorded consistently, they become usable data. That data can improve clinical care, refine patient-reported outcomes, guide trial design, and help develop therapies that address what patients need, not only what is easiest to measure.

So, our hope is that women with Parkinson’s will no longer have to fit into a model of care built around an average patient who may not represent them. Instead, their biology, symptoms, priorities, and lived experience should help shape the next generation of precision medicine in PD.

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