Advancing Parkinson’s Disease Research: APDA’s Commitment to Innovation
The American Parkinson Disease Association (APDA) is committed to furthering Parkinson’s disease (PD) research and proudly invests in the most promising clinicians and scientific projects focused on the discovery of the cause(s) and finding the cure(s) for PD. Our Centers for Advanced Research, along with a variety of funded grants and fellowships, are the backbone of our research program. APDA aims to fund early investigators – whether new to their careers or to PD research – giving them the initial funding needed to obtain the preliminary data necessary to secure a large grant.
Spotlight on APDA Research Grant Recipient: Dr. Roberta Marongiu
We recently had the opportunity to catch up with Roberta Marongiu, PhD, a former APDA Research Grant recipient, who updated us on her PD research initiatives. Dr. Marongiu is an assistant professor of genetics and neuroscience in the Neurological Surgery department at Weill Cornell Medicine in New York City. She also holds a secondary appointment in the Cornell Genetic Medicine department and the Feil Family Brain and Mind Institute. Using novel genetic, viral, and animal model approaches, her lab research focuses on the identification of the molecular mechanisms underlying the role of biological sex and menopause on neurodegenerative brain disorders like PD and Alzheimer’s disease.
Exploring the Role of Biological Sex in Parkinson’s Disease
APDA: You were the recipient of an APDA research award in 2018. Tell us about your work and what your research at that time uncovered.
Dr. Marongiu: The focus of my lab’s research is to understand the role of biological sex, particularly ovarian hormones, in PD. With APDA funding, we developed a novel PD mouse model with alpha-synuclein overexpression in the substantia nigra. Additionally, we used a novel mouse model of accelerated ovarian failure to mimic human menopause. By combining these models, we found that early menopause accelerates the onset and progression of PD in female mice, similarly to what we observed in males, which we believe parallels the human condition but has been so far highly debated. This work is being prepared for publication, including an analysis of the underlying mechanisms. My last interview with APDA was in 2019 and described this work in some more detail.
APDA: How has your research career progressed since you were awarded the APDA grant?
Dr. Marongiu: The APDA grant launched my career as an independent investigator, enabling me to establish models and techniques in the lab that I have since applied to other projects. It also allowed my lab to expand our research goals while keeping our primary research focus on the role and mechanisms of biological sex differences in PD. This work led to additional funding, including a grant from the National Institute on Aging to study the effects of accelerated ovarian failure in Alzheimer’s disease mouse models. Furthermore, we received a multi-million-dollar grant from Aligning Science Across Parkinson’s (ASAP) through The Michael J. Fox Foundation to characterize the influence of biological sex and ovarian hormones on the propagation of PD pathology from the gut to the brain.
APDA: You have collaborated with the Women’s Brain Foundation on initiatives regarding the care of women with PD. What were the results of that collaboration?
Dr. Marongiu: Last year, I began collaborating with the Women’s Brain Foundation and soon after accepted the pro-bono role of Scientific Lead for the organization in the US. Our first collaborative effort resulted in a peer-reviewed article published in Nature Medicine, titled “Using a sex- and gender-informed lens to enhance care in Parkinson’s disease.”
The innovation of this work also lies in its patient-centric approach, originating from the 2023 Policy Roundtable, which united patients, caregivers, policymakers, scientists, and healthcare professionals to address the unmet needs of women with PD. The research highlights sex differences in PD and highlights how the disease presents unique challenges for women, including differences in symptoms, progression, cognitive and emotional impacts, delayed diagnosis, and underrepresentation in research. This work sheds light on the urgent need for a sex- and gender-informed approach to PD diagnosis, care, and research. It calls for policy changes, more inclusive clinical trials, and improved healthcare strategies to address the unique challenges faced by women with PD and ensure equitable care for all PD patients.
APDA: Artificial Intelligence (AI) has the potential to transform how clinical care is provided with a lens towards personalized medicine. How is AI informing your work in regard to the clinical care of women with PD?
Dr. Marongiu: Since receiving APDA funding, my lab’s research focus has expanded to include new aims and methodologies. This includes an exciting collaboration with Dr. Chang Su at Weill Cornell Medicine. Our goal is to use Artificial Intelligence machine learning models to integrate the analysis of Big Data and gather a deeper understanding of the multivariate nature of sex differences in PD and the underlying mechanisms. Ultimately, we believe these approaches will transform the way treatments and cures are developed by adopting a more tailored and personalized approach for both women and men with PD.
Tips & Takeaways
- APDA is committed to furthering PD research and proudly invests in the most promising clinicians and scientific projects focused on the discovery of the cause(s) and finding the cure(s) for PD. APDA aims to fund early investigators – whether new to their careers or to PD research – giving them the initial funding needed to obtain the preliminary data necessary to secure a large grant.
- Former APDA Research Grant recipient Dr. Roberta Marongiu conducts research focused on understanding the role of biological sex, particularly ovarian hormones, in PD.
- The APDA Research Grant that Dr. Marongiu received helped launch her career as an independent investigator, allowed her lab to expand their research goals while maintaining their primary research focus, and led to additional funding from other sources.
- You can learn more about what we know about PD in women in this previous APDA blog post.
- You can support APDA’s mission, including our national research program, by making a donation here. Thank you.