Advances in Parkinson's Disease Research and Michael J. Fox's Story
Published on July 4th, 2023
⏱ 7 min read
by BigOmics
Neurodegenerative Diseases – Can They Be Eradicated in Our Lifetime?
Neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease pose massive problems for countless patients. With aging populations and rising healthcare costs, the need for urgent therapies to combat these diseases is growing, as their development is mainly fueled by aging. Over 50% of healthcare expenditure is already allocated to individuals over the age of 55. There are currently no drugs available for neurodegenerative diseases that can stop their progression. Existing drugs only offer symptomatic relief to certain patients.
However, there is hope. Pharmaceutical and biotech companies are working hard to develop drugs and therapies for neurodegenerative diseases, and they are making great strides. There are a number of factors contributing to this trend. Scientific advances to detect, measure, and treat neurodegenerative diseases are meeting a favorable regulatory environment and increased investment by governments and public funders in translational research and drug development.
In the past two years, we have seen the introduction of the first disease-modifying drugs for Alzheimer’s and amyotrophic lateral sclerosis (ALS). For example, lecanemab, a monoclonal antibody sold under the brand name Leqembi, is one of the first dementia drugs that appears to slow the progression of cognitive decline. [1]
Parkinson's Patient Influencers and Advocates – Meet the Michael J. Fox Foundation
In addition to research companies, patient representatives and advocates also contribute to the development of new drugs. Patient influencers are affected themselves and share their experiences, while patient advocates often work on a broader level, advocating for policy changes, patient-centered care, and improvements in healthcare access and quality.
Michael J. Fox, the famous actor who has Parkinson’s disease himself, has certainly been one of the most impressive patient-focused influencers for decades. The Michael J. Fox Foundation for Parkinson’s Research [2] is the largest private foundation supporting research into Parkinson’s disease. It has a single, urgent goal: Eliminate Parkinson’s disease in our lifetime. To achieve this, the Foundation advocates for the 6 million people living with Parkinson’s disease worldwide with optimism, urgency, resourcefulness, collaboration, accountability and persistence in problem-solving.
“Parkinson’s patients are the experts on what we have. We have a responsibility as patients to share our experience — what works for us, what we respond to, what we can contribute to research.”
– Michael J. Fox
Since its inception, the Foundation has already raised an incredible sum of well over $1 billion for Parkinson’s research. Recently, it was also instrumental in the development of a biomarker for Parkinson’s. [3] Researchers made a real breakthrough in understanding the disease when they discovered a new tool that can reveal a key pathology of the disease: abnormal alpha-synuclein – known as the “Parkinson’s protein”– in brain and body cells. The “α-synuclein seeding amplification assay” (αSyn-SAA) can detect the pathology in spinal fluid not only in people diagnosed with Parkinson’s disease, but also in people who have not yet been diagnosed with the disease (or who have no clinical symptoms but are at high risk of developing it). [4]
When Michael learned of his diagnosis in his twenties, he initially disclosed it only to his immediate family. “I am here to entertain, and I was afraid that this would no longer work if people felt sorry for me.” he explained at a recent talk. Soon, however, he reached out to the public and has been giving encouragement to many patients through his example ever since. After all, in addition to immediate medical help, a key unmet need for patients is the lack of focus on psychological and social complexities.
Michael speaks very openly about his problems with the disease and doesn’t sugarcoat anything: “I can walk, but when I’m distracted, I fall down. But if I find just one thing every day to be grateful for, then the day has been worthwhile. I’m used to winning, and I want to give people hope.”
The Molecular Basis of Neurodegeneration Is Crucial to Identify Biomarkers
The identification of biomarkers is key to early diagnosis and the development of effective therapies. And patient gene expression data is an important part of this process.
In addition to oncology, age-related and neurodegenerative diseases are therefore important focus areas for BigOmics. Vast amounts of omics data is generated in these areas, and there is a great need to analyze and process it effectively.
To read more in depth about this exciting topic, see our recent meta-analysis of two published datasets on Alzheimer’s and Parkinson’s, where we highlight similarities and differences between the two neurodegenerative diseases. We selected two transcriptomic datasets from the GEO database to analyze the similarities and differences between the two diseases, and show how large datasets can be used to provide further insights of clinical relevance.
The BigOmics platform, Omics Playground, can contribute to the development of therapies for neurodegenerative diseases by providing a comprehensive workflow specifically tailored for the analysis of RNA-Seq and proteomics data. Our goal is to develop intelligent tools that enable individuals to immerse themselves in advanced omics analysis. We streamline dataset integration and reveal key genes and proteins involved in disease biology. By supporting drug repurposing and unraveling the intricate mechanisms of novel agents, we are contributing to a deeper understanding of disease mechanisms.
Want to learn more about how RNA-Seq and proteomics provide invaluable insights for neurodegenerative research? Curious about how bioinformatics data discovery software can simplify the exploration of your RNA-Seq and proteomics data?
Unlock the full potential of your RNA-seq and proteomics data!
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References
[1] CNN: FDA advisers vote that lecanemab shows benefit as an Alzheimer’s treatment.
https://amp.cnn.com/cnn/2023/06/09/health/fda-committee-lecanemab-leqembi/index.html
[2] The Michael J. Fox Foundation for Parkinson’s Research
https://www.michaeljfox.org/
[3] Breaking News: Parkinson’s Disease Biomarker Found
https://www.michaeljfox.org/news/breaking-news-parkinsons-disease-biomarker-found?pn_cid=pn-a1b1R00000AlPyE
[4] Siderowf, Andrew et al. “Assessment of heterogeneity among participants in the Parkinson’s Progression Markers Initiative cohort using α-synuclein seed amplification: a cross-sectional study.” The Lancet. Neurology vol. 22,5 (2023): 407-417. http://doi.org/10.1016/S1474-4422(23)00109-6.