Hello fellow MSA warriors,
I want to share some news about a program that might give us all a bit of “Cautious Optimism” in our fight against Multiple System Atrophy (MSA). It’s called MyTrial, and it’s being developed by the incredible team at Khurana Labs, part of the Division of Movement Disorders at Brigham and Women’s Hospital in Boston. https://khuranalab.bwh.harvard.edu/bwhmytrial/
Before I dive into blogging about my experiences with the MyTrial program, I want to take a moment to explain what MyTrial is all about. Understanding the details of this innovative program will help you follow along with my journey and see how it might bring a bit of light to all of us living with Multiple System Atrophy (MSA).
What is MyTrial?
MyTrial is not your typical clinical trial. It’s an innovative approach to understanding and treating diseases like MSA. The scientists at Khurana Labs are using cutting-edge technology to get a very detailed picture of how MSA affects each of us. This process is known as “deep phenotyping,” and it involves tracking our disease progression with various clinical measurements, including biometrics, biomarkers from blood and spinal fluid, and advanced brain imaging techniques like MRI and PET scans.
The goal of MyTrial is to match the right patient with the right drug at the right time. This is in line with precision medicine, which aims to tailor treatments based on individual characteristics. The hope is that by understanding the natural history of our disease, they can provide us with the most effective treatments. This is a new program that they are just getting started, so they are currently enrolling a small number of patients, but they’re hoping to increase enrollment over the next couple of years.
https://www.brighamandwomens.org/neurology/movement-disorders/pamsa-multidisciplinary-clinic
The Science Behind MyTrial
The MyTrial program relies on the extensive infrastructure of the Harvard Biomarkers Study (HBS 2.0) and the Movement Disorders Genetics Clinic at Brigham and Women’s Hospital. These facilities are among the largest biobanks for Parkinson’s disease and related disorders in the world. They have the tools and expertise to perform whole-genome sequencing and other advanced genetic studies.
One fascinating aspect of MyTrial is the use of personalized stem-cell models. From a simple skin biopsy, the scientists can generate what’s called an induced pluripotent stem cell. This is a special type of cell that can turn into any cell type in the body, including the neurons affected by MSA. Additionally, they can capture the toxic proteins that are causing damage in our brains, like alpha-synuclein, using a technique called the “seeded amplification assay.” This allows them to create a personalized model of our disease in the lab, where they can test potential drugs against it.
Personalized Treatment Approach
What makes MyTrial unique is its “n-of-few” clinical trial approach. This means that instead of testing drugs on large groups of people, they focus on a few individuals, tracking their disease and responses to treatment very closely. This approach allows for a better understanding of how each patient responds to a drug, increasing the chances of finding effective treatments.
For another day: Another promising area of research in MyTrial is gene therapy. The scientists are working on optimizing techniques like gene knockdown and editing, which could potentially correct the genetic problems causing MSA. These therapies are tested in the personalized stem-cell models before being introduced to patients, reducing the risk and increasing the chances of success.
Why This Matters
As someone living with MSA, I know how challenging it can be to hold onto hope. But programs like MyTrial offer a glimpse of a brighter future. The detailed, personalized approach to understanding our disease means that treatments are tailored specifically to us. This could lead to more effective therapies and, ultimately, better quality of life.
I’m thrilled to share that I’ve recently started participating in MyTrial. Over the coming months, I’ll be blogging about my experiences and progress. I hope that by sharing my journey, I can provide insight and encouragement to all of you who are also battling MSA.
Follow along as I traverse this exciting new Horizon for MSA. It’s important for us to stay informed and involved in the advancements that could potentially change our lives.
Stay strong, stay hopeful, and let’s continue to support each other on this journey.
Warm regards,
~Coach~
Living Beyond the Diagnosis 
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