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Is mRNA the Key to Treating Parkinson’s Disease?


Parkinson’s Disease (PD) impacts as many as 500,000 Americans, though some estimates put that number as high as 1 million. There is currently no cure for PD, which means that healthcare providers are constantly on the lookout for effective therapeutic treatments to improve patients’ quality of life. Now, researchers at the Scripps Research Institute have developed a new method to target mRNA, potentially unlocking a number of therapeutic approaches for neurodegenerative diseases like PD. Read on to find out more about this groundbreaking study and its implications for PD patients and their caregivers.

What Is Parkinson’s Disease?

Parkinson’s disease is a progressive disorder that affects the nervous system, leading to symptoms including tremors, slurred speech, stiffness, and slowing of physical movement. The symptoms of PD worsen over time, and there is currently no cure for the disease. For these reasons, Parkinson’s is classified as a neurodegenerative disease — an illness that impacts the nervous system and turns increasingly severe as it progresses. Researchers have long explored therapeutic treatments for neurodegenerative diseases like PD; unfortunately, these diseases are often associated with specific proteins that are often difficult to target. One such protein is Alpha-Synuclein, or α-synuclein, a type of neural protein that accumulates in patients with PD, leading to dysregulated genetic encoding.

Finding Effective Treatments via mRNA Intervention

In the past, researchers have tried to target disordered proteins like α-synuclein, but results have been limited due to the proteins’ unique structures. These proteins lack small-molecule binding pockets, the stable three-dimensional structures that would ordinarily serve as targets for therapeutic drug compounds. With that in mind, α-synuclein proteins have been dubbed “undruggable” proteins.

But now, the Scripps research team has targeted these proteins in a new way: specifically, targeting messenger RNA, or mRNA, which is the genetic material that tells the human body how to make proteins. The team published their findings in a paper entitled “Decreasing the intrinsically disordered protein α-synuclein levels by targeting its structured mRNA with a ribonuclease-targeting chimera,” in the journal Proceedings of the National Academy of Sciences (PNAS).

mRNA Binding Targets Protein Building Blocks

As mentioned above, mRNA provides the instructions that allow the human body to build proteins. To successfully target the problematic proteins, the research team determined that they would need to target the encoding structure within mRNA before it could form the protein itself. That encoding SNCA mRNA has multiple ordered structure areas within its 5′ untranslated region, making it an excellent target for drug interventions.

To target the encoding mRNA, the team introduced a powerful duo: Synucleozid-2.0 and Syn-RiboTAC, an mRNA binding and degradation “team” designed to modulate α-synuclein via its mRNA. In other words, the researchers targeted the building blocks long before the “undruggable” proteins were actually formed, thus reducing the buildup of α-synuclein proteins and improving dysregulated gene expression.

Study Results Point to Improved Gene Expression

The Scripps team ultimately found that the mRNA binding duo restored expression in about half of the dysregulated genes, likely due to a notable reduction in α-synuclein levels. Ideally, regulating the protein through mRNA binding would also reduce cellular stress, thus providing a means toward therapeutic intervention for Parkinson’s disease.

Parkinson’s disease research still has a long way to go; however, studies like this one prove just how important hyper-focused medical research can be to improving patient care.

Scantox is a part of Scantox, a GLP/GCP-compliant contract research organization (CRO) delivering the highest grade of Discovery, Regulatory Toxicology and CMC/Analytical services since 1977. Scantox focuses on preclinical studies related to central nervous system (CNS) diseases, rare diseases, and mental disorders. With highly predictive disease models available on site and unparalleled preclinical experience, Scantox can handle most CNS drug development needs for biopharmaceutical companies of all sizes. For more information about Scantox, visit www.scantox.com.