Revolutionary neurotherapeutic advances presented at the ASENT 2022 Annual Meeting
In the relentless pursuit of healing the human brain, a dedicated community of scientists is breaking new ground, one discovery at a time.
Imagine a world where a single injection can halt a progressive neurological disease by instructing the body's own cells to produce a healing protein. This is not science fiction; it is the promising reality of antisense oligonucleotide (ASO) therapy, one of the many revolutionary topics taking center stage at the ASENT 2022 Annual Meeting1 .
This gathering, a premier nexus for neurotherapeutics, brought together the world's leading researchers, clinicians, and industry pioneers. Their shared mission is as challenging as it is crucial: to accelerate the development of life-changing treatments for the millions affected by disorders of the brain and nervous system.
From rare genetic conditions to widespread neurodegenerative diseases, the meeting showcased a field on the cusp of a transformative era.
Single injection treatments that instruct cells to produce healing proteins
A significant hurdle in developing brain therapies is the difficulty of measuring whether a drug is working. A compelling in-depth session sponsored by SynapCell highlighted how electroencephalography (EEG) is being reinvented as a powerful, precise tool for drug discovery1 .
Far from the simple brainwave monitor many imagine, modern EEG-based "biomarker platforms" can objectively assess a new compound's activity, mechanism, and potential efficacy with high sensitivity.
Researchers presented a detailed case study on using EEG to discover a new anti-dyskinetic drug for Parkinson's disease1 . The experiment followed a meticulous process:
Researchers first established a reliable animal model of Parkinson's disease that exhibits levodopa-induced dyskinesia—the involuntary movements that are a common side effect of long-term treatment.
Before administering the experimental drug, a baseline EEG was recorded. This measured the specific brainwave patterns, or biomarkers, associated with the dyskinetic state.
The new neurotherapeutic compound was then administered to the model. Following this, researchers conducted continuous EEG recordings to track changes in the brain's electrical activity in real-time.
The post-drug EEG data was rigorously analyzed and compared to the baseline. Sophisticated software identified statistically significant changes in the predefined biomarkers.
Crucially, the changes in EEG biomarkers were correlated with direct observation and measurement of the model's physical dyskinetic movements, confirming that the electrical brain signal was a true proxy for therapeutic effect.
The results were clear and promising. The experimental compound produced a significant and measurable change in the specific EEG biomarkers for dyskinesia1 . This objective data demonstrated that the drug was engaging its intended target in the brain and having a biological effect.
For the field, this validation is transformative. It means that instead of relying solely on subjective clinical observations in late-stage trials, researchers can now use these precise EEG biomarkers early in the drug discovery process.
They can quickly determine if a compound is likely to work, investigate how it is working, and make informed decisions about which therapies to advance, thereby accelerating the entire pipeline from lab to patient.
Estimated improvement in decision-making accuracy
| EEG Biomarker | Neurological Condition | Role in Drug Discovery |
|---|---|---|
| Auditory Steady-State Response (ASSR) | Schizophrenia | Measures neural synchrony; used to test drugs that aim to improve brain circuit function1 . |
| Beta/Gamma Oscillations | Parkinson's Disease (Dyskinesia) | Tracks abnormal rhythms linked to involuntary movements; assesses efficacy of anti-dyskinetic drugs1 . |
| Spike-Wave Discharges | Epilepsy | Quantifies seizure-related brain activity; used to screen potential anti-epileptic compounds1 . |
The advances presented at ASENT are powered by a sophisticated arsenal of research tools. The following details some of the key reagents and solutions fundamental to this work, explaining their critical function in the quest for new therapies.
Synthetically designed RNA-like molecules that can modulate gene expression.
Measurable indicators of a biological state or condition.
Modified viruses used as vehicles to deliver genetic material into cells.
Cells grown in a controlled environment to model disease or test compounds.
A major focus of the 2022 meeting was the dramatic progress in treating rare neurological disorders, an area long considered medically neglected. The Presidential Symposium, held in partnership with the National Organization for Rare Disorders (NORD), highlighted how personalized, gene-targeted therapies are now offering hope for conditions that once had none1 .
Speakers like Dr. Timothy Yu from Boston Children's Hospital discussed pioneering "N=1" studies, where a custom ASO therapy is designed for a single, unique patient—a truly bespoke medical approach1 .
Simultaneously, researchers are applying these advanced platforms to tackle widespread neurodegenerative diseases like Alzheimer's and Parkinson's. Another key session explored the emerging frontier of brain lipid pharmacology1 .
Given that lipids make up about 50% of the brain's dry weight, understanding their role is critical. Presenters shared work on drugs that target lipid pathways, such as activating specific enzymes or inhibiting others, to slow neurodegeneration and reduce inflammation1 .
| Therapeutic Approach | Mechanism of Action | Target Conditions |
|---|---|---|
| ASO Therapy | Binds to RNA to alter protein production, potentially correcting genetic defects. | Spinal Muscular Atrophy, ALS, other rare genetic disorders1 . |
| Gene Therapy | Uses viral vectors to deliver a functional gene to compensate for a non-functional one. | Various inherited neurological and neuromuscular diseases1 . |
| Lipid-Targeting Small Molecules | Modifies the brain's lipid environment to reduce toxicity and improve neuron health. | Parkinson's disease (GBA subtype), Alzheimer's disease1 . |
| Prescription Digital Therapeutics | Software-based interventions to manage disease symptoms or behavior. | Substance Use Disorder, Opioid Use Disorder1 . |
The path from a promising experimental result to an approved treatment remains long and complex. Sessions on regulatory science and drug development barriers acknowledged these challenges, particularly for complex neurodevelopmental diseases1 . However, the overwhelming sentiment was one of optimism, driven by data and technological convergence.
The collective work presented at ASENT 2022 paints a picture of a field evolving from generalized approaches to a future of precision neurology. The continued synergy between cutting-edge tools like EEG biomarkers, genetic technologies like ASOs, and a deeper understanding of fundamental brain biology is creating an unprecedented opportunity to develop effective therapies.
For patients and families waiting, this progress, showcased in the vibrant community of ASENT, signals that new horizons of hope are within reach.
For further reading on the latest in neurotherapeutics and to learn about upcoming meetings, you can visit the official ASENT website at www.asent.org4 .