Nisha Pandey

Health and Me

Drug-Resistant Epileptic Seizures Reduce By Over 90% With Experimental Gene Therapy

Experimental Gene Therapy Reduces Drug-Resistant Epileptic Seizures By Over 90%

An experimental <span>gene therapy</span> has shown tremendous potential in reducing the frequency of seizures in three patients with drug-resistant <span><a href="https://www.healthandme.com/health/epilepsy">epilepsy</a></span>.

The results have raised hopes for a new approach to treating epilepsy patients who do not respond to conventional anti-seizure medications.

All three participants experienced a reduction of more than 90% in their monthly seizure frequency after the treatment. No serious side effects were reported during the study.

The results were reported today in <em>The <span rel="noindex">New England Journal of Medicine</span></em> by Yang Dai, a neurosurgeon at <span>Xuanwu Hospital of Capital Medical University</span> in Beijing, and his colleagues. 

“It’s exciting that there’s such a dramatic effect, and that they see very few side effects,” says Matthew Walker, a neurologist at University College London who was not associated with the study.

<h2>How Does the Experimental Gene Therapy Work?</h2>

Also read: <a href="https://www.healthandme.com/health-news/tv-actor-anuj-thakur-dies-at-33-after-suffering-a-seizure-in-his-sleep-when-can-nocturnal-seizures-turn-fatal-article-156451146" data-type="tilCustomLink">TV Actor Anuj Thakur Dies At 33 After Suffering A Seizure In His Sleep: When Can Nocturnal Seizures Turn Fatal</a>

The treatment uses a modified virus called adeno-associated virus 9 (AAV9) to deliver genetic instructions directly into the part of the brain from where seizures stem.

The virus carries a gene that instructs specific neurons, known as excitatory neurons, to produce a receptor that are sensitive to a particular medication.

Once the gene is delivered, patients take clozapine, which is an antipsychotic medication. The drug activates the receptors, reducing the activity of the neurons and helping suppress the electrical signals that cause <a href="https://www.healthandme.com/health-news/swimmer-archie-goodburns-plea-prompts-uk-action-to-speed-up-brain-cancer-treatment-article-156346903" data-type="tilCustomLink">seizures</a>.

Researchers describe the medication as a kind of dimmer switch because it allows them to adjust the activity of the genetically modified brain cells.

Unlike conventional gene therapies, this approach gives researchers a way to regulate the treatment using medication.

<h2>What Did The Clinical Trial Find?</h2>

Also read: <a href="https://www.healthandme.com/health-news/woman-with-epilepsy-dies-after-following-chatgpts-advice-to-stop-medication-why-ai-cannot-replace-a-doctor-article-156425495" data-type="tilCustomLink">Woman With Epilepsy Dies After Following ChatGPT’s Advice To Stop Medication: Why AI Cannot Replace A Doctor</a>

The study was led by neurosurgeon Yang Dai of Xuanwu Hospital, Capital Medical University, in Beijing.

It involved three adults with drug-resistant <a href="https://www.healthandme.com/health-news/revolutionary-device-detects-brain-cancer-in-under-60-minutes-article-151036422" data-type="tilCustomLink">epilepsy</a> caused by hippocampal sclerosis, a condition in which scarring develops in the hippocampus, a brain region involved in memory and seizure activity.

All three participants had experienced epilepsy for 10 to 25 years. Despite trying at least two anti-seizure medications, their seizures still remained out of control.

They also experienced at least two seizures per month on average during the three months before entering the trial.

After gene therapy all three participants saw a reduction of more than 90% in their monthly seizure frequency.

<h2>Significant Discovery For People With Drug-Resistant Epilepsy</h2>

Also read: <a href="https://www.healthandme.com/health-wellness/can-chatgpt-replace-your-doctor-why-epilepsy-patients-should-never-alter-medication-on-ai-advice-article-156430331" data-type="tilCustomLink">Can ChatGPT Replace Your Doctor? Why Epilepsy Patients Should Never Alter Medication on AI Advice</a>

Epilepsy is a neurological disorder characterised by recurrent seizures caused by abnormal electrical activity in the brain.

Anti-seizure medications can help many people control their seizures. However, some patients continue to experience seizures despite trying multiple medications.

This is known as drug-resistant epilepsy. According to the <span>International League Against <a href="https://www.healthandme.com/health/epilepsy">Epilepsy</a></span>, it generally refers to a failure to achieve control of seizure after sufficient trials of two anti-seizure medications.

Surgery may be an option for patients with focal epilepsy if doctors can identify the brain region responsible for seizures.

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The new gene therapy takes a different approach by targeting the neurons responsible for seizures.

However, experts have raised concerns about the long-term use of clozapine. High doses of the drug have been associated with cardiovascular complications and other adverse effects.

Despite the reduction in seizures, researchers have warned against interpreting the findings as proof that the treatment is effective for all people with epilepsy.

The trial involved only three participants, making it too small to probe the treatment’s long-term safety or effectiveness.

All three also had a specific form of epilepsy related to hippocampal scarring on one side of the brain. The findings cannot automatically be applied to people with other forms of epilepsy or seizures originating in different brain regions.

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gene therapy

Epileptic seizure

epilepsy

epilepsy seizure

drug resistant epilepsy

xuanwu hospital of capital medical university

new england journal of medicine

international league against epilepsy

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An experimental gene therapy has shown tremendous potential in reducing the frequency of seizures in three patients with drug-resistant epilepsy. The results have raised hopes for a new approach to treating epilepsy patients who do not respond to conventional anti-seizure medications.All three participants experienced a reduction of more than 90% in their monthly seizure frequency after the treatment. No serious side effects were reported during the study.The results were reported today in The New England Journal of Medicine by Yang Dai, a neurosurgeon at Xuanwu Hospital of Capital Medical University in Beijing, and his colleagues. “It’s exciting that there’s such a dramatic effect, and that they see very few side effects,” says Matthew Walker, a neurologist at University College London who was not associated with the study.How Does the Experimental Gene Therapy Work?Also read: TV Actor Anuj Thakur Dies At 33 After Suffering A Seizure In His Sleep: When Can Nocturnal Seizures Turn FatalThe treatment uses a modified virus called adeno-associated virus 9 (AAV9) to deliver genetic instructions directly into the part of the brain from where seizures stem.The virus carries a gene that instructs specific neurons, known as excitatory neurons, to produce a receptor that are sensitive to a particular medication.Once the gene is delivered, patients take clozapine, which is an antipsychotic medication. The drug activates the receptors, reducing the activity of the neurons and helping suppress the electrical signals that cause seizures.Researchers describe the medication as a kind of dimmer switch because it allows them to adjust the activity of the genetically modified brain cells.Unlike conventional gene therapies, this approach gives researchers a way to regulate the treatment using medication.What Did The Clinical Trial Find?Also read: Woman With Epilepsy Dies After Following ChatGPT’s Advice To Stop Medication: Why AI Cannot Replace A DoctorThe study was led by neurosurgeon Yang Dai of Xuanwu Hospital, Capital Medical University, in Beijing.It involved three adults with drug-resistant epilepsy caused by hippocampal sclerosis, a condition in which scarring develops in the hippocampus, a brain region involved in memory and seizure activity.All three participants had experienced epilepsy for 10 to 25 years. Despite trying at least two anti-seizure medications, their seizures still remained out of control.They also experienced at least two seizures per month on average during the three months before entering the trial.After gene therapy all three participants saw a reduction of more than 90% in their monthly seizure frequency.Significant Discovery For People With Drug-Resistant EpilepsyAlso read: Can ChatGPT Replace Your Doctor? Why Epilepsy Patients Should Never Alter Medication on AI AdviceEpilepsy is a neurological disorder characterised by recurrent seizures caused by abnormal electrical activity in the brain.Anti-seizure medications can help many people control their seizures. However, some patients continue to experience seizures despite trying multiple medications.This is known as drug-resistant epilepsy. According to the International League Against Epilepsy, it generally refers to a failure to achieve control of seizure after sufficient trials of two anti-seizure medications.Surgery may be an option for patients with focal epilepsy if doctors can identify the brain region responsible for seizures. Limitations Of The Study   The new gene therapy takes a different approach by targeting the neurons responsible for seizures.However, experts have raised concerns about the long-term use of clozapine. High doses of the drug have been associated with cardiovascular complications and other adverse effects.Despite the reduction in seizures, researchers have warned against interpreting the findings as proof that the treatment is effective for all people with epilepsy.The trial involved only three participants, making it too small to probe the treatment’s long-term safety or effectiveness.All three also had a specific form of epilepsy related to hippocampal scarring on one side of the brain. The findings cannot automatically be applied to people with other forms of epilepsy or seizures originating in different brain regions.