Nisha Pandey

Alexander Disease: FDA Approves First Drug That Can Target The Root Cause Of The Rare Neurological Disease

Alexander Disease: FDA Approves First Drug That Can Target The Root Cause Of Rare Neurological Disease

The US Food and Drug Administration (FDA) recently approved the first drug for <span>Alexander disease</span>, a rare and progressive neurological disorder. Alexander disease is marked by symptoms like seizures, slow development, muscle weakness, difficulty in walking and problems with speech and swallowing.

The FDA approved <span>Zanvastro</span> (<span>zilganersen</span>) for children and adults afflicted with Alexander disease. Being the first approved treatment for the condition, the drug is designed to directly target the abnormal protein buildup, one of the major factors that drives the disease.

Talking about the landmark drug, Emily Freilich, MD, Director of the Division of Neurology I at the FDA’s Center for Drug Evaluation and Research said, “For patients with Alexander disease and their families, there have been no approved treatment options, only supportive care while the disease progresses. Today’s approval is a landmark moment for this community, offering the first therapy that addresses the underlying cause of this rare and serious disease.”

According to the FDA, Alexander disease is extremely rare as it affects fewer than one in a million people. Alexander disease is a <a href="https://www.healthandme.com/parenting/6-year-old-australian-kid-dies-from-rarely-seen-19th-century-disease-why-is-scurvy-still-a-risk-today-article-156036144" data-type="tilCustomLink">rare, genetic neurodegenerative disorder</a> that is caused due to mutations in the GFAP gene. GFAP gene promotes the production of glial fibrillary acidic protein, a type of protein found in the central nervous system.

GFAP is found primarily in astrocytes, a type of specialised cells in the brain and spinal cord that support and protect nerve cells. 

In Alexander disease, abnormal GFAP accumulates inside these cells. This leads to formation of cumulative protein called Rosenthal fibres. This disrupts the normal function of astrocytes and ultimately damages the nervous system.

In simple terms, the issue is not that the brain suddenly loses a particular protein. It is that a genetically altered version of GFAP is produced and progressively accumulates where it should not. This is why <a href="https://www.healthandme.com/health-news/flesh-eating-screwworm-threat-increases-in-us-why-illegal-horse-trade-is-raising-alarm-article-156100652" data-type="tilCustomLink">Alexander disease</a> is considered an astrogliopathy, a disorder which is primarily driven by astrocyte dysfunction.

Also read: <a href="https://www.healthandme.com/health-news/hhs-announces-us-fdas-first-ai-chief-heres-what-it-means-for-the-future-of-drug-regulation-article-156122844" data-type="tilCustomLink">HHS Announces US FDA’s First AI Chief: Here’s What It Means For The Future Of Drug Regulation</a>

The disease can present very differently depending on the timing of its onset. In infants and young children, it can cause delay in development, loss of previously acquired essential skills, seizures, unusually large head, muscle weakness, feeding difficulties and problems with movement. Some severe early-onset cases may progress rapidly.

Juvenile and adult forms can be slower and may involve speech and swallowing difficulties, abnormal way of walking, muscle stiffness, poor coordination, weakness, breathing or sleep problems and autonomic dysfunction.

As symptoms of Alexander disease may overlap with many other neurological disorders, its diagnosis remains challenging. Doctors typically diagnose it by combining key findings in brain MRI with genetic testing for a disease-causing <a href="https://www.healthandme.com/health-news/84-cancer-patients-report-benefit-from-ivermectin-mebendazole-what-the-study-found-why-more-trials-are-needed-article-156034007" data-type="tilCustomLink">GFAP variant</a>.

Also read: <a href="https://www.healthandme.com/health-news/small-cell-lung-cancer-is-one-of-the-hardest-cancers-to-treat-what-this-new-drug-combination-could-change-article-156122746" data-type="tilCustomLink">Small-Cell Lung Cancer Is One Of The Hardest Cancers To Treat: What This New Drug Combination Could Change</a>

<h2>What Makes The New Drug Different?</h2>

Until now, the treatment for Alexander Disease has largely focused on managing individual symptoms, like seizures, feeding problems and difficulties with movement, speech or swallowing. 

Taking a different approach, <a href="https://www.healthandme.com/health-news/small-cell-lung-cancer-is-one-of-the-hardest-cancers-to-treat-what-this-new-drug-combination-could-change-article-156122746" data-type="tilCustomLink">Zanvastro</a>, a type of RNA-targeted medicine is designed to reduce production of the abnormal GFAP protein. 

Its goal is to lower the amount of disease-driving protein before it can continue accumulating and damaging astrocytes. The drug is administered once every three months as an injection into the spinal canal.

Also read: <a href="https://www.healthandme.com/health-news/leptospirosis-cases-surge-in-manila-after-floods-what-to-know-article-156121295" data-type="tilCustomLink">Leptospirosis Cases Surge In Manila After Floods: What To Know</a>

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The <a href="https://www.healthandme.com/health-news/fda-approves-new-breast-cancer-treatment-that-targets-resistance-before-cancer-progresses-article-156100654" data-type="tilCustomLink">FDA's</a> approval arrived after a controlled study involving 49 patients aged two and older, along with an open-label substudy involving four children younger than two.

Among patients aged five and older who already had walking difficulties, those receiving Zanvastro had significantly better walking speed after 61 weeks than others.

For children aged two to four, researchers used broader measures of motor development because walking speed alone is not a reliable measure at that age. Those children showed improvement in motor skills, while the control group declined.

The most common side effects included vomiting, back pain, cough, headache and post-lumbar-puncture syndrome. The FDA also warned that aseptic meningitis has been reported.

Alexander disease has long been a condition in which doctors treated complications but had no approved therapy that aimed at the biological process causing the disease. 

Zanvastro does not reverse genetic mutations themselves. Instead, it targets one of the consequences of those mutations by reducing production of abnormal GFAP.

As Ionis Pharmaceuticals CEO Brett P. Monia said following the approval, the treatment allows the field to move “beyond managing individual manifestations of the disease to addressing its underlying biology.”

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The US Food and Drug Administration (FDA) recently approved the first drug for Alexander disease, a rare and progressive neurological disorder. Alexander disease is marked by symptoms like seizures, slow development, muscle weakness, difficulty in walking and problems with speech and swallowing.The FDA approved Zanvastro (zilganersen) for children and adults afflicted with Alexander disease. Being the first approved treatment for the condition, the drug is designed to directly target the abnormal protein buildup, one of the major factors that drives the disease.Talking about the landmark drug, Emily Freilich, MD, Director of the Division of Neurology I at the FDA’s Center for Drug Evaluation and Research said, “For patients with Alexander disease and their families, there have been no approved treatment options, only supportive care while the disease progresses. Today’s approval is a landmark moment for this community, offering the first therapy that addresses the underlying cause of this rare and serious disease.”What Is Alexander Disease?According to the FDA, Alexander disease is extremely rare as it affects fewer than one in a million people. Alexander disease is a rare, genetic neurodegenerative disorder that is caused due to mutations in the GFAP gene. GFAP gene promotes the production of glial fibrillary acidic protein, a type of protein found in the central nervous system.GFAP is found primarily in astrocytes, a type of specialised cells in the brain and spinal cord that support and protect nerve cells. In Alexander disease, abnormal GFAP accumulates inside these cells. This leads to formation of cumulative protein called Rosenthal fibres. This disrupts the normal function of astrocytes and ultimately damages the nervous system.In simple terms, the issue is not that the brain suddenly loses a particular protein. It is that a genetically altered version of GFAP is produced and progressively accumulates where it should not. This is why Alexander disease is considered an astrogliopathy, a disorder which is primarily driven by astrocyte dysfunction.Also read: HHS Announces US FDA’s First AI Chief: Here’s What It Means For The Future Of Drug RegulationSymptoms Of Alexander DiseaseThe disease can present very differently depending on the timing of its onset. In infants and young children, it can cause delay in development, loss of previously acquired essential skills, seizures, unusually large head, muscle weakness, feeding difficulties and problems with movement. Some severe early-onset cases may progress rapidly.Juvenile and adult forms can be slower and may involve speech and swallowing difficulties, abnormal way of walking, muscle stiffness, poor coordination, weakness, breathing or sleep problems and autonomic dysfunction.As symptoms of Alexander disease may overlap with many other neurological disorders, its diagnosis remains challenging. Doctors typically diagnose it by combining key findings in brain MRI with genetic testing for a disease-causing GFAP variant.Also read: Small-Cell Lung Cancer Is One Of The Hardest Cancers To Treat: What This New Drug Combination Could ChangeWhat Makes The New Drug Different?Until now, the treatment for Alexander Disease has largely focused on managing individual symptoms, like seizures, feeding problems and difficulties with movement, speech or swallowing. Taking a different approach, Zanvastro, a type of RNA-targeted medicine is designed to reduce production of the abnormal GFAP protein. Its goal is to lower the amount of disease-driving protein before it can continue accumulating and damaging astrocytes. The drug is administered once every three months as an injection into the spinal canal.Also read: Leptospirosis Cases Surge In Manila After Floods: What To KnowZanvastro's FDA Approval          The FDA's approval arrived after a controlled study involving 49 patients aged two and older, along with an open-label substudy involving four children younger than two.Among patients aged five and older who already had walking difficulties, those receiving Zanvastro had significantly better walking speed after 61 weeks than others.For children aged two to four, researchers used broader measures of motor development because walking speed alone is not a reliable measure at that age. Those children showed improvement in motor skills, while the control group declined.The most common side effects included vomiting, back pain, cough, headache and post-lumbar-puncture syndrome. The FDA also warned that aseptic meningitis has been reported.Alexander disease has long been a condition in which doctors treated complications but had no approved therapy that aimed at the biological process causing the disease. Zanvastro does not reverse genetic mutations themselves. Instead, it targets one of the consequences of those mutations by reducing production of abnormal GFAP.As Ionis Pharmaceuticals CEO Brett P. Monia said following the approval, the treatment allows the field to move “beyond managing individual manifestations of the disease to addressing its underlying biology.”

alexander disease: fda approves first drug that can target the root cause of the rare neurological disease

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Alexander Disease: FDA Approves First Drug That Can Target The Root Cause Of The Rare Neurological Disease

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