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Clinical care and therapeutic trials in PLS
Mary Kay Floeter1, Deborah Warden2, Dale Lange3
1National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration
|February 19, 2021
Summary
Primary lateral sclerosis (PLS) is a rare neurological disorder causing progressive motor neuron loss. Current research focuses on personalized multidisciplinary care and innovative clinical trials for symptomatic and disease-modifying treatments.
Area of Science:
- Neurology
- Neurodegenerative Diseases
- Clinical Trials
Background:
- Primary lateral sclerosis (PLS) is an ultra-rare central nervous system disorder.
- Characterized by progressive upper motor neuron degeneration, leading to significant limb and bulbar dysfunction.
- Spasticity is a core, often refractory, symptom, representing a major unmet clinical need.
Purpose of the Study:
- To outline the clinical care requirements for PLS patients.
- To highlight the necessity of a personalized, multidisciplinary management approach.
- To review recent advancements in PLS clinical trials, including symptomatic and disease-modifying therapies.
Main Methods:
- Review of clinical care needs and management strategies for PLS.
- Analysis of progress in ongoing and emerging clinical trials for PLS.
- Focus on advances in symptomatic treatments and disease-modifying therapies.
Main Results:
- Clinical care for PLS necessitates a personalized, multidisciplinary approach.
- Progress is being made in symptomatic treatments, particularly for spasticity.
- Innovative clinical trials are exploring both symptomatic and disease-modifying interventions.
Conclusions:
- Multidisciplinary care is essential for managing the substantial burden of PLS.
- Advancements in clinical trials offer hope for improved treatments for PLS.
- Further research into symptomatic and disease-modifying therapies is crucial for PLS patients.
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