Advancements in ALS Diagnosis and Prognosis
Event Type
Research Presentation
Academic Department
Physics
Location
Dana Science Building, 2nd floor
Start Date
26-4-2024 1:30 PM
End Date
26-4-2024 3:00 PM
Description
Under the direction of Dr. Brian Gentry
Amyotrophic Lateral Sclerosis (ALS) is a severe neurodegenerative disease affecting motor neurons, leading to progressive muscle weakness, paralysis, and eventual death, with an average life expectancy of 2-5 years post-diagnosis. Currently, ALS affects approximately 30,000 Americans, with an estimated 450,000 to 600,000 individuals globally living with this condition, resulting in approximately 12 cases per 100,000 individuals worldwide. Despite its severity, ALS remains challenging to definitively diagnose, often resulting in delayed intervention and limited treatment options that primarily aim to manage symptoms rather than halting or reversing disease progression. Moreover, these treatments are typically invasive and provide only modest effectiveness. Recent studies have highlighted the effectiveness of NfL, a protein found in nerve cells, as a diagnostic biomarker for ALS, showing elevated levels in cerebrospinal fluid and blood samples of ALS patients compared to healthy controls. This biomarker aids in early and precise prognosis, complementing metabolic approaches for treatment. Metabolic approaches, such as small molecule drugs and gene therapies, show promise in delivering targeted treatment to ALS-affected tissues by using nanoparticles in the brain and spinal cord. Early preclinical studies show promising results, suggesting motor function improvements and extended survival in ALS animal models following metabolic interventions. Therefore, using NfL as a diagnostic biomarker for early detection of ALS, along with nanoparticle-based metabolic treatments, offers significant potential for improving patient outcomes by enabling early intervention and targeted therapeutic delivery to ALS-affected tissues, potentially enhancing treatment effectiveness and extending survival rates.
Advancements in ALS Diagnosis and Prognosis
Dana Science Building, 2nd floor
Under the direction of Dr. Brian Gentry
Amyotrophic Lateral Sclerosis (ALS) is a severe neurodegenerative disease affecting motor neurons, leading to progressive muscle weakness, paralysis, and eventual death, with an average life expectancy of 2-5 years post-diagnosis. Currently, ALS affects approximately 30,000 Americans, with an estimated 450,000 to 600,000 individuals globally living with this condition, resulting in approximately 12 cases per 100,000 individuals worldwide. Despite its severity, ALS remains challenging to definitively diagnose, often resulting in delayed intervention and limited treatment options that primarily aim to manage symptoms rather than halting or reversing disease progression. Moreover, these treatments are typically invasive and provide only modest effectiveness. Recent studies have highlighted the effectiveness of NfL, a protein found in nerve cells, as a diagnostic biomarker for ALS, showing elevated levels in cerebrospinal fluid and blood samples of ALS patients compared to healthy controls. This biomarker aids in early and precise prognosis, complementing metabolic approaches for treatment. Metabolic approaches, such as small molecule drugs and gene therapies, show promise in delivering targeted treatment to ALS-affected tissues by using nanoparticles in the brain and spinal cord. Early preclinical studies show promising results, suggesting motor function improvements and extended survival in ALS animal models following metabolic interventions. Therefore, using NfL as a diagnostic biomarker for early detection of ALS, along with nanoparticle-based metabolic treatments, offers significant potential for improving patient outcomes by enabling early intervention and targeted therapeutic delivery to ALS-affected tissues, potentially enhancing treatment effectiveness and extending survival rates.