Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Parkinson's Disease: Treatment01:24

Parkinson's Disease: Treatment

436
Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of...
436
Alzheimer's Disease: Treatment01:22

Alzheimer's Disease: Treatment

287
Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
287

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

At-Home Versus in-Clinic Vital Capacity Measurement: Insights From the HEALEY ALS Platform Trial.

Muscle & nerve·2026
Same author

Select Contemporary Statistical Concepts in Heart Failure Clinical Trials: Insights From the Heart Failure Collaboratory.

JACC. Heart failure·2026
Same author

Optimizing Research Operations and Resource Utilization in ALS Care: Insights From the Tofersen Antisense Oligonucleotide Expanded Access Protocol.

Muscle & nerve·2026
Same author

Vagal Nerve Stimulation in Patients With Heart Failure and Reduced Ejection Fraction: The ANTHEM-HFrEF Trial.

Journal of the American College of Cardiology·2026
Same author

Phosphorylated Tau at Threonine 181 Is Elevated in Amyotrophic Lateral Sclerosis Plasma.

Muscle & nerve·2026
Same author

Oral Nirmatrelvir-Ritonavir for Covid-19 in Higher-Risk Outpatients.

The New England journal of medicine·2026

Related Experiment Video

Updated: Oct 9, 2025

Establishment of an Electrophysiological Platform for Modeling ALS with Regionally-Specific Human Pluripotent Stem Cell-Derived Astrocytes and Neurons
11:52

Establishment of an Electrophysiological Platform for Modeling ALS with Regionally-Specific Human Pluripotent Stem Cell-Derived Astrocytes and Neurons

Published on: August 26, 2021

2.4K

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development.

Sabrina Paganoni1,2, James D Berry1, Melanie Quintana3

  • 1Sean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA.

Annals of Neurology
|December 22, 2021
PubMed
Summary

The Healey ALS Platform Trial accelerates amyotrophic lateral sclerosis (ALS) treatment discovery by testing multiple investigational drugs simultaneously. This adaptive approach aims to rapidly identify effective therapies and biomarkers for ALS patients.

More Related Videos

Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis ALS
12:35

Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis ALS

Published on: March 17, 2012

28.2K
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
15:48

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

Published on: July 29, 2007

9.8K

Related Experiment Videos

Last Updated: Oct 9, 2025

Establishment of an Electrophysiological Platform for Modeling ALS with Regionally-Specific Human Pluripotent Stem Cell-Derived Astrocytes and Neurons
11:52

Establishment of an Electrophysiological Platform for Modeling ALS with Regionally-Specific Human Pluripotent Stem Cell-Derived Astrocytes and Neurons

Published on: August 26, 2021

2.4K
Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis ALS
12:35

Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis ALS

Published on: March 17, 2012

28.2K
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
15:48

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

Published on: July 29, 2007

9.8K

Area of Science:

  • Neurology
  • Clinical Trials
  • Drug Development

Background:

  • Traditional amyotrophic lateral sclerosis (ALS) drug development uses costly, time-consuming single-product clinical trials.
  • Existing methods for testing ALS therapeutics lack flexibility and efficiency.
  • Adaptive platform trials offer a novel, continuous approach to investigate multiple interventions for a single disease.

Purpose of the Study:

  • To introduce the Healey ALS Platform Trial, a novel adaptive trial for amyotrophic lateral sclerosis.
  • To describe the methodology for concurrently testing multiple investigational products in ALS patients.
  • To highlight the trial's goal of accelerating the identification of effective ALS treatments, biomarkers, and endpoints.

Main Methods:

  • The Healey ALS Platform Trial employs an adaptive platform design.
  • Multiple investigational products are tested concurrently in individuals with ALS.
  • This approach allows for continuous enrollment and evaluation of treatments.

Main Results:

  • The trial is designed to rapidly identify promising therapeutic candidates for ALS.
  • Biomarker discovery and validation are integrated into the platform.
  • Novel trial endpoints are being explored to improve efficiency.

Conclusions:

  • Adaptive platform trials represent a significant advancement in ALS therapeutic development.
  • The Healey ALS Platform Trial offers a more efficient and flexible model for drug discovery in ALS.
  • This innovative approach has the potential to expedite the delivery of new treatments to patients with amyotrophic lateral sclerosis.