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

Alzheimer's Disease: Treatment01:22

Alzheimer's Disease: Treatment

565
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...
565
Alzheimer's Disease: Overview01:26

Alzheimer's Disease: Overview

1.1K
Alzheimer's Disease (AD) is a continually advancing neurodegenerative disorder, distinguished by escalating memory loss, cognitive dysfunction, and dementia. The disease unfolds in three stages: preclinical, mild cognitive impairment (MCI), and dementia. Its onset is insidious, and the progression gradual, with the cause not well explained by other disorders.
The clinical diagnosis of AD hinges on the presence of memory and other cognitive impairments. Biomarkers, such as changes in Aβ...
1.1K

You might also read

Related Articles

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

Sort by
Same author

Life-course BMI trajectories and long-term weight gain in relation to hepatic steatosis in a rural community-based cohort in Southwest China.

Frontiers in public health·2026
Same author

Corrigendum to 'Wavelength-dependent photobiomodulation attenuates synovial inflammation in fibroblast-like synoviocytes and a collagenase-induced osteoarthritis model' [Journal of Photochemistry & Photobiology B: Biology Volume 272 (2025) 113276].

Journal of photochemistry and photobiology. B, Biology·2026
Same author

Trajectories of brain structure and function in young adult carriers of genetic frontotemporal dementia variants.

medRxiv : the preprint server for health sciences·2026
Same author

Highly diverse diazotrophs drive high N<sub>2</sub> fixation rates in a shallow submarine hydrothermal system.

Fundamental research·2026
Same author

Feasibility and Preliminary Effects of Inspiratory Muscle Training in Patients With Knee Osteoarthritis Awaiting Total Knee Arthroplasty: An 8-Week, Pilot Randomized Controlled Trial.

Archives of physical medicine and rehabilitation·2026
Same author

Plasma p-tau217 and glucose metabolism correlate in neocortical association areas in Alzheimer's disease.

Brain communications·2026

Related Experiment Video

Updated: Nov 15, 2025

Assessment of Spontaneous Alternation, Novel Object Recognition and Limb Clasping in Transgenic Mouse Models of Amyloid-&#946; and Tau Neuropathology
10:02

Assessment of Spontaneous Alternation, Novel Object Recognition and Limb Clasping in Transgenic Mouse Models of Amyloid-β and Tau Neuropathology

Published on: May 28, 2017

27.6K

Value-Generating Exploratory Trials in Neurodegenerative Dementias.

Lauren G Friedman1, Nicholas McKeehan1, Yuko Hara1

  • 1From the Alzheimer's Drug Discovery Foundation (L.G.F., N.M., Y.H., M.O., A.T., H.M.F.), New York; Chambers-Grundy Center for Transformative Neuroscience (J.L.C.), Department of Brain Health, School of Integrated Health Sciences, University of Nevada Las Vegas; ADM Diagnostics (D.C.M.), Inc. Northbrook, IL; Servier Pharmaceuticals (J.Z.), Boston, MA; Global Alzheimer's Platform Foundation (R.C.M.), Washington, DC; AgeneBio (R.C.M.), Inc. Baltimore, MD; AbbVie Inc. (D.W.), North Chicago, IL; Pentara Corporation (S.B.H., S.P.D.), Salt Lake City, UT; Berry Consultants (M.Q.), Austin TX; Keck School of Medicine of the University of Southern California (L.S.S.), Los Angeles; Global R&D Partners (M.G.), LLC, University of California, San Diego, La Jolla; Department of Neurosciences (H.H.F.), University of California, San Diego, La Jolla; Albert Einstein College of Medicine (J.J.), Bronx, NY; CognitionMetrics (J.J.), LLC; Department of Clinical Neurological Sciences and Robarts Research Institute (E.C.F.), Schulich School of Medicine and Dentistry, University of Western Ontario; Parkwood Institute (E.C.F.), Lawson Health Research Institute, St. Josephs Health Care, London, Ontario, Canada; Rodin Therapeutics (J.M.R.), Boston, MA; Association for Frontotemporal Degeneration (D.N., S.L.-J.D.), Radnor, PA; and Modus Outcomes LLP (J.T.M.), Cambridge, MA.

Neurology
|March 6, 2021
PubMed
Summary

Exploratory clinical trials in neurodegenerative dementias, like Alzheimer disease, use biomarker outcomes to assess drug efficacy early. This approach helps identify promising treatments and stop ineffective ones, saving resources for drug development.

More Related Videos

Basics of Multivariate Analysis in Neuroimaging Data
06:35

Basics of Multivariate Analysis in Neuroimaging Data

Published on: July 24, 2010

17.1K
Abbiategrasso Brain Bank Protocol for Collecting, Processing and Characterizing Aging Brains
12:28

Abbiategrasso Brain Bank Protocol for Collecting, Processing and Characterizing Aging Brains

Published on: June 3, 2020

17.8K

Related Experiment Videos

Last Updated: Nov 15, 2025

Assessment of Spontaneous Alternation, Novel Object Recognition and Limb Clasping in Transgenic Mouse Models of Amyloid-&#946; and Tau Neuropathology
10:02

Assessment of Spontaneous Alternation, Novel Object Recognition and Limb Clasping in Transgenic Mouse Models of Amyloid-β and Tau Neuropathology

Published on: May 28, 2017

27.6K
Basics of Multivariate Analysis in Neuroimaging Data
06:35

Basics of Multivariate Analysis in Neuroimaging Data

Published on: July 24, 2010

17.1K
Abbiategrasso Brain Bank Protocol for Collecting, Processing and Characterizing Aging Brains
12:28

Abbiategrasso Brain Bank Protocol for Collecting, Processing and Characterizing Aging Brains

Published on: June 3, 2020

17.8K

Area of Science:

  • Neuroscience
  • Clinical Pharmacology
  • Drug Development

Background:

  • Alzheimer disease and frontotemporal dementia drug development face challenges due to high failure rates in late-stage clinical trials.
  • Shared pathologies and similar challenges in early-phase trial design hinder predictive success for neurodegenerative dementias.
  • Traditional phase 2 and 3 trials often fail to demonstrate efficacy, leading to significant resource expenditure.

Purpose of the Study:

  • To discuss the role and design of exploratory clinical trials in neurodegenerative dementias.
  • To highlight how early-phase trials (phase 1b/2a) can improve drug development efficiency.
  • To provide recommendations for optimizing exploratory trial design and application.

Main Methods:

  • Focus on exploratory trials (phase 1b/2a) assessing pharmacologic effects using biomarker outcomes.
  • Utilize shorter treatment durations and fewer patients compared to traditional phase 2 studies.
  • Leverage data from advisory panels convened by the Alzheimer's Drug Discovery Foundation and the Association for Frontotemporal Degeneration.

Main Results:

  • Exploratory trials can establish go/no-go decision points, supporting proof of concept and dose selection.
  • These trials identify drugs lacking target engagement, suitable exposure, or acceptable safety profiles early on.
  • Early termination of ineffective drugs conserves valuable resources, particularly for academic and small biotechnology programs.

Conclusions:

  • Exploratory studies provide a staged approach to clinical development, offering crucial data for advancing compounds.
  • Optimized exploratory trial design can significantly improve efficiency and reduce costs in drug development.
  • Recommendations on outcome measures and statistical considerations are vital for enhancing the success of neurodegenerative dementia drug discovery.