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Related Concept Videos

Clinical Trials: Overview01:11

Clinical Trials: Overview

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Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
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Neurodegenerative disorders are progressive diseases that cause irreversible damage and loss to neurons in specific brain areas. Examples of these disorders include Parkinson's disease, Alzheimer's disease, Multiple Sclerosis (MS), and Amyotrophic Lateral Sclerosis (ALS). These disorders share characteristics such as proteinopathies, selective neuronal vulnerability, and a complex interplay between genetic and environmental factors. The primary therapeutic goal for these conditions is...
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Progressive multiple sclerosis: Six trials to watch.

Maria A Rocca1, Paolo Preziosa1, Massimo Filippi2

  • 1Neuroimaging Research Unit, Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy; Neurology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.

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New therapies for progressive multiple sclerosis (PMS) target CNS inflammation. Six trials investigate Bruton's tyrosine kinase inhibitors, CD40-CD40L blockade, and CAR-T cells to treat microglia and B cell pathology.

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Area of Science:

  • Neuroimmunology
  • Clinical Therapeutics
  • Translational Medicine

Background:

  • Progressive multiple sclerosis (PMS) is characterized by CNS inflammation, driving disease progression independently of relapse activity.
  • Current treatment paradigms for PMS are limited, representing a significant unmet clinical need.
  • Emerging therapeutic strategies aim to address the underlying immunopathology of PMS.

Purpose of the Study:

  • To review emerging therapies targeting CNS-compartmentalized inflammation in progressive multiple sclerosis (PMS).
  • To highlight pivotal clinical trials evaluating novel immunomodulatory agents for PMS.
  • To discuss the potential of these therapies to redefine PMS treatment by targeting key cellular pathways.

Main Methods:

  • Review of six pivotal clinical trials for progressive multiple sclerosis (PMS).
  • Focus on therapies including Bruton's tyrosine kinase (BTK) inhibitors, CD40-CD40L blockade, and CD19-directed chimeric antigen receptor (CAR)-T cells.
  • Analysis of the mechanisms of action targeting microglia and B cell pathology.

Main Results:

  • Several novel therapeutic classes are under investigation for PMS.
  • These agents target distinct immune pathways implicated in neuroinflammation and neurodegeneration.
  • Early data suggests potential for disease modification in progressive forms of MS.

Conclusions:

  • Emerging therapies targeting CNS inflammation show promise for treating progressive multiple sclerosis (PMS).
  • Modulating microglia and B cell activity represents a key strategy for future PMS treatments.
  • These advancements may significantly improve outcomes for patients with unmet clinical needs in PMS.