Therapeutic strategies in neurodegenerative diseases

Kristi M. Anderson, Mosley Lee

Research output: Chapter in Book/Report/Conference proceedingChapter

3 Scopus citations


Until the 1990s, progress in developing therapy for neurodegenerative diseases was slow and there were few clinical trials. However, with the advent of animal models and the recent advances in understanding the basic pathophysiological mechanisms underlying the diseases, potential therapies to prevent, delay the onset or slow the progression of neurodegenerative disease are being identified at an ever increasing rate. High throughput technologies are being used to screen large numbers of potential therapeutic agents and new developments in the realms of immune modulation, RNA interference, viral vector delivery of gene products and stem cell therapy hold great promise for the future. There are still many unanswered questions regarding the mechanisms of disease and why beneficial therapy in animal models has not translated well into human clinical trials. Neurodegenerative diseases are rare, and so identifying enough patients to obtain studies with adequate power has been difficult. Novel phase II designs are now being used to screen greater numbers of agents and to better define correct dosing before proceeding to phase III trials. Multicenter phase III trials are being designed with adequate power, and using meaningful validated outcome measures that reduce the high dropout rates of past trials. Agents are now being tested in combination in order to detect possible additive effects. Focus is also being given to the need to better define the best symptomatic therapies in randomized controlled trials. Further research will prompt more targeted therapies that we hope will soon provide truly meaningful breakthroughs for neurodegenerative disorders.

Original languageEnglish (US)
Title of host publicationNeuroimmune Pharmacology
PublisherSpringer International Publishing
Number of pages31
ISBN (Electronic)9783319440224
ISBN (Print)9783319440200
StatePublished - Jan 1 2016


  • Alzheimer’s disease
  • Amyotrophic lateral sclerosis
  • Animal models
  • Apoptosis
  • Clinical trials
  • Combination therapy
  • Dementia
  • Huntington’s disease
  • Inflammation
  • Neurodegeneration
  • Neuroprotection
  • Parkinson’s disease
  • RNA interference
  • Randomized controlled trials
  • Stem cell therapy
  • Symptomatic therapy
  • Therapeutics
  • Vaccination

ASJC Scopus subject areas

  • Medicine(all)
  • Immunology and Microbiology(all)
  • Neuroscience(all)
  • Pharmacology, Toxicology and Pharmaceutics(all)


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