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J Clin Neurol. 2016 Oct;12(4):381-392. doi: 10.3988/jcn.2016.12.4.381.

Harnessing Cerebrospinal Fluid Biomarkers in Clinical Trials for Treating Alzheimer's and Parkinson's Diseases: Potential and Challenges.

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Department of Pharmacology and Medicinal Toxicology Research Center, Incheon, Korea.
Hypoxia-Related Diseases Research Center, Inha University School of Medicine, Incheon, Korea.
Department of Thoracic Surgery, Inha University Hospital, Inha University, Incheon, Korea.
Department of Emergency Medicine, Inje University Ilsan Paik Hospital, Goyang, Korea.
Hypoxia-Related Diseases Research Center, Inha University School of Medicine, Incheon, Korea.


No disease-modifying therapies (DMT) for neurodegenerative diseases (NDs) have been established, particularly for Alzheimer's disease (AD) and Parkinson's disease (PD). It is unclear why candidate drugs that successfully demonstrate therapeutic effects in animal models fail to show disease-modifying effects in clinical trials. To overcome this hurdle, patients with homogeneous pathologies should be detected as early as possible. The early detection of AD patients using sufficiently tested biomarkers could demonstrate the potential usefulness of combining biomarkers with clinical measures as a diagnostic tool. Cerebrospinal fluid (CSF) biomarkers for NDs are being incorporated in clinical trials designed with the aim of detecting patients earlier, evaluating target engagement, collecting homogeneous patients, facilitating prevention trials, and testing the potential of surrogate markers relative to clinical measures. In this review we summarize the latest information on CSF biomarkers in NDs, particularly AD and PD, and their use in clinical trials. The large number of issues related to CSF biomarker measurements and applications has resulted in relatively few clinical trials on CSF biomarkers being conducted. However, the available CSF biomarker data obtained in clinical trials support the advantages of incorporating CSF biomarkers in clinical trials, even though the data have mostly been obtained in AD trials. We describe the current issues with and ongoing efforts for the use of CSF biomarkers in clinical trials and the plans to harness CSF biomarkers for the development of DMT and clinical routines. This effort requires nationwide, global, and multidisciplinary efforts in academia, industry, and regulatory agencies to facilitate a new era.


Alzheimer's disease; Parkinson's disease; biomarker; cerebrospinal fluid; clinical trial; disease-modifying therapy

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