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A High Throughput, Multiplexed and Targeted Proteomic CSF Assay to Quantify Neurodegenerative Biomarkers and Apolipoprotein E Isoforms Status
Published on: October 20, 2016
Central nervous system drug development: an integrative biomarker approach toward individualized medicine
B Gomez-Mancilla1, E Marrer, J Kehren
1Neuroscience-Biomarker Development, Novartis Pharma, CH-4002 Basel, Switzerland. baltazar.gomezmancilla@novartis.com
Abstract:
Drug development for CNS disorders faces the same formidable hurdles as other therapeutic areas: escalating development costs; novel drug targets with unproven therapeutic potential; and health care systems and regulatory agencies demanding more compelling demonstrations of the value of new drug products. Extensive clinical testing remains the core of registration of new compounds; however, traditional clinical trial methods are falling short in overcoming these development hurdles. The most common CNS disorders targeted for drug treatment are chronic, slowly vitiating processes manifested by highly subjective and context dependent signs and symptoms. With the exception of a few rare familial degenerative disorders, they have ill-defined or undefined pathophysiology. Samples selected for treatment trials using clinical criteria are inevitably heterogeneous, and dependence on traditional endpoints results in early proof-of-concept trials being long and large, with very poor signal to noise. It is no wonder that pharmaceutical and biotechnology companies are looking to biomarkers as an integral part of decision-making process supported by new technologies such as genetics, genomics, proteomics, and imaging as a mean of rationalizing CNS drug development. The present review represent an effort to illustrate the integration of such technologies in drug development supporting the path of individualized medicine.
Insights
Biomarkers and new technologies like genomics can help overcome challenges in central nervous system (CNS) drug development. These tools aid in rationalizing development and paving the way for personalized medicine approaches.
Area of Science:
- Neuroscience
- Pharmacology
- Biotechnology
Background:
- Central nervous system (CNS) drug development faces significant hurdles including high costs, unproven targets, and demands for value demonstration.
- Traditional clinical trials struggle with the complexity of CNS disorders, characterized by subjective symptoms, heterogeneous patient populations, and poorly defined pathophysiology.
- The limitations of conventional methods necessitate innovative approaches to improve the efficiency and success rate of CNS drug development.
Purpose of the Study:
- To review the integration of novel technologies and biomarkers in CNS drug development.
- To illustrate how these advancements can rationalize the development process and support personalized medicine.
- To address the challenges posed by the complexity and heterogeneity of CNS disorders in clinical trials.
Main Methods:
- Review of current literature on CNS drug development challenges.
- Exploration of the role of biomarkers in clinical decision-making.
- Discussion of enabling technologies such as genetics, genomics, proteomics, and imaging.
- Analysis of the shift towards individualized medicine in CNS therapeutics.
Main Results:
- Biomarkers and advanced technologies offer solutions to the inherent difficulties in CNS drug development.
- These tools enhance the rationalization of drug development pathways.
- Integration of these technologies supports the progression towards personalized medicine for CNS disorders.
- Improved signal-to-noise ratio in early-stage trials through biomarker utilization.
Conclusions:
- Biomarkers and new technologies are crucial for overcoming CNS drug development hurdles.
- The integration of genetics, genomics, proteomics, and imaging is key to rationalizing development.
- These advancements facilitate the move towards individualized medicine for CNS disorders.
- Adoption of these strategies can lead to more efficient and successful drug development.
