Biomedical
The basics of preclinical drug development for neurodegenerative disease indications
Peer Reviewed
Abstract
The article "The basics of preclinical drug development for neurodegenerative disease indications" provides an overview of the essential steps involved in transitioning a drug candidate from laboratory discovery to human clinical trials. It emphasizes the importance of preclinical studies in identifying lead candidates, developing scalable production methods, selecting optimal formulations, and determining appropriate dosing regimens. The article also highlights the necessity of assessing pharmacokinetics, safety, and toxicity patterns using rodent and non-rodent mammalian models. Additionally, it discusses the critical role of understanding a drug's ability to cross the blood-brain barrier for treating brain-targeted diseases. The authors stress the need for adherence to regulatory guidelines, such as those set by the U.S. Food and Drug Administration (FDA) and the International Conference on Harmonisation, to ensure the safety and efficacy of new drugs.
Key Questions about Preclinical Drug Development for Neurodegenerative Diseases
1. What is the role of preclinical development in drug discovery?
Preclinical development bridges laboratory drug discovery and human clinical trials. It involves identifying lead candidates, developing scalable production methods, selecting optimal formulations, and determining dosing regimens. This phase also includes assessing pharmacokinetics, safety, and toxicity patterns using animal models.
2. How are pharmacokinetics and safety evaluated in preclinical studies?
Rodent and non-rodent mammalian models are utilized to delineate the pharmacokinetic profile and general safety of drug candidates. These studies help identify toxicity patterns and determine the drug's mean residence time in the body, which depends on absorption, distribution, metabolism, and excretion properties.
3. Why is the ability to cross the blood-brain barrier important?
For drugs intended to treat Alzheimer's disease or other brain-targeted diseases, the ability to cross the blood-brain barrier is crucial. This property determines the drug's effectiveness in reaching the central nervous system to exert therapeutic effects.
4. What regulatory guidelines govern preclinical development?
Preclinical development activities are governed by regulatory guidelines such as the U.S. Food and Drug Administration (FDA) Good Laboratory Practices and international standards, including those set by the International Conference on Harmonisation. Adherence to these guidelines ensures the safety and efficacy of new drugs.
5. What funding opportunities are available for preclinical development?
Various funding options are available for investigators seeking to advance their drug candidates. Government programs like the Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) grants, as well as the National Institutes of Health (NIH) Rapid Access to Interventional Development Pilot Program, provide funding and services to assist in preparing preclinical programs and documentation. Private foundations also increasingly fund preclinical work.
