Novel Developments to Enable Treatment of CNS Diseases with Targeted Drug Delivery.

Authors: Meyer AH, Feldsien TM, Mezler M, Untucht C, Venugopalan R, Lefebvre DR

The blood-brain barrier (BBB) is a major hurdle for the development of systemically delivered drugs against diseases of the central nervous system (CNS). Because of this barrier there is still a huge unmet need for the treatment of these diseases, despite years of research efforts across the pharmaceutical industry. Novel therapeutic entities, such as gene therapy and degradomers, have become increasingly popular in recent years, but have not been the focus for CNS indications so far. To unfold their full potential for the treatment of CNS diseases, these therapeutic entities will most likely have to rely on innovative delivery technologies. Here we will describe and assess approaches, both invasive and non-invasive, that can enable, or at least increase, the probability of a successful drug development of such novel therapeutics for CNS indications.

Introduction

Purpose Other

Study Objective To describe and assess invasive and non-invasive approaches for delivering novel therapeutics (e.g., gene therapies and degradomers) across the blood–brain barrier to improve drug development for CNS diseases.

Outcomes and Safety

Summary of Outcomes This review did not report specific biological or behavioral effects from experiments but summarized invasive and non‑invasive approaches to improve CNS drug delivery, and it did not test or identify any focused ultrasound parameters as successful.

Safety-related matter The provided text does not mention any safety concerns or adverse effects; it focuses on delivery challenges for CNS therapeutics and approaches (invasive and non-invasive) to improve drug development.

Brain Region

Visualization unavailable

Ultrasound Parameters

Focal Characteristics Focal depth: None, Focal length: None, Aperture size: None

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