Related Experiment Video
Updated: May 13, 2026

Nanoparticle Delivery of an Oligonucleotide Payload in a Glioblastoma Multiforme Animal Model
Published on: September 27, 2024
Which drug or drug delivery system can change clinical practice for brain tumor therapy?
1Gaffin Center for Neuro-Oncology, Hadassah Hebrew-University Medical Center, Ein Kerem, P.O. Box 12000, Jerusalem 91120, Israel. siegal@hadassah.org.il
Abstract:
The prognosis and treatment outcome for primary brain tumors have remained unchanged despite advances in anticancer drug discovery and development. In clinical trials, the majority of promising experimental agents for brain tumors have had limited impact on survival or time to recurrence. These disappointing results are partially explained by the inadequacy of effective drug delivery to the CNS. The impediments posed by the various specialized physiological barriers and active efflux mechanisms lead to drug failure because of inability to reach the desired target at a sufficient concentration. This perspective reviews the leading strategies that aim to improve drug delivery to brain tumors and their likelihood to change clinical practice. The English literature was searched for defined search items. Strategies that use systemic delivery and those that use local delivery are critically reviewed. In addition, challenges posed for drug delivery by combined treatment with anti-angiogenic therapy are outlined. To impact clinical practice and to achieve more than just a limited local control, new drugs and delivery systems must adhere to basic clinical expectations. These include, in addition to an antitumor effect, a verified favorable adverse effects profile, easy introduction into clinical practice, feasibility of repeated or continuous administration, and compatibility of the drug or delivery system with any tumor size and brain location.
Insights
Improving drug delivery to brain tumors is crucial for better treatment outcomes. Overcoming the central nervous system
Area of Science:
- Neuro-oncology
- Pharmacology
- Drug Delivery Systems
Background:
- Primary brain tumor prognosis remains poor despite advances in anticancer drug discovery.
- Experimental agents often fail due to inadequate drug delivery across the blood-brain barrier.
- Physiological barriers and efflux mechanisms limit drug concentration at the tumor site.
Purpose of the Study:
- To review leading strategies for enhancing drug delivery to brain tumors.
- To assess the potential of these strategies to change clinical practice.
- To outline challenges for drug delivery, especially with anti-angiogenic therapies.
Main Methods:
- Systematic review of the English literature.
- Critical review of systemic and local drug delivery strategies.
- Identification of challenges in combined treatment modalities.
Main Results:
- Limited success of current experimental agents is attributed to poor central nervous system drug delivery.
- Various strategies are being explored to overcome physiological barriers.
- Combined therapies, including anti-angiogenic treatments, present unique delivery challenges.
Conclusions:
- New drugs and delivery systems must demonstrate antitumor efficacy and a favorable safety profile.
- Ease of clinical implementation, repeated administration, and compatibility with tumor characteristics are essential.
- Successful strategies require overcoming physiological barriers to achieve significant clinical impact beyond local control.
Related Concept Videos
Drug Delivery: Parenteral Route
There are three primary parenteral routes: intravenous (IV), intramuscular (IM), and subcutaneous (SC). The IV route introduces the drug directly into the bloodstream, ensuring immediate action. The IM route...
Modified-Release Drug Delivery Systems: Site-Targeted
Drug Delivery Systems: Different Types
Modified-Release Drug Delivery Systems: Classification
Oral Drug Delivery Systems: Continuous-Release Systems
Drug Delivery: Overview
Enteral delivery involves administering drugs directly through swallowing, sublingual placement, or buccal application. Orally administered drugs predominantly navigate the gastrointestinal...

