Q&A: ‘Groundbreaking’ intranasal brain cancer treatment bypasses blood-brain barrier

Key takeaways:

• NeOnc’s nebulizer bypasses the blood-brain barrier to deliver drugs directly to olfactory nerve endings.
• Although the nebulizer is patient administered, NeOnc is raising awareness among health care providers.

Bypassing the blood-brain barrier remains an obstacle for novel therapeutics. Healio spoke to Thomas Chen, MD, PhD, founder and CEO of NeOnc, to find out how a novel nasal spray formulation to treat brain cancer overcomes these challenges.

Healio: How and why was the decision made to create the novel therapy as a nasal spray as opposed to other formulations?

Chen: The decision to develop NEO100 for delivery via a nasal nebulizer was driven by the unique challenges posed by the blood-brain barrier, which significantly limits the effectiveness of traditional drug delivery methods for brain cancer. The nebulizer allows for direct delivery of the drugs to the olfactory nerve endings, which pass through the cribriform plate at the base of the skull and connect directly to the brain. This method leverages the olfactory nerve pathways to bypass the blood-brain barrier, ensuring that the therapeutic agent reaches the brain more effectively.

Also, NEO100 comprises a patented composition of a proprietary synthesis of perillyl alcohol, which has a small molecular size, allowing it to bypass the blood-brain barrier effectively when delivered intranasally. This method minimizes systemic side effects and enhances the overall safety profile of the treatment, making it an ideal choice for treating central nervous system diseases like brain cancer.

Traditional methods often struggle with the barrier, which acts as a protective shield preventing many drugs from reaching the brain. Our nasal nebulizer formulation offers a noninvasive, efficient and effective alternative.

Healio: How and why was the decision made for this particular therapy to address a condition as serious as brain cancer?

Chen: Brain cancer, particularly high-grade gliomas like glioblastoma, represents one of the most formidable challenges in oncology because of the protective nature of the blood-brain barrier. Traditional chemotherapy and targeted therapies struggle to cross this barrier, leading to suboptimal treatment outcomes.

The nasal delivery method offers a groundbreaking solution by bypassing it entirely, allowing the drug to reach the brain directly and target tumor cells more effectively. NEO100 acts as a regulator for neurologic pathways associated with tumor cell growth and demonstrates significant therapeutic efficacy in high concentrations. Given the critical need for more effective brain cancer therapies and the promising results from preliminary studies, NeOnc chose to focus on this severe condition.

Healio: Can you elaborate on the mechanism of action that allows for the bypassing of the blood-brain barrier?

Chen: NeOnc’s nasal delivery system for NEO100 uses the olfactory nerve pathways to bypass the blood-brain barrier. The olfactory nerves, responsible for our sense of smell, extend from the nasal cavity through the cribriform plate at the base of the skull and into the brain.

By administering the drug as a nasal spray, it travels along these pathways directly to the brain. This allows the drug to reach the temporal lobe and the cerebrospinal fluid, facilitating widespread distribution throughout the brain. Also, when delivered intra-arterially, the drugs can create a temporary opening in the blood-brain barrier, allowing larger molecule therapeutics to pass through. This dual capability enhances the delivery and effectiveness of the treatment by ensuring higher concentrations of the drug at the tumor site while minimizing systemic exposure and side effects.

Furthermore, NEO100 can act as a solvent for traditional large-molecule therapeutics in low concentrations, allowing them to bypass the barrier and be conjugated with other CNS therapeutics to create compound formulations capable of higher penetration and more significant effects on the cancer itself.

Healio: Although it is possible for patients to self-administer the therapy, are clinicians aware of and able to administer the treatment as an alternative?

Chen: Currently, not all clinicians are fully aware of the availability of this therapy because it is still undergoing clinical trials. However, NeOnc is actively working to raise awareness among health care providers about the potential benefits and availability of this treatment. The company encourages clinicians with eligible patients to contact NeOnc for participation in ongoing clinical trials. As NEO100 progresses through the trial phases and moves toward FDA approval, NeOnc plans to implement comprehensive educational initiatives to ensure that clinicians are well informed and prepared to administer the treatment, if needed.

Healio: How do you anticipate the application of the self-administered nasal spray will impact the care continuum for patients and clinicians going forward?

Chen: I would anticipate that this delivery method has the potential to revolutionize the treatment landscape for brain cancer and other neurodegenerative conditions because it offers several advantages, including ease of use, reduced need for frequent hospital visits and improved patient compliance.

By empowering patients to administer their treatment at home, it alleviates the burden on health care systems and enhances the quality of life for patients. Also, the success of this delivery method in treating brain cancer opens the door to applying similar approaches to other CNS diseases, such as Alzheimer's disease, Parkinson’s disease and multiple sclerosis.

The FDA’s fast track and orphan drug designations for NEO100 highlight its potential to address unmet medical needs and expedite the review process, bringing these groundbreaking therapies to market more quickly.

For more information:

Chen can be found at: https://neonctech.com/leadership/thomas-chen/.