This technology offers a non-surgical treatment for hydrocephalus by using drugs to enhance the removal of harmful macromolecules from the brain’s ventricles, targeting specific cellular transport mechanisms to reduce fluid buildup and disease progression.
Background: Hydrocephalus is a neurological disorder characterized by the abnormal accumulation of cerebrospinal fluid (CSF) within the brain's ventricles, leading to increased intracranial pressure and potentially severe neurological impairment. The condition affects individuals across all age groups, from infants to the elderly, and can arise from congenital malformations, trauma, infection, or tumors. The standard of care for hydrocephalus has remained largely unchanged for decades, relying primarily on surgical interventions such as the implantation of shunts or endoscopic procedures to divert or remove excess CSF. While these procedures can be life-saving, they are invasive, carry significant risks of infection and mechanical failure, and often require repeated surgeries over a patient's lifetime. The limited availability of effective, non-surgical treatments underscores a critical need for alternative therapeutic strategies, particularly for patients who are poor surgical candidates or who develop hydrocephalus in the context of complex medical conditions, such as after traumatic brain injury. Current pharmacological approaches to hydrocephalus are notably inadequate. The only widely used drug, acetazolamide, acts as a diuretic to reduce CSF production but has shown limited efficacy in clinical trials and is often associated with significant side effects. Moreover, existing treatments do not address the underlying pathophysiology of hydrocephalus, such as the accumulation of macromolecules in the ventricles, which is increasingly recognized as a key factor in disease progression. Surgical interventions, while effective at relieving pressure, do not resolve the buildup of these pathological macromolecules and may not prevent long-term neurological damage. As a result, there is a pressing need for novel therapeutic approaches that can directly target the mechanisms responsible for macromolecular accumulation and clearance within the brain, offering the potential for safer, more effective, and less invasive management of hydrocephalus.
Technology Overview: This technology introduces a novel pharmacological approach for treating hydrocephalus by enhancing the removal of macromolecules from the brain’s ventricles. Unlike traditional treatments that focus on reducing CSF production, this solution targets the underlying accumulation of macromolecules implicated in the pathology of hydrocephalus. The strategy involves manipulating key transport mechanisms—specifically efflux transporters, endocytosis, and exocytosis—either individually or in combination. By modulating these pathways, the therapy aims to decrease the buildup of harmful macromolecules within the ventricles, thereby alleviating the symptoms and progression of hydrocephalus. This approach is designed to be adaptable, potentially allowing for combination therapies or use as an adjunct to surgical interventions, and may also be applicable to other neurological conditions characterized by abnormal macromolecular accumulation. What differentiates this technology is its shift from the conventional focus on CSF volume management to directly addressing the molecular contributors to hydrocephalus. By identifying and targeting the transport mechanisms responsible for macromolecular clearance, this solution leverages a deeper understanding of hydrocephalus pathophysiology. The ability to pharmacologically modulate efflux transporters and related pathways represents a significant advancement, offering a non-invasive alternative for patients who are poor surgical candidates or who develop hydrocephalus following trauma. Its originality, adaptability, and potential for broader neurological applications set it apart as a transformative step in hydrocephalus management.
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Advantages: • Provides a non-surgical pharmacological treatment option for hydrocephalus, reducing reliance on invasive surgery. • Targets the removal of macromolecules from brain ventricles by modulating efflux transporters, endocytosis, and exocytosis. • Aims to decrease the accumulation of pathological macromolecules implicated in hydrocephalus progression. • Potentially effective for post-traumatic hydrocephalus and other neurological conditions involving macromolecular buildup. • Enables combination therapies targeting multiple clearance pathways for enhanced treatment efficacy. • Offers an alternative for patients who are poor candidates for surgical intervention. • Represents an original approach with potential broad clinical and military applications.
Applications: • Non-surgical hydrocephalus treatment • Adjunct therapy for hydrocephalus surgery • Treatment for post-traumatic hydrocephalus • Therapy for macromolecule-related brain disorders
Intellectual Property Summary: Patent application: 63/794,421, application filed on 4/25/2025 Know-how based
Stage of Development: TRL 2 Identifying and targeting the transport mechanisms responsible for macromolecular clearance.
Licensing Status: This technology is available for licensing.