The PROBLEM

Barriers to Pharma-based Therapy for CNS-Based Diseases

The key to most any problem is the ability to be able to bring an effective solution to bear on the problem in order to eliminate the disrupting concern. It does not matter if the problem is a burnt-out light bulb or a life-threatening disease, the process is the same: Identify the issue, define the best solution, deliver the solution to the problem, and implement the cure.

The Challenge

In pharma-based therapeutics for most CNS-based diseases, such as brain cancer, Parkinson’s, dementia, and epilepsy, this four-step process has remained much the same since the earliest beginnings of medicine. Though great progress has been made in diagnostics (defining the problem), determining an effective therapeutic (the solution), and implementing therapy (the cure), little has changed in the all-important step of therapeutic delivery. For the most part, pharma-therapeutics are delivered either by oral ingestion, direct injection, or intravenous drip. In each of these methods, two major obstacles must be overcome. The first is the systemic delivery of the therapeutic agent. Though a therapeutic, such as a chemotherapy medicine for a malignant non-operable tumor, should ideally target the tumor and the tumor alone, by systemic delivery in order to make its way to the disease target the therapeutic is first forced to pass through the digestive system, in the case of oral medications, or through the entire venous system, in the case of injection or intravenous delivery.

This form of delivery creates two major concerns. The first being the potency of the therapeutic once it finally is able to be taken up by the tumor. Having to pass through the digestive system before entering the circulatory system and then making its way to the tumor, much of a therapeutics potency can be compromised. Not a problem, just make the therapeutic more potent when given to counteract the degradation effects of delivery. But this creates the second issue, side-effects and toxic exposure of healthy tissue and organs to the therapeutic. Because of the systemic nature of the delivery, all of the patient’s body is exposed to the drug being used. Since most tumor medications are in essence a type of poison designed to kill the tumor that same poison is effecting all other cellular tissue it encounters. Hence the adage, ”We hope to kill the disease before we kill the patient.” Harsh indeed, but in essence true. A patient’s ability to be able to tolerate a toxic chemotherapeutic is greatly determined by the status of their overall health and immune system status. For someone in poor health, advanced cancer stages, or compromised immunity, these therapeutics, though potentially life-saving, cannot be implemented because of the potential effect on the rest of the body.

Illustration of the Blood-Brain Barrier

The Blood-Brain Barrier

These hurdles remain part of the difficult equation physicians face when developing a treatment protocol involving pharma-therapeutics. However, when it comes to CNS- and brain-based diseases, there is another even more insurmountable problem; the Blood-Brain Barrier. The Blood-Brain Barrier (BBB) is a semipermeable barrier of endothelial cells that prevents solubles in the circulating blood from crossing into the vertebral cavity and the central nervous system. The BBB is highly selective in that it allows oxygen and nutrient molecules to pass through but it prevents potentially harmful molecules such as pathogens, large molecules, and hydrophilic molecules (water-soluble) that could carry disease. The BBB is an incredibly intricate and important part of the body’s defense mechanism for the protection of the CNS and the brain, but because of its nature, it is also a barrier to physicians in their ability to deliver lifesaving pharma-therapeutics to this area of the body. Since most pharma-therapeutics are large molecule formulations and/or water-soluble, they are unable to effectively pass through the BBB. This limits therapy then to direct injectable such as intralesional and intratumoral chemotherapy or implanted Ommaya reservoir pumps. These though are limited to those types of cancers that are accessible via injection or proximity pump location. Unfortunately, many CNS and brain cancers tumors are too deep-rooted to be treated in this manner.

The Solution

NeOnc Technologies is hoping to provide a major leap forward in providing a solution posed by current pharma-therapeutic delivery for CNS-based diseases. By creating a proprietary molecular wrapper technology to protect and encase the therapeutic agent during delivery and one which can cross the BBB, along with addressing a three-prong delivery solution Neonc holds the promise to improve the physician’s ability to cure and to extending and enhancing the patient’s quality of life.