A Novel Way for Biologics to Target the Central Nervous System

Biologics Development

How can therapeutic biologics be delivered effectively into the central nervous system?
Image source: Flickr CC user NICHD

The central nervous system may be comprised of only two components, but those pieces are crucial for bodily function. After all, the brain is the master organ while the spinal cord sends information to our muscles and glands. As a result, injury to either of them can leave long-lasting effects on the body.

Injury isn’t the only thing that can negatively impact the central nervous system, however. A wide array of diseases and disorders can cause adverse effects. Parkinson’s, Huntington’s and Alzheimer’s disease are all examples of illnesses that target the central nervous system. Alzheimer’s disease, in particular, has been a focus of heavy study due to its correlation with old age. Considering the fact that the Baby Boomer generation is about to hit retirement age, you can imagine efforts to find effective treatment options will only increase in the years to come.

Difficulties in Using Biologics to Treat Central Nervous System Disorders

Unfortunately, diseases affecting the central nervous system are notoriously difficult to treat. There are a couple reasons for this. First is the fact that modern drug discovery has focused primarily on target based approaches to identify new putative drugs.1 That means it requires identifying potential sites of drug action. While the method has proven effective for some forms of drug development, things have been slower when it comes to CNS disorders.

Why? Because target-based drug discovery generally requires a thorough understanding of how the target interacts on a molecular level. When it comes to conditions involving the CNS, however, many genetic associations can be involved. For example, there are around 70 genetic associations with Alzheimer’s. Determining how those associations might be involved or interact with each other is challenging enough; imagine choosing only one as a prime target site!

The other impediment to drug discovery is central nervous system permeation.2 It has traditionally been difficult to treat CNS diseases due to the presence of the blood-brain barrier, which can keep many therapeutics—including biologics—from reaching their targets. Because of this, any drug discovery must take this limiting factor into account. Potential candidates must either be designed to cross the blood-brain barrier or have a delivery system that can shuttle it across.

Using Gene Therapy in Conjunction with Biologics to Target the Central Nervous System

Earlier this year, Medgenics expanded its collaboration with Harvard University to apply its gene therapy technology to the central nervous system.3 The therapeutic platform uses the patient’s own tissue to create and deliver proteins into the body. Even though the research is still in its early stages, the company hopes this technology could provide a successful vehicle to deliver biologics to the difficult-to-treat CNS. According to the firm, their system is less likely to instigate an immune response than other existing therapies.

Developing therapeutic biologics for the central nervous system can be a challenge. But considering the wide number of CNS disorders, finding ways to circumvent the biological hurdles are paramount. And in addition to the biological challenges, there are also workflow challenges to biologics development. For example, a lot of complex data is generated during research. Numerical results, empirical observations and structural models are all details collected over the course of a project.

The BIOVIA Biologics Solution can help efforts within this arena. As a fully integrated and centralized system, it manages all kinds of data, from initial characterization results to formulation development. It can also keep track of the manufacturing process and related testing. This leads to better understanding of your data, which in turn leads to better insight into processes and faster decision-making.

Are you interested in learning how the BIOVIA Biologics Solution can support your game-changing biologics development? Contact us today to learn more.

  1. “Challenges in the Search for Drugs to Treat Central Nervous System Disorders,” January 30, 20089, http://jpet.aspetjournals.org/content/329/2/404.full
  2. “Central nervous system diseases and the role of the blood-brain barrier in their treatment,” June 25, 2013, http://www.hoajonline.com/neuroscience/2052-6946/1/3
  3. “Gene therapy tech could be ‘game-changer’ for CNS biologics, says Medgenics,” April 21, 2015, http://www.biopharma-reporter.com/Bio-Developments/Medgenics-TARGTs-central-nervous-system-in-expanded-Harvard-deal

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