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Therapeutic Advances in CNS

Central Nervous System (CNS) disorders are diseases that attack the brain, the spine and the nerves that connect them. There are more than 600 diseases of the nervous system. Some, such as Huntington’s, Parkinson’s and Multiple Sclerosis, affect motor abilities while others, such as Alzheimer’s and dementia, impair cognitive abilities.

Currently there is widespread excitement about the advances in CNS research and discovery of novel research in CNS over the past five years. These findings are bringing greater venture capital funding and are resulting in large pharma rebuilding their R&D platforms to address these debilitating diseases that affect so many people in our aging populations.

America’s biopharmaceutical research companies are developing nearly 450 new medicines to prevent and treat neurological disorders, according to a report by the Pharmaceutical Research and Manufacturers of America (PhRMA)¹.

Dr. Adam Rosenberg, CEO of Rodin Therapeutics believes today’s science allows for more personalized medical management, starting with more precise targeted diagnosis that will help drive discovery of more specific novel CNS drug candidates. Dr. Rosenberg believes the possibilities are great with accelerated biomarker development to improve patient selection and target engagement; better enabling tools like imaging and cloud computing; and the vital need of renewed VC investment in the CNS field. Read Dr. Rosenberg’s inspiring blog post here.

Gene Therapy

Genetic research and subsequent gene therapies could revolutionize the treatment for diseases afflicting the brain. Advances such as genetic sequencing and new DNA editing technologies are giving scientists extensively more information on brain behaviors and feedback.

Current research has the potential to increase the quality of life and delay cognitive decline for patients diagnosed with neurological disorders. For example, scientists are beginning to understand more about the genes that affect the development of Alzheimer’s disease. Such discoveries could lead to new research pathways to help find ways to slow, delay or reverse the effects of the disease. According to a Alzheimer’s Association 2015 report², a treatment that delays the onset of Alzheimer’s by five years could reduce the cost of care for the disease by $367 billion a year in 2050.

A Patient’s Story

Dr. Halina Frydman was diagnosed with CNS Lymphoma after her personality, memory and behavior changed dramatically. Dr. Frydman was enrolled in a clinical trial consisting of methotrexate, a chemotherapy drug that was given in very high doses; along with the chemotherapy drugs vincristine and procarbazine, and a monoclonal antibody called rituximab3.

What the clinical trial uncovered was that underlying brain tissue is not necessarily destroyed by tumors; therefore effective treatment really can restore people to functionality.

Clinical Trial Management

The foundation of therapeutic advances in CNS lies in novel drug discovery methods. But what about when the molecules reach clinical trials? So much investment and time goes into getting the CNS indicated therapeutics into studies in man, it would be terrible if clinical sites dropped the ball on simple things like managing the temperature controlled drugs on-site.

According to Good Distribution Practices (GDP) requirements, if stability data of a product in development requires certain temperature handling, the clinical site must have documented proof those investigational CNS medicines have been kept within defined temperatures in storage and during transport to worldwide clinical sites. If they don’t, clinical sites and their sponsors face serious consequences from regulatory authorities and ethics committee violations that could impact trial timelines and delay the novel therapeutics entry to market.

Unlike earlier lifecycle challenges, maintaining drug stability during clinical trials is very manageable with robust technology that is readily available. As the CNS investigational medicines are distributed to clinical trials sites, temperature monitoring plays an important role in maintaining product stability.

Lundbeck, an international pharmaceutical company renowned for their contribution to the CNS field, has had sufficient monitoring in place for years. Their compliant processes include easy to read temperature excursion alarms; using a multi-level data logger to make sure of available stability data; and storing temperature data in a traceable database serving as an audit trail. Read the full post on How Lundbeck Stopped Wasting QA Resources in Clinical Supply.

For stability and temperature management on-site, while the CNS investigational medicines are in storage in refrigerators or freezers, there is new technology in the field of digital data loggers, called independent monitoring. These are stand-alone, self-powered systems – virtually ‘hands-free’ base and sensor systems. They allow clinical staff to spend more time with patients, instead of manually recording temperatures. Reports are generated by simply connecting the base to a computer using a USB connection.

See how new Independent Monitoring for Rooms & Equipment for clinical trial sites works, watch tutorial videos.

Quality control in the ‘last mile’ of delivering novel CNS medicines is a challenging time. Companies like Lundbeck are implementing processes and technology that help minimize risk to patient, while ensuring clinical trial timelines are met.

Exciting Times

Scientific advances and new technologies are dramatically changing how medicines are discovered. This new information is critical to the development of new treatments for neurological disorders. Greater knowledge of how diseases work at the genetic and molecular level has allowed researchers to pursue new targets for therapy and better predict how certain biopharmaceuticals will affect specific subpopulations of patients.

Halina Frydman was lucky to be involved in the right clinical trial with advanced therapeutics. Today she has returned to a full life of teaching, researching, writing, publishing and dancing with her husband.


  1. Pharmaceutical Research and Manufacturers of America (PhRMA); WASHINGTON, D.C. (July 30, 2013); Nearly 450 Medicines for Neurological Disorders

  2. Alzheimer’s Association; 2015; Changing Trajectory of Alzheimer’s Disease: How Treatment by 2025 Saves Lives and Dollars

  3. Memorial Sloan Kettering Cancer Center; «Halina’s story about Primary Central Nervous System (CNS) Lymphoma»

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