The development of biological therapies for neurological diseases: moving on from previous failures

ABSTRACT

Introduction: Although years of research have expanded the use of biologics for several clinical conditions, such development has not yet occurred in the treatment of neurological diseases. With the advancement of biologic technologies, there is promise for these therapeutics as novel therapeutic approaches for neurological diseases.

Areas covered: In this article, the authors review the therapeutic potential of different types of biologics for the treatment of neurological diseases. Preclinical and clinical studies that investigate the efficacy and safety of biologics in the treatment of neurological diseases, namely Alzheimer’s disease, amyotrophic lateral sclerosis, Parkinson disease, multiple sclerosis, and stroke, were reviewed. Moreover, the authors describe the key challenges in the development of therapeutically safe and effective biologics for the treatment of neurological diseases.

Expert opinion: Several biologics have shown promise in the treatment of neurological diseases. However, the complexity of the CNS, as well as a limited understanding of disease progression, and restricted access of biologics to the CNS has limited successful development. Therefore, more research needs to be conducted to overcome these hurdles before developing effective and safe biologics for neurological diseases. The emergence of new technologies for the design, production and delivery of biologics will accelerate translating biologics to the clinic.

KEYWORDS:

• Biologics
• neurological diseases
• blood-brain barrier
• antibodies
• gene therapy

Article highlights

• Several types of biologics have been evaluated in preclinical models and clinical trials for the treatment of neurological disease, yet none of these biologics have been approved for the clinical use.

• Monoclonal antibodies are the most common of biologics that have gained high research interest for treating neurological diseases because of their specificity and safety.

• Enhancing brain removal of faulty protein aggregates using passive and active immunization is a promising approach for the treatment of AD and PD.

• Cells and gene therapies are novel biological approaches that have been tested extensively as therapeutic alternatives for ALS and stroke.

• CNS delivery, clinical pharmacology aspects, complexity of the diseases, and high risk of severe adverse reactions are the most common challenges in developing biologics for treating neurological diseases.

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Disclaimer

The article reflects the views of the authors and should not be construed to represent the views or policies of the FDA.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose

Additional information

Funding

This manuscript has not been funded.