Translating Cellular Therapies from Bench to Bedside for Amyotrophic Lateral Sclerosis

Abstract

The last decade has witnessed an increasing number of biologic (e.g., cell- or viral vector-based) therapeutics supported by preclinical efficacy data for the treatment of afflictions to the CNS. While some international investigators have undertaken preliminary clinical safety studies, published literature indicate varying degrees of rigor with respect to study design and technical approach. To our knowledge, ours is the first group to have systematically generated preclinical validation data for a delivery approach and translated this into a Phase I trial attempting to covalidate the safety of a direct, targeted delivery approach, as well as a cell-based therapeutic. This article discusses the rationale for cell-based therapy in amyotrophic lateral sclerosis and several of the unique considerations encountered during this process.

KEYWORDS::

• amyotrophic lateral sclerosis
• intraspinal microinjection
• spinal cord
• stem cell therapy
• targeted delivery

Financial & competing interests disclosure

Neuralstem, Inc. (MD, USA) provided financial assistance for microinjection platform construction and is funding the Phase I clinical trial that is described and is currently underway. N Boulis has received an inventors‘ fee for the microinjection platform and floating cannula. N Boulis is also eligible for royalties associated with future licensing of these technologies. The authors have no other 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 apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Additional information

Funding

Neuralstem, Inc. (MD, USA) provided financial assistance for microinjection platform construction and is funding the Phase I clinical trial that is described and is currently underway. N Boulis has received an inventors‘ fee for the microinjection platform and floating cannula. N Boulis is also eligible for royalties associated with future licensing of these technologies. The authors have no other 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 apart from those disclosed. No writing assistance was utilized in the production of this manuscript.