Cell-based Therapy for Neurodegenerative Diseases

Background

• Problem: Amyotrophic lateral sclerosis (ALS) is a severe degenerative disease with current treatment being two FDA approved drugs with limited efficacy.
• Solution: Adipose-derived stem cell conditioned media (ASC-CM) is a promising, close-to-IND-ready therapy for ALS.
• Unique value proposition: The ASC-CM formulation has a consistent profile of factors that can influence both the central nervous system and the peripheral nerves.

Technology Overview

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease with a global incidence of about 1 per 50,000 people. There are only two FDA approved drugs for the treatment of ALS with only marginal therapeutic benefits in a subset of patients. These, along with other therapies in development, target primarily the central nervous system, allowing the peripheral nervous system to continue to degenerate.

An IU researcher at the Indiana University Schools of Dentistry and Medicine developed a formulation of adipose-derived stem cell conditioned media (ASC-CM) that prevents early disease pathology in an ALS transgenic mouse model. The ASC-CM was developed to contain a unique and consistent profile of a combination of paracrine factors that could influence both the central and peripheral nervous systems. The consistency and reliability of the conditioned media product provides a distinct advantage over cellular therapies.

The researchers are expecting to initiate a Phase I clinical trial in the near future with an improved formulation of their published pre-clinical work. The Phase I study design has already been reviewed favorably by the FDA in a pre-IND meeting. This work in developing the profile of factors for the conditioned media can be expanded to other neurodegenerative diseases, greatly increasing the market value of this highly promising technology.

Further Details:

• Adipose-derived stem cell conditioned medium for the treatment of amyotrophic lateral sclerosis: pre-clinical evidence and potential for clinical application
• Systemic Dental Pulp Stem Cell Secretome Therapy in a Mouse Model of Amyotrophic Lateral Sclerosis
• Adipose-derived stem cell conditioned medium impacts asymptomatic peripheral neuromuscular denervation in the mutant superoxide dismutase (G93A) transgenic mouse model of amyotrophic lateral sclerosis

Benefits

• Dual action on both the central nervous system and the peripheral nerves
• Near IND-ready formulation with FDA conceptual approval
• Demonstrated efficacy in preventing ALS disease pathology in vivo
• Adaptable platform that can be expanded for multiple neurodegenerative diseases

Applications

• Treatment of amyotrophic lateral sclerosis
• Potential therapy for other neurodegenerative diseases

Opportunity

• Looking to partner to facilitate the initiation of the Phase I clinical trial, and for further development for treatment of ALS as well as other neurodegenerative diseases.