Stem Cell Therapy for Amyotrophic lateral Sclerosis (ALS)

Stem Cell Therapy for Amyotrophic lateral Sclerosis (ALS)

Mesenchymal stem cells isolated from human umbilical cord (hMSCs) are modified for the treatment of Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease).


ALS is a devastating neurological disorder with no known etiology or treatment.  ALS usually strikes during adulthood and life expectancy is most often less than 5 years after diagnosis.  Current treatments fail to effectively improve prognosis, and thus new therapies are desperately needed.  The unique modification of mesenchymal stem cells isolated from human umbilical cord (hMSCs) showed promising results for maintaining neuromuscular junctions in ALS mouse models.


Modified hMSCs could be used as treatment for ALS patients.

How it works:

KU researchers have developed a condition to modify human umbilical cord mesenchymal stem cells (hMSCs) to increase secretion of synapse organizer and neurotrophic factors.  Combinatorial treatment of the synapse organizer and the neurotrophic factors may result in improvement of symptoms in ALS patients.  In vivo data generated in the mouse model of ALS demonstrates that these hMSCs maintain innervation of neuromuscular junctions.

Why it is better:

The proposed therapy incorporates the maintenance benefits of modified stem cells, neurotrophic factors, and a new target, which is highly unique to this study.  Umbilical cord derived stem cells have a number of advantages compared to other types and sources of stem cells in that they possess combined fetal and adult stem cell properties and therefore have fewer limitations and are similar to early embryonic cells in surface marker portrait and differentiation potential.  They are also the richest in cell material and most homogenous compared to other type of stem cells.  Umbilical cord stem cells are also far more available and ethically clean as compared to other described stem cell sources.

Other Applications:

Therapeutics for motor neuron disease, and neuromuscular disease patients

Additional Details

Owner: University of Kansas

IP Protection Status: Pending Patent