Stuart Prime, Research Team Leader at Axol, recently spoke at the 2nd Annual ALS Drug Development Summit in Boston, MA. Dr Prime presented Axol’s breakthrough Platforma project, using iPSCs to build in vitro models of the neuromuscular junction for ALS research and drug discovery.
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressing, devastating disease that affects around 450,000 people worldwide 1. It is characterized by the loss of motor neuron function, needed to activate skeletal muscle. This causes patients to lose control of their limbs, swallowing and breathing, resulting in terminal paralysis and death 2.
There is currently no cure for ALS; current therapies seek to manage symptoms or extend life expectancy, usually by just a few months 2. Despite the recent FDA approvals of Relyvrio and Qalsody, there are still challenges in addressing the translational gap for the wider ALS patient population.
Recognizing these challenges, the conference brought together industry experts, scientists and researchers from around the world to discuss challenges and breakthroughs in ALS drug development.
Axol’s new platform for ALS disease modeling
With a talk entitled “Human iPSC 2D & 3D neuromuscular junction modelling for assessing C9orf72 ALS hyperexcitability in vitro“, Dr Prime presented Axol’s new platform for ALS disease modeling and drug discovery.
Dr Prime discussed how iPSC-derived motor neurons and skeletal muscle were used to build 2D and 3D cell culture models of the neuromuscular junction (NMJ). This included discussion of iPSC characterization (to generate physiologically relevant cells) and the various functional assays used to assess the 2D and 3D co-cultures as models of hyperexcitable ALS subtypes.
Given the central role of the NMJ in ALS disease onset, Axol’s new platforms have enormous potential as models for ALS research and drug discovery. It also highlights Axol’s continued investment in developing better human disease models using human iPSCs.
Products used in this project
- axoCellsTM Motor Neurons – ax0078 (control), ax0074 and ax0073 (ALS lines)
- Motor Neuron Maturation Accelerator – ax0179
Want to explore our work with ALS disease modeling? Click here to find out more.