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‘ALS on a Chip’ to Potentially Improve Personalized Medicine

August 27, 2020

A University of Central Florida research lab has developed “human-on-a-chip” technology that can be used to help assess the potential of new personalized therapy candidates for ALS. The microfluidic device contains human-derived nerve cells (cultivated from ALS patients’ human stem cells) and muscle cells, which together have formed neuromuscular junctions, physiology associated with ALS and other neurogenerative diseases. Muscle fibers on the chip – mini muscles – can be contracted by activation of the motoneurons or direct electrical stimulation.

Organs on a chip give researchers a way to research disease and drugs on human physiology without experimenting on human beings or using animals as rough substitutes. For every 50 drugs determined to be safe for animals, only one proves safe in humans, according to the lab.

“To our knowledge, this is the first study to demonstrate that while different ALS mutations display various phenotypes, all have the common point-of-origin deficit at the NMJ for each mutation,” said James J Hickman, Ph.D., Chief Scientist at Hesperos and Professor at the University of Central Florida. “This is also the first data to demonstrate the efficacy of the Deanna protocol [a holistic supplement regime] for the treatment of ALS, utilizing a clinically relevant assay system.”

Recording ‘skips’ and fatigue in the cells

With the neuromuscular junction on a chip, UCF researchers were also able to simulate in-clinic tests where a patient is given a task to do with increased speed to detect spasticity or loss of muscle strength. To carry this out, researchers stimulated the motoneurons at increasing frequency and monitored the muscle to record “skips” as well as accelerated fatigue, the UCF lab said.

This work echoes ALS-on-a-chip drug discovery work at Harvard and MIT as well as federally funded research to grow blood-vessel and nerve cells on a chip to model fetal spinal cord development, also for ALS research. In the latter work,

“[e]ach cell type was then injected into one of two chambers embedded side-by-side in thumb-sized, plastic tissue chips and allowed to grow. Six days after injections, the researchers found that the growing neurons exclusively filled their chambers while the growing blood vessel cells not only lined their chamber in a cobblestone pattern reminiscent of vessels in the body, but also snuck through the perforations in the chamber walls and contacted the neurons. This appeared to enhance maturation of both cell types, causing the neurons to fire more often and both cell types to be marked by some gene activity found in fetal spinal cord cells.”

ALS affects thousands

ALS stands for Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease. The condition destroys motor neurons, the large nerve cells that message muscles, causing increased muscle weakness throughout the body. A little more than 5,000 Americans are diagnosed with ALS every year, and as many as 30,000 Americans may have the disease at any time. Ninety percent of ALS cases are sporadic, meaning they’re not inherited.

iSpecimen supports research into ALS and other diseases by providing researchers with human biospecimens and deidentified data from patients with various types of the disease, as well as normal healthy controls. These biospecimens include peripheral blood mononuclear cells (PBMCs), which can be induced to pluripotency. We blogged about R&D into ALS treatment and the wildly successful Ice Bucket Challenge that helped fund it here.

Learn about the iSpecimen Marketplace where you can browse millions of richly annotated, de-identified human tissue and biofluid biospecimens, in addition to hematopoietic and immune cell products and COVID-19 samples. You can join for free and creating a login is easy. Request a quote or custom collection today.