Creating a Cure: Japan's advances in overcoming neurodegenerative diseases
Creating a Cure
Japan’s advances in overcoming neurodegenerative diseases
“We are developing a revolutionary synthetic human nervous system... This could have a significant impact on new drug discovery.”— KAWADA Jiro, CEO & Founder, Jiksak
In May 1939, ailing ironman Lou Gehrig shocked the baseball world when he abruptly pulled himself out of the line-up, ending his consecutive games played streak at 2,130. Within a month, he was diagnosed with Amyotrophic Lateral Sclerosis (ALS).
He was told that ALS would lead to paralysis and difficulty swallowing and speaking, but his mental faculties would remain sharp. He was also told the disease was incurable. Less than two years later, Lou Gehrig would die, but his legacy would live on in the search for a cure. ALS would even be called “Lou Gehrig’s disease” in some parts of the world.
Yet despite ALS having been discovered in the 1860s, and entering the public conscious as Lou Gehrig and Stephen Hawking battled it, this lethal disease still infects about 120,000 people each year. Life expectancy after diagnosis still averages just two to five years, too.
ALS is not slowing down
By 2040, it is forecasted that ALS cases globally will increase by 69 percent to 376,674. In about 90 percent of cases, ALS arises with no apparent cause—toxins, viruses and lifestyle may play a role, but these are hard to pinpoint. The other 10 percent of cases are passed down through mutated genes.
ALS most often affects people between the ages of 40 and 70, and with aging populations in developed nations on the rise, this projection is likely to be an underestimate.
THE WORLD'S AGING POPULATION
The sheer complexity of ALS is why it remains such a debilitating disease. As ALS affects motor neurons throughout the body, there is no single test that can determine whether someone has it. Even after symptoms appear, there are only two mainstream drugs on the market approved to treat ALS: riluzole and edaravone. Both are expensive - when launched in Japan in 2017, ALS drug edaravone was priced at $35,000, and neither provide a cure. Meanwhile, the rarity of the disease makes it difficult to find patients to conduct new clinical trials.
Even when drugs do advance to the trial stage, most fail. In fact, as few as 10 percent of all drugs currently pass through every phase of clinical trials, compounding global efforts to combat diseases like ALS with losses in the millions—if not billions—of dollars.
“The Nerve Organoid is a three-dimensional neural tissue that resembles the human body environment, applying stem cell technology and organ-on-a-chip technology which allows experiments to take place”— TOITA Sayaka, Medical Researcher, Jiksak
New technology strikes a nerve
Finding cost-effective drugs to treat and cure the disease remains paramount. In Japan, emerging bioengineering technology is changing the way researchers study the disease and fast-tracking drug discovery, edging the world closer to a cure.
Medtech startup Jiksak Bioengineering, based in Kanagawa Prefecture, is championing a novel synthetic human nervous system called the “Nerve Organoid.” This organ-on-a-chip device imitates the human nervous system to help conduct clinical trials and drug tests that will aid neurogenerative research for ALS, Parkinson’s and Alzheimer’s disease.
In ALS patients, the axons of motor neurons start to shrink even before motor function symptoms appear. The brain stops telling the mouth muscles how to chew, the leg muscles how to move—even the muscles that allow us to breathe stop receiving signals. No one yet understands what causes these axons to degenerate, though.
The Nerve Organoid
Three-dimensional Neural Tissue
The Nerve Organoid is a three-dimensional neural tissue, generated from neurons derived from stem cells (cells that have the ability to develop into many different cell types). Through doing so it offers an environment that resembles the human nervous system, enabling experiments to take place.
Helping Examine the Behavior of Axons
With this three-dimensional structure, researchers can examine the behavior of axons, long, hair-like projections of nerve cells that send electrical signals from the brain and spinal cord to muscles. Axons, in other words, tell our muscles what to do.
Enabling Revolutionary Experiments and Testing
That is what makes the Nerve Organoid so revolutionary. The chip-sized structure allows medical researchers to experiment on axons, cells and cell nuclei; trial new drugs like never before; and potentially fast-track treatments without having to rely on patients for testing.
Already, one group of Japanese researchers has discovered a novel gene to target in the fight against ALS using the Nerve Organoid technology. Jiksak has stepped into the ring with its own ALS program, too, targeting the neuromuscular junction in drug discovery.
20mm long Nerve Organoid integrated
Japan Engineered innovation
The bioengineering technology coming out of Japan is helping to compile data and build a comprehensive picture of ALS like the world has never seen. This data will not just aid ALS research, though. It is facilitating clinical trials for other neurogenerative diseases like Parkinson’s and Alzheimer’s as well.
Could new drugs that extend patients’ lifespans or even cure them of neurogenerative diseases like ALS be on the horizon? With breakthroughs in bioengineering like Japan’s Nerve Organoid, the solutions suddenly seem closer than ever.