Overview

Amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) is a relentless and fatal neurodegenerative disease which carries with it an average life expectancy of two to five years from diagnosis.

ALS damages and destroys long nerve cells, known as motor neurons, in the brain and spinal cord. As their name implies, motor neurons are in charge of movement. The long motor neurons extend from the brain, all the way down to the spinal cord and then to muscles throughout the body. When these motor neurons degenerate and die, it becomes difficult—and then impossible—to control movement. Loss of motor neurons causes paralysis, resulting in difficulty speaking, swallowing and eventually breathing, leading to respiratory failure and ultimately death.

For the thousands of patients living with ALS, there is no existing therapy that can cure or effectively treat their condition.

There is currently no cure for ALS, and only limited treatments to slow or mitigate the symptoms. Rilutek, the only FDA-approved compound to treat ALS, extends ALS patients’ lifespans by a maximum of three months.

Progress Towards A Cure

With more than $62 million in funding from Proposition 71 and the California Institute for Regenerative Medicine (CIRM), researchers are working to develop ways to slow or halt the progression of ALS.

Scientists are using stem cells to find ways to prevent damage and support the function of motor neurons before they die. A team at Cedars-Sinai Medical Institute, led by Dr. Clive Svendsen, is using a strategy to implant a type of stem cell, known as a neural progenitor cell, that can home to the site of dysfunction or injury. These specific neural progenitor cells have also been engineered to secrete a powerful growth factor called glial-derived neurotrophic factor (GDNF). The idea is to use these engineered cells as Trojan Horses: after implantation, the cells will migrate to the site of injury, where they will secrete high levels of GDNF and prolong the life of the motor neurons.

This trial was approved by the FDA in October 2016 and is expected to commence in mid-2017.

The scientists hope to slow the progression of motor neuron death, thereby slowing or halting the progression of ALS.

Another research project at the University of California, San Diego (UCSD), led by Dr. Larry Goldstein, Dr. Samuel Pfaff, and Dr. Martin Marsala, aims to protect surviving neurons in people diagnosed with ALS from further degeneration. The team intends to take human embryonic stem cells, and then turn them into a type of stem cell known as neural stem cells. These develop into neurons and support cells known as astrocytes: support cells that envelope and provide vital nutrients to motor neurons.

By transplanting neural stem cells into ALS patients, the scientists hope to slow the progression of motor neuron death, thereby slowing or halting the progression of ALS.