Harvard researchers have identified a key enzyme that kills cells in patients with amyotrophic lateral sclerosis (ALS). The Centers for Disease Control and Prevention (CDC) estimates that around 30,000 people in the U.S. are currently living with ALS (also known as Lou Gehrig’s disease). This neurodegenerative condition affects mainly older individuals between the age of 55 and 75.
While there is no cure for ALS, researchers at Harvard published the results of a new study that revealed an enzyme responsible for cell death. This discovery is paving the way for new ALS treatments to slow down the progression of the condition.
RIPK1 Enzyme and ALS
ALS is a degenerative condition that attacks cells in the brain and the spine, ultimately leading to muscle weakness, paralysis, and respiratory failure. In patients with ALS, cells are damaged and killed, thus inhibiting cell-to-cell signal communication within the body. Prior to this study, scientists discovered that enzyme RIPK1 was an essential regulator of inflammation and cell death in those with ALS. However, it was unknown how this mechanism occurred at the cellular level.
According to researchers, the RIPK1 enzyme interrupts the production of myelin, a gel-like material that protects neuron axons from injury. In essence, RIPK1 takes away the protective material that shields cells from damage, making the cells susceptible to attacks. Scientists discovered that this mechanism occurs as a chain reaction through the disruption of oligodendrocytes, the cells responsible for producing myelin. This leads to progressive damage of healthy cells over time.
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| RIPK1 disrupts the production of the protective myelin on the nerve cell’s axon. Source: health.pa.gov |
RIPK1 and Future Treatments
RIPK1 could help researchers develop new ALS treatments that slow down the progression of the condition. According to scientists, RIPK1 can be blocked in mice by a chemical called necrostatin-1. To test the new potential ALS cure, researchers studied genetically altered mice that would ultimately develop ALS. By treating these mice with necrostatin-1, scientists discovered that they could restore the cell’s protective material myelin and stop cell damage. Necrostatin-1 also prevented limb weakness in the mice treated.
According to researchers, RIPK1 may be responsible for other degenerative conditions caused by axonal damage, such as multiple sclerosis, spinal muscular atrophy, and Alzheimer's disease. The discovery of RIPK1 could potentially lead scientists to find a functional cure for ALS and other degenerative diseases.
