Causes of ALS: Growth Factor Deficiency

Growth factors are natural proteins or steroid hormones that stimulate cell growth and proper cell development. They’re also important for regulating a variety of cell processes.

The idea that the cell growth factor VEGF (vascular endothelial growth factor) might play a part in starting or maintaining ALS came from the discovery of ALS symptoms in mice carrying a human mutated VEGF gene. As the mutant mice underproduced that growth factor, they also developed progressive loss of muscle function and motor neuron death.

In humans, three different mutations in the VEGF gene have been marked as significant risk factors for ALS, following a large study of European patients.

VEGF isn’t the only growth factor implicated. Like VEGF, other growth factors such as IGF-1 (insulin-like growth factor) and GCLN (glial cell line-derived neurotrophic factor) also appear to have nerve-protective properties. In principle, loss or deficiency of these factors would make motor neurons more vulnerable to damage.

Ongoing clinical trials of VEGF should tell if it’s a useful therapy.

Learn more about the research projects funded and coordinated by the Packard Center for ALS Research at Johns Hopkins, and targeted at finding the causes of ALS and a cure. Subscribe to ALS Alert to stay informed about ALS research and clinical trials.