Antibodies can help fight incurable disease suffered by Stephen Hawking
Biologists from Israel synthesized antibodies to the defective protein SOD1, which causes amyotrophic lateral sclerosis, a condition from which the famous physicist suffered. The resulting antibodies bind specific regions of this protein, preventing its aggregation and the development of the disease.The proposed antibodies were tested on mice and showed high efficacy in preventing the development of amyotrophic lateral sclerosis. They bound to epitopes, fragments of the SOD1 protein that only defective copies have, preventing their accumulation. At the same time, "healthy" copies of the SOD1 protein did not bind with these antibodies and continued to perform their functions. The synthesized antibodies reduced the amount of the defective protein in the cells, restored motor neurons, and led to a slowing of the development of the disease.
Normally, the SOD1 protein is required in our bodies, including nerve cells, to protect against oxidation. With some mutations, the formation of this protein is impaired, and such defective molecules stick together and accumulate in the nerve cells. This leads to the development of the currently incurable disease amyotrophic lateral sclerosis, which gradually destroys motor neurons, causing paralysis of muscles, including respiratory ones.
Currently, to reduce the toxic effects of the defective SOD1 protein, drugs block the expression of this protein in nerve cells, including the "healthy" form of this protein, which deprives the nervous system of protection against oxidation. The development of antibodies will allow the use of targeted therapies targeting only misfolded proteins, reducing the risk of collateral oxidative damage to the nervous system.