They are testing in people a new drug against ELA

Washington, July 17 (Prensa Latina) Scientists test a new drug in people to fight a hereditary form of amyotrophic lateral sclerosis (ALS) after testing in mice with improved survival and functions neuromuscular, reports The Journal of Clinical. Investigation.

Research, conducted by the University of Washington School of Medicine, launched phase two clinical trials to determine if the drug can help people with this disease, caused by mutations in the SOD1 gene.

Previously, the drug was tested on mice and with only one or two doses, the effects were impressive, says Timothy Miller, one of the project's specialists.

"We have high hopes. We have completed the first phase of safety testing, and now we are working to find the right dose, "said neurologist Timothy Miller.

Specialists have provided two compounds known as antisense oligonucleotides to genetically modified rodents with a mutated form. of the SOD1 gene that prevented them from walking and feeding autonomously.

In comparison with others who received a placebo, mice that consumed the active drug maintained their weight, lived longer and improved their muscle function. Neurological damage increased, in all cases it was faster in those who did not receive treatment.

According to Robert Bucelli, of the team of scientists, the human test is designed to badess the safety of the use of oligos in people and it ensures that until the tests Have identified no obvious danger.

Now, different doses are being tested to find the most effective way to reduce SOD1 levels without causing unacceptable side effects.

About 10% of ALS cases are hereditary. Of these, 15.0 percent are caused by mutations in the SOD1 gene, which cause too much activity in the protein, suggesting that reducing their levels could help patients with mutations in this gene. gene.

oda / mfg