Olesoxime

Olesoxime

0Olesoxime (also known as TRO19622) is a drug that was under development for the treatment of Amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. It is a cholesterol-like compound that targets mitochondria, the powerhouses of cells, to boost their function and protect against damage.

History

Olesoxime was developed by the French biotechnology company Trophos. The development of the drug began in the early 2000s, with the aim of treating neurodegenerative diseases. In 2014, the company was acquired by Roche, a Swiss multinational healthcare company, which continued the development of Olesoxime.

Mechanism of Action

Olesoxime works by interacting with two proteins in the outer membrane of mitochondria, VDAC (voltage-dependent anion channel) and TSPO (translocator protein). By binding to these proteins, Olesoxime stabilizes the mitochondria and helps to maintain their function, even in conditions of stress. This is particularly important in neurodegenerative diseases, where the function of mitochondria is often compromised.

Clinical Trials

Several clinical trials have been conducted to test the safety and efficacy of Olesoxime. In a Phase 2 trial involving patients with ALS, Olesoxime was found to slow the progression of the disease and improve survival rates. However, a Phase 3 trial was halted in 2017 due to difficulties in demonstrating a clear benefit of the drug.

Future Prospects

Despite the setback in clinical trials, research into Olesoxime continues. Scientists are exploring its potential use in other neurodegenerative diseases, such as Spinal muscular atrophy (SMA) and Huntington's disease.

See Also

• Amyotrophic lateral sclerosis
• Neurodegenerative diseases
• Mitochondria
• Clinical trials