Professor Luis Barbeito of the Institut Pasteur in Montevideo, Uruguay, presented the findings at the 27th International Symposium on ALS/MND (Motor Neuron Disease) in Dublin, Ireland, in December of 2016.
Masitinib is an oral treatment that inhibits proteins known as tyrosine kinases that play important roles in cells. Preventing the proteins’ activation helps fight cancer, inflammatory diseases and disorders of the central nervous system (CNS).
Researchers have learned that masitinib reduces inflammation in immune-system cells such as mast cells and microglia that can promote the development of ALS.
“Beyond our recently published findings, we have acquired additional preclinical data showing neuroprotective effects of masitinib in ALS,” Barbeito said in a news release. “We have now shown that masitinib generates its observed neuroprotective effect in ALS by regulating neuroinflammation in the peripheral nervous system [the part of the nervous system that regulates muscles] as well as the central nervous system and that it also penetrates the blood-brain barrier to a greater extent than previously thought. Overall, these data provide further compelling pharmacological rationale for the recently reported positive phase 3 interim analysis.”
A previous study of rats carrying a genetic mutation that leads to ALS showed that masitinib inhibited microglia and prolonged the lives of animals that developed the paralysis associated with the disease.
Meanwhile, interim data from a Phase 2/3 trial (NCT02588677) showed that masitinib and the ALS treatment riluzole improved patients’ quality of life and ability to survive. Researchers used the Amyotrophic Lateral Sclerosis functional rating scale, which measures progression of the disability, quality of life and survival, to assess patients who had taken 48 weeks of treatment.
Masitinib was granted Orphan Drug status in 2016 by the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products.