What is the clinical effect of bosutinib in treating ALS?

ALSclinically refers to amyotrophic lateral sclerosis, a progressive neurodegenerative disease caused by the loss of motor neurons, for which there is an urgent need to develop effective drugs. Drug repurposing based on induced pluripotent stem cells (iPSC) identified the Src/c-Abl inhibitor Bosutinib (Bosutinib) As a candidate drug for the molecularly targeted treatment of ALS, Bosutinib has been approved for the treatment of chronic myelogenous leukemia (CML).

This is the first study to use the Src/c-Abl inhibitor bosutinib in patients with ALS in a novel clinical trial design that combines safety studies in oncology with a 3+3 design and efficacy studies in neurology. The overall safety and tolerability of bosutinib in ALS was assessed using a 3+3 design, in which the maximum tolerated dose and phase 2 recommended dose was determined to be 300 mg. It was found that a subset of patients responded well to bosutinib, as demonstrated by clinical stability maintained over 12 weeks, and treatment response patients could be distinguished by plasma NFL levels, which may be a potential predictor of drug efficacy.

The originator drug of bosutinib is not marketed in the country, so it cannot be included in medical insurance. Currently, the original drugs sold overseas include Turkish version and European version. The price of the Turkish version may be more than RMB 2,000 per box of 500mg*28, and the price of the European version may be more than RMB 30,000 (the price may fluctuate due to the exchange rate). There are also bosutinib generic drugs produced overseas in other countries. Their pharmaceutical ingredients are basically the same as those of the original drug. The price of 500mg*30 per box may be more than 2,000 yuan (the price may fluctuate due to the exchange rate), and the price is relatively cheap.