T-ALL is a blood cancer that frequently develops from children to the AYA generation (adolescents and young adults).Although treatment with a combination of bone marrow transplantation and anticancer drug is being performed, the 1990-year survival rate of T-ALL treated from 2001 to 35 is XNUMX%, and the prognosis is extremely poor.In particular, since patients with central nervous system (brain) metastasis of leukemia cells cannot be expected to survive for more than XNUMX years, the development of a new therapeutic agent effective for patients with brain metastasis has been eagerly desired.

　In previous studies, the group found that lysine-specific demethylase (LSD1) was involved in the development of T-ALL, and in this study, inhibition of LSD1 resulted in the development of T-ALL. I thought I could prevent it.As a result of the examination, we succeeded in creating a new LSD1 inhibitor that has excellent transferability to the brain, high specificity, and is effective even at low concentrations.This LSDXNUMX inhibitor showed the effect of suppressing the growth of leukemia cells metastasized to the brain and prolonging the survival time in leukemia model mice.If the safety and efficacy in humans are verified by clinical trials in the future, it can be expected to have a significant life-prolonging effect as the world's first molecular-targeted drug effective for brain metastasis.

　This result is an important discovery that leads to improvement of the prognosis of T-ALL, and a patent has been filed under the name of "a novel compound having lysine-specific demethylase 1 inhibitory activity, its production method and its use". There is.It is currently in talks with a pharmaceutical company for clinical application.

Paper information:[Clinical Cancer Research] Eradication of central nervous system leukemia of T-cell origin with a brain-permeable LSD1 inhibitor