ACT001, the new drug candidate originated from the research group of Professor Chen Yue from the National Key Laboratory of Medicinal Chemistry and Biology of Nankai University and the College of Pharmacy, has recently obtained an accelerated approval from the State Food and Drug Administration, after a re-application by the cooperative enterprise for clinical trial of the treatment, combined with radiotherapy of brain metastases from solid tumors.

The Treatment of Brain Metastases from Solid Tumors (2017): guidelines from the European Association of Neuro-Oncology has stated that the incidence of newly diagnosed brain metastases is 3-10 times that of newly diagnosed primary malignant brain tumors, and most brain metastases patients’ life expectancy is limited. That is because the appearance of brain metastases is often regarded as a sign of the end stage of tumor metastases. Lung cancer and breast cancer are the most common cancers with brain metastases. The clinical treatment methods for them mainly include surgery and radiotherapy.

“However, due to the presence of the blood-brain barrier, most drugs are difficult to enter the brain, so they are often not very effective for intracranial tumors. Except for a small number of patients with driver gene mutations receiving certain targeted drug treatments, there is no standard treatment for brain metastases from solid tumors. Hence, there is a very broad and urgent clinical need”, Associate Professor Zhang Quan, a member of Professor Chen Yue's team, said.

In 2013, the Research on the Discovery and Derivatization of Sesquiterpene Lactone Compounds that can Cross the Blood-Brain Barrier and Their Anti-glioma Stem Cell Activity, a general program chaired by Zhang Quan, has been approved by the National Natural Science Foundation of China. This approval greatly supported the team, making it dedicated to the discovery of drug candidates that can enter the brain.

After 9 years of efforts, the team of Nankai University has independently developed the new anti-glioma drug ACT001, which can break through the blood-brain barrier and enter the brain. Clinical trials have been carried out in Tianjin Medical University Cancer Institute & Hospital and in several hospitals in Australia. Professor Wang Ping of Tianjin Medical University Cancer Institute & Hospital presided over the phase I clinical study in China. On this basis, Tianjin Medical University Cancer Institute & Hospital will carry out another two 1b / 2a clinical studies. ACT001 has obtained the Orphan Drug certification for treatment of glioblastoma in the European Union and in the United States, and the phase 2 clinical trial scheme has also received ethical approval.

(Reported by Junhui Wu, Translated by Xinghua Li, Edited by Davide Francolino and JianjingYun)