Brain cancer drug cleared for phase III clinical trials

A brain cancer treatment drug developed by scientists in Tianjin has been approved to enter phase III clinical trials, with early data showing it can extend the lifespan of some brain tumor patients by more than 50 percent.

"This is the world's first drug of its kind and Tianjin's second innovative drug to receive Breakthrough Therapy Designation. It can cross the blood-brain barrier to treat brain tumors," said Chen Yue of the State Key Laboratory of Medicinal Chemical Biology at Nankai University in Tianjin.

Chen's team spent 15 years developing the drug, known as ACT001, which can treat patients with brain metastases from solid tumors such as small cell lung cancer and non-small cell lung cancer. He added that Australia, the United States, Canada and Germany have also applied to conduct clinical studies of the drug.

The blood-brain barrier, which protects the brain from foreign substances, makes it difficult for most drugs to enter and poses major challenges for developing new therapies. "Even if some substances manage to get in, the brain's pump-like mechanism will expel them," Chen said. Because of this, radiotherapy remains one of the most common treatments for brain tumors, but it has not significantly improved survival rates for patients with advanced disease.

Chen noted that SCLC is a rapidly growing cancer with early metastasis and a strong tendency to spread to the brain, causing more than 200,000 deaths worldwide each year. Between 50 and 80 percent of SCLC cases develop brain metastases. Once the cancer spreads to the brain, survival is typically less than a year and treatment becomes far more difficult.

Scientists at Accendatech, which developed ACT001, said the drug can work in combination with immunotherapy, chemotherapy and radiotherapy, enhancing anti-tumor immunity and extending survival.

"For patients with brain metastases from small cell lung cancer enrolled in the phase IIb study, compared to radiotherapy alone, ACT001 combined with radiotherapy extended median survival by 53 percent, meaning half of the patients survived more than 9.5 months," Chen said. "If further combined with immunotherapy drugs, survival can be extended by 75 percent."

Limited data also showed that patients with NSCLC brain metastases achieved a doubling of survival time in the same study.

"This is a broad-spectrum anticancer drug. It makes a breakthrough in treating patients with brain metastases. Although data from earlier studies is limited, theoretically it could also benefit patients with other types of cancer," Chen said.

The phase III ACT001 study, recently approved by the National Medical Products Administration, will involve about 50 hospitals across China, including the Shandong Cancer Hospital in Jinan, Shandong province, Cancer Hospital of the Chinese Academy of Medical Sciences in Beijing and Sun Yat-sen University Cancer Center in Guangzhou, Guangdong province.

Chen explained that before a new drug can be approved, it must pass three phases of clinical trials. About 40 percent of drugs are eliminated in phase I, 70 to 80 percent in phase II, and 30 to 40 percent in phase III.

"It is estimated that in about 18 months, we will obtain the phase III trial results and apply for market approval," Chen said. SCLC patients with brain metastases can apply to participate in the trial through contact information listed on the study's website.

ACT001 has received five important research credentials from China, Europe and the US, including orphan drug designation, rare pediatric disease designation, fast track status and Breakthrough Therapy Designation. The rare pediatric disease designation from the US Food and Drug Administration was the first of its kind awarded to a drug developer from China.

"For those with metastatic and primary brain tumors, ACT001 has the potential to become the first drug to sensitize radiotherapy, chemotherapy and immunotherapy, offering safer and more effective treatment," Chen said.

Wang Xiaojing contributed to this story.