Cell Discov: Zhao Yao's team at Huashan Hospital and Guo Guoji's team at Zhejiang University have discovered a new drug for targeted treatment of refractory intracranial choroidal tumors

On September 20, the team of Professor Zhao Yao of the Department of Neurosurgery of the National Medical Center for Neurological Diseases and Huashan Hospital affiliated to Fudan University and the team of Professor Guo Guoji of Zhejiang University published a research paper online in Cell Discovery (Impact Factor 38.

The research team used single-cell RNA sequencing technology to find for the first time a cluster of stem cell-like cells in malignant cells of skull-base chorma, and this cell cluster was closely related
to the radiation therapy resistance of tumors.

On this basis, Professor Zhao Yao's team conducted a Phase I clinical trial in Huashan Hospital, and confirmed in 3 chordoma patients that taking p-EMT inhibitors (YL-13027) can significantly slow tumor growth and maintain a progression-free state
for at least 6 months.

This study is the first time that stem cell subsets and p-EMT processes exist in skull fundus chordates at the single-cell level, and the therapeutic effect of radiotherapy sensitizers and p-EMT inhibitors has been verified in the laboratory, and subsequent clinical trials have further confirmed the clinical effects
of p-EMT inhibitors.

Skull base chordoma (SBC) is a rare malignant bone tumor
originating from residual tissue in the ridge cord of the slope or saddle area.

In addition, residual tumors are not sensitive to conventional radiation therapy and there is no recognized effective drug, resulting in patients with skull base chordomas often facing the dilemma
of "no cure" after several "surgery-relapse".

"Although skull base chordoma is a relatively rare tumor, our Huashan Hospital, as a national neurological disease medical center, still has many patients with skull base choratoma who come to visit every year
.

The research team used single-cell RNA sequencing technology to determine a total of 90,691 cells from 12 skull base chordoma samples (from 6 patients) and mapped the single-cell transcriptome of skull base chordomas
.

The research team used non-negative matrix decomposition (NMF) to cluster chordoma tumor cells and found that there were 8 modules in the chordoma tumor cells, which for the first time confirmed the presence of the p-EMT process
in the choratoma.

On this basis, Professor Zhao Yao's team further carried out a Phase I, one-arm, open-label clinical trial
in Huashan Hospital.

Dr.

Original Source:

Zhang Q, Fei L, Han R, Huang R, Wang Y, Chen H, Yao B, Qiao N, Wang Z, Ma Z, Ye Z, Zhang Y, Wang W, Wang Y, Kong L, Shou X, Cao X, Zhou X, Shen M, Cheng H, Yao Z, Zhang C, Guo G,Zhao Y.