Cover News Reporter Wu Yujia
"Diffuse tenosynovial giant cell tumor is highly destructive to normal tissue and has a wide range of damage. It is often difficult to remove completely with surgery. The recurrence rate is very high, which seriously affects the patient's limb function and quality of life." Recently, it was reported by China The Center for Drug Evaluation of the State Drug Administration (CDE) has identified it as a breakthrough therapeutic drug for the treatment of unresectable tenosynovial giant cell tumor ABSK021. In the ongoing clinical trial, Associate Professor Zhou Yong of the Department of Orthopedics, West China Hospital of Sichuan University accepted During the interview, he said, "This drug is of great significance for patients with giant cell tumors of the tendon sheath that cannot be completely removed by surgery and obtain good therapeutic effects."
It is understood that giant cell tumors of the tendon sheath are divided into localized and diffuse, with an incidence rate of approximately Forty-three per million, of which diffuse disease accounts for about one-tenth, it is a rare disease. Although giant cell tumor of the tenosynovium is a benign tumor, because the early symptoms are insidious, patients seek medical treatment late. When they seek medical treatment, the tumor is often huge and has a wide range of invasion, making complete resection difficult. Moreover, the tumor is ineffective to radiotherapy and chemotherapy, so the recurrence rate and disability rate are very high.
"Currently, surgery has reached a bottleneck in this situation," Associate Professor Zhou Yong said, "First, tumors often invade the important blood vessels and nerves surrounding the limbs, and widely invade normal tissues. The risk of blood vessel and nerve damage during surgical resection is very high. If it is high, there may be ischemic necrosis of limbs or loss of limb function after nerve damage; secondly, tumors often invade the joint synovium, joint spaces, cartilage, bones, muscles, and tendons of the limbs, wrapping blood vessels and nerves, even if staged Despite multiple surgical resections, the recurrence rate is still high. Repeated recurrences not only cause pain and severe functional impairment of the patient's limbs, but may even lead to complete amputation of the normal structures. This disease mostly occurs in young and middle-aged people, which is where most people live their careers. In the prime time, the disease has a huge impact on patients' physiology and psychology, and patients' treatment needs are very high, so innovative treatments are urgently needed."
Research shows that overexpression of CSF1 is the main cause of tenosynovial giant cell tumor. Blocking the CSF1/CSF-1R pathway provides a new option for systemic drug treatment of TGCT. Currently, Heyu Pharmaceutical's ABSK021 for the treatment of tenosynovial giant cell tumor has completed a clinical phase 1a dose escalation trial in the United States, and is currently conducting a phase Ib multi-cohort expansion phase study simultaneously in China and the United States.
Associate Professor Zhou Yong gave an example, “There was a patient with multiple recurrences of diffuse tendon sheath giant cell tumor of the knee joint. He was unable to walk when he was enrolled and came in a wheelchair. After two courses of clinical trial treatment, the tumor shrank significantly and the patient Another patient with a giant tenosynovial tumor of the hip joint had a significantly thicker limb on the affected side than the contralateral side before enrollment. After one cycle of medication, the patient's tumor shrank significantly and both limbs were basically the same. Thickness, the effect is very obvious. Therefore, based on the good therapeutic effect and safety shown in the current clinical trials, we very much hope that this drug can benefit the majority of patients as soon as possible. "
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