In a significant development in the field of oncology, a novel small molecule drug targeting the primary complications of various malignant tumors has been developed by research teams from East China Normal University and Fudan University.

The achievement has recently led to a global equity partnership worth $100 million with the American biopharmaceutical company Supercede Therapeutics.

The research team has developed a novel oral small molecule drug that can effectively alleviate symptoms of cancer cachexia, a disease usually developed among late-stage cancer patients.

Cancer cachexia, characterized by lack of appetite, significant weight loss, muscle atrophy and fat degradation, often culminates in multiple organ failure and death in the advanced stages of the disease. The occurrence of cancer cachexia not only diminishes the effectiveness of chemotherapy and radiotherapy, shortening the survival rate of cancer patients, but also severely impacts quality of life.

According to Professor Zhang Xiongwen from the Shanghai Center for Molecular Therapy and Drug Discovery at ECNU, 50 percent to 80 percent of late-stage cancer patients develop cancer cachexia.

When cancer cachexia occurs, the TGF-β family ligands in the blood activate ActRIIB on the surface of skeletal muscle cells, triggering various forms of protein degradation and inhibiting protein synthesis.

The research team has developed the oral small molecule drug that acts as an inhibitor targeting ActRIIB, thus reducing muscle atrophy, improving grip strength, and alleviating other symptoms of cancer cachexia.

In comparison to traditional large molecule antibody drugs that are costly to produce and require stringent transportation and storage conditions, this new drug is made of small molecules and is more suitable for oral administration, offering advantages such as convenient transport and storage, good stability, and high membrane permeability. Moreover, the anticipated market price of this new drug is only one-third of traditional large molecule antibody drugs, making it more accessible to the general patient population, according to the research team.

Source: Jiefang Daily