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Neuroblastoma, an embryonic tumor of the peripheral sympathetic nervous system, is the third most common pediatric cancer and the most common cancer in infants. This cancer is mostly diagnosed in infancy, an average of 1 to 2 years of age. It is estimated that there are 700 to 800 new cases of neuroblastoma in the United States each year.
Although the survival rate of medium- and low-risk neuroblastoma is very high, the 5-year survival rate of high-risk neuroblastoma is about 50%, and the likelihood of recurrence is high even with high-intensity multimodal treatment. There is currently no standard treatment for patients with relapsed/refractory neuroblastoma.
On March 25, 2022, according to a press release from Asia Healthcare, the FDA awarded the targeted inhibitor allizomadlin (APG-115) rare pediatric disease (RPD) designation for the treatment of neuroblastoma.
Alrizomadlin is a novel oral MDM2-p53 inhibitor that has shown anti-tumor activity in in vitro and in vivo models of neuroblastoma.
Alrizomadlin selectively inhibits the interaction of the MDM2 protein with p53, leading to tumor suppressive activity (this activity is blocked due to the overexpression of MDM2 in tumors). Alrizomadlin has also shown antitumor activity in preclinical studies of solid tumors and acute myeloid leukemia.
In a Phase 2 clinical trial (NCT03611868), patients with multiple solid tumors who did not respond to immunotherapy received a combination of allizomadlin and pembrolizumab (Pembrolizumab) showed antitumor activity and were well tolerated.
Currently, a Phase 1 clinical trial (NCT02935907) is underway, and the drug is used as monotherapy to treat patients with advanced solid tumors or lymphomas. Early trials of Alrizomadlin in combination with other drugs are also recruiting patients. AsunTech plans to conduct a clinical study in neuroblastoma patients in the near future.