Children with acute myeloid leukemia (AML) who develop drug resistance generally lack treatment options and are deemed incurable. Recently, The CUHK’s HK-HOPE developed a method to obtain the sensitivity of a patient’s cancer cells to a basket of targeted drugs within three days. In combination with genomic analyses, AML patients are matched with the most effective drugs according to their individual conditions.
AML is a rare but highly aggressive blood cancer that accounts for five percent of pediatric cancers. In Hong Kong, there are about 10 new cases per year. Chemotherapy with stem cell transplantation has remained the standard of care for decades. Advances in medical development have significantly improved the overall survival rate of newly diagnosed AMLpateints to 70 percent. However, the chance of survival drops below 40 percent for patients who develop relapses.
The research team of CUHK’s HK-HOPE performed comprehensive drug and genomic profiling of bone marrow biopsies from 52 children with AML and integrated it with their clinical parameters to deliver the first high-dimensional gene-drug-clinical dataset. It has been made freely accessible to all researchers around the globe to stimulate further basic, translational and clinical research on this rare blood cancer.
From the dataset, the researchers also uncovered a children-specific pattern of drug response, linkage to genetic mutations, and active targeted agents with immediate clinical relevance. The study identified a wealth of targeted agents from an approved drug library showing better effectiveness over standard chemotherapeutics against pediatric leukemia. The findings enable the prioritization of new drugs to be tested in upcoming clinical trials.
Through the drug and genomic analysis, the team developed the first precision medicine for pediatric leukemia patients. This enables evidence-based drug selection for relapse cases that can extend patients’ survival or even cure the disease.
Professor Leung Kam Tong, Assistant Professor of the Department of Pediatrics at CU Medicine, said that with that testing method, they could obtain drug sensitivity testing results within three days. In combination with genomic analysis, they could match the correct drugs to individual leukemia patients who had no further treatment options in a timely manner, bringing hope to those sick kids.
Professor Li Chi Kong, Professor of the Department of Paediatrics at CUHK’s Faculty of Medicine (CU Medicine), stated that a 14-year-old AML patient using the new precision medicine approach had experienced a relieving condition with his survival extended by two and a half years, fulfilling the patient’s wish.
After treatment, another 14-month-old patient also experienced disease remission with significantly prolonged survival.
Professor Albert Martin Li, Director of HK-HOPE and Chairman of CU Medicine’s Department of Pediatrics, added that they had successfully implemented precision medicine-guided management in childhood AML patients by making good use of valuable data and that the personalized medicine approach could be extended to other leukemia types.
