The study reveals that brain tumors in babies are molecularly unique and could be candidates for targeted treatments.
A new study published in Cancer Discovery shows a possible alternative to chemotherapy for infants with high-grade glioma tumors. The study, funded by the CRIS Cancer Foundation, The Brain Tumour Charity, Children with Cancer UK, Great Ormond Street Hospital Children’s Charity and Cancer Research UK, reveals that brain tumors in babies are molecularly unique and could be candidates for targeted treatments.
High-grade gliomas, tumors of the glial cells, are a rapidly growing type of brain tumor that 150-180 children in North America are diagnosed with each year. More than half of all brain tumors in children and gliomas. Children who are diagnosed as infants, less than 12 months, have a better chance at survival, with only 20% of older children living past five years.
“It’s especially cruel when cancer affects such small children, so it is encouraging that this study offers some hope for very young patients and their families,” said Professor Paul Workman, Chief Executive of The Institute of Cancer Research, London. “The research offers a better understanding of the biology of brain tumors in infants and finds that they might potentially be treated with new targeted drugs. That’s vitally important because infants are particularly vulnerable to the side effects from standard treatments such as chemotherapy, and we need much better, kinder options.”
This new research has found that when cancer is looked at in infants, the tumors are biologically different than those in older children. Scientists discovered that infant tumors could be targeted with precision medicines because of the genetic make-up of the tumors, meaning no chemotherapy, which can have devastating side-effects and be harmful while their brain is still developing.
“Our study offers the biological evidence to pick out those infants who are likely to have a better outcome from their disease, so these very small children and their families can be spared the harmful effects of chemotherapy,” said Professor Chris Jones, Professor of Pediatric Brain Tumour Biology at The Institute of Cancer Research, London.
Scientists at The Institute of Cancer Research in London, working with colleagues at the Hopp Children’s Cancer Center Heidelberg in Germany, the UCL Great Ormond Street Institute of Child Health and St. Jude Children’s Research Hospital in the U.S., looked at 241 infants diagnosed with glioma brain tumors. This is the largest and most comprehensive study of infant gliomas to date. Research showed a genetic difference from the samples of infant and children’s tumor samples, though they look similar under a microscope.
Of the 241 samples, 130 of them had entirely different genetic make-up from other forms of childhood brain tumors. Around 65 of the 130 unique types of tumors had specific molecular weaknesses like ALK and NTRK gene fusions that can be targeted with non-chemo medicines.
In research with mice, they found mice treated with Lorlatinib had a significant shrink in tumor size, compared to those treated with chemotherapy, who’s tumors kept growing, although at a slower rate.
In ‘mini tumors’ grown from patient samples, tumors with NTRK and other genes were sensitive to targeted treatments.
A small number of patients from the study were able to be successfully treated with ALK or NTRK targeted drugs.
These results could mean new hope for families affected and are set to change the World Health Organization’s diagnostic guidelines with brain tumors in infants to be classed separately from other childhood brain tumors.
“Chemotherapy is currently a good treatment option for babies with brain tumors – but our study has found that some children could be spared this treatment. With further testing, I’m hopeful that existing targeted drugs could expand our arsenal of options to treat these smallest of patients,” said Dr. Matthew Clarke, Clinical Research Fellow in the Glioma Team at The Institute of Cancer Research, London.
