Precision Medicine Helps Kids with Brain Cancer

Precision Medicine Helps Kids with Brain Cancer

By Admin at 23 Feb 2017, 14:27 PM


While many pediatric cancers have seen improved survival rates in the last 30 years, brain cancer still remains a leading cause of pediatric cancer death, accounting for 25 percent of all cancer-related deaths in children.

Part of the problem, according to Pratiti Bandopadhayay, MBBS, PhD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center, in Forbes, is “the treatments that we and everyone else use are decades old — radiation and chemo.”

Bandopadhayay co-authored a study to determine if precision medicine, which involves targeting the genetic abnormalities in an individual’s tumor, could help — with promising results. Researchers sequenced the genes of 203 tumors from children with brain cancer.

More than half (56 percent) had “clinically relevant” genetic abnormalities, meaning they could likely be targeted by drugs currently on the market or being tested in clinical trials. In the case of medulloblastomas, a common type of tumor that accounts for about 20 percent of pediatric brain cancers, 89 percent had targetable genetic abnormalities.

Two different genetic tests — OncoPanel and OncoCopy — were used to analyze the tumors, revealing 44 cancer mutations and 20 rearrangements, which describe abnormal DNA patterns that may promote cancer. Dana-Farber oncologist Rameen Beroukhim, a study co-author, told Forbes:

 

"The more comprehensive sequencing we can do, the more likely we'll be to detect new gene alterations that may be causing these brain tumors … As we get more sequencing into the clinic, hopefully that will help us to develop new treatment approaches."

 

BRAF was one of the most commonly mutated genes in the pediatric brain tumors, which is noteworthy because it’s the target of the drug Tafinlar (dabrafenib). While this drug is approved by the U.S. Food and Drug Administration (FDA) to treat melanoma, clinical trials are looking at its use in pediatric brain tumors.

Preliminary research testing dabrafenib among children with low-grade glioma positive for BRAF mutation showed the drug may shrink tumors or stop their growth. Currently, only a limited number of pediatric cancer centers routinely offer patients genomic testing, but as research increasingly shows its merits the hope is that this will soon change.

The research could also lead to the establishment of FDA-approved gene-targeted treatments for pediatric brain cancer, of which none currently exist. Because of this, insurance companies may deny coverage for what are deemed experimental or non-FDA-approved treatments — a crisis that increased research may help to solve.

In the meantime, gene sequencing tumors in pediatric brain cancer patients is set to be a game changer in the diagnosis and treatment of this disease.

In related news, a Gateway-funded clinical trial has been cited in the prestigious New England Journal of Medicine, and other major news sources, for breakthrough success using immunotherapy to fight aggressive brain cancer.


Sources:

Neuro-Oncology January 19, 2017
Annals of Oncology October 13, 2016
Forbes January 19, 2017
EurekAlert January 19, 2017


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