We’ve known for a while now how to make precision medicines, and now a group of scientists has discovered that sequencing the genes of brain tumors found in children could lead to specific treatments that target their genetic deficiencies. This is a fantastic breakthrough as could mean the introduction of many revolutionary treatments.
This recent study involved sequencing 200 tumor sample genes from children who have brain cancer. Out of the 200, researchers found that more than half of the tumors had genetic abnormalities that could affect the diagnosis or treatment they receive. Pratiti Bandopadhayay, a pediatric neuro-oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center where the study was led, says, “The reason we did this trial was that brain tumors are a leading cause of death in children, and the treatments that we and everyone else use are decades old – radiation and chemo. Our approach to try and improve on that is to target the individual tumors of each child.”
Precision medicine is hot right now, and a new initiative has just been signed called the 21st Century Cures Act which will devote $4.8 billion in federal funding to finding more specialized treatments for patients based on their genes. But, it’s still rarely used in oncology, and even less in pediatrics. Statistics show that brain tumors were responsible for as many as a quarter of all children’s cancer deaths between 2008 and 2012. This needs to change, so hopefully, Bandopadhayay’s study will help do that. During their research, they found 44 cancer mutations and 20 different rearrangements.
Tafinlar is a drug that was approved by the FDA back in 2013. It was created mainly to target the mutated gene BRAF and was approved in 2013 by the FDA. There are currently several ongoing clinical trials involving Tafinlar being carried out in pediatric brain tumors, and so far have shown promising results. Among those involved in the trial was a seven-year-old girl who had been diagnosed with a particular type of brain tumor called disseminated ganglioglioma. The results showed that within just three months of receiving Tafinlar treatment improvements in her health were seen.
All children that receive treatment at the Dana-Farber Center are offered genetic sequencing of their tumors routinely, which makes them special as not many other centers are set up for this type of testing. But, Bandopadhayay is hopeful that this new research will encourage more oncologists and practices to start using tumor sequencing. Rameen Beroukim, an oncologist at Dana-Farber and co-author of the paper, says, “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.”
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