Team Jack Credited Nationally for Funding Groundbreaking Research

Dr. Susan Chi, MD, Pediatric Neuro-Oncologist Harvard Medical School

Team Jack Credited Nationally for Funding Groundbreaking Research

Monday, March 6, 2017

The Team Jack Foundation has invested in multiple research projects across the United States. One of the Foundation’s earliest grants was to Dana Farber Cancer Center in Boston, MA. The following is a report of research made possible by this grant. This excerpt was reported online on January 19, 2017 by the journal Neuro-Oncology:

Precision medicine—in which diagnosis and treatments are keyed to the genetic susceptibilities of individual cancers—has advanced to the point where it can now impact the care of a majority of children with brain tumors, a new study by investigators at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center suggests.

In the largest clinical study to date of genetic abnormalities in pediatric brain tumors, researchers performed clinical testing on more than 200 tumor samples and found that a majority had genetic irregularities that could influence how the disease was diagnosed and/or treated with approved drugs or agents being evaluated in clinical trials. The findings demonstrate that testing pediatric brain tumor tissue for genetic abnormalities is clinically feasible and that in many cases the results can guide patients’ treatment.

The need for new approaches to testing brain cancer in children is urgent, the study authors say. “Although there has been a great deal of progress over the past 30 years in improving survival rates for children with cancer, advances in pediatric brain cancer haven’t been as dramatic,” says co-lead author Pratiti Bandopadhayay of Dana-Farber/Boston Children’s, who is also an instructor in pediatrics at Harvard Medical School (HMS). “In a recent study, brain tumors accounted for 25 percent of all pediatric deaths attributed to cancer. In addition, many of the current therapies can result in long-term difficulties in cognitive or physical functioning,” adds Bandopadhayay.
The researchers plumbed the genomes of 203 pediatric brain tumor samples, representing all major subtypes of the disease. They analyzed the DNA for irregularities in 300 cancer-related genes, and also studied how many copies of genes were missing or overabundant within the tumor cells. Results showed that 56 percent of the samples had genetic abnormalities that were clinically relevant—that could impact a patient’s diagnosis or be targeted by drugs already in clinical use or under study in clinical trials. (Many of these drugs cross the blood-brain barrier, the dense web of cells that can prevent medicines from exiting the bloodstream to reach the brain.)

Among the findings:
Alterations were found in the gene BRAF, one of the most commonly mutated genes in pediatric brain tumors, and one for which several targeted drugs are being tested. Testing revealed clinically relevant abnormalities in 89 percent of medulloblastomas, which account for nearly a fifth of all brain tumors in children.
“The importance of genomic profiling in the diagnosis and treatment of pediatric brain cancers is reflected in the World Health Organization’s recent decision to classify such tumors by the genetic alterations within them, rather than by broad tumor type” says study co-senior author Dr. Susan Chi of Dana-Farber/Boston Children’s, an assistant professor of pediatrics at HMS. “Targeted therapies are likely to be most effective when they’re matched to specific abnormalities within tumor cells. Our findings show that precision medicine for pediatric brain tumors can now be a reality.”

Precision Medicine in Action:
The Team Jack Foundation is also currently funding a clinical trial testing one of these therapies that targets a mutation in child brain tumors.
The MEK162 trial is testing a MEK1/2 inhibitor and is currently in Phase II, which evaluates the effectiveness of the therapy. This study is spearheaded by Dr. Nathan Robison and the Children’s Hospital of Los Angeles and includes 15 different institutions across the United States.

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