Meddling with medulloblastoma

SANTA CLARA, Calif.—Another childhood cancer is coming underfire thanks to a new partnership, with NextBio, the Winship Cancer Institute ofEmory University and the Aflac Cancer Center teaming up to targetmedulloblastoma, the most common type of brain tumor seen in children. Thepartners will use NextBio Clinical for the interpretation of molecular andgenomic data from children with medulloblastoma in hopes of finding biomarkerscapable of predicting the cancer's metastasis.

"Emory and the Aflac Cancer Center's ability to performgenomic studies on patients and then to use NextBio Clinical's correlationengine to compare the genomic profile of primary tumors with that of metastatictumors, both across our data and across the large amount of data that NextBiohas curated from the public domain, makes achieving our goal of improvingoutcomes for people with medulloblastoma seem nearer in sight," says Dr. TobeyMacDonald, director of the Brain Tumor Program at the Aflac Cancer and BloodDisorders Center of Children's Healthcare of Atlanta, associate professor ofPediatrics at Emory University and principal investigator of the study.

Medulloblastoma is the most common pediatric brain tumor,and primarily affects children between the ages of five and nine years. Thishigh-grade type of tumor affects approximately 500 children in the UnitedStates each year, and accounts for 20 percent of all brain tumors in childrenunder the age of 19. Though genetic and chromosomal changes have beenidentified that might play a role in its growth, the cause of medulloblastomais unknown, and there is no way to predict its metastasis.

The current standard of care for pediatric patients, afterremoving as much of the tumor surgically as possible, is radiation therapy.According to the American Brain Tumor Association, "with current therapies, 70percent to 80 percent of children with average-risk medulloblastoma can beexpected to be alive and free of disease five years from diagnosis. Even inthose children with high-risk disease, effective therapy is possible andresults in long-term disease control in as high as 60 percent to 65 percent ofpatients."

However, as noted by MacDonald in a press release, "Theproblem with giving radiation to all children with medulloblastoma is that itcauses long-term side effects and toxicity in young growing brains." Thediscovery of biomarkers combined with a summary of each patient's data wouldallow physicians to predict whether a child stands to benefit from radiationtherapy enough to make it worth the risk of associated damage.

That's where NextBio comes in. NextBio Clinical will gatherthe data collected on patients and will condense it into a usable report, saysSaeid Akhtari, president, CEO and co-founder of NextBio. The company collectsthe data in an anonymized, private cloud that is HIPAA-compliant.

"We take the individual patient's clinical data andmolecular profiling data, and we generate a report for the tumor board, forpathologists or oncologists, that would help the decision process for thatindividual patient," says Akhtari.

The issue with genomic profiling, according to Akhtari, isthe sheer amount of data that is generated and how to handle it. Combining themolecular profiling and clinical data means scientists "could have billions ofdata points" for each patient. It is difficult to work with so much data, andfor most researchers there is also an issue of how to compare such volumes ofdata between patients, says Akhtari.

"NextBio Clinical platform uses a big-data technology approachto solve exactly these types of perplexing problems that doctors andresearchers have spent years trying to address," Dr. Alpana Verma-Alag, head ofclinical development at NextBio, said in a press release. "This study will lookat clinical and genomic data from real patients, as well as data from mousemodels and frozen human tissue samples, and then will correlate these data setswith other data from the public domain. Our goal at NextBio has been to notonly make this type of study possible, but also to make it very easy andefficient to perform. To help change the course of a cancer that largelyaffects children would be a great accomplishment, and NextBio is very proud tobe part of such an effort."