New Biomarker Assay May Help Predict PFS in Neuroblastoma Patients

A five-gene neuroblastoma B5 assay for NB-mRNA may help improve definition of disease status and correlates independently with progression-free survival (PFS) in patients with relapsed/refractory neuroblastoma, according to a new study published in Clinical Cancer Research.

Researchers conducted a systematic comparison of disease burden quantification in relapsed neuroblastoma patients by standard evaluations compared to a new TaqMan® Low Density Array assay for mRNA of five neuroblastoma genes (NB5 assay). They found that the NB5 assay may enhance standard evaluations and provide independent prognostic information in this patient population.

Investigators at The Saban Research Institute of Children’s Hospital Los Angeles have developed and tested this new biomarker assay for quantifying disease and detecting the presence of neuroblastoma even when standard evaluations yield negative results for the disease. Study investigator Araz Marachelian, MD, of the Children’s Center for Cancer and Blood Diseases, said there is some remaining tumor in the body that often times cannot be detected with standard tests and physicians have a hard time predicting if a patient is likely to relapse.

“We have found that we could use this assay in patients with relapsed or refractory neuroblastoma to predict the likelihood of progression. Once this assay is validated further, the hope is that we can share the results of the assay to help patients, families, and physicians make treatment decisions,” Dr. Marachelian told OncoTherapy Network.

Previously, assays used for detecting disease screened for only one NB-associated gene at a time, which was less effective. Instead of running five different tests, the research team figured out a way to test for multiple neuroblastoma-associated genes simultaneously using a different technology platform.

Standard clinical evaluations such as scans (CT, MRI, and metaiodobenzylguanidine) and bone marrow evaluation can be limited in their ability to estimate PFS. Dr. Marachelian, who is also an assistant professor of Clinical Pediatrics at the Keck School of Medicine of the University of Southern California, said this new assay could have the potential to act as an advance warning system. She said it can allow clinicians to determine if patients are getting worse and be poised to take action. Alternately, if the clinicians see things are getting better or the disease is no longer detectable even with this very sensitive test, then it may be possible to decrease treatment and protect the patient from unnecessary exposure to toxicity and side effects.

Now that that the team has validated the new assay for its predictive ability, they have begun running the test for each individual therapeutic to see how the results of the assay change in response to different kinds of treatments.