Clinical Trials and Tumor Banking

The clinical trials effort for the PLGA Program is spear-headed by Mark Kieran, MD, PhD. Our clinical trials arm has two specific aims, the first is to increase the Program’s access to biological materials that can be used to support the scientific investigations; the second aim is to improve the translation of laboratory findings into clinical trials for children with low-grade gliomas.  More information on the clinical efforts, including trials currently recruiting can be found at Dana-Farber/Boston Children’s Cancer and Blood Disorder Center.

Some highlights of our clinical trials include:

    1. A completed trial for KIAA1549-BRAF truncation/fusion recurring tumors (results are being made ready for publication soon)
    2. A completed trial for patients with neurofibromatosis type 1 and recurrent or progressive glioma.
    3. A trial targeting tumors with the BRAF V600E mutation.  This particular therapy targets the mutated BRAF itself, rather than the downstream effectors.
    4. A trial for patients with mutations in FGFR1 and low-grade glioma, in concert with other hospitals in the USA.
    5. A trial undergoing regulatory review this year will target the downstream effectors of the truncation/fusion form of BRAF, in patients scheduled to undergo surgery.  This will allow us to determine the concentration of drug penetrating the brain and the percentage of target inhibition in resected samples.
This figure illustrates the accrual of tumors for research purposes to the PLGA Program over the past seven years, with a breakdown showing each tumor type.

This figure illustrates the accrual of tumors for research purposes to the PLGA Program over the past three years, with a breakdown showing each tumor type.
(Click on image to enlarge it.)


Hayley Malkin

    Our tumor banking efforts continue to improve over the past year, thanks to the work of a dedicated pediatric neuro-oncology tissue banking research co-ordinator. Hayley Malkin manages the accrual of participants to the clinical protocol and the distribution of tissue among groups of researchers. All new patients are offered participation in this study at surgical resection or biopsy. Brain tumor patients are also approached to discuss banking during outpatient visits to the Jimmy Fund Clinic. If a participant has excess tumor tissue to donate to research studies, the research coordinator acts as a liaison between clinical and basic science research staff to deliver tissue samples to the groups with active research projects. She also performs a detailed abstraction of clinical data to accompany the excess tissue, building a resource, which may be used in to create cohorts for projects or correlate clinical and molecular information.

Selected Publications

Diffuse intrinsic pontine glioma: a reassessment
J Neurooncol. 2014 Aug;119(1):7-15.

Targeting BRAF in pediatric brain tumors
Am Soc Clin Oncol Educ Book. 2014:e436-40.