ABC2, Exosome collaborate to explore RNA biofluid-based diagnostic technology in brain cancer

Feb 6 2012

Accelerate Brain Cancer Cure (ABC2) and Exosome Diagnostics are collaborating with leading academic medical centers to accelerate clinical validation of Exosome's blood and cerebrospinal fluid-based molecular diagnostics technology in brain cancer. 

The collaboration will explore the capabilities of Exosome RNA biofluid-based diagnostic technology for early identification, progression monitoring and disease risk stratification in glioma, the most common form of brain cancer.

Brain cancer is the leading cause of death among children and young adults under age 20. This year, more than 200,000 people in the United States will be diagnosed with either a primary or metastatic brain tumor.  There are more than 120 different types of brain tumors, making specific diagnosis and effective treatment extremely complicated. In many cases, accessing brain tissue via biopsy carries significant risk or is not surgically feasible.  The ability to sample a brain cancer's genetic characteristics through a blood or cerebrospinal fluid sample could contribute greatly to driving advances in clinical treatment and drug development.

This collaboration will bring together world-leading clinicians, researchers and industry participants to develop the potential of stable, high-quality disease-specific RNA harvested from exosomes found in blood and cerebrospinal fluid. The joint effort will support near-term, in-vitro diagnostic validation of known tumor and immune-derived clinical biomarkers for brain cancer.

"We are impressed with the catalytic approach of Exosome Diagnostics and our academic partners," said Max Wallace, chief executive officer of Accelerate Brain Cancer Cure.  "The ability to identify and track specific pathway mutations over time could significantly improve brain cancer patient care." 

As early as 2007, Exosome Diagnostics' researchers from Massachusetts General Hospital reported detecting key gene mutations in the blood of brain cancer patients.  Subsequent studies involving a multi-center investigative effort led by Dr. Bob S. Carter, professor and chief of neurosurgery at University of California, San Diego, and Drs. Fred Hochberg and Xandra Breakefield of Massachusetts General Hospital, have shown blood and cerebrospinal fluid exosome populations containing virtually the entire disease-specific population of the transcriptome can be accessed safely multiple times, from diagnosis through tumor progression, without the need for a surgical procedure.  These studies were conducted as part of Exosome Diagnostics' neuro-degenerative disease program examining biofluid-based exosomes for tumor and immune response genetic abnormalities in brain cancer, Alzheimer's disease and traumatic brain injury patients.

"Accessing the stable RNA contained in blood and CSF exosomes gives us a significant advantage when it comes to detecting and understanding genetic changes inside the brain compartment caused by a tumor or immune response without the need for surgical biopsy," added James McCullough, chief executive officer of Exosome Diagnostics.  "Collaborating with ABC2 helps ensure we are asking the right questions and structuring our clinical studies properly from the beginning for this first critical disease target in our neuro-degenerative disease program."

Dr. Bob Carter noted, "We are excited about the prospects of this model for collaboration involving a leading foundation, academic partners, and Exosome Diagnostics.  By leveraging the strengths of each arm of this triad, we will be able to more quickly bring tumor specific genetic information into the hands of practicing clinicians."

In January, ABC2 and Exosome Diagnostics hosted the first in a series of meetings with leading investigators to discuss the state of the brain cancer field, the prospective near and long-term clinical applications of exosome technology, performance requirements and barriers to clinical validation. Participating in the New York City meeting were senior principal investigators from leading academic institutions including the University of California, San Diego, Harvard Medical School and Massachusetts General Hospital, Memorial Sloan-Kettering Cancer Center, Johns Hopkins, Yale University, MD Anderson Cancer Center, Mt. Sinai Hospital, Henry Ford Hospital, University of Miami, University of Florida and Dana-Farber Cancer Institute.