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Recently in Brain Tumor Research

Published on December 19, 2013 in Research, Our Impact

…A round-up of recent news from across the brain tumor research field

NBTS-grantee Tocagen Gives Update on Experimental Brain Tumor Treatment

In 2010, the National Brain Tumor Society awarded one of our Innovation Grants to the San Diego-based biopharmaceutical company, Tocagen.

This was the first time that we had directly funded a biopharmaceutical company, and chose to do so after the company had shown exciting results from pre-clinical studies of a treatment they had originally licensed from researchers at UCLA and USC.  It was our hope that by providing some extra funding to their work, Tocagen could accelerate their R&D efforts on this treatment and hopefully provide brain tumor patients with a much-needed new weapon in the ongoing battle to eradicate this disease.

Just last month, Tocagen presented findings from two Phase 1 studies (60 patients enrolled across two clinical trials) of their Toca 511 at the Society for Neuro-Oncology’s (SNO) annual meeting. Early results from these studies in patients with recurrent GBM demonstrate that the treatment is safe and well tolerated, and the preliminary survival data are encouraging, as well.
> Get information on participating in Tocagen’s clinical trials

We spoke with Tocagen officials at SNO, as well as this past week, and are happy to hear their continued progress. We’re told the company will spend the next several months completing their Phase 1 studies, and then look to move into later stage trials.

“I am pleased with the progress we have made with our clinical programs, and the available survival data provide optimism for subsequent dose groups in these studies,” said Harry Gruber, M.D., CEO of Tocagen. “The results from our ongoing clinical studies, taken together with the depth of our preclinical research, provide momentum for the continued investigation of this novel gene therapy for patients with brain cancer.”
> Learn more about how Toca 511 works (San Diego Union-Tribune article)

Agenus Data Makes Waves

Earlier this week, Agenus, a Massachusetts-based biotech company, announced data from their Phase 2 single-arm trial of Prophage G-200, a vaccine treatment also for recurrent GBM. The data was published in Neuro-Oncology, the official journal of SNO.

The announcement was making waves across the community because of the positive data, but like the Tocagen news, it is still in its early stages of the testing required for potential approval from the U.S. Food and Drug Administration (FDA). However, the company’s announcement that it is already moving into a larger, NCI-sponsored trial is encouraging, nonetheless.

Dr. Andrew T. Para, MD, PhD, formerly of UCSF, and now the Chair at Northwestern Neurosurgery – the study’s lead clinical investigator – was the Keynote Speaker at the National Brain Tumor Society 2010 Summit, and we were also part of the original funding collaboration for the project (though not this trial).
> Learn more

NBTS Innovation Grant Supports New Model for Pediatric Research

National Brain Tumor Society’s most recent Innovation Grant winner, Dr. David H. Gutmann (Washington University School of Medicine) and his team, have generated a first-of-its-kind mouse model for pediatric low-grade gliomas (LGG). To date, existing models of pediatric LGG have unfortunately been unreliable in predicting how potential drug interaction will translate into actual humans, and even lack inclusion of the signature genetic alteration identified in pediatric pilocytic astrocytoma, a type of LGG.

The new transgenic mouse model contains this characteristic alteration – a specific BRAF fusion that represents the most common abnormality found in pilocytic astrocytoma. Thus, this new model provides researchers with a much-needed experimental platform to study the role of the BRAF fusion in developmental neurobiology and pediatric neuro-oncology.

“The availability of this novel mouse strain in which the signature genetic mutation can be expressed in specific cell types and at specific times offers the potential to generate small-animal models of pediatric low-grade glioma for future drug discovery and evaluation,” said Dr. Gutmann.

This new mouse model was published in the October 2013 Issue of Genesis, The Journal of Genetics and Development. Dr. Gutmann’s new model is already helping other researchers focusing on pediatric LGG, including Dr. Stefan Pfister of the German Cancer Research Center (DKFZ).

“David’s model may indeed be considered a breakthrough in the LGG field,” said Dr. Pfister. “We are very grateful for his collaboration.”

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