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Brain tumor summit unites cancer specialists, investors to ‘innovate, collaborate and accelerate cures’
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by John Andrew Boockvar, MD, and Marc Symons, PhD
As many of you know, in most cases of brain cancer, treatment options largely are limited to surgery, radiation and chemotherapy.
We, along with our fellow Feinstein Institutes researchers, are working to discover new and better treatments for patients by designing and testing new drugs and drug delivery methods.
We also host the annual Brain Tumor Biotech Summit, which brings innovators in neuroscience and oncology together with investors whose generous funding makes it possible for us to advance the science.
Our sixth annual summit took place June 7 in New York.
Leaders in neurosurgery, neuroscience, oncology and industry professionals from across the world gathered to exchange ideas of promising new therapies in the treatment of brain tumors and other central nervous system diseases.
The coming together of investors and cancer specialists — as we look for new treatments for brain tumors and CNS diseases — is vitally important.
Those who attend the summit do so because they are committed to advocating for patients who suffer from these incurable diseases. They rally around the mission of joining together to innovate, collaborate and accelerate cures.
Over the course of the day, attendees heard from dozens of pre-eminent researchers who push the frontiers of neuroscience and oncology, and they heard from leaders in the investment community who have insights into the commercial aspects of medical research.
Topics included immunotherapy and precision medicine for the treatment of glioblastoma; autologous tissue transfers to bypass the blood-brain barrier for human glioblastoma; artificial intelligence in the neuroimaging of glioma; intraoperative radiotherapy for treatment of surgically resected brain metastases; and the use of 5 ALA imaging dye during brain tumor removal surgery to better distinguish cancer cells from healthy tissue.
Robert Maki, MD, PhD, of Northwell Health kicked off the day by giving an overview of early-phase clinical trial design.
Because the treatment of brain tumors is uniquely challenged by the presence of the blood-brain barrier — which strongly limits efficient delivery of drugs to these tumors — Maki emphasized the need for neoadjuvant trials that allow for assessment of drug penetration and/or target down-regulation after surgery.
As so many clinical trials for the treatment of brain tumors fail, it is critical to be able to rapidly determine which drugs or novel drug delivery methods stand a chance to make a therapeutic difference.
Glioblastoma treatment
One focus of the summit was immunotherapy for the treatment of glioblastoma.
Whereas immunotherapy — and, in particular, various T-cell checkpoint therapies — has had remarkable success over the past decade in cancers such as melanoma and lung cancer, results from clinical trials of immunotherapy for the treatment of glioblastoma have been very disappointing.
As was discussed in a number of presentations, the main reason for these failures lies in the strongly immunosuppressive nature of glioblastoma.
In his keynote address, William Curry, MD, of Massachusetts General Cancer Center, presented preclinical studies demonstrating that one way to overcome immunosuppression is to combine PD-1 checkpoint blockade with an OX-40 stimulating antibody and GVAX vaccination strategy.
Harry Gruber, MD, of Tocagen Inc. reported on another approach to overcome immunosuppression, using direct delivery of the retroviral replicating vector Toca 511 (Tocagen) to the tumor, resulting in local production of 5-FU that directly kills tumor cells and depletes immune-suppressive myeloid cells.
Steve Kalkanis, MD, of Henry Ford Health System led a subsequent panel of speakers focused on precision medicine techniques in glioblastoma. His research focuses on extracranial vesicles called exosomes that can be retrieved from a blood draw to help to determine the molecular fingerprint of a tumor and whether it is responding to treatment.
Boockvar and his team from Lenox Hill Hospital Brain Tumor Center and Donald and Barbara Zucker School of Medicine at Hofstra/Northwell presented early data on novel techniques to cross the blood-brain barrier, including impressive results from intra-arterial bevacizumab with blood-brain barrier disruption for newly diagnosed glioblastoma.
Boockvar also presented two clinical trials using autologous extra-cranial tissue flaps that are placed in the surgically resected glioblastoma cavity to provide vasculature that lacks an intact blood-brain barrier to the milieu and microenvironment of the tumor. This can aid in drug delivery and antigen exposure to the systemic immune system.
Other highlights
The summit also included a patient caregiver forum moderated by Sherese Fralin, MSN, of Lenox Hill Neurosurgery at Northwell Health.
Giving a voice to caregivers and patients at scientific meetings is an all too rare event, and this was highly educational for clinicians and researchers.
The theme of this forum was what providers can do better for patients and their loved ones.
Wrenching testimonies from caregivers pointed to the urgent need for the implementation of novel clinical trial design, such as adaptive clinical trials that require the enrolment of dramatically fewer control patients who do not receive experimental treatments in addition to a need for improving post-care access to information for patients and caregivers.
This was followed by a panel from our investors, industry, biotech and pharma colleagues.
These experts provided optimistic guidance on the state of venture capital funding for early-stage neuro-oncology intellectual property and companies. They emphasized the importance of working with local tech transfer offices early to help get a drug or idea rapidly into the clinic.
Another highlight of the meeting was the presentation by Christopher Filippi, MD, of Northwell Health, who discussed the use of deep-learning convolutional neural networks, an artificial intelligence approach that allows accurate detection of genetic mutations in gliomas solely based on MRI data.
He vowed that soon MRI will be only 5 minutes long and that your cell phone held over an image would be able to help see the molecular phenotype of each tumor.
Conclusion
The summit provided a unique opportunity for creativity between people who are not usually in the same room.
It provided a locale for spontaneous meetings, discussions and action plans between great minds.
When bench scientists, clinicians and investors collaborate, they create the potential to ignite the discovery and development of novel treatments.
Now is the time for the hard work of making these discussions realities. We hope to do this at the Feinstein Institutes Brain Tumor Biotech Center by making use of nanocells, the novel drug delivery modality developed by EnGeneIC — one of the presenting biotech companies — to deliver therapeutic microRNAs to glioblastoma tumors.
Curry and Filippi already have arranged a meeting at Massachusetts General to further advance artificial intelligence in neuroimaging for glioblastoma.
And, importantly, we have listened to our patients and caregivers and have begun the “brain tumor buddy” program to match patients and caregivers with a buddy to help them navigate through this challenging ordeal.
We look forward to continuing to advocate for our patients and families and push the envelope to provide better treatments for our patients with these devastating illnesses.
It is our hope that, in 5 years, we will develop and implement a number of methods to decrease immunosuppression and overcome tumor heterogeneity and the blood-brain barrier so that we can improve not only the therapies but the lives of our patients who face these devastating diseases.
For more information:
John Andrew Boockvar, MD, is vice chair of the department of neurosurgery at Lenox Hill Hospital; director of the Brain Tumor and Pituitary/ Neuroendocrine Center at Lenox Hill Hospital; and professor of neurosurgery and otolaryngology/head and neck surgery at Donald and Barbara Zucker School of Medicine at Hofstra/Northwell. He can be reached at jboockvar@northwell.edu.
Marc Symons, PhD, is professor at The Karches Center for Oncology Research at Feinstein Institutes for Medical Research; co-director of Brain Tumor Biotech Center at Feinstein Institutes for Medical Research; and professor of molecular medicine and neurosurgery at Donald and Barbara Zucker School of Medicine at Hofstra/Northwell. He can be reached at msymons@northwell.edu.
Disclosures: Boockvar and Symons report no relevant financial disclosures.