Cancer Types | Brain Cancer - National Foundation for Cancer Research

Brain Cancer

Brain Cancer

People of all ages are diagnosed with brain cancer, but there is more frequency among children and older adults. Brain cancer is the second most commonly diagnosed cancer in children (after leukemia).

Key Facts

  • Of the nearly 80,000 brain tumors diagnosed in the U.S. each year, approximately 32% are considered malignant – or cancerous.
  • An estimated 23,890 malignant tumors of the brain and spinal cord will be diagnosed in the U.S. in 2020, with around 18,020 deaths expected to result from the diagnosis.
  • Overall, the chance that a person will develop a malignant tumor of the brain or spinal cord in his or her lifetime is less than 1% (about 1 in 143 for men and 1 in 185 for women).
  • Survival rates vary widely depending on the type of tumor.
  • Glioblastoma (GBM) is the deadliest type of brain cancer, accounting for 48.3% of all malignant brain tumors and the five-year average survival rate is only 5% or less.
Source: American Cancer Society’s Cancer Facts & Figures 2020 and American Brain Tumor Association’s Brain Tumor Statistics
Silver Brain Cancer Ribbon
will be diagnosed in 2020
deaths expected in 2020
% lifetime risk of brain cancer

Brain Cancer Research

In addition to specific projects listed below, genomics research is helping us attack brain cancers – and all types of cancer. NFCR has distinguished itself from other organizations by emphasizing long-term, transformative research and working to move people toward cancer genomics.

Dr. Rakesh Jain
Dr. Rakesh Jain

Dr. Rakesh Jain a renowned expert in understanding the tumor’s abnormal microenvironment, previously discovered that an imbalance of vessel growth in tumors results in leaky blood vessels (edema), lack of oxygen and immunosuppression. His research is now determining why a new revolutionary immunotherapy is not effective in GBM. In models of GBM, his team discovered the abnormal vessels limit the immune system’s T cells to kill tumors.Treating GBM models with blood vessel growth inhibitors that he previously discovered, led to more normal tumor vessels and improved outcomes when combined with the immunotherapy. Dr. Jain’s research has high potential to improve treatment outcomes and increase survival in GBM patients.


Unlike other cancers, effective treatment options for GBM patients have improved little in recent decades. NFCR has been a pioneer and founding supporter of GBM AGILE clinical trial — a platform different from ‘traditional’ clinical trials in many ways: it can evaluate multiple therapies simultaneously; allow researchers to quickly identify drugs that are showing promising results; and seamlessly transition them to a confirmatory stage designed to support fast drug approval, saving patient’s precious time. NFCR is a strategic partner with the Global Coalition for Adaptive Research (GCAR), the official sponsor of GBM AGILE. In 2019, eleven GBM AGILE trial sites opened in the US and rapid expansion will be available at nearly 40 sites in the US and Canada. We are hopeful that GBM AGILE will serve as a model trial platform that can be applied to other cancers—giving patients hope for treatments that are best suited for their care.

Paul Fisher, M.Ph., Ph.D.
Paul Fisher, M.Ph., Ph.D.
Dr. Web Cavenee
Web Cavenee, Ph.D.

Dr. Paul Fisher discovered IL/24, an immune modulator gene that causes primary and metastatic tumor cells to commit ‘cell suicide’ but leaves healthy cells untouched. IL/24 has multiple anti-cancer effects including sensitizing tumor cells to radiation, chemotherapy and immunotherapy. Dr. Fisher is developing an IL/24 gene therapy that also includes a gene that fluoresces (lights up) when IL/24 locates and destroys tumor cells for a detection- and treatment-monitoring approach (known as theranostics). Another therapy combines IL/24 with a patient’s own immune T cells (adoptive cell therapy) to supercharge T cells to fight cancer even more effectively.

Dr. Fisher and Dr. Web Cavenee have focused lL/24 research for a new treatment for GBM. With support from the NFCR AIM-HI Translational Research Initiative, IL/24 gene therapy will advance soon to a Phase I clinical trial to provide GBM patients hope for a new effective treatment.

Dr. Fisher also discovered the gene, MDA-9/ Syntenin, that promotes the deadly spread (metastasis) of many cancers. Dr. Web Cavenee and he developed an innovative drug called PDZ1i to block the gene’s signals for metastasis. PDZ1i may be effective in treating numerous metastatic cancers. Radiation treatment can induce GBM cells to invade healthy tissue. PDZ1i treatment, in combination with radiation, results in profound survival benefits in pre-clinical models of GBM. With support from the NFCR AIM-HI Translational Research Initiative, the scientists are furthering their research on PDZ1i treatment towards clinical trials to benefit patients.

Cesare Spadoni, Ph.D.
Cesare Spadoni, Ph.D.

Brain and other cancers of the central nervous system account for 26% of childhood cancers with medulloblastoma as the most common type. Dr. Cesare Spadoni’s team is focused on developing a therapy with the ‘2Hit approach’ – a compound or combination of agents that attack two or more therapeutic targets in medulloblastoma cancer cells. The 2Hit approach aims to simultaneously increase effectiveness and reduce potential drug resistance. Ongoing research has identified 3 synergistic combinations that inhibit several targets. Next steps with bioinformatics (large sets of biological data) will identify a lead compound against two targets in medulloblastoma models. The team of scientists are hopeful a new treatment for this childhood cancer is on the horizon in the next two years.

W. K. Alfred Yung, M.D.
W. K. Alfred Yung, M.D.

NFCR-affiliated scientist Dr. W.K. Alfred Yung focuses his research on drugs that target a gene called PI3K, which is a key factor in about 30% of GBM cases. To identify potential targets for drug development, his team collected glioma stem cells (GSCs) from GBM patients and developed a special panel of cell lines to investigate patterns of resistance to P13K inhibitors. Results from the P13K studies have shown GSCs contain increased levels of Wee-1, a protein that controls cell division and growth. Combination of a P13K inhibitor and a Wee-1 inhibitor resulted in greater inhibition of cell growth and the cancer cells were induced into cell suicide. Similar benefits with this combination treatment were observed in complex GBM tumor models. These findings reveal molecular targets and designs for combination therapies that could lead to new treatments for GBM patients.

Related Content

DelMar Pharmaceuticals Receives $500,000 Loan from the National Brain Tumor Society and National Foundation for Cancer Research to Support VAL-083’s Participation in a Pivotal Study for Glioblastoma Sponsored by the Global Coalition for Adaptive Research

SAN DIEGO, June 24, 2020 /PRNewswire/ — DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) (“DelMar” or the “Company”), a biopharmaceutical company focused on the development of new solid tumor cancer therapies announced today it has received a $500,000 loan from the National Brain Tumor Society (NBTS) and the National Foundation for Cancer Research (NFCR) to support VAL-083’s preparation for participation in the Global Coalition for Adaptive Research’s (GCAR) sponsored trial, Glioblastoma (GBM) Adaptive Global Innovative Learning Environment (GBM AGILE) study. On June 4, 2020, the Company announced that VAL-083, its “first-in-class,” small-molecule chemotherapeutic with a novel mechanism of action, was selected by GCAR as the third investigational therapy to participate in GBM AGILE, in which the compounds will be simultaneously evaluated across multiple international trial sites of which 25 are currently activated. DelMar intends to utilize GBM AGILE, which is an adaptive registration clinical trial, to serve as the basis for VAL-083’s new drug application submission and registration. “It means a great deal to all of us involved with VAL-083’s development to receive support from the National Brain Tumor Society and the National Foundation for Cancer Research as these organizations are two of the leading advocacy and funding partners for GBM AGILE,” commented Saiid Zarrabian, Chief Executive Officer of DelMar Pharmaceuticals. “This funding is an important milestone as it enables us to accelerate VAL-083’s participation in GBM AGILE, which is expected to reduce VAL-083’s pivotal trial completion and regulatory submission timeline by up to 18 months.” GBM AGILE is an international effort in newly diagnosed and recurrent GBM, utilizing an FDA approved master protocol to evaluate multiple therapies against a common control arm. As an approved registrational study, positive results from the VAL-083 arm of GBM AGILE are expected to be utilized to file for FDA approval. This study employs a cost-efficient, seamless phase 2/3 adaptive trial design with a Stage 1 learning and adapting phase and a Stage 2 expansion and confirmation phase. The effort is led by top-tier key opinion leaders in the GBM field and has the collective support of an international group of more than 130 clinicians, researchers, biostatisticians, imagers, pathologists, leaders from government and industry, and patient advocates. GCAR, a 501(c)(3) organization, is the international trial sponsor, and provides financial support for the program infrastructure, as well as trial oversight and management.  Comprised of the world’s foremost clinical, translational, and basic science investigators, GCAR strives to support the development of novel treatments to fight against rare and deadly diseases like GBM where patient prognosis is poor and treatment options are limited. “We are supporting the inclusion of VAL-083 in GBM AGILE adaptive clinical trial platform as it is consistent with our mission to support research for, and ultimately enable delivery of, effective treatments to patients with brain tumors. We are particularly pleased to lend our support to VAL-083 given the significant unmet medical need that exists for patients with GBM,” commented David Arons, Chief Executive Officer of the National Brain Tumor Society. Sujuan Ba, President & Chief Executive Officer of the National Foundation for Cancer Research added, “We are dedicated to facilitating the development of therapies for all cancers, and are pleased […]

Scorpion Venom: The Newest Treatment for a Deadly Cancer

GBM AGILE – Changing the Way We Fight Brain Cancer

GBM survival rates have not improved in any meaningful way in over 30 years despite desperate efforts. Glioblastoma Multiforme, referred to simply as GBM, is the deadliest type of brain cancer. GBM is widely regarded as incurable and universally fatal, killing 95% of patients within five years of diagnosis. It is a fast-acting cancer that attacks cells meant to support the health of nerve cells within the brain. GBM survival rates have not improved in any meaningful way in over 30 years despite desperate efforts. For years, researchers tried and failed to even come close to finding a potential cure for this disease. Each clinical trial required participation from GBM patients for the better part of a decade. Unfortunately, for a fast-acting and rare disease like GBM, the time required for a successful clinical trial is rarely feasible. Years of hard work and hundreds of millions of dollars were being flushed down the drain, leaving researchers and patients frustrated and seemingly hopeless. Fortunately, the Global Coalition for Adaptive Research (GCAR) took a major leap and decided to challenge the standard clinical trial by introducing GBM AGILE. GCAR is a nonprofit organization comprised of some of the world’s foremost clinical, translational, and basic science investigators, including the National Foundation for Cancer Research. Together, this international group developed GBM Adaptive Global Innovative Learning Environment, or GBM AGILE. GBM AGILE is the world’s first global adaptive clinical trial platform for GBM. It changes the model of traditional clinical trials by evaluating multiple therapies simultaneously, ultimately creating a flexible and adaptable trial approach. This new approach allows researchers to identify drugs that are showing promising results and seamlessly transition to a confirmatory stage designed to support drug approval. Similarly, researchers are able to pinpoint and cull the use of underperforming drugs with minimal time and resources being wasted. Simply put, GBM AGILE is patient-centric and provides a streamlined method for researchers to utilize data connectivity within the trial to answer many questions concurrently. “GBM AGILE allows us to get more researchers involved and allows there to be enough patients enrolled in trials to get statistically significant results,” stated GBM patient Dwayne Osgood. “This sharing of data and information could allow the research to move faster, and ultimately bring lifesaving and life extending treatments to patients faster.” GBM AGILE was the first of its kind, paving the way for further adaptive trials for other cancers. While such trials are not yet commonplace, Congress and the U.S. Food and Drug Administration are focused on accelerating their development to benefit patients. It is anticipated that systems like GBM AGILE will replace the current clinical trial model and become the standard for all cancer trials. While the GBM AGILE team is dedicated to improving outcomes and treatment options for GBM patients, they aim to apply the trial design to other rare and deadly diseases. GBM patients interested in participating in GBM AGILE should explore the current trial sites and contact the research team to learn more. As a founding member of GBM AGILE’s sponsoring body, NFCR has continued to take a leading role in this unprecedented global effort—Dr Sujuan […]