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GBM- AGILE

Meet Carmen Rice: A Courageous and Inspirational GBM Survivor

This year marks the 13th anniversary of Carmen Rice’s survival from Glioblastoma Multiforme (GBM) – the deadliest brain cancer that is widely regarded as incurable and universally fatal. GBM kills 91% of patients within three years of diagnosis and NFCR is part of a robust, international coalition working on innovative ways to research the disease. In the cancer research world, too often stories do not have optimistic endings, which is why our work continues until we find cures. Yet Carmen’s thirteen-year survival is nothing less than miraculous and we are truly honored to share her incredible story with you.

Carmen’s Story

In 2004, Carmen Rice began experiencing severe headaches, nausea and dizziness.  She remembers having lunch in a local restaurant and a moment later waking up in the hospital bed not knowing what happened or how she got there. She had a grand mal seizure.  Scans revealed a small brain tumor.  Days later, Carmen underwent brain surgery to remove the tumor.

The tumor removed proved to be GBM, a very aggressive, malignant tumor. Carmen was told she had only six months to live. Terrified, yet determined to beat this, she began an aggressive treatment regimen of radiation and chemotherapy.

Carmen remained in remission until 2008, when during a routine check-up, a new growth appeared in the same site as the original tumor. Since GBMs tend to return to the same site as the original tumor, Carmen and her doctors assumed this was a recurrence. In December 2008, Carmen underwent her second brain surgery. She spent Christmas and New Year’s in the hospital praying that she would once again recover. Her prayers were answered when the final pathology reported that this new growth was not malignant.
“There are amazing new discoveries being made each day. Thank you NFCR, donors, advocates, doctors and scientists for saving the lives of so many people like me. You give me hope and inspiration to move forward.” –Carmen Rice

(Left to Right) NFCR CEO Franklin C. Salisbury, Jr., NFCR President Dr. Sujuan Ba, Carmen Rice and Darrell Rice.

Fast forward to today, Carmen continues to live a happy, healthy and active life. She enjoys every moment and is an enthusiastic speaker within the cancer community, inspiring others with her positive outlook and message of hope. Carmen talks about how grateful she is to NFCR for the groundbreaking research done in the GBM field.

In her latest speech about her survival story, Carmen passionately discussed NFCR and her support of our work – specifically GBM AGILE.  Carmen is confident – as are we – that together we will find a cure for GBM.

Today’s Research Will Lead to Tomorrow’s Cures

GBM AGILE

Led by the best and brightest cancer researchers, GBM AGILE is a revolutionary global collaboration to test and develop new brain cancer treatments.  Its personalized approach will allow us to accelerate the discovery of targeted treatments for individual patients.

This global coalition has attracted over 150 participants from more than 40 leading cancer institutions across three continents. It implements a new generation of clinical trials – called “adaptive trials” – which allow patients to be enrolled more quickly, receive treatment with multiple anti-cancer drugs simultaneously and does not require years of follow-up to determine whether a new experimental treatment is beneficial. This revolutionary approach accelerates research for curing the aggressive form of cancer GBM and will serve as a new clinical research model for combating other cancers as well. As a founding member of the coalition, NFCR has continued to take a leading role in this unprecedented effort. It is anticipated that patient enrollment may start in the fall of 2017. Stayed tuned!

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4 Must-Know Facts About the Deadliest Brain Tumor

Glioblastoma multiforme- also known as GBM– is the most lethal form of brain cancer in adults. This devastating brain cancer spreads into other parts of the brain very quickly and is usually not surgically curable. Typically, radiation and chemotherapy are given with the hopes of delaying tumor progression. If you or a loved one is diagnosed with GBM, here are 4 important facts you need to know:

Brain Tumor Statistics

  • GBM kills 95% of patients within five years of diagnosis, with more than half dying within the first 15 months after diagnosis.
  • Although GBM can occur in any age group, it is most common in adults between the ages of 45-70.[i]
  • This year, more than 12,000 people living in the U.S. and tens of thousands more worldwide will receive a diagnosis of GBM. [ii]

1. Connect with a doctor(s) who specializes in GBM.

As with any rare or aggressive cancer, it’s best to seek care from a doctor who spends all of his or her time treating that particular disease. When it comes to GBM, there are many nuances to both its diagnosis and treatment that require expert knowledge.  Second opinions are also extremely important as they will either confirm what you’ve already been told or present different options to weigh.

2. Know the facts.

GBMs are extremely complex tumors.  Complete surgical removal of the entire tumor is extremely difficult because these tumors have finger-like tentacles that extend into surrounding normal brain tissue.  GBMs are also made up of a wide mix of different cell types that often differ from patient to patient.  Additionally, when GBMs recur or grow back—and they almost always do–the molecular profile of the tumor changes dramatically.  As a result, response to treatment varies considerably and today’s five-year survival for GBM is less than 5%.

3. With GBM AGILE, the future looks promising.

Currently there is no cure for GBM.  Although there have been hundreds of standard clinical trials for GBM, survival rates for patients with GBM have not improved in any meaningful way in more than 30 years… GBM AGILE brings tremendous hope to patients battling GBM today.

What is GBM AGILE?

Over 150 researchers from more than 40 leading cancer institutions across four continents have come together to find cures for GBM by launching the first-ever global, adaptive clinical trial that will revolutionize how brain cancer treatments are tested and developed. This new type of clinical trial platform is called GBM Adaptive Global Innovative Learning Environment, or GBM AGILE for short. By offering the ability to access to the newest, most promising treatments quickly and in real-time, GBM AGILE will provide people living with brain cancer new hope where little currently exists. While adaptive trials are not yet commonplace, Congress and the FDA are focused on accelerating their development to benefit patients, including GBM AGILE.

4. NFCR is attacking GBM head on.

With GBM AGILE, medical research is being transformed right now by the biggest global collaboration in the history of brain cancer research. NFCR has provided seed funding for this revolution and as part of the executive steering committee, is helping to develop and manage this groundbreaking adaptive trial protocol.

The GBM AGILE team is currently in discussion with the USA’s FDA (Food and Drug Administration) to create a process that will make it faster for patients to access the promising drugs that are being tested in this clinical trial. This will be done by allowing seamless transition from Phase II to Phase III of the trial which does not traditionally happen in the current clinical trial format. The protocol is nearly complete, and patient enrollment is expected to begin in January, 2018 giving patients worldwide the best chance to treat their disease.

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7 Facts You Need to Know About Brain Tumors

What is Brain Tumor?

  • Of the nearly 80,000 brain tumors diagnosed in the U.S. each year, approximately 32% are considered malignant – or cancerous.
  • 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%.
  • Glioblastoma multiforme (GBM) is the deadliest type of brain cancer, accounting for 45% of all malignant brain tumors.

Here’s a list of seven facts you need to know about brain tumors. And make sure you read about the work NFCR is doing to combat the deadliest brain cancer with GBM AGILE.

1. Primary brain cancer is rare.

primary malignant brain tumor is a rare type of cancer accounting for only about 1.4% of all new cancer cases in the U.S.  The most common brain tumors are known as secondary tumors, meaning they have metastasized, or spread, to the brain from other parts of the body such as the lungs, breasts, colon or prostate.

2. The cause of brain cancer is usually unknown.

Most people diagnosed with a primary brain tumor do not have any known risk factors. However, certain risk factors and genetic conditions have been shown to increase a person’s chances of developing one, including:

  • The risk of a brain tumor increases as you age.
  • People who have been exposed to ionizing radiation—such as radiation therapy used to treat cancer and radiation exposure caused by atomic bombs– have an increased risk of brain tumor.[i]
  • Rare genetic disorders like Von Hippel-Lindau disease, Li-Fraumeni syndrome, and Neurofibromatosis (NF1 and NF2) may raise the risk of developing certain types of brain tumors. Otherwise, there is little evidence that brain cancer runs in families.[ii]

3. Typically brain tumors don’t have obvious symptoms.

Headaches that get worse over time are a symptom of many ailments including brain tumors. Other symptoms may include personality changes, eye weakness, nausea or vomiting, difficulty speaking or comprehending and short-term memory loss.

Even benign or non-cancerous tumors can be serious and life threatening. If you experience these symptoms, speak with your doctor right away. 

4. Brain tumors can occur at any age.

Primary brain tumors—those that begin in the brain—can develop at any age, but they are most common in children and older adults. While brain tumors are one of the most common cancers occurring in children 0-14 years, the average age of diagnosis is 59 years. [iii]

5. Cell phones aren’t proven to cause brain cancer.

Although long-term studies are ongoing, to date there is no definitive evidence that cell phone use increases the risk of cancer. However, if you are concerned about the possible link between cellphones and cancer, consider limiting your use of cellphones — or use a speaker or hands-free device.

6. Survival rates vary.

There are different types of primary brain cancer and survival rates vary significantly depending on the type of cancer. Some types of brain cancer, such as meningioma, anaplastic ependymoma and oligodendroglioma, are highly treatable, while others are less responsive to treatment.

7. With GBM AGILE, the future looks promising.

Glioblastoma multiforme (also known as GBM) is the deadliest of all (primary) brain cancers and is widely regarded as incurable and universally fatal, killing 95% of patients within five years of diagnosis.

To combat this deadly disease, NFCR is part of a robust, international coalition working on innovative ways to defeat GBM utilizing a rigorous adaptive trial platform known as GBM AGILE (Adaptive Global Innovative Learning Environment).  Led by some of the best and brightest brain cancer researchers in the world, GBM AGILE is re-engineering the way clinical trials are conducted to develop more effective treatments faster than ever before.

To learn more about adaptive clinical trials and GBM AGILE, click here.

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 is a leader in the field of tumor biology – specifically in anti-angiogenic therapy, which looks at thwarting certain types of blood vessel formation. Dr. Jain has been studying the role angiogenesis plays in glioblastoma multiforme (GBM), the deadliest form of brain cancer. Dr. Jain’s research is helping doctors better tailor the use of anti-angiogenic therapies by identifying the characteristics that cause resistance for GBM patients. Dr. Jain and his team have identified molecular resistance pathways that may direct the development of new drugs that target these pathways and could extend the benefits of anti-angiogenic therapies for patients.
Because GBM invades healthy tissues near the tumor, Dr. Jain’s current NFCR-funded research is focused on testing inhibitors that could prevent invasion. Vessel co-option is a process by which cancer cells migrate through and around nearby healthy tissue. Dr. Jain is identifying genes and pathways that facilitate vessel co-option in order to prevent invasion and improve GBM therapies.

The Director of NFCR’s Scientific Advisory Board, Dr. Web Cavenee, has partnered with NFCR-funded scientist Dr. Paul B. Fisher to discover a new pharmacological agent that could – with additional chemistry – lead to a new drug to prevent radiation-induced invasion of GBM cells. The researchers have tested their pharmacological agent in combination with radiation and have seen profound survival benefits in pre-clinical models.

Previously NFCR-funded scientist Dr. W.K. Alfred Yung’s research focused on drugs that target a gene called PI3K, which is a key factor in about 30% of GBM cases. 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. The researchers are figuring out the molecular profile of these GSCs to identify potential targets for drug development. Results from the P13K studies have shown that the molecular profile of GSCs contain increased levels of Wee-1, which is a protein that controls cell division and growth. Following these results, the team then combined a P13K inhibitor with a Wee-1 inhibitor and found there was a greater inhibition of cell growth and the cancer cells were induced into cell suicide. Plus, when they tested the same inhibitors on complex GBM tumor models, they discovered similar benefits. These findings reveal molecular targets and designs for combination therapies that could lead to new treatments for GBM patients.

 

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[1] http://www.mayoclinic.org/diseases-conditions/brain-tumor/symptoms-causes/dxc-20117134

[2] http://www.cancercenter.com/brain-cancer/risk-factors/

[3] http://www.abta.org/about-us/news/brain-tumor-statistics/?referrer=https://www.bing.com/

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Understanding Adaptive Clinical Trials

Testing new treatments is key to successfully treating cancer. Unfortunately, the current model for conducting clinical trials — evaluating the effectiveness of therapies on human subjects — is inefficient, expensive and suffers from high failure rates. To overcome these challenges, the practice of adaptive trials is emerging and has become one of the most promising advancements in cancer research today.

Challenges with traditional clinical trials

Currently, it takes approximately $2.5 billion dollars, 10+ years and thousands of patient volunteers to bring one drug to market.[i] Despite this high cost, over half of phase III oncology trials have negative results or fail to complete. In addition, traditional trials cannot be modified once they begin. Patients get only one opportunity and receive only one treatment.

Adaptive trials literally adapt as evidence accumulates

Adaptive trials do not require years of follow-up to determine whether a new experimental treatment is beneficial. They test multiple treatments and combinations of treatments on patients in parallel and are designed to be continuously updated as researchers use what they learn from each patient to make real-time adjustments as the trial proceeds.

They are flexible by design so adjustments can be made in terms of dosage, subject population or sample size as needed.

In an adaptive trial, if one treatment regimen seems to be more successful, researchers can increase the number of participants receiving that treatment. This is especially important for patients with aggressive cancers as life expectancies are too short for traditional trial timetables. With adaptive trials, ineffective treatments can be shut down early, and new treatments can be initiated quickly enabling patients to receive the most promising treatments faster.

In a latest article published on Science Magazine: “Congress and FDA nominee heap love on ‘adaptive trials”, GBM AGILE is featured as one of the ongoing clinical trials using this new adaptive trial design.

GBM AGILE revolutionizes brain cancer trials

Glioblastoma multiforme (also known as GBM) is the deadliest brain cancer and is widely regarded as incurable and universally fatal, killing 95% of patients within five years of diagnosis. To combat this deadly disease, NFCR is part of a robust, international coalition working on innovative ways to defeat GBM utilizing adaptive trials known as GBM AGILE.

Led by the best and brightest cancer researchers, GBM AGILE is a revolutionary global collaboration to test and develop new brain cancer treatments. Its personalized approach will allow us to accelerate the discovery of targeted treatments for individual patients. This global coalition has attracted over 150 participants from more than 40 leading cancer institutions across three continents. This revolutionary approach accelerates research for curing the aggressive form of brain cancer GBM and will serve as a new clinical research model for combating other cancers as well.

In addition to GBM AGILE, there are other adaptive trials underway for certain cancers and tumor types, with more coming soon.  Other trials include:

  • I-SPY TRIAL is an adaptive clinical trial for women with newly diagnosed, locally advanced breast cancer.
  • Lung-MAP is a large adaptive clinical trial testing several new treatments for patients who have advanced stage squamous cell lung cancer.
  • Precision Promise is the first large-scale precision medicine adaptive trial for patients with pancreatic cancer.

While adaptive trial features are not yet commonplace, Congress and the FDA are focused on accelerating their development to benefit patients.

[i] https://www.scientificamerican.com/article/cost-to-develop-new-pharmaceutical-drug-now-exceeds-2-5b/

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NFCR President Wins International Cooperation Award from the Chinese Medical Doctor Association

NFCR President receives the inaugural award from Society for Neuro-Oncology of the Chinese Medical Doctor Association for her outstanding contribution to international cooperation in March 2017.

From left to right: Wenbin, Li, Tao Jiang, Sujuan Ba, Webster Cavenee

 

Dr. Jiang Tao, the Chairman of the Society for Neuro-Oncology of the Chinese Medical Doctor Association and the vice director of the department of neurosurgery at Beijing Tiantan Hospital presented Dr. Ba with the award.

Dr. Wenbin, Li, the general secretary of the Chinese Medical Doctor Association and the director of glioma program at Beijing Shijitan Hospital, provided remarks at the event. He said, “Congratulations to NFCR President and COO Dr. Sujuan Ba for receiving the inaugural Award for Outstanding Contributions of International Cooperation from the Chinese Medical Doctor Association. Dr. Ba’s outstanding work has helped to build trust and alliances between the U. S. China, and other countries through diversified platform, ensuring that each country’s unique advantages are brought together to enable highly effective and productive international collaborations.”

The award recognizes Dr. Ba’s efforts leading and facilitating numerous high-impact international collaborative projects over the past two decades, including building a high-quality cancer tissue biorepository in Asia and a recent cross-continent adaptive clinical trial platform, GBM AGILE (which stands for Glioblastoma Multiforme, Adaptive Global Innovative Learning Environment).

Dr. Li also explained how “Dr. Ba has led the creation of the Tissue Bank Consortium in Asia and the Joint Tissue Bank Facility at Tianjin Cancer Hospital and Institute to promote best practices for biorepositories and biobanks and for building international collaboration platforms for tissue-based cancer research. The project has been proclaimed an exemplary international platform for private, public and government partnership for international collaboration in the fields of cancer biomarkers and therapeutic development.”

Dr. Ba currently serves on the executive committee for GBM AGILE and is co-chair of the Patient Focused Research and Advocacy Committee. GBM AGILE – a revolutionary clinical trial design – provides a learning environment that proactively generates new knowledge, identifies promising therapies and quickly moves them to advanced clinical trial phases.

Dr. Li finished his remarks by saying, “Dr. Ba has been instrumental in establishing, expanding and escalating the scale and scope of these and other highly influential international collaborative work. These projects have and will continue to make substantial impact in accelerating translational and clinical cancer research, bringing better prevention methods, improved diagnostic tools and more effective treatment strategies to cancer patients around the world.”

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NFCR’s Dr. Web Cavenee Honored at Prestigious Gray Gala

On Friday, November 11, 2016, the National Brain Tumor Society (NBTS) held its annual Gray Gala to recognize and celebrate the achievements of change-makers across the brain tumor community.  Pioneers in brain tumor research were honored, including Dr. Web Cavenee, Chairman of NFCR’s Scientific Advisory Board and former NFCR-funded research fellow.  Dr. Cavenee was awarded the 2016 Feldman Founder’s Award for Adult Brain Tumor Research.

David Arons, CEO NBTS; Dr. Web Cavanee, Ludwig Cancer Research and Chairman of NFCR Scientific Advisory Board; G. Bonnie Feldman, Founder of NBTS; Dr. David Louis, Pathology Chair Mass General Hospital)
Pictured above from left to right: David Arons, CEO NBTS; Dr. Web Cavanee, Ludwig Cancer Research and Chairman of NFCR Scientific Advisory Board; G. Bonnie Feldman, Founder of NBTS; Dr. David Louis, Pathology Chair Mass General Hospital)

“It was an honor to witness the celebration of the scientists, advocates and volunteers all joined together to support the brain tumor community,” said Dr. Sujuan Ba, President and COO of NFCR.

Dr. Sujuan Ba spoke to fellow leaders at the Gray Gala about this collaboration and stressed, “NFCR is proud to partner with the National Brain Tumor Society to fight GBM, one of the most deadly cancers.” Dr. Sujuan Ba is particularly enthusiastic about an innovative undertaking both the NFCR and NBTS are supporting: GBM AGILE.  Led by the best and brightest cancer researchers, GBM AGILE is a revolutionary global collaborative program to test and develop new brain cancer treatments. Its adaptive and personalized approach will cut several years of the clinical testing and reveal potentially lifesaving treatments far faster than has ever been possible. Additionally, the learning from GBM can be used for other cancers using similar approaches to save more lives.

Pictured: G. Bonnie Feldman, Founder of NBTS (left) and Dr. Sujuan Ba
Pictured: G. Bonnie Feldman, Founder of NBTS (left) and Dr. Sujuan Ba
Pictured: David Arons, CEO of NBTS (left) and Dr. Sujuan Ba
Pictured: David Arons, CEO of NBTS (left) and Dr. Sujuan Ba
Dr. Sujuan Ba with Key leaders of GBM AGILE pictured from left to right:
Dr. Alfred Yung (MD Anderson Cancer Center and NFCR Fellow);
Dr. Brian Alexander (Dana-Farber Cancer Institute);
Dr. Web Cavanee (Ludwig Cancer Research and Chairman of NFCR Scientific Advisory Board)
Dr. Sujuan Ba with Key leaders of GBM AGILE pictured from left to right: Dr. Alfred Yung (MD Anderson Cancer Center and NFCR Fellow); Dr. Brian Alexander (Dana-Farber Cancer Institute); Dr. Web Cavanee (Ludwig Cancer Research and Chairman of NFCR Scientific Advisory Board)
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Women in Science – Dr. Sujuan Ba – CRS Luncheon 2011

NFCR’s  Dr. Sujuan Ba Energizes Women in Science at CRS Annual Luncheon

sujuanbaAugust 2011 –Sujuan Ba, Ph.D., Chief Operating Officer of the National Foundation for Cancer Research, was the featured speaker at the  Controlled Release Society (CRS) Women in Science Forum Luncheon.  Dr. Ba described her tenacious climb through the pharmaceutical and scientific fields, arriving at her current position where she oversees NFCR’s organizational strategic and tactical operations, fundraising and partnership development, and financial planning and management. Dr. Ba’s talk, entitled “Forging a Path on the Road Less Traveled By a Scientist,” included segments on her establishment of a powerful network of research centers and laboratories in the United States, Europe, and Asia – a network which has enabled NFCR Scientists to focus on critical aspects of cancer research.

Dr. Ba was selected by CRS for her innovative and collaborative approach to merging scientific thought with an entrepreneurial spirit.  She encouraged the attendees to never stop learning and to continue to look for the next opportunity for growth, while outlining the key skills necessary for women to succeed in science.  The talk was part of the 38th Annual Meeting & Exposition of the Controlled Release Society, held on July 30 – August 3, 2011 at the Gaylord National Hotel and Convention Center, National Harbor, Maryland.  The event was sponsored by Roche.

The Controlled Release Society (CRS) is the premier society worldwide for delivery science and technology. More information on Dr. Ba can be found on their website.  http://www.controlledreleasesociety.org/meetings/Archives/2016AnnualMeeting/program/Pages/WomeninScience.aspx

2016 Update: Dr. Sujuan Ba continues to forge paths on the road less traveled by a scientist.
She has since been promoted to President and Chief Operating Officer at NFCR. As a woman in the executive suite as well as an entrepreneur, Dr. Ba continuously innovates.  She continues to work collaboratively across cultures and countries in a tireless effort to disrupt cancer’s deadly path. When President Barack Obama announced a new cancer moonshot during his State of the Union Address January 12,2016, NFCR was already well positioned to be available to help on the international cancer research platform due to the GBM-AGILE initiative and her already established science connections in China.

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