Barrow Neurological Clinical Trial:
In early February 2017: Dr. Joseph Zabramski’s team at Barrow Neurological Institute was the recipient of a $75,000 grant from the Be Brave for Life Foundation in support of their continued research into a new treatment for vascular brain tumors, or CCMs (Cerebral Cavernous Malformations). They recently completed necessary preparation and design for a clinical research protocol at Barrow that promises to provide insight into the mechanism of propranolol’s effect on CCMs. Patients who are scheduled for surgical resection for symptomatic brain stem CCMs will be offered enrollment in a clinical protocol. If the patient chooses to participate he/she will be divided into one of two groups; the treatment group will receive propranolol 20mg orally three times per day, and the control group will receive placebo three times per day. Samples of the surgically resected CCMs from these two groups will be collected and will be subjected to global messenger RNA and micro RNA (see Appendix B) expression profiling to identify regulatory networks involved in effects produced by propranolol therapy for CCMs. The initial goal will be to enroll 20 patients (10 in the treatment group and 10 in the placebo group). The research team expects to complete enrollment by summer of 2017. Knowledge regarding the potential mechanisms of propranolol’s effect on CCMs is essential to obtaining government funding for a Phase III clinical trial.
Be Brave for Life’s financial support will provide for the administrative and medical staff required—including but not limited to a clinical nurse research coordinator—to accurately collect and monitor results of the proposed research protocol. The costs of genomic studies will also be offset with the support from Be Brave for Life. Our support will enable Dr. Zabramski and his team to take this important step in discovering new treatment options for people living with CCMs.
Past Funds Granted in March 2016: Be Brave for Life donated $40,000 to Dr. Joseph Zabramski’s lab at Barrow Neurological Institute, BNI, in Phoenix, Arizona. Dr. Zabramski is one of BNI’s leading neurosurgeons and researchers. When Be Brave for Life reached out to Riley’s neurosurgeon, Dr. Robert Spetzler, he immediately directed us to Dr. Zabramski. He and his team are conducting a Phase II clinical trial to establish the feasibility and safety of using an oral medication, propranolol, as an alternative approach to the treatment of Cerebral Cavernous Malformations (CCMs) or vascular tumors in adults and children. If the trial hypothesis is confirmed, this study will pave the way for a Phase III trial – one step closer to delivering this medication to patients with CCMs, people like Riley.
The advancements of 2016: It has been an exciting year for Dr. Zabramski and his research partners, Barrow’s Robert Spetzler, MD, Translational Genomics Research Institute’s Matthew Huentelman, PhD, and University of Utah’s Yashar Kalani MD, PhD. A number of clinical trials have demonstrated that propranolol (a low-cost and FDA-approved medication with a long history of widespread clinical use) can significantly shrink cutaneous hemangiomas in infants, and that the drug is extremely safe in this population. Because of the similarities between cutaneous hemangiomas and CCMs we proposed that propranolol may be an ideal candidate for treating both types of lesions.
Based on initial positive research results using oral propranolol published in the April 2016 issue of World Neurosurgery, and with financial support of Be Brave For Life, Dr. Zabramski and his team have continued investigating the use of oral propranolol in a small number of patients at Barrow who are unwilling or unable to consider surgical intervention. Recently, the team presented a proposal for a Phase III clinical trial of oral propranolol at the 12th Annual Scientific Meeting of the Angioma Alliance on November 10th, 2016 in Washington, D.C. The presentation entitled, “A Proposed Trial of Oral Propranolol for Symptomatic Cerebral Cavernous Malformations not Amenable to Surgical Resection,” was well received and there is international interest in this clinical trial. Following the meeting, Drs. Zabramski and Kalani met with the program director of the National Institute of Neurological Disorders and Stroke and National Institutes of Health, Jim Koenig PhD, to discuss government funding for this study. The team is also working with several groups in Italy to submit a joint funding request to the National Institutes of Health later this year.
Boston Children’s Hospital National Trial:
On February 23, 2017: Dr. Edward Smith and his research team were the recipients of a $64,000 grant by the Be Brave for Life Foundation. Over the past few years, he and his team have identified proteins in urine that signal the presence of multiple conditions, including unique diagnostic “signatures” for vascular disease and brain tumors. Moreover, Dr. Smith’s focus has expanded recently to translating his biomarker discoveries into novel therapeutics to treat these conditions. Be Brave for Life’s financial support is helping to further his research in this area! The first neurosurgical urinary biomarker clinical trial in the US and the first in which a neurosurgeon – Dr. Smith – is the principal investigator continues to enroll patients. Dr. Smith is examining the accuracy of urinary biomarkers in identifying pediatric brain tumors and predicting disease progression.
Our support has fueled novel research. Moreover, it has helped Dr. Smith and his team leverage their discoveries to attract independent funding. The National Cancer Institute and Pediatric Brain Tumor Consortium continue to fund the multi-center clinical trial, and the American Brain Tumor Association and the foundation Voices Against Brain Cancer are critical sources of support for the urinary biomarker studies. Their goal is to continue to garner such independent funding made possible by our grant.
Past Funds Granted In March 2016: Be Brave for Life donated $40,000 to Dr. Edward Smith at Boston Children’s Hospital. Dr. Smith’s lab focuses primarily on pediatric brain tumors and vascular disease. He is particularly interested in detecting brain tumors early, as well as why some brain tumors are benign and why others are invasive. Through his efforts they have developed a non-invasive urine test to try to detect tumors earlier and are now testing it in a national trial. With Be Brave for Life’s donation he has begun to expand this work to benign brain tumors so that they can be detected earlier and treated more effectively, while saving healthcare dollars.
“MASTERING INFLUENCE AND PERSUASION”
In early September 2015, Riley reached out to her snowboard coach, Lucyann Murray, to ask for her support of Be Brave for Life and to invite her to the Hike-A-Thon. Lucyann was so inspired by Riley’s spirit and determination, she chose to pitch Be Brave for Life to her graduate school peers as the charity they would support as part of her “Mastering Influence and Persuasion” class at Yale University Graduate School. Over a six week period, she and her team worked to market, create awareness and raise money for Be Brave for Life. Below is the pitch she used to convince her classmates to take on Riley’s cause. Lucyann and her team raised over $3,500 for Be Brave for Life, $1,500 more than any of the other teams.
As you know, I am a masters student at yale. SOM and FES.
As you may not know, I coach a snowboard competition team.
I get to spend every weekend in the winter sharing my passion with young athletes chasing their dreams… I also spend a lot of time chasing them.
I want to share a story about an 12 year old girl, Riley, whose determination to chase her dreams is stronger, bolder, and braver than any I have ever seen.
At the beginning of this past winter, I watched Riley take a fall while trying a new trick that would have knocked any of the boys out for the day. Instead of giving up, strong, bold, and brave Riley got right back up… and as we say on the mountain, she nailed that trick.
In January, Riley had her second craniotomy to remove a benign tumor on her brain stem. Emerging from surgery with double vision and other sensory complications, we were sure Riley’s season was over.
Riley was not. Six weeks later, with unwieldy determination, Riley was back on the mountain, following me right in my tracks with the biggest grin I have ever seen.
Six months later, Riley and her friend Sarah have raised over $20,000 for research on pediatric brain tumors.
I ask you to join me in supporting Riley. Strength, boldness, and bravery are qualities you and I will need daily as we take on the world as leaders for business and society. We can empower the next generation as they inspire us. Together, inspired and empowered, we will defy limits.