Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation have partnered to fund more than $3.3 million in Diffuse Intrinsic Pontine Glioma (DIPG) research grants. Their first round of grants was announced in 2017 and included a research grant awarded to Dr. David Ashley, the Director of the Preston Robert Tisch Brain Tumor Center at Duke University.
In recent years, the Duke University team has developed an immunotherapy treatment that uses a modified form of the poliovirus to treat brain tumors. This treatment has received significant attention, including two segments on 60 Minutes. In 2017, the Duke team began a clinical trial using the poliovirus vaccine in children with high-grade gliomas, but DIPG patients were excluded due to a risk of inflammation. In this study, “Recombinant Attenuated Poliovirus Immunization Vectors Targeting H3.3 K27M in DIPG,” Dr. Ashley works to modify the poliovirus to effectively target the H3.3 K27M mutation in DIPG. This mutation occurs in approximately 80-percent of DIPG tumors.
“The Duke team has been doing groundbreaking work in developing the polio virus for treatment of brain tumors in adults,” said Defeat DIPG co-founder, Mark Mosier. “When we saw the stories on 60 Minutes, we knew that we needed to bring this treatment to DIPG. We are very encouraged by the initial work on this project, and we are excited about the possibility that DIPG patients will receive the polio virus treatment in the future.”
Dr. Ashley spoke with the Defeat DIPG ChadTough team to provide an update on this project:
Q: Can you provide an overview of your research project, specifically the excitement around the polio vaccine and the adjustments you’re working on to make this a possible treatment for DIPG?
Dr. Ashley: “The polio virus, in its original form, is a rapidly replicating virus. It causes a lot of inflammation it is an entero-virus, entering patients through the gastrointestinal tract. Matthias Gromier and his colleagues changed that part of the original virus that caused brain toxicity through taking part of the virus out and exchanging it for part of the common cold virus. So, we were able to maintain the inflammatory parts of it, but take away the parts of it that cause the injury to the brain and spinal cord cells.”
“The other interesting part is that the modified polio virus is able to attack cancer cells almost exclusively. Almost every human cancer cell has the entry receptor on it. This means the virus can get into the cancer cell very easily, replicates like crazy and causes inflammation, causes an immune response, and that’s the basis of the use of the polio virus. With use in adults, and with three children we’ve treated now in a pediatric study, we inject the modified virus directly into the brain tumors of the patients. That does seem to be successful in approximately a quarter to a third of patients in causing long-term responses of disease stability in glioblastoma.
“In thinking about DIPG, there’s a couple of issues. One is the delivery of something into the brain that can cause a lot of inflammation. That’s why we haven’t gone immediately to introducing this modified virus directly into the brain. The other is, DIPG does have this target that we’re hoping to exploit: the H3.3 K27M mutation. So, what we hope we are able to do is exploit the inflammation that’s caused by polio virus and add that bit of the H3.3 K27M mutation into the viral vector — into the virus itself — and use it as an immunization, not unlike the way we give original polio vaccine.
“The reason that we think this might be more effective than just using peptides, that under investigation for this illness, is that the virus is much more inflammatory than peptides by getting into the immune cells and it activating the immune cells. We think it’s really a clever way of administering a vaccine against the H3.3 K27M target in DIPG. That is the basis of this study.”
Q: Will you be injecting this vaccine directly into the tumor?
Dr. Ashley: Rather than using the polio virus for a direct injection into the brain tumor, we’re going to be using the polio virus construct in a vaccination schedule. Ultimately, the patient would get a vaccine just like they would get a polio vaccine — just an injection into the muscle. Then we think there will be immune responses to the virus and in turn to the mutant H3.3 K27M.”
Q: Is this particular mutation present in all DIPGs or only certain mutations of DIPG?
Dr. Ashley: “This is a mutation that’s in the vast majority of DIPG – approximately 80 percent. In fact, outside DIPG, this particular mutation is carried in other high grade tumors in childhood as well. So we would hope that this could be something that could be helpful for the majority of patients with DIPG. So, where to from here? The next step will be to go to the FDA to understand what other evidence or studies they’d like to see before we move toward clinical trial.”
Q: You recently submitted your manuscript for publication – what does that paper include?
Dr. Ashley: “We’ve created the construct, done the work rebuilding the virus, and then we’ve been able to do parallel experiments in mice and in human cells. We used a model system with a protein that we know works to immunize mice against tumors, so we showed that we could use the virus in that situation and get immune effects. Then, in addition to that, we’re able to take human cells and infect them with the human virus and show that we’re able to derive a very robust immune response in human cells … in a dish, if you like. So, we have two levels of evidence that this is going to work. One is in animals and the other is in a dish with human cells.”
Q: Can you articulate how important foundation funding is for research of this type of disease?
Dr. Ashley: “The answer is twofold. One, it’s a very rare disease. Although it’s horrible for families and the patients, obviously, it’s difficult to get funding for these sort of rare diseases, because public health institutions and large organizations tend to focus on the ‘big-ticket items,’ the big public health scourges. Second, it’s really hard to get initial startup funding to do this type of research, because it is pretty innovative and high risk. We didn’t know that this would work. We thought it would, we had hypothesised it would, but before you’ve got preliminary data to support your hypotheses, it’s really hard to get national peer-reviewed funding for this type of work. The funds that the Defeat DIPG and ChadTough Foundations provide allows us to do early, innovative work like this in rare diseases that otherwise would never get done.”
Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation have partnered to fund more than $3.3 million in Diffuse Intrinsic Pontine Glioma (DIPG) grants. Their first round of grants were announced in 2017 and included a fellowship awarded to Zach Reitman, MD, PhD (Harvard University, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center).
Reitman spoke with Defeat DIPG ChadTough team to provide an update on his project, “Prioritizing PPM1D mutations as a target for new DIPG therapies,” for foundation supporters:
Q: Can you provide an overview of your research project?
A: “Happy to do so. In both the lab I’m working in and around the world, much has recently been learned about the biology of DIPGs through genomic sequencing. This work has revealed what we think drives these tumors to grow inappropriately. I’m focusing on one of the findings of this research, which is that one particular gene called PPM1D is likely to be a key driver of DIPG.
“My project is to find out more about how and why this PPM1D gene drives DIPG tumor growth. We’re growing DIPG tumor cells in a petri dish, and in mice, as laboratory models of DIPG. We’re then using genetic techniques to experiment with the PPM1D gene to find out how PPM1D is driving these model DIPG tumors to grow.
“This is really important because it will give us some clues as to why these deadly brain tumors are forming. I think it will also give us some important information on what the best way is to treat these tumors. For instance, we are finding out whether treatments that affect what the PPM1D gene is doing are likely to be effective treatments for DIPG.”
Q: How did you come across this idea?
A: “It’s really an amazing story. A lot of researchers became interested in looking at the genetics of DIPGs over the past 10 years. One of the reasons this was done is because the technology to sequence the genome and look at the genetics of tumors has dramatically improved over the past several years .
“This inspired several groups to carefully collect tumor tissue from patients with DIPG, and then to apply some of these new genomics technologies to uncover what really makes these DIPGs tick. At the time, I was working in a lab at Duke University. We carried out some genomic analyses of DIPGs, and we found this fascinating change in the PPM1D gene that I’m studying now.
“So this idea all dates back to that finding. One of the neat parts of the story is that several other groups were also looking at the genomics of DIPG patients and found that this same PPM1D gene is mutated in up to a quarter of DIPG patients worldwide. That really built up our confidence that this is really an important piece of the biology of DIPG, and not just something that was unique to the handful of DIPG tumors we had studied. It’s really a remarkable story where new technology came around, led researchers around the world to conduct some bold investigations with the permission of the patients and their families, and it led to paradigm-shifting discoveries.
“I’m now following up one of those discoveries and I’m hopeful this will help identify effective treatments for this deadly tumor.”
Q: One important thing you mentioned is collaboration across different labs and different researchers. Chad Carr’s tumor was sequenced at Michigan and they found PTEN as a key driver. What are your thoughts are around the different mutations of DIPG and the different people studying DIPG across a number of institutions?
A: “It’s so important for different researchers to study DIPG for a few reasons:
“First, I think studying diverse key drivers in DIPG is really important. This is because what we’ve found is that genetically, each DIPG is a little bit different – each patient’s tumor can have a different key driver or drivers. Some tumors, like Chad’s tumor, have PTEN as a key driver. Other tumors might have PPM1D, the gene I’m working on, as a key driver. Some tumors might have both. Others might have neither, or something else. This is why we need different groups to study the different key drivers in DIPG. We need to identify treatments that could work on tumors that have PTEN as a key driver. And we need to identify treatments that could work on patients with PPM1D as a key driver. The same goes for a number of other important drivers in DIPG. That way, when a child is diagnosed with a DIPG, there is going to be a high likelihood that research is being done that could be important for that child’s tumor.
“Second, I think having multiple researchers focusing on DIPG gives us more “shots on goal.” In cancer research, it’s very hard predict which research projects are going to ultimately result in an improvement in the standard of care for patients. In fact, most lines of research don’t ultimately lead to an improvement in how patients do. I think this speaks to the magnitude of the challenge we are up against. But there have been some amazing success stories in other types of cancer. Some types of cancer that were incurable in the past are now curable due to successful research efforts. I think by supporting many diverse ideas on how to improve care for DIPG patients, you maximize the chances that at least one of them will result in a success story for DIPG.
“Third, DIPG is a rare tumor. If only one institution studied it, you’d only have a few patients to glean information from, and you wouldn’t get a very complete picture of the biology of the disease or what treatments are going to work on the majority of DIPG patients. It’s so important to have many different institutions working together on this problem so we can combine our knowledge.”
Q: Can you go over your method and what you’re looking to produce out of your project? The answers you’re hoping to generate?
A: “One of the key methods we’re using is called CRISPR. This is a technique that’s emerged in the past few years in the molecular biology community that lets us edit the genome. A little bit of background: every cell in our body — or every cell in a DIPG tumor — contains approximately three billion pieces of DNA, or letters of DNA. CRISPR lets us very precisely edit a specific letter of that DNA. It’s a game changer.
“The CRISPR technology lets us ask some therapeutically imporatant questions. For instance, it lets us ask if a particular letter of DNA is important for DIPG cells to grow. One of the central aims of my work is to test whether a few important letters in the PPM1D gene are important for the growth of DIPG tumors using CRISPR. We’re using CRISPR to edit this gene and see if that causes the tumor to stop growing. Our hope is to produce a publication describing this, describing what we see, and we think that this will be valuable to determine whether treatments that affect this particular gene are worth pursuing.”
Q: Can you explain what happens after you publish a paper? How does that work in the grand scheme of DIPG?
A: “While our ultimate goal is to identify new treatments for DIPG, publications are an important milestone because they allow us to disseminate new research findings. This benefits the DIPG research community, because it gives other scientists a chance to build on our results. This also gives other groups a chance to validate our work and make sure its reproducible. An important part of the publication process is that the research is peer reviewed by other scientists, who make sure the work is rigorous and provide some feedback that can be very helpful to future work. And we continue to get feedback from the research community after the publication, which can be helpful for ongoing work and could even result in fruitful research collaborations.
“In some cases, publications can provide information for folks in the pharmaceutical industry in order to help them develop the best new cancer therapies. An important piece of background for this project is that pharmaceutical companies have already been developing chemicals that specifically target PPM1D, which is the gene we’re studying. This could eventually lead the way to a drug that targets PPM1D in the clinic. But there’s still a long way to go to get a drug that’s ready for clinical trials in kids. Developing a drug to that point is costly and takes years. A pharmaceutical company has to have a information that indicates that their drug likely to be helpful in order to make the investment to continue to pursue drug development.
“With our project, we hope that we can figure out if a drug that targets PPM1D is likely to be useful in kids with DIPG. If it is, it will provide a rationale that it might be worth the investment to develop a drug further. If we find out this isn’t likely to be a good strategy, it might help guide resources to be allocated towards more promising projects.
“Another interesting finding is that PPM1D also seems to be important in other types of cancers like breast cancer and some gynecologic cancers. So if we can show in our lab that it’s also important in DIPG, we can say, ‘Hey, look, this is a really important drug target. If a drug is developed that hits this target, it might be helpful for kids with DIPG in addition to these other tumor types. We’re hoping to provide a really fundamental biological insight that could prioritize whether this way of treating these tumors should be pursued further.”
Q: How will the Defeat DIPG ChadTough Fellowship impact your career?
A: “As I carry out this research project, the Defeat DIPG ChadTough Fellowship is providing me with research training that will help me in my career goal of establishing a laboratory aimed at identifying new treatment strategies for tumors like DIPG. As I mentioned above, I think work by multiple independent research groups is critical for the overall success of DIPG research. Training the next generation of research leaders is important to maintain this research community, and make sure that the very best people are going into DIPG research.
“A diverse set of skills is needed to lead a team that can tackle a terrible disease like DIPG. These skills do include a deep understanding of experimental laboratory techniques. But they also include skills to manage a team of researchers, to plan out long-term research projects, to understand how findings in the laboratory are translated to the clinic, and to effectively communicate results. And one needs experience in managing a budget to make sure funds are spent appropriately and effectively.
“Mastering these skills doesn’t happen overnight. For me, it started with my MD and PhD degrees at Duke University, which I followed with clinical residency training in the Harvard Radiation Oncology Program. The Defeat DIPG ChadTough fellowship is now giving me a chance to further master laboratory skills, to supervise a small group of researchers, and to start managing research funds. This is all being done under the supervision of my research mentors, Dr. Pratiti Bandopadhayay and Dr. Rameen Beroukhim, who both have a successful track record of leading research teams at the Dana-Farber Cancer Institute. By enabling me to gain this experience and to successfully complete meaningful projects, the fellowship is helping me towards my goal of one day leading a research group and identifying new treatment strategies for DIPG.”
Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation are excited to have Zach Reitman engaged in this research project, uncovering important data for DIPG research. Both foundations look forward to what he will do in the field in the future.
Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation are funding seven new Diffuse Intrinsic Pontine Glioma (DIPG)-specific research projects totaling more than $2.2 million over the next three years (2019-21). To date, the Defeat DIPG ChadTough partnership has committed $3.3 million to 13 DIPG research projects, ranging in focus from immunotherapy to fundamental biology of DIPG to epigenetics.
“To see that number – that we’ve come together to give $3.3 million to DIPG-specific research – is incredible,” said Tammi Carr, co-founder of The ChadTough Foundation. “Family foundations are the driving force behind finding a cure for this monster, so to be able to award this much over the past two years is an amazing feeling.”
The seven projects to be funded include: two research grants ($600,000 over three years), one fellowship ($150,000 over two years), and four new investigator grants ($250,000 over two years), which are new to this year’s Defeat DIPG ChadTough grantmaking cycle.
“A big part of our funding strategy is to increase the number of researchers dedicating their focus to DIPG,” said Defeat DIPG co-founder Mark Mosier. “New investigator grants are directed toward individuals in their first independent faculty position or those who have never researched brain tumors, which means we’re reaching a new group of scientists.”
Another important part of their strategy was to make longer-term commitments to funding the researchers awarded grants. All of the Defeat DIPG ChadTough grants are multi-year grants, allowing researchers to spend more of their time in the lab and less seeking additional funding for future years.
“As families that have been through the devastation of this disease, we recognize the urgency for answers,” said Jason Carr, co-founder of The ChadTough Foundation. “We put our grant application in front of as many talented researchers as we could, and structured our grants to set them up for success.”
All projects are reviewed by the Defeat DIPG Scientific Advisory Council made up of Darell Bigner (Duke University School of Medicine), Suzanne Baker (St. Jude Children’s Research Hospital), Oren Becher (Northwestern University’s Feinberg School of Medicine), Cynthia Hawkins (Hospital for Sick Children), Duane Mitchell (University of Florida College of Medicine), Michelle Monje (Stanford University), and Javad Nazarian (Children’s National Medical Center).
The Defeat DIPG ChadTough partnership was announced in 2017 with the funding of six projects (three research grants, three fellowships), totaling over $1 million over two years (2018-19). The Carr and Mosier families originally connected in the months following their sons being diagnosed with DIPG in September 2014. They quickly uncovered their shared passion for honoring their sons through raising research dollars.
Defeat DIPG and ChadTough bring together nine families with children who have fought or are fighting DIPG that are actively raising research dollars:
Michael Mosier Defeat DIPG Foundation is led by co-founders Mark and Jenny Mosier, and includes their Defeat DIPG Network chapters: Addison Grace Defeat DIPG Foundation (PA), Anthony’s Avengers Defeat DIPG Foundation (IL), Avery Huffman Defeat DIPG Foundation (WA), Connor Man Defeat DIPG Foundation (TX), and Vivian Rose Weaver Defeat DIPG Foundation (WA).
“Each family has channeled their grief into raising money for research,” said Jenny Mosier, Executive Director of Michael Mosier Defeat DIPG Foundation. “While nothing will erase the loss we feel, standing together to make tangible progress in this fight is one thing that keeps us going.”
Learn more about The ChadTough Foundation at chadtough.org and Michael Mosier Defeat DIPG Foundation at defeatdipg.org.
- Stefanie Galban, University of Michigan, “Targeting Cancer Stem cells in Diffuse Intrinsic Pontine Glioma (DIPG).
- Nalin Gupta and Daniel Lim, University of California San Francisco, “Use of Long Non-coding RNA (IncRNA) as a Therapeutic Target in DIPG”
New Investigator Grants
- Sameer Agnihotri, University of Pittsburgh, “Therapeutic Targeting of Metabolic Vulnerabilities in DIPG”
- Pratiti Bandopadhayay, Dana-Farber Cancer Institute, “Characterizing long non-coding RNAs as therapeutic targets in DIPG”
- Sujatha Venkataraman, University of Colorado-Denver, “MIC2 inhibition mediated apoptosis in DIPG”
- Nicholas Vitanza, Fred Hutchinson Cancer Research Center, “Optimal combinatorial targeting of HDAC inhibition and radiation in DIPG”
- Nneka Mbah, University of Michigan, “Therapeutic Targeting of the Disrupted Metabolic State in DIPG to Induce Ferroptotic Cell Death”
Michael Mosier Defeat DIPG® Foundation is pleased to announce the exciting news that it has been awarded a $71,370 grant by Select Equity Group Foundation. This is the second year of generous support from Select Equity Group Foundation, after an initial 2017 grant of $79,300 to support the Foundation’s DIPG research initiatives.
The 2018 Celebration Grant will play a critical role in supporting the Foundation’s growth through infrastructure expansion. Since its creation in 2015, the Foundation has seen substantial increase in revenue, as well as geographic expansion of its operations through the addition of chapters across the country. By strengthening its operations through staff hiring and further development of corporate partnerships, the Foundation will be even better situated to expand its impact on research funding to transform our understanding of the deadliest pediatric brain cancer, diffuse intrinsic pontine glioma (DIPG).
Select Equity Group Foundation’s mission is to make a positive impact by actively engaging all employees in identifying and supporting charitable organizations of excellence. Select Equity Group Foundation uses a democratic process as the primary mechanism for its grantmaking, whereby its employees nominate charities for consideration. Select Equity Group Foundation conducts a rigorous research process, including application by invitation and discussions with leadership before an award can be considered. To date, Select Equity Group Foundation has awarded more than $20 million in grants to nonprofit organizations in a range of areas.
Tracy Thomas, Associate General Counsel at Select Equity Group, championed Michael Mosier Defeat DIPG Foundation during their annual celebration. Tracy also volunteered for the Foundation this year at our 2nd Annual Defeat DIPG Dream Big Gala. We greatly appreciate Tracy’s efforts to share with her colleagues the critical need for investment in finding a cure for this devastating disease. We also thank Robert Wilson, Executive Director of Select Equity Group Foundation, as well as all of the Select Equity Group employees who supported the award of this grant.
Michael Mosier Defeat DIPG® Foundation is a 501(c)(3) nonprofit organization that supports research initiatives designed to find a cure for DIPG brain tumors. DIPG impacts hundreds of children each year in the U.S. alone, typically between the ages of 4 – 11 years old. These kids face a median survival of 9 months from diagnosis and overall survival of less than 1%.
The Defeat DIPG Foundation Board of Directors makes funding decisions, after consideration of recommendations from its Scientific Advisory Council (SAC). The SAC consists of preeminent doctors in the field of brain cancer and is led by Chair Darell D. Bigner, M.D., Ph.D., Director of The Preston Robert Tisch Brain Tumor Center at Duke University. Defeat DIPG Foundation has experienced significant growth since its creation in 2015, with the addition of five Defeat DIPG® Network chapters across the United States in Illinois (Anthony’s Avengers Defeat DIPG® Foundation), Pennsylvania (Addison Grace Defeat DIPG® Foundation), Texas (Connor Man Defeat DIPG® Foundation), and two in Washington (Avery Huffman Defeat DIPG® Foundation and Vivian Rose Weaver Defeat DIPG® Foundation).
There has been a significant increase in research focused on DIPG, including notable advancements of knowledge about the biology of the disease just in the past five years. With increased access to tumor tissue, more sophisticated technology, and greater information sharing between institutions, there is hope that children facing DIPG will soon have more options and hope for extended survival.
To learn more, visit DefeatDIPG.org.
The 2nd Annual Defeat DIPG Dream Big Gala, presented by DARCARS Automotive Group, took place on Saturday, November 3, 2018, at the Hyatt Regency Bethesda. Thanks to our sponsors, attendees and other supporters, we are thrilled to report that we raised over $435,000. We are Dreaming Big for a cure for DIPG brain cancer, and these funds will help us make that dream a reality.
We looked out on a room of 450 people at the gala, and we felt a powerful sense of hope and determination. With so many banding together to take on this horrible disease, we know that progress will be made.
We thank every DIPG family who allowed us to honor their child and especially those who attended, even knowing how difficult it can be on your already broken hearts. Three amazing DIPG families stood on stage and shared their stories with our guests, and it was their willingness to open their hearts that made the whole night. We give our heartfelt thanks to Lauren Gibson, Tammi & Jason Carr, and Rich & Nancy Engler. Dara, Chad, and Luke are an inspiration to us all.
Mitch Albom was unable to join us in person, but he took the time to send a powerful video about the beautiful Chika Jeune who fought DIPG so bravely. The love he and his wife had for Chika shined through, and we were honored to have her story as part of the event.
We thank every person who spent the night at the gala, sponsored DIPG family tickets, bought auction items, and otherwise donated to our mission.
We thank our Gala Host Committee that poured endless hours into making this event a success: Jacqueline Chan, Jenny Ellickson, Amy Elliott, Marguerite Laurent, Lori McGill, Amanda Posner, Amy Rogstad, and Jennifer Saulino.
We thank our Event Emcee Tommy McFly (TommyShow.com) and our generous sponsors:
- PRESENTING “VISIONARY” SPONSOR: DARCARS Automotive Group
- PLATINUM “HOPE” SPONSORS: PhRMA, [D3] District Design + Development, and NextGen LED
- BAR “STARGAZER” SPONSOR: Total Wine & More
- GOLD “IMAGINE” SPONSORS: Bayou Bakery, Good Stuff Eatery, and Hyatt Regency Bethesda
- TABLE SPONSORS: Ceros Financial Services, Inc., Children’s National Medical Center, Debevoise & Plimpton, Ernst & Young, FTI, Jack Silverman, DDS, K&L Gates, Next Level Rentals & Management, and Friends from Covington & Burling LLP.
- IN-KIND SPONSORS: CONTEND; Electra Entertainment; Sarah Marcella Creative; Barry Artistic Media Design; and BaseCamp DC
- SUPPORTERS: Cynthia Schnedar & Mark Polston; Lewis Asset Management; Sports and Orthopaedic Therapy Services; Central Maryland Oral and Maxillofacial Surgery, P.A.; Dominos; Scott Bilbrey; and Pepco
The Live Auction was run by the talented auctioneer Ernie Rogers and his team, who entertained the crowd and helped us raise some serious funds for DIPG research! Thank you to all who donated incredible Live Auction packages, including Lauren Phillips & Family; Maggie O’Neill; Chef Michael Friedman of The Red Hen and All-Purpose DC; Chef Erik Bruner-Yang; Total Wine & More; Jackson Family Wines, and Hyatt Hotels.
We thank Michael for being our light and inspiration, along with each and every child who has confronted this disease. Yes, we Dream Big. But these kids deserve that and so much more. Together, we will Defeat DIPG.
Save the Date: the 3rd Annual Defeat DIPG Dream Big Gala will be on Saturday, October 26, 2019, at the North Bethesda Marriott.
Michael Mosier Defeat DIPG Foundation has made a grant to support development of ONC201, an experimental drug currently being tested to treat DIPG. We have made this grant in collaboration with the Musella Foundation, The Cure Starts Now, Cancer Commons, and xCures.
Our objective in funding this program is to increase the availability of ONC201 for DIPG patients. The first goal is to facilitate enrollment in ONC201 clinical trials for any patient who is eligible for a trial. But we also recognize that many DIPG patients will not be able to enroll in a trial. For those patients, our goal is to assist them in getting access to ONC201 through an expanded access program where medically appropriate.
Oncoceutics has issued the following press release to announce the grant.
Philadelphia, PA (September 10, 2018) – Oncoceutics, Inc. announced the receipt of a Small Business Innovation Research (SBIR) Phase IIB Bridge Award from the National Cancer Institute (NCI). The NCI SBIR Bridge Award will allow Oncoceutics to expand and accelerate its clinical trials evaluating ONC201 in patients with a specific type of lethal brain cancer called H3 K27M-mutant glioma.
The awarded project, “Adaptive Clinical Efficacy Evaluation of ONC201 in Recurrent High-Grade Glioma” (grant number 2R44CA192427-04), builds upon the work supported by previous NCI SBIR Fast-Track grant that supported Oncoceutics’ effort demonstrating the activity of ONC201 in high-grade gliomas, including those with the H3 K27M mutation that is commonly present in the midline region of the brain. Each year, the NCI’s SBIR Development Center presents the Phase IIB Bridge Award to a select number of companies based on NCI and external expert review of the scientific merits and commercial potential of the technology. Awardees are required to allocate third-party investment to match the Bridge funding prior to submitting the funding application which occurred in mid-2017. The NCI Bridge Award will provide $3 million to Oncoceutics to advance the clinical development of ONC201 over the next 3 years.
Patients with H3 K27M-mutant glioma often have significant neurological symptoms from their disease and lack proven therapeutic options other than palliative radiotherapy. However, emerging clinical results have shown that some patients treated with single agent ONC201 have stable disease, have had their tumor shrink, and/or have had improvements in neurological symptoms, such as paralyses of peripheral and cranial nerves. This has been observed in adults and children treated with ONC201, including children with diffuse intrinsic pontine glioma (DIPG), a type of high-grade glioma that almost uniformly (80-90%) harbors the H3 K27M mutation.
“We are extremely pleased to see continued support from the NCI for the ONC201 clinical program in H3 K27M-mutant gliomas based on the clinical activity observed in our Phase II study supported by the Fast-Track SBIR grant,” said Patrick Wen, MD, Director, Center For Neuro-Oncology, Dana-Farber Cancer Institute.
“The emerging clinical experience with ONC201 to treat gliomas at our institution and other leading cancer centers around the country is exciting,” added said Yazmin Odia, MD MS, Lead Physician of Medical Neuro-Oncology, Miami Cancer Institute. “The dismal prognosis of midline gliomas and the dearth of therapeutic options means that this therapy could be practice-changing for neuro-oncology. We are eager to follow up on the radiographic and clinical improvements in biomarker-defined patients that we have seen with ONC201 as a single agent in our ongoing clinical trials. Future co-operative group efforts between NRG Oncology and the Children’s Oncology Group to test this drug in children and adults with newly diagnosed H3 K27M-mutant midline gliomas are in the planning process.”
In addition to the support from the NCI, Oncoceutics has also received support from The Musella Foundation, a non-profit organization that helps brain tumor patients through education, advocacy, and financial support. The Musella Foundation has supported Oncoceutics’ development of ONC201 in high-grade gliomas for several years from bench to Phase II clinical trials. The Musella Foundation, in collaboration with Cancer Commons, Michael Mosier Defeat DIPG Foundation, The Cure Starts Now Foundation and xCures, has committed to contribute at least $1 million in hopes of accelerating the development of ONC201.
“Having represented and supported brain tumor patients’ interests for several decades, it has been frustrating to experience the failures of new treatments that have been developed to impact the outcome of this disease,” said Al Musella, Founder and President of the Musella Foundation. “As highlighted in a recent ODAC meeting, the FDA, in collaboration with the NCI, is tasked with determining whether a molecular target is or is not considered substantially relevant to the growth or progression of pediatric cancer. We are excited to work with Oncoceutics that is developing a molecularly targeted agent, ONC201, that demonstrates the potential to advance the concept of Precision Medicine in patients that harbor the H3K27M mutation, a genetic aberration that is considered substantially relevant for the outcome of this disease. We are looking forward to contribute to making this drug available for as many patients as possible.”
Oncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds that selectively target G protein-coupled receptors for oncology. The first lead compound to result from this program is ONC201, an orally active DRD2 small molecule antagonist that is well-tolerated and effective against advanced cancers. The company completed a successful Phase I study in solid tumors and has underway a number of Phase I/II and Phase II clinical programs in both solid and hematological malignancies. Oncoceutics and collaborative groups have received more than $7 million in grants over the last two years, including grants from the National Cancer Institute, the U.S. Food and Drug Administration, the Pennsylvania Department of Health, and The Musella Foundation. In addition, outside interest in the company’s portfolio has resulted in several R&D alliance agreements between Oncoceutics and leading comprehensive cancer centers, including The University of Texas MD Anderson Cancer Center and the Fox Chase Cancer Center. The company has established a robust intellectual property position, including several issued patents. Visit oncoceutics.com for more information.
About The Musella Foundation
The Musella Foundation For Brain Tumor Research & Information, Inc is a 501(C)3 nonprofit public charity dedicated to helping brain tumor patients through emotional and financial support, education, advocacy and raising money for brain tumor research. Visit virtualtrials.com for more information.
About Cancer Commons
Cancer Commons is a nonprofit collaborative of patients, physicians, and scientists, dedicated to improving patient outcomes by tightly coupling clinical research and care. We arm patients and their physicians with the knowledge they need to achieve the best possible outcomes, help them access the relevant treatments and trials, and track their results to continuously learn. Vistit cancercommons.org for more information.
About Michael Mosier Defeat DIPG Foundation
Michael Mosier Defeat DIPG Foundation is committed to finding a cure for brainstem tumors known as diffuse intrinsic pontine gliomas (DIPG). Nearly every day one child in the United States is diagnosed with DIPG and another child dies from it. The Foundation seeks to make a difference and defeat DIPG both by raising awareness of DIPG and by providing funding for research into effective treatments for DIPG. Visit defeatdipg.org for more information.
About The Cure Starts Now Foundation
The Cure Starts Now Foundation is a 501(c)3 nonprofit organization with international chapters in nearly 40 locations around the world. It is has funded universal cancer cure strategies starting with cancers such as DIPG and organizes the biennial International DIPG Symposium showcasing innovative research methods. You can learn more at www.thecurestartsnow.org.
xCures is developing an AI-based methodology and platform to run ‘Virtual Trials’, which continuously learn from the clinical experiences of all patients, on all treatments, all the time. Each patient’s treatment regimen is adaptively planned by a ‘Virtual Tumor Board’ to optimize their individual outcome, and these plans are coordinated across the whole patient population to maximize collective learning. Visit xcures.com for more information.
Michael Mosier Defeat DIPG Foundation had a lot to celebrate on this year’s 4th of July, with over $115,000 raised to fund research for a cure for DIPG, thanks to the generosity of Washington Capitals player John Carlson and his wife, Gina.
In June, the Washington Capitals won the first Stanley Cup in franchise history. Following hockey tradition that has developed over the years, each team member gets the chance to spend a day with the prized Stanley Cup. John Carlson, a star defenseman on the Washington Capitals, chose to spend part of his day with the Stanley Cup to benefit Michael Mosier Defeat DIPG Foundation.
John Carlson was the highest-scoring NHL defenseman during the 2017-18 regular season and playoffs, and he has spent his whole career with the Washington Capitals since he joined the team as a first-round draft pick in 2008. Caps fans were thrilled to learn that John recently signed a new eight-year contact with the Capitals and will be continuing as a key contributor to the team’s success.
Carlson, along with his family, brought the Stanley Cup to a fundraiser organized by Michael Mosier Defeat DIPG Foundation on the grounds of Community Partner Bethesda Row. Over 1,000 fans gathered to view the Cup and to continue the celebration of the Capitals’ victory. Grant Paulsen, who hosts NBC’s pre- and post-game Capitals coverage and Grant and Danny on 106.7 The Fan, did an outstanding job as the emcee of the event. General admission tickets included free food and entertainment, along with a view of the Stanley Cup. Limited VIP tickets were also available and gave fans the exciting opportunity of a photo with John Carlson and the Stanley Cup. In the first ten minutes of ticket sales, over $40,000 of tickets were purchased. The event sold out entirely in 3 days.
The Capitals released a phenomenal video that captures some of John Carlson’s day with the Stanley Cup. In addition to the fundraiser at Bethesda Row, Carlson visited a fire department, Children’s National Medical Center, and a Kenwood neighborhood parade (whose participants also generously contributed to Defeat DIPG Foundation as part of their event).
Beyond the significant funds raised to help find treatments for children fighting this devastating childhood brain cancer, this event offered a unique opportunity to raise awareness of DIPG to a broad audience. News outlets including Fox 5 DC, NHL.com, WTOP, Bethesda Magazine, and featured the event and the work of the Foundation.
The Foundation also had the opportunity to partner with new sponsors who were valued contributors to the event. Community Partner Bethesda Row was an essential partner and co-host. Mamma Lucia Restaurants provided complimentary food for all attendees, gift cards for VIP attendees, and a donation of all proceeds from beverages sold at the event; Medium Rare donated food and beverages for the VIP tent; and Waldorf Toyota was a generous financial supporter. Thank you to Sarah Marcella Creative for generously donating time and services for the VIP photography, Harry Greenspun for general event photos, and Everything Entertainment for their photo booths. We also are deeply grateful for the support of the Montgomery County Police Department for their support.
Vivian Rose Weaver Defeat DIPG Foundation, in Husum, WA, is excited to announce that a generous anonymous supporter has donated $1 million that will be used towards DIPG-specific research funding in 2018. Vivian Rose Weaver Defeat DIPG Foundation was inspired by, and established in honor of, Vivian Rose Weaver, who is a smart, articulate, funny, sweet, thoughtful, imaginative, precocious, 3.5-year-old little girl who was diagnosed in February 2018 with a brainstem tumor called diffuse intrinsic pontine glioma (DIPG). DIPG is the deadliest form of pediatric brain cancer. Vivian Rose Weaver Defeat DIPG Foundation is a chapter of Michael Mosier Defeat DIPG Foundation and part of Defeat DIPG Network.
Co-Founders Katie and Simon Weaver are focusing on Vivian’s care, and her little sister Lucie, as their top priority. But they are also already making a substantial impact on the field of DIPG research by raising crucial funds to find a cure.
And the Weavers are not stopping with $1 million.
Vivian Rose Defeat DIPG Foundation would like to raise an additional $1 million this year, with an overall 2018 goal of $2 million raised for research. To put these numbers in perspective, the overall funding for DIPG research in any given year has typically been – at most – $2-5 million in a year. This is .0005% of the total funding for cancer research.
The funds raised will support the most promising DIPG research initiatives through the Defeat DIPG grantmaking process. All grant applications go through a rigorous review by a preeminent group of brain tumor experts that serve on the Defeat DIPG Scientific Advisory Council to ensure funds are used efficiently for initiatives with high scientific merit.
We call on everyone to join this effort. And, it couldn’t be easier.
An initiative called the #LemonFaceChallenge, started by a little girl named Aubreigh who is also fighting DIPG, has triggered worldwide attention to DIPG brain tumors. It has been heartening to see professional sports teams and coaches, members of the media, and so many others participate. The challenge is in the mold of the Ice Bucket Challenge, which raised $115 million in the summer of 2014 for ALS (amyotrophic lateral sclerosis or Lou Gehrig’s disease), dramatically increasing available funds for research for that disease.
Vivian Weaver, who inspired the $1 million donation to DIPG research, along with her parents Simon and Katie, are asking you to help us generate even more funds to find a cure. Vivian and the Weaver family are taking #LemonFaceChallenge and calling on you to keep the challenge going.
Here’s what you do:
- Take a video of yourself, your kids, your friends, your co-workers – anyone you can who will participate in the #LemonFaceChallenge, which means you take a big bite out of a lemon wedge!
- Tell them you are donating to Defeat DIPG and doing the video to find a cure for children facing DIPG, the deadliest form of pediatric brain cancer.
- Share the video and tag as many friends as you can and ask them to take the challenge.
- Donate to Vivian Rose Defeat DIPG Foundation through the Facebook fundraiser pinned to the top of their Facebook page (which has no processing fees), through their website, or through any member of Defeat DIPG Network.
DIPG is the deadliest form of pediatric brain cancer, with a median survival from diagnosis of 9 months and a near 0% survival overall. DIPG typically strikes children between ages four and eleven. Because of its location in the brainstem where all motor activity is controlled, DIPG is inoperable. The disease progresses by taking over a child’s motor functions one-by-one, typically starting with vision and balance problems, before moving to partial paralysis, followed by the inability to chew, speak, swallow, move and eventually breathe – all of this while the child remains mentally intact.
Raising $2 million through this initiative is a lofty goal. But, if kids like Vivian can fight hard every single day to beat this brain tumor, we can fight just as hard to make sure there are effective treatments for kids like her.
By supporting Defeat DIPG Network, you have the opportunity to move the needle and help us save lives of our precious children. Together, we will substantially increase research funding while also greatly increasing awareness of this devastating disease.