New ONC201 Expanded Access Program to Open with Funding from Defeat DIPG and Partners

Michael Mosier Defeat DIPG Foundation, with its chapters and partner families, has made a grant of $100,000 to support the opening of a new Expanded Access Program for ONC201, an experimental drug currently being tested to treat DIPG. From 2018 to 2020, Defeat DIPG has provided $266,666 to support development of ONC201, with a goal of increasing the availability of ONC201 for DIPG patients. Expanded access is a mechanism for patients to access this experimental therapy when the patient is unable to participate in a clinical trial and meets eligibility criteria.

Oncoceutics has issued the following press release to announce the grant.


Philadelphia, PA (November 5, 2020) – Oncoceutics, a clinical-stage drug discovery and development company, announced today that it has received grants from Michael Mosier Defeat DIPG Foundation, Dragon Master Foundation and The ChadTough Foundation to fund an Expanded Access program for ONC201 in the United States. 

The program will allow Oncoceutics to work with physicians practicing in the United States to provide ONC201 to patients with H3 K27M-mutant glioma, including DIPG, who meet certain eligibility criteria and who cannot otherwise access ONC201 by participating in an ongoing clinical trial. The Expanded Access program has been made available to select hospitals across the United States, though accrual is not yet open to patients. A list of centers that are in the process of initiating the program can be found on clinicaltrials.gov along with their contact information: click here. Patients and their families can reach out directly to these centers to discuss treatment options and enrollment timelines, as enrollment activities must be completed by the treating physician and their respective institutions in collaboration with Oncoceutics before a patient can be treated.

“At Oncoceutics, we have made it our mission to help patients with advanced cancers such as H3 K27M-mutant glioma and DIPG, and we are dedicated to advancing the clinical and regulatory development of ONC201 so that many more patients will have access to the treatment,” said Lee Schalop, MD, Chief Executive Officer of Oncoceutics. “We are grateful for the philanthropic support from the Michael Mosier Defeat DIPG Foundation, Dragon Master Foundation and The ChadTough Foundation as it enables us to provide ONC201 to more patients today while we are working towards our goal of obtaining regulatory approval to help brain tumor patients with ONC201 as a novel therapy.”

“Patients with DIPG and other brain cancers face the devastation of a challenging prognosis coupled with very few treatment options,” said Mark Mosier, Chairman of the Board of Directors of Michael Mosier Defeat DIPG Foundation. “Defeat DIPG, Dragon Master Foundation, and The ChadTough Foundation are funding the ONC201 Expanded Access program to help ensure patients have access to as many options as possible.”

Expanded Access – sometimes called “compassionate use” – provides a pathway for patients to receive an investigational medicine for a serious disease or condition. Expanded Access is often made available when: there are no comparable or satisfactory alternative therapies to treat the disease or condition; patient enrollment in clinical trials is not possible; potential patient benefit justifies the potential risk of treatment; and providing the investigational medicine will not interfere with investigational trials that could support the medicine’s marketing approval for the treatment indication.

ONC201 is an investigational medicine that is being evaluated in Phase I and Phase II trials. Investigational products have not yet been approved or cleared by FDA, and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

Requests for Expanded Access to ONC201 must be made by a U.S. licensed physician. Oncoceutics does not currently supply ONC201 outside of the U.S. Physicians can request access for a patient by sending an email to info@oncoceutics.com. For more information about the Expanded Access program, please refer to Oncoceutics’ Expanded Access Policy. FAQs about accessing ONC201 can be found here.

About Oncoceutics

Oncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds called imipridones that selectively target G protein-coupled receptors for oncology. ONC201 is an orally active small molecule DRD2 antagonist and ClpP agonist in late-stage clinical development for H3 K27M-mutant glioma with additional indications under clinical investigation. ONC206 is the second clinical-stage imipridone that is under clinical investigation for central nervous system tumors. The company is supported by grants from NCI, FDA, The Musella Foundation, Michael Mosier Defeat DIPG Foundation, Dragon Master Foundation, The ChadTough Foundation, the National Brain Tumor Society, and a series of private and public partnerships. Visit Oncoceutics.com or contact Press@oncoceutics.com for more information.

About Michael Mosier Defeat DIPG Foundation

Michael Mosier Defeat DIPG Foundation works to find a cure for brainstem tumors known as diffuse intrinsic pontine gliomas (DIPG), the deadliest form of childhood brain cancer.  The Foundation focuses on funding innovative research to find treatments for DIPG and raising awareness of this devastating disease.  Visit defeatdipg.org for more information.

About Dragon Master Foundation

Dragon Master Foundation’s mission is to find and accelerate cures for cancers and other diseases by fostering and rewarding a community of collaboration and innovation. We seek to spread awareness, expedite research and kinder treatments, and improve the quality of life for patients and their families. We invite your engagement at www.dragonmaster.org. Dragon Master Foundation’s support included contributions from Friends of Emily Hood, Mithil Prasad Foundation, Musella Foundation for Brain Tumor Research, National Brain Tumor Society, Storm the Heavens Fund, and Team Cozzi Foundation.

About The ChadTough Foundation

The mission of The ChadTough Foundation is to inspire and fund game-changing research to discover effective treatments for pediatric brain cancer, with an emphasis on Diffuse Intrinsic Pontine Glioma (DIPG). Visit www.ChadTough.org for more information.

First Round of Fellowship Grants Concludes Successfully

Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation, with their chapters and partner families, first joined efforts in late 2017 to jointly fund promising research focused on DIPG.  In the past three years, the foundations have awarded more than $6.1 million to fund DIPG-specific research projects through their grant program.

Three of the initial grant awards were fellowships, which are designed to encourage outstanding scientists to choose a career involving DIPG research.  They were awarded to Dr. Jamie Anastas of Boston Children’s Hospital, Dr. Zach Reitman of Dana-Farber Cancer Institute, and Dr. Chen Shen of Northwestern University.  

In addition to supporting important areas of DIPG research, Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation fund fellowships to encourage emerging scientists to study pediatric brain cancer, with a focus on DIPG.  By increasing the researchers working on a cure for this devastating disease, the foundations hope to accelerate progress. 

The first round of fellowships was a success: the studies will inform new research areas for DIPG and each of the three scientists will continue their career with research to find a cure for DIPG.

Dr. Jamie Anastas: “Targeting chromatin regulation to treat DIPG” 

Dr. Anastas studied how the histone mutation commonly found in DIPG affects how the tumor cells function. After screening 1,300 chromatin regulators, she identified multiple proteins that are necessary for DIPG cells to proliferate and survive, but are dispensable for normal cell growth. Her research focused on determining the roles of these proteins in DIPG development.

In talking about the importance of her Fellowship funding, Dr. Anastas explained, “Prior to receiving support from the Defeat DIPG ChadTough Fellowship program and other non-profits focused on supporting brain tumor research no one in my lab had studied brain tumors and we had no funding for the projects that I was keen to develop. Funding from these organizations has given me the freedom to initiate multiple high risk projects aimed at identifying novel therapeutic targets in DIPG and has prepared me to continue these studies as an Assistant Professor. ​ Without support from foundations like Defeat DIPG and ChadTough I can’t imagine these projects would have ever gotten off the ground, so I am extremely grateful.​

  • Summary of key findings: According to Dr. Anastas, certain chromatin binding proteins play a critical role in regulating DIPG growth and differentiation state. Dr. Anastas’s studies indicate that some of these factors bind regions of the genome where H3K27M mutant histones are enriched, suggesting that these factors play a direct role in promoting H3K27M-dependent up-regulation of stem and proliferation genes. Further studies reveal that loss of these chromatin regulators induces neurite-like processes, cell cycle arrest, and a transcriptional program enriched in neurons and oligodendrocytes compared to more progenitor-like cells. These data suggest the stem-like nature of brain tumor cells is not permanent and that abnormal transcription induced by H3K27M mutant histones might be reversed via chromatin-targeted therapies.
  • Next career steps: Dr. Anastas is currently wrapping up her postdoctorate work at Boston Children’s Hospital and Harvard Medical School, where her research focuses on determining the roles of aberrant epigenetic regulation in DIPG. In January 2021, she is looking forward to launching an independent brain tumor research lab as an Assistant Professor in the Department of Neurosurgery and in the Center for Cell and Gene Therapy at Baylor College of Medicine. In her prior work, after screening 1,300 chromatin regulators, she has identified multiple proteins that are necessary for DIPG cells to proliferate and survive, but are dispensable for normal cell growth.  Dr. Anastas is excited to follow-up on additional hits from these screens in her own lab with the long-term goal of developing new therapies to target these pathways driving DIPG tumorigenesis. “I’m also enthusiastic about continuing my work aimed at understanding why DIPG tumors sometimes initially respond to certain therapies, but then frequently develop resistance. A second major goal of my lab will be to search for ways to overcome tumor drug resistance through combination therapies and other strategies,” Dr. Anastas explained.

Dr. Zach Reitman: “Prioritizing PPM1D mutations as a target for new DIPG therapies” 

Dr. Reitman studied the role the PPM1D mutation plays in helping DIPG tumors grow. In this project, he tested whether targeting the PPM1D gene slows DIPG cell growth to determine whether a PPM1D inhibitor should be developed as a potential treatment for DIPG.

Dr. Reitman describes the importance of the Defeat DIPG ChadTough Fellowship as follows: “It has been really helpful to have the Defeat DIPG ChadTough Fellowship to carry out rigorous, in-depth DIPG research and to help initiate a new DIPG research lab.  This Fellowship helps individuals to establish their expertise in an area of DIPG research that can help them compete for larger grants from the National Institutes of Health and other organizations as they advance in their career.  In a way, it’s an investment that could pay off many-fold in terms of research dollars if the Fellow goes on to secure larger grants later in their career to push this important research forward.

  • Summary of key findings:  According to Dr. Reitman, “We found that PPM1D truncating mutations are associated with a requirement for PPM1D itself in DIPG cell lines. This provides evidence to suggest that PPM1D could be a therapeutic target in DIPG. We also carried out genetic screens that identify critical genetic interactions with PPM1D, revealing an increased dependency on the molecular pathways that control the response to DNA damage in cells that harbor mutant PPM1D. This suggests that strategies to target the DNA damage response pathway should be investigated as therapeutic approaches in DIPG.”
  • Next career steps: Dr. Reitman’s fellowship helped him transition to a new physician-scientist position at Duke University. In this faculty role, his ultimate goal is to establish a research program focused on new therapeutic strategies for brain tumors, with a special emphasis on pediatric brain tumors and on DIPG. Dr. Reitman and his team and building up experimental models of DIPG that enable ongoing experiments investigating the biology of DIPG and potential new treatment strategies.  These include experimental models that are derived from human DIPG tissues, and mice that are genetically engineered to develop DIPGs.  They are also carrying out mechanistic experiments looking at ways to improve the response of these tumors to radiation therapy, to increase its effectiveness and reduce its side effects.  Some of this work is funded by a New Investigator Award from the Defeat DIPG, ChadTough, and SoSoStrong Foundations.  

Dr. Chen Shen: “Dissection of ATRX in Diffuse Intrinsic Pontine Glioma” 

Dr. Shen focused on the ATRX protein and its role in driving DIPG tumor growth. Her hypothesis was that the loss of ATRX works with the histone mutation to promote DIPG growth. She investigated whether the loss of ATRX impacts the DIPG tumor’s response to radiation. 

  • Summary of Key Findings:  According to Dr. Shen, “The RNA-seq results suggested that, ATRX status affects the histone markers, although both the ATRX WT and the deficient group harbor the H3.3K27M mutation. Thus, further experiments will be done to reveal the effects of this negative enrichment of histone markers.”
  • Next career steps: Dr. Shen is continuing her work in Dr. Oren Becher’s lab at Lurie Children’s Hospital of Chicago.  She is expanding on the DIPG research she completed as part of her fellowship.

Defeat DIPG and ChadTough are thrilled to see the growth in research focused on DIPG.  In addition to the three fellowships that have concluded, the funding partners are excited about the four additional fellowships that are currently in process: Dr. Nneka Mbah of Michigan Medicine, Dr. Alan Jiao of Boston Children’s Hospital, Dr. Eshini Panditharatna of Dana-Farber Cancer Institute, and Dr. Xu Zhang of Columbia University.

Researchers require seed funding to investigate new ideas, and available funding can influence which focus areas young researchers choose.  Foundations like Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation play a critical role in driving promising scientists towards a field of DIPG and pediatric brain cancer research by supporting these researchers early in their careers.

Innovative DIPG Trial Opening Soon

Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation, with their chapters and partner families, have granted over $600,000 for the Diffuse Midline Glioma – Adaptive Clinical Trial (DMG-ACT), a new collaboration through the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and the DIPG Centre of Expertise in Zurich.

Left to Right: Katie Gaskin, Mark Mosier, Jenny Mosier, Dr. Sabine Mueller, Dr. Carl Koschmann, Jason Carr

This innovative project will support preclinical and clinical work across multiple institutions to move combination therapies for DIPG into the clinic as quickly as possible. Scientists across the world will share their latest preclinical data, greatly enhancing opportunities for research impact and improved patient outcomes.

The DMG-ACT is an international, adaptive clinical trial for diffuse midline gliomas. DMGs are fast-growing, cancerous tumors found in the midline of the brain, and they include diffuse intrinsic pontine glioma (DIPG) tumors.

“We’ve developed a clinical trial protocol, with multiple arms, where we will use evidence from multiple labs to bring the best combination therapies forward,” says Dr. Sabine Mueller, pediatric neuro-oncologist of University of California, San Francisco and Clinical Program Head at DIPG Centre of Expertise in Zurich. “Initially, our main concern is if drugs are really getting into the tumor of these devastating diffuse midline gliomas. If we do not see any early signs of survival impact, we will also look at drug penetration to decide if an arm should be adjusted or closed.” 

The other key focus of this program will be to offer combination therapies at each stage of the disease (newly diagnosed, post-radiation, and upon recurrence). In addition to new promising combinations, many critical studies will be performed (biopsies, blood draws, and lumbar punctures) for real-time analysis at labs around the world to learn which treatments are working and why. This trial setup is unprecedented for pediatric diffuse midline glioma. 

“PNOC has a very well organized and motivated clinical trial infrastructure in San Francisco and Zurich that allows trials to open quickly, complete quickly, and share data quickly. This allows us to speed up translating discoveries in the lab to the bedside and share our data with colleagues around the world in real time,” said Michigan Medicine pediatric oncologist Dr. Carl Koschmann, who serves as a PNOC site co-principal investigator.  PNOC is an international consortium with study sites within the United States, Canada, Europe and Australia.

Like many PNOC trials, DMG-ACT will require or offer sequencing along with the therapy, depending on the diagnosis. This data will be deposited into a central PNOC database that allows researchers within and outside of PNOC to access de-identified data. Researchers will then be able to work together to design the future generation of trials while also re-evaluating current trials.

The protocol for DMG-ACT is different from typical clinical trials in that it studies smaller groups of patients (15 to 20). Patients are then added or transitioned to other treatments based on initial findings. This design allows the trial to proceed much quicker than typical early-phase clinical trials.

As part of this trial, a consortium of more than 10 international labs (the DMG-ACT Pre-Clinical Team) has been studying single and combinatorial treatments on models of DMG. This data is then shared with the DMG-ACT Clinical Team. The drugs that are the most promising pre-clinically are then selected for use in combination in an arm of DMG-ACT. Once selected, the Pre-Clinical Team works to develop markers of treatment response to look for in the tissue and liquid samples that clinicians will collect from patients.

Researchers at PNOC are currently finalizing the protocol and agreement with sponsors of the initial combinations. The first arm of the trial should be open for enrollment early fall 2020 and includes the combination of ONC201 and panobinostat.

“Simply put,” said Dr. Koschmann, “the trial would not be opening this year without the current and future support of Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation. Their early and generous support of DMG-ACT allowed us to remove all brakes to get all aspects of the infrastructure created and get the trial open, with the international scale of pre-clinical and clinical aspects we felt our patients needed. For those of us involved with this trial, we feel this is the trial that will finally provide the jumps in improved survival for DMG that was seen in leukemia 40 to 50 years ago. It is fitting that Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation are part of our team for this.”

Frequently Asked Questions

Q: When will the trial open for enrolling patients?

We are currently estimating that the trial will be finalized in Fall 2020.  The earliest site openings are expected in late 2020, with broader openings in 2021.

Q: How can I find out if my child qualifies for this trial?

When the trial is finalized, more detailed information, including eligibility criteria, will be available through www.pnoc.us and www.clinicaltrials.gov

Q: What can I do to be ready to enroll when the trial opens?  

When the trial opens, patients (or their parents) may contact their nearest PNOC site, and let them know you would like to be considered for the DMG-ACT trial PNOC sites are listed at www.pnoc.us, or email PNOC at PNOC_Regulatory@ucsf.edu for further details.

Q: My child is currently enrolled in a different trial.  Can I switch to DMG-ACT so my child can get a combination therapy?

There are three cohorts to the trial in its current form: (1) newly diagnosed (before radiation), (2) post-radiation (before progression), and (3) at progression/relapse. It is less likely that a patient would be eligible for one of these cohorts if currently on a clinical trial without progression.

Q: Is it possible for my child to be involved in the trial, yet receive treatments at our own cancer center?

Treatment/enrollment will only be through PNOC sites. It is possible that some of the care can be performed locally.

Q: Will there be a required washout period before starting the trial?

Yes, washout periods will be similar to other clinical trials and will depend on which arm you enroll.

Dr. Sujatha Venkataraman: A Researcher’s Drive to Defeat DIPG After Loss of Son to Childhood Cancer

Dr. Sujatha Venkataraman, a researcher at the University of Colorado, was awarded a New Investigator grant jointly funded by Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation, with our chapters and partner families. The New Investigator grant allows funding for either newly independent DIPG researchers in establishing new DIPG research labs or established researchers who have not previously conducted brain tumor research to encourage them to start DIPG research.

We had the opportunity to talk with Dr. Venkataraman to get a more in-depth look at her research and the inspiration behind her work.

Q) We understand you were inspired to switch your research focus from adult breast and prostate cancer to pediatric cancer after you lost your child to the horrible disease. What inspired you to focus your energy on DIPG?

SV: My son, Rishi, was a happy 2-year-old when he was diagnosed with a devastating cancer — stage 4 neuroblastoma. During that time, I was doing my postdoctoral fellowship at the University of Iowa investigating the role of reactive oxygen species and hypoxia in adult prostate and breast tumors. The diagnosis took a toll on our family. He was given many rounds of chemotherapy and radiation, and he went through a bone marrow transplant, all in vain.

Rishi Venkataraman

After each chemotherapy round, I had to give him an injection to increase his white blood cell count, and those were the hardest moments in my life. The instant Rishi saw the needle in my hand, he used to scream and cry, “Mummy, please no!” He wouldn’t come to me for hours after I gave him the shot. My heart shattered into pieces. I tried to stay strong and think “one day,” when he becomes free from cancer and grows up into a big boy, he will understand that I was only trying to help him. That day never came.

Cancer is terrifying, and when it is diagnosed in children, it is devastating. As a mother who lost her young son to cancer, I say it’s the parent’s worst nightmare. I felt numb to everything. The tragic loss of my loving son changed my life. I don’t want any parent to go through this pain. That is when I decided I want to continue my research in pediatric cancer.

When we moved to Denver and after I joined the University of Colorado’s pediatric neuro-oncology division, I attended a neuro-oncology conference where I learned about the devastating brain tumor called DIPG, which affects children and has a median survival of only 11 months. I didn’t have any DIPG tumor lines to work with, so I contacted many centers where DIPG work was in progress, received cell lines, and started my research work in studying DIPG tumorigenesis. If, through my research work, I could save at least one child and one parent from losing their child, I have achieved my goal in life.

Q) Your current work centers on identifying the genes that cause DIPG and to identify the pathways that make the DIPG tumor aggressive after relapse from radiation treatment. Can you tell us a little more about this work and what you have discovered thus far?

SV: We have performed studies to identify pathways that are upregulated by radiation. We find that DIPG cells undergo senescence (loss of a cell’s power of division and growth) but not complete cell death after radiation. These senescent cells then reactivate and become more resistant to therapy. We have identified two specific pathways that drive the increased aggressiveness of the DIPG cells. We are now testing several new agents to target these pathways.

Q) What do you hope will be the conclusion of your research? How might the findings in your research impact DIPG patients? Will it drive changes in treatment?

SV: We hope that by the end of this project we will have established that several specific pathways drive DIPG cell aggressiveness in the face of radiation. We hope to show that suppressing these pathways resensitizes DIPG cells to radiation and other therapies. We plan to generate the preclinical data needed to justify additional large-scale studies and early-phase clinical trials for these new agents.

Q) What do you think have been some of the biggest breakthroughs in DIPG research over the last few years?

SV: In my opinion, the most significant breakthrough in DIPG biology was the discovery of characteristic mutations in the histone H3 protein, found in more than 80% of DIPG patient tumors and associated with a poor prognosis. The identification of H3K27M mutations and the resulting epigenetic changes now present numerous and promising druggable targets. Similarly, the identification of mutations that accompany the H3K27M mutation, like the ACVR1 receptor kinase mutation in a subset of DIPG tumors, presents opportunities for intervention. A recent study tracing the origin of DIPG tumor cells using single-cell RNA sequencing and another targeting cell surface antigens on DIPG tumor cells promise to reveal druggable targets. But even with the increasing number of breakthroughs in understanding the biology of DIPG, the standard of treatment has not changed, and there is still no improvement in patient survival.

Q) How important is private funding, such as the Defeat DIPG ChadTough New Investigator grant, in moving forward with your research?

SV: Brain tumors are the leading cause of death in children, but pediatric brain tumor research is acutely underfunded. Only 4 percent of federal funding is dedicated to childhood cancer research, and only a fraction of that is allocated to pediatric brain tumor research. This lack of funding hinders many of the critical studies that are needed to improve the outcome of children with brain cancer.

Private funding from foundations like The ChadTough Foundation and Michael Mosier Defeat DIPG Foundation is critically important as it enables more researchers to explore their innovative hypotheses and helps them obtain federal funding to move their ideas from the bench to the bedside. Without the funding, our project would likely have not been performed at the speed that it was. We will have a publication submitted describing data funded by the foundation within the next 6 months. The funding was critical as it enabled us to develop robust preclinical proof of principle data that will form the basis of additional NIH grants. We are hopeful that the successful completion of this work will form the basis of a clinical trial through the Pediatric Brain Tumor Consortium (PBTC) phase 1 trials.


Michael Mosier Defeat DIPG Foundation Announces New Oregon Chapter: Levi Harden Defeat DIPG Foundation

Michael Mosier Defeat DIPG®Foundation, a nonprofit whose mission is finding a cure for the deadliest pediatric brain cancer, DIPG (diffuse intrinsic pontine glioma), announced today the addition of a chapter in Gresham, Oregon, to its Defeat DIPG®Network:  Levi Harden Defeat DIPG®Foundation. Today would have been Levi Harden’s fourth birthday. Levi passed away from DIPG in October 2019, at three years old, just two months after diagnosis.

The new chapter adds to Defeat DIPG Network’s existing presence across the United States, in Maryland and Washington, D.C. as Michael Mosier Defeat DIPG Foundation, in Illinois as Anthony’s Avengers Defeat DIPG®Foundation, in Texas as Connor Man Defeat DIPG®Foundation, and in Washington as Avery Huffman Defeat DIPG®Foundation and Vivian Rose Weaver Defeat DIPG®Foundation. The Defeat DIPG Network has raised over $7.4 million for DIPG research in less than 5 years.

The Oregon chapter, which will operate as Levi Harden Defeat DIPG Foundation, is founded in memory of Levi Harden, son of Jenica and Nathan Harden. Levi loved sports, and from just two years old he could play miniature golf, hit a golf ball across the yard, play baseball, shoot hoops for hours and throw football with a perfect spiral. Levi was full of laughter and love and adored his big brother Clayton and new little sister Erin who was born around three weeks before his diagnosis with DIPG. 

The Harden Family
(left to right: Jenica, Erin, Levi, Nathan, and Clayton)

“We believe the best way to honor our son’s memory is to continue his fight against the disease that took his precious life,” says Jenica Castillo-Harden, Levi’s mother who will serve as Director of Levi Harden Defeat DIPG Foundation. “We are committed to finding a cure for DIPG because we believe that every child deserves a chance to grow up.  No family should have to hear their child’s doctor say, ‘there is nothing we can do.’ We know that together with our Defeat DIPG partners, we can change the future for kids with DIPG.” 

Jenny and Mark Mosier created Michael Mosier Defeat DIPG Foundation in June 2015 to fund DIPG research and promote awareness of the disease, after the passing of their 6-year-old son, Michael. With its geographic expansion and growth of existing initiatives, the Foundation expects to continue to increase its capacity to fund essential childhood cancer research.  

The Foundation, with its chapters, has announced over $7 million in DIPG-specific research funding, in partnership with The ChadTough Foundation. Two grants are also in partnership with SoSo Strong Pediatric Brain Tumor Foundation. Michael Mosier Defeat DIPG Foundation works with a preeminent Scientific Advisory Council of brain tumor experts that advises its Board of Directors on how to maximize its resources to fund research for a cure for DIPG. 

The Mosier, Harden, Gaskin, Huffman, Olympia, and Weaver families will work to grow the already powerful base of support in each of their communities, and to honor and unite all children and families who have had to confront this disease.  

“It is an honor to stand side-by-side with other families who share such a deep passion and dedication for finding a cure for DIPG,” says Jenny Mosier, Executive Director of Michael Mosier Defeat DIPG Foundation. “The devastating experience of losing our own children inspires our work to improve the dismal prognosis of less than 1% survival for children with DIPG. By driving more resources to DIPG research, we are pushing the field forward to find treatments for these children.” 

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.

For decades, treatment for DIPG has remained the same and has been ineffective. The entire amount spent annually on DIPG research – approximately $3 – 5 million – is less than 0.0005% of the total funding for cancer research.  In just the past few years, due to better medical technology and increased access to tumor tissue, researchers have made real advances in their understanding of this disease.  There is finally hope for progress in finding a cure.

Learn more about our new chapter at levidefeatdipg.org.

Stanford’s Dr. Michelle Monje Reports on Breakthrough DIPG Research Funded by Defeat DIPG and ChadTough

Michelle Monje, MD, PhD, is a pediatric neuro-oncologist at Stanford University and one of the world’s top researchers in the study of high grade-gliomas.  In 2017, Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation awarded a research grant to Dr. Monje for her project titled “The Tumor Microtube Network in DIPG:  Targeting a Possible ‘Achilles Hill’ Required to Defeat DIPG.”

Through this research, Dr. Monje discovered that deadly brain tumors integrate themselves into the brain’s electrical network and then hijack signals from healthy nerve cells to fuel their own growth. Her findings were published in Nature and featured on NPR, and The Defeat DIPG ChadTough team had an opportunity to discuss this project with her:

Q: You recently had a study published in Nature.  Can you tell us about that?

MM: We have, for many years in my lab, been trying to understand the way that DIPG and other pediatric high grade gliomas interact with the normal brain, particularly the abnormal cells in the developing childhood brain. One thing that we’ve learned in the past and that we’ve published, is that neural activity, the activity of the brain itself, very robustly promotes the growth of DIPG and other childhood brain tumors. One of the important mechanisms that we discovered that is responsible for this is the brain activity dependent release of a particular kind of growth factor.

This is a growth factor that under normal circumstances helps to promote brain plasticity, and this overall process might have roles in learning and memory and brain development, but the cancer is hijacking it and taking advantage. When we looked at it, this mechanism seems to be so important. If we disrupt it, DIPG really can’t grow. So we’ve been trying to understand why that’s true, and that prompted us to look at the cellular consequences of exposure to this molecule. 

One of the cellular consequences was upregulation of genes in the tumor cells that enable them to form networks. In adult glioblastoma this network formation had been described as occurring and it was kind of shocking to people because a previous conception of cancer is that one cell goes bad and it divides in this mindless way while some of the different cells take on different functions in the tumor. It’s homogeneity but basically it’s just continuous growth. What the paper in adult glioblastoma showed was that, in fact, the tumor cells were connecting with each other and forming kind of a cooperative network. What we’ve discovered is the extent to which that same kind of network formation was happening in DIPG between the tumor cells, and how that might interface with how the tumor interacts with the normal brain. The study also tries to understand if this is indeed important for DIPG growth and might represent a therapeutic target.

Q: Have you seen research trickling down to other pediatric brain cancers yet? Have you seen it making an impact?

MM: Yes, I think actually in the work I was just describing for you, that there’s a lot of cross pollination between different kinds of high grade gliomas. I described sort of a cross pollination between adult and pediatric high grade gliomas, but some of what we’ve discovered in DIPG we have found to be equally relevant to other forms of pediatric high grade gliomas. That starts some collaborations to look for similar physiologies or pathophysiologies in ependymoma and the broad area of understanding how the normal brain cells are interacting with the cancer cells. I think this is something that all pediatric brain tumor researchers need to think about; many of them are thinking about. A lot of what we’re learning in DIPG is helping to inform that.

Q: Chad Carr and Michael Mosier were diagnosed a little over 5 years ago.  Are there differences in treatment today versus 5 years ago?

MM: In the past 5 years, a number of laboratory studies on DIPG have identified new promising treatments for DIPG, from immunotherapy to novel drugs targeting epigenetic, metabolic and microenvironmental vulnerabilities of DIPG. Several new clinical trials based on these laboratory studies are now opening. We know more about the biology of DIPG than ever before, and soon that new knowledge may lead to effective therapy.

Q: What is the importance of private funding, such as the Defeat DIPG ChadTough grant you received for this project, in moving the field of DIPG forward?

MM: Private funding enables researchers to be more nimble and pursue new ideas more rapidly.

Q:  What do you think the impact will be of this groundbreaking discovery? Will it drive changes in treatment for DIPG patients?

MM: Our new appreciation that DIPG integrates into neural circuitry opens up a whole new dimension of possible therapeutic targets that I am hopeful will make a difference in outcomes for children with this terrible brain cancer. 

Researchers, Advocates, and Families Gather for Congressional Briefing & Summit for DIPG Awareness Day

Families, researchers, government regulators, and advocates gathered in Washington, DC, on February 13, 2020, for a multi-part event for the DIPG community, organized by the DIPG Advocacy Group.  There was an excellent turnout from throughout the country, a powerful example of collaboration and persistence of families who will not stop pushing to do better for the children facing DIPG (diffuse intrinsic pontine glioma). 

Jenny Mosier, Dr. Sabine Mueller, Dr. Eugene Hwang, and Dr. Carl Koschmann

In the morning, a Congressional Briefing was held to educate Congressional offices and advocate for passage of H. Res. 114, a resolution to support establishing May 17 as DIPG Awareness Day and encouraging greater consideration for pediatric and high mortality-rate cancers in the research grant process with public and private funding sources.  We thank Congresswoman Jackie Speier for her support for this important event.  There are nearly 100 co-sponsors for the bill to date, but additional support is needed.  Following the briefing, attendees gathered for a State of DIPG Summit that involved discussion of hot topics in the community, such as research initiatives, challenges facing patients and families, development of ONC201, the FDA regulatory framework, data-sharing, and advocacy opportunities.  A listing of speakers is below.

A highlight of the day was hearing from three patients:  young adults who are currently fighting DIPG and DMG (diffuse midline glioma).  Jace Ward, Anjalie Bartee, and Katie Bedingfield spoke with strength and passion about the reality for young adults and children fighting this difficult disease.  They emphasized the urgency of driving research forward and drawing more attention to the needs of this vulnerable population.

Jace Ward, Katie Bedingfield, and Anjalie Bartee

“I can’t promise I’ll be back here next year.  Which is exactly why I respectfully ask you to co-sponsor H. Res. 114 before you leave for the weekend. DIPG won’t wait until this is convenient. DIPG won’t wait until we are ready. While we have been ‘waiting’ to take a solid stand, DIPG has been taking the sight, the hearing, the speech, the ability to swallow and eventually the breath of thousands of kids across this country.” — Jace Ward, 21 years old, fighting DIPG since May 17, 2019.

Katie Gaskin, Stacey Sands, and Antonio Halek

A number of other families in the midst of the fight with their child made an incredible effort to take part in the event, including the Basha family from New York (with Elita in attendance), Halek family from Illinois (with Noah in attendance), and Stacey Sands from Kansas (in honor of Hudson).  And there were countless families who carry the heartbreak of losing a child who took part in memory of their precious children.  The power of standing together as a united community was palpable.  

Michael Mosier Defeat DIPG Foundation was proud to sponsor this event alongside other organizations:  The ChadTough Foundation, Dragon Master Foundation, Jack’s Angels Foundation, Julia Barbara Foundation, Smashing Walnuts Foundation, and Team Cozzi Foundation.  We thank Covington & Burling LLP for generously hosting the State of DIPG Summit and Akin Gump LLP for supporting the Congressional Briefing effort.  We appreciate the hospitality and great food from We, The Pizza, a restaurant owned by The Mendelsohn Family who are steadfast supporters of the fight to Defeat DIPG.

YOU CAN TAKE ACTION

There are two immediate action items where all can contribute to the coordinated efforts to establish May 17 as DIPG Awareness Day, as a catalyst for increased attention to this tragic disease.

Support H. Res. 114 – Federal Recognition

The DIPG Advocacy Group has detailed information on how you can engage with your Congressional representative to urge their participation as a co-sponsor for H. Res. 114.  Please visit the DIPG Advocacy Group website for instructions on how to contact the legislator who represents your district, either to request their support or to thank them for signing on to the resolution. 

Support “DIPG Across the Map” – State Level Recognition  

We call on everyone to use their stories and voice to help get all 50 states to recognize May 17 as DIPG Awareness Day.  To sign up for this project, please visit www.defeatdipg.org/dipgacrossthemap.  Michael Mosier Defeat DIPG Foundation organizes this project – a collaboration of foundations and individuals throughout the country – as a companion effort to our federal efforts.  We will provide step-by-step instructions and materials to assist in advocating within your state.  In 2019, 32 states recognized May 17 as DIPG Awareness Day.  We hope to increase that number in 2020, with the collective effort of the DIPG Community.

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Congressional Briefing Speakers

Janet Demeter (Jack’s Angels Foundation), Sabine Mueller (University of California San Francisco and [zurich]), Adam Resnick (Children’s Hospital of Philadelphia and Children’s Brain Tumor Tissue Consortium), Charles Keller (FILL IN), Malcolm Smith, David Arons (National Brain Tumor Society), Jace Ward (DIPG patient), Jenny Mosier (Michael Mosier Defeat DIPG Foundation), Elizabeth and William Psar (Julia Barbara Foundation, and Jill, Cam, and Phebe Morin (Luke’s Posse). Video available at this link.

State of DIPG Summit Speakers

Mark Mosier (Michael Mosier Defeat DIPG Foundation), Janet Demeter (Jack’s Angels Foundation), Elizabeth Psar (Julia Barbara Foundation), Paul Miller (advocate), Jenny Mosier (Michael Mosier Defeat DIPG Foundation), Sabine Mueller, MD, PhD (University of California San Francisco and [zurich]), Eugene Hwang, MD (Children’s National Hospital), Carl Koschmann, MD (University of Michigan), Paul Cozzi (Team Cozzi Foundation), Katie Gaskin (Anthony’s Avengers Defeat DIPG Foundation), Anjalie Bartee (patient), Katie Bedingfield (patient), Wolfgang Oster, MD, PhD (Oncoceutics), Amanda Haddock (Dragon Master Foundation), Adam Resnick (Children’s Hospital of Philadelphia and Children’s Brain Tumor Tissue Consortium), Gregory Reaman, MD (U.S. Food and Drug Administration), Jonathan Agin (Max Cure Foundation, Oncoheroes Biosciences), Danielle Leach (National Brain Tumor Society), and Jace Ward (patient). Video available at this link.

Defeat DIPG, ChadTough Award $2.8 Million for Ten New DIPG-Specific Research Projects

Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation are funding ten new Diffuse Intrinsic Pontine Glioma (DIPG)-specific research projects totaling more than $2.8 million over the next three years (2020-22).  Two of the new grants will be made in partnership with SoSo Strong Pediatric Brain Tumor Foundation.

To date, the Defeat DIPG ChadTough grant program has committed $6.1 million to 23 DIPG research projects.  In addition, Defeat DIPG and ChadTough have committed $600,000 to help fund an innovative collaboration between PNOC (Pacific Pediatric Neuro-Oncology Consortium) and the DIPG Centre of Expertise in Zurich, supporting preclinical and clinical work across multiple institutions.

“By working together, we are amplifying our capacity to make significant investments in DIPG-specific research,” says Defeat DIPG Executive Director Jenny Mosier. “It is the generosity, passion, and commitment of the Defeat DIPG and ChadTough supporter networks that propel our shared mission of finding a cure for this devastating disease.”  

The ten projects to be funded (listed below) include:  two research grants ($600,000 over three years), four new investigator grants ($250,000 over two years), and four fellowships ($150,000 over two years).

Jason Carr, President of The ChadTough Foundation, explains, “Our grant program is designed to push the field forward by investing in research that is likely to fuel progress and add to our understanding of this disease, while also ensuring we empower the next generation of researchers to bring this over the finish line for our children.  We hope for a cure in the near term, but we realize we need a pipeline of researchers who have the expertise and drive to follow through with this work as long as needed”

Defeat DIPG ChadTough Fellow Eshini Panditharatna,
Dana-Farber Cancer Institute
Photo by: Dana-Farber

All projects for the Defeat DIPG ChadTough grant program are reviewed by the Defeat DIPG Scientific Advisory Council, an unparalleled group of experts in pediatric brain cancer who evaluate each application for scientific merit.  “Our Scientific Advisory Council brings a breadth of experience and expertise that sets our grant program apart,” says Mark Mosier, Chair of the Defeat DIPG Board of Directors. “Their rigorous review of the many applications we receive ensures we are using donor funds as efficiently and effectively as possible.”

The Defeat DIPG Scientific Advisory Council is chaired by Suzanne Baker (St. Jude Children’s Research Hospital) and includes David Ashley (Duke University School of Medicine), 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 (University Children’s Hospital Zurich, Children’s National Medical Center).

The Defeat DIPG ChadTough grant program was structured with guidance from the Defeat DIPG Scientific Advisory Council to ensure the grant amount, duration, and criteria were shaped to achieve the most meaningful results to push the field forward.  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.  Researchers submit progress reports to ensure the studies are proceeding as anticipated.

Defeat DIPG and ChadTough bring together 11 families with children who have fought or are fighting DIPG that are actively raising research dollars.  

“We are grateful for the opportunity to work together with this incredible set of families who contribute so much,” says Tammi Carr, Co-Founder and Board Member of The ChadTough Foundation, “They have all been through such a difficult experience with their own child’s battle, yet they harness their grief and passion to make a difference for families who will face DIPG in the future.”

Michael Mosier Defeat DIPG Foundation is led by co-founders Mark and Jenny Mosier, and includes their Defeat DIPG Network chapters:  Katie Gaskin and Ruben Cardoza (Anthony’s Avengers Defeat DIPG Foundation (IL)), Amanda and Brandon Huffman (Avery Huffman Defeat DIPG Foundation (WA)), Alexisand Peter Olympia (Connor Man Defeat DIPG Foundation (TX)), and Katie and Simon Weaver (Vivian Rose Weaver Defeat DIPG Foundation (WA)).

The ChadTough Foundation is led by co-founders Jason and Tammi Carr, and includes partner families Gina Hatzivasilis and Sam Reinhold (Team Benjamin) Connie and James Jones (Team Carter), Jeff and Shannon DelVerne (Team Colt), Brad and Nettie Boivin (Team Julian), and Tom and Amanda Ruddy (Team Tommy).

SoSo Strong Pediatric Brain Tumor Foundation is a nonprofit established in honor of Sophia Ann Myers and is co-funding two of the ten grants awarded in this grant cycle.

Learn more about The ChadTough Foundation at chadtough.org and Michael Mosier Defeat DIPG Foundation at defeatdipg.org.

RESEARCH GRANTS

  • Hideho Okada, University of California San Francisco, “Next-generation CAR T cell therapies for treatment of DIPG, utilizing sequential “prime-and-kill” circuits to achieve safe and effective tumor targeting”
  • Daphne Haas-Kogan, Dana-Farber Cancer Institute, “Dependence of DIPGs on DNA polymerase q for DNA repair defines a new therapeutic target”

NEW INVESTIGATOR GRANTS

  • James Stafford, University of Vermont, “Onc201 in DIPG; establishing mechanism, enhancing efficacy and determining long-term phenotypic consequences.”
  • Zachary Reitman, Duke University, “Enhancing the efficacy of radiation therapy for DIPG”
  • Stephen Mack, Baylor College of Medicine, “Interrogating the Role of HERV Activation in H3K27M Pediatric Glioma.”
  • Matthew Dun, University of Newcastle (Australia), “Unlocking oncogene addition to identify synergistic treatment targets for the treatment of DIPG.”

FELLOWSHIPS

  • Eshini Panditharatna, Dana-Farber Cancer Institute, “Targeting epigenetically induced vulnerabilities in DIPG.”  (Mentor:  Mariella Filbin)
  • Chan Chung, University of Michigan, “Targeting DIPGs by interrupting metabolic pathways.”  (Mentor: Sriram Venneti)
  • Alan Jiao, Boston Children’s Hospital, “Dissecting mechanisms of H3K27M oncohistone function in DIPG)  (Mentor: Yang Shi)
  • Xu Zhang, Columbia University, “Mechanistic studies on the WNT5A signal pathway in DIPG tumor.”  (Mentor: Zhiguo Zhang)

Defeat DIPG, ChadTough Announce $3.4 Million in New DIPG Research Funding

Michael Mosier Defeat DIPG Foundation and The ChadTough Foundation, and their chapters and partner families, are thrilled to announce the funding of 11 new DIPG-specific research projects totaling $3.4 million over the next 3 years. This brings the total research dollars committed through the Defeat DIPG ChadTough partnership to more than $6.7 million in the past 3 years. Two of the new grants will be made in partnership with SoSo Strong Pediatric Brain Tumor Foundation.

The foundations will share more details on these new projects in the upcoming weeks. This round of funding includes $2.8 million through the Defeat DIPG ChadTough research program to fund: two research grants ($600,000 over three years), four new investigator grants ($250,000 over two years), and four fellowships ($150,000 over two years). All projects are reviewed by the Defeat DIPG Scientific Advisory Council, a preeminent group of experts in the field.

Defeat DIPG and ChadTough are also providing over $600,000 for an innovative new collaboration through PNOC (Pacific Pediatric Neuro-Oncology Consortium) and the DIPG Centre of Expertise in Zurich, to support preclinical and clinical work across multiple institutions to move combination therapies for DIPG into the clinic. 

We are pleased to make this announcement on behalf of all of our valued Defeat DIPG Network chapters: Anthony’s Avengers Defeat DIPG Foundation (IL), Avery Huffman Defeat DIPG Foundation (WA), Carson Hall Defeat DIPG Foundation (KS), Connor Man Defeat DIPG Foundation (TX), and Vivian Rose Weaver Defeat DIPG Foundation (WA).  We are also very grateful to work with the ChadTough partner families, Team Tommy (the Ruddy family), Team Juliam (the Boivin family), Team Colt (the DelVerne family) and Team Carter (the Jones family). 

We thank all of our supporters who make this important researching funding possible. It is because of your generous contributions that we are making progress towards finding a cure.

Defeat DIPG Raises Over $230,000 for DIPG Research on #GivingTuesday

Thanks to our amazing Defeat DIPG supporters, we raised over $230,000 (and counting) for DIPG research on #GivingTuesday. We started #GivingTuesday, a global day of giving on December 3, with a goal of raising at least $150,000 for DIPG research. In the afternoon, we were very grateful to get another $20,000 in matching dollars to increase our overall goal to $190,000.

But our amazing Defeat DIPG community was not done there, and we exceeded our goals!

Thank you to the Rives FamilyDARCARS Automotive GroupSoSo Strong Pediatric Brain Tumor FoundationTough Like Jack Foundation, and the William & Mildred Kaplan Charitable Foundation for committing matching funds to make our #Give2DefeatDIPG initiative possible. 

Thank you to all of the DIPG families and supporters who created fundraisers to support this effort. We had over 55 Facebook fundraisers created to support the #Give2DefeatDIPG initiative on Giving Tuesday, in honor of dozens of different children who have fought DIPG.

All members of Defeat DIPG® Network offer you our deepest appreciation for your support, not only on Giving Tuesday but all year long. You donate, volunteer, attend events, and so much more. It is all of these contributions combined that allows Defeat DIPG® Foundation to grow and meet our mission: funding research for a cure for the deadliest childhood brain cancer, DIPG.

We are not yet at the finish line, but we are getting there faster because of you. 

Together, we will Defeat DIPG.