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.