Case Report in the Journal of Neurosurgery Highlights Potential of ONC201 in H3 K27M-mutant DIPG

Last fall, we announced our collaboration with Musella FoundationxCuresThe Cure Starts Now FoundationMichael Mosier Defeat DIPG Foundationand Oncoceutics to help patients access ONC201, a new, experimental treatment for a type of brain tumor known as diffuse intrinsic pontine glioma (DIPG), as well as other gliomas with a genetic mutation known as H3 K27M. Oncoceutics now reports the publication of a case study of a 10-year-old patient for whom ONC201 provided noteworthy results:

Philadelphia, PA (April 9, 2019) – Oncoceutics announced today the publication of an article entitled “First clinical experience with DRD2/3 antagonist ONC201 in H3 K27M–mutant pediatric diffuse intrinsic pontine glioma: a case report” in the Journal of Neurosurgery (authored by Matthew D. Hall, M.D., MBA). The article summarizes the medical history of a 10-year-old girl with a diffuse intrinsic pontine glioma (DIPG) brain tumor. Following radiation therapy and treatment with ONC201 on a compassionate use basis, she developed near complete resolution of her presenting neurological symptoms for almost one year, enabling her return to school and participation in many normal activities. Continue reading…


Researchers Tap Genomic Technology to Develop Personalized Treatments for Pediatric Glioma

Excerpt:

“Researchers at UCSF Benioff Children’s Hospitals are using next-generation genomic technology to develop targeted therapies for high-grade pediatric glioma.

“Sabine Mueller, MD, PhD, adjunct associate professor of neurology, pediatrics and neurosurgery at University of California, San Francisco, and colleagues aim to treat as many as 44 children and young adults with this disease.”

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Liquid Biopsy Shows Promise for Monitoring Childhood Brain Tumors

Excerpt:

“New research by investigators at the University of California, San Francisco and the Children’s National Health System, has provided early evidence that liquid biopsy testing could help doctors monitor how well treatments are working in kids with diffuse midline gliomas.

“Brain cancers present a challenge for longitudinal monitoring, because obtaining repeat biopsy samples is dangerous and difficult. But liquid biopsy techniques have now opened the possibility of tracking these and other tumors over time based on analysis of tumor genetic material that is shed into the blood or other body fluids.”

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Monoclonal Antibody Treatment in Newly Diagnosed Pediatric High-Grade Glioma

Excerpt:

“In a phase II study reported in the Journal of Clinical Oncology, Grill et al found that the addition of bevacizumab (Avastin) to radiotherapy plus temozolomide (RT + TMZ) did not improve event-free survival in pediatric patients with newly diagnosed high-grade glioma.

“In the international open-label study, 121 patients aged 3 to 18 years with localized nonbrainstem disease from 51 sites in 14 countries were randomized between October 2011 and February 2015 to receive RT + TMZ with (n = 62) or without (n = 59) bevacizumab 10 mg/kg every 2 weeks. RT + TMZ consisted of RT at 1.8 Gy 5 days per week and TMZ at 75 mg/m2 per day for 6 weeks followed by a 4 weeks off, then up to twelve 28-day cycles at 150 mg/m2 per day on days 1 to 5 in cycle 1 and 200 mg/m2 per day on days 1 to 5 in cycles 2 to 12. The primary endpoint was event-free survival on blinded central radiology review. Results are reported as of 12 months after the enrollment of the last patient.”

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Study Splits Incurable Childhood Brain Tumors Into 10 New Diseases

Excerpt:

“Scientists have found that deadly childhood brain tumours are actually 10 different diseases that should each be diagnosed and treated based on their specific genetic faults.

“The major new study has important implications for treatment, since personalising care for each type of   is likely to be much more effective than grouping them all together as one.

“A team at The Institute of Cancer Research, London, found stark differences among ‘s ‘high grade’ , or gliomas, and that they could be split into at least 10 different cancers.”

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Potential Treatment for Brain Cancer as Drug Shrinks Tumours

Excerpt:

“An international team of researchers has found a drug previously approved to treat breast cancer could also be used to shrink medulloblastoma, a common form of childhood brain tumour.

“The discovery, made by The University of Queensland’s Institute for Molecular Bioscience and the Fred Hutchinson Cancer Research Center in Seattle, has led to a clinical trial using the drug palbociclib to treat children with medulloblastoma, the most common malignant brain tumour found in children.”

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Researchers Assess Pinworm Medication for Adults, Children With Glioblastoma

Excerpt:

“Researchers at Johns Hopkins University are conducting the first clinical trials to evaluate the potential of a pinworm medication for the treatment of children and adults with newly diagnosed glioblastoma.

“Mebendazole has been used for more than 40 years to treat parasitic infections.

“Although the medication requires further testing in patients with cancer, results of a phase 1 trial have shown the medication is safe for and tolerated by adults with glioblastoma. An additional phase 1 trial is underway to assess the agent in children.”

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New Insight Into Life-Threatening Childhood Brain Cancer

Excerpt:

“The most common type of malignant childhood brain cancer has been identified as seven separate conditions each needing a different treatment, new research has revealed.

“A study by Newcastle and Northumbria universities has found that childhood medulloblastoma can be separated into seven different subgroups which all have their own biological and clinical characteristics.”

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Further Reductions in Radiotherapy to Young Children With Brain Tumors Less Successful

Excerpt:

“A team of investigators has determined that young children participating in a clinical trial to assess the effectiveness of reduced radiotherapy did worse when there were deviations from the treatment protocol. Results of the study will be available online in advance of publication by Pediatric Blood & Cancer on April 4.

” ‘This study shows that attention to the timing, dose, and location of radiation therapy is crucial,’ Kenneth K. Wong, MD, a radiation oncologist at Children’s Hospital Los Angeles and first author on the study.

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