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…


Promising Developments for Brain Tumor Drug ONC201

In September, we announced our collaboration with Musella FoundationxCures, and Oncoceutics to help patients access ONC201, a potential new 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.

Since then, several news stories have reported promising developments for ONC201. Check out the coverage:

  • The Philadelphia Inquirer: “Cancer therapy shows promise for some brain tumors”
  • Atrium Health: “Finding hope in the face of brain cancer: After being diagnosed with a brain tumor, Amanda’s future was uncertain. But participating in a Phase 2 clinical trial has given her more time, more hope and a new mission.” (Also covered on WSOC-TV)
  • Michigan Medicine: “Brain cancer patient finds hope with emerging new therapy”
  • Omaha World-Herald: “Experimental drug ‘nothing short of miraculous’ for father of 4 with super-rare brain tumor”

To learn more about our ONC201 project, click here.


Immune Cells Track Hard-To-Target Brain Tumours

Excerpt:

“Instructing the immune system to recognize and kill tumours, an approach termed cancer immunotherapy, has transformed the clinical treatment of certain types of malignancy. Prominent among these therapies are immune-checkpoint inhibitors, which block the action of proteins that dampen immune-cell responses against tumours. For example, antibodies can be used to interfere with the inhibitory protein PD-1, which is present on T cells, a type of immune cell that attacks tumours. Immune-checkpoint inhibitors have been most successfully used to treat cancers, such as melanomas, that are well infiltrated by T cells and have a large number of genetic mutationsA subset of these mutations might generate neoantigens — altered protein sequences that are uniquely produced in cancer cells and are recognized as foreign by the immune system.”

Go to full article.

If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


ASCO and Friends of Cancer Research Applaud NCI’s Expansion of Clinical Trial Eligibility Criteria

Excerpt:

“ASCO and Friends of Cancer Research (Friends) applaud the National Cancer Institute’s (NCI) recent revision of its clinical trial protocol template to broaden eligibility criteria for cancer clinical trials. The protocol template was expanded to help increase the opportunity for participation in NCI-funded clinical trials for patients with certain health-care conditions, as well as to provide an opportunity for patients younger than age 18 to participate in adult clinical trials in certain circumstances.”

Go to full article.

If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


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.”

Go to full article.

If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


The Promise of Plerixafor in Glioblastoma Treatment

A Q&A with Adan Rios, MD; Professor in the Division of Oncology-Department of Internal Medicine of The University of Texas McGovern Medical School at Houston, Texas Medical Center; adan.rios@uth.tmc.edu

Q: Glioblastoma multiforme (GBM) remains a scourge with a typically rapid fatal course resistant to most therapy. All solid tumors must receive sufficient blood supply to grow. Plerixafor is an FDA-approved drug that may inhibit tumor angiogenesis. How might plerixafor be sensibly used off-label as an adjunctive therapy for GBM?

A: Glioblastoma multiforme (GBM) is a CNS (central nervous system) tumor with post-therapy median time to progression of 7 months and median overall survival of 15 months. I decided to use plerixafor for the prevention of recurrence of GBM in one patient treated with standard chemo-radiotherapy five years ago and since then have studied this patient and this subject in depth. Continue reading…


Super ASK Caregiver: Sheena Advocates for the Best Possible Care for Her Mother’s Glioblastoma


In the summer of 2016, Sheena’s father called her from the emergency room. Her mother Shobha, a fitness trainer in the Bay Area, had experienced a seizure, and the doctors suspected a brain tumor. “I packed and moved back home from the east coast the next day,” Sheena says.

Shobha later had surgery to remove the tumor and received a diagnosis of glioblastoma (GBM). Her oncologist prescribed a standard GBM treatment of radiation and chemotherapy, but she had a bad reaction to the chemotherapy drug Temodar, and had to stop the medication.

“That threw us off completely because there are very few treatment options for GBM,” says Sheena, who has a master’s degree in public health and had previously worked in an oncology department. Shobha’s doctor advised that she wait and watch for tumor growth, but she and her family weren’t comfortable with inaction.

“I’ve had my own medical challenges and learned over the years how to advocate for myself,” Sheena says. “People assume that you should do whatever the doctor says, which is a good place to start, but I’ve found that you shouldn’t stop there.”

The family began looking into other treatment options for Shobha. “Every night, we researched clinical trials on ClinicalTrials.gov, and called the medical centers with trials in the morning,” Sheena says.

Meanwhile, they sought guidance from ASK Cancer Commons’ Emma Shtivelman and from previously featured Super Advocate Stephen Western. Emma and Stephen helped Shobha’s family sort through the many complex enrollment requirements for each trial, and narrow the options to trials with potentially more promising treatments.

“They were really our brainstorming partners through all of this because we didn’t know who else to talk to,” Sheena says. “It was a huge comfort to have them available as we navigated this new, scary territory.”

Sheena’s family with Dr. Kesari (center)

Shobha ended up receiving treatment with an immunotherapy drug and a device called Optune. She also tried a number of other therapies, and worked with oncology nutritionist Patrice Surley, to incorporate dietary changes and supplements. It wasn’t an easy transition, but improving the odds for her was the main focus.

Meanwhile, although she couldn’t do many of the exercises herself, she continued teaching fitness classes out of her home studio as she had for 15 years, while Sheena and her father worked from home.

“She would teach while sitting with her cane, and her clients said that her workouts got a lot more difficult now that she didn’t do the exercises with them,” Sheena says. “They love her. Working also focused her mind on something positive and off the difficult diagnosis and treatments.”

Compassionate Use

A year later, Shobha’s symptoms gradually returned, and her family pushed for an MRI, which revealed that the tumor had regrown. To their dismay, the size of the tumor disqualified her from participating in a particularly promising clinical trial that involved surgery plus injection of MDNA55, a toxin that targets glioblastoma cells.

At this point, Shobha had started travelling to Los Angeles regularly to receive second opinions from Santosh Kesari, MD, PhD, a neuro-oncologist recommended by glioblastoma survivor and patient advocate Greg Cantwell.

“Dr. Kesari really, really fought for my mother,” Sheena says. After battling paperwork, back-and-forth calls, and a rollercoaster of promise and disappointment, Dr. Kesari, her neurosurgeon Dr. Achrol, and their team were able to arrange for Shobha to receive the MDNA55 treatment through the U.S. Food and Drug Administration (FDA)’s compassionate use program.

The treatment worked well for Shobha for a year, though she experienced some side effects. She is currently undergoing treatment in Los Angeles for a different trial.

Lessons for Caregivers

Brain tumors are incredibly challenging on the patient and patient’s family due to any number of unexpected symptoms that can show up depending on the tumor location, and medication side effects, including physical limitations such as walking or using the bathroom to cognitive decline, memory and speech issues, vision issues, and extreme personality changes. For this reason, it is also one of the most financially difficult diseases on the patient and family. Professional caregivers can be hired but are usually not covered by insurance.

Through all of this, Sheena and her father have learned some key aspects of being caregivers. They recommend that families prepare for a lot of emotional ups and downs, which can come with any difficult diagnosis. Everyone deals with such news differently, so they urge families to try to be kind to one another as they adjust to the word “cancer.”

Dr. Kesari leads a Q&A at a Brain Tumor Awareness Tea
organized by Sheena and her family

From a practical standpoint, after every scan, Sheena suggests getting multiple copies of the cd so that it’s ready to quickly mail out to brain tumor centers, if needed. It’s helpful to keep an updated Word/Excel file of all the latest results, labs, scans etc., so that precious time isn’t wasted collecting that data, in case it is needed for clinical trials and new physician appointments. She also says she wishes she had registered her mother as a patient in more hospitals when she was feeling well, so that they could easily get appointments and more opinions later on when they needed them urgently.

For seeking out information on treatment and other aspects of the glioblastoma experience, Sheena recommends online support groups (such as those found at Inspire and Facebook), as well as services like ASK Cancer Commons and people like Stephen and Greg.

She suggests connecting with the hospital social worker early to help pursue options, such as disability benefits, paid family leave, unemployment benefits, or other government programs, should they be needed. On the same note, it may be helpful to connect with close family, friends, a pastor or priest, and other key people who can help. “Many people are very willing to help, they often just don’t know how,” Sheena says. “So, it’s okay to ask for specific help.”

Crucially, she emphasizes the importance of finding doctors who are a good fit. “It really has to feel like they’re on your team and you can be very open with them.”

And lastly, Sheena advises always advocating for the best possible care. “So often, we put the responsibility on the doctor and just assume that’s the way it is. But it really is your own responsibility,” she says. “It’s an attitude shift. We have to be proactive in the process, and always remember that there is hope.”

***

Super Patients are cancer survivors who learned to be more engaged in their own care. Cancer Commons believes every patient can be a Super Patient or benefit from a Super Caregiver or Super Advocate. We hope these stories will provide inspiration and hope for your or your loved one’s own treatment journey.


Options to Treat a Glioblastoma

A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY; email: musella@virtualtrials.com, phone: 888-295-4740

Q: You direct an established foundation that supports research and information about brain tumors. What would you do if you yourself were diagnosed with a glioblastoma multiforme (GBM)?

A: Now that GBMs are in the news again, I would like to discuss what I would do if it happened to me—a newly diagnosed GBM in an adult in otherwise good shape. There are several choices.

    1. Standard of care: Surgery, radiation, Temozolomide. Chance of 5 year survival is about 5%.
    2. Standard of care PLUS Optune. Bumps my chance of 5 year survival up to 24.9% (if used over 90% of the time) with no added toxicity.
    3. Phase 3 Clinical trials: There are now about nine phase 3 trials for newly diagnosed GBM. Some have impressive phase 1 and phase 2 data. By the time a treatment gets to phase 3, it has shown enough promise in earlier trials that the sponsor is willing to risk a lot of money to test in a phase 3 trial. Most have two big downsides: 1) Most have a control group of patients who receive the old standard of care so that some of the participants do not get the experimental treatment. 2) Most do not allow you to use Optune, so you are trading a known benefit for a chance at an unknown benefit.
    4. Phase 1 or 2 trials: There are about 75 of these trials in the USA. There are many interesting choices here, but we do not have enough data to make an informed decision on which one to try. We do have early results from some phase 1 trials, which are much better than those seen with standard therapies, but it is not likely that any one of these alone will make a big difference in survival for most patients. We do not (under the current system) have the ongoing results of these trials—we only get the results a few months after the trial is over. And while inside the trial, we cannot combine them with other treatments.
    5. Off label use of drugs approved for other diseases. There are many choices here and a rational approach might be to select a “cocktail of drugs” based on a genomic analysis of my tumor.
    6. Cocktail approach involving experimental and approved treatments. Right now, this is impossible or very difficult to obtain. However, if it were possible, this would be my approach. Especially if we had a registry of all of the patients, the treatments tried, and the outcomes so we can learn from every patient.

Continue reading…


Patient Foundation Collaboration to Advance ONC201 in DIPG and other H3 K27M-mutant Gliomas

Musella Foundation, Cancer Commons, xCures and Oncoceutics announced the initiation of a collaboration to develop ONC201 as a new treatment for diffuse intrinsic pontine glioma (DIPG) and other H3 K27M-mutant gliomas.

Critical to the collaboration is $1M in funding from The Musella Foundation, Michael Mosier Defeat DIPG Foundation and The Cure Starts Now Foundation. Cancer Commons and xCures will contribute additional resources to the success of the collaboration. “The Musella Foundation, Michael Mosier Defeat DIPG Foundation and The Cures Starts Now Foundation are excited to partner with Oncoceutics to translate a new molecularly-targeted therapeutic concept,” said Al Musella, DPM, President and Founder of the Musella Foundation.

Due to the tumor location within the midline region of the brain, as well as patients with a specific mutation called H3 K27M, patients often have significant neurological symptoms from their disease. There are no proven therapeutic options other than palliative radiotherapy. However, emerging clinical results have shown that some patients with H3 K27M-mutant glioma treated with single agent ONC201 have had their tumor stabilize or shrink and have had improvements in neurological symptoms, such as paralyses of peripheral and cranial nerves. Continue reading…