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.”

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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…


NCCN Guidelines Recommend Optune in Combination with Temozolomide as a Category 1 Treatment for Newly Diagnosed Glioblastoma

Excerpt:

“NovoCure (NASDAQ: NVCR) announced today that the National Comprehensive Cancer Network (NCCN) has updated its clinical practice guidelines to recommend Optune® in combination with temozolomide as a category 1 treatment for newly diagnosed glioblastoma (GBM) in its globally recognized Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Central Nervous System Cancers.

“NCCN panel members designated alternating electric field therapy, or Optune, as a Category 1 treatment recommendation for patients with newly diagnosed GBM in conjunction with temozolomide after maximal safe resection and completion of radiation therapy in patients with newly diagnosed GBM.”

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Tumor-Treating Fields for Glioblastoma Do Not Negatively Impact Quality of Life

Excerpt:

“The addition of tumor-treated fields to standard therapy with temozolomide prolonged deterioration-free survival without negatively influencing health-related quality of life among patients with glioblastoma, according to a secondary analysis of a phase 3 clinical trial published in JAMA Oncology.

“However, tumor-treating fields, or TTFields (Optune, Novocure) — alternating electrical fields delivered via four transducer arrays at an intermediate frequency of 200 MHz (1-3 V/cm) placed on the shaved scalp of patients and connected to a portable medical device — also caused skin irritation in more than half of patients.”

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Super Patient and Storyteller Adam Hayden Takes an Active Approach to Glioblastoma Treatment


On the day after Christmas of 2014, Adam Hayden experienced his first strange “episode.”

“We had just carried all the presents upstairs, and as I was walking towards our bedroom I was struck by this strange lightheadedness that I hadn’t experienced before,” he says. “I didn’t collapse, I didn’t pass out, I just kind of folded down to the ground.”

Adam and his wife Whitney, parents of three young children, chalked up the incident to holiday stress. But over the next year and a half, he kept having similar inexplicable episodes. Finally, in May of 2016, an MRI scan revealed a 71-milimeter tumor in the parietal lobe of Adam’s brain. The episodes had been seizures—symptoms of the tumor.

“When you have an MRI scan like that, things start to move really quickly,” Adam says. A few days after the scan, he was in the operating room to remove the tumor. The impact of the surgery on his brain left him in a wheelchair, and he needed intensive physical therapy to restore his ability to walk.

Meanwhile, Adam’s tumor tissue was analyzed, and on June 10, 2016, he received a diagnosis of glioblastoma. He began a standard treatment protocol of initial chemotherapy and radiotherapy followed by a long-term, 11-cycle chemotherapy regimen to keep the tumor from growing back.

“My oncologist said we could go to 12 cycles of maintenance chemo, but there has not been a randomized controlled trial validating that more is better, so taking the side effects into account, my wife and I decided to stop at 11 cycles,” Adam says. “That was in July, and I have not been on any active treatment since then.”

In late 2016, Adam’s oncologist suggested that he supplement his standard treatment with Optune, a device that had recently been approved by the U.S. Food and Drug Administration. Optune is a portable device worn on the head that produces an electrical field intended to disrupt cell division, thereby preventing tumor growth.

Adam says that his experience with Optune’s manufacturers was “terrific,” and he used the device for over two months. Ultimately, however, he and his wife decided it wasn’t for them.

“What the clinical trials show is that Optune provides a median of five months’ extra survival, sometimes much more and sometimes less, depending on the patient,” Adam says. “But the kids were afraid of how it looked, it was cumbersome to handle, and it was just an obstacle to daily living, so after hours and hours of talking about it, we made the decision to discontinue.”

Writing as medicine

As a graduate-trained philosopher with a penchant for writing, Adam was compelled to document his glioblastoma experience on a blog he named Glioblastology. He credits his father and grandfather, both pastors, with instilling him with a knack for storytelling.

“My grandfather was a loud, larger-than-life figure with a belly laugh, and storytelling was commonplace around the dinner table any time we visited,” Adam says. So when Adam received his glioblastoma diagnosis, he says, it felt natural to share his story online.

“People say, ‘it’s really courageous of you to live your journey publicly,’ but I was already living on social media, and it would have been stranger if I dropped off the map.”

Adam started writing immediately. “The day after my surgery, as soon as I could lift my head off the pillow, I asked my wife, ‘where’s my journal?’ ” he says. “It was therapeutic for me to write and tell my story.”

But Adam worried that he’d lose his readers’ interest if he just told his story over and over again. So he began to use his blog to put his story into a different context. “I began to write with the goal of teaching clinicians, patients, and others how to find value in their experiences.”

After publishing many posts, Adam was thrilled to discover that what he was doing on his blog—and what so many other cancer patients do—had a name: narrative medicine. In this approach, narrative structure is applied to patients’ experiences with illness and treatment in an effort to enhance the healing process.

Adam became especially interested in the works of narrative medicine practitioner Rita Charon, MD, at Columbia University. After studying Charon’s work, Adam was honored to present at a medical ethics conference, with Charon in the audience. An example of Adam’s take on narrative medicine is featured in his guest post at the science communication blog, PLOS SciComm.

Adam is actively involved in brain tumor and narrative medicine communities, including the National Brain Tumor Society, and he has shared his perspective in many presentations and publications. Recently, he wrote an open letter to Senator John McCain after news broke of McCain’s own glioblastoma diagnosis.

Over the course of his treatment, Adam has also found valuable support in the #BTSM (Brain Tumor Social Media) community on Twitter, which was co-created by fellow brain tumor patients Liz Salmi and Charlie Blotner. Community members interact with each other regularly by tagging their tweets with “#BTSM” and by participating in live monthly tweet chats, which Adam now co-moderates.

“If there’s something that you’re wondering about or that you think others in the brain tumor space could learn from, you can tweet it and tag it with #BTSM, and you’ll get thoughts and support from the community,” Adam says.

Thinking and talking about brain cancer

Adam says that his background in philosophy has helped shape his approach to glioblastoma treatment. As a graduate student studying the philosophy of science, he developed an acute sense of the limits of human knowledge and understanding.

“I was equipped with this humble way of viewing scientific theories that taught me to ask questions and never be too confident in the answers, knowing that throughout history, our theories have been evolving,” Adam says.

“So when I received my diagnosis, I was able to push past the doom of wondering what would happen to me. I realized that I didn’t have to be defined by whatever I was reading about the latest glioblastoma research since it is always transforming, and I could focus on how I would approach it.”

Adam has also taken a thoughtful approach to discussing brain cancer with his kids. He has found ways to frame his disease using words and ideas his kids can understand, instead of avoiding the topic altogether.

For instance, when Adam was recovering from his surgery, his five-year-old son expressed his concern about his dad’s wellbeing. Off the top of his head, Adam came up with the idea to compare his surgical incision and staples to a knee-scrape—a concept familiar to most kids.

“We talked about what happens when you fall and skin your knee; you put a Band-Aid on it and it scabs, and after a while the scab falls off,” Adam says. “So I told him, ‘that’s going to happen with daddy’s head.’ ”

Adam says he would advise other parents with cancer to be honest and frame things in terms of their kids’ level of understanding.

The “invisibility” of brain cancer

Despite his advanced diagnosis, Adam has not experienced many of the symptoms commonly associated with glioblastoma.

“So many people in the brain tumor community have suffered language processing issues, cognitive impairment, and more as a result of either the tumor or the treatment,” he says. “Fortunately I have tolerated treatment well, and while I do have some motor and sensory symptoms, I’m just about as sharp as I’ve always been.” Adam and his wife have connected with a palliative care doctor to proactively prepare themselves for cognitive decline, a near universal symptom of disease progression for people living with a malignant brain tumor.

The seizures that portended his diagnosis still play a big role in his life. Specifically, Adam regularly experiences focal seizures, in which he gets lightheaded and dizzy with tingling, numbness, and weakness on the left side of his body. The seizures are more likely to occur if he has been working hard, such as by writing.

“I can feel the onset of a seizure pretty well,” Adam says. “I know what my triggers are, and I remind myself to schedule my days around that.” Medication is also helpful in preventing seizures, but Adam notes that many fellow brain tumor patients find these treatments prohibitively expensive.

With his symptoms well-controlled, Adam is often reminded that people tend to assume that everyone they meet is healthy and able-bodied, unless they see an obvious sign otherwise.

“Most people’s picture of a cancer patient is someone who is sickly, thin, and nauseated,” Adam says. “What I try to highlight often in my presentations is that with brain cancer, we may look pretty good on the surface, even though we’re sick. We need to be cognizant of people’s visible disabilities, but also invisible disabilities that don’t always present themselves as obviously.”

Read Adam’s writings and learn more about his activities at his blog, Glioblastology. You can also follow him on Twitter and follow his wife’s writings on her blog, Faith, Hope, and Wine.

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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.


Combination of Optune® with Temozolomide Demonstrates Unprecedented Five-Year Survival for Newly Diagnosed Glioblastoma Patients

Excerpt:

“Novocure (NVCR) announced today results from its phase 3 pivotal EF-14 trial adding Optune to temozolomide for the treatment of newly diagnosed glioblastoma (GBM), including results from health-related quality of life analyses, were presented at the American Society for Radiation Oncology’s (ASTRO) 2017 Annual Meeting in San Diego. This marks the first presentation of EF-14 five-year survival and quality of life data at a radiation oncology conference.

“A late-breaking oral presentation focused on Novocure’s EF-14 phase 3 pivotal trial, which demonstrated unprecedented five-year survival results in newly diagnosed GBM. Patients treated with Optune in combination with temozolomide experienced a significant extension of overall survival without added toxicity compared to patients treated with temozolomide alone. The data also showed that Optune-treated patients were able to maintain quality of life for longer compared to patients treated with temozolomide alone.”

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Proposed Phase II Trial Aims to Explore Optune System in Grade III Recurrent Glioma

Excerpt:

“Researchers are hoping that a proposed phase II study exploring use of the Optune system in patients with recurrent grade III malignant glioma will expand the indications for the tumor treating fields (TTFields) device beyond its current FDA-approved use in recurrent grade IV glioblastoma.

“Daniel O’Connell, MD, a neuro-oncologist at UCLA’s Geffen School of Medicine, discussed his proposal with Targeted Oncology at the 21st Annual Scientific Meeting of the Society of Neuro-Oncology (SNO) held November 17 to 20, 2016 in Scottsdale, Arizona.”

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