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 learned 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 cancer dietitians 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 and his 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 preparing for a lot of ups and downs, as any difficult diagnosis causes the entire family to be on emotional rollercoaster of hope and disappointment. Different people deal with such news differently, so they urge families to just 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.


Laser Interstitial Thermal Therapy Shows Promise for Glioblastomas

Excerpt:

“Magnetic resonance imaging-guided laser interstitial thermal therapy (LITT) appears to be safe and effective for glioblastomas in select patients and may add an average of 2 months to life expectancy compared with the current standard of care, according to a new report published in the journal Neurosurgery.

” ‘We showed that the procedure is well tolerated and that recurrent patients had a meaningful clinical benefit that seems to be better when compared with previously published data on the current standard of care,’ said Eric Leuthardt, MD, senior study author and a professor of neurosurgery, neuroscience, biomedical engineering, and mechanical engineering & applied science at Washington University School of Medicine in St. Louis, Missouri.”

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.


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. Continue reading…


First Patient Dosed in Phase I/II Trial of Novel Immunotherapy Combination in Newly Diagnosed Glioblastoma

Excerpt:

“The first patient has been dosed in a phase I/II open-label, multicenter trial investigating a novel immunotherapy combination in patients with newly diagnosed glioblastoma (GBM). Fifty patients have been accrued in the trial, as of May 31, 2018, which will be conducted at 25 sites across the nation.

“This study aims to investigate the efficacy of INO-5401, a T-cell activating immunotherapy agent encoding multiple antigens in GBM, and INO-9012, an immune activator encoding IL-12, in combination with the PD-1 inhibitor cemiplimab (REGN2810).”

Go to full article.

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Doctors Try Genetically Modified Poliovirus As Experimental Brain Cancer Treatment

Excerpt:

“A genetically modified poliovirus may help some patients fight a deadly form of brain cancer, researchers report.

“The experimental treatment seems to have extended survival in a small group of patients with glioblastoma who faced a grim prognosis because standard treatments had failed, Duke University researchers say.

” ‘I’ve been doing this for 50 years and I’ve never seen results like this,’ says Dr. Darell Bigner, the director emeritus of the The Preston Robert Tisch Brain Tumor Center at the Duke Cancer Institute, who is helping develop the treatment.”

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New Approach to Treating Glioblastoma Could Add Years to Patients’ Lives

Excerpt:

A new way of treating glioblastoma — the deadly brain tumor currently ailing Arizona Sen. John McCain — with a personalized vaccine is giving some patients in a clinical trial more time.

“The vaccination, called DCVax-L, is made out of a patient’s own cells and uses them to jumpstart the immune system and attack the tumor. In the trial, some patients survived for more than 36 months — more than a year and a half longer than current life expectancy after glioblastoma diagnosis.”

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MimiVax Announces Positive Interim Results from Phase II Trial of SurVaxM Immunotherapy for the Treatment of Newly Diagnosed Glioblastoma

Excerpt:

“MimiVax LLC, a clinical-stage biotechnology company developing immunotherapeutics and targeted therapies for cancer treatment, today announced positive interim results from a multicenter Phase II study of SurVaxM in patients with newly diagnosed glioblastoma (nGBM). These promising interim results support further development of SurVaxM in combination with standard therapy as a potential treatment for glioblastoma. A randomized trial of SurVaxM in glioblastoma is planned, pending completion of this study and review at the end of Q4 2018.”

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Study Evaluating VBI-1901 Enrolling Recurrent GBM Patients in Second Dose Group

Excerpt:

“An Independent Data and Safety Monitoring Board (DSMB) unanimously recommended that the Phase 1/2 clinical trial evaluating VBI Vaccines’ VBI-1901 continue to enroll patients with recurrent glioblastoma (GBM) in a second study arm.

“The positive review recommended the study continue without modification, after scanning through all safety data from the first group of patients, who received the lowest VBI-1901 dose. Enrollment now has commenced for the second, intermediate-dose study arm.”

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.