Expanded Access Program for brain tumor patients – thinking outside the box
October 4, 2021
Erin Anderson was diagnosed with glioblastoma in late 2018. Since then, she’s had eight brain surgeries, and gone through rounds of radiation and chemotherapy. “This cancer is unique,” she said. “It’s aggressive and likes to come back. What we’ve learned is that even though our plans change in ways we don’t want them to, there is just so much hope out there. There are medical professionals who are committed to helping us and there are technological and medical advances that people didn’t have 5 or 10 years ago – or even months ago.”
The medical professionals dedicated to helping Erin include neuro-oncologist Elizabeth Neil, MD, Department of Neurology, and Neurosurgery Department Head Clark C. Chen, MD, PhD. They have thrown everything in their arsenals against Erin’s cancer, including:
- Performing minimally invasive laser ablation procedures that helped Chen destroy tumor tissues without using large incisions
- Using 5ALA (5-aminolevulinic acid), which makes glioblastoma tumor cells bright red, enabling Chen to remove the entire tumor, and
- Having Erin wear the Optune® device, which creates electric fields that disrupt cancer growth.
Thinking outside the box
When her tumor recurred after her last surgery, Chen (pictured at left) applied for the use of an experimental drug for Erin through the U.S. Federal Drug Administration’s (FDA) Expanded Access Program (EAP). “Erin had gone through so many different treatments that we had to think outside of the ‘box’ of available trial and treatment options,” he said.EAP, also known as compassionate use, enables patients to access treatment options before they receive full FDA approval. In this context, the benefits and side effects of these treatments are not fully investigated. To ensure patient safety, each EAP request must undergo multiple rounds of independent review, including by the sponsoring company, the FDA, and the University of Minnesota Institutional Review Board. “To make sure all brain tumor patients treated at the University have access to EAP, M Health Fairview devotes dedicated funds and personnel to support the process,” said Chen.
The approved EAP gave Erin an opportunity to be treated with a virus engineered to express a cytokine called interleukin-12. “In mouse models, this virus effectively cures glioblastoma,” said Chen. “The University of Minnesota enrolled patients in the phase 2 clinical trial for this virus. I saw impressive results for some of those participants, which is why I applied for EAP access for Erin.”
Complexity and uncertainty
Chen performed a surgery and injected the engineered virus directly into Erin’s tumor. “Following that, I get an infusion of an immunotherapy called OPDIVO® every two weeks,” said Erin. “I will keep doing that as long as it’s working and as long as it’s easy for me to tolerate.” OPDIVO helps T-cells activated by the injected virus seek out and kill cancer cells. “Through this combination, we are educating immune cells to fight off cancer cells, in ways similar to how vaccines educate our body to fend off the flu,” explained Chen.
MRIs taken after the treatment showed no evidence of glioblastoma recurrence for the first time in a long time.
Erin was tolerating the new treatment well when she noted some drainage from her surgical incision. Because she is receiving a therapy that is not fully studied, there was a great deal of complexity and uncertainty in the interpretation of her laboratory results after she noticed the drainage. Chen reviewed each of the clinical decisions with the sponsoring company and reported them to the FDA. Ultimately, he performed surgeries to explore the site of the virus injection. “During these procedures, I saw pools of liquified, dead tumor cells where the tumor used to be,” Chen said. “I have not seen this phenomenon in other glioblastoma patients.”
“You have reasons to be hopeful.”
Because of the complexities associated with her treatment, Erin was hospitalized for more than a month after she noticed the wound drainage. Her stay was during Covid containment measures, which made it especially upsetting because it took her away from her girls, Clara (5), and Julia, (3). Thanks to the efforts of Chen, her care team, and the hospital staff, Erin was able to visit with her girls outside the hospital. “It was fun to see the kids smiling and laughing,” she said. “It lifted my spirits and made me feel not so sad about being away from them. You can’t give a hug over Facetime.”
Jarett, Erin’s husband, texted the photo of the three of them to Chen that day. “I cannot begin to express what it meant to me to see Erin’s smile as she embraced her two adorable daughters,” he said.
Coping with it all
Since her diagnosis, Erin copes with what she’s been through by going to support groups, writing daily journals for her girls, attending church, and sometimes, “just crying it out.” In addition, Jarett does a lot of the research that helps inform Erin’s healthcare decisions. “When we face these scary things like recurrences, we know what our best bets might be,” she said.
What Erin reminds herself about during the scary moments of recurrence – and encourages others who are facing something similar – is to keep your hope up because there are still options. “And yes, maybe the dreadful news will come that you’ve exhausted all the options but, in the meantime, you have a lot in your arsenal,” she said. “And we have intelligent people like Dr. Chen and Dr. Neil who are committed to finding new solutions for their patients and being at the forefront of glioblastoma research. You have reasons to be hopeful.”