June 7, 2019 | Mia Brozovich Nacke 

June 7, 2019 | Mia Brozovich Nacke

COMMUNITY OUTREACH, EVENTS, PEDIATRICS, RESEARCH AND INNOVATION.

Families who have lost children to Diffuse Intrinsic Pontine Glioma, a fatal brain tumor with a median survival of 9-10 months, met with Stanford pediatric neuro-oncologist Michelle Monje to learn about new clinical trials and express optimism that accelerated funding will yield treatment results.

DIPG families visit the Monje Lab. From left to right, Marisa Martinez, Xavia Martinez-Lusinchi, Kirsten Finley, Zach Major, Robby Larson, Keagan Finley and Janet Demeter.

On May 17, DIPG Awareness Day, four families who donated their late children's brain tumor tissue to science convened at Stanford to hear firsthand from pediatric neuro-oncologist Michelle Monje, MD, PhD, about research developments and new clinical trials that may hold the key to unlocking treatment for Diffuse Intrinsic Pontine Glioma (DIPG). 

DIPG, a highly aggressive tumor of the brain stem, is the leading cause of childhood brain tumor deaths. There is no cure for DIPG; radiation treatment can extend a child's life by mere months.

To help call awareness to DIPG-and learn more about groundbreaking research-congressional staff from Sacramento and Washington, DC, joined DIPG families in Stanford's Lorry I. Lokey Stem Cell Research Building for a presentation by Monje, followed by a tour of the Monje Lab.

During a Monje Lab tour, Michelle Monje, MD, PhD, center, talks with Brian Perkins, District Director, Office of Congresswoman Jackie Speier (second from left), and visiting families who are raising awareness and funds for DIPG.

Joining the roundtable were the offices of U.S. Representatives Jackie Speier and Anna Eshoo, California State Senators Jerry Hill and Scott Wilk, and California Assembly member Marc Berman.

Several of the families present have started foundations to support DIPG research and assist other families in navigating the brutal illness. They know all too well that a DIPG diagnosis is fatal.

Each year in the U.S., DIPG strikes 200-400 school-aged children. DIPG peaks at around age 7, but occurs in both younger and older children. According to Monje, we now know DIPG can also strike adults. With standard therapy, median survival is 9-10 months. Five-year survival rates are below 1 percent.

Discoveries drive human trials

Researchers at work in the Monje Lab, part of the Department of Neurology & Neurological Sciences at the Stanford University School of Medicine.

Identifying effective chemotherapy for DIPG has been challenging. Because the tumor grows in the brainstem, a region that controls essential body functions such as breathing and heartbeat, and because these cancer cells grow tangled with healthy cells, the tumor cannot be surgically removed. While other pediatric cancers have seen vast improvements in survival rates with new treatments, the survival rates for DIPG children have not improved in 40 years.

Monje, who has been a leader in DIPG research for the past 10 years, is hoping to change this. In 2009, Monje's research team was the first in the world to grow laboratory cultures of human DIPG cells from deceased donors. This gave scientists their first opportunity to study the cells' growth, as well as a platform to identify and test chemotherapy agents that fight DIPG. Monje's team also created mouse models of DIPG to allow detailed studies of the tumor's growth and to test possible treatments.

The team is currently running a Phase 1 clinical trial in children with DIPG of the drug panobionstat, which slows DIPG growth and increases survival in mice with the tumor. Recently, Monje and her colleagues demonstrated that engineered immune cells, called chimeric antigen receptor T cells or CAR-T cells, can eradicate several types of brain tumors in mice, including DIPG. The team is planning a human clinical trial for this treatment.

A horizon of hope

Speaking to the families who had traveled from as far away as Florida to raise awareness for a disease that strikes at the heart of childhood, Monje asked, "Why have we failed DIPG children for so long?

"DIPG is ‘the worst of the worst.' Historically, DIPG has been difficult to study. Eleven years ago, when I started studying this disease, there were no cell cultures, no experimental mouse model systems. Finally, we have the resources to begin to untangle the biology of this disease." 

Kirsten Finley, Marisa Martinez and her daughter Xavia, and Janet Demeter share a moment together during DIPG Awareness Day. 

"As we see in this room, that families of children affected by DIPG are extraordinarily giving. Families who have been affected by this disease have pushed the needle forward."
Michelle Monje, MD, PhD

The donations of DIPG families will help other generations of children, said Monje. Research has already shown that DIPG shares a mutation with other high-grade gliomas like spinal cord and thalamic gliomas, which has led to a reclassification of these cancers. These somewhat less common but still fatal diseases may benefit from DIPG research.

With the aim of giving DIPG children a longer period of good quality of life - and envisioning a cure within our lifetime - the Monje Lab openly shares tumor tissue resources that generate cell cultures with over 200 labs around the world.

Meet the Kids: Images of children lost to DIPG, including Kayne Finley (top left), Jack Demeter (top right) and Zamora Martinez-Lusinchi (middle row, second from right)

According to the National Pediatric Cancer Foundation, only 4% of federal government cancer research funding goes to study pediatric cancer. The Monje Lab benefits from diverse funding streams, including from the National Institutes of Health (NIH); DIPG families have also stepped up by raising research funds. But the funding gap for DIPG, and pediatric cancer research in general, remains significant.

Congresswomen Speier and Eshoo, longtime supporters of increased federal funding for childhood cancer research, both addressed letters to the families gathered at the event and the Packard Children's Hospital community. Noting that DIPG Awareness Day is intended to encourage research into DIPG and pediatric cancers in general, Speier praised the Monje Lab's groundbreaking work. 

Stanford pediatric neuro-oncologist Michelle Monje, MD, PhD

"Thanks to the work of Stanford researchers, including notably Dr. Michelle Monje and her team, there is reason for hope."
U.S Congressional Representative Jackie Speier

"What we need to do," Speier suggested, "is support NIH research into this important childhood disease, and to encourage the FDA to support the testing and ultimately the approval of needed treatments."

Eshoo encouraged the families to continue their advocacy, which she acknowledged is vitally important to children facing deadly diseases.  "Children are not just small adults," stated Eshoo, adding that drugs need to be studied specifically for their use in children.

The State Senate and Assembly offices of Jerry Hill and Mark Berman presented Monje and the Monje Lab with Certificates of Recognition to acknowledge the impact of the Monje team's research. 

Moonshot for kids

The families spoke candidly about how meaningful Monje's work is to their mission to encourage public engagement and support for DIPG research, trials and treatment. Having faced DIPG's devastating statistics head on, they agree that greater awareness and scaled-up funding would be game-changers.

"When my son Kayne was diagnosed," said Kirsten Finley, "it felt helpless, hopeless and like nothing you did would make a difference. Your leadership and commitment to saving these children, Dr. Monje, makes a huge case for more NIH funding. We have to attack and address it."

To draw attention to DIPG and raise research funds, Kayne's brother Keegan is bicycling cross-country with friends. "We all know that this disease is underfunded," he told the audience. Older than most DIPG patients when diagnosed at age 17, his brother became an advocate for brain cancer awareness while undergoing clinical trial treatments. Kayne used his 366 days after diagnosis to start college, speak to schools and businesses about DIPG, and meet with his state's governor to urge funding for childhood cancer. 

Marisa Martinez comforts her daughter Xavia, in front of a photo of her late daughter and Xavia's sister Zamora, who was diagnosed with DIPG at age 7.

Janet Demeter, who lost her 3-year-old son Jack to DIGP, hosts a talk radio show dedicated to childhood cancer and raises awareness of DIPG through her foundation. Demeter coined the phrase "Moonshot4Kids" to evoke the Cancer Moonshot initiative and illuminate how we must aim high to fast-track new therapies for children with cancer.

"Every child is a life, not a number. Someday in the future, the experience for families with a DIPG diagnosis for their child will be one of hope."
Janet Demeter, mother of DIPG child Jack

On May 23, the United States Senate passed S. Res. 223, a resolution expressing support for the designation of May 17, 2019 as "DIPG Pediatric Brain Cancer Awareness Day." On February 12, Rep. Jackie Speier and Rep. Dave Joyce introduced a similar bipartisan resolution, H. Res. 114, in the House of Representatives. H. Res. 114 currently has 42 cosponsors.

To learn more about how to raise awareness and funds for DIPG Research in your community, contact Lucile Packard Foundation for Children's Health: https://supportlpch.org.