New hope for pediatric cancer treatment

Cassidy still remembers the agony of watching her son, Austin, undergo cancer treatment. Diagnosed with acute lymphoblastic leukemia at age 5, Austin endured three and a half years of radiation and chemotherapy at Children’s Hospital Colorado. He felt so sick and exhausted that he missed most of his kindergarten year. He spent multiple birthdays and holidays in the hospital, and he lost his hair twice.  

The intensive treatment regimen was successful in sending the young boy’s cancer into remission, but the journey to healing was agonizing. While Cassidy is very grateful for the expert care her family received at Children’s Colorado, she wishes there could be an easier way.

“I don’t think there’s anything worse than seeing your child in pain,” said Cassidy. “The things that cured him also made him so sick.”

Today, there is new hope.

Physician-scientists at Children’s Colorado are working tirelessly to find more effective, less invasive treatments for pediatric cancer, and they are gaining ground by using an unexpected weapon: the body’s own immune system. With sufficient funding, researchers believe they can advance new therapies that turn kids’ bodies into cancer-fighting machines.

Cancer’s worst enemy

Dr. Michael Verneris is a pediatric hematologist/oncologist and bone marrow transplant expert at Children’s Colorado. He’s also one of the world’s leading researchers in immunotherapy, a promising new treatment approach that harnesses the body’s immune system to fight cancer and other diseases. Immunotherapy has the potential to drive radical progress in treating pediatric cancer — and that could mean a future in which kids like Austin are cured with far fewer side effects.

From the beginning of his career, Dr. Verneris was keenly focused on finding ways to leverage the immune system and human stem cells to defeat cancer. “It’s what I have thought about every day of my life for the last 25 years,” said Dr. Verneris, who is the Barton Family Chair in Bone Marrow Transplant.

Back then, immunotherapy was largely dismissed as an implausible treatment that wouldn’t work outside the lab. Today, thanks to the pioneering research of Dr. Verneris and others, it’s being hailed as a breakthrough treatment that’s transforming the field of medicine as we know it.

Dr. Verneris was the first to discover how to reduce the recurrence of leukemia after bone marrow transplant by using stem cell therapy.  Now he and his team are working to find new ways to train the body’s immune system to hunt down and kill cancer cells – and his discoveries are already saving lives.

Supercharged T-cells

Much of Dr. Verneris’ current research focuses an emerging immunotherapy treatment known as CAR T-cell therapy. This pioneering therapy involves removing the immune cells from a patient’s blood – specifically the T-cells – and then genetically modifying them in a laboratory so that they are supercharged to seek out and destroy cancer cells. By adding a special receptor that binds to cancer cells, known as a chimeric antigen receptor (CAR), the reinfused T-cells can lock onto the cancer cells like a missile.

Approved by the Food and Drug Administration (FDA) for pediatric use in 2017, the first commercially available CAR T-cell therapy achieved an astonishing 80-90% remission rate in relapsed leukemia patients after just four months of treatment. The response rate was remarkable, given the critical state of the patients who participated in the initial trials.

“These patients hadn’t responded to any other therapies,” said Dr. Verneris. “They literally had an expected lifespan of weeks or months going into the trial, so those remission results were extraordinary.”

Moreover, patients in the trial were able to avoid many of the side effects of chemo, which Dr. Verneris says could be a game-changer for patients in the future.

“When a child is diagnosed with leukemia, they are looking at a treatment journey of two to three years,” said Dr. Verneris. “The majority of patients can be cured, but it comes at come cost – both in terms of exposure to a lot of drugs and pain, and the personal cost of turning a family’s life upside down. It’s frankly devastating. Immunotherapy could change that.” 

The Children’s Colorado Center for Cancer and Blood Disorders is one of very few hospitals that currently has CAR T-cell therapy available as a treatment option. And if you ask Dr. Verneris, he’ll tell you that we’re only just beginning to understand the many potential benefits of immunotherapy.

Boundless possibilities

With the help of philanthropy, the cellular therapy team at Children’s Colorado is working on additional treatments that improve cancer remission rates. About half of the children on the initial CAR T-cell trials relapsed within a year, so now Dr. Verneris is working on new cellular therapies that target different leukemia mutations, as well as solid tumors and brain tumors. By expanding treatment options, Dr. Verneris and his research team on the Anschutz Medical Campus hope to improve cure rates for even the most relentless of cancers.

His research lab is also studying the benefits of using CAR T-cell therapy at the outset of treatment, as opposed to waiting until the patient has exhausted all other options. A randomized clinical trial recently launched at Children’s Colorado and other sites is studying whether infusing CAR T-cells early on can improve outcomes in cancer patients with a high risk of relapsing. 

“We envision a world where kids get diagnosed, they get some induction chemo, then they go right to CAR T-cell therapy,” said Dr. Verneris. “We believe many of these patients will have a positive benefit and may not need additional therapy. That’s what this trial will help to determine.”

Dr. Verneris says that cancer is just one of many potential applications of CAR T-cell therapy. His lab regularly collaborates with other research teams to build on his team’s success and support new immunotherapy treatments for blood disorders, autoimmune conditions and even organ transplants. The possibilities seem as endless as Dr. Verneris’ dedication to finding new cures.

“The excitement in this field is just boundless at this point,” said Dr. Verneris. “It’s a really exciting time to dream about what’s possible and how we can make this therapy even better.”