The Latest in CAR-T Cell Therapy for Pediatric Cancer

Although significant strides have been made in the treatment of children with cancer over the years, survival for some cancers, such as solid tumors, and rare, aggressive cancers, have seen little improvement. Those who do survive pediatric cancers, are often faced with long-term health complications from the current standard of care treatments of chemotherapy, surgery and radiation therapy.1 For children who relapse, survival rate for some cancers is unacceptably low – near 0% – with few second-line treatments available.

But, a promising new cancer treatment that harnesses the power of the immune system may be a game changer.

Chimeric antigen receptor (CAR) T cell therapy is a way of taking the body’s own immune cells, which fight infection, and reprogramming them in a lab to find and kill cancer cells. 

Today, CAR-T cell therapy is approved and being used in pediatric oncology for children as well as adolescents and young adults (AYA) with blood cancers, such as lymphoma and certain types of leukemia, often resulting in full remission.2 Researchers are currently working on extending this therapy to improve outcomes for many high-risk pediatric solid tumors.

How Does CAR-T Cell Therapy Work?

A diagram showing the process of car-t cell therapy

CAR-T cell therapy is a type of immunotherapy using cutting-edge cell engineering to enable the body’s own immune system to heal itself. 

The process begins with removing types of white blood cells, called T cells, from the patient’s blood. T cells kill diseased cells by using the protein receptors on their surface to latch onto antigens, which are protein fragments found on the surface of all cells. When a T cell latches onto an abnormal cell, it turns “on” and destroys that cell while minimizing damage to surrounding healthy cells.

But, cancerous cells often look like normal cells. To combat their deception, scientists use CAR-T cell therapy, to attack specific cancer cells by inserting instructions in the patient’s T cells to recognize and latch onto the diseased cell. 

Once reprogrammed, the T cells have special receptors called chimeric antigen receptors (CARs) on their surface. Then, these newly engineered CAR-T cells are grown to multiply in a lab. Afterwards, millions of them are returned into the patient’s bloodstream, where the new receptors allow CAR-T cells to seek out and latch on to a specific antigen on the patient’s tumor cells and destroy them.3

Recent Breakthroughs in CAR-T Cell Treatment

Over ten years ago, 6-year-old Emily Whitehead became the first pediatric patient to receive CAR-T cell therapy.5 The treatment was an unprecedented success: the CAR-T cells completely eradicated her acute lymphoblastic leukemia and put her into full remission. 

Five years after Emily was treated, the U.S. Food and Drug Administration (FDA) approved the CAR-T cell therapy product used to treat Emily, ushering in a new era in the treatment of cancer. Since then, a total of six CAR-T cell therapies were also approved. 

With every patient treated using this novel therapy for cancer, along with continued innovation in CAR-T cell design, exciting new developments in CAR-T cell treatments are leading to more hope for pediatric cancer patients and their families.

Here are the latest breakthroughs showing how CAR-T cells are paving the way to become the next great advancement in cancer treatment:



Continuing Impressive Results from New Cancer Treatment for Blood Cancers

CAR-T cell therapy has resulted in continuing remarkable success for some pediatric blood cancers, such as lymphoma, certain types of leukemia, and most recently, multiple myeloma (MM), the second most common type of blood cancer in the United States. CAR-T cell therapy has shown extraordinary responses with refractory MM. Continuing research is underway to further improve patient outcomes.6

 

Earlier CAR-T Cell Therapy Produces More Effective Results

Recent, large clinical trials have shown that after initial chemotherapy, CAR-T cell therapy may be more effective than current conventional treatments. With ongoing trials proving similar positive outcomes, researchers have noted that these dramatic results could signal a change in the future with CAR-T cells being used earlier in the course of the disease, which means it could become a frontline treatment for many more types of cancers soon.7

 

CAR-T Cell Treatment Results in Solid Tumors Are Improving

Solid tumors like diffuse intrinsic pontine glioma (DIPG) tumors are extremely difficult to treat due to its aggressive nature and location in the brain. While CAR-T cell treatment results in solid tumors are limited, progress is improving and heading in the right direction. 

The latest developments include the first patient infused directly with CAR-T cells into the brain to treat DIPG, which resulted in both proving the treatment was tolerable and that it inhibited growth of the cancer cells for a period of time.8 

Also, a recent collaboration of using GD-2 (a sugar molecule found on the surface of DIPG tumors) with CAR-T cell therapies in neuroblastoma and central nervous system gliomas have shown promising results in several clinical trials with dramatic reduction in size of DIPG tumors and improvement in symptoms.

 

CAR-T Cell Treatment of Relapsed or Refractory Cancers Is Underway

In 2020, the FDA approved the first CAR-T cell treatment for relapsed and refractory cancer. Some cases of these groups of clinical trials resulted in CAR-T cell therapy being so effective that they didn’t require further types of treatments.9 CAR-T cells as first- or second-line of treatment could significantly transform the treatment picture for relapsed or refractory cancers that conventional therapies initially fail to cure.10

 

Solving Kids’ Cancer’s Role in Advancing Research in Pediatric Cancer Treatment

Over a decade since receiving CAR-T cell therapy, Emily Whitehead is now a bright 17-year-old applying for college. Her 10-year survival shows the incredible progress of this new cancer treatment and the long-term hope it offers. Although there is still much work to be done, CAR-T cells have been a lifesaving treatment to many, like Emily, who only a few years ago would have succumbed to their disease. 

At Solving Kids’ Cancer, we believe the cures are in the science. That’s why we’re committed to funding breakthrough research and clinical trials to find cures for children with the deadliest childhood cancers. 

Over the past decade, we have funded three DIPG cancer-specific clinical trials and eight additional trials that included the study of DIPG and other types of pediatric brain tumors. Additionally, one of our more recent landmark pediatric research projects involves testing a new CAR-T cell therapy specifically targeting ETMR and medulloblastoma cancer cells. 

 

Help us raise awareness and funding to give more kids a second chance at life — because every kid deserves to grow up.

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Sources
1 Childhood Cancer Fact Library https://cac2.org/interest-groups/awareness/childhood-cancer-fact-library/
2 Study Shows CAR T-Cell Therapy Is Effective at Putting Childhood Leukemia Into Remission
https://www.cancer.net/blog/2022-12/study-shows-car-t-cell-therapy-effective-putting-childhood-leukemia-remission#:~:text=Leukemia%20Into%20Remission-,Study%20Shows%20CAR%20T%2DCell%20Therapy%20Is%20Effective,Putting%20Childhood%20Leukemia%20Into%20Remission&text=Results%20from%20a%20recent%20study,children%20with%20high%2Drisk%20disease.
3 CAR T-cell Therapy and Its Side Effects https://www.cancer.org/treatment/treatments-and-side-effects/treatment-types/immunotherapy/car-t-cell1.html
4 First-Ever CAR T-cell Therapy Approved in U.S. https://aacrjournals.org/cancerdiscovery/article/7/10/OF1/6073/First-Ever-CAR-T-cell-Therapy-Approved-in-U-S
5 Emily Whitehead, First Pediatric Patient to Receive CAR T-Cell Therapy, Celebrates Cure 10 Years Later https://www.chop.edu/news/emily-whitehead-first-pediatric-patient-receive-car-t-cell-therapy-celebrates-cure-10-years#:~:text=Search-,Emily%20Whitehead%2C%20First%20Pediatric%20Patient%20to%20Receive%20CAR%20T%2DCell,Celebrates%20Cure%2010%20Years%20Later
6 CAR T-Cell Therapy for Patients with Multiple Myeloma: Current Evidence and Challenges https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9439649/
7 Should CAR T Cells Be Used Earlier in People with Non-Hodgkin Lymphoma? https://www.cancer.gov/news-events/cancer-currents-blog/2022/nhl-car-t-cells-belinda-transform-zuma7
8 Could CAR-T-cell therapy offer hope to children with cancer? https://www.nature.com/articles/d41586-022-04344-6
9 CAR T-cell therapy boosts cure rates for children with aggressive blood cancer https://connect.uclahealth.org/2022/11/03/car-t-cell-therapy-boosts-cure-rates-for-children-with-aggressive-blood-cancer/
10 Paediatric Strategy Forum for medicinal product development of chimeric antigen receptor T-cells in children and adolescents with cancer: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration
https://pubmed.ncbi.nlm.nih.gov/34840026/
A man and a little girl are sitting in front of a wooden wall.

Ahana’s Story of Strength

By duda • November 20, 2024
Ahana’s Story of Strength with High-Risk Neuroblastoma Every day, over 1,000 children worldwide are diagnosed with cancer,1 and for many families, that diagnosis means facing an uncertain future. At just 20 months old, Ahana was diagnosed with a rare and aggressive cancer that had already spread through her small body. Her family’s search for life-saving childhood cancer treatments led them from South Africa to Spain and possibly toward a clinical trial in New York. Ahana’s journey shows the importance of supporting organizations like Solving Kids’ Cancer, which works to find, fund, and advocate for advanced treatments that give hope to families fighting against tough-to-treat rare pediatric cancers.
A poster for 2024 year in review for solving kids ' cancer

2024 Year-in-Review

November 11, 2024
Advancing Childhood Cancer Research: SKC’s 2024 Milestones
A group of children are holding a sign that says `` lace up for kids 2024 ''.

2024 Lace Up for Kids Recap

October 1, 2024
Lace Up for Kids: Your Impact on Childhood Cancer Research At Solving Kids’ Cancer, our mission has always been driven by hope — hope for better treatments, hope for breakthroughs, and most importantly, hope for every child fighting cancer. This year’s Lace Up for Kids campaign was nothing short of inspiring. Together, we’ve raised over $264,000 across the entire campaign, funding innovative childhood cancer research and providing more treatment options for children in need.  From coast to coast, nearly 4,000 participants have stepped up to lace up their gold shoelaces and make a difference. Whether it was through schools, sports teams, or community groups, the show of support has been overwhelming throughout the years. Over 7,823 donations have been made to accelerate new treatments, and the hashtag #CareWearShare has been used 13,864 times, spreading the message far and wide!
A girl is standing in front of a car in a parking lot.

Stories of Impact: Erin Martin

September 2, 2024
Gold Laces for Childhood Cancer Awareness Month: Erin’s Story 
A group of children are standing in a circle with their shoes on the floor.

Smithtown Goes Gold for Childhood Cancer

August 8, 2024
Smithtown Goes Gold for Childhood Cancer Awareness Month!
A poster for lace up for kids with a butterfly on it

Lace Up for Kids 2024

August 6, 2024
Childhood Cancer Awareness Month: Lace Up for Kids 2024
A man standing on top of a mountain with the words osteosarcoma survivor to surgeon dr. kurt weiss ' story

Osteosarcoma Survivor Dr. Kurt Weiss

August 1, 2024
Osteosarcoma Survivor to Surgeon: Dr. Kurt Weiss’ Story
Jacob 's journey : a childhood cancer survivor 's story

National Cancer Survivors Month

May 10, 2024
Jacob’s Journey: A Childhood Cancer Survivor’s Story Every battle has its heroes, and among the bravest are young children like Jacob Mozer, who faced stage 4 high-risk neuroblastoma when he was barely old enough to walk. Diagnosed at only 16 months old, Jacob’s early years were filled with constant medical appointments and intense treatments. Despite these challenges, today, Jacob is a shining example of the remarkable progress in pediatric oncology. He’s not merely surviving; he’s thriving as a pharmacy resident, driven to give back to the medical community that gave him a second chance at life. During National Cancer Survivors Month this June, we honor and celebrate fighters like Jacob who have battled cancer and emerged stronger. This month also serves to amplify the conversation about pediatric cancer survivorship and push for vital research that continues to save lives. Solving Kids’ Cancer (SKC) plays an essential role in this effort, supporting children like Jacob by funding crucial clinical trials that enhance survival rates and improve the quality of life for those fighting fatal childhood cancers.
A close up of a person holding a test tube in a laboratory.

Trailblazing Treatments for Pediatric Brain Tumors

May 2, 2024
Trailblazing Treatments for Pediatric Brain Tumors Picture a fortress built to guard precious treasure but accidentally also keeps out the very allies it needs in a time of crisis. This is the challenge faced by medical researchers when dealing with the brain’s natural defense — the blood-brain barrier — especially in children with brain tumors. At Columbia University Medical Center, Dr. Luca Szalontay is leading the charge to breach this fortress with groundbreaking approaches. Recently, Dr. Timothy Cripe from Solving Kids’ Cancer’s podcast, This Week in Pediatric Oncology (TWIPO) , interviewed Dr. Szalontay to delve deeper into these innovative treatments. This Brain Tumor Awareness Month, we’re shining a light on these important advances in treating children’s cancer. Understanding the Challenge Treating pediatric brain tumors like diffuse midline glioma (DMG) is especially tough because of the brain’s own protective shield, known as the blood-brain barrier. This barrier keeps out most chemotherapy drugs as well as large molecules, like antibodies, making it hard to treat the tumors effectively. Dr. Szalontay describes the dilemma: “Our field hasn’t been able to advance as quickly as it has with leukemias or other solid tumors because the drugs don’t penetrate the brain efficiently.” She adds on the nature of this blood-brain barrier, “It’s evolutionary, very important to protect us from different toxins and harmful agents, but this is a shield which protects the tumor as well.” Even when drugs work well in lab tests, they struggle to get to the tumor in strong enough doses without harming the body. Dr. Szalontay also points out that DMG tumors are found in very sensitive areas of the brain, making traditional surgery or radiation risky because they could damage vital brain functions. This makes finding safe and effective treatments for these tumors even more challenging. So, what’s being done to tackle these obstacles and help kids fight these tumors?
A group of stick figures are holding hands around a globe.

World Health Day: Pediatric Cancer’s Global Divide

April 4, 2024
World Health Day: Pediatric Cancer’s Global Divide Imagine this: Two children, born at the same moment, oceans apart. In a country brimming with medical resources, one child quickly gets top-notch treatment for cancer. Meanwhile, across the globe, the other child faces a drastically different reality, with limited access to basic care. This isn’t just a thought experiment — it’s the harsh truth of pediatric cancer treatment worldwide. Observed every year on April 7, World Health Day shines a light on the mission to make healthcare accessible for all. This day is particularly significant in the context of pediatric cancer, where vast disparities in treatment availability highlight the global challenge of ensuring every child has the opportunity for health and healing. The Stark Contrast in Survival Rates: A Look at Pediatric Cancer Statistics Every day, more than 1,000 children are diagnosed with cancer, and the chances of survival vary greatly depending on where they live.1 Pediatric cancer survival rates serve as a clear indicator of the disparities between high-income countries (HICs) and low- and middle-income countries (LMICs). In wealthier nations such as the United States and Western Europe, the 5-year survival rate for children with cancer is over 80%, thanks to advanced healthcare systems and access to the latest treatments.2 But, here’s a troubling childhood cancer statistic: in LMICs where 80% of children with cancer live, fewer than 20% survive.1 This gap is due to several factors, including late diagnoses or, tragically, no diagnosis at all. Many children succumb to the disease without access to pain medication, and the burdens of travel and the high costs force most families to abandon care. These challenges are compounded by limited access to necessary treatments,3 as well as a lack of specialized equipment and trained healthcare professionals needed for effective pediatric cancer treatment. Addressing this divide requires a concerted global effort to improve early detection, make treatments more affordable, and enhance healthcare infrastructures, giving every child a fair chance to beat cancer. Navigating the Access Maze Access to cancer care shouldn’t be a treasure hunt. Yet, for many in LMICs, it feels like that — a relentless search for something seemingly out of reach. Here’s the reality revealed in childhood cancer statistics worldwide: many areas lack enough doctors who specialize in children’s cancer4 and the prices of cancer drugs can be sky-high.2 Even getting to a hospital can be an ordeal, with some families traveling hundreds of miles. And the financial strain? Devastating. The cost of care can quickly add up to more than a year’s income, forcing households into deep financial distress. In many LMICs, the lack of comprehensive insurance coverage means families are often left to pay for pediatric cancer treatment entirely out-of-pocket.5 Families often face the impossible choice between their child’s health and their financial survival. Solving Kids’ Cancer: Bridging the Gap Solving Kids’ Cancer (SKC) isn’t standing by; we’re on the front lines, actively working to make a difference in the global pediatric cancer landscape. Beyond funding research, advocating for policy changes, and fostering global partnerships, SKC is committed to educating and empowering the community to take action. We understand that while we might not have a global foothold to address every aspect of the pediatric cancer divide directly, there’s power in collective action. Through our efforts, we aim to catalyze advancements in pediatric cancer treatments and care that could dramatically change outcomes for children worldwide. • Research: We direct funds into cutting-edge studies, searching for breakthrough treatments that promise a brighter future for all kids, regardless of where they live. • Advocacy: We advocate for children’s needs by steering research funding towards unmet challenges and collaborating with experts to prioritize all children, including expanding trials to international sites to maximize access.2 • Global Partnerships: We don’t do it alone. By teaming up with researchers, hospitals, and other organizations across the globe, we’ve helped fund clinical trials that have extended to over 250 institutions across 15 countries worldwide . Uniting for a Cause Solving Kids’ Cancer extends its mission beyond research by fostering community and collaboration. Our signature event, Lace Up for Kids , held annually during September Childhood Cancer Awareness Month, is a powerful testament to this mission, drawing communities together to stand in solidarity against childhood cancer. Additionally, supporters can create their own fundraiser and have a platform to engage their local communities, sparking crucial conversations and garnering support for the cause. These initiatives highlight an important fact: While the challenge of pediatric cancer is significant, each of us has a part to play. By working together and supporting each other, we can fund the research needed to possibly change the direction of pediatric cancer treatment and care worldwide. World Health Day: A Chance for Every Child The fight against pediatric cancer is a stark reminder of the work ahead of us.6 It’s a battle that Solving Kids’ Cancer is dedicated to, but we need more than just our efforts — we need a global movement. This World Health Day, let’s rally for every child’s right to fight cancer on an even playing field. Donate today to help fund more innovative research and one day find a cure for childhood cancer — because every child, no matter where they are born, deserves a chance — a chance to fight, a chance to survive, and a chance to thrive.
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