- First patients join international multi-center trial testing CAR T-cell therapy in young people with solid tumors.
- The study will be the largest of its kind, evaluating a personalized approach that re-trains the body's own defenses to recognize and attack cancer.
- The trial is a major step towards better treatment options to improve children and young people’s chances of survival and quality of life.
WASHINGTON--(BUSINESS WIRE)--The first two patients have been enrolled in a new clinical trial taking place in the US and UK. Up to 60 eligible children and young people with cancer will receive a personalized investigational therapy that harnesses the immune system to attack tumors.
Set to explore whether CAR T-cell immunotherapy could be effective as a treatment for children and young people with difficult-to-treat solid tumors*, the new trial includes participants up to early adulthood, with the first patient in their early 20s. The hope is that these investigational therapies will prove more effective at treating these cancers and reduce long-term side effects, compared to current standards of care. The trial is being led by a pioneering team of international experts, brought together by Cancer Grand Challenges - a global research initiative founded by Cancer Research UK and the National Cancer Institute, part of the National Institutes of Health, in the U.S. to tackle the biggest challenges in cancer. This is the first clinical trial funded by Cancer Grand Challenges and will be the world’s largest to test CAR T-cell therapy in children and young people with solid tumors.
Led by the Cancer Grand Challenges NexTGen team, the “multi-trial” consists of three trials running in parallel across the UK and the US. The UK arm of the NexTGen clinical trials is led from the UCL Cancer Institute at Great Ormond Street, in partnership with University College London Hospitals. The other arm, SABRE, is headed by Holly Meany, MD, and Catherine Bollard, MBChB, MD, both of Children’s National Hospital.
The third US arm of the trial is being led by a team at Dana-Farber Cancer Institute and Boston Children’s Hospital.
Dr. Catherine Bollard, senior vice president and chief research officer of Children’s National Hospital, co-team lead of NexTGen, said:
“We know that CAR T-cell therapies have been game-changing for pediatric leukemia and lymphoma, but that same success hasn’t yet been seen in solid tumors. That’s why our NexTGen team at Children’s National Hospital is thrilled to open a new clinical trial for children with relapsed or refractory solid tumors. This ‘first in human and child’ study targets not one, but two cancer markers — combining two T-cell therapy platforms to push the field forward.
While this is an early safety and feasibility study, we hope it brings new options and new hope to patients and families who have exhausted every other treatment. Together with our partners at UCL and Boston Children’s, we’re proud to help launch one of the largest international CAR T-cell programs focused specifically on young people with solid tumors.”
Unlike blood cancers, which affect the blood, bone marrow or immune system and don’t usually form lumps, solid tumors grow in organs or tissues and often form masses.
Over the past 30 years, there has been very little improvement in survival rates for children with these tumors. Current treatment options – many of which were designed for adults decades ago – can cause long-term health effects, such as infertility and learning difficulties. For those who see their cancer return, there are few other treatment options available.
CAR T-cell therapy is a type of treatment where a patient’s own T cells, a type of immune cell, are reprogrammed in the lab to better recognize and destroy cancer cells.
This form of treatment has been transformative for some people with cancer, particularly those with certain blood cancers. However, there are still challenges in broadening its use in different types of cancer and ensuring more patients can benefit.
Solid tumors are surrounded by complex defenses that are much harder for the immune system to break through. The NexTGen team is developing new ways to break down these barriers, helping the modified T cells infiltrate solid tumors and do what they’re designed to do — destroy cancer cells.
Ryan Schoenfeld, Chief Executive Officer at The Mark Foundation for Cancer Research, said:
“For far too many children living with these types of cancer, current treatments are not good enough. This trial offers a real sense of hope. By harnessing the power of CAR T-cell therapy in innovative ways, the NexTGen team is pushing the boundaries of what's possible for childhood cancer treatment.
“We’re proud to support this ambitious global effort through Cancer Grand Challenges. Children and families affected by these cancers deserve better, and they deserve it now.”
The international team of scientists, co-funded by The Mark Foundation for Cancer Research, recently recruited its first two patients into the trial.
The trial aims to recruit patients across two sites in the UK (GOSH and UCLH) and two in the US (Children’s National Hospital, Washington and the Dana-Farber Cancer Institute). Patients and their families should speak to their doctor to find out more about taking part in this study.
Dr. Dinah S. Singer, Ph.D., NCI Deputy Director for scientific strategy and development, said:
“It is a significant milestone for the first Cancer Grand Challenges-funded clinical trial to begin to enroll patients. When we first set the challenge in 2020, we emphasized the need to develop better treatments for children and young people with solid tumors that are effective and less toxic.
In only six years, this global team of eminent scientists identified new cancer targets, developed novel CAR T-cell therapies, and is now testing them in the largest trial of its kind.
At such incredible pace and precision, their work shows the bold, collaborative science Cancer Grand Challenges was created to achieve. Thank you to the researchers from Team NexTGen, the patient advocates who have championed this work, and our partners at The Mark Foundation for Cancer Research, for making this work possible.”
About the Cancer Grand Challenges NexTGen team
The NexTGen team is a global collaboration between researchers across the UK, US and France to tackle the challenge of finding better treatments with fewer long-term side effects for children and young people with solid tumors.
The team is funded by Cancer Research UK, the National Cancer Institute (NCI) in the US and The Mark Foundation for Cancer Research, through Cancer Grand Challenges.
Notes to Editor
*The trial will look at rhabdomyosarcoma, Ewing sarcoma or desmoplastic small round cell tumor.
About Cancer Grand Challenges
Co-founded in 2020 by two of the largest supporters of cancer research in the world: Cancer Research UK and the National Cancer Institute, part of the National Institutes of Health, Cancer Grand Challenges supports a global community of diverse, world-class research teams to come together, think differently and take on some of cancer’s toughest challenges. These are the obstacles that continue to impede progress and no one scientist, institution or country will be able to solve them alone. With awards of up to £20m, Cancer Grand Challenges teams are empowered to rise above the traditional boundaries of geography and discipline to make the progress against cancer we urgently need.
About Cancer Research UK
- Cancer Research UK is the world’s leading cancer charity dedicated to saving lives through research, influence and information.
- Cancer Research UK’s pioneering work into the prevention, detection and treatment of cancer has helped save millions of lives.
- Cancer Research UK has been at the heart of the progress that has already seen survival in the UK double in the last 50 years.
- Cancer Research UK supports research into the prevention and treatment of cancer through the work of over 4,000 scientists, doctors and nurses.
- Together with its partners and supporters, Cancer Research UK is working towards a world where people can live longer, better lives, free from the fear of cancer.
For further information about Cancer Research UK's work or to find out how to support the charity, please call 0300 123 1022 or visit www.cancerresearchuk.org.
About The Mark Foundation for Cancer Research
The Mark Foundation for Cancer Research, a charitable organization based in New York City, actively partners with scientists worldwide to accelerate research that will transform cancer prevention, diagnosis, and treatment. Since 2017, The Mark Foundation has awarded over $275 million in grants to investigators at more than 115 academic institutions across 18 countries, with research programs focusing on early career support, team science collaboration, new technology innovation, and therapeutics discovery. Additionally, The Mark Foundation maintains a growing portfolio of investments in early-stage cancer diagnostics and therapeutics companies, including several that have transitioned from grantee projects into commercial development.
To learn more, please visit www.themarkfoundation.org
About Children’s National Hospital
Children’s National Hospital, based in Washington, D.C., was established in 1870 to help every child grow up stronger. Today, it is one of the top 10 children’s hospitals in the nation and ranked in all specialties evaluated by U.S. News & World Report. Children’s National is transforming pediatric medicine for all children. The Children’s National Research & Innovation Campus opened in 2021, a first-of-its-kind pediatric hub dedicated to developing new and better ways to care for kids. Children’s National has been designated four times in a row as a Magnet® hospital, demonstrating the highest standards of nursing and patient care delivery. This pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty care locations in the D.C. metropolitan area, including Maryland and Virginia. Children’s National is home to the Children’s National Research Institute and Sheikh Zayed Institute for Pediatric Surgical Innovation. It is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. In 1987, Children’s National founded Safe Kids Worldwide, a non-profit dedicated to reducing unintentional injuries among children through comprehensive national and global education, research and advocacy. As a non-profit, Children's National relies on generous donors to help ensure that every child receives the care they need.
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