2 Clarke Drive
Suite 100
Cranbury, NJ 08512
© 2025 MJH Life Sciences™ and OncLive - Clinical Oncology News, Cancer Expert Insights. All rights reserved.
A love of collaborating with colleagues from around the globe and a fiery passion to help children diagnosed with cancer led Ching-Hon Pui, MD, to dramatically improve outcomes in pediatric oncology.
Ching Pui, MD was clearly brilliant from a young age. Hailing from a large family the need to become inependant very early on in his life would prove critical as his intellectual skills developed. Though he claims it is no longer true, he had a near-perfect photographic memory in his youth. He regularly bested Chinese chess masters, becoming a master himself. He can play chess in his head without looking at the board, and he always won by thinking 20 to 30 steps ahead. This skill is what Pui credits with helping him write manuscripts: He can express the appropriate result, present his findings in a way that “sells” his papers, and likens it to a game. “I have a strategy for writing an article,” he said.
Pui also credits some individuals in his early days for his interest and pursuit in medicine as an adult. When he was a fifth-grade student at Saint Timothy’s Primary School in Hung Hom in Hong Kong Pui ran into his English instructor while waiting for his bus home. “She smiled and told me I was a good English student. Well, that wasn’t exactly true,” he said. However, Pui said it was her kindness and encouragement that spurred him on to become a better and more focused student from that day forward. “I didn’t want to disappoint her. I really have to thank her. She really changed me.”
He also considers his colleagues at St Jude Children’s Research Hospital (St Jude) as some of his most impactful mentors, including the late Donald Pinkel, MD, who was the first medical director in St Jude’s history and a prior inductee to the Giants of Cancer Care program. Pinkel inspired Pui to “challenge the conventional wisdom."
Another mentor Pui gives credit to is the late Rhomes (John) Aur, MD, who stressed the integral role that meticulous medical recordkeeping and clinical data sets can play in treat- ment protocols and research. Pui also credits 4 other changemakers in the field for research opportuni- ties that helped to nurture his career during critical junctures: Joseph V. Simone, MD (a 2019 Giant recip- ient); Arthur W. Nienhuis, MD; William E. Evans, MD, PharmD; and James R. Downing, MD.
Additionally, Pui credits St Jude as an institution for his career trajectory and fulfillment. “I am blessed to be working at an institution where patients are treated with the best available approaches, regardless of their ability to pay. As a result, I have seen near-miraculous recoveries from practically incurable conditions.”
These poignant encounters and surroundings were not the only catalysts to push Pui into pediatric oncology. When Pui was in middle school, he happened upon a 1969 French film called The Christmas Tree. Despite what the title implies, it is a dramatic film about a boy who is diagnosed with leukemia after witnessing the dropping of an atomic bomb while on vacation with his father.
Pui’s passion was ignited after watching the film, understanding even then that he might be able to do something for children with leukemia. Many years later, while already at St Jude and resting in an on-call room, the same film was playing on the room’s television and Pui watched it again. This time, with his education and growing experience, he knew he could help children like the boy depicted in the heart-wrenching film.
The coincidence of the film and “working daily with skilled cancer specialists and young patients with leukemia was the catalyst that caused me to become a pediatric oncologist,” Pui said.
His work ethic and dedication are revered. “I had the pleasure of working alongside and collaborating with Dr Pui for [more than] 40 years at St Jude, and we coauthored [more than] 200 papers together. Dr Pui is the most knowledgeable person on the planet regarding the treatment of children with leukemia. He is brilliant, but he also works longer and harder than anyone else in the field,” said Evans, who is Emeritus Faculty and former president/CEO of St Jude. “Dr Pui was often invited by other institutions and international pediatric cancer cooperative groups to attend their meetings, because he often knew things about their studies that they did not, so they had him there to bring new insights as they analyzed their data and began planning their next treatment protocol. He is indeed a Giant in cancer treatment and research.”
Pui noted that, in situations like these, he is often the only pediatric oncologist in the room. In 2024 alone, Pui stated that he designed 4 to 5 protocols. He also mentioned that he has authored or coauthored more than 1000 papers in his lifetime, and he credits “a good team of investigators” with helping him to be so prolific. He has done all of this while still treating patients.
Jun J. Yang, PhD, also of St Jude and a frequent collaborator with Pui, said that “Pui’s career has been marked by many achievements that have fundamen- tally changed the way we understand pediatric ALL [acute lymphoblastic leukemia] and the way these children are treated. As a master clinician, he has led consecutive frontline ALL protocols at St Jude for the past 40 years, and each one of them has resulted in paradigm-shifting treatment regimens adopted subsequently throughout the world. Dr Pui is an inspiration to many of us. He is a true Giant of Cancer Care.”
One of those studies, and perhaps his most notable, has been the TOTXV study (NCT00137111). Launched in June 2000 and concluded in 2007, TOTXV is the 15th study in a series of clinical trials at St Jude focused on children with ALL and the use of “risk-directed” therapy.
“Our team developed the inaugural clinical trial employing minimal residual disease–directed therapy, significantly elevating survival rates to surpass the 90% mark [and] dramatically improving outcomes without adding new drugs,” Pui said. “We can directly reduce the overtreatment or undertreatment of patients and allow for less toxicity.” He noted that the study also spearheaded the complete elimination of prophylactic cranial irradiation.
“No one has done more for children with leukemia around the world than [Dr] Pui....He has been responsible for increasing the survival rate of pediatric ALL from 40% to 94%, improving survivor- ship by removing cranial irradiation from standard treatment, and advocating for China to cover treatment of children with ALL,” said James Downing, MD, current president and CEO of St Jude. “After more than 40 years, he is continuing his work through the Department of Global Pediatric Medicine at St Jude.”
More than 20 years ago, in 1991, Pui was invited by Project HOPE (a US organization) to four different hospitals in Shanghai, China to teach and share medical materials for Chinese physicians to learn from. By the year 2000, the Shanghai Children’s Medical Hospital was open and a sanctioned site for St. Jude’s, who purchased equipment for the facility to conduct their research. It was during a brief encounter while there that he was moved to act after seeing a distraught, almost inconsolable parent. When he inquired about her distress, he learned that her child had been diagnosed with ALL but was turned away from the facility because of the inability to afford the cost of treatment. Essentially, her child had been sent home to die.
Pui spoke with Zhu Chen, PhD, who was then the director of the Shanghai Institute of Hematology and an academician at the Chinese Academy of Science, and then became Ministor of Health, and vice chairman of the Standing Committee of National People’s Congress, and an honorary director of Shanghai Institute of Hematology, and discussed the prohibitive cost of treatment. This conversation with Chen indirectly led to China quickly making ALL one of the first diseases covered by national insurance. At the time, less than 30% of Asian children diagnosed with ALL were being treated.1
Pui noted that, in 2003, he asked Mr. Paul Marcus to establish the Partner in HOPE Foundation Limited (Hong Kong) to support the treatment of underpriv- ileged children with acute lymphoblastic leukemia. The endeavor was incredibly successful, with 84% of the original 155 patients treated at the time, still alive today.2
Pui and Chen’s continued friendship and the close collaboration between St Jude and Shanghai Children’s Medical Center led to many more ideas that prompted change. Approved in 2017, the National Children’s Medical Center in Shanghai opened on September 26, 2024. It is a 500-bed facility, making it the largest pediatric cancer center in the world—surpassing the Children’s Cancer Hospital Egypt 57357 at 345 beds.3 Pui has been invited to the opening ceremonies and plans to attend.
Pui highlighted his love of collaborating with colleagues from around the globe, stating that “together, we can advance the field much faster.” He and his colleagues also forged the Ponte di Legno study group, which brought 15 international study groups together to engage in collaborative research. With the support of the VIVA foundation for children with cancer, a Singapore-registered charity and St Jude Children’s Research Hospital, the St Jude-VIVA Forum has been organized to support Pui to provide education to hundreds of pediatric oncologists in Asia for the past 18 years. It is an annual forum to this day.
Here, he made sure to acknowledge Jennifer Yeo, the founder of the VIVA Foundation for Children with Cancer (in Singapore) and the VIVA China Chil- dren’s Cancer Foundation, “that support my work in Asia and China.”
In 2014, the China National ALL Study Group was formed and supported by the China Children’s Cancer Foundation, a Hong Kong based charity to help Pui to find solutions to critical problems for children in China with ALL via large clinical trials. One study that the group conducted, CCCG-ALL-2015 (ChiCTR-IPR-14005706), specifically concluded that outcomes were drastically improved without the need for preventive cranial irradiation, achieving a 5-year survival rate of 91%.4 Another phase 2 trial (ChiCTR2000032211), regarding the coadministra- tion of CD19 and CD22 chimeric antigen receptor T-cell protocols, demonstrated that “complete remis- sion was achieved in 99.0% of the 194 patients with refractory leukemia or hematologic relapse, all nega- tive for minimal residual disease.”5 The group has published 21 papers since its inception.
Pui has no plans to stop his work any time soon and says he hopes to “find a cure for the remaining 10% of patients with refractory or relapsed ALL, develop affordable and effective treatment for patients in developing countries, and replace toxic chemo- therapy with novel effective and less toxic treatments to improve...quality of life.” He also said that his only “bad” days are when his patients are suffering.
His work, or his entire life’s passion, is without ques- tion predicated on his relationship with his patients. “I admire the resilience of my patients. No matter how sick they are, they amaze me with their ability to bounce back—a trait that has taught me to be stubborn and slow to give up,” Pui said.
He takes great care in building a rapport with them and their families and has confessed to performing magic tricks for his youngest patients to put them at ease during their cancer journey. One patient even traveled by public transit to meet him at an event and thank him many years after treating her. Those moments are what inspire him most.
Pui recalled another patient, who is reportedly now a “happy teenager who just graduated as valedictorian from his university.” His story has a happy ending because of Pui’s drive and tenacity to stay the course and adjust the treatment plans, but there was a time when the outcome looked bleak.
Many years prior, it seemed that all hope had been lost. “The patient needed intensive chemotherapy followed by a bone marrow transplant for the treatment of relapsed leukemia. His mother was the donor. During the intensive chemotherapy, he was found to have serious liver problems that could require a liver transplant, and he lost his gallbladder. After the bone marrow transplant, he needed to have his spleen removed and developed a rare complication that left him completely paralyzed and in severe pain. He required respiratory support because he was unable to breathe on his own. Worst of all, leukemia cells reappeared in his bone marrow. It was virtually hopeless and painful to watch him suffer. I was grasping at straws trying to find new ways to manage these complications. Fortu- nately, the transplanted cells from his mother had begun to grow and instilled hope that they would grow strong enough to overcome the residual leukemia, and that his organs might recover with time.” They did, and Pui counts this as a “miracle.”
Pui has clearly been a force for life-changing advancements within the world of pediatric oncology and continues to be a guiding light for others in the treatment of patients with ALL. Although he gives credit to many for his career and achievements, he cites his brother, David Y.H. Pui, PhD, a renowned mechanical engineer with the University of Minnesota, as the individual he admires the most. His brother was there through it all, encouraging Pui in all things, including moving to the United States to start his career. “He is why I’m here,” Pui said.
Pui considers any free time as an opportunity to further drive the achievement of his goals rather than an opportunity for rest. He enjoys researching, analyzing data and creating papers, developing clinical trials, and expanding knowledge for the leukemia community. Outside of work, Pui enjoys classical music, swimming, and watching movies during his travels. His passion for curing any child diagnosed with ALL is what truly brings him the most joy.