Precision Medicine and Advances in Pediatric Leukemia Treatment

Jump To Section

• Dramatic Improvement in Childhood Leukemia Cure Rates
• Three Pillars of Progress in Leukemia Therapy
• The Critical Role of Supportive Care in Treatment
• The Major Challenge of Treatment Toxicity and Side Effects
• Understanding Long-Term and Delayed Side Effects
• Future Goals: Curing Every Child with Less Toxic Therapy
• The Need for Investment in Biology and Research

Dramatic Improvement in Childhood Leukemia Cure Rates

Pediatric leukemia treatment has undergone a revolutionary transformation over the past four decades. Dr. Shai Izraeli, MD, notes that in the 1960s, very few children were cured of leukemia. Today, the prognosis is overwhelmingly positive, with modern protocols curing almost 90% of children diagnosed with the most common type, acute lymphoblastic leukemia (ALL). This unbelievable progress represents one of the great success stories in modern medicine, turning a once-fatal diagnosis into a highly treatable condition.

Three Pillars of Progress in Leukemia Therapy

Dr. Shai Izraeli, MD, identifies three fundamental pillars that drove this remarkable progress. The first was the establishment of international, collaborative chemotherapy treatment protocols. These protocols, often run through national or multicenter groups, allowed oncologists to learn from each successive study and systematically improve outcomes every few years. The second pillar was the integration of genetics into diagnosis, revealing that leukemias that appear identical under a microscope can be very different diseases genetically. This understanding allows therapy to be adapted to the specific genetic subtype of leukemia a child has, a cornerstone of precision medicine.

The Critical Role of Supportive Care in Treatment

The third pillar of progress is the dramatic advancement in supportive care. Dr. Shai Izraeli, MD, emphasizes that you cannot treat leukemia effectively without a robust support system. This includes specialized intensive care units, infectious disease teams, and pharmacology experts. He notes that at Schneider Children's Medical Center, the entire hospital works in concert to support oncology patients. This comprehensive supportive care is essential for managing the intense side effects of chemotherapy and preventing treatment-related mortality.

The Major Challenge of Treatment Toxicity and Side Effects

Despite the high cure rates, current childhood leukemia treatment remains incredibly toxic. Dr. Shai Izraeli, MD, explains that therapy typically involves 10 to 12 different chemotherapy medications administered over two years. This intensity means children often require care in the ICU. A significant problem is treatment-related mortality, where at least 20% of deaths are caused by the complications of therapy itself, not the leukemia. This highlights the urgent need to develop less toxic treatment strategies.

Understanding Long-Term and Delayed Side Effects

The focus in pediatric oncology extends far beyond initial cure to ensuring a healthy life decades later. Dr. Izraeli tells parents the goal is for their children to live long enough to have grandchildren. This long-term perspective makes managing delayed side effects critical. For example, 20-40% of adolescents cured of leukemia suffer from bone fractures and other skeletal problems due to prolonged steroid use. Other serious delayed effects include neurocognitive issues from brain-directed therapy and an increased rate of secondary cancers later in life.

Future Goals: Curing Every Child with Less Toxic Therapy

Dr. Shai Izraeli, MD, states that the field is now entering a new era with two primary goals. The first is to cure every single child diagnosed with leukemia, a goal he believes is achievable within the next 20-30 years without exaggeration. The second, equally important goal is to drastically lower the toxicity of therapy. This means developing targeted treatments based on a deeper biological understanding of the disease to replace the blunt, toxic instruments of traditional chemotherapy.

The Need for Investment in Biology and Research

Achieving these ambitious goals requires a significant and sustained investment in basic biology and clinical research. Dr. Izraeli concludes that now is the time to push forward, using the tools of precision medicine to identify new vulnerabilities in leukemia cells. This research is the key to designing smarter, more effective, and far less toxic treatments that will ensure not only survival but also a high quality of life for every child who defeats leukemia. The conversation with Dr. Anton Titov, MD, underscores that the future of pediatric oncology lies in this targeted, research-driven approach.

Full Transcript

Dr. Anton Titov, MD: Precision medicine era arrived in pediatric leukemia treatment. It means we hope to cure every child from leukemia, and do so with fewer side effects. Leading child cancer expert. You study childhood leukemia. It's a very complicated disease.

Dr. Anton Titov, MD: What is new in pediatric leukemia diagnosis and treatment? Where has the progress been made over the last 40 years?

Dr. Shai Izraeli, MD: When I was a child, very few children were cured from leukemia. This is in the 1960s. Now, 40 years or so later, we cure most children with leukemia. Almost 90% of children with leukemia are cured today. The most common type of leukemia is acute lymphoblastic leukemia.

That's unbelievable. This was achieved because of three processes. First, from the beginning, children have been treated according to international chemotherapy cancer treatment protocols. For example, in the case of the United States, national cancer treatment protocols, multicenter cancer treatment protocols of pediatric cancer treatment.

Changes were made to the cancer treatment protocols every couple of years. You can learn from previous experience and apply it to the next.

Dr. Shai Izraeli, MD: Then the second pillar of the progress of treatment of leukemia occurred. These pediatricians who were the pioneers, they were geneticists. We learned that not all leukemias are the same. You may have two children whose leukemias look the same in the microscopes and blood counts, etc.

Dr. Anton Titov, MD: But they are very different leukemias by genetics. Now we adapt leukemia therapy to the genetic subtype of leukemia.

Dr. Shai Izraeli, MD: The third pillar of leukemia therapy progress that has been critically important. It is the progress in child cancer treatment in general. But it is important especially in leukemia. It is the development of supportive care. You cannot just treat leukemia without support system.

We have here in Schneider Children's Medical Center great supportive treatment capabilities. I sometimes joke that the whole hospital is working for me. It is intensive care unit, infectious disease unit, pharmacology unit, etc. You need the supportive care.

We are now in this stage. We can cure the majority of children with leukemia.

Dr. Shai Izraeli, MD: We are in the beginning of new era in leukemia treatment. But it is also a new era in general for child cancer treatment. In this new era we can really hope, without exaggeration, to cure every child with leukemia over the next 20 - 30 years.

The second very big challenge is to lower toxicity of therapy. We need to lower toxicity of therapy because now our leukemia treatment is incredibly toxic. We treat leukemia with 10 to 12 different cancer medications or types of chemotherapy, usually over two years.

Quite often our kids with leukemia go to intensive care units. We have long term toxicities. For example, up to 20 to 40% of adolescents that are being cured from leukemia have afterwards fractures. They have other problems in their bones due to steroids used in leukemia treatment.

At least 20% or more of the deaths occur because of leukemia treatment.

Dr. Shai Izraeli, MD: There is a treatment-related mortality, treatment-related death in child leukemia. We have a problem of delayed side-effects. We have to remember that child leukemia therapy is very different from adult medicine.

If I am now diagnosed with cancer, a reasonable goal would be to keep me around for 10 - 20 years. I tell this to the parents who come to our department. Our goal is that their children will have grandchildren. It's a completely different dimension.

We have to think about delayed cancer treatment side effects.

Dr. Shai Izraeli, MD: Delayed leukemia treatment side effects are related to treatment of the brain, for example. We have been talking about it.

Dr. Anton Titov, MD: Delayed cancer therapy side effects are secondary cancers. There is increased rate of secondary cancer.

Now our goal is to cure every child with leukemia. We have to do that with less toxic therapy.

Dr. Anton Titov, MD: It is now the time to invest a lot in biology and research to achieve cure for children with leukemia.