Pediatric Cancer

Pediatric Cancer

Pediatric cancer care offers one of the most striking examples of progress in modern medicine. In the 1950s, less than 10 percent of children with cancer were cured. Today, nearly 80 percent will survive a cancer diagnosis.

Federally-funded clinical trials have been especially important in pediatric cancer. Most of today's effective treatments for childhood cancers were developed through trials conducted by the National Cancer Institute's Clinical Trials Cooperative Groups. Currently, 50 to 60 percent of eligible children with cancer are enrolled in these trials.

While overall progress has been impressive, some childhood cancers remain very difficult to treat, and one in five children ultimately does not survive. Continued research is essential to develop more effective, targeted, and safer treatments, so that even more children with cancer have the potential to live full and productive lives.

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2017

First gene therapy for cancer

First gene therapy for cancer

In a landmark trial, the chimeric antigen receptor-modified T cell (CAR-T) therapy tisagenlecleucel (Kymriah) causes complete remissions in a majority of young patients with B-cell acute lymphoblastic leukemia (ALL) that progressed despite previous treatment with standard therapies. Soon after, tisagenlecleucel becomes the first gene therapy to be approved by the FDA.

CAR-T therapy works by genetically modifying the patient’s own immune T cells to target a specific cancer protein and ultimately trigger the cancer cell’s destruction. Importantly, this “living therapy” needs to be given to the patient only once, because CAR T cells continue to multiply in the patient’s body, so the anticancer effects of CAR T cells persist and can even increase over time.

2016

Adding second stem cell transplantation lowers relapse risk for high-risk neuroblastoma

Adding second stem cell transplantation lowers relapse risk for high-risk neuroblastoma

A federally funded clinical trial finds that adding a second autologous stem cell transplant (ASCT) to standard neuroblastoma therapy can reduce the chance of recurrence within three years for children at high risk. An improvement within this three-year timeframe is especially important because most neuroblastoma recurrences develop within 2 to 3 years of diagnosis, and patients who do not experience relapse in this time period have a much better chance of long-term survival. Neuroblastoma is the second most common solid tumor in children and the most common cancer in infancy.  

Childhood cancer survivors living longer and healthier

Childhood cancer survivors living longer and healthier

A federally funded analysis of over 34,000 five-year childhood cancer survivors shows significant gains in long-term survival over three decades, owing to steady refinements in treatment. The rate of death from any cause within 15 years of childhood cancer diagnosis declined by half (from 12% to 6%) among survivors treated in the 1990s compared to those treated in the 1970s. While treatment advances played a major role, reductions in treatment-related health complications likely had the greatest impact on long-term survival. This includes lower dose of radiation for certain cancers and less use of anthracycline chemotherapy. These advances helped make it possible for more than 400,000 childhood cancer survivors to be alive today in the United States.

2013

Potential new approach to treating childhood leukemia found

Potential new approach to treating childhood leukemia found

An early clinical trial shows that a new approach called chimeric antigen receptor-modified T cell (CAR-T) therapy led to complete remission of acute lymphoblastic leukemia (ALL) that had not responded to previous aggressive treatment in two children. This is the first report on the use of CAR T-cell therapy in patients with ALL. 

CAR-T therapy works by genetically modifying the patient’s own immune T cells to target a specific cancer protein.

2010

Late effects of childhood cancer substantially reduce life expectancy

Late effects of childhood cancer substantially reduce life expectancy

A modeling study based on data from the long-running Childhood Cancer Survivor Study shows that life expectancy for survivors of childhood cancer is about 10 years shorter, on average, than in the general population. This effect is due to an increased risk of heart and lung problems and second cancers later in life. The findings increase the urgency of long-term health screenings for childhood cancer survivors, who now number over 300,000 in the U.S.

Review finds major success against childhood cancers

Review finds major success against childhood cancers

An analysis of childhood cancer statistics reveals that an estimated 38,000 childhood cancer deaths have been averted in the United States between 1975 and 2006. Cancer death rates also declined by more than 50 percent in this period. These achievements are attributed to improved drugs, treatment strategies and past investments and collaboration in clinical trials. At the same time, however, childhood cancer incidence increased significantly, with one form of leukemia, acute lymphoblastic leukemia (ALL), rising most quickly.

Investigators stressed the need for new, innovative treatment strategies, and effective targeted drugs in particular, to ensure that death rates continue to fall.

2009

Targeted drug helps children with hard-to-treat leukemia

Targeted drug helps children with hard-to-treat leukemia

Adding the targeted drug imatinib (Gleevec) to standard, intensive chemotherapy is found to dramatically improve outcomes for patients with acute lymphoblastic leukemia (ALL) who have a genetic mutation known as the Philadelphia chromosome. After three years, 80 percent of patients who received both therapies in the study were alive, compared to 30 percent who received chemotherapy alone. While further research is needed, the results offer hope to children with this hard-to-treat form of cancer.

First immunotherapy for children with neuroblastoma

First immunotherapy for children with neuroblastoma

A study shows that a new immunotherapy – called chimeric anti-GD2 antibody ch14.18 – can reduce risk of recurrence and increase survival for patients with high-risk neuroblastoma, a cancer of the nerve tissue. This therapy targets a specific molecule on neuroblastoma cells for destruction by the immune system. The treatment has become a standard of care for children with high-risk neuroblastoma who have completed aggressive chemotherapy and radiation treatment. Clinical trials to refine the approach and possibly improve its efficacy are underway.

2007

Children with neuroblastoma benefit from less aggressive treatment

Children with neuroblastoma benefit from less aggressive treatment

A large, international trial finds that some children with neuroblastoma (a cancer of the nerve tissue) can receive a much less aggressive chemotherapy regimen than the previous standard treatment, without reducing their survival. Reducing chemotherapy, surgery and radiation helps children avoid serious, long-term side effects such as hearing loss. Today, certain key factors, such as age, stage of the tumor and specific tumor characteristics help determine which children with neuroblastoma can have reductions in therapy.

2005

New treatment option for children with rare forms of leukemia and lymphoma
Research sheds light on long-term health problems of childhood cancer survivors

Research sheds light on long-term health problems of childhood cancer survivors

Twelve years after its launch, the Childhood Cancer Survivors Study reports that childhood cancer survivors' risk of long-term moderate and severe health problems – including heart problems, second cancers and scarring of the lungs – was five times greater than that of their healthy siblings. The same study later shows that less than half of survivors undergo the recommended screenings for many of these conditions. Thanks to this and other studies, oncologists and primary care providers are increasingly focused on monitoring the long-term health of childhood cancer survivors.

2003

Adding two drugs to standard chemotherapy improves survival for children with early-stage Ewing sarcoma

Adding two drugs to standard chemotherapy improves survival for children with early-stage Ewing sarcoma

Scientists show that adding two additional drugs (ifosfamide and etoposide) to the standard four-drug combination chemotherapy regimen (doxorubicin, vincristine, cyclophosphamide and dactinomycin) significantly increases five-year survival rates in patients with early-stage Ewing sarcoma (a form of bone cancer, that most often occurs in the legs, spine, ribs, or pelvis). This new combination soon becomes the standard treatment.

2001

National Cancer Institute consolidates clinical research on childhood cancers

1999

Intensive chemotherapy, with radiation and stem cell transplantation, improves neuroblastoma survival

Intensive chemotherapy, with radiation and stem cell transplantation, improves neuroblastoma survival

An NCI study shows that treatment with intensive chemotherapy, together with radiation therapy and autologous stem cell transplantation, lowers the risk of recurrence and improves survival in children with high-risk neuroblastoma (a cancer of the nerve tissue). Adding stem cell transplantation to neuroblastoma therapy makes it possible for patients to recover from the intense chemotherapy needed to effectively fight the disease. In this approach, doctors harvest healthy stem cells from the patient's own bone marrow before chemotherapy and then re-implant them intravenously after treatment. The infusion of stem cells helps rebuild the patient's blood-production and immune system, which are both impaired by chemotherapy.

1998

New chemotherapy regimen reduces side effects for children with Hodgkin lymphoma

New chemotherapy regimen reduces side effects for children with Hodgkin lymphoma

A National Cancer Institute-funded study shows that a chemotherapy combination regimen known as ABVD (with the drugs doxorubicin, bleomycin, vinblastine and dacarbazine) can safely replace the previous standard treatment for patients with advanced Hodgkin lymphoma. This new combination dramatically lessens the side effects of cancer therapy for children with the disease, without affecting overall treatment outcomes.

1993

Major study tracks long-term health of childhood cancer survivors

Major study tracks long-term health of childhood cancer survivors

As more and more children are cured of their cancer, researchers realize the need to better understand the long-term health effects of their cancer – and cancer treatment – as they age. In 1998, researchers from centers across the United States and Canada launch the Childhood Cancer Survivor Study, the first major study to monitor the long-term health of childhood cancer survivors. Over the coming years, their research sheds light on major long-term risks for these survivors, from increased risk of heart disease and stroke to secondary cancers later in life. These results enable physicians to monitor for these effects and provide more timely care.

1991

Genetic discovery helps guide treatment decisions for common pediatric tumors

Genetic discovery helps guide treatment decisions for common pediatric tumors

Researchers discover a link between a gene called MYCN and response to treatment for neuroblastoma, a cancer that most commonly arises in the adrenal gland or in the surrounding nervous system tissue. Neuroblastoma is one of the most common cancers among infants and young children. Because of this finding, doctors are now able to distinguish patients who need intense therapy from those who can be treated with a less aggressive approach, sparing many children from unnecessary side effects of treatment.

1987

Improved combination chemotherapy regimens boost pediatric leukemia cure rates

Improved combination chemotherapy regimens boost pediatric leukemia cure rates

Cure rates for pediatric acute lymphoblastic leukemia (ALL) climb above 80 percent with the introduction of combination chemotherapy regimens.

In the same time period, cancer remissions (which may or may not be permanent) also climb as high as 99 percent, with the help of ongoing advances in supportive care. Supportive care dramatically eases the side effects of treatment and enables greater numbers of patients to complete their full chemotherapy regimen on schedule.

Chemotherapy before surgery helps children with bone cancer avoid amputation

1986

Scientists discover genes linked to increased risk of childhood and adult cancers

Scientists discover genes linked to increased risk of childhood and adult cancers

Researchers identify two inherited gene mutations associated with an increased risk of developing childhood and adult cancers – one for retinoblastoma, a cancer of the eye, and another for Li-Fraumeni syndrome, a condition that predisposes children and adults to several different cancers. These discoveries make it possible to screen for the mutations in children with a family history of the diseases, creating the potential for earlier detection and more effective treatment.

1975

Successful use of adjuvant therapy in osteosarcoma

Successful use of adjuvant therapy in osteosarcoma

Researchers find that adjuvant chemotherapy – treating cancer with chemotherapy following surgery for early-stage cancers – offers major benefits for children with osteosarcoma (cancer of the bone). This approach prolonged the time that most patients in the study remained free of cancer recurrence after their surgery. In 1986, results from a long-term randomized trial establish adjuvant chemotherapy as the standard of care for all children with osteosarcoma.

1972

New research network seeks cures for soft tissue cancers in children

New research network seeks cures for soft tissue cancers in children

The NCI establishes the Intergroup Rhabdomyosarcoma Study Group (IRSG) to investigate rhabdomyosarcoma and other forms of sarcoma – tumors of the soft tissues that support and connect the body – in patients age 21 and younger. By pooling resources, expertise and patients, the group accelerates the development and evaluation of new classification strategies, surgical approaches, and therapies.

1969

Formation of the National Wilms Tumor Study Group

Formation of the National Wilms Tumor Study Group

This new NCI-funded Cooperative Group aims to improve survival for children with Wilms tumor, a rare kidney cancer that most often occurs in young children, around age 3 or 4.

This group is later the first to demonstrate the benefit of combination adjuvant chemotherapy (administrating chemotherapy after surgery) for patients with Wilms tumor. Over subsequent decades, the group completes five major clinical trials involving hundreds of children that provide key insights on assessing the disease, most effective use of surgery, chemotherapy and radiation therapy, and managing the long-term effects of treatment in these young patients. Thanks to the Group's research, five-year survival rates for Wilms Tumor climb from 40 percent in the mid-1960s to nearly 90 percent by the early 2000s.

1967

Treating central nervous system helps achieve first cures for the common childhood leukemia

Treating central nervous system helps achieve first cures for the common childhood leukemia

Researchers at St. Jude Children's Research Hospital first show that adding central nervous system radiation and intrathecal therapy (chemotherapy that is injected into the fluid-filled space between the layers of tissue that cover the brain and spinal cord) to standard chemotherapy, provides prolonged remissions and the first long-term cures for acute lymphocytic leukemia (ALL). ALL is the most common form of childhood leukemia.

1958

Combining chemotherapy drugs found to improve leukemia survival

Combining chemotherapy drugs found to improve leukemia survival

NCI scientists pioneer the use of combination chemotherapy – in which multiple drugs are administered together – after discovering that this approach leads to remissions in both children and adults with acute leukemia. Their findings set the stage for modern chemotherapy, in which drug combinations, dosing and scheduling have been carefully refined to maximize their effectiveness while minimizing side effects.

1955

U.S. government establishes network of researchers to pursue childhood cancer cures

U.S. government establishes network of researchers to pursue childhood cancer cures

The Clinical Trials Cooperative Group Program is established by the U.S. National Cancer Institute. This nationwide cancer research network soon becomes instrumental in testing the safety and benefits of promising new cancer therapies. The Children's Cancer Group, a key part of the new program, is tasked with developing new treatments for childhood cancers. Over the next 55 years, research conducted through the program helps to dramatically increase childhood cancer cure rates, from less than 10 percent in the 1950s to nearly 80 percent today.

1947

First-ever remission of pediatric leukemia

First-ever remission of pediatric leukemia

Sidney Farber, a physician at Children's Hospital Boston, achieves the first partial remission of pediatric leukemia in a 4-year-old girl using the drug aminopterin. He soon documents 10 cases of remission in a landmark scientific paper. Until this time, children with acute leukemia usually died within weeks of being diagnosed. While early remissions prove temporary, they pave the way for therapies that cure thousands of patients in the decades to come, allowing most childhood cancer patients to live long, healthy lives.