Rare Tumors in Children: Progress Through Collaboration

Alberto S Pappo1, Wayne L Furman2, Kris A Schultz2

  • 1Alberto S. Pappo and Wayne L. Furman, St Jude Children's Research Hospital, Memphis, TN; Kris A. Schultz, Children's Hospital of Minnesota, Minneapolis, MN; Andrea Ferrari, Instituto Nazionale Tumori, Milano, Italy; Lee Helman, National Cancer Institute Center for Cancer Research, Bethesda, MD; and Mark D. Krailo, Keck School of Medicine, Los Angeles, CA. alberto.pappo@stjude.org.

Insights

Investigating rare pediatric tumors is challenging due to their diversity. Current collaborative efforts show limited success, prompting exploration of new strategies like independent registries for studying childhood cancers.

Area of Science:

  • Pediatric Oncology
  • Rare Cancer Research
  • Clinical Trial Design

Background:

  • Rare pediatric tumors represent a significant challenge in childhood cancer research, accounting for approximately 10% of all pediatric malignancies.
  • The histological and clinical heterogeneity of these rare tumors complicates the study of their biological and clinical characteristics.
  • Existing national and international cooperative initiatives have faced limitations in advancing knowledge about rare pediatric cancers.

Purpose of the Study:

  • To review current national and international collaborative strategies for studying rare pediatric cancers.
  • To explore alternative mechanisms for enhancing research efforts in rare pediatric oncology.
  • To identify novel approaches for investigating the biology and clinical features of rare childhood tumors.

Main Methods:

  • Review of existing literature and initiatives from cooperative groups like the Children's Oncology Group and European Cooperative Study Group for Pediatric Rare Tumors.
  • Analysis of the effectiveness of current collaborative programs in rare pediatric cancer research.
  • Exploration of alternative study mechanisms, including independent registries and disease-specific clinics.

Main Results:

  • Current collaborative programs have demonstrated partial effectiveness in advancing the study of rare pediatric tumors.
  • There is a recognized need for alternative and enhanced strategies to overcome the limitations of existing collaborative efforts.
  • Independent registries and National Cancer Institute-sponsored clinics are being explored as promising alternative mechanisms.

Conclusions:

  • Enhanced collaborative strategies are crucial for advancing the understanding and treatment of rare pediatric cancers.
  • Alternative approaches such as independent registries and specialized clinics show potential for improving research outcomes.
  • Continued innovation in study design and collaboration is necessary to address the challenges posed by rare childhood tumors.

Related Concept Videos

Tumor Progression02:07

Tumor Progression

Tumor progression is a phenomenon where the pre-formed tumor acquires successive mutations to become clinically more aggressive and malignant. In the 1950s, Foulds first described the stepwise progression of cancer cells through successive stages.
Colon cancer is one of the best-documented examples of tumor progression. Early mutation in the APC gene in colon cells causes a small growth on the colon wall called a polyp. With time, this polyp grows into a benign, pre-cancerous tumor. Further...
7.9K
Tumor Progression02:07

Tumor Progression

3.6K
Treatment Resistant Cancers02:56

Treatment Resistant Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
3.9K
Treatment Resistent Cancers02:56

Treatment Resistent Cancers

1.5K
Rous Sarcoma Virus (RSV) and Cancer01:03

Rous Sarcoma Virus (RSV) and Cancer

Rous Sarcoma virus or RSV was discovered by F. Peyton Rous in the year 1911 as a filterable transmissible agent that could cause tumors in chickens. He won a Nobel Prize for this discovery in 1966. His experiments clearly demonstrated that some cancers could be caused by infectious agents and led to the discovery of many more cancer-causing viruses in animals as well as humans.
RSV is a retrovirus that contains two copies of a plus-strand  RNA genome. Its genome consists of four main open...
6.6K
Rous Sarcoma Virus (RSV) and Cancer01:03

Rous Sarcoma Virus (RSV) and Cancer

6.0K