The Facts

What it Means to be Rare

While over 10,000 known rare diseases exist, only a fraction have effective treatments due to limited funding. Pediatric cancers, all considered rare, are particularly challenging, with diverse subtypes complicating treatment development and trial participation.

Amidst these obstacles, hope persists, and practical solutions for raising awareness and funding rare causes are within reach.

The Reality

The majority of pediatric cancer funding is for the largest population of those impacted by Leukemia and Lymphoma, which have the highest survival rates. Cancers with the lowest survival rates are given the least amount of national funding posing challenges to align with corporate pillars.

Low National Funding = Less Research, Less Clinical Trials & Less Treatment Options

Because all pediatric cancers are considered rare, developing treatments and finding enough patients to participate in clinical trials is difficult. Rare pediatric cancers affect the least amount and therefore have less awareness and funding from all sources.

The Rarity = Less Patients for Trials, Less Awareness & Less Money Raised

The reality of rare pediatric cancer may seem daunting. With organizations like ISF, there is hope.

Types of Rare Pediatric Cancer

Ewing sarcoma is a type of cancerous tumor that primarily affects the bones or the soft tissues surrounding bones. It most commonly arises in the legs, pelvis, ribs, arms or spine. Ewing sarcoma can metastasize (spread) to other parts of the body, including the lungs, bones and bone marrow.

Learn more about Ewing Sarcoma.

Neuroblastoma is a very rare type of cancerous tumor that originates from immature nerve cells called neuroblasts. While it can occur at any age, it’s most commonly diagnosed in early childhood.

This cancer can develop in various regions of the body including the chest, spine, spinal cord and abdomen, but it frequently originates in the adrenal glands located on top of the kidneys.

In some instances, neuroblastoma can be inherited, meaning it’s passed down through families.

Learn More about Neuroblastoma

Osteosarcoma is the most common type of bone cancer in children and adolescents. It is a malignant tumor that arises from bone cells, typically occurring in the wide ends of long bones such as the femur (thigh bone) and tibia (shin bone) in the lower limbs, as well as the humerus (upper arm bone) in the upper limbs.

One of the significant challenges with osteosarcoma is that it can metastasize, or spread, to other parts of the body. Approximately 15-20% of patients have metastatic disease at the time of diagnosis. The most common site of metastasis for osteosarcoma is the lungs, but it can also spread to other bones beyond the initial site of the tumor. 

Early detection and prompt treatment are essential to improve outcomes and reduce the risk of metastasis.

Learn More about Osteosarcoma.

Rhabdomyosarcoma is a type of cancerous tumor that develops in the soft tissues, particularly in muscles. It originates from immature cells that are destined to become skeletal muscle cells. It is the most common soft tissue sarcoma in children, but it can occur at any age.

There are two primary types of rhabdomyosarcoma:

  • Embryonal rhabdomyosarcoma is most commonly seen in children under the age of 5. It often affects areas such as the head and neck, bladder, vagina, prostate and testicles. 
  • Alveolar rhabdomyosarcoma can occur in individuals of all age groups and often affects the large muscles of the arms, legs and trunk.

Alveolar rhabdomyosarcoma tends to be more aggressive and may have a poorer prognosis compared to embryonal rhabdomyosarcoma.

Learn more about Rhabdomyosarcoma.

Wilms tumor is a relatively rare cancer, but it is the most common type of kidney cancer in children.

Learn more about Wilms Tumor.

Desmoid tumors (also known as aggressive fibromatosis) are considered non cancerous, meaning they do not metastasize to distant parts of the body like cancerous tumors do. However, they can still be locally aggressive, meaning they can invade nearby tissues and structures and cause significant morbidity.

Desmoid tumors arise from the connective tissue, specifically from cells called fibroblasts. These tumors can develop anywhere in the body where there is connective tissue, but they are most commonly found in the abdominal wall, arms, legs and sometimes in the chest wall.

Learn more about Desmoid Tumors.

DIPG, or diffuse intrinsic pontine glioma, is a rare, fast-growing brain tumor that develops in the pons, a part of the brainstem that controls vital functions like breathing and heart rate. This aggressive cancer primarily presents with symptoms such as difficulty walking, cranial nerve palsies, and eventually impacting critical bodily functions.

Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive type of cancer that primarily affects children, adolescents and young adults. It is characterized by the formation of small, round, blue cells surrounded by dense fibrous tissue (desmoplasia).

DSRCT most commonly arises in the abdomen or pelvis, although it can also occur in other locations such as the chest, extremities or retroperitoneum (area behind the abdominal cavity). The tumor typically arises from the lining of the abdominal cavity (peritoneum) and may involve multiple organs within the abdomen and pelvis.

Learn more about Desmoplastic small round cell tumor.

Hepatoblastoma is a type of liver cancer that primarily affects infants and young children, typically up to the age of 3. Hepatoblastoma arises from immature liver cells called hepatoblasts, which are involved in liver development during fetal development.

Lear more about Hepatoblastoma.

Adrenal cortical carcinoma (ACC) is a rare type of childhood cancer that originates in the adrenal cortex, which is the outer layer of the adrenal glands. 

Adrenal Glands: The adrenal glands are small, triangular-shaped glands located on top of each kidney. They produce hormones that regulate various bodily functions, including metabolism, blood pressure and response to stress. The adrenal cortex, specifically, produces hormones such as cortisol, aldosterone and androgens.

Learn more about Adrenal Cortical Carcinoma.

Soft tissue sarcoma is a rare type of cancer that originates in the soft tissues of the body. Soft tissues include muscles, tendons, fat, blood vessels, nerves and connective tissues. Soft tissue sarcoma can occur in any part of the body, but it most commonly affects the arms, legs, trunk or abdomen.

Learn more about Soft Tissue Sarcoma.

Families at the Forefront