Solving Kids' Cancer - every kid deserves to grow up

These 3 deadly childhood cancers are complex diseases that need special attention to solve

The broad term "cancer" comprises over 200 different genetically and molecularly variant diseases

Pediatric cancers are especially unique. Pediatric cancers:

• Are not preventable and do not result from lifestyle choices
• Are difficult to detect – children present with these cancers after they have already formed and often metastasized
• Are the leading cause of death by disease in children

There are currently no real solutions for these deadly childhood cancers

There has not been a significant breakthrough in the deadliest childhood cancers in 30 years

• One of the deadliest childhood cancers which has not seen therapeutic advancement is neuroblastoma
  • Is the most common cancer in infants
  • Because there is no system for detecting neuroblastoma, most children present at Stage 4 with metastases¹
  • Despite aggressive treatment, the survival rate of advanced stage neuroblastoma in children >1 year of age is only 30%^2,3
  • Current neuroblastoma relapse survival rate: 0-10% (rates vary by institution)

Therapies being used are often adult hand-me-down agents that are overly toxic for children. These agents are also not aimed at treating the unique pathology of children’s cancers

• Number of effective therapies available to all children with these deadly forms of cancer: 0

There is no standardized treatment for deadly childhood cancers — the only option children have for treatment is participation in clinical trials

Translational research transforms scientific discoveries arising from laboratory, clinical, or population studies into clinical applications to reduce cancer incidence, morbidity, and mortality.

The Translational Research Working Group (TRWG), National Cancer Institute (NCI)

The status quo is unacceptable

In today’s clinical research landscape, progress is too slow and promise too rare

• Average time for a therapeutic discovery to be tested in humans: 5 to 7 years

The possibility of discovering appropriate therapies can be limited by accepted research standards (eg, large scale, multicenter, single-treatment, placebo-controlled, narrow eligibility criteria)

Basic research generally receives more funding than translational or clinical research

• Existing ideas need to be identified, prioritized based on viability, and progressed into therapeutic development in humans

The usual players are not in the position to solve the problem alone

Pediatric oncologists are, justifiably, focused on treating their patients, not necessarily on developing and prioritizing new therapeutic discoveries

Research scientists and academic institutions have everyday demands which prevent them from investigating many promising basic research ideas

• Rewards in this field are given for scientific discoveries, not practical applications of these discoveries

Pharmaceutical and biotech companies are not incentivized financially to develop treatments in small populations such as children with cancer

Conventional nonprofit organizations are not necessarily designed to progress therapeutic development

Parents are forced to become experts in their child’s treatment

• With their child’s well-being as their first priority, parents bring a valuable perspective of ethics and a sense of urgency to clinical research

Children cannot advocate for themselves like adults with deadly or debilitating diseases can

To date, there has not been a single group, institution, or research entity effectively and solely dedicated to the task of solving kids’ cancer

Children need help now

Children suffering from these forms of cancer today are often forgotten about in the pursuit of a cure