Raymond A. Wood Foundation

Cycle 2

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https://www.rawoodfoundation.org

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Childhood hypothalamic-pituitary brain tumors can affect the function of the pituitary gland and other nearby structures in the brain. Symptoms include gradual changes in vision, fatigue, excessive urination and headaches. Children with these tumors may grow slowly and may be smaller than expected.

Last updated 04/30/2026

Clinical

Disease Class

Endocrine diseases

Body Systems

Endocrine

Metabolic

Muscular / Skeletal

Nervous / Sensory

Renal / Urinary / Excretory

Reproductive

Organs

Brain

Known Genetic Link

No, the disease is not known to have a genetic basis

Causative Genes

None specified / unknown

Contributory Genes

None specified / unknown

Type of Inheritance

Not specified / unknown

Newborn Screening

Not applicable

Disease Mechanism(s)

Abnormal cell proliferation

Age of Onset

Adulthood (age 18-64)

Early childhood (age 1+-5)

Middle childhood (6-11)

Average Age at Diagnosis

Adulthood (age 18-64)

Early childhood (age 1+-5)

Middle childhood (6-11)

Life Expectancy

Elderly (age 65+)

Affected Sex(es)

Female

Intersex

Male

National Prevalence

1001-10000

Global Prevalence

10000+

National Incidence

Less than 10

Global Incidence

Less than 10

Populations and/or ancestry with higher prevalence

African-American in the US, certain African countries and Japan

Symptoms / Phenotypes

adipsia / hypodipsia

anxiety

balance problems

behavioral changes

excessive hunger / hyperphagia

growth delay / deficiency

headaches / migraines

intellectual disability

polydipsia

seizures / epilepsy

thermal dysregulation

vision problems

weight gain

Biomarkers

Diagnostic

· Yes for a type pf craniopharyngioma called papillary craniopharyngioma. However, the biomarker development is at the research stage. Lack of funding to validate that biomarker

Prognostic

· Imaging data can help for some prognostic

Therapeutic

· Yes there are some biomarkers to assess therapeutic efficacy for tumor treatments. Other biomarkers, essentially questionnaires serve as biomarkers for efficacy of drugs against hyperphagia. Other anthropometric measures such as weight or BMI for weight loss...

Existing Therapies

Regulatory Agency-Approved for Symptom Relief

Therapies in Development

Devices/medical equipment

· blood sodium meter

Dietary & metabolic therapies (medical food, dietary restriction, supplements, etc.)

Other (please specify)

· adjuvant therapy

Radiation therapy

· focused radiotherapy (e.g. proton therapy)

Repurposed drug

Small molecule therapy (novel small molecule drugs)

· Setmelanotide

Surgical & interventional

· Hypothalamic-sparing surgery

Therapeutic Development Stages

Awaiting final regulatory decision

Therapeutic Development Role

Meetings with regulators (e.g., FDA listening sessions, PFDD meetings)

Other

Outcome measures development

Recruitment and outreach to patients

Organizational & Research

Cell Lines

None

Cell Lines, Institution

None

Cell Lines, share

N/A

Disease Model

None

Disease Model, share

N/A

Organizational Challenges

Additional Human and funding resources would allow expanding on translational research efforts to advance the development of meaningful therapies

Clinical Trial Role

Focus group

Outcome measures, development

Recruitment and outreach, patients

Recruitment and outreach, trial sites/physicians

Results dissemination, publication

Study protocol design, review

Clinical Trial Types

Phase 2

Phase 3

Biobank, Institution

Not specified

Biobank, Involvement

Funded

Center of Excellence, Institution

None

Registry

Yes, we have a registry that we created

Data Collected, Registry

Imaging data

Longitudinal natural history data

Medication usage

Patient contact info

Patient-reported data

Data Entered by, Registry

Other

Patients

Platform, Registry

IAMRARE

Natural History Study

Yes, we have a natural history study that we created

Data Collected, Natural History Study

Imaging data

Medication usage

Prospective data

Platform, Natural History Study

IAMRARE

FDA Patient Listening Session

Yes

FDA Patient-Focused Drug Development (PFDD) Program

ICD Codes

We use an ICD-10 code capturing the family of diseases to which our disease belongs

Diagnostic Guidelines

Yes, we have guidance available on our website

Science Advisory Board Policies

Not Specified

Research Network Policies

Not specified

Patient Priority Survey

Yes

Patient Priority Survey, share

Yes, will share

Research Roadmap

Yes we have a Research Roadmap, and will share policies

None

Europe