Adrenal Cancer: When Healing Goes Awry

The same cells that repair your body from stress can become the source of a rare cancer.

Rare Disease Medical Research Treatment Advances

The Silent Glands That Govern Our Health

Perched atop our kidneys like tiny triangular caps, the adrenal glands wield power far beyond their modest size. These crucial endocrine organs produce hormones that regulate essential bodily functions—from managing our stress response through cortisol to controlling blood pressure via aldosterone and influencing sexual characteristics with androgens. When adrenal cancer strikes, it disrupts this delicate hormonal balance, often with devastating consequences.

Despite its rarity—affecting only 0.5-2 people per million annually—adrenal cancer presents substantial challenges for patients and clinicians alike 4 . The cancer's heterogeneity means that outcomes vary dramatically between individuals, with some experiencing aggressive disease progression while others live with indolent tumors for years. Recent research has unveiled fascinating new insights into how these cancers begin, not from specialized hormone-producing cells, but from their supporting cast—the very cells tasked with maintenance and repair 1 . This article explores the latest advances in understanding, diagnosing, and treating this complex disease.

Incidence Rate

0.5-2

per million people annually

The Cellular Surprise: When Healers Become Harmful

For decades, scientists believed that the specialized cells producing adrenaline and noradrenaline in our adrenal glands were the primary suspects in cancer formation. But a groundbreaking 2025 study from Karolinska Institutet revealed a surprising twist: the true origin may lie in the gland's support cells known as sustentacular cells 1 .

These glial-like cells were found to act like stem cells after birth, regenerating hormone-producing tissue—a capacity previously thought to exist only during embryonic development. This regenerative ability, while essential for maintaining healthy tissue, creates a vulnerability where genetic mutations can redirect healing toward cancer formation 1 .

The Molecular Switch

The researchers identified a critical interaction between two proteins—DLK1 and NOTCH—that acts as a cellular switch, determining whether sustentacular cells generate healthy tissue or contribute to tumor formation 1 . This discovery is particularly significant because DLK1 has previously been identified as a target for immunotherapy in neuroblastoma, suggesting potential parallel treatment strategies for adrenal cancers 1 .

"In tumors with germline VHL mutations, subsets of these support cells showed loss of chromosome 3p, the 'second hit' leading to VHL inactivation. This suggests they may be the origin of certain tumors," explains Dr. Michael Mints, co-corresponding author of the study 1 .

Cellular Transformation Process
Normal Function

Sustentacular cells support and maintain healthy adrenal tissue

Regenerative Capacity

These cells demonstrate stem cell-like properties after birth

Genetic Mutation

Mutations in genes like VHL disrupt normal cellular regulation

Transformation

DLK1-NOTCH interaction malfunctions, leading to tumor formation

Decoding the Disease: Modern Diagnostic Approaches

Diagnosing adrenal tumors presents unique challenges due to their biological diversity and rarity. The diagnostic journey typically begins when patients present with symptoms or when incidentalomas (unexpectedly discovered adrenal masses) are found during imaging for other conditions.

Recognizing the Signs and Symptoms

Adrenal cancer symptoms generally fall into two categories: those caused by tumor growth and those resulting from hormone imbalances 3 .

General symptoms may include:
  • Persistent abdominal or back pain
  • Unexplained weight loss or decrease in appetite
  • A palpable lump or swelling in the abdomen
  • Generalized fatigue and weakness
Hormone-related symptoms vary based on which hormones are overproduced:
  • Cortisol excess: Weight gain, pink or purple stretch marks, muscle weakness
  • Aldosterone excess: High blood pressure, low potassium levels
  • Androgen excess: In women—excessive facial hair, acne; in children—early puberty
  • Estrogen excess: In men—breast enlargement, reduced libido 3 5
Advanced Diagnostic Technologies

Modern adrenal tumor diagnosis employs sophisticated laboratory techniques and imaging studies:

Biochemical profiling

now utilizes two parallel approaches:

  • Automated immunochemical methods (CLIA, ECLIA) that provide widely available, inexpensive screening tools
  • Advanced chromatographic techniques like liquid chromatography-mass spectrometry (LC-MS/MS) that can determine multiple hormone profiles during a single analysis 2
Molecular diagnostics

have revolutionized our understanding of adrenal tumors. Next-generation sequencing (NGS) has revealed that up to 35-40% of pheochromocytoma patients carry genetic variants, far higher than the previously estimated 10-15% 2 . Newly identified genes including CSDE1, H3F3A, MET, MERTK, and IRP1 have expanded our knowledge of the genetic basis of these tumors 2 .

Genetic Alterations in Adrenal Tumors
Tumor Type Key Genetic Alterations Clinical Implications
Pheochromocytoma CSDE1, H3F3A, MET, MERTK, IRP1 Up to 40% have germline variants, requiring genetic counseling
Aldosterone-producing adenomas KCNJ5, CACNA1D, ATP1A1, ATP2B3, CACNA1H 90% of APAs have somatic variants; treatment targeting possible
Primary bilateral macronodular adrenal hyperplasia ARMC5, KDM1A Enables early detection in relatives; 100% of GIP-responsive cases have KDM1A variants
Adrenocortical carcinoma TP53, BRD9, TERT, CTNNB1, CDK4, FLT4, MDM2 50% of advanced cases have potentially targetable alterations

Precision Medicine: Tailoring Treatment to the Tumor

The treatment landscape for adrenal cancer has evolved significantly from a one-size-fits-all approach to personalized strategies based on tumor characteristics, staging, and genetic markers.

Surgical Interventions

When detected early, complete surgical removal of the tumor offers the best chance for cure 5 . The complexity of surgery depends on tumor size, location, and involvement of surrounding structures. For localized adrenocortical carcinoma, complete resection is the primary goal, while for pheochromocytomas, careful preoperative management is crucial to prevent dangerous blood pressure fluctuations during surgery 2 .

Systemic Therapies

For advanced or metastatic disease, several treatment options exist:

  • Mitotane: This adrenolytic drug remains a cornerstone for treating adrenocortical carcinoma, both as adjuvant therapy after surgery and for advanced disease.
  • Cytotoxic chemotherapy: The combination of etoposide, doxorubicin, and cisplatin with mitotane is commonly used for aggressive adrenocortical carcinoma 4 .
  • Targeted therapies: Based on individual tumor genetics, targeted treatments are increasingly being explored, especially for tumors with specific molecular alterations 2 .

Emerging Prognostic Tools

Two novel approaches presented at the 2025 ESMO Sarcoma & Rare Cancers Congress show promise for predicting outcomes:

Circulating Tumor DNA (ctDNA) Detection

In metastatic pheochromocytomas and paragangliomas, ctDNA has proven to be a significant prognostic marker. Patients with detectable ctDNA tumor fraction had a 24-month overall survival rate of just 24%, compared to 85% for those without detectable ctDNA 4 .

Survival Comparison
With ctDNA:
24%
Without ctDNA:
85%
The BUCEN Scoring System

For adrenocortical carcinoma, the BUCEN system incorporates four easily measurable parameters:

  • Burden (based on tumor size and spread)
  • Cortisol excess
  • ECOG performance status
  • Neutrophil-to-lymphocyte ratio

This scoring system effectively stratifies patients by predicted outcomes and treatment responses, with scores of 3 or more associated with significantly shorter survival 4 .

The BUCEN Scoring System for Adrenocortical Carcinoma
Parameter Scoring Criteria Points
Tumor Burden Based on tumor size, local recurrence, and metastases 0, 1, or 2
Cortisol Excess Present or absent 1 if present
ECOG Performance Status 0, 1, or 2-3 0, 1, or 2 respectively
Neutrophil-to-Lymphocyte Ratio <5 or ≥5 1 if ≥5

Inside the Lab: Tracing Cancer to Its Origins

The groundbreaking discovery that sustentacular cells can initiate adrenal cancer emerged from meticulous research combining multiple advanced techniques. Let's examine the key experiment that revealed this unexpected connection.

Methodology: A Multi-Faceted Approach

The research team employed complementary methods to unravel the complex cellular relationships:

  1. Genetic tracing in mice: Specialized breeding techniques allowed researchers to label specific cell types and track their progeny over time.
  2. Single-cell sequencing of human tissue: This cutting-edge technology enabled researchers to analyze gene expression in individual cells.
  3. Comparative analysis: Researchers compared genetic data from VHL-related paragangliomas with healthy tissue 1 .
Results and Analysis

The experiments yielded several crucial findings:

  • Sustentacular cells demonstrate stem cell-like properties postnatally.
  • In patients with VHL mutations, subsets of sustentacular cells showed loss of chromosome 3p.
  • The DLK1-NOTCH interaction serves as a critical regulatory switch 1 .
Key Research Reagents and Their Applications
Research Tool Function/Application Research Context
Single-cell RNA sequencing Profiles gene expression in individual cells Identified cellular subtypes and lineages in healthy and tumor tissue
Genetic tracing models Tracks cell lineages and fate over time Demonstrated postnatal chromaffin cell regeneration from sustentacular cells
Immunostaining (SF-1, Ki-67) Identifies cell types and proliferation markers Differentiated adrenal cortical cells and measured proliferation rates
Plasma ctDNA analysis Detects tumor DNA fragments in blood Used as prognostic marker in metastatic pheochromocytoma/paraganglioma
LC-MS/MS Precise hormone measurement Enabled comprehensive steroid profiling for diagnosis
Future Horizons: Personalized Medicine and Novel Therapeutics

The evolving understanding of adrenal cancer biology is paving the way for more targeted and effective treatments. Several promising directions are emerging:

Targeting Developmental Pathways

The discovery of the DLK1-NOTCH interaction as a key switch suggests new therapeutic possibilities 1 .

Immunotherapeutic Approaches

Since DLK1 has been identified as an immunotherapeutic target in neuroblastoma, similar strategies might prove effective for adrenal cancers 1 .

Integrated Diagnostic Platforms

Combining imaging, biochemical profiling, genetic analysis, and liquid biopsies for comprehensive patient-specific profiles.

Conclusion: From Insights to Impact

The landscape of adrenal cancer research and treatment is transforming at an unprecedented pace. What was once a poorly understood rarity is now revealing its secrets, from the surprising origins of tumors in support cells to the genetic switches that determine healing or harm. These discoveries are not just academic exercises—they're paving the way for real clinical advances that offer hope to patients facing this challenging diagnosis.

As research continues to unravel the complexities of adrenal cancer, the medical community moves closer to a future where each patient's treatment is as unique as their tumor, where detection happens earlier, and where therapies are more effective and less toxic. The journey from scientific insight to clinical impact continues, powered by collaborations across institutions and countries that pool knowledge about these rare diseases. For patients and families affected by adrenal cancer, these advances promise a future with more options and better outcomes.

References