Rare Oncology News

Spotlight On

Melanoma

Melanoma is a malignancy of pigment-producing cells (melanocytes) located predominantly in the skin, but also found in the eyes, ears, GI tract, leptomeninges, and oral and genital mucous membranes.

Prevalence

<1 / 100 000

< 100

US Estimated

< 100

Europe Estimated

Age of Onset

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ICD-10

C43.9

Inheritance

Autosomal dominant

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Autosomal recessive

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Mitochondrial/Multigenic

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X-linked dominant

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X-linked recessive

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5 Facts you should know

FACT

1

New cases of melanoma are increasing annually at almost 6%.
It is estimated that the number of new melanoma cases diagnosed in 2021 will increase by 5.8 percent.

FACT

2

Melanoma can be colorless.
While it’s true that many melanomas are dark brown to black in color, some melanomas have no color and appear as pink spots or bumps.

FACT

3

The majority of melanoma-related deaths occur in patients initially diagnosed with early-stage disease.
There are an estimated 130,000 patients diagnosed with invasive, non-metastatic cutaneous melanoma annually in the US. Most of these patients (estimated to be more than 100,000) are diagnosed with early-stage (Stage I-II) disease. Despite recent advances in treatment, the majority of melanoma-related deaths occur in patients initially thought to be low risk.

FACT

4

The false-negative rate of the SLNB procedure is reported to be between 5-21%.
While SLNB provides prognostic information the procedure has significant limitations including no survival benefit, low sensitivity, and a surgical complication rate of 11%.

FACT

5

Genomic testing more accurately identifies high-risk melanoma tumors.
When compared with traditional staging, genomic testing can provide a more accurate and personalized assessment of the risk of tumors metastasizing or recurring.

Melanoma is also known as...

Melanoma

Hereditary Cutaneous Melanoma; Familial Cutaneous Melanoma; Hereditary Cutaneous (Skin) Melanoma; Familial Melanoma

What’s your Rare IQ?

Approximately what % of melanoma patients undergoing an SLNB surgical procedure will receive a
negative result?

Common signs & symptoms

Nevus

Mole

Abnormal hair morphology

Abnormality of the hair

Abnormality of the lymphatic system

Dry skin

Freckling

Abnormality of extrapyramidal motor function

Neoplasm of the breast

Breast tumor

Retinopathy

Noninflammatory retina disease

Current treatments

The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.

Encorafenib + binimetinib

(Brand name: Braftovi + Mektovi) - Manufactured by Array BioPharma, Inc.
FDA-approved indication: June 2018, approved in combination for the treatment of patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation, as detected by an FDA-approved test.

Pembrolizumab

(Brand name: Keytruda) - Manufactured by Merck, Sharp & Dohme Corp
FDA-approved indication: Treatment of patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor.

Trametinib

(Brand name: Mekinist) - Manufactured by GlaxoSmithKline, LLC
FDA-approved indication: Treatment of patients with unresectable or metastatic melanoma with BRAF V600E or V600K mutations as detected by an FDA approved test.

Nivolumab

(Brand name: Opdivo) - Manufactured by Bristol-Myers Squibb Co
FDA-approved indication: Treatment of patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor. And treatment of patients with melanoma with involvement of lymph nodes or metestatic disease who have undergone complete resection.

Top Clinical Trials

TitleDescriptionPhasesStatusInterventionsMore Information
IO102-IO103 in Combination With Pembrolizumab Versus Pembrolizumab Alone in Advanced Melanoma (IOB-013 / KN-D18)Phase 3, multicenter, international, open-label, randomized, 2-arm trial investigating the safety and efficacy of IO102-IO103 in combination with pembrolizumab as first-line treatment for patients with previously untreated unresectable or metastatic (advanced) melanoma.Phase 3RecruitingDrug: IO102-IO103|Drug: PembrolizumabMore Info
Efficacy of Daromun Neoadjuvant Intratumoral Treatment in Clinical Stage IIIB/C Melanoma PatientsThe trial aims to evaluate the efficacy of Daromun neoadjuvant treatment followed by surgery and adjuvant therapy to improve in a statistically significant manner the recurrence-free survival (RFS) of Stage IIIB/C melanoma patients with respect to the standard of care (surgery and adjuvant therapy).Phase 3RecruitingDrug: Daromun|Procedure: Surgery|Drug: Adjuvant therapyMore Info
A Clinical Trial of Three Study Medicines (Encorafenib, Binimetinib, and Pembrolizumab) in Patients With Advanced or Metastatic MelanomaThe purpose of this study is to learn about the effects of three study medicines (encorafenib, binimetinib, and pembrolizumab) given together for the treatment of melanoma.Phase 3RecruitingDrug: Encorafenib|Drug: Binimetinib|Drug: PembrolizumabMore Info
Study to Compare Adjuvant Immunotherapy of Bempegaldesleukin Combined With Nivolumab Versus Nivolumab After Complete Resection of Melanoma in Patients at High Risk for RecurrenceThe main purpose of this study is to compare the efficacy of bempegaldesleukin plus nivolumab versus nivolumab in patients with completely resected Stage IIIA/B/C/D, or Stage IV cutaneous melanoma who are at high risk for recurrence.Phase 3RecruitingBiological: Bempegaldesleukin|Biological: NivolumabMore Info
CMP-001 in Combination With Nivolumab Compared to Nivolumab Monotherapy in Subjects With Advanced MelanomaCMP-001-011 is a Phase 2/3 study of CMP-001 intratumoral (IT) and nivolumab intravenous (IV) compared to nivolumab monotherapy administered to participants with unresectable or metastatic melanoma.Phase 2|Phase 3RecruitingDrug: CMP-001|Drug: NivolumabMore Info
Neoadjuvant Ipilimumab Plus Nivolumab Versus Standard Adjuvant Nivolumab in Macroscopic Stage III MelanomaThis phase II/III trial studies the side effects of nivolumab and ipilimumab when given together with or without sargramostim and to see how well they work in treating patients with stage III-IV melanoma that cannot be removed by surgeryPhase 3RecruitingDrug: Neoadjuvant ipilimumab + nivolumab|Drug: Adjuvant nivolumabMore Info
A Phase II/III Trial of Nivolumab, Ipilimumab, and GM-CSF in Patients With Advanced MelanomaThis phase II/III trial studies the side effects of nivolumab and ipilimumab when given together with or without sargramostim and to see how well they work in treating patients with stage III-IV melanoma that cannot be removed by surgery.Phase 2|Phase 3RecruitingBiological: Ipilimumab|Biological: Nivolumab|Biological: SargramostimMore Info
Evaluation of the Length of Treatment With PD-1/PD-L1 Inhibitors in Patients With Advanced Solid TumorsThe investigators are proposing a trial that will randomize patients who have disease stability to stop treatment at 1 year or continue treatment until disease progression. The investigators anticipate that the results of this study will answer questions regarding the optimal duration of treatment. therapy.Phase 3RecruitingDrug: Continue PD-1/PD-L1 Inhibitors treatment|Other: Discontinue PD-1/PD-L1-1 inhibitorMore Info

Top Treatments in Research

AgentClass/Mechanism of ActionDevelopment StatusCompanyClinical StudiesMore Information
IO102-IO103/PembrolizumabIO102-IO103 is an investigational cancer vaccine designed to target the immunosuppressive mechanisms mediated by key immunosuppressive proteins such as Indoleamine 2,3-dehydrogenase (IDO) and PD-L1.Pembrolizumab (Keytruda) is a humanized monoclonal antibody to programmed cell death receptor 1 (PD-1), which results in an increased immune reactivity that can break tolerance and is used in the immunotherapy of cancer.Phase 3IO BiotechMore InfoMore Info
DaromunDaromun (Philogen) is a combination of Darleukin, a human vascular targeting monoclonal antibody (L19) fused to interleukin-2 (IL-2), and Fibromun, the L19 antibody linked to tumor necrosis factor (TNF). Phase 3PhilogenMore InfoMore Info
Encorafenib/Binimetinib/PembrolizumabEncorafenib is a small molecule BRAF inhibitor that targets key enzymes in the MAPK signaling pathway. Binimetinib is an uncompetitive, small molecule inhibitor of selective mitogen-activated protein kinase (MEK1/2). .Pembrolizumab (Keytruda) is a humanized monoclonal antibody to programmed cell death receptor 1 (PD-1), which results in an increased immune reactivity that can break tolerance and is used in the immunotherapy of cancer.Phase 3PfizerMore InfoMore Info
Bempegaldesleukin/NivolumabBempegaldesleukin is a PEGylated interleukin-2 (IL-2) acting as a CD122-preferential IL-2 pathway agonist designed to activate and proliferate CD8 + T cells and NK cells. Nivolumab is a human recombinant monoclonal immunoglobulin G4 antibody to the programmed cell death receptor-1 (PD-1) which has distinctive immunomodulatory activity and is used in cancer immunotherapy.Phase 3Nektar TherapeuticsMore InfoMore Info
CMP-001/NivolumabAnti-Qβ-coated CMP-001 induces IFN-α production by pDCs which has secondary effects on a variety of cells including monocytes. Nivolumab is a human recombinant monoclonal immunoglobulin G4 antibody to the programmed cell death receptor-1 (PD-1) which has distinctive immunomodulatory activity and is used in cancer immunotherapy.Phase 2|Phase 3Checkmate PharmaceuticalsMore InfoMore Info
Neoadjuvant ipilimumab + nivolumab/Adjuvant nivolumabIpilimumab, is a monoclonal antibody medication that works to activate the immune system by targeting CTLA-4. Nivolumab is a human recombinant monoclonal immunoglobulin G4 antibody to the programmed cell death receptor-1 (PD-1) which has distinctive immunomodulatory activity and is used in cancer immunotherapy. Phase 3The Netherlands Cancer InstituteMore InfoMore Info
Ipilimumab/Nivolumab/SargramostimIpilimumab, is a monoclonal antibody medication that works to activate the immune system by targeting CTLA-4. Nivolumab is a human recombinant monoclonal immunoglobulin G4 antibody to the programmed cell death receptor-1 (PD-1) which has distinctive immunomodulatory activity and is used in cancer immunotherapy. Phase 2|Phase 3National Cancer Institute (NCI)More InfoMore Info
Continue PD-1/PD-L1 Inhibitors treatment/Discontinue PD-1/PD-L1-1 inhibitorPD-1 inhibitors and PD-L1 inhibitors are a group of checkpoint inhibitor anticancer drugs that block the activity of PD-1 and PDL1 immune checkpoint proteins present on the surface of cells.
Phase 3Antoinette J Wozniak, University of PittsburghMore InfoMore Info