Very Rare Cancer Consortium


Very Rare Cancer Consortium (VRCC): A New York Genome Center multi-institutional initiative to investigate the molecular drivers of rare disease.


While tremendous progress has been made in curing many common cancers, treatment options still remain especially limited for those diagnosed with very rare cancers.  As less than one in one million people are diagnosed with very rare tumors, individual medical centers see too few patients to investigate the driving causes of the disease. As a result, most of these diseases are poorly studied and novel treatments have not been devised or tested.

The Very Rare Cancer Consortium (VRCC) aims to leverage the combined expertise from multiple institutions across New York City to advance efforts to improve research for very rare cancers. Through a network of 20 member institutions, this project will help to collect samples of very rare cancers in sufficient numbers to provide answers to important scientific questions, build research cohorts, and reduce the toll taken by understudied diseases.


The Very Rare Cancer Consortium (VRCC) will conduct genomic analyses on very rare cancer cohorts for retrospective and prospective cases, potentially producing insights to inform treatments for patients. The goal is ultimately to identify novel treatments or new indicators for already approved therapies.

Recent advances in genomic sequencing technologies have revolutionized our understanding of tumor biology and impacted the prevention, risk assessment, early detection, diagnosis, and treatment of disease. As the first institution in New York State to receive Department of Health approval to offer a comprehensive whole genome and transcriptome oncology clinical test, the New York Genome Center (NYGC) is uniquely positioned to investigate the molecular drivers of very rare cancer types. Our unified group of world-renowned clinicians and researchers across NYC collectively seeking to pinpoint the genetic cause for rare diseases.

What is a “Very Rare” Cancer?

  • Cancers occurring in unusual locations in the body
  • Cancers occurring in unusual ethnic groups
  • Cancers occurring at an unusual age
  • Cancers due to an unusual environmental
  • Cancers with very unusual clinical course
  • Cancers occurring in unusual gender

This innovative initiative aims to effectively acquire and comprehensively study rare cancers through a network of interacting partners.

  • The VRCC Has An Unprecedented Capacity To Study Very Rare Tumors In Large Enough Cohorts To Enable Informed Genomic Testing.
  • Studies In Phase I Of This Project Will Rapidly Profile Rare Cancers From Already Available Samples And Generate Disease-Specific Datasets.
  • Prospective Recruitment Of Patients With Suspected Cases Of These Tumors Will Allow For Molecular Diagnosis To Better Inform Treatment In Phase II.
  • The NYGC Is Uniquely Qualified To Generate And Analyze A Unique Multimodal Dataset, Linking Genomics To Clinical And Functional Data.

The consortium, with the Genome Center Cancer Group (GCCG), has identified over 15 rare and understudied tumor types with poor treatment options. It will initially focus on three cohorts as a sufficient number of stored samples were already identified throughout several NYC institutions.  The New York Genome Center will build on the framework established in the Polyethnic-1000 study, including: a network of clinicians, a city-wide infrastructure for sample collection, and an institutional review board (IRB)-approved protocol to guarantee patient protection.

Initial studies include but are not limited to: Epithelioid hemangioendothelioma, Metaplastic breast cancer, Choroid plexus carcinoma, Appendix cancers, Renal medullary carcinoma, Adult Wilms tumors, Adult neuroblastoma,  Juvenile granulosa tumors, Breast implant-associated lymphomas, Malignant Sertoli/Leydig cell tumor, Malignant PEComa, Male breast cancer, Neurofibromatosis Type 1 (NF1), and Histiocytic Sarcoma.

The NYGC aims to partner with institutions both nationally and internationally to acquire, and collectively investigate more of these very rare samples. Additional profiling methods will be added to increase the number of cases sequenced, and we plan to create a deeper engagement with the patient advocacy community.