Intimal sarcoma · ultra-rare · under-researched

No one with this cancer should have to start from zero.

Intimal sarcoma is one of the rarest cancers there is.

So rare that there is no reliable natural-history data, no shared registry, and almost nothing to design a trial around. We are building the missing foundation: a structured, molecularly-linked base of real cases that turns scattered stories into something science can act on.

A patient-founded, non-profit initiative. We connect people and data to the centers already leading sarcoma research — we don't duplicate them.

Fewer than 1 in a million
Estimated incidence
Most oncologists never see a single case.
No registry
No dataset exists
No entity-specific natural-history dataset exists today.
Every case counts
Each one moves the field
At this rarity, each documented case measurably moves the field.
The problem

Rarity isn't just bad luck. It's a research dead-end — unless someone breaks it.

When a cancer is this rare, the usual machinery of medicine stalls. Cases are scattered across countries and clinics. No single center sees enough patients to draw conclusions. There are no reliable survival statistics, no map of how the disease recurs, no biobank of tissue to test drugs against. Funders and companies stay away because there is nothing to build a study on.

The result is that every new patient — and every treating physician — effectively starts from scratch. We think that is a solvable problem, and that the missing piece is not a breakthrough drug but organized information.

Our first case, published in full

We are asking people for their data. So here is ours first.

Below is the complete molecular profile of one intimal sarcoma of the pulmonary artery, completely resected, from a comprehensive next-generation sequencing panel. Identity, institution, dates and country are withheld; nothing else is.

One case proves nothing on its own. But the entire published molecular literature on this disease amounts to roughly 120 sequenced cases worldwide — so a single fully reported profile is not a footnote here. It is also the format we think every case deserves, and the starting point for a comparison we would like to run with you.

CASE IS-001

Intimal sarcoma, pulmonary artery

Complete (R0) resection · 517-gene DNA panel · 40% tumour content
  • MDM2 — amplified, 13.98 copies (12q15). The defining alteration of this entity; reported in about 71% of published cases. Expected
  • CDK4 — amplified, 12.83 copies (12q14.1). Frequent co-amplification; reported in 43–81% of cases. Expected
  • YAP1 — amplified, 27.53 copies (11q22.1). Discordant The highest amplification in this profile — higher than either of the two alterations that define the disease. Not listed as recurrent in any published intimal sarcoma series.
  • PDGFRA — not amplified (4q12). Discordant Against a reported background frequency of about 60%. PDGFRA amplification is within this panel's coverage, so this is a true negative and not a blind spot.
  • STAT6 — amplified, 9.88 copies (12q13.3). Consistent with a passenger on the 12q amplicon.
  • ELF3 — homozygous deletion, 0 copies (1q32.1). Not previously described in this entity.
  • Tumour mutational burden — 1.9 mutations per megabase. Low.
  • Genomic instability metric — 4%. Low.
  • Microsatellite status — MSS (stable).

Comparison frequencies from Koelsche et al., Modern Pathology 2021 (n=35, genome-wide copy number and methylation profiling): https://doi.org/10.1038/s41379-021-00874-y — This is a single case reported for comparison and hypothesis generation. It is not medical advice and carries no inference for any other patient.

What stands out

YAP1 is the loudest signal here. At 27.53 copies it exceeds both MDM2 and CDK4 — the two alterations that define the disease. YAP1 amplification is a documented driver in roughly 10% of soft-tissue sarcomas broadly, particularly dedifferentiated liposarcoma and undifferentiated pleomorphic sarcoma, but it does not appear as recurrent in intimal sarcoma series. Either it is genuinely rare here, or nobody has been looking for it.

PDGFRA is absent, and that matters. PDGFRA amplification is one of the three canonical alterations of this entity, present in about 60% of reported cases. Its absence here shifts the presumed driver landscape entirely onto cell-cycle control — and would argue against a PDGFR-directed approach in this particular case.

Copy-number driven, immunologically quiet. Low mutational burden, low genomic instability, microsatellite stable. A profile shaped by amplification rather than mutation. This is worth stating openly because it tempers optimism about single-agent immunotherapy in cases that look like this one.

Why we published this. Every rare-disease initiative asks patients to hand over their most sensitive information to strangers on the internet. It seems fair to go first. If this page is worth anything, it is worth something because it contains real numbers — and because the next case, and the one after that, make the comparison sharper.

→ See the full clinical course (Case IS-001)

Treatment timeline, metabolic imaging response, pathology, genomics and serial labs — one completely resected pulmonary artery intimal sarcoma. Identity, institution and country withheld.

The plan

From scattered cases to a trial-ready field — in six steps.

Each step is a concrete asset that makes the next one possible. We are not trying to run the science ourselves; we are building the foundation the leading centers need — and connecting it to the expert cohorts that already exist.

1

A shared case base

Real intimal sarcoma cases, documented in a consistent, consented format, so patterns become visible where today there is only anecdote.

2

Natural-history knowledge

How the disease actually behaves: where it recurs, what treatments were used, how people responded. The map that doesn't exist yet.

3

A genotype–phenotype picture

Linking each case to its molecular profile (MDM2 / CDK4 and beyond) to learn which subtypes might respond to which targeted approaches.

4

Tissue and models

Connecting cases to tissue, cell lines and models: a shared resource labs worldwide can use to test drugs the field cannot screen today.

5

A basis for real trials

The dataset and biobank that make a study designable and fundable, including inclusion of intimal sarcoma in broader molecularly-defined trials.

6

One connected, international effort

Feeding this foundation into the expert cohorts already leading the field, so the whole rare-disease community pulls in one direction.

One principle guides everything: connect, don't silo. The world already has brilliant sarcoma centers and cohorts. Our job is to be the connective tissue — to organize what patients know and hold, and channel it to the people who can turn it into treatments. Structured and consented, never scattered.

What you can do

Two questions we are actively trying to answer.

Both are answerable — but only by people who have been through this disease or who work on it. If either applies to you, one message genuinely moves this forward.

Have you lived with intimal sarcoma for more than three years?

If you, or someone in your family, is three or more years out from an intimal sarcoma diagnosis — we especially want to hear from you. In a disease where most published survival is measured in months, the people who did well are the most valuable information that exists, and almost none of it has ever been written down.

What was done, where, and in what order. That's it.

Tell us your course →

Do you have a molecular report? Send us your numbers.

You have just seen ours. If you or your relative has an NGS or FISH report for an intimal sarcoma, the amplifications and copy numbers on it are the single most useful thing you can share — no names, no records, just the values.

We will add it to the comparison and send you back what it looks like against every other case we hold.

Send your profile →

Clinician or researcher? If you have treated even one case, or your work touches MDM2/CDK4-amplified sarcoma, we would like to hear what you would do with material and funding. We would rather support the right experiment than commission the obvious one. contact@intimasarc.com

Non-profit initiative for intimal sarcoma. Built to organize knowledge and connect it to the centers advancing sarcoma research. This site provides information and community, not medical advice. Always discuss decisions with your own care team. No personal health data is collected without explicit consent.
Organisations & Resources

Where to turn, and who is doing the work.

Switzerland

Swiss Sarcoma Network — swiss-sarcoma.net
SAKK – Swiss Group for Clinical Cancer Research — sakk.ch
Krebsliga (Swiss Cancer League) — krebsliga.ch

International

Ultra Rare Sarcoma e.V. (Germany) — ultra-rare-sarcoma.de
Deutsche Sarkom-Stiftung — sarkome.de
SPAGN – Sarcoma Patient Advocacy Global Network — sarcoma-patients.org
Sarcoma UK — sarcoma.org.uk
Sarcoma Foundation of America — curesarcoma.org

Research & molecular diagnostics

NCT Heidelberg – Precision Oncology / MASTER Programme — nct-heidelberg.de
SARC – Sarcoma Alliance for Research through Collaboration — sarctrials.org

Patient Initiatives

Chordoma Foundation — chordomafoundation.org
Founded in 2007 by a chordoma patient and his mother. It built the field almost from nothing: a tissue bank, cell lines, drug screening, clinical trials. The clearest existing proof that a patient-founded organisation can turn an ultra-rare cancer into a researchable one, and now widely cited as a template for others.
Rare Cancer Research Foundation / pattern.org — rarecancer.org
Runs patient-initiated tissue donation: a patient can direct their own tumour tissue from surgery into research, and the foundation turns it into models that scientists can actually use. Relevant to anyone whose tissue is currently sitting in a hospital archive doing nothing.
osteosarc.com — osteosarc.com
A patient with osteosarcoma published his complete tumour data — sequencing, pathology, imaging — openly on the web so that anyone could analyse it. The most direct precedent for what we have done with Case IS-001.
Fibrolamellar Cancer Foundation — fibrofoundation.org
Founded by the family of a patient with fibrolamellar carcinoma, an ultra-rare liver cancer. Research it funded identified the fusion gene that drives the disease. The defining molecular discovery of that entity came out of a small patient foundation.
Every Cure — everycure.org
Founded by a physician who nearly died of Castleman disease and found his own treatment among drugs that were already approved for something else. It now searches existing medicines for unrecognised uses systematically. Relevant to any rare cancer where the most realistic near-term option is a drug that already exists.

Run or know of a patient-founded initiative in another ultra-rare cancer? Tell us and we will add it.