Reviewing the scientific foundation and clinical validation of patient-derived organoid testing for cancer therapy selection
Our approach is built on decades of peer-reviewed research in organoid biology, functional drug testing, and precision oncology. Below you'll find the landmark studies that validate why functional testing works.
Why functional drug screening is moving from research labs to clinical decision-making
You have a patient with advanced cancer. Multiple treatment options exist. Genomic testing shows several mutations—but which drug will actually work?
Too often, the answer comes after months of ineffective treatment, disease progression, and diminished quality of life.
What if you could test dozens—or hundreds—of drugs against a patient's actual tumor cells before making a treatment decision?
That's the promise of patient-derived organoid testing. And it's no longer theoretical.
Here are seven landmark studies proving this approach works.
Study: Vlachogiannis et al., Science, 2018
What they did: Grew organoids from 71 patients with gastrointestinal cancers and tested them against standard chemotherapy regimens.
Why it matters:
This wasn't a small pilot—this was 71 real patients whose organoid results matched their clinical outcomes with remarkable precision. If the organoid said a drug wouldn't work, it didn't. Period.
"We can now test the tumor, not just sequence it." — Lead researcher
Study: Tiriac et al., Cancer Discovery, 2018
The setup: Direct comparison in pancreatic cancer—arguably one of the hardest cancers to treat.
Genomic mutations alone were poor predictors of chemotherapy response. Functional organoid testing identified sensitivities that sequencing completely missed.
Why it matters:
Your genomic report might show KRAS mutations or BRCA alterations. But does that mutation make the tumor sensitive to Drug X? Organoids answer that question definitively.
Study: van de Wetering et al., Cell, 2015
What they built: The first large-scale organoid biobank of colorectal cancer patients.
Why it matters:
This answered the scalability question. Yes, you can culture organoids reliably. Yes, you can screen hundreds of drugs. Yes, the results translate to the clinic.
OncoForma's 1,800+ drug panel? This study proved that kind of breadth is not only possible—it's practical.
Study: Boj et al., Cell, 2015
The expansion: Organoid testing validated across pancreatic, colorectal, and other GI cancers.
Why it matters:
This isn't a one-trick pony. Whether you're treating colon cancer or ovarian cancer, the biology works. The predictive power holds.
Study: Raufi et al., Cancer Discovery, 2023
The clinical trial: 75 patients with refractory cancers received organoid-guided therapy.
Why it matters:
This is the "it saves lives" study. Not just laboratory correlation—actual patients living longer because their doctors used functional testing.
When a colleague asks "does this actually help my patients?"—point them here.
Study: Yao et al., Cancers, 2022
What they analyzed: Every major organoid prediction study published to date.
Why it matters:
This is the reproducibility proof. Nineteen independent research groups, different cancers, different countries—and the same result: organoids predict outcomes.
Study: Huang et al., JAMA Oncology, 2021
The question: Is functional testing cost-effective?
Why it matters:
Payers care about this. Health systems care about this. Testing a tumor upfront costs $5,000-8,000. Giving 3 rounds of ineffective chemo? $150,000+.
The math isn't close.
Seven independent research groups. Thousands of patients. Multiple cancer types. The conclusion is consistent:
Organoid drug testing predicts clinical outcomes better than genomics alone.
The question is no longer "does this work?"
The question is: "Why aren't we using this for every patient?"
At OncoForma, we've built our platform on this foundation of evidence. Our approach:
We're not replacing your clinical judgment. We're giving you data that makes that judgment more informed.
Interested in how organoid testing could help your patients?
Questions about our clinical evidence? Contact our scientific team