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Home Science & Environment Medical Research

New AI tool uses routine blood tests to predict immunotherapy response for many cancers

January 7, 2025
in Medical Research
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New AI tool uses routine blood tests to predict immunotherapy response for many cancers
Schematic of the study design and analysis. a, Cohort collection. Top: a real-world cohort (MSK-I) from MSKCC was used for model development. Middle: two real-world cohorts from MSKCC (MSK-II) and MSHS were used. Bottom: 10 global phase 3 clinical trials were used. ITT, intention-to-treat. b, Feature selection analysis. Top: number of features collected in the MSK-I cohort for model development. Bottom: 47 features were tested for the association with overall survival using the Cox proportional hazards regression or clinical benefit using the Cochran-Mantel-Haenszel test. Systemic therapy history was adjusted as a confounding factor in both tests. c, Machine learning analysis. Top: model construction with separate models for predicting overall survival and clinical benefit. Middle: model performance comparison using ROC and AUC (receiver operating characteristic and area under the receiver operating characteristic curve). Bottom: model performance evaluation. Among the two machine learning models, the one that performed the best on the hold-out test set was subjected to the analyses. Credit: Nature Medicine (2025). DOI: 10.1038/s41591-024-03398-5

Doctors around the world may soon have access to a new tool that could better predict whether individual cancer patients will benefit from immune checkpoint inhibitors—a type of immunotherapy—using only routine blood tests and clinical data.

The artificial intelligence–based model, dubbed SCORPIO, was developed by a team of researchers from Memorial Sloan Kettering Cancer Center (MSK) and the Tisch Cancer Institute at Mount Sinai.

The model is not only cheaper and more accessible, it’s significantly better at predicting outcomes than the two current biomarkers approved by the U.S. Food and Drug Administration (FDA), according to findings published in Nature Medicine.

“Immune checkpoint inhibitors are a very powerful tool against cancer, but they don’t yet work for most patients,” says study co-senior author Luc Morris, MD, a surgeon and research lab director at MSK. “These drugs are expensive, and they can come with serious side effects.”

So the key is patient selection—matching the drugs with patients who are most likely to benefit, Dr. Morris says.

“There are some existing tools that predict whether tumors will respond to these drugs, but they tend to rely on advanced genomic testing that is not widely available around the world,” he adds. “We wanted to develop a model that can help guide treatment decisions using widely available data, such as routine blood tests.”

Collaborating to make checkpoint inhibitor therapy work for more cancer patients

Checkpoint inhibitors target the immune system rather than the cancer itself. These drugs work by taking the brakes off immune cells, allowing them to better fight cancer. MSK clinicians and scientists played a key role in bringing the new class of drugs to patients.

The new study was jointly overseen by Dr. Morris and Diego Chowell, Ph.D., an Assistant Professor of Immunology and Immunotherapy, Oncological Sciences, and Artificial Intelligence and Human Health at the Icahn School of Medicine at Mount Sinai, and a former postdoctoral fellow at MSK.

Q&A with Dr. Morris

Memorial Sloan Kettering Cancer Center spoke with Dr. Morris about the team’s prediction model and next steps for the research.

Why did you develop this new model to predict checkpoint inhibitor response?

It was clear there was room for improvement.

There are currently two FDA-approved biomarkers for predicting response to checkpoint inhibitors: tumor mutational burden (the number of mutations in a tumor) and PD-L1 immunohistochemistry (evaluating the expression of the programmed death-ligand 1 protein in tumor samples).

Both require samples of the tumor to be collected. Meanwhile, genomic testing to assess mutations is expensive and not available everywhere, and there is a lot of variability evaluating PD-L1 expression.

Instead, our model relies on readily available clinical data, including routine blood tests performed in clinics around the world—the complete blood count and the comprehensive metabolic profile. We found that our model outperforms the currently used tests in the clinic.

The simplicity and affordability of this new approach could help ensure more equitable access to care while also reducing costs and helping ensure patients receive treatments most likely to benefit them individually—whether that ends up being a checkpoint inhibitor or some other type of therapy.

How was the model developed?

SCORPIO was initially developed by our team by collecting data from MSK patients, because of the length and depth of experience oncologists here have treating patients with these drugs. Collaborating with the team at Mount Sinai, we used a type of artificial intelligence called ensemble machine learning, which combines several tools to look for patterns in clinical data from blood tests and treatment outcomes.

The model was developed using a rich resource of retrospective data from more than 2,000 patients from MSK who had been treated with checkpoint inhibitors, representing 17 different types of cancer. The model was then tested using data from 2,100 additional MSK patients to verify that it was able to predict outcomes with high accuracy.

Next, we applied the model to nearly 4,500 patients treated with checkpoint inhibitors in 10 different phase 3 clinical trials from around the world.

Further validation was done with additional data from nearly 1,200 patients treated at Mount Sinai.

In total, the study included nearly 10,000 patients across 21 different cancer types—representing the largest dataset in cancer immunotherapy to date.

We did this extensive testing and validation because our goal was not just to develop a predictive model, but to develop one that would be widely applicable to patients and physicians in different locations.

What are the next steps?

We plan to collaborate with hospitals and cancer centers around the world to test the model with additional data from a wider variety of clinical settings. The feedback we receive will help us to continue to optimize the model.

Additionally, work is underway to develop an interface that is readily accessible by clinicians, regardless of where they’re located.

More information:
Seong-Keun Yoo et al, Prediction of checkpoint inhibitor immunotherapy efficacy for cancer using routine blood tests and clinical data, Nature Medicine (2025). DOI: 10.1038/s41591-024-03398-5

Provided by
Memorial Sloan Kettering Cancer Center


Citation:
New AI tool uses routine blood tests to predict immunotherapy response for many cancers (2025, January 7)
retrieved 7 January 2025
from https://medicalxpress.com/news/2025-01-ai-tool-routine-blood-immunotherapy.html

This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no
part may be reproduced without the written permission. The content is provided for information purposes only.



New AI tool uses routine blood tests to predict immunotherapy response for many cancers
Schematic of the study design and analysis. a, Cohort collection. Top: a real-world cohort (MSK-I) from MSKCC was used for model development. Middle: two real-world cohorts from MSKCC (MSK-II) and MSHS were used. Bottom: 10 global phase 3 clinical trials were used. ITT, intention-to-treat. b, Feature selection analysis. Top: number of features collected in the MSK-I cohort for model development. Bottom: 47 features were tested for the association with overall survival using the Cox proportional hazards regression or clinical benefit using the Cochran-Mantel-Haenszel test. Systemic therapy history was adjusted as a confounding factor in both tests. c, Machine learning analysis. Top: model construction with separate models for predicting overall survival and clinical benefit. Middle: model performance comparison using ROC and AUC (receiver operating characteristic and area under the receiver operating characteristic curve). Bottom: model performance evaluation. Among the two machine learning models, the one that performed the best on the hold-out test set was subjected to the analyses. Credit: Nature Medicine (2025). DOI: 10.1038/s41591-024-03398-5

Doctors around the world may soon have access to a new tool that could better predict whether individual cancer patients will benefit from immune checkpoint inhibitors—a type of immunotherapy—using only routine blood tests and clinical data.

The artificial intelligence–based model, dubbed SCORPIO, was developed by a team of researchers from Memorial Sloan Kettering Cancer Center (MSK) and the Tisch Cancer Institute at Mount Sinai.

The model is not only cheaper and more accessible, it’s significantly better at predicting outcomes than the two current biomarkers approved by the U.S. Food and Drug Administration (FDA), according to findings published in Nature Medicine.

“Immune checkpoint inhibitors are a very powerful tool against cancer, but they don’t yet work for most patients,” says study co-senior author Luc Morris, MD, a surgeon and research lab director at MSK. “These drugs are expensive, and they can come with serious side effects.”

So the key is patient selection—matching the drugs with patients who are most likely to benefit, Dr. Morris says.

“There are some existing tools that predict whether tumors will respond to these drugs, but they tend to rely on advanced genomic testing that is not widely available around the world,” he adds. “We wanted to develop a model that can help guide treatment decisions using widely available data, such as routine blood tests.”

Collaborating to make checkpoint inhibitor therapy work for more cancer patients

Checkpoint inhibitors target the immune system rather than the cancer itself. These drugs work by taking the brakes off immune cells, allowing them to better fight cancer. MSK clinicians and scientists played a key role in bringing the new class of drugs to patients.

The new study was jointly overseen by Dr. Morris and Diego Chowell, Ph.D., an Assistant Professor of Immunology and Immunotherapy, Oncological Sciences, and Artificial Intelligence and Human Health at the Icahn School of Medicine at Mount Sinai, and a former postdoctoral fellow at MSK.

Q&A with Dr. Morris

Memorial Sloan Kettering Cancer Center spoke with Dr. Morris about the team’s prediction model and next steps for the research.

Why did you develop this new model to predict checkpoint inhibitor response?

It was clear there was room for improvement.

There are currently two FDA-approved biomarkers for predicting response to checkpoint inhibitors: tumor mutational burden (the number of mutations in a tumor) and PD-L1 immunohistochemistry (evaluating the expression of the programmed death-ligand 1 protein in tumor samples).

Both require samples of the tumor to be collected. Meanwhile, genomic testing to assess mutations is expensive and not available everywhere, and there is a lot of variability evaluating PD-L1 expression.

Instead, our model relies on readily available clinical data, including routine blood tests performed in clinics around the world—the complete blood count and the comprehensive metabolic profile. We found that our model outperforms the currently used tests in the clinic.

The simplicity and affordability of this new approach could help ensure more equitable access to care while also reducing costs and helping ensure patients receive treatments most likely to benefit them individually—whether that ends up being a checkpoint inhibitor or some other type of therapy.

How was the model developed?

SCORPIO was initially developed by our team by collecting data from MSK patients, because of the length and depth of experience oncologists here have treating patients with these drugs. Collaborating with the team at Mount Sinai, we used a type of artificial intelligence called ensemble machine learning, which combines several tools to look for patterns in clinical data from blood tests and treatment outcomes.

The model was developed using a rich resource of retrospective data from more than 2,000 patients from MSK who had been treated with checkpoint inhibitors, representing 17 different types of cancer. The model was then tested using data from 2,100 additional MSK patients to verify that it was able to predict outcomes with high accuracy.

Next, we applied the model to nearly 4,500 patients treated with checkpoint inhibitors in 10 different phase 3 clinical trials from around the world.

Further validation was done with additional data from nearly 1,200 patients treated at Mount Sinai.

In total, the study included nearly 10,000 patients across 21 different cancer types—representing the largest dataset in cancer immunotherapy to date.

We did this extensive testing and validation because our goal was not just to develop a predictive model, but to develop one that would be widely applicable to patients and physicians in different locations.

What are the next steps?

We plan to collaborate with hospitals and cancer centers around the world to test the model with additional data from a wider variety of clinical settings. The feedback we receive will help us to continue to optimize the model.

Additionally, work is underway to develop an interface that is readily accessible by clinicians, regardless of where they’re located.

More information:
Seong-Keun Yoo et al, Prediction of checkpoint inhibitor immunotherapy efficacy for cancer using routine blood tests and clinical data, Nature Medicine (2025). DOI: 10.1038/s41591-024-03398-5

Provided by
Memorial Sloan Kettering Cancer Center


Citation:
New AI tool uses routine blood tests to predict immunotherapy response for many cancers (2025, January 7)
retrieved 7 January 2025
from https://medicalxpress.com/news/2025-01-ai-tool-routine-blood-immunotherapy.html

This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no
part may be reproduced without the written permission. The content is provided for information purposes only.


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