In short
Sargramostim is being studied in clinical trials for several cancer-related uses. These trials look at safety, how well treatment works, and immune responses in people with cancers such as leukemia, breast cancer, lung cancer, mesothelioma, neuroblastoma, ovarian cancer, colorectal cancer, and pseudomyxoma peritonei.
Key points
- Sargramostim is being studied in several cancer clinical trials, not as a standalone drug description but as part of treatment combinations. The trials include early and later phases, from Phase 1 to Phase 4. They focus on cancers such as colorectal cancer, lung cancer, breast cancer, ovarian cancer, mesothelioma, acute myeloid leukemia, neuroblastoma, and pseudomyxoma peritonei. The studies measure safety, survival, cancer control, and immune response. Some trials are completed, while others are still authorised. Many of the studies test Sargramostim together with vaccines, immunotherapy, or other cancer medicines.
Overview of Sargramostim trials
The trial data show Sargramostim being used in cancer research across several different studies. It is usually tested as part of a combination approach, together with vaccines, immunotherapy, or other cancer treatments.
These studies are interventional trials, which means the research team gives a study treatment and then measures the results. The trials are designed to learn about safety, effectiveness, and immune response in people with specific cancers.
Conditions being studied
Sargramostim appears in trials for a wide range of cancers and cancer-related conditions. These include unresectable locally advanced or metastatic colorectal cancer, advanced or metastatic non-small cell lung cancer, breast cancer, ovarian cancer, malignant pleural mesothelioma, acute myeloid leukemia, high-risk neuroblastoma, and pseudomyxoma peritonei.
Some studies focus on people whose disease has returned, has not responded fully, or is at high risk of coming back. For example, one breast cancer study includes people with residual disease or high risk of recurrence after standard treatment.
Trial phases and what they mean
The listed studies include Phase 1, Phase 1/2, Phase 2, Phase 3, and Phase 4 trials. Early-phase studies mainly look at safety and the best treatment plan, while later-phase studies focus more on whether the treatment helps patients.
For example, the colorectal cancer study is Phase 1/2 and aims to define the recommended Phase 2 dose and the maximum tolerated dose, then test early efficacy. The breast cancer study is Phase 3 and compares outcomes in a larger group of patients at high risk of recurrence.
Main outcomes measured
The trials measure several important outcomes, depending on the cancer and phase. Common outcomes include progression-free survival, overall survival, objective response rate, safety, and immune response.
Safety outcomes include treatment-emergent adverse events, serious adverse events, dose-limiting toxicities, and treatment stops caused by side effects. Some studies also use imaging or standard response rules such as RECIST or modified RECIST to see whether the cancer is shrinking, stable, or growing.
In the pseudomyxoma peritonei study, researchers also measure T cell responses in blood and skin, which is a way to see how the immune system reacts to the treatment.
Who is being studied
The trials include adults with specific cancer types and, in some studies, very specific disease settings. Some participants have metastatic disease, meaning the cancer has spread, while others have disease that is unresectable, meaning it cannot be fully removed with surgery.
Several studies focus on people after first-line treatment or after standard therapy has already been given. One mesothelioma study looks at patients whose disease progressed after first-line platinum-based chemotherapy.
Key trial examples
In the colorectal cancer study, Sargramostim is part of the STC-1010 plus immunotherapy strategy with standard chemotherapy, and the main goals are safety in Phase 1 and 12-month progression-free survival in Phase IIa. This trial is authorised and plans to enroll 100 participants.
In the non-small cell lung cancer study, Sargramostim is given with UV1 vaccination, and the trial compares progression-free survival in patients with stage IIIB/IIIC or stage IV disease. This Phase 2 study is completed and enrolled 141 participants.
In the breast cancer study, Sargramostim appears in a Phase 3 trial of GLSI-100 for HER2/neu positive patients with residual disease or high risk of recurrence after trastuzumab-based therapy. The main outcome is invasive breast cancer-free survival, which tracks time until breast cancer comes back, spreads, or causes death.
In the ovarian cancer study, Sargramostim is part of a maintenance treatment comparison that looks at progression-free survival between treatment arms. This Phase 2 trial is authorised and plans to enroll 188 participants.
In the mesothelioma study, Sargramostim is used with nivolumab and ipilimumab, with or without UV1 vaccine, to study progression-free survival in inoperable malignant pleural mesothelioma. The study is Phase 4 and includes 118 participants.
In the acute myeloid leukemia trial, Sargramostim is listed among the study drugs in a Phase 3 comparison of Galinpepimut-S versus best available therapy, with median overall survival as the main outcome. The trial includes people in second or later complete remission, or complete remission with incomplete platelet recovery.
In the neuroblastoma study, Sargramostim is given with antibody treatment for high-risk patients with refractory bone or bone marrow disease, and the main outcome is objective response rate. This Phase 2 trial is authorised and plans to enroll 122 participants.
In the pseudomyxoma peritonei study, Sargramostim is part of a Phase 1 vaccine and immunotherapy approach that focuses on safety, tolerability, and immune activation. This is the smallest study listed, with 10 participants planned.
What these studies may add
Together, these trials show that Sargramostim is being explored mainly in cancer combination studies rather than as a single treatment. The research aims to learn whether adding Sargramostim can improve cancer control, support immune response, or help maintain remission in different patient groups.
The studies also show that patient groups are carefully selected, often by cancer type, stage, prior treatment, or risk of recurrence. This helps researchers compare results in people with similar medical situations.