Top GI News From September: GIST, Pancreatic and Colorectal Cancers

The top gastrointestinal (GI) cancer news from September highlighted major advances, including long-term results with Gleevec (imatinib) in gastrointestinal stromal tumors (GIST), an effective pancreatic cancer vaccine and new U.S. Food and Drug Administration (FDA) designations in pancreatic and colorectal cancer.

Long-Term Effectiveness of Gleevec in Advanced Gastrointestinal Stromal Tumors

For patients with advanced GIST, treatment with Gleevec has been shown to provide long-term effectiveness. Researchers reported that patients who underwent complete surgical removal (resection) of metastasis and achieved a complete response experienced a doubling of overall survival time.

Results from the phase 3 BFR14 study, which tracked outcomes for 20 years, were recently published in Annals of Oncology.

The BFR14 study, launched in 2002, enrolled 434 patients with advanced or unresectable GIST who received Gleevec. At a median follow-up of 219 months (about 18 years), the median overall survival was 75.3 months, or just over 6 years. Survival rates were 33.9% at 10 years, 19.8% at 15 years and 13.1% at 20 years.

ELI-002 Cancer Vaccine Shows Strong Immune Response in Pancreatic Cancer

An experimental off-the-shelf vaccine, ELI-002 7P, has demonstrated positive results for patients with pancreatic cancer. According to an announcement from Elicio Therapeutics, the vaccine generated strong mKRAS-specific T-cell responses in 89 of 90 evaluable patients, or about 99%.

The findings come from the phase 2 AMPLIFY-7P trial, which enrolled both minimal residual disease-positive and -negative patients. This included patients who either did or did not have very small amounts of cancer remaining that could be detected by a blood test.

“This vaccine works like most vaccines hopefully work, which is to engage and stimulate an immune response against a cancer protein,” explained Dr. Zev Wainberg in an interview with CURE. “That includes T-cells, B-cells, and other immune cells recognizing cancer cells as foreign and engaging against them.” Wainberg is the first author of a study on ELI-002 2P, another version of the vaccine, which was published in Nature Medicine.

FDA Grants Orphan Drug Designation to Utidelone for Pancreatic Cancer

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to utidelone for the treatment of pancreatic cancer, according to a news release from Biostar Pharma.

Utidelone, Biostar’s lead pipeline drug, had already received orphan drug designation for breast cancer brain metastases and gastric cancer.

At last year’s CSCO Annual Meeting, early results from a phase 2 trial testing utidelone with gemcitabine as a first-line treatment for advanced, unresectable pancreatic cancer showed encouraging findings. Among 11 patients who had completed their first evaluation, three achieved a partial response and five achieved stable disease. This resulted in an objective response rate of 27.27%, a disease control rate of 72.72%, and a median overall survival of 9.57 months.

The FDA grants orphan drug designation to drugs and biologics that aim to prevent, diagnose, or treat rare diseases. This status offers benefits for sponsors, including tax credits for clinical trials, exemptions from certain fees, and up to seven years of market exclusivity after approval.

FDA Grants Fast Track Designation to Alnodesertib for Colorectal Cancer

The FDA has also granted fast track designation to the ATR inhibitor alnodesertib in combination with low-dose irinotecan for the treatment of adult patients with ATM-negative metastatic colorectal cancer in the third-line setting, according to a news release from Artios.

The fast track program is designed to speed up the development and review of drugs that may address urgent medical needs for serious conditions.

This designation was based on results from the ongoing phase 1/2a STELLA study, which is testing alnodesertib plus irinotecan. In addition to showing benefits in third-line metastatic colorectal cancer, clinical responses were also seen in seven other solid tumor types with ATM deficiency. The combination so far has been well tolerated, shown a favorable safety profile, and appears suitable for long-term use.

References

1. “Gleevec Associated With Long-Term Effectiveness in Treating GIST” by CURE Media Group, September 2025.
2. “Vaccine Elicits Immune Responses in Pancreatic Cancer” by CURE Media Group, September 2025.
3. “FDA Grants Orphan Drug Designation to Utidelone in Pancreatic Cancer” by CURE Media Group, September 2025.
4. “Alnodesertib Wins FDA Fast Track Status in ATM-Negative Colorectal Cancer” by CURE Media Group, September 2025.

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