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Published on: November 8, 2024
A Drosophila platform identifies a novel, personalized therapy for a patient with adenoid cystic carcinoma
Erdem Bangi1, Peter Smibert1, Andrew V Uzilov2,3
1Department of Cell, Development, and Regenerative Biology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Abstract:
Adenoid cystic carcinoma (ACC) is a rare cancer type that originates in the salivary glands. Tumors commonly invade along nerve tracks in the head and neck, making surgery challenging. Follow-up treatments for recurrence or metastasis including chemotherapy and targeted therapies have shown limited efficacy, emphasizing the need for new therapies. Here, we report a Drosophila-based therapeutic approach for a patient with advanced ACC disease. A patient-specific Drosophila transgenic line was developed to model the five major variants associated with the patient's disease. Robotics-based screening identified a three-drug cocktail-vorinostat, pindolol, tofacitinib-that rescued transgene-mediated lethality in the Drosophila patient-specific line. Patient treatment led to a sustained stabilization and a partial metabolic response of 12 months. Subsequent resistance was associated with new genomic amplifications and deletions. Given the lack of options for patients with ACC, our data suggest that this approach may prove useful for identifying novel therapeutic candidates.
Insights
This study developed a Drosophila model for adenoid cystic carcinoma (ACC) to screen for effective drugs. A three-drug combination therapy showed a 12-month stabilization in an advanced ACC patient.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Adenoid cystic carcinoma (ACC) is a rare salivary gland cancer with challenging surgical treatment.
- Current therapies for recurrent or metastatic ACC exhibit limited efficacy, highlighting an unmet therapeutic need.
Purpose of the Study:
- To establish a patient-specific Drosophila model for advanced adenoid cystic carcinoma.
- To identify a novel therapeutic strategy for adenoid cystic carcinoma using a Drosophila-based drug screening platform.
Main Methods:
- Development of a patient-specific Drosophila transgenic line modeling five major ACC variants.
- Robotics-based high-throughput screening to identify a synergistic drug combination.
- Administration of the identified drug cocktail to an advanced ACC patient.
Main Results:
- A three-drug cocktail (vorinostat, pindolol, tofacitinib) rescued transgene-mediated lethality in the Drosophila model.
- The patient exhibited a sustained 12-month stabilization and partial metabolic response.
- Acquired resistance was linked to new genomic amplifications and deletions.
Conclusions:
- A Drosophila-based therapeutic screening approach can identify potential treatments for adenoid cystic carcinoma.
- This strategy offers a promising avenue for discovering novel therapeutic candidates in rare cancers with limited options.
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