"N of 1": First Basket Study Delivers on Promise of Precision Medicine
The confluence of the basket study design and genomic sequencing is speeding delivery of targeted treatments to patients underrepresented in trials.
Ivette G. initially attributed her nausea and belly pain to eating food she was unaccustomed to while visiting her native Peru, then her expanding waistline to winter weight gain.
But when her abdominal and back pain grew stronger than ibuprofen could relieve, she met with her obstetrician/gynecologist. Referred for an ultrasound, results of additional scans pointed to ovarian cancer that had metastasized to the liver, peritoneal cavity, and lymph nodes.
She was 2 days shy of her 37th birthday and had recently given birth to a boy.
Scheduled for a hysterectomy, a friend of her husband's urged her to get a second opinion. Three second opinions, a negative endoscopy, a negative colonoscopy, and a liver biopsy later, Ivette was diagnosed with stage 4 pancreatic neuroendocrine tumor (NET). Her oncologist at Mount Sinai Beth Israel in New York City started her on oxaliplatin, bevacizumab, 5-FU, and leucovorin.
An oncologist at Memorial Sloan Kettering Cancer Center (MSK) who specializes in NETs confirmed her chemotherapy regimen was state-of-the-art.
Ultimately, after Ivette's tumor was sequenced and identified as positive for the BRAF V600 mutation, she was enrolled in MSK's “basket” study and was started on vemurafenib, four pills twice daily.
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Her tumor volume declined by 25%. Due to rash and joint pain, her dose was reduced to 3 pills twice daily. Ivette, now 40 years old, is completing her fifth cycle of vemurafenib and her tumor remains stable.
Excited about the possibilities this treatment is opening up, she told Cancer Therapy Advisor she is encouraging other patients with NETs to have their tumors sequenced. “There is hope,” she said. “Physicians are really trying hard to find out the best way to treat people.”