Dr. Bui is a Clinical Assistant Professor of Medicine at the Stanford Cancer Institute and a specialist in the Sarcoma and Developmental Therapeutics programs. Dr. Bui earned an undergraduate degree in Computer Science at Stanford University and went on to earn his medical degree from the University of Texas Southwestern Medical Center. He completed Internal Medicine residency at Stanford Hospital and Hematology/Oncology fellowship at the University of California San Diego, where he performed extensive research in bioinformatics to analyze tumor sequencing data. His research background and interests are in the field of bioinformatics as applied to large data sets and the study of novel compounds in rare malignancies.

Clinical Focus

  • Medical Oncology
  • Sarcoma
  • Phase 1 Trials

Academic Appointments

Boards, Advisory Committees, Professional Organizations

  • Member, American Society of Clinical Oncology (ASCO) (2014 - Present)
  • Associate Member, Stanford Cancer Institute (2018 - Present)
  • Member, Connective Tissue Oncology Society (CTOS) (2017 - Present)

Professional Education

  • Residency: Stanford University Internal Medicine Residency (2014) CA
  • Board Certification: American Board of Internal Medicine, Medical Oncology (2017)
  • Fellowship: UCSD Hematology and Oncology Training (2017) CA
  • Board Certification: American Board of Internal Medicine, Hematology (2017)
  • Medical Education: University of Texas Southwestern Medical School Registrar (2011) TX
  • Board Certification: American Board of Internal Medicine, Internal Medicine (2014)
  • B.S., Stanford University, Computer Science (2007)

Clinical Trials

  • A Study of AL102 in Patients With Progressing Desmoid Tumors Recruiting

    The current study is designed to evaluate the efficacy and safety of AL102 in patients with progressive desmoid tumors.

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  • Phase 1 Study of SQ3370 in Patients With Advanced Solid Tumors Recruiting

    The purpose of this study is to evaluate the safety, tolerability, and preliminary activity of SQ3370 in patients with advanced solid tumors.

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  • A Phase II Clinical Trial Evaluating the Combination of Olaparib and Temozolomide for the Treatment of Advanced Uterine Leiomyosarcoma Not Recruiting

    This phase II trial studies olaparib and temozolomide in treating patients with uterine leiomyosarcoma (LMS) that has spread to other places in the body (advanced or metastatic) or cannot be removed by surgery (unresectable). PARPs are proteins that help repair DNA mutations. PARP inhibitors, such as olaparib, can keep PARP from working, so tumor cells can't repair themselves, and they may stop growing. Chemotherapy drugs, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving olaparib and temozolomide may work better than giving either drug alone in treating patients with LMS.

    Stanford is currently not accepting patients for this trial. For more information, please contact Site Public Contact, 650-498-7061.

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  • A Study to Test the Safety of the Investigational Drug Selitrectinib in Children and Adults That May Treat Cancer Not Recruiting

    This research study is done to test the safety of the new drug selitrectinib in children and adults with cancer having a change in a particular gene (NTRK1, NTRK2 or NTRK3). The drug may treat cancer by interfering with the effect of the NTRK genes on cancer growth. The study also investigates how the drug is absorbed and processed in the human body, and how well and for how long the cancer responds to the drug. This is the first study to test selitrectinib in humans with cancer, for whom no other effective therapy exists.

    Stanford is currently not accepting patients for this trial. For more information, please contact Cancer Clinical Trials Office (CCTO), 650-498-7061.

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  • ADI-PEG 20 in Combination With Gemcitabine and Docetaxel for the Treatment of Soft Tissue Sarcoma, Osteosarcoma, Ewing's Sarcoma, and Small Cell Lung Cancer Not Recruiting

    The investigators have recently demonstrated that argininosuccinate synthase 1 (ASS1) expression is silenced in 88% of all sarcomas (n=708), and that this loss is associated with a decreased overall survival. Using the extracellular arginine depleting enzyme PEGylated arginine deiminase (ADI-PEG20), an extracellular arginine depleting enzyme, the investigators demonstrated ADI-PEG20 induces a prosurvival metabolic reprogramming in ASS1-deficient sarcomas that redirects glucose into the serine/folate pathway directing the carbons from glucose into pyrimidine biosynthesis, thus sensitizing cells to death by the pyrimidine antimetabolite gemcitabine by using metabolomics. The synthetic lethality was increased by the addition of docetaxel. Therefore a phase II clinical trial of ADI with gemcitabine and docetaxel, a standard second line therapy for soft tissue sarcoma will be conducted to determine if the clinical benefit rate of gemcitabine and docetaxel is improved by the metabolic changes induced by ADI-PEG20. Recently published data shows that priming ASS1-deficient tumors with ADI-PEG 20 and docetaxel improves the effect of gemcitabine. Therefore, a cohort of patients consisting of ten patients diagnosed with either osteosarcoma or Ewing's sarcoma (ideally five of each), and five patients diagnosed with small cell lung cancer will be included as an exploratory cohort. Enrollment to Cohort 2 will occur concurrently with Cohort 1.

    Stanford is currently not accepting patients for this trial. For more information, please contact Nam Bui, M.D., 713-412-8721.

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  • Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) Not Recruiting

    This study evaluates nirogacestat in the treatment of desmoid tumor/aggressive fibromatosis (DT/AF). Half of the participants will receive nirogacestat while the other half will receive placebo.

    Stanford is currently not accepting patients for this trial.

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  • Olaparib in Treating Patients With Advanced Glioma, Cholangiocarcinoma, or Solid Tumors With IDH1 or IDH2 Mutations Not Recruiting

    This phase II trial studies how well olaparib works in treating patients with glioma, cholangiocarcinoma, or solid tumors with IDH1 or IDH2 mutations that have spread to other places in the body (metastatic) and usually cannot be cured or controlled with treatment (refractory). Olaparib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

    Stanford is currently not accepting patients for this trial. For more information, please contact Cancer Clinical Trials Office (CCTO), 650-498-7061.

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  • Phase III Trial of Anlotinib, Catequentinib in Advanced Alveolar Soft Part Sarcoma, Leiomyosarcoma, Synovial Sarcoma (APROMISS) Not Recruiting

    THIS STUDY IS CURRENTLY RECRUITING PATIENTS WITH ALVEOLAR SOFT PART SARCOMA ONLY AND IS NO LONGER RECRUITING PATIENTS WITH SYNOVIAL SARCOMA OR LEIOMYOSARCOMA. This study evaluates the safety and efficacy of AL3818 (anlotinib) hydrochloride in the treatment of metastatic or advanced alveolar soft part sarcoma (ASPS), leiomyosarcoma (LMS), and synovial sarcoma (SS). All participants with ASPS will receive open-label AL3818. In participants with LMS or SS, AL3818 will be compared to IV dacarbazine. Two-thirds of the participants will receive AL3818, one-third of the participants will receive IV dacarbazine.

    Stanford is currently not accepting patients for this trial.

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All Publications

  • Use of a computer model and care coaches to increase advance care planning conversations for patients with metastatic cancer Gupta, D., Fardeen, T., Teuteberg, W., Seevaratnam, B., Asuncion, M., Alves, N., Rogers, B., Neal, J. W., Fan, A. C., Parikh, D., Patel, M. I., Shah, S., Srinivas, S., Huang, J. E., Reddy, S. A., Ganjoo, K. N., Bui, N., Hansen, J., Gensheimer, M. F., Ramchandran, K. LIPPINCOTT WILLIAMS & WILKINS. 2021
  • Phase 1 trial of SQ3370 in solid tumors. Guminski, A., Wang, D., Bui, N., Bhadri, V., De Silva, M., Steffner, R., Yee, N. A., Srinivasan, S., Oneto, J., Saville, M., Subbiah, V. AMER ASSOC CANCER RESEARCH. 2021
  • Phase II trial of pegylated arginine deiminase in combination with gemcitabine and docetaxel for the treatment of soft tissue sarcoma. Van Tine, B., Hirbe, A. C., Luo, J., Oppelt, P., Weiss, M. C., Eulo, V., Toeniskoetter, J., Haarberg, S., Abaricia, S., Ruff, T., Bomalaski, J. S., Johnston, A., Kuo, C., Shiu, C., Ingham, M., Bui, N., Chawla, S. P., Schwartz, G. K., Ganjoo, K. N. LIPPINCOTT WILLIAMS & WILKINS. 2021
  • NCI protocol 10250: A phase II study of temozolomide and olaparib for the treatment of advanced uterine leiomyosarcoma. Ingham, M., Allred, J. B., Gano, K., George, S., Attia, S., Burgess, M., Seetharam, M., Boikos, S., Bui, N., Chen, J., Close, J., Cote, G., Thaker, P. H., Ivy, S., Das, B., Shapiro, G., Kochupurakkal, B., Trepel, J. B., Pommier, Y., Schwartz, G. K. LIPPINCOTT WILLIAMS & WILKINS. 2021
  • Prognostic relevance of the hexosamine biosynthesis pathway activation in leiomyosarcoma. NPJ genomic medicine Tolwani, A., Matusiak, M., Bui, N., Forgo, E., Varma, S., Baratto, L., Iagaru, A., Lazar, A. J., van de Rijn, M., Przybyl, J. 2021; 6 (1): 30


    Metabolic reprogramming of tumor cells and the increase of glucose uptake is one of the hallmarks of cancer. In order to identify metabolic pathways activated in leiomyosarcoma (LMS), we analyzed transcriptomic profiles of distinct subtypes of LMS in several datasets. Primary, recurrent and metastatic tumors in the subtype 2 of LMS showed consistent enrichment of genes involved in hexosamine biosynthesis pathway (HBP). We demonstrated that glutamine-fructose-6-phosphate transaminase 2 (GFPT2), the rate-limiting enzyme in HBP, is expressed on protein level in a subset of LMS and the expression of this enzyme is frequently retained in patient-matched primary and metastatic tumors. In a new independent cohort of 327 patients, we showed that GFPT2 is associated with poor outcome of uterine LMS but not extra-uterine LMS. Based on the analysis of a small group of patients studied by 18F-FDG-PET imaging, we propose that strong expression of GFPT2 in primary LMS may be associated with high metabolic activity. Our data suggest that HBP is a potential new therapeutic target in one of the subtypes of LMS.

    View details for DOI 10.1038/s41525-021-00193-w

    View details for PubMedID 33941787

  • Uterine Leiomyosarcoma with FN1-Anaplastic Lymphoma Kinase Fusion Responsive to Alectinib and Lorlatinib. Case reports in oncology Testa, S., Million, L., Longacre, T., Bui, N. 2021; 14 (2): 812-819


    Uterine leiomyosarcoma (LMS) is a rare malignant neoplasm of the female genital tract poorly responsive to conventional chemotherapy and radiotherapy, with an overall poor prognosis. Pazopanib is at the moment the only FDA-approved targeted molecular therapy for uterine LMS, given the exceedingly rare occurrence of actionable genetic mutations in this type of cancer. Here, we describe the first reported case of metastatic uterine LMS with an FN1-anaplastic lymphoma kinase (ALK) fusion mutation occurring in a 63-year-old woman with a history of uterine leiomyomas. The patient progressed on several lines of therapy, including conventional chemotherapy, pazopanib, and the first-generation ALK inhibitor crizotinib. Interestingly, the patient showed a remarkable 16-month response to second generation ALK inhibitors alectinib and lorlatinib. This case demonstrates that ALK inhibitors can be an effective therapeutic strategy for patients with ALK fusion-positive uterine LMS that has progressed on conventional chemotherapy.

    View details for DOI 10.1159/000516758

    View details for PubMedID 34248545

  • Evolving role of entrectinib in treatment of NTRK-positive tumors. Future oncology (London, England) Chawla, N., Bui, N. Q., Seetharam, M. 2021


    Targeted therapy has shown to be a very effective treatment in tumors with specific genomic drivers. Trkhas proven to be one such target. Efforts to target the Trk fusion with specific inhibitors have shown remarkable responses in a tumor agnostic fashion, with responses seen even in patients with intracranial metastasis. Entrectinib is a first-generation Trk inhibitor with impressive activity in early phase trials performed in patients with NTRK fusion positive solid tumors and ROS1 positive non-small-cell lung cancers with subsequent approval for those indications. Entrectinib was also found to be effective in treatment of brain metastasis and generally well tolerated.

    View details for DOI 10.2217/fon-2020-0936

    View details for PubMedID 33896226

  • Evaluation of Absolute Lymphocyte Count at Diagnosis and Mortality Among Patients With Localized Bone or Soft Tissue Sarcoma. JAMA network open Brewster, R., Purington, N., Henry, S., Wood, D., Ganjoo, K., Bui, N. 2021; 4 (3): e210845


    Importance: Host-related immune factors have been implicated in the development and progression of diverse malignant neoplasms. Identifying associations between immunologic laboratory parameters and overall survival may inform novel prognostic biomarkers and mechanisms of antitumor immunity in localized bone and soft tissue sarcoma.Objective: To assess whether lymphopenia at diagnosis is associated with overall survival among patients with localized bone and soft tissue sarcoma.Design, Setting, and Participants: This retrospective cohort study analyzed patients from the Stanford Cancer Institute with localized bone and soft tissue sarcoma between September 1, 1998, and November 1, 2018. Patients were included if laboratory values were available within 60 days of diagnosis and, if applicable, prior to the initiation of chemotherapy and/or radiotherapy. Statistical analysis was performed from January 1, 2019, to November 1, 2020.Exposures: Absolute lymphocyte count within 60 days of diagnosis and antimicrobial exposure, defined by the number of antimicrobial agent prescriptions and the cumulative duration of antimicrobial administration within 60 days of diagnosis.Main Outcomes and Measures: The association between minimum absolute lymphocyte count at diagnosis and 5-year overall survival probability was characterized with the Kaplan-Meier method and multivariate Cox proportional hazards regression models. Multivariable logistic regressions were fitted to evaluate whether patients with lymphopenia were at greater risk of increased antimicrobial exposure.Results: Among 634 patients, the median age at diagnosis was 53.7 years (interquartile range, 37.5-66.8 years), and 290 patients (45.7%) were women, with a 5-year survival probability of 67.9%. There was a significant inverse association between lymphopenia at diagnosis and overall survival (hazard ratio [HR], 1.82; 95% CI, 1.39-1.40), resulting in a 13.5% 5-year survival probability difference compared with patients who did not have lymphopenia at diagnosis (60.2% vs 73.7% for those who never had lymphopenia). In addition, poorer survival was observed with higher-grade lymphopenia (grades 3 and 4: HR, 2.44; 95% CI, 1.68-3.55; grades 1 and 2: HR, 1.60; 95% CI, 1.18-2.18). In an exploratory analysis, patients with increased antibiotic exposure were more likely to have lymphopenia (odds ratio, 1.96; 95% CI, 1.26-3.07 for total number of antimicrobial agents; odds ratio, 1.70; 95% CI, 1.10-2.57 for antimicrobial duration) than antimicrobial-naive patients.Conclusions and Relevance: This study suggests that an abnormally low absolute lymphocyte count at diagnosis is associated with higher mortality among patients with localized bone and soft tissue sarcoma; therefore, lymphopenia may serve as a reliable prognostic biomarker. Potential mechanisms associated with host immunity and overall survival include a suppressed antitumor response and increased infectious complications, which merit future investigation.

    View details for DOI 10.1001/jamanetworkopen.2021.0845

    View details for PubMedID 33666664

  • Long-Term Remission with Ipilimumab/Nivolumab in Two Patients with Different Soft Tissue Sarcoma Subtypes and No PD-L1 Expression. Case reports in oncology Zhou, M., Bui, N., Lohman, M., van de Rjin, M., Hwang, G., Ganjoo, K. 2021; 14 (1): 459–65


    Checkpoint inhibitor therapy has been shown to improve outcomes in multiple solid malignancies; however, data are limited in soft tissue sarcoma. We present two cases of patients with advanced soft tissue sarcoma of different subtypes (dedifferentiated liposarcoma and myxofibrosarcoma) with zero percent PD-L1 expression by immunohistochemistry who were treated with ipilimumab and nivolumab followed by maintenance nivolumab. Both patients had failed multiple lines of systemic treatment and experienced long-term remission after starting ipilimumab and nivolumab. Genetic testing revealed that no genetic mutations were found in common between the two cases. One patient received concurrent cryoablation, which may have sensitized his tumor to immunotherapy. Checkpoint inhibitor therapy may improve outcomes in soft tissue sarcoma regardless of PD-L1 status, especially when combined with cryoablation. Studies are needed to evaluate whether treatment response varies by sarcoma subtype and what molecular markers can be used to guide patient selection.

    View details for DOI 10.1159/000512828

    View details for PubMedID 33790767

  • Management Strategies for Patients With Epithelioid Hemangioendothelioma: Charting an Indolent Disease Course. American journal of clinical oncology Yurkiewicz, I. R., Zhou, M., Ganjoo, K. N., Charville, G. W., Bolleddu, S., Lohman, M., Bui, N. 2021


    Epithelioid hemangioendothelioma (EHE) is a malignant vascular neoplasm representing ∼1% of sarcomas. Due to its rarity, its clinical course is not well characterized and optimal treatment remains unknown.This was a retrospective review of patients with EHE treated at Stanford University between 1998 and 2020. Demographic characteristics, pathology results, treatment modalities, and clinical outcomes were collected from the electronic medical records.A total of 58 patients had a mean age of 50.6 years and a slight female predominance (52%). Primary disease sites were liver (33%), soft tissue (29%), lung (14%), bone (9%), and mediastinum (9%). A majority (55%) had advanced or metastatic disease. Median overall survival (OS) was 16.9 years, with OS 89% at 1 year, 68% at 5 years, and 64% at 10 years. The longest median OS was associated with soft tissue sites and shortest with lung and mediastinal disease (P=0.03). The localized disease had improved median OS compared with metastatic disease (P=0.02). There was no OS difference between tumors >3 cm and those equal or smaller (P=0.85). Surgery was a common treatment (71%), while radiation and ablation were sometimes used (28% and 9%, respectively). The median time to initiating therapy of any kind was 68 days. The median time to systemic therapy was 114 days.We report on the clinical characteristics and outcomes of patients with EHE at a large academic center. Treatment options included surgical excision, liver transplant, ablation, radiation, and systemic therapy. A subset of patients had indolent disease not requiring treatment upfront.

    View details for DOI 10.1097/COC.0000000000000827

    View details for PubMedID 34028371

  • Surgical resection of leiomyosarcoma of the inferior vena cava: A case series and literature review. Surgical oncology Zhou, M., Javadi, C., Charville, G. W., Bui, N. Q., Harris, E. J., Poultsides, G. A., Norton, J. A., Visser, B., Lee, B., Dua, M. M., Ganjoo, K. N. 2021; 39: 101670


    We review our institution's experience in treating leiomyosarcomas involving the inferior vena cava, and we offer guidance on the management.A text-based search was performed to identify all patients who underwent surgical resection between January 2002 and October 2020. Clinicopathologic data, intraoperative variables, and outcomes were extracted from chart review.Twelve of 16 patients (75%) had localized disease; the remaining had limited metastatic disease. Seven of 16 patients (44%) received neoadjuvant chemotherapy or radiation; three patients had partial responses, and four patients had stable disease using RECIST 1.1 criteria. IVC reconstruction was performed in 14 of 16 patients (88%); IVC was ligated for the remaining two patients. Half of all patients had R0 resection on final pathology; the remaining had R1 resections. Progression-free survival (PFS) and overall survival (OS) were not statistically different between patients with R0 and R1 resection. Median PFS was 1.8 years (95% CI 0.89 - not reached); median OS was 6.5 years (1.8 - not reached). Only one patient (6%) experienced local disease recurrence; 4 of 16 patients (25%) experienced disease recurrence distally without local recurrence.Resection of IVC leiomyosarcomas at a sarcoma referral center with experience in vascular reconstruction can lead to many years of recurrence-free survival. Surgical resection should be offered to patients with a low volume of metastatic disease to reduce local complications from the primary tumor, many of which exert significant mass effect on surrounding organs. For patients with metastatic disease or large, high-risk tumors, neoadjuvant chemotherapy can provide a biologic test of disease stability prior to resection.

    View details for DOI 10.1016/j.suronc.2021.101670

    View details for PubMedID 34710646

  • Nivolumab plus ipilimumab for soft tissue sarcoma: a single institution retrospective review IMMUNOTHERAPY Zhou, M., Bui, N., Bolleddu, S., Lohman, M., Becker, H., Ganjoo, K. 2020; 12 (18): 1303–12
  • SQ3370-001 IS A MULTI-CENTER OPEN-LABEL PHASE I DOSE-ESCALATION STUDY TO TEST A NOVEL INTRATUMORAL AND SYSTEMIC APPROACH TO TREAT ADVANCED SOLID TUMORS Bhadri, V., Bui, N., Guminski, A., Oneto, J., Murthy, R., Sankhala, K., Saville, M., Srinivasan, S., Steffner, R., Subbiah, V., Wang, D., Yee, N. BMJ PUBLISHING GROUP. 2020: A253–A254
  • NCI protocol 10250: A phase II study of temozolomide and olaparib for the treatment of advanced uterine leiomyosarcoma. Ingham, M., Allred, J. B., Gano, K., George, S., Attia, S., Burgess, M., Boikos, S., Bui, N., Chen, J., Close, J., Seetharam, M., Thaker, P. H., Schwartz, G. K. LIPPINCOTT WILLIAMS & WILKINS. 2020
  • Increasing Clinical Trial Accrual via Automated Matching of Biomarker Criteria. Pacific Symposium on Biocomputing. Pacific Symposium on Biocomputing Chen, J. W., Kunder, C. A., Bui, N. n., Zehnder, J. L., Costa, H. A., Stehr, H. n. 2020; 25: 31–42


    Successful implementation of precision oncology requires both the deployment of nucleic acid sequencing panels to identify clinically actionable biomarkers, and the efficient screening of patient biomarker eligibility to on-going clinical trials and therapies. This process is typically performed manually by biocurators, geneticists, pathologists, and oncologists; however, this is a time-intensive, and inconsistent process amongst healthcare providers. We present the development of a feature matching algorithmic pipeline that identifies patients who meet eligibility criteria of precision medicine clinical trials via genetic biomarkers and apply it to patients undergoing treatment at the Stanford Cancer Center. This study demonstrates, through our patient eligibility screening algorithm that leverages clinical sequencing derived biomarkers with precision medicine clinical trials, the successful use of an automated algorithmic pipeline as a feasible, accurate and effective alternative to the traditional manual clinical trial curation.

    View details for PubMedID 31797584

  • Increasing Clinical Trial Accrual via Automated Matching of Biomarker Criteria Chen, J. W., Kunder, C. A., Bui, N., Zehnder, J. L., Costa, H. A., Stehr, H., Altman, R. B., Dunker, A. K., Hunter, L., Ritchie, M. D., Murray, T., Klein, T. E. WORLD SCIENTIFIC PUBL CO PTE LTD. 2020: 31-42
  • Successful treatment of HIV-negative Kaposi sarcoma with ipilimumab and nivolumab and concurrent management of baseline psoriasis and bullous pemphigoid. JAAD case reports Tabata, M. M., Novoa, R. A., Bui, N. Q., Zaba, L. C. 2020; 6 (5): 447–49

    View details for DOI 10.1016/j.jdcr.2020.03.001

    View details for PubMedID 32382643

    View details for PubMedCentralID PMC7200441

  • Nivolumab plus ipilimumab for soft tissue sarcoma: a single institution retrospective review. Immunotherapy Zhou, M. n., Bui, N. n., Bolleddu, S. n., Lohman, M. n., Becker, H. C., Ganjoo, K. n. 2020


    Aim: To analyze the efficacy of checkpoint inhibitors in soft tissue sarcoma. Materials & methods: We retrospectively reviewed patients with advanced soft tissue sarcoma treated with ipilimumab and nivolumab. All patients who received at least one cycle were included. Results: One patient had a complete response and five had a partial response, for an objective response rate of 15%. Clinical benefit rate was 34% with a median duration of 12.0 months (range: 4.5 to 28.9+ months [mo]). Median overall survival was 12.0 months (95% CI: 4.5-23.7+ mo). Median progression-free survival was 2.7 months (95% CI: 2.3-4.5+ mo) by Response Evaluation Criteria in Solid Tumors 1.1 and 2.9 months (2.5-6.0+ mo) by immune-related Response Evaluation Criteria in Solid Tumors. Adverse events of any grade were seen in 58% of patients, the most common being fatigue (21%) and cough (10%), 5% of patients experienced a grade 3 adverse event (AE) (hyperglycemia) or grade 4 AE (myocarditis). Conclusion: Ipilimumab/nivolumab combination showed efficacy and was well tolerated in advanced soft tissue sarcoma.

    View details for DOI 10.2217/imt-2020-0155

    View details for PubMedID 32967520

  • Dose escalation of tinostamustine in patients with advanced solid tumors Mita, A., Loeffler, M., Bui, N., Mehrling, T., Rimmel, B. J., Natale, R. B., Kummar, S. AMER ASSOC CANCER RESEARCH. 2019
  • Multi-institutional analysis of outcomes in patients with dedifferentiated chondrosarcoma (DDCS). Bui, N., Dietz, H., Hirbe, A. C., Ganjoo, K. N., Van Tine, B., Keedy, V., Davis, E. J. AMER SOC CLINICAL ONCOLOGY. 2019
  • A phase II study of ADI-PEG 20 in combination with gemcitabine and docetaxel for the treatment of soft tissue sarcoma. Van Tine, B., Bui, N., Prudner, B., Bomalaski, J. S., Wu, B., Chawla, S. P., Ganjoo, K. N., Oppelt, P., Hirbe, A. C. AMER SOC CLINICAL ONCOLOGY. 2019
  • Chromatin dysregulation and DNA methylation at transcription start sites associated with transcriptional repression in cancers NATURE COMMUNICATIONS Ando, M., Saito, Y., Xu, G., Bui, N. Q., Medetgul-Ernar, K., Pu, M., Fisch, K., Ren, S., Sakai, A., Fukusumi, T., Liu, C., Haft, S., Pang, J., Mark, A., Gaykalova, D. A., Guo, T., Favorov, A., Yegnasubramanian, S., Fertig, E. J., Ha, P., Tamayo, P., Yamasoba, T., Ideker, T., Messer, K., Califano, J. A. 2019; 10
  • Next-Generation Sequencing of Tissue and Circulating Tumor DNA: The UC San Diego Moores Center for Personalized Cancer Therapy Experience with Breast Malignancies MOLECULAR CANCER THERAPEUTICS Shatsky, R., Parker, B. A., Bui, N. Q., Heisten, T., Schwab, R. B., Boles, S. G., Kurzrock, R. 2019; 18 (5): 1001–11
  • Contemporary management of metastatic soft tissue sarcoma. Current problems in cancer Bui, N. Q., Wang, D. S., Hiniker, S. M. 2019


    Soft tissue sarcoma (STS) is a rare, heterogeneous cancer that can have high rates of distant metastases. Optimal treatment planning requires detailed knowledge of distinct sarcoma histologies as well as the wide array of therapeutic options through surgical, medical, radiation, and interventional oncology. In this review article, we discuss the contemporary management of metastatic STS and the underlying data behind these recommendations. All patients with metastatic STS should be discussed in a multidisciplinary tumor board at an experienced sarcoma center. For patients with oligometastatic disease, there should be strong consideration for definitive local therapy such as surgical resection, stereotactic body radiation therapy, or ablative procedures. In cases with widespread metastases, cytotoxic chemotherapy represents the standard treatment for STS patients with traditional chemotherapies, such as anthracyclines, gemcitabine/docetaxel, ifosfamide, and dacarbazine, still being the most commonly used drugs today. The recent approvals of trabectedin, eribulin, and pazopanib have expanded the therapeutic armamentarium for metastatic STS. Histology-directed treatment is crucial for certain subtypes of STS which are highly sensitive to targeted therapy and relatively insensitive to chemotherapy. Despite the significant progress that has been made in metastatic STS in the past decade, overall prognosis is poor and there is a critical need for novel therapeutics.

    View details for DOI 10.1016/j.currproblcancer.2019.06.005

    View details for PubMedID 31248634

  • Editorial. Current problems in cancer Bui, N. n. 2019; 43 (4): 249
  • A clinico-genomic analysis of soft tissue sarcoma patients reveals CDKN2A deletion as a biomarker for poor prognosis. Clinical sarcoma research Bui, N. Q., Przybyl, J., Trabucco, S. E., Frampton, G., Hastie, T., van de Rijn, M., Ganjoo, K. N. 2019; 9: 12


    Background: Sarcomas are a rare, heterogeneous group of tumors with variable tendencies for aggressive behavior. Molecular markers for prognosis are needed to risk stratify patients and identify those who might benefit from more intensive therapeutic strategies.Patients and methods: We analyzed somatic tumor genomic profiles and clinical outcomes of 152 soft tissue (STS) and bone sarcoma (BS) patients sequenced at Stanford Cancer Institute as well as 206 STS patients from The Cancer Genome Atlas. Genomic profiles of 7733 STS from the Foundation Medicine database were used to assess the frequency of CDKN2A alterations in histological subtypes of sarcoma.Results: Compared to all other tumor types, sarcomas were found to carry the highest relative percentage of gene amplifications/deletions/fusions and the lowest average mutation count. The most commonly altered genes in STS were TP53 (47%), CDKN2A (22%), RB1 (22%), NF1 (11%), and ATRX (11%). When all genomic alterations were tested for prognostic significance in the specific Stanford cohort of localized STS, only CDKN2A alterations correlated significantly with prognosis, with a hazard ratio (HR) of 2.83 for overall survival (p=0.017). These findings were validated in the TCGA dataset where CDKN2A altered patients had significantly worse overall survival with a HR of 2.7 (p=0.002). Analysis of 7733 STS patients from Foundation One showed high prevalence of CDKN2A alterations in malignant peripheral nerve sheath tumors, myxofibrosarcomas, and undifferentiated pleomorphic sarcomas.Conclusion: Our clinico-genomic profiling of STS shows that CDKN2A deletion was the most prevalent DNA copy number aberration and was associated with poor prognosis.

    View details for DOI 10.1186/s13569-019-0122-5

    View details for PubMedID 31528332

  • Reviewing the role of healthy volunteer studies in drug development. Journal of translational medicine Karakunnel, J. J., Bui, N., Palaniappan, L., Schmidt, K. T., Mahaffey, K. W., Morrison, B., Figg, W. D., Kummar, S. 2018; 16 (1): 336


    BACKGROUND: With the exception of genotoxic oncology drugs, first-in-human, Phase 1 clinical studies of investigational drugs have traditionally been conducted in healthy volunteers (HVs). The primary goal of these studies is to investigate the pharmacokinetics and pharmacodynamics of a novel drug candidate, determine appropriate dosing, and document safety and tolerability.MAIN BODY: When tailored to specific study objectives, HV studies are beneficial to manufacturers and patients alike and can be applied to both non-oncology and oncology drug development. Enrollment of HVs not only increases study accrual rates for dose-escalation studies but also alleviates the ethical concern of enrolling patients with disease in a short-term study at subtherapeutic doses when other studies (e.g. Phase 2 or Phase 3 studies) may be more appropriate for the patient. The use of HVs in non-oncology Phase 1 clinical trials is relatively safe but nonetheless poses ethical challenges because of the potential risks to which HVs are exposed. In general, most adverse events associated with non-oncology drugs are mild in severity, and serious adverse events are rare, but examples of severe toxicity have been reported. The use of HVs in the clinical development of oncology drugs is more limited but is nonetheless useful for evaluating clinical pharmacology and establishing an appropriate starting dose for studies in cancer patients. During the development of oncology drugs, clinical pharmacology studies in HVs have been used to assess pharmacokinetics, drug metabolism, food effects, potential drug-drug interactions, effects of hepatic and renal impairment, and other pharmacologic parameters vital for clinical decision-making in oncology. Studies in HVs are also being used to evaluate biosimilars versus established anticancer biologic agents.CONCLUSION: A thorough assessment of toxicity and pharmacology throughout the drug development process is critical to ensure the safety of HVs. With the appropriate safeguards, HVs will continue to play an important role in future drug development.

    View details for PubMedID 30509294

  • Disruption of NSD1 in head and neck cancer promotes favorable chemotherapeutic responses linked to hypomethylation. Molecular cancer therapeutics Bui, N. n., Huang, J. K., Bojorquez-Gomez, A. n., Licon, K. n., Sanchez, K. S., Tang, S. N., Beckett, A. N., Wang, T. n., Zhang, W. n., Shen, J. P., Kreisberg, J. F., Ideker, T. n. 2018


    Human papillomavirus (HPV) negative head and neck squamous cell carcinoma (HNSCC) represents a distinct classification of cancer with poor expected outcomes. Of the 11 genes recurrently mutated in HNSCC, we identify a singular and substantial survival advantage for mutations in the gene encoding Nuclear Set Domain Containing Protein 1 (NSD1), a histone methyltransferase altered in approximately 10% of patients. This effect, a 55% decrease in risk of death in NSD1-mutated versus non-mutated patients, can be validated in an independent cohort. NSD1 alterations are strongly associated with widespread genome hypomethylation in the same tumors, to a degree not observed for any other mutated gene. To address whether NSD1 plays a causal role in these associations, we use CRISPR-Cas9 to disrupt NSD1 in HNSCC cell lines and find that this leads to substantial CpG hypomethylation and sensitivity to cisplatin, a standard chemotherapy in head and neck cancer, with a 40 - 50% decrease in IC50. Such results are reinforced by a survey of 1,001 cancer cell lines, in which loss-of-function NSD1 mutations have an average 23% decrease in cisplatin IC50 compared to cell lines with wild type NSD1. This study identifies a favorable subtype of head and neck cancer linked to NSD1 mutation, hypomethylation and cisplatin sensitivity.

    View details for DOI 10.1158/1535-7163.MCT-17-0937

    View details for PubMedID 29636367

  • A multicenter phase II study of Q3 week or weekly paclitaxel in combination with bevacizumab for the treatment of metastatic or unresectable angiosarcoma. Rare tumors Bui, N. n., Kamat, N. n., Ravi, V. n., Chawla, S. n., Lohman, M. n., Ganjoo, K. N. 2018; 10: 2036361318771771


    Paclitaxel (P) and bevacizumab (B) are agents that provide clinical benefit in advanced angiosarcoma (AS). The objective of this study was to assess the efficacy and safety of P-B in two different scheduled regimens. Patients were to receive P 200mg/m2 IV with B 15mg/kg IV every 21 days (Regimen A) or P 90mg/m2 IV weekly D1, 8, 15 with B 15mg/kg IV D1 of a 28 day cycle (Regimen B) x6 cycles. Maintenance B followed at a dose of 15 mg/kg intravenously once every 21 days. The primary end point was 4 month non-progression rate (NPR). A total of 16 patients were enrolled. 4 month NPR was 62.5% with median overall survival 16 months and median progression free survival 5.06 months. 11 patients made it to cycle 3 and were evaluable for response with 1 CR (9%), 4 PR (36%), 2 SD (18%), and 6 PD (36%). There were ten grade 3 toxicities and four grade 4 toxicities. The breakdown between the two regimens revealed comparable efficacy and safety. Paclitaxel and Bevacizumab is an active regimen in angiosarcoma. Q3 week and weekly paclitaxel appear similar in efficacy and safety.

    View details for PubMedID 29760870

  • Cell-Free DNA from Ascites and Pleural Effusions: Molecular Insights into Genomic Aberrations and Disease Biology MOLECULAR CANCER THERAPEUTICS Husain, H., Nykin, D., Nam Bui, N., Quan, D., Gomez, G., Woodward, B., Venkatapathy, S., Duttagupta, R., Fung, E., Lippman, S. M., Kurzrock, R. 2017; 16 (5): 948-955


    Collection of cell-free DNA (cfDNA) from the blood of individuals with cancer has permitted noninvasive tumor genome analysis. Detection and characterization of cfDNA in ascites and pleural effusions have not yet been reported. Herein, we analyzed cfDNA in the ascites and pleural effusions from six individuals with metastatic cancer. In all cases, cfDNA copy number variations (CNV) were discovered within the effusate. One individual had a relevant alteration with a high copy amplification in EGFR in a never smoker with lung cancer, who showed only MDM2 and CDK4 amplification in a prior tissue biopsy. Another subject with metastatic breast cancer had cytology-positive ascites and an activating PIK3CA mutation identified in the tissue, blood, and ascites collectively. This individual had tumor regression after the administration of the mTOR inhibitor everolimus and had evidence of chromotripsis from chromosomal rearrangements noted in the cell-free ascitic fluid. These results indicate that cfDNA from ascites and pleural effusions may provide additional information not detected with tumor and plasma cell-free DNA molecular characterization, and a context for important insights into tumor biology and clonal dynamic change within primary tumor and metastatic deposits. Mol Cancer Ther; 16(5); 948-55. ©2017 AACR.

    View details for DOI 10.1158/1535-7163.MCT-16-0436

    View details for Web of Science ID 000400713500017

    View details for PubMedID 28468865

  • Strategies to Overcome Bypass Mechanisms Mediating Clinical Resistance to EGFR Tyrosine Kinase Inhibition in Lung Cancer MOLECULAR CANCER THERAPEUTICS Husain, H., Scur, M., Murtuza, A., Bui, N., Woodward, B., Kurzrock, R. 2017; 16 (2): 265-272


    The vast majority of patients with metastatic lung cancers who initially benefit from EGFR-targeted therapies eventually develop resistance. An increasing understanding of the number and complexity of resistance mechanisms highlights the challenge of treating tumors resistant to EGFR inhibitors. Resistance mechanisms include new, second-site mutations within EGFR (e.g., T790M and C797S), upregulation of MET kinase, upregulation of insulin growth factor receptor (IGFR), HER2 amplification, increased expression of AXL, BIM modulation, NF-κB activation, histologic switch to small-cell cancer, epithelial-to-mesenchymal transition, PDL1 expression with subsequent immune tolerance, and release of cytokines such as TGFβ and IL6. Herein, we review the growing body of knowledge regarding EGFR bypass pathways, and the development of new drugs and combination treatment strategies to overcome resistance. Mol Cancer Ther; 16(2); 265-72. ©2017 AACR.

    View details for DOI 10.1158/1535-7163.MCT-16-0105

    View details for Web of Science ID 000395563700002

    View details for PubMedID 28159915

  • Molecular insights into desmoid tumors. Oncotarget Bui, N. n., Kummar, S. n. 2017; 8 (53): 90608–9

    View details for DOI 10.18632/oncotarget.21293

    View details for PubMedID 29207578

    View details for PubMedCentralID PMC5710859

  • Chart review versus an automated bioinformatic approach to assess real-world crizotinib effectiveness in ALK-positive NSCLC. JCO clinical cancer informatics Bui, N. n., Henry, S. n., Wood, D. n., Wakelee, H. A., Neal, J. W. 2017; 2017

    View details for DOI 10.1200/CCI.16.00055

    View details for PubMedID 28868517

  • Evolution of early phase clinical trials in oncology. Journal of molecular medicine (Berlin, Germany) Bui, N. Q., Kummar, S. n. 2017


    The therapeutic armamentarium for the treatment of cancer has rapidly evolved with the advent of molecularly targeted and immuno-oncology agents. Dramatic and prolonged responses observed in patients with advanced cancers have created excitement and promise for expedited development of effective new treatments. However, this has also necessitated a rethinking of our early phase clinical trial designs and the process of optimally developing a novel agent. In this review, we discuss the current state and future directions of phase I clinical trials in oncology. Firstly, we cover the statistical methodologies behind rules and model-based dose escalation designs, and what the future holds for optimal dose selection beyond targeting the maximum tolerated dose. Next, we discuss the recent adoption of seamless expansion strategies to expedite drug development timelines, highlighted by the pembrolizumab KEYNOTE-001 trial, and potential pitfalls with this approach. Finally, we delve into the concepts behind genomic matching trials, including early success stories and the challenges that lie ahead.

    View details for PubMedID 29177698

  • Novel Treatment Strategies for Brain Metastases in Non-small-cell Lung Cancer CURRENT TREATMENT OPTIONS IN ONCOLOGY Bui, N., Woodward, B., Johnson, A., Husain, H. 2016; 17 (5)


    Brain metastases are common in patients with non-small cell lung cancer (NSCLC), and due to associated poor prognosis, this field is an important area of need for the development of innovative medical therapies. Therapies including local approaches through surgical intervention and/or radiation and evolving systemic therapies have led to improvements in the treatment of brain metastases in patients with lung cancer. Strategies that consider applying advanced radiation techniques to minimize toxicity, intervening early with effective systemic therapies to spare radiation/surgery, testing radiosensitization combinations, and developing drug penetrant molecules have and will continue to define new practice patterns. We believe that in carefully considered asymptomatic patients, first-line systemic therapy may be considered before radiation therapy and small-molecule targeted therapy may provide an opportunity to defer radiation therapy for recurrence or progression of disease. The next several years in oncology drug development will see the reporting on of brain penetrant molecules in oncogene-defined non-small cell lung cancer. Ongoing studies will evaluate immunotherapies in patients with brain metastases with associated endpoints. We hope that continued drug development and carefully designed clinical trials may afford an opportunity to improve the lives of patients with brain metastases.

    View details for DOI 10.1007/s11864-016-0400-x

    View details for Web of Science ID 000374565200002

    View details for PubMedID 27085533

  • Reactivation of hepatitis B virus after withdrawal of erlotinib CURRENT ONCOLOGY Bui, N., Wong-Sefidan, I. 2015; 22 (6): 430-432


    Reactivation of hepatitis B virus (hbv) is a reported complication for patients undergoing chemotherapy, particularly immunochemotherapy with anti-CD20 agents such as rituximab. However, as the use of molecularly targeted agents increases, the risk of viral reactivation is less clearly defined. Here, we present the case of a 62-year-old woman with newly diagnosed EGFR mutation-positive metastatic non-small-cell lung cancer (nsclc). Per interview, our patient had a remote history of hbv infection. She was started on erlotinib and developed profound diarrhea leading to renal failure that required hospital admission and temporary discontinuation of erlotinib. At 8 days after erlotinib cessation, she had a marked spike in her liver function tests, with viral serologies that were consistent with hbv reactivation. Although erlotinib and other tyrosine kinase inhibitors (tkis) are not classically associated with hbv reactivation, hbv reactivation can occur even in the setting of tki withdrawal. Before tki initiation, careful patient screening in those at risk for hbv should be performed to attenuate preventable hepatotoxicity and to differentiate between other causes of hepatotoxicity (for example, drug-induced toxicity).

    View details for DOI 10.3747/co.22.2665

    View details for Web of Science ID 000366983500022

    View details for PubMedID 26715877

    View details for PubMedCentralID PMC4687665

  • Dorsal aesthetic lines in rhinoplasty: a quantitative outcome-based assessment of the component dorsal reduction technique. Plastic and reconstructive surgery Mojallal, A., Ouyang, D., Saint-Cyr, M., Bui, N., Brown, S. A., Rohrich, R. J. 2011; 128 (1): 280-288


    Preservation or reconstruction of the middle nasal vault structure and internal nasal valve after dorsal reduction is challenging. The purpose of this study was to retrospectively analyze a series of 100 consecutive rhinoplasty cases with respect to preservation or restoration of the dorsal nasal lines following component dorsal reduction. A new quantitative mathematical application for subject digital images was performed.Medical information and digital images were obtained from 100 consecutive primary rhinoplasty patients from one author (R.J.R.) with University of Texas Southwestern Medical Center Institutional Review Board consent. All postoperative subject digital images were taken at more than 1-year follow-up. Preoperative and postoperative digital images of the dorsal nasal aesthetic lines were analyzed using a software application that quantitated various facial anatomical features compared with landmark measurements unique for each subject (pupil-to-pupil distance). Dorsal line symmetry, nose width, and variation of deformities on each side of the face were determined.Mean subject dorsal line symmetry was 68 percent preoperatively and 94 percent postoperatively. Only 32.5 percent of dorsal lines were harmonious preoperatively, whereas 97 percent of dorsal lines were harmonious postoperatively. Identification of dorsal lines postoperatively versus preoperatively was similar in 74.6 percent, improved in 15.7 percent, and decreased in 9.7 percent. Nasal width lines were similar in 36 subjects, 21 subjects had wider nasal width lines, and 43 subjects had narrower width lines after surgery.Component dorsal hump reduction procedures result in reliable and reproducible clinical outcomes. Quantitative assessments provide evidence that improved and harmonious curves of dorsal aesthetic lines are achievable.Therapeutic, IV.(Figure is included in full-text article.).

    View details for DOI 10.1097/PRS.0b013e318218fc2d

    View details for PubMedID 21701345