2019 Top Ten Finalists
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 This year, the level of scientific rigor, quality, and innovation in all of the nominated studies was truly outstanding. The Clinical Research Forum congratulates the following studies who were finalists: 

 

A Null Mutation in SERPINE 1 Protects Against Biological Aging in Humans

Douglas E. Vaughan, MD, Northwestern University Feinberg School of Medicine

Journal Citation: Khan, S. S., Shah, S. J., Klyachko, E., Baldridge, A. S., Eren, M., Place, A. T., Aviv, A., Puterman, E., Lloyd-Jones, D. M., Heiman, M., Miyata, T., Gupta, S., Shapiro, A. D., & Vaughan, D. E. (2017). A null mutation in SERPINE1 protects against biological aging in humans. Science advances, 3(11), eaao1617.

 

Application of exosomal PD-L1 as a predictor for anti-PD-1 therapy

Xiaowei Xu, MD, PhD, Perelman School of Medicine at the University of Pennsylvania

Journal Citation: Chen G, Huang AC, Zhang W, Zhang G, Wu M, Xu W, Yu Z,  Yang J, Wang B, Sun H, Xia H, Man Q, Zhong W, Antelo  LF, Wu B, Xiong X, Liu X, Guan L, Li T, Liu S, Yang  R, Lu Y, Dong L, McGettigan S, Somasundaram R, Radhakrishnan R,  Mills G, Lu Y, Kim J, Chen YH, Dong H, Zhao Y, Karakousis  GC, Mitchell TC, Schuchter LM, Herlyn M, Wherry EJ, Xu X*, Guo W* (*co-senior authors). Exosomal PD-L1 contributes to immunosuppression and is associated with anti-PD-1 response. Nature. 2018 Aug;560(7718):382-386. doi: 10.1038/s41586-018-0392-8. Epub 2018 Aug 8.

 

Blood-based tumor mutational burden as a predictor of clinical benefit in non-small-cell lung cancer patients treated with atezolizumab

David Gandara, University of California at Davis, School of Medicine

Journal Citation: Atezolizumab versus docetaxel in patients with previously treated non-small-cell lung cancer (OAK): a phase 3, open-label, multicentre randomised controlled trial. Rittmeyer A, Barlesi F, Waterkamp D, Park K, Ciardiello F, von Pawel J, Gadgeel SM, Hida T, Kowalski DM, Dols MC, Cortinovis DL, Leach J, Polikoff J, Barrios C, Kabbinavar F, Frontera OA, De Marinis F, Turna H, Lee JS, Ballinger M, Kowanetz M, He P, Chen DS, Sandler A, Gandara DR; OAK Study Group. Lancet. 2017 Jan 21;389(10066):255-265. doi: 10.1016/S0140-6736(16)32517-X. Epub 2016 Dec 13.

 

Fingolimod Phase III Study for Pediatric Multiple Sclerosis

Tanuja Chitnis, Massachusetts General Hospital

Journal Citation: Chitnis T, Arnold DL, Banwell B, Brück W, Ghezzi A, Giovannoni G, Greenberg B, Krupp L, Rostásy K, Tardieu M, Waubant E, Wolinsky JS, Bar-Or A, Stites T, Chen Y, Putzki N, Merschhemke M, Gärtner J; PARADIGMS Study Group. N Engl J Med. 2018 Sep 13;379(11):1017-1027. doi: 10.1056/NEJMoa1800149.

 

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study.

Craig M. McDonald, University of California at Davis, School of Medicine

Journal Citation: McDonald CM, Henricson EK, Abresch RT, Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP, Cnaan A, Gordish-Dressman H; CINRG Investigators.  Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study.  Lancet. 2018 Feb 3;391(10119):451-461. doi: 10.1016/S0140-6736(17)32160-8. 

 

Molecular Detection of Blood Cancer Cells After Transplant Predicts Outcomes

Eric Duncavage, Washington University School of Medicine

Journal Citation:  Duncavage EJ, Jacoby MA, Chang GS, Miller CA, Edwin N, Shao J, Elliott K, Robinson J, Abel H, Fulton RS, Fronick CC, O'Laughlin M, Heath SE, Brendel K, Saba R, Wartman LD, Christopher MJ, Pusic I, Welch JS, Uy GL, Link DC, DiPersio JF, Westervelt P, Ley TJ, Trinkaus K, Graubert TA, Walter MJ. Mutation Clearance after Transplantation for Myelodysplastic Syndrome.  N Engl J Med. 2018 Sep 13;379(11):1028-1041. doi: 10.1056/NEJMoa1804714. PMID: 30207916

 

Prevention of metastasis in men with prostate cancer

Matthew R. Smith, MD, PhD, Massachusetts General Hospital Cancer Center

Journal Citation: Smith MR, Saad F, Chowdhury S, et al. Apalutamide treatment and metastasis-free survival in prostate cancer. N Engl J Med 2018; 378: 1408-18.

 

Safety and Efficacy of Methadone vs Morphine for Treatment of Neonatal Abstinence Syndrome

Jonathan M Davis, MD, Tufts Medical Center

Journal Citation: Davis JM, Shenberger J, Terrin N, et al. Comparison of Safety and Efficacy of Methadone vs Morphine for Treatment of Neonatal Abstinence SyndromeA Randomized Clinical Trial. JAMA Pediatr. 2018;172(8):741“748. doi:10.1001/jamapediatrics.2018.1307

 

Structural violence, racial disparities, and biological mechanisms of disease

Alexander C. Tsai, Massachusetts General Hospital and Harvard Medical School

Journal Citation: Bor J, Venkataramani AS, Williams D, Tsai AC. Police killings and their spillover effects on the mental health of black Americans: a population-based, quasi-experimental study. Lancet 2018 Jul 28;392(10144):302-310.

 

The effect of broad based genomic sequencing of tumor samples from patients with advanced non small cell lung cancer on survival

Cary P. Gross, MD, Yale University School of Medicine

Journal Citation: Presley CJ, Tang D, Soulos PR, Chiang AC, Longtine JA, Adelson KB, Herbst RS, Zhu W, Nussbaum NC, Sorg RA, Agarwala V, Abernethy AP, Gross CP. Association of Broad-Based Genomic Sequencing With Survival Among Patients With Advanced Non-Small Cell Lung Cancer in the Community Oncology Setting. JAMA. 2018 Aug 7;320(5):469-477. doi: 10.1001/jama.2018.9824.

 

Trial Assigning Individualized Options for Treatment (TAILORx)

Joseph A. Sparano, M.D., Albert Einstein College of Medicine

Journal Citation: Sparano, Joseph A., Gray, Robert J., Makower, Della F., Pritchard, Kathleen I., Albain, Kathy S., Hayes, Daniel F., Geyer, Charles E., Dees, Elizabeth C., Goetz, Matthew P., Olson, John A., Lively, Tracy, Badve, Sunil S., Saphner, Thomas J., Wagner, Lynne I., Whelan, Timothy J., Ellis, Matthew J., Paik, Soonmyung, Wood, William C., Ravdin, Peter M., Keane, Maccon M., Gomez Moreno, Henry L., Reddy, Pavan S., Goggins, Timothy F., Mayer, Ingrid A., Brufsky, Adam M., Toppmeyer, Deborah L., Kaklamani, Virginia G., Berenberg, Jeffrey L., Abrams, Jeffrey, Sledge, George W. (2018)Adjuvant Chemotherapy Guided by a 21-Gene Expression Assay in Breast Cancer. New England Journal of Medicine 379:2, 111-121. https://www.nejm.org/doi/full/10.1056/NEJMoa1804710