Eric J. Small, MD

Doris and Donald Fisher Distinguished Professorship in Clinical Cancer Research; Stanford W. Ascherman and Norman R. Ascherman Endowed Chair, UCSF

Professor In Residence of Medicine and Urology; Deputy Director and Chief Scientific Officer; Co-Leader, Prostate Cancer Program, UCSF Helen Diller Family Comprehensive Cancer Center

Cancer Center Program Membership

Prostate Cancer

Research Summary

My interests lie in clinical and translational research in advanced prostate cancer, including trial design, and development of novel agents. I piloted the early trials targeting the androgen receptor in advanced prostate cancer, developing ketoconazole and contributing to an understanding of its mechanisms of activity and resistance. I subsequently was centrally involved with the development of two novel androgen signaling inhibitors, abiraterone acetate and apalutamide, from first in human studies to FDA approval. 

I have also been involved in the early application of immunotherapy to prostate cancer, developing sipuleucel T from first in human studies through FDA approval, and undertaking the first in human testing of ipilimumab, the first immune check point inhibitor to be clinically developed. 

Understanding the mechanisms of resistance in advanced prostate cancer has been a research focus. I was principal investigator of a $10 million multi-center Stand Up to Cancer (SU2C)/Prostate Cancer Foundation Dream Team Award undertaking deep genomic characterization of prostate cancer metastases, in order to elucidate patterns of resistance in abiraterone and/or enzalutamide treated patients. Important observations from this, and subsequent work have included unexpectedly high prevalence rates of treatment associated small cell/neuroendocrine prostate cancer (t-SCNC), as well key structural variants which contribute to progression of castration-resistant disease, observed in the largest Whole Genome Sequencing series to date of metastatic advanced prostate cancer specimens. 

I hold a number of leadership roles, serving as Co-Leader of the UCSF Prostate Cancer Program, and as Deputy Director of the UCSF Helen Diller Family Comprehensive Cancer Center. I previously served as Chief of the Division of Hematology and Oncology in the UCSF Department of Medicine for 10 years, and most recently have assumed the position of Chief Scientific Officer in the UCSF Helen Diller Family Comprehensive Cancer Center. Nationally, I serve on the ASCO Board of Directors, and chair 4 External Advisory Boards for other NCI-Designated Cancer Centers.

Education

Stanford University, Stanford, CA, BS, 1980, Biology
Case Western Reserve University of Medicine, Cleveland, Ohio, MD, 1985, Medicine
Beth Israel Hospital, Boston, MA, Internship and Residency, 1988, Internal Medicine
University of California San Francisco, CA, Fellowship, 1991, Hematology/Oncology

Honors & Awards

  • 1978
    Biomedical Research Grant, The Jackson Laboratory, Bar Harbour, ME
  • 1980
    NIH Research Support Grant, Case Western Reserve University, Cleveland, OH
  • 1984
    Alpha Omega Alpha Honor Medical Society
  • 1997
    Prostate Cancer Foundation Therapy Consortium
  • 2006-present
    Stanford W. and Norman R. Ascherman Endowed Chair

Selected Publications

  1. Chi KN, Castro E, Attard G, Smith MR, Sandhu S, Efstathiou E, Roubaud G, Small EJ, de Santana Gomes AP, Rathkopf DE, Saad M, Gurney H, Jung W, Kim W, Dibaj S, Wu D, Zhang J, Lopez-Gitlitz A, Francis P, Olmos D. Niraparib and Abiraterone Acetate plus Prednisone in Metastatic Castration-resistant Prostate Cancer: Final Overall Survival Analysis for the Phase 3 MAGNITUDE Trial. Eur Urol Oncol. 2025 May 05.  View on PubMed
  2. Feng EM, Vo-Phamhi J, Subramanian AN, Dias M, Foye A, Vinson J, Hong JC, Freedland SJ, Alumkal JJ, Beltran H, Morrissey C, Nelson PS, Chinnaiyan AM, Aggarwal R, Small EJ, Quigley DA, Sjöström M, Zhao SG, Chen WS. Racial variation in the advanced prostate cancer genome. Prostate Cancer Prostatic Dis. 2025 Mar 31.  View on PubMed
  3. Chi KN, Rathkopf D, Smith MR, Efstathiou E, Attard G, Olmos D, Lee JY, Small EJ, Pereira de Santana Gomes AJ, Roubaud G, Saad M, Zurawski B, Sakalo V, Mason GE, Francis P, Wang G, Wu D, Diorio B, Lopez-Gitlitz A, Sandhu S. A plain language summary of the results from the MAGNITUDE study assessing how effective and how safe niraparib and abiraterone acetate with prednisone is in patients with metastatic castration-resistant prostate cancer. Future Oncol. 2025 Apr; 21(9):1013-1031.  View on PubMed
  4. Aggarwal RR, Vuky J, VanderWeele D, Rettig M, Heath EI, Quigley D, Huang J, Chumber A, Cheung A, Foye A, Leung S, Abbey J, Dorr A, Nasoff M, Hunter J, Wang S, Flavell RR, Fong L, Liu B, Small EJ. Phase I, First-in-Human Study of FOR46 (FG-3246), an Immune-Modulating Antibody-Drug Conjugate Targeting CD46, in Patients With Metastatic Castration-Resistant Prostate Cancer. J Clin Oncol. 2025 May 20; 43(15):1824-1834.  View on PubMed
  5. Sharifi MN, Feng E, Rydzewski NR, Taylor AK, Sperger JM, Shi Y, Helzer KT, Bootsma ML, Carreno V, Chang AH, Nunamaker LA, Blitzer GC, Shang TA, Subramanian A, Bjartell A, Josefsson A, Wikström P, Feng E, Kohli M, Yang R, Dehm SM, Small EJ, Aggarwal R, Quigley DA, Lang JM, Zhao SG, Sjöström M. Adverse prognosis gene expression patterns in metastatic castration-resistant prostate cancer. Mol Oncol. 2025 Feb 22.  View on PubMed
  6. Feng FY, Smith MR, Saad F, Mobadersany P, Tian SK, Yip SSF, Greshock J, Khan N, Yu MK, McCarthy S, Brookman-May SD, Bourla AB, Todorovic T, Yamashita R, Huang HC, Royce TJ, Showalter TN, Griffin J, Mitani A, Esteva A, Small EJ. Digital Pathology-Based Multimodal Artificial Intelligence Scores and Outcomes in a Randomized Phase III Trial in Men With Nonmetastatic Castration-Resistant Prostate Cancer. JCO Precis Oncol. 2025 Jan; 9:e2400653.  View on PubMed
  7. Chou J, Robinson TM, Egusa EA, Lodha R, Zhang M, Badura M, Mikayelyan M, Delavan HM, Swinderman J, Wilson C, Zhu J, Das R, Nguyen M, Loehr A, Golsorkhi T, Simmons A, Abida W, Chinnaiyan AM, Arkin MR, Small EJ, Quigley DA, Yang L, Kim M, Ashworth A, Feng FY. Synthetic Lethal Targeting of CDK12-Deficient Prostate Cancer with PARP Inhibitors. Clin Cancer Res. 2024 Dec 02; 30(23):5445-5458.  View on PubMed
  8. Kwon DH, Trihy L, Darvish N, Hearst E, Sumra S, Borno HT, Bose R, Chou J, de Kouchkovsky I, Desai A, Ekstrand B, Friedlander T, Kaur G, Koshkin VS, Nesheiwat S, Sepucha K, Small EJ, Aggarwal RR, Belkora J. Patients Can Administer Mobile Audio Recordings to Increase Knowledge in Advanced Prostate Cancer. Cancer Med. 2024 Nov; 13(22):e70433.  View on PubMed
  9. Gillessen S, Turco F, Davis ID, Efstathiou JA, Fizazi K, James ND, Shore N, Small E, Smith M, Sweeney CJ, Tombal B, Zilli T, Agarwal N, Antonarakis ES, Aparicio A, Armstrong AJ, Bastos DA, Attard G, Axcrona K, Ayadi M, Beltran H, Bjartell A, Blanchard P, Bourlon MT, Briganti A, Bulbul M, Buttigliero C, Caffo O, Castellano D, Castro E, Cheng HH, Chi KN, Clarke CS, Clarke N, de Bono JS, De Santis M, Duran I, Efstathiou E, Ekeke ON, El Nahas TIH, Emmett L, Fanti S, Fatiregun OA, Feng FY, Fong PCC, Fonteyne V, Fossati N, George DJ, Gleave ME, Gravis G, Halabi S, Heinrich D, Herrmann K, Hofman MS, Hope TA, Horvath LG, Hussain MHA, Jereczek-Fossa BA, Jones RJ, Joshua AM, Kanesvaran R, Keizman D, Khauli RB, Kramer G, Loeb S, Mahal BA, Maluf FC, Mateo J, Matheson D, Matikainen MP, McDermott R, McKay RR, Mehra N, Merseburger AS, Morgans AK, Morris MJ, Mrabti H, Mukherji D, Murphy DG, Murthy V, Mutambirwa SBA, Nguyen PL, Oh WK, Ost P, O'Sullivan JM, Padhani AR, Parker C, Poon DMC, Pritchard CC, Rabah DM, Rathkopf D, Reiter RE, Renard-Penna R, Ryan CJ, Saad F, Sade JP, Sandhu S, Sartor OA, Schaeffer E, Scher HI, Sharifi N, Skoneczna IA, Soule HR, Spratt DE, Srinivas S, Sternberg CN, Suzuki H, Taplin ME, Thellenberg-Karlsson C, Tilki D, Türkeri LN, Uemura H, Ürün Y, Vale CL, Vapiwala N, Walz J, Yamoah K, Ye D, Yu EY, Zapatero A, Omlin A. Management of Patients with Advanced Prostate Cancer. Report from the 2024 Advanced Prostate Cancer Consensus Conference (APCCC). Eur Urol. 2025 Feb; 87(2):157-216.  View on PubMed
  10. Roy S, Malone S, Wing K, Chowdhury S, Kishan AU, Sun Y, Wallis CJD, Mohamad O, Jia AY, Swami U, Zaorsky NG, Morgan SC, Ong M, Agarwal N, Spratt DE, Small EJ, Saad F. Prior Local Therapy and First-Line Apalutamide in Patients With Nonmetastatic Castration-Resistant Prostate Cancer: A Secondary Analysis of the SPARTAN Randomized Clinical Trial. JAMA Netw Open. 2024 Oct 01; 7(10):e2439434.  View on PubMed
  11. Rathkopf DE, Roubaud G, Chi KN, Efstathiou E, Attard G, Olmos D, Small EJ, Saad M, Castro E, Kim W, Wu D, Bertzos K, Dibaj S, Zhang J, Francis P, Smith MR. Patient-reported Outcomes for Patients with Metastatic Castration-resistant Prostate Cancer and BRCA1/2 Gene Alterations: Final Analysis from the Randomized Phase 3 MAGNITUDE Trial. Eur Urol. 2024 Sep 23.  View on PubMed
  12. Shrestha R, Chesner LN, Zhang M, Zhou S, Foye A, Lundberg A, Weinstein AS, Sjöström M, Zhu X, Moreno-Rodriguez T, Li H, SU2C/PCF West Coast Prostate Cancer Dream Team, Alumkal JJ, Aggarwal R, Small EJ, Lupien M, Quigley DA, Feng FY. An Atlas of Accessible Chromatin in Advanced Prostate Cancer Reveals the Epigenetic Evolution during Tumor Progression. Cancer Res. 2024 Sep 16; 84(18):3086-3100.  View on PubMed
  13. Karsh LI, Bevans KB, Saad F, Chung BH, Oudard S, Brookman-May SD, McCarthy SA, Smith MR, Chi KN, Small EJ, Agarwal N. Prostate-specific antigen and health-related quality of life in individuals with advanced prostate cancer treated with apalutamide: a plain language summary of the SPARTAN and TITAN studies. Future Oncol. 2024; 20(35):2689-2698.  View on PubMed
  14. Zhu X, Farsh T, Vis D, Yu I, Li H, Liu T, Sjöström M, Shrestha R, Kneppers J, Severson T, Zhang M, Lundberg A, Moreno Rodriguez T, Weinstein AS, Foye A, Mehra N, Aggarwal RR, Bergman AM, Small EJ, Lack NA, Zwart W, Quigley DA, van der Heijden MS, Feng FY. Genomic and transcriptomic features of androgen receptor signaling inhibitor resistance in metastatic castration-resistant prostate cancer. J Clin Invest. 2024 Aug 13; 134(19).  View on PubMed
  15. Zhao SG, Bootsma M, Zhou S, Shrestha R, Moreno-Rodriguez T, Lundberg A, Pan C, Arlidge C, Hawley JR, Foye A, Weinstein AS, Sjöström M, Zhang M, Li H, Chesner LN, Rydzewski NR, Helzer KT, Shi Y, West Coast Dream Team Consortium, Lynch M, Dehm SM, Lang JM, Alumkal JJ, He HH, Wyatt AW, Aggarwal R, Zwart W, Small EJ, Quigley DA, Lupien M, Feng FY. Integrated analyses highlight interactions between the three-dimensional genome and DNA, RNA and epigenomic alterations in metastatic prostate cancer. Nat Genet. 2024 Aug; 56(8):1689-1700.  View on PubMed
  16. Zhang M, Sjöström M, Cui X, Foye A, Farh K, Shrestha R, Lundberg A, Dang HX, Li H, Febbo PG, Aggarwal R, Alumkal JJ, Small EJ, SU2C/PCF West Coast Prostate Cancer Dream Team, Maher CA, Feng FY, Quigley DA. Integrative analysis of ultra-deep RNA-seq reveals alternative promoter usage as a mechanism of activating oncogenic programmes during prostate cancer progression. Nat Cell Biol. 2024 Jul; 26(7):1176-1186.  View on PubMed
  17. Subramanian A, Zhang M, Sharifi M, Moreno-Rodriguez T, Feng E, Rydzewski NR, Shrestha R, Zhu X, Zhao SG, Aggarwal R, Small EJ, Ding CC, Quigley DA, Sjöström M. A prostate cancer gastrointestinal transcriptional phenotype may be associated with diminished response to AR-targeted therapy. bioRxiv. 2024 Jun 03.  View on PubMed
  18. Crawford ED, Bryce AH, Hussain MH, Agarwal N, Beltran H, Cooperberg MR, Petrylak DP, Shore N, Spratt DE, Tagawa ST, Antonarakis ES, Aparicio AM, Armstrong AJ, Boike TP, Calais J, Carducci MA, Chapin BF, Cookson MS, Davis JW, Dorff T, Eggener SE, Feng FY, Gleave M, Higano C, Iagaru A, Morgans AK, Morris M, Murray KS, Poage W, Rettig MB, Sartor O, Scher HI, Sieber P, Small E, Srinivas S, Yu EY, Zhang T, Koo PJ. Expert Perspectives on Controversies in Castration-Sensitive Prostate Cancer Management: Narrative Review and Report of the First US Prostate Cancer Conference Part 1. JU Open Plus. 2024 Apr; 2(4).  View on PubMed
  19. Bryce AH, Crawford ED, Agarwal N, Hussain MH, Beltran H, Cooperberg MR, Petrylak DP, Shore N, Spratt DE, Tagawa ST, Antonarakis ES, Aparicio AM, Armstrong AJ, Boike TP, Calais J, Carducci MA, Chapin BF, Cookson MS, Davis JW, Dorff T, Eggener SE, Feng FY, Gleave M, Higano C, Iagaru A, Morgans AK, Morris M, Murray KS, Poage W, Rettig MB, Sartor O, Scher HI, Sieber P, Small E, Srinivas S, Yu EY, Zhang T, Koo PJ. Expert Perspectives on Controversies in Metastatic Castration-Resistant Prostate Cancer Management: Narrative Review and Report of the First US Prostate Cancer Conference Part 2. JU Open Plus. 2024 Apr; 2(4).  View on PubMed
  20. Chang H, Marquez J, Chen BK, Kim DM, Cheng ML, Liu EV, Yang H, Zhang L, Sinha M, Cheung A, Kwek SS, Chow ED, Bridge M, Aggarwal RR, Friedlander TW, Small EJ, Anderson M, Fong L. Immune Modulation with RANKL Blockade through Denosumab Treatment in Patients with Cancer. Cancer Immunol Res. 2024 Apr 02; 12(4):453-461.  View on PubMed

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