Howard J. Federoff

picture of Howard J. Federoff

Distinguished Professor, Neurology
School of Medicine


MD/PhD, Albert Einstein College of Medicine
M.S., Albert Einstein College of Medicine, 1977, Biochemistry

ORCiD: 0000-0002-7595-6520

Phone: (240) 281-2598
Email: federoff@uci.edu

University of California, Irvine
Coltrane Court
Mail Code: 3950
Irvine, CA 92617
Research Interests
gene therapy, neurodegenerative diseases
Awards and Honors
USPHS T321982
ACS Special Postdoctoral Award
Alpha Omega Alpha Election
Arthur Kornberg Research Award, University of Rochester
Founding member, Parkinson’s Disease Gene Therapy Study Group
Director, Parkinson’s Disease Gene Therapy Study Group
Grass Lecturer, Kansas City Chapter of the Society for Neuroscience
Abreu Memorial Keynote Lecturer; The University Texas Medical Branch
Selection, Chair of NIH Recombinant DNA Advisory Committee (RAC)
Recipient, Bernard Sandberg Award, American Society of Neural Therapy and Repair
President, American Society of Neural Therapy and Repair
President, American Society Experimental Neurotherapeutics
Selection, Chair of NIH Gene Therapy Resource Program
Elected Fellow of American Association for the Advancement of Science (AAAS)
Elected Fellow of the National Academy of Inventors (NAI)
Appointed O’Neill Institute Senior Scholar, Georgetown Law Center
Recipient, Outstanding Alumni Award, Earlham College
Elected Board Chair, Association of Academic Health Centers
Fellow of the American Neurological Association
Awarded “Pioneer in Medicine” from the Society of Brain Mapping and Therapeutics
Designated Distinguished Professor of Neurology, University of California, Irvine
Roy Bakay Distinguished Lecturer, ASNTR, Sand Key, Florida
Short Biography
Howard J Federoff, MD, PhD is a distinguished professor of neurology at the University of California, Irvine. He is the former CEO of UCI Health, vice chancellor for Health Affairs and dean of the UCI School of Medicine. Prior to joining UCI Health, Federoff was executive vice president of Health Sciences and executive dean at Georgetown University. Federoff has published more than 275 peer-reviewed and invited articles, and serves on editorial boards of five journals. He co-founded MedGenesis Therapeutix and Brain Neurotherapy Bio, both advancing therapeutics for neurologic diseases. He became CEO of the regenerative medicine company, Aspen Neuroscience, Inc, in San Diego. Aspen is developing an autologous iPSC drug product for Parkinson’s disease. Federoff chaired the NIH Recombinant DNA Advisory Committee, the NHLBI Gene Therapy Resource and the Board of the Association of the Academic Health Centers. He has served as an advisor/director for several companies. He is an elected Fellow of the American Association for the Advancement of Science and the National Academy of Inventors. He received his MD, MS and PhD in biochemistry from the Albert Einstein College of Medicine in New York. He completed his residency and clinical and research fellowships at Massachusetts General Hospital and Harvard Medical School.

Howard J Federoff, MD, PhD is a distinguished professor of neurology at the University of California, Irvine. He is the former CEO of UCI Health, vice chancellor for Health Affairs and dean of the UCI School of Medicine. Prior to joining UCI Health, Federoff was executive vice president of Health Sciences and executive dean at Georgetown University. Federoff has published more than 275 peer-reviewed and invited articles, and serves on editorial boards of five journals. He co-founded MedGenesis Therapeutix and Brain Neurotherapy Bio, both advancing therapeutics for neurologic diseases. He became CEO of the regenerative medicine company, Aspen Neuroscience, Inc, in San Diego. Aspen is developing an autologous iPSC drug product for Parkinson’s disease. Federoff chaired the NIH Recombinant DNA Advisory Committee, the NHLBI Gene Therapy Resource and the Board of the Association of the Academic Health Centers. He has served as an advisor/director for several companies. He is an elected Fellow of the American Association for the Advancement of Science and the National Academy of Inventors. He received his MD, MS and PhD in biochemistry from the Albert Einstein College of Medicine in New York. He completed his residency and clinical and research fellowships at Massachusetts General Hospital and Harvard Medical School.


Howard J Federoff, MD, PhD is a distinguished professor of neurology at the University of California, Irvine. He is the former CEO of UCI Health, vice chancellor for Health Affairs and dean of the UCI School of Medicine. Prior to joining UCI Health, Federoff was executive vice president of Health Sciences and executive dean at Georgetown University. Federoff has published more than 275 peer-reviewed and invited articles, and serves on editorial boards of five journals. He co-founded MedGenesis Therapeutix and Brain Neurotherapy Bio, both advancing therapeutics for neurologic diseases. He became CEO of the regenerative medicine company, Aspen Neuroscience, Inc, in San Diego. Aspen is developing an autologous iPSC drug product for Parkinson’s disease. Federoff chaired the NIH Recombinant DNA Advisory Committee, the NHLBI Gene Therapy Resource and the Board of the Association of the Academic Health Centers. He has served as an advisor/director for several companies. He is an elected Fellow of the American Association for the Advancement of Science and the National Academy of Inventors. He received his MD, MS and PhD in biochemistry from the Albert Einstein College of Medicine in New York. He completed his residency and clinical and research fellowships at Massachusetts General Hospital and Harvard Medical School.

Howard J Federoff, MD, PhD is a distinguished professor of neurology at the University of California, Irvine. He is the former CEO of UCI Health, vice chancellor for Health Affairs and dean of the UCI School of Medicine. Prior to joining UCI Health, Federoff was executive vice president of Health Sciences and executive dean at Georgetown University. Federoff has published more than 275 peer-reviewed and invited articles, and serves on editorial boards of five journals. He co-founded MedGenesis Therapeutix and Brain Neurotherapy Bio, both advancing therapeutics for neurologic diseases. He became CEO of the regenerative medicine company, Aspen Neuroscience, Inc, in San Diego. Aspen is developing an autologous iPSC drug product for Parkinson’s disease. Federoff chaired the NIH Recombinant DNA Advisory Committee, the NHLBI Gene Therapy Resource and the Board of the Association of the Academic Health Centers. He has served as an advisor/director for several companies. He is an elected Fellow of the American Association for the Advancement of Science and the National Academy of Inventors. He received his MD, MS and PhD in biochemistry from the Albert Einstein College of Medicine in New York. He completed his residency and clinical and research fellowships at Massachusetts General Hospital and Harvard Medical School.
Publications
Roger A Barker 1 , Anders Bjorklund 2 , Don M Gash 3 , Alan Whone 4 , Amber Van Laar 5 , Jeffrey H Kordower 6 , Krystof Bankiewicz 7 , Karl Kieburtz 8 , Mart Saarma 9 , Sigrid Booms 10 , Henri J Huttunen 10 11 , Adrian Kells 5 , Massimo S Fiandaca 5 , A Jon Stoessl 12 , David Eidelberg 13 , Howard Federoff 14 , Merja Voutilainen 9 , David T Dexter 15 , Jamie Eberling 16 , Patrik Brundin 17 , Lyndsey Isaacs 18 , Leah Mursaleen 18 19 , Eros Bresolin 18 , Camille Carroll 20 , Alasdair Coles 21 , Brian Fiske 16 , Helen Matthews 18 , Codrin Lungu 22 , Richard K Wyse 18 , Simon Stott 18 , Anthony E Lang 23 GDNF and Parkinson's Disease: Where Next? A Summary From a Recent Workshop. J Parkinsons Dis. 2020 Jun 3. doi: 10.3233/JPD-202004.
 
Matthew A Edwardson 1 2 , Xiaogang Zhong 3 , Massimo S Fiandaca 4 5 6 , Howard J Federoff 4 7 , Amrita K Cheema 8 9 , Alexander W Dromerick 10 11 12 Author Correction: Plasma microRNA Markers of Upper Limb Recovery Following Human Stroke. Sci Rep 2020 Feb 7;10(1):2484. doi: 10.1038/s41598-020-58943-2.
 
Gresenz CR, Mitchell JM, Marrone J, Federoff HJ. Health Econ. Effect of early-stage Alzheimer's disease on household financial outcomes. 2020 Jan;29(1):18-29. doi: 10.1002/hec.3962. Epub 2019 Oct 24. PMID: 31650668
 
Nashine S, Subramaniam SR, Chwa M, Nesburn A, Kuppermann BD, Federoff H, Kenney MC. 3. PU-91 drug rescues human age-related macular degeneration RPE cells; implications for AMD therapeutics. Aging (Albany NY). 2019 Sep 2;11(17):6691-6713. doi: 10.18632/aging.102179. Epub 2019 Sep 2. PMID: 31477635
 
Heiss JD, Lungu C, Hammoud DA, Herscovitch P, Ehrlich DJ, Argersinger DP, Sinharay S, Scott G, Wu T, Federoff HJ, Zaghloul KA, Hallett M, Lonser RR, Bankiewicz KS. Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinson's disease. Mov Disord. 2019 Jul;34(7):1073-1078. doi: 10.1002/mds.27724. Epub 2019 May 30
 
Adelman ZN, Albritton LM, Boris-Lawrie K, Buchmeier MJ, Cannon P, Cho M, DiGiusto D, Donahue JK, Federoff HJ, Hammarskjold ML, Hardison AD, Hearing P, Lee B, Lee DA, Porteus MH, Ross LF, Ross SR, Wooley DP, Zoloth L (2018) Protect NIH's DNA advisory committee. Science. 2018 Oct 26;362(6413):409-410. doi: 10.1126/science.aav2483. No abstract available. PMID: 30361364
 
Fiandaca MS, Gross TJ, Johnson TM, Hu MT, Evetts S, Wade-Martins R, Merchant-Borna K, Bazarian J, Cheema AK, Mapstone M, Federoff HJ (2018) Potential Metabolomic Linkage in Blood between Parkinson's Disease and Traumatic Brain Injury. Metabolites. 2018 Sep 7;8(3). pii: E50. doi: 10.3390/metabo8030050. PMID: 30205491
 
Fiandaca MS, Mapstone M, Mahmoodi A, Gross T, Macciardi F, Cheema AK, Merchant-Borna K, Bazarian J, Federoff HJ. (2018) Plasma metabolomic biomarkers accurately classify acute mild traumatic brain injury from controls. PLoS One. 2018 Apr 20;13(4):e0195318. doi: 10.1371/journal.pone.0195318. eCollection 2018.
 
Fiandaca MS, Mapstone M, Connors E, Jacobson M, Monuki ES, Malik S, Macciardi F, Federoff HJ. (2017) Systems Healthcare: A Holistic Paradigm for the Future. BMC Syst Biol. 2017 Dec 19;11(1):142. doi: 10.1186/s12918-017-0521-2. Review, PMID:29258513
 
Mapstone M, Lin F, Nalls MA, Cheema AK, Singleton AB, Fiandaca MS, Federoff HJ. (2016) What success can teach us about failure: the plasma metabolome of older adults with superior memory and lessons for Alzheimer's disease. Neurobiol Aging. 2016 Nov 21. pii: S0197-4580(16)30294-9. doi: 10.1016/j.neurobiolaging.2016.11.007.
 
Sailer A, Scholz SW, Nalls MA, Schulte C, Federoff M, Price TR, Lees A, Ross OA, Dickson DW, Mok K, Mencacci NE, Schottlaender L, Chelban V, Ling H, O'Sullivan SS, Wood NW, Traynor BJ, Ferrucci L, Federoff HJ, Mhyre TR, Morris HR, Deuschl G, Quinn N, Widner H, Albanese A, Infante J, Bhatia KP, Poewe W, Oertel W, Höglinger GU, Wüllner U, Goldwurm S, Pellecchia MT, Ferreira J, Tolosa E, Bloem BR, Rascol O, Meissner WG, Hardy JA, Revesz T, Holton JL, Gasser T, Wenning GK, Singleton AB, Houlden H. (2016) A genome-wide association study in multiple system atrophy. European Multiple System Atrophy Study Group and the UK Multiple System Atrophy Study Group. Neurology. 2016 Oct 11;87(15):1591-1598.
 
Fiandaca MS, Zhong X, Cheema AK, Orquiza MH, Chidambaram S, Tan MT, Gresenz CR, FitzGerald KT, Nalls MA, Singleton AB, Mapstone M, Federoff HJ. (2015) Plasma 24-metabolite Panel Predicts Preclinical Transition to Clinical Stages of Alzheimer's Disease. Front Neurol. 2015 Nov 12;6:237. doi: 10.3389/fneur.2015.00237.
 
Su X, Chu Y, Kordower JH, Li B, Cao H, Huang L, Nishida M, Song L, Wang D, Federoff HJ. (2015) PGC-1a Promoter Methylation in Parkinson's Disease. PLoS One. 2015 Aug 28;10(8):e0134087. doi: 10.1371/journal.pone.0134087
 
Nalls MA, McLean CY, Rick J, Eberly S, Hutten SJ, Gwinn K, Sutherland M, Martinez M, Heutink P, Williams NM, Hardy J, Gasser T, Brice A, Price TR, Nicolas A, Keller MF, Molony C, Gibbs JR, Chen-Plotkin A, Suh E, Letson C, Fiandaca MS, Mapstone M, Federoff HJ, Noyce AJ, Morris H, Van Deerlin VM, Weintraub D, Zabetian C, Hernandez DG, Lesage S, Mullins M, Conley ED, Northover CA, Frasier M, Marek K, Day-Williams AG, Stone DJ, Ioannidis JP, Singleton AB; Parkinson's Disease Biomarkers Program and Parkinson's Progression Marker Initiative investigators*. Lancet Neurol. 2015 Aug 10. pii: S1474-4422(15)00178-7. doi: 10.1016/S1474-4422(15)00178-7. [Epub ahead of print], PMID:26271532
 
Fiandaca MS, Kapogiannis D, Mapstone M, Boxer A, Eitan E, Schwartz JB, Abner EL, Petersen RC, Federoff HJ, Miller BL, Goetzl EJ (2014) Identification of preclinical Alzheimer's disease by a profile of pathogenic proteins in neurally derived blood exosomes: A case-control study, Alzheimer’s Dementia, Aug 14. pii: S1552-5260(14)02469-8. doi: 10.1016/j.jalz.2014.06.008.

195. Fiandaca MS, Kapogiannis D, Mapstone M, Boxer A, Eitan E, Schwartz JB, Abner EL, Petersen RC, Federoff HJ, Miller BL, Goetzl EJ (2014) Identification of preclinical Alzheimer's disease by a profile of pathogenic proteins in neurally derived blood exosomes: A case-control study, Alzheimer’s Dementia, Aug 14. pii: S1552-5260(14)02469-8. doi: 10.1016/j.jalz.2014.06.008.

195. Fiandaca MS, Kapogiannis D, Mapstone M, Boxer A, Eitan E, Schwartz JB, Abner EL, Petersen RC, Federoff HJ, Miller BL, Goetzl EJ (2014) Identification of preclinical Alzheimer's disease by a profile of pathogenic proteins in neurally derived blood exosomes: A case-control study, Alzheimer’s Dementia, Aug 14. pii: S1552-5260(14)02469-8. doi: 10.1016/j.jalz.2014.06.008.
 
Mapstone M, Cheema AK, Fiandaca M, Zhong X, Timothy R. Mhyre TR, MacArthur LH, Hall WJ , Fisher SG, Peterson DR, Haley JM , Nazar MD, Rich SA, Berlau DJ, Peltz CB, Tan MT, Kawas CH, and Federoff HJ (2014) Plasma phospholipids identify antecedent memory impairment in older adults, Nature Medicine, doi:10.1038/nm3466, epub March 9.
 
Macarthur L, Ressom H, Shah S, Federoff HJ (2013) Network modeling to identify new mechanisms and therapeutic targets for Parkinson's disease. Expert Rev Neurother. Jun;13(6):685-93.
 
Scholz SW, Mhyre T, Ressom H, Shah S, Federoff HJ. (2012) Genomics and bioinformatics of Parkinson's disease. Cold Spring Harb Perspect Med., 2(7), a00949

189. Scholz SW, Mhyre T, Ressom H, Shah S, Federoff HJ. (2012) Genomics and bioinformatics of Parkinson's disease. Cold Spring Harb Perspect Med., 2(7), a00949

189. Scholz SW, Mhyre T, Ressom H, Shah S, Federoff HJ. (2012) Genomics and bioinformatics of Parkinson's disease. Cold Spring Harb Perspect Med., 2(7), a00949
 
Sailer A, Scholz SW, Gibbs JR, Tucci A, Johnson JO, Wood NW, Plagnol V, Hummerich H, Ding J, Hernandez D, Hardy J, Federoff HJ, Traynor BJ, Singleton AB, Houlden H. (2012) Exome sequencing in an SCA14 family demonstrates its utility in diagnosing heterogeneous diseases. Neurology, 79(2):127-31.
 
Galpern WR, Corrigan-Curay J, Lang AE, Kahn J, Tagle D, Barker RA, Freeman TB, Goetz CG, Kieburtz K, Kim SYH, Piantadosi S, Comstock Rick A, Federoff HJ. (2012) Sham Neurosurgical Procedures in Clinical Trials for Neurodegenerative Diseases: Scientific and Ethical Considerations. Lancet Neurology, 11(7): 643-50
 
Rogawski, MA and Federoff, HJ (2011) Disclosure of Clinical Trial Results When Product Development is Abandoned. Science Transl Med. 3(102)
 
Richardson RM, Kells AP, Rosenbluth KH, Salegio EA, Fiandaca MS, Larson PS, Starr PA, Martin AJ, Lonser RR, Federoff HJ, Forsayeth JR, Bankiewicz KS. (2011) Interventional MRI-guided Putaminal Delivery of AAV2-GDNF for a Planned Clinical Trial in Parkinson's Disease. Mol Ther. 19:1048-57
 
Zheng B, Liao Z, Locascio JJ, Lesniak KA, Roderick SS, Watt ML, Eklund AC, Zhang-James Y, Kim PD, Hauser MA, Grünblatt E, Moran LB, Mandel SA, Riederer P, Miller RM, Federoff HJ, Wüllner U, Papapetropoulos S, Youdim MB, Cantuti-Castelvetri I, Young AB, Vance JM, Davis RL, Hedreen JC, Adler CH, Beach TG, Graeber MB, Middleton FA, Rochet JC, Scherzer CR; Global PD Gene Expression (GPEX) Consortium. (2010) PGC-1a, a potential therapeutic target for early intervention in Parkinson's disease. Science Transl Med., 2(52):52ra73
 
Lee, B, Shin, JH, Jackalina, V, Petrucelli, L, West, AB, Ko, HS, Lee, YI, Maguire-Zeiss, KA, Bowers, WJ, Federoff, HJ, Dawson, VL and Dawson, TM (2010) Inhibitors of Leucine Rich Repeat Kinase 2 (LRRK2) Protect Against LRRK2-Models of Parkinson’s Disease. Nat Medicine 16(9):998-1000
 
Kells, AP, Eberling, J, Su, X, Pivirotto, P, Bringas, J, Hadaczek, P, Narrow, WC, Bowers, WJ, Federoff, HJ, Forsayeth, J, Bankiewicz, KS (2010) Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF. J. Neurosci. 14;30(28):9567-77
 
Zaia JA, Federoff H.J. (2009). Death in a gene-therapy trial. N Engl J Med. 29;361(18):1811
 
Sudol, KL, Mastrangelo, MA, Narrow, WC, Frazer, ME, Levites, YR, Golde, TE, Federoff, H.J., Bowers, WJ (2009) Generating Differentially Targeted Amyloid-beta Specific Intrabodies as a Passive Vaccination Strategy for Alzheimer's Disease. Mol Ther., 17(12):2031-40
 
Eberling, JL, Kells, AP, Pivirotto, P, Beyer J, Bringas J, Federoff H.J., Forsayeth J, Bankiewicz KS. (2009) Functional Effects of AAV2-GDNF on the Dopaminergic Nigrostriatal Pathway in Parkinsonian Rhesus Monkeys, Hum Gene Ther., 20(5):511-8.
 
Fiandaca MS, Varenika V, Eberling J, McKnight T, Bringas J, Pivirotto P, Beyer J, Hadaczek P, Bowers W, Park J, Federoff H., Forsayeth J, Bankiewicz KS (2009) Real-time MR imaging of adeno-associated viral vector delivery to the primate brain, Neuroimage, 47 Suppl 2:T27-35
 
Wuertzer, C.A., Sullivan, M., Qiu, M., and Federoff, H.J. (2008) CNS delivery of vectored prion-specific single-chain antibodies delays disease onset. Mol. Ther., 16(3):481-6
 
Harvey, B.K., Chang, C.F., Chiang, Y.H., Bowers, W.J., Morales, M., Hoffer, B.J., Wang, Y. and Federoff, H.J. (2003) HSV Amplicon Delivery of Glial Derived Neurotrophic Factor is Neuroprotective Against Ischemic Injury. Exp. Neurol. 183(1):47-55.
 
Brooks, A.I., Stein, C.S., Hughes, S.M., Heth, J., McCray, P.M. Jr, Sauter, S.L., Johnston, J.C., Cory-Slechta, D.A., Federoff, H.J. and Davidson, B.L. (2002) Functional Correction of Established CNS Deficits in an Animal Model of Lysosomal Storage Disease Using Feline Immunodeficiency Virus-based Vectors. Proc. Natl. Acad. Sci. (USA) 99(9):6216-21.
 
Richfield, E.K., Thiruchelvam, M.J., Cory-Slechta, D.A., Wuertzer, C., Gainetdinov, R.R., Caron, M.G., DiMonte, D.A., and Federoff, H.J. (2002) Behavioral and Neurochemical Effects of Wild-Type and Mutated Human ?-Synuclein in Transgenic Mice. Exp. Neurol. 175(1):35-48
 
Brooks, A.I., Cory-Slechta, D.A. and Federoff, H.J. (2000) Gene-experience Interaction Alters the Cholinergic Septohippocampal Pathway of Mice. Proc. Natl. Acad. Sci. (USA) 97(24):13378-83.
 
Brooks, A.I., Chadwick, C.A., Gelbard, H.A., Cory-Slechta, D.A. and Federoff, H.J. (1999) Paraquat Elicited Neurobehavioral Syndrome Caused by Dopaminergic Neuron Loss. Brain Res. 823:1-10.
 
Brooks, A., Muhkerjee, B., Panahian, N., Cory-Slechta, D., and Federoff, H.J. (1997) Nerve Growth Factor Somatic Mosaicism Produced by Herpes Virus-Directed Expression of cre Recombinase. Nature Biotech. (1): 57-62.
Other Experience
CEO
Aspen Neuroscience 2019—2020

CEO
UCI Health 2016—2018

Last updated
01/01/2021