Novel therapies of neurological and psychiatric diseases


Stéphane Palfi Head Stéphane Palfi

Tél.: +33-(0)1 49 81 22 03

Fig3-site-Equipe-Palfi-1024x945Our team works on translational medicine projects, concerning neurodegenerative diseases, essentially Parkinson disease, Huntington disease,…. and Psychiatric disorders

Our major research programs relevant for Parkinson’s disease and related disorders as well as Psychiatric disorders are funded by national and/or European institutions as well as by private partnerships.

The aim of those projects are:

1- The neurological and neurosurgical assessment of biotherapies for neurodegenerative diseases at initial phase of clinical development following the demonstration of safety and efficacy in non-clinical experiments.

2- The development of neuromodulation approaches for drug-resistant psychiatric and neurological diseases using optogenetic technology to better understand brain circuitry abnormalities and define new therapeutic targets.

3- The development of new neurosurgical tools and devices to assess or administrate drugs, viral vectors, cells as well as electrical or light sources devices with the help of neuronavigation system and surgical robot.



Fig2-site-Equipe-Palfi                                       Fig1-site-Equipe-Palfi




Selected publications

Palfi S, Gurruchaga JM, Ralph GS, Lepetit H, Lavisse S, Buttery PC, Watts C, Miskin J, Kelleher M, Deeley S, Iwamuro H, Lefaucheur JP, Thiriez C, Fenelon G, Lucas C, Brugières P, Gabriel I, Abhay K, Drouot X, Tani N, Kas A, Ghaleh B, Le Corvoisier P, Dolphin P, Breen DP, Mason S, Guzman NV, Mazarakis ND, Radcliffe PA, Harrop R, Kingsman SM, Rascol O, Naylor S, Barker RA, Hantraye P, Remy P, Cesaro P, Mitrophanous KA.Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet. 2014 Mar 29; 383(9923):1138-46.

Lancet. 2014 Mar 29;383(9923):1138-46. doi: 10.1016/S0140-6736(13)61939-X. Epub 2014 Jan 10.

Jarraya B, Boulet S, Ralph GS, Jan C, Bonvento G, Azzouz M, Miskin JE, Shin M, Delzescaux T, Drouot X, Hérard AS, Day DM , Brouillet E, Kingsman SM, Hantraye P, Mitrophanous KA, Mazarakis ND, Palfi S. Dopamine Gene Therapy for Parkinson’s disease in a Non-human Primate without Associated dyskinesia. Science Transl Med 1, 2ra4 (2009).

Sci Transl Med. 2009 Oct 14;1(2):2ra4. doi: 10.1126/scitranslmed.3000130.

S. Palfi, E. Brouillet, B.Jarraya, J. Bloch, C. Jan, M. Shin, F. Condé, X. Li, P. Aebischer, P.Hantraye and N. Déglon. Expression of an N-terminal fragment of mutated huntingtin in the lateral putamen is sufficient to produce abnormal movement in non-human primates. Mol Ther. 2007 15(8):1444-51

Mol Ther. 2007 Aug;15(8):1444-51. Epub 2007 May 1.

X. Drouot, S. Oshino, B. Jarraya , L. Besret, H. Kishima, P. Remy, J. Dauguet, J. P. Lefaucheur, F. Dollé, F. Condé, M. Bottlaender, M. Peschanski, Y. Kéravel, P. Hantraye and S. Palfi. Functional recovery in a primate model of Parkinson’s disease following motor cortex stimulation. Neuron 2004 44; 769-778, 2004.

Neuron. 2004 Dec 2;44(5):769-78.

S. Palfi, L. Leventhal, S. Y. Ma, Y. Chu, M. Emborg, R. Bakay, N. Déglon , P. Hantraye , P. Aebischer and J. H. Kordower. Lentivirally delivered GDNF increases the number of striatal dopaminergic neurons in primate models of nigrostriatal degeneration. Journal of Neuroscience 22: 4942-4954, 2002.

J Neurosci. 2002 Jun 15;22(12):4942-54.

Kordower JH, Emborg ME, Bloch J, Ma SY, Chu Y, Leventhal L, McBride J, Chen EY, Palfi S, Roitberg BZ, Brown WD, Holden JE, Pyzalski R, Taylor MD, Carvey P, Ling Z, Trono D, Hantraye P, Deglon N, Aebischer P.Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 290 (5492):767-73, 2000.

Science. 2000 Oct 27;290(5492):767-73.

Kordower J.H., S Palfi, E Chen, S Ma, T Sendera, E J Cochran, E J Mufson, R Penn, C G Goetz and C D Comella. Clinico-pathological findings following intraventricular GDNF treatment in patient with Parkinson’s disease. Annals of Neurology .46 : 419-424, 1999.

Ann Neurol. 1999 Sep;46(3):419-24.

Palfi S., F Condé, D Riche, E Brouillet, C Dautry, V Mittoux, M Peschanski, and P Hantraye. Fetal neural grafts reverse cognitive deficits in a primate model of Huntington’s disease. Nature medecine. 4: 963-966, 1998.

Nat Med. 1998 Aug;4(8):963-6.