Program of the workshop

BrainVectors workshop:  Neurotrophic factor gene delivery for neurodegenerative diseases: still a promising clinical paradigm?

 Program 

 September 12th

12:00-13:00 Registration and posters hanging.

12:00-13:30 Lunch

13:45 -14:00 Welcome addresses: 

Liliane Tenenbaum, Lausanne University Hospital,  BrainVectors coordinator

Nicole Déglon, Head of the Center for Neuroscience Research, Lausanne University Hospital.

Renaud Du Pasquier, Head of the Neurology Service, Lausanne University Hospital.

Session 1: Neuroprotective gene therapy for neurodegenerative diseases   Chairman: Marc Levivier

14:00-14:45  Mart Saarma, Helsinki (FI),   Potential of neurotrophic factors for Parkinson’s disease

14:45-15:30  Krys Bankiewicz, San Francisco (CA-USA),  AAV-mediated neurotrophic factor gene delivery : updates of clinical trials for Parkinson’s and Alzheimer diseases.

Coffee break

15:45 -16:30 Stéphane Palfi, Paris (FR),  The place of gene therapy for Parkinson’s disease among existing treatments: history of the ProSavin clinical trial .

16:30-17:00 Pavlina Konstantinova, Amsterdam (NL),   Public acceptance of Gene therapy products: risk:benefit ratio.

17:00-18:30  Round table:  The future of neurotrophic gene therapy for Parkinson’s disease. Moderator: Liliane Tenenbaum.

 Mart Saarma, Helsinki (FI),  Krystof Bankiewicz, San Francisco (CA-USA),  Harald Petry, Amsterdam (NL), Stéphane Palfi, Paris (FR), José-Luis Lanciego, Pamplona (SP).

September 13th

 Session 2: Optimization of neurotrophic factor gene delivery  Chairmen:Mart SaarmaandSebastian Kügler

9:00-9:30 Tomas Gonzales-Hernandez, Tenerife (SP)   Dose- and time-dependent effects of GDNF on the rat mesostriatal system: a rationale for controlling GDNF release.

9:30 -10:00 Sebastian Kügler, Göttingen (D)   Discontinuous GDNF delivery by GeneSwitch vectors

10:00-10:30 Mikko Airavaara, Helsinki (FI) Will new neurotrophic factors reveal the same limitations? The example of CDNF.

Coffee break

Session 3:  The BrainVectors industry-academia joint program Chairmen:Eric Kremer and Otto Merten

11:00– 11:15 Liliane Tenenbaum, Lausanne (CH)   Presentation of the BrainVectors consortium

11:15-11:45 Eric Kremer, Montpellier (F) Why a dog virus could be a neurobiologist’s best friend?

11:45-12:15 Cecilia Lundberg, Lund (S) Post-transcriptional regulation of transgene expression

12:15-12:45 Atze Das, Amsterdam (NL)  Tet systems for doxycycline-controlled gene expression

13:00-14:30 Lunch

Short communication by BrainVectors fellows

14:30 -14:45 Marie Humbert-Claude, Lausanne (CH) Pharmacological control of GDNF biological  effects in the brain by clinically-acceptable dox doses using a sensitive inducible AAV vector.

14:45 -15:00   Diego Pignataro, Pamplona, (SP) The development of new molecular tools for AAV-mediated CNS transduction.

15:00-15:15:Shelby Shrigley, Lund (SE) Inducible and cell-specific expression of GDNF using lentiviral vectors.

 15:30-17:00  Poster session+ coffe

 18:30 – 22:00   Social event  (restricted to invited speakers).

Session 4: Preclinical evaluation of cellular and molecular therapies in the CNS . Chairperson:  Liliane Tenenbaum

9:30– 10:00  José-Luis Lanciego, Pamplona (SP)  Gene therapy approaches for the treatment of Gaucher-related synucleinopathies like Parkinson's disease and dementia with Lewy bodies.

10:00-10:30  Jocelyne Bloch, Lausanne (CH)   Preclinical evaluation of adult brain cell autotransplantation in the MPTP non-human primate model.

Coffee break

11:00-11:30  Nicole Déglon, Lausanne (CH)   Molecular therapies for Huntington’s disease.

11:30-12:00  Bernard Schneider, Lausanne (CH)   Gene therapy for amyotrophic lateral sclerosis:
the importance of non-cell autonomous disease mechanisms.

12:00-12 :30  Nicolas Serratrice, Lausanne (CH) Evaluation of helper-dependent canine adenovirus vectors in nonhuman primate brain.

12:30-12 :40  Nicole Déglon, Lausanne (CH)    Presentation of the Swiss Transmed gene therapy network.

Lunch 12:45-14:00

Session 5: Gene therapy clinical trials setup and monitoring  Chairmen: Hueseyin Firat  and Otto Merten

 14:00-14:30 Ana Filipa Rodrigues, Oeiras (PT)   Impact of the cell source on the production of clinical-grade vectors: cGMP considerations and cell line engineering perspectives.

14:30-15:00   Saliha Moussaoui, Huningue (F)  Peripheral biomarkers of dementia of Alzheimer and Parkinson types.

15:00 – 15:30  Marie-Lise Gougeon , Paris (F) Immune response after rAAV2/5-hNAGLU injection in the brain of children with Sanfilippo type B syndrome.

15:30-16:45.   Round table:  Final conclusions and future directions of brain gene therapy: expectations and limitation issues

Krystof Bankiewicz, Hueseyin Firat, Nicole Déglon, Marie-Lise Gougeon and Marc Levivier.

Moderator: Pavlina Konstantinova.

End of BrainVectors workshop

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 All participants are kindly invited to attend the seminar of Krystof Bankiewicz, at the Auditorium Mathias Mayor in the “Département des Neurosciences Cliniques“(same building) in the frame of the:

Colloque du Département des Neurosciences Cliniques / Seminars of the Clinical Neurosciences Department.

 Chairman:  Marc Levivier.

 17:00-18:00   Krystof Bankiewicz,  San Francisco (CA-USA)   Image-guided vector delivery to the putamen with AAV2-AADC  vector in Parkinson’s disease and AADC deficiency.