Stem cells: just 10 years from research to first clinical trials

I-Stem research laboratory was celebrating its 10 years in cell therapy R & D last October 27th in Paris: the right time to organize a press conference gathering international top medical opinion leaders in order to make an update on their pioneering work and the major steps forward to innovative stem cell therapies.

Cross funded by AFM-Téléthon and INSERM in 2005, I-Stem also joined the AFM-Téléthon Biotherapies Institute for Rare Diseases last 2012 which is an unrivalled strike force and a world reference hub in translational biomedical research and innovative medicines. 70 scientists work at I-Stem making it the biggest and leading lab in France dedicated to research on embryonic stem cells (ESC) as well as induced pluripotent cells (iPSC) up to date.

Its teams are launching clinical trials in rare genetic disorders affecting the eye and the skin aiming at getting treatments from 2017. They also prepare a trial in a neurodegenerative disease (Huntington) to get treatments from 2019. I-Stem is headed by Dr Marc Peschanski who was facilitating this meeting.He first reminded that in spite of a late start in 2005, France could catch up in the area of stem cells therapeutical research. He also mentioned the consortium INGESTEM (Infrastructure Nationale d’Ingénierie des Cellules Souches Pluripotentes) created in 2012, whose objective is to produce iPSC and differentiated cells for clinical applications.

Pr P. Menasché (Chief of the heart failure surgery unit, Georges Pompidou European Hospital) then made an update on applications currently studied in the cardiology area. The first French clinical trial using ESC has started last 2014, based on some initial work done by I-Stem in 2007 and funded by AFM-Téléthon. It has included a first patient suffering from serious heart failure last October 2014. In this trial, human embryonic stem cell (hESC)-derived cardiac progenitors are incorporated into a biocompatible fibrin gel patch. This is attached to the infarcted (damaged) portion of the patients heart, to anchor the cells. A pericardial flap, made of autologous cells from the patients, covers the patch, providing trophic factors to the ES progenitors.

Pr J.A. Sahel (chief of the “Institut de la Vision” – Inserm, CNRS, Université Paris VI), a member of the French Académie des Sciences, then presented a cell therapy phase I clinical trial project for retinitis pigmentosa developed in collaboration with I-Stem. It aims at evaluating the tolerance to a hESC-derived retinal pigmented epithelium cell transplantation. These cells being attached to a biological support (amniotic membrane) in these patients.

At last, Pr A. Benaceur-Griscelli (chief of the Stem Cell Hub at hospital Paul Brousse, Villejuif), also explained the prospects held out by discovering of the cell reprograming technology for pluripotent cells induction by S. Yamanaka’s team. iPS cells enable to enlarge applications for modeling numerous human pathologies, reproducing the dysfunction processes of damaged tissue, and developing targeted medicine. She also told participants about the project to set up a national Haplobank (iPSC cells  selected from matched HLA healthy donors). It aims at producing cell lines that are to be broadly available for manufacturing cell therapies matching the widest possible number of récipients.

As a conclusion to a fruitful debate, M. Peschanski came back on the AFM-Téléthon and Bpifrance partnership announcement made in the beginning of October. It’s about co-funding industrial facilities for developing and producing gene and cell therapies, and to build up a new economic sector. Indeed, clinical trials have multiplied and existing facilities do not fit anymore with the new needs for these innovative biotherapies. Sticking to its ambition of curing, AFM-Téléthon has made the decision to invest in this project for the benefit of the patients.

 

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