14 Jun 2012

First advances in cell therapy

Illustration for article: First advances in cell therapy
Cell therapy has been the subject of many research projects. The first human therapeutic trials have begun. The French Muscular Dystrophy Association (AFM-Téléthon) supports this research, which is already bearing fruit.

Rebuild skin

In 2009, Marc Peschanski's team at I-Stem was able to produce a complete epidermis thanks to human embryonic stem cells. The aim: to use this new possibility as an alternative therapy, for example to cure people who suffered major burns or patients with a rare skin disease.

By using human embryonic stem cells (hESC), I-Stem's researchers wer able to get keratinocytes similar this the ones found in human epidermis. Keratinocytes allow the permanent renewal of the skin.

These hESC have two basic characteristics : they can renew themselves indefinitly and they can differentiate to any cell type of the human body. Hence, hESC represent a major hope for regenerative medicine and for the potential identification of new pharmalogical treatment.

Myoblasts promising for DMD...

A trial led by Pr Jacques Tremblay, in Quebec province (Canada) showed that myoblast (muscle stem cells) transplant could have a positive impacts on patients suffering from Duchenne muscular dystrophy. The myoblast were collected from a compatible donor (father or mother). The tranplant took place in the biceps and required an anti-rejection treatment.

The results were published in 2006, in the Journal of Neuropathology and Experimental Neurology. They show that dystrophin, the protein that is non-functional for Duchenne's patients, was expressed normally with the transplant.

... And for heart attack

In 2006, Philippe Menasché's team at the European Georges Pompidou hospital in Paris (HEGP), published encouraging results of a phase I trial, in the Journal of the American College of Cardiology (JACC). Each patient received an injection of his own myoblasts in the portion of the heart (which is a muscle!) affected by the infarction. This trial, supported by AFM-Téléthon, led to a phase II trial performed by Myosix in Europe and the United States.

Meanwhile, Pr Patricia Lemarchand, at the Institut du Thorax (Inserm - Nantes, France) accomplished a cell therapy clinical trial in 2010, based on 101 persons who had survived a heart attack. Stem cells from their own bone marrow were injected  in the injured area of the heart. The results of this trial (BONAMI), supported by AFM, were published in the European Heart Journal.  Three months afther the attack, cell therapy proved its positive effect on cardiac muscle. Patients who received a cell therapy treatment were found to have a better recovery than the ones who didn't. Those results suggest that the cells that were injected stimulated and increased the recovery capacity of the cardiac muscle after the attack.

Another strategy was developed by Pr Lemarchand, in association with Pr Roncalli, from Toulouse's hospital, aimed at patients suffering from severe heart failure. They injected autologous stem cells directly in the cardiac muscle, in order to improve its functional performance. This strategy is being assessed.

Huntington: embryonic stem cells are restoring hope

In 2008, AFM-Téléthon announced in a joint press release with the Inserm (French National Institute for Health and Medical Research) and Evry-Val d’Essonne University in the Essonne département, that French researchers had succeeded in differentiating human embryonic stem cells in vitro into neuronal precursors and had grafted them into a rat's brain. There, the precursors continued their differentiation into GABAergic neurons in the striatum. This discovery, published in the Proceedings of the National Academy of Sciences (PNAS) on 13 October 2008, opens a new therapeutic pathway for treating Huntington's chorea, a rare neurodegenerative disease linked to the production of an abnormal protein called huntingtin.