Interviews

Interview Marc Lechuga, Engineer CECS

Principal Investigator "High Throughput Screening Team"

1. On which pathology is your research focused on I-Stem ?

I work on the screening program of identification of potentially therapeutic molecules (High Throughput Screening or HTS). This program develops the other therapeutic option of against the rare diseases: the drug.
This field of expertise relates to all target pathologies of I-Stem. The feasibility and the availability depend on the detection of the cellular effects of a mutation.

2. What are your expectations from stem cells within your research area ?

The relevance in terms of effectiveness of a compound against any given pathogenesis is strongly related to the scientific value of the studied pathological model.  The study of the effects of modulating molecules with respect to an enzymatic activity obviously leads to less useful results than the screening of these molecules on a functional target like a cell model. The stem cells make possible to reach cellular models of rare diseases and “to configure them” to the standards of the HTS, i.e. to direct them towards differentiations which support the intrinsic relevance of the model and/or allows the handling them in micro container (microplate) using automated facilities.

3. What is specifically relevant in hES cells research ?

Currently, the majority of drugs on the market result from screenings of molecules on cells. In industry, these targets are often GMO of animal origin containing of human genes and emerge of this fact on a pharmaceutical development long and tiresome so much because of the model is very distant from the patient. Istem have human embryonic stem cells likely to offer an additional since they are able to differentiate in any tissue. In addition, some of our lines carry causal mutation of rare diseases. These characteristics make them valuable tools with no comparison with usual models existing in conventional laboratories public or private.

4. Which aims are you expecting to reach within the next 10 years  ?

The development of drugs until its sale authorization requires on average eight years. The orphan drug status allows to save time within the clinical trials and the lawful steps. I think the will be able to save an invaluable time on the discovery of the most efficient molecules towards the drug. I hope that two molecules resulting from my activity will be in lane to clinical tests as well as three to five screening campaign per year.


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