According to Friday’s press release on the American site listing clinical trials, the first clinical trial using CRISPR-Cas 9 in humans has been launched at Ratisbonne Hospital in Germany. This experimental treatment, CTX001, was developed by CRISPR Therapeutics, and the study is jointly sponsored by Vertex Pharmaceuticals, i.e. two American companies.
In May 2018, the request to conduct this clinical trial in the United States was put on hold by the FDA (seeFDA remains cautious about CRISPR-based treatment for sickle cell anaemia), but in Europe, the process is going ahead: the (phase 1/2) clinical trial has enrolled twelve adults with beta-thalassemia. This hereditary disease is caused by a gene mutation causing a reduction in the quantity of haemoglobin in the blood – a molecule essential for the transportation of oxygen. Rather than directly correct this pathogenic mutation, the experimental treatment targets another DNA zone: it lifts the restriction on haemoglobin production which can substantially reduce the symptoms of this condition. It might be possible to treat sickle cell anaemia- another haemoglobin-related disorder – in the same way (see CRISPR: a potential remedy for sickle cell anaemia?). Patients do not witness direct treatment administration. Instead, their blood cells are collected, treated in vitro and then reinjected.
The race for clinical trials using CRISPR is up and running. The American company, Editas Medicine, should soon be launching a clinical trial using the CRISPR technique to treat a rare form of blindness (see Genome editing: what is the status of human treatment projects?).
Note from Gènéthique:
The first clinical trials using CRISPR appear to have got off to an enthusiastic start. But do we have the necessary hindsight to apply this type of technique to humans? Curing beta thalassemia via CRISPR: has the time come to switch to human trials?
STAT, Sharon Begley (31/08/2018)