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Researchers and clinicians at Leiden University Medical Centre (LUMC) are about to begin a milestone clinical study. It will be the first time a stem cell gene therapy developed in the Netherlands is used in a clinic. The therapy will be used to treat children with SCID, a rare disorder where children are born without essential immune cells.
‘We are the first group worldwide to have developed stem cell gene therapy for this type of SCID, RAG1-SCID to be precise. It is also the first stem cell gene therapy initiated by the Netherlands that will actually be used in patients,’ says Professor of Stem Cell Biology Frank Staal. The researchers have spent a total of 15 years developing the therapy and consider the opening of the study a milestone. ‘We can finally start and treat our first patients. Because it is a very rare disorder in newborns, the first patient should probably still be born,’ says Staal.