In 1984, Richard Mulligan devised a method to introduce foreign DNA into cells using a retrovirus. Anderson saw this as a blessing and wanted to try this out in clinical trials, for which he applied to the NIH’s Human Gene Therapy Subcommittee to allow him to proceed. The committee however declined his request but did offer him a hearing to put his case.
He then managed to convince the committee members to grant him the permission to go ahead with his experiments. In 1989 he conducted the first human gene therapy safety test where a harmless marker was injected into a 52 year old man. The test succeeded and the following year he treated a 4 year old girl, Ashanti DeSilva, who was suffering from an immune disorder caused by a defective ADA gene. At the time the only solution was to treat the patient with an artificial PEG-ADA supplement, though Anderson set out to make a more permanent cure. He introduced a healthy functioning copy of the gene using a modified retrovirus.
Presently DeSilva is alive and well, but still taking her PEG-ADA supplements, the issue is still not clear as to whether it was the gene therapy treatment carried out by Anderson or the supplement itself that has been keeping her going all these years. The trial received a mixed reception, but nonetheless it was a shot in the arm for gene therapy enthusiasts all over the world.