W. French Anderson, M.D., a gene therapy pioneer, will be released from prison on May 17 after serving almost 12 years of a 14-year sentence, having been convicted in 2006 of molesting the young daughter of one of his colleagues.
Dr. Anderson has always maintained his innocence, and despite his appeals being rejected by nine courts, he plans to pursue exoneration by pleading his case before the U.S. Supreme Court.
Dr. Anderson, the founding editor of Human Gene Therapy, published by Mary Ann Liebert, Inc. publishers, led the NIH team that conducted the first human gene therapy trial in 1990. The researchers used retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children suffering from severe combined immunodeficiency (ADA-SCID).
According to the NIH, SCID is a group of rare disorders caused by mutations in different genes involved in the development and function of infection-fighting immune cells. Infants with SCID appear healthy at birth but are extremely susceptible to severe infections. The condition is fatal, usually within the first year or two of life, unless infants receive immune-restoring treatments, such as transplants of blood-forming stem cells, gene therapy, or enzyme therapy.
The 1990 trials demonstrated that the number of T cells normalized along with cellular and humoral immune responses. Treatment was halted after two years, but follow up studies showed that the integrated vector and expression of the ADA gene in T cells continued. However, some scientists have labeled the results of the trial ambiguous as the two children still receive low does of PEG-ADA by injection.
Under a CRADA, Dr. Anderson in 1991 founded a company, Genetic Therapy (GTI), which set up a manufacturing facility near the NIH where he worked as a government scientist. Four years later, he was granted a gene therapy patent (covering genetically engineered human cells that encoded a marker or therapeutic to be expressed in vivo), which was owned by the government and was licensed to GTI. Novartis later purchased GTI for $325 million and subsequently received (in 2016) the first FDA-approved gene therapy license in the U.S.
Dr. Anderson took up a position at the University of Southern California (USC) in Los Angeles in 1992 where Novartis continued to fund his gene therapy research.
He has stated that his intention is that he wants to resume a career in science.
