1984-1986: In 1984, Randall Willis and collaborators partially cured Lesch-Nyhan disease—an enzyme deficiency that causes neurological problems and self-mutilation behavior—in human cells, and in 1986, Philip Kantoff and colleagues corrected another enzyme deficiency in human blood cells. Now to try the methods in people...
Funded in part by the US Public Health Services.
1990: The first FDA-approved gene therapy trial takes place at the NIH, when two girls with a type of severe combined immunodeficiency syndrome (SCID) caused by an enzyme deficiency are infused with modified versions of their own blood cells that carries genes for the functioning version of the enzyme. The effects are temporary but observable. For the long term, the patients still have to receive enzyme injections to treat their disease.
Funded by the NIH.
2002: Further forays in using retroviruses for gene therapy, however, bring tragedy. Two of 10 children treated with gene therapy for SCID in a French trial develop leukemia, researchers announced in 2002, and it is discovered that the virus had inserted genes in several unexpected places around the genome, leading the cells to become cancerous. After this incident, and the death in 1999 of a young man in a clinical trial, apparently because of an immune reaction to the treatment, gene therapy research grounds nearly to a halt.
Despite the pall these events laid over the field, scientists work to find safer ways to practice gene therapy. Lentiviruses, a class of retroviruses, show particular promise because they often did not provoke an immune response, and stringent testing of modified cells for suspicious gene insertions is now a necessary stage of experiments.
2009: A resounding success brings gene therapy back into the limelight: Jean Bennett and Albert Maguire announce that 12 patients with a degenerative disorder leading to total blindness were treated with viruses bearing a replacement gene, and all regain some vision.
Funded in part by the National Center for Research Resources.
2011: Another success makes headlines: David Porter and Carl June report that immune cells modified with gene therapy had cured two terminal leukemia patients of their cancer. Though the treatment is still in the very early stages, its striking results, along with the success of the blindness study two years prior, bring new attention to gene therapy’s potential.
The National Cancer Institute is now funding a study to determine what specific characteristics led to that trial’s success, comparing two sets of modified immune cells in patients. Porter, June, and their collaborators are currently recruiting patients.
Funded in part by the NIH.
This post is part of a blog carnival called Public Science Triumphs, a project that shows the great value of public funding of basic science at a time when Congress is considering deep cuts to agencies like the National Science Foundation and the National Institutes of Health.
Annalee Newitz of io9, the inspiration for the series, writes: “Did you use a browser to zoom around on the internet today? Have you ever been vaccinated? If you answered yes to either of those questions, your life has already been made better through publicly funded science in America. Public science is basic scientific research funded by governments, and just in America alone it's led to breakthroughs in everything from medicine to clean energy. But now public science is under threat. Here's why we can't afford to lose it.”
The budget proposal comes out on November 23rd. If you’d like to learn more about the tremendous successes of publicly funded science and why it should not be cut, check out Maggie Koerth-Baker’s post on space dust or Keith Veronese's post on single-molecule chemistry.