A Michigan woman stricken by skin cancer has become the first person to receive a direct injection of genetic material - opening the door toward routinely using DNA as a disease-fighting drug, doctors have announced.
University of Michigan researchers said that they injected fat particles carrying a gene that they hope will trigger a cancer-fighting immune response into one of the woman's skin tumors. The woman, whose name was withheld, tolerated the procedure well and has been discharged from the University Hospital, researchers said.Previous efforts at gene therapy have involved a complicated procedure of removing cells from patients and then using a crippled virus to carry genes into those cells. The genetically modified cells are then infused back into the patient.
"For the first time, we have treated a human disease by introducing genetic material, DNA, directly into a patient. This approach marks a beginning - we have begun to use DNA as a drug," said Dr. Gary Nabel, head of the Michigan team.
The gene injected into the woman's tumor codes for HLA-B7, one of the proteins that tells the immune system whether or not to reject foreign tissue like transplanted organs. Researchers hope that the HLA-B7 gene will be recognized as foreign by the woman's immune system and thereby stimulate an attack on her tumors.
Emphasizing that direct gene injection, along with other types of gene therapy, is in its early stages of development, Nabel cautioned that "it is premature to expect that it will cure" the woman's cancer, a deadly type of skin cancer called malignant melanoma.
Deoxyribonucleic acid, or DNA, contains the blueprints for all life. Humans' DNA is packaged in 23 pairs of chromosomes which contain about 100,000 genes.
Another University of Michigan team also announced this week that a Canadian woman has become the first patient to undergo gene therapy for a rare, life-threatening cholesterol disorder.
The 29-year-old Quebec woman suffers from a severe form of familial hypercholesterolemia .
FH is an inherited disease in which the liver cannot clear low density lipoprotein (LDL), commonly called "bad" cholesterol, from the body. The steep cholesterol levels put patients at very high risk for heart attacks and strokes, often before age 10.
About one in a million Americans are stricken by severe FH. Currently, a liver transplant is the only effective treatment for such patients.
Stormie Jones perhaps is the best known American with severe familial hypercholesterolemia. The Texas girl underwent the world's first heart-liver transplant at Children's Hospital of Pittsburgh in 1984, when she was 61/2 years old. She died in November 1990 of heart rejection.