Gene Therapy:
The Future of Modern Medicine
Although gene therapy has made progress over the year and will continue to do so, there has been debate surrounding the scientific phenomenon.
Timeline
1970 & 1972
Stanfield Roger had introduced the concept of replacing damaged genes with good, effective ones aka gene therapy was born. This was originally thought up to treat those suffering from genetic disorders. Then in 1972, Theodore Friedmann and Richard Roblin wrote and published an article titled "Gene therapy for human genetic disease?", which specified Stanfield Roger's proposal of gene therapy.
1986
Studying gene therapy was still an ongoing process at this point. Animals were chosen as the test subjects by the research team to observe how accurately and safely the correct genes were being delivered into bone marrow cells. Although this process did not harm the animals there was unfortunately too small of a number of cells that received the correct gene. Therefore, this method was disregarded as a useful treatment.
1989
The researchers teamed up with Dr. Steven Rosenberg to assess the safety behind gene therapy in cancer patients. They cultured tumor infiltrating lymphocytes (TIL cells) from patients diagnosed with malignant melanoma. From this point on, they created a virus, which acted as a vector, for the purpose of placing a DNA marker into the TIL cells. What the researchers gained from this was that TIL cells were most useful in cancer treatments and that the created virus was of use in humans.
1993
Researchers used gene therapy on newborns that had ADA deficiency. This time the correct genes were delivered to the immature blood cells that had been separated from their umbilical cords. It was done in the hopes that these genes will have more benefit for them in the long haul.
1985
The collaborations of Drs. W. French Anderson and Michael Blaese from the National Heart, Lung, and Blood Institute and the National Cancer Institute united as one in order to prove that cells from a patient stricken with ADA can be fixed in tissue culture. So they proceeded to use a retrovirus as a vector to transport the correct genes to the cells.
1988
Since using bone marrow cells proved to be an ineffective method, the team decided to use white blood cells in place of it. This proved to be a much greater success because the number of cells that received the correct gene increased by a dramatic amount. After this, they began researching for ways for this to be done in humans.
1990
Two female patients, who were at the ages of four and nine, were treated by Drs. Anderson, Blaese, and and Kenneth Culver for their ADA deficiency. The doctors used a virus as a vector to transport the precise ADA genes to the damaged cells. Both of the girls received the treatments for two years.
1999
Eighteen year old Jesse Gelsinger became the first person to die undergoing gene therapy due to vector associated toxicity. He suffered from nitrogen metabolism disorder ornithine transcarbamylase (OT) deficiency. After receiving the treatment, he suffered from a severe inflammatory response, followed by multiple organ failure. A later investigation revealed violations in protocol and procedure. The consequences that followed were the head of the investigation, James Wilson, being suspended, the University of Pennsylvania and the Children’s National Medical Center paying a $500,00 fine each since they were involved in the trial.