US Scientists Use Gene Editing Tool to Fix Mutation in Human Embryo.

Oregon scientists use breakthrough gene editing procedure to fix birth defect in embryo. According to a CBS TV News story, August 2 2017, researchers from the Oregon Health and Science University ran an experiment that safely repaired a mutated gene in a human embryo. The experiment used a procedure is called CRIPR cas 9, which is a genetic editing tool. The scientists edited out a defective mutation of DNA in the embryo. In a next step, the DNA automatically repaired itself without the mutation. The successful experiment demonstrated that you are scientists are technically capable of repairing mutations of DNA in embryos. This sort of thing was once considered science fiction, but now it is reality. Clearly the FDA will need to weigh in on whether or when medical clinical trials might begin in the US. There are many bio ethical considerations that have to be made before this experimental method comes to the marketplace. For example this type of technology could be a way of curing certain types of disease conditions such as enabling a person with HIV to have an immune system that could prevent the onset of AIDS or perhaps this technique could be used to fight certain forms of cancer. Down the line in the far future people might consider the possibility to alter human features to their liking. Scientist in other countries such as China, South Korea and others are using the gene editing technology to suit the needs of their localities.

The FDA Approved a New Gene Therapy to Treat Blood Cancers.

CBS News reported today that the FDA has approved a new type of therapy that will treat pediatric patients with a specific form of leukemia. The therapy is called CAR – T therapy. The concept behind the therapy is that patients have blood with T cells removed from their bloodstream and sent to a factory in New Jersey. The factory uses a specific type of DNA gene therapy that enables the T cells to recognize leukemia cells. The genetically modified T cells are then infused back into the patient. So the therapy works like a living drug that is injected into the patient that goes out and attacks cancer cells. 

The drug company, Novartis is working with doctors and their patients to deliver this new type of DNA therapy. So far, the therapy is producing some remarkable results by putting the cancer into remission. 

Dr. David Agus, from USC’s Westside Cancer Center, was very excited about about the promise for this new therapy. In addition to the therapy itself, the doctor explained that Novartis had developed an innovative payment plan based on the value of the treatment. That is, if the therapy works for a certain amount of time then the patient would pay a fee. If the therapy does not work there is no charge. This innovative therapy and payment system costs about $495,000. Whereas this fee is very large, it compares to be far less than a bone marrow transplant, which costs about $800,000. Novartis said that this new therapy will be tested for other diseases in the near future starting with lymphoma and other similar cancers. Dr. Agus is hopeful that this therapy could be used to treat other diseases such as diabetes or other conditions.

FDA Expanded Approval to a Cancer Immunotherapy

CBS’s doctor David Agus said in a May 30 story of CBS This Morning, that the FDA had recently expanded the uses for the immunotherapy known as KeyTruda for additional indications or medical conditions. All cancer cells operate in a type of stealth mode that allows it to hide from immunity cells that would normally eat it. The therapy targets cancer cells that have a biomarker that enables them to switch on a gene function that says it’s OK for the immune system to eat it. The therapy was originally used to fight lung cancer and some forms of skin cancer. The new FDA approval would expand the list to treat cancer searches kidney cancer, endometrial cancer, colon cancer and a growing list of other cancers. This immunotherapy enables patients previously without hope, to get an extended life of 1 to 3 years. Dr. Agus said that the cancer always returns, because the cancer mutates into a different form that is not suitable for treatment with this immunotherapy.