A little backstory on the little girl if you haven't seen the news, she was diagnosed with acute lymphoblastic leukemia and had undergone a chemotherapy regime and also a bone marrow transplant. For all intensive purposes these treatments were unfortunately unsuccessful. Fearing the loss of their young girl the parents consulted with the girls physicians and they decided to try a new route with a experimental therapy that was being tailored for cancer treatment by researchers University College London, Gene Editing.
So what is Gene Editing and how does it work? Well the basic idea falls into two points: cell attack and molecular scissors.
Cell Attack: immune system T-cells are removed from the patients body and then engineered to attack cancerous cells. They are engineered to have a extra gene that causes them to express certain protein receptors that recognize proteins present on the surface of cancer cells. Then these T-cells are placed back into the patient.
Molecular Scissors: specific DNA sequences are cut with "molecular scissors", this induces mutations that disable certain genes. During cancer therapy there are many drugs that target the patients immune system by recognizing T-cell surface proteins, so this technique can be used to disable certain genes that result in those surface antigens allowing them to be placed back into the patient without the risk of being destroyed by the therapy.
Overall this experimental cancer therapy was tremendously risky but fortunately has had a very positive turnout for the little girl. The research team is hesitant to say that this therapy is truly successful and awaits the start of full clinical trials in 2016 before they give their final judgement.
1. Reardon, S. (2015, November 6). Leukaemia success heralds wave of gene-editing therapies. Retrieved November 8, 2015, from http://www.nature.com/news/leukaemia-success-heralds-wave-of-gene-editing-therapies-1.18737
2. Le Page, M. (2015, November 5). Gene editing saves girl dying from leukaemia in world first. Retrieved November 8, 2015, from https://www.newscientist.com/article/dn28454-gene-editing-saves-life-of-girl-dying-from-leukaemia-in-world-first/
Although there are some skeptical of the treatment, I applaud the research team for taking a risk and trying a new therapy. I was reading in an article about the therapy that the goal is to produce T-cells from a single donor that can then be "gene edited" and used in multiple recipients. This would save the patient from undergoing another surgery and having their own T-cells extracted. If the research team can prove that this therapy is effective, it will be a beneficial therapy for numerous cancer patients. However, even if the treatment works the first time, how would the researchers confront cases of remission? Do you think the researchers can also look at techniques that might lower the risk of remission in patients who receive the therapy?
ReplyDeleteThis post strikes a chord with me as it pertains very closely to my thesis on stem cell transplant as a treatment for acute lymphoblastic leukemia! It turns out there is a massive amount of research currently being performed to not only determine new ALL treatments such as Gene Editing, but also to determine in which situations each treatment is appropriate. As you mention, many of these experimental treatments are incredibly risky physiologically as well as ethically. The question of children being able to provide informed consent and participate in a clinical trial plays a large role in ALL research as I'm sure this girl's family experienced. Along these lines, I recently read a CNN article about a little boy with ALL who was experiencing pain and nausea due to chemotherapy and was given THC oil to relieve these symptoms. Amazingly, the THC not only resolved the child's pain and nausea, but also increased his hematopoietic cell counts causing his mother to stop all chemotherapy treatment which, of course, ignited a Child Protective Services issue due to the stigma regarding marijuana and THC. Along the lines of genetics and ALL, CNN also recently published an article about 10-year-old twin girls who were both diagnosed with ALL suggesting a congenital element to this aggressive disease. Thank you, Jordan, for sharing and adding to my research on ALL treatments!
ReplyDeletehttp://www.cnn.com/2014/01/15/health/cannabis-landon-riddle/
http://www.cnn.com/2015/09/29/health/dove-family-twins-leukemia/