Are we close to eradicating sickle cell disease once and for all? Here is some positive news.
I’ve been following what is happening with genome editing (also called gene editing). This is highly complex but in simple terms it is about a group of technologies that gives scientists the ability to change an organism’s DNA. These technologies allow genetic material to be added, removed or altered at pre-determined locations in the genome.
Several approaches to genome editing have been developed. A recent one is known as CRISPR-Cas9, which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9. The CRISPR-Cas9 system has generated a lot of excitement in the scientific community because it is faster, cheaper, more accurate and more efficient than other existing genome editing methods.
Scientists have announced that trials have begun with regards to incurable diseases using this technology. Two cancer patients have become the first humans outside China to be treated with a new genome editing technique.
Living here in Bahrain where sickle cell disease is a major health issue and personally having to live with multiple myeloma I find this news very exciting. Treatment associated with both diseases could be radically advanced when it comes to prevention and treatment.
There is in place opposition to this work due to the implications of manipulating human genes that could be passed down to future generations. Concerns are centred around scientists interfering with nature and as a result affecting those who have yet to be born.
In the current trial scientists are modifying T cells, a white cell that is critical when it comes to the immune system. The cells are removed from the body, manipulated in the laboratory and then returned to the body where they attack and destroy the cancer.
Currently, there are only two patients involved in the trial now underway. One has multiple myeloma and the other has sarcoma. This work is being conducted in the University of Pennsylvania and scientists have confirmed that they will share their results at the appropriate time.
There is great excitement across the world regarding this advance and many are hopeful we will know what this technology can provide before the end of 2019. Presently, much is unknown, and the trials may or may not work but one thing is for sure we appear to be on the cusp of a breakthrough.
It seems that the day is approaching when many of the incurable diseases of today will no longer be incurable. As I alluded to above, this advance does bring with it ethical questions. Being able to eradicate diseases that are due to inherited DNA is a stunning achievement and potentially allows many to live happy and fulfilled lives.
Finally, I am sure we are just around the corner from a grand debate as to the rights and wrongs of this technology once it is proven. This breakthrough will obviously stir up strong emotions on the side of those who support this technology as well as those strongly against. One thing is for sure, we are getting closer and closer to eradicating inherited diseases that cause misery to those who have to live with an accident of birth.