photo credit: GOSH UK website
I read an article of magnificent research done and a happy scientific success story. I thought I would spend a little time explaining the science and sharing it with you my readers as it's just truly magnificent that it worked.
Let me give you a bit of background about the story first. Baby Layla was born in June of 2014, a healthy 7lb 10 oz baby happy and content, but at about 3 months Layla was taken to the doctors with a suspected tummy bug. However, the prognosis was far bleaker and every mothers greatest fear they diagnosed her with Infant Acute Lymphoblastic Leukaemia (ALL). It was an extremely aggressive form of the ALL and she was sent to GOSH (Great Ormond Street Hospital) immediately and they started her on Chemotherapy. And despite every effort the cancer came back, there were no treatment options left. Her parents were given the news that there was nothing left.
However, researchers and her parents weren't willing to give up just yet. The doctors told the family about an extremely recent and experimental treatment that had only been tried on mice. It was however successful in mice and was being developed for a clinical trial in humans. The doctors had to meet with the ethical committee to get clearance in order to try this experimental treatment, but everyone was desperate to save Layla and it was the only option left. The emergency ethics committee agreed to let them try.
Here comes the science if you have any questions please leave them in comments, but here is my best attempt at explaining what they did.
They infused 1 ml of genetically engineered cells called UCART19 which are T-cells (immune cells) from donors. UCART19 cells have been modified using a molecular tool called TALEN to act like very accurate scissors going into the Tcells and at specific genes in order to make them behave in two specific ways. They hide from the powerful leukaemia drugs that would usually kill them and are reprogrammed to only target and fight against leukaemia cells. They are basically cloaked missiles within the weakened body that hide from the crossfire that has already been fighting the disease and come in with a sneak attack. They had to keep her in isolation to ensure she didn't get sick with something else as her immune system was extremely compromised and all they could do was wait. Two weeks went by and nothing then tada she developed a rash! That was a sign that it was working and I bet the most beautiful sight any of them had seen in far too long.
After about two months Layla is Cancer free. This may not be a permanent state, as it's an extremely aggressive disease, but any day turned into a potential future with 1 ml of experimental genetically engineered T-donor cells.
I'd like to appauled personally everyone involved I find your innovation and willingness to make the effort for this little girl amazing!
Want to read more head over to the GOSH website