Unstoppable cancer in a girl is cured by cutting-edge treatment.
Unstoppable cancer in a girl is cured by cutting-edge treatment. A groundbreaking new kind of medicine has successfully treated a teenage girl’s untreatable cancer for the first time.
For Alyssa’s leukemia, every other treatment had failed.
To create a new living drug, physicians at Great Ormond Street Hospital performed a feat of biological engineering known as “base editing.”
Alyssa is still being monitored in case the cancer recurs, even though it is no longer detectable six months later.
Alyssa, a 13-year-old Leicester resident, was diagnosed with T-cell acute lymphoblastic leukemia in May 2016.
T-cells are meant to act as the body’s watchdogs, spotting and eliminating threats, but in Alyssa’s case, they turned dangerous and out of control.
Her cancer spread quickly. The bone marrow transplant and chemotherapy treatments failed to remove it from her body.
The only option left without the experimental drug would have been to keep Alyssa as comfortable as possible.
I would have eventually died, Alyssa said. Her mother, Kiona, recalled that she had been dreading Christmas last year, “thinking this is our last with her.” Then, she “just cried” through her daughter’s 13th birthday in January.
The large group of medical professionals and researchers used this resource to create a brand-new variety of T-cells that could track down and eliminate Alyssa’s cancerous T-cells.
They began by modifying healthy T-cells that were obtained from a donor.
- The T-cell targeting mechanism was disabled in the first base edit so they would not attack Alyssa’s body.
- The second eliminated a chemical label known as CD7 on all T-cells.
- The third modification was an invisibility cloak that stopped chemotherapy drugs from destroying the cells.
The T-cells were instructed to search for anything in her body that had the CD7 marking so they could all be destroyed, including the cancerous ones. This was the result of the final stage of genetic modification. Because of this, the marking must be removed from the therapy; otherwise, it would self-destruct.
With the second bone marrow transplant, Alyssa’s immune system, including her T cells, will be rebuilt if the treatment is successful.
A second bone marrow transplant was given to Alyssa after she had been in remission for a month to help her immune system recover.
Alyssa spent 16 weeks in the hospital and could not visit her brother, who was still in school, for fear that he might have brought in germs.
After the three-month checkup revealed signs of cancer once more, there were concerns. Her most recent two investigations, however, have been transparent.
“Simply put, you come to value everything. I’m just so happy I’m here right now, “Alyssa said.
“It’s absurd. Incredibly, I’ve had this opportunity, and I’m grateful for it as it will eventually benefit other kids as well.”
Most kids with leukemia respond to the standard therapies, but it’s thought that up to a dozen could benefit from this therapy each year.
Only Alyssa is the first of ten individuals to receive the medication as part of a clinical trial.
Dr. Robert Chiesa, of Great Ormond Street Hospital’s bone marrow transplant division, said: “It is incredibly thrilling. This is a relatively new area of medicine, and it’s fascinating that we can use the immune system to combat cancer.”
However, the technology only partially realizes the potential of base editing.
Dr. David Liu developed base editing at the Broad Institute, they told me it was “a little surreal” that people were receiving treatment only six years after the technology was created.
Each of the base edits in Alyssa’s therapy involved shattering a portion of her genetic code to render it useless. However, there are more complex applications where you can correct a flawed instruction rather than turning off one.
Therefore, base editing is already being tested for sickle-cell disease, familial high cholesterol, beta-thalassemia, and other conditions.
As science was now taking “key steps towards taking control of our genomes,” according to Dr. Liu, “therapeutic applications of base editing are just beginning.” He added that it was “humbling to be part of this era of therapeutic human gene editing.”
