Girl With Fatal Leukemia Given New Life-Saving CRISPR Treatment Declared Cancer Free In Just 28 Days

News Scientist

In another win for science, a young teenage girl with cancer, who was not responding to common types of treatment, has been given the most amazing news.

13-year old leukemia patient, Alyssa, from the United Kingdom, is the latest success story when it comes to CRISPR gene editing technology, also known as base editing. Incredibly, she now has no detectable cancer cells in her body.

She was given a dose of genetically edited immune cells, which were made to attack the leukemia, in a method that has been used with other types of cancer.

Leukemia happens to be a type of cancer that affects the bone marrow tissue, caused by mutated immune cells. Usually, this type of blood cancer is treated by killing the bone marrow cells within a patient’s body before they are given a bone marrow transplant from a donor. Usually, children tend to respond well to this type of treatment. But there are cases, just like this one, when it works at first but comes back, like Alyssa, or it doesn’t work at all.

So when this type of treatment fails, doctors may opt to use this Nobel Prize-winning option, which is the CAR-T cell therapy, instead. Alyssa was actually the first patient to be enrolled in this new clinical trial. It was also funded through the Medical Research Council’s (MRC) Development Pathway Funding Scheme.

Alyssa was given a dose of common immune system weapons, referred to as T cells, which were modified to attack the cancerous cells in her young body. And in order to avoid the normally extensive price tag on such treatments like these, the Great Ormond Street Hospital team from the University College London modified the donor T cells for her. Notably, the CAR T-cells were originally from a healthy donor.

Moreover, they were edited using a new base-editing technology which allowed them to actually hunt down and destroy the cancerous T-cells before they could attack each other.

Unbelievably, just 28 days after Alyssa’s life-saving treatment, she was placed in remission, and not too long after, was given her second bone marrow transplant to help bring her immune system back to better health.

This is considered quite miraculous considering had she not done this treatment, her only option would have been to be put in palliative care, which was basically to wait until she took her final breath of life.

According to Robert Chiesa, who was one of the doctors that treated Alyssa, he said in a statement released by the Great Ormond Street Hospital, “This is quite remarkable, although it is still a preliminary result, which needs to be monitored and confirmed over the next few months.”

New Atlas

Now, not even a year since her treatment, Alyssa has no detectable cancer, and is home and enjoying her life just like a young teenager should. However, she will still need to continue to follow up with her doctors and continue to check through the next few years to make sure that she is truly cancer free forever.

As for this study, the researchers presented their data for the very first time at the American Society of Hematology during their annual meeting in December, which was held in New Orleans in the United States. The hope is that with further research, and hopefully other success stories in patients using this type of treatment, it can be offered to others undergoing the same cancer diagnosis.