Famous billionaire and Twitter CEO Elon Musk was left stunned by the major breakthrough made by a UK-based research hospital, which led to the remarkable recovery of a 13-year-old girl with leukaemia. Alyssa, from Leicester, had been treated with all the conventional cancer therapies, from chemotherapy to a bone marrow transplant (BMT). But despite dozens of rounds of treatment, nothing seemed to work for good. When her cancer came back, researchers at GOSH, University College London and Great Ormond street decided something radical was needed.
In a first-of-its-kind trial, the 13-year-old received a brand new treatment which appeared to work wonders. Cathie Wood, the founder and CEO of asset management firm Arkinvest, tweeted: “Last month, BBC reported that GOSH, a research hospital in the UK, base edited the genome of a 12-year-old girl, Alyssa, suffering from leukemia.
“She had failed dozens of therapies and had no more options. Seven months later, she is cancer-free. Not many investors know about it.” But now, arguably the most prominent “investor” on the planet certainly does. Mr Musk, also the CEO of Tesla and SpaceX, simply responded: “Wow”.
Mr Musk’s interest could be significant for the future of this revolutionary form of cancer treatment. The billionaire’s cash, if he chose to invest, could go a long way as it is hoped that the technology will be used to treat more children in the future and become more widely available, and extra funding would be sure to help get it off the ground.
In the groundbreaking trial, Alyssa became the first person in the world to receive what are known as ‘universal’ CAR T-cells. These were pre-manufactured from a healthy volunteer donor.
The cells were gene-edited via a new base-editing technology and fitted with a Chimeric Antigen Receptor (CAR). This allowed them to recognise, hunt down and destroy cancerous T-cells without attacking each other.
T-cells are part of the body’s own defence system and develop from stem cells in the bone marrow. These can be trained to seek out cancerous cells. But traditional CAR T cell therapy cannot be used to treat leukaemia as these specific cells need to be designed to attack other T cells. Instead, they fight each other before they can prove useful.
However, the base-editing technique makes precise changes to the DNA of T cells, allowing special T cells to be hidden from other lines of attack, such as other cancer treatments and, crucially, other base-edited T cells.
This allows them to do their job without fighting each other, meaning the special T cells can indeed seek out and attack cancerous T cells successfully. It is hoped that this form of treatment can lead to fewer side effects than conventional cancer therapies and that it will become a treatment option for more cancers and more children.
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Just 28 days after receiving the CAR T cells, Alyssa went into remission and received a second bone marrow transplant, which restored her immune system.
Ahead of the treatment, she said: “Once I do it, people will know what they need to do, one way or another, so doing this will help people – of course I’m going to do it,” she said ahead of the treatment.”
She told ITV’s This Morning: “I just wanted to make a difference. What is the point of having a life that doesn’t have any meaning.”
Alyssa added: “If it wouldn’t help me, it would help somebody else, but then also it was another chance for me as well. And then even just doing it, it would make such a difference to the world. Even if I didn’t survive it, then again, my life would have had a meaning.”
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Seven months on, Alyssa is at home recovering from her treatment. The family is reportedly feeling positive that the leukaemia is now undetectable, although they are aware she will need to be closely monitored for the coming months and years.
Dr Robert Chiesa, Consultant in Bone Marrow Transplant and CAR T-cell therapy at GOSH, said: ”Only after she received her CD7 CAR-T cell therapy and a second bone marrow transplant in GOSH she has become leukaemia free. This is quite remarkable, although it is still a preliminary result, which needs to be monitored and confirmed over the next few months.
“The entire team here at GOSH are extremely happy for Alyssa and her family and it’s been a privilege to work with them over the past few months. We have been very impressed by how brave she is and nothing makes me happier than seeing her outside the hospital, going back to a more normal type of life.”
