New Base Editing Gene Therapy Can Cure Teenager's incurable Leukemia
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According to researchers at University College London a new base editing gene therapy can cure teenagers of incurable leukemia. A UK teenager suffering from incurable leukemia has become the first person to be treated with this new revolutionary theory that uses “base-edited” immune T-cells from a donor.

According to Waseem Qasim, a University College London researcher working on the project, “This is a great demonstration of how, with expert teams and infrastructure, we can link cutting-edge technologies in the lab with real results in the hospital for patients. It’s our most sophisticated cell engineering so far and paves the way for other new treatments and ultimately better futures for sick children.”

Although the technology behind this new treatment was announced less than five years ago, it’s revolutionary in more ways than one. Researchers originally called it “CRISPR 2.0.” It is much more targeted and precise than traditional CRISPR gene editing techniques, with researchers saying it functions essentially like a pencil rather than cutting and pasting.

Our genes are a sequence of 3 billion base pairs, made up of chemical units that can be denoted by the letters A, C, G, and T. Normally, if CRISPR was like using scissors to cut strings into different lengths, then base editing is like erasing letter combinations or adding new ones – this way you can edit single letters in baes without changing the length of the DNA.

 Alena Pance, a geneticist from the University of Hertfordshire explained, “Base editors allow precise nucleotide substitutions in the DNA without having to break the DNA to force the cell machinery to repair it and in the process make mistakes, which is the main mechanism of CRISPR-Cas9. This property of base editors and the advances to improve their accuracy means that the exact wanted changes can be made, leaving no trace on the DNA otherwise, reducing the potential of collateral effects.”

In a recent case study, base editing was used to create a new treatment for a thirteen-year-old patient with T-cell acute lymphoblastic leukemia, an incurable form of cancer where patients’ T cells do not mature effectively and disrupt other blood cells.

For the past few years, we’ve seen remarkable breakthroughs in gene therapies for cancer. In order to combat this, scientists use a technique of editing a patient’s T cells to target specific cancers without damaging the rest of their white blood cells. As you probably know, however, it can be extremely difficult to ensure that modified T-cells don’t destroy other modified cells because of the risk for t-cell acute lymphoblastic leukemia.

In this study, researchers were able to make several novel edits to T cells supplied by a healthy adult donor. The base edits changed the surface of the cells so they were indistinguishable from regular T cells.

Other base edits to the T cells removed markers that were specific to the donor, turning the cells into a “universal” treatment. This means it can be stockpiled rather than made per the patient’s specific needs and is quicker and cheaper than current treatments.New Base Editing Gene Therapy Can Cure Teenager's incurable Leukemia_1Although gene therapy has been successfully done in the past, prior examples of it were slow and time-consuming. Harvesting immune cells from a patient, modifying them genetically, and then transplanting them back into the patient is an onerous process that limits its prevalence.

The article discusses new research on a bank of cells that can be used to treat many patients. In other words, this is a scalable, commercialized, and standardized medicinal product.

A groundbreaking clinical trial is underway with the first patient being administered base-edited T cells. Initial reports from the young patient who was enrolled in the trial are staggering, going from having exhausted all available treatments to having a remarkable recovery in a matter of weeks.

The patient, Alyssa, was cured of leukemia in just one month after receiving the experimental therapy. Six months later, she is still in complete remission.

Preliminary clinical trials show that base editing of leukemia cells can be achieved. However, this is just the beginning of technology. Three other trials are underway testing base editing to treat sickle-cell anemia, high cholesterol, and beta-thalassemia.

One researcher who was instrumental in developing this base editing technology just a few short years ago, David Liu, said these few therapeutic applications for the technology are only just the beginning of what base editing can achieve.

“The vast majority of known genetic diseases require precise target gene corrections as opposed to gene disruptions or deletions,” Liu said at a recent conference for gene editing. “[These emerging technologies] give us hope that one day we may no longer be so beholden to the errors in our DNA.”

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