How a breakthrough in gene-editing from a Harvard lab saved the life of a girl with leukemia

Alyssa, 13, became the first patient to receive modified cell therapy to treat her leukemia in May 2022.Great Ormond Street Hospital for Children

  • A 13-year-old girl was the first patient in the world to receive a cell therapy called base editing in May.

  • The experimental treatment put his leukemia into remission for six months and more.

  • Base editing is a new gene editing technology that could lead to cures for many diseases.

A British teenager’s leukemia has gone into remission after she received an experimental cancer therapy that used a new gene-editing technology called base editing.

Alyssa, 13, was the first patient ever to receive background modified cell therapy after enrolling in a clinical trial in May. A month after the infusion and subsequent bone marrow transplant, her cancer, called T-cell acute lymphoblastic leukaemia, has gone into remission and the Leicester resident remains in remission six months later, Great said on 11 December. Ormond Street Hospital for Children. .

The staggering success is an early sign of the potential of base editing, a new technology discovered less than a decade ago in a Harvard University lab. Basic editing allows scientists to make ultra-precise changes to individual letters of DNA in cells. The goal is to make genetic changes that could treat or cure disease.

In addition to leukemia, biotech companies aim to use base-editing therapies to treat sickle cell disease and some types of cardiovascular disease.

Alyssa, a teenager from Leicester, England, received modified cell therapy to treat her leukaemia.

Alyssa, a teenager from Leicester, England, received modified cell therapy to treat her leukaemia.Family’s

In Alyssa’s case, her treatment used baseline editing to genetically modify T cells, a type of immune cell, from a healthy donor. These cells were modified to allow them to destroy cancer cells without attacking her own immune system, and then infused into Alyssa.

“It is our most sophisticated cell engineering yet and paves the way for other new treatments and ultimately a better future for sick children,” said Dr. Waseem Qasim, professor of cell and gene therapy and consultant immunologist at Great Ormond Street Hospital. a declaration.

Basic editing is an ultra-precise version of CRISPR gene editing

The basic editing was discovered in the laboratory of Harvard genomics researcher David Liu.

In November 2013, a 26-year-old chemist named Alexis Komor exchanged emails with Liu to figure out a research project, Insider reported. Komor and Liu sketched the basic editing idea within days, which kickstarted years of research that culminated in the publication of their findings in April 2016 in Nature, a leading scientific journal.

A headshot of Alexis Komor, core editing developer and genomics researcher at the University of California, San Diego

Alexis Komor, pioneer of basic editing and researcher at the University of California, San Diego.Courtesy of Alexis Komor

That paper has now been cited more than 3,400 times and helped launch several biotech companies, including Beam Therapeutics, cofounded by Liu, and Verve Therapeutics.

“It’s really crazy that this Frankenstein genome editing tool put together from all these different parts works so well,” Komor previously told Insider.

The technology addresses some of the limitations of CRISPR-Cas9, the Nobel Prize-winning gene-editing technology discovered in 2012.

CRISPR-Cas9 works by cutting a disease-causing gene from the DNA double strand before stitching it back together. Instead of performing a complete cut, base editors nick a single strand of DNA, simultaneously changing a single letter of DNA on the other strand. The cell then repairs that cut and uses the newly edited DNA as a template.

The technology offers researchers an unprecedented level of specificity, being able to make changes to the basic elements of the genetic code, or to nucleotides dubbed A, C, G and T. Researchers often compare basic editing with the using a pencil and eraser, whereas the traditional CRISPR system is more like using scissors and glue.

Despite these benefits, even basic editing has its limitations. Basic editors can only change a single letter of the genetic code, and researchers still haven’t figured out how to make all the changes. As of now, base editors can only make a few changes to nucleotides, limiting their potential. For example, basic editing may change an A to a G but not an A to a T.

Newer technology, such as prime editing, can write and edit longer strokes of letters.

Big Pharma sees “huge potential” in basic editing

The potential of background editing doesn’t stop with Alyssa, as researchers are beginning to test background editing therapies in a range of conditions. Earlier this month, the FDA cleared a different T-cell therapy similar to Alyssa’s treatment to begin trials in humans.

“Seeing more patients like Alyssa experience remission from this treatment would mean a lot to both the researchers who developed the background editing and the patient communities who could benefit from it,” Liu told Insider in an email.

Big Pharma has also shown interest, with Pfizer agreeing in January 2022 to pay $300 million upfront to partner with Beam on several core editing therapies.

Mikael Dolsten, Pfizer’s chief scientific officer, told Insider at the time that he expects baseline editing to impact large patient populations over time, and not just rare diseases.

“This is a healing technology and it can really transform genetic medicine for the future,” Dolsten said. “It has enormous potential.”

Read the original article on Business Insider

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