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

Alyssa, 13, was 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 benefit from a cell therapy called Basic Edition in May.

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

  • Basic editing is a new gene-editing technology that could help cure many diseases.

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

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

The spectacular success is a harbinger of the potential of basic editing, a new technology discovered less than a decade ago in a lab at Harvard University. Basic editing allows scientists to make ultra-precise changes to single letters of DNA in cells. The goal is to make genetic adjustments that could treat or cure diseases.

Beyond leukemia, biotech companies aim to use base-editing therapies to treat sickle cell disease and certain 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.Belonging to the family

In Alyssa’s case, her treatment used base 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 its own immune system, then infused into Alyssa.

“This is our most sophisticated cell engineering to date and paves the way for other new treatments and ultimately a brighter 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 core edition was discovered in the lab of Harvard genomics researcher David Liu.

In November 2013, a 26-year-old chemist named Alexis Komor exchanged emails with Liu to find a research project, as Insider reported. Komor and Liu sketched out the basic editing idea in just a few days, sparking years of research culminating in the publication of their results in April 2016 in Nature, a leading scientific journal.

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

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

This article has now been cited over 3,400 times and helped launch several biotech companies, including Beam Therapeutics, co-founded by Liu, and Verve Therapeutics.

“It’s really crazy that this Frankenstein genome-editing tool assembled 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 pathogenic gene from the DNA double helix before stitching it back together. Instead of making a full cut, base editors cut a single strand of DNA, while simultaneously modifying a single letter of DNA on the other strand. The cell then repairs this nick and uses the newly edited DNA as a template.

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

Despite these advantages, the basic edition also has its limitations. Basic editors can only modify a single letter of the genetic code, and researchers have yet to figure out how to make all the changes. Currently, base editors can only make certain changes to nucleotides, which limits its potential. For example, the base set can change an A to G but not an A to T.

Newer technology, like master editing, can write and edit longer portions of letters.

Big Pharma sees ‘tremendous potential’ in basic edition

The potential for base editing doesn’t stop with Alyssa, as researchers are beginning to test base editing therapies in a variety of conditions. Earlier this month, the FDA cleared another T-cell therapy similar to Alyssa’s treatment to begin the first human trials.

“To see more patients like Alyssa go into remission from this treatment would mean so much both to the researchers who developed the Core Edition and to the patient communities who could benefit from it,” Liu told Insider in a post. E-mail.

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

Mikael Dolsten, chief scientific officer of Pfizer, told Insider at the time that he expected the Core Edition to impact large patient populations over time — not just rare diseases.

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

Read the original article on Business Insider

Leave a Reply

Your email address will not be published. Required fields are marked *