New Gene Treatment Saves 1-Year-Old With Aggressive Form Of Cancer
A baby cancer patient on the verge of death may have been saved by an innovative gene-editing technique.
London's 1-year-old Layla Richards had an aggressive form of blood cancer called relapsed acute lymphoblastic leukemia, The Wall Street Journal reports.
When chemotherapy and a bone marrow transplant proved unsuccessful, doctors predicted she would die in a matter of months or even weeks.
Researchers from University College London and doctors at Great Ormond Street Hospital for Children sought a solution in French biotech firm Cellectis.
The firm developed a gene-editing tool called TALEN that alters the genes of donor immune cells so they can kill leukemia cells but not attack the recipient's healthy cells.
This tool, which had only previously been tested on HIV patients, additionally protects the donor's immune cells from leukemia drugs that would normally kill them.
Doctors at Great Ormond Street Hospital injected the new cells and a few weeks later, Layla's leukemia had gone into remission.
Layla remains cancer-free three months after treatment and is now recovering at home, according to The Washington Post.
Dr. Paul Veys of Great Ormond Street Hospital told The Wall Street Journal,
We'll start talking about a cure only a year or two from now.
Cellectis is currently obtaining funds for clinical TALEN trials expected to begin at some point next year.
Matt Kaiser, head of research at UK charity Bloodwise, said such studies will determine the long-term effects of TALEN as well as what kind of cancer patients respond to it.
Accessibility would never be an issue for TALEN, as the healthy donor cells can be shipped in tiny vials to doctors, who would then need to perform just a single injection.
Layla's miraculous recovery will be presented at the American Society of Hematology's annual meeting in Orlando next month.
Citations: Gene Editing Credited With Eliminating an Infants Aggressive Cancer in London (The Wall Street Journal), Baby close to dying from leukemia has no signs of cancer 2 months after designer cell therapy (CBC), Untested designer cell therapy saves a baby close to dying from leukemia (The Washington Post)