Alyssa, a young girl diagnosed with acute lymphoblastic leukemia, and she was admitted to Great Ormond Street Hospital for Children
The first person in the world to be infused with base edited immune cells was admitted to the Great Ormond Street Hospital for Children in London.
According to a University College London news release those edited cells are given to the patient to find and destroy T-cells in the body.
Base editing has been life-changing for Alyssa. The UK teen was diagnosed with T-cell acute lymphoblastic leukemia, or T-ALL, in 2021. T-ALL accounts for 10% to 15% of all ALL cases, and is one of the most common children’s cancers.
After she received chemotherapy and a bone marrow transplant, Alyssa’s cancer persisted, leaving palliative care as her only remaining option for treatment.
“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 in a hospital news release.
The findings of Alyssa’s case were presented at the American Society of Hematology’s annual meeting, and the team at Great Ormond Street hopes to enroll at least 10 more patients to trial the therapy.
Base editing is an even more precise gene editing technique than CRISPR and has fewer risks of unwanted effects on the chromosomes and thus less risk of side effects.
CRISPR-Improves Cancer Immunotherapy Through Living CAR T Cells: A Medical Miracle for Kopp and Bartolome
“This is quite remarkable, although it is still a preliminary result, which needs to be monitored and confirmed over the next few months,” said Dr. Robert Chiesa, a consultant in bone marrow transplant and CAR T-cell therapy at Great Ormond Street, in the statement.
Bartolome is a former NBA basketball player. “It sounded like something from a science fiction movie. Bartolome says that he thought that was cool.
For their part, Kopp and Bartolome are thrilled. Kopp’s been in remission for more than two years. “Now, I joke that I’m genetically modified, because I’ve been a Homeopath all my life, pretty much,” he says. There is a small amount of cells that can change my life. Wow. Just, truly, medical miracle.”
With the use of the new genetic engineering tool, it has been shown that it can be used to treat a number of genetic diseases, including sickle cell disease. Some attempts to use CRISPR to treat other diseases have not gone well recently.
This therapy is a different form of therapy than drugs and it’s called a living therapy. “You’re injecting into your patient a drug that is alive, that can persist for weeks to months and sometimes beyond that — for years.” McGuirk and others are hoping CRISPR can make better CAR T-cell living drugs, such as versions that are more potent and effective at treating more common cancers.
The CAR T-cell treatments could be cheaper than the custom made ones. “I’m totally excited about this. This would be a game-changer that way, with a total new approach,” says Dr. Carl June is a CAR T-cell pioneer at the University of Pennsylvania who is not involved in the studies that included Kopp and Bartolome.
Source: https://www.npr.org/sections/health-shots/2022/12/13/1140384354/crispr-improves-cancer-immunotherapy-car-t-cell
The Immunotherapy of Car-T Cell Lymomas: How Well Does It Work? Dr. Iyer and Fyodor Urnov
McGuirk says that this is the most exciting time in his career. I have always been very excited by the work we’ve been doing. But this is unprecedented.”
The research is being led by Dr. Iyer at the University of Texas M.D. Anderson Cancer Center in Houston. Both studies are being sponsored by CRISPR Therapeutics in Boston. “We have not had a therapy like this of such promise in T cell lymphomas,” Iyer says.
“The prospects are much brighter than anyone could have dreamed of 10 years ago,” agrees Fyodor Urnov, a gene-editing scientist at the University of California, Berkeley, who was not involved in the research. The field is progressing quickly.
“The results so far are encouraging,” Sadelain says. “However, the rate of responses is not as good as you obtain with a patient’s own cells. We need more investigation.
More research is needed to find out how well off-the-shelf therapies work, how long they last, and what can be done to improve them.
“When you consider the number of patients that would have been killed, that’s a big advance,” McGuirk says. “None of us are satisfied with that. We need to do better, better better.” For example, he says, some of the shortcomings might be overcome by giving patients more than one infusion.
Source: https://www.npr.org/sections/health-shots/2022/12/13/1140384354/crispr-improves-cancer-immunotherapy-car-t-cell
Bartolome’s birthday: on the day the doctors couldn’t find cancer in his body. He didn’t know he had cancer
Bartolome will never forget the day the doctors said they couldn’t find cancer in his body. That was more than a year ago. It was a life-changing event. He says that he was bubbling up inside. That day was great. Since then, I just thank my lucky stars.