A Cell Therapy Untested in Humans Saves a Baby With Cancer
This is not even at the early clinical trial stage, it was basically a moon-shot because nothing else was working. But hopeful:
Sounds almost too good to be true. But could we be on the cusp of a new era of genetic engineering as "the next big thing" like computers and the internet had been? I think we're just scratching the surface of what may be possible if they can customize cells like this. They made all sorts of modifications to these T cells to target the cancer but not healthy cells. And it appears to have worked in this case. I don't know if this can apply to all cancers or just certain kinds but it's exciting. Maybe it could even make chemo obsolete?The girl, Layla Richards of London, had acute lymphoblastic leukemia. Doctors tried the usual chemotherapy, a bone marrow transplant and a new type of biotech drug, but nothing worked. Layla did not have enough T cells to allow doctors to extract them and make a personalized therapy.
Her parents, Ashleigh Richards and Lisa Foley, were counseled to consider palliative care to keep her comfortable until she died, Dr. Qasim said. But the parents were not willing to do that.
In what might be a lucky coincidence, the hospital was already manufacturing Cellectis’s cells in preparation for clinical trials. “We had them in our freezers,” Dr. Qasim said. He is also a professor at University College London, which worked with Cellectis on the treatment.
So, with permission from her parents and Cellectis, Layla became the first person to receive the treatment, on what is sometimes called a compassionate use basis.
After getting the cells around the time of her first birthday in June, Layla went into remission, with no trace of leukemia found in her blood or bone marrow. She later had a second bone marrow transplant to help her immune system recover and is now at home.
Cellectis’s therapy uses T cells from a healthy donor that are first modified to target leukemia cells in much the same way Novartis and the others modify a patient’s own cells.
But to make its treatment “universal,” Cellectis then takes another step using another new technology that is generating huge excitement — genome editing. This refers to using molecular scissors to make precise changes to DNA, just as one might edit a word in a document. The genome editing technique that has gotten the most attention is known as Crispr-Cas9, though Cellectis used an older approach known as Talens.
A big concern with a nonpersonalized therapy is that the transplanted T cells will recognize the patient’s own cells as “foreign” and attack them. Cellectis used genome editing to knock out a gene so the transplanted T cells could not do that. It also inactivated another gene to help the transplanted T cells survive in the patient’s body.