Untested Therapy Saves Baby Dying from Leukemia

Baby Layla is seen at Great Ormond Street Hospital (GOSH) in London in this October 28, 2015 handout photo by the hospital released on November 5, 2015. (Sharon Lees/Great Ormond Street Hospital/Reuters)Baby Layla is seen at Great Ormond Street Hospital (GOSH) in London in this October 28, 2015 handout photo by the hospital released on November 5, 2015. (Sharon Lees/Great Ormond Street Hospital/Reuters)

A few weeks ago MedCareerNews reported on a new immunotherapy clinical trial that’s turning patients’ bodies into cancer fighting agents and now a similar technique has just saved the life of a lucky little girl.

Five months ago, Layla Richards’s parents were told that their infant daughter was about to die. She had been undergoing treatment for acute lymphoblastic leukemia since she was just 14 weeks old. She’d been subjected to chemotherapy, flown across the U.K. for experimental procedures, undergone bone marrow transplants and endured countless blood tests. The majority of her year-long life had been spent in intensive care units. And still, cancer seemed to have conquered her small body. Her doctors were out of ideas.

But the little girl’s family didn’t give up and asked the doctors to try anything, no matter the risk. “Anything” turned out to be a highly experimental and possibly ineffective therapy that had never before been tested on anything larger than a lab mouse. It might not have any effect on Layla’s leukemia, the doctors warned. It might even make her sicker.

New immunotherapy treatment

Her parents approved the treatment and Layla Richards, a 1-year-old from London, became the first person in the world to be treated for cancer using “designer immune cells,” according to a write-up in “Nature,” which noted that the method has been tried on patients with HIV. If her body remains cancer-free, as it has been for the past several months, she could become the first person to be cured by them. According to Nature, the team will present the case in December at a meeting of the American Society of Hematology.

The treatment involves using tiny molecular “scissors” to edit genes and make immune cells especially capable of hunting out and destroying cancer, represents a promising new front in the fight against cancer. If it works — not just in Layla, but in future lab tests and clinical trials — doctors might one day be able to pluck a vial of genetically engineered immune cells from a shelf and then inject them into a patient to wage microscopic war against previously unresponsive cancers.

Other oncology research

A similar treatment, known as CAR T-cell therapy (T-cells are the immune cells that fight off cellular abnormalities and infections, CAR is the protein that gets added to T-cells that equips them to kill cancer) is currently in clinical trials at the University of Kansas. But as the New York Times pointed out, those treatments require that immune cells be extracted from a patient, shipped to a plant to be altered, sent back and reinjected into the patient — a process that can be lengthy, logistically challenging and expensive. In addition, patients who have already undergone several rounds of excruciating and exhausting chemotherapy may not have enough healthy T-cells left for this course of treatment.

Instead of relying on the patient’s weakened immune cells, the researchers developed a bank of pre-engineered, one-size-fits-all T-cells from healthy donors. Using a genome editing tool called TALEN, they cut the T-cells to render them impervious to leukemia drugs, which would ordinarily kill them, and paste in new programming that directs them to hunt down and fight against cancer. The editing also prevents the donor cells from attacking anything else it comes across (like the patient’s own cells) a concern whenever someone is injected with cells that aren’t theirs.

These genetically engineered “designer cells” can be bottled up in vials and shipped to doctors, who then inject them into patients suffering from certain severe cancers — no additional assembly required.

The first “off the shelf” banks of donor T-cells, called UCART19, were still being tested when Layla’s parents asked if there was anything else the doctors at GOSH could do for their daughter, according to a hospital press release. A consultant immunologist at GOSH who had been working on the therapy, Waseem Qasim of UCL, suggested UCART19. The idea was approved by an ethical panel, and a short while later Layla was injected with a 1 milliliter vial of the cancer-fighting cells.

Two weeks later, her cancer gone, Layla underwent an operation to replace the bone marrow that had deteriorated during treatment. She’s not “cured” yet — that won’t be certain for months or even years — but she’s well enough to go home, the hospital announced Thursday. “Her leukemia was so aggressive that such a response is almost a miracle,” Paul Veys, Layla’s lead doctor, told Reuters.

Qasim, the UCL researcher, cautioned that UCART19 therapy still has a long way to go before it becomes a treatment option: “We have only used this treatment on one very strong little girl,” he said in the hospital’s press release. “… But this is a landmark in the use of new gene engineering technology and the effects for this child have been staggering. If replicated, it could represent a huge step forward in treating leukemia and other cancers.”

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