Two infant girls with leukemia cured using immunotherapy treatment.

Two Babies With Leukemia Cured Using Breakthrough Treatment Involving Genetically Modified Cells

Two baby girls with leukemia have been cured using a breakthrough treatment involving a special form of gene-editing therapy, which uses modified white blood cells to target cancerous cells, Medical XPress reported.

A European team of researchers wrote on a scientific journal for Science Translational Medicine the details on how they were able to cure two baby girls, both of whom have been cancer-free for 18 and 12 months respectively since undergoing the breakthrough treatment. The research team is affiliated with various institutions in the United Kingdom.

The scientific journal read that “molecular remissions were achieved within 28 days in both infants,” a relatively short space of time compared to previous cases where gene-editing therapy was used.

“Two infants with relapsed refractory CD19+ B cell acute lymphoblastic leukemia received lymphodepleting chemotherapy and anti-CD52 serotherapy, followed by a single-dose infusion of UCART19 cells. Molecular remissions were achieved within 28 days in both infants, and UCART19 cells persisted until conditioning ahead of successful allogeneic stem cell transplantation. This bridge-to-transplantation strategy demonstrates the therapeutic potential of gene-editing technology.”

So far, the two baby girls show no signs of acute lymphocytic leukemia. As such, both infants wouldn’t be alive today if it weren’t for the hard work of medical experts from London’s Great Ormond Street Hospital and Cellectic.

Many other drug companies used the same treatment numerous times in the past but the white blood cells they used were extracted from patients also undergoing treatment.

The two babies cured of leukemia, however, were injected with white blood cells taken from healthy donors.

The therapy involved two changes, the first one being the disabling of the gene that prompts the immune system to attack the invading cell after recognizing it as a foreign body. The second change causes the edited cells to detect and attack cancer cells. The last change involves the turning off of the body’s mechanism to develop grant-versus-host disease.

The treatment used to cure the infants of leukemia wasn’t without its fair share of risks and negative effects. For one, their immune systems could have eliminated or responded negatively to the white blood cells injected into their bodies. That reportedly was not the case — at least initially.

The first infant did have one bad immune reaction to the white blood cells after two months, but it was immediately alleviated by injecting steroids and performing bone marrow transplantation. Bone marrow transplantation was administered to flush out edited cells from the infants’ bodies.

Fox 13 reported that a bone marrow registration drive had been staged in Lehi to help the two girls afflicted with leukemia. The girls’ families launched the registration drive to sign up donors and raise awareness of the need for bone marrow donors.

According to DKMS,70 percent of patients with a blood-related illness need donors from outside their family to acquire bone marrows that can save their lives.

Since undergoing treatment, the two girls remain perfectly healthy, and are happy living with their respective families.

As it stands, the treatment used to cure the two young girls of leukemia was experimental and is not approved for general use yet. The treatment was only allowed because no other effective methods were available. It is, however, a big step forward in terms of cancer treatment. In fact, various forms of CAR-T cell therapy are already being administered and tested, and experts say that results have been “promising.” According to Medical XPress, some of them may be approved soon.

While the British researchers haven’t discovered what causes leukemia in children, what they accomplished in treating the two young girls paves the way for better treatments in the future.

Leukemia is one of the most difficult cancers to diagnose and treat, but thanks to this latest breakthrough, people can breathe a little easier knowing there’s always hope that it can be cured.

[Featured Image by janzwolinski/Thinkstock]

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