Stories about: acute lymphoblastic leukemia

Landmark moment for science as the FDA approves a gene therapy for the first time

Leukemia blast cells, which could now be destroyed using a first-of-its-kind, FDA-approved gene therapy called CAR-T cell therapy
Leukemia blast cells.

Today, the Food and Drug Administration approved a gene therapy known as CAR T-cell therapy that genetically modifies a patient’s own cells to help them combat pediatric acute lymphoblastic leukemia (ALL), the most common childhood cancer. It is the first gene therapy to be approved by the FDA.

“This represents the progression of the field of gene therapy, which has been developing over the last 30 years,” says gene therapy pioneer David A. Williams, MD, who is chief scientific officer of Boston Children’s Hospital and president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “It’s a realization of what we envisioned to be molecular medicine when this research started. The vision — that we could alter cells in a way to cure disease — is now coming true.”

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Do children with Down syndrome need modified chemo for leukemia? No, says study

leukemia down syndrome acute lymphoblastic leukemia

Clinicians have long known that children with Down syndrome carry an elevated risk of developing acute lymphoblastic leukemia (ALL), the most common pediatric cancer. Research consistently shows that children with Down syndrome are more likely to suffer complications from chemotherapy. At the same time, some studies have suggested that children with Down syndrome and ALL may have a higher chance of relapsing.

What to do with this knowledge has been a source of controversy. Should patients with ALL and Down syndrome receive treatment modified to minimize toxicity from chemotherapy? Or should they be given the same treatment as other children with ALL to minimize the chance for relapse? Recent study results from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center suggest that full-dose chemo is preferable.

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Progress in the treatment of childhood leukemia

Although treatments for childhood cancer patients are improving, cancer remains the leading cause of death by disease in children. Doctors and researchers are also focused on decreasing the toxicity of these treatments, which can have side effects years after a child finishes treatment.

“The war against childhood cancer is hardly over,” says Kimberly Stegmaier, MD, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “We need to do better.”

Stegmaier, who focuses her research on identifying new drug targets and new drugs for childhood leukemiaEwing sarcoma, and neuroblastoma, recently discussed advances in childhood cancer treatment in a Science, Innovation, and Discovery Talk (SID Talk) at Dana-Farber. During the TED Talk-style presentation, Stegmaier explained some of her research in the treatment of sub-microscopic acute lymphoblastic leukemia (ALL) as well as genetic targets in childhood cancers.

“What you can do in an environment where you have chemists, biologists, and clinicians adjacent and working collaboratively is very powerful,” says Stegmaier. “That’s why I’m here today—we need to cure 100 percent of kids, and we can’t do this alone.”

This story originally ran on Dana-Farber Cancer Institute’s Insight blog.

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