Improving care for patients with acute lymphocytic leukemia

It took three months and multiple bone marrow biopsies to determine Becky Yu's exact diagnosis. Her treatment included joining a clinical trial that has now defined the new standard of care for certain adults with newly diagnosed acute lymphocytic leukemia (ALL).

The therapy she received was approved by the FDA in June 2024, shortly before the results of the clinical trial Yu participated in were published in the New England Journal of Medicine. Penn Medicine patients, including Yu, and clinicians, including leukemia specialist Selina Luger, who was a co-senior author on the study, were major contributors to making this cancer treatment advance possible. It will be the newest FDA-approved therapy on a growing list that can be substantially credited to research contributions at Penn Medicine.

By the time Yu began treatment, she was ready. The first phase of treatment for ALL is called induction therapy and involves intense chemotherapy to induce cancer remission. It's followed by four to six months of consolidation therapy, then at least two years of maintenance therapy.

Luger, a professor of hematology-oncology at the Perelman School of Medicine, explains the lengthy treatment regimen is designed to protect against the cancer returning, which still happens too often: "Despite the fact that we get most of these patients into remission, there's a high chance of relapse even in those with the best results after induction therapy," Luger explains. "If patients relapse, there's a high chance they won't survive, so we want to be able to improve their outcomes."

To combat this challenge, Luger and colleagues on the ECOG-ARIN Cancer Research Group, a national clinical trial collaborative funded by the National Cancer Institute, designed a clinical trial that adds an immunotherapy drug, called blinatumomab, to consolidation chemotherapy for a particular group of patients with ALL.

"Cooperative group trials allow us to enroll patients through many different types of hospitals, including large academic centers, like Penn Medicine, and smaller community programs that may only see one or two patients each year with a given diagnosis," says Luger. "It's much more relevant to real-world care than a single-institution study."

After receiving her diagnosis at Penn, Yu learned she was a candidate for the clinical trial, and she chose to enroll. In order to participate, Yu had to receive a continuous IV infusion (24 hours a day) for four 28-day cycles-four months in total in addition to the standard chemotherapy. Carrying the pump bag around was just like carrying an extra purse, she said. The tradeoff for such a large commitment is that the immunotherapy doesn't have the same side effects as chemotherapy, such as hair loss or nausea.

Yu completed the clinical trial in spring 2021 and continues to enjoy life cancer-free. In December 2022, Luger's colleagues presented the clinical trial results at a national oncology meeting, showing that adding blinatumomab to consolidation chemotherapy improved overall survival. As the published study showed, after three years, 85% of patients who received the immunotherapy drug were still alive, compared to 68% who received chemotherapy alone. These study results supported the FDA's decision to approve blinatumomab for patients with CD19-positive, Philadelphia chromosome-negative, B-cell precursor ALL in the consolidation phase.

"Based on what we learned from this study, we've improved the outcomes for patients with ALL and more ALL patients will survive while avoiding additional treatment toxicities," Luger says. "This progress is only possible through clinical trials."

Read more at Penn Medicine News.