An immunotherapy with game-changing – life-saving – potential for patients with relapsed B-cell ALL
CREDIT: ADOBE STOCK
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A new immunotherapy is providing another path to remission for relapsed B-cell ALL. Dr. Laura Hogan shares news of a long-awaited breakthrough for patients with the same pediatric cancer she had.
Saving lives is the ultimate goal for all pediatric oncologists. But Laura Hogan, M.D., a pediatric oncologist at Stony Brook University has a unique perspective on the B-cell acute lymphoblastic leukemia (ALL) she studies – because she’s a survivor.
Dr. Hogan was only 4 when she was diagnosed with B-cell ALL. |
But she still remembers the realities of treatment – three years of toxic chemotherapy, months in the hospital and the fear that that cancer might one day come back. Many of the patients in the rooms around her died.
Now, decades later, she’s a pediatric oncologist at Stony Brook Children’s and part of the Children’s Oncology Group (COG) team working to find more effective and less toxic treatments for the disease. One of the biggest challenges is that about 15% of patients with B-cell ALL relapse.
Now, decades later, she’s a pediatric oncologist at Stony Brook Children’s and part of the Children’s Oncology Group (COG) team working to find more effective and less toxic treatments for the disease. One of the biggest challenges is that about 15% of patients with B-cell ALL relapse.
“It’s devastating for families to face the reality that their child’s cancer has returned, and to realize that the chances of survival are much lower than at initial diagnosis,” Dr. Hogan said.
Patients with relapsed B-cell ALL begin treatment with a series of intense chemotherapy which often requires weeks or months in the hospital, followed either by a lower-dose maintenance chemotherapy -or sometimes a bone marrow transplant. During this time patients are at high risk for infection and other toxicities. That's why scientists began looking to immunotherapy, which uses a patient's own immune system or external immune cells and agents to target and destroy cancer cells. This treatment holds the promise of precisely targeting cancer cells with fewer side effects than chemotherapy. |
Fulfilling COG’s promise to pursue the most difficult cancers to treat
When Blinatumomab, a new immunotherapy specifically designed to treat B-cell ALL, was initially being investigated, Dr. Hogan saw an opportunity. She joined the COG team led by Patrick Brown, M.D., Lia Gore, M.D. and Mignon Loh, M.D. studying Blinatumomab’s effectiveness for B-cell ALL patients whose cancer had relapsed.
Blintumomab works by attaching itself to a specific protein, which lives on the surface of cancerous B cells and to T cells, which are part of the patient's normal immune system. When the two get close enough, the T cells attack the cancer.
The study was divided into two groups. One for the high-risk and intermediate-risk patients and another for the low-risk patients. But the study closed early for the high-risk and intermediate-risk patients when the results were clear, Blinatumomab was far superior to the standard chemotherapy and and less toxic.
“There was a double-digit increase in remission. The same was true for overall survival – we saw double-digit increases,” Dr. Hogan said. “This is exactly why I pursued pediatric cancer research – because I knew in my career we were going to save lives.”
In June 2017, the Food and Drug Administration used the data from this study to provide full approval to blinatumomab for relapsed B-cell ALL treatment in children and adults.
Even more exciting was, after the initial first treatment, a large majority of kids were sent home after a couple of days in the hospital where they were monitored to make sure there were no complications.
“These patients didn’t have the nausea, pain, or discomfort that comes with chemotherapy,” she said. “That’s worth a lot to us as physicians and to our patients.”
Even better results than expected
Drs. Hogan and Brown we're also excited to see some unexpected but positive results of their study, including:
- Patients who had immunotherapy were less likely to have minimal residual disease – small numbers of cancer cells left in the bone marrow after treatment.
- The immunotherapy group experienced far fewer complications like serious infections compared to the chemotherapy group.
- Patients who received immunotherapy were in better health going into the final step of treatment, the bone marrow transplant.
“Being able to proceed to a bone marrow transplant greatly increases the chance for these patients to achieve remission. Immunotherapy gives us an avenue for more kids to have that chance,” she said. “I love that insights made in the lab about specific molecules related to cancer lead to an incredible outcome: we have more kids surviving cancer.”
More research on the horizon for B-cell ALL
The Children’s Oncology Group is already identifying ways to integrate Blinatumomab into B-cell ALL treatment plans earlier. They hope to further reduce the toxicity of treatments for patients whose cancer relapses, increase the rate of disease-free survival, and learn more about how to use the immune system to fight cancer.
“What's fascinating and unique about COG is that we are able to create new knowledge and make progress together," Dr. Hogan said. "Like all COG studies, this was a collective effort of researchers across the world that participate in COG. Doctors at participating institutions and patients are also an important part of the mission and collaboration.”
Dr. Hogan is also intentional in her declaration that donors play a significant role in all of COG’s research, enabling faster progress and better care for patients.
“The pediatric cancer community has been the driving force of many treatment successes. Without donors, we would not have the funding we need to gain knowledge and make progress. This is just the beginning for Blinatumomab. We’re essentially at the starting point of the potential for us to learn about how immunotherapy can help B-cell ALL patients,” Dr. Hogan said.
Reflecting on the study, her own experience as a patient, and the science that captivates her, Dr. Hogan is confident that pediatric cancer research is the right place for her.
“In medical school, I kept an open mind to make sure I didn’t choose pediatric cancer for personal reasons. But pediatric cancer was where I saw the greatest opportunity to be part of major advances over the lifetime of my career,” she said. “Because of donors and patient families and the Children’s Oncology Group, I look forward to many more years of tangible progress ahead and more kids surviving.”