Because of a “high-impact initiative” funded completely by St. Baldrick’s donations, researchers are now able to accelerate discoveries in childhood cancers and open more clinical trials for children with cancer.
To learn more about how this new initiative is helping save children’s lives, we checked in with Dr. Michael Kelly, pediatric oncologist and hematologist at Children’s Hospital of Wisconsin, one of the 130 research institutions that opened four of the high-impact studies.
Q: What are high-impact studies?
A: High-impact studies involve patient populations with select diseases that need improvement in patient care or therapy outcomes. In these studies, researchers evaluate different drugs and unique drug combinations in patients with rare and difficult childhood cancers.
Q: What’s an example of a high-impact study?
A: Outcomes for patients with relapsed leukemia are relatively poor. A drug called bortezomib has been shown to be effective in adult patients diagnosed with relapsed leukemia. One of our high-impact studies evaluates the safety and effectiveness of bortezomib in combination with re-induction chemotherapy in pediatric patients with relapsed acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma.
Q: What phase is the bortezomib clinical trial in now?
A: Bortezomib is being tested in two disease groups. The drug is still in the phase II trial for patients with relapsed ALL. However, Bortezomib is being used to treat pediatric and young adult patients newly diagnosed with acute myeloid leukemia (AML) as part of the most recent phase III COG therapy protocol for that disease.
Read about how a high-impact study helped cure a childhood cancer patient with AML.
Q: How essential was St. Baldrick’s funding for the high-impact initiative?
A: St. Baldrick’s funding has helped close the gap of real cost associated with clinical trial participation and enrollment. Until this particular initiative, a majority of the costs associated with running clinical trials at institutions were not covered. Honestly, that affects the number of institutions that can open a particular clinical trial.
The reimbursement that institutions receive from St. Baldrick’s funding for opening high-impact clinical trials have increased the number of institutions that can open and enroll patients on these trials. Increased institutional participation leads to more patients enrolled in a shorter time frame and allows us to evaluate the safety and effectiveness of new agents more quickly, leading to better therapies for children with cancer.
The high-impact studies are just one of St. Baldrick’s Childhood Cancer Research Outcomes. Learn more.