Grant Awarded to Study New Drug for Lymphoblastic Leukemia Treatment

As a pediatric oncologist and clinical researcher, Maureen O’Brien, MD, has dedicated her career to developing more effective treatments for children with high-risk and relapsed leukemias. With a new proposal specifically targeting acute lymphoblastic leukemia (ALL), Dr. O’Brien has been awarded the R50 Clinical Scientist Award by the National Cancer Institute (NCI) to lead multiple clinical trials.

Funding for her proposal, titled “Pediatric acute lymphoblastic leukemia: Improving outcomes through incorporation of novel targeted therapies for relapsed and newly diagnosed disease,” will support Dr. O’Brien’s leadership of multiple ALL clinical trials for the Children’s Oncology Group (COG) — the world’s largest organization exclusively researching childhood cancer. As the Vice-Chair for Precision Medicine and Novel Agents for the COG ALL Committee, Dr. O’Brien is leading the frontline trial for children and adolescents with newly diagnosed high risk B-cell ALL (B-ALL), guiding the development of new trials for children with Down syndrome and B-ALL, and developing new trials for children experiencing a first relapse of B-ALL.

The overall goal of the new generation of clinical trials for B-ALL is incorporation of immunotherapies and targeted therapies to both improve outcomes and decrease toxicities.

In the frontline trial for high-risk B-ALL, the highly active, FDA-approved antibody-drug inotuzumab ozogamicin will replace two of the most toxic chemotherapy blocks. In the trial for children with Down syndrome, who suffer more toxicity from treatment than children without Down syndrome, inotuzumab ozogamicin will replace the traditional first month of chemotherapy followed by the immunotherapy drug blinatumomab. This strategy will also be studied in children with a first relapse of B-ALL. Through these clinical trials, Dr. O’Brien hopes to determine how  inotuzumab ozogamicin can best be incorporated into existing treatment regimens.

“The optimal way to incorporate this drug into complicated chemotherapy regimens for newly diagnosed and relapsed patients is not known and requires carefully designed clinical trials,” says Dr. O’Brien. “We hope to determine whether this drug can replace some of the most toxic chemotherapy agents used for ALL treatment to improve outcomes and decrease side effects.”