The treatment landscape for Acute Lymphocytic Leukemia (ALL) is evolving rapidly, with recent approvals and clinical trial results poised to improve patient outcomes. Several companies are diligently working toward developing novel ALL treatment therapies.
FDA Approves Obecabtagene Autoleucel for R/R B-cell ALL
In November 2024, the U.S. Food and Drug Administration (FDA) approved obecabtagene autoleucel (Aucatzyl) for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). This approval marks the first indication for this chimeric antigen receptor (CAR) T-cell therapy, also known as obe-cel. The FDA's decision was based on the results of the Phase 1/2 FELIX clinical trial, which included patients with ALL who experienced relapse after a remission lasting no longer than 12 months, relapsed or were refractory within 12 months after at least two prior systemic therapies, or had recurrent or refractory disease at least three months following allogeneic stem cell transplantation.
Groundbreaking Results from COG Clinical Trial
In December 2024, the Children's Oncology Group (COG) presented transformative findings from a major clinical trial at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego, Calif. These results are expected to change the standard of care for children with acute lymphoblastic leukemia (ALL). This breakthrough is regarded as one of the most significant advancements in pediatric oncology in decades, made possible by 25 years of contributions to St. Baldrick's Foundation.
BESPONSA Approved for Pediatric Patients
In March 2024, the FDA approved Pfizer's BESPONSA for pediatric patients aged 1 year and older with relapsed or refractory CD22-positive B-cell ALL. BESPONSA had previously received priority review and orphan drug designation. It was initially approved in 2017 for treating relapsed or refractory B-cell precursor ALL in adult patients.
Ongoing Clinical Trials and Emerging Therapies
Numerous companies are currently involved in developing therapies for ALL. Emerging therapies in various phases of clinical trials include:
- CTA30X (Nanjing Bioheng Biotech)
- 131-I-Apamistamab (Actinium Pharmaceuticals)
- UCART22 (Cellectis S.A)
- AUTO1 (Autolus Limited)
- TBI-1501 (Takara Bio Inc.)
- Navitoclax (AbbVie)
- Daratumumab (Janssen Research & Development, LLC)
- CPX-351 (Jazz Pharmaceuticals)
- Orca-T (Orca Biosystems, Inc.)
These therapies utilize various routes of administration, including infusion, intravenous, oral, and subcutaneous, and encompass molecule types such as gene therapies, small molecules, and monoclonal antibodies. These novel approaches aim to improve outcomes for patients with ALL who have limited treatment options.
