Clinical Trial Corner: New Treatments in Leukemia, Lymphoma and Brain Cancers

Trials enrolling patients with relapsed leukemia, lymphoma and brain cancers: © stock.adobe.com.

The landscape of cancer treatment continues to evolve rapidly, bringing forward new options for patients with difficult-to-treat diseases. In this edition of Clinical Trial Corner, we highlight notable advances from ongoing studies across a variety of cancers, including acute myeloid leukemia, lymphoma and high-grade gliomas.

Early data from cutting-edge therapies such as off-the-shelf CAR natural killer cell treatments, precision CRISPR-based approaches and innovative targeted radiotherapies show encouraging safety and effectiveness.

These trials are actively recruiting patients at leading cancer centers worldwide, expanding access to novel treatments for people facing cancer. As research progresses, these developments may translate into meaningful improvements in outcomes and quality of life. Stay informed with CURE as we track these important steps forward in clinical research.

FDA Grants Orphan Drug Designation to SENTI-202

Encouraging early data from the ongoing phase 1 trial of SENTI-202, an off-the-shelf CAR NK cell therapy for relapsed or refractory acute myeloid leukemia (AML), has helped support the FDA’s decision to grant the treatment orphan drug designation. This designation is expected to help accelerate development of potential new treatment options for people with AML, a disease that often has few options once it returns or resists treatment.

According to Senti Biosciences CEO Dr. Timothy Lu, SENTI-202 has shown promising early results, and the orphan drug designation supports continued progress. AML remains a difficult disease with limited options for those whose cancer has relapsed or not responded to treatment.

SENTI-202 is designed to target cancer cells while sparing healthy bone marrow and is actively recruiting patients at sites including UCLA Medical Center in Los Angeles, MD Anderson Cancer Center in Houston, and Peter MacCallum Cancer Center in Melbourne, Australia.

LyL314 Shows High Rates of Durable Complete Responses

Lyell Immunopharma announced positive results from a phase 1/2 trial of LYL314, a CAR T-cell therapy for relapsed or refractory large B-cell lymphoma. Among patients treated in the third- or later-line setting, the overall response rate was 88%, with 72% achieving a complete response. Of those, 71% remained in complete response at 6 months or longer.

The treatment showed a manageable safety profile, with no cases of severe cytokine release syndrome and low rates of severe neurotoxicity, which resolved quickly with standard care.

These data support the ongoing pivotal PiNACLE trial of LYL314 in third- or later-line patients with large B-cell lymphoma. The company also plans to start a pivotal trial in the second-line setting by early 2026. Investigators highlighted that LYL314 could offer meaningful benefits for this hard-to-treat cancer, especially in patients with high-risk features like primary refractory disease or stage 4 cancer. Full results will be presented at the International Conference on Malignant Lymphoma in Lugano, Switzerland.

Trial sites in the U.S. include UCLA, UC Irvine, University of Iowa, University of New Mexico, Montefiore Medical Center, Huntsman Cancer Institute at University of Utah and VCU Massey Cancer Center.

GLPG5101 New Trial Findings in ATLANTA-1 for R/R Non-Hodgkin Lymphoma

New findings from the ongoing ATALANTA-1 study of CAR-T therapy, GLPG5101 will be presented at the 2025 European Hematology Association Congress. In 61 patients with relapsed or refractory non-Hodgkin lymphoma, results showed low rates of severe side effects. Nearly all patients received fresh, early-memory-enriched CAR-T cells, with 89% infused within 7 days, avoiding cryopreservation and bridging therapy. The attrition rate was just 5%, much lower than typical CAR-T trials.

Most side effects were blood-related, with few severe cases of cytokine release syndrome or neurological symptoms. This decentralized approach and rapid delivery of stem-like cells may expand access to CAR-T therapy for more patients with relapsed or refractory lymphoma, as per the release.

The study is recruiting patients at multiple locations across the United States and Europe, including Tufts Medical Center and Beth Israel Deaconess Medical Center in Boston; Antwerp University Hospital and UZ Leuven in Belgium; and Academisch Medisch Centrum, Leiden University Medical Center, and Erasmus MC in the Netherlands.

First Patient Dosed in Phase 1b Trial Evaluating SNIPR001

On June 12, 2025, SNIPR Biome announced dosing of the first patient in its phase 1b trial of SNIPR001, a CRISPR-based oral therapy aimed at preventing bloodstream infections from drug-resistant E. coli in patients with hematological cancer undergoing stem-cell transplantation. The randomized, double-blind, placebo-controlled study is underway at eight U.S. centers enrolling 24 patients colonized with fluoroquinolone-resistant E. coli.

Infectious complications remain a major threat in hematologic cancer care, with no approved preventive treatments for bloodstream infections. SNIPR Biome’s precision CRISPR technology offers a novel approach to this urgent need, supported by CARB-X funding. Early Phase 1a data showed promising safety and target engagement.

Participating recruiting sites include City of Hope in Duarte, University of Minnesota in Minneapolis, Weill Cornell Medicine in New York, and MD Anderson Cancer Center in Houston. Additional sites not yet recruiting are University of California San Francisco, Johns Hopkins University in Baltimore, UPMC in Pittsburgh, and Fred Hutchinson Cancer Center in Seattle.

Iopofosine-101 Shows Improved Progression-Free Survival

On June 11, 2025, Cellectar Biosciences announced initial results from the CLOVER-2 phase 1 trial testing iopofosine-131 in pediatric patients with relapsed or refractory high-grade gliomas. In seven patients who received at least 55 millicuries of the drug, the average progression-free survival was 5.4 months — more than double the typical 2.25-month median seen in this population. Overall survival averaged 8.6 months and remains ongoing. Three patients who received multiple dosing cycles showed an average progression-free survival of 8.1 months and overall survival of 11.5 months, with two achieving an objective tumor response.

The treatment was well tolerated with manageable blood-related side effects and no serious damage to the heart, kidneys, or liver. These early findings suggest that iopofosine-131 may offer clinical benefit for children and young adults facing aggressive brain tumors with limited options.

The trial is ongoing at multiple sites across the United States, Canada, and Australia, including Lucile Packard Children's Hospital in Palo Alto, Memorial Sloan Kettering Cancer Center in New York, Duke University in Chapel Hill, Cincinnati Children's Hospital Medical Center, Texas Children's Hospital in Houston, University of Wisconsin Hospital and Clinics in Madison, Children’s Hospital at Westmead in New South Wales, and the Hospital for Sick Children in Toronto.

References

1. “Senti Bio Granted U.S. FDA Orphan Drug Designation for Use of First-in-Class Off-the-Shelf Logic-Gated Selective CD33 OR FLT3 NOT EMCN CAR-NK Cell Therapy SENTI-202 to Treat Acute Myeloid Leukemia,” Senti Bio, June 18, 2025.
2. “Lyell Immunopharma Announces Positive New Clinical Data Demonstrating High Rates of Durable Complete Responses from the Phase 1-2 Trial of LYL314 for the Treatment of Aggressive Lymphoma,” Lyell Immunopharma, June 17, 2025.
3. “Galapagos to Present New ATALANTA-1 CAR-T Data at EHA 2025 Highlighting Low Toxicity and Rapid Decentralized Delivery of Fresh Early Memory-Enriched GLPG5101 in Relapsed/Refractory NHL,” Galapagos, June 12, 2025.
4. “SNIPR-BIOME Announces First Patient Dosed in Its Phase 1b Clinical Trial of SNIPR001 in Patients with Hematological Cancer,” SNIPR BIOME, June 12, 2025.
5. “Cellectar Biosciences Provides Update on CLOVER-2 Phase 1 Clinical Trial of Iopofosine I-131 in Pediatric Patients with Relapsed Refractory High-Grade Glioma,” Cellectar Biosciences, June 11, 2025.

For more news on cancer updates, research and education, don’t forget to subscribe to CURE®’s newsletters here.