News and Expert Insights in Rare Cancer Cell Therapy for Rare Cancer Day


In observance of Rare Cancer Day, held annually on September 30, catch up on the past few months’ news and expert insights related to cell therapies in development for these indications.

Over the past year, CGTLive™ has covered news related to the cell therapies in development for a variety of rare cancer types and conducted interviews with experts to learn about the clinical development process for these novel treatments. According to the National Organization for Rare Disorders, more than 1 out of every 4 cancer diagnoses are for rare cancers and 25% of all deaths from cancer are attributed to rare cancers.1 Despite these numbers, funding for research for individual rare cancer types tends to be low and great unmet need remains for many patients with these diseases.

For Rare Cancer Day, observed annually on September 30 by the patient and clinician communities, the CGTLive™ team has selected some of our news coverage and interviews from the past few months on cell therapies in rare cancer, in order to offer a snapshot of the therapeutic progress being made in this field.

Liso-cel Achieves Durable Responses in Patients With R/R Follicular Lymphoma and R/R Mantle Cell Lymphoma

In June of this year, Bristol Myers Squibb reported that lisocabtagene maraleucel (liso-cel; Breyanzi), a CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy currently marketed in the United States and European Union for large B-cell lymphoma indications, produced durable responses among patients with relapses/refractory (r/r) follicular lymphoma (FL) treated in the phase 2 TRANSCEND FL clinical trial (NCT04245839) and patients with r/r mantle cell lymphoma (MCL) treated in the phase 1 TRANSCEND NHL 001 clinical trial (NCT02631044).2

In TRANSCEND FL, 101 patients with FL that was r/r after 2 prior lines of therapy were evaluated for efficacy. The overall response rate (ORR) in this group was 97% (95% CI, 91.6-99.4; one-sided P < .0001) and the complete response (CR) rate was 94% (95% CI, 87.5-97.8; one-sided P < .0001). At a median follow-up of 16.6 months, the median duration of response was not reached. Furthermore, at 12 months of follow-up, 81.9% of patients whose disease responded to the therapy had sustained responses. At a median follow-up of 17.5 months, median progression-free survival (PFS) was not reached; 80.7% of the patients achieved 12-month PFS.

In TRANSCEND NHL 001, 74 patients with MCL that was r/r after 2 or more prior lines of therapy were included in the efficacy analysis for the trial’s MCL cohort. At a median follow-up of 16.1 months, the ORR for these patients was 86.5% (95% CI, 76.5-93.3; one-sided P < .0001) and the CR rate was 74.3% (95% CI, 62.8-83.8; one-sided P < .0001). All of the patients in this group had previously been treated with a BTK inhibitor. The patients received liso-cel at 2 dose-levels: 50x106 CAR-positive viable T-cells and 100x106 CAR-positive viable T-cells.

Multi-Base–Edited CAR T-Cell Therapy Trial Doses First Patient With R/R T-ALL/T-LL

Just earlier this month, Beam Therapeutics announced that it had dosed the first patient with r/r T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL) in its phase 1/2 trial (NCT05885464) of BEAM-201.3

BEAM-201 is an anti-CD7 allogeneic chimeric antigen receptor CAR-T therapy and is the first quadruplex-edited, allogeneic CAR T-cell therapy candidate to enter clinical-stage development. The news also marked the first treatment with a base editing CAR T-cell candidate in the United States.

“As the first patient dosed with a Beam therapeutic candidate and the first patient in the US to receive a base editing therapeutic, this represents a major milestone for the company, the scientists that made this possible, and the patients we hope to serve,” John Evans, chief executive officer, Beam, said in a statement.3

Invectys Initiates Clinical Trial for Solid Tumor CAR-T IVS-3001, Which Includes Cohorts for Renal Cell Carcinoma and Ovarian Carcinoma

On June 21, 2023, Invectys initiated a phase 1/2a clinical trial (NCT05672459) for IVS-3001, a CAR-T therapy intended to treat solid tumors, according to a July 13 announcement from the company.4

IVS-3001 targets HLA-G, a checkpoint molecule that under normal conditions is only expressed during pregnancy to protect the fetus from an immune response but is also expressed by some cancers. The clinical trial, which has begun recruiting patients, includes indication-specific cohorts for patients with HLA-G-positive clear cell renal cell carcinoma and HLA-G-positive epithelial ovarian carcinoma, but is also open to patients with other types of HLA-G-positive solid tumors.

“CAR-T cells have delivered stunning results in blood cancers, but their promise in solid tumors has yet to be fulfilled,” Jake Kushner, MD, an endocrinologist and medical director of McNair Interests whose appointment as Invectys’ new CEO was announced simultaneously with the trial’s initiation, said in a statement.4 “Invectys has the potential to turn this promise into a reality and breathe new hope into the cancer care field.”

Mary “Nora” Disis, MD, on Evaluating CAR-T PRGN-3005 in Ovarian Cancer

This year, at the American Society of Clinical Oncology (ASCO) 2023 Annual Meeting, held June 2-6, in Chicago, Illinois, results were reported from a phase 1/1b clinical trial (NCT03907527) evaluating investigational CAR-T PRGN-3005 for the treatment of platinum-resistant ovarian cancer, fallopian tube carcinoma, and primary peritoneal carcinoma.5

In an interview with CGTLive’s sister publication OncLive™, Mary “Nora” Disis, MD, director of University of Washington Medicine’s Cancer Vaccine institute, who coauthored the research presented, discussed the key findings and their implications for applying CAR-T for the treatment of patients with solid tumors. CGTLive also recently covered the details of the trial itself.

J. Andrew Livingston, MD, on Potential Advantages of TCR NK Therapy for Synovial Sarcoma and Myxoid/Round Cell Liposarcoma

In an interview with CGTLive, J. Andrew Livingston, associate professor in the department of sarcoma medical oncology and the department of pediatrics patient care at the University of Texas MD Anderson Cancer Center, discussed challenges with developing therapies, and specifically cell therapies, in the field of sarcoma. He also spoke on the potential advantages of T-cell receptor natural killer (TCR NK) therapies compared with CAR-T in this field.

1. Celebrate Rare Cancer Day with NORD. National Organization for Rare Disorders (NORD) website. Accessed September 29, 2023.
2. Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) delivers deep and durable responses in relapsed or refractory follicular lymphoma and mantle cell lymphoma in TRANSCEND clinical trials presented at ICML 2023. News release. Bristol Myers Squibb. June 17, 2023. Accessed June 23, 2023.
3. Beam Therapeutics announces first patient dosed in Phase 1/2 trial of BEAM-201 in relapsed, refractory T-ALL/T-LL. News release. Beam Therapeutics. September 5, 2023.
4. Invectys announces initiation of phase 1/2a clinical trial in solid tumors and change in leadership. News release. Invectys, Inc. July 13, 2023. Accessed July 18, 2023.
5. Liao JB, Stanton SE, Chakiath M, et al. Phase 1/1b study of PRGN-3005 autologous UltraCAR-T cells manufactured overnight for infusion next day to advanced stage platinum resistant ovarian cancer patients. Presented at: the American Society of Clinical Oncology (ASCO) 2023 Annual Meeting, held June 2-6, in Chicago, Illinois. Abstract #5590
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