Novel CAR-T Cell Therapy to Be Studied in Blood, Breast Cancers

The first patient was dosed in a clinical trial evaluating PRGN-3007 in patients with ROR1-positive cancers.

The first patient has been dosed in a phase 1/1b trial examining PRGN-3007 in patients with advanced ROR1-positive blood and solid cancers, according to a press release from Precigen, the pharmaceutical company manufacturing the novel drug.

The target population for the trial, which is set to determine the best dose of the drug, is patients with chronic lymphocytic leukemia, mantle cell lymphoma, diffuse large B-cell lymphoma and solid tumors, including breast adenocarcinomas and triple-negative breast cancer.

ROR1 is a protein that can sometimes be found on breast and blood cancer cells, and, according to the National Institutes of Health, high ROR1 expression has been associated with tumor metastasis and aggressive disease in patients with breast cancer.

"The PRGN-3007 study targets a broad range of hematological and solid tumor indications, and this milestone helps us move closer to our vision for UltraCAR-T, which aims to deliver a library of personalized autologous UltraCAR-T therapies using overnight manufacturing at the patient's medical center,” Helen Sabzevari, president and CEO of Precigen, said in the press release.

PRGN-3007 is a CAR-T cell therapy that utilizes Precigen’s UltraCAR-T platform, which is a gene delivery system that manufacturers CAR-T cell therapy overnight — an improvement over the average of 9 to 14 days for this type of therapy to be made, according to Penn Medicine.

CAR-T cell therapy works by taking a person’s immune (T) cells and re-engineering them to find and fight cancer cells. The T cells are then re-infused into the patient’s body. Additionally, PRGN-3007 also inhibits PD-1 gene expression, which helps cancer cells hide from the immune system.

READ MORE: What Is CAR-T Cell Therapy, and What Can Patients With Cancer Expect?

According to the press release, the design of PRGN-3007 will hopefully help patients avoid side effects as well as the high cost of checkpoint inhibitors (a type of immunotherapy drug) by eliminating the use of multiple drugs.

The study will be conducted in two parts; the first part will evaluate increasing doses of PRGN-3007, while the second part will be looking at the overall response rate (percentage of patients whose disease shrinks or disappears as a result of treatment) and disease control rate (percentage of patients whose cancer disappears, shrinks or does not grow) over a 12-month period.

Researchers are expecting to enroll approximately 88 patients. To be eligible, they must have little or no impediment to completing their daily tasks, have a life expectancy of 12 weeks or more at the start of enrollment, have adequate organ function, not have recently had steroids, not be pregnant and be able to understand and sign an informed consent.

"We are excited to work with Precigen and announce that the first patient, a (chronic lymphocytic leukemia) patient, has been dosed in the first-in-human study of PRGN-3007 UltraCAR-T," Dr. Javier Pinilla-Ibarz, senior member, lymphoma section head and director of immunotherapy, malignant hematology department at Moffitt, and principal Investigator for the PRGN-3007 clinical study, said in the press release. "ROR1 is a promising target for addressing a wide variety of tumors and we are hopeful that the PRGN-3007 study will further the development of this novel CAR-T treatment, which combines intrinsic PD-1 inhibition and ease of administration from the validated overnight manufacturing of UltraCAR-T performed at our medical center bringing therapy to patients within one day."

Researchers plan on competing the study by January 2026.

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