In this article
FDA Grants Orphan Drug Designation to A2B530 for CEA-Positive Colorectal Cancer
In March 2024, a news release said that A2B530 (A2 Biotherapeutics), a CAR T-cell therapy, had been given Orphan Drug Designation to treat colorectal cancer that expresses carcinoembryonic antigen (CEA) and has lost HLA-A*02 expression in people who have germline heterozygous HLA-A*02(+) disease.
Researchers think that autologous logic-gated cell therapy can target tumor cells while protecting healthy tissue. This is because it has a built-in safety switch that can keep healthy tissue from getting hurt. In the phase 1/2 EVEREST-1 (NCT05736731) study, this idea will be tested.
You may like to read: CAR T-Cell therapy in China
The FDA’s decision to give Orphan Drug Designation confirms the huge unmet need for better treatments for people with colorectal cancer,” said William Go, MD, PhD, chief medical officer of A2 Bio, in a news release. This designation backs up our promise to use our cutting-edge technology platform to create new cancer treatments for people whose cancers are hard to treat.
The open-label, phase 1/2 EVEREST-1 study is looking at A2B530 as a possible treatment for solid tumors like colorectal cancer, pancreatic cancer, and non-small cell lung cancer. It also looks at other types of solid tumors that produce CEA but not HLA-A*02. The people who are now in the EVEREST-1 study were first in the BASECAMP-1 (NCT04981119) study, where their T cells were gathered, processed, and kept for later use.
You may like to read: CAR T-Cell therapy for multiple myeloma in China
The main goal of the phase 1 study is to find the safest and most effective dose. Safety and effectiveness must be maintained as the main goals of the phase 2 study. The amount of solid tumor cells that the recommended dose can kill while still saving healthy cells.
Colorectal cancer, which is a word for both colon and rectal cancer, happens when polyps (groups of cells growing together) form in the colon or rectum and turn into cancer. Older age, being black, having a history of polyps or cancer in your body or in your family, inflammatory bowel disease, genetics, diabetes, obesity, a normal Western diet, and smoking and drinking are all things that put people at risk.
Surgery, radiation, targeted therapy, and immunotherapy are the most common ways to treat colorectal cancer. However, many of the current methods for this cancer and others with solid tumors can kill patients.
You may like to read: CAR T Cell therapy cost in China
Researchers who worked on the study think that this CAR T-cell therapy is safer than other targeted treatments. This is because the CAR T-cells kill tumor cells without hurting healthy cells because they have a built-in safety switch that keeps healthy tissue from getting hurt.
How CancerFax Helps
CancerFax is a specialist cancer access and patient-navigation platform. We help patients and families understand their options, organise medical records, coordinate hospital communication, and support cross-border treatment planning where appropriate.
We help collect and organise reports, scans, pathology, biomarker results, and treatment history for structured case review.
We communicate with hospitals or trial teams to assess whether a case may be suitable for further screening.
We support appointment coordination, document submission, translation, and direct communication with international departments.
For international patients, we help with practical coordination — travel planning, hospital admission guidance, and local support.
If this option is not suitable, we help explore other relevant treatments, clinical trials, or advanced care pathways.
From inquiry through to follow-up, our coordinators provide a single point of contact for the family.
CancerFax does not guarantee treatment access, eligibility, or clinical outcome. Our role is to help patients access accurate information, structured review, and appropriate specialist pathways.
About Susan Hau
Susan Hau is a distinguished researcher in the field of cancer cell therapy, with a particular focus on T cell-based approaches and cancer vaccines. Her work spans several innovative treatment modalities, including CAR T-cell therapy, TIL (Tumor-Infiltrating Lymphocyte) therapy, and NK (Natural Killer) cell therapy. Hau's expertise lies in cancer cell biolo…
✓ Reviewed for medical accuracy by the CancerFax review panel.
Medical Disclaimer
This article is for educational purposes only and should not replace medical advice, diagnosis, or treatment from a qualified oncology specialist. Every patient's case is different. Treatment decisions should always be made after a review of complete medical records by the treating medical team.
Treatment availability, eligibility, timelines, and access can change. Any clinical trial participation depends on detailed review and approval by the trial hospital or investigator.