Blueprint for Breakthroughs is a LinkedIn newsletter published by Adrienne B. Mendoza, MHA, SVP BioBridge Global and Chief Operating Officer (COO), BBG Advanced Therapies
Originally published on LinkedIn on July 15, 2025, Adrienne discusses the progress of cell therapies in sarcoma treatment
July is Sarcoma Awareness Month – a time to amplify the voices of those affected by this rare, often aggressive cancer, and to spotlight innovation in sarcoma treatment, including cell and gene therapies that could reshape the future of care.
Sarcomas make up less than 1% of all adult cancers, yet they present a disproportionate number of treatment challenges. With more than 70 distinct subtypes, sarcomas can arise anywhere in the body, from bone to soft tissue, and often affect younger patients. Many go undiagnosed until late stages, and for those with advanced or metastatic disease, options are limited.
But the tide is turning, and new access models could bring these therapies to the forefront.
Progress in the Pipeline
The landscape of sarcoma treatment is changing rapidly, with several notable breakthroughs in cell therapy over the past year. Right now, there is both scientific momentum and real progress for patients who previously had few options. It’s exciting.
Groundbreaking FDA Approval for Solid Tumors
In August 2024, Adaptimmune’s Tecelra (afamitresgene autoleucel, or afami-cel) became the first engineered T-cell receptor (TCR) therapy approved for any solid tumor. This historic approval, based on the SPEARHEAD-1 trial, offers a new option for adults with metastatic synovial sarcoma expressing the MAGE-A4 antigen and specific HLA types. Patients who had exhausted chemotherapy options saw a 43% overall response rate, marking a major milestone for both the company and the field.
CAR T-Cell Therapy Trials for HER2, GD2, and B7-H3 Targets
HER2 is a protein involved in cell growth, and while HER2 is best known in breast cancer, it can also be found in other cancers, including certain types of sarcoma and gastric (stomach) cancer. In sarcoma, some tumors express HER2, and this has become a target.
In April 2024, researchers at Baylor College of Medicine, Texas Children’s Hospital Cancer, and Houston Methodist reported promising results from the HEROS 2.0 phase I trial. This study evaluated HER2-targeted CAR T cells in high-risk sarcomas, demonstrating both safety and clinical benefit. These findings highlight the progress being made for patients with difficult-to-treat sarcomas.
There are multiple CAR T-cell therapies targeting HER2, GD2, and B7-H3 are in early-phase clinical trials for sarcoma subtypes, including osteosarcoma, Ewing’s sarcoma, and rhabdomyosarcoma. These trials are showing encouraging results, though most remain investigational as of the writing of this article in July 2025.
TCR Therapy Options for Soft Tissue Sarcoma
At the #ASCO2024 meeting, Memorial Sloan Kettering Cancer Center and collaborators shared encouraging results from the IGNYTE-ESO phase 2 trial of letetresgene autoleucel (lete-cel), a TCR therapy targeting NY-ESO-1. Tested in synovial sarcoma and myxoid/round cell liposarcoma, the therapy achieved a 40% overall response rate, further illustrating the rapid expansion of engineered T-cell therapies for sarcoma.
Ongoing Collaboration and Pipeline Growth
MD Anderson Cancer Center, and Memorial Sloan Kettering Cancer Center are actively leading trials of CAR T and TCR therapies for sarcoma, often in partnership with biotech firms, such as Adaptimmune. These collaborations are accelerating both discovery and patient access, with multiple early-phase and pivotal trials underway.
Access: The Missing Piece
Breakthroughs are only as powerful as our ability to deliver them. Cell therapies require a highly coordinated, patient-specific process, starting with the harvest of the patient’s or donor’s cells (the crucial ingredient in cell therapy) through a special immune cell collection procedure known as leukapheresis. This step is still largely limited to patients who can travel to major academic centers with built-out infrastructure. For rare cancer patients, especially those in rural or underserved regions, that can mean the difference between getting treatment and going without.
BBG Advanced Therapies, has introduced the Mobile Leukapheresis Center as an emerging solution, bringing cell collection capabilities closer to patients and supporting participation in early-phase trials through to late phase and commercial therapies. It’s a vital option for broadening access to advanced therapies.
Designing for Rare, Not Just Common
Most cell and gene therapy infrastructure in the United States has been built around high-prevalence diseases like leukemia or lymphoma, with specialized centers concentrated in a limited number of major hospitals. As a result, access remains highly uneven: there are currently about 20 states in the US with no cell and gene therapy infrastructure at all. For patients in these regions, participating in clinical trials or receiving advanced therapies often means traveling hundreds or even thousands of miles.
To truly democratize access, including for rare cancers like sarcoma – the available infrastructure and ecosystem must evolve. That means:
- Establishing more regionalized and community-based access points for cell collection, clinical coordination, and patient support, beyond just the largest academic centers.
- Developing flexible testing and manufacturing processes that can accommodate early-phase programs and adapt quickly to new therapies as they move from research to clinical use.
- Designing process development specifically for low-volume, high-urgency populations—like those with rare sarcoma subtypes—so that these patients are not left behind as the field advances.
With more clinical trials opening and new CAR and TCR constructs under active development, cell therapy for sarcoma is no longer just a future possibility – it is an emerging reality.
Now is the time to build the access model that will support these advances and ensure that innovation reaches every patient who needs it.
Let’s Work Together to Build Systems with Reach
Sarcoma may be rare, but the courage and resilience of those affected—and the innovation now unfolding—are anything but. We are standing at a pivotal moment: the science is moving forward, and the tools to transform lives are within reach. Yet, progress will only matter if it reaches every patient, everywhere.
Let’s not allow geography or infrastructure gaps to dictate who benefits from tomorrow’s breakthroughs. Let’s work together to ensure that hope, access, and advanced therapies are not privileges for the few but rights for all, no matter where they live or what disease they face.
If you are a patient, caregiver, clinician, or advocate who wants to help shape this future, or if you have questions about access, mobile cell collection solutions, or clinical trial opportunities, please reach out to me. Your voice and experience matter, because we can make the promise of next-generation sarcoma care a reality for every patient who needs it.
