Baylor College of Medicine Secures $1.4m to Fund a CAR T Cell-based Clinical Trial Investigating Advanced Sarcoma in Children

Baylor College of Medicine Secures $1.4m to Fund a CAR T Cell-based Clinical Trial Investigating Advanced Sarcoma in Children TrialsiteN

A Baylor College of Medicine and Texas Children’s Cancer Center research team has been awarded a $1.4 million gift over three years from the Faris Foundation. Dr. Meenakshi Hedge, assistant professor of pediatrics—hematology and oncology and her team will use the funds to support a clinical trial of a novel form of immunotherapy for children with recurrent or refractory sarcomas. Known as the HEROS 3.0 trial, the team will investigate chimeric antigen receptor (CAR) T cells that are targeted against HER2, a growth protein that is found on the surface of select cancer cells. These specialized altered T cells designed to fight cancer will be administered in combination with immune checkpoint inhibitors, such as the PD1 antibody.

What is Sarcoma?

Sarcoma is represented by a type of malignant tumor that arises from transformed cells of mesenchymal (connective tissue) origin. A broad term, connective tissue can include bone, cartilage, fat, vascular or hematopoietic tissues. Consequently many subtypes can materialize. They are classified based on specific tissue and type of cell from which the tumor originates.    

Quite rare, the risk of a previously healthy person receiving a new diagnosis of bone cancer is less than 0.001%, while the risk of receiving a new diagnosis of soft tissue sarcoma is between 0.0014—0.005%.

The American Cancer Society estimates that in the United States there will be 3,500 new cases of bone sarcoma in 2019 and 12,750 new cases of soft tissue sarcoma. Bone sarcomas represent a small percentage of all new cancer diagnoses (about 0.2%). They can affect all age groups. However, most high-grade bone sarcomas, including Ewing’s sarcoma and osteosarcoma are more common in children and young adults

Origin of the Drug

This HERO trial is actually the result of over a decade of research by Hedge and her mentor Dr. Nabil Ahmed, associate professor of pediatrics—hematology and oncology at Baylor. IN a prior clinical trial, children and young adults with advanced HER2 expressing sarcoma tolerated the HER2 CAR T cell infusion well, and some had clinical benefit.

HEROS 3.0 Trial

This is the first clinical trial in humans involving the study of HER2 chimeric antigen receptor T cells in combination with immune checkpoint blockade to treat advanced sarcoma. The research team seeks to identify the optimal dose and assess the safety of CAR T cells plus immune checkpoint blockade in children with high-risk sarcoma.

In this current clinical trial, in addition to defining the safety and anti-tumor effect of the combination therapy, the research team will study whether the combined approach can boost the vaccine effect. The information could help Hedge and her team improve the way CAR T cell treatments are delivered to children with sarcomas and other solid tumors.

Long Term Outcome Potential

Longer term, the trial results, if positive, could impact the effectiveness of CAR T cells against solid tumors. This could lead to more positive clinical outcomes. Children from this study could benefit from the discoveries and treatment approaches developed by physician-scientists and researchers at Texas Children’s Network.

Lead Research/Investigators

Meenakshi Hedge, MD, assistant professor of pediatrics—hematology and oncology, co-leader HEROS 3.0,  Baylor College of Medicine, Texas Children’s Cancer Center

Shoba Navai, MD, assistant professor of pediatrics—hematology and oncology, co-leader HEROS 3.0,  Baylor College of Medicine

Nabil Ahmed, MD, associate professor of pediatrics—hematology and oncology, Baylor College of Medicine

Sujith Joseph, PhD, Baylor College of Medicine

Jason Yustein, MD, PhD, director of the FarisD. Virani Ewing Sarcoma Center 

Call to Action: If you are based in this part of Texas and have a child in the family struggling with sarcoma, this study may be feasible.