Siteman Kids is the First Site of Multicenter CAR-T Trial for Autoimmune Diseases

Access to the novel immunotherapy ­_­— which “has the potential for cure” — is the latest offering by the national leader in pediatric hematology, oncology, and cell therapies.

Siteman Kids at St. Louis Children’s Hospital, along with WashU Medicine, is the first site to test a novel immunotherapy drug for the treatment of autoimmune diseases such as systemic lupus erythematosus, juvenile myasthenia gravis, juvenile dermatomyositis, and ANCA-associated vasculitis in children and young adults.

The clinical trial, called HELIOS Descartes-08 (202510129), evaluates dosing levels of an autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets the B-cell maturation antigen (BCMA). Significantly, no neoadjuvant chemotherapy is needed prior to the start of Descartes-08 therapy.

“We are the first site in a multicenter clinical trial that will offer qualified patients an infusion of mRNA-modified CAR-T cells that target plasma cells,” said WashU Medicine hematologist/oncologist Melissa Mavers, MD, PhD, site principal investigator and a leader on the childhood cancers specialist team at Siteman Cancer Center. “This trial is exciting because this is the first available therapy targeting plasma cells in autoimmune disease and has the potential for cure for these patients.”

Earlier results of the Descartes-08 therapy, developed by Cartesian Therapeutics, have been promising. The FDA has granted it Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation.

To be eligible for the clinical trial, a patient must:

• Be age 12 or older
• Diagnosed with systemic lupus erythematous, juvenile myasthenia gravis, juvenile dermatomyositis, or ANCA-associated vasculitis
• Have undergone systemic treatment

The trial will be administered through the Pediatric Hematopoietic Cell Transplant and Cellular Therapy Program at Siteman Kids at St. Louis Children’s Hospital.

Other Major Advancements

The clinical trial launch follows these recent advances at Siteman Kids:

Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma (ALL/LBL)

The FDA granted Breakthrough Therapy Designation for WU-CART-007, an allogeneic CAR-T therapy for T-cell malignancies developed by WashU Medicine researchers at Siteman Cancer Center. WU-CART-007 is an off-the-shelf, anti-CD7 CAR T-cell therapy for children and adults diagnosed with relapsed or refractory T-cell lymphoblastic leukemia or T-cell lymphoblastic lymphoma (R/R T-ALL/LBL). The FDA designation, announced in January, came after global clinical trials found that WU-CART-007 (also known as soficabtagene geleucel, or sofi-cel), resulted in 73% of adults and adolescents in the trials achieving full remission after receiving a full dose of the cellular therapy. WashU Medicine pediatric hematologist/oncologist Thomas Pfeiffer, MD, co-led the pediatric component of the clinical trial at Siteman Kids.

Learn more: U.S. FDA Grants to Wugen’s WU-CART-007 Breakthrough Therapy Designation

Brain Tumors

Global consultations and referrals continue to expand the reach and impact of an International Neuro-Oncology Tumor Board that originated at Siteman Kids. WashU Medicine neuro-oncologist Mohamed Shebl Abdelbaki, MD, director of the Pediatric Neuro-Oncology Program at Siteman Kids, began the monthly tumor board meetings in early 2021. To date, more than 2,500 health care specialists from 71 institutions in 41 countries have participated in the meetings, which have resulted in several advancements in best care practices for the management of complex brain tumors in infants, children and young adults.

Also available at Siteman Kids is participation in the CONNECT1906: PEP-CMV vaccine phase II clinical trial that is testing a CMV-directed peptide vaccine in children with recurrent medulloblastoma or newly diagnosed high-grade gliomas or diffuse intrinsic pontine gliomas. The multicenter study advances investigations into whether CMV-targeted vaccines can serve as a novel immunotherapeutic approach and boost responses against these cancers in pediatric patients. Eligible patients will receive a single, 5-day course of oral chemotherapy followed by PEP-CMV injections. Siteman Kids’ site principal investigator is Eric Thompson, MD.

For patients with malignant brain tumors, Siteman Kids also is involved in a multicenter phase I trial investigating the safety and dosing of intra-tumoral injections of NK cells in patients with recurrent or progressive tumors located in the upper part of the brain. Siteman Kids is one of only seven institutions offering this trial through the Pediatric Neuro-Oncology Consortium. WashU researchers are known internationally for their breakthroughs in the development and use of NK cell immunotherapies. Site principal investigators are WashU Medicine neuro-oncologists Mohamed Shebl Abdelbaki, MD, and Michael Angelo Huang, MD, MS.

“These are just a few of the leading-edge research efforts and collaborations we have underway at Siteman Kids,” said Jorge Di Paola, MD, chief of the Division of Pediatric Hematology and Oncology and the Elizabeth Finney McDonnell Endowed Chair in Pediatric Hematology Oncology at Siteman Kids at St. Louis Children’s Hospital and WashU Medicine. “We have 12 basic and translational research labs solely dedicated to advancing understanding and treatments for a wide range of pediatric cancers and more than 280 clinical trials available.”

For more information regarding clinical trials at Siteman Kids, email [email protected] or call 800-600-3606 to make a referral.

Learn about all active trials.