Isometric 3D illustration of CAR-T cell therapy in multiple myeloma. Photo: iStock.com
Physicians and researchers at Winship Cancer Institute of Emory University announced today they have administered the first investigational in vivo CAR-T cell therapy in the United States for relapsed and refractory multiple myeloma, marking an important milestone in the development of next-generation cellular therapies.
As part of the Phase 1 inMMyCAR study (NCT07075185), the patient received KLN-1010, an investigational therapy developed by Kelonia Therapeutics designed to generate chimeric antigen receptor T-cell (CAR-T) cells directly inside the patient’s body. Unlike traditional CAR-T therapy, which requires collecting a patient’s T cells, sending them to a manufacturing facility for engineering and waiting for the cells to be returned, in vivo CAR-T aims to streamline the treatment process, reduce delays in care, and eliminate the need for lymphodepleting chemotherapy.
Advancing In Vivo CAR-T Technology
“This represents the kind of innovation that can help shape the future of cancer treatment,” said Sagar Lonial, MD, chief medical officer of Winship Cancer Institute. “We are proud that Winship was selected as a leading site for this important clinical trial and that our team was able to move quickly to bring this opportunity to patients.”
KLN-1010 was developed using Kelonia’s in vivo gene placement system (iGPS) platform which combines in vivo lentiviral delivery technology with optimized CAR constructs to achieve potent, highly specific, and efficient transduction of T cells inside the body.
The study, which will assess the safety and efficacy of KLN-1010, first opened in Australia before expanding to sites in the United States. Winship was the second U.S. site activated for the trial and was the first site to administer KLN-1010 to a patient in this country. Winship leaders credited the rapid trial activation to the coordinated efforts of the myeloma program, clinical trials office, and research teams.
What This Means for Patients with Multiple Myeloma
Nisha Joseph, MD
According to Nisha Joseph, MD, hematologist and multiple myeloma specialist at Winship Cancer Institute, the investigational therapy could represent an important evolution in CAR-T treatment if ongoing studies continue to show positive results.
“Traditional CAR-T therapies have been highly effective for many patients, but they require multiple steps including cell collection, manufacturing time, and preparative chemotherapy,” Joseph said. “An in vivo approach is designed to create those therapeutic cells directly within the patient, which could make this type of treatment more accessible and more efficient in the future.”
Preliminary findings presented at the American Society of Hematology annual meeting reported encouraging early responses among the first patients treated, with the therapy appearing well tolerated. Researchers caution that the treatment remains investigational, and additional study is needed to determine long-term safety and effectiveness.
“This is an exciting advancement in the multiple myeloma space,” said Ajay Nooka, MD, MPH, associate director of clinical research of Winship Cancer Institute and director of the Myeloma Program at Emory University School of Medicine's Department of Hematology and Medical Oncology. “It is important for eligible patients with relapsed myeloma to have access to promising investigational therapies, and for Winship to remain at the forefront of bringing those opportunities to patients.”
Multiple myeloma is a cancer of plasma cells, a type of white blood cell found in bone marrow. Although treatment advances have improved outcomes, many patients eventually need new options after their disease returns or stops responding to therapy.
As Georgia’s only National Cancer Institute-designated Comprehensive Cancer Center, Winship offers patients access to a broad portfolio of cancer clinical trials, including studies evaluating emerging immunotherapies and precision treatments.