Source: FDA Approves First Mesenchymal Stromal Cell Therapy to Treat Steroid-refractory Acute Graft-versus-host Disease, U.S. FDA, 18 Dec 2024. Summary written by Future Health GCC.

What happened

On 18 December 2024, the US Food and Drug Administration approved Ryoncil (remestemcel-L). It is the first mesenchymal stromal cell therapy approved in the United States.

What it treats

Ryoncil is approved for children aged two months and older with steroid-refractory acute graft-versus-host disease. This is a serious complication that can follow a blood stem cell transplant from a donor, when the donor’s immune cells attack the patient’s organs and steroids have not helped. The cells in Ryoncil come from the bone marrow of healthy adult donors.

What the study found

The approval was based on a study of 54 children. Each received eight infusions over four weeks. Twenty-eight days after starting treatment, 16 children (30%) had a complete response and 22 (41%) had a partial response.

The most common side effects were infections, fever, bleeding, swelling, stomach pain and high blood pressure. Infusions have to be monitored by a doctor.

What it means for families

For the first time, the US regulator has approved a treatment made from mesenchymal stromal cells, the same broad family of cells studied in umbilical cord tissue. Ryoncil itself is made from donor bone marrow, and it is approved for one serious condition in children.

Read the original article on U.S. FDA →

This is a summary of published research, written by Future Health GCC. It is not medical advice. Speak to your doctor about any treatment.

Photo: U.S. FDA, via Wikimedia Commons (public domain).