Ryoncil® is for children 2 months and older who get steroid-refractory acute graft-versus-host disease (SR-aGvHD) after a stem cell transplant
Rye Brook, N.Y., January 3, 2025 – The U.S. Food and Drug Administration recently approved remestemcel-L-rknd (Ryoncil®) for the treatment of acute graft-versus-host disease (aGvHD) that does not respond to standard steroid treatment in children 2 months and older.
“This new treatment will help more families successfully navigate the challenge of GvHD after their child has a stem cell transplant,” says Dr. Gwen Nichols, The Leukemia & Lymphoma Society’s (LLS) Chief Medical Officer.
Acute GvHD is a common and very serious complication of donor stem cell transplants, an important treatment option for children with certain types of blood cancer.
Acute GvHD usually occurs within three months of a stem cell transplant, though it can come on later. It is caused when the donor’s cells (the graft) see the patient’s healthy cells (the host) as foreign and attack them. GvHD can be very serious, attacking major organs.
Learn more about the risks and signs of acute graft-versus-host disease
This new medication is the first one approved specifically for children whose GvHD does not respond to steroids. It is also the first mesenchymal stromal cell (MSC) therapy approved in the United States. Scientists are working to develop more treatments using MSCs, which can help repair, replace or even regenerate damaged cells, tissues and organs.
LLS is working to develop more treatments specifically for children with blood cancer
The LLS Dare to Dream Project is transforming treatment and care, so children not only survive but thrive after blood cancer treatment. “Children’s bodies work in different ways than adults, which means we need different treatment approaches for them,” says Dr. Nichols.
LLS is leading the first-of-its-kind pediatric acute leukemia master trial, called PedAL, which is testing multiple treatments for kids with acute leukemia simultaneously to speed up the research process. LLS also makes comprehensive investments in academic and biotechnology company research bringing us closer to having more effective and personalized blood cancer treatments for all kids.
Through Dare to Dream, LLS also powers meaningful policy changes at the state and federal levels to ensure that more kids with blood cancer and their families have better access to care. LLS also provides free education and support services to provide children and young adults with blood cancer and their families with a much-needed lifeline to thrive from diagnosis, through treatment and beyond.
“Every new treatment for children with blood cancer, like this one for acute GvHD, is an important step forward in our mission,” says Dr. Nichols. “But improving outcomes also relies on making sure families have access to the best care and the information they need to make the best treatment choices for their children.”
If you or a loved one need personalized disease, treatment or support information, you can contact one of our Information Specialists: https://www.lls.org/support-resources/information-specialists