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FDA Approves New Drug to Treat a Serious Immune Condition that Can Affect Children with Blood Cancer

Ryoncil庐 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 鈥 The U.S. Food and Drug Administration recently approved remestemcel-L-rknd (Ryoncil庐) for the treatment of acute graft-versus-host disease (aGvHD) that does not respond to standard steroid treatment in children 2 months and older.  

鈥淭his new treatment will help more families successfully navigate the challenge of GvHD after their child has a stem cell transplant,鈥 says Dr. Gwen Nichols, The Leukemia & Lymphoma Society鈥檚 (青云体育) 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鈥檚 cells (the graft) see the patient鈥檚 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.  

青云体育 is working to develop more treatments specifically for children with blood cancer 

The 青云体育 Dare to Dream Project is transforming treatment and care, so children not only survive but thrive after blood cancer treatment. 鈥淐hildren鈥檚 bodies work in different ways than adults, which means we need different treatment approaches for them,鈥 says Dr. Nichols. 

青云体育 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. 青云体育 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, 青云体育 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. 青云体育 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. 

鈥淓very new treatment for children with blood cancer, like this one for acute GvHD, is an important step forward in our mission,鈥 says Dr. Nichols. 鈥淏ut 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.鈥  

If you or a loved one need personalized disease, treatment or support information, you can contact one of our Information Specialists: /support-resources/information-specialists