Graft-versus-host disease (GVHD) is counterintuitive
In the weeks and months following a transplant, a major concern is the recipient's immune system rejecting the "foreign" biological material. But in GVHD, the opposite happens: transplanted tissue unleashes a horde of T cells that spark a cascade of inflammation, within 100 days. Typically, GVHD follows a bone marrow transplant (BMT).
Eighty Percent Mortality
BMT has been used for more than half a century to treat and possibly cure certain cancers and single-gene conditions like sickle cell disease, immune deficiencies, and lysosomal storage diseases. BMT and hematopoietic stem cell (HSC) transplantation also enable a cancer patient to withstand higher doses of chemotherapy or radiation.
Acute GVHD develops in about half of the 30,000 or so patients who receive a BMT from a donor worldwide each year. In children the complication can be particularly fierce. A blistery rash can become so extreme that the skin peels away, as can the intestinal lining, causing abdominal pain, diarrhea, and nausea and vomiting. Hepatitis may develop.
Only 20% of children who have steroid-resistant acute GVHD survive. But a treatment of mesenchymal stromal/stem cells (MSCs), called remestemcel-L (RYONCIL™), from Mesoblast Limited, is boosting survival to the 65-75% range among severely affected children, according to recent clinical trial findings.
On August 13, FDA's Oncologic Drugs Advisory Committee, an independent panel of experts who take a first peek at phase III clinical trial results, voted overwhelmingly to advise the agency to continue along the path to approval for RYONCIL. FDA's final meeting is slated for September 30.
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