β-thalassemia is one of the world’s most common blood disorders. Researchers noticed it shares similarities with brain disorders like Alzheimer’s disease. Both involve abnormal proteins that build-up in cells.
St. Jude scientists turned that observation into a possible treatment approach. They found a drug called rapamycin that’s widely used to treat other diseases. This drug is also used with organ transplant patients. Researchers showed the drug eased symptoms in mice with β-thalassemia. The drug also helped reduce the build-up of toxic proteins in developing blood cells from people with β-thalassemia.
“The next step is to design a clinical trial to test whether rapamycin is safe and effective for treating people with β-thalassemia,” said Mitchell Weiss, MD, PhD, St. Jude Department of Hematology chair.
A report on this work appeared in Science Translational Medicine.