Alternative treatment cures rare blood disease for first time

pharmafile | March 22, 2017 | News story | Research and Development congenital dyserythropoietic anaemia 

A Chicago man has become the first person ever documented to be cured of congenital dyserythropoietic anaemia (CDA), a rare and debilitating blood disorder, using a method of treatment which avoids the traditional combination of radiation and high-dose chemo to prepare a patient for a stem cell transplant.

35 year old David Levy had suffered for over 30 years with CDA, a disease disorder causing the body to underproduce red blood cells leading to progressive organ damage and eventual early death. As a result of regular blood transfusions to treat the illness, Levy suffered from iron poisoning, as well as severe fatigue, heart palpitations and an enlarged liver.  

As a result of his intolerance to traditional treatments for the disease, Levy was denied the possibility of a stem cell transplant.

Dr Damiano Rondelli, the Michael Reese Professor of Haematology at the University of Illinois explained: “For many adult patients with a blood disorder, treatment options have been limited because they are often not sick enough to qualify for a risky procedure, or they are too sick to tolerate the toxic drugs used alongside a standard transplant.”

Work performed at the University of Illinois Hospital & Health Sciences System in curing sickle cell patients had led to a new treatment which enabled transplanted donor cells to gradually take over a patient’s bone marrow, rather than eliminating the original cells in preparation for the transplant.

Since the treatment in 2014, Levy’s health has returned significantly: “I still have some pain and some lingering issues from the years my condition was not properly managed, but I can be independent now,” he remarked. “That is the most important thing to me.”

Rondelli noted the promise this treatment present for future patients of the disease: “The use of this transplant protocol may represent a safe therapeutic strategy to treat adult patients with many types of congenital anaemias – perhaps the only possible cure.”

Matt Fellows

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