“Without iron, life itself wouldn’t be feasible,” says Barry Paw, MD, PhD. “Iron transport is very important because of the role it plays in oxygen transport in blood, in key metabolic processes and in DNA replication.”
Although iron is crucial to many aspects of health, it needs the help of the body’s iron-transporting proteins. Which is why new findings reported in Science could impact a whole slew of iron disorders, ranging from iron-deficiency anemia to iron-overload liver disease. The team has discovered that a small molecule found naturally in Japanese cypress tree leaves, hinokitiol, can transport iron to overcome iron disorders in animals.
The multi-institutional research team is from the University of Illinois, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Brigham and Women’s Hospital and Northeastern University. Paw, co-senior author on the new paper and a physician at Dana-Farber/Boston Children’s, and members of his lab demonstrated that hinokitiol can successfully reverse iron deficiency and iron overload in zebrafish disease models.
“Amazingly, we observed in zebrafish that hinokitiol can bind and transport iron inside or out of cell membranes to where it is needed most,” says Paw.
This gives hinokitiol big therapeutic potential. …
In some children the body’s machinery for making red blood cells just doesn’t work right. Conditions like Diamond Blackfan anemia or thalassemia can leave the body anemic, struggling to keep up with its own demands for oxygen. And the misshapen red blood cells of sickle cell disease can get stuck in small blood vessels and cause anemia, organ damage and great pain.
Right now, the most effective way to care for these blood disorders is with blood transfusions. But unlike trauma or surgery, a single transfusion doesn’t solve the problem for people with life-long anemias or sickle cell. Most people with thalassemia, for example, have transfusions every month for their entire life.
“After about 20 transfusions, you reach a point where the body is overloaded with iron from all of the extra hemoglobin that’s been introduced into it,” says Ellis Neufeld, MD, PhD, director of the Thalassemia Program at Dana-Farber/Children’s Hospital Cancer Center (a partnership of Boston Children’s Hospital and Dana-Farber Cancer Institute). “The body has no way to actively remove iron on its own, so the iron starts to build up.” Over time, this can damage the liver, heart, pancreas and other major organs.
Over the last 40 years, a lot of work at DF/CHCC and elsewhere has gone into what’s called chelation therapy: drug-based treatments that scrub the blood of excess iron. Right now there are three chelating drugs in broad use: deferoxamine, deferasirox and deferiprone. They work well for many patients, but have their disadvantages. …