Technological Landscapes: Advanced Intravenous Iron-Deficiency-Anemia Therapy
Lexington-based AMAG Pharmaceuticals, Inc. is a biopharmaceutical company focused on the development and commercialization of a therapeutic iron compound to treat iron-deficiency-anemia.
There are diseases and then there are super-diseases. To visualize the scope of diseases that affect millions is almost incomprehensible. To visualize the scope of super-diseases that affect billions of people staggers the mind. When a disease impacts the lives of one out of three people on the entire Earth it is apocalyptic. Yet, that is the scourge of iron-deficiency-anemia (IDA).
According to the Mayo Clinic, IDA (low red-blood-cell levels) is characterized by extreme fatigue, weakness, poor appetite, shortness of breath, headache, dizziness and lightheadedness. The result is a loss of work efficiency, pregnancy issues, increased risk of death for children, and deteriorated physical and cognitive development. As more and more people in a society are affected, the national productivity decays and the national economy is eventually devastated.
Because the disease is most commonly driven by malnutrition, IDA strikes at the heart of the poor. The World Health Organization (WHO) estimates that “in developing countries every second pregnant woman and about forty percent of preschool children are estimated to be anemic.”
IDA also impacts the lives of people in developed countries. Its impact is not necessarily due to malnutrition but occurs in other more devious ways. For example it is prevalent in those cases where adults suffer from chronic kidney disease (CKD). Approximately one out of every eight adults in the United States is affected by CKD.
Treatment for IDA, aside from improved dietary habits, is primarily by means of iron pills (oral iron supplements). However, the side-effects of such therapy include constipation, heartburn, cramping, nausea and vomiting. People simply stopped taking their pills.
A better IDA therapy was needed and Lexington-based AMAG became a leader in this vital research. The company applied their nanotechnology expertise in ultra-small, semi-synthetic coated, iron-oxide particles to develop a product called Feraheme (ferumoxytol). AMAG’s primary focus was on an intensive research, development, sales and manufacturing program to produce an effective IDA treatment for adults who suffer with CKD.
The subsequent successful treatment of CKD was expanded into a recent AMAG advanced global clinical study (six countries) of patients with IDA who were afflicted by a number of different underlying causes. The new study obtained superior IDA therapeutic results on the basis of efficacy and safety. This set of positive results validated the previously established efficacy of Feraheme in the treatment of adult CKD. The door was opened to a more universal application of Feraheme.
In opposition to iron pills, Feraheme is delivered intravenously (IV) so that larger amounts of iron can be delivered with a greater increase in hemoglobin levels. The hemoglobin increase is due to the high chemical absorptivity of the nano-particles in the creation of hemoglobin. Thus, the more available the iron, the more hemoglobin is generated, and the more oxygen is carried by the red blood cells to all other parts of the body leading to improved health.
Most importantly and concurrently, the level of side effects is dramatically reduced because of Feraheme’s unique physiochemical structure. Its structure is defined by a National PBM Drug Monograph on Feraheme. The monograph indicates that it is a super-paramagnetic iron-oxide. Super-paramagnetism is another extraordinary result of working with nanometer-sized materials, in this case magnets. This nano-tech phenomenon is similar to the one addressed in an earier Technological Landscapes column, on "The Revolution in Lighting."
The desired property of the iron-oxide, when cased in a protective, specially designed carbohydrate shell, is that less free iron (iron not contained in hemoglobin) is released into the body than with other intravenous iron preparations. It is the amount of free iron that is believed to cause the dreaded side-effects, especially those that cause serious problems such as the risk of infection due to increased microbial growth.
The carbohydrate shell protects the Feraheme until it enters and is encapsulated within the liver, spleen and bone marrow. The Feraheme is eventually released into naturally-occurring iron pools and is then transformed into hemogloblin.
In July 2012, when AMAG announced its positive preliminary results from their global IDA registration program, Lee F. Allen, MD, Ph.D., and chief medical officer for AMAG stated:
"We will seek approval for Feraheme (from the FDA) for the treatment of a broader population of patients with iron deficiency anemia and a history of an unsatisfactory response to oral iron therapy…we believe that Feraheme could provide an important clinical benefit with an improvement in the quality of life for this patient population, and could be a valuable therapeutic alternative to currently approved IV irons for the treatment of their iron deficiency anemia.”
Clearly, any therapeutic advancement by AMAG to decrease this world-wide IDA epidemic would be greatly welcomed.