Enzyme Replacement Therapy Video
MYDICAR® is a genetically-targeted enzyme replacement therapy for advanced heart failure, one of the most expensive and debilitating conditions facing the U.S. health care system. Heart failure will cost about $39.2 billion in direct and indirect costs in 2010, primarily because patients are chronically in and out of intensive care units. Candidates for MYDICAR are advanced heart failure patients who continue to deteriorate from their disease despite optimal drug therapy, including those who are eligible for either an implantable mechanical support device or a heart transplant. Both, are highly invasive procedures for which Medicare reimburses at a rate of $200,000 per patient.
MYDICAR treatment involves a one-time outpatient infusion in a cardiac catheterization laboratory, similar to undergoing an angiogram. MYDICAR is designed to restore levels of an enzyme known to play a key role in the progression of heart failure. Repairing this molecular defect in preclinical studies reversed the disease and restored cardiac function. In the Phase 2 CUPID clinical trial in 39 patients, the frequency of death, worsening heart failure, heart failure-related hospitalizations, heart transplant, and need for a mechanical heart pump was dramatically lower and sustained for patients on high-dose MYDICAR compared with placebo. Furthermore, patients’ ability to exercise, symptoms of heart failure and quality of life improved.
MYDICAR’s molecular target is an enzyme found in the sarcoplasmic reticulum (SR) that is critical to the contraction of the cardiac muscle cell. The SR is a specialized part of a cell (cellular organelle) that regulates the contraction and relaxation of cardiac muscle cells by coordinating the outflow (contraction) and inflow (relaxation) of calcium ions (Ca2+). The heart muscle’s ability to contract, and thus to pump blood and maintain oxygenation of the body, is determined by a continual re-loading of the SR with Ca2+ to “stage” for the next cycle of contraction. The key factor that enables the re-loading of the SR with Ca2+ is the enzyme SERCA2a. Numerous human studies have established a clear association between depleted SERCA2a enzyme in cardiac cells and the progression of end-stage heart failure.
...The mean duration of hospitalization in the MYDICAR® high dose group during the 12-month period was 0.4 days per patient compared with 4.5 days per patient in the placebo group. This finding is especially noteworthy because heart failure is the leading cause of hospitalization in Americans 65 years of age and older...