Selected Medicines in Development for Heart Disease and Stroke

Long-term Treatment for Chronic Heart Disease – Coronary heart disease is the single largest killer of Americans, according to the American Heart Association, causing about one in every six deaths in the United States. Atherosclerosis, an inflammatory disease characterized by the building-up of plaque within the walls of the arteries, is the underlying cause of most heart attacks and strokes. One novel medicine in development is a selective inhibitor of an enzyme found in blood and atherosclerotic plaque. Elevated levels of the enzyme are involved in the development and progression of atherosclerosis.

Preventing Heart Attacks – Each year, more than 1 million Americans suffer a new or recurrent heart attack (myocardial infarction), according to the American Heart Association. Clinical evidence has shown that a high number of circulating platelets are associated with an increased incidence of heart attack and thrombotic stroke. A potential first-in-class medicine in development simply reduces the circulating platelets without interfering with platelet function. This approach is thought to prevent thromboembolic events without increasing the risk of bleeding.

Stem Cell Therapy – A potential treatment for ischemic heart disease (and being tested in heart failure patients) uses cells from the patient’s own bone marrow to target damaged tissue in the heart. The cells are inserted using an investigational catheter that enables local delivery of cell and gene therapies. It is believed that a patient’s immune system will not attack the newly transplanted cells because they are native to the patient.

Preventing Thrombosis – A common complication of surgery can be hospital-acquired deep vein thrombosis (DVT), where a blood clot forms in the deep vein (usually in the leg) that partially or totally blocks the flow of blood. According to the U.S. Centers for Disease Control and Prevention (CDC), 200,000 to 400,000 Americans each year suffer from DVT. A new potential first-in-class medicine in development is designed to prevent DVT from occurring. It is an oral, direct Factor Xa inhibitor (an anticoagulant that limits blood clotting) that acts at the central point in the coagulation cascade, to regulate thrombin generation, which leads to clot formation.

Improving Heart Function – Nearly 6 million Americans are living with congestive heart failure, according to the American Heart Association. Patients with congestive heart failure due to myocardial infarction often have scar tissue in the heart, limiting the heart’s ability to pump blood. Currently, heart transplants are the only existing cure for late-stage congestive heart failure. A new treatment in development delivers human stem cells (myoblasts) to form new heart muscle to restore cardiac function to the damaged heart.

Inhibiting Platelets – Acute coronary syndrome (ACS), which includes acute myocardial infarction (heart attack) and unstable angina (chest pain and discomfort caused by a low supply of oxygen to the heart muscle), affects more than 700,000 Americans, according to the American Heart Association. Acute coronary syndrome may develop slowly over time by the building up of plaques in the arteries in the heart, causing the arteries to narrow and make it more difficult for blood to flow through them. If the plaque in the coronary arteries ruptures it can cause a heart attack. A blood clot will form on the site of the rupture, blocking the flow of blood through the artery. Many patients suffering from acute coronary syndromes are managed using percutaneous coronary intervention (PCI) and a stent to relieve clogged arteries. A new medicine in development is an anti-platelet that inhibits platelet activation and prevents them from clumping and sticking together, causing clogged arteries that can lead to heart attack or stroke. Another medicine in development is a first in class antiplatelet treatment for ACS that selectively inhibits both platelet activation and aggregation in a reversible manner, thereby preventing major adverse cardiovascular events.

Lowering Blood Lipids – More than 102 million American adults have a total blood cholesterol level over 200, according to the American Heart Association. High cholesterol levels are a major risk factor of heart disease. The CDC estimates that a 10 percent decrease in total cholesterol levels population-wide could result in a 30 percent reduction in the incidence of coronary artery disease. A potential medicine in development is a selective inhibitor of a protein that plays a major role in transferring high-density cholesterol lipoprotein (HDL, or good cholesterol) to low-density lipoprotein cholesterol (LDL, or bad cholesterol). High HDL levels can help decrease the risk of cardiovascular disease.

Treating Inherited High Cholesterol – Familial hypercholesterolemia (HFH) is a genetic disorder that causes extremely high levels of low-density lipoprotein (LDL or “bad”) cholesterol in the blood. Patients with FH have mutations in the gene that normally “encodes” a protein to remove LDL from the blood. A first in class antisense inhibitor directed at the protein that plays a pivotal role in the production of LDL cholesterol is under development. The medicine has also shown that the inhibition of the protein itself is anti-inflammatory and may suggest an additional cardiovascular benefit. Current treatments leave FH patients at risk of cardiovascular events.

Gene Therapy for Heart Failure – Congestive heart failure is the leading cause of hospitalizations in the United States costing $39.2 million in 2010, according to the CDC. A genetically-targeted enzyme replacement therapy is being tested to restore levels of a specific gene to promote a failing heart to pump better and minimize the severity of heart failure. In all forms of late-stage heart failure, levels of the gene decline resulting in deficient heart function.

* From New Medicines in Development for Heart Disease and Stroke, PhRMA, 2011

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