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Drug Discovery Programs

Related Topics

Fibrosis Overview

Liver Disease

Kidney Disease

Lung Disease

Other Diseases

Other Fibrotic

In addition to the diseases listed separately, there are several other diseases that could benefit from drugs that treat inflammation and fibrosis.

Skin (Scleroderma)
Scleroderma is a chronic, systemic, autoimmune disease. One of the most visible indicators of the disease is a thickening or scarring of the skin. There is a localized form which is relatively mild. It is found in only a small number of places on the skin or muscles and does not normally spread elsewhere. The patient’s internal organs are usually not affected.

Systemic scleroderma occurs in two forms. Limited systemic scleroderma involves cutaneous scarring affecting mainly the hands, arms, and face.  It can have complications such as: Raynaud's phenomenon, esophageal dysfunction, thickening of the skin on fingers and toes, and the presence of small dilated veins near the surface of the skin. Pulmonary arterial hypertension, the most serious complication for this form of scleroderma, can occur in up to one-third of patients.

The other form, Severe diffuse systemic scleroderma, progresses quickly and, in addition to affecting large areas of the skin, often affects the kidneys, esophagus, heart, and/or lungs.

Scleroderma is difficult to diagnose because it is often similar to other autoimmune diseases.  There may be many misdiagnosed or undiagnosed cases. Current estimates are that scleroderma affects approximately 300,000 people in the US.  Approximately, one third of those people have the systemic form of scleroderma. Female patients outnumber male patients at 4-to-1.

Currently, there is no cure for scleroderma, but there are treatments available to help particular symptoms.  Most of the compounds in development are aimed at the complications of systemic scleroderma such as Raynaud’s phenomenon and pulmonary involvement.

Fibrosis of the Eye
Ocular tissues are also susceptible to fibrotic diseases. Visual impairment can be the end result of optical infection, inflammation or metabolic disease. This visual impairment can be caused by fibrosis in either the front or back of the eye.

In the anterior part of the eye, visual loss can be caused by corneal opacification and glaucoma. In glaucoma, fibrosis of the tracts that carry intraocular fluid from the eye is believed to cause most of the damage. These tracks become scarred and fluid can build up causing an increase in intraocular pressure. The cornea is covered by a layer of cells both on the inside and outside. Fibrovascular growths on the surface of the cornea secondary to an astigmatism or obstruction can lead to an obstruction. Estimates are that over 2.2 million Americans have glaucoma..

In the back of the eye, fibrovascular scarring can contribute to macular degeneration and diabetic retinopathy. 2010 US estimates for diabetic retinopathy were approximately eight million and growing. Estimates for age related macular degeneration in Americans over 65 is 12-15 million.

While many of these eye diseases have treatments, they are not always a cure and there is a continuing need for new therapies.

Fibrosis of the Heart
Cardiac fibrosis is one of the most interesting and potentially necessary areas for intervention. While the development of cardiac fibrosis is similar to that in other organs such as the lungs and kidneys, research in this area seems to be further back than fibrosis of these other organs. Cardiac fibrosis is defined as the overgrowth of the extracellular matrix (ECM) in the heart. This is present in most types of heart disease. This fibrosis causes increased rigidity that ultimately results in progressive heart failure.

Heart failure (HF), also known as congestive heart failure (CHF), is a condition where the heart cannot pump enough blood to meet the body's needs. Over time, HF leaves the affected heart too weak or stiff to fill and pump efficiently.

An estimated 5.1 million Americans over age 20 have heart failure. American Heart Association projections show that by 2030, heart failure prevalence will increase 25%. Survival of those with a HF diagnosis has improved over time, but the death rate is still approximately 50% within 5 years.
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