Disease background

Fibrotic lung disease.

Rare lung diseases often affect the interstitium, a lace-like network of tissues that extends throughout both lungs. This network provides support to microscopic air sacs where oxygen is absorbed from inhaled air and carbon dioxide is removed and exhaled. Interstitial lung disease (ILD) causes a thickening of this interstitium, which can be caused by scarring (fibrosis), inflammation, or extra fluid. The most common symptom of ILD is shortness of breath, which worsens as the disease progresses; other symptoms include a persistent dry cough, weight loss, and fatigue. The symptoms of ILD interfere with daily life and can be very distressing for both patients and their families. Our focus at Vicore is to develop therapies to help patients who are diagnosed with these life-changing diseases.

There are several forms of ILD, including idiopathic pulmonary fibrosis, pulmonary sarcoidosis, and diffuse systemic sclerosis.

 

Idiopathic pulmonary fibrosis

The most common type of pulmonary fibrosis is idiopathic pulmonary fibrosis (IPF) which is a severe and devastating disease with no known cause. Debilitating symptoms, including breathing difficulties and persistent dry cough, typically appear between the ages of 50 and 70 years and, while the disease is more common in men, the number of cases in women is increasing. It has been estimated that as many as 111,000 people are currently living with IPF in the EU, with around 35,000 new cases diagnosed each year. In the USA, approximately 100,000 people are currently living with IPF, with up to 40,000 new diagnoses per year.

Unfortunately, a diagnosis of IPF is often delayed because the symptoms are similar to other lung diseases. Although IPF is a progressive disease, the rate of progression varies among patients. Sadly, the prognosis for patients diagnosed with IPF is poor, with most patients surviving for only 3-5 years after diagnosis.

Currently, there is no cure for IPF, and only two approved medications are available, pirfenidone and nintedanib. Both medications have been shown to slow the progression of the disease; however, side effects have limited their use. Supportive treatments include oxygen therapy using a portable oxygen tank and immunization against pneumonia and influenza to avoid infection. Patient management plans consist of keeping active, following a healthy diet, and taking plenty of rest. Clearly, new treatments are urgently needed to reduce the significant burden of this disease.

Systemic sclerosis (SSc)

Systemic sclerosis is a rare, chronic, and progressive disease, which may affect a number of different parts of the body such as the skin, joints, lungs, and heart. SSc is difficult to diagnose, and treatment options are very limited. It is more common among women than men, and is most common in women aged 60-70 years.

Interstitial lung disease develops in around 50% of patients after approximately five years, and is the main cause of death in patients with SSc. High blood pressure affecting the blood vessels in the lungs and heart is also a serious complication of SSc. Very few treatment options are available to improve life expectancy at present.

 

Pulmonary sarcoidosis

The cause of pulmonary sarcoidosis (PS) is unknown. It typically affects the lungs and lymph glands. Inflammation leads to abnormal clumps of inflamed tissues, with scarring and stiffness of lung tissue. The disease can exist without showing any symptoms, be short-lasting and need minimal or no treatment, or it can last for many years and require continuous treatment.

There are significant racial and gender differences in the severity of PS, and women are more often affected than men. The chronic progressive form of the disease results in breathing difficulties and a severe, persistent dry cough, debilitating symptoms that often appear between the ages of 30 and 55 years.

As with IPF, diagnosis of PS is often delayed because the symptoms can be mistaken for other lung diseases, or discovered by chance in patients without symptoms. While there is no cure for PS, corticosteroids are often used to reduce inflammation and relieve symptoms, and the disease can disappear on its own over time in about half of cases. However, when corticosteroids fail to work the disease can be devastating, and lung transplantation may offer the only chance for long-term survival.

 

Vicore drug development programmes

Our aim is to develop new therapies for patients with rare lung diseases; if you would like to hear more about our experimental treatments you can contact us at info@vicorepharma.com.