The Cystic Fibrosis Foundation describes CF as a genetically-acquired chronic disease that impacts the lungs and gastrointestinal tract, causing these body systems to produce abnormally thick, and viscid mucus that has the tendency to consolidate in the lungs and block the secretion of pancreatic enzymes, resulting in malabsorption. Today, about 30,000 American children and adults, and over 70,000 people are affected all over the globe. While treatment for the disease has made tremendous strides over the past few decades, cystic fibrosis is still a deadly disease that affects most patients from early childhood and dramatically affects quality of life and lifespan.
In order to further accelerate research and development of viable, effective CF therapies, Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), a subdivision of the Cystic Fibrosis Foundation, recently revealed that they will be prolonging the grant for their partnership with Proteostasis Therapeutics, Inc., which is geared towards developing state-of-the-art treatments for the most prevalent form of cystic fibrosis, Delta F508.
Proteostasis Therapeutics will carry on with their dedication to research and development for the Delta F508 form of CF, particularly on the molecular process behind fixing the faulty intracellular CFTR protein that underlies the disease in order to return it to proper functioning.
When the Delta F508 variation is considered, the faulty protein in CFTR does not proceed to the correct site on the cell’s exterior, causing an obstruction in the movement of secretions into the airways. It is estimated that about 90% of people living with cystic fibrosis carry at the least one copy of the this mutation. Thus, the funding and research effort behind this particular project could bear out wide-ranging positive outcomes throughout the entire cystic fibrosis patient population.
Aside from the partnership with Proteostasis, the CFFT is putting money into a series of future treatment modalities designed to combat cystic fibrosis using a multitude of approaches, including partnering with Pfizer, Genzyme and Vertex to boost research and development of breakthrough drugs.