Telethon Kids Institute Spin-off Receives Sizable Investment to Test Cystic Fibrosis Treatment

Respirion Pharmaceuticals, a spin-off of the Telethon Kids Institute, will receive an investment of over $20 million from the Australian government’s Medical Research Commercialization Fund (MRCF)  as well as the US Cystic Fibrosis Foundation. The spin-off company has developed a therapy that utilizes a new inhaled drug to target antibiotic-resistant infections that wear down the lungs and  hence, restrict breathing.  Utilizing the antibiotic tobramycin with an additive that helps break down bacterial protective biofilms within the lungs.

MRCF has committed up to 20M Australian dollars in stages in collaboration with Telethon Kids Institute.  The Cystic Fibrosis Foundation (CF) in the U.S. will commit up to $3 million. The new investigational therapy was tested at Western Australian Department of Health.  An initial clinical trial evidenced an average 16% improvement in the lung function of patients.

The Telethon Kids Institute press release noted that Respirion will coordinate clinical trials with CF Foundation Therapeutics Development Network—the largest CF clinical trial network in the world. The CF Foundation is the world’s leader in the pursuit of  the CF cure—it has contributed to nearly every drug on the market today.

Who is the Telethon Kids Institute?

Telethon Kids Institute  is an Australian medical research institute focused on the prevention of pediatric disease and the development of improved treatments to improve the health and wellbeing of children.

What is Cystic Fibrosis?

It is a genetic disorder that affects mostly the lungs, but also the pancreasliverkidneys, and intestine. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. Other signs and symptoms may include sinus infectionspoor growthfatty stoolclubbing of the fingers and toes, and infertility in most males. Different people may have different degrees of symptoms.

CF is inherited in an autosomal recessive manner. It is caused by the presence of mutations in both copies of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Those with a single working copy are carriers and otherwise mostly normal. CFTR is involved in production of sweat, digestive fluids, and mucus. When the CFTR is not functional, secretions which are usually thin instead become thick. The condition is diagnosed by a sweat test and genetic testing.] Screening of infants at birth takes place in some areas of the world.

There is no known cure for cystic fibrosis. Lung infections are treated with antibiotics which may be given intravenously, inhaled, or by mouth. Sometimes, the antibiotic azithromycin is used long term. Inhaled hypertonic saline and salbutamol may also be useful. Lung transplantation may be an option if lung function continues to worsen.[Pancreatic enzyme replacement and fat-soluble vitamin supplementation are important, in the young. Airway clearance techniques such as chest physiotherapy have some short-term benefit, but long-term effects are unclear. The average life expectancy is between 42 and 50 years in the developed world. Lung problems cause death in 80% of people with cystic fibrosis.

According to the Telethon Kids Institute press release “More than 70,000 people worldwide suffer from CF with approximately 1000 new cases of CF being diagnosed each year in the U.S. alone. Recent breakthroughs in CF therapy have extended the median survival age from around six years in the 1950s to more than 40 years today. However, at this stage there is no cure and many lives are still cut short by the disease due to persistent lung infections and loss of lung function.”

Dr. Barry Clements, Respirion Founder was quoted “…persistent lung infections and the inevitable decline in lung function remain urgent unmet needs in Cystic Fibrosis.” Dr. Clements served patients at Perth Children’s Hospital for over 30 years.