MECFA Blog

News & Articles

Proteostasis Therapeutics Provides Clinical Enrollment Update

BOSTON, Dec. 20, 2018 /PRNewswire/ -- Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today provided a clinical enrollment update. The Company, while remaining blinded to data from the first cohort, now expects to report complete efficacy and safety data from the Phase 1, randomized, double-blind, placebo-controlled study of its proprietary combination therapy triplet (PTI-808, PTI-801, and PTI-428) and from the high-dose doublet study in subjects with CF in the first quarter of 2019. The C

Aridis Enrolling CF Patients to Test AR-501 in Chronic Lung Infections

Aridis Pharmaceuticals has enrolled the first healthy participant in its Phase 1/2a clinical trial to evaluate the antibacterial potential of its investigational candidate, AR-501 (gallium citrate), against chronic lung infections in patients with cystic fibrosis (CF). The study (NCT03669614) is expected to enroll approximately 48 healthy adult volunteers and 48 adult CF patients with chronic lung infections across 15 sites in the United States. Participants will be randomized to receive one of three doses of AR-501, or a placebo, self-administered once a week using a hand-held nebulizer. The company expects to announce results from Phase 1 during the fourth quarter of 2019, and from Phase 2

Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests

The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically measures the sweating dependent on CFTR, called C-sweat rate, than using classic sweat tests or lung function measurements, a study reports. This kind of test was sensitive enough to reveal that Kalydeco also amplifies CFTR function in healthy individuals, an effect that standard salt measurements in sweat failed to detect. The study, “Sweat rate analysis of ivacaftor potentiation of CFTR in non-CF adults,” was published in the journal Scientific Reports. Hundreds of different mutations in the CFTR gene have been found to cause cystic fibrosis (CF). Some mutations

Phase 3 Trial of RDEB Therapy EB-101 to Start in 2019, Abeona Therapeutics Says

Abeona Therapeutics plans to start a Phase 3 trial in 2019 testing the cell therapy candidate EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB). The multi-center, randomized study, named VITAL, will compare treatment with EB-101 to untreated wounds in the same patient. In total, 10-15 patients are expected to enroll. VITAL’s primary outcome will be the proportion of treated wounds showing more than 50% healing at three months. Secondary goals will include an investigator global assessment of wounds, and changes in pain and itch compared to the treatment’s start (baseline). Patients with RDEB have mutations in the COL7A1 gene, which causes no functional type VII coll

People with Rare Diseases Are Humans, Not Commodities

If you keep up with CF research, you may have seen the news about Phase 3 of the triple combination Vertex therapies. In these results, patients had a robust lung function boost after trialing a triple combination therapy treatment. More importantly, these results were tested in a broad population of people with CF, meaning the upcoming therapy may be accessible to the largest population of people with CF yet. While these results are worthy of celebration, there’s another element to this news to consider — business entanglements. Since CF is considered a rare, orphan disease, its research money is delegated differently than diseases that affect millions of people. There are financial incenti

Triple Combo Treatment Improves Lung Function in Patients with Cystic Fibrosis

An investigational triple combination treatment regimen for cystic fibrosis (CF) improved patients’ lung function in 2 phase 3 studies, according to Vertex. Based on the data, the regimen, which combines the next-generation corrector VX-659, tezacaftor, and ivacaftor, met the primary endpoint of improvement in lung function (percent predicted forced expiratory volume in 1 second [ppFEV1]) in both studies. In 1 study, the triple combination was evaluated in patients 12 years of age and older with 1 F508del mutation and 1 minimal function mutation for 24 weeks. The study randomized 385 patients, with 382 patients administered at least 1 dose of either VX-659 triple combination regimen

NuvoAir Redefines Home Monitoring for Cystic Fibrosis Patients

STOCKHOLM--(BUSINESS WIRE)--Dec 4, 2018--THE FINDINGS from a pilot study into a new way of caring for those with cystic fibrosis (CF) have just been released. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181203005846/en/ An innovative partnership between NuvoAir and London’s Royal Brompton Hospital was launched last year. It allowed patients taking part in the study the freedom to test themselves at home with the simple-to-use spirometer. This hand-held device, when connected to software via Aria - an app exclusively developed by NuvoAir - can accurately measure the state of the patient’s lungs and predict worsening. Once this

Mucus Protects Lungs by Forming Layers, Preventing Worsening of Lung Diseases, Studies Reveal

Two new studies highlight the important role of mucus in cleaning and protecting the lungs, and how its accumulation and impaired clearance can contribute to the worsening of lung diseases such as chronic obstructive pulmonary disease (COPD). In the studies, researchers show how mucus in the lungs forms a protective layer to trap and prevent bacteria from getting in contact with the lung epithelium. They also emphasize that this mucus eventually needs to be cleared from the body — likely the reason why, after a cold, people develop a cough to expel mucus. Failure to get rid of this mucus means that it becomes stagnant allowing bacteria trapped in the mucus to grow, which can promote the wors

Study Reveals How Cystic Fibrosis Affects Young Women’s Sexual Health

Girls with cystic fibrosis (CF) reach puberty six to eight months later than healthy girls, about 16 percent of young females with CF encounter problems during sex, and 78 percent of these women would like to have children someday. These are among the key findings of an unprecedented survey conducted in 2015-16, and made public for the first time by its author, Traci M. Kazmerski, MD, an assistant professor of pediatrics at UPMC Children’s Hospital of Pittsburgh. The report — “Sexual and reproductive health care utilization and preferences reported by young women with cystic fibrosis” — is based on a survey of 188 females with CF ages 15 to 24 years old. The study took place at five sites: U

PARI Devices used in SHIP Study of Hypertonic Saline in Preschoolers with Cystic Fibrosis

MIDLOTHIAN, Va., Nov. 29, 2018 /PRNewswire/ -- A recent study in preschoolers with cystic fibrosis (CF) conducted by Cystic Fibrosis Foundation Therapeutics used PARI's LC Sprint Jr. nebulizer, Vios PRO compressor, and PARI's 7% hypertonic saline. The study called SHIP (Saline Hypertonic in Preschoolers) showed that PARI's 7% hypertonic saline inhaled twice daily using the PARI LC Sprint Jr. and the Vios PRO compressor resulted in a sustained and significant improvement in a lung function test called the lung clearance index over 48 weeks. "PARI was the first company in the USA to receive clearance to market by the FDA for hypertonic saline 7% inhalation solution in unit-dose form, which inc

European Union Expands Kalydeco’s Approval to Certain CF Patients Ages 1-2

Kalydeco (ivacaftor) by Vertex Pharmaceuticals has been approved in the European Union for children ages 1-2 with cystic fibrosis (CF) who carry at least one of nine mutations in the CFTR gene — G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R — that prevent the CFTR chloride channel from working properly. “For the first time, EU physicians can now treat the underlying cause of CF earlier than ever, helping to improve clinical outcomes in children as young as 12 months,” Reshma Kewalramani, MD, executive vice president and chief medical officer at Vertex, said in a press release. The approval was based on results from the ongoing Phase 3 ARRIVAL (NCT02725567) trial that e

Cystic Fibrosis Patients Exposed to Higher Risks of Liver Cancer

Researchers from Colorado, in the United States, shared the results of a study that showed that patients with cystic fibrosis would be at higher risk of developing liver cancer. The Children’s Hospital in Aurora, Colorado, USA, has allowed researchers to conduct research that revealed the link between cystic fibrosis and cirrhosis and liver cancer. According to the study’s report cited by EurekAlert, the research aimed to identify factors related to an increased risk of developing advanced liver disease when the patient already had cystic fibrosis. Mucoviscidosis is a rare genetic disease that mainly affects the respiratory tract and digestive system. As the scientists reported, liver cancer

New Mucus Study Could Hold the Key to Clearing Your Phlegm

Mucus, you fickle fiend—usually, you’re there keeping us safe from whatever nasties sneak inside of us. But then you turn, becoming a source of discomfort, canceled plans, and overall malaise. How I wish we knew you better. I’m far from the only one with this opinion, and a team of scientists has now published a study analyzing the chemistry of mucus with the hope of finding better ways to clear it. But while their results could lead to therapies for common cold-induced discomfort, they could more importantly help those with severe diseases like chronic obstructive pulmonary disease (COPD) and cystic fibrosis. “Mucus is a very good thing,” study author Brian Button from the University of Nor

Featured Posts
Recent Posts
Archive
Search By Tags

Support Zahra's run for Palestine CF patients.

  • Facebook Clean
  • Twitter Clean
  • Instagram - Grey Circle
  • YouTube - Grey Circle
  • LinkedIn Clean