MECFA Blog

News & Articles

Antibiotic treatment for Stenotrophomonas maltophilia in people with cystic fibrosis.

Stenotrophomonas maltophilia is one of the most common emerging multi-drug resistant organisms found in the lungs of people with cystic fibrosis and its prevalence is increasing. Chronic infection with Stenotrophomonas maltophilia has recently been shown to be an independent predictor of pulmonary exacerbation requiring hospitalization and antibiotics. However, the role of antibiotic treatment of Stenotrophomonas maltophilia infection in people with cystic fibrosis is still unclear. This is an update of a previously published review. The objective of our review is to assess the effectiveness of antibiotic treatment for Stenotrophomonas maltophilia in people with cystic fibrosis. The primary

Claire’s Place Foundation Launches COVID-19 Emergency Fund for People with Cystic Fibrosis

Claire’s Place Foundation medical expert recommends all with cystic fibrosis self-quarantine immediately Claire’s Place Foundation, a non-profit organization providing support to children and families affected by cystic fibrosis (CF), has created a COVID-19 Emergency Fund to support the cystic fibrosis community during the COVID-19 outbreak. The fund will help the CF community during the COVID-19 crisis with grocery cards, supplies and access to medications. “Our Board Member Richard K. Mathis, M.D., who has been caring for CF patients for over forty years, recommends all patients with CF institute a voluntary self-quarantine period for at least 14 days in the home immediately,” said Claire’

COVID-19: NGO Commends Nana Addo For Suspending Foreign Travels For Gov’t Officials

The Management and Board of Community Focus Foundation Ghana (CFF-GH) has applauded the President of Ghana, H.E Nana Addo Dankwa Akufo-Addo for suspending foreign travels of government officials amidst the outbreak of the Coronavirus (COVID-19). COVID-19, since it was first recorded in China in January 2020, has gone on to spread to over 80 countries across the various continents of the world. While scientist continues to work on its vaccine, the deadly disease has affected millions and has killed thousands. On Thursday, March 12, 2020, the Ministry of Health through a press release announced that Ghana has recorded its first two cases of the Coronavirus which has been declared as a global p

New CRISPR Tool Fixes CFTR Mutations in CF Patients’ Stem Cells, Study Finds

A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of cystic fibrosis (CF) — in stem cells from CF patients, a study shows. The new approach has the ability to correct mutations without the need to excise the affected region, the researchers said. The study, “CRISPR-Based Adenine Editors Correct Nonsense Mutations in a Cystic Fibrosis Organoid Biobank,” was published in the journal Cell Stem Cell. CRISPR-Cas9, a system used by microbes to defend themselves against invading viruses, has been adapted and is currently at the forefront of many therapeutic approaches for human diseases. The technique allows for the correction of g

New drugs aim to disarm the immune system’s ‘atomic bomb’ cells

Seeking a new treatment for people who have dangerous blockages in their coronary arteries, doctors in London are trying to disarm the body’s own defenders. The 90 patients in the study receive the usual treatments for heart disease, such as small tubes called stents to prop open their narrowed arteries. But half of the patients in the phase II trial, conducted by clinical pharmacologist Albert Ferro of King’s College London and colleagues, also pop pills targeting a class of immune cells called neutrophils. Researchers think that by invading the fatty obstructions, or plaques, in clogged arteries, neutrophils make them even more dangerous. The drug is designed to steer the cells away. Inter

Inhaled Messenger RNA Treatment Fast-Tracked for Cystic Fibrosis

The Food and Drug Administration has granted Fast Track designation to MRT5005 (Translate Bio), an inhaled messenger RNA (mRNA) therapeutic being investigated for the treatment of cystic fibrosis. MRT5005 is designed to address the underlying cause of cystic fibrosis, regardless of genetic mutation, by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization. A phase 1/2 trial investigating the safety and tolerability of single and multiple escalating doses of MRT5005 is currently ongoing. Interim results from 12 patients showed that the treatment was generally well tolerated with cough and

COVID-19 in Chronic Lung Disease: What You Need to Know

Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and novel coronavirus disease (COVID-19) have been of major concern to the healthcare community at large, but particularly to clinicians who specialize in infectious disease and pulmonary and critical care medicine. We spoke with Charles Dela Cruz, MD, PhD, of Yale University School of Medicine in New Haven, Connecticut, regarding the risks posed by COVID-19 to patients living with chronic lung disease, as well as precautions both patients and healthcare providers can take to prevent the spread of the SARS-CoV-2 virus and its potential subsequent complications. Dr Dela Cruz is an associate professor in the Pulmonary, Critical Care

This new treatment helps 90% of cystic fibrosis patients breathe better

Vertex’s Trikafta was the second-fastest FDA approval in history, and could help some 27,000 patients. In October 2019, Vertex Pharmaceuticals received FDA a­p­prov­al for Trikafta, a cystic fibrosis treatment that could help 90% of patients with the deadly genetic disorder. Cystic fibrosis, which affects about 30,000 people in the U.S. (and has a life expectancy of 44 years for patients born today), is caused by mutations in the CFTR gene that lead to a defective protein being produced. That protein doesn’t allow chloride to flow in and out of cells normally (particularly those that line the lungs) and prevents water from hydrating the cellular surface, leading to a buildup of thick mucus a

CFTR Amplifiers May Be Potent CF Mono or Combo Therapy, Study Says

Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) — do so by stabilizing the template used for its production so the protein works better in cells and there’s more of it, a study into the mechanisms of amplifiers reported. Specifically, this groups of CFTR modulators work by interacting with a specific protein, PCBP1, that binds to RNA during crucial steps in protein production. These findings support nesolicaftor (PTI-428), an investigational and small molecule oral amplifier by Proteostasis Therapeutics, to treat CF either alone or in combination with other CFTR (cystic fibrosis transmembrane conductance regulator) therap

UK NICE to Announce Decision on Vertex’s Therapy Trikafta in December

The National Institute for Health and Care Excellence (NICE) in the U.K. is currently in the process of evaluating Vertex Pharmaceuticals’ triple combination therapy — known as Trikafta in the United States — for the treatment of cystic fibrosis (CF). The institute expects to deliver its recommendation on Dec. 16, 2020. NICE has the responsibility of giving advice to the National Health Services (NHS) in England on whether new therapies should be made available for physicians to prescribe. NICE’s advice is also largely followed by the health authorities in Northern Ireland and Wales. Trikafta is a next-generation triple combination therapy — elexacaftor, tezacaftor, and ivacaftor combo — des

FDA Grants Fast Track Status to Translate Bio’s RNA-Based Therapy MRT5005

The U.S. Food and Drug Administration (FDA) has granted fast track designation to MRT5005, an investigational RNA-based treatment for cystic fibrosis (CF), the therapy’s developer, Translate Bio, has announced. Fast track is given to medications that aim to treat serious or life-threatening conditions and that have the potential to fill unmet medical needs to expedite their development and regulatory approval. Clinical development programs that are granted fast track may be eligible for certain benefits, including additional meetings and communication with the FDA. And, if other criteria are met, they may also receive accelerated approval, priority review, and rolling review of a Biologics L

Vertex Announces Availability of Cystic Fibrosis Medicine KALYDECO® (ivacaftor) in New Zealand

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that KALYDECO® (ivacaftor) will be available to eligible patients with cystic fibrosis (CF) in New Zealand from March 1. Clinicians are able to apply for patient access using the exceptional circumstances framework set up by the Pharmaceutical Management Agency (PHARMAC). "Today is an important day for the CF community in New Zealand. For the first time in New Zealand CF patients will have access to a medicine that treats the underlying cause of their disease. We appreciate the engagement and collaboration from PHARMAC, and other involved parties, to bring this important medicine to patients," said Ludovic Fenaux, Senior Vice

Arch Scientist Publishes Data on the Pre-Clinical Efficacy of AB569 in Killing of Drug Resistant Bac

TORONTO, Feb. 21, 2020 (GLOBE NEWSWIRE) -- Arch Biopartners Inc., ("Arch" or the "Company") (TSXV:ARCH, OTCQB:ACHFF), announced today that Dr. Daniel Hassett's team at the University of Cincinnati (UC) College of Medicine, and its collaborators, have published details regarding the efficacy of AB569 in killing various multidrug resistant bacteria. The paper demonstrates key data on the synergistic effects of acidified sodium nitrite and EDTA (active ingredients of AB569) on destroying all bacteria tested as well as identifying bacterial properties that confer susceptibility to the drug. Details of these findings are reported in the prestigious journal of the Proceedings of the National Acad

Cystic Fibrosis And Infertility

Although, cystic fibrosis does not have any permanent,-cure, prevention can reduce the severity of complications Cystic fibrosis is a genetic disorder that affects various parts of the body, mainly the lungs, pancreas, intestines, liver, and kidneys. The disease is caused by a mutation in the gene which is responsible for affecting a protein that regulates the movement of salt in the cells. In complicated cases, individuals with cystic fibrosis may have bronchiectasis, chronic Infections, nasal polyps, hemoptysis, pneumothorax, and even infertility, especially in males. Infertility And Cystic Fibrosis (CF) Approximately 97 per cent men with cystic fibrosis are infertile. However, many medica

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