Groundbreaking research has found that the majority of people with cystic fibrosis can be treated successfully.
Researchers at Queens University in Belfast and at the Cystic Fibrosis Unit at St Vincents Hospital Dublin, have identified how a combination of three drugs, known as triple therapy, can tackle the underlying cause of the incurable disease.
The drug, Trikaftatm, targets the root cause of cystic fibrosis (CF), a genetic condition that clogs up the lungs and digestive system, making breathing difficult and often resulting in an early death for those affected.
CF is an inherited chronic disease that primarily affects the lungs and digestive system of about 1,300 children and adults in Ireland and 70,000 worldwide.
That figure, according to Philip Watt of Cystic Fibrosis Ireland, is set to increase by 75% in adults and 25% in children in this country according to ongoing studies by 2025.
This new drug Trikaftatm will benefit 90% of sufferers worldwide, lung function is expected to increase dramatically and will result in a 60% decrease in hospitalisation. This is a hugely welcome advancement. Many of the trials by the Queens University researchers were carried out at several centres in Ireland including St Vincents Hospital Dublin.
Dr Francis Collins, an American researcher who discovered the CF gene in 1989 has already said this is the breakthrough in research he has been waiting for. Many of the trials prior to this did not achieve as much as this study has.
Dr Collins who is the director of the National Institutes of Health writing in The New England Journal of Medicine and the highly respected Lancet medical magazine over the weekend, said these findings indicate that it may soon be possible to offer safe and effective molecularly targeted therapies to 90% of persons with cystic fibrosis.
He said that this should be a cause for major celebration and the best day ever for all of us traveling down this long road together will be the day when the more than 70,000 persons with cystic fibrosis worldwide do not need to take drug therapy at all and there finally is a permanent cure for cystic fibrosis that works for everyone.
Mr Watt added: The Food and Drugs Administration in the US has approved this drug five months ahead of time and now the European Medicines Agency is reviewing it. As a result we would expect this drug to be on the market here by the Summer of next year to those over 12-years-of-age. We would be hopeful that will include children aged as young as two in the future.
The numbers of adults and children increasing in Western Europe is down to advancements in related drugs and quality of services.
Talks and negotiations with the Health Service Executive on this new announcement will, Im sure, commence as a pipeline deal on future drug advancements was agreed with them in 2017 when Orkambi was finally approved. So far we are not privy to the potential costs of this new drug.
However, it is estimated that the drug may cost around $311,000 or 279,000 annually by Vertex, a pharmaceutical company that produces other CF drugs.
Ireland has the highest incidence of CF in the world with one in 19 Irish people being said to carry one copy of the altered gene, with three times the rate of the United States and the rest of the EU.
The organisation has pointed out that it is important to have a network of centres of expertise in dealing with disease.
A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections; and obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.
Researchers believe it is possible to help control the symptoms and delay complications to make the condition easier to live with.
Dr Damian Downey, is a Clinical Senior Lecturer at the Wellcome-Wolfson Institute for Experimental Medicine, Queens University Belfast and co-author on the worldwide trial, set-up to assess the safety and efficacy of a new triple-drug combination called Trikaftatm in patients with CF aged 12 years and older.
The study involved a four-week, randomised, active-controlled trial in 107 patients who had two copies of the (F508del) most common gene mutation.
Dr Downey said: The trial was a success in demonstrating that this drug combination can potentially treat up to 90% of people with CF by addressing the underlying cause of their disease.
This new triple therapy has the potential to transform the lives of people with CF. It results in a significant improvement in lung function and quality of life and also reduces the frequency of chest infections. This treatment will likely alter the future of CF care.
See more here:
Research finds majority of people with cystic fibrosis can be treated successfully with three-drug combo - BreakingNews.ie
- Gene Therapy for Genetic Disease: The Long and Winding Road [Last Updated On: August 17th, 2024] [Originally Added On: October 10th, 2011]
- Gene Doping: Super Athletes in 2008 Beijing Olympics - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 14th, 2011]
- The 3 Rs of DNA: Molecules to Medicine - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 15th, 2011]
- BIT 3.1 Entrez Gene - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 15th, 2011]
- Professor Ian Everall - Gene expression in schizophrenia - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 16th, 2011]
- Gene Therapy Promising Treatment (Part 2 of 4) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 16th, 2011]
- Cancer Research @ NC State's Centennial Biomedical Campus - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 16th, 2011]
- Gene Therapy Promising Treatment (Part 1 of 1) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 19th, 2011]
- HiTech_U3L3_Biotech in Medicine - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 19th, 2011]
- Sri Sathya Sai Medical Camps Worldwide - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 20th, 2011]
- Dr. Joseph M. DeSimone: 2008 Winner, $500000 Lemelson-MIT Prize - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 21st, 2011]
- Non-Hodgkin's Lymphoma Cancer Survivor Testimony - (Sanoviv Medical Institute) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 22nd, 2011]
- Vaccines: Medicine for the Healthy - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 24th, 2011]
- For Your Health - Health Care Informatics - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 24th, 2011]
- A Century of Stem Cells - Johns Hopkins Medicine - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 24th, 2011]
- Functional Repair of Human Donor Lung by IL-10 Gene Therapy (EX VIVO Lung) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 25th, 2011]
- Science Talk: Vaccine Made With Synthetic Gene Protects Against Deadly Pneumonia - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 25th, 2011]
- Health Matters: Family Medicine - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 26th, 2011]
- Dr. Jeffrey Myers Explains Genetic Variations in Head and Neck Cancer - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- The Emerging Network of Data for Understanding the Interactions of Genes and Drugs - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Burzynski_ Cancer Is Serious Business Part 4/8. wmv - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Pt2: Do you understand the new genetics? Maybe you don't have that gene! - Andrew Shelling - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Alien Genetic Takeover of Humanity! - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Burzynski | 2-DVD Set Extended Edition Montage Preview of New Material | Cancer Is Serious Business - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Genes, Genomes and the Future of Medicine - Richard Lifton, MD, Ph.D. - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Burzynski The Movie ~ Cancer Is Serious Business ~ High Quality - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Designing Life for Mars Colonization (Brainstorm Ep20) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Dedication Event: Santa Monica UCLA Medical Center [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- DR. Burzynski The Movie - Cancer Is Serious Business (PT.1) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- burzy - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Alien Genetic Takeover: The End of Humanity - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Burzynski_ Cancer Is Serious Business Part 1/8 .wmv - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Mike Schmidt - ECMO Patient - Penn State Hershey Medical Center - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Disrupting the HIV Virus (Brainstorm Ep24) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Diagnosis and Treatment of Hepatorenal Syndrome - Moving Targets? - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Big Bucks, Big Pharma: Marketing Disease and Pushing Drugs - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Roles of RNA in Gene Regulation - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Diatom PCR Colony Screen - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Kleanthis G. Xanthopoulos, Ph.D., CEO, Regulus, talking on the impact of microRNA medicines - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Burzynski_ Cancer Is Serious Business Part 2/8 .wmv - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Sickle Cell Anemia: Stem Cell Gene Therapy - A Patient's Perspective - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- Gene-based lung cancer treatment video - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- HIV Resistant Genes...Rhesus Negative, Excess PK [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- Futures in Biotech 83: Bioinformatics: Essential Gene names Skewed in a Network of Blame - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- Keeping Blood Young, Fighting Sickle Cells (Brainstorm Ep28 - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 31st, 2011]
- News@Northwestern - NUGENE - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 31st, 2011]
- UK Medical Student Mary Burchett's Research is Personal - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 31st, 2011]
- Cannabis 4 Crohn's: 2 Years As Medical Marijuana Patient, Dr Mind Bender [Last Updated On: August 17th, 2024] [Originally Added On: November 8th, 2011]
- Medical Haptical Robot for the Eye - Eye-Rhas, - TU Eindhoven - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 8th, 2011]
- The Ghost in Your Genes - Horizon - BBC - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 9th, 2011]
- The Ghost in Your Genes - BBC Horizon - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 13th, 2011]
- Burzynski: Cancer Is Serious Business (Trailer) - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 16th, 2011]
- Genes, Ancestry, and Personalized Medicine with Dr. Michael Kane - Interview Part 1.mp4 - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 25th, 2011]
- The Suicide Gene - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 29th, 2011]
- Ard Louis on Dawkins and Selfish Genes - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 30th, 2011]
- TEDxLex - Dr. Alicia L. Bertone - Gene Jockeying for a Win in Regenerative Medicine.mp4 - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 3rd, 2011]
- Introduction to Gene Network - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 7th, 2011]
- The demise of a dogma. The deaf somatic gene listens - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 7th, 2011]
- Future of health care, hospitals, medicine and wellbeing - by Dr Patrick Dixon - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 8th, 2011]
- Electro-Medicine : Biological Physics : Neurological Research Advancements - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 12th, 2011]
- Haemophilia gene therapy breakthrough - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 15th, 2011]
- Duke researchers: Gene discovery explains how fruit flies retreat from heat - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 16th, 2011]
- Focus on Nikolaus Rajewsky - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 18th, 2011]
- Deadly Monopolies Medical Ethicist Harriet Washington on How Firms are Taking Over Life Itself - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 23rd, 2011]
- Open fetal surgery: past, present and future - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 23rd, 2011]
- The Human Genome and Individualized Medicine - David Valle, MD - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 14th, 2012]
- Environmental Contributors to Autism Spectrum Disorders - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 20th, 2012]
- When Cancer Runs in the Family - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 20th, 2012]
- Genome Enabled Electronic Medical Record (GenE EMR) - William Knaus - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 22nd, 2012]
- Nuvigil withdrawal - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 23rd, 2012]
- Cancer Cure Documentary - Dr. Burzynski Antineoplaston Therapy - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 26th, 2012]
- Next Generation Cancer Gene Sequencing for Clinically Actionable Mutations - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 26th, 2012]
- Fibrocell Science, Inc. Announces Recognition for LAVIV™ (azficel-T) at The 2012 Cell & Gene Therapy Forum, Washington ... [Last Updated On: August 17th, 2024] [Originally Added On: January 31st, 2012]
- Gene Mutation Linked to Inappropriate Lipid Buildup in Liver [Last Updated On: August 17th, 2024] [Originally Added On: February 1st, 2012]
- 'Goldilocks' gene could determine best treatment for tuberculosis patients [Last Updated On: August 17th, 2024] [Originally Added On: February 3rd, 2012]
- James Wilson, MD, Ph.D., on Gene Therapy as a Disruptive Technology - Video [Last Updated On: August 17th, 2024] [Originally Added On: February 3rd, 2012]
- 'Goldilocks' gene used to find drug treatment that is 'just right' for TB patients [Last Updated On: August 17th, 2024] [Originally Added On: February 4th, 2012]
- Gene therapy, a totally Italian success - Video [Last Updated On: August 17th, 2024] [Originally Added On: February 4th, 2012]
- Electro-Medicine : Neurons function revealed - Video [Last Updated On: August 17th, 2024] [Originally Added On: February 4th, 2012]
- Gene mutation discovery sparks hope for effective endometriosis screening [Last Updated On: August 17th, 2024] [Originally Added On: February 7th, 2012]