Repaired Stem Cells Grow New Working Liver Cells

UK scientists took stem cells made from the skin cells of patients with an inherited liver disease called alpha1-antitrypsin deficiency, used "molecular scissors" to effect a "clean" repair of the gene mutation that causes the disease, and showed, both in test tubes and in mice, that the gene worked correctly when the stem cells made new cells that were almost like liver cells. Nature reports the study, led by researchers from the Wellcome Trust Sanger Institute and the University of Cambridge, in its 12 October online issue.

The study is significant because it uses a tidy method that leaves no remnants of the repair mechanism behind, which could otherwise introduce unacceptable risks in a clinical setting: thus it is a new way of making a "clean correction" to the defect gene.

Read Full Article Here

13th October 2011

Reproduced with permission from the British Lung Foundation

Patients helped with breathing proplems

DESPITE suffering from an incurable lung condition, alpha-1 uk support group member Michael Jones dreams of walking Hadrian’s Wall.

Now, thanks to a new specialist rehabilitation group, he is   edging closer to achieving his goal next summer.

The 60-year-old, of Stainton, Carlisle, suffers from a genetic condition called Alpha-1 Antitrypsin Deficiency, which can lead to emphysema and other serious complications.

His best bet to ward off further illness is to stay fit – which is why he was referred to the new pulmonary rehabilitation course at Brampton Community Hospital.

Here he has been working with health professionals to tackle his condition and regain his independence.

“If you keep exercising and keep well you can stave off emphysema,” said Michael.

“My ambition is to complete the Hadrian’s Wall walk by the end of next summer.

Read Full Article Here

Posted - 20th Feb 2011

John Doyle 17/01/1944 – 24/01/2011

We are saddened to announce the passing of our dear friend John Doyle co-founder member of the Alpha-1 UK Support Group. John was great personal friend to many of us and also a great friend to the group. He worked tirelessly for many years to help make the lives of alphas everywhere a little better, he will be sadly missed by us all. We were all blessed to have known John and to be able to call him friend....a dear friend. Our thoughts are with Mary and the family....for John now its eternal peace, may God Bless him.

Posted - 24th Jan 2011

Cochrane study poorly designed, ignores wealth of data, does disservice to rare disease patients, says Alpha-1 Foundation

MIAMI, FL – The Alpha-1 Foundation today challenged a newly published review which questions the value of augmentation therapy for Alpha-1 Antitrypsin Deficiency (Alpha-1).

The article, “Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease” by Peter C. Gøtzsche and Helle Krogh Johansen, was published this week by the Cochrane Library.

“The Cochrane Library has been respected in the scientific community for carefully performed reviews based on solid evidence,” said Foundation President and CEO John Walsh.

“But this report is so flawed in its methodology that it may threaten the reputation of the Cochrane Library. The report does a disservice to rare disease patients everywhere. We hope that therapies for other rare conditions won’t become victims of the same poorly designed analysis.”

Robert Stockley, MD, Director of Research and Development at Queen Elizabeth Hospital, Birmingham, UK, had this critique:

“This conclusion was based on retrospective analysis of published data from only two small pilot placebo-controlled studies that were not powered to evaluate the effectiveness of augmentation therapy. This flies in the face of carefully crafted guidelines from the American Thoracic Society, the European Respiratory Society, the American College of Chest Physicians, and the American Association for Respiratory Care – all prestigious organizations that recommend augmentation therapy for the treatment of patients with lung disease due to Alpha-1. The guidelines are based on the totality of the evidence, scientific understanding of the disease, correcting the biochemical defect, and a wealth of observational studies.”

Danish researcher Asger Dirksen, MD, originally listed as a co-author of the Gøtzsche review, had his name removed before publication.

Dirksen, who was the lead author of both augmentation studies cited by Gøtzsche, said today:

“After seeing the first draft I realized that our points of view were so far apart that collaboration with Peter Gøtzsche and his wife (Helle Krogh Johansen) would not be possible.”

Full Article including Complete Press Release

Grifols, Talecris press release on planned acquisition

BARCELONA, Spain and RESEARCH TRIANGLE PARK, NC—Grifols a global healthcare company and leading producer of plasma protein therapies, and Talecris, a US-based biotherapeutics products company, today announced that they have signed a definitive agreement through which Grifols will acquire Talecris for a combination of cash and newly-issued Grifols non-voting shares having an aggregate value today of approximately $3.4 billion (euro 2.8 billion), creating a global leader of life-saving and life enhancing plasma protein therapeutics.

The combination of Grifols and Talecris will create a vertically integrated and diversified international plasma protein therapies company, bringing together complementary geographic footprints and products, as well as increased manufacturing scale. Grifols’ leading global footprint will benefit from Talecris’ strong presence in the United States and Canada. Grifols’ available manufacturing capacity will enable Talecris to increase production in the near term. As a result, the combined company will be better able to meet the needs of more patients with under-diagnosed disease states around the world.

Full Article

Talecris releases letter to Alpha-1 community outlining research strategy, including recombinant alpha-1 protein Dear Alpha-1 Community:

I am writing to update the community on Talecris’ Alpha-1 research program. The relationship we have with the Alpha-1 community is important to us and we want to keep you informed of several changes as we prioritize our research program. We are in the final stages of planning studies with our intravenous, plasma-derived alpha-1 proteinase inhibitor that will evaluate safety and efficacy in a large patient population. We plan to evaluate a higher dose in these studies, as we know this is an important issue for the community. These studies are a commitment to regulatory authorities, but are also based on our commitment to furthering scientific knowledge in Alpha-1. Talecris is also investing in a new, recombinant technology for producing alpha-1 antitrypsin protein from cells in culture, as opposed to human plasma. While still several years from being considered for human studies, we plan to evaluate the protein produced by these cells for both intravenous and inhaled forms of therapy. Because of the above commitments, which include studies that are required by the regulatory authorities, we have decided not to pursue studies evaluating inhaled, plasma-derived alpha-1 antitrypsin. At this time, Talecris has decided to focus on the research programs described above. We hope you agree that the research path we have chosen is the best one for Talecris and the Alpha-1 community. We continue to increase our investment in the Alpha-1 and respiratory therapeutic areas. With your ongoing support, we will continue to advance the science and therapeutic options for Alpha-1.

Sincerely,

Lawrence SternChairman and CEOTalecris Biotherapeutics

Alpha-1 UK Support Group receives generous donation from Patron

This week we can share some very exciting news, we are extremely fortunate to have received a more than generous donation from our Patron Professor R. A. Stockley and the ADAPT Project.

On Tuesday April 13th 2010, the Committee were delighted to accept a substantial donation on behalf of all our members. This amount of money will be used to aid alphas in practical ways, and also to publish information in order to promote understanding of the condition throughout the wider alpha community.

10K run in memory of my brother - Kelvin Buck

A WOMAN from Beverley is preparing for a 10K challenge to raise money for a charity close to her heart.

Karen O'Connor, 54, will take part in Jane Tomlinson's Hull 10K in memory of her brother, Kelvin Buck, 52, of Beverley, who died in October last year.

Kelvin, who had chest problems, was diagnosed with alpha-1 antitrypsin deficiency, an inherited disorder that can cause lung and liver disease, in 1993.

He underwent a double lung transplant at Newcastle's Freeman Hospital in April last year.

Although the operation was initially successfully, Kelvin then suffered chronic rejection. Click here to read more

Campaign targets Notts lung disease hotspots

This is a campaign that one of our members Joe Lyons has been involved with, keep up the good work Joe: -

HEALTH experts are targeting the areas of Notts worst affected by lung disease as part of a new campaign. Love Your Lungs is being launched today in the county to tackle its high rates of chronic obstructive pulmonary disease (COPD). It is the fifth biggest killer in the UK and includes debilitating lung diseases such as chronic bronchitis and emphysema. New research by Nottingham's business information and credit data giant, Experian, has revealed the areas where risk of COPD is highest. It shows the majority are in north Notts – though there are also hotspots in Arnold and Gamston. Click Here to read more.

Kamada Announces Enrollment Of First Patients In Its Pivotal Study For Inhaled AAT In Europe

Kamada, a bio-pharmaceutical company engaged in the development, manufacturing and marketing of specialty life-saving therapeutics, announced today that it has enrolled the first patient into its pivotal clinical trial with its new breakthrough compound of inhaled alpha-1 antitrypsin (AAT) delivered by an Investigational eFlow Nebulizer System (PARI Pharma GmbH), in patients with alpha-1 antitrypsin deficiency. Click Here to read more.

Oxygen in aeroplanes should be free as air

BY LAURA HANNAM ( Milton Keynes News )

A man who suffers from lung disease is campaigning to stop airlines charging passengers who need to use oxygen when travelling.
John Mugford, 58, from Emerson Valley, has enlisted the help of local
MP Dr Phyllis Starkey to petition airline companies to stop preventing
passengers from bringing their own oxygen cylinders on to planes and
charging hundreds of pounds extra for them to use the oxygen that the
airline provides. To read full story Click Here