Important Message from our Trustees

Dear Alpha-1 Patients,

We have repeatedly noticed that there still appears to exist confusion about the status of ADAPT and the available options for Alphas to receive specialist care in the NHS. We would like to clarify the situation.

ADAPT has always been a largely industry-funded research programme with no formal links to the NHS. There is no NHS process which allows the “transfer” of patients from a research programme, eg. ADAPT, to an NHS service, eg. specialist Alpha-1 clinics.

It is important to understand that every patient who has been attending ADAPT or any other research programme is free to choose where they want to attend specialist Alpha-1 NHS clinics – an automatic ‘transfer’ to a particular NHS service by a local doctor without prior discussion with the patient about their preferred choice is not appropriate.

You and your local doctor should be aware of the available choice that now exists in the NHS for Alpha-1 patients:  specialist multidisciplinary NHS clinics are operational, for example, at Addenbrooke’s Hospital in Cambridge, at the Royal Free Hospital in London and, as part of the new West Midlands Specialist NHS Alpha-1 Service, at the University Hospital in Coventry and at the University Hospital in Birmingham. All these centres have the necessary expertise and experience to provide optimal care for Alphas.

Please read the front page article of our latest Newsletter for detailed information about the differences between attending research programmes and NHS clinics, and about some (but not all) of the available specialist NHS Alpha-1 clinics in England and how to get referred there (include link to the newsletter).

It is important that you are fully aware of the available options in order to make an informed choice about your care!

If you have any further questions, please feel free to contact us via Facebook the Yahoo Group or

Board of Trustees

Alpha-1 UK Support Group

Published: 27/09/2015

West Midlands Specialist NHS Alpha-1 Service Now Operational at Two Centres

Further to our announcement in April of the establishment of a multidisciplinary specialist NHS clinics for Alpha-1 patients in the West Midlands, we are pleased to announce that this service is now offered at two centres: Alpha-1 clinics at University Hospital Coventry are run by Dr David Parr and have been operational since May and, as of this week, Dr Alice Turner runs Alpha-1 clinics at the University Hospital Birmingham.

Both clinics are available to patients from across England, and patients can get referred to the clinic of their choice by their GP or local specialist.

The following links provide information and details of how to get referred and make an appointment:

University Hospitals Coventry and Warwickshire
Further information – 02476 967697

University Hospital Birmingham
Further information – 0121 371 3885

Published: 18/09/2015

CSL Behring Receives Marketing Authorization for Respreeza® in Europe

Respreeza® is a maintenance treatment for severe Alpha-1 Antitrypsin Deficiency patients and has been shown to slow the progression of emphysema

CSL Behring announced today that the European Commission (EC) has granted marketing authorization in all European Union (EU) member states for Respreeza® to treat patients with Alpha-1 antitrypsin deficiency (AATD). Respreeza®, a highly purified Alpha-1 protein derived from human plasma, is the only Alpha-1 proteinase inhibitor that has been proven in a prospective double blind, placebo controlled trial (the RAPID study) to significantly reduce the loss of lung tissue, slowing the progression of emphysema due to AATD. AATD is a hereditary condition marked by a lack of the Alpha-1 antitrypsin protein, whose main function is to protect the lungs from inflammation.

“AATD is a potentially debilitating disease and many affected individuals suffer from serious lung disease,” said Professor Helmut Teschler, MD, Director of the West German Lung Centre at the University of Essen. “With the approval of Respreeza®, healthcare professionals can now provide patients with severe AATD in Europe with a next generation Alpha1-proteinase inhibitor (A1-PI) that provided additional evidence that this augmentation therapy can slow the accelerated loss of lung tissue.”

Frank Willersinn, M.D., Alpha-1 Global Steering Committee Chair and patient representative in Europe added, “We are so glad that Respreeza® has been approved by the European Medicines Agency. We commend CSL Behring for their long-lasting commitment to the Alpha-1 community, now bringing their established Alpha 1-antitrypsin product to Europe, allowing it to be a cornerstone for treatment of AATD patients in the near future.”

CSL Behring has marketed A1-PI for 12 years as Zemaira® in the United States, where it is indicated for chronic augmentation and maintenance therapy in adults with A1-PI deficiency and clinical evidence of emphysema. In US, Zemaira® is contraindicated in patients with a history of severe systemic reaction to the product or to A1-PI protein, including anaphylaxis. Due to the risk of severe hypersensitivity, Zemaira® is also contraindicated in immunoglobulin A (IgA)-deficient patients with antibodies against IgA.

About Respreeza®

In EU, Respreeza® is indicated for maintenance treatment, and to slow the progression of emphysema in adults with documented severe A1-PI deficiency (e.g. genotypes PiZZ, PiZ(null), Pi(null,null), PiSZ). Patients are to be under optimal pharmacologic and non-pharmacologic treatment and show evidence of progressive lung disease (e.g. lower forced expiratory volume per second (FEV1) predicted, impaired walking capacity or increased number of exacerbations) as evaluated by a healthcare professional experienced in the treatment of A1-PI deficiency. Respreeza® is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients and IgA deficient patients with known antibodies against IgA, due to the risk of severe hypersensitivity and anaphylactic reactions.

About the RAPID Study

The RAPID study (Randomized, Placebo-controlled Trial of Augmentation Therapy in Alpha-1 Proteinase Inhibitor Deficiency) enrolled 180 severely deficient alpha-1 patients in the U.S. and Europe who were treated for 2 years with Respreeza® or Placebo. The study, published in Lancet in June 2015, showed a significant reduction in the rate of lung tissue loss as measured by CT scan lung density. In those patients who continued into the extension study, where all subjects received Respreeza®, the benefit was maintained for another 2 years.

Full Story: CSL Behring 25/08/2015

Step Closer to First UK Drug License Augmentation Therapy

One step closer to the first UK drug license for augmentation therapy for patients with severe alpha-1 antitrypsin deficiency

CSL Behring has announced today that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting a market authorisation to CSL’s alpha-1 antitrypsin augmentation therapy product Respreeza®.

Receiving a positive CHMP opinion (See EMA Website) marks an important milestone in the process of Respreeza® obtaining a market authorisation (= drug license) in all EU Member States, including the UK. The CHMP positive opinion will be transmitted to the European Commission (EC) to start the EC decision-making process on granting a centralised European drug license for Respreeza®, which we hope to be concluded later this year.

Lutz Bonacker, Senior Vice President & General Manager Commercial Operations, Europe, CSL Behring said: “CSL Behring continues to make strides towards fulfilling our promise to improve the lives of our patients. This positive opinion from CHMP brings us closer to providing Respreeza® as a new treatment option to the AATD community in Europe.”

However, once a drug license is granted by the EC, access to Respreeza® to UK patients will be subject to reimbursement of the drug by the NHS. As use of Respreeza® will be indicated in adults with documented severe alpha-1 antitrypsin deficiency and evidence of progressive lung disease as evaluated by a healthcare professional experienced in the treatment of Alpha-1, it is important that patients gain access to the care provided by a physician with sufficient experience in the field of Alpha-1, e.g. at the recently established West Midlands Alpha-1 Services

Published: 29/06/2015

Trial Results Alpha-1 Augmentation Therapy

The Lancet publishes RAPID trial results showing effectiveness of Alpha-1 augmentation therapy.

The Alpha-1 Foundation on Thursday congratulated the investigators and sponsor of the RAPID Trial, demonstrating the effectiveness of augmentation therapy in slowing emphysema due to Alpha-1 Antitrypsin Deficiency, on the publication of the trial in The Lancet, one of the world’s oldest and most prestigious medical journals.

The Lancet article was posted online this week.

When the results were first announced at the international conference of the American Thoracic Society (ATS) in 2013, lead author Kenneth Chapman, MD, director of the Asthma and Airway Centre of the University Health Network in Toronto, Canada, called the trial “the most rigorous evidence to date that augmentation therapy slows the progression of emphysema in patients with Alpha-1 Antitrypsin Deficiency. The effect of A1-PI seen in this trial was both clinically and statistically significant, finally confirming its benefit in preventing the loss of lung tissue in patients with this potentially debilitating disease.”

The Alpha-1 Foundation hailed the trial results and The Lancet publication. “We congratulate CSL on this landmark clinical trial, which provides the strongest evidence yet that augmentation therapy preserves the lung tissue of individuals with Alpha-1-related lung disease,” said John Walsh, Foundation president and CEO. “We hope these results will support Alpha-1 communities around the world in their efforts to win access to therapy.”

Walsh said, “The Foundation has communicated its support for acceptance of the RAPID data as proof of efficacy of augmentation therapy to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (the European equivalent of the FDA), and has continued a dialogue with both agencies to urge acceptance of these findings.”

CSL Behring sponsored the trial, which randomly assigned 180 Alpha-1 patients to receive either the augmentation product Zemaira or a placebo for a two-year period, followed by a two year of open-label extension study in which all subjects were offered augmentation therapy. “We applaud CSL for its commitment of time and effort to conduct this multinational, multi-year study, and we’re delighted that The Lancet has published the RAPID Trial results,” said Walsh. 

The Lancet article says that an interim analysis of data from the two-year extension trial suggest that early treatment with augmentation therapy shows persistent efficacy in patients with Alpha-1 and emphysema. In addition, when patients who had been receiving a placebo in the original two-year trial switched to treatment with augmentation therapy, their lung density decline (which was more rapid than the treated group in the main study) slowed to the same rate as the treatment group. “These findings should encourage early introduction of augmentation therapy [in patients with emphysema due to Alpha-1] and should stimulate further research into optimum dosing,” says the Lancet article.

Robert Sandhaus, MD, PhD, clinical director of the Alpha-1 Foundation and medical director of AlphaNet, was a co-author of the Lancet article.

The RAPID Trial is the first well-powered randomized, placebo-controlled trial to use CT scan lung density as the primary outcome measure. CT scans are currently considered the most sensitive measure of emphysema detection. The trial was conducted at 28 sites in the United States, Europe, Canada, Australia and Russia.

The multi-center, multi-national trial randomized patients with homozygous Alpha-1 (ZZ) to receive either alpha-1 antitrypsin augmentation therapy intravenously at 60 mg/kg weekly or a placebo over two years. CT scan lung density was measured at baseline, three months, one and two years. Secondary endpoints included spirometry, changes in exercise capacity and the rate of pulmonary exacerbations over two years.

The annual rate of lung density loss was significantly less in augmentation-treated patients (-1.45 +/- 0.24 units vs. -2.19 +/-0.25 units; p = 0.017, one-sided). Secondary outcome variables and adverse events were not significantly different between groups, according to the article. There was one death in the augmentation therapy group and three in the placebo group.

Attached is the full article published in The Lancet, with an accompanying editorial by Ron Crystal, MD, Department of Genetic Medicine, Weill Cornell Medical College.

Source: Alpha-1 Foundation 28/05/2015


New Specialist NHS Clinics for Alpha-1

New NHS Funded Multi Disciplinary Service for Alpha-1 Patients

Specialist NHS clinics for patients with alpha 1-antitrypsin deficiency will be running in the West Midlands from the end of May 2015 to cover all aspects of alpha 1. The clinics will be run at University Hospital Coventry by Dr David Parr and at University Hospital Birmingham by Dr David Parr and Dr Alice Turner. The services at both hospitals will be run in conjunction with hepatologists (liver doctors) and dermatologists (skin doctors) and there will be close links with the transplant services and the Genetics Department at University Hospital Birmingham.

Bookings for the clinic in Coventry can be made from the end of May, and an update will follow on a start date for clinics in Birmingham. Patients wishing to be seen in either of the clinics should see their GP to arrange a referral through ‘Choose and Book’, which will enable them to book into the alpha 1 clinic of their choice. Further information can be obtained by contacting Wendi Wallace in Coventry (02476 967697) or Rebecca Bray in Birmingham (0121 3713885). The following links can also be used to access information about the two hospitals and each clinic;

Coventry –

Birmingham –

Please Note: Birmingham web link is not live yet

The ADAPT research programme will continue to be based in Birmingham, and patients attending either NHS clinic who would also like to participate in alpha 1 research will still have the opportunity to do so.

Published: 28/04/2015

Clinical Trials Begin to Treat Alpha-1 Liver Disease

TAP collaborator Arrowhead begins clinical trials on ARC-AAT to treat Alpha-1 liver disease. A groundbreaking trial of a potential treatment for Alpha-1-related liver disease has begun in Australia.Research poster design

Arrowhead Research Corporation, a biopharmaceutical company developing targeted RNAi therapeutics, on Monday announced it has started dosing in a Phase 1 clinical trial of ARC-AAT, the company’s candidate for treating Alpha-1-related liver disease.

Arrowhead partnered with The Alpha-1 Project (TAP), the venture philanthropy subsidiary of the Alpha-1 Foundation, in June 2014 to develop treatment for Alpha-1 liver disease. Under the agreement, TAP will partially fund the development of ARC-AAT. In addition to the funding, TAP is making its scientific advisors available to Arrowhead, assisting with patient recruitment for clinical trials thanks to the Alpha-1 Foundation Patient Research Registry, and engaging in other collaborative efforts that support ARC-AAT development.

Source: Alpha-1 Foundation 23/02/2015

Cabinet Health Secretary commits to improving care

In a landmark seminar at the Scottish Parliament last week, the Alpha-1 Alliance moved one step closer to achieving its goal of improving health services for patients of the condition in Scotland – with a personal commitment to improve care for the rare disease from Health Secretary Alex Neil MSP.

The event, which was chaired by Dr Aileen McLeod MSP, served as a platform to discuss the Alliance’s policy report on the unmet medical need of Alpha-1 patients in Scotland – which was informed by the findings of the Alliance’s 2013 survey of Scottish Alpha-1 patients, relatives and carers.

Members of the Alpha-1 patient community, specialists clinicians, government officials and politicians came together to talk about what steps can practically be taken to establish a nationally commissioned specialist service for Alpha-1. The Alliance was pleased to welcome a number of MSPs to the event, including Conservative spokesman for Health and Community Care Nanette Milne, Linda Fabiani, Iain Gray, Christian Allard and Minister for Children and Young People Aileen Campbell.

Karen North, an Alpha-1 patient and Chair of the Alliance, and Fay Whittaker, an Alpha-1 patient and member of the Alpha-1 UK Support Group, both spoke of their experiences with the condition and about the need for better care for Scottish Alphas.

Professor MacNee, Chair of the Alliance Scotland and Professor of Respiratory and Environmental Medicine at the University of Edinburgh, provided further evidence of the high unmet medical need experienced by Scottish Alpha-1 sufferers in his presentation on the 2013 survey’s findings –confirming the pressing need to establish a national specialist service.

Cabinet Secretary for Health and Wellbeing Alex Neil MSP made a firm commitment to improve services for Scottish Alpha-1 patients, pledging to implement a ‘step change’ in Alpha-1 care. Mr Neil also indicated his intention to initiate a dialogue with the health ministers of the other UK nations to accelerate access to treatment.

“This is a really significant and encouraging commitment to receive from a government Minister” said Chair of the Alliance Karen North “the Alliance will now be doing everything it can to ensure that these changes are impended as quickly as possible”.

Following an overview on the context of specialised commissioning presented by Elizabeth Porterfield, a government official at the Quality Unit of the Scottish Government Health and Social Care Directorates, the event ended with a Q&A session between the audience and panel speakers.

The Alliance will now begin to formally engage with NHS Scotland regarding the establishment of a specialist service for Alpha-1.

Source: Alpha-1 Alliance 24/11/2014

Policy Report On Alpha-1 Care Scotland Published

The Alpha-1 Alliance today released its policy report on the current level of care for Alpha-1 patients in Scotland. The report draws upon the findings of a survey of Scottish Alpha-1 patients, relatives and carers conducted by the Alliance in 2013 – the first of its kind in Scotland. It highlights both the high level of unmet medical need for Alpha-1 in Scotland and how, despite high levels of existing expertise in the condition, patients are all too often let down by poorly coordinated and inefficient service provision.

In response to these findings, one of the report’s main recommendations is the creation of a multi-dis
ciplinary nationally commissioned specialist service for Alpha-1. This service, the report concludes, would be instrumental in ensuring uniformly high levels of care for Scottish Alpha-1 patients.

The publication of this report constitutes a major milestone in the Alliance’s campaign to raise awareness of Alpha-1 and the unmet medical need that patients experience, and to improve care and treatment for the condition in Scotland.

The Alliance will be discussing the report’s findings and recommendations with those affected by the disease, politicians and senior Alpha-1 clinicians at a seminar at the Scottish Parliament on the 19th of November 2014.

The policy report can be accessed and downloaded here

The details of the parliamentary seminar are as follows:

Wednesday 19th November, 6-8pm
The Fleming Room
The Scottish Parliament
Edinburgh EH99 1SP

If you would like to attend and haven’t signed up yet, please register by contacting the Alpha-1 Alliance at or by calling 020 3021 1627 by the 12th of November.

Source: Alpha-1 Alliance 04/11/2014

Alpha-1 Researcher Maurizio Luisetti Passes Away

Maurizio LuisettiProminent Alpha-1 researcher Maurizio Luisetti, MD, of Pavia, Italy, passed away on Monday, Oct. 20, after a brief illness.
He was 61 years old.
“We have lost a dear friend and pioneer in Alpha-1 research,” said Alpha-1 Foundation President John Walsh. “Maurizio was a true gentleman and the ultimate diplomat who was a constant source of wisdom and encouragement as a member of our scientific leadership.”

An advisor to the Foundation, Luisetti was considered the “father” of the Italian Alpha-1 patient and research community, said Foundation Scientific Director Adam Wanner.

Luisetti was medical director at the Clinic of Respiratory Diseases, IRCCS Policlinico San Matteo for 20 years, and head of the clinic’s Laboratory of Biochemistry and Genetics for nearly 30 years in Pavia, where he coordinated an Alpha-1 research registry, testing program and research network. He was an adjunct professor at the University of Pavia’s School of Respiratory Diseases.

In October 2006, he became a professor at University of Turin and in 2010 was appointed professor of respiratory diseases at University of Pavia. In 2010, Luisetti was appointed director of the clinic’s Pneumology Foundation.

In addition to Luisetti’s research and more than 190 publications, his many achievements included an appointment as international governor of the American College of Chest Physicians in 1996, and being a founding member of the Alpha-1 International Registry. He was head of the Italian Registry for Severe Deficiency of Alpha-1 Antitrypsin’s Coordination Center.

Luisetti served on the editorial boards of American Journal of Respiratory and Critical Care Medicine, Sarcoidosis, International Journal of COPD, Pulmonary Medicine, Monaldi Archives of Chest Disease, and Medicina Thoracica.

Luisetti, who organized many scientific conferences, was to be chair of the second Biennial International Research Conference on Alpha-1, to take place concurrent with the 2015 Alpha-1 International Patient Congress, April 9-11 in Barga, Italy.

He was a pervasive presence at the 2013 International Patient Congress in Barcelona, speaking on topics including “Contributions to the understanding of the natural history and genetic epidemiology of AATD over 50 years,” and “The status of Alpha-1 testing in Europe and the U.S. at the 2013 International Patient Congress.”

“Gordon and Maurizio were such good friends,” said Sally Everett, an Alpha, referring to her husband Gordon Snider, MD, whose important role in Alpha-1 research led the Alpha-1 Foundationto establish a research grant in his name this year. Snider passed away in 2013.

“They shared a passion for research on COPD and Alpha-1,” Everett said. “A truly sad day for his family, colleagues and all of us with Alpha-1 and COPD worldwide.”

Fellow researcher Francesco Bonella, MD, in announcing Luisetti’s passing, said, “Besides his outstanding competence and high international profile, Maurizio was a person with extraordinary humanity and commitment towards his patients and coworkers. He was always friendly and ready to help and cooperate. He strongly believed that progress in research depends on cooperation and networking.

“He will be sadly missed,” said Bonella, senior clinical researcher at Wissenschaftlicher Arzt-Prüfarzt Interstitial and Rare Lung Disease Unit. “We have to continue our work with passion and determination in his memory. Thank you, Maurizio, for all you have done and all you gave us in these years.”

Funeral arrangements are being handled by the Bonizzoni Funeral Home in Via Ciapessoni, Pavia. Luisetti’s funeral will be held at 3 p.m. Wednesday at the Church of San Lanfranco in Pavia.

Source: Alpha-1 Foundation October 20, 2014