Dr Ravi Mahadeva has resigned as Chair of the Alpha-1 Alliance due to a conflict of interest in relation to his work with NHS England’s Specialised Respiratory Disease Clinical Reference Group (CRG) and the ongoing campaign of the Alpha-1 Alliance.

Dr Mahadeva has assured the Alliance of his ongoing support for improving access to health services for patients affected by Alpha-1 Antitrypsin Deficiency (Alpha-1), which he will continue to pursue at CRG level.

The Alpha-1 Alliance would like to take this opportunity to thank Dr Mahadeva for his services and his dedication in supporting the Alliance and the Alpha-1 Community and we look forward to continuing to work with him in his capacity as a CRG member.

Karen North, Trustee of the Alpha-1 UK Support Group said: “The Alpha-1 Alliance campaign to establish a nationally highly specialised service and ensure access for patients to optimal integrated clinical care and associated therapies remains unaffected and will proceed as planned.”

A new Chair to the Alliance will be appointed in due course.

Source: Alpha-1 Alliance 29/05/2014

The Secretary of State for Health, Jeremy Hunt MP, met with representatives of the Alpha-1 community last Tuesday (13^th May 2014) at the Department of Health to discuss the unmet medical need of Alpha-1 patients and the associated requirement to establish a highly specialised service for patients affected by the condition.

The meeting was attended by Mark Pawsey MP, Margaret Millar (co-founder of the group Alpha-1 Advocacy & Action) and Karen North (Trustee of the Alpha-1 UK Support Group), both from the Alpha-1 Alliance’s Executive Committee, Dr David Parr (Consultant Respiratory Physician at the University Hospital Coventry) and the Alliance’s Secretariat.

During the meeting, Margaret Millar and Karen North, representatives of the patient community, shared their experience of living with Alpha-1 and the impact the disease has on their lives.

Margaret Millar, an affected patient who lost a sister to the condition, highlighted the burden the disease places on herself and her whole family, describing living with Alpha-1 as a “steady decrease of quality of life”. She said: “I had to give up work because of the disease, I often have chest infections, my social life suffers and I have difficulties walking my dog”.

Karen North, who also suffers from Alpha-1,  shared a similar experience to Margaret’s, describing the challenges imposed by the disease: “Everyday tasks many people take for granted such as bathing, shopping and walking on even the smallest incline will leave me breathless and sometimes gasping for air”.

Dr Parr, a respiratory consultant and Alpha-1 expert, briefed the Secretary of state on the clinical aspects of the disease and the current management of patients with Alpha-1 in the NHS. Dr Parr explained that the rarity and complexity of the disease meant that patients often experience a significant delay in receiving a correct diagnosis and in obtaining expert advice on the disease and its clinical management.  He advised that patients desire and need access to an integrated multidisciplinary team based in dedicated centres where clinical expertise can be concentrated to facilitate optimum care. 

In the meeting, the Alliance also expressed its concerns over the current uncertainty and lack of clarity of the commissioning process for highly specialised services and its application process. The Health Secretary requested to be kept updated on the progress of the campaign.

Source: Alpha-1 Alliance 29/05/2014

As part of the campaign in the Scottish Parliament, Members of the Scottish Parliament (MSPs) Malcolm Chisholm and Graeme Dey wrote to the Cabinet Secretary for Health and Wellbeing, Alex Neil MSP, on behalf of their constituents, to raise the issue of the level of care provided to patients with Alpha-1 Antitrypsin Deficiency (Alpha-1) in Scotland.

The key points in the Minister’s response letter are as follows:

• The Minister reiterated the Scottish Government’s commitment to raising the profile of rare diseases such as Alpha-1 and referred to the UK Rare Disease Strategy. An Implementation Plan for Rare Diseases in Scotland is currently being developed with the aim of delivering the commitments set out in  the Rare Disease Strategy, published in November 2013, to improve services for people living with a rare disease and their families.

•  The Minister’s letter provided an overview of the current care arrangements for patients with Alpha-1.  A Managed Clinical Network (MCN) approach is in place in Scotland that brings together professionals from primary, secondary and tertiary care with patients and carers, to work in a coordinated fashion across geographical, organisational and professional boundaries. The National Advisory Group of Respiratory MCNs (NAG RMCN) was also highlighted as a structure that helps to ensure a coordinated approach with the aim of improving respiratory services, serving as a platform for  local MCNs and other key stakeholders to discuss issues, find solutions and share best practice.

•  The Minister highlighted the Scottish Government’s interest in welcoming study proposals in Alpha-1 from the research community to further research in this area.

To access a copy of the Minister’s letter on Alpha-1, please follow the link.

Source: Alpha-1 Alliance 20/05/2014

As part of the Alliance’s campaign in Scotland, the Alpha-1 Alliance met with the following MSPs on Thursday 8th May in the Scottish Parliament:

• Bob Doris MSP, SNP Member for Glasgow Region
• Malcolm Chisholm MSP, Labour Member for Edinburgh North and Leith
• Collin Beattie MSP, SNP Member for Midlothian North and Musselburgh
• Iain Gray MSP, Labour Member for East Lothian

During the meeting, the Alliance briefed Parliamentarians on the current care of patients with Alpha-1 Antitrypsin Deficiency (Alpha-1) and on the need for the establishment of a highly specialised service in Scotland.

The Alliance will continue its meeting programme in the Scottish Parliament, and further meetings with MSPs have been scheduled for June 25th, 2014.

Source: Alpha-1 Alliance 19/05/2014

Kamada Announces Preliminary Results from Phase II/III Pivotal Trial in Europe and Canada of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency

Kamada Ltd., a plasma-derived protein therapeutics company focused on orphan indications, announces preliminary top-line results from the phase II/III pivotal clinical trial in Europe and Canada of the company’s proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD or inherited emphysema).  

The endpoints selected for this trial were based on scientific advice from the European Medicines Agency (EMA) and include those deemed to be clinically meaningful, such as frequency, time to first, duration and severity of exacerbation events, among others. A preliminary analysis of the results indicates clinically meaningful signs for inhaled AAT efficacy as well as additional positive signs in specific study populations. In a very important secondary end point, frequency of severe exacerbation was approximately 50% lower in the AAT group versus placebo. With regards to the primary endpoint of “time to first moderate or severe exacerbation,” early data do not show differences between the two treatment groups. 

Kamada’s announcement of the results:-

MTB McCartan Turkington Breen Solicitors collectively have raised an amount for the Alpha-1 UK Support Group that far exceeded expectations.

Ernie Waterworth, Ernie Telford , Dianne Wilson and Katie Campbell completed the 8 mile Belfast Marathon walk yesterday with John McShane completing the full marathon.

On behalf of the Alpha-1 UK Support Group a big thank you to everyone who took part and supported the event raising awareness and funds for Alpha-1. Through your kind support we are able to continue funding our programmes of supporting research, campaigning for better access to treatment, providing information and supporting Alphas and their families. We know there is so much more we can and need to do to promote better knowledge and understanding of Alpha-1 Antitrypsin Deficiency but we are limited by the funds we receive, so your support is valued and very much appreciated.

John L Mugford
Alpha-1 UK Support Group Chairman/Trustee

Minister Answers Parliamentary Questions on Alpha-1

Alpha-1 Alliance meets Andrew Lansley MP to discuss need for a national service

Source: Alpha-1 Alliance 26/03/2014

Alpha-1 Alliance in discussions with NHS England

Alpha-1 Awareness UK will be hosting two events this year:-

Further Information:

For our Information Day this year we are going to the Birmingham Area, which we hope will be easily accessible for a large number of our members.  

We have once again have an excellent selection of speakers, who we feel sure you will find interesting and informative. 

Doctor Alice Turner  Clinician Scientist and Honorary Consultant Physician Queen Elizabeth Hospital Research Laboratories, Birmingham 

Doctor Bibek Gooptu  King’s College London and Birkbeck College, University of London 

Doctor John Hurst  Reader in Respiratory Medicine Royal Free Campus, University College London Medical School 

Chris Brown-Martin  PiZZ, Nominated European Lung Champion 2012 and leader of the Kayak Challenge 2013 

Brian Gunson Project lead Alpha-1 Standard of Care Munro & Forster

Further Information: