A clinical research trial of a potential new therapy for adults affected by Alpha-1 Antitrypsin-related lung disease has opened in the UK and has started enrolling patients.

Seven trial sites – in Birmingham, Cambridge, Coventry, Edinburgh, Leicester, London and Southampton – are already open, and it is anticipated that additional sites will be added in due course.  There are many sites spread across the UK and Scotland, so you have the opportunity to hopefully find a site nearby and minimise the travel time for study visits.

All details are available on the UK website www.astraeus-alpha1.com, where you will be able to find more details, check if you are eligible for the study and get in contact with a trial centre near to you. There are also details on the official clinical trial registry:  www.clinicaltrials.gov/ct2/show/NCT03636347.

All patients interested in participating in this study are at liberty to contact any of the study centres to discuss the trial.  If you are interested in participating in this study, please use the website link above to find a suitable trial site or contact any of the study centres directly to discuss the trial.  Your preferred study centre does not have to be the same centre where you receive your regular care for Alpha-1 and may be chosen based on ease of access and personal preference.

The Astraeus Study is testing the safety of a research drug and whether it has the potential to reduce lung damage and slow the progression of lung disease caused by Alpha-1 Antitrypsin Deficiency.  The potential treatment, alvelestat, is taken as tablets and has already been tested in people with other lung diseases.

You must meet the study inclusion criteria which, amongst others, include the age range of between 18 to 75 years and a diagnosis of Alpha-1 Antitrypsin Deficiency lung disease.  The referral section of the Astraeus website will help you make the initial determination and also direct youtowards the different physicians leading the trial centres.  The physicians at the different trial centres will be able to advise if you are eligible based on the study criteria, which have been agreed by the regulatory ethics committees here in the UK.

Enrolled participants will receive all study-related care, testing and study medication at no cost.  You will receive study drug for about 12 weeks and you will be in the study for approximately 20 weeks in total.  You will need to visit the research site about 9 times and receive 2 phone calls during the study.

Location (Alphabetical)	Study Doctor	Contact Details
Birmingham	Dr. Alice Turner	0121 371 3885
Cambridge	Prof. Ravi Mahadeva	0122 334 8119
Coventry	Prof. David Parr	0247 696 6205
Edinburgh	Dr. Roberto Rabinovich	0131 536 3143
Leicester	Prof. Bibek Gooptu	0116 229 7137
London	Prof. Nicholas Hopkinson	0207 352 8121
Southampton	Prof. Tom Wilkinson	0238 120 5341

 For more information, please see:  www.astraeus-alpha1.com.

 

This remarkable new autobiography recalls the days when rugby league could be brutal and was regularly described as ‘The toughest sport in the World.’

It follows Keith from his days at Hull KR in the mid-sixties where he packed down with legends such as Peter Flanagan and Frank Foster, to the climax of his unique career, playing for Maitland in Australia during the seventies, in the highly competitive Newcastle league.

 

 

Rugby MP Mark Pawsey last week led the first ever parliamentary debate on alpha-1 antitrypsin deficiency, a rare hereditary illness which affects the lungs and breathing of those with this condition.

Alpha-1 antitrypsin deficiency is caused by a genetic abnormality which means that the protein alpha-1 antitrypsin, normally released into the blood to protect from inflammation, is instead trapped in the liver. This can not only cause significant damage to a patient’s liver but also leads the lungs becoming vulnerable to pollutants in the air. In particular, those with alpha-1 are particularly susceptible and sensitive to cigarette smoke.

Symptoms of alpha-1 are often mistaken for asthma or chronic obstructive pulmonary disease, leading to the incorrect treatment which can lead to a patient’s health deteriorating. Because of this, Mark has become a supporter of the Alpha-1 UK Support Group, which seeks to raise greater awareness of the condition and a delegation of alpha-1 patients visited Parliament to hear the debate.

Speaking after the debate, Mark said:

“Alpha-1 antitrypsin deficiency was first brought to my attention in 2012 by Sarah Parrin, when she attended my constituency advice surgery with her son, Stephen Leadbetter. Despite having presented symptoms of alpha-1 over eight years before, he had only just been formally diagnosed with the condition. That delay in diagnosis had real consequences for his health as he was growing up, and demonstrates the scale of the challenge facing patients with alpha-1.”

Mark continued:

“After meeting with Stephen and Sarah, I resolved to find out more about this condition and met with Professor David Parr, who is a consultant respiratory physician at University Hospital Coventry and Warwickshire, and an expert in Alpha-1. From there, I have become a supporter of the Alpha-1 UK Support Group, which recently met in Brandon in the constituency. I am especially pleased that a number of those with alpha-1 were able to attend the debate and it was a great pleasure to meet with them and hear directly about how alpha-1 affects their day-to-day lives.”

Mark concluded:

“I would like to thank the Nigel Adams MP, who spoke on behalf of the Government, for his supportive response although we all recognise that there is more work to be done. I particularly hope the Government will now ensure that the specialised NHS alpha-1 service which was due to be in place by April 2017 will be introduced as swiftly as possible. However, one of the most important things which all of us can do is be more aware of this condition and that is why I was delighted to have support from across the House during this important debate.”

Professor David Parr, Clinical Director for Cardio-Thoracic Services and Consultant Respiratory Physician at University Hospitals Coventry and Warwickshire commented:

“I know from my experience of seeing alpha-1 patients from all over the country in our multi-disciplinary AATD service at UHCW that there needs to be greater awareness of this complex condition amongst healthcare professionals. I very much welcome Mark’s continuing support of the alpha-1 community in their quest to obtain better access to optimal care.”

Sandra Nestler-Parr, Trustee of the Alpha-1 UK Support Group added:

“Mark has been a strong supporter of the alpha-1 patient community in the UK for many years, and we are very grateful that Mark has brought the challenges faced by patients with alpha-1 antitrypsin deficiency to the awareness of a wider audience through last week’s parliamentary debate.”

The full transcript of the debate is available at: https://hansard.parliament.uk/Commons/2018-10-31/debates/D79EB4FC-284C-4B5F-8B68-0BFAAB302325/Alpha-1AntitrypsinDeficiency

 

Video of the debate is available at: https://www.markpawsey.org.uk/news/mark-pawsey-mp-raises-awareness-rare-illness-parliament

 

Dear Alpha-1 patients in the UK,

WE NEED YOUR SUPPORT! URGENTLY!

We are delighted to inform you that, further to Mark Pawsey MP’s attendance at our recent patient meeting in Coventry, Mark has secured a Parliamentary debate on “Treatment of alpha-1 antitrypsin deficiency”, to be held next Wednesday from 16.00 – 16.30 at Westminster Hall in the Houses of Parliament (https://calendar.parliament.uk/calendar/Commons/All/2018/10/31/Daily). Matt Western MP who was also at our recent meeting will also be in attendance at the debate.

This is fantastic news, as this event will have cross-party support. We have worked closely with Mark Paswey to prepare the debate.

The key aim of the debate is to get as many MPs as possible to attend, to further raise awareness of Alpha-1 and to highlight the challenges the Alpha-1 patient community faces.

Unfortunately we were only informed about this on Friday and haven’t got much time to prepare. This is where we need your help. How can you do this?

1. Find out who your local MP is (if you don’t know just google your constituency and Member of Parliament)

2. Personalise the letter below and email it to your MP as soon as possible!!

3. On Tuesday, call your MP’s office to ensure that they received your email and to reiterate that you would like to encourage your MP to attend the debate.

4. If you receive a response from your MP ahead of the debate on Wednesday, please email us at info@alpha1.org.uk to inform us whether your MP is planning to attend, so that we can plan accordingly.

You can watch the debate on Parliament TV (https://www.parliamentlive.tv/Commons), which will be live streamed.

Thank you for your support.

The Alpha-1 UK Support Group

 

Dear [MPs Name], 

Westminster Hall Debate on Treatment for Alpha-1 Antitrypsin Deficiency
16:00 – 16:30, Wednesday 31^st October, 2018

I am one of your constituents and am writing to kindly ask you to attend a Westminster Hall debate on Treatment for Alpha-1 Antitrypsin Deficiency, moved by Mark Pawsey MP, to be held on Wednesday, 31^st October 2018, at 4pm.

I live in [town/village/borough] and I suffer from Alpha-1 Antitrypsin Deficiency (Alpha-1), a rare and complex genetic disorder that can cause lung and liver disease in adults and children, and occasionally affects other organs and may require organ transplantation. Severely affected individuals experience significantly reduced life expectancy, severe disability, and great limitations on multiple other aspects of their lives. Currently, no specific treatment for Alpha-1 is available in the UK.

Due to its rarity, Alpha-1 is often misdiagnosed, with an average delay of 6-7 years before patients receive the correct diagnosis, frequently resulting in sub-optimal care of Alpha-1 patients for many years. A nationally highly specialised NHS service for Alpha-1, to improve patients’ access to co-ordinated multi-disciplinary clinical expertise, was approved by the Department of Health’s Prescribed Specialised Services Advisory Group in March 2016, to be operational in April 2018. However, the development of this service has still not commenced.

Only one specific treatment for Alpha-1 exists: intravenous alpha-1 augmentation therapy. This treatment has been available to patients in many countries, including the US, Germany, Spain, Italy, Portugal, Austria and others for many years. The only augmentation therapy product with a license in the UK (which was granted in August 2015) is currently undergoing evaluation by the National Institute for Health and Care Excellence under the Highly Specialised Technology (NICE-HST) appraisal process. On 26^th September 2018, NICE published its draft guidance not to recommend this treatment for use in the NHS. The evaluation is currently out for consultation, but we are very sceptical whether the treatment will be recommended for reimbursement by the NHS.

I very much hope that you will recognise the life-changing impact that this disease has on patients like myself, and attend the Westminster Hall debate on Wednesday to learn more about the challenges of Alpha-1 patients and what you can do to support patient experience.

Yours sincerely,

[Your name]

In light of very recent events, we would like to provide some important information about upcoming clinical trials in Alpha-1 in the UK.

Currently, there are three trials in AATD planned to commence by pharmaceutical/biotech companies (Mereo Biopharma, Arrowhead Pharmaceuticals and CSL Behring). These trials will be undertaken in several different centres with Alpha-1 expertise across the UK.

It has come to our attention that Dr Turner from UH Birmingham implied in a recent email to an AATD patient that these trials will be conducted in Birmingham only and that patients should get in touch with her team now if they want to be considered for participation in any of these trials. This is misleading and FACTUALLY INCORRECT. Patients have no advantage for being chosen to participate in a clinical trial whatsoever if they provide their contact details to Birmingham or to any of the prospective trial centres at this point; in fact, there are several reasons why it may be counter-productive to do so.

The general process for patient recruitment is always the same and as follows: Once a trial has been formally approved, the company conducting the trial will publicly announce more detailed information about the trial, the trial centres and contact information at each trial centre. Once this information is released, patients interested in participating in the trial can then contact the trial centre of their choice (or several different centres if they wish). For logistical reasons, most patients tend to choose a trial centre that is geographically close to where they live, as most clinical trials require regular visits to the trial centre – however, the choice of centre lies entirely with the patient.

A few important points to keep in mind when you think about which trial centre to choose:

• Every clinical trial needs to follow a “trial protocol” which sets out how the trial is conducted, how often patients need to visit the centre, which tests are being performed etc. Each trial centre needs to strictly comply with this protocol. Patients will therefore have the exact same experience throughout the clinical trial, irrespective of the trial centre they choose.
• Every centre where the trial is conducted will have undergone a detailed screening process to ensure that the clinical staff have relevant and sufficient expertise and competence in AATD and that the technical, administrative and ethical requirements are met by the centre.
• A clinical study is less likely to be successful if the majority of patients attend a single trial centre. The more evenly distributed the patient numbers are across different centres, the better. There are several reasons why it is advantageous for the trial if patients choose a trial site geographically convenient for them: if patients have to travel a long way to the trial centre, they are more likely to miss study visits or drop out of the trial.
• The better the patient community advertises AATD trials, the more likely it is that the trials are successful, because we reach a wide range of patients, rather than only those who ‘register’ with a specific centre, as Dr Turner misleadingly suggested in her email to the Alpha-1 patient. All trial centres will offer the opportunity to discuss the trial in detail with each patient, once patient recruitment is allowed to start. 
• The AATD patient community has campaigned for many years to increase disease awareness and clinical expertise across the UK. As a result of this campaign, we now have several very experienced AATD centres across England, with two additional satellite centres currently being set up in the South and the North of England. It is crucially important for patients to utilise all centres, in order to help them gain more expertise and to ensure that all centres remain sustainable.
• Please note that patients who participate in clinical trials do not need to move their routine specialist NHS care to this trial site (even if this is suggested by the trial centre). It is entirely up to the patient to choose which doctor’s care they wish to be under.

We are in regular and frequent communication with all companies who are planning to conduct trials in AATD in the UK. As soon as they are in a position to publish specific information on these trials, including details about the trial centres and contact details, this will be available on our website. For current information on upcoming trials:
• Read the update in our recent newsletter (pages 2 and 3, https://alpha1.org.uk/attachmen…/…/51/A1UK_Newsletter2018.pdf),
• Watch the presentations that Mereo Biopharma, Arrowhead Pharmaceuticals and CSL Behring gave on their respective clinical trial programme at our recent annual patient group meeting – fast-forward to minute 9 on video 2 https://www.facebook.com/pg/alpha1uksupportgroup/videos/. This video includes a list of all prospective UK trial centres for the upcoming Mereo Biopharma trial – fast-forward to minute 23 of video 2.
• As soon as we have any update on any trials relevant in the UK, we will post it in this group.

Karen O’Hara, on behalf on the Alpha-1 UK Support Group, supporting all Alphas in the UK

Clinical utility of alpha-1 proteinase inhibitor…

Clinical utility of alpha-1 proteinase inhibitor in the management of adult patients with severe alpha-1 antitrypsin deficiency: a review of the current literature – the authors are Professor David Parr and Dr Beatriz Lara who are the Alpha-1 specialists who operate the NHS Alpha-1 Clinic at University Hospitals Coventry & Warwickshir

https://www.dovepress.com/articles.php?=33769

New Report! 

Our Trustee, Dr Sandra Nestler-Parr is a co author on this report published on 14th July 2017
Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report

http://www.sciencedirect.com/science/article/pii/S1098301517302644

PARTY IN THE PARK CHILDREN’S DAY
Saturday 19^th August 2017

Folly Farm, Kilgetty, Wales

The Alpha-1 UK Support Group are holding our 2^nd Party in the Park specifically targeted at our younger members and their families.

Folly Farm has both indoor and outdoor areas.  The zoo has over 750 animals and you can get up close to furry and feathery farmyard friends in their barn  The vintage fairground has 16 different rides and there are eight adventure play areas.  All set in 120 acres so there should be something to entertain everyone.

https://www.folly-farm.co.uk/

For further information or to register to attend please contact Jemma Coad info@alpha1.org.uk

 EU ALPHA-1 AWARENESS DAY | 25 APRIL 2018

 For the first time in history, the European Alpha-1 Community celebrates an EU Alpha-1 Awareness Day, on 25 April 2018. Among other activities, the main “kick-off” event takes place the day prior (24th) in the European Parliament from 15:00-17:00hrs. MEP Marek Plura (Poland) will host a round table discussion for MEPs, during which time patient representatives will provide updates from the following countries: Austria, Belgium, Germany, Italy, the Netherlands, Italy, Romania, Spain, Switzerland and the United Kingdom. The event is organized by RPP Health Care and sponsored by Alpha-1 Global, a program that facilitates collaborative efforts among Alpha-1 associations around the world. 

The EU Parliament gathering is directly related to the development of the Alpha-1 European Expert Group Recommendations; a document that was created to raise awareness among governments, health systems, health professionals and society in general about the importance of diagnosing, treating and investigating Alpha-1 Antitrypsin Deficiency (AATD). 

Combined action is sought in Europe in support of the early diagnosis and correct treatment of people with AATD, especially considering that there are still European countries that do not reimburse plasma derived augmentation therapy for AATD patients with severe lung disease and skin diseases such as panniculitis.

 Early diagnosis 

The Recommendations address the complications of uncontrolled pulmonary and liver diseases. “The main difficulty of handling Alpha-1”, the document reflects, “is the early diagnosis, which most patients do not obtain until they already have severe symptoms despite the low cost and effectiveness of diagnostic means.”

The challenge is to consolidate approaches for the training of health professionals and patients, and facilitate the exchange of knowledge, in addition to promoting diagnosis and extending the network of centers of excellence for Alpha-1. “The problem of Alpha-1 is not so much the number of affected patients as their quality of life, which can be significantly impacted by the delay in diagnosis and limitations in access to treatment.” 

The document proposes to the member states the development of a plan for the diagnosis of AATD, within the framework of their Rare Diseases programs. It also alerts health professionals to the need to perform the deficit tests for all patients with COPD, bronchial asthma, bronchiectasis and liver diseases of unknown origin, as well as people with a family history of AATD. 

The European Commission is requested to update its CORDIS study (2002) to find the current status of genetic testing for rare diseases and to issue subsequent recommendations for countries to promote an approach based on maintaining a high level of human health and not discriminating against patients taking effective steps to avoiding both human and health system costs.

Pollution and lung patients 

Air quality (interior and exterior), toxicity of chemical products in the workplace and pollution, are other aspects on which the Recommendations reflects, taking into account that “in environments with contaminated air, the organs of patients with a deficit degenerate in a significantly faster way than those of the remaining patients with COPD. ” Poor air quality is a “silent killer that currently affects 90% of residents in EU cities, especially lung patients,” says UK parliamentarian Seb Dance. “One of the measures that policymakers can adopt to improve the health of Alpha-1 patients is to achieve the highest standards of indoor and outdoor air quality.” 

Coordination and patient care 

The Recommendations also express the need for EU countries to coordinate their efforts. The creation of an EU stamp is requested for the accreditation of Centers of Excellence and its registration in the Orphanet database, as is already the case with Reference Centers in Rare Diseases. Implementing the Cross-Border Healthcare Directive, to support the rights of patients to have the best possible care, is another of the demands included in the document.

Translated from the article by Elena Goyanes on the Alpha-1 Spain website:
https://alfa1.org.es/dia-europeo-del-deficit-de-alfa-1-antitripsina/

 

 

Here is Linda’s Story……….

Linda was Trustee of the Alpha-1 UK Support Group and was always there, in person, on the end of the phone, by email and Facebook, to offer support and friendship to all who came within her sphere, no matter what walk of life they were from and no matter how ill, she herself was feeling….