Latest News (Test)

What is happening with clinical trials in the UK as we slowly emerge from COVID-19, and how can you continue to support clinical research into new treatment options?

The last year has seen intense focus on the development of treatments and vaccines for COVID-19, along with respiratory clinicians continuing to be on the frontline of treating patients with COVID.  Consequently, research into Alpha-1 and many other diseases has largely been on hold.  As significant progress has been made in the UK and globally with COVID-19 vaccination programmes and with Lockdown continuing to ease, it is good to hear that clinical trials are finally getting up and running again.

Mereo continues to enrol their ongoing Phase 2 ASTRAEUS trial.  This trial explores whether a new oral treatment is a promising potential effective and safe future therapy for Alpha-1.  The trial is well underway and the focus now is to complete the trial, which means that – if it is successful – the next step that Mereo plans is to conduct a Phase 3 trial to support potential regulatory approval of this novel oral therapy for the treatment of Alpha-1.

The ASTRAEUS trial, like so many other things for all of us, has been delayed because of the COVID pandemic.  But now, with the vaccine programme well underway, clinical trial sites here in the UK will soon be able to start screening potential study participants again.  This is great news because getting this trial done will allow Mereo – hopefully by the end of the year if enough people join – to have the necessary data to understand if this possible option of a new treatment can be progressed to a pivotal study and might become available to us in due course. 

As you will be aware, the UK has historically been in the forefront of research into potential new therapies for Alpha-1.  Mereo’s ASTRAEUS trial is no exception:  the UK has the highest number of participating sites and the largest number of Alphas volunteering to join this trial in any country globally.  We would like to extend a huge thank-you to everyone who has already participated.  The effort and commitment of every single Alpha that has already joined ASTRAEUS and those who will hopefully join in the future to help Mereo complete the study is hugely appreciated and adds to the collective knowledge that is so important to develop new treatment options.

We strongly encourage you to consider participation in the ASTRAEUS trial.  You can find out more at the following websites Astraeus Study Portal and on ClinicalTrials.gov.

We would like to sincerely thank all Alphas who continue to come together to help ensure that the UK continues to be a world leader in scientific research and discovery.

While a cure for Alpha-1 would, of course, be the ultimate goal for all of us, these technologies are still a way in the future.  In the meantime, we need to focus our attention on the other, more tangible possible therapeutic options.  ASTRAEUS is a relatively short trial and it is ‘almost there’.  Supporting it will hopefully provide a treatment option in the near term for the Alphas of today and tomorrow, so that we are ready for when the next wave of hopefully new, future curative options could be a reality for all of us.

European Pathways of Care in AATD Study

Patients : Please share this study with your clinicians and encourage them to participate.

Identifying the different pathways of care through which patients with AATD are diagnosed and managed within the different healthcare systems across Europe is a challenge but at the same time would provide information invaluable both for AATD experts and patients

In collaboration with EARCO and AATD core group of the European Respiratory Network for rare respiratory diseases (ERN-LUNG), we intend to try and collect such a data. A survey aimed at professionals involved in the patients referral, AATD diagnosis and management has now been created, and aims to understand:

1. How often patients with AATD are referred for specialist diagnostic, assessment and treatment

2. The type/availability of AATD testing methods in different countries and how these are implemented

3. If patients are routinely followed in ERN-LUNG reference centres, who is involved in the provision of AATD therapy, and if response to treatment is routinely assessed

Data obtained from the survey will be reported in a research manuscript and will provide much-needed information for improving the care for patients with AATD across Europe.

If you are involved in the diagnosis and management of patients with AATD, i.e., AATD testing, genetic counselling, pulmonology/primary care, etc., we would like to ask you to complete this survey using the following link:

https://www.surveymonkey.co.uk/r/EurPathwaysAATDSurvey

Please do forward this link to colleagues outside of AATD expert centres who refer patients for AATD diagnostic, their perspective is most valued as well

We would appreciate if you could complete the survey by July 15th

Survey development was supported by CSL Behring,

Thank you

Joanna Chorostowska-Wynimko, MD PhD

Maria Sucena, MD PhD

Annual Meeting Update 2020

Annual Social Gathering & Information Day 2020

 

As many of our veteran members will know we have held our annual event in September every year for over 20 years (yes, we’ve really been supporting alpha’s and their families for that long) and this year is no exception. However, despite historically hosting their annual meeting in the Spring, Alpha-1 Awareness decided to schedule theirs at the very same weekend as us. This is especially awkward for those of you who are members of both groups and don’t want to miss out on either meeting. With that in mind, and with the additional challenge of COVID meaning that meetings now have to be virtual events, we have carefully considered how we can best serve the alpha-1 community and our members most effectively until the COVID-related restrictions ease off.

 

Our Trustee Tanya Jones started to host virtual coffee mornings/evenings during the COVID lockdown and many of our members took advantage of getting together for a chat, so far we have hosted over 30 of these meetings. Hearing from you during these sessions has given us insight into what may be important to you, and we have considered what we can do to try and help especially as winter approaches.

 

We are very excited to announce that the Alpha-1 UK Support will be holding the following virtual events in the coming weeks instead of our Annual Social Gathering & Information Day this year:

 

Chair Yoga – weekly lunchtime sessions
This event will be led by Ali Graves who is a fully qualified yoga teacher

 

Managing Your Mental Health & Wellbeing
This event will be led by Matthew Vickers a Professional Psychotherapist

 

Alpha-1 UK Support Research Conference
This event will review current research activities and clinical trials internationally and within the UK. There will be presentations from multiple companies followed by a live Q&A session Chaired by Professor David Parr.

 

We will announce further details for each event independently in our Facebook Group and via email to our members.

 

 

Coronavirus (COVID-19) 27th April 2020

Alpha-1 coronavirus (COVID-19) update as of the 27th April 2020:


Further to the previous guidance relating to the risks of COVID-19 that was posted on the 8th April, there is little new that I have to report on the potential repercussions of infection with COVID-19 in patients with alpha-1 antitrypsin deficiency (AATD). Whilst there have been a few isolated reports of patients with AATD having been infected with the virus, the information remains insufficient to draw general conclusions with any degree of confidence. In order to address this current lack of information on COVID-19 in Alphas, it has been agreed following discussion with the EARCO joint chairs, Dr Marc Miravitlles and Dr Timm Greulich, that EARCO members across Europe will provide any information and experience they gain of COVID-19 infection in their patients to me as the central contact who is leading this initiative. This information can be then used to build a picture of the effects of COVID-19 on Alphas as quickly as possible. Until then, the informal expert consensus view remains unchanged from the summarised points in the previous update dated the 8th April 2020, namely:

  • There is no reason, at the present time, to suspect that alpha-1 antitrypsin deficiency (AATD) poses an increased risk of contracting infection with COVID-19.

  • In the event of infection with COVID-19, AATD patients with lung disease (emphysema / ‘COPD’ / bronchiectasis) are likely to have the same risks of developing worsening symptoms and respiratory failure as non-deficient patients with a comparable severity of lung disease. It is, currently, not possible to say whether an acute illness would be worse in these AATD patients than in the comparator patients with normal alpha-1 antitrypsin levels.

  • In the event of infection with COVID-19, AATD patients who develop a pneumonia may be more likely to experience worse long-term lung damage than people with normal levels of alpha-1 antitrypsin.

  • COVID-19 infection is, in some cases, associated with abnormalities in the blood tests that are used to assess liver function. There are no reports that indicate these abnormalities are caused by a clinically significant hepatitis and, to our knowledge, no deaths have been attributed to liver failure arising from viral hepatitis caused by COVID-19.

I have not yet received any answers in response to the emails I sent to Mark Pawsey MP and Matt Western MP to raise their awareness that government guidance in the UK on ‘shielding’ does not currently address the concerns of AATD patients. However, I anticipate that the information arising from the above EARCO initiative will provide some evidence-based guidance to politicians that can be used to lobby on behalf of the AATD community.

Alphas who either know or believe they have been infected with COVID-19 can contact me directly by email (david.parr@uhcw.nhs.uk). Please note that, on account of the exceptional pressure of clinical work during this crisis, requests for advice on matters relating to personal clinical issues cannot be addressed by email: patients are advised to either arrange a referral to the Coventry AATD service or, if they already attend the Coventry AATD Service, to book a virtual clinic appointment through my secretary (Jennifer.ChesterMorgan@uhcw.nhs.uk Tel No. 02476 966205).

Please note that the information in this posting should only be considered up to date at the current time. I will continue to provide regular updates on this website of any significant changes to the current COVID-19 situation.

Professor David Parr, University Hospital Coventry & Warwickshire

Coronavirus (COVID-19) April

Alpha-1 coronavirus (COVID-19) update as of the 8th April 2020:


Further to the previous guidance relating to the risks of COVID-19 that was posted on this website, there continues to be a complete lack of information that relates specifically to the risks for patients with alpha-1 antitrypsin deficiency. Following a discussion with other alpha-1 experts, I can confirm that we are not yet aware of any alpha-1 patients becoming ill with COVID-19 and, therefore, no clinical experience or objective clinical information is yet available to support any evidence-based guidance or advice. Nevertheless, the informal expert consensus view remains, at the current time, the following:

  • There is no reason, at the present time, to suspect that alpha-1 antitrypsin deficiency (AATD) poses an increased risk of contracting infection with COVID-19.

  • In the event of infection with COVID-19, AATD patients with lung disease (emphysema / ‘COPD’ / bronchiectasis) are likely to have the same risks of developing worsening symptoms and respiratory failure as non-deficient patients with a comparable severity of lung disease. It is, currently, not possible to say whether an acute illness would be worse in these AATD patients than in the comparator patients with normal alpha-1 antitrypsin levels.

  • In the event of infection with COVID-19, AATD patients who develop a pneumonia may be more likely to experience worse long-term lung damage than people with normal levels of alpha-1 antitrypsin.

  • COVID-19 infection is, in some cases, associated with abnormalities in the blood tests that are used to assess liver function. There are no reports that indicate these abnormalities are caused by a clinically significant hepatitis and, to our knowledge, no deaths have been attributed to liver failure arising from viral hepatitis caused by COVID-19.

I have contacted Mark Pawsey MP and Matt Western MP (both of whom have previously demonstrated their support for patients with AATD) to raise their awareness that government guidance in the UK on ‘shielding’ does not currently address the concerns of AATD patients. I am awaiting their responses. In the meantime, further information can be obtained at: 

www.gov.uk/government/publications/guidance-on-shielding-and-protecting-extremely-vulnerable-persons-from-covid-19/guidance-on-shielding-and-protecting-extremely-vulnerable-persons-from-covid-19

Patients who consider that they fall into the UK Government’s ‘extremely vulnerable’ category should also visit the website www.gov.uk/coronavirus-extremely-vulnerable and complete the form (or call 0800 028 8327). AATD patients who are concerned they may fall into the ‘extremely vulnerable’ category, but are unsure, should contact their NHS consultant for advice.

This information should only be considered up to date at the current time, since the landscape is changing rapidly. The web links included in the previous posting direct you to live updates relating to the government’s general advice.

Professor David Parr, University Hospital Coventry & Warwickshire

Coronavirus (COVID-19) (2)

COVID-19 INFORMATION FOR ALPHA’S


The following is a statement from Professor David Parr, Univsersity Hospitals Coventry & Warwickshire 

Information on the impact of novel coronavirus (COVID-19) on patients with alpha-1 antitrypsin deficiency is not yet available. It is unlikely that AATD confers an increased risk of becoming infected with the virus. However, the impact of infection with COVID-19 in AATD is likely to be greater than in the general population. Patients with emphysema are less likely to cope with the development of a viral pneumonia and it is likely that the lasting effects of a viral pneumonia will be greater in patients with AATD than in the general population (ie AATD patients are likely to be at high-risk).


The landscape is rapidly evolving and alphas are advised to monitor events on a daily basis, since the likelihood of infection increases with the increasing number of cases in the UK. Advice is available to employers from Public Health England, the NHS and the UK government. Information for the public is widely available on the internet and the following link should provide a reliable source of current information:

www.gov.uk/guidance/coronavirus-covid-19-information-for-the-public

World COPD Day 2019 (2)

Chronic Obstructive Pulmonary Disease (COPD) Day

20th November 2019

 

COPD is a disease that affects a large number of people in the UK. COPD has many possible causes, but one of the lesser known causes is alpha-1 antitrypsin deficiency, or AATD. AATD may affect many Brits, but only some very severely, and can lead to a number of other health issues for sufferers.  

Karen O’Hara, Chair of the Alpha-1 UK Support Group, and Andrew Deans, Lead Research Nurse in Edinburgh, were interviewed about COPD and the often undiagnosed link of COPD and AATD.

AATD is a rare, genetic disorder caused by a defective protein produced in the liver. The World Health Organisation (WHO) recommends that all COPD patients should be tested for AATD at least once in their life. However, these recommendations are generally not strictly followed, and AATD is therefore under-diagnosed in most countries, including the UK. Just a small percentage of patients will have severe AATD, but an early and correct diagnosis is critical to enable targeted interventions to prevent disease progression. It is common for people with AATD to get COPD at a much younger age than people with COPD due to other causes.

Please click the link below to hear the full interview with Karen and Andrew.

Click Here

 

Mereo Alvelestat Study Update

UK sites now open for new drug trial (Astraeus Study) to explore oral compound for Alpha-1 lung disease

 

8 clinical trial sites now open across the UK for ASTRAEUS Study 

Alpha-1 UK Support Group and Alpha-1 Awareness are pleased to note that all eight of the planned clinical trial sites to test alvelestat in Alpha-1 lung disease (the Astraeus Study) across the UK are now open, following the addition of a site in Exeter. This ensures that Alphas in the South-West of England are also given access to the clinical trial via a regional centre.                 

The eight Astraeus sites are spread geographically across the UK, from Edinburgh in Scotland, to Coventry and Birmingham in the middle of the country, down to Southampton and Exeter.  For a list of participating sites, please see www.astraeus-alpha1.com/en-GB/public/Referral

If you are interested in participating in the trial, you are free to approach any site of your choice, no matter where you are usually treated for your Alpha-1.  You can do this
via the Astraeus site.  Given the requirement to visit the centre regularly during the 12-week trial duration, you might want to consider a trial site that is close to you, or you might not mind travelling further – the choice is yours.

If you are told that your first-choice centre is full, please see if your second choice of site is accepting participants – again, this can be found on the Astraeus site. You can also contact us if you encounter any issues.

Lastly, please remember that a clinical trial is a lot of work for the physician concerned as well as a big commitment from the participating patients, so whichever centre you sign up for the trial at, you should be confident that you will be able to manage travel to that site for every study visit.  Dropping out of a trial before the anticipated trial duration weakens the result of a trial.

As mentioned in our previous post back in March, 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 with either PiZZ or Null genotype.  The referral section of the Astraeus website will help you make the initial determination and also direct you towards 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.

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.

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 nine times and receive two phone calls during the study.

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

Please remember that patient participation in clinical research studies is critical for the development of new treatments for Alpha-1 – we need you!

Sandra Takeda Keynote

Alpha-1 UK Support Group would like to congratulate our Trustee, Sandra Nestler-Parr, who today has delivered the keynote opening presentation at Takeda’s President’s Forum in Tokyo, Japan

 

Sandra delivered the keynote opening presentation on the topic “Putting patient centricity at work: An advocate’ view”. Takeda is a Top 10 global pharmaceutical company. With their recent acquisition of Shire, Takeda also acquired Aralast, an intravenous Alpha-1 augmentation therapy product available in the US. For Sandra to be invited to speak at this closed global leadership meeting is a great honour and recognition of her longstanding dedicated advocacy work in the rare disease patient community. It also offered a great platform to raise awareness of the unmet needs of the Alpha-1 patient community. In addition, I am very pleased to announce that Sandra will donate her generous honorarium for this speaker engagement to our patient charity. Please join me in thanking Sandra for this generous offer, particularly as she took annual leave from her day job in order to be able to travel all the way to Japan to give this presentation.

Mereo Alvelestat Study

Alpha-1 UK Support Group & Alpha-1 Awareness are very pleased to announce exciting news about a new drug trial for the UK

 

UK trial sites open for ASTRAEUS Study into Alpha-1 Antitrypsin Lung Disease

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.