NZAPA had the pleasure of being invited to attend the 2nd alliance meeting. This year the meeting was held in the beautiful city of Berlin in Germany at the historical Langenbeck-Virchow-Haus and organised by Patientenverband FAP. We were all there to participate in a 4 day conference that involved patients, carers, doctors, researchers and the sponsoring pharmaceutical companies (Alnylam Pharmaceuticals, Akcea Therapeutics, Eidos Therapeutics, Intellia Therapeutics and Pfizer Pharma GMBH).
For the benefit of posterity and background information, my name is Jaime Christmas and my husband, Aubrey, has the hATTR Amyloidosis disease (G47v). Diagnosed back in 2013 and since then having undergone a liver transplant, he continues to deteriorate in his condition as Amyloidosis progressively attacks his heart, nerves, gastrointestinal tract and autonomic system. As treatment is very slow in reaching us here in New Zealand, the advancing disease progression and with time running out, we created NZAPA (NZ ATTR Amyloidosis Patient Association) as an avenue to not only champion treatment for him but also for other families afflicted with this fatal disease here New Zealand.
This article is just a snippet of what we experienced while attending the conference and no doubt, we feel very enlightened, educated and empowered by what we have learnt. Having made vital connections with the pharmaceutical companies involved in the treatment drugs, we are encouraged that in due time (sooner than later) despite the daunting challenges we fear we must presently face, treatment will be made available to all you champions walking the journey with this dreaded disease.
We do recognise that time is ticking away and we hope that we have done justice in this update to spur you on to not give up and that together we can make a difference in each others lives.
September 1st Opening Welcome by Roland Straube, Jean-Christopher Fidalgo and Hartmut Schmidt on behalf of the organising committee.
In attendance were 300 participants from 21 nationalities and involved over 200 doctors.
The organisers warm welcome created a good atmosphere for all of those in attendance and everyone just milled around getting to know one another. Having the disease as a common denominator perhaps made it ‘easier’ as the ability to share stories and experience each other’s struggle allowed the evening to just flow. There were patients and carers from Japan, South Korea, Venezuela, Netherlands, England, Germany, Argentina, Austria, Brazil, Canada, Columbia, France, Israel, Italy, Macedonia, Mexico, Portugal, Spain, Sweden and the United States. Despite the long and costly journey to get there, both Aubrey and I feel very honoured and blessed to be representing not only ourselves but New Zealand. More so when we were joined by our Australian counterpart, Vince and Terri O’Donnell!
Monday, Sept 2nd: Sessions 1-4 For Patients
Day two of conference were filled with slides after slides kicking off ‘session 1’ with a round table presentation on an overview of the Alliance activities since its creation and the launch of their website,
Session 2 presentation covered,
- Which medication is available/not-available and accessible in which countries
- Awareness and diagnosis
- Standard for cure and care
- Research and Trials
- AL Amyloidosis
Fortunately for you, the entire session 2 and 3 have been recorded for your viewing. We highly recommend that you find an evening and listen to what the experts have to share, teach and say.
For your viewing,
One of the main take from this session are the stark differences in the ATTR medication map which highlights the differences regarding medical access between each countries.
Tuesday, Sept 3rd: Sessions 8-11 For Patients and Doctors
To summarise on an otherwise lengthy all day session, the topics covered
- towards an integral service model for cure and care;
- For patients with amyloidosis, no one need can be the same and therefore the setting up of a ‘centre’ must look at what the institution should provide or can provide in terms of services to the patient e.g educational nurse, psychologist, amyloidosis expert, etc
- The amyloidosis expert centre can look at medical, paramedical and educational services.
- Patient organisation can support educational and information services, as well as advocacy.
- Private and public institutions to whom references can be made on where to go to receive support or services which the expert centre cannot fully provide such as financial or legal advice, insurance matters, access to technical aids for disability, etc.
2. Gastrointestinal & nutritional challenges
- Gi symptoms are also associated with an impaired quality of life
- Poor nutritional status associated with a decreased survival time
- Patients with low mBMI (<700) are poor candidates for liver transplantation
3. Early diagnosis
- Treatment is most beneficial early in the disease development
- Early identification leads to early treatment that leads to better prognosis
- University Medical Centre Groningen in the Netherlands are working on a pilot using an Electronic Health Report (EHR) algorithm and Best Practice Alert to identify patients with systemic amyloidosis. This alert system would screen the medical history and problem list that patients visiting the hospitals in the Netherlands face by using the EHR for:
- Neurological problem (polyneuropathy or carpal tunnel syndrome or spinal canal stenosis)
- Cardiac problem (atrial fibrillation or cardiomyopathy)
- Age >50
- Exclusion: Diabetes, amyloidosis
A Best Practice Alert (BPA) will alert the clinician specialist when the EHR is opened and all criteria is fulfilled.
Wednesday, Sept 4th: Satellite Workshop for Patients; How to Start and Mature a Patient Organisation
This half day session covered,
- Steps to set up a patient association
- How to use social media
- How to mature a patient association
- Fundraising issues and ideas
For obvious reasons, we will not elaborate on this at this juncture but feel free to contact me should you want to receive details (jaime @amyloidosis.co.nz)
In total, we hope that this write up can provide a small view into the benefits of having made the journey to attend the Amyloidosis conference hosted by the alliance on behalf of the patients and carers here in New Zealand.
The benefits we received are,
- That we are not alone in this journey
- That progress are being made globally (Europe, United States and Japan) to highlight this disease and create treatment drugs
- The importance of NZAPA playing a role in a global scale via being a part of the European Alliance or forming a sister alliance that covers this region
- The role, activities and management of the Alliance
- How ATTR Amyloidosis are managed in Europe and the rest of the other countries involved
- How disease identification are managed and the future of ATTR
- Treatment options currently available overseas & how to bring it into the country
- The implementation of precision medicine in this field
Our attendance enabled us to make contact with the pharmaceutical patient advocacy representatives. We worked hard trying to communicate the need for treatment to become available to us here in NZ. The inadequate care (via our local doctors, government agencies, nurses, etc) that we face are indeed appalling and we must work together in finding a way to allow treatment to come to us. Unfortunately we also realise that the hurdle not only lays with them (drug companies) but also within our own government agencies. PHARMAC has to approve local treatment (more so for funding purposes) and our medical specialists themselves must be diligent in communicating the need for treatment. The only way forward is for NZAPA to be able to receive as much support as we can locally from patients, carers, their friends and families so that we can be a voice to fight for the rights to be treated. We want to:
- Push for education of this disease amongst our doctors and nurses.
- Allow the drug companies to fund the set up of support centres for patients and carers
- Facilitate local workshops/ conference to host amyloidosis experts to teach those in the medical field as well as patients and families on how to mitigate this disease
- Create a nation-wide awareness of this disease
- Most importantly, push for treatment for patients
Thank you for your time in partaking in this journey with us. We hope we have done justice in this update but if you have any questions at all, please do not hesitate to contact me at ‘email@example.com‘ Together we can achieve greatness.
PS The next alliance meeting will be held in 2021 in London.