The importance of fundraising

Sounds like a silly title – the importance of fundraising. Obviously, fundraising is important. Without money, there’s not that much we can do.

But fundraising plays a purpose for rare disease patient groups (and most other charities) that goes beyond raising funds. It’s about raising awareness, telling others about the issue you’re focusing on, using your supporters to spread the word.

This is something the AKU Society is increasingly trying to do. This Sunday, we have 10 runners taking part in the London 10k, a major sporting event with more than 25,000 runners participating and more than 40,000 spectators. It takes place in the middle of London and should be a fantastic event.

We got the idea from other rare disease patient groups. They encouraged us to register with the London 10k. One of these rare disease patient groups raises £25,000 a year thanks to the London 10k – that’s a substantial amount of funding that can be used as the charity wants in order to further its mission. I don’t think we’ll get anywhere near that amount this year, but it’s a good target to aim for in future years.

We do most of our fundraising for these events via our JustGiving page at www.justgiving.com/alkaptonuria. If you scroll down, you’ll see that there’s a whole bunch of supporters raising funds for us.

And if you’d like to sponsor me for the London 10k, you can donate on my justgiving fundraising page at: www.justgiving.com/Nicklondon10k. So dig deep!

A successful AKU meeting in Cardiff

We had a fantastic AKU meeting in Cardiff on Wednesday evening that brought together more than 70 metabolic consultants, scientists, patients, industry representatives and NHS advisors to hear about and discuss the latest advances in AKU research and treatment. The meeting was co-sponsored by Swedish Orphan Biovitrum Ltd and the AKU Society and was a pre-meeting ahead of the annual conference of the British Inherited Metabolic Disease Group.

Prof James Leonard chaired the meeting. He’s a world-class expert on metabolic diseases with a tremendous reputation, so we felt privileged that he led the meeting. He also has experience of treating AKU patients for much of his working life, hence his input was highly valuable.

Prof Jim Gallagher, head of the AKU Science Team at the University of Liverpool, presented the pathophysiology of AKU. He focused on the latest solid-state nuclear magnetic resonance (ssNMR) studies on AKU as well as the latest promising developments in our AKU animal model.

I then did a short presentation on the growth of the AKU patient movement. I emphasised the support the AKU Society provides to patients, particularly through online tools such as the AKU Online Community, but also our part in developing research and international collaboration.

Dr L Ranganath, our Medical Director, presented the results of a clinical study on AKU patients that led to the successful development of a disease severity score index. This is highly significant for our work as this severity score index means we now have a method for assessing, in a robust way, the evolution of the disease among AKU patients. This will allow us to assess the effectiveness of treatments in new clinical trials.

The meeting finished with a presentation by Dr Wendy Introne from the National Institutes of Health on the recent trial of Nitisinone, a drug that reduces by 95% homogentisic acid, which is the culprit molecule in all the damage caused by AKU. We then went on, after the meeting, to discuss plans for a new trial for Nitisinone in order to obtain marketing authorisation so that patients can access it as the first ever treatment for AKU.

 

Changemakers

I’m just about to leave for the Eurostar for another trip – the last of a long series of work trips for now. I’m going to Paris for three days to attend the Changemakers Week organised by Ashoka, the global network of social entrepreneurs. It promises to be a particularly exciting event.

For those of you who don’t know Ashoka, it’s a fantastic organisation that brings together social entrepreneurs from around the world, providing them with support, access to resources and advice for their work. I’ve been a fellow of Ashoka for 18 months now, and they’ve been excellent, providing introductions to key people and access to their networks.

The Changemakers week, under the patronage of the French president Nicolas Sarkozy, will bring together more than 1,000 participants at the campus of HEC, one of France’s leading business schools. Talks will cover everything from health to energy and housing and will include major networking sessions to meet new people and form new alliances.

Anyway, I’d better go. Got a train to catch!

Why patient groups should attend the Eurordis summer school

I’m just back from a terrific week of seminars at the Summer School for Clinical Trials for Rare Diseases, organised by Eurordis. There were around 50 participants from a range of rare disease patient groups from 20 countries across Europe. If you’ve never attended a Eurordis summer school, and you’re a patient group, then definitely keep an eye out for the next one.

The speakers were excellent, including members of the European Medicines Agency Scientific Advice Working Party, Committee for Orphan Medicinal Products, Committee for Human Medicinal Products and Committee for Advanced Therapies. We covered issues such as the life cycle of drug development (phase 1, 2 and 3 trials, etc), the theory behind randomised controlled trials and their applicability to rare diseases, the statistics of clinical trials, and the regulatory process.

We had group discussions on issues such as ethics and clinical trials, with case studies.  We also looked specifically at clinical trials in ‘non-standard situations’, which was particularly relevant to the small population sizes of rare diseases. We then looked at different forms of regulatory approval, such as marketing authorisation, conditional approval, and exceptional circumstances.

From our point of view, it was absolutely brilliant. The AKU Society and our partners are working on building a new clinical trial for a potential treatment, hence understanding the regulatory process is crucial to our success. The summer school provided a crash-course in all the key information needed to build a solid trial and go for regulatory approval.

If you’re interested in finding out more, go to the Eurordis e-learning website at: http://www.eurordis.org/content/e-learning.

 

Eurordis summer school on clinical trials for rare diseases

I got back late last night from London as we had a meeting with the NHS about our bid for setting up a National AKU Service in Liverpool. We’re cautiously optimistic as everyone seems to agree that the project is well put together and promising. It would make such a huge difference to the lives of AKU patients in England as they would have access to a centralised team of AKU specialists and treatments.

The plan is that AKU patients would be able to come to Liverpool each year for a full series of tests with a range of specialists (rheumatologists, metabolic consultants, physiotherapists, etc) to cover the range of health problems they face. If all goes well, the patients would also have access to the latest treatments.

More about all this in a later post as it’s still too early to tell whether the proposal will be approved.

I’m going to be away for the next week, probably without much blogging, as I’ve been invited to the Summer School on Clinical Trials for Rare Diseases organised by the European Organisation for Rare Diseases (EURORDIS). This is a crucial workshop as they will train 40 or so patient groups in all aspects of how clinical trials are set up, implemented and approved.

For the AKU Society, this is a major benefit as we’re already starting to plan new trials and applications to the European Commission for funding them. You can read all about the EURORDIS summer school here: http://www.eurordis.org/content/eurordis-summer-school-patient-advocates.

It’s non stop at the moment for the AKU Society. This week, Oliver Timmis, our AKU Communications Project Manager, was in Brussels for a workshop on how to apply to funding to the European Commission. Later this month we have the AKU meeting in Cardiff, which promises to be excellent.

Hopefully, we’ll be able to rest a bit in July!

 

Using social media for clinical research

Here’s an article article I’ve just come across from the Alzheimer Research Forum. It’s about using social media to carry out research into treatments: http://www.alzforum.org/new/detail.asp?id=2791.

The research team from PatientsLikeMe, an online medical data sharing platform, used data volunteered by patients on the site to evaluate the efficacy of off-label lithium use for amyotrophic lateral sclerosis (ALS), as discussed in the April 24 online Nature Biotechnology journal. ‘Because in 2008 a small clinical study suggested that lithium might slow disease progression, some ALS patients decided on their own to try the drug. PatientsLikeMe built a data collection tool where participants could enter information about their symptoms, treatment, and outcomes, using the Revised Functional Rating Scale to measure disease progression. After 12 months, 149 people who took the drug met inclusion and exclusion criteria for analysis.’

The analysis showed no significant treatment effect from lithium, which was in agreement with the findings of other, larger clinical trials, which suggests that the online registry approach for the study was useful. For the authors of the article, this confirms that online data-sharing registries may be useful complements to traditional clinical trials.

I agree. We need as much data as we can gather about diseases, and even more so for rare diseases. Building platforms that can capture such data in a reliable way is therefore crucial. More about this in subsequent posts as we progress with our plans for an online registry.

The challenge of being a small rare disease patient group

We’re moving office today. Our old office was in a place in Cambridge called the Social Enterprise Centre. The facilities were a bit run down, but it was a fun place to work, with lots of other small charities and budding businesses and a great sense of entrepreneurship. It was in a building earmarked for demolition. That’s why the social enterprise that ran it (called FutureBusiness) managed to get such a low rent for its tenants. The landlord was willing to rent it out for very cheap while he sorted out selling it.

So a couple of months ago we were given notice that they were going to demolish the building in order to build a hotel. FutureBusiness managed to find new office space in the top floor of a DIY warehouse. It’s temporary accommodation while they raise finance to build a brand new social enterprise centre in the north of Cambridge. We’re doing the move today, with one of our volunteers (an AKU patient) hiring a van and recruiting his sons to help us shift our stuff.

Why do I tell you this story? To show you some of the challenges facing rare disease patient groups. Like most other small charities, we have very little money and depend on the goodwill of others to find us affordable office accommodation and other resources. We depend on the work of volunteers, including dedicated AKU patients, to help us move forward.

And yet, the AKU Society is one of the more developed rare disease patient groups. Most other ones don’t even have an office or dedicated staff. How can we expect them to make a significant difference for their patients if they don’t have the resources to do so?

That’s why I’m convinced of the urgent need to build the capacity of the rare disease patient group sector. We need foundations that will support them with more than just a few hundred pounds. We need affordable training for their staff and volunteers. We need a concerted effort at organising patient groups and helping them interact with academia and industry.

A few of us are getting together over the coming months to see what we can do. If you’re interested, drop me an email at nick@akusociety.org.