Ataxia UK has launched a new a collaborative drug discovery programme in Friedreich’s ataxia, a recessive inherited condition that causes progressive damage to the nervous system, with pharmaceutical company Pfizer and researchers from three major universities as part of its 2020 initiative. Here, Dr Julie Greenfield, Research Projects Manager at Ataxia UK tells AMRC more about this project and why working with industry, they hope, will help them achieve their goal.
The drug discovery programme
The 2020 initiative, was developed in 2012 as Ataxia UK’s research strategy and aims to find a treatment or cure for one or more of the ataxias by 2020. This new drug discovery project will see over the next three years scientists Dr Paola Giunti, University College London, Professor Richard Festenstein, Imperial College London and Dr Michele Lufino, Oxford University working together with Pfizer scientists to develop a new potential therapy for patients with Friedreich’s ataxia.
“When we launched our new research strategy at the end of 2012, we were very keen to be ambitious in our aims of finding treatments for the ataxias,” said Dr Greenfield. “As part of this initiative, we planned to engage more with pharmaceutical companies as we feel that it is only through working in partnership that we can achieve our vision.”
With Pfizer already having an interest in ataxia, having launched a Rare Disease Consortium back in 2013, they were a natural research partner for Ataxia UK.
“In the beginning it was a case of keeping in touch and seeing what we could do for each other,” said Dr Greenfield. “Then when they launched a new consortium, we thought it would be an ideal opportunity for us to bring together a group of experts with complimentary expertise and apply to this scheme.”
Although currently only a drug discovery programme, having the backing of a large pharmaceutical company is a real boost for the charity and big a step towards realising their 2020 goal.
“We’re hoping if it does go well, further investment will be made, and it could be taken all the way to a potential drug,” said Dr Greenfield.
It’s not all about the money
Ataxia UK has also put a small amount of funding towards the programme to maximise its chance of success, but the main role provided has been an advisory one.
“We have a lot of experience of the condition, both from being in touch with patients and also from understanding the research funding landscape,” said Dr Greenfield. “We feel this is helpful to pharmaceutical companies. In addition we can also be helpful in liaising between people with ataxia who are members of our charity, researchers and industry.”
Keep going and keep engaging
When it comes to charities collaborating with industry, the key is to keep going and to keep engaging according to Dr Greenfield.
“We ran an international ataxia research conference in March this year to bring together academic researchers, pharma companies and charities and it was great to see conversations happening between the them,” said Greenfield. “It was at a rare disease conference in Oxford that conversations between us and Pfizer Rare Disease Unit first began.”
What’s encouraging, according to Dr Greenfield, is that industry is becoming more receptive to rare diseases like the ataxias and are seeing the benefits to working with charities. This can be seen even by the fact pharmaceutical companies are employing staff to work with patients and patient groups.
“This is particularly good for smaller charities that don’t have the vast amounts of money but do have the expertise,” said Dr Greenfield. “That is still valuable and companies are understanding the value and appreciating it and now want to work in partnership.”
Want to know more?
Here at AMRC, we understand that for charities, working with industry can be hard. We’ve been working with many of you to understand the challenges you face so that we can support you in the best way possible. Our Essential Partnership report is full of really useful information on how to set up these collaborations, alongside almost 40 case studies showcasing how others have done this.
For more information on the drug discovery project. To contact Dr Julie Greenfield on the topic.
Want to know more about working with industry? Talk to Andy
 Rare disease consortium members include Oxford University, University College London, King’s College London, University of Cambridge, Imperial College London and Queen Mary University of London