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Scientific advice is crucial in the rare disease world. With few researchers working on any given rare disease it is important for patient groups to build connections with the scientific community. This helps them to understand their disease, gives them a platform to encourage appropriate patient focussed research, and also helps the scientists themselves truly understand the impact of their work.

Scientific advice is just as vital to Findacure. While we don’t focus on a single rare disease, we do run a number of scientific projects on different conditions and we are planning to expand this as our drug repurposing programme develops. Independent scientists with a knowledge of rare diseases and human biology help us to identify the most promising projects, based on the best science, and to ensure that the work we do is relevant to the clinical and research communities, as well as patient groups.

Luckily, we have been well supported by our Scientific Advisory Board since it was formed three years ago. This group of eleven clinicians and researchers have lent their support to Findacure and the idea that research into rare diseases is fundamental to medical science. Board members such as Dr Farid Khan have given talks at our scientific conferences, Professor Sir Tom Blundell supported our first networking event with a lightning talk on structural biology, and Dr Anil Mehta, among others, has acted as a judge in our student essay competition. All have provided invaluable support to Rick since he started at Findacure, and we’re grateful to each and every one of them for their time and advice.

Given their service on the board for over three years, and our increased focus on drug repurposing for rare diseases, we decided at the end of 2016 that we would reform the board. We have formed a smaller Science Advisory Committee, to allow us to meet on a regular basis. The committee will review all of Findacure’s scientific projects and provide crucial support on developing our drug repurposing programme. These changes gave existing Science Advisory Board members the opportunity to step back, while allowing new expertise to benefit the charity. The eagle-eyed amongst you will have noticed our new committee members on our website, but we thought it would be great to introduce them all here.

Dr Bruce Bloom is the President and Chief Scientific Officer of the US charity Cures Within Reach. Bruce has continued in his role as a scientific adviser, with Cures Within Reach being a partner in the development of our Rare Disease Drug Repurposing Social Impact Bond (SIB).

Dr David Cavalla has 30 years of experience in drug development and drug repurposing, invaluable experience for our own repurposing project. He is author of a recent book on Off-label Medicines – a complex area allowing doctors – to prescribe drugs outside the normal bounds of the therapeutic label. This is a practice that many rare disease patients rely on for their medicines.

“I gladly joined the Findacure SAC because the team at Findacure understands that there is huge untapped clinical potential for rare disease patients in drug repurposing.  Our work with Findacure allows us to pioneer new financial and other incentives to encourage scientists, physicians, government and industry to participate in the repurposing of generic drugs to create effective treatments for rare disease patients.” – Dr Bruce Bloom

Dr Mark Edwards is medically qualified, and has 25 years of experience in pharmaceutical research and development. He now provides strategic advice to organisations in the public, private and charitable sectors, and has a wealth of contacts in the life sciences world, all of which will be hugely beneficial to Findacure.

Dr Kathryn Johnson is another new committee member, and new to Findacure. She is a neonatal paediatrician based in Leeds with experience in both rare diseases and clinical trial delivery. With the majority of rare diseases affecting children, many from birth, an understanding of the challenges of paediatric research will be a huge help to the charity.

“Rare disease drug development is a rapidly expanding and exciting subject and a great field to be involved in at the present time. As a paediatrician I want to ensure that all children with rare disease have the opportunity to benefit from the work Findacure is doing.” – Dr Kathryn Johnson

Dr Edmund Jessop has been working at NHS England since 1981, and is heavily involved in the commissioning of specialised services for some of the very rarest diseases. He brings a wealth of experience in the functioning of the NHS that has already proved invaluable to our SIB project.

Margaret Ogden, a patient herself, has extensive experience in both rare disease science and patient engagement. Her ability to speak from multiple perspectives will be a valuable asset on our Scientific Advisory Committee.

As a volunteer in patient and public involvement activities for over a decade, I have developed a passion for research into rare diseases. I was diagnosed with Wegener’s Granulomatosis, a rare autoimmune disease, in 2002 and believe that patients affected by rare diseases should be a fundamental focus at the heart of research into diagnosis, treatment and cure. Over the years my interest and involvement has become more science based, prompting my membership to Findacure and subsequent appointment to the Scientific Advisory Committee. I hope to use my experience and knowledge to the important work of this Committee.” – Margaret Ogden

Prof. Lakshminarayan “Ranga” Ranganath has continued in his position as a scientific advisor. Prof. Ranga runs the National Alkaptonuria Centre in Liverpool and is leading on a drug repurposing clinical trial. This experience is vital for our SIB project, as well as Prof. Ranga’s clear focus on patient need and patient experience.

Our final committee member is Dr Kamlesh Sheth, a long-time supporter of Findacure who is currently in his second year as a peer mentor, and has previously spoken at one of our training workshops. Kamlesh has over 25 years of experience in the life science industry, and is a fellow of the Faculty of Pharmaceutical Medical.

“Why did I decide to work with Findacure? We share the same vision and passion to develop treatments for rare disease patients and empower patient advocacy groups.” – Dr Kamlesh Sheth

Everyone at Findacure is really excited to work with this new advisory committee, and want to thank them for getting involved with Findacure for the next two years.

The science of rare diseases – our revamped scientific advisory committee

by Rick Thompson time to read: 4 min