The Wellcome Trust unveiled this week that rare disease research projects put forward by Pfizer and Lilly are the first to win funding under its new Pathfinder Awards scheme.

The Pathfinder Awards are designed to help get pilot projects that could potentially lead to the development of innovative products for the treatment of orphan or 'neglected' diseases off the ground, in the wider hope that this will help 'de-risk' future development in these areas.

Under the scheme, academic leads link up with companies that have relevant specialist knowledge and technologies to help facilitate the development of a specific product.

The Wellcome Trust will contribute up to £100,000 to each partnership, but whatever the amount the company involved must provide a matching contribution.

Neurological disorders

Lilly's winning project is centered on the development of a line of human stem cells that can be used to study Neurodegeneration with Brain Iron Accumulation (NBIA), a rare group of severe neurological disorders that affect children and young adults.

Despite the fact that a number of genes have already been linked to NBIA the mechanisms of the disease remain little understood and currently there are no treatments to help patients.

Working together with Professor John Hardy from the Department of Neuroscience at University College London, researchers at Lilly will develop an induced pluripotent human stem cell line to help shed more light on the underlying mechanism behind NBIA, and thus accelerate the development of new treatments for these disorders.

Researching Homocystinuria

The other winning project will see a team of researchers at Pfizer work with Dr Wyatt Yue at the Structural Genomics Consortium, University of Oxford, to tackle a rare, hereditary metabolic condition called Homocystinuria, which is characterised by an inability to metabolise the protein methionine and for which there is no cure.

The team plan to investigate methods of restoring normal metabolism and preventing the progression of the disease, and as such will study the enzyme affected by the disease and how to restore its normal function.

Commenting on the first round of the Awards, Ted Bianco, Director of Technology Transfer at the Wellcome Trust, said: "Orphan and neglected diseases present a huge challenge and we believe that successful collaborations between academia and industry will be vital for finding new treatments and diagnostics". 

"This is a key priority for the Pathfinder Awards and we are delighted that the first two projects to be supported under the scheme will foster such exciting cross-sectoral collaborations.”

The next deadline for applications is October 12 this year, and applicants should be notified of the outcome sometime in December, the Wellcome Trust said.