Ice bucket challenge...will it help?

Iain Moal offers his take on the Ice Bucket Challenge The ALS ice bucket challenge, a campaign to raise funds and awareness for amyotrophic lateral sclerosis (ALS), has taken social media by storm and spurred a host of celebrity endorsements. Thousands have doused themselves with ice cold water before challenging others to do the same, or reach into their pockets, inciting enthusiasm and cynicism in equal measure. ALS charities have reported massive increases in donations, much of which will be ear-marked for research. Looking past the media frenzy, detractors have questioned whether the endeavour will have any impact at all, pointing out that the boost in donations is a drop in the ocean compared the amount spend on medical research. However, they fail to realise just how much ALS research, and rare disease research in general, relies upon funds from public and charitable bodies. With drug pipelines drying up, pharmaceutical companies cannot act if it isn't in their financial interest, and there simply aren't enough ALS patients to make it worth their while. Failure can strike at any time on the decade long journey from finding a molecular target for a disease to having a compound that is proven safe, and even then half fail the final hurdle of actually being shown to be effective. Approaching US$2 billion, the cost of developing a drug from scratch makes the donations from the ice bucket challenge seem insignificant, but it isn't the only option. Drugs rarely hit only their intended target, and unforeseen effects can be opportunities to repurpose existing or obsolete therapeutics, or medicines that have been abandoned in the drug development gauntlet. After all, many have enjoyed benefiting from the extraneous effects of sildenafil, originally developed for hypertension and better known as Viagra, but these effects were obvious. An accidental ability to treat ALS won't be found serendipitously. The pharmaceutical industry lacks the profit motive to lay the ground work in tackling rare diseases, which collectively affect hundreds of millions. For many of them, prospects are bleak and without the innovation of researchers supported by charities and public institutions, it will remain that way. Whether the ice bucket challenge succeeds in gaining long-term supporters remains to be seen, but there is no doubt that it will open up new avenues of exploration which just might hasten the breakthrough that so many are hoping for. Dr Iain H. Moal is involved in the initial stages of a project aiming to find drug candidates for rare diseases including ALS as part of the Protein Interactions and Docking group at the Barcelona Supercomputing Centre.