Drug Company reports that RNAi could be a treatment for patients with SOD1 ALS

May 20, 2004

Recently media reports have discussed a new technology that may lead to a treatment for patients with ALS who have the SOD1 mutation.  The technique is known as RNA interference (RNAi).

The SOD1 mutations cause ALS in 2% of all people with ALS, or 20% of all familial ALS. Results in cell culture were very encouraging, showing that RNAi was effective in destroying the mutant SOD1 and preventing cell death in a toxicity model that may be associated with this genetic mutation.

While the initial work with RNAi is very encouraging and hopeful as a future treatment for people with the SOD1 mutation, the technical challenge of how to deliver RNAi to lower the levels of mutant SOD1 in humans remains. Currently, experimental efforts are underway to use viral delivery (gene therapy) as a system to administer RNAi. The ALS Association is funding several laboratory studies to address this area, visit http://www.alsa.org/research/projects.cfm? for more information.

A representative of the company developing the technology, CytRx, made a brief report on their RNAi work at an ALSA Advocacy Day workshop. The company plans to take all the necessary steps to initiate clinical trials in the near future.

More research is needed to develop a system that will deliver the RNAi compound to the proper locations and to further test animals; however, the technology is exciting and does offer realistic hope for patients with the SOD1 mutation. If successful, RNAi technology, which greatly reduces the genetic mutation, potentially could be effective with yet-to-be-discovered genetic mutations in ALS that, like the SOD1 mutation, cause a gain of toxic function.