Identifying possible drugs to treat MND

The MND Research Institute have two programmes developing new drugs, and these work in tandem with other initiatives nationally and internationally.

(i) Cell models

When a new MND gene is discovered, or we learn about a new biological pathway that is involved (see Understanding the patient and disease process), we can use that knowledge to search for a way to cancel out the problem. An effective way to do this searching is through using cell models. These are laboratory cells of various types that can be manipulated to understand why the MND gene or pathway affect MND, and what could be done to stop that happening. Traditionally, these studies have been done in a single laboratory, and do not always show the same results when tested elsewhere, because the conditions are not always the same, or the cells used might differ. That means we cannot easily tell if the reason a second experiment did not confirm the findings of a first is because of a difference in the way the second experiment was carried out, or because that gene or pathway really does not work like that. In a global first, UK MND Research Institute researchers have developed a network of cell lines across multiple laboratories, all using the same sets of cells and all using the same protocols. That means any findings in one laboratory can really be checked in another in a reliable way.

What we gain from this:

We can now quickly and reliably confirm laboratory findings that suggest how to change what is happening in the cells in MND, a crucial step in developing new treatments.

Identifying possible drugs to treat MND

(ii) Developing Possible Drugs

The results from cell studies are then fed into a programme of work that identifies molecules or gene sequences that affect the different cell processes that matter. Depending on the responses, the number of possible molecules is narrowed down, and from those, a few will show promise in being non-toxic at likely doses, able to cross into the brain if given by mouth or injection, and worth understanding in more detail.

What we gain from this:

This method generates a set of possible drugs that could be tested in people. If these are known already (repurposed drugs) that is a quick route to trial. Otherwise the process is more laborious as great care has to be taken in ensuring safety and a lot of testing is needed.