A wave of new ideas?

There has been a great deal of focus over the last few months on the work of IONIS pharmaceuticals who are developing a gene silencing drug (currently known as IONIS HTTrx); this is largely because they were the first to complete a successful phase one clinical trial and announce very promising results. In the subsequent media storm, the work of another company, Wave Life Sciences, appears to have been overlooked.

Wave are also developing a gene silencing drug which is just going into phase one clinical trials. This drug is different from the one IONIS is developing in one important way. The IONIS drug blocks the production of both the normal and faulty copy of Huntingtin (it’s the faulty copy of this protein that makes brain cells become sick and then die). IONIS have done lots of work to show that reducing the level of normal Huntingtin doesn’t cause harm in adults and the phase one clinical trial seems to confirm that.

It does however remain an open question as to whether this will be safe in the longer term.

Wave are taking a different approach; their drug only targets the faulty gene and therefore only reduces faulty Huntingtin. It also uses a slightly different approach to targeting the gene which makes this possible, but whereas IONIS are using one drug that can treat everyone with HD, Wave will need five variations of their drug to achieve he same thing. In their phase one trial which is getting off the ground, they are using two variations which can treat about 65% of people with HD.

The goal of both treatments is the same; to lower the levels of faulty Huntingtin with the expectation this will have an impact on the way the condition progresses and the symptoms it causes.

For people living with HD, this is all good news; both approaches may be successful, both may have potential drawbacks, but the chances of finding an approach that works are certainly improved by two different companies working on the problem. One thing is for sure, if Wave Life Sciences isn’t already on your ‘treatment radar’, it’s time to give them a ping.