Wave is currently testing a new treatment known as ‘Precision medicine’. The drug only targets the disease-causing protein and leaves the healthy protein alone.
Video interview below!
In July 2017, Wave Life Sciences started their clinical trial known as ‘Precision HD’. The trial is a so-called Phase I clinical trial – which means it is a first-in-human trial testing a specific drug.
Phase I is conducted with a small number of patients (Roche’s Huntingtin lowering trial was for instance carried out in 46 participants). In this phase, the researchers are mainly concerned with safety and tolerability.
For instance: how does the body react to the drug? How big should the dosage be? Is there any side effects? And so on.
Wave will hopefully have answers to these questions in the first half of 2019.
So what’s new?
Wave’s treatment is taking a so-called Huntingtin-lowering approach. In other words: the level of Huntingtin protein is lowered in the brain. This protein is responsible for causing Huntington’s disease.
With that being said, all humans have a certain level of Huntingtin in their brain – but only a small percentage has Huntington’s disease.
In a human being there are always two copies of the Huntingtin gene which codes for the protein Huntingtin. In a person without Huntington’s disease the two genes, or alleles, will both code for healthy Huntingtin, or wild-type Huntingtin.
In a person with HD, one of the alleles will code for mutant Huntingtin, while the other will code for healthy Huntingtin. This mutant Huntingtin causes Huntington’s Disease.
Read more: Wave partners with Takeda
So instead of shut-down the total level of Huntingtin, Wave only target the mutant Huntingtin – which is a totally unique and new approach. This precise targeting is the reason for the term ‘precision medicine’.
The specific role of the healthy Huntingtin protein is not fully understood but it’s most likely there for a reason. That’s why researchers might believe the healthy, wild-type Huntingtin is important for the long-term care in people with HD.
There are many exciting approaches being tested in the Huntington research scene – and Wave’s approach is definitely one of them.