The announcement that UniQure’s clinical trial had achieved success at slowing the advancement of Huntingon’s disease has brought the community a new sense of hope. But alongside this are complex feelings of grief and uncertainty for a future that had seemed inevitable but now carries with it a question mark. Erin Paterson gives her personal and candid thoughts on this recent breakthrough

By Erin Paterson

In the week following the news of UniQure’s trial successfully slowing the progression of Huntington’s disease (HD), I found myself breaking down in tears. I have been going about my life, doing the groceries, going to the gym, cleaning the house…and the tears keep coming unexpectedly. I am happy about the news, but that is not how it feels in my body. I have come to realise I am experiencing grief.

There are so many mixed emotions in the community — and that is completely understandable.

This is the positive story the HD community has been waiting for. UniQure announced that its investigational gene therapy can slow progression of the disease. The therapy is being used in clinical trials to lower huntingtin (a disease gene linked to HD), while also exploring safety. As a clinical trial it is not available to the community yet, but this is the first time a disease-modifying treatment has been on the horizon. This is a huge advancement for the HD community and we are all celebrating.

But…

We are still in the trenches taking care of our sick loved ones. Whether you are parenting a child with juvenile-onset Huntington’s disease (JHD), caregiving for a parent or supporting multiple people impacted by the disease, the reality of our daily lives has not changed. For many of those we love and care for, this breakthrough has not arrived in time.

There is fear that, if it’s approved, this treatment may not be accessible for everyone. We worry that it might not be available in our country, we might not qualify for treatment, we will not be able to afford it or our symptoms will be too advanced. We also know there are a lot of hurdles to overcome before this treatment reaches the community. It needs to go through peer review and be approved by the FDA — and that’s just in the USA. Then it has to be approved and rolled out around the world. It feels treacherous to get our hopes up too high.

This announcement brings to mind all of those who have already suffered and lost their lives to this disease. The potential treatment is too late for them, but that doesn’t stop us from wishing that things were different, that we could have them back, that they could have benefitted from this as well. Imagine how different our lives could have been.

HD is a part of our identity. Whether we are gene positive, gene negative or untested, we have worked really hard to accept our fate in life. This announcement represents a fundamental shift in how we see ourselves. Although the shift is good, it’s uncomfortable. Now there is the possibility of a brighter future, and that can be unsettling because we don’t know what our future looks like anymore.

No matter how this news has impacted you, no matter what emotions you are experiencing, even if you can’t quite identify what you are feeling, it’s OK to feel the way that you do. Your emotions are NOT wrong.

Living with HD in the family is very complex, and so is thinking about this potential treatment. Not only do we need to consider the impact for ourselves personally, we also have to think of everyone in our family.

And soon, if we are lucky, and this treatment is approved in the countries where we live, we will face another set of important questions. Do I want to go through this treatment? Is this the right treatment for me? It’s a very personal decision, and everyone has the right to make their own choices around accessing treatment, or not, just as they do with deciding to go through genetic testing or having children who are at risk. This potential treatment involves a lengthy brain surgery. Perhaps now that huntingtin-lowering could be a viable strategy, it will push along other treatments in development, and maybe those won’t be as invasive.

There are so many unknowns.

For now I am focusing on the renewed HOPE this announcement has given me and trying to shift my thinking from being someone who is 100% guaranteed to get HD, because I am gene positive, to someone who just MIGHT not. I have to come to terms with this new reality.

I am beyond grateful to everyone who has worked so tirelessly to get us to this point, including all the scientists searching for a treatment, the drug companies investing in finding effective treatments and the brave individuals who participated not only in this clinical trial but in all the ones that have come before. I am grateful as well for the patient advocacy organisations that have been supporting us along the way, not just with understanding clinical trials but also with the complexity of our everyday lives. We are all part of a wonderful community and we will be there to support each other no matter what.

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