Collaboration between industry and patient groups

During the May Awareness Month this year I have been visiting 3 pharmaceutical companies with upcoming or ongoing trials in HD. It has been inspiring to meet with all the people who work to realize therapies for pateints.

 

We have had fruitful discussions about facilitators and hurdles in the long process from laboratory to patients. I have told stories and tried to express the experiences from people affected about how the disease is influencing life choices and everyday life and family relations. During my last visit to Basel this week, I visited the Novartis Pavillion to see the exhibition over the history of medical knowlegde about the human body and where we are in drug development. 

 

What stuck out to me is that we have come along way. Nevertheless, we still dont have any disease modifying therapies allthough we are moving in the right direction. 

 

The 3 companies I been visiting, Ferrer, Uniqure and Novartis have all different approaches to how to treat HD. Different therapies, different modes of action. Two are pills or oral medicines, one is given to patients through brain surgery procedures. Two are for daily in-take, one is a one time treatment. Two are targeting the toxic Huntingtin protein, one is aming to strenghten cell functions. I think we need them all – there will not be one treatment fixing it all. 

 

We see the same in other diseases like Multiple Scleroses (MS) for instance. The first therapy for MS was approved in 1993. Today there are a large number of medicines approved and the number is growing. Many MS patients can live more or less unaffected by the disease thanks to good therapies. I think the same will be the case for HD.

 

However, I realize that HD families has been waiting for a looong time and some are loosing hope. One of the wonderful HD-CAB advocates, Tanita Allen, wrote in a recently published article: “Hope is oxygen” She also express that hope is a major reason for people to sign up for a clinical trial. We need to support each other and provide information to nurish hope. In order to accelerate development of medicines, we need to take part.  Another member of the HD community, BJ Viau, presented a bold vision: “Huntington will be the fastest clinical trial recruiting community in the Rare Disease world”.

 

I believe that is possible. And that is why I visit drug developers and discuss how we can collaborate. I want trials to be done in a way that takes best possible care of the participants needs and wishes. We want trials to be recruiting as efficient and fast as possible. Because time matters. Slow recruitment is one of the main contributors to time delays in trials. And we dont have any time to loose. Only by taking a pro-active approach can the community help speeding things up. Because time matters!