Bright Lights Big City: HD-COPE descends upon New York City

The HD-COPE committee held its second annual meeting in the Big Apple, February 21-24, 2019. This year the committee welcomed members from Africa, Australia, China and South America. Now making it an international power house in the HD community. 

Written by Dina de Sousa, member of HD-COPE

HD-COPE, the Huntington’s Disease Coalition for Patient Engagement, was formed last year and met for the first time in London. 

Read more: HD-COPE’s first meeting

This year the second annual meeting was in NYC and the team expanded to include members from Africa, Australia, China and South America. Expanding its wings to reach Huntington disease (HD) families in the all corners of the world.

Plans for the future

HD-COPE is a global coalition to give families who are affected by HD a direct and impactful voice in HD clinical research.

In New York, the coalition had four days of brain storming, team building, presentations and meeting with two pharmaceutical companies: Roche Pharmaceuticals and Wave Life Sciences.

The two companies is currently testing different approaches to lower the level of mutant Huntingtin protein. 

During the meetings, it was forward planning into the future with their clinical trials.

Families – the ultimate experts

Up until recently pharma would have these discussions among themselves and come up with what they thought were reasonable solutions.

But who are the experts in a disease? Pharma may know the science, but families are the ultimate experts.

And with HD being such a complex disease this is even more so. Unless you are living with the disease or caring for someone it is very hard to understand the magnitude of what goes on.

Like an iceberg, what people see on a daily basis is the tip out of the water, what really happens is the enormity of the iceberg below the surface. There is no “one size fits all” in HD.

Pharma is listening

Families have a voice and Pharma is listening. As a team we were able to share our opinions, thoughts to their questions, concerns and vice versa.

We helped structure the course of their ideas and plans for their clinical trials. For instance: what would be a reasonable clinic day for a patient/carer? And what are the moral and ethical issues with the trials?

Meeting with CHDI

One of the afternoons we had the opportunity to visit CHDI Foundation’s office.

CHDI, Cure Huntington’s Disease Initiative, is a non-profit, philanthropic foundation with ONE aim: to rapidly discover and develop drugs that delay or slow the progression of Huntington disease.

How many other rare diseases have this kind of team in their court? Very few. CHDI work as a “virtual” biotechnology model funding third party research organizations.

Robi Blumenstein, President, gave us an introduction, history and vision of CHDI. Robert Pacifici, CSO, an overview of some of the research and Eileen Neacy, COO, an update on Enroll-HD.

A new pill?

All of the presentations at CHDI were top notch but the one that caught my attention.

Anu Bhattacharyya from PTC Therapeutics told us about their upcoming approach to lower Huntingtin. Their aim is to develop a small molecule, which can be delivered through a pill. Wouldn’t that be great?

This just reinforces that there are so many other companies all with same aim, lowering Huntingtin, but via different administration.

Read more: Huntington disease research

I firmly believe that a treatment for HD will be via a cocktail of drugs and not just one.

A unique team

It was wonderful to reunite with all team members and meet new ones.

In amongst all the work there was also time to socialise, eat, drink, chat, solve the world problems and strengthen our friendships.

I can speak for all of us on the team that it is an honour to work so closely with so many people with one common goal: a treatment for Huntington disease.

Led by an exceptional advisory board, HD-COPE is a ground-breaking initiative. The first of its kind worldwide and we are privileged to learned from each other and share our thoughts and have an impact with the pharmaceutical companies.

We are on the precipice of something great in HD history. This has all been possible due to such a strong HD community, patients, carers, professionals, scientists etc.

We are unique as a rare disease team.

Dina de Sousa