Working collaboratively to accelerate discovery of disease-modifying therapies for Parkinson’s (and related conditions)

November 28, 2024

Mention Parkinson’s disease and word association leads many people to “Michael J Fox”. The well-known Canadian actor has made a substantial contribution to entertainment since the 1980s from his role as Alex Keaton on the sitcom Family Ties through to the films Back to Future (and sequels) and Teen Wolf, among countless other film and TV appearances. But his greatest legacy will undoubtedly be as a Parkinson’s advocate, especially through the Foundation that bears his name, The Michael J Fox Foundation for Parkinson’s Research, which has invested over US$2B into research over the last couple of decades, driving significant progress.

It was thus a great honour to be invited to attend the inaugural NeuroImpact Coalition Workshop at the MJFF offices in New York City this month, as part of a global coalition of non-profits working together, strategically, to overcome hurdles and drive the development of the first-ever disease-modifying therapies for Parkinson’s (treatments that can stop or slow progression of the disease). Together with representatives from our partners MJFF, Cure Parkinson’s (UK), and Shake it Up Australia, as well as Parkinson’s UK and new friends from Parkinson’s Canada, Parkinson’s Foundation and Critical Path Institute (among others), the various challenges in getting such treatments approved were discussed over two days.

The workshop covered not only Parkinson’s, but also the closely related diseases Dementia with Lewy Bodies (DLB) and Multiple Systems Atrophy (MSA). Importantly, there were representatives of all three disease communities present, to share the needs and priorities of people with these diseases and their care partners. Adding academic heft to the discussions were several academic representatives from Europe, US and the UK, while industry representatives from several pharmaceutical companies actively developing therapies for Parkinson’s and related conditions also shared their perspectives and priorities.

Although the MJFF offices are in central Manhattan, the building was fairly non-descript with no signage or showy glitz—this is a Foundation that is serious about research investment and outcomes, and not there for show. Many of the staff work remotely coming into the office a couple of times a month, so they occupy just two floors. We were in the Boardroom, and there were just a few giveaway signs of where we were, including the orange wall paint (on brand), a collection of TV awards, and Marty McFly’s trademark boots (see image). But it was a packed agenda with lots to cover, so it was straight down to business after an early breakfast.

What made this meeting special, in addition to the collaborative nature of it, was the learnings brought to it from experts in other fields, in particular Alzheimer’s disease and multiple sclerosis. In a relatively short time, these fields have progressed from where Parkinson’s research is now, to having genuine disease-modifying therapies. There was much to learn from these experts, both in terms of the mistakes made and the successes. There remain significant challenges to getting disease-modifying therapies approved for Parkinson’s, not least of which include identifying and validating biomarkers of disease progression (we had a first win there last year), recognizing different biological subtypes (and enrolling patients and targeting therapies accordingly), determining how best to measure the effect of any treatment over a short enough period to make a clinical trial feasible, and even defining what “meaningful benefit” might look like.

The perspectives of diverse stakeholder from patients to their families and caregivers, to clinicians, payers (in NZ this is Pharmac), regulators (FDA in the US, EMA in Europe, Medsafe here in NZ), and industry (the pharmaceutical companies who will likely need to be involved as partners to push candidate drugs through the later stages of clinical development), all needed to be taken into account. After all, we need the drugs that show promise to ultimately be proven to be safe and effective, in a meaningful way for patients, leading to approval and ideally acceptance under whatever local payment systems exist. Regulators will consider safety as well as whether a new treatment can work better than existing treatments, while the payers take a health economics perspective (does the benefit justify the cost).

Breakout sessions on day 2 then explored how the learnings and experiences shared by diverse presenters could be applied to each of the disease areas being discussed, including robust discussions around the selection of clinical trial outcome measures and how to define benefit.

Some fabulous new contacts were made and existing relationships were strengthened as we all worked to develop an actionable roadmap for how to assess the cumulative benefits of new treatments for Parkinson’s, DLB and MSA. From a NZ context, it has become clear that we need to create a database of people with Parkinson’s who would want to participate in a clinical trial, including information on genetics, symptoms, lifestyle and life history among other key pieces of the puzzle. As we continue to grow as an organisation, enabling us to fund more research, including clinical trials, this resource will become invaluable to identify the people most likely to benefit, and to attract international investment here for new drug trials. The future starts now, and we have much to do, but it is an exciting time and wonderful being part of such a proactive global community.

Daniel (CEO)