The other day I was asked which moments from my time at CureGRIN stand out as especially meaningful.
One, I replied, was seeing Radiprodil move into the first Phase 3 clinical trial for a drug especially designed for GRI Disorders. The other was when CureGRIN decided to expand beyond GRIN to also seek treatments and cures for people living with GRIA, GRIK and GRID Disorders.
Taken together, these two milestones highlight both a gap and an opportunity. While the Radiprodil study is significant for people with Gain-of-Function GRIN Disorders, more than 70% of CureGRIN’s population is not currently eligible for the trial.
And it’s becoming increasingly clear that different disorders and variants will not only require different medicines, but also different roadmaps to get to those medicines.
CureGRIN’s board recently voted to approve a new strategic plan – including a new mission statement and ambition statement – that directly addresses this reality over the next four years.
The plan does this in a few key ways:
- Gene-specific advisory boards: In addition to our broad Science and Medical Advisory Board, we are setting up four distinct advisory groups dedicated to each gene family (GRIA, GRID, GRIK and GRIN). These new groups will identify and prioritize the top gaps towards clinical trial readiness for each disorder. Addressing these gaps could involve funding scientists to answer a specific research question or developing scientific assets such as a new mouse model. We believe this approach will allow us to accelerate progress on disorders with larger populations like GRIN1 and GRIN2B, while allowing disorders with smaller populations like GRIA1 or GRIK2 to catch up. The key is to focus on what matters most for each disorder, rather than applying a one-size-fits-all model across our genes.
- Recognition of non-traditional trials: It would be ideal if private biopharma companies would run clinical trials that would cover every GRI variant, but that’s probably not going to happen any time soon. That’s why our new plan recognizes the value of non-traditional trials. This could involve a single family, or small group of families, fundraising to pay scientists and physicians to develop and administer a genetic treatment. While non-traditional trials don’t usually lead directly to regulatory approval that would help a large patient population, they can provide a proof-of-concept for biopharma that a type of treatment can work.
- Focusing on what matters most. Our previous strategic plan included three pillars, one focused on science and research, one focused on support and community, and one focused on fundraising and operations. Our new strategic plan expands to five different pillars, of which three are focused on getting to treatments and cures through science and research.
Keith McArthur
Executive Director
