Growing Forward: Channeling Hope in Every Season
It was a productive and impactful summer at the Channeling Hope Foundation! In this newsletter, we’re sharing updates on our research and advocacy efforts, highlighting progress across our community, and providing resources to support families as we enter the next season.
Summer of Science
Our scientists stayed extremely busy finalizing the first-ever clinical trial protocol to test a treatment for an NALCN-related disease. Three years ago, Jeremy Tanner (Chief Scientific Officer) set out to build an international, multi-disciplinary team to repurpose existing FDA-approved drugs for CLIFAHDD. After a series of experiments in the U.S. and Europe and clinical surveys completed by caregivers, all the hard work has paid off! On August 28, 2026, the FDA approved the Investigational New Drug (IND) application for our first clinical trial:
IND clearance means the FDA has reviewed the safety and mechanistic data supporting the repurposing of aprepitant for CLIFAHDD and authorized the research team at UTHealth San Antonio, led by Drs. Megan Iammarino and Jeremy Tanner, to proceed with enrolling participants.
This is real, tangible progress toward the first clinical trial for CLIFAHDD. Thank you to everyone in this community who has contributed to our “clinical trial readiness.” Every survey completed, dollar donated, and minute spent building community and inspiring scientists have made this moment possible. Families who would like to learn more, including how to participate, should ensure they have completed our patient contact registry and indicated interest in clinical trial research.
We "rounded" out the summer by convening 25 scientists from 3 continents and 7 time zones at a research roundtable. Held a few times each year, these meetings strengthen collaborative relationships, increase knowledge sharing across scientific disciplines, and lead to new research projects inspired by our community, including ones funded by the NIH (National Institutes of Health)! NALCN Research Roundtables are open to any scientist/research clinician interested in NALCN science. Email info@channelinghope.org to be invited.
Fundraising Fun
Throughout the summer months, families around the globe shared meaningful mobility milestones and other moments of progress—reminders of the individuals at the center of our work.
They also led fundraising events to contribute to our $2.8 million Raising Hope Campaign. Thousands were raised from selling t-shirts (in Tennessee), lemonade (in Kansas), and flowers (in the UK). We also launched Hope Giving Society to amplify and sustain our efforts through consistent, dependable, monthly giving. We invite everyone in our community – scientists, families, health providers – to give what they can so that we can keep advancing NALCN research at a record pace.
Silvie sold flowers that she grew to support the Raising Hope Campaign
Advocacy in Action
Shayanne Martin (Executive Director) took her advocacy on the road this summer, completing the Archer Fellowship Program, a premier public service and leadership program that supports University of Texas System graduate students to live, study, and intern in Washington, D.C. She researched and advocated for FDA policy reforms that would accelerate evidence generation, clinical research, and regulatory review for ultra-rare disease treatments. She also interned with the RARE Foundation (formerly EveryLife Foundation for Rare Diseases) to develop resource materials on Patient-Focused Drug Development (PFDD) and regulatory flexibility for rare diseases.
From left to right, Shayanne with RARE Foundation's Director of Policy Research after a Rare Disease Caucus Congressional Briefing, speaking at a public FDA meeting on drug repurposing, and graduating from the Archer Fellowship
Before returning to Texas, Shayanne spoke at an FDA meeting on drug repurposing to advocate for a regulatory system built for patient-led science:
Back-to-School Resources We Love
Though there is no limit to what our community can do. This month we are highlighting incredible back-to-school resources from rare disease partners.
Rory sporting an accessible backpack fit for wearing or riding along.
The Pediatric Epilepsy Surgery Alliance (PESA) offers a free, self-paced course on navigating the IEP process (now available in English, Spanish, Mandarin, Tagalog, and Vietnamese) that walks you through what to ask for, how evaluations work, and how to advocate when you disagree with the school.
- IEP Course: learn.pesalliance.org/p/iep-101
- Facebook Group: facebook.com/groups/509508329448387
- How to Get a 1:1 Aide: epilepsysurgeryalliance.org/2021/07/12/school-aide
You are your child's strongest advocate. Let these tools carry some of the weight so you can focus on supporting their learning.
Global Genes offers RARE Toolkits, a library of free guides, videos, and podcasts built for the rare disease community, including toolkits on self-advocacy and bringing your family's story to policymakers. This is good reading for parents navigating school systems or preparing to advocate at any level.
NORD (National Organization for Rare Disorders) has Caregiver Resources such as free webinars, fact sheets, and guides on self-advocacy, insurance navigation, and care coordination, plus the RareEdu® learning platform and a caregiver respite program that helps fund professional in-home care.
The Child Neurology Foundation Back to School guide provides practical tips for the first day (teacher info sheets, pre-visits, updated action plans), a plain-language walk-through of IEPs and 504 plans, guidance on Medicaid services in schools, and a free Special Education Resource Guide.
DEE-P Connections Education Resources include webinars and slide decks on building your child's IEP (including one geared toward children with severe DEEs), plus links to the Council of Parent Attorneys and Advocates (COPAA) for families who need legal support.
Changing Seasons, Constant Commitment
The seasons turn, but our focus on building a global community of families and researchers to advance care and develop treatments for individuals affected by NALCN channel-related diseases never changes.
Our work is not slowing down with the arrival of fall. In many ways, this next season represents an opportunity to build on the momentum of the past several months and continue moving the field forward.
No matter the season, the team at the Channeling Hope Foundation is here to help. If you ever need anything, please reach out on social media or email emily@channelinghope.org.