CHF Newsletter - March 2024
Channeling Hope Foundation held a second "Friends and Family" webinar in February. The webinar was recorded for those that could not attend. Here's a summary of what we covered in the webinar.
INCLUSION PILLAR REVISITED
The Channeling Hope community of researchers and families are driven to advance care and develop treatments for those affected by any NALCN-related disease. The NALCN channel protein interacts with 3 other neuronal proteins (UNC79, UNC80, and FAM155A) to form the "NALCN Channelosome." This entire channel complex appears necessary for normal functioning of the ion channel and thus healthy neuron firing. As a patient advocacy organization and community, we strive to advance care, research, and treatment for patients and families affected by all diseases due to abnormal NALCN channel functioning, including due to mutations in the associated proteins (UNC79, UNC80). We wanted to better emphasize this in our messaging.
SCIENTIFIC UPDATES
Scientific updates and priorities were presented by Dr. Jeremy Tanner, CHF Chief Scientific Officer (2 minutes, 15 seconds). In the webinar, he explained the field of drug repurposing and discovery. Last year, Drs. Tanner, Arnaud Monteil (CHF's Scientific Advisory Board Director), Brandon Holmes (CHF Scientific Advisory Board), and Josiah Gerdts (CHF Scientific Advisory Board), collaborated with the team at the Center for Innovative Drug Discovery at the University of Texas Health San Antonio in San Antonio, Texas, USA, to try to discover potential drug candidates that can modulate NALCN activity. The team was awarded a $75,000 pilot grant to start this research.
“Drug discovery research is collaborative teamwork, requiring expertise from different disciplines, such as strong biomedical research and structural biology, to identify and validate targets.” – Dr. Daohong Zhou, Co-Director of Center for Innovative Drug Discovery
Dr. Tanner also shared how donations to Channeling Hope are supporting the generation of patient-derived iPSCs (stem cell lines) to serve as an open resource to the scientific community to support further research and treatment development for NALCN-related diseases. Dr. Tanner discussed the CHF Scientific Advisory Board and CHF's future plans to start a Patient Registry for NALCN-related diseases. This will allow patients in our community to contribute clinical data to support medical research that is necessary to better understand NALCN-related diseases and to test treatments.
RARE AS ONE GRANT
The Board of Directors of Channeling Hope Foundation recently completed an application for a five-year "RARE AS ONE" grant through the Chan Zuckerberg Initiative. Rare as One is in Cycle 3 of funding and is specifically looking for patient-led disease advocacy organizations that represent channelopathies diseases, a heterogeneous group of disorders resulting from the dysfunction of ion channels located in the membranes of all cells and many cellular organelles.
Rare As One grantee organizations will (1) accelerate research within individual disease areas and (2) work across specified areas of scientific focus to develop shared research priorities and proposals.
Ambassadors of Hope: Call for Volunteers
There is so much to do to keep Channeling Hope Foundation moving forward for the benefit of the patients. The list of needs for our organization was shared with all the webinar attendees to recruit volunteers. Any support, no matter how small, helps advance the mission of CHF! If you would like to get involved, please email us at channelinghopefoundation.org.
THANK YOU!
We were overwhelmed by the positive response from our community after this webinar. In addition to moving the science along, we are making the following a priority in the coming weeks:
Provide talking points on what we're raising money for and how donations have already supported research
Involve new family members in our volunteer efforts - If we haven't yet spoken one-on-one, please contact us so we can discuss how you can get involved.
Show our gratitude to our donors
Rare Disease Day 2024
Our first Rare Disease Day as an organization was a huge success! Several families raised awareness by sharing their stories, some for the first time. A rare disease diagnosis is difficult to process and it often takes time to be okay opening up about it. Many families started new fundraisers to honor of their family member with an NALCN-related disease. All donations go directly to supporting the mission of the Channeling Hope Foundation.
Our mission is to build a global community of NALCN families and researchers to advance care and develop treatments for individuals affected by NALCN-related diseases.
Thank you to the following organizations who supported community fundraisers:
- Webster/Rochester, NY, USA: Ridge Donuts, Schutt’s Apple Mill, Secret Ingredient Cupcakery
- Montezuma, KS, USA: The Sister's Boutique, Coffee Connection
Tyler Diedrich
If you wish you submit something to your local newspaper or news broadcast station on Channeling Hope Foundation or NALCN-related diseases, please contact Jill Wahl, Director of Marketing and Communications, for assistance and support at channelinghopefoundation.jwahl@gmail.com
