The mission of Chelsea’s Hope is to improve the lives of those affected by Lafora Disease and help accelerate the development of treatments.
Chelsea’s Hope Lafora Children Research Fund is an IRS 501(c)3 non-profit organization. EIN: 27-1008382
Chelsea’s Hope c/o Dr. Donohue
976 Maywick Dr.
Lexington, KY 40504
Chelsea’s Hope Advisory Board Meets to Discuss Research Goals
/by Chelsea's HopeThe Chelsea’s Hope Advisory Board met in April to provide guidance and support for the organization’s clinical and research goals in 2025:
The primary topics of discussion were preparing for ION283 clinical trials, validating biomarkers for Lafora disease progression, and developing guidelines for seizure management for Lafora patients.
ION283 Safety Study
The ION283 Safety Study now has six patients enrolled. Dr. Berge Minassian, who directs the study at the University of Texas Southwestern Medical Center, assured the board that patients will be enrolled as soon as funds are available. Also, Noventia is continuing its preparations to launch a clinical trial for ION283 in Europe. The timeline and details of this trial are still under discussion. Members of the Advisory Board will make themselves available to Noventia when requested to provide expertise and insight to ensure the best care for Lafora patients.
Four patients wait to join the ION283 Safety Study.
Biomarker Research Goals
Before any potential Lafora therapy can begin clinical trials to establish efficacy, biomarkers for Lafora disease progression must be identified and validated. Chelsea’s Hope and the broader Lafora community have assisted with global efforts to collect patient samples for biomarker analysis. The data from the two-year Natural History Study sponsored by Ionis is now ready for analysis. Members of the Advisory Board have committed to participate in working groups over the summer to review the data and recommend promising biomarkers.
Centers of Excellence
While our organization continues to support the development of therapies, there are steps that we can take now to improve the quality of patient care worldwide. This year, Chelsea’s Hope will sponsor a series of discussion panels to establish Centers of Excellence for Lafora Disease and guidelines for patient care. The Advisory Board recommended that the organization prioritize the development of guidelines for seizure management in Lafora disease. Dr. Roberto Michelucci will direct this effort with support from Chelsea’s Hope as well as a cohort of clinicians who treat Lafora patients.
Questions?
For more information about the Chelsea’s Hope Advisory Board,the projects, or the research goals outlined in this news post, please contact our Scientific and Executive Director, Dr. Kit Donohue (katherine@chelseashope.org).
Announcing a Town Hall: Fully Funding the Safety Study
/by Christine KellyWe hope you will join us for a Zoom Town Hall next Wednesday, May 21st, at 4 p.m. ET, to discuss fully funding the safety study. Register to attend today!
We have less than a month to fundraise to enroll the final four patients on the original timeline. Learn more about our fundraising initiatives and how you can get involved when you join our Town Hall next week.
Six out of 10 patients have been enrolled in the ION283 Safety Study.
We will use Zoom’s closed caption translation feature. Please do not hesitate to join us if your first language is not English.
Wondering about what’s next for ION283 development? Check our ION283 Safety Study FAQs and stay tuned for more information about a Q&A in June. We will not discuss that next week; we will focus on fundraising planning.
Read 2024 Impact in our Annual Report
/by Christine KellyChelsea’s Hope recently published its 2024 Annual Report. Inside, you’ll find a letter from our President, a breakdown of our 2024 impact, financials, and what we’re working towards in 2025.
To our generous donors, dedicated research network, and community members, this is your impact, too. Every new resource or hopeful advancement to treatments is thanks to your support!
We often say that together, we fight Lafora disease. That is true more than ever as we are united as a worldwide Lafora community to support the ION283 safety study.
Much of the work you’ll read about in the Annual Report was possible thanks to grants from the Chan Zuckerberg Initiative. We also received event support from the Danny Did Foundation, Vibe Bio, Tempo Zero, A.I.L.A., France Lafora, and Association Cel-luz. We appreciate all our 2024 supporters and your continued partnership to reach a cure!
2025 Goals
Last year, despite progress, also left us mourning the passing of more children. We will keep fighting for a better future in their memory.
Our goals for 2025 are to:
Are you interested in getting involved? Stay connected by signing up for our mailing list and following our social media for regular updates. We always welcome donations and volunteers to support our cause.
We remain committed to improving the lives of those affected by Lafora and helping accelerate the development of treatments. Thank you for your belief in our mission!
Safety Study Update – February 2025
/by Christine KellyWe are excited to share an update about the ION283 Safety Study: the team at UTSW can continue enrolling patients.
In December 2024, the first patient in the Lafora Disease Safety Study at the University of Texas Southwestern Medical Center (USTW) received their first dose of ION283, a treatment designed to halt disease progression. Last month, that patient completed their one-month check-up with no safety issues detected. This allowed a second patient to receive their first dose of ION283 in January. Earlier in February, the second patient completed their one-month safety check. The safety board at the university reviewed the safety data, giving an all clear to proceed as planned with the study.
The first ten families who applied to participate in the study will receive notification from the team at UTSW when there is enough money available to enroll them in the study. We are pleased to share that the third patient is en route to Dallas, and the fourth has been notified.
As a community, we must continue raising funds so all 10 patients can receive treatment. The data collected from this study will help accelerate the approval process so that we can expand access for other patients to receive this drug. However, we need to complete the enrollment for this study first and get the data we need. So far, we have raised more than $977,000 as a community. We must reach 1.5 million dollars by May 2025 to fully fund the study.
We will continue to keep the Lafora community informed about the progression of the Safety Study. Any questions not answered in our FAQs about the study can be directed to Chelsea’s Hope at info@chelseashope.org.