safety study Archives - Chelsea's Hope Lafora Children Research Fund

Chelsea's Hope Lafora Children Research Fund's 2024 Annual Report

Read 2024 Impact in our Annual Report

April 11, 2025/by Christine Kelly

Chelsea’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.

“While we celebrated this milestone, we acknowledge that many families are still without a path to treatment for their loved ones. Every child matters. We will continue to support all of our Lafora families and push for expanded access to treatments and new therapy development.” – Jenifer Merriam, President

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:

Get the ION283 safety study fully-funded (we are more than halfway there)
Establish Clinical Centers of Excellence & Guidelines for Clinicians
Publish collected data in order to:
- Drive biomarker development
- Determine Lafora prevalence rates

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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!

Download the 2024 Annual Report

Safety Study Update – February 2025

February 20, 2025/by Christine Kelly

We 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.

Read Press Release

Text says 'ION283 Safety Study ASO Therapy for Lafora Disease' over a background image of the Dallas, Texas, skyline

Now Recruiting: ION283 Safety Study

November 17, 2024/by Christine Kelly

The application packet for the ION283 safety study is now available!

You can view the application as a PDF or download a Word document.

Please read the email below from Dr. Minassian:

“Dear Lafora disease patients and families,

We are now ready to receive referrals for consideration for inclusion into the clinical trial: Safety and Efficacy of Intrathecally Administered ION283 in Patients with Lafora Disease

This email relates to the prescreening phase. Below are the requirements for the prescreening. If a patient passes the prescreening, they will be placed on a list of prospective patients to participate in the trial. Each patient invited to participate will still need to meet broader inclusion-exclusion criteria at the time of screening in Dallas. These final inclusion-exclusion criteria are listed in the above website.

Only referrals from neurologists will be accepted, except where it is impossible for the patient to have a neurologist (we are thinking of the patient in Gaza), in which case another doctor or local medic can make the referral.

Attached is a document that contains a prescreening table and the Lafora Disease Performance Scale, both of which should be completed by the referring neurologist. All parts of the document must be completed. All documents and video mentioned in the prescreening table must be provided at the time of referral precisely as described.

Referrals must be sent by the neurologist from his or her email address to LDrecruiting@utsouthwestern.edu as a single email containing all the attachments. Please note that our email system cannot accept files that are larger than 50 Mb total. Therefore, make the video short and of low quality. Just enough to show the patient walking ten steps independently.

The list of prospective participants will be generated on a first-come first-served basis, where first-come means that all the above requirements are included in the referral. Any referral that has any component missing will not be accepted and will be returned.

The study will only include 10 participants. Therefore, the first 10 on the first-come list will be invited to Dallas for formal screening toward potentially entering the study. If any patient among the first 10 fails to meet inclusion-exclusion criteria at the time of final screening in Dallas, then this will open a slot for a subsequent patient on the list.

The upper age limit is 18. We understand this to mean any patient before their 19^th birthday at the time of final screening in Dallas. In case among the first 10 patients passing prescreening there is a patient whose turn in the list means they will age out of their 19^th birthday at the time of the final screen in Dallas, such a patient may be moved up the list as long as this does not affect the age-criterion admissibility of the remaining patients.

Thank you for considering participation in this clinical trial.

Berge A. Minassian, MD

Principal Investigator

University of Texas Southwestern”

Our deepest gratitude goes to Dr. Minassian, Dr. Joshi, Dr. Verma, Dr. Messahel, and the entire team at UTSW for making this possible. We are also thankful for Ionis’ drug donation and our community’s collective fundraising efforts. We can’t stop now!

Please help us share this information, and good luck to everyone applying.

View PDF Application Download Application as a Word Document

BECAUSE WE HAVE HOPE Lafora Holiday Season Auction 2024

Announcing Holiday Season Auction

October 29, 2024/by Christine Kelly

This November, we are hosting a holiday season auction to raise money for the upcoming ION283 safety study.

Bidding will open on November 1st and continue through the 30th. 100% of the profits will go toward the safety study.

Each piece has been donated by the Lafora families or affiliates. Check out our page to see what items are already available for bidding. We invite you to bid on an item to purchase or share this auction with your family and friends via this link: https://givebutter.com/c/laforaauction/auction.

How it will work

Some items will have a unique bidding timeline, but the entire online auction will run throughout November. Generous supporters have donated the items from around the world!

You will have to register to bid here: https://givebutter.com/c/laforaauction/auction.

Please review the details and shipping for each item you bid on. Some items have additional shipping costs, while others only ship to certain countries.

Can you help us?

This is an open call for in-kind donations for our holiday season auction! We request artwork, books, clothing, jewelry, tickets, vacations, and passes to events in your country.

Please email us with the subject “November Auction Donation” to get involved. You can donate items until November 24th.