ION283 Safety Study

ASO therapy for Lafora disease

Chelsea’s Hope was thrilled to learn that Dr. Minassian’s team submitted an Investigational New Drug application to the FDA for a safety study of ION283 ASO therapy. All we know is what we learned at the April 2024 Research Roundtable. You can read the summary to see what Dr. Minassian shared with everyone in attendance. 

Rest assured, you know as much as we do and we will communicate any new information we learn as soon as possible. Please feel free to send us any inquiries you have about the study if your question isn’t answered below. We will wait as patiently as possible until May 11th and hope the FDA approves the study. We are as eager as you are to find out more! 

This is an exciting step forward! It will ensure a brighter future for the entire Lafora community.

Frequently Asked Questions about the ION283 Safety Study

IONIS logoThis will be a 2-year study to assess the safety and effectiveness of ASO genetic therapy in Lafora disease. Other forms of ASO therapy have been successful in treating other genetic diseases; hence Ionis Pharmaceuticals has developed a new ASO drug called ION283 for treatment of Lafora disease. ION 283 has been shown to prevent Lafora Body accumulation in preliminary studies of Lafora disease animal models.

Berge A. Minassian, M.DDr. Berge Minassian is the Chief of Child Neurology at UT Southwestern Medical Center. He has been active in neurogenetics throughout his entire career and is a dedicated clinician treating Lafora disease patients. He announced that on April 11th, his team submitted an Investigational New Drug application to the FDA for a safety study of ION283 ASO therapy.

The inclusion/exclusion criteria will be available on clinicaltrials.gov after the FDA review process concludes on May 11th. As a general guideline, patients who are still able to walk on their own are likely to be eligible for the study. Patients will need to be recommended by their neurologists in order to participate in this study. More information will be available on ClinicalTrials.gov if the FDA approves the study.

Blue text says 'UT Southwestern' above gray text that says 'Medical Center' The safety study will be held at UT Southwestern Medical Center in Dallas, Texas, and participants will have to be present in Dallas for treatment and assessment for about five days every three months. Participants would be responsible for covering travel costs, including travelers’ health insurance and accommodation; however, both Chelsea’s Hope will work to locate resources for families who need financial support to cover travel costs. UT Southwestern has a lot of experience with international patients and has previously partnered with Ronald McDonald House, which provides accommodation and meals for patients and their families. 

The study will last for 2 years.

The plan is that a total of 10 patients will be enrolled in the safety study, though this may be subject to change.

The estimate is 1.5 million USD for running the 2-year study; however, this number could change! While Ionis is providing the drug free of charge, this is the budget required to cover the clinical costs associated with the study.

Blue text says 'UT Southwestern' above gray text that says 'Medical Center'You will be able to donate through a foundation at UT Southwestern to support running the study. Chelsea’s Hope will send out details when the site for receiving funds is launched. If you haven’t already, sign up for emails from Chelsea’s Hope.

No. To keep the process fair, you cannot essentially pay to have your child join the study. Donations to the fund will have no impact on who is selected to participate in the trial! 

Following FDA review, the information about the safety study will be found on clinicaltrials.gov, and will have the inclusion/exclusion criteria, protocol, and contact information.  Chelsea’s Hope will assist with fundraising for the study, and we will send out details when the site for receiving funds is launched at UT Southwestern. We will continue to communicate any information we learn with you, so please make sure you’ve signed up for our mailing list and indicated your email preferences.

Connect with Chelsea’s Hope for updates on the study, and help us spread the word!