Currently, the Lafora community can fundraise. Fill out this survey HERE if you are interested.
Families, one effective way of fundraising is sharing your story. Please get in touch with us if we can help you in any way!
The estimated cost of the Safety Study is $1.5 million USD. Please remember that donations do not guarantee a spot for your loved one in the trial. We encourage donations through a patient organization.
Chelsea’s Hope has an online fundraiser where you can donate, and we will transfer the money to UTSW. You can also join our fundraising efforts here: givebutter.com/LaforaSafetyStudy/Join.
We are thrilled to share the collective fundraising efforts of our worldwide Lafora organizations: TempoZero, A.I.L.A., Cel-Luz, France Lafora, Chelsea’s Hope, and the Weiss family.
Together, we have raised $977,000 for the safety study.
Because of your support, we are almost two-thirds of the way to the estimated budget! We can’t stop now!
We cannot stress the urgency enough. We must reach the estimated budget ASAP to support all ten patients.
The FDA approved the ION283 Safety Study in August!
During the 2024 Lafora Disease Science Symposium, the team at University of Texas Southwestern (UTSW) received the official National Clinical Trial number from the FDA for the ION283 Safety Study: NCT06609889. Families and clinicians can now find official information about the eligibility criteria and the study protocol on clinicaltrials.gov. The essential eligibility criteria are:
1) Patients must be between 10-18 years of age at the time of enrollment (meaning they are at least 10 years old and have not yet turned 19 years old)
2) Patients must have a genetically confirmed diagnosis showing a mutation in either EPM2A or EPM2B/NHLRC1
3) Patients must be able to walk independently (meaning they can walk 10 steps without assistance)
The additional update we have is that Ionis announced on September 4, 2024, that they reached an agreement to license ION283 to Noventia Pharmaceuticals. Read Ionis’s statement to the Lafora community HERE.
The licensing of ION283 will not impact Dr. Minassian’s Safety Study at UT Southwestern. That will continue as planned, and we will update our community as soon as enrollment is open on clinicaltrials.gov.
On October 14, Dr. Souad Messahel kindly joined us for a Q&A. The meeting recording and transcript are available upon request. Requests can be emailed to info@chelseashope.org. View her slides HERE.
We are excited to share that the first patient is in Dallas and received their first dose of ION283 in December. We are working on getting permission to share updates with the community.
Families, the UTSW team will contact you directly if they can enroll your loved one. All information is kept confidential. Please join us in respecting the privacy of the safety study participants and in celebrating the news from Dr. Minassian that the safety study has started!
Has the enrollment process started?
Yes! It began on November 17, 2024. Learn more and apply here: https://chelseashope.org/now-recruiting-ion283-safety-study/.
How will the enrollment process work?
What is the safety study?
This 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. ION283 has been shown to prevent Lafora Body accumulation in preliminary studies of Lafora disease animal models.
Who is leading it?
Dr. 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, at the April 2024 Research Roundtable, his team submitted an Investigational New Drug application to the FDA for a safety study of ION283 ASO therapy.
He shared on August 14th that the FDA approved the ION283 Safety Study.
What are the criteria to join?
The inclusion/exclusion criteria are available on clinicaltrials.gov.
Patients will need to be recommended by their neurologists in order to participate in this study.
Where will the study take place?
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.
How long is the study?
The study will last for 2 years. Once the study receives approval, patients will be enrolled on a rolling basis, one per month, as the funds are available.
How many patients will be enrolled?
The plan is that a total of 10 patients will be enrolled in the safety study. The FDA will require a preliminary evaluation after the first two patients are enrolled in the study. If those evaluations show good safety and tolerance of the drug in the first two patents, then the remaining 8 patients can be enrolled.
What is the cost?
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.
Can the safety study be expanded?
That depends on a number of factors. After the FDA approves the protocol, the investigators at UTSW can submit amendments to expand the study. These amendments could include expanding the age range and the number of patients in the safety study. These amendments will depend on the safety data collected from the initial patients enrolled in the study. It will also depend on how much ION283 drug is available. Enough drug for 10 patients is guaranteed, however, additional drug for more patients would need to be negotiated with the pharmaceutical company.
How can I donate?
Chelsea’s Hope created a fundraising page in anticipation of the Safety Study. Lafora France also has a dedicated fundraising page.
You can make tax-deductible donations in your preferred country. Our organizations will then donate to the UTSW foundation once we have confirmation that the study is active.
You will also 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.
If I donate, will my child be guaranteed a spot in the safety study?
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!
How can I learn more?
The information about the safety study will be found on clinicaltrials.gov soon, 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.
Besides donating, how can I support the study?
Connect with Chelsea’s Hope for updates on the study, and help us spread the word!
What does the licensing of ION283 mean for the Lafora community?
Who is Noventia Pharma?
Noventia Pharma is “a privately owned European specialty pharmaceutical company committed to advancing research to bring hope to those living with serious diseases… In seeking a partner, Ionis was looking for an organization who had the commitment and capability necessary to execute a global development program to evaluate ION283 in Lafora disease.” – Ionis
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, Post Office Box 348626, Sacramento, CA 95834
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 […]