Sloane is an adorable 3 year old with KAND. She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. Her mother, Megan, has offered to share their experience and journey with everyone to help us understand the process and to share in their hopes, fears, challenges, and successes that come along with ASO treatment.
Hello KIF1A Community!
We are quickly approaching Sloane’s ASO dosing and I thought it might be helpful to “blog” about our experience so you all can follow along to see what the process is like.
I wanted to give a brief timeline of our experience to date.
Timeline:
- December 2021– We met with Dr Chung in NYC and discussed and applied to n-Lorem. They also drew Sloane’s blood to create IPSCs (patient cell line).
- September 2022– We were contacted by n-Lorem to obtain blood from Sloane, my husband, and me.
- March 2023– We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane. However, we needed to wait for Susannah to finish her trial before we could submit to the FDA for Sloane’s ASO use.
- February 2024– n-Lorem submitted an IND (investigational new drug) to the FDA for Sloane.
- March 2024 – The FDA approved the IND, and the administering doctor (Dr. Bain) submitted to the ethics review board at Columbia University Medical Center for treatment approval.
Future Dates:
- May 2024 – Initial dosing scheduled for May 9th.
- June 2024 – Second dose scheduled.
- August 2024 – Third dose scheduled. Going forward dosing will be quarterly (every 3 months ongoing).
We will move to New York for 1-2 months come the beginning of May. My husband will stay with my older children (1st grade and Pre-K) while they finish school and my mom will come with me to NYC. That has been a little daunting because I’m the primary caregiver and coordinator for all my children. My mom will be coming to NYC with me until my husband and older kids can join at the end of the school year. I’m also a little nervous about getting around the city with a non-ambulatory, nonverbal, three year old. But we are so excited for her to get this treatment we know the challenges will be worth it.
Before receiving her first ASO dose, the research team will conduct baseline cognitive and physical assessments as well as a baseline EEG. We will also have a movement tracker that Sloane will be wearing so they can monitor changes in her movement outside of physical assessments. Sloane will get her first dose May 9th.
I know a lot of people are wondering about cost. The actual drug is covered 100% by n-Lorem but all hospital and administration costs are either billed to insurance or we will pay out of pocket for them. We are also paying for our housing. We are staying for 1-2 months assuming everything goes well. Because this is still very experimental we want to be close to the hospital and the administering physician should we need them. However, it is my understanding that once the process is refined, patients should be able to go in and out in a couple days.
Please reach out with any questions. We want to be an open book during this process. We will follow up post-dosing and will hopefully be reporting back with really positive changes we see coming soon!
With Hope,
Megan, Sloane, and family
Thank you for sharing this!
Praying for you all — Sloane is tough as nails, and her parents are doing a great job with everything, managing the chaos!
Perhaps you already know it, but even her name is an old Celtic derivative meaning “warrior”, and I know she’s a strong one.
We are all praying for Sloane and the Dr.’s during this journey. Prayers for her entire family as well for strength and perseverance. She is definitely a warrior!! Love you guys!
Warrior Kirt and his family from Kansas!