RACING TO CURE
NIEMANN PICK TYPE-C
The Life For Liam And Friends Foundation is a registered nonprofit charitable organization with the mission to support individuals and families who are impacted by Niemann Pick Type-C and help the world find a cure for this devastating disease through helping connect NPC families, spreading awareness, educating the public, and supporting medical research.
For Medical Research
University of Iowa
Stead Family
Children’s Hospital
Partnered With
Donations Fund Research & Development of a New Gene Therapy to Cure NPC
Niemann Pick Type-C (NPC) is a rare fatal disease that attacks the Brain, Nervous System and Organs. Often called "Childrens's Alzheimers", the disease progresses rapidly from birth, interrupting neurological development as the cells in the brain and nervous system die off. This incurable disease causes damage that is irreversible and is ultimately fatal. Historically, Children with NPC do not live 5 years past the diagnosis.
Due to the complexity of the disease and inconsistency of disease progresson, NPC kiddos often struggle for years before getting a confirmed diagnosis. A few experimental treatment have shown to help slow down the rate of progression of the disease if given early enough. With NPC every day counts.
We are extremely blessed to have learned Liam's diagnosis early and we are grateful there is a strong international community established around NPC.
A cure doesn't exist for Niemann Pick Type-C, yet! We are set on finding one and we are getting close!
Recent advancements in the field of genetics and gene therapy have tremendous potential to cure diseases like NPC at the root of the disease, the DNA.
Genetic disorders can be cured by either editing the DNA, gene editing, to correct mutated genes or by delivering the missing mRNA caused by the mutated genes. We are researching the use of mRNA as a gene therapy to cure NPC and the data so far is very promising!
Niemann Pick Type-C
The Research
A genetic disease requires a genetic solution
University of Iowa
Stead Family
Children’s Hospital
We've partnered with Dr. Mark Schultz and the Shultz Laboratory at the University of Iowa Stead Family Children's Hospital to research and develop a new gene therapy using the latest mRNA technology.
Children with Niemann Pick Type-C are missing the NPC1 proteins which are responsible for breaking down and clearing out of cholesterol from the cells of the body. If the cholesterol builds up enough, the cell will inevitably die. When this happens in the brain and nervous system it causes neurodegeneration.
The new gene therapy that we are researching & developing leverages new advancements in mRNA technology and medicines to help the body produce the missing NPC1 proteins so the cells can break down and clear out the cholesterol.
If this gene therapy is successful children with Niemann Pick Type-C would be functionally cured of the disease by receiving regular doses of the treatment.
01
SPREAD
AWARENESS
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02
COMMUNITY
ENGAGEMENT
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03
TREATMENT
DEVEMOPLMENT
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MISSION &
PRIORITIES
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Research Progress & Updates
December 2023:
Heartfelt Gratitude and Exciting Progress Update! As 2023 comes to a close and we reflect on the past year, our hearts are filled with immense gratitude for the outpouring of love, support, and generosity we've received. From family and friends to kind-hearted strangers who connected with Liam's story, your support has been the cornerstone of our journey in raising awareness and funds for this cause close to our hearts. We're thrilled to highlight how much we accomplished in one short year. Through collective efforts, we have not only successfully raised nearly $60,000 for the Schultz Laboratory at the University of Iowa to start this important research, but have made strides in developing a groundbreaking treatment for Niemann-Pick disease (NPC). Thanks to all the generous donations, the Shultz Lab has been able to develop a viable mRNA treatment, test & optimize the treatment in cell lines, and conduct preliminary testing in mice. The data and results from Dr. Shultz’s work has been very encouraging and gives us an incredible amount of hope that we are going to save Liam. Now, we're gearing up for a crucial phase of our research this next month. We'll be starting a full mouse model trial of the treatment's effectiveness, focusing on the liver of mice afflicted with NPC. This is a pivotal step in the process, bringing us nearer to comprehending the full potential and efficacy of the mRNA treatment for NPC. Your support of our mission has been the driving force behind these milestones. Each donation, regardless of its size, has been a vital part of this success. Help us keep this extraordinary work moving forward, there is still a long way to go. The road to develop new treatment is lengthy and daunting, but with the support from each one of you, our confidence in making a meaningful impact has never been stronger. A heartfelt thank you from our little family! Your support means the world, not just to us, but to everyone whose lives could be changed by this research.
July 2023:
We're thrilled to share some amazing news from the Schultz laboratory at the University of Iowa Stead Family Children’s Hospital! In a time frame much shorter than we anticipated - only 3 months! - the team has made significant strides in our quest for an effective treatment for Niemann-Pick Type C (NPC). They have successfully packaged NPC1 mRNA into a particle, which is capable of entering cells and restoring the missing NPC1 protein. This critical breakthrough paves the way for the correction of the characteristics like cholesterol accumulation we see in NPC. This achievement propels us into the next stages of our research - cellular dosing studies and then animal model testing. The journey is still a long way to go, but every step forward fuels our hope and resolve. These next stages will required significantly more funding, we need to raise an additional $34,000 to start the cellular dosing studies. That research will take about a year, in which time we will need to raise another $150,000 to start animal model testing of the impact of the treatment on the liver and brain. Unfortunately the research is on hold until we can fund the next stage. Your generous support has made this possible. It's you who have empowered us and the incredible scientists at the Schultz laboratory to push the boundaries of what is possible. So, we dedicate this victory to each and every one of you, our donors and supporters. Together, we're writing a new chapter for NPC. But we still need your help to raise the funds to continue the research! Here’s how you can continue to support Liam and help make this new treatment a reality for NPC: Follow Liam Everywhere: Twitter: @Life4Liam @JordanAMitchell @jlezeu Facebook: Jordan Andrew Mitchell, Jen Lezeu Instagram: rarekido, mitchjet, jlezeu Share ALL of our posts about Liam and NPC: Reposting, liking, and commenting on all of the post we make about our efforts and NPC will help spread awareness and connect with people outside our network of friends. Donate to Liam or the Shultz Lab directly to help fund the research: Life4Liam GoFundMe: Our Life4Liam GoFundMe is still active while we work on setting up our nonprofit. gofundme.com/f/Life4Liam Donate Directly to University of Iowa: You can now donate directly to the University of Iowa’s Stead Family Children’s Hospital and Dr Shultz’s! Donate to NPC Research
March 2023:
We wanted to thank everyone who has shared, donated and sent the kind messages for Liam. Our hearts are so full with gratitude. We currently have raised $26,220 in the last 5 months which is a huge accomplishment to us. We would like to reach our goal of $30k before the month of April so we can start the first year of testing and research. We would like to share with you a bit more detail of what we will be doing during the first year and how your donations are supporting Liam and NPC kiddos towards a cure. As we have mentioned previously, we are working with a scientist that is working on getting a treatment into clinical trials for NPC. One option we have started exploring is a new type of therapy called lipid based mRNA therapy. The idea is to package mRNA into tiny particles called lipid nanoparticles, or LNPs, which can be injected into the body. Once inside the body's cells, the mRNA can tell the cells to make the missing protein, which could potentially help alleviate the symptoms of the disease. This method is cheaper and faster than other gene therapy options. A recent study, ‘mRNA Treatment Rescues Niemann-Pick Disease Type C1 in Patient Fibroblast’ (https://pubmed.ncbi.nlm.nih.gov/36125338/) had great results showing mRNA treatments can have a tremendous impact. The goal for the research by Dr. Mark Shultz at University of Iowa (https://medicine.uiowa.edu/pediatrics/profile/mark-schultz-1) is to see if healthy NPC1 mRNA can be delivered using lipid nano particles to help correct the problem of cholesterol building up in cells. We are ecstatic that mRNA treatments for NPC research is underway. We absolutely could not have started this so soon without your generosity and support! Please continue to share and help Liam.
Ways to Donate
We are extremely grateful for the generosity and support that has poured out over the past few years. We have been able to establish a few different ways you can donate. Directly to the Life For Liam & Friends Foundation, to the Schultz Lab at University of Iowa Stead Family Children’s Hospital, or through Behind the Blue.
