We Need A Mouse
Originally posted on August 15, 2017
Grace Niewijk recently interviewed me about the incredible scientists at The Jackson Laboratory and how they helped our KIF1A families develop a mouse model. We talked about KIF1A.ORG’s campaign #WeNeedAMouse and our quest for mouse models so scientists can study KIF1A, and ultimately find a cure for our daughter’s extremely rare neurogenetic disease, KIF1A Associated Neurological Disorder.
Grace asked me to tell her about the campaign, how it got started and what is was like to meet the scientists at Jax.
So, I told her.
I was contacting every scientist, physician and researcher who was even remotely mentioned in KIF1A literature and asking them for help. Some replied, and when they did the first two questions were always the same:
- How many kids have the disease?
- Is there a mouse model?
I heard time and time again that nothing can really be done without a mouse model to study the disease. I also heard from others in the rare disease community about The Jackson Laboratory, so I went on their site and asked the person on the helpline “How much for a KIF1A mouse?” I figured it was a good starting point. We found ourselves constantly saying to friends and family “We need a mouse.” So, my wife Sally and I decided to start a hashtag #WeNeedAMouse and asked people to take out their phone and shoot a little video saying that phrase, “We Need A Mouse.” It started with the families then it spread to friends, then to strangers off the street and anybody who we could get to make a video. We had musicians record little songs, we even had puppeteers and their puppets shoot a video.
After the first week there were too many videos to post on our site, so they all went to our Facebook feed. I sent the videos weekly to JAX, and one day I got a phone call from a team of JAX scientists led by Cat Lutz. They went over the logistics of a mouse, and explained the role of mutant organisms in finding treatments for neurodegenerative disease. They explained that we didn’t just need one mouse model, but we likely needed several with different genetic mutations. I was doing the math in my head and just as the devastation of cost hit me, Cat Lutz said (and I will always remember the impact of hearing this sentence), “What we’re saying, Luke, is that we want to do this for the families, take the money you’ve raised and spend it on something else your organization needs.” It was the first tangible goal our foundation met. I was crying, so I’m not sure if they even heard me say thank you.
After that phone call, KIF1A families made “Thank You JAX” videos and sent them to Cat’s team up in Maine. Families in our community are so truly thankful to the scientists working on KIF1A, because without them we would not have any hope for treatment. At that time we knew of about 50 families in the world. We all made “Thank You Jax” videos. That moment was so important. It was one of the first steps up a seemingly impossible mountain, and we all did it together.
The critical element to all of this was (and is) collaboration. The team at JAX collaborates closely with our physician and our champion, Wendy Chung. Dr. Chung and her team research KIF1A and know all of the families. She is also one of few physicians with clinical knowledge of our rare disease, and is our daughter’s doctor. This collaboration set everything in action: we were able to use the money from #WeNeedAMouse to support a team that works relentlessly to understand the disease and to discover treatment. Without this collaborative effort we would still be screaming from the mountain top about KIF1A and our need for research. Now scientists actually return my call because KIF1A.ORG and all our families can say “we have a mouse model.”
If we didn’t have collaborators like Wendy and Cat working so hard for our kids, we would be even further away from discovery than we are now — and I am terrified that we’re still far away from treatment. But we keep working to raise awareness and money, and we know sooner than later a fleet of scientists will be working together around the clock to bring treatment to our KIF1A kids.
I’m so thankful for everybody at JAX. They go to work in the morning with a mission to help our kids — kids who are racing against time and fighting so hard every single day.
Learn more about our relentless mission to find treatment for Susannah and kids like her at www.KIF1A.org.
