I think Rose’s favorite day last week was Saturday. We arrived at 10:30am, forty-five minutes outside of Austin to a ranch that specializes in helping children with special needs. They use horses, and it’s called hippotherapy. The use of “hippotherapy” (using the movement of the horse) can help to engage sensory, neuromotor and cognitive systems and in turn, promote functional outcomes.. The horse's movement helps children with speech delays and disorders. It’s science that isn’t completely understood. But in the distance, was a 15 year old girl with a rare disease getting off her horse. She stepped off and introduced herself. I thought…”I wonder if Rose will ever do that one day… introduce herself.” 

They brought out a pony, and showed Rose how to brush it. She was excited and overwhelmed. She wore a helmet for 5 minutes, which is a huge win, and even made a few brush strokes with the brush. For Rose, even the tiniest steps are celebrated, they’re wins. In science, it’s the same. They may seem like small steps. But they all add up to moving mountains, we just have to have the patience. Rose doesn’t have the ability to regulate her emotions, so when she would get frustrated and scream.. I’d put on her favorite song and I sat it down on the saddle. She went back to focusing on the pony at hand.  

There was a freeze in Austin not too long ago. It just so happened to be the same time we were getting neurons shipped to us from Europe to the lab I started, Everlum Bio. We use these basic neurons to help us grow and test the cells that are considered “healthy” and compare that data to Rose’s. Unfortunately, the freeze screwed up shipping and we ended up with a box of contaminated dead cells. That set us back a couple of weeks. But this is how this works. Sometimes it feels like there’s something in the air that wants to prevent me from getting to a cure... And you take your deep breath, reorder the neurons, and smile and say... NOT TODAY!!!! The company sent us a second shipment for free. Kudos to Bit Bio, for being so awesome. 

We designed the potential drugs in a computer. We manufactured the most promising ones (that you funded) and we treated Rose’s cells in a dish with them. This is just the first step. Next will be optimizing our favorite designs for low toxicity and efficacy. (Is it safe, and does it work)

That means going into the Rosie mice. It means starting a large animal study, because the FDA will likely require two different animal species to prove a drug is safe and it works. It also means that things are about to get real expensive. We have to raise 1.8 million dollars to do the rest of this work, and treat Rose’s disease. It’s daunting. But I’m hoping some people step up this year for Rose and get us closer. 

500,000 - people reached with our social media campaigns

10,000 - people reached with Casey’s speeches and presentations at major conferences

1000 - drug designs we can work from

100 - oligos printed

50 - scientists working with us and advising

20 - oligos being confirmed as working

10 - Rosie humanized mice

5 - Cell lines for scientists to work on HNRNPH2

2 - Brain organoids

1 - Rose with hope, for thousands of children in the future. 

You have stood by us and are bringing this to fruition. Rose was alone in her fight. Our family was alone. But because of you, we are surrounded by an immense amount of hope. To think what we do now for Rose, will affect thousands of children and families in the future. A true impact that never stops helping. You are doing this!! 

Yours in the fight always,

Casey McPherson 

1401 Lavaca St, Suite 890
Austin, TX 78701

tocurearose.org