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Nanci Ryder has visited my lab at USC two times.
The first time she could walk and talk a bit, and write on a sketch pad. The next time, a year and a half later, in 2017, she couldn’t move at all. But she’s always brought Team Nanci members, friends like Renee Zellweger, Courteney Cox and Don Diamont. We don’t get to visit with ALS patients that often in the lab, let alone stars. They asked such great questions and were interested in our work. For us, it’s been a phenomenal exchange of information, but seeing Nanci is such a reminder of how fast ALS progresses. There are others dying every day. It makes us realize even more how urgent our work is.
That is why sharing this news is so exciting.
We have made a major discovery in our search to find a cure for ALS. Feb. 5 on Nature.com, we announced that we have used stem cell technology to create nerve cells in a Petri dish from an actual ALS patient. Previously, you could not study living nerve cells of an ALS patient because the nerve cells are so inaccessible when a person is alive. You can’t get to them so you can’t study them, and therefore you cannot see what is happening with the disease. Studying nerve cells after a patient dies is OK, but it doesn’t allow you to really do experiments and figure out what is going on. Instead of extracting nerve cells, we took blood cells and converted them to nerve cells by going through a stem cell intermediate. In that way, we can make real nerve cells from an ALS patient in a lab. By doing that, the cells can recapitulate all of the things we can see in a patient, and it becomes a platform for studying ALS.
We made another discovery, which is really key and can be used for the most common form of the disease. We found out how ALS is causing the nerve cells to degenerate, and by doing that we were able to find a new protein target. With this new target, we can use a drug to bind to it that we think can change the disease course for ALS patients. The next step is to try to get the drug into clinical testing, but first we need to make this chemical more stable for the body. We have launched a startup company that is hopeful that they can change the structure of the chemical to last longer in the human body and make it more drug-like. A similar drug has already been in clinical trials for use to treat Crohn’s disease so we know that this approach is safe, which might make it easier to go through the phases, which still could take years. The average drug-approval process takes about 10-15 years, but we hope that we can show a very good effect in phase II and that might speed up the process.
It takes time, and we want to make sure that the drug is safe and effective to give it the best chance at succeeding. I’m hopeful. I’m hopeful enough that we started a company to move forward with this process. There are people working right now whose job it is to get this drug to ALS patients. This is one of the first, if not the first, example of someone finding a new target.
The funding that we used to find this new protein target and the new drug, a large chunk of that money came from an ALS therapeutic grant from the Department of Defense. The reason they have this grant is that the ALS Association lobbied to have that as part of the Department of Defense’s funding portfolio. The ALS Association funnels money into research as well as lobbying, so the work Nanci and her friends have done in contributing more than $600,000 has been invaluable in this process. Because of the awareness Nanci delivers with Team Nanci, it creates huge public support for curing ALS, and that support drives the Department of Defense to give a portion of its budget to the disease.
We are a direct beneficiary of that money, and that money has led to our discoveries. It’s a circuit and every piece is critical to make it complete, even if it takes a while to see the pieces come together. Each piece makes all the difference, and so does each new discovery.
Thank you, Nanci, and thank you everyone who has contributed to this fight. We are on our way.
A version of this story first appeared in the Feb. 14 issue of The Hollywood Reporter magazine. To receive the magazine, click here to subscribe.
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