“The Search for a Cure for ALS” on the Learn the Technology Podcast

Yentli Soto Albrecht is a scientist working to eliminate ALS before it eliminates her. She recently sat down with Brandon Krakowsky on the Learn the Technology podcast to talk about where the field stands and what it will take.

Three takeaways she thinks matter most for the community of patients, carriers, and families:

💡 1. ALS is not one disease, which is much of why it has been so hard to treat.

Its causes range from genetics, like her own C9orf72 mutation, to environmental exposures and head injuries, with more genetic causes still undiscovered. One treatment may not work for everyone, so much of the work is matching patients to the approaches most likely to help them.

💡 2. Patients can directly power the search for a cure.

With CureC9’s partner BrainXell, she is turning patient skin cells — including cells from carriers like her, and from her father — into the cell models researchers and companies need to test new drugs. You can contribute your own cells and have treatments developed on them, and it removes the year of paperwork that often blocks researchers from accessing patient cells, because they can simply buy vials. Learn more at CureC9.com.

💡 3. The hardest roadblock is not the science, it is whether patients can take part.

So much of what slows the field down is access to patients, samples, and data. When you share yours and get connected early enough to be eligible for trials, you move the whole field forward. You are not a bystander in this, you are central to it.

She left the conversation hopeful. Baby KJ at CHOP was recently treated for a fatal genetic disease with a custom gene therapy in about six months, in work led by one of her mentors, Dr. Kiran Musunuru — and that is the speed the field needs to bring to ALS and FTD.

If this speaks to you, watch the conversation below and reach out.