#ScienceSaturday posts share relevant and exciting scientific news with the KAND community. This project is a collaboration between KIF1A.ORG’s Research Engagement Team Leader Alejandro Doval, President Kathryn Atchley, Science Communication Volunteer Aileen Lam and Chief Science Officer Dr. Dominique Lessard. Send news suggestions to our team at impact@kif1a.org.

KIF1A.ORG In The News

A look into a 3-year-old boy’s battle with KIF1A, a rare disease without a treatment or cure

In this article, team Ethan shares their story of their son’s KIF1A journey with their local community. Thank you for sharing your story to advocate on behalf of the entire KIF1A community!

KIF1A.ORG Now Hiring for a Research Engagement Director

We’re now recruiting for a full-time Research Engagement Director! If you have scientific, research and/or communications experience and you want to join a community of people driving progress against neurodegenerative disease, check out this announcement.

KIF1A-Related Research: From the Archives

KIF1A/UNC-104 Transports ATG-9 to Regulate Neurodevelopment and Autophagy at Synapses

Instead of sharing a recent piece of KIF1A-related research this week, we’re highlighting an influential piece of KIF1A literature from the past. This week’s topic? Autophagy! We can think if autophagy as our neuron’s waste management system. The day-to-day functions of our nervous system lead to a buildup of waste products, just like we accumulate trash and recycling in our personal living spaces. Eventually, these waste products must be addressed in our neurons, and either be recycled into new products or degraded and broken down. Without autophagy our cells could quite literally be full of cellular garbage!

The paper we are featuring today, led by KIF1A Research Network member Dr. Andrea Stavoe, highlights the important role that KIF1A plays in facilitating neuronal autophagy, using a C. elegans worm model for experimentation. Specifically, in this study KIF1A is shown to carry and transport a key protein for autophagy called ATG-9. In other words, KIF1A makes sure that ATG-9 is in the right place at the right time to ensure successful autophagy. Furthermore, these findings clarify the role of autophagy in neurodevelopment. Want to learn more about autophagy and why it was the topic of a Nobel Prize in 2016? Check out the video below!

Rare Disease News

How to rescue Big Pharma drug castoffs from the scrap bin through nonprofit partnerships: report

In the process of drug development, it is not an uncommon event for potential drugs and compounds to be “shelved” or halted in a development pipeline for a variety of reasons. In fact, “the U.S. National Institutes of Health (NIH) estimates 80% of all drugs that enter human testing are never approved for use”! Just because these drugs are sitting on the shelf for one application, does not mean that they couldn’t be beneficial for other applications, like repurposing in the rare disease space. As is so, this article we are featuring today highlights a new report out of The Milken Institute, proposing a potential path forward for these types of shelved medicines that could offer hope for patients like ours. The article goes on the discuss the many roles played in the processes by various stakeholders such as investors, government agencies, and non-profit organizations. To read more, click the button below!

“Drugs that can improve people’s lives should not be sitting on drug company shelves… Finding a development path for promising drugs, regardless of their commercial potential, is simply the right thing to do for society.”

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