Paralyzed Mice Walk Again Thanks to New Drug

In a medical first, paralyzed mice with a “severe spinal cord injury” have regained the ability to walk. The startling outcome is thanks to a new drug developed by researchers at Northwestern University, who hope to approach the Food and Drug Administration about human trials as soon as next year.

“The aim of our research was to develop a translatable therapy that could be brought to the clinic to prevent individuals from becoming paralyzed after major trauma or disease,” said study leader Samuel Stupp, in a report by Agence France-Presse via Sciencealert.

BACKGROUND: BIONICS AND STEM CELLS

Earlier this year, The Debrief reported on a quadriplegic patient who was able to use robotic arms to feed himself. Such improvements in brain machine interfaces, artificial intelligence and robotics are giving hope to similarly challenged individuals who typically rely on others for even their most basic needs. However, until now, the idea of a treatment that could fully restore full mobility has remained elusive. Thus far, attempts t restore spinal cord connections using stem cells and other regenerative drug cocktails have tried but failed to achieve measurable results.

 

ANALYSIS: MAKING PARALYZED MICE WALK AGAIN

Published in the Journal Science, the Northwestern team describes a multi-pronged approach where “a mouse model of paralyzing human spinal cord injury” is treated with a scaffold that bears two peptides already known to promote nerve generation. The first helps promote the formation of new blood vessels, while the second reduces scarring.

According to the AFP piece, this means that instead of trying stem cells or other previously unsuccessful methods to restore the spinal cord connection, the Northwestern team “used nanofibers to mimic the architecture of the extracellular matrix – a naturally occurring network of molecules surrounding tissue that is responsible for supporting cells.

Each of these nanofibers are 10,000 thinner than a human hair, and each is coated with hundreds of thousands of the two regenerating peptides. The matrix is then placed in a gel-like substance that can be injected into the tissue surrounding the spinal cord injury.

In the lab trials, the team used mice whose spinal injury had occurred at least 24 hours earlier, since spinal cord accident victims often experience delays in receiving treatment.

Four weeks after the injection, the mice who had received the treatment were able to walk with more or less the same ability as before their injury. The untreated mice remained paralyzed.

OUTLOOK: HUMAN TRIALS AS SOON AS 2022

The researchers hope that their successful lab results could usher in a wave of “supramolecular drugs,” like theirs, where many therapeutic compounds work together in one drug treatment.

“The challenge will be how the FDA will look at these therapies because they’re completely new,” said Stupp, whose Northwestern team expects to approach the FDA with their first human trials in 2022. “[T]here is nothing out there to help spinal cord injury patients, and this is a huge human problem.”

Follow and connect with author Christopher Plain on Twitter: @plain_fiction