A new treatment targeting a rare, aggressive form of ALS is delivering results far beyond what researchers imagined. Some patients aren't just stabilizing — they're regaining lost abilities.
Recovery, Not Just Delay
In ALS clinical trials, the best-case scenario is usually slowing the disease. But this time, researchers saw something unprecedented: actual functional improvement in some patients.
A Mutation With Devastating Power
The drug targets a rare mutation in the FUS gene, which causes one of the most aggressive forms of ALS — often striking teenagers and young adults with rapid progression.
Meet the Drug: Ulefnersen
Formerly called jacifusen, this experimental therapy was developed through a collaboration between Columbia University and Ionis Pharmaceuticals. Its name honors the first patient who received it.
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One Woman’s Remarkable Comeback
After starting injections in 2020, a young woman who had lost the ability to walk and breathe independently regained both — defying the expected course of her condition.
Preventing ALS Before It Starts
Another patient, a man in his 30s, began treatment before showing symptoms. Three years later, he remains symptom-free — and tests show his muscles are functioning better than expected.
Biomarkers Tell the Story
Patients on the drug saw up to an 83% drop in neurofilament light, a biomarker of nerve damage — clear evidence the drug is hitting its target in the nervous system.
Slower Progression, Longer Lives
Even among patients who didn’t fully recover, several experienced slower disease progression and extended survival — a meaningful gain in quality and quantity of life.
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A Safe and Tolerable Option
The drug caused no serious side effects in the study participants, adding to its promise as a long-term treatment option if approved.
Global Trials Now Underway
With early results in hand, Ionis has launched a global clinical trial led by Dr. Neil Shneider — the neurologist and scientist behind the therapy’s development.
It All Began With One Patient
The journey started with Jaci Hermstad, whose twin had died from ALS. She became the first person to receive the drug — and inspired a treatment that could now help many more.
Rewriting the Rules With Precision Medicine
Ulefnersen uses antisense technology to silence the FUS gene and stop toxic proteins from forming. It’s a powerful example of what’s possible when science meets personal medicine.
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This Article Is Based on Information From News Medical
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