A breakthrough recently came in the medical world as doctors say Huntington’s disease – a cruel hereditary disorder that brings with it a combination of ALS, Parkinson’s, and Alzheimer’s symptoms – can now, in part, be cured. However, potential patients in Mississippi and across the country are awaiting FDA approval to be able to take advantage of the treatment.
The treatment was found to be a preliminary success in September after various trials across Europe and North America. Called AMT-130 and produced by developer uniQure, the gene therapy showed a 75% slowing of disease progression in Huntington’s patients by reducing toxic protein in the brain, according to research from the University of Alabama at Birmingham and the University College London.
Huntington’s was long considered untreatable and a disease that often leads to death within 10 to 30 years of onset, but according to Dr. Victor Sung at UAB, the new breakthrough brings optimism to a community of patients who more times than not struggle to find hope.
“We certainly remain cautious about interpretation of these results since they are only in a small number of patients, but the 75% disease-slowing is a big number and still very much a reason to be excited,” said Sung, who heads UAB’s Huntington’s Disease Clinic and has spent decades looking for a cure.
Of the 29 early-stage patients included in trials, some received a low dose of AMT-130 and others a higher dose, which is delivered to the brain during a surgery that last up to 10 hours. After three years, those who received the higher dose were reaching up to the 75% mark in disease progression and experiencing more movement of their extremities and thinking and daily functioning abilities without significant side effects, Sung noted.
Heather Thurgood Wilmoth, who works for SuperTalk Mississippi Media but is also the wife and stepmother of people suffering from Huntington’s, is actively working with others impacted by the disease to urge the FDA to accelerate the approval process. The FDA had originally scheduled uniQure to present its findings in the first quarter of 2026 but has since delayed the meeting, Wilmoth said.
“The FDA, back in 2024, seemed to agree that this was something that was needed and we needed something for the Huntington’s community, because there is no cure. They can treat symptoms, but they can’t treat the disease,” she explained. “For whatever reason, they’ve gone back on that and said that they were not going to take a look at AMT-130 early next year. We just want a fair review from the FDA.”
Wilmoth is one of the leading local forces behind a pair of petitions addressed to the FDA that have received over 41,000 signatures combined and urges the agency to approve AMT-130, extending “a lifeline for thousands of families who have waited generations for a glimmer of hope.”
She’s also been in contact with various congressional members to see if they can encourage the FDA to move forward. According to her, the office of Rep. Michael Guest (R-Miss.) has already sent an inquiry to the agency.
“Congressional members could be integral because this would hopefully push the FDA or encourage the FDA to take a fair review of AMT-130. AMT-130 is the first thing we’ve had as hope in this community,” Wilmoth said, tearing up. “It’s the closest thing we have to hope. Now it may not help [my husband] but it could help our daughter, it could help other people who are just starting this journey with Huntington’s.”
A spokesperson for the FDA said in an email to SuperTalk Mississippi News that neither they nor any other officials within the agency can comment on AMT-130 or when it might be considered for approval. Around 200 families across Mississippi are affected by Huntington’s while an estimated 41,000 suffer from the disease in the U.S.