Tag: als
Roberta Flack’s ALS diagnosis has made it impossible for her to sing
A press statement confirmed the singer’s condition and insisted that “it will take a lot more than ALS to silence this icon”
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Soft robotic device stimulates muscles, sparks hope for ALS and MS patients
Today, muscle atrophy is often unavoidable when you can’t move due to severe injury, old age or diseases like amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS). However, Harvard researchers see hope in soft robotics that could someday stretch and contract the muscles of patients unable to do so themselves.
The Harvard engineers tested a new mechanostimulation system on mice, successfully preventing or assisting in their recovery from muscle atrophy. The team implanted the “soft robotic device” on a mouse’s hind limb, which they immobilized in a cast-like enclosure for around two weeks. While the control group’s untreated muscles wasted away as expected, the actively stimulated muscles showed reduced degradation. The researchers believe their system can eventually lead to implants helping humans with atrophy.
Its promise stems from its ability to induce a small mechanical muscle strain that mirrors natural stimulation during exercise. Moreover, while keeping atrophy at bay, the device didn’t lead to any severe tissue inflammation or damage.
“There is a good chance that distinct soft robotic approaches with their unique effects on muscle tissue could open up disease or injury-specific mechano-therapeutic avenues,” said David Mooney, Ph.D., the paper’s senior author and Harvard’s Wyss Institute engineering faculty member.
Dubbed MAGENTA (short for “mechanically active gel-elastomer-nitinol tissue adhesive”), the anti-atrophy system includes an engineered spring made from nitinol, a shape memory alloy (SMA) that can rapidly actuate when heated. Researchers control the spring with a wired microprocessor unit that determines the frequency and duration of muscle contractions and stretches.
The system also includes an elastomer matrix forming the device’s body and providing insulation for the heated SMA. In addition, a layer of “tough adhesive” keeps MAGENTA aligned with the muscles’ natural movement axis while transmitting stimulation deep into muscle tissue.
“While untreated muscles and muscles treated with the device but not stimulated significantly wasted away during this period, the actively stimulated muscles showed reduced muscle wasting,” said first-author and Wyss Technology Development Fellow Sungmin Nam, Ph.D. “Our approach could also promote the recovery of muscle mass that already had been lost over a three-week period of immobilization, and induce the activation of the major biochemical mechanotransduction pathways known to elicit protein synthesis and muscle growth.”
The team also experimented with a wireless version, using laser light rather than electrical wiring to actuate the SMA spring. Although this approach showed reduced effectiveness due to fat tissue absorbing some of the laser light, the researchers believe this approach still holds potential and warrants further research.
FDA Approves ALS Drug Whose Study Was Partly Funded By Ice Bucket Challenge
In November, Amylyx submitted a drug application to the FDA for the medication, then called AMX0035, as an oral ALS treatment, seeking approval based on a Phase 2 trial that included 137 people with ALS who received either the drug or a placebo for 24 weeks. The study was funded in part by a grant from the ALS Ice Bucket Challenge, the viral social media campaign that started in 2014 involving people dumping buckets of ice water over themselves to raise awareness and money around ALS. The trial also showed that the drug was generally well-tolerated, but there was a greater frequency of gastrointestinal events in the group getting the medication. Amylyx is now continuing to study its safety and efficacy in a Phase 3 trial. In March, the Peripheral and Central Nervous System Drugs Advisory Committee voted 6-4 that a single Phase 2 trial did not establish the conclusion that the drug is effective in treating ALS.
One key difference between the FDA advisory committee’s March and September meetings is that in the later meeting, Amylyx indicated that if the drug was approved but its Phase 3 trial results fail to confirm the drug’s benefits, the company would consider withdrawing the drug from the market, Lynch said. She added, however, that the company didn’t say specifically what it would view as a failure. “So at the vote, the advisory committee members switched, and most of them said, ‘Yes, we are now convinced that this product should be approved.’ And when they were asked why they changed their minds, some of them said, ‘Well, the company said they would withdraw,'” she said. “And they were also convinced by patients’ testimonies that they very much want to try this drug.” But overall, the FDA’s approval was based on Phase 2 trial data, which, Lynch said, may send a message to other pharmaceutical companies that they don’t need robust Phase 3 trial data to get products on the market. Although people with ALS want access to this promising drug, there are concerns that such a message could open the door more broadly to the approval of medications that have not been proved to work, says Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania. “The FDA could later withdraw those products if needed, she said, but doing so without voluntary company agreement is ‘a huge pain’ and often requires a very lengthy process,” reports CNN.
Read more of this story at Slashdot.