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[ USA ] Potassium Channels as Highly Promising Therapeutic Target for ALS – Alleviation of Neural Hyperexcitability; Cambridge, Massachusetts

Human body fluids are aqueous solutions containing electrolytes (ions). Normally, ions cannot move in and out of cells, but there are “holes” in the cell membrane called ion channels. These channels open and close in response to external stimuli, and ions can enter and leave the cell through the channels only when these holes are open. It is known that this creates the senses of smell, taste, and touch. In addition, the movement of ions in and out of cells is essential for the brain to think and muscles to move. This flow of ions in and out of cells is called “ion permeation. There are different types of ion channels: channels that allow sodium (Na+) ions to pass through well are called Na+ channels, and channels that allow potassium (K+) ions to pass through well are called K+ channels. In other words, channels selectively allow Na+ and K+ ions to pass through. This topic is about one such channel, the potassium channel.

Potassium channel Kv7.2/3 channels, otherwise known as M currents, are responsible for suppressing neuronal excitability. As such, they are currently of great interest as potential therapeutic targets for amyotrophic lateral sclerosis (ALS). A number of studies have shown that activating these channels may alleviate the hyperexcitability observed in ALS motor neurons and slow disease progression. This is because hyperexcitability destroys motor neurons and exacerbates neurodegeneration.

In ALS patients, excessive levels of motor neuron excitability are often observed due to impaired potassium channel function, including reduced activation of K v7.2/3 channels. Kv7 has already been validated as a therapeutic target to suppress hyperexcitability in epilepsy.

Last December 2024, QurAlis https://www.quralis.com/, a company located in Cambridge, Boston’s neighboring city, launched the world’s first Phase 1 Clinical Trial (Phase 1 Clnical Trial) of Kv7 precision therapy in ALS patients. QurAlis is a young biotech company founded in 2016 by several world-renowned neurobiologists from Harvard Medical School.

The trial, called QRL-101, is the only Kv7 ion channel opener trial currently being studied in ALS patients with disease progression due to neural hyperexcitability (reportedly 50%). The trial is also being conducted in healthy volunteers for biomarker evaluation, not only in ALS but also in epilepsy. QuaAlis expects to present top-line data from the trial in the first half of 2025. QuaAlis believes that data from ALS patients in the trial, as well as the first data from healthy volunteers, will be essential in determining optimal drug dosing, allowing evaluation of efficacy and bringing much-needed new treatment options to ALS patients as soon as possible. The news article quotes the CEO of QurAlis as saying that he believes the data will be essential for knowing the optimal drug dosage and evaluating its effectiveness.

https://www.prnewswire.com/news-releases/quralis-doses-first-patient-with-als-in-phase-1-clinical-trial-evaluating-qrl-101-a-first-in-class-kv7-precision-therapy-for-als-302321243.html

Kasper Roet, PhD, CEO and Founder of QurAlis

 

Team members in QurAlis  

The table below shows ongoing research by QurAlis that covers ALS and epilepsy, as well as FTD and PSP.

I will follow the news about this trial in the first half of this year and in the second half of the year.

Reporter: Nobuko Schlough , USA on Jan 28, 2025

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