CaV1.1-linked hypokalemic periodic paralysis: causes and treatment approaches
Official title: Disease Pathogenesis and Modification for CaV1.1-Associated Hypokalemic Periodic Paralysis
Funded at University of California Los Angeles · NIH-11235194 · Data updated 2026-11-12
This project studies how CaV1.1 gene mutations cause low‑potassium muscle weakness and explores ways to stop attacks and slow permanent weakness in people with hypokalemic periodic paralysis.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Los Angeles NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project start | July 1, 2012 |
| Project end | November 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $551,662 |
| NIH project number | 5R01AR063182-13 |
| Project ID | NIH-11235194 on NIH RePORTER |
Who could benefit from this research
✓Could help
People diagnosed with hypokalemic periodic paralysis who carry CaV1.1 (CACNA1S) mutations are the primary candidates to benefit or participate.
✕Less likely to help
People whose muscle weakness is due to other causes or who lack CaV1.1 mutations (for example those with only SCN4A mutations or unrelated neuromuscular disorders) may not benefit directly.
What this research studies
Researchers at UCLA are studying the faulty calcium channel (CaV1.1) caused by CACNA1S mutations that lead to low‑potassium paralysis. They measure abnormal 'gating‑pore' leak currents in laboratory models and use computer simulations to see how small leaks depolarize muscle cells. The team tests strategies to block or compensate for the leak in cells and animal models and watches how those changes affect muscle excitability. Results are intended to point toward targeted therapies to prevent attacks and reduce long‑term permanent muscle weakness.
Why it matters
Potential benefit: If successful, the work could lead to treatments that prevent low‑potassium attacks and reduce long‑term muscle weakness in affected patients.
How similar studies have gone: Prior studies identified the gating‑pore leak mechanism and current symptom treatments give limited relief, so directly targeting the gating pore is a relatively new and unproven therapeutic approach.
Where this research is happening
-
University of California Los Angeles
Los Angeles, United States Active
Researchers
- Principal investigator: Cannon, Stephen C. — University of California Los Angeles
- Study coordinator: Cannon, Stephen C.
Related research
Other NIH-funded projects on the same topics.
-
Neuromuscular Junction Problems in Hyperkalemic Paralysis
A clinician-scientist is pursuing five years of advanced training and research to better understand and care for people with neuromuscular diseases related to hyperkalemic paralysis.
-
Restoring movement after paralysis with AI-powered copilots
A patient-facing AI-aided system would help people with paralysis perform everyday tasks by interpreting the user’s intent and guiding assistive robots.
-
CoPE II: Personalizing patient-reported outcomes for vocal fold paralysis care
Aimed at tailoring patient-reported outcome measures to each person with unilateral vocal fold paralysis to better guide treatment decisions.
-
Mobile brain-computer interface to help veterans with paralysis control devices
Aimed at improving a mobile brain-computer interface to help veterans with paralysis better control assistive devices at home.
-
Preventing awareness during paralysis in emergency ventilated patients and reducing PTSD
This project will change emergency department medication defaults to cut back on a paralytic drug and see if that lowers the chance that adults put on ventilators wake up while paralyzed and later develop PTSD.
-
Muscle stem cells to repair paralyzed vocal cords
This project uses muscle-derived stem cells to rebuild and strengthen paralyzed vocal cords in adults with vocal fold paralysis.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.