Health · Medical Research · 3 hrs ago
Drug Combination Improves Parkinson’s-Like Symptoms in Mice
Researchers tested a two-drug treatment in mice with Parkinson’s-like movement problems.
The study was conducted by a team including scientists at Harvard Medical School, and the mice developed symptoms after researchers triggered a buildup of the protein alpha-synuclein in their brains.
Parkinson’s damages dopamine-producing brain cells, and problems with cells’ energy-producing mitochondria may disrupt how they use oxygen.
The drugs reduced oxygen delivery to tissues to try to limit this mismatch, and treatment began after the mice had developed movement problems.
Over six weeks, treated mice improved in tests of movement, while untreated mice continued to worsen.
The experiment did not show whether the drugs protected dopamine-producing cells, and one anxiety-like behavior measure did not improve.
The approach remains untested in people, and the researchers say more work is needed to assess its safety and effectiveness before human trials.
An experimental drug combination improved movement problems in mice with Parkinson’s-like symptoms.
Treatment began after the symptoms had developed and continued for six weeks.
The combination reduced excess oxygen in the brain and a marker of fat damage in brain tissue.
The study did not establish whether the drugs protected dopamine-producing nerve cells.
The researchers said the approach remains preclinical and needs much more study before it can be tested in humans.
- Who
- Hong Wang and colleagues tested the drug combination in mice.
- What
- Two compounds improved movement tests in mice with Parkinson’s-like symptoms.
- When
- Treatment began six weeks after the researchers triggered symptoms and lasted six weeks. The study was published on October 22, 2026.
- Where
- In mice; the study’s location is not stated.
- Why
- The researchers aimed to reduce a mismatch between oxygen delivery and oxygen use in cells.
This story does not have two clearly opposing sides.
Researchers reported that mice with Parkinson’s-like symptoms benefited from breathing air with 11 percent oxygen.
Researchers began giving the mice the drug combination orally each day.
Treated mice improved in pole and cage-hang tests, while mice given a liquid control kept getting worse.
The study was published in PNAS.
- Drug combination
- GBT601 and PT2399
- Treatment start
- Six weeks after movement problems developed
- Treatment duration
- Six weeks
- Low-oxygen comparison
- 11 percent oxygen; ordinary air contains about 21 percent
- Other disease model
- Leigh syndrome mice had median lifespan increase from 62 to 158 days
What people are saying
Our current working hypothesis is that when there is mitochondrial dysfunction, oxygen is not being consumed properly, and this leads to an oxygen mismatch, i.e., oxygen delivery is greater than oxygen utilization
Hypoxia can be dangerous, so an important challenge is being able to achieve hypoxia therapy that is safe and effective
All of our work to date is pre-clinical, in mouse models, and much more work is required before moving this concept into humans.