This common parasite could be used to treat brain diseases
A study published in Nature Microbiology suggests that Toxoplasma gondii, a common parasite, may hold potential for treating brain diseases. The research indicates that this single-celled organism can cross the blood-brain barrier and infiltrate brain cells. This unique ability could be harnessed to deliver therapeutic proteins into these cells, potentially revolutionizing treatment approaches for neurological disorders.
Overcoming the blood-brain barrier challenge
The blood-brain barrier, a protective layer of cells lining the brain's blood vessels, often limits treatment options for neurological conditions. This barrier acts as a gatekeeper, preventing many drugs from reaching neurons. However, Toxoplasma gondii's ability to cross this barrier could provide a new avenue for drug delivery into the brain. The parasite is known to infect all animals and can be transmitted through contaminated food or water.
Engineering Toxoplasma for protein delivery
In a significant development, scientists have genetically modified Toxoplasma to produce a hybrid protein. This protein combines one of the parasite's secreted proteins with MeCP2, a protein that regulates gene activity in the brain. The engineered parasites were found to release this hybrid protein into neurons grown in a laboratory setting as well as in the brains of infected mice.
Potential treatment for Rett syndrome and other disorders
A deficiency in MECP2 causes Rett syndrome, a rare brain development disorder. Current gene therapy trials are using viruses to deliver the MeCP2 protein to treat this condition. If Toxoplasma can deliver a form of MeCP2 protein into brain cells, it may provide another treatment option for this currently incurable condition, and other neurological problems such as Alzheimer's and Parkinson's disease.
Challenges and risks in using Toxoplasma for treatment
Despite its potential, using Toxoplasma for medical purposes presents significant challenges. The parasite can cause a severe, lifelong infection that is currently incurable and potentially damaging to critical organs including the brain, eyes, and heart. Additionally, up to one-third of people worldwide already carry Toxoplasma in their brain which could disqualify many of them from using it for treatment due to developed immunity against future infection.