Patients with Huntington’s disease — and their doctors — need to know if a therapy is working. Currently, however, that's very difficult to do.
But now, researchers at Stanford have found a way to test the effectiveness of treatment on patients with Huntington’s disease. The study appears in The Journal of Experimental Medicine.
“Clinical trials for patients with Huntington’s take a long time,” said Daria Mochly-Rosen, PhD, senior author of the study. “There is a need to figure out early on in the treatment whether the therapy is having an effect and if you are dosing patients correctly.”
Mochly-Rosen and the lead author of the study, Marie-Hélène Disatnik, PhD, identified half a dozen biomarkers in the body accesible through a blood or urine sample that can indicate the progress of the disease. They found that levels of biomarkers such as mitochondrial DNA differed greatly between healthy mice and mice with Huntington’s disease. They also discovered biomarker differences between several human patients with Huntington's and those of healthy controls.
“We don’t need to wait until patients have severe disease progression to understand if their treatment is working,” Mochly-Rosen said. “We hope that the use of these biomarkers will guide us.”
In addition, the researchers were able to test the efficacy of a drug they had previously developed called P110 by examining its effect on the biomarkers. They found that treating mice with Huntington's disease with P110 improved their biomarker readings, suggesting that P110 was helping treat the disease.
They now plan to study a larger number of patients to confirm these results.
“We hope that these biomarkers can help in clinical trials of other neurodegenerative diseases as well,” Mochly-Rosen said.
Previously: Tiny drug-bearing balls of gold may be just the right size to quench dementia, "The uncertainty was killing me": A student's tale of genetic testing for Huntington’s disease and When proteins go bad: Quality control inside the cells
Photo by PublicDomainPictures