"The work could lead to the first treatment that actually targets the underlying defect in Fragile X syndrome and not just the symptoms," Dr. Paribello said.
UCR's Douglas Ethell, an assistant professor of biomedical sciences, noted that effective therapies for Fragile X syndrome are few and far between. "This is a good time for identifying highly effective therapeutic strategies that might work in Fragile X patients," he said. "We are excited that our research has the potential to affect many lives."
Fragile X affects 1 in 4000 males and 1 in 6000 females of all races and ethnic groups. About 1 in 259 women carry Fragile X and could pass it to their children. About 1 in 800 men carry Fragile X; their daughters will also be carriers.
Minocycline belongs to a group of antibiotics that has been used in people for more than fifty years to treat Lyme disease, acne, and other skin infections.
Minocycline may have beneficial effects in other disorders where higher-than-normal brain levels of MMP-9 are found. It is currently under study for treating rheumatoid arthritis, multiple sclerosis (MS), Parkinson's disease, and several other neurodegenerative conditions.
"In the future, new compounds that more specifically target MMP-9 can be developed and tested," Douglas Ethell said.
Next in their research, the Ethells and their colleagues plan to refine the therapeutic strategy in Fragile X mice to determine the optimal age, if any, to administer minocycline. They will also explore other MMP inhibitors that may be more effective than minocycline.
"We will investigate whether a combination of MMP inhibitors with other drugs, such as fenobam, can help mature the synapses in Fragile X mice," Iryna Ethell said.
|Contact: Iqbal Pittalwala|
University of California - Riverside