Toronto Star

The clinical trial that’s offering fresh hope to families

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The people: 300 volunteers 100 are paisa mutation carriers who receive the drug.

100 are paisa mutation carriers who receive a placebo.

100 are non-carriers from the family who receive a placebo. 0 know if they are carriers. 0 know if they are receiving the drug or a placebo.

The theory: That the buildup of amyloid plaque in the brain is the main cause of Alzheimer’s, and removing it will prevent or delay dementia.

The medicine: A drug called crenezumab, designed to sweep the amyloid out of the brain. It is licensed by Genentech, an American subsidiary of the Swiss pharmaceut­ical company Roche.

The method: Administer­ed by injection every two weeks for five years.

The participan­ts: May already have amyloid buildup in the brain, but have no symptoms.

The good news:

Though crenezumab failed in a clinical trial last year in people who already had dementia, the news was positive for Lopera because people in the study with milder forms of dementia showed better responses. The hope: If the drug works, it will be immediate good news for the paisa families. Further testing would be required to determine if and how it could work for people with non-familial, late-onset Alzheimer’s. The tests:

1. PET scan to measure amyloid in the brain

2. FDG (fluorodeox­yglucose)-PET scan to assess brain functionin­g by glucose levels

3. Spinal tap to check levels of amyloid and tau, another protein, in the cerebrospi­nal fluid

4. MRI to assess whether the brain is shrinking

5. Cognitive tests to check for cognitive decline The timeline: The trial ends five years from the day the 300th patient receives his or her first injection, likely in 2021. Amy Dempsey

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