Developing a test to evaluate therapeutic delivery

Frontotemporal dementia (FTD) is the most common form of dementia for people younger
than 60. Up to half the people who develop this type of dementia have widespread and severe
buildup of a toxic protein in their brains. This toxic protein causes neurons’ degeneration,
leading to patient death in a few years. Research shows that by removing the toxic protein,
neurons have a chance to survive. That means that the neurons within the brain regions that
were affected by FTD can be saved, hence stopping or slowing the progression of patient’s
symptoms.
We have developed a therapy that can clear this toxic protein directly inside of the neurons.
One of the problems related to this therapy is that there are no images (like MRI), blood tests
or biopsy methods available to follow it up. This means that we cannot “see” it, and that it is
very difficult to tell whether or not the treatment is working. Patients, their doctors and families have to live in the uncertainty of “wait and see” over time, not only causing major
distress for them, but also impairing our ability to conduct proper clinical trials with new
dementia treatments.
We aim to develop a new test that can inform doctors and patients about treatment’s
efficacy. This would be the first test of its kind in the world. We are designing this test such
that a simple blood sample can be used, making it feasible for a patient with dementia, for
rapid translation into the clinical setting and for real and effective world use.
Furthermore, there are many other toxic proteins causing different types of dementia. Once
proven to be successful, our method can be extended into other types of dementia,
significantly advancing the evaluation of any future new therapy.