Often times it seems that research for the rarest of conditions is brushed aside. Primary attention is given to more common illnesses which affect more people. For instance, in the world of dementia diagnoses, we hear about alzheimer’s research more than we hear of research for progressive supranuclear palsy (PSP) or corticobasal degeneration (CBD). But things are changing and more research is underway for these rare conditions.
The first ever International Research Symposium for these conditions provided many updates on the latest research research. It was held October of this year in London and its brought a lot of hope to the PSP and CBD communities.
There are currently three different antibodies being studied for PSP and CBD treatment. These include UCB107, ABBV-8E12, and BIIBO92 which are all humanized IgG4 which bind in different regions of the tau. All of these potential therapies have phase 1 or phase 2 clinical trials underway but there are no major updates to be shared. Most of the trials are safety trials, first examining if the treatment will produce any adverse effects for patients.
At this point, it is still unclear which of these three different antibodies will be most effective for PSP and CBD patients but all showed efficacy in trials with mice.
Despite their potential, there is some controversy regarding these trials because they are utilizing patients who have already progressed very far in the disease. Some say that this is not the most ideal population to study. However, researchers testify that they don’t have much of a choice in terms of which population they utilize. They simply have to be one hundred percent certain the patients they are studying have the correct form of the disease. Right now, they can’t be positive with patients in the early stages.
That means the most pressing research needed for these conditions is what early symptoms are unique to the illnesses. If we can confirm diagnosis earlier, we can begin to study the efficacy of drugs in younger patients as well. Thankfully, there are some ongoing observational studies which should give us more information on the early stages of these diseases.
In sum, much of the research for these conditions is still in the early stages. However, even so, these clinical trials do mark progress and we’re excited to see their outcomes.
The research symposium also gave an update on the patient registry which was founded in 2015. It now has over 1,900 individuals registered which is incredible progress. In addition to providing researchers with more information on patient experiences, the founders of the registry hope that it will foster collaboration among researchers. This registry should also help spread awareness about these rare conditions within the scientific world.
You can read more about the registry, specifics on each individual clinical trial, and additional updates from the International Research Symposium here.
There’s lots more to be done, but we’re making strides for PSP and CBD.