I thought the webinar was excellent. I hope many of you were able to attend either by the combo of web and phone, or by phone alone (as I did).
Apparently CurePSP intends to post Dr. Golbe’s PowerPoint presentation to its website some time soon. I do not believe a recording of the webinar was made, unfortunately.
“PSP” was mentioned much more often than CBD. It was unclear if some of the information was PSP-only or applied equally to both PSP and CBD. I think each item would need to be addressed with Dr. Golbe.
The following are some notes I took during the presentation. The notes include what I could recall of the questions and answers. If any of you attended the webinar, please share your notes and key take-aways!
There are two types of PSP: Richardson’s Syndrome and PSP-Parkinsonism. The most common type is RS. One-third of those with PSP have PSP-Parkinsonism (PSP-P). PSP-Parkinsonism looks like Parkinson’s Disease. People with PSP-P live longer. They are less likely to have dementia or visual problems, and they are less likely to fall. Those with PSP-P are more likely to experience tremor. (Robin’s note: If the head of the CurePSP Scientific Advisory Board is using the “two types of PSP” terminology, I conclude that it has gained acceptance among PSP experts.)
On average, those with PSP live 7 years after symptom onset. Broken down, those with RS live 5.9 years after symptom onset and those with PSP-P live 9.1 years after symptom onset. On average, those with PSP are diagnosed 3.5 years after symptom onset. This does not allow for much time for treatment. If you have an earlier age of onset, you survive longer.
In order to get people diagnosed earlier, we need to educate MDs. If MDs see unexplained falls, they should immediately think of PSP. MDs should check for eye movement problems.
There’s a list of supportive features. These are not required for a PSP diagnosis. They are simply “common symptoms” that can be “helpful clues” to an MD in making a diagnosis. (Examples: symmetric bradykinesia, retrocollis.)
In all his years caring for those with PSP, Dr. Golbe has never seen someone with Whipple’s Disease, though it is commonly screened for as part of the PSP diagnosis.
A defect in the protein tau causes: 1) disintegration of microtubules, and 2) clumping. The disintegration of microtubules causes problems in the brain. The tau tangles are the result of problems with tau.
There are three key issues in PSP:
1) too much 4-repeat tau is produced.
2) there is too much oxidation. (Rusting is one form of oxidation.) Too much oxidation damages molecules.
3) the mitochondria is not working properly.
These three key issues lead to the creation of tau tangles. Any treatment in PSP likely to have some success in slowing the disease progression needs to effect one of these three key problems.
The incidence of PSP is 1 per 100,000 people. The prevalence of PSP is 6 per 100,000 people. By comparison, Alzheimer’s Disease is 800 per 100,000 people. (Robin’s note: I believe 800 is a prevalence number.) The incidence of CBD is thought to be less than 1 per 100,000 but the incidence and prevalence of CBD have never been studied.
Of the brains at the PSP Brain Bank where the patients was diagnosed in life with PSP, only 70% actually had PSP. Those who were misdiagnosed actually had: CBD (8%), a continuum of DLBD/PD (3%), ALS (1%), and FTD (1%). FTD is “another tau disorder.” (Robin’s note: Some FTD disorders are not tau disorders but are ubiquitin disorders, so Dr. Golbe’s comment is confusing to me.)
In looking at first-degree relatives of those with PSP, 39% showed abnormalities on various rating scales. This 39% figure suggests that something genetic is going on.
There is evidence of environmental toxins. An example of this is tropical fruit. There is currently research going on into the dietary habits of those with PSP.
In research conducted 20 years ago, there was a *slim* suggestion that those with PSP were less likely to be educated, and more likely to be a factory worker or exposed to toxins used on a farm.
We can’t biopsy the brain and diagnose PSP or CBD. Confirmation of the diagnosis is only possible on post-mortem exam of brain tissue. (Robin’s note: It seems that one or two people on the CBGD_support Yahoo!Group have indicated that their loved ones *did* receive a biopsy and CBD was confirmed.)
A research paper will be published soon looking at the use of CSF (cerebrospinal fluid) in diagnosing PSP.
A family with a history of PSP was studied. We know the general location but not the specific gene associated with PSP.
Standard medication given to those with PSP and CBD includes levodopa (Sinemet) and amantadine. Levodopa sometimes helps with the slowness and stiffness. Amantadine can help sometimes for a few months.
Other medication has unproven benefits:
* amitriptyline (Elavil). Due to side effects from this medication, Dr. Golbe has stopped using it.
* ChEIs. Sometimes these help with dementia.
* SSRIs can help disinhibition.
* CoQ10 is focused on slowing the progression of PSP. It does *not* help with symptoms.
Namenda seems to be poorly tolerated in those with PSP.
Not all symptoms progress at the same rate. There is huge variability.
Sense of smell is reduced a little in PSP.
There are PSP animal models in a type of fish, fly, and worm, and in rats.
An ’05 study showed that lithium reduced hyperphosphorylation of tau. The NIH decided to fund a lithium trial in PSP and CBD. No site involved in the study has received approval yet to proceed recruiting patients. It was noted that the info on the NIH-funded lithium trial is not on the curepsp.org website.
CurePSP is funding the PSP Genetics Consortium. The goal was to have 1000 PSP brains to include in the study. They’ve ended up with 1300 brains. This is a DNA study, a genome study.
There was a question about estimating survival time in someone with PSP. Dr. Golbe replied that the best tool for prognosticating is the PSP Rating Scale he developed. Generally, patients decline at a rate of 10-12 points per year. When a patient gets to 70 points, they usually live for six months. Often the issue is the immobility leads to medical complications. (Robin’s note: You can find the PSP Rating Scale on the CurePSP website — https://www.psp.org/materials/rating_scale.pdf I used this with my dad and found it accurate.)
There was a question about the experimental drug Rember that was discussed at a recent Alzheimer’s Disease conference. Dr. Golbe said he and others are skeptical of the results reported at the conference. There are problems with the design of the trial and the data. The analysis was also suspect. The principal investigator of the trial has a conflict of interest.
There was a question about stem cell research. (Robin’s note: I’m not confident I got all of Dr. Golbe’s statements down correctly so if others listened in please help me out here!) Dr. Golbe noted that there are currently no treatment trials for stem cell therapy. We can’t replace all the dead cells in the brain. He said that stem cell therapy in the early stages of PD is relevant to the PSP world. The closest approach with relevance to PSP is to encourage cells to produce chemicals.