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There is a lot of excitement in the PSP community at the moment about new research, and for once I think some of it is justified.
I recently read an article in Being Patient about the latest PSP clinical trials and the possibility that we may finally be moving towards treatments that do more than simply deal with symptoms.
That is a big sentence.
At the moment, there is no approved treatment that stops or slows PSP. We treat the consequences. We manage stiffness, eye problems, swallowing, balance, sleep, pain and dozens of other problems as they appear.
But we do not currently treat the disease itself.
Researchers are now trying to change that.
One of the most advanced drugs is NIO752, being developed by Novartis. It is designed to reduce the production of tau, the abnormal protein that builds up in PSP, and it has now moved into a major Phase III trial.
There are also several other possible treatments being tested, or prepared for testing, through a new PSP clinical trial platform.
I am not going to pretend I understand every molecule, antibody or biological pathway involved.
I understand the important bit.
For the first time in a long time, there are serious attempts to slow PSP itself.
That is genuinely exciting.
As someone living with PSP and trying to advocate for others affected by it, I am thrilled by that progress.
Better treatments matter. Better biomarkers matter. Earlier and more accurate diagnosis matters enormously.
If researchers can identify PSP earlier and then actually do something to slow it down, the experience of being diagnosed with PSP could look very different in the future.
That is the hopeful part.
Then comes the more personal part.
It is unlikely to help me.
There are two main reasons.
The first is that the two major new trials attracting much of the current attention are specifically concentrating on PSP-RS, or Richardson syndrome, the most common form of PSP.
I seem to have PSP-P, or PSP-Parkinsonism.
PSP-P has been included in some previous research, but it is not the focus of these major new trials. So there is already a gap between the research being celebrated and the particular form of PSP that I appear to have.
The second reason is timing.
I am now approximately six and a half years into PSP.
The major trials are generally looking for people much earlier in the disease. The NIO752 Phase III study, for example, is aimed at people whose symptoms began less than five years ago and who are still at a relatively earlier stage of PSP.
The new PSP trial platform has similar restrictions.
I understand why.
If you are trying to discover whether a drug slows a progressive disease, it makes sense to start before too much damage has already occurred.
Scientifically, that is entirely logical.
Personally, it puts me on the wrong side of the line.
Even if I had the right type of PSP, I would almost certainly be too advanced for the current trials.
Neither of these major programmes currently offers me an obvious trial route in Israel either, although in truth that is probably the least important obstacle.
Then there is the calendar.
Even if NIO752 proves successful, there are still years of testing, analysis, regulatory approval and practical decisions about how a treatment would actually reach patients.
Even if everything goes well, we are probably talking about the early 2030s before a treatment could become meaningfully available internationally.
Nobody yet knows exactly when, or indeed whether, that will happen.
By then I would be more than eleven years into PSP.
There is also another question that nobody can yet answer.
If a drug can slow the production of tau, how useful will it be once PSP has already caused many years of neurological damage?
Slowing what happens next is one thing.
Repairing what has already happened is something very different.
Importantly, the fact that people with more advanced PSP are largely excluded from these trials does not prove that a successful treatment could not help someone later in the disease.
We simply do not know.
So I have mixed emotions about all of this.
I am excited and genuinely hopeful. I am delighted for the PSP community, and grateful to the researchers, pharmaceutical companies, charities and, particularly, the patients volunteering for these trials.
But there is sadness too.
It is difficult to live with an incurable progressive disease, hear that treatments may finally be getting closer, and not wonder whether they will arrive in time for you.
For me, the answer is probably no.
I would be lying if I said that did not sting a little.
There may even be a touch of envy.
Not because I want anybody else to miss out. Quite the opposite. I desperately want these treatments to work.
I just wish I could be at the right stage, with the right type of PSP, at the right time to benefit as well.
I think I am allowed to admit that.
But there is another way of looking at it.
When I was diagnosed, the conversation was essentially about coping.
Perhaps somebody diagnosed in five or ten years will have a very different conversation.
Perhaps their neurologist will be able to say:
“We have caught this early. There is something we can give you that may slow it down.”
That would be extraordinary.
It would change what a diagnosis of PSP means.
Maybe not for me.
But for somebody.
As both a patient and an advocate, I can live with feeling two things at once.
I can be sad that this progress may come too late for me, while still being genuinely delighted that it may come in time for somebody else.
That is still hope.
