On hope…Again

(After receiving a lot of feedback on the therapy mentioned in the article below I have decided to re-release this piece.)

Seemingly every few months the same story plays out in degenerative disease communities around the world. News circulates of a breakthrough therapeutic development, of resurrections performed in a line of mice or miraculous recoveries in a handful of patients. Foundation or pharma dollars pour in, anticipation builds, hope rises. Only for the story to slowly fade as results trickle in that fail to live up to expectations.

One such story is playing out right now in the Parkinson’s community. The therapy this time is focused ultrasound, a new tool that allows surgeons to non-invasively lesion parts of the brain, supposedly cutting off the faulty wiring believed to be responsible for certain symptoms. Groups have been filling up online with stories of patients paying up to thirty five thousand dollars for the procedure. Many of those patients then rush back to the group, eager to announce their results. Their stories then inspire many more patients to dig deep into their bank accounts to get the procedure. Neurodegenerative diseases can be hell to live with and these anecdotes are awfully enticing. Even as I read again them a part of me wonders, maybe they are on to something this time? To hell with the clinical trial process and whatever my doctor might say, the proof is in the pudding. These are real people that I can reach out and talk to who seem to have had their lives instantly transformed for the better. Maybe it is worth a shot?

But then the questions come. Is what this patient is experiencing a placebo? Even if it does have some benefit, how long will it last? Just how safe is it? And how do I know this will work for me?

So, what to do? How to figure out what to believe and what not to believe? Where should I place my hope?

These questions are a regular part of living with a degenerative condition. Time is the enemy and new and improved therapies all seem to be perpetually 5-10 years away. It can be comforting, even therapeutic, to pin ones hopes to something new.

Eventually though I come around and remember that we have a tried and tested means of separating fact from fiction. Science and the scientific method. It is a boring answer I know. Science moves at a crawl and is too often blanketed by jargon, paywalls and rigid hierarchies. But for all its faults, it is the best tool we have for knowing where to place one’s faith.

I look to the pandemic for hope in what science can do. The vaccines being injected into millions of arms around the world did not come from a belief in what might happen if we just give them to people. It came from a detailed and thorough understanding of biology and how our bodies interact with the virus. That is why we can confidently move forward and intervene in what nature has wrought.

My hope is that all members of the neurodegenerative disease community can develop the patience, rigor and fortitude needed to do the same for these ailments. It is a tough ask, especially for patients who should continue to be impatient by always demanding more, by pushing to reform our fractured healthcare systems and the exclusive nature of science. But before we go flying off to distant clinics to shell out thousands for experimental procedures, remember that these are experiments and you are the subject being used to fill in all the unknowns we have about the procedure you are paying for.

As for the scientists, my hope for you is to keep in mind that educating and including the public must be part of everything you do. From climate change to the vaccine rollout to future cures for neurodegenerative diseases, for science to do all that it can do, resources and time must be devoted to explaining why each is important. The good news is it doesn’t take much. For a tiny fraction of the resources being spent advancing science, a much broader impact can be had by devoting some of those resources to educating as many people as possible as to why that science is important.

I’ll leave by paraphrasing one of the great philosophers of our time whose aphorisms are proving to be quite prescient these days…

“To alcohol education, the cause of, and solution to, all of life’s problems.”

Homer Simpson

Note added after initial posting

I received a lot of feedback on this article and it made me to realize that I inadvertently clumped Focused Ultrasound in with a lot of pseudoscience. That was not my intention. Below is a thoughtful response I received from Dr. Julian Lo that includes a bounty of useful information on FUS for those that want to learn more…

Without evidence, one might categorize this procedure along with the Herbal Garden cure-for-all scams. But I have yet to see an article about it in Skeptical Inquirer or Quackwatch. So, let’s look at the research and judge for ourselves. But first, let’s be clear that we are talking about PallidoThalamic Tractotomy (PTT, the Swiss procedure), not Pallidotomy or Thalamotomy that exists in many treatment centers around the world. The start of using FUS on the brain was in 1964 by Russell Meyers and William Fry.

https://sonimodul.ch/parkinson/ 

Dr. Jeanmonod explained why and how PTT works. The earliest publication on PTT and PD that I could find is this 

https://www.cambridge.org/…/C476825097C1495F66035B93CCC…

What are the differences?

  • Parkinsonian tremor – target in the thalamus (thalamotomy)
  • Parkinsonian dyskinesia – target in the globus pallidus (pallidotomy) or subthalamic nucleus
  • Parkinsonian tremor or akinesia – target in the pallidothalamic tract

Source:https://www.fusfoundation.org/diseases-and-conditions/neurological/parkinsons-disease

Unfortunately, many sites (look up treatment sites there), including all 14 U.S. sites, using the necessary equipment ExAblade, have chosen to do only pallidotomy and/or thalamotomy (lesioning the brain in a different place, with different effects). In Canada, there is one commercial treatment site and 3 clinical trial sites; none is for PTT. In contrast, Japan has done PTT for dystonia (2019) https://pubmed.ncbi.nlm.nih.gov/31207377/ in their Tokyo Women’s Medical University Hospital and for PD (2019) in the same hospital https://pubmed.ncbi.nlm.nih.gov/30336294/. Part of their conclusion, “PTT might be useful in patients who do not desire device implantation.” The case study of 51 consecutive surgeries from SoniModul Clinic in Switzerland has been published.

https://www.frontiersin.org/articles/10.3389/fsurg.2019.00076/full

What we learn is that, well, read the abstract at least. From the conclusion, “MRgFUS PTT was shown to be a safe and effective intervention for PD patients, addressing all symptoms, with varying effects.” Despite the publicity for FUS-PTT (and side effects from the other procedures (see below)), Insightec (the maker of the ExAblate) is still doing clinical trials on pallidotomy, not PTT, for dyskinesia. This trial involves 20 sites worldwide with 92 test subjects.

https://clinicaltrials.gov/ct2/show/NCT03319485…

and on Thalamotomy for tremor dominant PD:

https://clinicaltrials.gov/ct2/show/NCT01772693…

A recent publication of the more common thalamotomy, not the PTT, indicates this procedure is plagued with side-effects. 

https://www.nejm.org/doi/full/10.1056/NEJMoa2016311?source=nejmtwitter&medium=organic-social&fbclid=IwAR1gtDn3WD1T8xZrW-x3-4Xce2mgI3Po-msYe2fETUJfGCT0dDXwHki2KQk

This is disappointing. My belief is that PTT is a trade secret of SoniModul of Switzerland. They are under no obligation to teach the rest of the world about their technology. But Japan has been learning and performing PTT on patients. Other countries may be starting the learning process. PTT has passed the placebo-effect stage, with patients already having success more than 5 years after the surgery. The question is when it will become available in your country, having skillful surgeons, for all PD patients, especially the PD patients who are still responding to levodopa. So, to all advocates, I’d suggest that you give PTT serious consideration.

“ To alcohol education, the cause of, and solution to, all of life’s problems.” – Homer Simpson

Yes, choose education at your own risk.

8 comments

    1. This article is well written and well reasoned. However, it’s not like some random guy set up a table on a street corner and started performing focused ultrasound procedures. Two forms of Focused Ultrasound for Parkinson’s Disease have undergone rigorous clinical trials and are FDA approved in the United States. A third form (PTT) has been approved and used in Switzerland for nearly a decade and is now the subject of clinical trials in Japan. We all need to decide for ourselves, but I remain very encouraged and excited.

      1. Appreciate the feedback and I agree, though it was not the purpose of the article, I did not properly lay out the case for Focused Ultrasound and may have inadvertently clumped it in with a lot of pseudoscience. It is a promising line of therapy that deserves a proper clinical trial. Thank you for providing some added context. I will be adding an additional edit shortly that provides more.

  1. On Sat, Jan 16, 2021 at 11:34 AM Tomorrow Edition wrote:

    > Benjamin posted: ” Seemingly every few months the same story plays out in > degenerative disease communities around the world. News circulates of a > breakthrough therapeutic development, of resurrections performed in a line > of mice or miraculous recoveries in a handful of” >

  2. I have wrote the initial text, and wrote with the intention of call the attention for this new tecnique. (Sorry about my english, i’m not a native speaker) Jump on to this decision was no light and not easy to make. And only could do it because i had finantial help. After 13 years of diagnostic, where i tried everything, pills, alternative medicine, thc, cbd, HDT (high dose thiamine) Vitamins, exercise (almost runned a marathon with 10 years of PD), learned to eat well, i have made my search and my choices acording with it was avaliable right now, as after 13 years of fight, i knew my situation would degradate fast, and had to make an option. My neuro pushed me into DBS, but with 47 years, i knew i didn’t wanted to be dependent on an electronic device to move myself for the rest of my life. I deal with electronic devices, and they failllure, a lot…And i have seen to many stories if bad DBS results. And this are not all roses, it has side effects, althoug the benefits still are much larger. And doctors said the brain needs to reballance, and that needs time. only a week went trough. My main simptoms on my treated side really have gone. No more tremor, no dyscnesia at all, no distonia on foot, no rigidity, but… it seems that my brain doesn’t know well the limits of my body, my fine dexitricity is strange ( i have to look to understand what i’m doing. Hopping this all vanishes with time (Drs. told me 3 months at least) and i still suffle walk (especially with my untreated side, unless i’m doing fast walk, and i am a little more stoppet overall. Some other minor but important changes… urinary urgency has stopped, a bit of return of smell sense and improved sexual drive (sex and parkinson, we always know how it starts but we never know how it ends, right). I have 3 children and i need to be here for them. this was clearly my best option available at this time. Fell free to contact-me and i will put you on a video call so you can check for your self. Cheers

Leave a Reply to Marco Serrabulho Cancel reply