PFS Research in 2021: PFSNetwork fundraising for research projects with the PFSFoundation in Summer

Dear fellow PFS patients,

2021 is shaping up to be perhaps the most promising year for PFS research and awareness ever.

While the moderator team are pleased with the progress made since we addressed the forum in December, it is still entirely unacceptable that there remains a stark lack of clinical understanding of this debilitating disease.

No patient should have to feel unheard by their clinician when explaining their devastating symptoms are caused by a supposedly safe cosmetic drug. No patient should have to suffer for months, years or even decades. No patient should have to take their own life because suitable treatment options do not exist.

While this painful reality remains for now, we are taking important steps to increase understanding and awareness of our problem.

Over the past two years, we have taken advice from over a dozen scientists to weigh up what and who we can endorse as the most practical offers to further investigate our condition. This in itself is no small feat.

We are pleased to announce that we are intending to support two opportunities designed by leading researchers that we would be very excited to have involved in investigating PFS further. The first will be a lower cost study, in the mid-five figure range, that we hope to begin fundraising for in the summer so we can hopefully begin later this year, and the admin team have already pledged a substantial amount of the funds required. The second will be a larger study and a longer-term fundraising goal. We are also thrilled that the PFS Foundation has agreed to support us by handling fundraising for this landmark endeavour. This will be dedicated fundraising and not affect usual donations to the Foundation, so donations should not be currently made for these coming studies. We encourage any patients who may be able to contribute large sums, whether directly or through their family, to get in touch directly via PM.

While we cannot provide specifics yet for several months, and a lot depends on the availability of the researchers, this is without a doubt a watershed moment in our long journey.

Looking back

Our message as a team working hard over several years has been clear and consistent: there is no magic bullet for this complex condition and only by collectively taking small steps can we hope to find relief and peace from this disease that has taken so much from us all.

In recent times, these collective small steps have included developing the patient information pack to provide to clinicians, PFS being effectively noted on Wikipedia for the first time, and the development of our survey which has received over 400 responses, and will now be analysed and interpreted to provide the world’s largest dataset about our condition.

When we provided our end of year update, we outlined several immediate goals that were crucial in laying the groundwork for these upcoming studies. They were:

  • Launch a new information site and social channels for PFSNetwork (pfsnetwork.org), our “parent” brand.
  • Launch our first series of patient video stories on YouTube.
  • Create the first-ever professionally made animated explainer video to quickly outline what PFS is and how it affects patients’ lives.
  • Receive study proposals that we can support and fundraise for through our platform.
  • Find a technical volunteer to help with the survey data analysis.

We are so pleased to have had success with these important efforts through the hard work of our team, and our community pulling together. This was a key aim of our organisation since 2018. Our new website, pfsnetwork.org has been live since Rare Disease Day (February 28th) and coherently presents the current understanding of PFS and the impact of PFS on patients’ lives in a clear way. Six patients spoke publicly about the devastating impact of PFS on their lives - we cannot thank those who participated enough - and we are thrilled with the message and quality of our explainer video.

To have received interest and potential for investigation from a number of high-calibre researchers is an overwhelming success for us. Our team has shouldered the burden of providing propeciahelp to patients for many years, at huge effort and cost. We could not support the first nor simply the most enthusiastic offers. Our team all have to be confident in the proposals, the scientists carrying them out, and how any donations from our community will be handled. Axo has a long-standing relationship with the Foundation, and with their help, we are all confident it’s time to move forward in this regard.

While necessary steps sometimes appear slow, we hope it is apparent there is a critical momentum that we hope will turn the tide on PFS understanding and awareness, and pave the way for a future where patients never have to face what we have alone.

Now, we must try to increase the pace of these steps.

Moving forward

For a long time, we expressed our concern that the investigation of PFS and the enthusiasm to do so could be left in the hands of very few doctors and scientists that do not account for or acknowledge the breadth of patient symptoms. Individuals may have strong opinions, but the fallout of this disease is shouldered by us. Any further delay in effective investigation is an increase in our burden.

It is important to note that our decisions have been informed by over two years of consultation with leading molecular biologists and centres. This is not a proposal we have pulled from a hat, haphazardly. Everything that will be pursued has been recommended to us by more than one accomplished scientist - independently - and builds on recommendations in the literature of PFS.

Please note the conversations were provisional, and one of the researchers is currently busy with projects until summer so we cannot make concrete promises or share details yet. I can say we deeply hope to finalise a patient cell focused investigation that will require the participation of patients who developed the syndrome relatively quickly and severely. It will correct inappropriate decisions by the investigators in the Harvard study.

Once we have received the study design proposal, hopefully by summertime, both PFSNetwork and the Foundation will present it to our community to begin fundraising. While we are several months away, it is important that we inform you of these coming proposals now. We are acutely aware of the damage suffered by PFS patients, and how it often costs us our lives, including our careers and financial security. Several of our admins are seriously disabled by the extent of the harm. We understand it is not easy to contribute large sums at short notice.

However, the moderator team have already pledged a substantial amount, so the study will be well underway. We encourage patients to begin thinking about what you, or your family, may be able to contribute when the time comes. The first study cost is relatively low. We are hopeful that we can raise the amount through our patient community, and they will be promoted with a clear progress banner where the current survey participation banner is.

Other projects: awareness above all

Since the launch of our new site, you may have seen me banging the drum about speaking publicly about your condition. I again implore every patient to ask themselves this simple question: “What is more important - my life or my anonymity?”

These kinds of complex and stigmatising issues are not resolved by the affected population remaining silent and anonymous. That is simply a fact. No one else will solve this for us. There is no doctor out there who will have an understanding that doesn’t yet exist, and no bureaucratic manoeuvre that can simply take care of this.

For years before I got involved I watched the community circle the drain with tenuous ideas, self-made pharmaceutical or alternative therapies that may prove effective for some but devastating for our worst affected, and constant new groups that most often completely disappear. Personally, it is frustrating to watch precious energy and time being wasted on fruitless endeavours that attempt to put a roof on a house before the concrete slabs have even been laid.

There is one incredibly powerful thing that every PFS patient who is persistently and badly affected can do, which is speaking publicly about what has happened to you. To support this, I am starting a new PFS video podcast that will interview patients, patients’ families or family survivors, and clinicians, in an effort to develop more awareness and a single source of well-presented content about the impact of PFS. We have already lined up the first guests for the series, and I am personally very excited to begin.

If you would like to speak, please reach out to me privately. I would like to again thank our four new brave patients - @Toughluck24, @teddy25, @pokertje and @anon74895881 - who came forward last week. Your efforts are deeply appreciated.

You may also be aware that Tzinkman has been severely unwell over the past six months, and has sadly not improved. SkinDiesel is also badly affected and has not been able to contribute as he would like recently. So if you are enthusiastic and would like to contribute to our recent momentum, please reach out.

In closing

I must again reiterate the importance of collectivism in surmounting this formidable problem. Year after year effort is duplicated that distracts and disappears. As the admin team, we can’t always communicate our discussions publicly, for many reasons, so we appreciate that it sometimes feels like no progress is being made. But there are clear and beneficial ways to help right now. Please also sign up to the mailing list for PFS Network, if you haven’t already.

We have so much to look forward to in 2021, if we work together. For perhaps the first time in our history, there is real, tangible momentum on every front.

Best,
Mitch

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Extremely well written Mitch, considering everything!!!
Im up for contributing in anyway i can and i admit too often going my own way in order to try to lift the lid on this publicly.
My brain is as castrated as my manhood so IT skills and concentration have been hammered as well as many other cognitive functions but if there is anything you or the guys can think of give me a shout.
I tried to contribute financially recently but had trouble with it, my brain!!! I will provide larger amounts if i gain full confidence in the direction of the forum. I doubt im alone in this.
Can you send me a link please and ill retry.
It may also be worth moving it to a more prominent place so that it gets the viewers attentiion early on in their journey.
I know a few people who are quite well off who like to donate to the likes Macmillan on birthdays via FB etc.
Getting away from the ED side which is often peoples sole view and showing the true suffering which i think the videos do will make people realise that this is a worthwhile cause for donation!!!
Maybe pull together a vid clip with a supporting narrative from the work thats already been done soley for donation purposes. . We could then fan it out via fb, twitter etc. Just a thought.
Sorry to hear about tzink and skind- just awful.

Thanks for the update once again mate great piece!!

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Thanks for the kind words as always Ryan. We all appreciate the massive awareness efforts you’ve put in over the years - you tell the story of PFS so well.

We are not ready to start fundraising yet, so I don’t have a link, but please start thinking about what you and others you know may be able to contribute!

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Thanks Mitch I will, !! My sister is very supportive and one of the few thats been in my corner/ sees the devestation. She would definitely contribute from a family members perspective. Let me know and ill give you her contact details. She gave input to the book that MJ Payne is in the process of writing.

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Are any of the new research projects going to overlap with what the Baylor project is doing?

Hi @lakehouse do you mean will we be working with Baylor, or will the study designs be of similar nature?

the objectives of the study.

are they capturing similar data to baylor? or different?

Unfortunately we can’t reveal that just yet @lakehouse as the researcher is busy with other projects for a couple of months.

We will inform the patient community as soon as we can, but this post was intended to share the initial good news with the group, and to ask all patients to start thinking about what they or their families could contribute when the time comes. Most likely in the Summer.

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OK, so bottom line - if you’re planning to donate a significant sum, donate specifically to this new initiative when the time comes. Meaning - not the PFS foundation or the PFS network?

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Almost @M_C. Although the likely projects will be arranged by PFS Network (PH admins), in collaboration with the researchers who will design the study, the fundraising will be conducted through the Foundation.

Since PFS Network is not a registered charity, we will not fundraise ourselves. Setting up a nonprofit is an option for us, but more and more organisations are unnecessary when we have a longstanding cooperation well with the PFSF. So when the time comes, we’ll be using up a ringfenced fundraising account with the Foundation and they will administrate the funds. However, the funds will need to go through the specific links we provide here as the PFSF will be setting up a second account to earmark these funds for the studies.

TL;DR. Hold your dollars for now, and when the time comes, we’ll let you know how you can donate to this exciting new study. All plans will be fully outlined beforehand.

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Okay fair but I’m curious

  1. would this study be private and deliver results very fast?

  2. would this study at least potentially help us get closer to finding therapeutics?

Given all the fervour surrounding Baylor and other studies in the past, I don’t think it would be wise of me to say anything further unfortunately. We will provide full details at the time - this is just a heads up there will be an opportunity we’ll be supporting in the summer.

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Excuse my ignorance, but what would be the point of fundraising future studies without a. Knowing whether the Baylor epigenetics study will come out, and if so when and b. What will be contained within the study.

Fair point man, but we’re at a point whereby we have to move on, with our without Baylor. If Baylor comes out and reveals something significant, great. If it doesn’t, we continue as we are.

There’s also the aspect of the relevant technology being much more advanced and also cheaper than it was at the time the Baylor study was initiated, so continuing in this direction will hopefully yield far more detailed and reliable results.

Something similar to CRISPR?
Also thanks for addressing my questions, I thought maybe you admins knew something the rest of us didn’t but it doesnt seem like it.

Not CRISPR, no. CRISPR is not yet widely available to the public and is a gene editing technology, not a diagnostic tool.

I assure you man, no moderator knows anything about Baylor. There’d be absolutely no reason for any of us to keep any info regarding that study to ourselves. We’re as in the dark as you are.

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Thanks for replying again, could you tell me the name of this new diagnostic technology?

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You mentioned interviewing clinician in your post; I haven’t seen this doctor’s video posted on the forum yet. Maybe you could get in contact with him?

(https://www.youtube.com/watch?v=Cybeqpn1txQ&t=11s)

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Thank you…you have my support when contributions are needed.

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Any updates on when this will get started?