Author Topic: Lecture on amyloidosis at International Society of Nephrology meeting  (Read 11259 times)

Pat Martin

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  • Diagnosed: Dec 15
Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #15 on: August 12, 2017, 12:36:15 pm »
This Lecture is a MUST WATCH for amyloidosis patients and a big Thank you to Miriam for making us aware of this Superb lecture by Professor Sir Mark Pepys and the ISN link.
Pat you were not wrong when you pointed out that the lecture was very accessible and understandable to us non medical patients.  I feel it provides hope for Amyloidosis sufferers that there are new positive treatments on the horizon.
The comment regarding Proteinuria to Nephrologists: "THINK AMYLOIDOSIS" was interesting to me as for 2/3 years pre-diagnosis, maybe longer, I had fizzy urine. I had a Medical MOT at during that time and was told that I was fit and healthy. I wish I had known about proteinuria at this time, as I would have mentioned this change during the tests, but, would these changes have been picked up? I am glad the Specialists are becoming more aware as this will filter down to the general public, which can only be good news.

Best wishes to all

Pat Martin

patpinchin

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #16 on: August 13, 2017, 01:46:15 pm »
Spot on Pat.

Thank goodness that after quite a struggle, you were diagnosed in time. It should have been earlier as we and you now know.  That's the difficulty Drs face when a patient presents with a set of non specific rather vague symptoms relating to many other more common diseases. Amyloidosis as a rare disease is not usually a part of Drs' diagnostic thinking. Amyloidosis Awareness is clearly a real challenge.

During Sir Mark's lecture you couldn't fail to be struck by the sheer passion and enthusiasm in his voice. This is a brilliant Dr/Scientist who had dedicated more than 40 years his life to amyloidosis.
Dogged determination, energy and perseverance were in there too. As my daughter Chloe, a seasoned fundraiser for the Amyloidosis Research Fund said, 

"You don't achieve things like that by being laid back!"

As a retired teacher, i fully understand how well you have to understand your subject yourself to be able to explain it very well to others. Sir Mark is also a very good teacher. It is quite something when a gifted talented  Dr/Scientist can explain something complex in a way which ordinary patients can understand. The SAP scanned images were very appropriate to illustrate what Professor Hawkins described at the AL Amyloidosis Infoday in 2015 as " unprecedented clearance" of amyloid from the liver and spleen. "Amazing," is an over-used word these days to describe really basic things. In the true sense of the word it really was amazing to see that sticky hard insoluble amounts of deposited amyloid had cleared away quite quickly from the affected organs with this new combination treatment.

Whilst listening to the lecture, I was reminded of one of Sir Mark's well known sayings from several years ago...........

"it is easier quicker and cheaper to put a man on the moon that it is to invent and develop a new medicine for patients!" 

Would that Sir Mark's simple message "THINK AMYLOIDOSIS" does become a part of every Dr's diagnostic thinking.  What a breakthrough life saver that would be to promote amyloidosis awareness and earlier diagnosis amongst the medical community and ordinary people.  :)


« Last Edit: August 13, 2017, 04:45:41 pm by patpinchin »
Pat

nvroberts

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #17 on: August 15, 2017, 10:56:26 am »
Not seen the lecture yet, but I will.

The "Think amyloidosis" message is very important. I had abnormal and increasing amounts of protein in my urine for about 3 years before anybody thought of testing for amyloidosis. My kidney function was down to about 25% before I was recommended for a biopsy that showed the amyloidosis. Up until then my GP had just assumed the problem was a result of my type-2 diabetes. I don't blame them for that, it is the obvious conclusion, but it is a pity nobody spotted the real problem earlier.

patpinchin

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #18 on: August 18, 2017, 03:53:06 pm »
In addition to his excellent so very well illustrated explanation of the development and mechanism of miridesap and dezamizumab, it was very heartening to hear at the end of his lecture, Sir Mark express immense appreciation to the Medical Research Council, other orgainisations, not to mention GSK for the financial and enthusiastic support to enable his new treatment to reach phase 2 in clinical testing. As always, Sir Mark made a point of including his indebtedness to donors and organisers, mostly NAC patients, family members and friends of the numerous voluntary fundraising activities for the Amyloidosis Research Fund at the NAC. So now ordinary NAC patients who are also NAC fundraisers and donors have been heard about by the many Nephrologists from across the world at the ISN conference in New Mexico in April 2017.

It is not perhaps well enough known by patients especially newcomers of the importance of the Amyloidosis Research Fund on which the NAC depends to suppport its clinical work and research. If it has escaped your notice, next time you go to the NAC look out for a large poster in a glass case near the double entrance doors at the NAC publicising the Amyloidosis Research Fund. Just to give newcomers an idea of how much the NAC depends on charitable work and donations to the ARF, it is worth reading the fundraising news and a personal letter of thanks written to Wayne Shaw last year from Sir Mark. Wayne and his small team at the Window Company in Manchester had made the Amyloidosis Research Fund their chosen charity of the year raising over £8,000 from a number of different fundraising activities. The cheque on behalf of the Window Company was presented personally to Sir Mark by NAC patient Lesley Toft.

You can read about this is on page 8 and much else besides in this NAC newsletter.
http://www.ucl.ac.uk/amyloidosis/pdfs/nac_newsletter_8I
« Last Edit: August 18, 2017, 04:00:35 pm by patpinchin »
Pat

Miriam Vered

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #19 on: September 11, 2017, 10:17:15 am »
I've posted the full lecture on the NAC Patient Information website now. it can now be viewed directly there, no need to register for anything.
just click here


( http://www.amyloidosis.org.uk/essentials/lecture-prof-sir-mark-pepys/)


patpinchin

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #20 on: September 12, 2017, 10:25:54 am »
Thank-you so much for this Miriam.

I hope now that Sir Mark's inspirational and informative lecture will be heard and watched by many more NAC patients, because you have made it so very easy to access on the NAC website. It's rightful home!

Sir Mark's lecture is mind blowingly impressive. Although aimed at a medical audience, Sir Mark understands exactly how to explain the mechanism of miridesap plus dezamizumab in patient friendly language especially for something as complicated as amyloidosis. The history of his research and the development of his new treatment, a world first, is also very interesting too for those for whom it is new.

 I feel extremely privileged to have met Sit Mark and to be a patient at the NAC which Dr Mark Pepys (as he was at the time) established as the first specialised amyloidosis clinic in the U.K. way back in the early 70s.  So much has progressed both with the now very well established NAC clinic (1999) as the National Referral Centre for amyloidosis and in clinical research for amyloidosis, which of course patients do not see. However it's so important that all NAC patients know and understand at least something about the world class scientific developments taking place at the NAC right under patients' noses tho' behind closed doors as is rightly the case.

I urge all forum members to listen to and read the slides in Sir Mark's keynote lecture. I hope Miriam you will also include the NAC website link in the next issue of the NAC newsletter which all NAC patients receive. :)
« Last Edit: September 15, 2017, 11:45:06 am by Miriam Vered »
Pat

nvroberts

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #21 on: September 15, 2017, 08:07:02 am »
Just viewed the lecture. As everybody says, it is extraordinarily informative even to a layman, and offers great hope for all of us.

The "Think amyloidosis" message, it seems to me, needs to be spread far and wide, and not just to specialist nephrologists.  In my own case, as I think I have said elsewhere on this site, substantial protein loss was detected several years ago, suggesting kidney problems, but as I am also type-2 diabetic my GP, quite naturally one might argue, blamed this on the diabetes and tried to treat it with various changes to my drug regime.  It was only after at least 3 years, when my kidney function declined to about 20%, that I was sent for a biopsy and amyloidosis was diagnosed.  So the first thing I am going to do is insist that the head of my local G.P. practice views Sir Mark's lecture, in the hope that diagnosis of amyloidosis in the kidneys can be more effective.

The next question that any amyloidosis patient who has seen the lecture will ask is, of course, "Fantastic; where do I get some of these new drugs".  We all understand that it is not that simple, and the treatment has to go through the standard clinical trial processes before it can be prescribed on a more general basis.  But is there anything that we, as patients, can do to speed up this process?

Miriam, thanks to Sir Mark for his extraordinary work, and is there anything we can do to help get this disease defeated more rapidly?

Miriam Vered

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #22 on: September 18, 2017, 09:47:00 pm »
I've passed on your message to Sir Mark. He and the other NAC  consultants are all very grateful to you and to other patients and relatives who are involved in raising amyloidosis awareness amongst your own doctors and of course in invaluable fundraising. They are also very aware of the potential "patient power" may have at some point in the future for overcoming regulatory or other obstacles to advance drug access. But they feel it is necessary to be careful to exert such power judiciously, at the appropriate time for maximum effect. There is no need for it at the moment as we await the results of the ongoing trials.

patpinchin

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #23 on: September 19, 2017, 10:12:02 am »
Thank you Miriam.

I fully understand.
Pat

patpinchin

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Re: Lecture on amyloidosis at International Society of Nephrology meeting
« Reply #24 on: September 19, 2017, 01:46:43 pm »
In reply to NV Roberts......

Please do tell us your name. It is so much nicer to "talk to" a named person.

You are right, extraordinary world class scientific expertise and passion for amyloidosis is very evident in Sir Mark's lecture. A dedicated team of talents characterises the whole NAC team.

 I too have forwarded the link to Sir Mark's lecture to my local Haematologist who correctly diagnosed me without the slightest hesitation thanks to a prompt referral from my GP to whom I have also forwarded the link. Dr Jack always tells me he passes around amongst his hospital colleagues the amyloidosis newsletters from the NAC and AL Amyloidosis Matters published by Myeloma UK which I give him 5 times a year. Owing to my diagnosis the GPs  in my local practice have all been well informed about amyloidosis since 04 when I was their first ever amyloidosis patient. As a direct result of receiving all the amyloidosis newsletters published annually, my GP has told me that in service training in the GP practice has included a session on amyloidosis every few of years where the newsletters and NAC website have provided so much of the important information disseminated amongst the GP group, particularly to new Drs. I would like to think that my local GPs might well listen to Sir Mark's lecture in another in service trainng session. 

Your remark about accelerating new treatments reminded me of the remark made by Sir Mark in 2013 in a letter to a veteran fundraiser for the Amyloidosis Research Fund at the NAC. Sir Mark has a unique capacity to come out with memorable poignant remarks as he did then when he wrote.....

"Sadly, drug development, that is the invention of new medicines and all the work needed to make them available for patients, is generally agreed to be the slowest, most difficult and most expensive undertaking conducted by the human race! It is quicker and easier to send a man to the moon."

You can read Sir Mark's complete letter here:

http://www.ucl.ac.uk/amyloidosis/pdfs/nac_newsletter_2

I think our Drs are as frustrated as are we patients about the legislative hoops Drs and scientists have to jump through and the "glacial pace" of drug development to accelerate new treatments for patients. Professor Hawkins told attendees at the AL Amyloidosis  Infoday in 2016 that typically it takes at least 20 years or longer for the development of new treatments from test tube in the lab to patient.


















« Last Edit: September 20, 2017, 11:27:39 am by Miriam Vered »
Pat