National
Amyloidosis
Centre
News
Panel discussion held at the 6th UKAN Meeting (March 2014) on the place of stem cell transplantation and
chemotherapy for AL amyloidosis. The discussion was chaired by Prof Philip Hawkins, Director of the NAC,
far left.
Panel members, from left to right:
Dr Stefan Schoenland (Heidelberg, Germany),
Dr Giovanni Palladini (Pavia, Italy) and Prof Ronjon Chakraverty (Professor of Haematology and Cellular
Immunotherapy, Royal Free Hospital).
Introduction
The 6th Annual UK Amyloidosis Network (UKAN) Meeting, hosted by the NAC
in March 2014, was well attended by doctors and researchers from around the
UK, with guest lecturers from amyloid centres in Europe and the US. Lectures
included state of the art updates on all types of amyloidosis and a lively panel
discussion addressed the place of stem cell transplantation versus
chemotherapy for AL amyloidosis. Patient members of the UK Amyloidosis
Advisory Group (UKAAG) were present and later met with the NAC consultants
for discussions on how the NAC can continue to excel in fulfilling patient
needs.
NAC consultants and scientists from the Wolfson Drug Discovery Unit
attended and lectured at the XIV International Symposium on Amyloidosis, in
Indianapolis, USA, from 27 April - 1 May 2014.
Decades of world-leading research at the NAC have led to huge strides
forward in understanding and treatment of amyloidosis. Doctors around the
UK are increasingly aware that existing treatment options can improve
symptoms and prolong life. Excitingly, more new treatments than ever before
are now in various stages of development and trial.
Our website includes recently updated information for patients on all the
ongoing clinical trials at the NAC, many of which are currently recruiting
patients. See http://www.ucl.ac.uk/amyloidosis/nac/clinical-research.
This newsletter discusses some of the imaging scans available at the NAC and
some recent clinical research studies. There is an update on the upcoming
Land’s End to John O’Groats bike ride, scheduled for July, which will kick-start
the ambitious project aiming to raise funds for a new MRI suite at the NAC.
We are also happy to report on some of the successful fundraising for the UCL
Amyloidosis Research Fund. This issue’s patient story tells Sandra Morgan’s
inspiring tale of how a combined heart and kidney transplant 21 years ago
gave her a chance to live a full life, despite a diagnosis of a rare type of
amyloidosis at age 35.
ISSUE 4: June 2014
IN THIS ISSUE
Introduction
1
Strides in imaging
2
Conquering CAPS
4
Patient story:
Sandra Morgan
Professor Sir Mark Pepys’s
perspective
5
Fundraising news
8
Donations
National Amyloidosis Centre, UCL Division of Medicine, Royal Free Campus, Rowland Hill Street, London NW3 2PF, UK
www.ucl.ac.uk/amyloidosis
6
12
National Amyloidosis Centre News
2  Issue 4: June 2014
Strides in imaging
DPD scans
The heart is in constant motion, contracting and relaxing
as it pumps blood around the body. SAP scanning under
routine conditions is not useful for assessment of
amyloid deposits in the heart because the organ is
moving, because it contains a lot of blood with the
labelled tracer within it and because it takes some time
for SAP to enter the cardiac amyloid deposits.
heart tests appear normal. DPD scans are less useful for
detecting and evaluating AL amyloid deposits in the
heart, so we do not routinely perform DPD scans on
patients who are known to have AL amyloid affecting the
heart.
At the NAC, the DPD tracer is scanned on the same Single
Photon Emission Computerised Tomography (SPECT)/CT
gamma camera as that used for the SAP scan.
However, a different radioactive
marker that does detect amyloid in
the heart has recently been identified.
This marker is called 99mTc-DPD, or
just DPD.
DPD is a “bone-seeking” tracer.
When it is injected into the
bloodstream it is mainly taken up by
bones all through the body. It is
taken up most strongly in bone
regions in which there is disease or
abnormality. In some countries in
Europe DPD is used routinely in bone
scans to detect a variety of bone
illnesses. In the UK it was used for
bone scans in the past, but at present
other tracers are preferred.
The procedure is as follows:
• The DPD tracer is injected into the
David Hutt, Lead Nuclear Medicine Technician at
the NAC. Together with Professor Hawkins,
David developed the DPD scan as a standard test
for cardiac amyloidosis.
Over the last 30 years there have
been several case reports in which bone-seeking tracers
appeared to home in on amyloid deposits in the heart, as
well as detecting bone abnormalities. However, these
were generally viewed as incidental findings occurring in
patients who underwent bone scans for other reasons
than amyloidosis.
In recent years this approach has been investigated
systematically for the first time. Since 2010 we have
performed over 1000 DPD scans on patients at the NAC
with suspected amyloid in the heart. They are also
performed in some other European amyloidosis centres.
The results have been impressive, and have contributed
significantly to our ability to evaluate patients with
cardiac amyloidosis.
It turns out that DPD is very sensitive at detecting
particular amyloid deposits in the heart. DPD scans are
most useful when the amyloid deposits are of TTR type.
Asymptomatic ATTR amyloid deposits can be detected in
the heart by DPD scans at an early stage, when other
patient’s vein.
• It is necessary to wait 3 hours after
injection of the tracer before we can
begin scanning.
• For each scan, the patient lies
down on the scanner and there is a
scan consisting of a “whole body
sweep”, which shows the entire body.
• The scanning camera is rotated
and a type of scan called SPECT of the
heart is performed while the patient
remains in the same position. At the
same time a routine high resolution
X-ray CT scan is obtained.
The combination of the 3D SPECT DPD
scan and the standard X-ray 3D CT provide detailed, 3dimensional information, showing both the anatomy
(structure) of the heart and the pathology (amyloid
deposits) in the heart.
Since DPD scans can detect ATTR amyloid deposits in the
heart before other tests, they may be helpful in guiding
treatment decisions, including timing of liver
transplantation in:
•
•
Asymptomatic patients who are known to carry
mutations in the TTR gene that cause familial
amyloid polyneuropathy.
Patients with early familial amyloid polyneuropathy
who do not yet have heart failure symptoms.
DPD scanning and SPECT are safe tests. There is some
exposure to radiation from the tracer and from the CT
scan, but the exposure is minimal and is comparable with
the annual background radiation people are exposed to
in some parts of the country.
National Amyloidosis Centre News
CMR (cardiac magnetic resonance) scans
CMR is a method whereby a magnetic field and radio
waves are used to obtain detailed pictures of the heart.
Information provided by CMR is more detailed and
accurate compared with echocardiography. Further, in
some patients, echocardiography may not be able to
determine whether heart wall thickening is due to
amyloidosis or to another cause such as hypertension. In
such patients, CMR can distinguish between these
different causes of heart wall thickening. In CMR
imaging, a non-radioactive tracer called gadolinium is
used to enhance the images. Amyloid deposits in the
heart produce a very characteristic appearance after a
short delay ‘late gadolinium enhancement’ (LGE) . Other
patterns of LGE occur in other types of heart disease, for
example if there is fibrosis, or scarring of the heart
muscle.
Experienced doctors can recognise the
particular pattern of LGE which is associated with cardiac
amyloid deposits, and researchers at the NAC have
developed sophisticated new methods using the
gadolinium tracer to quantify the precise amount of
amyloid in the heart.
A new scoring system to improve diagnosis of
cardiac amyloid
A recent NAC study showed that CMR imaging may help
doctors to tell whether amyloid deposits in the heart are
of AL or ATTR type. These are the two types of
amyloidosis that most frequently affect the heart. In the
past, the vast majority of patients with amyloid in the
heart had AL amyloidosis, but in the last 5 years we have
seen a marked increase in new cases of ATTR amyloid
affecting the heart amongst patients referred to the NAC.
Over the same time period, CMR has become more
widely available. AL and ATTR amyloidosis are different
diseases with very different treatments, so it is crucial to
distinguish between these conditions.
The NAC team, together with colleagues from
St George’s University Hospital, London, looked at CMR
scans from 100 cardiac amyloid patients, about half with
AL amyloidosis and half with ATTR amyloidosis. The
scans had been performed in 46 different hospitals
around the UK. As discussed above, amyloid deposits in
the heart cause a characteristic CMR finding called LGE.
The researchers developed a new scoring system based
on the LGE patterns they observed. They called it the
Query Amyloid Late Enhancement (QALE) score. When
used together with other clinical and CMR features, the
Issue 4: June 2014  3
QALE score was very accurate in distinguishing between
ATTR and AL amyloid.
This could prove helpful to a growing number of patients,
because of the increasing availability of CMR.
Echocardiography is also widely available, but does not
always reliably distinguish between amyloid and
non-amyloid cardiomyopathy, and cannot distinguish
between AL and ATTR amyloid. The DPD scan which can
help distinguish between AL and ATTR amyloid in the
heart, is only available at the NAC, where personnel have
sufficient experience in scan performance and
interpretation, and has only been studied in a relatively
limited number of patients so far. CMR is now available
in hospitals all around the UK, and the scans analysed in
this study were performed in centres without specialist
amyloid protocols. So QALE scoring may become helpful
in diagnosis and management of many patients, although
it will not replace biopsy as the gold standard diagnostic
test.
In the future it may also be possible to use MR to
accurately measure the size of the amyloid deposits
within both the heart wall and other body tissues. Such
measurements could then be repeated to follow the
build-up of amyloid deposits and their regression with
treatment.
Before the scan, contrast
material may be injected
into the patient’s vein.
During the scan, patients
lie still inside a closed
“tunnel” type of scanner
for up to one hour.
The pictures produced by the computer can then be
examined by doctors. CMR is safe and painless, and does
not involve any exposure to radiation.
CMR is not currently available in the NAC, so we refer
patients to another hospital for this type of scan if
necessary.
The success of our research has led to an ever increasing
need for this new investigation. The Land’s End to John
O’Groats bike ride, planned for July 2014 (details on
page 10) will kick off our fundraising efforts for a new
MRI suite at the NAC. This will reduce the need for
patients to travel, shorten waiting times for scans and
allow us to scan more patients.
4  Issue 4: June 2014
National Amyloidosis Centre News
Conquering CAPS: An NAC success story
How NAC research led to effective treatment for a rare cause of amyloidosis
NAC doctors recently published a report of the impressive success they have had in treating a condition which
can lead to development of AA amyloidosis.
Cryopyrin-Associated Periodic Syndrome (CAPS) is a very rare, inherited disease, usually diagnosed in childhood.
Untreated, patients with CAPS experience daily flu-like symptoms from early childhood, and sometimes go on to
develop AA amyloidosis.
In 2001, ground-breaking research by the NAC team, led by Professor Philip Hawkins and Dr Helen Lachmann, the
fever syndrome specialists, led to identification of the gene family, mutations in which cause CAPS. Based on this
new understanding of the disease, Professor Hawkins went on to identify the first ever effective treatment for
CAPS. This is a very rare achievement for any doctor and is vitally important even though it is such a rare disease.
Subsequently the NAC doctors played a crucial role, in collaboration with the pharmaceutical company, Novartis,
in development of a new drug, canakinumab, which effectively ‘switches off’ the underlying disease process
causing CAPS symptoms. Patients treated with canakinumab injections every 2 months have found that this
incredibly effective drug has turned their lives around. In addition to significantly improving their day to day
quality of life, long term canakinumab treatment will prevent the development of the most serious complication
of CAPS - AA amyloidosis.
The NAC doctors share a specialist paediatric fever clinic with Dr Paul Brogan and Professor Nigel Klein at the
Great Ormond Street Hospital for Children, where they collaborate in treatment of children with CAPS. The NAC
and Great Ormond Street doctors recently published a report of the impressive success of this profoundly
effective treatment in relieving the symptoms of patients with CAPS.
Response of rash to canakinumab in a patient with the Cryopyrin-Associated Periodic Syndrome (CAPS)
Immediately before the administration of the initial dose of canakinumab, this patient had a typical
urticarial rash (Panel A). With 24 hours after the administration of a single dose of canakinumab, the rash
had almost completely disappeared (Panel B). From Lachmann H J et al. Use of Canakinumab in the
Cryopyrin-Associated Periodic Syndrome. N Eng J Med, 2009; 360: 2416-2425 (Copyright © 2009
Massachusetts Medical Society). Reprinted with permission.
National Amyloidosis Centre News
Issue 4: June 2014  5
Patient story: Sandra Morgan
“My heart and kidney transplant gave me 21 years of life … and counting”
It was in September 1989 when I was 32 years old that I
first noticed my feet and ankles beginning to swell. I
went to see my GP a few times about this, before he
referred me to the Renal Unit at the Royal Devon and
Exeter Hospital. From here I was referred in April 1990
to the Immunological Medicine Unit at London’s
Hammersmith Hospital, which was then the national
referral centre for amyloidosis. Here I was seen by
Professor Mark Pepys, and Dr Philip Hawkins (now
Professor)
and
underwent
numerous blood tests and scans.
Up until then I had not felt unwell
at all. I just had swelling of the feet
and ankles and was retaining fluid.
After a few more trips and tests at
Hammersmith, plus tests on my
family in Devon, I was diagnosed
with ApoAI amyloidosis, as was my
father, who had had a stroke and
heart defect a few years earlier. I
was told I would need a kidney
transplant at some point in the
future as they had found amyloid in
the kidneys.
In 1991 the fluid problem was
getting worse and I was struggling
to walk, getting very breathless, and had collapsed a few
times. I was admitted to the Hammersmith Hospital for
more tests, where they discovered I had heart failure due
to amyloid. I was told in September 1991 that I now
needed a combined heart and kidney transplant.
Professor Pepys then undertook a campaign to get me on
to the heart transplant list at Harefield Hospital. Initially
they were reluctant to take me on as they had never
tried to transplant an amyloid patient and they did not
know if it would work. Current thinking at the time was
that the amyloid would re-appear in the transplanted
organs immediately. I was 35 with a young 12 year old
daughter, and Professor Pepys fought long and hard for
my case to be brought to the forefront. This was 22
years ago when amyloid was not as well known as it is
today.
In February 1992 I was put on the transplant list for a
combined heart and kidney transplant. In July of 1992 I
was admitted to Hammersmith Hospital with right-sided
heart failure due to excessive water retention. I needed
to be drained through a catheter and had an intravenous
drip of diuretic. I was in hospital for five weeks, and
during that time I was sent to Harefield Hospital again to
be assessed for transplant.
I had had enough of hospitals as I had been away from
home now for over five weeks. Thankfully I was sent
home to await the outcome of their
decision.
While I was at home waiting I went
into the Royal Devon and Exeter
Hospital where I was told I was
suffering from kidney failure. I had
turned yellow and was bloated out
with fluid. They took me in to the
Renal Unit to be fitted for
peritoneal dialysis but the catheter
was put into the wrong place and
did not work. The following day,
while waiting to go to theatre for
the re-fitting of the catheter, I was
told they had found a match for me
in Harefield, and I was rushed by
ambulance
from
Exeter
to
Harefield.
On arrival they took me straight in and got me ready for
the transplants. This was the 14th October 1992. After
the operation I found out from my husband that I had
three professors in the theatre with me: Professor Sir
Magdi Yacoub doing the heart, Professor Peter Morris
from Oxford doing the kidney and Professor Pepys,
watching and taking notes and samples.
The next thing I remember is coming round three days
later in ITU. During this time I was on a cocktail of
painkillers and other drugs that gave me terrible
nightmares and weird dreams. For the next five weeks I
had biopsies through the neck every week for the heart,
plus kidney biopsies.
I had to travel from Exmouth to Harefield every two
weeks for the first 6 months, then once a month for the
next six months. This was a strain on the family as I was
away for two days each time. They took heart biopsies
by entering through my neck.
National Amyloidosis Centre News
6  Issue 4: June 2014
Since then the heart and kidney have all been good.
There have been a few little drug adjustments and the
side effects of the medications have caused me all kinds
of problems from gout to skin cancers, facial bloating
from steroids, facial hair and more, often resulting in
more medication!
The skin cancers are now appearing more frequently and
I have regular visits to the dermatologist for a head to
toe examination. I have had over twenty lesions cut out
and my skin is very thin due to all the medication.
I have been to see the eye specialist as there is
amyloidosis in my eyes.
I have lived a full life: I have been on holidays to Florida
and Los Angeles, I have learnt to drive, I enjoy walking
and have great times with all my grandchildren.
My transplanted kidney has now come to the end of its
life and I am on the transplant list for another kidney.
My sister Janice is a good match and we are hoping to
get the transplant done in the near future.
I have had a fantastic 21 plus years of life. This would
not have been possible without the determination of
Professor Pepys and his team who pushed for me to have
the transplants.
I would like to give Professor Sir Mark Pepys a huge
thank you for giving me my life back.
Just after writing this, Sandra received her second
kidney transplant from her sister, and Sandra, the
new kidney and her sister are all doing well!
Professor Sir Mark Pepys’s perspective
“I phoned the heart transplant team every single week to push for Sandra’s transplant”
I became interested in amyloid and amyloidosis in 1976,
when I was Head of the Immunology Department at the
Royal Free Hospital School of Medicine. When I moved
to the Royal Postgraduate Medical School at
Hammersmith Hospital in 1977 and set up the
Immunological Medicine Unit there, I started to see
patients with amyloidosis. In 1986 I invented the SAP
whole body scan for diagnosing and monitoring
amyloidosis and showed that it worked experimentally.
Philip Hawkins joined me soon afterwards to study for his
PhD degree and for his project I assigned him to establish
the method in human patients. Under my supervision he
first developed it further in experimental models and
then rapidly and successfully transferred the technology
to patients. It is worth noting that it is fortunate that we
did this in 1986-7 because current regulations and
constraints in medical research, intended to prevent
harm to patients, incur such huge development costs
that this harmless and amazingly helpful test would
never have become available for clinical use. In fact we
have safely performed over 29,000 SAP scans, with
immense benefit to many thousands of patients.
During the course of his studies Philip did wonderful
work in developing and perfecting the SAP scan and also
gained great experience in diagnosing and managing
patients with amyloidosis. By the time he got his PhD
degree in 1989 I had no hesitation in appointing him to a
Consultant Physician post and putting him in charge of
looking after all the amyloidosis patients who were then
being referred to me. Having the SAP scan enabled us
for the first time to understand properly the natural
history of amyloidosis and its response to treatment,
uniquely better than anywhere else in the world. As a
result more and more patients were referred and we
became the de facto national referral centre for this
disease. Eventually, when we moved back to the Royal
Free Hospital, by then a part of University College
London, I established the UK NHS National Amyloidosis
Centre in 1999, with Philip as the ideal person to head it.
He has done this with great skill and success, so that he is
now the leading clinical specialist in amyloidosis in the
world and runs the world’s leading centre for
amyloidosis.
Sandra Morgan is one of our most remarkable patients.
We diagnosed her hereditary apolipoprotein AI
amyloidosis on her referral to us in 1990. It was already
quite advanced, despite her young age of only 35, and
over the next couple of years she rapidly deteriorated
into heart and kidney failure. Apart from support for the
function of failing organs, the only treatment for
National Amyloidosis Centre News
amyloidosis, then and now, is to try to reduce the
abundance of the precursor protein which forms the
amyloid fibrils that cause the disease. If this can be
achieved, accumulation of amyloid is halted and organ
function may be stabilised, unless the damage is already
too severe. ApoAI is produced by the liver and the small
intestine and unfortunately no treatment or intervention
is available to reduce it. All that could be done for
Sandra was to replace the functions of her failing organs
and fortunately both kidney and heart transplantation
are possible. Given her young age and the fact that she
had a young daughter I thought that she was an ideal
candidate for transplantation, even though this had
never been done in such patients before. Firstly it would
save her life and secondly, although it was certain that
amyloid would be deposited in the transplanted organs, I
did not believe that there was any reason why this
should not take as long to terminally damage them as it
had from her birth, that is up to 30 years.
Philip and I therefore referred Sandra to Harefield, the
UK’s leading heart transplantation centre and they put
her on the transplant list. Her condition continued to
deteriorate and she was in grave kidney and heart
failure, an extremely difficult and challenging situation to
manage. However I was determined that she should get
the chance that the transplants would provide and from
April 1992 I phoned the heart transplant team, either
Sir Magdi Yacoub or his colleagues, every single week,
telling them of her desperate situation and the urgent
need for transplantation, until finally we heard on
14 October 1992 that suitable donor organs had finally
become available.
While they rushed Sandra to the hospital,
Professor Sir Magdi Yacoub, one of the world’s leading
cardiac surgeons, and Professor Sir Peter Morris, the
Regius Professor of Surgery at Oxford University, arrived
to perform the operations. Two surgeons were needed,
one for the heart and the other for the kidney. I arrived
at Harefield at 11.30 pm to watch the operation and to
receive Sandra’s old amyloid laden heart when it was
removed to be replaced by the new healthy donor organ.
They started with the heart transplant and Sir Magdi
completed this uneventfully in 45 minutes, from start to
finish. Amazing! Sir Peter then went to work on the
kidney transplant which, in theory, is a very simple
operation. However patients with systemic amyloidosis
usually have amyloid in the walls of their blood vessels so
that there is often much bleeding when they undergo
operations, and this can be very difficult to control.
Issue 4: June 2014  7
Indeed this was the case in Sandra and the kidney
transplant was only completed at about 4 am the next
morning. Quite an ordeal for Sir Peter and the whole
team but fortunately no problem for Sandra! Sir Peter
and I still remember it vividly whenever we meet but
Sandra immediately had adequate kidney function and
continued to have it until recently, 21 years later.
As Sandra tells in her story, she has lived a full and
rewarding life with reasonable quality, although
complicated by the many unpleasant side effects of the
drugs required to protect the transplants from rejection.
She has just received a second kidney transplant from
her sister and should continue to be well for many years
to come.
From the medical side, we have learned a lot from
Sandra, both from her amyloid laden heart which was
removed, and from her long and complicated clinical
course. This knowledge has greatly benefited other
patients. She was and still is a model patient and it has
been a great pleasure to know her, and a privilege for
Philip and me to have been able to look after her
successfully.
Despite this success story, amyloidosis of all types
remains a serious unmet medical need. We badly need
treatments that can eliminate the damaging amyloid
deposits from the tissues and organs, so that organ
failure requiring transplantation is no longer required. I
have been working towards this since 1984 and finally, in
2005, invented a new approach which works very well in
experimental models, eliminating massive amyloid loads
from vital organs without having any adverse effects.
GlaxoSmithKline, one of the world’s largest leading
international pharmaceutical companies, licensed this
invention in February 2009 and we have been working
very closely with them to develop it for clinical testing
since then. The first clinical trial, to show safety and
tolerability of the treatment in amyloidosis patients, is
currently in progress and the results so far, including in a
patient with apoAI amyloidosis, are very encouraging.
The process of drug development is extremely complex,
extremely slow and extremely expensive, so it will be a
while before we know for sure that our approach will be
effective and safe, and can then become available for
routine treatment. However we hope that in future, the
combination of earlier diagnosis of amyloidosis, before
there is irreversible organ damage, and availability of a
treatment to eliminate existing amyloid deposits, may
greatly improve the outlook for this serious disease.
National Amyloidosis Centre News
8  Issue 4: June 2014
Fundraising News
The Big McCycle 2014 — My Comeback Tour
A 500 mile patient journey from Dunfermline to London to raise awareness and funds for the
UCL Amyloidosis Research Fund
By Mark McConway
Two conversations in
March 2011, separated
by a fortnight, changed
my life!
Returning on the train to Scotland, I considered what I
had been told about treatment and, for the first time
since becoming ill, some of the clouds began to lift.
Whilst there were no guarantees on offer, there was
sufficient reason to be optimistic about the future. Yes,
chemotherapy might be unpleasant but I pledged myself
to ‘co-operating’ with the treatment fully and without
complaint.
I also pledged myself to getting well enough to ride my
bicycle back to London for my appointment at the NAC
two years hence.
Mid-March 2011,
Dunfermline
at
Queen
Margaret
Hospital,
“The kidney biopsy has tested positive for amyloidosis.
It’s a very rare and serious blood disease. In fact, it’s
potentially life shortening. I’m sorry to tell you that it’s
incurable at the moment.”
“What do you mean by life-shortening?”
“Without treatment, the average life expectancy is
around 15 months. We're referring you to the National
Amyloidosis Centre in London.”
End of March 2011 at the NAC, Royal Free Hospital,
Hampstead, London
“There is a heavy load of amyloid present in your liver,
kidneys, heart and spleen. It's systemic AL amyloidosis which means that it's being produced in your bone
marrow. Whilst we can't offer you a cure, we are
confident that we can halt the progress of the disease
and treat the organs that have been affected. To get you
back to ‘near normal’ will take a couple of years but
you'll need to start chemotherapy straight away.”
By the time that second conversation took place, my
body was filled with fluid, my limbs and stomach were
swollen, my cholesterol had gone through the roof at 25;
I was being violently sick regularly and I could walk less
than 50 feet without assistance or being pushed in a
wheelchair.
Given that my muscles had weakened to the extent that I
couldn’t lift a few bath-towels without becoming
exhausted – and I needed to sleep for several hours
every day - the notion that I would be able to ride 500
miles in 24 months time seemed ridiculous to all but a
few people who knew me.
My fifth cycle of chemotherapy finished in July 2011. In
October of that year, I did my first bike ride of 5 miles.
Inevitably, it was exhausting – but fun. As the weeks
went on, I increased the mileages and, within another
few months, I could cycle 30-50 miles on a mountain bike
in winter conditions, without distress. I decided that,
with training, I could make the ride to the NAC in London
in May 2013.
At this stage, I floated the idea past my friend and cycling
buddy, Tom McLucas, who had just recently retired from
the fire service. He agreed to do it with me and we
started training properly.
Fate decreed that May 2013 would not be my time for
doing the ride as I got a virus in December 2012 which
laid me low until the start of April 2013. There was no
time left to restart the interrupted training. Instead, I
visited the NAC by train, as normal, in May and was told
that there had only been a very small decrease in the
amount of amyloid in my affected organs.
Notwithstanding this, I felt hugely improved and was
beginning to regain some much-needed muscle.
National Amyloidosis Centre News
Issue 4: June 2014  9
In order to keep the project alive for another twelve
months, we resolved to continue the training, extending
our 3-4 hour rides to 5-6 hours and spending the winter
nights on turbo trainers in front of a projector screen
with ‘Sufferfest’ training videos.
Fast forward to February 2014. By
this time, we had a name for our
project - “The Big McCycle” - and had
enlisted the support of my brother,
Matt, who would become our
logistics support with his van and
‘navigator of last resort’ with his
collection of OS Maps. Early April
saw our JustGiving site go live and we
were in the final stages of
preparation, getting bikes serviced,
cycling tops printed and longer days
in the saddle.
Our Route
We set out from Cairneyhill in Fife on April 29th and
followed a scenic route down the East Coast of Scotland
and North East England as far as South Shields, before
heading inland towards York, then dropping South to
Lincoln, Peterborough and ultimately, London.
On arriving at the Royal Free Hospital, we were met by
Professor Philip Hawkins. Along with his team at the NAC
– and with Professor Sir Mark Pepys at the Wolfson Drug
Discovery Unit – Professor Hawkins is at the forefront of
the pioneering research to find a cure for amyloidosis.
In summary, we cycled 507 miles (if you include the
wrong turns, diversions, etc) in 6 days and 2 hours. The
weather was mixed with us being shrouded in fog and
battered by wind and rain on two days. We averaged 80
miles a day and had three long days of 11 hours each,
where the weather, terrain and traffic made progress
slow.
Wherever we went, our printed cycling tops got people
talking about amyloidosis and a few gave us ‘on the spot’
donations. We’ve raised £2,000 so far for the UCL
Amyloidosis Research Fund, between on and off-line
donations (www.justgiving.com/Mark-McConway) and
the webpage remains open until the end of July 2014.
Bespoke poems in aid of amyloidosis
By Chloe Bullock
I write bespoke poems for any occasion: birthdays, anniversaries, births, christenings, marriages, Mother's/Father's/Valentine's
Day, farewells, memorial services, or anything else you like! I can also provide poems for use by organisations and causes for
marketing purposes. I write the poems in exchange for a donation to the UCL Amyloidosis Research Fund.
My mother suffers from amyloidosis, and I wanted to use my creativity in order to raise money for this charity on an on-going
basis. My poems have always proved popular with friends and family, so I decided to widen my audience and raise some
much-needed funds for this important cause at the same time. My poems are likely to bring a tear to your eye but will
hopefully raise a smile too!
How does it work?
Contact me by email: hellobespokepoems@gmail.com then I will organise to get back in touch with you by Skype, email or
phone to find out the exact nature and purpose of the poem you are looking for. I will ask for specific details about the
person/organisation/occasion for which it is required in order to tailor it perfectly to your needs. Once I have enough
information I will put pen to paper, then email/call you with the draft poem once it is ready. We can then tweak it together,
according to your personal requirements. When you are happy with the finished article I ask that you make a donation via our
page on justgiving.com of minimum £15 per poem. Once you have donated I will send you your poem by email.
If you have a Facebook account type: "Bespoke poems in aid of amyloidosis" into the search bar. When you reach the page,
click on "about" and you will find samples of my poems and reviews there.
National Amyloidosis Centre News
10  Issue 4: June 2014
Channel distance swim for amyloidosis in Horsham pool
By Pat Pinchin
Those who know Martin Bolton-Smith well might have known a knee injury
sustained during training for the Brighton Marathon in 2013 would not stop
him from taking on a very different fundraising challenge for the UCL
Amyloidosis Research Fund. Martin’s recent new challenge was closer to
home in Horsham swimming pool. Martin, no stranger to starting intensive
preparation for his sporting challenges from scratch, believes this was the
toughest one yet.
With his characteristic perseverance and determination, and with the help
and advice of Alastair, his swimming instructor at Horsham Pool, Martin
spent an arduous 6 months learning how to swim front crawl. This also
involved learning correct breathing, stroke and turning techniques needed
to swim the 22 miles distance of the English Channel (Dover to Calais) in
9 weeks, starting on December 2nd and finishing on January 30th. Martin
had to build up his endurance and confidence too in order to complete the
120 x 25 metre lengths each week, 1408 in total.
Just days into the challenge, Martin was already finding that the swim was the hardest challenge he had ever done both
mentally and physically. There was nothing in the swimming pool to take his mind off how much it was hurting. Water, chlorine
and walls of the pool are not in the least inspiring as compared with interesting countryside and cheery greetings from
passers-by when out running. The words of a friend “EVERY STROKE IS A STROKE NEARER TO A CURE FOR AMYLOIDOSIS”
helped keep Martin focused on the 40-45 minutes it took to cover the 64 lengths of each mile.
In mid-January, with “the coast of France in sight” and just 12 miles left to go, Martin admitted that it took this venture to
enable him to understand just how challenging long distance swimming is, but that this particular venture would be memorable
for years to come. To his great relief, the countdown had begun and by now Martin was ticking off the days. On January 30th,
24 days ahead of his original 12 week schedule, Martin had completed his 22 miles, feeling a sense of deep satisfaction that this
particular fundraising journey had been successful. Most importantly, he had also raised £804.00 for the UCL Amyloidosis
Research Fund.
Amyloidosis MRI Cycling Challenge:
Land’s End to John O’Groats
The Land’s End to John O’Groats (or End to End) is one of the classic long
distance cycle challenges. It is called LEJOG if ridden north, and JOGLE if
ridden south. There are many routes and it has been cycled, walked,
hopped, roller-skated and even driven on lawn mowers!
On the 6 July 2014 a team comprising staff and patients from the NAC,
led by John Plant (patient), Thirusha Lane (Lead Nurse) and David Hutt
(Lead Nuclear Medicine Technician), will be cycling from Land’s End to
John O’Groats to help raise funds towards the purchase of an MRI
scanner. This epic challenge will cover 977 miles, climbing 50,300 feet
(almost twice the height of Mount Everest), arriving in John O’Groats 14
days later on the 19 July.
National Amyloidosis Centre News
Issue 4: June 2014  11
We have made great progress in developing new MRI techniques to specifically diagnose and quantify amyloid deposits
non-invasively, crucially including amyloid within the heart, which has not been possible previously. This research is already
facilitating the development of new treatments, and avoiding the need for potentially dangerous biopsies in many patients.
Currently our patients have to travel to another London hospital to undergo MRI. We wish to create our own MRI facility in
order to greatly reduce the travel burden and the waiting time involved, and to enable us to scan many more patients.
How can you help?
1.
Would you like to join the team for part of the ride? If so, contact John Plant (john.h.plant@gmail.com). There are
still places available for people to join for a day or two. You don’t need a carbon fibre racing bike to join part of the ride
- many have ridden successfully and comfortably on light hybrid bicycles.
2.
Would you like to meet the team as it comes through major towns? This is where we really need the help of patients
and relatives. Are you able to contact local media (newspapers, radio, and television) to raise interest? We would be
happy to provide information or be interviewed. This will also give patients and relatives an opportunity to meet one
another. If you are able to meet the team as they ride through a town near you, and if you would be happy to share
your story, please get in touch. A few small media events have been set up but we still need more coverage in the
bigger cities listed below. The more media coverage we can get the more people will become aware of amyloidosis and
the work of the NAC.
3.
Would you like to help by fundraising locally? You could organise a local event or appeal to local businesses. Some of
our collaborators in the pharmaceutical and related industries (Isis Pharmaceuticals, Alnylam Pharmaceuticals and The
Binding Site) have agreed to sponsor us and we will be happy to wear sponsor logos on our jerseys, in order to
advertise.
5.
Please sponsor us at our JustGiving page at http://www.justgiving.com/nac-mri-cycling-challenge. You can also text
your donation by texting TDJP99, followed by the amount you wish to give (e.g. £10), to 70070. If you would prefer to
send a cheque please make it payable to “UCL Amyloidosis Research Fund” and send it to Beth Jones at the address on
the following page.
Find out more about the Amyloidosis MRI Cycling Challenge and follow our blog at http://amyloidosismri.blogspot.co.uk/.
We will be using a programme called Buddy Beacon which allows people to follow the ride. It uses GPS and updates every
20 minutes. There will be a link to this on the blog page. We will blog and upload pictures along the way, where possible.
Interested people could follow the blog and send messages (which would be nice for the team!). We have also set up a
twitter account (Thirusha Lane @lejog4amyloid) which we will attempt to update daily.
The route in brief
Day
1
2
3
4
5
6
7
8
9
10
11
12
13
14
/
Date
Start
6 July
7 July
8 July
9 July
10 July
11 July
12 July
13 July
14 July
15 July
16 July
17 July
18 July
19 July
Land’s End
Fowey
Moretonhampstead
Glastonbury
Monmouth
Clun
Runcorn
Conder Green
Keswick
Moffat
Balloch
Glencoe
Inverness
Lairg / The Crask
Passes
Penzance
Ends
St Austell
Plymouth
Exeter
Taunton
Wells / Chepstow
Bristol / Tintern
Hereford
Shrewsbury
Chester
Warrington
Bolton
Lancaster / Ambleside
Windermere
Carlisle
Gretna
Glasgow
Crianlarich
Fort William
Spean Bridge
Bonar Bridges
Altnaharra
Thurso
Fowey
Moretonhampstead
Glastonbury
Monmouth
Clun
Runcorn
Conder Green
Keswick
Moffat
Balloch
Glencoe
Inverness
Lairg / The Crask
John O’Groats
National Amyloidosis Centre News
12  Issue 4: June 2014
Donations
To ensure that your donations go directly and exclusively to the NAC,
please send directly to us or contact Beth Jones on 020 7433 2802 or beth.jones@ucl.ac.uk.
All university based medical research depends on funds that are
fought for in open competition (grants) from the
Government-funded Medical Research Council and other
charitable bodies. Their renewal depends on a successful
research programme and the NAC has an excellent track record
in this respect. However, there are constant shortfalls and every
penny from other sources is received with sincere gratitude, and
is used specifically and in its entirety for amyloidosis research.
You can make an online donation at:
http://www.ucl.ac.uk/amyloidosis/support-us
or make a donation by post. A gift aid form is included here and
is also available online or from:
Beth Jones
National Amyloidosis Centre
Division of Medicine, Royal Free Campus
University College London
Rowland Hill Street
London NW3 2PF UK
UCL Medical School is part of University College London (UCL)
which is a registered charity. Due to changes in the Budget
nearly all gifts given to UCL now qualify for the Gift Aid
Scheme. This increases the amount of the gift by 25% without
any extra cost to the donor. It does so by allowing UCL to
claim back the basic rate tax paid by the donor. To qualify for
Gift Aid the donor must be a UK taxpayer and his/her
combined income and capital gains tax bill must equal or
exceed the amount UCL claims back on the gift. The gift can
be for any amount and applies to one-off gifts and regular gifts
made over a number of years. For UCL to claim the tax
benefits the donor must make a "Gift Aid Declaration". One
declaration will cover all future gifts and may be cancelled at
any time. Cheques should be drawn in favour of "UCL
Development Fund". This money is then transferred to the
Amyloidosis Research Fund together with the reclaimed tax. If
a donor does not qualify for the Gift Aid Scheme or does not
wish to contribute in this way, cheques should be made
payable to: "UCL Amyloidosis Research Fund".
 Newsletter funded by a bequest from Laura Lock 