News and Stories - Summer 2018 Amyloidosis Brought My Family Together - Amyloidosis Foundation
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News and Stories - Summer 2018
Amyloidosis Brought My Family Together
My name is Jason Conway walking up the
and I am a husband, father, bleachers. My
teacher, and coach. I am daughter didn’t
48 years old and have lived ignore the symptoms
a pretty normal, healthy life. and made sure to tell
I have always been active my wife when we got
and have taken care of my- home.
self. I am also an amyloidosis
patient. In hindsight, I’m glad
she did because the
next day I was admit-
My life changed drastically in
ted into the hospital myopathy is hereditary and
January of 2015. I was with
with cardiac related con- there is no one in my family
my daughter at my son’s
cerns. The doctors diag- who suffers from it. Libbi
basketball game and I
nosed me with cardio- pushed to have a cardiac
started having cardiac prob-
myopathy. My wife, Libbi,
lems. Being a guy, I just MRI done.
disagreed with this diagno-
wanted to ignore the racing (Continued on page 7 )
sis because cardio-
heart and the difficulty of
FDA Approves Treatment for Hereditary (hATTR) Amyloidosis
Alnylam Pharmaceuticals, other treatments become represents one of most
Inc. received approval for available soon. European rapidly advancing aspects
ONPATTROTM (patisiran) from regulators have also rec- in today’s biology and
the U.S. FDA in August for the ommended approval, with pharmaceutical develop-
first-ever treatment for pa- a final decision coming ment. By silencing the mes-
tients suffering from heredi- shortly. senger (RNA), this treat-
tary (hATTR) amyloidosis. ment will prevent the
This is exciting news for the ONPATTRO was shown to problematic protein from
TM
entire amyloidosis commu- improve polyneuropathy being made.
nity, as other companies are (numbness, pain, weakness
getting closer to having in hands and feet) in For more details on this
patients and allow them to product and to see if you
sustain activities of daily qualify for treatment,
living. This drug is based on please visit their website:
a natural cellular process of www.alnylamassist.com.
gene silencing that AF
Education • Awareness • Support • Research
www.amyloidosis.org
2
News from the Amyloidosis Foundation
Website Update Fall Webinar
Have you visited the AMYLOIDOSIS 2030:
“Newsroom” page on our A SyFy Special
website? Here you will find
the latest updates on re- October 15, 11am (EST)
search and industry news in
A look 12 years into the
future at how the risks of AL
and ATTR will be managed
and the diseases treated.
Anniversary Open House
Hosted by Raymond
September 20, 2018
Comenzo, MD, Professor of 3:30pm—7pm
Medicine
and Pa-
Fountains Golf & Banquet
the amyloidosis community. thology
Clarkston, MI
We’ve added links to this at Tufts
page on our social media University
Join us to celebrate this
channels to keep everyone School of
special occasion. Our AF
informed about any future Medicine.
board members will attend,
treatments, clinical trials,
plus patients, donors, volun-
etc. Please follow us on so-
Please share with your fam- teers, friends, family and
cial media (details below)
ily & friends. Register today members of our local com-
and bookmark our website
online: www.amyloidosis.org munity. See you there! AF
on your laptop or tablet.
Patient Resources
The foundation has several programs that Our comprehensive website has
benefit patients and their families. All of information for patients, caregivers
these are provided free of charge. and physicians featuring:
Webinar recordings posted on our website Treatment Centers (US / International)
Updated informational pamphlets Support Groups
Toll Free Number 1-877-AMYLOID Newsletters
Listing of experienced physicians that Webinars
specialize in amyloidosis. Email us anytime
with questions: info@amyloidosis.org Fundraising Toolkits
Follow Us Stay connected for all the latest
information on Amyloidosis:
Web: www.amyloidosis.org
Twitter: @Amyloidosisfdn
Facebook: @amyloidosisfdn
www.amyloidosis.org
3
President’s Corner Board of Directors
We are so thrilled with the news that the FDA has approved President
a drug for hATTR patients. Years of research and develop- Mary E. O'Donnell
ment, including many patients in clinical trials, have lead to
this opportunity. Hopefully more new treatments will follow Treasurer
Dante Burchi
soon, we know you share in our excitement.
Charlotte Haffner
Make sure to register for our next webinar on October 15,
AMYLOIDOSIS 2030: A SyFy Special, hosted by Dr. Comenzo. Silva Pregja
You won’t want to miss this topic, plus we have plenty of Mark Sutherland
time scheduled for your questions.
Fall is coming soon which means it’s time for the 2nd annual
AF Run/Walk in Michigan on October 13th and the 3rd an-
nual Pittsburgh Amyloidosis Research Benefit on October 26.
You can find all the details and links to register online on our
website, www.amyloidosis.org. Thanks as always for your
support!
Sincerely,
Mary E. O’Donnell
Scientific Advisors
Save the date! Merrill Benson, MD
Raymond Comenzo, MD
3rd Annual Pittsburgh Amyloidosis Research Benefit
Lawreen Connors, MD
Friday, October 26, 2018 Rodney H. Falk, MD
Join us and enjoy a gourmet dinner, live music, wine Morie Gertz, MD
raffle, silent auction, and more—all in the name of
Mathew Maurer, MD
raising funds for amyloidosis research.
Giampaolo Merlini, MD
Montour Heights Country Club
Vaishali Sanchorawala, MD
Tickets $175 Douglas Sawyer, MD, PhD
Buy your tickets today: www.amyloidosis.org Jonathan Wall, PhD
Our newsletter is published quarterly (Spring, Summer, Fall and Winter) by the
Amyloidosis Foundation. We welcome letters, articles and suggestions.
Please contact us anytime at: info@amyloidosis.org, 1-877-AMYLOID (877-269-5643)
or 7151 N. Main Street, Ste. 2, Clarkston, MI 48346
If you wish to receive a printed newsletter, please send us an email:
info@amyloidosis.org
www.amyloidosis.org
4
Four Infrequently Asked Questions, by Raymond Comenzo, MD
1. Who is this man?
This is Don Brockman, the patient with AL amyloidosis
whose vision for finding a cure for amyloidosis led him
and his wife, Mary O’Donnell, to form the Amyloidosis
Research Foundation in 2003.
2. What happened to Mr. Brockman?
Mr. Brockman sought treatment for AL amyloidosis at all
the major centers and with all of the key AL investiga-
tors of that day. He bravely enrolled in a clinical trial for
a new drug and died while on the trial. Patients who
Don Brockman, co-founder of the enroll in clinical trials are brave by definition and give
Amyloidosis Foundation substantial meaning to their lives and deaths.
3. What happened to the Amyloidosis Research Foundation?
Under the leadership of Don’s widow, Mary, the ARF became the Amyloidosis Founda-
tion. Now, 15 years later, the AF has an unparalleled record of achievement—they
have distributed over $1.7 million in grants for global research into all forms of
amyloidosis.
4. How close are we to achieving Don’s vision for a cure?
We are closer to achieving a cure for AL amyloidosis than ever before, and many
pharmaceutical companies are developing novel agents that will move us even
closer. Many—but not all—AL patients are living for 10 or more years after being diag-
nosed and treated. The other major type of systemic amyloidosis, ATTR, is the current
focus of scientific research. As new drugs come into clinical development, the pace of
their success depends on the bravery of patients and caregivers willing to participate
in clinical trials as Don Brockman did. The AF has been the major driving force over the
past 15 years in pursuit of cures for both AL and ATTR.
Thank you, Don Brockman
Please show your
support for the AF
by donating $15 for
15 years—to raise
funds for research,
patient education
and help to spread
awareness for this
rare disease.
www.amyloidosis.org
5
The Amyloidosis Foundation
is proud to announce that
we will be participating in
the National Organization
for Rare Disorders, Inc.
(NORD) Rare Summit in
October in Washington,
D.C.
During the 2018 NORD Rare
Summit, over 700 leaders
from FDA, NIH, industry, pa-
tient groups, payers and
attendees will explore the
research institutions will ad- disease community. We
new and innovative ways
dress the New Era of Patient- look forward to making
in which patients and care-
Focused Innovation. To- new connections and
givers are helping drive
gether, NORD Rare Summit learning from this event. AF
progress for the rare
Fundraising on Facebook is Easy, Give it a Try!
Have you ever donated Since 2017, the Amyloidosis each fundraiser averaging
online to a fundraiser? For a Foundation has had great just over $400! Crowd fund-
cause or group that you feel online support from friends ing has grown dramatically
strongly about supporting? and family members — over the years, and we are
Maybe a friend or family starting fundraisers in honor thankful for our generous
member has sent you a do- or memory of a loved one donors.
nation link, to share details for
or their own birthday or
an event or asked you to Next time you are on FB, use
anniversary. So far we have
help? Well, here is your the details below to start
raised over $63,000—with
chance to make a difference. your own fundraiser! AF
https://www.facebook.com/fundraisers/
www.amyloidosis.org
6
Daughter Raises Donations for Amyloidosis Research
In 2015, Addi Lacy had just with the help of her mom’s
started the third grade. She Facebook page.
decided to start a bracelet
fundraiser to support the
Amyloidosis Foundation as
part of a school project. Her
father, Josh, was celebrating
his second "birthday", receiv-
ing a stem cell transplant in
2013. She wanted to honor
him and start a fundraiser as
a birthday gift for him on this
special day.
Addi is now 11 years old and
in the sixth grade. She has
sold her bracelets at church,
on a family cruise and even
school sports games, to
swam with the dolphins!
family members and online
Their son, Chet, just turned
seven and they all give
thanks everyday for Josh’s
Addi and her
health.
family recently
moved from What a special girl Addi is,
Texas to Wis- with such a big heart. We
consin to be are in awe of her spirit, kind-
closer to fam- ness and creativity. Thank
ily. They cele- you for your support and the
brated Josh’s donations you continue to
fifth birthday send the AF. AF
Don’t Miss Out - Make Plans to Join Us in October!
We cordially invite you to followed by the 5k at 9:45 We still have sponsorships
participate in our 2nd Annual a.m. Awards will be given available! All the details are
Amyloidosis Foundation for top qualifiers as well as online at our website,
Zombie Run for Your Life! best costume. www.amyloidosis.org. AF
5k/10k Walk/Run. This event This year is special
will raise money for our re- as we celebrate our
search grant program. The 15th anniversary!
event will be held Saturday,
October 13, 2018 at Inde- Registration includes:
pendence Oaks County Run for Your Life! Long
Park in Clarkston, MI. The sleeve tech shirt, medal,
10k will kick off at 9:30 a.m., prizes plus food & drink.
www.amyloidosis.org
7
Amyloidosis Brought My Family Together
(Continued from page 1)
amyloidosis has given to I am of the opinion that my
I think the doctors agreed just me. Amyloidosis has story is rather boring and
to appease Libbi. That brought my family closer insignificant. I am simply a
cardiac MRI was crucial to together. guy who had the unfortu-
my amyloidosis diagnosis. nate luck of getting amy-
It has also given me reason loidosis. That’s really how I
Amyloidosis quite often gets to tell my children, Kelsey, feel. However, out there in
misdiagnosed. However, that Ethan, and Seth that I love my world of supporters, I
was not the case for me. Dr. them more frequently. The am sure there are many
Wheat, a cardiologist in Val- girls that I coached experi- differing views. I suppose
paraiso, Indiana read my MRI enced my amyloidosis jour- one thing that we can all
and diagnosed me with amy- ney with me. They were agree upon is that bad
loidosis. I can still vividly re- given the opportunity to things do happen and with
member the day that this oc- see that even though horri- the help of others we are
curred. Libbi and I sat in Dr. ble things can happen, a
able to carry what we
Wheat’s office. I knew some- strong person with a great have been given. AF
thing was really wrong with support group can perse-
my heart, but I figured my di- vere. This is an incredibly Libbi Conway wanted to
agnosis would include a valuable life lesson. give back and support
change in lifestyle and medi- amyloidosis patients and
cine. I was not prepared for families, knowing first-hand
the onslaught of new words what it feels like. She
and phrases that included decided it was time to
amyloidosis and oncologist. plan something fun – to
help others facing this rare
Since my diagnosis I have disease. “Cocktails 4 a
received 16 weeks of chemo- Cause” was born!
therapy and a stem cell trans-
plant at Northwestern Memo- The event in July included
rial Hospital. This was in con- a great night of cocktails,
junction with multiple medi- live music and amazing
cations and very long hospital food – celebrating
stays. Currently, I am in Although I don’t open up with friends and family. The
hematologic remission and too much to my students goal was to raise funds for
receive maintenance che- about my condition, I have the Amyloidosis Founda-
motherapy once a month to had students confide in tion and Northwest Memo-
maintain remission. me that a parent is going rial Hospital, where Jason
through or is getting ready was treated. We are so
I would not wish amyloidosis to go through chemother- grateful for people like the
on anyone. Amyloidosis is the apy. These students are Conway’s, who want to
hand that I have been dealt. scared and unnerved. give back
I cannot change this, I can and support
only carry it. The general However, when I tell them others going
consensus is that amyloidosis that I am receiving che- through this
only takes. However, I be- motherapy, it brings a same jour-
lieve that in some ways, sense of calm to them. ney. AF
www.amyloidosis.org
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