News and Stories - Spring 2019

[Pages:8]News and Stories - Spring 2019

FDA approves Vyndaqel? and VyndamaxTM

On May 6, 2019, the U.S. Food and Drug Administration (FDA) approved both VYNDAQEL? (tafamidis meglumine) and VYNDAMAXTM (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-CM)

in adults to reduce called amyloid in the heart

cardiovascular

and is defined by restrictive

mortality and car- cardiomyopathy and

diovascular-related progressive heart failure.

hospitalization.

Previously, there were no

VYNDAQEL and medicines approved to

VYNDAMAX are treat ATTR-CM; the only

two oral formula- available options included

tions of the first-in- symptom management,

class transthyretin and, in rare cases, heart (or

stabilizer tafamidis, heart and liver) transplant.

and the first and only

It is estimated that the

medicines approved by prevalence of ATTR-CM is

the FDA to treat ATTR-CM. approximately 100,000

people in the U.S and only

Transthyretin amyloid

one to two percent of

cardiomyopathy is a rare, those patients are

life-threatening disease

diagnosed today.

characterized by the buildup of abnormal de- For more information, visit: posits of misfolded protein AF

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Education ? Awareness ? Support ? Research



2

AF Board Member integral in Tennessee legislation

In 2008, Charlotte was a

"I asked Dr. Fredi if it was

healthy, active 55-year-old fatal, and he held back for

who loved the outdoors, a minute, and he said, `I'm

especially taking care of afraid it could be,'"

her horses. Then, one day Charlotte remembers. "I

she started feeling different: said to him, `Well, it won't

fatigued, retaining fluid,

be this time.'"

shortness of breath.

Eleven years later, Char-

The following months

lotte has undergone heart

dragged on. Routine testing transplant and peripheral

offered no explanation for blood stem cell transplant.

her heart failure. Her

She helped found VAMP

condition deteriorated. "I (Vanderbilt Amyloidosis

sat on the hillside behind Multidisciplinary Program);

my house and prayed. I

joined the Board of

was dying, and I knew it," the Amyloidosis Founda- She also convinced

Charlotte says.

tion (AF), where she has "a Tennessee State

platform to help other

Representative Glen

Then she was referred to patients"; and became the Casada to sponsor a

cardiologist Joseph Fredi, first patient enrolled in a

resolution that led to

MD. He had an idea what first-in-human, phase 1

Tennessee becoming the

was wrong. Tests confirmed clinical trial testing a new first state to declare March

it. Charlotte has primary AL amyloid-binding peptide for Amyloidosis Awareness

amyloidosis.

detecting amyloid.

Month. AF

Patient Resources

Our comprehensive website has

The foundation has several programs that benefit patients and their families. All of

information for patients, caregivers and physicians featuring:

these are provided free of charge.

Treatment Centers (US / International)

Webinar recordings posted on our website Support Groups

Updated informational pamphlets

Newsletters

Toll Free Number 1-877-AMYLOID

Webinars

Listing of experienced physicians that specialize in amyloidosis. Email us anytime with questions: info@

Caregiver/Patient Binder Fundraising Toolkits

Follow Us

Stay connected for all the latest

information on Amyloidosis:

Web:



Twitter: @Amyloidosisfdn

Facebook: @amyloidosisfdn

Instagram: @amyloidosisfoundation



President's Corner

Mary E. O'Donnell

I have personally been a part of the amyloid world for over 17 years. Although we still need great strides and advances, the picture is completely different than in 2002. In the past year, 4 drugs have been approved for hATTR; 2 for hATTR polyneuropathy and 2 for hATTR cardiomyopathy and ATTR wild type. Trials are in progress for AL amyloidosis that show promise, and hopefully will progress to FDA approval.

Early diagnosis is still critical, so we continue to work at raising awareness of all types of systemic amyloidosis in the medical community. Your generosity not only supports innovative research but helps our awareness activities.

3

Board of Directors

President Mary E. O'Donnell

Treasurer Dante Burchi Charlotte Haffner Daniel J. Lenihan, MD, FACC Adrienne Molteni, RN Mark Sutherland

March was Amyloidosis Awareness Month.

We began in 2018/2019

by passing state resolutions

to recognize Amyloidosis

Awareness Month in

Tennessee, Michigan,

Indiana, Florida and

Louisiana. We have

advocates working on

similar legislation in the following states for 2019: AK, CA,

GA, KY, NE, NY, PA, UT, VA and WI. If you're interested in

helping to pass this legislation in your state, please

contact kathi@

AF

Scientific Advisors

Merrill Benson, MD Raymond Comenzo, MD Lawreen Connors, MD

Rodney H. Falk, MD Morie Gertz, MD

Mathew Maurer, MD Giampaolo Merlini, MD Vaishali Sanchorawala, MD Douglas Sawyer, MD, PhD

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@, 1-877-AMYLOID (877-269-5643) or 7151 N. Main Street, Ste. 2, Clarkston, MI 48346

If you wish to receive an electronic version, please send us an email:

info@



4

Rare Disease Week on Capitol Hill

In February, one of our staff The film, `My Turn', was

stories to navigating

members traveled to

about a former professional insurance.

Washington DC for Rare Disease Week on Capitol Hill. She participated in many activities and events advocating and giving a voice to amyloidosis

hockey player, and his firsthand perspective on how his life has changed since his rare disease diagnosis. His wife was on a panel for a discussion after the film

Monday was a Legislative Conference. It was a full day packed with many speakers and breakout sessions. Some things

patients, caregivers, family because her

and friends. As you know, husband lost

Washington DC is often

his battle 2

gridlocked with partisan months prior.

bickering. However,

There were

amyloidosis isn't a partisan many

issue, it doesn't discriminate. questions

It affects both Republicans from viewers

and Democrats alike.

regarding

The festivities began on Sunday evening with a documentary screening.

everything from those diagnosed telling their

(Continued on page 5)

*NEW* Caregiver Binder and Resources!

Suggestions From Other

The AF is pleased to announce that we have a new resource for caregivers and patients.

Medical

and sections to fit your Lab Results

`Medications' section for information you receive

The binder was designed

Questions

by the Amyloidosis

the calendar to keep track

Foundation staff, a team

from the of appointments and when

who strive to make care-

doctor

to order medications and

giving easier for caregiv-

ers and patients. Each section has links to PDF files

cards and handouts.

plastic business card sleeve to hold business cards. You

that are designed so that Take It, Share It, Use It

may also write notes on the

you can print your own

copy and keep in a 3 ring binder to take with you to doctor's appointments.

Download yours today! If it with other caregivers and you don't have internet

access, we can even mail

Here's how it works: Keep make caregiving easier you a copy! Contact us at

documents in one location and more effective

info@ AF



5

Rare Disease Week on Capitol Hill (Continued from page 4)

covered were the Health

and FDA for the

Policy Forecast, Healthcare's annual discretion-

challenging issues, how the ary funding in the

new Congress will impact health policy in 2019, how to advocate effectively, and building effective partnerships. One breakout session was a deep dive into policy of NIH and FDA Appropria-

Fiscal Year 2020. Many personal stories were shared, tears were shed, and our impact on Capitol Hill was felt. Tuesday

tions. An investment is needed in rare disease biomedical research

evening ended with a meeting on `Rare Diseases: Perspectives on

event to view beautiful pieces while supporting

conducted at NIH and FDA, Progress'. This meeting

a cause we all care so

which ultimately leads to

focused on advancements deeply about.

treatments and facilitates patient access to treatments.

Another session was `Access

in rare disease and the current development

Thursday, February 28 was

landscape. The program Rare Disease Day.

provided discussions about Naturally, it was also `Rare

the challenges and oppor- Disease Day at the Nation-

to Treatment of Rare

tunities in rare disease

al Institute of Health' (NIH).

Diseases - A spotlight on Rare research and the needs of It featured interactive

Cancers'. This was an

the patient community. panel discussions on

excellent session as Multiple Myeloma was discussed. Treatments for MM have increased significantly, yet it still isn't curable.

collective research

Wednesday was a Rare Disease Congressional Caucus Briefing where

models for rare diseases, patient registries, and rare

many legislators and direc- cancer initiatives. A

tors from NIH and FDA

common theme was that

On Tuesday, she proceeded to meetings with Michigan legislators. While there, Kathi

discussed how they are fighting rare disease, tools and techniques in diagnosing rare diseases and

"Rare diseases are not rare", considering there are over 7,000 rare diseases. 30 million people

met with 2 Senators (and/or challenges for caregivers. in the US are living with a

Legislative Aides), and 2 House Representative Aides. The two main areas discussed were the Rare Disease Congressional Caucus that works to raise awareness of rare diseases,

This is an area that the Amyloidosis Foundation is working on. We want to support the caregiver with ideas and resources. In the evening, there was a `Rare Artist Reception' where

rare disease, that's 1 in 10 Americans, or 10% of the US population. In addition, 95% of these diseases do not have a FDA approved drug treatment. We are trying to change that...all

namely amyloidosis, and

artwork was displayed from of the rare diseases

asking for their support for the rare disease communi- together, including

robust funding for the NIH

ty. This was a wonderful

amyloidosis. AF



6

Utah Amyloidosis Symposium 2019

On May 17, one of our staff needs to change.

Session 2 started with Jo

members attended the 2nd Annual Utah Amyloidosis Symposium at the University of Utah in Salt Lake City.

Another point that he made was that AL should be searched for by physicians before the onset

Abraham, Clinical Medical Director for the Division of Nephrology; Director, Nephrology Fellowship Program; Director,

The event was sponsored by of symptoms. Once there is Nephrology, Utah

the Amyloidosis Foundation, symptomatic organ

Amyloidosis Program,

University of Utah Healthcare, involvement, it can

University of Utah Health/

the Huntsman Cancer

portend a poor outcome. Huntsman Cancer

Institute, Amyloidosis Support Groups and Amyloidosis Research Consortium.

The objective of the Utah Amyloidosis Symposium was to develop a forum that will catalyze, advocate, and provide education that promotes change, development and improvement for the early diagnosis and management of amyloidosis.

Tibor Kovascsovics,

Institute, Salt Lake City, UT.

Director, Hematology, Utah Dr. Abraham spoke about

Amyloidosis Program,

kidney involvement with

University of Utah Health/ amyloidosis, including

Huntsman Cancer

ALect2, a type of amyloido-

Institute, Salt Lake City, UT sis mainly associated with

was the next speaker on kidney damage.

the agenda. He spoke

about novel agents for the Many experts agree that

treatment of AL amyloido- ALECT2 amyloidosis should

sis. He mentioned that

be considered while look-

CAEL-101 has specifity of ing for a diagnosis when a

antibody binding for AL. He patient has evidence

Speakers included many top has seen a 97% cardiac of renal (kidney) disease. A

physicians and researchers in response and a 50% renal patient's symptoms may

the field of amyloidosis. The response.

appear as renal failure or

first speaker was Giovanni

"nephrotic syndrome".

Palladini, Acting Director, Amyloidosis Research and

Vaishali Sanchorawala, Director, Autologous Stem

Nephrotic syndrome is a group of symptoms that

Treatment Center,

Cell Transplant Program; relate to kidney issues

Foundation "Istituto di

Director, Amyloidosis

including: protein found in

Ricovero e Cura a Carattere Center, Boston University the urine, high cholesterol

Scientifico (IRCCS) Policlinico School of Medicine,

levels, low blood pro-

San Matteo" and Department Boston, MA spoke about tein levels, and swelling.

of Molecular Medicine,

Hematology, the role of

University of Pavia, Italy. He stem cell transplantation Luke Gessel, Specialist,

spoke on median survival and (SCT), and Grand Rounds.

Gastroen-

mentioned that amyloidosis is Following this session was a the "great imitator". His slides question/answer period included one that had some with a multidisciplinary discouraging statistics that panel discussion on how to 32% of patients need to see 5 approach the treatment to or more physicians to reach a patients with advanced

diagnosis. All agreed that this (Stage IV) AL amyloidosis.

terology, Utah Amyloidosis Program, University of Utah Health/ Huntsman



7

(Continued from page 6)

University of Utah Health/

Cancer Institute, Salt Lake Huntsman Cancer Institute,

City, UT was the next

Salt Lake City, UT, told

speaker. He spoke about about the survival rates

gastrointestinal involvement. between the different types

With amyloidosis, the small bowel is the most affected gastrointestinal organ.

of amyloidosis with cardiac manifestations. He mentioned that cardiac AL is a

The next speaker was Chakravarthy Reddy, Specialist, Pulmonology, Utah Amyloidosis Program, University of Utah Health/

medical emergency. He role of genetic counseling

also told about the non- in amyloidosis.

invasive testing called

Pyrophosphate scintigraphy She is a Genetic Counselor,

(PYP scan).

Utah Amyloidosis Program,

University of Utah Health/

Huntsman Cancer Institute, Salt Lake City, UT.

Session 4 began with Kelsey Huntsman Cancer Institute, Barrel, Specialist, Neurolo- Salt Lake City, UT.

He spoke about the different ways that amyloidosis manifests as pulmonary issues. It was insightful to patients, doctors and researchers alike.

The speakers of session 3 started with Mathew Maurer, Medical director of The HCM Center New YorkPresbyterian Hospital/

gy, Utah Amyloidosis Program, University of Utah Health/ Huntsman Cancer Institute, Salt Lake City, UT.

Her main focus was the burden and management in amyloidosis neuropathy, which starts in the toes and moves to the feet, legs and hands.

Isabelle Lousada, CEO and President, Amyloidosis Research ConsortiumBoston, MA started session 5 with a talk on quality of life in amyloidosis, followed by a patient panel discussion about patient referrals.

Each one had a different story, but there were many similarities. Most went to

Columbia University Medical Sami Khella, Chief, Depart- several physicians and spe-

Center, New York, NY. Dr. ment of Neurology, Penn cialists before being diag-

Maurer is also on the Scien- Presbyterian Medical

nosed. It was noted to the

tific Advisory Board for the Center, University of

physicians in the audience

AF.

Pennsylvania School of

that they need to look at

He spoke about Cardiology Grand Rounds and the ad-

Medicine, Philadelphia, PA, spoke of the impact of

symptoms in a multidisciplinary fashion.

vances in TTR (transthyretin) gene silencers in neuropa- Closing out the symposium

protein stabilization and

thy and quality of life with

was session 6, with a

degradation of amyloid fi- patients diagnosed with

Multidisciplinary Panel

brils for ATTR amyloidosis. So hereditary amyloidosis. He

Discussion about referral

much has changed in the told about the scales used patterns for early diagnosis,

last few years. There have for a clinical practice,

access to therapy and

been many advances.

which focuses on weakness research collaboration. It

versus those used in clinical

Jose Nativi, Director, Cardio- trials.

vascular Intensive Care Unit;

Director, Cardiology, Utah Katelyn Swade ended this

Amyloidosis Program,

session talking about the

was a day full of learning for all of those in attendance. We are looking forward to next year's event.

AF



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