Externally-Led Patient-Focused Drug Development Meeting - SEPTEMBER 16, 2019 HYATTSVILLE , MD
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Externally-Led Patient-Focused Drug Development Meeting FO R M Y E LOPROLIFE R ATIV E N EOPL A S M S S E P TE MB E R 1 6 , 2 01 9 H YAT T S V ILLE , MD
W E LCOM E LE T T E R
Dear Myeloproliferative Neoplasms community member:
On behalf of the MPN Research Foundation, I want to welcome
you to the Externally-Led Patient-Focused Drug Development
Meeting for the MPNs! We are so pleased that you have been
able to join us in person for this event and taking part via
the webinar. We and our partners - Leukemia & Lymphoma
Society, MPN Advocacy & Education International, MPN Cancer
Connection, and MPN Education Foundation – are eager to put
a spotlight on the stories of people living with Polycythemia
Vera, Essential Thrombocythemia and Myelofibrosis.
We know that it will be impossible to bring to bear the individual
experiences of the hundreds of thousands of people living
with an MPN in the U.S. alone, but we want to ensure that the
professionals in the Food and Drug Administration have the
benefit of what we as advocates hear daily.
People with PV, ET, and MF are living with this chronic blood
cancer. They often suffer in silence. They live with a variety
of symptoms and health risks as well as side effects of their
treatments. Finally, they are eager for more therapeutic options
and, eventually, a cure.
By being here today or taking part in the webinar, you are
helping every one of the people living with an MPN. Thank you
for making a difference.
Sincerely,
Michelle Woehrle, MPA
MPN Research Foundation
2 Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms
AG EN DA
8:30 am Registration
Opening Remarks
9:30 am
Michelle Woehrle, Executive Director, MPN Research Foundation
Clinical Overview of MPNs
9:40 am
Robyn Scherber, MD, MPH
Introduction and Meeting Overview
9:55 am
James E. Valentine, J.D., M.H.S., Meeting Moderator
10:05 am Audience & Remote Demographic Polling
Patient Panels and Audience Discussion
Topic 1: Living with an MPN
Panel Discussions
10:15 am • Living with Polycythemia Vera
• Living with Essential Thrombocythemia
• Living with Myelofibrosis
10:45 am Audience and Remote Polling
11:00 am Moderated Audience Discussion
12:00 pm Lunch
12:50 pm Living with MPN Video
Meghana Chalasani
1:00 pm
Center for Drug Evaluation and Research, U.S. Food and Drug Administration
Topic 2: Current & Future Treatments
1:05 pm • Panel Discussion
• Audience & remote polling
1:40 pm Moderated audience discussion
Recap and Closing Remarks
3:00 pm
Ruben Mesa, MD, FACP and John Mascarenhas, MD
3:20 pm Farewell Remarks
3:30 pm Meeting concludes
Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms 3
ABOUT THE MPN RESEARCH FOUNDATION www.MPNRF.org Our Mission The mission of MPNRF is to stimulate original research in pursuit of new treatments - and eventually a cure - for the blood cancers polycythemia vera, essential thrombocythemia, and myelofibrosis, known collectively as myeloproliferative neoplasms (MPN). Through a combination of MPN cancer research, advocacy, and education, we bring together patients, researchers, and clinicians around the common goal of realizing new treatment options and a cure for MPNs. Our History Diagnosed with polycythemia vera in 1997, Chicago businessman Robert Rosen was shocked to discover that little research was being conducted on PV, ET, and MF and that there were no advocacy groups working to assist people with these rare blood cancers. Our history as an organization that was founded by patients for patients continues to influence everything we do. In addition to funding promising MPN research, we work to educate and empower patients, family members, doctors, and researchers across the MPN community. Together, we’re committed to change the prognosis for people living with an MPN. Our Research Impact With more than $13 million in funding and over 60 funded research projects, we have made a significant difference in advancing our understanding of the causes of MPNs, the development of new drug therapies and in cutting edge research that has been published in leading scientific journals. Trusted Resource for Patients Founded by patients for patients, the MPN Research Foundation works every day to educate and empower patients, caregivers, doctors, and researchers throughout the world. MPNRF strives to provide the MPN community with the latest news in MPN research, treatments, drug discovery, community events, support groups, and educational resources. 4 Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms
O U R PART N E R S
MPN Advocacy and Education International is dedicated to providing the knowledge, support, and
resources patients will need as they adjust to living with an MPN through educational symposia
in several cities each year, website access, free webcasts of each program, collateral materials, and
direction to people, resources and other organizations that can help. MPN Advocacy and Education
International identifies unmet needs in the MPN Community and provides advocacy support to
individuals and groups unable to represent themselves.
The MPN Education Foundation is a non-profit organization run by volunteer MPN patients. It was
incorporated in early 2004 at the urging of Dr. Ayalew Tefferi of Mayo Clinic (Rochester). The initial
thrust of the Foundation was to continue the information/education/support goals of Dr. Harriet
Gilbert through her MPN Research Center, which unfortunately died when she did in 2003. Our
co-founder, Joyce Niblack(who passed in 2009), was a patient volunteer for Dr. Gilbert’s center and
Editor of the MPD VOICE newsletter.
MPN Cancer Connection (MPN-CC) was created by David Wallace. He is an outspoken “patient
advocate” who has Polycythemia Vera, an uncommon MPN blood cancer. Speaking from the
patient point of view, he experiences areas that need improvement and acts upon those needs.
MPN-CC partners with organizations who provide valuable resources to fellow patients. Funds
raised are used to create more educational programming and resources for patients.
The Leukemia & Lymphoma Society (LLS) is the world’s largest voluntary health agency dedicated
to blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin’s disease and myeloma, and
improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research
around the world and provides free information and support services for patients and caregivers.
Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms 5
SP E AK ER S
Mich elle Woehrl e , M PA
Michelle Woehrle has been working with the MPN Research
Foundation since 2007 in a variety of roles, most recently as
Executive Direc tor. PV patient and founder Bob Rosen was
Michelle’s mentor and was the f irst person with an MPN she
met (that she knew of ). Her passion is executing the mission of
MPNRF in a way that is true to the greatest unmet needs of the
people living with PV, ET and MF. Michelle has a Master ’s in Public
Administration with a focus in nonprof it management . She lives
in the suburbs of Chicago with her husband and two little boys.
Jam es Valentin e , JD, M H S
Mr. Valentine assists medical product industr y and patient advocacy
organization clients in a wide range of regulator y matters, including
new drug and biologic development and approval issues. Before
joining his current f irm in 2014 , Mr. Valentine worked in FDA’s Off ice
of Health and Constituent. Affairs where he facilitated patient
input in benef it-risk decision-making and ser ved as a liaison to
stakeholders on a wide range of regulator y policy issues. There, Mr.
Valentine administered the FDA Patient Representative Program,
launched the Patient-Focused Drug Development program, and
developed the FDA Patient Network.
PIVOTAL.
SCIENCE.
incyte.com
6 Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms
SP E AK ER S
Ru ben Mesa, M D, FAC P
Dr. R uben M esa i s t h e di rec to r o f UT Healt h San A nto n i o M D
An derson Can cer Center. He has been i nvo l ved i n MPN re sea rch
for m ore th an 20 years . He l ed t h e devel o pm ent o f Nati on a l
Compreh ensive Can cer Netwo rk’s pan el gu i del i n es , t h e f i rst
U. S . guidelin es o n t h e di a gn o s i s an d t reat m ent o f MF, PV a n d
ET. Dr. M esa h as been t h e pri n ci pal i nves t i gato r o r co - p ri n c i pa l
investigator of m o re t han 70 cl i ni cal t ri al s . He co - l ed th e
research team l ea di n g to t h e F DA’s approval o f fedrat i n i b a n d
ruxolit inib for PV an d MF. Dr. Mes a was el ec ted to s i t o n th e
National B oard o f Di rec to rs fo r t h e Leu kemi a an d Ly mp h om a
Soc iety an d is C hai r o n t h e Steeri n g Co mmi ttee fo r my M PN ,
th e M PN R esea rch Fo u n dat i o n’s o nl i n e pat i ent regi s t r y.
Robyn Sch erbe r, M D, M PH
Dr. Robyn Scherber is an ac tive clinician and researcher
focusing on chronic myeloid blood cancers, specif ically the
myeloproliferative neoplasms. She currently sees a variety of
patients with myeloid disease at UT Health San Antonio MD
Anderson Cancer Center, including myelodysplastic syndrome
and acute leukemia. She has a broad por tfolio of research
experience that has included the investigation of novel treatment
strategies to improve quality of life. These effor ts include
conduc ting investigational trials that evaluate the impac t of
pharmacologic and/or integrative care approaches to therapy.
John Mascaren h a s, M . D.
Dr. John Mascarenhas is an Associate Professor of Medicine
at the Icahn School of Medicine at Mount Sinai (ISMMS) and a
member of the Tisch Cancer Institute. Dr. Mascarenhas is the
Direc tor of the Adult Leukemia Program and Leader of Clinical
Investigation within the Myeloproliferative Disorders Program
at Mount Sinai, under the direc tion of Dr. Ronald Hoff man. As
a clinical investigator in malignant hematology with a focus in
translational research involving myeloproliferative neoplasms
(MPNs) and evolution to acute myeloid leukemia (AML), he is
focused on the evaluation of rationale based novel therapies for
patients with MPNs and AML .
Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms 7
PAT IE N T PA N E LI ST S
David K - PV Patie n t
D avi d i s a 54 -yea r- ol d market i n g an d techn o l o g y b u s i n es s exec u t ive
l i vi n g i n Co l o ra do. A f ter l o s i n g s i g ni f i c ant wei g ht an d s everal yea rs
o f ex pe ri e n ci n g vi s u al d i s t u rban ces , h e was event u al l y d i a g n osed
w i t h JAK 2 po si t i ve PV i n Ju n e 201 9 an d s t ar ted t reat m ent w it h
ph l e bo to m i e s a n d a co mp o u n d to co nt ro l t hro mb o t i c co mp lic a t ions.
H e h a d be e n wo rki n g i n a d eman d i n g f i el d b u t ha d to s to p work d ue
to t h e m e n t a l fo g c au s ed by fat i g u e an d v i s u al d i s t u rban ces . Even
n ow w i t h t h e sym p to ms m o s t l y al l ev i ated , h e i s co n cern ed h is life will
n eve r g e t ba ck to n o rmal .
Vicki B - ET Patie n t
Vi cki i s a m e di ca l jo u rnal i s t l i v i n g i n New Yo rk . S h e was d i a gn osed
w i t h Esse n t i a l T h romb o cy t h emi a 8 years a g o. Her p ro fes s i o na l skills
h e l p h e r to i nve st i g ate ever y an g l e o f h er d i a g n o s i s w i t h E T, a n d seek
i n fo rm a t i o n to h e l p h er u n d ers t an d h ow to mana g e h er d i s ea se b est
a n d w h a t h e r f u t u re may h o l d , es p ec i al l y as s h e fears s h e may b e
pro g re ssi n g to Mye l o f i b ro s i s .
Vivienn e W - PV Patient
Vivienne has been living with PV for 20 years. She was put on a JAK2
inhibitor four years ago and it’s done a great job controlling her spleen
and blood counts, but she now struggles with skin lesions, GI problems
and other quality of life challenges. She is thankful for the years that
she has sur vived and the wonderful people that she has met on her PV
journey, including MPNRF, her suppor t group, her doc tor and his off ice
staff, the scientists, the FDA and pharmaceutical companies, all who
have made her journey possible. She is grateful to her husband Richard
and her family, who have always understood her limitations.
B ridg et B - ET Pati e n t
B ri dg e t wo rks i n f i nan c i al s er v i ces o p erat i o ns an d l i ves i n East
S t . Lo u i s. S h e wa s d i a g n o s ed w i t h E T i n 201 5 an d has ex p eri e n ced
di sco rda n ce be twe en d o c to rs ’ o p i ni o ns o n h ow to b es t t reat h er
di sea se a n d sym ptoms . S h e’s b een s t ab l e o n a p l atel et red u cin g d r u g
a l o n g w i t h a n a spi ri n reg i m en b u t s t i l l ex p eri en ces cl o tt i n g issues,
a n d h e r pl a te l e t s are n o t wel l co nt ro l l ed . Her f req u ent sy mp toms
i n cl u de fa t i g u e , i tchi n g , nu mb n es s i n h er han d s an d l eg s , j o int pa in,
a n d pro bl e m s w i t h co n cent rat i o n.
8 Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms
PAT IE N T PA N E LI ST S
Ru th Fein R - MF Pa ti e n t
Ru t h h a s l i ve d w i t h an M P N f o r 2 3 y ear s . S he w as f i r s t d i ag nosed wit h
E T , w h i c h t r a n si t i o n ed t o P V , and r ec ent l y t o M F . S he has t w o a d u lt
so n s l i vi n g i n Ma n hat t an and s he l i v es w i t h her hu s b and i n Sa ra t og a
S pr i n g s, N Y . H e r MP N ex p er i enc e i nc l u d es a mi s d i ag no s i s f rom b one
pa i n a n d m i g r a i n e s , a maj o r t hr o mb o t i c ev ent , and a s er i o u s G I
h e m o r r h a g e f o l l o w i ng s u r g er y f o r c o l o n c anc er l as t y ear . S t i ll, she liv es
h e r da y- t o - da y l i f e , “ p r i mar i l y s y mp t o m f r ee.” S he i s a f r eel anc e wr it er
a n d c o m m u n i ca t i o n s c o ns u l t ant s p ec i al i z i ng i n heal t h and r enewa b le
e n e r g y. R e ce n t l y, sh e b eg an l o b b y i ng f o r r ar e d i s eas es and e xp loring
w a ys t o h e l p o t h e r s l i v i ng w i t h M P Ns .
Karrie S - ET Patie n t
K a r r i e i s 4 8 ye a r s o l d and w as d i ag no s ed w i t h E T w hen s he w as
pr e g n a n t w i t h h e r f i r s t c hi l d at ag e 3 7 . T w o y ear s l at er , her d ia g nosis
ch a n g e d t o P V. H e r M P N p o s ed s i g ni f i c ant hu r d l es f o r her i n a c hiev ing
h e r g o a l t o h a ve m o re c hi l d r en. S he t hen f o u nd a d r u g t hat help ed
co n t r o l h e r di se a se a nd al l o w ed her t o c ar r y her s ec o nd c hi l d t o
t e r m . S h e ’ s n o w f o c u s i ng o n mak i ng t he mo s t o u t o f l i f e and b ring ing
a w a r e n e ss t o pa t i e nt s l i k e her w ho may no t r eal i z e t hat t her e a r e
t h e r a pe u t i c o pt i o n s av ai l ab l e.
David A - PV Patie n t
Living with diagnosed PV since 2005, but neurocognitive issues for
15+ preceding years. A regular blood donor since age 18, he always felt
better after donation, same after starting therapeutic phlebotomies.
Started a JAK2 inhibitor in 2016 against intractable pruritus but also
had a 70% spleen response, although he had no spleen symptoms.
Had to discontinue the JAK2 inhibitor in 2019 due to life threatening
cryptococcal infection. He has remained on a phlebotomy regime
without problems since diagnosis. David is President of the all-patient/
volunteer MPN Education Foundation, and is an online and DC local
support group leader.
Dian e R - PV Patie n t
D i a n e w a s di a g n o sed w i t h an M P N 1 4 y ear s ag o and has s een it c ha ng e
f r o m o n e f o r m t o ano t her . S he has s o me s u c c es s w i t h m ed ic a t ion
c o n t r o l l i n g h e r bl o o d c o u nt s b u t no t her s y mp t o ms . Her b i g g es t c onc erns
a r e t h e po ssi bl e n e g at i v e s i d e ef f ec t s o f c u r r ent t r eat ment o p t ions a nd
t h a t t h o se sa m e t r e a t ment s d o n’t al l ev i at e her o ng o i ng M P N s y mp t oms.
Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms 9
PAT IEN T PA N E LI ST S
Nan cy D - MF Pat i e n t
N a n cy i s 52 yea rs o l d , i s JA K 2 p o s i t i ve, an d has b een l i v i n g wit h a n
MPN fo r 33 yea rs. S h e was d i a g n o s ed w i t h PV i n 1 986 d u ri n g a rou t in e
bl o o d te st fo r m o n o. S h e t h en p ro g res s ed to M F 26 years l ater. Af ter
m a ny di f fe re n t t h erap i es , i n cl u d i n g two cl i ni c al t ri al s , n o t hi n g ha d
be e n a bl e to sh ri n k h er s p l een u nt i l h er c u rrent t reat m ent , t h e
re ce n t l y a pprove d JA K 2 i nhi b i to r. S h e was recent l y d i a g n o s e d wit h
t h e ASXL1 g e n e a n d i s co ntemp l at i n g a s tem cel l t rans p l ant , b ut d oes
n o t c u rre n t l y qu a l i f y d u e to h er mas s i ve s p l een an d am o n g ot h er
co m pl i c a t i o n s. H e r o t h er M PN sy mp to ms have b een mana g ea b le
w i t h ea ch t h e ra py al o n g t h e way.
Ned W - MF Patie n t
Ned, trained as a chemist , is semi-retired as an executive in the
pharmaceutical and medical device industr y. He is 76 and was
diagnosed with PMF in November 2018 . He entered a clinical trial
for a new experimental drug for MF in late March 2019. This has
required travel f rom Nor thern Utah to UCLA ever y 3 weeks. He feels
his constitutional symptoms have been signif icantly reduced and his
spleen volume has also decreased, however he has had to begin red cell
transfusions. Overall he is currently living an almost normal life with
the disease.
Morg an G - MF Pa ti e n t
Mo r g a n i s a 52 ye ar o l d mar r i ed mo t her o f t hr ee. S he l i v es in t he
C h i c a g o l a n d a r e a and w o r k s as a P r o j ec t M anag er f o r a mee t ing s
a n d e ve n t c o m pa ny . S he w as d i ag no s ed i n Janu ar y 2 0 1 9 and t a k es a
co m po u n d to co n t ro l t hro mb o t i c co mp l i c at i o ns and as p i r i n d a ily . Her
MP N e x pe r i e n ce i n c l u d es l i v i ng w i t h d ai l y s y mp t o ms o f b o ne p a in,
n i g h t sw e a t s, f a t i g u e, j o i nt p ai n, nu mb nes s / t i ng l i ng i n hand s a nd
f e e t , a n d i ssu e s w i t h c o nc ent r at i o n. Her b i g g es t c o nc er n i s liv ing wit h
t h e u n kn o w n pr o g r es s i o n o f an M PN.
10 Patient Focused Drug Development Meeting for Myeloproliferative NeoplasmsmyMPN PAT I E N T R EG I ST RY
www.MPNRF.org/mympn-register
About myMPN
myMPN empowers patients to change their prognosis by sharing their experience of living with an
MPN. The development of targeted disease therapies will be expedited when firsthand information
about disease symptoms and progression are available from patients.
What is myMPN?
• It’s a digital hub for patients to record and anonymously share their unique MPN journey with
the research community.
• Participants can access a secure online portal with a personalized dashboard. As they complete
surveys listed on the dashboard, the registry will provide insights into how the user’s MPN
experience compares to other registry users.
• All patient data is protected and only shared according to individual user privacy settings.
• myMPN is a place for eligible patients to connect with upcoming drug trials and observational
studies that will help increase our knowledge about PV, ET, and MF.
• Participation is currently limited to those inside the U.S. and over the age of 18.
• myMPN is not yet mobile-friendly, so please use a laptop or desktop to access your account.
What are the benefits of joining myMPN?
• myMPN functions as a repository for each participant’s experience with an MPN. Each
completed survey is accessible by the user going forward. Participants can download their
symptom, event and other data to share with their doctor or for other purposes.
• The registry provides participants with the opportunity to inform the research and drug
development community with data they need to move better MPN treatments (and, potentially,
cures) through the discovery pipeline more quickly.
• The platform offers the research community the ability to follow-up on specific topics of interest.
For example, a researcher may request additional information on the experience of a group of
patients who have used a particular therapy.
• Participants will learn about clinical trials that may be of interest to them.
Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms 11TH AN K YO U TO O UR SP ONSORS GOLD SILVER BRONZE 12 Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms
NOT ES
Patient Focused Drug Development Meeting for Myeloproliferative Neoplasms 13MPN RESE ARCH FOUNDATION 18 0 N MICHIGAN AVE, SUITE 18 70 CHIC AGO, IL 6 0 6 01 W W W. MPNRF.ORG
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