annual report 2015 - NCL

ANNUAL REPORT 2015
of the charitable NCL Foundation
FOR A FUTURE WITHOUT CHILDHOOD DEMENTIA
TRAINING
|
RESEARCH
|
FUNDING
|
FUNDRAISING
NCL FOUNDATION
For a future without childhood dementia
ABOUT THE CHILDHOOD DEMENTIA NCL
Neuronal Ceroid Lipofuscinosis (NCL) is a genetic metabolic disease. This disease, also known
as childhood dementia, is characterized by a
gradual degeneration of nerve cells. The cause
of NCL is a malfunction in the cellular waste
disposal system. Due to the deposits the cellular
processes cannot function properly.
The first symptom is the complete loss of vision
at primary-school age followed by years of degeneration. The children forget things they have
learned and gradually lose the ability to speak,
walk and act. They also suffer epileptic seizures.
In the end, they usually lose their fight against
NCL at the age of 20 to 30.
In Germany, there are currently 700 children
suffering from NCL, which is why childhood
dementia counts as a rare disease. As a consequence, the pharmaceutical industry does
not pay much attention to NCL which makes
finding a therapy very difficult. This is where the
NCL Foundation comes into play to promote
research into this disease – for a future without
childhood dementia.
GREETINGS FROM THE FOUNDER
On 1 October 2015, our Federal President honored me with The Order of Merit of the Federal
Republic of Germany. This is a great acknowledgement of what we were able to achieve
with the joint commitment of many people.
Since 2002, the founding year of the NCL
Foundation and the year in which my son was
diagnosed with NCL, our main goal has been
to drive research towards finding a therapy to
successfully treat NCL.
Therefore, I would like to thank everyone who –
each in their own way – contributes to achieving this goal. I also want to encourage them
to continue to cooperate with us. Because it is
worthwhile, especially for the affected children.
In 2015, we again got a step closer to achieving
our goal – a future without childhood dementia
– both regarding research and information and
education.
This report is meant to give you an impression of what we have achieved this year. I look
forward to the challenges ahead of us and to
overcoming them one by one.
Yours, Dr. Frank Husemann
The President of the Federal Republic of Germany,
Joachim Gauck, presented Dr. Frank Husemann
with The Order of Merit of the Federal Republic of
Germany.
Allocation of donations in 2014
The figure below shows how donations were
allocated in the financial year 2014. In line with
the foundation’s main focus, we invest the main
part of the donations in research. The figure
does not include the indirect research funding,
i. e. co-operations with third parties initiated by
us, and sponsoring contributions such as free
printing, waived telephone charges etc..
RESEARCH EXPENDITURE 264.8
TRAINING 82.5
EVENTS 53.7
Thorsten Schröder
(News anchor and presenter at the German TV
station “ARD”) ©Henning Scheffen Photography
“I have been actively supporting the NCL
Foundation for four years now. I think it’s
important that rare diseases such as NCL are
not forgotten, which is why I draw attention
to NCL childhood dementia in whatever
way I can – usually on my bike.”
PROJECT SUPPORT 37.7
ADMINISTRATION 22.8
TRAVEL EXPENSES 12.6
in EUR thousand
2ND PLACE AT
THE ASPIRIN SOCIAL AWARD 2015
Every year, the Bayer Cares Foundation
tenders the Aspirin Social Award to honor
the best aid and consultancy services in the
German health care sector. We are happy
that our school project was awarded second
place.
TRAINING · RESEARCH
The NCL Foundation‘s training initiatives
SCHOOL PROJECT
For rare diseases such as NCL nothing is more important than awareness. For that reason one of our
core goals is to inform health care providers, pupils, students and the general public with targeted
initiatives.
KEY FIGURES OF 2015
220
597
510
HEALTH CARE PROVIDERS
INFORMED
200
PAGE VIEWS FOR OUR CERTIFIED
ONLINE TRAINING COURSE
INFORMATION MATERIALS DISTRIBUTED
AMONG HEALTH CARE PROVIDERS
60
LIFE SCIENCES STUDENTS
INFORMED
66
368
PUPILS INFORMED
IN COLOGNE
PUPILS INFORMED
IN HAMBURG
PUBLICATIONS (E.G. IN THE GERMAN
NEWSPAPER “DIE ZEIT”, THE MAGAZINE)
13TH NCL RESEARCH CONGRESS IN HAMBURG
The objective of our work is to connect
researchers and pool knowledge. This is
why our NCL Congress, which was held for
the 13th time on 2 November 2015, is one of
our most important events. 30 international
researchers and 6 PhD students/postdoctoral
researchers we support exchanged views
about innovative approaches in NCL research
and started new co-operations. This is essential for research into rare diseases.
© Simon Baumann Fotografie
© Bayer Aspirin Sozialpreis
“It is repeating the relevant topics in a
concrete way but at the same time it is also
an approach that pupils would be denied
otherwise.” Maik Zaborowski, teacher and
long-standing partner at the Hamburg
secondary school Matthias-Claudius-Gymnasium, firmly believes in the concept “NCL
macht Schule” (a school project initiated by
the NCL Foundation). This project provides
pupils a sensitive and exciting approach
to childhood dementia and raises their
awareness of rare diseases. Thanks to the
support by the Bayer Science & Education
Foundation, “NCL macht Schule” is now also
offered at schools in Cologne, so even more
pupils get the chance to participate in this
unique project.
Overview of the international research activities
ONGOING FUNDED PROJECTS
D: Cologne, Dresden, Hamburg, Münster
USA: Houston
COMPLETED PROJECTS
D: Berlin (2x), Cologne, Dresden, Düsseldorf,
Frankfurt, Hamburg (2x), Leipzig, Regensburg,
Würzburg
IL: Rehovot (2x)
UK: London (4x)
USA: Iowa City, New York City (2x)
LABORATORIES AND
CONGRESSES VISITED IN 2015
CH: Basel, Lausanne
D: Cologne, Dresden, Frankfurt, Hamburg
LU: Luxemburg
NL: Nijmegen
PT: Lissabon
UK: Cardiff, London
USA: Boston, Galveston, Houston, Waltham
INFOBOX
Our research activities spread around the
world. Apart from funded projects, we visit
congresses and laboratories on a regular
basis to improve research networking.
RESEARCH · FUNDING
GOAL: Within the next 3 years develop assays supporting therapy development
THE NCL FOUNDATION’S KEY RESEARCH AREAS
E
I N XP
A
R
DEVELOP
ASSAYS
SUPPORTING
THERAPY
DEVELOPMENT
VE
D E ES
EA L M
D IS C EL
R E LO P
O LE
D E VA
LS NT
T
PS IF Y
SE WI
AS T H
ES
C
CLN L AR
3 F IF
U
RE T
D RCH
AN SEA
E
NG N
YI C T I O
N
EN
I D LA I
ER D
OV HER
OT
ALBERTO DI RONZA (HOUSTON, USA)
Project: Postdoctoral fellow Alberto di Ronza conducts research
into the molecular function of the CLN31 protein. So far, its function
is poorly understood. Dr. di Ronza focus is on the role of CLN3 in
intracellular organelles that play a key role in the degradation and
recycling of metabolites. His work might reveal novel routes for therapy and biomarkers. The latter are essential
for conducting successfully clinical trials in
CLN3 patients. This project received the 5th
NCL Research Award.
Supervisor: Dr. Marco Sardiello, Baylor
College of Medicine
Funding partners: AstraZeneca, Beyond
Batten Disease Foundation, Charity Dinner,
Art Auction, Town & Country Foundation
ABUZAR KALEEM (HAMBURG, GERMANY)
Project: PhD student Abuzar Kaleem continues with promising
work that was and still continues to be conducted at the Center
for Molecular Neurobiology inHamburg (ZMNH). He examines the
interaction between CLN31 and another protein that is known in
association with Alzheimer’s disease (AD).
The main goal is to understand the role of
this interaction and what potential role CLN3
plays in this AD-relevant molecular pathway.
Supervisor: PD Dr. Guido Hermey, Center for
Molecular Neurobiology Hamburg (ZMNH)
Funding partners: Dorit & Alexander Otto
Stiftung
CRISTINA GOLFIERI (DRESDEN, GERMANY)
Project: Postdoctoral researcher Cristina Golfieri works with
human induced pluripotent stem (hiPS) cells2 and focuses on the
retina. hiPS cells are derived from skin cells of affected NCL patients.
Dr. Golfieri grows three-dimensional retina-like structures from
hiPS cells to examine CLN3-dependent pathological changes in the
retina such as lipofuscin deposition, a hallmark of cellular changes
in NCL. Hopefully, the human retina model
will facilitate the development of a therapy
that prevents or halts loss of vision in NCL.
Children experience this burden already
around school age and it has a major impact on their lives.
Supervisor: Dr. Mike Karl, German Center
for Neurodegenerative Diseases (DZNE)
Funding partner: Ein Herz für Kinder e.V.
GEMMA GÓMEZ GIRÓ (MÜNSTER, GERMANY)
Project: PhD student Gemma Gómez Giró’s goal is to create brain
neurons from human induced pluripotent stem (hiPS) cells2 with
different CLN3 disease-associated mutations. The goal is to better
understand CLN3-related pathological
processes within neurons and hiPS-derived
brain-like structures. These models should
also provide a basis for target validation
and drug discovery in academia and the
pharmaceutical industry.
Supervisor: Prof. Dr. Hans Schöler, Max
Planck Institute for Molecular Biomedicine
Funding partners: Batten Research Fonds,
Berti Vogts, Münsteraner Krimi-Cup
KATHARINA DANNHAUSEN (COLOGNE, GERMANY)
Project: PhD student Katharina Dannhausen examines the
influence of immunoregulatory substances on disease progression
in the retina of NCL mouse models. She focuses on modulating the
role of specific and highly specialized immune cells in the retina,
the microglial cells. These cells are thought to play a key role in the
NCL disease process in the retina. A further goal is to generate and
study NCL patient-derived microglial cells
derived from hiPS cells2.
Supervisor: Prof. Thomas Langmann, University Hospital of Cologne – Project team
Experimental Immunology of the Eye
Funding Partners: Ein Herz für Kinder
e.V., Hans and Marlies Stock Foundation,
Kämpgen Foundation, Marga and Walter
Boll Foundation
1)
CLN3 gene – The gene mutated in juvenile NCL. The majority of patients have a deletion in this gene. This deletion results in a CLN3 protein that is truncated and dysfunctional. Other patients carry e.g. non-sense mutations, that also result in a truncated protein or missense mutations, that result in a functionally different and dysfunctional protein.
2)
hiPS cells – human induced pluripotent stem cells are generally derived by reprogramming skin fibroblasts into stem cells. These stem cells can be cultured under different conditions,
such that they will develop into e.g. nerve cells. They offer unique potential for facilitating drug discovery as hiPS-derived models are human cell-based. These models can offer a good
alternative to using animal models.
FUNDRAISING
Highlights in 2015
JANUARY
Charity Partner MeridianSpa Hamburg – As
this year’s charity partner of the gym and spa
we were benefitted at many events such as a
prize draw in the context of the autumn party.
FEBRUARY
12th Charity Art Auction in Hamburg – Under the motto “Life Artists” many paintings
and photographs were auctioned off at the
event‘s venue JENSENs
Lagerhaus. Among
them were artworks
by Armin MuellerStahl, Romero Britto
and Heinke Böhnert.
Under the patronage of
senator Prüfer-Storcks
amazing proceeds of
23,000 Euros in total
were raised!
Host Birgit Saatrübe and auctioneer Joseph Weber
©Leila Ivarsson
Ritzenhoff co-operation with Jan Josef Liefers Together with our ambassador Jan Josef
Liefers and Berlin artist Olaf Hajek, Ritzenhoff created a collection of elegant champagne glasses.
APRIL
11th Charity Dinner “Cooking for Kids” in
Hamburg – Under the patronage of Dr.
Karl-Joachim Dreyer
(chairman of the HASPA
supervisory board), this
year’s traditional Charity Dinner took place at
the Okzident restaurant
within the Museum of
Ethnology in Hamburg.
More than 70 guests
enjoyed to be entertained by mentalist
Thorsten Havener
Thorsten Havener
and host Melissa
© Simon Baumann
Ortiz-Gomez.
Fotografie
MAY
1st Andreas Köpf Cup – To celebrate the anniversary of the physiotherapeutic practice
Köpf, the Köpfs hosted a charity golf tournament at the Golfclub Hamburg-Holm. More
than 5,000 Euros were raised for NCL research.
JUNE
Warsteiner and EDEKA Struve working
together for a good cause – Under the motto
“Every crate counts” the brewery donated
1Euro per crate.
Marriott DOM – Lots of fun for the good cause:
the raffle proceeds of this year’s DOM
organized by the Hamburg Marriott Hotel
were donated to the NCL Foundation.
JULY
10th NCL-Golf-Trophy – At the 10th anniversary
of this golf tournament at Castanea Resort
Adendorf that took place under the patronage
of the Lower Saxony Minister of the Interior
and Sports Boris Pistorius and Adendorf’s
mayor Thomas Maack, we were pleased to
welcome the actors Katrin Wrobel, Gerry
Hungbauer and Oliver Hörner and were
able to raise a large amount of money for our
foundation.
3rd “Thriller Cup” in Münster – Jan Josef Liefers, Werner Schulze-Erdel and the Volksbank
Münster sent out invitations for the 3rd Thriller
Cup in Münster. 115 golf enthusiasts, among
them Gerhard Delling, Uli Stein, Anna Loos
and Caroline Beil,
accepted the invitation and convened
at the golf course
Aldruper Heide e.V..
The day was rounded off with a gala
Werner Schulze-Erdel, Anna at the Mövenpick
Hotel Münster.
Loos and Jan Josef Liefers
© Joachim Busch
Host Dr. Martin Wilhelmi and Oliver Hörner at the
10th NCL-Golf-Trophy
© Simon Baumann Fotografie
Donations for a cause – On his mountainbike,
Jan Hähnlein travelled from Erfurt to Berlin
and Almira Schwarzer walked the Way of St.
James to raise money for our foundation.
AUGUST
Golf tournament of the Round Table 4 – For
the 4th time the members of the RT4 around
Arne Hansen organized an exclusive charity
golf tournament including a helicopter ride
and creative charity games.
SPRINGER Bio-Backwerk – Again the longestablished company SPRINGER put up our
donation boxes in their stores in Hamburg.
SEPTEMBER
8th Vintage Car Festival – For the 8th time now
visitors were able to take a look at vintage
cars in the park of the Randel restaurant. In
co-operation with
the Old Tablers
300 Hamburg the
NCL Foundation organized this event
for visitors both old
One of the vintage cars: a
and young.
Mercedes Cabriolet
Charity run of the secondary school Gymnasium Süderelbe – The students charity run was
one of many exciting events in the context of
our school project.
Duck race – Thanks to the RT-115, the OT-215
and Leos Dortmund 1,554 rubber ducks
floated along the river Lippe for the benefit of
our foundation.
2nd Charity Dinner “Cooking for Kids” in Düsseldorf – Under the patronage of Mayor Thomas
Geisel, 15,000 Euros were raised at the exclusive restaurant Lido 1960. Ventriloquist Daniel
Reinsberg, musical
star Zodwa Selele
and chef de cuisine
Florian Ohlmann
were applauded for
their performance
and gourmet
Musical star Zodwa Selele
kitchen.
© Colin Vennes Fotografie
OCTOBER
Charity Nite in Walldorf – For the first time,
the Ladies’ Circle 17 Heidelberg organized
a Charity Nite. The proceeds were donated to
the refugee hostel in Walldorf and the NCL
Foundation.
Restaurant event “Cooking for Kids” – Under
the patronage of TV
chef Ralf Zacherl, 11 restaurants participated in
our event and donated
part of the proceeds of
a specific meal to our
foundation.
Ralf Zacherl © Florian Bolk
NOVEMBER
Charity Cycling Marathon – Under the motto
“We cycle for life” 40 teams pedaled for us at
the MeridianSpa Wandsbek – cheered on by
the organizer Torsten Statz. Among others,
the NCL Foundation’s team included ARD news
anchor Thorsten Schröder.
DECEMBER
Advent Reading – This year’s advent reading
again took place at Ansgar Church, this time
with actress Saskia Fischer. The collected money was donated to the foundation Alzheimer
Gesellschaft Hamburg and the NCL Foundation.
CONTACT
DONATION ACCOUNT
NCL Foundation
NCL Foundation
IBAN: DE50 2005 0550 1059 2230 30
BIC: HASPDEHHXXX
(Hamburger Sparkasse)
Holstenwall 10
20355 Hamburg, Germany
T: +49 (0) 40 696 66 74-0
F: +49 (0) 40 696 66 74-69
E: contact@ncl-stiftung.de
For donations of EUR 200 or more we will send you a
receipt deductible for tax purposes. We therefore request
you to state your name and address in the purpose field
of the bank transfer form.
www.ncl-stiftung.de
www.ncl-foundation.com
Numerous sponsors and voluntary helpers have once again supported us in 2015.
Concerts, fundraising activities, sports events and many other projects help make
NCL known and promote NCL research. We would like to thank all active, loyal
and new supporters for their help.
HONORARY FOUNDATION COUNCIL
a&c Druck und Verlag • A. Behrens & G. Felsch • Adler Vertrieb • Airwedding • Alarmzentrale Hamburg • Alison Frey • arielgrafik • A-ROSA • Art Consulting • Barceló •
Barkassen-Meyer Touristik • Bartels Druck • Best Western Hotel Hamburg International •
betterplace.org • Bilderwerk Hamburg • Blue Café • Buchbinder • Budnianer Hilfe • Caffé
Chicco D‘Oro • Carl Tesdorpf • Carl Zeiss Microscopy • Cha Chã Restaurants • Charity Conception Birgit Saatrübe • Chilli Club • Christian Brinkmann • Christian Damerius • Claudia
Musekamp • Colin Vennes • Comedia MDS IT • Confiserie Heilemann • das druckhaus
digital • Datadruck • Delinat • Derag Livinghotel • Derag Livinghotel De Medici • dgroup
• die kleine konditorei • DIE ZEIT • DMV Eventbedarf • Dominic Packulat • druckriegel •
east Hotel & Restaurant • Eichhorn Solutions • Empire Riverside • engelmassage • Evert
Martin
Hartleif
Dr. Frank
Husemann
Dr. Christian
Borchard
Alexander
Rößler
Dr. Rainer
Kuhn
Veldhuijzen • Feinwerk Catering • Fiete Deichgraph • Fiftyfifty Galerie • Fontenay • Foss
(Finanances)
(Chair)
(Taxes)
(Legal)
(Research)
• Galerie Mensing • geiger notes • gibbins european architects • Gilhaus Eventmarke-
Doll • Friends of Britain • FuH Hamburg • Gabriele von Loeper • Galerie Anne Mörchen
ting • Glaxo-SmithKline • Gruner + Jahr • Hamburg Airport • Hamburger Abendblatt
FOUNDATION OFFICE
• HanseRent • Hartwig GmbH • HASPA • Hawesko • Hogan Lovells • Home Flowers •
Hotel St. Annen • Hotel Zum Alten Flecken • ibis Berlin Hauptbahnhof • Indoo Eisarena
• Inner Wheel Club Siegen • Insa Hagemann • Iris-Alexandra Hass • Jimdo • Jo Röttger •
John Bock • Jürgen Joost • Kerstin Michaelis • Kerstin von der Linden • Kirsten Nijhof •
Kleinhuis´ Restaurant im Baseler Hof • KPMG • Kreativkonfekt • Kunsthaus an der Alster
• Kunsthaus Lübeck • Kunstverein Düsseldorf • Labor Lademannbogen • Leo-Club Askanier • Lieselotte-Paulsen Stiftung • Lindner Hotel Am Michel • Madison Hotel Hamburg
• Magnolia Blumen • Maritim Hotel Dresden • Marley‘s Restaurant • MedLearning • Mercure Hotel Bochum City • Mercure Hotel Hamburg City • Messebau Feith • Mia Ohlsen •
Mika • Mondo Agit • Monster • Multicolor • Multiple Box • NH-Hotel City • Noah Wunsch
• Nordlite • NW-Zentrum • Olympus • Parkhotel Hohenfeld • PerLease • Peter Jensen •
Philippi Design • Phoenix Reisen • Platinum Oliver Rolf • Prima Quartett • Restaurant
26 • Restaurant Del • Restaurant Engel • Restaurant Filini • Restaurant Lenz • Restaurant
Dr. Frank Stehr
Dr. Herman v. d. Putten*
(Chair)
(Head of Research)
M: +49 (0) 178 - 341 60 57
frank.stehr@ncl-stiftung.de
M: +49 (0) 163 - 7 38 30 83
herman.vanderputten@ncl-stiftung.de
Tiziana Serio
Tanja Stern
Anne Erichsen
(Education, Fundraising)
(Event management)
(Event management trainee)
M: +49 (0) 177 - 24 63 758
tiziana.serio@ncl-stiftung.de
M: +49 (0) 163 - 51 09 785
tanja.stern@ncl-stiftung.de
M: +49 (0) 157 - 3 75 20 367
anne.erichsen@ncl-stiftung.de
Speicher 52 • Robinson Clubs • Rotaract Dortmund • Rotaract Lüneburg • Rotary Club
Bargteheide • Rotary Club Rhein-Selz • Round Table 1 • Round Table 22 • Round Table
70 • Round Table Deutschland • Scharlau • Serenaders • SIDE Hotel • Simon Baumann •
Smartchecker • smits • Stadthotel Münster • Stepstone • Stichting Beat Batten! • Sven
Helferich • S 1 Event • Tee-Shaker • Telefónica • TEREG • Thomas Lehne • Tom Thomsen
• Town & Country Stiftung • United Charity • Verband Hamburger Amateurtheater • VI
VADI Hotel München • Vladi Private Islands • WEMPE • Werner Berges • Yin Seafood • Zur
Kloster-Mühle (extract)
* The research unit is supported by the Auerbach
Foundation, which has been supporting the NCL
Foundation for many years.
HONORARY SCIENTIFIC ADVISORY BOARD
Translated from German into English by Sylvia
Lass within the initiative PerMondo. Sponsored
by Mondo Agit offering translations from German
into English.
Cover Photo: School project “NCL macht Schule”
© Bayer Aspirin Sozialpreis
Prof. Dr. Dr. Robert
Steinfeld
Prof. Dr. Beverly
Davidson
Prof. Dr. Rudolf
Martini
(University Clinic Göttingen)
(Children‘s Hospital of Philadelphia,
(University of Würzburg)
USA)
The printing and the compilation of the annual
report have been entirely sponsored by Max
Siemen printers, Vacanceselect Reisen and the
design agency sternklar. Many thanks!