For a Future without Childhood Dementia

1. CLN3
For a Future without Childhood Dementia
Dr. Frank Stehr, Dr. Herman van der Putten, NCL Foundation, Hamburg, Germany, www.ncl‐foundation.com
The NCL Foundation
On 7 August 2002 the NCL Foundation officially
declared its fight on the deadly children’s disease
Neuronal Ceroid Lipofuscinosis (NCL), the most
common form of childhood dementia. NCL is one of
the rare diseases known in medical circles as “orphan
diseases”. These disorders are virtually unknown and
receive usually no attention. As a result the approxi‐
mately 700 children with NCL in Germany (worldwide
approx. 70,000) are left more or less on their own.
Conclusion
With marginal financial input, the NCL Foundation
recruits new laboratories and multidisciplinary
expertise. The majority of these new recruits have
gone on to obtain additional funding from other
sources and are staying in the field of NCL research.
The NCL Foundation initiates collaborations between
researchers, clinicians, and companies making
substantial contributions to the field of NCL research.
Our initial projects in fundamental science have
reached the pre‐clinical phase. The next step will be
therapy. This will require a coordinated and
international effort.
Acknowledgements
We would like to thank all families for not giving up
and holding up the fight against Batten Disease. You
are the real experts and are in the position to make a
difference. Thank you for encouraging researchers to
develop a cure for the kids. Additionally, we would
like to extend our thanks to all volunteers, young
researchers, sponsors and private donors. They help
us to support research to find a cure.
Strategies – Overview
The charitable foundation aims to further research
against this fatal disease by
• informing medical experts to prevent false
diagnoses.
• building an NCL‐network of medical specialists to
collect and coordinate existing expertise.
• initiating research concerning possible cures by
creating research fellowships or other initiatives.
• increasing public awareness of NCL in order to
promote the early diagnosis of the disease.
The NCL Foundation funds research work
The NCL Foundation actively supports funding of research work in order to ramp up
developments in current research and thus obtain new scientific findings. The Foundation
functions as a neutral coordinating body for the global interlinking of expert knowledge. It thus
primarily funds (post‐)doctoral students with the objective of boosting basic research. Over and
above this, the Foundation initiates and accompanies collaborative research projects by
providing and bringing together experts. The money raised from the Foundation’s own charity
events against NCL and with the help of active partners and sponsors is also used to provide
financial support.
Scientists still face the challenge of understanding the NCL form of childhood dementia and,
ultimately, of also developing treatment and healing methods. Research is thus the most
important area of the NCL Foundation’s work.
NCL
Foundation
Education Networking
Research Funding Fundraising
• Medical education
• School projects
• Publications
• PhD fellowships
• Collaborations
• NCL Research Award
• NCL workshops
• PhD symposia
• Consulting
• PR Work
• Sponsors
• Charity Events
university
university
university
university
Research institute
Company
Training
Early diagnosis of the disease is crucial for the timely initiation of counter measures to delay
symptoms. This is the reason why the Foundation promotes and organizes NCL advanced
training courses and seminars for various medical disciplines and specialist consulting
physicians. These include national expert conferences at which the NCL Foundation is
represented with its own speakers to publicise the disease. Contacts with paediatricians and
ophthalmologists in particular are important since they play a key role in diagnosing NCL. Over
and above this, human geneticists provide parents of sufferers with the advice they so
desperately need, making them additional important partners of the NCL Foundation. In
addition to physicians, pupils, teachers
and students are important partners
of the Foundation in the battle
against NCL.
Within the scope of the “Study
partnerships for senior study profiles”
The pupils not only learn more about
rare diseases but also have the
opportunity to get active themselves.
Networking
The NCL Foundation has many years’ experience in
the fields of knowledge transfer, initiating research
and research networks. The Foundation has
successfully initiated at least 50 new collaborations.
The vast majority are cooperation projects between
university groups. New laboratories and experts have
thus been won for work in the field of NCL,
collaborating with established NCL groups.
NCL Research Award
Dr. Frank Husemann, the
Foundation’s founder,
presents the 1st NCL
Research Award to winner
Dr. Vydehi Kanneganti.
The winning research idea
came from Prof. Jeffrey
Gerst’s Rehovot,
Israel‐based laboratory.