Research and Mission
Mission of KAKS in Research
KAKS aim is to support research projects in a field, where either due to the rarity of the disease or/and lack of patent protection, commercial interest is basically non-existent. Since its foundation KAKS initiated, financed, supported and completed retinoblastoma based research projects in search of for innovative therapies and diagnostic methods. Within these research projects important components were identified, the understanding of the retinoblastoma, its mechanisms and diagnosis pathways was deepened and also the late effects of the tumour disease were identified. The KAKS funding strategy and use of the (limited) KAKS resources has proven especially effective on two levels: Projects that are promising but still too small for the large foundations, or projects that cannot wait for the 12-18 month period an approval procedure of a large foundation typically takes. These projects are examined for funding eligibility immediately and then interim financed by KAKS. For example, the project growth inhibitors has now found its way into an extensive DKS application after successful research funded by KAKS. We are applying the same concept for the project mechanisms of the tumour development. Furthermore, the concept of participation of KAKS in large research projects leads to an increase amount in projects funded by the big foundations. Accordingly various projects were co-financed by KAKS as well.
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Early diagnosis for second tumors
– Max-Planck-Institut Potsdam and University Clinic Essen, Germany
Our research project tumor marker for early diagnosis is sponsored by BILD
hilft e.V. with a donation of €224,000. We are using this amount to develop
a simple blood test for early diagnosis of tumors. We are cooperating in
this research project with the University of Essen, and the Max Planck
Institute in Potsdam.
Background: Different GD2 Markers have already been identified for
retinoblastoma cells. Anti GD2 antibodies from the patient blood bind to
synthetic immobilized GD2 – synthesized by the Max Planck Institute. Such
antibodies are produced by the patients as a response to a new tumor.
Furthermore, RNA from the GD2 synthesis can be found in the blood of the
patient. With the data so far obtained we are presently analysing in
parallel approaches which GD2 biomarker can be found in blood, bone marrow
or cerebrospinal fluid (e.g. a specific level of an anti-GD2 antibody) or
RNA (from GD2 synthesis) in the blood of the retinoblastoma patient.
Status: GD2 Synthesis for the immunetest is completed. GD2 spiking control
was successful as well as first RNA Marker tests in blood.
Sponsor: BILD hilft e.V. Ein Herz für Kinder
Analysis of the Diagnosis Pathway
– University Clinic Essen, Germany
This study has been completed. It had the goal to analyse the diagnosis
pathway of children affected by retinoblastoma in order to improve early
diagnosis. The results show that the most frequent first symptoms are
leukokoria and cross-eyedness. First symptoms are mostly recognised by
parents and often not diagnosed or misinterpreted by pediatricians even
though the children frequently visit pediatricians in the first months of
their lives. For the study data of 1049 patients between 1992 and 2011 were
analysed. This study also shows that the delay until diagnosis has not
significantly changed in the last 20 years.
We have various projects that are intended to change this situation. The
White eye campaign tackles the problem by disseminating symptom information
to parents and paediatricians. The smart phone white eye detector app is
aimed at the same goal and presently we are starting an educational program
for all paediatricians.
– Bryan Shaw, Baylor University USA and University Clinic Essen Prof. Kremenz:
This is a co-operation with the originators of the white eye detector App
for testing and distribution.
Furthermore we have initiated an evidence-based study of the white eye
detector App in paediatric wards.
Status: Co-operation with Brian Shaw et al. started and study plan
Retinoblastoma-Inhibitors in Tumor Cell-lines
– University Clinic Essen, Germany – Dr. Temming
Aim of this project is testing promising tumor inhibitors SYK, PLK1 and BET
Bromodomain inhibitors in various retinoblastoma cell lines. In addition the
inhibitors are be tested in osteosarcoma cell lines as an example of a
common second tumor of Rb patients. The data generated are being used for
further development of clinical candidates.
Status: First positive results are available and are being used for further
Prevention of radiotherapy induced secondary tumours in retinoblastoma animal models
– Dr. Sylvie Julien, Prof. Dr. Ulrich Schraermeyer, Prof. Dr. H. Peter Rodemann – University Clinic Tübingen, Germany
First stage: Development of a new Animal model
Second stage: Testing the radioprotective effect of p-Tyrosin in the animal
Status: Stage I successfully completed. First positive results in stage II
Sponsor: Deutsche KinderkrebsStiftung and KinderAugenKrebsStiftung
Innovative treatment options in transgenic and orthotopic animal models
– University Clinic Essen, Germany – PD Dr. rer. nat. Alexander Schramm
Chemotherapy with only one active ingredient is not promising since the
RB-tumors build up resistance quickly. Chemotherapy with multiple active
ingredients can be applied to reduce tumor size in order to then apply
laser- thermal- or cryo-therapy. However chemotherapy has significant side
effects. The study aims at testing various chemotherapeutics in cell culture
and animal models and identifying the mechanisms of resistance. In addition,
the substance beta-Lapachon from the bark of the lapacho tree is tested
Sponsor: Deutsche Kinderkrebsstiftung and KinderAugenKrebsStiftung
Late effects study
– Prof. Bornfeld, Prof. Lohmann
The KinderAugenKrebsStiftung is co-sponsor of a DKS study that intends to
optimize long-term support and improvement of long-term prognosis and life
quality of RB-cancer survivors. The long-term effects of different therapies
were identified with respect to the second tumor risk including individual
genetic disposition. In addition, patients and parents were taught
preventative measures. In the first phase the children and adolescent’s
collective was 570 patients]
Sponsor: Deutsche Kinderkrebsstiftung and KinderAugenKrebsStiftung
Bedeutung von Selen-Methionin für die Induktion der DNA-Schadensreparatur
– Prof. Dr. H. Peter Rodemann, Dr. Sylvie Julien, Prof. Dr. Ulrich Schraermeyer – Universitätsklinikum Tübingen
Bei einer Nachbeobachtungszeit von 50 Jahren ist die Radiotherapie mit einem relativ hohen Risiko für durch die Strahlenbehandlung induzierte Sekundärtumoren verbunden (Draf et al. 2010). Diese haben i.d.R. ihren Ursprung in den während der Strahlentherapie induzierten nicht lethalen Mutationen und Entwicklung genetischer Instabilitäten in Normalgewebezellen, die nach einer entsprechenden Latenzzeit und zu einer Tumorentwicklung führen können. In dem Projekt wird überprüft, inwiefern durch die Verabreichung von Selen-Methionin (SeMet) vor der Strahlenbehandlung die Akkumulation residueller DNA-Schäden, die Ausgangspunkt für eine genetische Instabilität und nachfolgende Tumorentwicklung sein können, durch eine verbesserte DNA Schadensreparatur in Normalgewebezellen von Retinoblastom-Patienten reduziert bzw. unterbunden werden kann. Hierdurch können mechanistische Hinweise erhalten werden, die es ermöglichen, eine chemopräventive Strategie mittels Selen-Methionin zu entwickeln, durch die die Entstehung von Radiotherapie-induzierten Sekundärtumoren bei Retinoblastom- Patienten potenziell unterbunden kann. Somit besteht auf Basis des Projektes die Möglichkeit, auf der Beantwortung von Klinik-relevanten Grundlagenfragestellungen eine zeitnahe klinische Umsetzung zu erreichen.
gefördert von der KAKS und der Kinderkrebsstiftung
Establishing a cell culture model for differentiation of mural retina from
– UK Essen, Germany – Dr. Laura Steenpass, Dr. Petra Temming, Dr. Deniz Kanber
In retinoblastoma cells both copies of the RB1 gene are in activated. This
however is not sufficient to transform the cells into tumor cells. Thus far
it is unclear which molecular mechanisms contribute to tumor occurrence.
In order to find out more about these mechanisms and the primary
retinoblastoma cell, a model with stem cells is to be developed in order to
make available the initial retinoblastoma tissue for further testing. The
effects of different mutations on the development of the retina will then be
analysed to understand the cancer better.