Research projects underway

CHECT funded retinoblastoma research

These projects are currently being funded by CHECT. For past research please have a look at our previous retinoblastoma research page.

Targeted Antibody-Drug Conjugates for Retinoblastoma

Project leader Dr Gail Seigel
Award £25,000
Duration One year, Sept 17


The overall goal of this project is to improve upon current therapy for retinoblastoma by targeting toxic molecules (ADCs) directly to the retinoblastoma tumour. The ADCs are designed to kill retinoblastoma tumour cells, but spare surrounding normal cells. In turn, this will result in a more efficient eradication of the RB tumour with fewer side effects for the patient. Our experiments are designed to test the effectiveness of ADCs through the study of retinoblastoma tumour cells in test tubes, as well as in actual tumours. By the end of this project, we will know whether ADCs designed for retinoblastoma have potential to move toward clinical trials and human patients.

Status: In progress

Eloise Patterson Project

Project leader Dr Zerrin Onadim
Award £24,800
Duration One year, Nov 15


Records held at the Royal London Hospital will be studied alongside those from the Childhood Cancer Research Group to investigate the way in which risk of tumours occurring in later life depends on different genetic mutations associated with heritable Rb, on the treatment (radiotherapy, chemotherapy) used in treating Rb, and to calculate statistical estimates of these risks.

It is hoped this will lead to clinicians and geneticists having better information available to them when assessing the risks of second tumours occurring. This could potentially lead to earlier diagnosis and treatment of these second cancers.

Status: In progress

Support the development of a national system to collect and audit data on children diagnosed with retinoblastoma within the national cancer registration service.

Project leader Dr Helen Jenkinson
Award £8,841.36
Duration Six months, Sept 2015


The national RB register will replace existing in-house databases used at Birmingham Children’s Hospital and the Royal London Hospital, thus providing a single national resource for understanding and addressing concerns such as:

  • the effectiveness of treatment plans; through comparison between treatment centres
  • the impact and efficacy of specialised commissioning
  • the link between the heritable form and individual genotype on the risks of disease progression and secondary cancers in later life

It will provide a template for the creation of other site-specific registers for other rare childhood cancers, such as Wilms disease and neuroblastoma.

Status: In progress

Modelling Retinoblastoma using human induced pluripotent stem cells (iPSC)

Project leader Dr Sandy Hung
Award £34,315
Duration 18 months, June 2015


It has been a challenge to develop a good cell model system for retinoblastoma (Rb), despite it being the first cancer to have its gene identified. Model systems are important tools which enable scientists to better understand the diseases and develop more effective treatments. However, current models for RB (e.g. mouse), cannot accurately reproduce what is observed in RB patients due to differences in disease mechanisms between species.

This project proposes to make induced pluripotent stem cells (iPSCs) from skin cells of Rb survivors with known heritable Rb mutations, to make different cell types, including the retinal (eye) cells that become cancerous in Rb. Ultimately, this model system could enable researchers to test potential treatments. A successful human Rb model would also, in the future, enable researchers to gain insight into the development of second cancers commonly seen in Rb survivors.

Status: In progress

Autonomic reflexes and cardio-respiratory instability during super-selective intra-arterial chemotherapy in the management of children with Rb.

Project Leader: Dr S P McGuirk et al. at Great Ormond Street Hospital and Birmingham Children’s Hospital.
Award: £18,000
Duration: One year from May 2014.


Previous studies have reported that intra-arterial chemotherapy is a safe and effective treatment in children with advanced disease. It causes complete regression of the tumour and preservation of vision in most cases. However, we have observed that a significant proportion of children developed severe adverse cardio-respiratory responses during the procedure. These reactions have occurred only during the second catheterisation procedure and are characterised by the sudden development of acute bronchospasm, impaired ventilation and marked haemodynamic instability.

Patients have been resuscitated with intravenous fluids, intravenous atropine and intramuscular epinephrine with rapid restoration of normal cardio-respiratory function. None of the children had any adverse sequelae following these events.

We do not currently understand why these responses occur, why they occur in some children but not others, or why they are only clinically evident during the second catheterisation procedure. We believe that they represent an autonomic reflex response, which is qualitatively similar to the trigemino-cardiac and trigemino-respiratory reflexes observed during corrective surgery for strabismus, cranial and maxillofacial surgery, and skull base surgery.

We propose to undertake this prospective case-controlled study to examine the pathophysiological events in detail. In particular, this study will determine the incidence of the trigemino-cardiac and trigemino-respiratory reflexes and quantitatively measure the effect of these reflex responses on haemodynamic and respiratory function. Ultimately, this information may allow us to identify adverse responses before they become clinically apparent, thus improving the safety of this procedure. The findings of this study are, therefore, of potentially immediate clinical application.

Status: In progress. See the following article in Pediatric Anesthesia.

Understanding and overcoming barriers to living with an artificial eye

Project leaders: Tara Shea and Laura Rouse
Funding awarded: £14,339
Duration: One year from April 2014


Understanding the experiences of children and their families following enucleation will help to develop some clear best practice guidelines regarding clinical management of this population. We hope to promote innovation in the development of clinical resources to support adaptation to living with an artificial eye. By better understanding the scope of the barriers (physical, mental and emotional) that may be encountered the research team can explore the support services and strategies used by children and their families in order to address these barriers.

In order to understand the experiences of children and their families following enucleation the study will involve conducting interviews with three groups: children over five years of age who have experienced a unilateral enucleation; parents of children who have experienced enucleation; and teachers of children who have experienced enucleation.

Status: In progress