Networking strategies to fight a fatal children disease

Aims: The NCL Foundation connects researchers, clinicians and companies to develop therapies and ultimately, cure the children disease called Neuronal Ceroid Lipofuscinosis (NCL). This lysosomal storage disorder leads to blindness, dementia, epilepsy and loss of motor functions and finally to a premature death.

Methods: After the start of the NCL Foundation in 2002 we analyzed the existing NCL research landscape to discover research gaps. We performed intensive literature reviews, actively participated in scientific conferences, and met with individual investigators in their laboratories. These activities pointed out to research avenues that still need to be strengthened and in some cases, started. As a result, investigators in clinical practice, academia and the pharmaceutical industry have been recruited into the field of NCL research. Today, the foundation continues to recruit key experts and often brings them together helping to lay the groudwork for exchanging new ideas, creating new collaborations and initiating multidisciplinary activity.

Results: Altogether 35 new collaborations have been initiated since the founding of the NCL Foundation in 2002. Predominantly these collaborative movements are between university laboratories (66%). The remaining teamworks are between a university groups and a research institutes (17%) or commercial companies (17%). By now 9 national NCL conferences and one international PhD symposium have been organized by the foundation.

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 sourcees and are staying in the field of NCL research. The NCL Foundation initiates collaboration between researchers, clinicians and companies making substantial contributions to the field of NCL research through personal networking. Our first basic research projects have reached the pre-clinical phase. The next step will be therapy.

Neuronal Ceroid Lipofuscinosis - Batten disease - lysosomal storage disorder