New Foundation collaboration

Genetic Cures for Kids

The Foundation is collaborating with Genetic Cures for Kids Inc. (GC4K) a new charity that is funding research to find a cure for SPG56 HSP.

What started out less than 12 months ago as an inquiry from a family in the HSP community about one of their children recently diagnosed with this type of HSP, has led to the family establishing the Genetic Cures for Kids foundation and funding research to find a cure.

GC4K, a registered charity, is pursuing two streams of research. The first is studying human HSP stem cells with the purpose of characterising the cellular impairment, then identifying drug candidates to effectively treat the condition. Alan Mackay-Sim is providing guidance and input on this program. The second is a gene therapy research stream for which there are early promising indications, but a long road ahead is expected before establishing a successful genetic cure not just for SPG56, but also for the vast majority of diseases where the quest for a gene therapy cure is in progress.

As with any research, there is no guarantee of success. The Foundation is fortunate to have this opportunity to develop understanding, knowledge and competency on gene therapy development for HSP without cost. As this is a significant emerging potential therapy, there are substantial benefits to the HSP community foreseeable through our involvement and collaboration with GC4K.

The Foundation committee carefully deliberated the nature and extent of our relationship and involvement with the new GC4K entity, including the issues of potential conflict of interest and adherence to our core value of providing equitable support across the HSP community. It was decided that the Foundation would neither receive funding from, nor give funding to GC4K. We would support GC4K with knowledge and information.

The HSP Research Foundation is neither giving to nor receiving funds from GC4K. We are providing information and guidance on researchers, clinicians, resources and regulators to help the family navigate and accelerate the steep learning curve involved to get the charity going.

The strategic opportunity for the HSP Research Foundation is to have a front row seat for the development of gene therapies for the HSPs without having to invest in this emerging technology. It gives us direct connection with leading researchers on gene therapy, helping us develop knowledge, expertise and competency for making good decisions for the future regarding the development of gene therapies, with potential benefits for the whole HSP community.

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