Our hope

Batten’s disease currently has no cure. However, scientific advances & promising new research give us a glimmer of hope.

This consists of therapies and experimental stage treatments which require a significant financial investment and therefore our main objective is to raise funds through our association Hodeilargi.

An experimental genetic therapy in the USA.

Science has advanced regarding the development of genetic therapies focussing on genetic disorders such as Batten’s an other similar diseases.

In the case of Batten CLN6 disease that affects both Hodei and Ilargi, there is a line of research in gene therapy with potential to slow the course of the disease, developed in the United States by Amicus Therapeutics together with the Research Institute of Nationwide Children’s Hospital.

At Nationwide Children’s Hospital in Columbus, Ohio, a clinical trial for this gene therapy was conducted with 13 patients from around the world.

This gene therapy program for Batten CLN6 from Amicus Therapeutics has now been stopped after analyzing the long-term results and concluding that the stabilization that was initially observed has not been maintained over time. A review seems necessary to continue with this line of research which, however, seems promising.

With this gene therapy, an attempt is made, through inoculation in a single dose, to transport a gene to the patient’s brain through an adenovirus that has been modified to eliminate the ability to infect. In this way, it is expected to replace the affected CLN6 gene that causes Batten disease with a correct copy.

Other lines of complimentary research: Molecular therapy.
New treatments are being investigated which could prove effective in the treatment of Batten’s, starting off with existing treatments and borrowing others from the treatment of other illnesses.

Given that these composites are mostly safe for use on humans, the possibility exists that they could be soon used in this clinical trial. This creates an interesting new perspective in the search for a cure and treatments for Battens, but which require a significant investment to achieve desired results.

More Information: Compounds Similar to Flupirtine Seem Promising for Treatment of Batten Disease

This would be one of the lines of investigation which, together with other organisations and affected families, we would like to be able to finance to provide treatments to compliment genetic therapy.

The Charlotte and Gwenyth Gray Foundation is promoting some of these initiatives.

Learn more: Scientist Wins Grant to Study CLN6 Batten, Advance Early Trials – Batten Disease News

Currently in Europe, a consortium is being formed that aims to continue with some studies on Batten disease, including the participation of a team from the University of Salamanca.

More information: Investigador de la USAL avanza en identificación de nuevos mecanismos neurodegenerativos en la enfermedad de Batten (europapress.es)

The future: cellular therapy.

Science is advancing in regenerative treatments through the transplanting of stem cells, which could help not only in the fight against Batten’s but also for other similar neurodegenerative illnesses.
This research is extremely promising, although they imply massive costs & are currently less well-developed.

However, these treatments are a real possibility for the future, and we must endeavor to highlight these rare illnesses and contribute as much as possible to successful research and treatment in the near future.

With your help, we are closer to achieving this goal.