Gene therapy developed by Lincoln scientists receives US FDA approval for in-human clinical trial

A Lincoln research team has received US FDA approval for in-human clinical trials of their gene therapy for the treatment of CLN5 Batten disease, a fatal neurodegenerative childhood disease.

The CLN5 form of Batten disease appears early in a child’s life and causes brain degeneration manifesting in devastating symptoms including vision loss, seizures, dementia, abnormal movements and inability to communicate. Sufferers typically die in their teens.

Until now there has been no cure and no hope of treatment, but the Lincoln-developed gene therapy is a potentially transformative treatment for the CLN5 patient community.

Over the past decade, Professor David Palmer and Doctors Nadia Mitchell and Samantha Murray have been developing their gene therapy in sheep with a naturally-occurring form of the disease. Continue reading

Lincoln researchers receive grants to develop treatments for Batten disease

Two Lincoln University researchers, Dr Nadia Mitchell and Dr Samantha Murray, have been awarded three grants totalling more than $477,000 for research into the understanding and treatment of neuronal ceroid lipofuscinosis, or Batten disease.

There are 13 forms of Batten disease, a family of fatal neurodegenerative diseases primarily affecting children.  Each is caused by the lack of a particular gene and there is no cure.

The disease, often appearing early in a child’s life, causes brain degeneration manifesting in devastating symptoms including vision loss, seizures, dementia, abnormal movements and inability to communicate. Sufferers typically die in their teens.

The research grants have been awarded by Batten Disease Support and Research Association (BDSRA) Australia, Cure Kids New Zealand and Neurogene Inc (USA).

Drs Mitchell and Murray are developing a gene therapy that appears to slow and even halt the progress of the disease in sheep with a naturally occurring form of the disease.

Dr Mitchell explains:

“The brains of sheep with Batten disease shrink, as do the brains of humans with the condition. When we replace the missing gene in affected sheep before they display symptoms, in most cases the disease can be prevented. When we replace the gene after the sheep begin to display symptoms, the therapy slows the progress of the disease.

“The implications of our research are hugely significant, and offer considerable hope to the families of Batten disease sufferers.”

Dr Mitchell’s team is working with an American pharmaceutical company, Neurogene Inc, to gain US  Food and Drug Administration approval to begin the first human trial of the gene therapy for a common form of Batten disease.  If the trial is successful it is hoping similar treatments and delivery routes can be tested in patients with other forms of the disease.

Patients are currently being recruited for the trial from around the world.

While Neurogene Inc is the principal sponsor, Cure Kids New Zealand funds Dr Murray’s project grant to better characterise the retinal disease in affected sheep. Technical support for both projects will be enabled by the awarding of the BDSRA grant.

BDSRA spokesperson Dr Ineka Whiteman said the organisation was pleased to award their grant to Dr Mitchell and her team at Lincoln University.

“The research being undertaken by Dr Mitchell and her team is truly world-class, and will help provide the essential data required for translating animal model research into human clinical studies.”

Source:  Lincoln University