Longevity Briefs: Bioviva Announces Results from Human Dementia Gene Therapy Trial

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Longevity briefs provides a short summary of novel research in biology, medicine, or biotechnology that caught the attention of our researchers in Oxford, due to its potential to improve our health, wellbeing, and longevity.

Why is this research important: In a recent youtube video released last week, the results of Longevity-focused biotech Bioviva’s recent clinical trial looking into potential gene therapy treatments for dementia, were announced.

In this short video, Dr. Patrick Sewell, director of clinical affairs at Integrated Health System, a company partnered with Bioviva, gives us the low-down on the Human Dementia Study, which was trialling two types of gene therapy to help combat this age-related neurodegenerative disease.

What did the researchers do: Using viral vectors, the scientists introduced two types of gene therapy into 5 patients with mild to moderate cognitive impairment; human Telomerase Reverse Transcriptase (hTERT) and Klotho gene transfer therapy.

What do these do?

hTERT helps to maintain and even repairs parts of the chromosome called telomeres. Telomeres are short sequences of non-coding DNA which protect the chromosome from damage, and whose shortening have been implicated with accelerated ageing.
The Klotho gene produces Klotho proteins. These reduce the build up of beta-amyloid plaques in the brain, sticky protein aggregations which impede brain cell function and which are a hallmark of dementia.

Together, the scientists hoped that these therapies would help combat the progression of cognitive impairment most people will experience at some point in their life.

Key takeaway(s) from this research: The revealed a number of extremely promising results:

Median telomere length lengthened in 4 of the patients, whilst maintaining the same length in the fifth
The shortest telomeres (those closest to triggering senescence) showed significant elongation in all 5 patients
Biological age was significantly reduced in 4 of the patients
Cognitive testing demonstrated significant improvement in all 5 patients

As well as showing these exciting results the therapies were well tolerated, with brain MRI indicating no obvious structural alterations, or negative patient experiences.

Overall these results are a very promising first step along the path of developing gene therapies for age-related diseases, However, it must be kept in mind that this study only included 5 individuals. Follow-up studies involving more patients will provide more substantial evidence on whether these gene therapies have the potential to alleviate the burden of neurodegenerative disease on society, and individuals.