\u201cIt may be one of the first dementias to have a definitive treatment, a cure if you like, a really transformative treatment that allows people to live much longer and much more normal lives,\u201d says Prof James Rowe, a consultant neurologist at Cambridge\u2019s Addenbrooke\u2019s hospital.<\/p>\n
FTD mainly affects the front and sides of the brain and, unlike Alzheimer\u2019s disease, does not begin with memory loss, which tends to occur later. It is characterised by progressive loss of language and changes in personality and behaviour.<\/p>\n
Lead scientist Andrea seen filtering solution inside the labs of gene-therapy company AviadoBio. Photograph: Alicia Canter\/The Guardian<\/p>\n
Most cases are diagnosed in people aged 45 to 65, but it can affect people in their 20s and 30s. There are an estimated 20,000 to 40,000 people living with FTD in the UK, and between 1m and 2m in the world.<\/p>\n
Rowe says: \u201cIt\u2019s a double-edged sword: the young onset, the high genetic burden and rapid illness are also features that perhaps make it more tractable to treat.\u201d<\/p>\n
The Die Hard and Pulp Fiction actor Bruce Willis, who recently celebrated his 70th birthday, was diagnosed with FTD two years ago, with his family calling it a \u201ccruel disease\u201d. They have not said whether he has a genetic form of FTD.<\/p>\n
The gene therapy developed by AviadoBio, which was spun out of Prof Christopher Shaw\u2019s research lab at King\u2019s College London in 2021, targets a type of FTD known as FTD-GRN. This is caused by mutations of a gene that lead to a deficiency of progranulin (GRN), a protein that is essential for maintaining healthy brain cells.<\/p>\n
AviadoBio, which employs 60 people, signed an exclusive licence agreement with the Japanese pharma firm Astellas<\/a> last October to develop the therapy. It is now recruiting patients for its clinical trial in the UK, as well as the US, Poland, Spain, Sweden and the Netherlands.<\/p>\n The first patient received the infusion in Warsaw in March 2024, out of six patients who have had the treatment so far, in Poland and the US. All patients will be followed for up to five years as part of the trial. AviadoBio expects to publish the first data next year.<\/p>\n Julia and Jessica Crawford. Photograph: supplied<\/p>\n Three years ago, Jessica Crawford, from Beverley in Yorkshire, lost her mother to FTD, caused by mutations of another gene, C9orf72. In 2014, when her mother was 58, her behaviour changed; the family suspected depression. Previously very sociable, she stopped going out and started playing games like Candy Crush or watching TV shows \u201cover and over,\u201d her daughter recounts.<\/p>\n Her mother initially did not want to see a doctor, and was only diagnosed with FTD in February 2019. By this time she was so confused she once put raw chicken in a sandwich. \u201cGetting the diagnosis wasn\u2019t easy because FTD wasn\u2019t well known; my mum was aceing in the memory tests,\u201d Crawford says. But her mother became increasingly confused and lost the ability to speak, and to communicate at all. Crawford became her full-time carer in 2020, until her mother deteriorated so much that she had to go to a care home in late 2021, and died the following year.<\/p>\n Crawford, 33, found out that she herself carries the gene mutation, and with her husband decided to conceive through IVF with a pre-implantation genetic diagnosis. When their five embryos were screened, four had the gene mutation, and the fifth resulted in the birth of their son. The couple donated their other four embryos to science and she takes part in GENFI \u2013 a long-running UK-led global study of families with FTD across 40 sites.<\/p>\n AviadoBio itself was born out of the research done at King\u2019s by Shaw, a neurologist who has focused on FTD and amyotrophic lateral sclerosis (ALS) for more than three decades, and Dr Youn Bok Lee and Dr Do Young Lee from the UK Dementia<\/a> Research Institute\u2019s centre based at King\u2019s. ALS, the most common form of motor neurone disease, has also been linked to mutations of the GRN gene and leads to muscle weakness, paralysis and eventually death.<\/p>\n skip past newsletter promotion<\/a><\/p>\n Sign up to Business Today<\/p>\n Get set for the working day \u2013 we’ll point you to all the business news and analysis you need every morning<\/p>\n Privacy Notice: <\/strong>Newsletters may contain info about charities, online ads, and content funded by outside parties. For more information see our Privacy Policy<\/a>. We use Google reCaptcha to protect our website and the Google Privacy Policy<\/a> and Terms of Service<\/a> apply.<\/p>\n after newsletter promotion<\/p>\n Shaw acts as the company\u2019s chief scientific and clinical adviser, and while the Lees are no longer involved in the firm\u2019s research, all three remain shareholders.<\/p>\n Dr David Cooper with Robin the skull, seen with placements for the cannula during surgery. Photograph: Alicia Canter\/The Guardian<\/p>\n David Cooper, AviadoBio\u2019s chief medical officer, says: \u201cIt was something that hit me when I first joined the company, you look at the MRI scans of patients with a GRN mutation, the frontal and temporal parts of their brain are just melting away \u2026 So we really need earlier treatment, an, earlier diagnosis and a more organised healthcare approach to deal with it.\u201d<\/p>\n AviadoBio\u2019s lead product, known as AVB-101, is infused directly into the brain by a neurosurgeon using a cannula as thin as a strand of angel hair pasta, during a 90-minute procedure guided by MRI. It delivers a functional copy of the progranulin gene to restore appropriate levels of the protein to affected areas of the brain. It is a once-only treatment, and no immunosuppressant drugs are needed subsequently.<\/p>\n \u201cThe patients who have FTD are born with almost half of the progranulin levels that you and I might have,\u201d says chief executive Lisa Deschamps. \u201cOur goal in the study is to supplement the GRN gene and restore as much progranulin in these individuals as possible to normal levels to reduce the neurodegeneration effect.<\/p>\n Other medications in development at AviadoBio include two gene therapies, from Philadelphia-based Passage Bio and Eli Lilly-owned Prevail Therapeutics, but they do not target the thalamus, the \u201crelay station\u201d in the brain. Passage Bio\u2019s therapies are delivered directly to the cerebrospinal fluid in a single treatment. Denmark\u2019s Vesper Bio has developed an oral capsule<\/a>, designed to act on the GRN gene, that is being trialled at University College London Hospital.<\/p>\n![\"Catheter](\"https:\/\/www.europesays.com\/uk\/wp-content\/uploads\/2025\/06\/330.jpg\")
Catheter placement on a patient. Photograph: AviadoBio<\/p>\n