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news, breaking news, Nikita Hunter, Isla Hunter, Batten disease, CLN1, BDSRA Australia, Taysha Gene Therapies, gene therapy, clinical trial

A BALLARAT couple have made an emotional appeal for their daughter to be part of a groundbreaking search halfway around the world. Nikita and Harley Hunter received a heartbreaking blow in April this year when they were told that the youngest of their three children – Isla, two years old – suffered from a rare and incurable neurodegenerative disease known as Batten. But the experimental gene therapy being developed in the United States offers a silver lining for the Hunter family. Texas-based Taysha Gene Therapies is currently seeking FDA approval for an investigative application for a new drug that would allow them to conduct a clinical trial of gene therapy for children with CLN1 – the form of Batten’s disease from which Isla suffers. RELATED Gene therapy for CLN1 disease is a one-time treatment in which a healthy copy of the defective gene is delivered to the brain inside a viral “transporter”. The healthy copy of the gene leads to the production of the deficient key brain enzyme, thereby slowing or preventing further brain degeneration. “To think that Isla could be a part of something like this, which has the potential to not only help her but other children with CLN1 as well, is amazing,” said Nikita. “We would love Isla to have the opportunity to participate in a clinical trial. We would go anywhere. It would be a game changer for our family.” The Batten Disease Support and Research Association Australia is a strong advocate for Isla and another young Victorian girl with CLN1, whose family contacted them after reading Isla’s story. BDSRA Australia Director of Research, Medical and Scientific Affairs Dr Ineka Whiteman said that while the trial was not yet open to patient recruitment, she was in regular contact with Taysha. “We don’t just sit around and wait for them to approach us to see if the girls would like to participate in the trial, we just don’t think that will happen. We have to be proactive,” she said. “I regularly reach out to them as well as the members of the Advisory Board (BDSRA Australia) here to keep the conversation going and to make sure the girls stay on Taysha’s radar.” But Dr Whiteman said time was running out. “Time is neurons. The more a patient progresses, the more neurons that have been lost usually cannot be regenerated. Treatment will only stop the disease or slow its progression from the time therapy begins. “she said. “It’s a race against time. Dr Whiteman said two Australian children with another form of Batten disease had traveled abroad in the past to participate in different clinical trials. But acceptance into the trials was not easy, with many variables such as the number of places available, the level of disease progression and other selection criteria, the exclusion criteria and the location of the patient. site, all potentially affecting girls’ eligibility. However, the prospect of gene therapy was “a real hope” for families like the Hunters. “It’s exciting and it gave them more hope than the families before them had when they were diagnosed with CLN1 disease,” said Dr Whiteman. “For BDSRA Australia this would be a wonderful outcome for the families we represent.” As the anxious wait for the family continues, Nikita said she has been overwhelmed by the community’s response to Isla’s story. “I’ve had people I don’t know kiss me, cry with me, people I wouldn’t expect to have reached out to both Harley and me to send their love, greetings and thoughts.” , she said. “We have had donations and fundraisers on behalf of our beautiful daughter, so from the bottom of our hearts, thank you to everyone who made a difference and showed us what the love and spirit of community. ”It’s easy to lose faith in a situation. like ours, but we will never give up the fight to give Isla all she deserves. Thank you to everyone who supported us along the way, none of this has gone unnoticed. “- A GoFundMe page has been set up to help the Hunter family. To learn more or to donate click here.


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