Genetic Researcher Professor Ernst Wolvetang and his team at UQ’s Australian Institute for Bioengineering and Nanotechnology (AIBN) have secured almost $1 million from the Medical Research Future Fund (MRFF) to continue testing gene therapies with “mini-brain” organoids for children with Hereditary Spastic Paraplegia Type 56 (SPG56).
SPG56 is an ultra-rare neurodegenerative disease that normally begins in childhood and continuously worsens throughout life. Commonly, children with SPG56 lose the ability to sit, stand, walk or talk, and this is what happened to four-year-old Australian Tallulah Moon Whitrod, whose family started a campaign in 2021 to find a cure for this currently incurable disease.
The grant comes after Prof Wolvetang has already dedicated 18 months to the collaborative SPG56 gene therapy project Our Moon’s Mission initiated by Tallulah’s parents Golden and Chris Whitrod who lead the foundation Genetic Cures for Kids (GC4K).
Golden Whitrod said that the MRFF meant this vital research benefiting Our Moon’s Mission could continue within a time that still held hope for her daughter and other children living with SPG56.
“We are absolutely thrilled with the announcement of this MRFF and it’s high time the importance of this organoid research being led by Prof Wolvetang and his team at the AIBN is recognised for the life-saving potential it has,” Golden said.
Chris Whitrod said Prof Wolvetang’s brain organoid approach, using tiny, synthetic organs grown from a patient’s own cells gave his family and foundation the greatest hope for a treatment that could work for Tallulah.
“This incredible approach to personalised medicine gives researchers hundreds of brain organoids with the same genetic make-up as my daughter’s own brain, and with these SPG56 diseased “mini-brains” they can safely test the best gene therapies and develop a cure without ever risking her health,” Chris said.
Professor Wolvetang said the MRFF grant would help them continue this vital research, and the SPG56 project could serve as a pilot to develop a replicable framework to treat other rare diseases, of which there are over 7000.
“The AIBN is aiming to lead the nation in the field of personalised medicine and this testing will help us create a pathway to faster, more accurate treatments for children with SPG56 and other forms of HSP,” Professor Wolvetang said.
“Once we demonstrate the power and accuracy of this approach for one genetic disease, it could open the door for testing of other gene therapies for a range of genetic neurological conditions.”
The Whitrods, who aim to see their SPG56 gene therapy research project translate to the clinic in Australia in 2024, said they hoped the organoid research they are using would become more accessible and accepted by regulatory bodies to help other parent-led research programs in the future.
“The regulatory approval process still relies too heavily on controversial, expensive and time-consuming animal testing,” Golden said.
“It’s a challenging and expensive road for parents trying to save their kids from a rare disease, and when organoids can be developed this rapidly and tested extensively with only a drop of a blood, this makes rare disease cheaper, faster and more accurate- something the 300 million living without rare disease treatments need desperately”, Golden said.
Professor Wolvetang’s MRFF-funded project will focus on pre-clinical testing of the safety and efficacy of adeno-associated virus (AAV)-based gene therapy for HSP-SPG56 patients, with the hope of accelerating the time to clinical trial approval.
“This is a project that is founded on a genuine collaboration between parents, patient advocacy groups, researchers and the wider community, and we anticipate that its success will further promote community support for stem cell models,” Professor Wolvetang said.
