What eye treatments are in sight? Ask the experts
University student and runner Billy Morton is leading the charge to raise $150,000 in a single day for research into gene therapies to protect and restore vision. He has a rare genetic disease that is causing his eyesight to deteriorate.
A new gene therapy being developed by Melbourne scientist Dr Raymond Wong aims to treat eye diseases like this by regenerating lost light-sensing cells in the eye.
We’re partnering with the Centre for Eye Research Australia on World Sight Day to raise funds to bring his ideas closer to clinical trial. Triple your impact by making a donation on Thursday 14 October. More below.
Raymond will join a panel of ophthalmologists and scientists to share the latest eye research and answer audience questions through an online forum event on the eve of World Sight Day. Questions like: what’s next for treating glaucoma, age-related macular degeneration, and inherited retinal diseases? Join the forum and ask the experts. Read on for details.
The winners of our 2021 Metcalf Prizes for Stem Cell Research will be announced next month. They’re named for the late pioneering stem cell scientist Professor Don Metcalf, whose work transformed cancer treatment. Read on for a patient’s perspective on how his discoveries are saving lives.
In other news,
- US scientists observing mouse hair follicles have discovered an unexpected mechanism of ageing,
- stem cell-derived mini organs are continuing to assist COVID-19 research
- biotech start-up companies are working to overcome the challenges of mass-producing reprogrammed stem cells in the quantities needed for clinical use.
These and more in our regular round up of stem cell news from around the world.
Dr Graeme L Blackman AO
Chairman, National Stem Cell Foundation of Australia
In this bulletin:
- Support gene therapy research focused on the eye’s light receptors
- What are the latest developments for treating eye diseases? Ask your questions at a free online public forum
- How Don Metcalf changed cancer treatment
- Stem cell news from around the world
Support gene therapy research focused on the eye’s light receptors
Triple the impact of donations given on 14 October - World Sight Day
Melbourne university student and long-distance runner Billy Morton, 22, was first diagnosed in his early teens with a rare genetic disease that is causing his eyesight to deteriorate. At the time there was no prospect of a treatment, but progress in gene therapy research is providing new hope.
Now Billy is leading the charge to raise $150,000 in a single day for research into gene therapies to protect and restore vision. He is the face of the Centre for Eye Research Australia’s (CERA) World Sight Day campaign, which aims to raise funds to develop and trial pioneering gene therapies in Australia.
Every dollar donated on this day will be matched by donations from the National Stem Cell Foundation of Australia and the CERA Foundation, tripling the impact of your donation.
You can make a donation online at: charidy.com/HopeinSight.
Funds raised will support the research of Dr Raymond Wong and his team who are developing a new gene therapy to regenerate photoreceptor cells in patients like Billy.
Billy, with the rare genetic disease choroideremia, is one of 190 million people worldwide with diseases causing the death of light-sensing photoreceptor cells in the eye.
“Photoreceptors are tiny cells in the retina at the back of our eye, which pick up light and send the signal from the retina to the brain, enabling us to see,’’ explains Raymond.
“Currently there is no cure for blindness once photoreceptors are lost but my research is aiming to change that.’’
Billy says that gene therapy research makes him hopeful and optimistic that it will make a difference.
“Hopefully there will be research that will benefit me, but if not, it is exciting to know that it could make a difference to other people,” he said.
“Raymond and his colleagues at CERA have an outstanding scientific track record” said National Stem Cell Foundation of Australia General Manager Graeme Mehegan. “It’s a pleasure to partner with them to give real hope for people with vision loss on this World Sight Day.”
What are the latest developments for treating eye diseases? Ask your questions at a free online public forum
Online event: Hope in Sight online forum
When: 7:00 pm – 8:00 pm (AEDT), Wednesday 13 October
More than 13 million Australians have one or more chronic eye conditions, according to government data. Of these, 131,000 people have partial or complete blindness.
If you or a loved one are among these, you likely have questions.
Can stem cells treat blindness? What is gene therapy? Can progressive eye diseases like glaucoma or age-related macular degeneration be prevents, halted, or even reversed?
Send in your questions, and join an online event to hear the answers from a panel of experts from the Centre for Eye Research Australia.
- Dr Carla Abbott is a clinician-scientist and expert in structure and function of the retina and optic nerve. Her research areas include age-related macular degeneration (AMD), inherited retinal diseases (such as retinitis pigmentosa (RP)) and glaucoma.
- Associate Professor Lauren Ayton is a clinician-scientist with research interests in retinal disease, low vision and vision restoration
- Dr Tom Edwards is a vitreoretinal surgeon and scientist. His research looks at the potential of gene therapy to cure inherited retinal diseases, aiming to develop treatments that may halt or partially reverse some inherited causes of blindness.
- Professor Robyn Guymer AM is a clinician scientist who leads a team of researchers primarily investigating age-related macular degeneration (AMD), a condition that affects one in seven Australians over the age of 50 years.
- Professor Keith Martin is a clinician scientist ophthalmologist. His research is focused on developing new strategies to protect and regenerate the optic nerve in glaucoma, the leading cause of irreversible blindness worldwide. He was first in the world to demonstrate gene therapy and stem cell therapy could reduce retinal ganglion cell death in an experimental model of glaucoma.
- Dr Raymond Wong is a stem cell biologist. His research focuses on understanding the genetic signals that define retinal cells, and using cell reprogramming and stem cell technologies to study and treat retinal diseases.
- Associate Professor Peter van Wijngaarden is an ophthalmologist and medical scientist with research interests in diabetic retinopathy, Alzheimer’s disease and retinal imaging biomarker discovery.
Register via www.cera.org.au/tribe-events/hope-in-sight-online-forum/ and email questions to [email protected].
How Don Metcalf changed cancer treatment
The story of a journalist, his brother, and the man who helped 20 million cancer patients
Transplant day: Chris Kimball (left) pictured with his brother and stem cell donor Sean (centre) and with his donated stem cells. (Photo: Chris Kimball)
In 2011, journalist Chris Kimball noticed he was losing weight, along with other odd symptoms. The 35-year-old father of two young children found out he had an unusual and aggressive form of non-Hodgkins lymphoma, a cancer of the lymphatic system. And it was severe. It had spread to multiple lymph node sites, other organs and his bone marrow.
Within a week, Kimball had started treatment. He would need an intensive, brutal regime of chemotherapy followed by a bone marrow transplant to rebuild his immune system from scratch using donor stem cells from his brother.
Kimball’s road to recovery involved pain, fear, uncertainty, and several weeks in an isolation ward to protect him from infections.
“I don’t think you can under-estimate how full-on that process is,” he says.
“When you spike a fever and you have no immune system, it’s scary. It is the worst moment of your life and you’re completely isolated, waiting for that moment when you can start having some protection.”
Gruelling as Kimball’s treatment was, it would have been even tougher without a glycoprotein called granulocyte-colony stimulating factor (G-CSF). G-CSF stimulates the bone marrow to produce stem cells, white blood cells and other blood cells, and releases them into the bloodstream.
Colony stimulating factors—there are four different types—were discovered in the early 1980s by the pioneering Australian stem cell scientist Professor Donald (‘Don’) Metcalf, who pursued cancer research at Walter and Eliza Hall Institute (WEHI) in Melbourne. The Foundation’s Metcalf Prize for Stem Cell Research is named in his honour.
Stem cell news from around the world
Between newsletters, we share stem cell news on social media:
Here are a few stories we’ve shared recently:
New York Times: Losing Your Hair? You Might Blame the Great Stem Cell Escape. Paper.
Nature: The next frontier for human embryo research
The Conversation: New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many
New Scientist: The stem cell revolution isn't what you think it is
Nature: Stem-cell start-ups seek to crack the mass-production problem
Science Daily: Lab grown tumor models could improve treatment for pancreatic cancer.
EurekAlert: Repeated injury to airway stem cells could be major factor in chronic lung disease. Paper.
Caltech: Researchers build embryo-like structures from human stem cells. Paper.
Science Daily: When organoids meet coronaviruses
Massive Science: Precise CRISPR gene editing can correct the mutation that causes cystic fibrosis in mini-intestines. Paper.
About the Foundation
The National Stem Cell Foundation of Australia is an ATO-registered tax-deductible Health Promotion Charity dedicated to promoting the study and responsible use of stem cells to reduce the burden of disease.
The Foundation’s activities include:
- supporting research that pursues cures for as-yet-untreatable diseases
- building a community of people with a shared interest in stem cell science
- providing the Australian public with objective, reliable information on both the potential and risks of stem cell medicine.
We are working to build a community of people with a stake in stem cell science and to promote collaboration between scientists locally and internationally.
Please feel free to contact the Foundation’s General Manager Graeme Mehegan via [email protected].
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