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Centenary Institute - Medical Research
Centenary Institute - Medical Research

New treatment discovery for vascular disorder

Researchers from the Centenary Institute have discovered a potential new therapy for cerebral cavernous malformations (CCMs), a devastating disease that often affects young people and can result in stroke and seizures.

Using mouse models, the researchers found that use of the drug, CD5-2 helped normalise the vascular disorders, and inhibited the development and reduced the size of existing lesions.

The study was published in the highly respected international science journal PLOS Biology.

“CCMs are vascular lesions comprising clusters of abnormally thin and leaky blood vessels. Stroke or seizures can occur when blood from these vessels leaks into the surrounding brain tissue. We can’t predict when this will happen or how frequently,” said senior author on the research paper Professor Jennifer Gamble, Head of the Vascular Biology Program at the Centenary Institute.

“Most often, people don’t realise they have the disease until they have an event such as a seizure. Currently, there is little in the way of effective medical treatment for CCMs except for surgical removal of the most dangerous lesions which is limited by the size and depth of the lesions,” she said.

In the study, Professor Gamble and collaborators were able to show that a protein called VE-cadherin, critical to maintaining a healthy blood vessel lining, was seen at lower levels in mice with CCM lesions.

“We then used the drug CD5-2 to elevate VE-cadherin levels, resulting in a reduction in the lesions that were present in our CCM mice,” said Professor Gamble.

The drug CD5-2, developed by the Centenary Institute and collaborators was initially designed to combat the development of solid cancers by reducing and repairing damage caused by inflammation in blood vessels. It has since been found to have other potential benefits including as a treatment to prevent sight-loss in people with diabetes.

“Our research journey with CD5-2 is exciting,” says Professor Gamble. “CD5-2 is a drug that improves leaky blood vessels and this is a feature of many chronic diseases. We have now shown that CD5-2 is a potential novel therapy for CCMs, a disease with surgery as the only option, and that not always possible, especially if patients have multiple lesions. This discovery could lead to the first effective, non-invasive treatment option for CCMs which would be truly heartening for sufferers.”

Professor Gamble believes that CD5-2 may yield even further health benefits with research into the drug and other disease areas still ongoing.

Read the full media release here.

Access the publication here.

Dr Jaesung Peter Choi awarded prize in Washington DC

Photo: Dr Jaesung Peter Choi receives the award from Dr Brent Derry.

Centenary Institute researcher Dr Jaesung Peter Choi has been awarded “The FEBS Journal Poster Prize” at the 14th Annual Angioma Alliance CCM Scientific Meeting, held in Washington DC. The prize recognises the best poster presentation by a trainee.

Dr Choi was able to attend the meeting after receiving the Australian Vascular Biology Society (AVBS) Travel Award.

He was joined by Head of Centenary’s Cell Signalling Laboratory, Dr Xiangjian (XJ) Zheng.

The pair presented their most recent study, published in the scientific journal Science Advances, which identified a cancer drug as a potential treatment option for Cerebral Cavernous Malformations (CCM) – a leading cause of stroke in young people.

Read more about their recent breakthrough.

Stroke of genius: drug could target leading cause in young

A study led by researchers at the Centenary Institute has identified a drug currently used to treat cancer patients, as a potential treatment option for a leading cause of stroke in young people.

Cerebral Cavernous Malformations (CCM) occurs when abnormal and dilated thin-walled blood vessels form clusters in the brain; altering blood flow. The condition affects as many as 1-in-200 people, and can cause bleeding, epilepsy and stroke.

Currently, the only treatment for CCM is surgery, which is not always possible; highlighting the urgent need for non-invasive, pharmacological treatment options.

In a study published in highly-regarded scientific journal Science Advances, researchers from the Centenary Institute in Sydney and several other institutions in Australia and China, have identified the FDA-approved drug Ponatinib as a suitable candidate.

Lead author and Centenary Institute researcher, Dr Jaesung Peter Choi says it’s a significant step in the quest to find a suitable treatment for this debilitating disease.

“Our next goal is to synthesise derivatives of Ponatinib for specific use in CCM to maximise its efficacy, and to minimise any side effects,” says Dr Choi.

Read the full media release.

Dr Jaesung Peter Choi and Dr Xiangjian Zheng explain their research and the implications of their findings.

Metaflammation

Metaflammation is a new term that refers to low levels of inflammation throughout the body.

Your body use the essential repair process of inflammation to heal the body. When you have a full blown infection your levels are very high. Metaflammation is different. It is much much lower but constant.

This new aspect of inflammation has come to light, its connection with chronic metabolic diseases, like obesity and diabetes. One of the reasons this link is really important is that we are able to control with small molecule or biological therapeutics some aspects of inflammation. This approach might become really useful in treating some diseases that were thought to be diet or environment influenced and may become a devastating problem in the future.

Inflammation research: why it matters

Diabetes

A third of all diabetics will suffer from vision loss and potential blindness during their lifetime. This condition is caused by inflammation in the retina blood vessels.

Centenary’s Impact 

We have developed an exciting new drug which has the potential to mend the damaged blood vessels and prevent loss of sight.

Read more about our diabetes work here.

Centenary Institute scientists are working on many aspects of inflammation, including metaflammation. For more details on our research approaches see here.