Collaboration on One of the Leading Causes of Blindness Seeks to Find More Effective Treatments

Macular degeneration (AMD or ARMD)- is a medical condition which may result in blurred or no vision in the center of the visual field. Tunatura/Shutterstock

The Australian National University (ANU) has begun a collaboration with the Korean Biotech Company MDimune Inc. to develop new and more effective treatments for Age-related Macular Degeneration (AMD).

AMD is one of the leading causes of blindness globally and is expected to affect 288 million people in 2040.

Assoc. Prof. Riccardo Natoli, Head of the ANU Clear Vision Research Lab and his team, which includes Adrian Cioanca PhD, and Yvette Wooff PhD, are working with MDimune to find AMD treatments.

Yvette Wooff said that, at the moment, there are no treatments available for the more prevalent form of AMD, they dry/geographic AMD, which affects around 90 percent of all AMD cases.

“For the wet form of the disease, there is one available treatment form which involves regular expensive injections of anti-VEGFa drugs into the eye,” Wooff said.

“Advancements in drug development for dry-AMD largely lie in the complex and multifaceted nature of this disease, which can be caused or attributed to genetic, lifestyle and environmental risk factors.”

“Therefore, our goal is to develop therapeutics which can overcome these hurdles and eventually allow for targeted delivery to the retina,” she said.

U.S. scientists have identified two genes responsible for macular degeneration, the gradual deterioration of eyesight in the elderly that can lead to blindness. The research published in April 2007, in the Journal of the American Medical Association also showed that smoking and being overweight carries a strong risk of the condition in which the central part of the eye's retina degenerates. KAREN BLEIER/AFP/Getty Images

Treatment Using Cell-to-Cell Communication

ANU scientists believe that the molecular messages inside certain cells could serve as a therapeutic for not only AMD, but also other neurodegenerative diseases like Parkinson’s and Alzheimer’s.

Wooff said that all cells release extracellular vesicles as a form of cell-to-cell communication.

“These vesicles carry and transfer genetic messages such as RNA, DNA and microRNA between other cells in the same tissue as well as systemically through the body,” she said.

“As these vesicles under normal conditions function to relay messages of health, we aim to harness this ’molecular message of health' as a potential therapeutic.

“In retinal degenerations, we know that the molecular message of retinal health can be dysregulated or even lost.”

Consequently, Wooff said that they aim to re-supplement this molecular message and restore healthy communication pathways in the retina to slow the progression of degeneration by using extracellular vesicles derived from other cells that have naturally similar cargo, or by loading the essential healthy molecular cargo into vesicles from other cells.

The Role of MDimune

Wooff said that the essential healthy cargo could be introduced to the vesicles of other cells through a few different methods in the lab, which are fairly achievable.

“Simply, we can manufacture the molecular sequences missing or required and encapsulate them within vesicles from any cell type,” Wooff said.

“Think of vesicles as envelopes and the cargo as the mail inside. We have the mail, we have a few envelopes, but we need the stamp and address to really get it to the right house (cell).”

MDimune possesses cutting-edge BioDroneTM platform technology, which is a complex system that uses cell-derived vesicles (CDVs) to convey various drugs to specific parts of the body.

“Working with MDimune we have identified some great envelopes and will be looking to develop the stamp and address in the near future,” Wooff said.

She said that the ANU’s collaboration with MDimune has identified candidate vesicles that are really exciting and promising therapeutics because the vesicles have not only the potential to load therapeutic cargo but also naturally encapsulate important protective cargo that’s also found in retinal extracellular vesicles.

An employee of German technology company Bosch demonstrates the use of a Bosch eye care solution to check up the retina of an eye with a digital camera at the company's headquarters in Gerlingen near Stuttgart, southwestern Germany, on April 29, 2015. THOMAS KIENZLE/AFP/Getty Images

Direct Delivery of Medicine to the Retina

Associate Prof. Natoli said in an ANU news release that the partnership with MDimune advances the development of new drug therapies that could eventually cure AMD, which is a debilitating disease that causes loss of vision in an estimated 200 million people globally.

“This novel class of drug carriers and therapeutics are highly versatile and can be generated from various types of human cells, meaning they can be produced in large quantities,” Natoli said.

“We are excited at the possibility of the MDimune-developed BioDrone platform technology for use as a therapeutic and drug delivery for the eye.”

Wooff said that in many cases, vesicles would travel systemically and can target some or all cell types through specific ligand-receptor interactions.

However, Wooff said that their goal is to develop targeted therapeutics by modifying the surface of the vesicles and target to the retina so that the medicine can be delivered locally and systemically. She said that achieving this will prevent the need for invasive ophthalmic injections.

“We are thrilled to launch this collaboration to develop AMD therapeutics with the world-class research group at ANU,” said the Chief Scientific Officer at MDimune, Dr Seung Wook Oh.

“It will be a great opportunity for us to confirm the anti-inflammatory and regenerative capacity of stem cell-derived CDVs as therapeutics.

“Also, through our collaboration with ANU, we anticipate that we can facilitate the commercialisation of the BioDrone platform with global pharmaceutical companies.”