MRFF Funding Promotes Treatment Development for Patients with Rare Diseases

 

When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash University grant recipients with $2.44 million. The funding aims to meet unmet needs in patients with vasculitis, epilepsy, NAFLD/NASH, and muscle-wasting diseases.

MRFF Funding and Recipients

According to the Australian Government Department of Health, the Medical Research Future Fund (MRFF):

will support Australian researchers to make game-changing discoveries, develop a global biotech industry and enable the implementation of changes in healthcare.

Currently, the government pledged to donate $150 million over the next 10 years. The mission will specifically focus on research on treatments utilizing stem cells.

Vasculitis

The first grant given to Monash University staff was given to Dr. Kim O’Sullivan, Dr. Poh Yi Gan, Dr. Dragana Odobasic, and Professor Stephen Holdsworth. Through their research, the team will examine whether human amniotic epithelial cells (hAECs) could treat patients with vasculitis. In particular, the team is interested in understanding vasculitis caused by an immune reaction to myeloperoxidase.

Vasculitis refers to blood vessel inflammation, which can restrict blood flow, cause tissue and organ damage, or cause a dangerous, even fatal aneurysm. Researchers hope that hAECs would be safer and better tolerated than current treatments involving immunosuppressants. While hAECs are also immunosuppressive, they are also anti-microbial, safe, easy to collect, and relatively accessible to patients.

To determine the efficacy of hAECs in treating vasculitis, researchers will test hAECs on patient cells and mice models. Learn more about vasculitis here.

NAFLD/NASH

The MRFF granted funding to Associate Professor Rebecca Lim for the study of nonalcoholic fatty liver disease (NAFLD) and a more severe condition called nonalcoholic steatohepatitis (NASH).

NAFLD and NASH are characterized by the buildup of fat in livers of non-drinkers or people who don’t drink a lot. The condition, which leads to inflammation, can cause cirrhosis and liver damage. According to Monash University, about 25% of organ transplants in Australia result from liver disease. Learn more about NASH here.

In this case, Associate Professor Lim and her team seek to create a therapeutic option from hAECs. They release extracellular vesicles (EVs) which can prevent or inhibit fibrosis (scarring). Additionally, preliminary evidence shows that EVs may also prompt liver regeneration and promote better function.

By potentially creating more potent EVs, researchers hope to develop and test EVs as a treatment in mice models of NASH.

Epilepsy

For epilepsy treatments, the MRFF granted funding to Professor Patrick Kwan. Epilepsy, a disorder in which abnormal brain cell activity causes seizures, affects over 70 million people across the world. However, over 30% of patients have drug-resistant epilepsy. In his study, Professor Kwan will grow stem cells from patients with drug-resistant epilepsy, and healthy patients, in vitro (isolated / in glass) into neurons. Next, his team will determine any potential new drug therapies that could improve patient outcomes.

Muscular Disorders

For research on muscular dystrophies and myopathies, the MRFF offered funding to Professor Peter Currie, Professor Laurence Meagher, and Associate Professor Milkael Martino.

Costs associated with these conditions are estimated to be upwards of $435 million per year, and there are little to no treatments. Additionally, muscle wasting conditions such as sarcopenia or cachexia are emerging health crises for elderly citizens. However, researchers believe that stem cells could offer the chance for muscle regeneration.

In their study, this team will study how to regenerate, rebuild, and repair muscle stem cells in vivo. They will develop stem cell therapies using a cytokine called NAMPT, which will hopefully allow cell regeneration. Researchers also believe this will assist patients needing muscle grafts or similar procedures.


Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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