This blog post summarises the key insights from the latest Virtual Education Session (VES) led by Dr Hugo Stephenson, an Australian doctor, specialist in medical research, and founder of several biotechnology service companies, including MediGuard and Health Research Solutions. He is also an active advocate of drug safety research and consumer health awareness.
This article includes a brief summary of Scleroderma, a research update, and support resources for patients and families.
For more free resources, access our in-depth and targeted information brochures here.
Scleroderma Overview
As a doctor, and someone in the medical community, Stephenson acknowledged how people often see Scleroderma in a very medical context. There are some genetic factors that influence whether we develop Scleroderma, including specific coding within our DNA that increases our chances of developing it. This development starts with damage to blood vessels, then results in fibrosis, but there are still gaps in understanding as to why this is the case. What medics do know, however, is that even with the exposure factors that trigger Scleroderma development, not everyone who is genetically predisposed to Scleroderma will end up developing it.
This is only the physiological understanding, as Stephenson put it, of Scleroderma, based on the medical research of what is happening internally. However, Scleroderma doesn’t just impact our cells, it has a significant impact in many areas of our lives, including social, professional, and personal aspects. This is why it’s so important to view Scleroderma, and research it, holistically.
Scleroderma Research Update
Luckily, research today is factoring in these aspects, potentially providing better management measures that better cater to our day-to-day goals and benefit the loved ones around us, as well as ourselves.
Epidemiological research is investigating associations between Scleroderma and other conditions, deepening our understanding of its risk factors and if people with Scleroderma are more prone to developing other health conditions. When referring to this research, Stephenson noted that researchers are looking at developing AI and other things to help fill in the gaps of what is still unknown about Scleroderma, stating it is “all very exciting and optimistic”, and researchers know the importance of prioritising Scleroderma research because it’s still so unknown and underrepresented in the medical and research fields.
Cell transplants research, which involves transplanting stem cells into people with Scleroderma to see if it can reverse some of the fibrotic damage, and carty therapy, which Stephenson described as a “super targeted anti-inflammatory therapy” using white blood cells, are also being explored for Scleroderma management at the moment.
Drug discovery and testing is also another form of research which is highly prevalent in the field, with Stephenson saying researchers are using basic research to help identify and test potential drugs through several stages of research.
Preclinical research is being done in vitro, to see what drugs have an impact on our cells. Stephenson used the Scleroderma-based example of taking fibrocytes, which generate fibrous tissue, and adding different drugs to the petri dish they’re tested in to see what impact they can have on altering how the fibrous tissue gets laid down.
Preclinical in vivo research is also being done, which is studies done in living animals. These are completed to see if what has been observed in the petri dish translates into a living creature.
Then there is a three-phase process for human studies once a drug has worked in the first two tests. This tests if the drug is safe to be used by humans (Phase 1), if it is effective in some way (Phase 2), and then if the drug can get approved (Phase 3).
In some cases, there are also phase four studies that test the outcomes of approved drugs when they are used in different ways.
Stephenson also highlighted the options of non-medical interventions and how they’re attempting to be furthered through more research. There’s much that we could get from using medicines and drugs, but for those under different conditions, with access to a carer, for example, non-medical interventions might be more effective in increasing quality of life.
Limitations researchers are finding, however, are in a lack of participants with Scleroderma for these clinical studies.
One survey recently conducted by Stephenson found that out of the 34 people surveyed, roughly 35% had participated in one or more Scleroderma studies, and 10% had been in an observational study where they’re completing a series of surveys over time. However, only 2 of the 24 people had been in a clinical trial to test for new medicine, even though 97% said they’d be likely or very likely to participate in a clinical trial if invited.
Having direct patient engagement in this research and trial stage of medical testing is crucial in ensuring both collective and individual experiences are factored into research gaps and patterns. Stephenson also highlighted the significance of patient involvement in shaping research priorities and encouraged us to express our interest to healthcare providers to help further our desired research outcomes. This patient engagement can start by individuals tracking symptoms and patterns, as these observations can inform research questions and study designs.
Males specifically are significantly underrepresented in Scleroderma research, so Stephenson reinforced the importance for males with Scleroderma to engage in communities and research being conducted, rather than isolate themselves as some research shows they do. All-male support groups have recently become a priority to try and provide men with support that is more beneficial for them.
Finally, there seems to be a huge opportunity for collaboration between Australia and New Zealand when it comes to Scleroderma research and progress. The two countries have collaborated on research in other health areas, such as with brain cancer, so Stephenson believes a similar team-up could be possible for Scleroderma research. This will be especially significant for the people living with Scleroderma in New Zealand, as there is a severe underrepresentation of Scleroderma studies there.
Patient and Family Support
The last of Stephenson’s session was focused on how crucial it is to utilise support groups as someone with, or around someone with, Scleroderma. The number of known Scleroderma cases in Australia is 6,000, but there are less than 500 registered members in support groups, so it is crucial to help raise awareness of these resources and reach out to others with Scleroderma so they know they’re not alone.
We all know living with Scleroderma presents unique challenges, but being part of a supportive community can provide invaluable resources, understanding, and encouragement. By sharing experiences, insights, and resources, we can empower ourselves, others, and medical researchers to better navigate the journey of living with this autoimmune condition.
For those living with scleroderma, staying updated and informed can make a world of difference. Connecting with others can also be hugely beneficial. Find out more about support in your area here.
Our National Education Sessions or Virtual Education Sessions are offered at no cost. Virtual Education Sessions happen every month via Google Meet.
During these sessions, we’ve invited medical professionals and experienced legal experts to address frequent questions about Scleroderma and other topics related to it.
If you’d like to gain firsthand knowledge, our National Education Sessions and Virtual Education Sessions are available to you at no charge. Our Virtual Education Sessions are held every month through Google Meet.
These sessions provide an opportunity to engage with medical professionals and seasoned legal experts who will address common inquiries about scleroderma and related topics.