One of the reasons we never lose our hope, working in this industry where disease is one of our main topics is the astounding amount of medical advancements we learn about every single day. It’s true that clinical research is improving major disease prognosis by the hour – new breakthroughs happen almost every month to the point where it’s hard to keep track of all of them.

However, none of this means much to you or your loved ones if information can’t make its way to you, if it’s wrapped up in incomprehensible medical jargon or if it feels like those new advancements will never reach you.

We know how disheartening it can feel but we don’t want you to lose hope. For this reason, we at FindMeCure love updating you on what’s new and coming for the treatment of some major diseases previously misunderstood or under-researched.

Today on the blog we’re talking about some of the recent advancements for myasthenia gravis – both in terms of treatment and in terms of better understanding the disease itself. Keep reading to find out more about the most promising new therapies.     

Thymectomy might actually benefit people with MG. Thymectomy or the removal of the thymus gland – an organ where important for the immune system T-cells mature – was previously primarily performed when tumors were found in the thymus. However, a 2016 research study finally came up with more than anecdotal evidence that suggests MG patients with no thymomas can also benefit from the procedure.

AChR-positive MG patients were randomly assigned to two groups – one on prednisone alone, the second taking prednisone and undergoing thymectomy. The study wanted to evaluate the patients QMG score – a result which shows just how much MG affects the muscles – and their need for prednisone (a steroid used to decrease inflammation) over the course of 3 years.

Patients in the thymectomy arm of the trial not only did better on the QMG score, meaning they had better muscle tonus compared to the prednisone-only group but they also needed lower prednisone doses.

Lower prednisone doses may be great news for people who experience its side effects, as mild as they can generally be. And thymectomy in adulthood is generally safe and well tolerated since the thymus is most significant in childhood and adolescence.

Soliris approved for the treatment of refractory gMG. For the 10-15% of people with generalized Myasthenia Gravis who cannot tolerate or do not respond to most of the available MG treatments this is great news.

Soliris is currently targeting patients anti-AchR antibody-positive MG. What this means is that Soliris is a complement inhibitor – it interrupts the process of antibodies binding to important receptors that are supposed to carry out communication between the nerve cells and the muscles they control. Thus, Soliris prevents the immune system from attacking the AchR receptors with antibodies.

However, Soliris is also linked to life-threatening meningococcal infections, so meningococcal vaccination is recommended at least two weeks prior to the beginning of treatment. Children treated with Soliris are also at a higher risk for serious infections and the same goes for immunocompromised patients.

Zilucoplan goes to phase III clinical trial. This one is as recent as last month, for patients with gMG zilucoplan is showing enough promise to go to the next stage of clinical trials in late 2019. The main goal of phase III, which is expected to enroll about 130 participants, is to evaluate the patients’ ability to engage in daily activities as a result of the therapeutic effect of the drug.

Zilucoplan works much the same way drugs like Soliris does – it binds to a protein that plays a crucial role in the immune response, though to be part of the process of autoantibodies formation. The drug is known as complement inhibition because it aims at stopping the overreaction of the complement system.

The second phase of the trial zilucoplan demonstrates not only its safety and tolerability but it also proves it’s effective enough to warrant further testing. During the 12 weeks study patients who took the drug showed better QMG scores compared to the placebo control group and no serious adverse events as the result if zilucoplan were reported.

New research suggests that the gut microbiome is less diverse in people with MG. The gut microbiome has been continuously linked to the immune system with some evidence that seems to suggest it can actually help regulate it. While the link between the gut microbiome and other autoimmune diseases such as IBD has been established, new research is widening our perspective beyond inflammation of the digestive tract.

Compared to the control group, MG patients had a significantly less diverse microbiome. With reduced protective bacteria and abundance of the proinflammatory kind found in people with IBD, MG patients were at a disadvantage. These results serve as further evidence for the link between certain gut bacteria and autoimmunity.

Additional investigation into short-chain fatty acids found out that SCFAs linked to a kind of T-cells which help maintain the immune system in order are reduced in people with MG. Such findings are important because a deeper understanding of the link between the gut microbiota and the immune system can inspire new therapeutic approaches – just like it already has in the treatment of IBD.

Could microbiota replacement be on the horizon for MG patients? It’s too early to tell but either way, the more we understand all the factors at play, the better chance we have at treating it.

Another promising drug that is emerging for the treatment of MG is Rituxan. In a clinical research study it not only improved muscle strength but it also delayed disease worsening. The drug is targeting patients with refractory MG and according to the research results it allows those patients to lower their steroid doses.

Like many other drugs for MG rituxan interferes with a part of the immune response responsible for inflammation. By lowering the levels of B-cells, a type of white blood cells, rituxan can slow the progression of MG and reduce the need for other medications.

Patients positive for MuSK (muscle-specific tyrosine kinase) showed the greatest improvement in muscle strength with 89% reduction in their manual muscle testing score. Not only that but patients also needed lower doses of prednisone and the time between relapses was significantly extended.

With very few exceptions, all patients then recovered their B-cells count over a period of 12 months. Nevertheless, close monitoring of B-cells is strongly advised, considering the high risk of developing infections linked to prolonged B-cells depletion.  

If you want to be among the first to try out new promising therapies, receive innovative treatments and extensive monitoring and care, check out FindMeCure for clinical trials looking to enroll participants. Or, if you’re feeling less adventurous but you want to help further medical research and a better understanding of MG, you can participate in one of the other, non-invasive studies trying to identify biomarkers that make early diagnosis easier. You can become part of scientific progress by donating your time to research.

Leave a Reply

Your email address will not be published. Required fields are marked *

You may use these HTML tags and attributes: <a href="" title=""> <abbr title=""> <acronym title=""> <b> <blockquote cite=""> <cite> <code> <del datetime=""> <em> <i> <q cite=""> <s> <strike> <strong>