A peek into a septic shock clinical trial and a patient’s message of hope (TMM2016-Day2)

Author: Hamideh Emrani, 10/27/16

The second day of the Till & McCulloch Meetings (TMM) kicked off with a great talk by Dr. Masayo Takahashi of the Riken Center for Developmental Biology, on the generation of retinal progeny and photoreceptors from iPSCs and ESCs. However, for today, I would like to focus on two different talks. I am sure further posts will cover the other great presentations.

The next talk in the morning was delivered by Dr. Lauralyn McIntyre, Ottawa Hospital, on the use of mesenchymal stromal cells (MSC) in a phase I clinical trial for the treatment of septic shock. During my masters study I worked with MSCs and we used them for applications in treatment of tendon and ligament injuries. Thus, I have a personal bias towards MSC studies. (I should add that I am fully aware of the valid concerns around the homogeneity of MSC populations, and lack of well-defined purification methods, but I digress and this would need a separate panel for discussion.)

Septic shock is the most severe form of infection seen in Intensive Care Units with the high prevalence rate of 50,000 patients a year being affected in Canada. The problem is not the infection, but how the body responds to it. It usually involves multiple organ failure and high morbidity rates (20 to 40 percent). Septic shock is a complicated syndrome that is expensive to treat, and even when the patient survives it there are long term emotional, physical, mental health and social problems involved, according to Dr. McIntyre.

Because of the complex nature of the septic shock pathophysiology and the lack of complete understanding of the stages involved, over 100 phase II and III clinical trials have been conducted that have failed at the end. So there is a need for novel approaches in treatment strategies.

Thus, Dr. McIntyre collaborated with Dr. Duncan Stewart who had successfully used MSCs in animal models for sepsis. MSCs are a great candidate for such an intervention since they can localize to the area of injury, have limited differentiation potential compared to ESCs – thus lowering tumorigenicity – show low engraftments and, most importantly, have immunomodulatory properties.

Stewart’s team reported decreased mortality, lower levels of systemic inflammatory mediators, higher bacterial clearance and enhanced phagocytic ability of the immune cells. The main goal of using MSCs was to take advantage of their ability to distract the immune system for a while so that other cell types of the body, such as regenerating cells, will have a chance to repair the damage to the organs.

The phase I clinical trial “Cellular Immunotherapy for Septic Shock (CISS; pronounced like kiss)” was conducted using human MSCs. Unfortunately, the details and data of this trial have not been published yet. At this stage I am authorized to tell you that the Phase I trial found that MSCs appear safe to use in septic shock treatment. You will have to wait for the data to be published to know more. I am getting used to teasers for TMM2016 posts!

A highlight of today’s presentations was delivered by Tina Ceroni, who bravely shared her story of hardship when diagnosed with a non treatable disease, and her journey to recovery after receiving a stem cell treatment.

Tina was a healthy 28 year-old personal trainer who all of a sudden experienced rigidity in her muscles. Initially she thought her troubles were a result of her training. However, over a course of two years she became progressively worse and was finally diagnosed with Stiff Person Syndrome (SPS), which is a rare neurological disorder (one in every million). SPS is an auto-immune disease, meaning that the body’s own immune system attacks neuromuscular cells. Tina was having progressively more severe attacks and ended up in the hospital 47 times over the course of one year.

Accidentally, she found another person who had the same disease and had received the world’s first stem cell transplant to treat it. Tina was subsequently introduced to Dr. Harry Atkins, Ottawa Hospital, who had performed the first stem cell transplant for SPS treatment and he chose Tina to be patient number two. (Dr. Atkins is also involved in similar clinical trials for the treatment of other auto-immune conditions such as Multiple Sclerosis.)

Tina was transplanted with stem cells isolated from her own bone marrow. In order to encourage stem cell growth in her marrow, she was subjected to aggressive chemotherapy, medications and injections. Her stem cells were isolated and then purified in vitro. She received another aggressive regimen of chemotherapy to completely eliminate her immune system. Then she received the stem cell transplant and her immune system started to regenerate itself, and she has been symptom free since two years after the transplant. As she put it, the day that she received her stem cell transplant it was as if she was born again.

Tina told her story and described her immense appreciation for the stem cell research that led to the exploration of this technique. She was especially thankful for the support she received from Dr. Atkins and his team during her treatment. She was also thankful for having the opportunity to share her message of hope through TMM2016.

She also advocates for more inclusion of patients in the process of research and clinical trials. When patients are involved they can actively collaborate with their caregivers, clinicians and researchers in developing new protocols and raising funds for the research to bring about positive change and perhaps eliminate a disease. She received a well deserved standing ovation from the audience. Tina is actively raising funds for stem cell research and the stem cell and bone marrow transplant centre at the Ottawa Hospital through her foundation “Share a Cell.”

I chose to write about Tina’s talk because I think it is a great reminder for us all of the big picture and the importance of stem cell research. We are all working to ultimately treat debilitating and life threatening diseases and provide patients a second chance at a normal life.

 

 

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Hamideh Emrani

Hamideh Emrani

Hamideh is a scientific communicator and the founder of Emrani Communications, serving clients in Toronto (University of Toronto) and California (Stanford University). She earned her B.Sc. in Cell and Molecular Biology at UC Berkeley and finished her M.Sc. at the University of Toronto (U of T). She was an intern at the Carnegie Institute at Stanford University, honours research student at UC Berkeley and has won awards for best podium and best poster presentations at the Faculty of Dentistry and IBBME at U of T. She is passionate about science and loves to talk and write about it. You can follow Hamideh on Twitter at @HamidehEmrani.
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