Precision Medicine Approaches to Scleroderma Treatment
One of the hallmarks of Scleroderma is that every patient presents differently. In this episode, John Varga, MD, and Chase Correia, MD, share how they are developing personalized approaches for patients through research and multidisciplinary care. The foundation of their research is developing the tools and technologies to identify each patient's molecular and genetic markers in order to provide targeted precision medicine approaches to treatment. In the clinical arena, Northwestern Medicine Scleroderma Program has a comprehensive management team including specialists in rheumatology, pulmonology, cardiovascular and gastroenterology working together to provide the best care to each patient.
Featured Speakers:
Learn more about Chase Correia, MD
Chase Correia, MD | John Varga, MD
Chase Correia, MD is a Rheumatologist and co-Director of the Northwestern Medicine Scleroderma Program.Learn more about Chase Correia, MD
John Varga, MD is a Rheumatologist and Director of the Northwestern Medicine Scleroderma Program.
Learn more about John Varga, MD
Learn more about John Varga, MD
Transcription:
Precision Medicine Approaches to Scleroderma Treatment
Melanie Cole, MS (Host): The Northwestern Medicine scleroderma program is committed to conducting innovative research to increase knowledge of the disease providing advanced individualized patient care at Feinberg affiliated care sites. My guests in this panel discussion today are Dr. John Varga—he’s a rheumatologist and director of the Northwestern Medicine scleroderma program and lead investigator on many clinical trials—and Dr. Chase Correia—he’s a rheumatologist and co-director of the Northwestern Medicine scleroderma program. Gentlemen, thank you for being with us today. Dr. Varga, would you please set the stage for us as it were and tell us a little bit about scleroderma, the incidence and prevalence and what it is.
John Varga MD (Guest): Yes, thank you. It’s a rheumatic disease similar to lupus and other diseases that rheumatologists see. It effects primarily women in their 40s and 50s, and it occurs in about 20 individuals per million, and has a prevalence of about 200 people per million in this country with an estimated prevalence of about 60 to 100,000 people effected in any given time.
Host: Dr. Correia, which age groups have the highest prevalence of it? Is there any way to predict who might be susceptible to it?
Chase Correia, MD (Guest): It is most common in middle ages. So in patients who are in their 40s or 50s, but it can happen in any age including pediatric patients. Unfortunately, there's not a great way to predict who will get the disease. However, those who have other autoimmune diseases in their family are at a slightly higher risk.
Host: Dr. Varga, tell us how it’s diagnosed. Give us a little information on the clinical presentation.
Dr. Varga: Most people present with non-specific symptoms, but those include puffy hands or swollen hands and circulatory problems in the finger that are called Raynaud phenomenon. The problem is that the symptoms are very nonspecific and can often be dismissed or diagnosed as another rheumatic condition. Because of that people often don’t come to a definitive diagnosis for quite a long time, months to even years, which is a missed opportunity where early intervention could make a difference.
Host: That’s so interesting. So Dr. Correia, along those lines, tell us about what Northwestern Medicine scleroderma program is doing to help advance the understanding and treatment of this condition. Are there some current advances that we should know about that can help also with earlier detection?
Dr. Correia: First, the Northwestern scleroderma program is in involved in numerous clinical trials as well as clinical research studies. One of the things that we are particularly interested in is using single cell RNA transcriptomic analysis to better understand the genetic linkages with this disease. We are also engaged in some clinical trials that we are very excited about. Then finally I’d like to mention some recent research advances. One of which did also take place here at Northwestern, which is the census trial which is studying the Nintedanib for systemic sclerosis interstitial lung disease. That trial was just released in May and it showed that the rate of decline of lung function was halved using this new drug Nintedanib, and that’s currently going to the FDA to seek approval.
Host: That’s fascinating. Dr. Varga, do you have some predictors of treatment response based on some of these therapies? What have been your outcomes? What have you seen with your patients?
Dr. Varga: It’s very variable. One of the hallmarks of scleroderma is that every patient is different. So a lot of the emphasis we place in our research program is to develop personalized and predictive kind of medicine. That means developing the tools and technologies to identify each patient and their molecular and genetic markers that would predict number one, are they lucky to have progressive and aggressive disease and bad outcomes or more benign disease? Secondly, to predict which of the many different types of treatments that are now becoming available would be more available. So we’re using some very cutting edge technologies including a lot of studies looking at genetics, genomics, advanced imaging technologies that we’re developing at Northwestern, and very targeted early stage clinical trials where we try a new drug to see how an individual with a particular molecular background would respond to that treatment. So a very personalized and predictive approach.
Host: That certainly seems to be the future of precision medicine. So Dr. Correia, tell us about the multidisciplinary approach. What type of providers are involved in your research and in dealing with patients?
Dr. Correia: That’s one of my favorite things about being at Northwestern is we truly have a comprehensive management team as part of the scleroderma program. So first there’s Dr. Varga and I who are the rheumatologists who tend to be kind of the captain of the ship so to speak. Then because interstitial lung disease is such a prevalent condition in systemic sclerosis, we rely a lot on Dr. Dumati who’s a pulmonologist and also a specialist in systemic sclerosis interstitial lung disease. Then for patients how have pulmonary hypertension, we have Dr. Michael Cudicu who is a pulmonologist who specializes in pulmonary hypertension. For those who have cardiac involvement, we have Sanjeev Shaw in the cardiology department. There’s a lot of gastro involvement in this disease. It can effect the entire gastrointestinal system. So we have Dr. Horrano and Dr. Brenner in the GI department.
Host: Dr. Varga, looking forward to the next 10 years in the field, where do you see it going? What do you feel will be the most important areas of research. Give us a little blueprint.
Dr. Varga: I think what I see happening in the next 10 years is that there’ll be some new drugs discovered, but more importantly we are going to be what’s called repurposing existing drugs. In other words, drugs that are used for other rheumatic disease or even in cancer. We’ll discover that they might be appropriate treatments for certain kinds of patients. Using the genetic and genomic tools that we talked about, we might have a much better ability to predict and identify patients who might be more likely to respond and benefit from some of the existing drugs that we currently have. We will also have much better tools to essentially determine if the patient—when a specific treatment is going a good response where no response or actually is getting worse. Currently those are not done in any kind of a robust matter, but I think the technologies that are coming online would allow us to do that. I think these technological and precision management advances combined with some major progress in understanding the basic biology of what scleroderma is are going to lead to perhaps not a cure, but much better outcomes as well as our ability to predict complications before they occur and hopefully prevent them.
Host: Dr. Correia, first last word to you. What would you like other physicians to know about the scleroderma program at Northwestern Medicine and when you feel it’s important that they refer.
Dr. Correia: To us I think we firmly believe that the earlier that we can see new patients with this disease, the better. The outcomes are better. Our abilities to help them are better and we can head off some of the worst complications of this disease. The other thing is that we are particularly interested in patients with diffuse systemic sclerosis right now and where they welcome and consultations with other physicians with the few systemic scleroses.
Host: Dr. Varga, last word to you. What would you like other providers to know about when they do refer a patient, what they can expect from you and your team and Northwestern Medicine.
Dr. Varga: To echo my colleague, I think it’s critical to be able to get patients with scleroderma or scleroderma like conditions to a referral center really early. Because one damage occurs, it’s much more different generate and repair than [inaudible] the damage. So seeing patients very early, we pursue a very integrated, multi-disciplinary proactive approach to find out what is the specific characteristics of that individuals disease. What are the kind of treatments they might benefit from? We like to work very closely with our colleagues in rheumatology and other specialists throughout the team to provide the in proactive care that’s very personalized to each patient.
Host: Thank you gentlemen so much for coming on and sharing your expertise and explaining the program so very well for us. Thank you, again. That wraps up this episode of Better Edge a Northwestern Medicine podcast for physicians. To refer a patient and for more information on the latest advances in medicine, please visit nm.org to get connected with one of our providers. If you as a provider found this podcast informative, please share with other providers. Share on social media and be sure to check out all the other fascinating podcasts in the Northwestern Medicine library. Until next time, I'm Melanie Cole.
Precision Medicine Approaches to Scleroderma Treatment
Melanie Cole, MS (Host): The Northwestern Medicine scleroderma program is committed to conducting innovative research to increase knowledge of the disease providing advanced individualized patient care at Feinberg affiliated care sites. My guests in this panel discussion today are Dr. John Varga—he’s a rheumatologist and director of the Northwestern Medicine scleroderma program and lead investigator on many clinical trials—and Dr. Chase Correia—he’s a rheumatologist and co-director of the Northwestern Medicine scleroderma program. Gentlemen, thank you for being with us today. Dr. Varga, would you please set the stage for us as it were and tell us a little bit about scleroderma, the incidence and prevalence and what it is.
John Varga MD (Guest): Yes, thank you. It’s a rheumatic disease similar to lupus and other diseases that rheumatologists see. It effects primarily women in their 40s and 50s, and it occurs in about 20 individuals per million, and has a prevalence of about 200 people per million in this country with an estimated prevalence of about 60 to 100,000 people effected in any given time.
Host: Dr. Correia, which age groups have the highest prevalence of it? Is there any way to predict who might be susceptible to it?
Chase Correia, MD (Guest): It is most common in middle ages. So in patients who are in their 40s or 50s, but it can happen in any age including pediatric patients. Unfortunately, there's not a great way to predict who will get the disease. However, those who have other autoimmune diseases in their family are at a slightly higher risk.
Host: Dr. Varga, tell us how it’s diagnosed. Give us a little information on the clinical presentation.
Dr. Varga: Most people present with non-specific symptoms, but those include puffy hands or swollen hands and circulatory problems in the finger that are called Raynaud phenomenon. The problem is that the symptoms are very nonspecific and can often be dismissed or diagnosed as another rheumatic condition. Because of that people often don’t come to a definitive diagnosis for quite a long time, months to even years, which is a missed opportunity where early intervention could make a difference.
Host: That’s so interesting. So Dr. Correia, along those lines, tell us about what Northwestern Medicine scleroderma program is doing to help advance the understanding and treatment of this condition. Are there some current advances that we should know about that can help also with earlier detection?
Dr. Correia: First, the Northwestern scleroderma program is in involved in numerous clinical trials as well as clinical research studies. One of the things that we are particularly interested in is using single cell RNA transcriptomic analysis to better understand the genetic linkages with this disease. We are also engaged in some clinical trials that we are very excited about. Then finally I’d like to mention some recent research advances. One of which did also take place here at Northwestern, which is the census trial which is studying the Nintedanib for systemic sclerosis interstitial lung disease. That trial was just released in May and it showed that the rate of decline of lung function was halved using this new drug Nintedanib, and that’s currently going to the FDA to seek approval.
Host: That’s fascinating. Dr. Varga, do you have some predictors of treatment response based on some of these therapies? What have been your outcomes? What have you seen with your patients?
Dr. Varga: It’s very variable. One of the hallmarks of scleroderma is that every patient is different. So a lot of the emphasis we place in our research program is to develop personalized and predictive kind of medicine. That means developing the tools and technologies to identify each patient and their molecular and genetic markers that would predict number one, are they lucky to have progressive and aggressive disease and bad outcomes or more benign disease? Secondly, to predict which of the many different types of treatments that are now becoming available would be more available. So we’re using some very cutting edge technologies including a lot of studies looking at genetics, genomics, advanced imaging technologies that we’re developing at Northwestern, and very targeted early stage clinical trials where we try a new drug to see how an individual with a particular molecular background would respond to that treatment. So a very personalized and predictive approach.
Host: That certainly seems to be the future of precision medicine. So Dr. Correia, tell us about the multidisciplinary approach. What type of providers are involved in your research and in dealing with patients?
Dr. Correia: That’s one of my favorite things about being at Northwestern is we truly have a comprehensive management team as part of the scleroderma program. So first there’s Dr. Varga and I who are the rheumatologists who tend to be kind of the captain of the ship so to speak. Then because interstitial lung disease is such a prevalent condition in systemic sclerosis, we rely a lot on Dr. Dumati who’s a pulmonologist and also a specialist in systemic sclerosis interstitial lung disease. Then for patients how have pulmonary hypertension, we have Dr. Michael Cudicu who is a pulmonologist who specializes in pulmonary hypertension. For those who have cardiac involvement, we have Sanjeev Shaw in the cardiology department. There’s a lot of gastro involvement in this disease. It can effect the entire gastrointestinal system. So we have Dr. Horrano and Dr. Brenner in the GI department.
Host: Dr. Varga, looking forward to the next 10 years in the field, where do you see it going? What do you feel will be the most important areas of research. Give us a little blueprint.
Dr. Varga: I think what I see happening in the next 10 years is that there’ll be some new drugs discovered, but more importantly we are going to be what’s called repurposing existing drugs. In other words, drugs that are used for other rheumatic disease or even in cancer. We’ll discover that they might be appropriate treatments for certain kinds of patients. Using the genetic and genomic tools that we talked about, we might have a much better ability to predict and identify patients who might be more likely to respond and benefit from some of the existing drugs that we currently have. We will also have much better tools to essentially determine if the patient—when a specific treatment is going a good response where no response or actually is getting worse. Currently those are not done in any kind of a robust matter, but I think the technologies that are coming online would allow us to do that. I think these technological and precision management advances combined with some major progress in understanding the basic biology of what scleroderma is are going to lead to perhaps not a cure, but much better outcomes as well as our ability to predict complications before they occur and hopefully prevent them.
Host: Dr. Correia, first last word to you. What would you like other physicians to know about the scleroderma program at Northwestern Medicine and when you feel it’s important that they refer.
Dr. Correia: To us I think we firmly believe that the earlier that we can see new patients with this disease, the better. The outcomes are better. Our abilities to help them are better and we can head off some of the worst complications of this disease. The other thing is that we are particularly interested in patients with diffuse systemic sclerosis right now and where they welcome and consultations with other physicians with the few systemic scleroses.
Host: Dr. Varga, last word to you. What would you like other providers to know about when they do refer a patient, what they can expect from you and your team and Northwestern Medicine.
Dr. Varga: To echo my colleague, I think it’s critical to be able to get patients with scleroderma or scleroderma like conditions to a referral center really early. Because one damage occurs, it’s much more different generate and repair than [inaudible] the damage. So seeing patients very early, we pursue a very integrated, multi-disciplinary proactive approach to find out what is the specific characteristics of that individuals disease. What are the kind of treatments they might benefit from? We like to work very closely with our colleagues in rheumatology and other specialists throughout the team to provide the in proactive care that’s very personalized to each patient.
Host: Thank you gentlemen so much for coming on and sharing your expertise and explaining the program so very well for us. Thank you, again. That wraps up this episode of Better Edge a Northwestern Medicine podcast for physicians. To refer a patient and for more information on the latest advances in medicine, please visit nm.org to get connected with one of our providers. If you as a provider found this podcast informative, please share with other providers. Share on social media and be sure to check out all the other fascinating podcasts in the Northwestern Medicine library. Until next time, I'm Melanie Cole.