HCP Live

The American Society for Preventive Cardiology (ASPC) has announced their 2021 Virtual Summit on CVD Prevention will take place from July 23-25, 2021.

The ASPC emphasized the event will focus on the inclusiveness and the care of one and all at risk for cardiovascular disease.

The event is for a wide variety of physicians, researchers, and other healthcare professionals to engage with cutting-edge lectures, lively debates, and practical applications.

Presenters will explore the broad spectrum of preventive cardiology led by world-renowned experts in the field of cardiology.

PAD Evaluation, Treatment, and Management

Practical Application of New Prevention Therapies

Articles

The virtual sessions will cover a litany of topics in the greater spectrum of cardiovascular disease prevention and care.

ASPC 2021 Virtual Summit on CVD Prevention Taking Place July 23-25

 State of the Science expert webinar on the current and future state of inflammatory bowel disease (IBD).

Melmed, Director of Inflammatory Bowel Disease Clinical Research at Cedars-Sinai, was joined by his Cedars-Sinai colleagues 

for a 90-minute program on the following topics:

Biologics for IBD – when, which one, and in whom?

Emerging Therapies – the future is bright!

 for a recap interview on the subjects of the program, highlighting current biologic therapy options for IBD, and what future clinical research should entail.

Videos

Gil Melmed, MD: Biologics for Inflammatory Bowel Disease

Key opinion leaders consider the possibility of cure for patients with sickle cell disease receiving gene therapy, and end the discussion by providing some closing thoughts. 

There was another assumption in my question about the cost of gene therapy compared with the evolving standard of care. The assumption was that gene therapy is potentially curative. Is it?

We don’t know yet. I’m involved in quite a few trials, and I hesitate to use the word curative in any of them. What I tell patients very honestly is that I can guarantee they’ll be heavily disease modified if successful, but we won’t be able to determine cure. Then again, there are still some things about stem cell transplant. We know it’s a cure, because we know we biologically change the type of hemoglobin those individuals are producing, but we don’t completely know if it cures their organ dysfunction or if it completely cures some of the other adverse effects and complications of this disease. We have to look at it in those terms. We’re getting there. I don’t think we can quite say that. It’s important, and I tell patients all the time that all gene therapies are not created equal. One gene therapy is not the same as another, and we’re going have to figure out that there are going to be stopping rules. Some gene therapies shouldn’t continue, and there are going to be some that are better than others. We’re going to have make sure. I’m on the cure panel to create the way that we’re going to be able to analyze these gene therapies and compare them with one another, and I hope to compare them with stem cell transplant in a way that we can really understand the outcomes.

 I want to thank all of you for this very informative discussion. But before we conclude, I’d like to get final thoughts from each of you. Michael?

 I like Wally’s statement about utilitarian approach for this disease. At some level, we have to understand that the science that cures an individual with this disease is just as exciting as the science that informs a family about their risk of having a child with the disease so they can make an informed decision about whether they want a child with the disease. And that science is just as important as understanding environmental risk factors for mental health challenges that our families have. As we celebrate truly state-of-the-art science developed in the laboratory, I hope that we can start to celebrate, or at least have on the agenda, resources to better understand the social determinants of health in this population and to better understand ways to inform primarily a miseducated Black population in this country about their risk of having a child with sickle cell disease so that they can make informed decisions. Too often, I see a family whose newborn is 3 months old, and they say, “Nobody ever told me that I could have a child with sickle cell disease.” This happens in 2020, and there’s no effort by our leaders to address the lack of formal education and programs to better identify this.

Last, but not least, I’d like to comment on our burden as providers and leaders to understand the humanitarian requirement to expand the scope and research of sickle cell disease to the lower middle-income countries where 95% of the children with this disease are born and where pregnant women, in most of the countries in Africa, have a 10% rate of death. Nowhere in the world would that be tolerated, but on the continent of Africa, where pregnant women are associated with a 10% risk of death. I am excited about the basic science with curative therapy. I am cautious about expenditure of resources that don’t go to the greater good. That has to be a strategy where we learn to have all contingencies at the table when we talk about helping our families with sickle cell disease.

Thank you, Michael, for all those highlights that are deeply rooted in social justice. We really appreciate that. Julie, some last thoughts from you?

It’s an exciting time to be a provider in sickle cell disease. We are finally getting to offer our patients options; something they’ve never had. Elliott said it really well earlier: Sickle cell disease sadly embodies the health inequities in our country. My goal is to really improve access to care, and I believe if we can do it here, then we can translate these findings to other places. But with a population that’s as small as ours is, compared with some of the larger populations of individuals with sickle cell disease, we’re not doing a good enough job. We have to figure out how to do our job better so that we can spread this other places. The science is exciting, and it is going to provide the pathway to improving access to care.

Thank you, Julie. Wally, some final thoughts?

 Recently, I’ve been comparing our quest to climbing Mount Everest. We’ve got the backpacks and the Sherpas. We see the top of the mountain. We know what it means to go up there. We are nearing, perhaps, Base Camp 1. We’re picking up people and taking them with us to get to Base Camp 1. We need more people to come. After Base Camp 1, it’s going to get steeper and steeper, and we really have to begin with the end in mind and start the journey with enough resources, planning, and forethought to actually get to the top of Mount Everest.

I love the metaphor. Thank you, Wally. Elliott, take us home. What are your final thoughts?

I agree with what the other doctors said. You cannot stop science. There’s been an explosion of new therapies and understanding targets. These have the potential to benefit sickle cell significantly. That goes without question. So I support ongoing scientific trials. These drugs and therapies have real benefit.

What I’m troubled about is the historical, immoral approach to society about providing basic care prep for them. I have difficulty in focusing only on new therapies if the families don’t get the education, the mental health, and the basic standard of care that will allow them to make quality-of-life decisions. We can do better with developing an infrastructure for care. I don’t think we’re in that case, and I actually believe these drugs will pull the universities further into sickle cell care. In a lot of ways, it’s harder to get outpatient counseling and things done than it is to get on to a gene therapy trial right now. But just don’t forget where sickle cell was in the past. It was always a scientific disease that was great to study, but also remember what Michael said. We all remember what Linus Pauling, who made some incredible discoveries, did. He came out with a proposal that wasn’t thought out that trait carriers should have that tattooed across their foreheads so they would know that they were carriers.

I’m in favor of the science. It needs to be a partnership between the health care plan community and the scientists. We need an organization as we have for cancer in children. We need a real organization that works out multidrug trials. We need an organization that looks into the statistical and other variables that affect outcome and biomarkers. We need to approach this the same way we would cancer or leukemia. You would not do 1 drug at a time. You would have an organization that looked at that and correlated it. I’m excited that if we stick together, we’ll get there. Thank you.

Well, thank you. Thank you to all of you. I really appreciate your insights, and I’m sure our audience does as well. Thank you for watching this HCPLive® Peer Exchange. If you enjoyed the content, please subscribe to our e-newsletters to receive upcoming Peer Exchanges and other great content right in your in-box.

Potential for Cure with Gene Therapy and Final Thoughts 

What are the barriers, if any, to FMT [fecal microbiota transplantation] for the treatment of recurrent CDI [

That’s unfortunately an important point. The FDA [Food and Drug Administration], in the past 2 years, has issued 3 warnings and cautions about the use of FMT. The first 1, a result of transmission of multidrug resistant organisms to susceptible patients, unfortunately had some fatal outcomes in those patients because they were highly immunosuppressed. That was followed by additional reports of transmission of toxigenic E. coli [

], which also resulted in some fatal outcomes following FMT. Then most recently, because of the presence of COVID-19 in the stool, there has been a caution from the FDA about using stool that has been acquired from donors during this COVID epidemic as a caution to primarily prevent transmission of the coronavirus with fecal transplant. These have been unfortunate occurrences, and we’re improving the screening of the donors. Each time something like this happens, additional screening tests are added for the donors. It is benefitting the entire process of FMT by getting better donor control in terms of management. The other factor that we still don’t know about is whether there will be long-term changes in underlying illness in patients who have their microbiome changed. It will probably take 5 to 10 years before we can answer questions like that.

That brings to mind a question: Is there a difference in a response to fecal microbiota transplantation in refractory CDI versus recurrent CDI?

Again, the bulk of experiments with FMT have been for recurrent CDI. We have had relatively few studies of recalcitrant disease or refractory disease, and the ones that have been done have been largely done by administering FMT through colonoscopy. An investigator in Italy, Giovanni] Cammarota Agostino Gemelli of the IRCCS University Hospital Foundation, had done some early studies. By doing colonoscopy, you can tell if a patient has a pseudomembrane or not. About half of the patients, based on our early studies, will have pseudomembranes present if they have 

 diarrhea. What Cammarota found was that he needed to do multiple fecal transplants in these patients and try to resolve the pseudomembranes. He did it in an unusual way. He administered vancomycin while he gave fecal transplants. I’ve never quite understood how you can have both vancomycin being administered, which should be detrimental to your microbiota, and at the same time be administering fecal transplant. It’s always been a mystery, but it’s been much more difficult to treat refractory 

 infection than it is to treat prevention of recurrence.

Are there any common threads, key factors associated with the negative outcomes from fecal transplants?

Yeah. There are very few failures as everyone has pointed out. Generally, in comparator trials, the rates of response are up in the 80% to 90% range. One recent publication has shown that non-responders are probably people who have irritable bowel syndrome, and that they probably entered these trials inappropriately and have persistent diarrhea after a failure of the FMT. The other interesting finding about this study was that the responders seemed to do better if they’d had a long course of antibiotic therapy preceding FMT. That’s been found by other investigators as well. A long course of vancomycin or a long vancomycin taper and pulse course followed by FMT generally gives you the highest response rates.

If you enjoyed this content, you should subscribe. We have an e-newsletter, and you can receive upcoming 

 and other great content in your inbox—that’s right, electronically. I’ll see you next time. I’m Dr Peter Salgo. Thanks again for watching.