Episode 38: Authorized Therapies for Autoimmunity! With Dr. Peter Marks
We don't gain anything by preventing people from getting therapy; we gain by seeing people get therapies that help them. Enter Dr. Keith March, a leading expert in adult stem cell research and Dr. Peter Marks, the leader of the FDA's CBRER, and who discuss with Haylie Pomroy the FDA-approved therapies for autoimmunity, specifically regenerative medicine. Join in the conversation and get a glimpse of how diligent the FDA is in making sure therapies are safe. They ensure they have sufficient data to know that the products do what they say they're supposed to do.
Still, each person is unique. The efficiency of these therapies depends on your microbiome and overall health. If you want to know about the variety of FDA-approved therapies that can help you in your fight against autoimmunity, this episode's for you.
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Authorized Therapies for Autoimmunity! With Dr. Peter Marks
I run this amazing community where we try to be open and receptive to all kinds of things that can help us find true health in our health journey. I'm a huge advocate of natural foods, health, and nourishing the body. I also have been very candid about my health journey and struggle. I have ITP, which is an autoimmune disorder. My hematological oncologist at UCLA over the years has become a dear friend and a champion for me continuing to seek therapies that can provide wellness. What I feel like is to become an individual that's outrageously healthy, feeling vibrant, alive, and well.
In our community and my clinical practice, which had been in practice for many years, many of my clients have traveled all over the world to seek, I'm going to use the term progressive, maybe some out of box therapies that either weren't readily available or available at all in the United States. I'm honored and so thrilled to speak with you. I have been privileged to have docs in my corner that supported some of my ideas and choice of therapies for myself.
When I heard about some of the FDA initiatives and the True Trials initiative specifically, it made me to want to share with my community that there is hope on the horizon. That's the biggest thing that I want to convey to my community. Thank you for being here. It is a privilege and an honor. I have goosebumps and chills because it brings me a lot of hope, and I know it's going to bring a lot of my community a ton of hope.
Let's cut to the chase. Let's jump right in. I know my community knows a lot about you already, but can you give me a little bit of insight as to how you ended up where you're at in your field? What was your inspiration, passion?
As somebody who was trained as a medical scientist, a clinician and administrator and also been in drug development and biologics development, it was a very natural fit to take the deputy director position when they offered it to me several years ago. I don't look back because I love what I do for the variety of complex biologic products that we regulate. We don't just regulate them. We also do applied scientific research on the products, try to understand them, and make policies about how they're going to be regulated. To me, it may not be earth-shattering, but it floats my boat. It keeps me excited about coming and doing what I do every day.
It takes that inspiration because it's a lot of responsibility. You personally have a lot of responsibility to ensure safety and efficacy with things in the regenerative healthcare field. In your particular role, you are the steward for safety for us as patients. Would you say that's a correct assessment?
Yes. Probably at the top of the list is safety. We'd also like to make sure that things have efficacy, and they need to be made with appropriate quality. Safety, efficacy, quality are essentially our mottos.
A lot of people in my community were asking me, what do we mean by regenerative therapies? How does the FDA play a role in that? As I shared with you about my health journey, a lot of terms that have been used in the past, even anti-aging therapies, things of that nature, a lot of regenerative therapies have only been readily available outside of the United States. What would be considered a regenerative therapy?
First of all, the term regenerative therapy is quite broad. It includes everything ranging from drugs to biologics to cell therapies. Some even consider gene therapies in that mix or cell-based therapies. A lot of different things in that mix. Most of us, when we're focusing in and out of the way and stuff that we care about most are cell-based therapies, gene therapies, and cell-based gene therapies. That's not to say that there aren't other things out there that are regenerative therapies, tissue-based therapies. There are some devices that are probably even regenerative and some drugs, but for us, our center, we care about cells and tissues.
We've heard a lot about stem cell therapies. When I was diagnosed with ITP, I did try traditional therapies quite aggressively. It wasn't a phenomenal success for my body in particular. I was fortunate to have the team of docs that I do that are not only brilliant but my biggest cheerleaders, for me, continuing to seek out therapies. One of the reasons why I wanted to speak to you specifically is because this FDA authorization of certain types of trials is, in my opinion, I know I'm getting up there in age, a long time coming. What was the precipice? Why is it starting to happen in the United States?
What we're starting to see is people were coming to us and wanting to do these trials. For a while, people were playing with stem cells, hoping they were going to do things and using them as tools to get business, perhaps. People are trying to understand what they do, and are they bringing benefits? We don't gain anything by preventing people from getting therapy. We gain by seeing people get therapies that help them. The only the way you figure out whether something is helping people is by studying it in some type of a trial.
It doesn't have to be a randomized placebo-controlled trial, but by studying it and using some type of scientific method, that's how you figure out whether it works or not. What we want to see with stem cells is somebody studying something that's a defined thing. Seeing whether, at the end of the day, people will get better or not. Someone could look at mesenchymal stem cells administered by blood to make ITP better. It is an appropriate immunosuppressive treatment.
There you have something that's easy to measure. You can see the platelet count go back up. That's an easy example of you can do a rigorous trial and say, “We're going to give the stem cells on day one and 15, 30 days later, we're going to see what happens to the platelet count versus where it was at baseline.” If it's gone up, if it does that in multiple people, maybe the stem cells are doing something right.
If it doesn't, maybe you're having to go back to the drawing board and figure out what you need to do better next time. That's this generation of knowledge that is so critical here. It’s what's hopefully transformed the past couple of years from a field that wasn't very interested in rigorously understanding what was going on to now trying to understand rigorously what is going on.
I always tell my community, you have so much power as the patient. I would try to empower my client base and community so that they can ask questions. We do a health wish list self-assessment questionnaire so that people can understand what's going on in their health and also so that they can communicate to their physician more efficiently. I will share with you, I've traveled with clients that have gone everywhere from Moscow to Mexico to The Caribbean, many places out of the country seeking these types of therapies.
It is sometimes expensive. It can be extremely scary. Sometimes people feel a sense of desperation. They're willing to absorb that risk and take that on for this huge hope. Now there's this opportunity that's coming up where we can have FDA-authorized trials. They're regulated, watched, monitored, and given a blessing.
There are trials that are conducted under our investigation on the drug authority. They're like any other trial for any other drug where we make sure that the people who enroll in those trials are protected. They're being treated safely and we make sure that the trials are trying to answer the questions that are important. You can say you're doing a trial and not get any real information from it to play the game.
When you get back to medical tourism, I'll offer up that we have to be careful about not saying anything too sweeping about overseas trials because, in some places, they do good jobs. The problem is others don't. It's a little bit hit or miss. Are you educated enough, or are you willing to do the research to figure out who's doing it well and who's not doing so well? Hopefully, by having these trials available in the United States and more accessible, people will take advantage of them. There'll be done well.
It's a lonely process. I'll share that with you. A lot of my clients that have gone through this, don't even feel comfortable sharing with their physicians that they're going to either participate out of the country or in a non-FDA-authorized trial. They're going to try this experimental therapy without that supervision or support. One of the things that's exciting for our community is that with these being FDA-authorized, they can have a more open dialogue with their physician. I was fortunate. I had a team of docs that were my number one cheerleader in my process of using food as medicine, nutrients as medicine and supplementation. They all encouraged me to seek out what was best for my health and wellness.
What I find in my community is that so many people feel alone in this process. We now have this website where people can come and look for trials that could potentially be right for them. How can you help people in my community that are reading that say, “I want to start to have this conversation with my doctor?” With you speaking to me, this is going to help the legitimacy of their conversation. They're going to be going from A to Z right away. That's why I appreciate this so much, but how could someone broach this conversation with their physician?
A couple of things. First of all, many physicians, whenever they hear you're interested in something, they're concerned that you're getting the standard of care. That's probably something that we as physicians can probably be a little more open-minded about. I know that when I treated a lot of patients, I probably had the same attitude as perhaps some of your physicians had. As long as somebody is getting reasonable standard care from me, I'm okay if they want to try other things.
This stuff is not perfect because we don't always have the be-all-end-all. What I would want to do is make sure they know that when they go to participate in a trial that it's being done correctly and that usually means a combination of two things. If it's a new drug or biologic that is being studied in the United States, it's under our Investigational New Drug Application. If it's in some other countries, again, it's under those country's laws.
In the United States, if you're participating in research of an unapproved drug, it should be under an Investigational New Drug Application. That's number one. Number two, there are some types of institutional review or ethics committee approval of that trial. They do two separate things. One, what the FDA does is make sure that the product itself is named correctly and that the way it's being studied is going to get the answer.
We even find this in the supplement world, what's in the bottle needs to be what is said is in the bottle.
The Ethics Committee’s job with the IRB is to make sure that the way this is done is consistent with what we've come to expect in the United States, that someone is not taking unacceptable risks. It's complimentary. By having both of those together, we do right by people to make sure that they're getting the type of care they should be.
Can we talk about the types of things that may be beneficial for cell therapies? I shared with you that I have ITP. I have clients that have gone for arthritis, other autoimmune disorders, degenerative knee disorders. We've seen in other countries a lot of therapies around viral infections, chronic EBV, CMV. Are there things that are perfect for this or we're not sure yet?
We're not sure yet, but if it's studied well in a clinical trial, it's okay. There can be a variety of things here. That's part of why it's helpful to have something that's FDA-authorized in the US. We've given it the go-ahead. It's an FDA-authorized trial, or it's been approved by an Ethics Committee because what that says is enough people have thought it through that it's not a harebrained idea. That's what you're trying to get at here. This concept of certain types of stem cells having immunosuppressive properties, there's a fair amount of scientific evidence that would tend to suggest that.
Has it been documented beyond all doubt yet? No. That's why we're still seeing clinical trials ongoing with this, including things that have even been blocked before the agency in terms of new drug applications or biologics applications. It's a very exciting area. We don't have definitive answers. That's why, if you are participating in a trial, it's good to be participating in one that's going to help generate knowledge and help keep you safe.
I obviously have a particular passion for the autoimmune world because it's something that's affected one of my family members and me. Is there something in all of your vast experience that you get excited or interesting to see how this is going to pan out with?
The whole area is exciting. Personally, I'm very excited to see what will happen with the immunosuppressive properties, perhaps of some of the stem cell products. Even some of the cellular products that we see use, whether exosomes or cell fragments may have some immunosuppressive properties. We don't know yet. It'll be interesting to find out. Some of this is a matter of having them studied well, having this study in a given condition, and having people have the courage to study them in conditions where they'll get an answer.
One of the problems here is that a little bit of what goes on in regenerative medicine is a little bit like what goes on sometimes in British law. British lawyers never asked your client if you're guilty because you don't want to find out. Sometimes people don't seem to want to get an answer because if there are some diseases or disease entities that are perfect for getting an answer to this, you don’t have to do biopsies, you just take blood samples.
That's part of what has gone on here. The field needs to have the courage to fail. When I worked in the industry, one of the things I did besides doing early phase development was they realized that I could not insult sponsors. The businesspeople used to take me to do business deals and licensing visits. What I got to see was a lot of different things, but I also realize that about 1 in 20 things that get into phase one make it through the process. A lot of stuff has to fail. The stuff we're seeing failing in regenerative medicine shouldn't be viewed as a catastrophe. It's just nature taking its course here.
It's the courage to try. That's what I'm appreciating in a lot of the people that I'm seeing involved in this. To your point, there's people that are maybe way far out there, but maybe those people have been forging the path for people to have reasonable and science-based research around some of these therapies that are very exciting to see. I have this massive community. We do this thing, as we call it, a Dear Dr. Sanders letter. There was a physician that we work with within Ohio, that we were having a difficult time getting care for one of our clients. Every time they walk in, this appointment would be super short, and they couldn't articulate what they needed. It was a miss all the way around.
She was going to fire the doctor and change her practice. We felt like she was in a good place. We helped her formulate this doctor's letter. Our whole community uses it. What we do is we have people fill it out and it says, “This is what's going on with me. This is what I'm dealing with. This is what I'm hoping for in my health and wellness.” They print it out and give it to the doctor before the visit. We always have to send it ahead and it helps people effectively communicate their vision for their health.
If I were to give my community some instruction on how to fill out their Dear Dr. Sanders letter, you're saying, “I'm dealing with ITP or lupus or Crohn's or rheumatoid arthritis.” I want to broach the subject with my doctor. I don't want them to think that I've got this harebrained idea. I want them to take me seriously. I want them to take these trials that I've found on this website seriously. How could we distill this down onto our worksheet? How could we communicate that effectively? There's a huge divide between trials that are there. They are good trials. People are getting access to them. How could we communicate that?
I'm going to step back and give you a long answer. First of all, the Dear Doctor letter should probably say, “Doctor, how do you think I'm doing in the scheme of things?” Part of this is understanding. Sometimes patients can be doing well. They sometimes don't understand how well they're doing. For instance, for patients with ITP, with a platelet count of 75,000, that's great. For those of us who had to struggle with patients who can't get a platelet count above 15,000, that is stellar.
I wouldn't call it perfect, but I would say not bad. Could it be better? Yes. That helps you understand, are you doing okay? Good, not so good. That makes me at least help a patient understand what they might be willing to take the risk for. It helps me have that conversation. If I haven't been able to use my therapies to help someone's platelet count, let's use that as the example, say at a reasonable level, that will make sure that they don't have a bad bleeding. For those who might be reading, platelet counts are these numbers, but usually, one’s platelet counts and people with ITP get much below 30,000. Once they get to 15,000, we start to lay awake at night, as doctors can get worried. Even though usually nothing bad happens, but we worry.
I know my community is reading this. There are so many symptoms that I can share with you associated with maybe even if you fit into that paradigm of your labs being okay, or acceptable or not scary. There are so many things that lie in between there. The patient 24/7 lives with that, whether it's a symptom profile or a less-than profile.
Some people with ITP have lupus or other autoimmune diseases that are driving it. What they need to do is think where does your disease lie in the scheme of things. The question is if I were to use one of these therapies, would it interfere with what you're doing? That's oftentimes like a key question because if the answer to that is no, then why shouldn't I try it?
How do you consume an elephant? One bite at a time. Take a little bite right here and see if I can distill this down. If an individual is on therapy like prednisone or an anti-rejection drug-like Imuran or CellCept or something of that nature. Collectively people felt that mesenchymal stem cell therapy would not interfere with that particular medicine protocol. If I'm hearing you correctly, we're thinking that this would be how we would broach it with a physician.
I had a client come in one time and say, “I'm done taking my medication, help me find or navigate. I don’t care where we go in the world. I don’t want to take this anymore. I want health and wellness.” I said, “Hold on, breathing is important. Your platelet levels are important. This is a process.” What I want to bridge for our community is that there's not one or the other, but that it can be complimentary. Regenerative means to bring on the new, create the new, regenerate something that has gone awry, regulate the inflammatory and immune system in that situation. How can a person say, “Doc, I've been on this website. I see that there was this trial at University of Florida or Mayo Clinic. I'd like to broach this conversation?”
They can come with the trial. They'll be inclusion and exclusion criteria. That's all the point of the trials because the trials will say, “It's okay if the person’s on prednisone, no problem. You can stay on the current dose of prednisone” because that's the beautiful thing. If it's working, they should be able to taper the prednisone. That's the whole point of clinical trials that are written. It's a protocol that's done the same way. The idea would be that, “If the platelet count seems to be going up, you can taper down the prednisone.”
Would that be in the IND? Would that be in the paperwork?
The clinical trial itself that the physician is following and that clinical trial, someone will write, you can decrease the prednisone as you move along if the platelet count comes up. That's how some of the therapies, if you think about the platelet raising therapies, let people stay on. You couldn't take people off of it, but you could reduce the steroid treatments.
I can see my community members now that are going to go print out the studies and highlight them and take them to their docs. Do you think the most compelling reason that a physician would support this decision in a patient is because they are FDA-authorized? Do you think that is what's going to move us across this chasm?
It's going to help. Is it going to be perfect? No. I've had people come to me with FDA-authorized trials. It's a very good start. I’m like saying, “I'm going to someplace that does not have a stringent regulatory system. I'm going to try something there that doesn't have any relationship to anything FDA is doing.” This is something that we've said at FDA. It means people have thought about this when our folks say that a clinical trial can proceed, they’ve read through all the stuff about the drug that's being used or the biologic that's been used, they looked at the clinical trials protocol very carefully.
They think about it and say, “This can proceed or can't proceed.” If you're getting it, if it's out there, it's because it could proceed. It has a certain stamp that doesn't guarantee that it's going to work. If we knew things were going to work, we won’t have to study them. At least it says that somebody thought that what was being done is possible. It's not likely to hurt people.
It was brought to my attention that some colleagues of mine are participating in this website called True Trials. What was fascinating to me about it is it felt like a safe meeting place for physicians that are passionate about regenerative therapies and progressive, maybe even sometimes out-of-the-box medicine and patients that are looking to participate in some of these FDA-authorized trials. How do you view this particular website, and what service do you think it can provide?
We've talked a little bit before about the hit-or-miss nature of what happens if you go overseas to get a trial that is well done and done properly in terms of both the scientific and ethical aspects. This concept that’s being done on this website is this concept that the hit-or-miss is taken out of it. It doesn't say that the trial is going to work necessarily, but it does say that the trial has been authorized by FDA. It does say that an ethics committee or institutional review board has done what it's supposed to do. It takes at least that hit-or-miss nature out of it. You can at least rest assured that those key things are taken care of.
It's this meeting ground and this coming together of people that have these progressive passions and ideas in medicine and patients that are in need. I have to ask, and our community was having a deep conversation about this. I'm not necessarily asking you to predict other people's thoughts, but do you have some insight in your position as to why we don't see physicians recommending or referring out a lot for trials, even specifically in the regenerative therapy world but in trials in general?
This is a mindset that we've had in the United States. In a lot of cases, it's about the fact that we as physicians take a lot of ownership of our patients, which is a good thing. It also means that sometimes we don't let go when it's in the best interests of patients for the trial. It takes a certain amount of maturity and courage as a physician to say, “I've gotten you as far as I can, but it's okay. You can try something else. I'll support you through it.”
That's a very amazing way to look at it. In my case, in particular, my docs or my partners in my process of being well and getting healthy. I sought out individuals that had the same vision for me as I had for myself. In this case, would it be a good idea for physicians to participate in True Trials, physicians that are doing FDA-authorized trials? Should they put their research here and their information here?
This type of system is very helpful for patients. Right now, ClinicalTrials.gov is great, but it's got this large listing of trials. We're not able to police it at FDA to make sure that those trials, every trial is in there. You don't know whether it has an appropriate IND in place. Some of them, you don't even know if it has set up the right IRB or ethics committee review. Whereas this concept of having a site whereby somebody has gone through the trouble of saying, “We have gone and looked, made sure they have that Investigational New Drug Application in place.”
“We've made sure they have the ethics committee or Institutional Review Board approval.” Taking care of that piece of it helps essentially winnow down a lot of the issues. It’s helpful to patients to have all that uncertainty, especially in this area of regenerative medicine where there are some predatory practices that have been used with ClinicalTrials.gov, and that's been in the papers. This is a way of essentially eliminating that.
There's a lot of good things in that space. They are potentially could be bad things in that space. What we're looking at for our community are resources where some of the vetting has taken place. If it's going to be there, we know X, Y, and Z have been addressed.
I think of it as de-risking. We do that a lot.
The other thing is efficacy. I'm going to shift gears here. You are the leader in efficacy. We want to make sure that things work well. As you know, I am in the nutrition field. In any aspect of regenerative medicine, would it be safe to say that a healthier patient is going to have a better result?
That's a well-known fact in medicine, is that people who are healthier overall will do better generally.
That's why I always feel like I've been able to navigate these amazing relationships with physicians that a variety of different universities and institutions is because our goal as a community is to provide you as the physician with the healthiest patient, with the most nutrient-dense diet and micronutrients available so that the body can have a healing process or a health trajectory that's in a better direction. That's awesome. We both agree. Nutrition's awesome.
By de-risking the process of an individual looking into a trial, do you think that this is going to elevate the field of regenerative medicine because, to your point, regenerative medicine has had some negative issues in the past because of people that I wouldn't say are progressive, but are more maybe irresponsible with a twisted renegade.
It can help us here. The reason why is if enough trials get done carefully, eventually there will be trials that will be successful, and the field will start to progress into a place where you now have successes. There will be improved products. You’ll generally find people coming behind those products with more trials, and the trials should get better over time.
As we have trials that are successful, people will understand the science better. The next trials that come up behind them will hopefully improve upon the earlier trials. That's how we like things to work in medicine. It's taken a while to get there in regenerative medicine, in part because cells are complicated things. It's not easy, but it's not hopeless either. We can get there if we work systematically.
Anything that is organic in nature has such bio-diversity and more of maybe a profound impact within the body because maybe the body recognizes it more as self. There are many concepts around cell therapy that are fascinating, from biochemistry to physics to oncology. It's a fascinating study. Fast forward ten years from now, is it your vision? Is it your dream? I'll share mine with you that an individual that's dealing with lupus or ITP or Hashimoto's thyroiditis, those cell therapies are part of their protocol or a choice?
It may be a cell-based therapy or cell-derived therapy could very well be some part of that modulation. If we can find things that can do what our current drugs are doing, they can do so less toxically all the more the case. As somebody who has given a lot of prednisone in my day, there were a lot of people who would like to avoid chronic prednisone for many reasons. Avoiding diabetes, weight gain, and all the other stuff is among them. It will be hopeful that will be a way that things will progress. Do we know that it will work for sure? We don't, but it would be great if we can see things progress, whether it's a whole cell therapy or parts of cells, or even if it's the insight that we derive from this that allows us to deal with other biologics that help people. That will be great.
I always say that prednisone was the very first love of my life. It was one of those drugs that pulled me out of a horrific health crisis. It saved my life probably. It also took me to the brink of kidney failure. I had a negative response to it, unfortunately. Because of that, though, it drove me to go back and learn more about my biology and biochemistry, and fall in love with what can be supported and nurtured within my body. That's why I said it became my hematologist best friend.
I get so hopeful when I see these trials coming into the United States, as an individual that has helped thousands of people navigate their health journey, We are so biodiverse as people. Our responses to drug therapies, cell therapies, natural therapies, or nutritional therapies can be so different. When we try to put an individual in a box, we restrict the true potential.
In our community, we believe that the healthier patient that we can deliver to the physician, the better the outcome is going to be. We see this time and time again with the reduction of inflammation and increased fertility. As a physician in your role, why is there such a profound impact in people when they have strong sound nutrition?
I'm not a nutritionist, but as a physician, who's taking care of a lot of people, people who have good diets, take care of themselves, exercise regularly, they tend to do better. It is one of those things that we see the body gets taken care of well and gets what it needs. It tends to do better no matter what therapy you give to it. That's the fact. We see this in clinical trials. That’s why clinical trial sometimes even stratifies people by their weight, exercise levels.
If you compare groups and you didn't do that, and then you had one group that was all exercising, eating wonderful diets, and another group that was couch potatoes, eating potato chips, and drinking soda, even if you gave them the same therapy, you might get a different outcome. You have to control for those things.
It's all about fertilizing that soil to grow healthy cells, healthy tissue, and have healthy blood counts. It's about nurturing that.
It's these people taking care of themselves. It's an indicator of whether people take care of themselves.
It’s in a hopeful way. I'm fascinated by this True Trials website. For me, and I'm not good at these things, it's very easy to navigate. This is where we sent a lot of our community to look for trials that might pertain to them. Universities are where the trials are occurring and happening. Why would it be important for a patient to have the safest space like this to go?
There are so many websites you could end up on by accident. It's nice to have a place to go that is trusted. That's helpful not just be wandering the wilderness of the web looking for things but to have a site that has some clear validity to it, that's taken the time to do the right things of making sure that all the right parts are there for people to go to. When they get something off that website, they feel like, “It's something I can trust.”
It's much needed. A lot of my doc friends that have this passion and desire and are participating in these types of clinical trials are sometimes having issues with the recruitment of getting their trials filled. Is this a great place for doctors to position their trials to place in there? How is that going to help them get their trials filled?
You'd like to think that if you have a site that people start to trust, that will draw on traffic eventually to that site. Trials will fill a lot more quickly. People are going to go there, hopefully, because they know, that's where they can find trials that meet the criteria of being FDA-authorized and ethics committee or IRB approved. That's going to help. More people that go there and more people see trials, more people get on the trials, trials get done faster, you get answers faster. That helps the field of regenerative medicine overall because then you start to get the answers that you need. Things are going to fail. It's going to happen. There's no guarantee. The part that’s important about these websites, it's not a guarantee that the trial is going to work. To your terms, it's a field that things can grow there. That's what it's saying.
It allows for people to know that they have an option in the United States. It can be lonely going out of the country. It can be extraordinarily expensive going out of the country. Are these trials something that when a person participates in the trial, is it at an expense? Do people have to pay to be in a trial?
In general, people do not pay to be in these trials. Clinical trials should be covered by the group that's doing them. There is patient-funded research. Don't get me wrong. There are some times that it gets done. The way these trials should generally be done, when it's patient-funded, it's the foundation. Getting them on the foundation then spends the money. It’s not moving there. There are trials that the FDA sometimes does give approval for people to recruit the cost of the product itself that is being used.
The patient's insurance might have to pay for that product. That's only in specific cases where they have come to the FDA in advance. Many of the trials are done not at a charge to the patient. Standard care of things is often charged to patients or insurance or things that would have been. Things might be done anyway, and standard of care might be charged to the patient's insurance, but the things that are done specifically for the trial, generally, the patient should not have to pay for it.
If a patient were to go into a trial and didn't have insurance, but part of the standard in the trial was to have labs before and after, like an ITP checking for platelets, would that be something that typically they would work into the trial?
That would usually be worked into the trial and the investigators would have to work through that.
It wouldn't be like, “We can't run labs.” That's why it's so important because there is a standard here where maybe we're doing this in some other setting. Maybe they wouldn't have the labs checked. I'm open and receptive to people seeking out what's right for them. What I've found so many times, which has always kept me up a little bit at night, is oftentimes, people will do therapy. They'll come back to me to this country without having an additional follow-up call. We all ponder. We work hard with their physician stateside to say, “How can we pseudo put together some hypothesis or what parameters would we look at to see if this was first and foremost, hopefully not a negative impact on the body, but also potentially positive ones?”
This is very exciting to see that. I know my community members are going to be printing this out and highlighting all the things and having these great conversations with their doctors. Specifically, on True Trials website, if they go in and navigate the website and see a trial, would they print out information and take it to their doctor? Would they contact the clinic? Would they contact the places doing the trial? How does the logistics work? Walk me through it.
That you'll have to get from the trial site, the idea is that you find a trial, and there are ways to set off motion to contact the people who are involved with doing the trial.
Primarily, your role is to say that, “They're not going to be put on this website unless we have blessed them as far as they are following protocol, they have a particular protocol that they have to adhere to, and we've vetted the process and said, ‘Go forward and let's see if it’s going to work.’”
That's what's being said here. Trials would not show up there unless they made sure that the FDA has given the authorization that they can proceed and ethics committee or IRB approval is there.
I am so excited about this and to have someone like you watching over the process is going to make it such a profound endeavor. I'm excited for my community, for myself, and for the future of medicine. I want to thank you.
Thank you. We have a whole group of people that keep a close eye on these things, ranging from the scientists who review trials, compliance officers who go out to make sure that things are what they say they are. There's a large team behind this. It's a pleasure.
Thank you.
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Important Links:
- ClinicalTrials.gov
- Investigational New Drug Application
- CBER
- Investigational New Drug Application
- Center for Drugs
- Creating Your Health Wishlist
About Dr. Peter Marks
Peter Marks, M.D., Ph.D. is the director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration. The center is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies.
Dr. Marks and center staff are committed to facilitating the development of biological products and providing oversight throughout the product life cycle.
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We don't gain anything by preventing people from getting therapy; we gain by seeing people get therapies that help them. Enter Dr. Keith March, a leading expert in adult stem cell research and Dr. Peter Marks, the leader of the FDA's CBRER, and who discuss with Haylie Pomroy the FDA-approved therapies for autoimmunity, specifically regenerative medicine. Join in the conversation and get a glimpse of how diligent the FDA is in making sure therapies are safe. They ensure they have sufficient data to know that the products do what they say they're supposed to do.
Still, each person is unique. The efficiency of these therapies depends on your microbiome and overall health. If you want to know about the variety of FDA-approved therapies that can help you in your fight against autoimmunity, this episode's for you.
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Watch the episode here:
Listen to the podcast here:
Authorized Therapies for Autoimmunity! With Dr. Peter Marks
I run this amazing community where we try to be open and receptive to all kinds of things that can help us find true health in our health journey. I'm a huge advocate of natural foods, health, and nourishing the body. I also have been very candid about my health journey and struggle. I have ITP, which is an autoimmune disorder. My hematological oncologist at UCLA over the years has become a dear friend and a champion for me continuing to seek therapies that can provide wellness. What I feel like is to become an individual that's outrageously healthy, feeling vibrant, alive, and well.
In our community and my clinical practice, which had been in practice for many years, many of my clients have traveled all over the world to seek, I'm going to use the term progressive, maybe some out of box therapies that either weren't readily available or available at all in the United States. I'm honored and so thrilled to speak with you. I have been privileged to have docs in my corner that supported some of my ideas and choice of therapies for myself.
When I heard about some of the FDA initiatives and the True Trials initiative specifically, it made me to want to share with my community that there is hope on the horizon. That's the biggest thing that I want to convey to my community. Thank you for being here. It is a privilege and an honor. I have goosebumps and chills because it brings me a lot of hope, and I know it's going to bring a lot of my community a ton of hope.
Let's cut to the chase. Let's jump right in. I know my community knows a lot about you already, but can you give me a little bit of insight as to how you ended up where you're at in your field? What was your inspiration, passion?
As somebody who was trained as a medical scientist, a clinician and administrator and also been in drug development and biologics development, it was a very natural fit to take the deputy director position when they offered it to me several years ago. I don't look back because I love what I do for the variety of complex biologic products that we regulate. We don't just regulate them. We also do applied scientific research on the products, try to understand them, and make policies about how they're going to be regulated. To me, it may not be earth-shattering, but it floats my boat. It keeps me excited about coming and doing what I do every day.
It takes that inspiration because it's a lot of responsibility. You personally have a lot of responsibility to ensure safety and efficacy with things in the regenerative healthcare field. In your particular role, you are the steward for safety for us as patients. Would you say that's a correct assessment?
Yes. Probably at the top of the list is safety. We'd also like to make sure that things have efficacy, and they need to be made with appropriate quality. Safety, efficacy, quality are essentially our mottos.
A lot of people in my community were asking me, what do we mean by regenerative therapies? How does the FDA play a role in that? As I shared with you about my health journey, a lot of terms that have been used in the past, even anti-aging therapies, things of that nature, a lot of regenerative therapies have only been readily available outside of the United States. What would be considered a regenerative therapy?
First of all, the term regenerative therapy is quite broad. It includes everything ranging from drugs to biologics to cell therapies. Some even consider gene therapies in that mix or cell-based therapies. A lot of different things in that mix. Most of us, when we're focusing in and out of the way and stuff that we care about most are cell-based therapies, gene therapies, and cell-based gene therapies. That's not to say that there aren't other things out there that are regenerative therapies, tissue-based therapies. There are some devices that are probably even regenerative and some drugs, but for us, our center, we care about cells and tissues.
We've heard a lot about stem cell therapies. When I was diagnosed with ITP, I did try traditional therapies quite aggressively. It wasn't a phenomenal success for my body in particular. I was fortunate to have the team of docs that I do that are not only brilliant but my biggest cheerleaders, for me, continuing to seek out therapies. One of the reasons why I wanted to speak to you specifically is because this FDA authorization of certain types of trials is, in my opinion, I know I'm getting up there in age, a long time coming. What was the precipice? Why is it starting to happen in the United States?
What we're starting to see is people were coming to us and wanting to do these trials. For a while, people were playing with stem cells, hoping they were going to do things and using them as tools to get business, perhaps. People are trying to understand what they do, and are they bringing benefits? We don't gain anything by preventing people from getting therapy. We gain by seeing people get therapies that help them. The only the way you figure out whether something is helping people is by studying it in some type of a trial.
It doesn't have to be a randomized placebo-controlled trial, but by studying it and using some type of scientific method, that's how you figure out whether it works or not. What we want to see with stem cells is somebody studying something that's a defined thing. Seeing whether, at the end of the day, people will get better or not. Someone could look at mesenchymal stem cells administered by blood to make ITP better. It is an appropriate immunosuppressive treatment.
There you have something that's easy to measure. You can see the platelet count go back up. That's an easy example of you can do a rigorous trial and say, “We're going to give the stem cells on day one and 15, 30 days later, we're going to see what happens to the platelet count versus where it was at baseline.” If it's gone up, if it does that in multiple people, maybe the stem cells are doing something right.
If it doesn't, maybe you're having to go back to the drawing board and figure out what you need to do better next time. That's this generation of knowledge that is so critical here. It’s what's hopefully transformed the past couple of years from a field that wasn't very interested in rigorously understanding what was going on to now trying to understand rigorously what is going on.
I always tell my community, you have so much power as the patient. I would try to empower my client base and community so that they can ask questions. We do a health wish list self-assessment questionnaire so that people can understand what's going on in their health and also so that they can communicate to their physician more efficiently. I will share with you, I've traveled with clients that have gone everywhere from Moscow to Mexico to The Caribbean, many places out of the country seeking these types of therapies.
It is sometimes expensive. It can be extremely scary. Sometimes people feel a sense of desperation. They're willing to absorb that risk and take that on for this huge hope. Now there's this opportunity that's coming up where we can have FDA-authorized trials. They're regulated, watched, monitored, and given a blessing.
There are trials that are conducted under our investigation on the drug authority. They're like any other trial for any other drug where we make sure that the people who enroll in those trials are protected. They're being treated safely and we make sure that the trials are trying to answer the questions that are important. You can say you're doing a trial and not get any real information from it to play the game.
When you get back to medical tourism, I'll offer up that we have to be careful about not saying anything too sweeping about overseas trials because, in some places, they do good jobs. The problem is others don't. It's a little bit hit or miss. Are you educated enough, or are you willing to do the research to figure out who's doing it well and who's not doing so well? Hopefully, by having these trials available in the United States and more accessible, people will take advantage of them. There'll be done well.
It's a lonely process. I'll share that with you. A lot of my clients that have gone through this, don't even feel comfortable sharing with their physicians that they're going to either participate out of the country or in a non-FDA-authorized trial. They're going to try this experimental therapy without that supervision or support. One of the things that's exciting for our community is that with these being FDA-authorized, they can have a more open dialogue with their physician. I was fortunate. I had a team of docs that were my number one cheerleader in my process of using food as medicine, nutrients as medicine and supplementation. They all encouraged me to seek out what was best for my health and wellness.
What I find in my community is that so many people feel alone in this process. We now have this website where people can come and look for trials that could potentially be right for them. How can you help people in my community that are reading that say, “I want to start to have this conversation with my doctor?” With you speaking to me, this is going to help the legitimacy of their conversation. They're going to be going from A to Z right away. That's why I appreciate this so much, but how could someone broach this conversation with their physician?
A couple of things. First of all, many physicians, whenever they hear you're interested in something, they're concerned that you're getting the standard of care. That's probably something that we as physicians can probably be a little more open-minded about. I know that when I treated a lot of patients, I probably had the same attitude as perhaps some of your physicians had. As long as somebody is getting reasonable standard care from me, I'm okay if they want to try other things.
This stuff is not perfect because we don't always have the be-all-end-all. What I would want to do is make sure they know that when they go to participate in a trial that it's being done correctly and that usually means a combination of two things. If it's a new drug or biologic that is being studied in the United States, it's under our Investigational New Drug Application. If it's in some other countries, again, it's under those country's laws.
In the United States, if you're participating in research of an unapproved drug, it should be under an Investigational New Drug Application. That's number one. Number two, there are some types of institutional review or ethics committee approval of that trial. They do two separate things. One, what the FDA does is make sure that the product itself is named correctly and that the way it's being studied is going to get the answer.
We even find this in the supplement world, what's in the bottle needs to be what is said is in the bottle.
The Ethics Committee’s job with the IRB is to make sure that the way this is done is consistent with what we've come to expect in the United States, that someone is not taking unacceptable risks. It's complimentary. By having both of those together, we do right by people to make sure that they're getting the type of care they should be.
Can we talk about the types of things that may be beneficial for cell therapies? I shared with you that I have ITP. I have clients that have gone for arthritis, other autoimmune disorders, degenerative knee disorders. We've seen in other countries a lot of therapies around viral infections, chronic EBV, CMV. Are there things that are perfect for this or we're not sure yet?
We're not sure yet, but if it's studied well in a clinical trial, it's okay. There can be a variety of things here. That's part of why it's helpful to have something that's FDA-authorized in the US. We've given it the go-ahead. It's an FDA-authorized trial, or it's been approved by an Ethics Committee because what that says is enough people have thought it through that it's not a harebrained idea. That's what you're trying to get at here. This concept of certain types of stem cells having immunosuppressive properties, there's a fair amount of scientific evidence that would tend to suggest that.
Has it been documented beyond all doubt yet? No. That's why we're still seeing clinical trials ongoing with this, including things that have even been blocked before the agency in terms of new drug applications or biologics applications. It's a very exciting area. We don't have definitive answers. That's why, if you are participating in a trial, it's good to be participating in one that's going to help generate knowledge and help keep you safe.
I obviously have a particular passion for the autoimmune world because it's something that's affected one of my family members and me. Is there something in all of your vast experience that you get excited or interesting to see how this is going to pan out with?
The whole area is exciting. Personally, I'm very excited to see what will happen with the immunosuppressive properties, perhaps of some of the stem cell products. Even some of the cellular products that we see use, whether exosomes or cell fragments may have some immunosuppressive properties. We don't know yet. It'll be interesting to find out. Some of this is a matter of having them studied well, having this study in a given condition, and having people have the courage to study them in conditions where they'll get an answer.
One of the problems here is that a little bit of what goes on in regenerative medicine is a little bit like what goes on sometimes in British law. British lawyers never asked your client if you're guilty because you don't want to find out. Sometimes people don't seem to want to get an answer because if there are some diseases or disease entities that are perfect for getting an answer to this, you don’t have to do biopsies, you just take blood samples.
That's part of what has gone on here. The field needs to have the courage to fail. When I worked in the industry, one of the things I did besides doing early phase development was they realized that I could not insult sponsors. The businesspeople used to take me to do business deals and licensing visits. What I got to see was a lot of different things, but I also realize that about 1 in 20 things that get into phase one make it through the process. A lot of stuff has to fail. The stuff we're seeing failing in regenerative medicine shouldn't be viewed as a catastrophe. It's just nature taking its course here.
It's the courage to try. That's what I'm appreciating in a lot of the people that I'm seeing involved in this. To your point, there's people that are maybe way far out there, but maybe those people have been forging the path for people to have reasonable and science-based research around some of these therapies that are very exciting to see. I have this massive community. We do this thing, as we call it, a Dear Dr. Sanders letter. There was a physician that we work with within Ohio, that we were having a difficult time getting care for one of our clients. Every time they walk in, this appointment would be super short, and they couldn't articulate what they needed. It was a miss all the way around.
She was going to fire the doctor and change her practice. We felt like she was in a good place. We helped her formulate this doctor's letter. Our whole community uses it. What we do is we have people fill it out and it says, “This is what's going on with me. This is what I'm dealing with. This is what I'm hoping for in my health and wellness.” They print it out and give it to the doctor before the visit. We always have to send it ahead and it helps people effectively communicate their vision for their health.
If I were to give my community some instruction on how to fill out their Dear Dr. Sanders letter, you're saying, “I'm dealing with ITP or lupus or Crohn's or rheumatoid arthritis.” I want to broach the subject with my doctor. I don't want them to think that I've got this harebrained idea. I want them to take me seriously. I want them to take these trials that I've found on this website seriously. How could we distill this down onto our worksheet? How could we communicate that effectively? There's a huge divide between trials that are there. They are good trials. People are getting access to them. How could we communicate that?
I'm going to step back and give you a long answer. First of all, the Dear Doctor letter should probably say, “Doctor, how do you think I'm doing in the scheme of things?” Part of this is understanding. Sometimes patients can be doing well. They sometimes don't understand how well they're doing. For instance, for patients with ITP, with a platelet count of 75,000, that's great. For those of us who had to struggle with patients who can't get a platelet count above 15,000, that is stellar.
I wouldn't call it perfect, but I would say not bad. Could it be better? Yes. That helps you understand, are you doing okay? Good, not so good. That makes me at least help a patient understand what they might be willing to take the risk for. It helps me have that conversation. If I haven't been able to use my therapies to help someone's platelet count, let's use that as the example, say at a reasonable level, that will make sure that they don't have a bad bleeding. For those who might be reading, platelet counts are these numbers, but usually, one’s platelet counts and people with ITP get much below 30,000. Once they get to 15,000, we start to lay awake at night, as doctors can get worried. Even though usually nothing bad happens, but we worry.
I know my community is reading this. There are so many symptoms that I can share with you associated with maybe even if you fit into that paradigm of your labs being okay, or acceptable or not scary. There are so many things that lie in between there. The patient 24/7 lives with that, whether it's a symptom profile or a less-than profile.
Some people with ITP have lupus or other autoimmune diseases that are driving it. What they need to do is think where does your disease lie in the scheme of things. The question is if I were to use one of these therapies, would it interfere with what you're doing? That's oftentimes like a key question because if the answer to that is no, then why shouldn't I try it?
How do you consume an elephant? One bite at a time. Take a little bite right here and see if I can distill this down. If an individual is on therapy like prednisone or an anti-rejection drug-like Imuran or CellCept or something of that nature. Collectively people felt that mesenchymal stem cell therapy would not interfere with that particular medicine protocol. If I'm hearing you correctly, we're thinking that this would be how we would broach it with a physician.
I had a client come in one time and say, “I'm done taking my medication, help me find or navigate. I don’t care where we go in the world. I don’t want to take this anymore. I want health and wellness.” I said, “Hold on, breathing is important. Your platelet levels are important. This is a process.” What I want to bridge for our community is that there's not one or the other, but that it can be complimentary. Regenerative means to bring on the new, create the new, regenerate something that has gone awry, regulate the inflammatory and immune system in that situation. How can a person say, “Doc, I've been on this website. I see that there was this trial at University of Florida or Mayo Clinic. I'd like to broach this conversation?”
They can come with the trial. They'll be inclusion and exclusion criteria. That's all the point of the trials because the trials will say, “It's okay if the person’s on prednisone, no problem. You can stay on the current dose of prednisone” because that's the beautiful thing. If it's working, they should be able to taper the prednisone. That's the whole point of clinical trials that are written. It's a protocol that's done the same way. The idea would be that, “If the platelet count seems to be going up, you can taper down the prednisone.”
Would that be in the IND? Would that be in the paperwork?
The clinical trial itself that the physician is following and that clinical trial, someone will write, you can decrease the prednisone as you move along if the platelet count comes up. That's how some of the therapies, if you think about the platelet raising therapies, let people stay on. You couldn't take people off of it, but you could reduce the steroid treatments.
I can see my community members now that are going to go print out the studies and highlight them and take them to their docs. Do you think the most compelling reason that a physician would support this decision in a patient is because they are FDA-authorized? Do you think that is what's going to move us across this chasm?
It's going to help. Is it going to be perfect? No. I've had people come to me with FDA-authorized trials. It's a very good start. I’m like saying, “I'm going to someplace that does not have a stringent regulatory system. I'm going to try something there that doesn't have any relationship to anything FDA is doing.” This is something that we've said at FDA. It means people have thought about this when our folks say that a clinical trial can proceed, they’ve read through all the stuff about the drug that's being used or the biologic that's been used, they looked at the clinical trials protocol very carefully.
They think about it and say, “This can proceed or can't proceed.” If you're getting it, if it's out there, it's because it could proceed. It has a certain stamp that doesn't guarantee that it's going to work. If we knew things were going to work, we won’t have to study them. At least it says that somebody thought that what was being done is possible. It's not likely to hurt people.
It was brought to my attention that some colleagues of mine are participating in this website called True Trials. What was fascinating to me about it is it felt like a safe meeting place for physicians that are passionate about regenerative therapies and progressive, maybe even sometimes out-of-the-box medicine and patients that are looking to participate in some of these FDA-authorized trials. How do you view this particular website, and what service do you think it can provide?
We've talked a little bit before about the hit-or-miss nature of what happens if you go overseas to get a trial that is well done and done properly in terms of both the scientific and ethical aspects. This concept that’s being done on this website is this concept that the hit-or-miss is taken out of it. It doesn't say that the trial is going to work necessarily, but it does say that the trial has been authorized by FDA. It does say that an ethics committee or institutional review board has done what it's supposed to do. It takes at least that hit-or-miss nature out of it. You can at least rest assured that those key things are taken care of.
It's this meeting ground and this coming together of people that have these progressive passions and ideas in medicine and patients that are in need. I have to ask, and our community was having a deep conversation about this. I'm not necessarily asking you to predict other people's thoughts, but do you have some insight in your position as to why we don't see physicians recommending or referring out a lot for trials, even specifically in the regenerative therapy world but in trials in general?
This is a mindset that we've had in the United States. In a lot of cases, it's about the fact that we as physicians take a lot of ownership of our patients, which is a good thing. It also means that sometimes we don't let go when it's in the best interests of patients for the trial. It takes a certain amount of maturity and courage as a physician to say, “I've gotten you as far as I can, but it's okay. You can try something else. I'll support you through it.”
That's a very amazing way to look at it. In my case, in particular, my docs or my partners in my process of being well and getting healthy. I sought out individuals that had the same vision for me as I had for myself. In this case, would it be a good idea for physicians to participate in True Trials, physicians that are doing FDA-authorized trials? Should they put their research here and their information here?
This type of system is very helpful for patients. Right now, ClinicalTrials.gov is great, but it's got this large listing of trials. We're not able to police it at FDA to make sure that those trials, every trial is in there. You don't know whether it has an appropriate IND in place. Some of them, you don't even know if it has set up the right IRB or ethics committee review. Whereas this concept of having a site whereby somebody has gone through the trouble of saying, “We have gone and looked, made sure they have that Investigational New Drug Application in place.”
“We've made sure they have the ethics committee or Institutional Review Board approval.” Taking care of that piece of it helps essentially winnow down a lot of the issues. It’s helpful to patients to have all that uncertainty, especially in this area of regenerative medicine where there are some predatory practices that have been used with ClinicalTrials.gov, and that's been in the papers. This is a way of essentially eliminating that.
There's a lot of good things in that space. They are potentially could be bad things in that space. What we're looking at for our community are resources where some of the vetting has taken place. If it's going to be there, we know X, Y, and Z have been addressed.
I think of it as de-risking. We do that a lot.
The other thing is efficacy. I'm going to shift gears here. You are the leader in efficacy. We want to make sure that things work well. As you know, I am in the nutrition field. In any aspect of regenerative medicine, would it be safe to say that a healthier patient is going to have a better result?
That's a well-known fact in medicine, is that people who are healthier overall will do better generally.
That's why I always feel like I've been able to navigate these amazing relationships with physicians that a variety of different universities and institutions is because our goal as a community is to provide you as the physician with the healthiest patient, with the most nutrient-dense diet and micronutrients available so that the body can have a healing process or a health trajectory that's in a better direction. That's awesome. We both agree. Nutrition's awesome.
By de-risking the process of an individual looking into a trial, do you think that this is going to elevate the field of regenerative medicine because, to your point, regenerative medicine has had some negative issues in the past because of people that I wouldn't say are progressive, but are more maybe irresponsible with a twisted renegade.
It can help us here. The reason why is if enough trials get done carefully, eventually there will be trials that will be successful, and the field will start to progress into a place where you now have successes. There will be improved products. You’ll generally find people coming behind those products with more trials, and the trials should get better over time.
As we have trials that are successful, people will understand the science better. The next trials that come up behind them will hopefully improve upon the earlier trials. That's how we like things to work in medicine. It's taken a while to get there in regenerative medicine, in part because cells are complicated things. It's not easy, but it's not hopeless either. We can get there if we work systematically.
Anything that is organic in nature has such bio-diversity and more of maybe a profound impact within the body because maybe the body recognizes it more as self. There are many concepts around cell therapy that are fascinating, from biochemistry to physics to oncology. It's a fascinating study. Fast forward ten years from now, is it your vision? Is it your dream? I'll share mine with you that an individual that's dealing with lupus or ITP or Hashimoto's thyroiditis, those cell therapies are part of their protocol or a choice?
It may be a cell-based therapy or cell-derived therapy could very well be some part of that modulation. If we can find things that can do what our current drugs are doing, they can do so less toxically all the more the case. As somebody who has given a lot of prednisone in my day, there were a lot of people who would like to avoid chronic prednisone for many reasons. Avoiding diabetes, weight gain, and all the other stuff is among them. It will be hopeful that will be a way that things will progress. Do we know that it will work for sure? We don't, but it would be great if we can see things progress, whether it's a whole cell therapy or parts of cells, or even if it's the insight that we derive from this that allows us to deal with other biologics that help people. That will be great.
I always say that prednisone was the very first love of my life. It was one of those drugs that pulled me out of a horrific health crisis. It saved my life probably. It also took me to the brink of kidney failure. I had a negative response to it, unfortunately. Because of that, though, it drove me to go back and learn more about my biology and biochemistry, and fall in love with what can be supported and nurtured within my body. That's why I said it became my hematologist best friend.
I get so hopeful when I see these trials coming into the United States, as an individual that has helped thousands of people navigate their health journey, We are so biodiverse as people. Our responses to drug therapies, cell therapies, natural therapies, or nutritional therapies can be so different. When we try to put an individual in a box, we restrict the true potential.
In our community, we believe that the healthier patient that we can deliver to the physician, the better the outcome is going to be. We see this time and time again with the reduction of inflammation and increased fertility. As a physician in your role, why is there such a profound impact in people when they have strong sound nutrition?
I'm not a nutritionist, but as a physician, who's taking care of a lot of people, people who have good diets, take care of themselves, exercise regularly, they tend to do better. It is one of those things that we see the body gets taken care of well and gets what it needs. It tends to do better no matter what therapy you give to it. That's the fact. We see this in clinical trials. That’s why clinical trial sometimes even stratifies people by their weight, exercise levels.
If you compare groups and you didn't do that, and then you had one group that was all exercising, eating wonderful diets, and another group that was couch potatoes, eating potato chips, and drinking soda, even if you gave them the same therapy, you might get a different outcome. You have to control for those things.
It's all about fertilizing that soil to grow healthy cells, healthy tissue, and have healthy blood counts. It's about nurturing that.
It's these people taking care of themselves. It's an indicator of whether people take care of themselves.
It’s in a hopeful way. I'm fascinated by this True Trials website. For me, and I'm not good at these things, it's very easy to navigate. This is where we sent a lot of our community to look for trials that might pertain to them. Universities are where the trials are occurring and happening. Why would it be important for a patient to have the safest space like this to go?
There are so many websites you could end up on by accident. It's nice to have a place to go that is trusted. That's helpful not just be wandering the wilderness of the web looking for things but to have a site that has some clear validity to it, that's taken the time to do the right things of making sure that all the right parts are there for people to go to. When they get something off that website, they feel like, “It's something I can trust.”
It's much needed. A lot of my doc friends that have this passion and desire and are participating in these types of clinical trials are sometimes having issues with the recruitment of getting their trials filled. Is this a great place for doctors to position their trials to place in there? How is that going to help them get their trials filled?
You'd like to think that if you have a site that people start to trust, that will draw on traffic eventually to that site. Trials will fill a lot more quickly. People are going to go there, hopefully, because they know, that's where they can find trials that meet the criteria of being FDA-authorized and ethics committee or IRB approved. That's going to help. More people that go there and more people see trials, more people get on the trials, trials get done faster, you get answers faster. That helps the field of regenerative medicine overall because then you start to get the answers that you need. Things are going to fail. It's going to happen. There's no guarantee. The part that’s important about these websites, it's not a guarantee that the trial is going to work. To your terms, it's a field that things can grow there. That's what it's saying.
It allows for people to know that they have an option in the United States. It can be lonely going out of the country. It can be extraordinarily expensive going out of the country. Are these trials something that when a person participates in the trial, is it at an expense? Do people have to pay to be in a trial?
In general, people do not pay to be in these trials. Clinical trials should be covered by the group that's doing them. There is patient-funded research. Don't get me wrong. There are some times that it gets done. The way these trials should generally be done, when it's patient-funded, it's the foundation. Getting them on the foundation then spends the money. It’s not moving there. There are trials that the FDA sometimes does give approval for people to recruit the cost of the product itself that is being used.
The patient's insurance might have to pay for that product. That's only in specific cases where they have come to the FDA in advance. Many of the trials are done not at a charge to the patient. Standard care of things is often charged to patients or insurance or things that would have been. Things might be done anyway, and standard of care might be charged to the patient's insurance, but the things that are done specifically for the trial, generally, the patient should not have to pay for it.
If a patient were to go into a trial and didn't have insurance, but part of the standard in the trial was to have labs before and after, like an ITP checking for platelets, would that be something that typically they would work into the trial?
That would usually be worked into the trial and the investigators would have to work through that.
It wouldn't be like, “We can't run labs.” That's why it's so important because there is a standard here where maybe we're doing this in some other setting. Maybe they wouldn't have the labs checked. I'm open and receptive to people seeking out what's right for them. What I've found so many times, which has always kept me up a little bit at night, is oftentimes, people will do therapy. They'll come back to me to this country without having an additional follow-up call. We all ponder. We work hard with their physician stateside to say, “How can we pseudo put together some hypothesis or what parameters would we look at to see if this was first and foremost, hopefully not a negative impact on the body, but also potentially positive ones?”
This is very exciting to see that. I know my community members are going to be printing this out and highlighting all the things and having these great conversations with their doctors. Specifically, on True Trials website, if they go in and navigate the website and see a trial, would they print out information and take it to their doctor? Would they contact the clinic? Would they contact the places doing the trial? How does the logistics work? Walk me through it.
That you'll have to get from the trial site, the idea is that you find a trial, and there are ways to set off motion to contact the people who are involved with doing the trial.
Primarily, your role is to say that, “They're not going to be put on this website unless we have blessed them as far as they are following protocol, they have a particular protocol that they have to adhere to, and we've vetted the process and said, ‘Go forward and let's see if it’s going to work.’”
That's what's being said here. Trials would not show up there unless they made sure that the FDA has given the authorization that they can proceed and ethics committee or IRB approval is there.
I am so excited about this and to have someone like you watching over the process is going to make it such a profound endeavor. I'm excited for my community, for myself, and for the future of medicine. I want to thank you.
Thank you. We have a whole group of people that keep a close eye on these things, ranging from the scientists who review trials, compliance officers who go out to make sure that things are what they say they are. There's a large team behind this. It's a pleasure.
Thank you.
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Important Links:
- ClinicalTrials.gov
- Investigational New Drug Application
- CBER
- Investigational New Drug Application
- Center for Drugs
- Creating Your Health Wishlist
About Dr. Peter Marks
Peter Marks, M.D., Ph.D. is the director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration. The center is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies.
Dr. Marks and center staff are committed to facilitating the development of biological products and providing oversight throughout the product life cycle.
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