Marc: Physician, FSHD Muscular Dystrophy, Pathway to a Cure

Episode 11 May 10, 2020 00:56:45
Marc: Physician, FSHD Muscular Dystrophy, Pathway to a Cure
Freewheelin with Carden
Marc: Physician, FSHD Muscular Dystrophy, Pathway to a Cure
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Speaker 0 00:05 So they had the scooter at my disposal to use and then they also got me dragon dictation, which is a dictation software, allows me to do my medical documentation and then I had similar experiences with my residency at Spaulding rehab hospital in Charlestown. They were very good at making sure that I had everything I needed. Speaker 2 00:23 That's so awesome. It's really great to hear how schools are being more adaptive, more inclusive, more accommodating of your needs. They're being proactive about it. I think we are entering this paradigm shift at least in the last 10 years where it's kind of just expected. Speaker 3 00:41 Welcome to freewheeling with carton. This podcast share stories of people with various disabilities and shines a new light on accessibility topics. Our goal is to knock down barriers so we can roll through life a little easier and build a community. To do this together, please rate and follow this podcast or text card at (470) 588-1215 with comments and suggestions. We welcome you on your journey towards inclusion for all and now your host, Carden Wyckoff, global disability advocate and wheelchair warrior. Speaker 2 01:15 Welcome back to another episode of free willing with carton. I have Mark with me today joining virtually. Welcome Mark. Thank you so much for being here. Speaker 0 01:24 Thank you for having me. Speaker 2 01:26 And tell me a little bit about the disability that you have and also the industry that you're currently in. Speaker 0 01:32 Okay, so I have at the safety as well, which is for those who don't know a muscular dystrophy, I am a physician that works. I'm specializing in both physical medicine and rehabilitation and neuromuscular medicine and have recently made the transition to working in the biotech industry where I help develop drugs for rare diseases. Speaker 2 01:57 And that's a wonderful, you kind of like being on the other side, like me being a patient and also being a family member and waiting for this treatments. You're basically the people that I look to the hope waiting for those next news updates when drug trials and when other drugs are going to come out onto market. So I'm excited to learn a little bit more about what your journey has been like, how you got to where you are today. So tell me a little bit what it was like growing up and when did you kind of realize that you wanted to go into the medical field? Speaker 0 02:36 So growing up my mother also has FSHD so when I was very, very, very young, she was diagnosed. So we kind of had a suspicion when I was even small <inaudible> and I was able to run and I played hockey and baseball is that of skier? Mmm. I had some facial weakness and were like the doctors thought probably, but it wasn't until really I was around or 11 where I started having more significant symptoms, which is when kind of the slew of doctors started. I started seeing lots of different doctors and I was exposed to the medical field fairly early age just to try to kind of do the full assessment and get all the screening tests done and all the, uh, just making sure that everything else is working okay. So I, so I went to a lot of doctors then and it was, that was really my first exposure to the medical field and being able to see doctors. Speaker 0 03:26 I mean I saw my pediatrician every here, but other than that, not really much exposure to medicine. And from that point on I started getting more interested. I started reading more about it. My, as the muscle dystrophy progressed during my teenage years, there's a lot of things that were changing and things were getting more difficult and there was some some concern about going into the medical field. This is something that I was interested in but your normal vision of a physician is not someone that it's sick. And I was concerned that there was this last aspects of the job that might be a little bit challenging. And I remember I had this great conversation with my pediatrician where, so I was still 17 or something and I was looking at colleges and and I went to him, I said, I kind of want to be a doctor but I'm worried like do you think I can do it? And he came back to me with like absolutely being a doctor is all about thinking and being able to help people and diagnosing and the physical aspect of things can be worked on. And that is not a barrier. So that was really helpful in my, a journey to get into the medical field and he was very encouraging of that. And I continued to work on it. And with the college I, I did biomedical engineering, which allowed me to go and apply to medical school and how it went. Speaker 2 04:42 That's so great to have someone that allayed all those fears because I was premed in college as well. And that was always something on my mind of, you know, what is medical school going to be like, what are those physical tasks that you're going to have to do and what I need to, certain accommodations, what I not be able to graduate. Um, and so what were some of those things that you relied on throughout medical school and in your residency and postdoc? Speaker 0 05:11 So I've gone through many different kind of iterations of this, starting at a new school meeting to talk about accommodations and things. And at my medical school I was the first person at that school to have a physical disability that they had to deal with. <inaudible> it was, it was near them. And it was a learning process and we kind of had to work together and there was just certain things that we wanted to portray to the physicians that we're working with. The first two years of medical school was all in the classroom. So it was all just book learning. And I did that all through college. That was fine. But the second two years are you're in the hospitals, you're doing rotation, you're working with patients and some of those rotations are things like surgery and that requires you to stand for long periods of time and use your hands. Speaker 0 05:55 And those are things that I was worried about cause I have some hand weakness. Um, I'm still able to stand, but I was not able to stand for long periods of time at that point. And so just the concern of, of going into an operation that would take six hours and, and the expectation of medical students to stand there and hold something for six hours, which is not realistic. So we worked with the, uh, the physicians and there was combinations made. I was able to, if I needed to at certain points of the surgery, leave for a little while, then come back. Things like that. Fairly mild things, things that didn't really affect the overall kind of experience as a medical student. I knew going into medical school I was not going to be a surgeon, so it was a little bit less, uh, important for me to be there for the whole surgery. Speaker 0 06:38 I could do all the things like go take care of the patients on the floor and, and help out with the interns and things like that. But what really impressed me is when I got to residency. So residency is a match process. So you kind of interview at a bunch of places and then you, you, you ranked your list and then you get mashed into a certain place to go. And so I did physical medicine and rehab, which it's basically the medical profession that takes care of patients with disabilities. But in order to get into that, you have to do an intern year in internal medicine and they're kind of separate. So my intern year I did a a little kind of semi city sound like community hospital in length at all called Lincoln hall. It was in Pennsylvania and I had a really great experience with the, the, the, the leader of that, a residency because I matched and then two hours after I got my letter saying I was going to Lincoln off my answer and yet he called me on the phone and was very excited about me coming and he was telling me how he's going to bring me Lincoln all even before I start, we're going to talk about all the things we need to get done. Speaker 0 07:41 He's going to have me shadow an intern, we're going to get all the equipment in place on day one. You have all the supplies and all the technology that you need. Two, be successful as an intern. And I thought that was a really great approach. He didn't, people required me to reach out. He just called me and said, you tell us what you need and we'll get it. And some of the accommodations, um, that were well supply where things like, uh, they got me a electric scooter for longer distances because the hospital's a big place and although I'm, I'm walking and standing, um, to get from one end of the hospital to the other in the event of an emergency, like a code or something will take a long time to walk there. So they had the scooter at my disposal to use whenever I wanted. And then they also got me dragon dictation, which is a dictation software allowed me to do my medical documentation without having to type, which takes me longer because of some of the weakness in the hands. And then I had similar experiences with my residency here in Boston. I was a resident at Spalding universe, Spaulding rehab hospital in Charlestown, which is a big rehab hospital in the city. They were very good at making sure that I had everything I needed. Speaker 2 08:53 That's so awesome. It's great to hear how schools are being more adaptive, more inclusive, more accommodating of your needs and they're being proactive about it. And so like you really have to beg to get these accommodations or you have to prove to them that you need them. I think we're entering this paradigm shift at least in the last 10 years where it's kind of just expected, right. To allow for accommodations. Um, because of the disability rights act that we've in the United States and elsewhere in the world. Speaker 0 09:31 Yeah, I think it's great that they really tried. But one thing I will say is that they, a lot of times the schools really have, I have no idea what you're going to need and they really rely on you to be partnered with them too. Tell them what they, what, what you need and what you, what you think would be helpful going forward. Cause everyone's disability is a little different. And I think that was a misconception I had initially. I was like, well, you should know what I need. Um, I don't know what goes on at the school. I don't know what I'm gonna have to do from day to day. But in reality, that is something that is known and you can figure out. They don't know. They don't know what but what you need. So to be successful and you really need to partner with them on that to help advocate for yourself and get the things you need. Speaker 2 10:11 And what advice would you give to those who aren't good or not familiar with how to advocate for yourself? Because a lot of people, at least I know in the disability community sometimes are afraid of asking for help or feel like they can do everything themselves. So how would you help people who are struggling through that? Speaker 0 10:29 Well, I would probably say is that first of all, asking for help is in no way lessening your experience in terms of the education or the the learning opportunity or the growing of yourself. And you want to ask for help ahead of time if you can, because if you go into something saying that you can just do it and you'll get by. And even though that you're not able to do it to the level or to the, to the extent that everyone else can, but you could have experienced it more fully if you asked for a little help or a little assistance in one thing or the other, you're just missing out yourself. You're just, you're losing out on that extra bit of experience you could get if you, when and and reached for it and ask for it. With regards to how to reach out that can, it can be a challenge because every place has a different process to and kind of figuring that out. Different leaders have different strategies, but I don't think it's ever a bad thing to go to whoever is leading the program or supervising you to just say, look, I think I need some help with this stuff either. So I talked to you about it or who do I go to talk to and they should be able to advocate for you or at least be able to help you and guide you into the direction of where you want to advocate and where you want to talk and like who do you need to talk to. Speaker 2 11:46 And I'm also curious about what the workload was like for you and obviously enough sleep in medical school and having a disability, especially in progressive disease, right? Where sleep is so vital, good nutrition is vital. Making sure that you're in good, healthy spirits as well. So can you talk a little bit about that? Speaker 0 12:08 Yeah, that's, that's interesting. In the medical field, that could be tough, especially during the intern year, working long hours. Well, I will say as an intern, I didn't do much else other than work and sleep. That's the, that lasts about a year. Uh, I what gets to the hospital depending on the rotation between six and 7:00 AM and I wouldn't leave until about six or 7:00 PM and then I'd go home and I would eat dinner and then I would do a little bit more work from, it's a nine or 10 and then I'd go to bed and that was kind of my daily routine. You'd have maybe one one day off a week at most and you just kind of get into that routine and that there are breaks in throughout the year when you're on elective and you have a little bit more time. But um, I think he does get into that mindset of just like doing it over and over and over again. And as an intern, I didn't have any overnights. And then when I got to my second year, then I started having overnights and I did okay with the staying awake for 36 hours apart. But then I would crash afterwards and I would sleep for, for 16 hours. 17 hours. Speaker 2 13:15 Wow. That's a very long time being awake. Speaker 0 13:18 Yeah. It's maybe not 36 would be a long, long day, but normally it was like 28 to 30 hours. But we'd do it. You'd go to work in the morning, you work all day and you work all night and then you finish up in the morning and you go home. And it's, I think is like, it's a stop and go kind of profession. Especially when you're in the hospital and you're doing these overnight shifts. You're, you're, you're working hard. And then there's times when you're kind of just like mindlessly putting some stuff into the computer and then you work hard so you have little breaks in there and you're not doing it alone cause you're with your nurses and your health staff and, and there's so many other people around you. You just get energized and you want to help the patients and you're during that or just in that environment that you just want to do it. Speaker 0 13:59 But there are days when it's hard and there's days when you're tired. But, uh, I did my best to, there's some people I know that I trained with that would just, if they were on, they just stayed awake and they didn't try to sleep. They didn't try to do anything. They just were awake and they were on. If I got little, like 30 minute hour gaps in the workload, I would often try to, to uh, go to the call room and take a nap and sleep for a little bit and kind of arrange it so that I would have those little gaps if needed. Speaker 2 14:26 Nice. And then so you shifted a little bit, right? So you went through medical school and then you did, you're after medical school, you went into rehab, musculoskeletal degenerative diseases. What, what sparked that interest? Was it because of your own personal disability? Speaker 0 14:47 So I think so. It's hard to tell because you have so different things that influence you in your life. <inaudible> I was in physical medicine and rehab that was definitely sparked by my disability and my own situation. But I got into that and I started really looking at different areas and aye within the neuromuscular realm. I do EMGs as well, which I like and I mean I had, I felt like I had a lot of knowledge and I felt like I had a lot of personal experience that I could bring to patients and help patients in that sense and I just was very interested in the conditions I felt and the neuromuscular disease realm is an exploding realm where treatments are coming out every day, a couple of months for different diseases and I feel everything I've learned in fellowship last year is going to be obsolete and probably five to 10 years cause there's going to be treatments for all these conditions that I learned back a year ago. The only treatment was supportive care and rehab, but now with the treatments resume and Duchenne coming out and there's a lot of other diseases that are, are following suit soon with the same technologies that it's going to be really, I think a really exciting time to be in the field and to be involved in and to be able to help people. Speaker 2 15:58 It's such as an explosive field right now. We saw the SMA drug come out and Duchenne's has had a lot of activity with drug trials and now with FSHD the type that we have, we're in drug trials. So I'm interested in learning more about what it's like working for a pharmaceutical company and tell me what it's like on the other side because things obviously take a long time. And like w w I mean, just tell me a little bit about the whole process and why things do take such a long time. Speaker 0 16:35 Okay. Um, so my transition to pharmaceuticals was a little bit of a tough decision for me. When I was looking for jobs. I was looking at both like academic medical positions and pharmaceutical positions and I wasn't sure which way I was going to go. Ultimately I decided on going pharmaceutical one because they, uh, the pharmaceuticals allow me to have a day in the clinic every week, which I'm working on setting up now at six, a little bit of time to get set up, but I'm still gonna have that ability to see patients and take care of patients. But if I, if I'm able to work on one medication that gets FDA approved and is effective, I'll be able to, to help thousands of tens of thousands of people with that drug versus that's just a, it was a very appealing thought to me to be able to kind of work on that and take my knowledge and experience and work in a field where it kinda affects potentially so many people with regards to how long it takes. That is something I'm learning a lot about. Uh, I've only been in the pharmaceutical industry for about eight months now, so I'm still relatively new. But it is definitely a eyeopening to see why things take so long and it's, and believe me that the companies want to get their drugs approved if they're effective as soon as possible. It's not the date. Take a long time on purpose. It's, Speaker 2 17:59 yeah, Speaker 0 18:01 the studies take a while to design and then these studies have to be held at many different centers. So they're all over the world and each study site has their own process for approving the protocol and their own IRB, which is their internal route. The review board, which is a a board of physicians and scientists and layman and ethical agers that look at the post study and make sure that it's with, if it fits the core values of humanity and their university and every, every site needs to approve or the study, which takes a lot of time. And then you have to recruit for the study, which takes time as well because doctors are not seeing all their FSHD patients on one day and FSHD patients often only see their doctors once a year, maybe once, twice a year. So in order to fill the study, you have to be able to get those patients into the clinic to be screened and then to be incorporated into the study. Speaker 0 19:00 And that's where registries are really important. Help doctors kind of pull patients from those registries. Assault studies. But, but that's, I mean those are some of the things. And then once the study is complete, and believe me, if a study is positive, you're going to know about it because the company is going to be very vocal and very open about telling everyone that beta positive study. And then depending on what phase the studies at either have to do another larger study or they go to the FDA and, and the EMA, which is the equivalent for Europe and all that takes time and review. And the FDA, they have a tough job. The FDA, they have to look at all this data which was collected over three or four years. <inaudible> I make a judgment call of whether the drug is first safe and then sec second effective <inaudible>. Speaker 0 19:45 Okay. That takes a long time. I mean, I know just from me being in the pharmacy in the business for eight months, it's taken me a long time just to become familiar with the studies I'm working on. Whereas the FDA is not even working on these studies and they have to kind of review all the data and be comfortable saying this drug is safe. Okay. Thousands, hundreds of thousands of people to potentially take, which is a big, a big task. So it's a process and it takes time. Could it be more efficient? Sure. It could be, but I think everyone's trying to get drugs out as quickly as they can. And safely. It's a big, big thing. Speaker 2 20:20 Right. And I know with United States, and I'm not familiar if this is true with the EMA, but like you said, there's safety and then there's efficacy is, can you speak a little bit more about that? Why and United States sometimes we think drugs take a lot longer than the rest of the world. Speaker 0 20:37 So safety is such a big part of the approval process. And it was only recently, relatively recently, within the last of decades or even last decade or so, the efficacy was really brought into the picture. I in the past, if you could show that a drug was safe, sometimes it would be approved because as long as it's safe, what does it matter? But we're finding that more and more that these and these drugs have more and more side effects, that safety is not the whole picture. You need to have that to see, you need to have the appropriateness of the drug and the, the, the business I think, I mean the EMA is also relatively strict and in some ways more strict than the yep. Yeah. When it comes to approving medications, I'm not as familiar with the drug approval process in the rest of the world. Speaker 0 21:26 I think there is more, a little bit more leeway with when it comes to safety, safety and efficacy in some of those countries. But some of the things the FDA is doing, which is great, especially for rare diseases, it is, they have the fast track program. They have the orphan drug designation, which is allowing yep. These companies developing those types of treatments, kind of priority access to the FDA and priority reviews. So, and when an orphan drug asks for a meeting, it gets done much quicker than if a drug wasn't orphaned or, or uh, a rare disease get asked for a meeting much sooner. They get priority access and a little more leeway in terms of, so the standards, because if there's nothing else to treat the disease, then it's a little bit easier to get to get it. The approval. Speaker 2 22:12 And I know with those orphan drugs and the fast track, it gives so much hope for those families. Given our current situation right now and maybe you do or don't know about it, but I know lots of people want a vaccine for coven 19 and you know, what are your thoughts being in the medical professional field of why it takes so long and you kind of alluded a little bit to that. It takes, it takes time. Speaker 0 22:38 Yeah, there's actually a lot of things, and I'm not an expert in this by any means. I don't do vaccine development, but just the process. I mean they're doing their small phase two trials now, which are 40 maybe 50 patients where they're giving them the mean these are, these are healthy individuals giving them the vaccine. Speaker 0 22:59 Then they're going to have to test their hypothesis, which is which, which requires them to look at them over time to see if they get sick and to see if there's a control group. They get sick and if they think it's working, then they have to do the next step of, I'll test this vaccine in 400 500 people. Make sure that they don't see any trends in terms of side effects or risks of this vaccine. Because if you start rolling out the sex seed and start vaccinating millions of people, and then all of a sudden you start to see that 10% of the people are getting some terrible neurologic issue from it. That's a, that's a dangerous vaccine that's, that's a little bit risky. So you have to test this vaccine in larger groups, which we're still going to do. And even that, it's not going to take them, it took a year. But then you have to manufacturer the vaccine, which is a whole nother step. And we're going to need millions of doses of this vaccine to really fully counteract this virus. And that's going to take a long time to me. So if you think about all those factors, there's the timelines that have been put out there seem pretty realistic to me, although I'm not an expert in vaccine development. So Speaker 2 24:06 interesting. So vaccine development is different than a rare disease drug development. In terms of the production different or just the timeline or, Speaker 0 24:16 I think the production is different. I think just the size of the studies are different and in my rare disease studies that I work on, I'm usually looking at phase twos are about the same around 40 patients, but the phase threes are maybe a hundred 150 patients in neuromuscular disease at most just because if you're looking at the population they, this is not an patients out there to have studies larger and be able to recruit them in a in a a fair amount of time. And the FDA is aware of that. So when we come to and with a study of 120 130 patients, they're okay with that. Whereas other diseases, like if you're doing a study and high blood pressure, they, that's so common that they're going to want to see hundreds of, if not thousands of patients in that study. It's a really, we have a good sense of whether the drug's working or whether it's helpful or whether it's I'm having side effects. Speaker 2 25:06 Got it. And so when a drug is in development and in production, I should say, how long does it take to produce the drug? And then actually get it into the hands of patients. Speaker 0 25:18 So the kind of the timeline, and this is a scary number, but the timeline from from chemical development meeting in the laboratory actually developing the drug as a chemical all the way to FDA approval is normally between, I would say six to 10 years to really get a drug from cause. And that's including developing the drug in a laboratory, doing animal testing, going from animal testing to human testing, going through the FDA approval process. So it's, it's a long process and a lot of these, a lot of the drugs that you're seeing in clinical trials now were discovered in the lab eight, five, eight years ago. And it just takes time to Speaker 2 26:00 right Speaker 0 26:01 to get it through. And not all drugs go through that same process. There are drugs that have been discovered and were tested in other diseases didn't work, but now they're like, well, let's see if it works in this disease where we have reason to believe that it might help this other disease. So let's try it out. So in that process you have all the safety data, you have all the animal data, you don't need to go back and do all that again and you're able to just take this disease model and start testing a drug in those patients and that it can be a quicker the process. So there's many different ways of doing it. Speaker 2 26:31 Yeah. And I think that kind of route where, let's say one S one lab found a drug and they thought it was designed to treat a B, D but it ended up not. And then another lab found that and realized that it did help them. The fact that track to get that to into the hands of actual family members is much quicker. So what'd you say that is more common or would you say starting from ground zero is more common? Speaker 0 27:07 It's tough for me to tell that because I don't have a full picture of the entire landscape in neuromuscular, in the Northwest of the realm. I would say starting from ground zero is more common, mainly because the diseases are so rare and so specific. And I would say that's more recently in the past that hasn't been, I mean pregnazone was approved for Duchenne. Prednisone was definitely not developed for Duchenne that was developed for auto-immune and other things. Steroids. Okay. It was found that it works in my senior gravis. The same thing goes that these auto immune diseases, well my son gravis is an autoimmune disease, but they're using these treatments that were developed for other autoimmune diseases and just using them in my gravis and which is a neuromuscular junction or uh, another neuromuscular disease that causes weakness. So I think in the past there was a lot of repurposing of drugs in neuromuscular realm. But more recently a lot of the new treatments that are coming out are really targeted specific treatments for the disease. And in that, for instance, you really have to start from out of the bottom up. Speaker 2 28:10 This is so fascinating to learn about. I never really knew much about how the whole process works. So thank you for sharing. I'm also curious to know, so what do you specifically do in your, in the pharmaceutical field? So are you in the lab all day long or are you that's in drugs or you said you go to clinic one day. What does that mean? Speaker 0 28:32 Okay, so what I, my day to day job, so I worked, there's a medical associate medical director, so my job entails a lot of different things. One of my roles is I assist with the medical monitoring of studies meeting aye review all the patients that are getting enrolled in the studies and I reviewed the data that's coming in from, from the study visits and I am looking to make sure that there's no concerns about these drugs. So the medications we're treating the patients in these studies are not tested and then there can be unforeseen side effects and unforeseen adverse events and some of my job is to make sure that I'm, I'm looking for that and picking up on that and making sure that that that is brought the light. If something is starting to cause an adverse event that I'm seeing in a couple of different patients all across the country, I need to pick that up because if we have one patient that's at a site in California, one patient at a site in New York and one patient in a site, France, those individual doctors that are taking care of this patients are not going to pick that up as being maybe caused by this medication. Speaker 0 29:39 But since I'm able to see all the patients across all the studies, I'm able to kind of pick those things up. I'm looking at labs, things like that. Speaker 2 29:46 And what are some of those things that you're looking for? Maybe it's an increase in temperature or higher white blood cell count. Speaker 0 29:53 I mean those are, those are things. Yeah, exactly. So I'm looking maybe increase in blood pressure sometimes if you're looking okay. It really depends on the type of drug you're looking at. Well, you're looking at the liver function. A lot of drugs are processed in the liver after they're done doing their thing. Same thing with the kidneys. A lot of drugs are cleared by the kidneys and if we start seeing some numbers coming back from the blood tests of the, the liver, the kidneys that are not normal or concerning. Yeah, that is also, that is something that I need to watch out for. It also goes on the other side of things where I'm looking at looking for biomarkers or looking for signs that the drugs working or being helpful. A lot of the drugs, like in Duchenne you're looking at the creatine kinase or CK levels, which is a, a marker of of muscle damage. Speaker 0 30:37 When those are high, you know, there's a lot of muscle damage going on when they're low, you know that the muscles doing better. So we look at these numbers and we're seeing it. The numbers are going down from their baseline and if they're going down, that's a reassuring thing that maybe the drug is, it's helping or improving. Of course we're looking for a lot of things. We're looking for, uh, efficacy as I mentioned, which in that sense means a lot of things in terms of the function is, that's a big thing function. Making sure that the drug is not just causing a laboratory value to get better, but making sure that it's improving their quality of life, improving the function from day to day because that's really what's important. Speaker 2 31:15 Sure. Right. Keeping that quality of life is important and not have it. I do the reverse, so I'm also interested in knowing at what point do you see those adverse side effects in multiple patients and you're like, okay, we need to actually shut down this study, or do you, is it a hard closure or do you tell the researchers we got to redo this change tracks? How does that process work? Speaker 0 31:43 It's, it's complicated and varied based on the type of study you're doing and how serious the side effect is. So depending on the size of the, of the trial, in a phase two trial, you have a safety review committee, which has doctors that are, that sit outside the company to make sure that there's other people that can take a look at the safety data and kind of give their input and give their opinion. So it's not just the company making these calls. It's it's a group of doctors that are experts in the field where the company plays a role and then in bigger trials you have these, you have these committees that consists of doctors outside the company, whether it's where the or the company isn't even involved, so their, their, their liaison between them, but there's no one in the company that actually sits on that board to allow kind of unbiased assessment of the safety data. Speaker 0 32:31 When you actually look at the data, you're, it's a risk benefit assessment that needs to be done. You look at how much benefit you think the drug is giving versus risky. The side effects are now if you have a side effect of some mild, I don't know, mild itching after medication was administered that you might be able to live with if you're having a huge benefit on proving the quality of life or function. Whereas if you're seeing an increased risk of stroke or something, it's more serious. That might be something that you have to be more careful with. And then you, you do an assessment and there's many different levels of the assessment. You're looking at the subgroup of patients that are having those strokes. Are they different than the rest of the patients? Is this something that's predisposing them to have those strokes that we can maybe filter out from the rest of the study to make sure that we don't expose that risk or, or is it just kind of universal risk that was just unforeseen and we have to shut the study down? That could be something that's happening as well. Speaker 2 33:27 And could it ever be just maybe one person in particular reacts to it in an extreme way that you never thought about, but everyone else seems fine? Speaker 0 33:37 I think it would be relatively rare to stop the study based on one on patient experience because people react to things in different ways. People have different autoimmune reactions or allergic reactions. I think if we, if we have a patient that has a serious events and we think of as related to the drug, we would definitely include that in the, uh, kind of informed consent and any kind of drug label to kind of allow people to know that this is a possibility. But overall the benefits of the drug and the, the kind of percentage of people that have these serious adverse events are low enough and high enough or high enough and low enough respectively that the drug is still worth taking for the majority of the patients. And that's just, it's also something for doctors to say this happened in someone, patient needs to watch out and kind of you screen your patients for this to make sure it's not happening. So you can intervene quickly and sooner than later to kind of prevent any. Speaker 2 34:37 And I guess how much involvement does the pharmaceutical company have in recruiting these patients and these volunteers? Cause essentially you're signing up for the unknown. You have no idea how you're gonna react to this drug. Obviously you're doing it for science and trying to help cure your disease or to benefit others. So what advice would you give to patients to enroll in a drugs in a drug study? Speaker 0 35:02 So what I will say is that the kind of the pharmaceutical industry, they're not going to bring a drug to human trials until they have a fair, the amount of confidence that there's at least something there that they need to look at. I mean they, there is a sense of work in the kind of animal model, animal testing to kind of try to prove as much as they can in these animal models that there is something there that is being beneficial, that it's helping that is potentially going to help the humans. Now, animals are not the same as humans and there's no guarantee that's anything that looks like it works in animals is going to work in humans. We're much more complicated and there's different enzymes and there's different processes that go on. But the pharmaceutical industry is going to only bring things to human trials that they think are going to work. Speaker 0 35:53 So from that standpoint, you can have faith that they're not, they're just not throwing things out there because studies are very expensive and they take a lot of time and they're sure they're there. They're labor intensive for the company, so there's many people at, at a company that's working on this study and they're not going to want to do a study that's that's, they don't think it's going to succeed to begin with. That being said, from the standpoint of the direct patient interaction with the patients that are involved in the study, it's kind of a rule of thumb that the pharmaceutical, the employees themselves are not directly involved with talking to the patients. We try to supply as much information and as much support to the physicians at the individual hospitals as we can and we're always on. I'm always available. My cell phone is always available for any doctor and any of the sites that are participating in one of my studies to call me and ask me questions and talk to me about it. Speaker 0 36:44 Well, we really want to empower the physicians that are dealing one on one with the patients to be able to talk to them about the study, to be able to work through them because they're the ones that are there onsite. They're the ones that are going to be responding to the patients when they have questions or concerns and they're the ones that are going to be actually giving in administering the drug. We, and we were making it and we're issuing it, but they're the ones that are making the decision. It is safe for me to give my drug to this patient within the bounce of the, of our guidance. And so we, we really try hard to, um, and I was on the other end of this when I was, uh, a fellow at UC Davis. UC Davis has a big muscular research department and I was, I was the doctor that was giving the treatments. Speaker 0 37:25 I was the one helping screen the patients for the trials and helping pick the right trials for them especially. And I saw a lot of Duchenne patients at Davis and we had many, many Duchenne trials going on and picking which one is right for you is a, and also be challenging because often once you start one you don't qualify for any of the others anymore because now you're obviously study. So I've kind of seen both ends of that. And I think if you have questions, ask the doctor and they don't know something. They are, they have all the resources to come out and reach, reach out to the pharmaceutical companies to try to answer the question. Speaker 2 37:59 This is so cool. I just, I have all these like questions that keep popping up as you talk. Um, um, I'm thinking about, well, kind of going back to different drugs sometimes can benefit different diseases and they become aware in a, in a particular field. So is there some type of database that you guys are always scanning and looking at of different drugs that are coming on the market and realizing like, Hey, actually I know about XYZ disease that this drug may benefit from. Do you guys ever then reach out to researchers that are in that field to help match a drug to the disease? Are you like drug matchmakers? Speaker 0 38:46 So I don't do much of that, but there are companies out there that have developed platforms where they are, and I don't know the details about it, but I know a little bit they, they, they take, they have these big uh, and enzyme like panels where they're able to test a bunch of different drugs at once to see if it affects certain molecule or a certain process. And they had this big, big, uh, database where they kind of run through all these drugs and they, if they, if one pops up and they're like, Oh wow, this drug was used for to try to treat cancer, it didn't really work too well. But when we ran it through our model and ran it through our experiment, it looks like it reduces this expression of this gene and it may be helpful for, for, for this disease. Speaker 0 39:38 Okay. That's a model that's popping up in more and more smaller biotech companies because there are so many different drugs out there that are just sitting there because they, their initial indication was unsuccessful. And just the the company and the way a lot of biotech companies are, especially the smaller to mid sized one because they have one or two or maybe three at most kind of areas of expertise and that's the area they work in. So if they develop a drug and they try it out in their area of expertise and it doesn't end up working, then a lot of times that drugs will just get put on the shelf and not really do anything. But it will take another company to come and say, Hey we saw your data too bad. It didn't work for what you were working at it. But we have this expertise in this other area and we want to try it out. Speaker 0 40:23 See cause we think that it might have some benefit. And in those settings then there's, there's partnerships that are made and that's very common to have partnerships between biotech companies where they, they work together. And one company will be the manufacturer or the one that created the drug and other company will be the, the uh, area of expertise and you'll go into a partnership and they'll test the drug out and see if it works in those areas. And that's a very common occurrence in the biotech industry. Now that being said, could there be more transparency? Definitely. I mean w we the U S lives when we're in a capitalist, the system where these companies are out to make money in, could there be a central database where there is kind of all the status captain shared and openly exchange? Yeah. That, that, that could exist. Speaker 0 41:15 That doesn't currently exist. The way the data currently is shared is through publication and things being put out in journals. When I say there was a lot more data than what gets published at these biotech companies, there is a lot more data. Uh huh. So it's not always put out there, but often if, if a company, a lot of a lot of drug companies and pharmaceutical companies, if they see a drug and they see that it has this other effect that it might be working, they often will put but their dad out there so that other companies can compensate. Look, we see that this might be helpful. We want to try it out and in the same vein, it doesn't have to be other pharmaceutical companies. Often if a drug doesn't end up working for what we were using it for, but then there's a specific a researcher at a specific university that's like, well I'm doing this other study. I think your drug might work for me. At least my company is often open to that. We'll supply some drug for them to do some small trials on things and just looking for scientific discovery and finding any way that, that the molecules that we're developing can be used and that helps people. Speaker 2 42:18 And obviously these drugs cost money as you were talking about and how do you put a price tag on a drug that is successful. And then how do you get that into the hands of patients at an affordable price so they're not paying millions of dollars? And that's a million dollar question. I'm sure Speaker 0 42:37 it's a, it's a good question. It's a little bit outside of my area. I do see kind of the process a little bit. So there's a lot that plays into that. How much insurance is willing to pay for the certain drug. And again, it's a, the risk, it's a benefit cost analysis where you're looking at how much the drug benefits the patient versus how much the cost is going to be. And also how often the treatment is going to be. Especially now that we're getting these new gene therapy treatments that are, that our thoughts would be once a Don may, maybe not, but um, where and they're coming out with Mo with million dollar price tags but you're putting a price on, you're trying to say that this medication is the only medication we'll have to take and there'll be able to basically, I mean in the case of SMA one survive and otherwise they wouldn't. Speaker 0 43:26 It's really hard to put a price tag on that. That being said, I was at a conference a couple of years ago and there was this company that does these really in depth and I can't recall the name off the top of my head, the name of the company. It does these really in depth analysis of looking at the benefit that a drug gives versus the the cost of the drug. And some drugs are overpriced, some drugs are under priced, some drugs are priced just right. But what I will say is that they did some analysis of some of those hundreds of hundreds of thousands of dollars a year of drugs and because of their benefit, the price is right and that is where it should be. And unfortunately the health insurance system is not in a position where it, it makes it easy for people to always get them. And I think that's where the reform needs to be. And I mean the drugs are developed at a cost and that cost is not funded by the government. Speaker 2 44:21 Yes. It's like the glass store for drug pricing. Speaker 0 44:29 Yeah. I mean definitely. If you think if it, if it takes 10 years to develop the drug, you're, you're dealing with 10 years, you have 10 years of all the people salaries that worked on the drug and here's of materials and costs of, of running the studies and doing all the analysis. And so that that gets expensive. Um, and now, and what I say is there are companies out there that, I mean there's a price tag on the drug, but then they also have other programs to help people get the drug if they can't. I dealt with a couple of them when I was a physician treating patients and UC Davis open access programs for, for patients where their insurance just does not cover it or, or it's taking too long to get through or this, they're putting up so many red tape that is, can't get the insurance to cover it. There's options for getting on medications a lot of the times ahead of time without immediate payment and then continue to work on getting insurance coverage. And those are great programs and they, they allow patients to get on drugs earlier than they would normally be able to get on drugs. Speaker 2 45:33 And getting to that next step. I know I was actually invited to come speak at this panel in mid April, which obviously has gotten canceled because of travel restrictions. Um, but it brought patients, FDA, pharmaceutical clinicians, researchers, all in the same room. And to me that was so fascinating. I was really looking forward to doing that. What benefit do you from a pharmaceutical standpoint get out of around tables such as that? Speaker 0 46:06 So that's interesting cause cause I would bring many hats to that table. Right? Cause I would have the pharmaceutical, I would have the physician standpoint and I have have the patient standpoint all at one. So from the pharmaceutical standpoint, I think it's valuable to hear from all all people. What I will say is that pharma, at least in my company, the pharmaceutical industry does kind of mini round tables on a pretty regular basis. We often bring insurance experts into the company to talk to us about that kind of thing. The one thing that we do do what we do at my company, which is I think is great, is that every two to three weeks we bring a patient, the Boston who was on one of our studies to talk to us about how the study has affected them and to give us that perspective and, and how, and I've had a lot of that perspective being working as a physician and giving drugs, but I think it's good for the rest of the company that is not a position to kind of see that they get that kind of exposure. Speaker 0 47:06 And then at the same time whenever you're working on a study, you're, you have a stream of conversation with the FDA and, and things like that. But having everybody in one room would be really helpful so that everyone could hear what everyone's saying at the same time. Cause often there's a middleman and I feel that the pharmaceutical company often plays that role where the patients are telling them one thing. They go to the insurance company and they're saying, well, if we can't do that, this is what we <inaudible>. I think it would be more powerful if the insurance companies just hear from the patients and the FDA hears it from the patients, the pharmaceutical companies here for the patient, so everyone's really hearing it from the, the really the thing that matters, right? The thing that really matters is the patient and improving their function and quality of life and treating their condition and then all this other stuff is just the way we, we achieve that goal. Speaker 2 47:51 Yeah. Speaker 0 47:52 We have a system in place now it's producing drugs. Is it the best system? I don't know. And I think that's where that round table conversation can really help improve that by bringing all the different perspectives, perspectives together and allowing new ideas to be born from that and changes to we've had. Speaker 2 48:10 Yeah. I guess that was my followup question was does it actually benefit the pharmaceutical company to listen from patients perspectives of why this is good for them? Like, why should it be a patient pharmaceutical relationship versus just using the doctor as a middleman? Speaker 0 48:31 So having the patient come into the pharmaceutical industry, because I think it was really important. I mean, as, as someone who, so as other than the physicians in the pharmaceutical industry that see patients on the side, like I do the rest of the work, the rest of the team does a huge team that works in this industry and most of them are not physicians, most of them are. So there's a project management team, there's a pharmaco vigilance team, has a, a laboratory team, a research and development team. And, and they're, they're working on these, these treatments for patients, but they don't really get to see the patients. And I think it's important to bring the patients in so they get that exposure. And it can be a motivating and inspiring force for them too. Do you to do what they're doing and, and making sure and ensuring them that, that, that their work is important and that what they do makes a difference. And, um, yeah. So I think it's really important for okay, from the employees kind of mental health and kind of pride in themselves to be able to see and what they're doing is important and driving kind of a a positive and productive outcome. Speaker 2 49:37 That's so true. I remember going and meeting a few FSHD researchers. They had never met an FSHD patient before in their lives. And so they're in the lab all day long, treating a disease that I have and I'm like, how have you never interacted with someone who actually has this disease? Like how are you creating a drug out of thin air? Basically that is going to cure me. And I remember after they met me, it was their lives had totally transformed. They were like, now there's a real purpose. I can put a face to what I'm doing every day. And then that opened up the door for them to connect with all these other patients as well. So yeah, it brings, that brings that human element to it that it's not just carbon, hydrogen, oxygen and all these molecules and test tubes and liquids in the lab. It's actually, no, this is who you are benefiting and the generational impact that you're going to have if the drug does go to fruition. Speaker 0 50:46 Yeah. And the um, kind of getting away from the disease description. So a lot of times these researchers, like they, they read about the disease so they know kind of what the symptoms are and, but taking that context of a really hard and give them any complete picture and having that exposure to the patient just really, really important. And that's why one of the reasons why when I, when I moved back to Boston and I took my new job, I was very pretty insistent on having the opportunity to, to, to see patients and uh, continue to have that kind of part of my life and my career. Although, uh, it is taking a little while to get set up. Speaker 2 51:25 Well, I know this conversation totally took a one 80 of, we went from talking about you having the disability and then actually making a career out of it and wanting to help find cures for other diseases as well. And then just really diving deep into your career journey. And that's been so fascinating and eyeopening to learn about. So thank you. Do you have any other advice or anything else from the pharmaceutical industry lens that you look through? I know you look through many lenses. Yeah, Speaker 0 51:57 that's from the pharmaceutical industry. I would just say that our success depends on all participants and including patients. We are trying to develop treatments, but in order to do that we need, we need people to participate in the trials. And so I encourage everyone that is interested too, to reach out to your physician. There's a website of the trials.gov there's a great resource that you can look up pretty much any conditions you can think of and S and see all the clinical trials that are going on in the U S and some have many outside of the U S that are, and it tells you whether the recruiting, it tells you what sites they're recruiting at and, and could you contact information. So if you're interested in participating too, be able to reach out and talk to someone and potentially get screened and participate. So, yeah, but that's, that would be my advice is to try to get involved, uh, joint registries and to stay informed and keep up to date. Speaker 2 52:54 That's great advice. And kind of tying it back to that human element of who you are, obviously I know we have progressive diseases. Where do you think that your health is going to continue to progress and how does that impact your career? Can you foresee that at all? Speaker 0 53:11 Yeah. Okay. Um, it's a, it's a difficult question. I think about currently I'm still walking, I'm still ambulatory but it's a, I'm on a tight rope right now. Part of my training is EMGs electromyogram, which uh, if you're in the neuro neuromuscular world, you kind of know what they are about. Otherwise it's a test of the nerves and the muscles and they're, they're a little bit physically intensive requiring use of your arms and hands. And a lot of my weaknesses in my arms and my hands and I'm fortunate enough currently that I have enough grip strength that I can hold the needle and still do the study. But that is a, with that progression, I don't know if that's going to be realistic in the future. The nice thing about the testing is I can train people to do it and then I can still read the studies, like the still interpret the studies, just I could just have someone that assists me with doing them. Speaker 0 54:04 But from uh, from the rest of it point of view, like, like I started the conversation being a physician, it is very cerebral and very, I thought, uh, about being able to think through problems and solve problems and taken a lot of information and work through that information and then, you know, to come out with an output. So I think from that standpoint, I'm not worried about the progression affecting my career so much as I think I'll always be able to do what I do and do what I love and, and be able to participate in this. I think things over time will change. And the way I do some of it may change, but I think that overall there's, there's always a way to figure out how to do things. A little side note, as a, as I, as I mentioned earlier, I was an engineer in undergrad. Speaker 0 54:50 So as I was going through my early teens or later teenage years and early twenties is when I really kind of, I started developing the weakness in my hands. And this is before the dragon dictation software was really super. It was really good. So as an engineer, and this is, this is kind of how I take all all problems that I, I run into, I, uh, worked with like, uh, with a group too. I designed some braces for my hands that haven't been designed before. That allowed me to type on a keyboard for a while and that Speaker 2 55:20 how cool. Speaker 0 55:21 And that worked for a little while. Um, they're not ideal. Uh, they, after a period of time I would start to lose feeling in my fingertips, so I'd have to take them off. But yeah. Speaker 2 55:30 Yeah. Speaker 0 55:31 But it was, this is just an example of things like that where I think there's always a solution to problems and, um, it's just a matter of thinking about it in different ways until you find it. And bringing the right people and, and asking for the right help and asking for the right assists, the right minds. Think about things with you and come up with solutions. Speaker 2 55:52 Well, certainly I think that the career path that you chose is very much on par with having a progressive disease and having to problem solve and rethink and reassess and redo life all the time. I totally understand that and can relate to that every year is very different. So, I mean, do you adjust? It's, we're always thinking about how to do something better, smarter, faster, more efficient. Speaker 0 56:16 Yup. Yeah, that's, that's very true. Speaker 2 56:19 Well, cool. I didn't have anything else, but thank you so much. Speaker 3 56:23 Yeah. Thanks for having me. Thank you. Friends for listening. Please rate and follow this podcast or texted card and (470) 588-1215 with comments and suggestions. Tune in next week for another disability topic. Speaker 4 56:37 <inaudible>.

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