Why is Sickle Cell Disease Still Underfunded and Underresearched?

[00:00:00] Jared: Hello everyone, and welcome back to another enlightening episode of the Med Design Podcast, where we delve into the stories and innovations of the medical world's trailblazers and thought leaders. Today we're honored to have Dr. Jane Little with us, a distinguished professor of medicine and a leading voice in the realm of hematology, particularly in sickle cell disease research and care.

Dr. Little's dedication to the field is evident in her extensive work. From her academic pursuits at renowned institutions to her transformative roles that we'll dig into later. Her journey spanning the National Institutes of Health, Einstein School of Medicine and her current leadership at UNC is a testament to her unwavering commitment to advancing patient care and medical research.

Dr. Little's approach characterized by a blend of empathy and multidisciplinary care sets her apart in her community with a career rich in achievements and passion for improving the lives of those with sickle cell disease. Little offers a unique lens into the challenges and triumphs of those living with this chronic disease state.

In today's episode, we'll journey through her illustrious career, gain insights into the intricacies of sickle cell disease, and explore her vision for the future of hematology and patient care. So without further ado, let's dive in. Welcome, Dr. Little. We're happy to have you.

[00:01:09] Jane: Who is speaking today? Jared? I did not recognize myself in that intro, but I'm always happy to talk about Sickle cell.

[00:01:19] Jared: Appreciate your humbleness. And so just starting to dive in here can you, describe to us a little bit about your role at UNC and what you've got going on over there as well as, also what got you into, medicine in the first place, and how did you niche down into hematology and sickle cell disease?

[00:01:35] Jane: North Carolina has historically been a leader in care for people with sickle cell disease. About 50 years ago, children with sickle cell disease, which is a, an inherited anemia and vascular damaged disease. That's vasculopathy is the word for that. 50 years ago, children with sickle cell disease died across the world and they died from infections because the abnormal red cells that they had would destroy the main filtering system that we have in our body to help us deal with bacterial infections. And so little children would die from infections and if the infections didn't kill them, then strokes would kill them actually, because its vascular damage is really significant in little children. And so 50 years ago the government had a in the 1970s there was a real push to try to really inspire care for people with sickle cell and they started national kind of organizations and funding to try to support research in sickle cell.

And so like many diseases but more than most because you can actually track over the decades how more and more children survived. We saw that these kind of iterative and multi-centered studies on how to treat, how to prevent infections, how to prevent strokes really changed the life of people who had sickle cell disease.

So it's a real testament to those children and their families that they participated in these studies 'cause it's a little scary when somebody says, oh, we're gonna, we don't know what the right thing is. So we're gonna try this and see if that works. These patients and their families really hung in there.

And over the years you can see the life expectancy of people with sickle cell really expanding. So said another way. 50 years ago, everyone who took care of people with sickle cell disease primarily they were taking care of children. And so now you have this big enlarging population of young adults who are aging even more, who have sickle cell disease.

And it's was a new disease in a funny way, right? Because you had people who had never survived before. So they've been exposed to things that they hadn't experienced, that hadn't been experienced previously. And another disease like that would be cystic fibrosis, which is a lung disease that used to be really have little kids who died from it.

And now adults were surviving, children were surviving into adulthood. So that's a long way of saying that north Carolina many years ago was really a leader. UNC and Duke were really at the forefront of trying to care for adults with sickle cell disease. So there were many very eminent places across the country that cared for children.

But really there was a place in the Bronx and UNC, those and Duke. Those were really places that started to focus on adult care early. And so it's really been an honor to be part of the UNC program because it really is quite historically, rich in caring for adults. And so that's what we do is we care for adults with sickle cell disease with our partners at the pediatric center who care for kids.

In North Carolina, caring for sickle cell disease also means recognizing that people live a million miles away. So if you live on the coast, which many African Americans do, then you have to drive two or three hours to come one way to come to your appointment. And we have made an effort to try to really consolidate and coordinate care so that people with chronic illness are not spending all their time dealing with the illness and they get to get on with their lives.

So that's really been with my colleagues. One of whom I can see is here. Patrick Ellsworth with a number of us, have really worked hard to try to make care easier for people with sickle cell disease. And we're still behind other illnesses like cystic fibrosis and hemophilia have really had coordinated care for decades.

And sickle cell disease has not. And many of us think that's really been a place where racism has had an impact on people's lives. One of the many ways. And we see it every day, right? Like we are right next to the hemophilia clinic. They've had a registry that gathers data on their patients for 50 years.

They've had government funding for nurses and coordinators and social workers. And we still don't have that in sickle cell. We just keep pushing. But there are a lot of things that we're hoping will get addressed in the next decades.

[00:06:15] Jared: That's fantastic. There's already been so many improvements and I think when you're talking about 50 years ago, there was even I think more virulent racism than there is today, even though of course it still exists today.

And would you say that racism impacted the early stage research into sickle cell disease because people just didn't really even want to fund that sort of thing.

[00:06:37] Jane: Yeah. I think colleagues at Duke had published papers that, and at Johns Hopkins had published papers that really suggest that cystic fibrosis pound for pound had much more foundation funding, much more government funding.

And it's affecting children in our communities. It's hard to understand what the difference is, except that most of the kids with sickle cell disease are black, and those with cystic fibrosis are mostly white. I think the children with cystic fibrosis deserved every scrap of resources they got, but sickle cell deserved children with sickle cell deserved the same.

And so there's a lot of unfairness, that's still, it's still out there.

[00:07:18] Jared: Yeah, no doubt. And so just from your experience as well, what are some of the common challenges, facing people that are living with sickle cell disease, where in terms of managing the disease state and really trying to maintain the quality of life. For me, Sickle cell came up 'cause I'm a sports fan and I like football and like certain football players can't travel to play in like Denver for example. And so I know I noticed that apparently altitude has something to do with it.

[00:07:43] Jane: Yeah. That's a complicated question. So there are two parts of that. So like many genetic diseases, there's a carrier state.

There's a disease state and there's a carrier state. So in sickle cell it's called sickle cell trait, and probably 2 million Americans have sickle cell trait. People at UNC and elsewhere, UAB other places across the country are really trying to understand, does the carrier state of this gene, meaning that you have the mutated gene but you don't have the disease.

Does that have any negative impacts? What you're implying, and it's true, there was some poor football player for the Pittsburgh Steelers who almost died when he went to Denver, Colorado. And it's not the height, it's actually that the oxygen is less, that the air is quote thinner in Denver. And what we say in clinic is if you think of hemoglobin as just marbles in a bag, You imagine sickle hemoglobin as turning those marbles into little Legos.

When there is oxygen in the cell, the Legos are covered in a little coat and they can't snap together. When the oxygen pops off, then the Legos can snap together and you can get like a stick inside the red cell that normally is not there. It's never there. It's an unusual and abnormal thing in the red cell, and it can either poke a hole in the red cell or it can distort the red cell so that it's abnormal and it can scrape the blood vessels or blow up.

And so the blowing up part is what makes the anemia low and damages the blood vessel lining. Oxygen has a big role in whether or not those Legos stick together. And the concentration of the Legos in that red cell also has a big role. So if you inherit this, half of your hemoglobin molecules are going to be Legos and the other half are gonna be marbles.

I don't know if that makes sense. That's a great analogy. Yeah. There's some conditions in which it makes it easier for these molecules to snap together, and one of them is low oxygen. And there are also places in the body like the spleen, which is our bacterial filter, or the kidney, where the oxygen is low biologically or the environment is more acidic and that also makes the oxygen pop off the little marbles, so they become Legos. There are conditions even in people who don't have both. Genes in which even half the Legos will find each other. And that's trait. And so we don't know yet whether that has an impact on people. And that's really an important question.

So that, football players who wanna go to Denver can figure out whether they'd be safe for them or not. The other part of that question, I guess had to do with racism and or the impact of race on quality of life or the impact of this disease on quality of life? I can't presume to answer that 'cause I fortunately don't have the disease.

But I am always moved and impressed by the dignity and guts that people who have sickle cell disease show despite all of the complications of having multi-organ damage and pain, which is in this day and age especially, a fraught not diagnosis exactly, but a fraught complication.

A lot of trying to go to the emergency department when you have pain is very challenging these days and most people with sickle cell disease handle it much better than I would to be honest.

[00:11:16] Jared: That's a question I also had is what is, what does living with this on a day-to-day basis kind of feel like for those that you know don't have it?

You said pain, that's one thing. Multi-organ failure or damage. It'd be cool sometime for you to have a, somebody who has the disease 'cause they could really talk about it in a way that I cannot. What I see from the outside is that pain is very bedeviling for people.

[00:11:37] Jane: It's chronic. In adults, so it's every day you have aches and pains, and then you can have these flares that are really life-threatening and very severe, whether chronic or acute. They both are really interfere with your life, and they're not always linked to the other damage that we see. So pain isn't all, even though it makes people miserable, isn't always connected to some of the other side effects that are really life threatening.

So the pain is quality of life threatening, but the cardiopulmonary effects or brain effects or reproductive health effects, that those may be disconnected from the pain. So I think it's very hard as an individual, I would imagine, to have the pain not tell you. For you, that's the worst part of the disease.

But there may be other feature, those features of the disease that are just silent that you're not really able to see or grasp. So I think it's a very difficult disease in being creepy. Wow. So both has these painful episodes, but can also have slow underlying organ damage that's cumulative and that's really hard for people to cope with.

[00:12:53] Jared: What I'm curious of also is, you spoke of how patient outcomes have improved over the past five decades and what sort of, from a patient care level, what kind of treatments have led to this or I guess outcome to where now kids can at least live to be adults and live a longer life?

[00:13:09] Jane: Yeah, it's a little bit like, if you think about the major medical advancements that affect it. It's probably things like sewage, like managing sewers, like you think it might be a drug, but often it's public health stuff that makes such a big difference for populations and sickle cell isn't that different from that in that managing, preventing some of the worst consequences.

Really had a big impact in sickle cell because those consequences were so dire. So in children, they tested whether giving children penicillin for the first five years of their life would prevent some of the worst infections. And it did. And then they tested whether screening by ultrasound of the blood vessels to the brain in kids, because in children, the skulls are thin enough that you can do ultrasound through the skull and you can get some picture of how the blood vessels in the brain are doing. They began to understand that just like when you put your thumb over the end of the hose and you're trying to wash your car and you want more flow, more velocity so you can really clean it as a vessels beginning to block, you'll see the flow through it will increase, right?

The pressure and the flow will increase, and so that can be measured by ultrasound. They can see before the blood vessel actually occludes. They can see it being blocked because the flow rises or starting to be blocked. And they did tests back in the nineties and they said, fine, can we randomize kids to transfusions or no transfusions and see which is better, seeing if giving people non-sick blood protects them from progression to stroke?

And it did. And so these things were not, some amazing gene therapy that swept in and allowed, it was really just immunizing giving antibiotics, monitoring carefully for stroke. And so now you have 18 and 20 year olds who are surviving, but their underlying disease hasn't really been modified.

So in the last 20 years, the agent that has emerged in the most severe form of sickle cell, which is called sickle cell anemia. Where you have both genes are S and S. If the normal hemoglobin genes are A and A we're just gonna simplify it and say it that way. In, in sickle cells, S and S, there can be other mutated hemoglobins that, for instance, we talked about the Legos in a red cell.

Yeah. If you had 50 50 marbles and Legos Right, that's better. But one of the mutations that's a he hemoglobin mutation doesn't add more legos. It makes the red cells smaller. So there can even be other ways that you can get sickle cell disease, but the worst form is SS when you, both of them are Legos, as it were.

And in the worst form there's a medicine that over some months changes your hemoglobin. So that if you're lucky, up to 20% of the hemoglobin is no longer will jump into that Lego. So the little bumps get sought off maybe 20% of the hemoglobin so that as you're making that Lego right the little pieces are much smaller.

So you don't get that stick poking through the red cell anymore. You don't get the red cells exploding as much anymore, but that means that you have to take a pill every fricking day, three pills, and they're big, and they can make your hair thin, and they can make your nails have stripes on them, and you can gain weight.

Nothing comes without a cost. And for the patients, that's a big deal to have to take a pill every day. On the other hand, it can really lessen risk to the people who are able to take it successfully. They're less likely to have painful episodes, and they're less likely to have these pneumonia, like acute chest syndromes that can be lethal.

So all in all but you have to believe in it, right? It's if I take a pain medicine, I feel better right away if I take this medicine called hydroxyurea. It's gonna take months. And the pills may make me feel not great, and I have to believe that it's making me healthier. It may not make me feel healthier, but it may be making me healthier.

So that's a very difficult spot to be in. It's like blood pressure medicine, right? Like those medicines are very hard to take every day. Like you have to believe it. So we spent a lot of time talking to patients about why we think it might help them. But it's very, it's not an easy thing for anyone to do, in your whole life.

You have to take it

[00:17:54] Ty: anytime. You don't have that direct cause effect relationship. It just makes it that much harder to believe. Yep. For all of us. Yeah, exactly. That's anybody.

[00:18:03] Jane: And if you're chronically ill I think it's even more challenging. Because you get tired of it. Yeah.

[00:18:07] Ty: It's yeah.

It could you take a minute, we were talking about this before the podcast, but explain your background there because as you're talking about Legos and marbles, I'm looking at your background and it like the, for me as a visual person that's connecting the dots, but do you mind explaining your background image there?

[00:18:22] Jane: How I got to sickle cell? Oh no, sorry.

[00:18:24] Ty: The image you've got.

[00:18:25] Jane: Oh, my background. Oh yeah. I was thinking God, who, nobody cares about that. These, so these, wait, that is a sickle cell. That is a sickle cell. That is a sickle cell. It's amazing, right? Like they, you can see that they look different.

And this is from the original article in 1910. There was a young man who got his courage in his teeth and he moved from Guyana, which is an island in South America, I think it's an island nation. And he went up to New York and he was treated for jaws, which was an infection in his ankle, like a skin breakdown in his ankle.

And he made his way to Chicago where he went to dental school and he was in second year in dental school and he came down with pneumonia. When they looked at his blood smear, voila, they saw that his red cells looked different and they theorized that he had some, inherited condition or some red cell.

I don't think they theorized inherited, actually, I think they just theorized that he had a red cell condition that had explained this because they could see these abnormal red cells and unfortunately, he had all of the kind of side effects you can get with sickle cell, including kidney disease, and he had died from a pneumonia about 15 years later when he went back to Guyana to be a dentist.

He died. It's a very classic story of someone really struggling through a lot. The jaws were probably leg ulcers from the sickle cell. They weren't an infection probably. And actually these cells that are have a, like a target, make me think that he probably had one of these variations of sickle cell that we talked about.

Like I talked about how the red, there's one variation where the red cell shrinks and it's not the most severe form. He probably had another variation that allowed him to live to adulthood with some reasonable health. But still, it's not a free ticket if you don't have the full-blown SS.

He still had a lot of complications and he died young, as many patients do still unfortunately.

[00:20:37] Ty: Yeah, but It's remarkable. It's amazing that long in that, in 1910, it just, yeah. Yeah,

[00:20:42] Jane: that's, he must have been an amazing guy 'cause he must have had pain and complications as a kid and he still got on that boat, and went north, like the cold weather is really rough on people with sickle cell because there's something about evaporative cooling on the skin. Like you'll hear little kids who go swimming who have sickle cell, they'll come outta the water and they'll have pain because their skin cools down. And there's something about that makes the sickling happen or probably makes it happen.

And so I think of this guy going to New York must have been freezing. Oh my gosh. He's come from the Southern climbs and he was very gutsy, I think.

[00:21:18] Ty: Be that accomplished too. Wow.

[00:21:20] Jane: Yeah, I know that's right. A dentist in 1910, I can't imagine. Probably quite barbaric, honestly.

[00:21:25] Ty: We had a question I'd love to hear your thoughts on that.

Came in as from Brian Spencer. He asked, are federal, state institutional funds increasing for sickle cell research? And maybe, what about private capital? Do you see those trends happening or, yeah.

[00:21:39] Jane: That's a good question. Yes. I think it's exploded in the last decade and there are, there had been only one FDA approved medication for Sickle cell disease and that was hydroxyurea, which we talked about that increases the nons sickling hemoglobin.

In the last five to eight years, there have been three more medications added, and they're very interesting. One of them is, I still don't really know how it works. It's an antioxidant, I think. And it's really hard for people to take. It's an amino acid and it's messy and it's not one of those things that makes you feel great right away, but it appears to have decreased pain episodes by maybe 40% and people are able to tolerate it. Oh my gosh. We don't have, a lot of people are on it, honestly. It's too expensive and too obscure in a way. The second medication that came out in the last few years was Voxelotor. And remember how we talked about how much oxygen matters, that it's only when the oxygen pops off the Legos that they can stick together.

The Voxelotor changes the affinity of the Lego for the oxygen, so it makes the Lego hold onto oxygen longer so that it's harder for it to make that stick. I have but it's hard for patients to take because it can give people diarrhea and headaches and blah, blah, blah, and we don't know really the risk of this, but we've seen it a couple of times and it's been reported that if you stop it suddenly, so if you imagine we have this bag and it maybe has 80 Legos in it.

But you stop breaking down the hemoglobin because you're protecting it from losing its oxygen. So it doesn't make those sticks as much. There's not as much damage. And so you increase the amount of hemoglobin inside that red cell 'cause it's able to mature more fully and it feels so you're getting more Legos in there.

But they have this medication that's making them hold onto oxygen. Okay? But if you stop the drug, that effect goes away very fast. But the increased concentration of hemoglobin does not go away fast. And so you have now unprotected hemoglobin that now it clicks. And so we've had a couple of, so it's a tricky drug.

You have to take it reliably. We had somebody who just said, I felt crummy. I didn't wanna take it. And they stopped taking it for a few days. And most medications, it doesn't snap back at you. You know what I mean? It will. You won't get the benefit from it, but you don't actually feel, and that's not very common.

I don't want you to think that happens all the time, but it's something we worry about. And we know that it's an expensive medication that is sometimes hard for patients to get. And so we're very careful that to ask people to really try to take it daily and when we stop it, we try to think about the best ways to do that.

But the hemoglobin goes up with that medication. And then the last medication is something called omalizumab. And omalizumab actually doesn't affect hemoglobin. It doesn't affect oxygen. What affects is the interactions of white cells and the lining of your blood vessels. Because white cells are pain signalers, they will tell the body something's happening.

Look over here, there's something going on and omalizumab got in the way of that, gets in the way of that. And again, for a subset of people who have even those variant forms that we talked about, this can make the pain better. The first drug glutamine, l-glutamine was tested only on patients with the most severe form of sickle cell, but is used in both the variant form and the most severe form.

And we have a few patients on that. Voxelotor is only used primarily in people who have the most severe form of sickle cell, who have low hemoglobins 'cause it'll raise the hemoglobin and it's hoped to allow people to function better. And omalizumab is really the pain one that blocks white cells and is used in both SS and SC.

And again, we have a modest number of patients on that, but we try to get every patient who has the most severe form of sickle cell disease on hydroxyurea. Okay? So that tells you that the private capital has really come into this market and is but these are super expensive medications and I think the concern is it was 20 years ago when hepatitis C, which was this very common form of infectious hepatitis, was suddenly curable, but it was going to be 80 to a hundred thousand dollars to do that.

Medicaid. Many patients were on Medicaid and it was very hard to think of. They had to try to really think about how there, how did you get, how do you work through this? Many of our patients are on Medicaid and some of these medications are a hundred thousand dollars a year, and they're not curing the disease.

So they had a lot of trouble with Hepatitis C and that was 88 to a hundred thousand dollars once, and now we have a lot of medications that have a lot of promise and I think may have real benefits for our patients, but they're 80 to a hundred thousand dollars a year. Hydroxyurea is I don't know, under 5,000 a year, it's probably under $2,500 a year.

It's an old drug. So there's a lot of private capital. There are a lot of expensive medications. There's gonna be I think even more expensive gene therapy products emerging in the next five to 10 years.

[00:27:07] Ty: Was gonna ask about that. Yeah. If you had seen any promise with the gene therapy again, something about that.

[00:27:13] Jane: Yeah. It's super promising. It's just complicated. So a lot of the federal funding now is going towards curative therapy. Patients want it, the providers want it, families want it, everybody wants it. And I think that's terrific. I think there is a little bit that's been at the expense of quality of life as it is being lived now.

You know what I mean? There's always this, there's a lot of research money going into cure. But I think it's not. It's not, there's not a lot going into how do we improve what's happening right now. And the second thing is that we've learned in the last two, three years that these curative therapies are tricky.

They're super important and very valuable, but they have side effects that were unexpected. And unlike hemophilia and unlike cystic fibrosis, we don't have the database going back 50 years, that tells us what, how this disease, what the impact of the disease in its natural state is. And so our ability to understand really well what's happening with these curative therapies is somewhat limited by that.

And the second thing is because hemophilia, and you can see it in the hemophilia clinic, there's a long relationship. There's a calm and established process and we've, I think we've accomplished that in sickle cell too at UNC and other places have across the country too. But it's much harder because we didn't really have this national support to really keep clinics strong and make them part of the sickle cell community so that people felt like they could really trust what was happening when these new curative therapies come. So what, when I've given talks about this in the past the analogy I've made is that they really had a beautiful dock in hemophilia and cystic fibrosis when you had, when you're stuck on an island and they send out the curative boat to come get you in sickle cell, we have, let's say we have a hundred people on that island.

And they send out the boat to come and cure people on the island, and the boat can only pick up maybe two or three people. It's like a really beautiful, very expensive, really comfortable boat. But if it picks up 10 people, that's gonna be amazing. And we don't have the dock, we don't have all of this information underlying that whole process that really informs patients and the families and the community that they're part of this, that we've helped them build the dock. That there's a real relationship. And again, I think many of us across the country have tried to do that in our own regions, but it's not been a national hemophilia is a miracle the way they've done that across the country.

And I think it makes curative therapies easier. The scientists have done a phenomenal job. It's really amazing. And the patients who've undergone those are brave as can be. But we still don't know how it's all gonna shake out. And some of the reasons for that are lack of resources previously.

So we're still suffering from those accumulated debt in a way of what's happened.

[00:30:36] Ty: Yeah, you hear about technical debt, but that's like scientific debt almost.

[00:30:40] Jane: Yeah, it's social debt, I think. Social debt. Okay. Yeah, I think it's, I think these patients are really owed better from us as a community than they're currently getting. And I think that the curative stuff is really cool and we'll see where it goes, but there are some challenges to it, and also, it involves high dose chemotherapy, which most people don't realize. So that's not trivial for anybody to undergo.

Yeah. Makes people sterile. It's a big deal.

[00:31:08] Jared: That would be really hard to try to sell to somebody that has already been suffering so for so long already here's more suffering and hopefully, will help you get through this.

[00:31:16] Jane: Yeah, and I think people have been really brave.

And the other thing I did wanna say, if we're on an island of a hundred people, they're like, In Africa, there's six to 10 million people who have sickle cell disease. So we have a hundred thousand in the US. And so it's a rare disease here, but it's not a rare disease in Africa, sub-Saharan Africa, where kids are still dying.

So there's a lot of need across the world, I would say. And if the solutions that are being prepared are super expensive, you can see it'd be, would really struggle to scale effectively.

Yeah, I think that's right. HIV was very informative, right? The medications that were first used there were very expensive and very niche, and I think that, George Bush, senior, God love him started pepfar, which really helped make HIV meds, I don't know that whole history, so I'm probably saying something wrong, but I think it really helped.

Make HIV meds accessible across the Sub-Saharan continent so that, mortality's really gone down and the prevalence has really gone down. And that is totally to our credit. And it would be lovely if we could do the same thing. Malaria that gets a lot of, gets a lot of love I would say.

And it would be nice to have sickle cell get the same. Yeah.

[00:32:31] Ty: Jane, could you touch on maybe why you decided to jump on as we were doing the med+Design program at UNC, like you, you took a big leap of faith to jump in with us and do that. Do you mind talking through that journey you went through?

[00:32:42] Jane: Yeah. We, I feel like when you have a chronic disease, you have to keep thinking when you're taking care of a chronic disease, you have to keep thinking as creatively as you can about ways to help patients. And as we've discussed, one of the big chasms for patient is leaping from pediatric care, overdose adult care, and we were really, we have been struggling and we're still struggling with ways to do that.

And so when you're designed effort appeared as an option. We had no idea what we were doing. So we said, sure. And as you remember, the first year was a disaster.

[00:33:22] Ty: I wouldn't say it was a disaster. It was like the kind of like the first turn of the flywheel. It was the first okay,

[00:33:27] Jared: this is, I know you're such an optimist.

[00:33:31] Jane: Anyway, we never showed, we didn't know. I didn't understand what, I don't know. There were all these paths and journey, I don't know. I didn't know what the hell was going on and you guys were very gracious about it. And let us try again. And you also, I think for, I don't think we were the only ones who didn't make as many meetings as we wanted to.

And so you changed the way you conveyed information, which was really helpful because you could look at it at your own. And so we had good colleagues like Patrick again and others who really threw themselves into the effort and I think they all appreciated being made to take the time to think creatively about a problem that we were always thinking about in one way.

So that was very interesting and now we have to actually do it what we had talked about. And I think that's gonna take us a couple of years to really get going. But we have very engaged people on our team and I think it'll happen. And I think your process really helped us be a little less dull about it. Which I appreciated a great deal.

[00:34:38] Ty: Working with your team was one of the highlights of the med+Design program for us because, And then your team, you had a diverse group of stakeholders, the pediatricians, social workers, adult hematologists who were all coming together and each had their own viewpoint.

And I was just appreciated the diligent discussion that happened where, or debating about what truly is the problem? Then as a group coming to consensus about what the problem is and then going and talking to the actual patients themselves and then that's not actually the problem as we see it.

And it's getting like a lot of people just to come to like alignment on what that is and then start to come up with solutions and think the array of solutions we came up with were like, some of them were like, potentially expensive, but others were changes in care, which I thought was so fascinating that you could start to make an impact just by putting some smaller scale programs in place.

[00:35:34] Jane: No, it's, as ironically, I was not anywhere near that team. As so I could take no claim, no credit. But it's a very diligent group of people who really care a lot. And I think they really I agree with you. I thought one of the best things that that came out of that was making us go and talk to patients and to the nurses and people that weren't part of the core group, we just didn't have the ability to have a single patient be part of it. And so we instead went and talked to patients afterwards. And Caroline Hale, who's the social worker on our team, did that. And it was really great. I think it really helped shape our thinking and you guys made us do that.

You know how sometimes you ignore things until you're made to do them. And so I think that was part of what was so valuable. It forced us.

[00:36:22] Ty: For us, as we got feedback on how to really, there's the education part of this that we've been trying to figure out what's the best way to bring this along, bring people along on this experience and put the right checks and balances 'cause we're not experts at, anything to do with sickle cell. And so trying to put together a curriculum that at least helps to, make sense of some of the methods to try to discover new value and yet also have it be completely grounded in helping people. We were co-creating the material with your team as to try to make it so that it would be as helpful as possible, which is ultimately what, where we try to make an impact through, like trying to put these programs together.

No, I think you didn't, I think you really you guys were serious and supportive and very helpful and we really appreciated it. So I think we're all we're all grateful for the experience.

Yeah. We're grateful for the collaboration. We've got just a little bit of time left.

Let's see. We had another question come in and so taking us back to the previous discussion is there parallel research or other disease research that's benefiting sickle cell or vice versa? I

[00:37:25] Jane: think that I would say

that's always true, that both biologically and socially, you're never an island.

I would say personally that I have learned so much. I think I have gotten so much more from taking care of people with sickle cell disease than I've given to people with sickle cell. That it's ironic because it just changes the way you take care of people because you come to understand that if you don't actually talk to people about why you're doing things, they won't do them, and why would they?

And so you just become better at trying to educate patients about why you're doing what you're doing, and I have become clearer, I think in these after visit summaries. If you've ever gone to a physician and got that 15 page booklet that tells you to put on your seatbelt and love your mother and do all those things you should do.

I've tried to become much clearer in my communication about what I think is important in the next three months. I don't try to over list. I tend to be, and then you do this and then you do this. No, do this. You know what I mean? I think medically, clinically, one can learn an enormous amount from taking care of people with sickle cell disease.

And I think red cells have been foundation of a lot of what we've learned about molecular medicine, like going back. To the first kind of protein electrophoresis were on red cells because you could actually get a ton of protein from a red cell by just drawing blood. When you think about it, you're not gonna do a lot of research on the lung back in the old days, 'cause it was hard to get tissue. But you can get a red cell very easily. It's like the most prominent cell when you draw blood. And the red cell has been a source of a lot of knowledge. I think that has been applied generally in molecular biology and in science.

And that's true of sickle cell too. I think people have learned a lot from the disease.

[00:39:19] Jared: I know we are as Ty said, coming up closer on time something I did wanna pick your brain about as well, which is goes to the namesake of our first module, which is the empathy module.

I was curious if you could speak to the importance of having empathy for your patients, as a clinician particularly for populations that are typically underserved at a systemic level like those with sickle cell.

[00:39:43] Jane: Yeah, compassion is always valuable. And I'm actually, this may not surprise you.

I'm not the most empathetic person in the world. And I've learned from my patients and my colleagues all the time. Again, Patrick, who's on this, is is so lovely with people in all spheres of there, there are many people I work with that I just see the way they interact and you think, oh, that's lovely.

And again, I have learned a ton from sickle cell patients that has, that just being kind goes a long way for everybody. So yes, of course say yes to compassion. Yeah. It doesn't mean you can ever do everything people want, but you can always be compassionate about it and empathetic.

[00:40:30] Jared: Absolutely. And I guess just capping things off here, for folks that are, trying to follow in your shoes and try to have an impact on the next generation of sickle cell patients, try to advance the space forward as you have, been instrumental in doing, of what advice would you give to them to be successful throughout their career in order to, like I said, have a career of impact and something that changes like I said, so many lives at the end of the day.

[00:40:57] Jane: I wouldn't say that I've had that career, but I would say that then I think being stubborn and trying to redirect, when you get distracted by why am I parking in this lot? Why am I not in a better lot? You know what I mean? I really try not to get distracted, although I do sometimes by stupid things.

So you just keep your head down. And try to care about the right things. Absolutely. One fails constantly, but you keep trying.

[00:41:32] Jared: I think that's something we've learned a lot as well, but Yes. Yes. Yeah. Jane Dr. Little, sorry. Yeah. Thank you

[00:41:42] Jane: so much. Oh my God. I'm gonna have to sign off now.

[00:41:44] Jared: I know. We really appreciate you being here. Really appreciate the work that you do. And thank you so much for taking the time outta your busy schedule to join us today.

[00:41:52] Jane: Very much. No, we're very grateful to you guys too. You guys have been lovely partners in many things, so thank you.

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