The Closest Thing to a Cure for Type 1 Diabetes - Dr. Piotr Witkowski

Could Type 1 diabetes finally have a cure? In this episode of The Diabetes Diaries, Rachael Sood sits down with Dr. Piotr Witkowski, a transplant surgeon at the University of Chicago and the lead researcher behind the Eledon trial - a groundbreaking clinical trial involving islet cell transplant, and the medication Tegoprubart, that may change the future of Type 1 diabetes treatment forever. Dr. Witkowski breaks down what islet cell transplants are, how the procedure works, why insurance won’t cover it, and what it could mean for the millions of people living with Type 1 diabetes worldwide. This is the first time Dr. Witkowski has ever shared this story publicly. 

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Transcript

SPEAKER_01 0:01

Welcome to the Diabetes Diaries. I am your host, Rachel Seude. Joining me today is Dr. Pitar Watowski, a transplant surgeon at the University of Chicago, and the person behind what many in the diabetes community are calling the closest thing to a cure for type 1 diabetes the world has ever seen, the Elodon trial. Dr. Watowski has dedicated his career to solving one of the most complex problems in medicine. And today, for the very first time, he's sharing his story publicly. If you have type 1 diabetes or love someone who does, this conversation is for you. Dr. Watowski, welcome to the show.

SPEAKER_00 0:41

Thank you very much for having me and invitation.

SPEAKER_01 0:45

Thank you for being here and sharing your story. So, Dr. Watowski, every person living with type 1 diabetes has asked the same question their entire life. Where is the cure? You are the person behind this Elodon trial, the closest thing to a cure people with type 1 diabetes has have ever seen. And this is the first time you really are getting to share and speak about it. We want to hear from you. Tell us everything.

SPEAKER_00 1:11

So again, thank you very much for invitation and opportunity to talk about our results and our uh our findings of the of the of the trial of uh including new medication teguprubert. So it's not a cure yet. I have to um, I mean, unfortunately, I have to dis disappoint many many of you, many patients, but it helps a lot of patients. What we do, we transplant pancreatic eyelids, which we isolate from disease donor, and we transplant them to the patients.

SPEAKER_01 1:46

Where do you transplant them to?

SPEAKER_00 1:49

We infuse them into liver during minimally invasive procedures. So basically, interventional radiologist is placing a small catheter through the skin under local anesthesia, and we infuse eyelids into patient liver. Those cells, once they get into liver, um, they grow into the liver, and what's important, they produce insulin in an optimal amount and allows patients to control their blood glucose better, and in many of them allows to reverse diabetes. So, yes, our patients don't have diabetes anymore, and this is a major advantage, and give it gives them hope uh for long-term living without diabetes.

SPEAKER_01 2:33

Wow. So, how have you been able to achieve this? Because no one has done this before, because if someone's immune system attacked them one time, who's to say that it won't attack them again? Are you doing something different than ever was used?

SPEAKER_00 2:49

So, actually, this procedure we've been doing and optimizing for the last 25 years. Wow. The problem is that, I mean, there are many problems, but the main problem is that since those cells come from people, other people, our patient's immune system is able to recognize that they are not own cells and reject them the same way as it would reject other organs which are transplanted. Therefore, we need to use anti-rejection medication. And unfortunately, those medications have side effects, toxic to the patients. And that this was the main problem why and why this procedure hasn't been available to every patient with type 1 diabetics. We've been offering this only to selected patients, those who can control blood glucose and it's compromising their daily life despite the best effort and using the base, the best, uh, the best technology. So only for selected patients. What's new in this particular trial that we're testing new medication, which we hope is less toxic or non-toxic compar comparing to the previous medication. And this is what we see, and that's why it's different, and that's why it gives our patients a lot of hope, to the point that some of the patients they they do feel like they cure because they don't have side effects and then they don't have diabetes anymore. So they feel cured. For us, it's not really cured yet because patient needs this new medication, uh they need to get the infusion every three weeks. Um, but because there are no side effects, they have normal life as not diabetic patients. And for some of them, they never experience this. They developed diabetes when they were children and they don't remember life without diabetes. So there is a progress, it is a progress. Um, of course, this is just first 12 patients. These are uh early results. The longest patient now uh has been off insulin almost two years, and we will extend the participation for this patient for another year. So the patient will continue getting the new medication, and we hope that we'll stay off insulin. But we have to wait longer to learn if there are any unexpected uh side effects. We have to enroll more patients to confirm that this is safe and effective. What we also hope that this medication has potential to modify and the immune system so potentially in the future we can stop immunosuppression altogether completely. So this is the hope. We are not there yet, but this is where we hope to get in the future.

SPEAKER_01 5:43

Wow, it is so promising though, this type of therapy. And I something that I didn't realize is Tegorapart. Is that the name of it?

SPEAKER_00 5:51

Tegaprubart, yes.

SPEAKER_01 5:52

TegraPrubart is a medication that is not available to the public yet, but you're using it in an experimental way, but it has to be given, you said every three weeks.

SPEAKER_00 6:02

Right. So for now, um, we're giving patient uh uh this medication as infusion, one hour infusion, because it's a frame of the clinical trials in its new medication, patient needs to come to us every three weeks. In the future, the formulation of this drug can be changed to subcutaneous injection like insulin, and patient can could potentially take it at home uh without coming for infusion every three weeks.

SPEAKER_01 6:31

Okay. Do you see this drug being used in different situations other than just the uh transplanted eyelet cells? Do you think that there is promise to use it in other situations?

SPEAKER_00 6:43

Right. So this drug has been already tested in kidney transplantation comparing to the standard previous medication, and the results are really uh promising. They it protects kidney transplant as well as alt medication, but without the same toxicity. So there are kidney transplant patients who are already benefiting. And then I know there is um there is a plan to test it in and liver transplant patients as well. I'd like to go back to type 1 diabetes because, as you said, uh we are dealing with two problems the rejection against the eyelids from somebody else, but patients' own immune system attacked own eyelets previously, the autoimmunity. And we hope that this particular drug can control both, can control and prevent recurrence of autoimmunity and the same time prevent the rejection of the islets.

SPEAKER_01 7:38

So that's why it's so unique from other people.

SPEAKER_00 7:40

That's why it's so unique and so promising. The best part again, I'd like to highlight that there is really no um substantial toxicity which we see today, and that's why it's been uh so promising and and and uh cause a lot of emotions and hope and bring hope to the patient who's been waiting for the cure for a long time.

SPEAKER_01 8:05

Right. Do you think this is the cure for type 1 diabetes?

SPEAKER_00 8:09

It's just the beginning, it's not the cure today because in um I mean, although some patients feel cure, for us, cure is when we do something, some manipulation, yeah, and it will there will be no chance for recurrence. So I can tell you, I'm a surgeon. If I remove gallbladder, I cure patients from gallbladder disease. Because the gallbladder is gone. Because it's gone and disease is gone and it will never come back. Yeah, so this is the cure which we're aiming for. Do something and manipulate something so the diabetes has no chance to come back. Because today with anti-rejection medication, there is still some chance of recurrence.

SPEAKER_01 8:49

Yeah. Speaking to this, you know, there are, I want to say, nine million or more people in the world living with type one diabetes. Seeing something that you're doing coming to fruition, do you ever see this being available to the entire population? Is this feasible? Of course. Of course.

SPEAKER_00 9:06

Yeah, this is what we we do believe, and that's why we do the research. So um the next step is to use this drug and test the drug in more patients and complete uh clinical trials. There is uh there is phase one, phase two, and phase three clinical trials need to be completed and the results need to be assessed, and then the the drug can be available as a as a standard of care to any patients uh who require any type of, I mean, to require transplant, eyeless transplant or organ transplant in the future. So once the drug is approved, it it will be available, which makes eyeless transplantation more available to more patients.

SPEAKER_01 9:48

Yes. Can you talk to me about that? What has been, you know, I never like to mix uh medicine and religion and politics, but somehow um here we are. There's a lot of limitations to what physicians and scientists can do because doing good things, uh, we all want to give our heart away, but it costs money to do good things and it takes support and money and funding. Um, is there something, a barrier doing eyelet cell transplants that you have faced?

SPEAKER_00 10:18

Right. Unfortunately, the regulations related to the eyeless transplantation hasn't been optimal in the United States. Eyeless transplantation over the last 20 years became standard of care alternative procedure to whole pancreas transplant, rainbursts by national health system in Canada, Europe, Australia, Japan. But it's still not rainbursts in the United States. And the problem is because of the regulations in the United States. Eyeless has been regulated as a drug, not as organs, and it limited access to this procedure and path to the reimbursement. So now we leaders in the field and scientists and researchers and physicians, we've been advocating for adjustment of the of the regulations. More recently, breakthrough T1D join us uh in advocacy. And then so we already talked to the HHS, FDA, and HERSA and the administration to and ask them to look into regulation and and maybe adjust so it can be available and reimburse procedure as in other countries. This will allow us not only to do to help more patients um in the United States, but also progress the field faster. We can test um the research will be not as expensive. We can test the new approaches on more patients faster and progress field towards the queue. Wow. So we hope that this adjustment will happen soon. Because today, as you said, we've been able to do 12 patients only, and the progress is limited by number of patients. If eyeless transplantation is reimbursed today, we would enroll much more patients with the same funding and progress the fields faster.

SPEAKER_01 12:13

So there are only 12 people in this trial right now, like you said. What does a person actually go through from like the moment they say yes and enroll of this? Like what does their process look like?

SPEAKER_00 12:25

So it's a clinical trial. So everything happens based on the protocol. We follow the protocol is which is approved, has been approved by FDA and our ethics committee at the university. There is uh there are um inclusion and exclusion criteria. So every patient who would like to participate, we screen against those criteria, and only those who qualify can proceed. And then once they qualify, we invite them. Uh, I mean, first we invite them for screening. Then, once they qualify, we offer them eyeless transplantation. So when actually we start the anti-rejection medication first, then we look for a disease donor, then we isolate the eyelids, and if we have enough, when we have enough, we invite patients for the procedure. Um patient stays in the hospital only three days. Okay. Stays in Chicago for a week altogether, and then comes for infusion. Every three weeks, you said in the long term. That's correct. The good news is that this is minimal, minimally invasive procedure. There's no surgery, it's just infusion. So patient can return to work quickly, within a week can be back. Right.

SPEAKER_01 13:43

And they're not immunosuppressed or immunocompromised. Like, do people getting this, should they take precautions as far as like wearing a mask or washing hands, staying out of a crowd? Yes, yes.

SPEAKER_00 13:53

So so as we discussed, they're still taking anti-rejection medication, immunosuppression. So absolutely, they don't need to live in the bubble. They can have normal life, enjoy the life. However, yes, however, when they fly, yeah, we advise them to use N95 mask and avoid, basically avoid sick people. Yeah. But otherwise, they have no limitation.

SPEAKER_01 14:19

Okay.

SPEAKER_00 14:19

And daily life.

SPEAKER_01 14:21

That's something I wanted to make sure and get gather from you and info and make sure the public knows that this medication, it is an immunosy an anti-rejection medication.

SPEAKER_00 14:31

Still anti-rejection medication, but with less side effects. Okay. With less side effects in the short term, in the long term, we have to wait longer. But what is really important because I know patients are worried about leaving uh with immunosuppression, being immunocompromised. Um, they worry about the risk of infection. Infection may happen the same way as it may happen when they are diabetic. But what is very important, they they contact us as soon as they have infection. We implement regular treatment and they save and we keep them safe. Patients get into trouble if they they don't um they don't uh contact us when they sick, basically, and this is a dangerous situation. So we highlight that they need to stay with us uh in constant contact when anything happens to them.

SPEAKER_01 15:26

Okay. I think you may have answered this question, but what is the biggest obstacle standing between this trial and it becoming available to the masses of people living with type 1 diabetes? Because it seems since you've done this and achieved this, you've done this with 12 patients. Now your phone, you said, is ringing off the hook and emails, and there are people that want this.

SPEAKER_00 15:50

They need it, right? So we're getting phone calls from the most desperate patients whose life is completely disrupted despite the best efforts to control the blood glucose, and they they would love to participate in the trial. But the number of patients which we can accommodate today is very limited. Now, as I said, we will continue the trial. There will be more um patients enrolled, but again, it's it's maybe 20 patients. Um and then uh and then in this when it's approved by the FDA as a as a new drug, then it can be uh offered to a higher number of patients. But I have to highlight at the same time, we need eyeless transplantation to be reimbursed. Yeah. We hope that in the meantime the regulations will be adjusted and we can offer eyeless transplantation with the new drug to help more patients.

SPEAKER_01 16:48

So two things need to happen simultaneously. We're we're looking for almost a miracle, maybe. We're looking for this medication, TegeraPart, to be approved for the indication to use an island cell transplant therapy because it's being used in other indications.

SPEAKER_00 17:04

It's being tested, it's not approved for anything yet. Ah so it's being tested in parallel in kidney transplantation the same way as we tested in islets, but it haven't been approved for anything yet.

SPEAKER_01 17:16

So it just needs approval in general.

SPEAKER_00 17:18

Approval in general.

SPEAKER_01 17:19

What is what's going on? How is that process going? Are you involved in that?

SPEAKER_00 17:23

Or right, so we will be involved. So um the drug manufacturer, Elodon, is pharmaceutical molecules. It's it's about is organizing the the next step of the clinical trials to enroll more patients and and and obtain the license.

SPEAKER_01 17:41

Okay. So are you going to enroll, like you said, you're going to enroll maybe eight or ten more patients in another phase of this?

SPEAKER_00 17:47

Yes. So what we will do soon, very soon, we will uh we are opening another trial with the same medication, the same approach, specifically for patients with type 1 diabetes who already has kidney dysfunction. Today, because of toxicity of current medication, tacrolimus, we cannot offer any transplant, eyeless transplant to patients who already have kidney dysfunction, because tacrolimus will uh will completely damage the kidneys. Yeah so with the stego probert new medication who is which is not toxic to the kidney, we can help patients. So we hope that reversing diabetes, we can stop the progression of the kidney disease and maybe let the kidney recover, maybe the kidney will recover. So we are about to open the small pilots study again for 10 patients. They will be transplanted at the University of Chicago at University of Miami, and it's sponsored by Breakthrough T1D. And again, we need sponsors, we need research funding to continue doing this. But in parallel, the LADON, I'm sure they are already getting ready to enroll more patients for the clinical trial, which allowed them to apply for the uh license approval for the drug.

SPEAKER_01 19:08

I see. What needs to happen next, again, for this to become available for everyone living with type 1? Because that's what everyone wants to know. They want you in their living room giving them this procedure.

SPEAKER_00 19:21

So, so of course, but since this is still anti-rejection medication, I don't think we can use it, we should use it for any patients with type 1 diabetes, especially children, right? I mean, there is so many desperate parents calling us and asking for help. So, not today, but again, as I said, this is just the beginning. Once we learn more about this medication and we hope to optimize um this immunomodulation with Tego Probart or adding some other medication, or we will be possibly we will be able to stop anti-rejection medication, which gets us closer to the cure. And I'd like to add another aspect, right? We've been talking about cadaveric eyelets, which are not approved, needs to be uh regulation needs to be changed in order to be available. But in the meantime, there are many companies trying to develop stem cells-derived eyelets for transplantation. And if they are successful, hopefully those eyelets will be available as off-shell product. We can schedule the procedure for everyone because disease donors are limited and we can't help millions of patients with disease donor eyelets. But once stem cells eyelets are developed, optimized, and they they they work on a consistent basis, we can combine with some immunomodulation towards the cure. The cure is still not here, but uh but we we would love, we are progressing.

SPEAKER_01 20:50

Yes. So, yes, you brought up something. So I know a lot of people are using in multiple labs across the US and the world, they're doing deceased, they're getting skin like from the palms of hands, and they're taking that skin and modifying it, and we're calling that cell therapy. And they're injecting it right now in labs all over in mice. Would this medication be used in combination with cell therapy, do you think?

SPEAKER_00 21:16

Yes. So this is the goal that this medication will help stem cells-derived islets to progress definitely. Because, for example, now we've been testing uh stem cells-derived eyelets manufactured by de uh by Vertex with alt anti-rejection medications, and the outcomes can be much better if we use Tegoprubart in the future. So definitely.

SPEAKER_01 21:41

That could be used almost in a larger scale, hopefully, to more patients and and again, progress, progress, progress.

SPEAKER_00 21:49

We need the progress.

SPEAKER_01 21:50

Do you think somebody like me and you, do you think that I'm going to be prescribing these cell therapies maybe in the next five or ten years with this, like just in my office? Certain patients, like what would be your prediction? I'm serious.

SPEAKER_00 22:02

No, no, no, prediction is hard, and we promised our patients cure uh in five years, so many years ago. Yeah, of course, we do everything we can and we hope for the best. And can promise I can promise that we will work hard. Definitely, we work hard hard, work hard and do everything we can to progress, to provide a therapy which is more beneficial and less toxic.

SPEAKER_01 22:27

I thank you for all of your work in this. And I know, you know, since some of your patients have given their testimonials, you know, the world's eyes are on you, and it's really uh so much. Um you've given people hope. Uh, maybe not something you were even thinking or anticipating, but it's been um probably so overwhelming for you to know you're you're doing something to impact the world.

SPEAKER_00 22:49

Yes, I mean uh we feel the pressure, um, but it's not only me, it's our entire team. It's uh it's highly motivated and and stimulated, and we we really uh enjoy seeing our patients doing so well, it's so rewarding. Yeah. Uh research is tough because especially testing something new might be not effective. Right. And many patients uh and investigators uh experienced this in the past. We've been lucky that the results are so great. And again, it's stimulating, it's rewarding. Yeah, and uh, and and that's why we are available to our patients 24-7 and uh and ready to help them.

SPEAKER_01 23:31

Today at the American Diabetes Association, you're gonna be presenting clinical data at the scientific sessions to you know physicians and clinicians all from around the world. They want to hear and see your work. That's what this conference is all about. What do you think will be the most interesting part of your presentation today?

SPEAKER_00 23:50

Right. So um I can disclose since this will be air after the presentation, the results are really good. So we have 12 all 12 patients are are off insulin. We reverse diabetes in all 12 patients, they're doing well. There is nothing unexpected. Um, so the study continues, and then the longest patient has been off insulin close to two years, and we will extend the therapy for all of them longer. Um, so yeah, so this is just reassuring. In this particular trial, our patients have been open on social media, so the progress has been has been, I mean, the information about progress has been there. So I don't have any breakthrough. I can reassure that the outcomes are continue to be very exciting.

SPEAKER_01 24:37

Yeah. I think the last thing I want people to know is, you know, the world of social media has changed a lot of medicine. And a lot of clinicians, including people like yourself and scientists, you're not here to um be a king or a celebrity, right? You came to do something good, and that's your job, and you you took that oath. And it's definitely a weird or different feeling landscape that we're in now that people want to hear from you. And they, I hope that you feel I hope you feel important and valued, but I mostly wanted you to share your story because I think people do want to hear from you and your real feelings and experience with this. I think it is important to them.

SPEAKER_00 25:25

It is, thank you for that. But it is important to us, but it's also creates additional pressure and expectations. Um, but nevertheless, as I said, we do everything we can to help patients and hope for the best.

SPEAKER_01 25:42

Yeah. Well, thank you so much for your work and sharing all of these experiences. I appreciate you.