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Stem Cell Clinical Trials and New Therapies for Patients: Alpha Clinic Director’s Panel

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CIRM-funded Alpha Stem Cell Clinics are a network California medical centers that specialize in delivering stem cell clinical trials to patients. Sandra Dillon shares how their work has impacted treatments for her rare cancer. Maria T. Millan, M.D., Mehrdad Abedi, M.D., Daniela A. Bota, M.D., Ph.D., Sheila Chari, Ph.D., Noah Federman, M.D., Catriona Jamieson, M.D., Ph.D., Sean Turbeville, Ph.D., and Leo D. Wang, M.D., Ph.D., then discuss current clinical trials and the future of stem cell research.

Credit: University of California Television

Video Transcript:

[MUSIC] It’s my pleasure to start the afternoon session and I’ll introduce myself more formally in a little bit. But most importantly, I’d love to introduce Sandra Dillon, who is going to give us an account from the perspective, not only if a patient but a patient advocate, and a very important member of the whole ecosystem that continues to drive and accelerate research and advancements in this ecosystem as we heard about a little bit this morning. Sandra, please. [APPLAUSE] Hi, everyone. I think earlier today you had a panel discussion about space and I’d like to talk a little bit about time.

I have a rare cancer called myelofibrosis and it’s scarring my bone marrow and inhibiting my ability to make blood cells. This cancer is progressive, it is exhausting, and it is deadly. When I was first diagnosed with myelofibrosis, they had no cure. My only treatment options were to manage my symptoms as I got worse.

Left to run its course, myelofibrosis has a median survival of 5-7 years.

I was diagnosed at 28. At that time, I thought of my lifetime in terms of decades, like many of them. That was shrunk to years and just a handful of them. This is jarring. As my time began passing, every moment felt so impermanent and so precious, and how limited it was.

I wanted my time, this precious time, to mean something, to have value worth this sense of preciousness. I wanted to do something important, I wanted to change the world.

But how? I’m just this one little person and I have cancer that is killing me. Then it hit me.

If I could just find a bunch of really smart, really driven passionate people, we’re going to do something great, and we’re going to change the world. I could work with them and I could contribute. Even if it was small and even if it was short, my time would matter because I would be a part of something bigger. Now I was thinking of this in terms of my work.

My life might have gotten shorter but it wasn’t free.

At this time, I was still thinking of my cancer, something personal that was happening inside of me. Something that was stripping away my time and my energy and my life, something that I would endure along with everything I was losing. But that all changed. I had the opportunity to join a trial for a new drug to treat myelofibrosis. That trial is run by Dr.

Jamieson at UCSD with funding from CIRM. Suddenly I was surrounded by these really smart, really driven, compassionate people determined to find answers and to make a difference to save lives, to change the world. Suddenly my time, my cancer, my fight with it, it mattered. It was making a difference. I started that trial over 12 years ago.

Since then, this drug is FDA approved and it is helping others like me and I’m still here. [LAUGHTER] That acceleration of time from research to patient, that is by design and determination, and that changes lifetimes. I am grateful to be standing here in this building. I’m grateful for what it means. I am grateful to be here with you smart, driven, compassionate people.

I have time, so let’s all go change the world and beyond. [APPLAUSE] It is my distinct pleasure to introduce this afternoon’s panel to discuss the Alpha Clinics, which is a very special program that CIRM put in place years back. I’m Maria Millan and I have the pleasure and the honor of being the president and CEO of the California Institute for Regenerative Medicine, which is your agency. Just for a brief introduction for those of you who are not as familiar with CIRM, CIRM was created in 2004 through a three-billion-dollar bond initiative. This truly was created in California by Californians.

Fast-forward to today, what CIRM has been able to do is to build an ecosystem that in a patient-centric fashion continues to fuel and accelerate basic, translational, and clinical research, as well as build the future workforce and leadership of tomorrow with our amazing education programs which are supported by our institutions around California, as well as build critical infrastructure, and one of our crowding joules is the Alpha Clinic’s Network, which you’ll hear about more shortly. CIRM’s mission continues to be to accelerate world-class science and to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world. That means to the real world, to our diverse populations, to all Californians, including those from underserved populations. That has been a major focus for CIRM, especially in this new era. You’ll hear a little bit more about that in upcoming meetings from CIRM.

But meanwhile, just to introduce today’s session, I want to proudly announce that the field is continuing to grow.

I think when we started this whole endeavor, maybe 6-7 years ago, we were wondering if there were enough clinical trials in the cell and gene therapy space to warrant investing in infrastructure specifically to support cell and gene therapy clinical trials. Worldwide, the number of cell and gene therapy clinical trials is continuing to grow. This map of the world was provided by the Alliance for Regenerative Medicine, which tracks clinical trials. You can see that the majority of trials are in North America.

Surprisingly even today, most of these trials are being conducted by academic sponsors as well as government sponsors. If you’ll look at that, California has made a significant contribution to this progress. If you just even look at it in terms of clinical trials, not to mention the incredible science which you heard about this morning and the potential for that. The Alpha Clinics Network was launched in 2015, and since then has supported over 200 clinical trials to date, industry as well as academic trials. Over 1,000 participants in these trials.

We’re going to hear this afternoon from our esteemed panel some amazing examples of how this has all been made possible.

In this photo, I just wanted to take a little walk from memory lane. We just took a picture outside the auditorium today. As you’ll see on the bottom right hand, when you’re looking at the screen, is a picture in that same position in 2016 in our first Alpha Clinics Network symposium. Then you can see in the other pictures, all of the following years where there were these symposia at the various Alpha Clinics.

What you’ll hear about today is just a snapshot of what’s been made possible through this network and sharing knowledge, experience, and advancements. Without further ado, what I would like to do now is to allow our panelists to please introduce themselves briefly and where they’re from.

The list is here. If I may please ask our directors to please introduce themselves, starting with Dr. Abedi.

I’m Mehrdad Abedi. I’m the Director of Alpha Stem Cell Clinic at UC Davis and a bone marrow transplant. Hi, I’m Daniela Bota, I’m the director of the Alphas Stem Cell Clinic at UC Irvine and I’m a professor of neurology, neuropsychology. Catriona Jamieson and I go by Cat and I’m the director of the Alpha Clinic here at UC San Diego and also the director of the new Sanford Stem Cell Institute and very happy to know Sandra Dillon. Thank you for being here.

Hi, Noah Federman. I’ll be the new director of the UCLA Alpha Stem Cell Clinic.

I’m the current director of the Clinical Translational Research Center at UCLA and I’m a pediatric hematologist , oncologists by training. My name is Leo Wang from City of Hope. I’m also a pediatric oncologist and stem cell trans-planter and I’m from City of Hope.

Thank you so much. On this screen, can I please ask Sheila to first introduce? Hi, thanks Maria. I’m Sheila Chari. I’m editor and chief of the Journal Cell Stem Cell.

Then Sean Turbeville. Yeah. Thank you, Maria. I’m Sean Turbeville. I’m the vice president of medical affairs and policy at CIRM.

Thank you so much, everybody. I’m going to go ahead and kick off some of the panel discussions and I’d love for this to be interactive. Along the way, if there’s follow-on comments and conversations, we can continue to do that. But I’ll also want to make sure we cover a lot of the ground that we discussed, so I may move things along. But I wanted to just say that this has been an incredible symposium so far.

This morning we had the transformative science described by Dr. Blackburn herself and that how that has actually transformed how we look at aging and what’s made possible through understanding that biology.

Then of course, the amazing tools that were in progress and the signs of precision medicine and application of genomics that was presented after that. With all that, we then ended the morning with presentations from space, giving us a lot to think about regarding other dimensions and opportunities to add to the already incredible opportunities that we’re seeing with the advancement of cell and gene therapy and science. Having said that, I would like to ask the panelists from where you seat, what do you think are the most novel advancements that you’ve seen made possible within your program?

I’m going to go ahead and just let it be a free for all, but I guess maybe I could call on Daniela Bota first and then you can kick it off and tag somebody next.

Excellent. I’m happy to do that. As Maria said, we have been a network since 2014, 2015. When we all started, this network was just getting ourselves involved in cell and gene therapy.

For me, the most amazing things is how we had grown into this time. At UC Irvine, we started with one study in retinitis pigmentosa and now we are conducting 60 studies in 20 plus indications covering everything from neuroscience, which is our forte, to cancer and to other degenerative diseases.

The impact of this growth that we’re seeing in the State of California has empowered us to offer the therapies to many patients from all our counties around us. I think that I’m going to tag Cat. [LAUGHTER] Thank you, Daniela.

It’s great to have friends [LAUGHTER]. You can see that there’s actually quite a lot of camaraderie among this network because we have really shared the trials and tribulations. I have tried to do early-stage clinical trials, but also to develop new therapies with the partners in the room and our partners at CIRM as Sandra so eloquently mentioned and stated the importance for.

I think the most important part for our Alphas Stem Cell Clinic is focusing on the number two cause of death in this country and that’s cancer. How do you get cancer?

How do you eradicate it before it really gains hold? That gave us the idea to tackle stem cell properties in cancer, whether it’s with Sandra’s cancer or with other malignancies like we’ve worked on with Jim Bright Meyer and Mary Bright Meyer who are here in this room and they were in, I think one of the pictures there at City of Hope when we first presented how we make new therapies. I think the key is to be able to make the full depth and breadth of therapies. I think by some focus to regenerative medicine I would say we need people like Sandra to be able to have small molecules, biologic, cellular therapeutics, and sometimes in combination. Because the sustainability piece of targeting stem cell properties in cancer is not just important for us as scientists and physicians, but for all of us if we become patients.

We need to have that choice. It’s about freedom. I think with stem’s we have very broad portfolio, we’ve been able to do that with stem two schematic is the one unique thing that is now moving to cell and gene therapy, even as a biologic is a monoclonal antibody is substantially changed the lives of people with mantle cell lymphoma and CLM. We were able to share that with our network partners very quickly. That’s the case for my dad, is a case for Daniela and as we expand City of Hope of course, and expand to UCLA in terms of opportunities.

I think that the key here is sustainability of these relationships. We talk about erudites science. But actually, the luxury for us would be to give up on these relationships. That’s a luxury that patients can’t afford. I think if we stay united and continue to build these programs and then will make newer therapies faster.

Thank you so much. I’m going to go ahead and tag Dr. Wang. I wanted to specifically ask Dr. Wang, I think one of the really dramatic effects that we saw when we formed the network is how the City of Hope in particular became a go-to site for CAR T therapy clinical trials.

If you could describe that and actually you are involved in next-generation CAR T programs, so I’d love for the audience to hear about that program and why it’s so unique and uniquely developed within this setting? Yeah, sure.

I’m a new kid on the block. I’m assuming the directorship of City of Hope Alpha Stem Cell Clinic, which for years was led very capably by John Zaha, who’s a leader in the field of gene therapy. In 2013, I was actually in Boston, and the idea that an entire state could imagine and then realize the capacity to build a structure that would take lab ideas and turn them into medicines that actually helped people was remarkable.

Even in Boston where we feel like if things come from another place, they’re probably not as good.

Sorry, that’s a Boston joke. [LAUGHTER] I left Boston. I was actually very fortunate to participate in one of the UCLA gene therapy trials for X-linked chronic granulomatous disease and we treated one of the first patients in Boston. I was able to see firsthand the dedication that CIRM and the scientists here have towards taking these things that are great ideas and great publications, but then actually improving people’s lives.

And that was one of the reasons that I came to City of Hope, where as Maria said, City of Hope had been invested in building CAR T therapies for a long time. CAR T-cells are reprogrammed immune cells that can seek and destroy cancer cells. But turning, again, turning that from a high-profile publication in a journal to a medicine that actually helps patients is an enormous undertaking. It can’t really be done very easily at a single institution without an enormous network and support from a place like CIRM.

The contrast was stark, coming from a very highly regarded research institution, but coming then to an institution that is embedded within the office stem cell clinic network where the focus really is on translation.

It’s on lowering the barriers so that patients can get access to these cutting edge therapies and really making a difference. I’m honored to lead CAR T-cell therapy trial for children with brain tumors. These are devastating diseases and kids don’t have really great therapeutic options, unfortunately. Much of the time, survival is measured in months, if not weeks. There’s an urgent need for better therapies for these children.

There’s no place better than California to accelerate that timeframe to give them more time. Thank you. Thank you so much. Maybe in the next question, I’d love to hear all of the programs here have oncology programs and how it’s benefited from being within institutions that have such robust regenerative medicine programs. How, even as you’re in the clinics advancing the clinical trials, how the science, while it’s continuing to advance, have really both partnered with as well as informed the next-generation approaches and improvements.

I gave you a little bit of warning there so that you can prepare [LAUGHTER] and then I’m going to go ahead and let you tag the next person, please. [LAUGHTER]. I believe it’s Nolan. It’s a good segue. We were traveling to Boston the summer.

Leo and my daughters wanted to know where in California Boston was [LAUGHTER]. We sort of in California were so centric and everything outside of California is a different country and a different solar system, so to speak. I think that’s true. Just a little background about me. You’ve heard a little bit before.

I’m taking over for Dr. John Adams. I’m like Leo were both new to this, but I think both of us are pediatric oncologist, [LAUGHTER] and hematologists. But I think we specialize in the rare and the ultra rare. We are orphan disease physicians.

We deal with orphan therapeutics and both from industry and homegrown. What’s amazing to me is now I get to take credit for all of this stuff that’s going on at UCLA and beyond and watching patients benefit, watching our own communities benefit from the diseases that 10 years ago, 20 years ago, 30 years ago were death sentences or we had to do bone marrow transplants, e.g.

For severe combined immunodeficiency. These are trials led by Don Cohen, who’s I think deserves a Nobel prize or a Nobel Peace Prize depending on what you know of him.

But watching the patients that he would treat come in with looking like no infant I’ve ever seen. Then over six months to a year having a normal development and a normal immune system. All of these trials, and that’s just one example coming through UCLA. But all of these trials are funded by CIRM. They are really pioneered by investigators who on their own are amazing people, but they need help to accelerate their therapeutics into the clinical sphere to patients to cure them.

I think that’s where CIRM and the office stem cell clinics really in my role now I’m just honored to be here to help accelerate accelerate these therapeutics.

So I’ll stop there. Thank you so much and then I’m going to put it another [LAUGHTER] bait in the water about that statement, about Dr. Cohen, by the way, who is a Lifetime Achievement Award winner at the last American Society for gene and cell therapy. An amazing award.

But I wanted to say that Don’s platform for monogenic gene therapy is something that also has a multiplier effect across various institutions and investigators, including at UC San Diego, one of the homegrown programs that was made possible. We first heard about it, the alpha clinic Symposium, which I’m sure Dr. Jamieson will tell tell us about. I’d like to turn it over to Dr. Abedi.

Sure. I’m adult side of the hematology, oncology, bone marrow transplant. We joined Alpha clinic somewhere in the middle and that at the beginning, but not recently either. Maybe started. We have all these programs there in our university, in our medical center, but none of them are talking and none of them are actually moving forward.

We just didn’t have an infrastructure to get them going. Alpha stem cell clinic gave us this structure. We were able to go around and talk with every division, every department telling them that here we can get you going from the pre INT to INT to clinical trial. You provide all this structure for the cell therapy part of it. Having a storage areas, having manipulation areas, having GMP facility.

We can manufacture these things for you. We can get it to your patients directly. Having that infrastructure really jump-start a lots of clinical activities. Again, we started from very few to 30, 40 trials that we’re running right now, plus all the other industry sponsored trials that’s happening.

The all value that added was the network.

For the first time we were able to just go around. This has been the most collaborative, I’ve worked with many other collaborative groups there, but it has been the most collaborative, easiest group there. We had our cell phones, the directors have our cell phones and we’re talking so closely dip. Every trials were discussing and say, okay, we have this one. Our interests and to participate.

How can we get it together as soon as possible and moving forward? That has created a really good Environment. Going to the next level now with the Serum 2.0, now we are able to get a lot more done at this point. We have the infrastructure in place.

We have all the glitches of the clinical trial worked out to be able to do it centrally with all the other groups together and I think this could be a very exciting time.

Thank you so much. I think we made our way through the in-person directors and so I’m going to turn it over to Sheila, and from your perspective, you’ve been watching us watching the field both scientifically and from the clinical translation perspective. Love to hear your thoughts at this point. Yes.

Thank you, Maria, for inviting me to participate on this panel.

I realized I’m a bit of an outlier, but I really appreciate speaking with this group because at cell stem cell we share a lot of the same goals as Serum with regard to supporting basic discovery and promoting therapeutic development of stem cell technologies. If you don’t mind, I’m going to take a step back and just introduce the journal to some people who may not be as familiar with it and so cell stem cell is a leading stem cell bioengineering and Regenerative Medicine Journal and we’re published by Cell Press. We published about 100 articles a year and so this ranges from brief reports and articles reporting on rapid developments in the field and more relevant for this audience, we publish clinical and translational articles which are bench to bedside studies in regenerative medicine that span key inflection points across the translational continuum and so this ranges from pre-clinical IND enabling studies all the way to early phase clinical trials.

From a publishing perspective, the two elements of STEM that really stand out to me are these focus on strategic collaborations and enhancing access.

These strike me as essential ingredients for publishing at all stages of therapeutic development. Would I look served? That’s a little different for a field like regenerative medicine as opposed to a field that’s a little more mature like oncology, is that there’s great value in publishing early data that leads to a trial. For example, IND enabling studies as well as very small pilot trials and clinical trials with small numbers of patients. With what STEM is added, it’s this willingness and ability to publish at these very early stages is really enhanced by the de-risking and openness that comes from STEM funding, as well as these partnerships that everyone’s been talking about between the Alpha clinics and their academic collaborators.

I want to stress the importance of access here. Another aspect of this that has become more into my awareness recently is that publishing these early studies that lead to a clinical trial, it’s actually really important for a patient and a woman and so authors have told me that publishing a paper in Cell Stem Cell has been important for enrolling patients in first in human cell therapy trials using very potent stem cells because they could actually share with patients the safety and toxicity data on the very same cells that would be delivered to them.

The fact that this is published in a reputable journal has really helped give peace of mind to patients and concrete information that they can take and discuss with their doctors as they’re deciding whether or not to enroll in an experimental treatment. In many cases, what I’ve observed is this transparency just isn’t feasible without a partner like serve in the process. Thank you so much, Sheila.

As we always say, good medicine starts with strong science so thank you for allowing the community to be able to share the science with the broader community.

Sean is a recent arrival at STEM, comes from industry and in the medical affairs field and so it’ll be interesting to hear what Sean has to say in terms of his seven months so far, understanding what we have at STEM with the Alpha clinics and how he views this in the context of what it takes to bring therapies out to patients, clinical trials and therapies out to patients. Yeah, certainly. Thank you, Maria. First of all, as Maria mentioned, seven months on the job, haven’t had the opportunity to see every single site, but I can tell you from my experience what I’ve seen so far, enormous potential, and hats off to the Alpha clinics.

I had no idea quite frankly that the scale of activities all the way from manufacturing, obviously for the clinic to the bedside, but also the ability to almost commercially manufacture therapeutics.

When you think about the totality of the skill set and the expertise, there really is no limit with respect to possibilities with respect to therapeutic development. Incredibly impressed with what has been put in place today and look forward to some future activities besides. There are a couple of things, of course, that are really important to STEM and many of the Alpha sites are starting to take the lead on this. One is of course, is the community.

As Maria mentioned earlier, there’s a tsunami of cell and gene therapy trials right now approximately what, 23, 2,400, there’s more around the corner. FDA predicts that there’ll be approving 10-15, maybe 20 cell and gene therapy products a year and if you think about the infrastructure in the community, not only in the facilities, but also the patients there right now does not have the operational expertise to be able to fulfill those types of therapies to those patients out in the community so that’s a big push for STEM right now is to start teeing up, what is going to be needed out in the community, particularly the community care centers of excellence and to start building that infrastructure to support these therapies as they go out to the clinic.

The other thing and Sheila just mentioned on this, it ties in as Sheila’s comments. On the regulatory side to some extent, one is a big push for expanded access programs so those are becoming more and more, I think methodologically unique. I think competent authorities are now looking to expand the capabilities of those expanded access programs almost to the point where they can be enabling type of trials if you will, so I think that’s going to be one of the initiatives that we’d like to see as a value on or value add moving forward.

Then finally, this also leads into the publication and that is the opportunity with the real-world data generation. There’s an enormous push right now for us from the FDA’s of the world and the EMEAs who came out with their guidance documents, but the Alpha clinics could certainly set precedents with some of those filings utilizing the real-world data so that’s something that STEM would be really interested in moving forward. Thank you, Sean. Just to tack onto some of the conversation that was already started, it would be wonderful to talk about some aspects of what was already brought up. The platform type model, the platform model for bringing therapies and distinct places forward together with the standards that could be something that’s both understandable and brings comfort to the FDA, to the community that this research has gone through, what it needed to go through, but in a more accelerated fashion.

I’d love to hear from all of the panelists what you see as amazing starting points for this and where you see what we can go with this idea. Peter Marks, who’s the head of the Center for Biologics at FDA, had articulated recently that he believes at some point, especially for gene therapy type approaches, that there could be some. He meant that in the best of terms that cookbook similarly, protocols that we have for the lab, [inaudible].

Those of you who’ve been in the lab. A [inaudible] in terms of best practices, best methodology to get to a certain point that can be shared so that investigators and developers can then focus on the unique aspects of how to apply the front end resources and approaches and so that we can both de-risk that because now there’s aggregate body of evidence and confidence in front and aspects of this, as well as promote collaboration and a learning environment.

I’ve said enough, so now I’m going to turn it over to the panelists.

If I could please start with maybe Dr. Jamieson and then you could tag the next person. Thank you. I think Maria, by being such a stellar physician, as a liver transplant surgeon.

We both worked at the same institution. I think you’ve humanized the platform. The platform is all about people. We can talk about these advanced technologies and we sound really fancy. But it really comes down to what Meridad was saying.

We all have each other’s cell phones we’re on speed dial and I think when you look at the people who are really driving this, it’s the patients and we have incredibly brave people like Sandra who are willing to show up again and again to say this matters. But it also comes down to data. I would say the biggest opportunity here is to democratize data through existing electronic medical record platforms. I’m doing a pitch for Epic here, but there’s no potential to corrupt Epic is owned by one person who doesn’t take any outside investment, Judy Faulkner, and she has 150 million people on her epic platform.

I think if we make a patient like me with all the genomics data that’s available to patients on the platform, then we’ll really understand who’s potentially eligible for these trials for ultra rare diseases or even really common diseases where people find they get into really common bad problems where they stop responding.

Maybe Daniela has a trial for that, which she’s often proffered up and shared with our network. But that would be, I think the strength of what STEM has really put into place allowing us to bridge the gap between biotech and high-tech and have AI artificial intelligence platforms to analyze massive amounts of data, but make that available to the user, to the patient.

Again, we’re all going to be patients. I don’t use that pejoratively, I think that this is something that should empower us. Knowledge is power, and we really appreciate what CIRM has done to set up that communication platform.

I think that would be the strength for us right now. Dr. Federman, could you comment on a platform approach to some of another thing that we’ve heard from the FDA is they realized that with that unique aspect of cell and gene therapy, clinical trials often targeting smaller populations but having large effect sizes.

What are the opportunities with some non-typical BLA pathways for patients to access clinical trials and some examples and the Alpha clinic’s network that you’ve been able to use those other pathways? I mean, I think there’s so much to talk about.

[LAUGHTER] I would focus though on what CRM and Alpha stem cell clinics have already done and are doing, which is really removing roadblocks to clinical trials. Whether it’s at your institution across the whole network and working together. Examples of that are IRB reliance. Those of you that do clinical trials and that our patients on trials know that you have to sign a consent and each of those IRBs often have to go through your own institution.

You have to reinvent the will.

You have to reinvent the will with contracts. That’s master contracting, data safety. The list goes on and on and these are all barriers to getting your trial through the different roadblocks and getting it through activation and on-study support. From my opinion, and I say this joking, but I think there’s an element of truth to it. If academia can operate like industry, like SpaceX, even a fraction of the speed of SpaceX, we will cure cancer and many other diseases in a decade.

Maybe that’s a little optimistic. But I think the reality is that we can operate like industry, and one of the ways of doing it is by relying on each other and not reinventing the will. That’s my global thought. Thank you. I think when we’re talking about platforms, what we’re really talking about are interoperability and standardization and whether that’s true for data structures or clinical trial, regulatory affairs and activation, or whether it’s even true about clinical trial design.

It’s really important to be able to speak the same language so that we can learn from each other.

Noah and I are pediatric oncologists and there are about 15,000 kids under the age of 19 every year who get cancer in the United States, which means that in order to do really important clinical trials, we have to work together. I think on the pediatric side for a long time we’ve been working in these networks where we have common trials, we have common IRBs. We all do the same analysis on patient samples so that when I say, “Oh, I found this signal someone in Seattle,” it makes sense to that person.

For our CAR T trial, it is a single institution trial, but there are four other single institution, CAR T brain tumor trials, and we’re all working together to try to develop exactly what we’re talking about, interoperability and standardization.

I think many of those institutions are not inside of California, but with this network, what we can do is think about that from its inception. If multiple different centers are thinking about trials, first, how do we create efficiencies so that if they do open at multiple centers, it’s really the same trial that opens in a bunch of different places, and if it’s not the same trial, how do we make it so that the data goes back and forth, the samples can go back and forth? We all learn from the aggregate as opposed to just the individual.

Thank you. Dr.

Abedi. When we talk about the platform, I’m going to give a different angle on that. You probably know about the cost of these and affordability and access. Some of these BLA is going to have a price of 2-3 million per patient. How are we going to afford that?

Who’s going to afford that? California is going to go bankrupt and California is one of the richest, basically stated here. Can we use platforms to make everything accessible and affordable? This is something my group has been really focusing on of trying to have point-of-care, basically manufacturing point of care with a central monitoring at the same time there. If you’re manufacturing a product there, you have been doing this for the stem cell transplant for a long time.

The stem cell transplant is in my area. We collect the cells from a patient there. They sometimes manipulate, sometimes we don’t manipulate and we give it back to the patients there.

Can we do it just for the same thing for the CAR T-cells locally? Why we should centralize it and have a price tag of $500,000 just for the product itself there?

We are working on the platforms that we can manufacture individually, locally and a point of care in each center and Alpha clinic will be really a good place to start this and then have a central monitoring for the data, so when we go to FDA, in each center actually with AI, we can monitor these manufacturing, do Q&A analysis there and make sure they’re reproducibly coming off of the centers there to be able to have a BLA for products that we can have there. We’re thinking about price tags coming down to 50,000 versus $500,000 for products there, there is a huge difference there. Same thing with platform. Maria mentioned on Colin’s work and we’re looking at the same idea as well. We have more than 10,000 rare diseases there metabolic.

Are we going to have 10,000 companies with two million dollar price set for each one of them? Or can we use the same platform? Just individually come with the product there.

But the process will be exactly the same, standardized in all of the centers there. We just change the vector and for each product there and make this much more affordable there.

I think platforms and Alpha clinic will be really the best place to do this because of the resources and because of the collaborative effect that we have. I would like to close in a different idea of having a manual and maybe a common operating platform. That is in the way on which we approach our relations with the community. The communities of patients, the minority communities around us, the community of children, adults, and senior citizens.

Because many years we have done studies before the Alpha clinics in our own academic centers, and if you look at the results from the clinical trials were conducted, many of those clinical trials will not be applicable to me and you.

That’s majority of the patients will be Caucasian, middle age, white, and males. Why I’m saying it? Because we are here working on a different manual, on a different paradigms. On paradigms on which the work that we’re doing, it’s applicable to the people that we treat and the communities that we serve.

We are all considering novel ways to engage with our patients from community engagement studios to the participations of the patient advocates in our steering boards and steering committees to the discussions that we have with the physicians in the communities because the cost of travel and the economic toxicity to come at one of our wonderful large non-academic centers might be a little bit overwhelming for patients from our remote or medically underserved areas.

I think this is going to be something very wonderful when we are going to be able to have that manual. We already have a lot of it done and a common platform to work together to be able to distribute those therapies. Thank you so much and you bring up a very important point. As we were listening to the scientific presentations earlier today, one thing that came up is the diversity in terms of the cells and the diversity in terms of the background.

When we’re talking about doing the best science, especially when you’re talking about precision medicine, which a lot of the cell and gene therapy approaches are geared toward, how can we do that unless we have representation of what we’re really dealing with because then otherwise we’re developing treatments for very few?

I think that those are such extremely important points, Dr. Botha. I don’t know if Sheila and Sean, you have something to add. Thank you so much, Sheila. [OVERLAPPING].

Just build on some of these points that have been made about democratizing data and expanding access. Firms focus on this is really important, but I also think that publishing has a big role to play here. At the big picture level, obviously it’s very important for scientists to accurately communicate the state of regenerative medicine therapies to the public.

That means both focusing on the progress that’s been made, but also the hurdles to bringing therapies. But one more practical way of doing this is through publishing papers and commentaries that are openly and freely accessible to the public.

Operationally, there’s an important role for funders and publishers to pay and funders like CRM that value equitable access to treatments can also support access to information about the underlying sites with clear commitments and funds to enable open access publishing. On the other side, publishers also need to see the value and enabling open access to research and especially bio medically relevant research like what we’re discussing today. There’s been a lot of movement in this area in the past couple of years and even months.

From my part, I can say that since last year, all the cell press journals are either gold open access or hybrid with an option to publish open access. For example, at stem cell, you’d have the option to publish open access if your friends required it.

But these changes don’t just happen organically. It does take a real commitment from leaders like Sam and other funding agencies and partnerships with the publishers to really continue to expand access for all. I saw there was a question in the chat precisely about this. I very much do support democratizing the results of these trials and basic science research. Thank you, Sheila.

Sean, did you have a comment to that topic? Yeah, certainly. I think all the panelists are spot on when you think about the platform. When I think about the platform, I think about the journey, the patient journey.

All the way from being diagnosed to going into clinical trial, all the clinical care, and now to post-marketing, which is becoming just as important.

That is critical that Alpha clinics are at great position when you think about that 360 journey for that patient. Not just an early-stage, but also with cell and gene therapies, particularly for the competent authorities are asking for post-marketing follow-up data, and the payers will as well. The Alpha clinics are in a great position to be able to provide those services. Thank you so much. That was an amazing response to a very important question about how we can optimize collaboration in platforms as well as increase access.

I think so for the final leg of this panel, I’d love to hear from the panelists where you see the near future and the future future with space or without space.

[LAUGHTER] I think you called it I float research path? [LAUGHTER] [OVERLAPPING] You know how we like to float ideas? Yeah, [OVERLAPPING] about research. Where do you see we’re going in the near future and the not-so-distant future for the field, both in technology as well as approaches?

Yeah, I’d love to comment on that. I think that we’ve worked a lot on where stem cells go awry and give rise to malignancies are really dysfunctional stem cells, as in the case of hereditary stenosis. I think the way the field is going is something we were talking about at lunch, which was our mini pre-panel and precision medicine and actually applying all aspects of sequencing to patient care starting at birth.

That’s something that’s happening here at Brady’s Children’s Hospital that actually served highlighted and said you should work with ratings. I think it’s these opportunities to understand disease in its infancy and really tackle it in the very beginning stages, so we don’t have people with advanced disease that don’t even understand they have a disease because then it’s much harder to tackle.

That’s where key partners that are really rocket scientists literally apply their very clever knowledge, whether it’s Mike Roberts working with us at NASA national labs or NSF or other organizations. I think bringing new partners to the table and bringing industry to the table. I keep looking at Jim Bright Meyer because he’s been our constant partner together with Maria. But remember, there are philanthropists, there’s academic funding, but there’s also industry.

I think if we engage the partners early, that’s where we’re going.

Develop these technologies together and really have them vetted by Sheila. Now no pressure Sheila, but really make them open access plans where we say this is where we’re going, this is where we think we’re going to get it right, and tell us if we’re getting it wrong. This is where we keep each other honest here because our partners will say, maybe you’re a little off track. I tried that it didn’t work and try this instead, I think the future is more about sharing, sharing data, sharing platform, sharing technologies. But with that tripartite funding structure, industry, academia, philanthropy, and we’re very fortunate to live in a place that is in Boston, I think where we share.

That’s a terrible dig of Boston, but I think Boston is a great place.

I was just there three weeks ago. But they’re coming here to say, what’s the magic sauce? What are you doing? Why are things getting done so fast?

While ceramide is a huge catalyst, it’s developing a lot of interest. Yeah. It’s absolutely clear that the sky is no longer the limit in terms of how much we can help illustrate scientifically. I really look forward to being part of this as we move into even just discoveries that I can’t even imagine right now. I think the other part of it, the responsibility that we all have as scientists, clinicians, and public health stewards is making sure that your zip code doesn’t determine your health outcome.

We’ve got to do a better job of making sure that the best new therapies don’t go to the people who have the most money or have the most connections and that’s a responsibility I think that we all feel very, very keenly and are really grateful to serve in investing heavily in. I would like to add to that one thing that we haven’t discussed, which is our responsibility to raise the next generation of clinical professionals, the people that will administer those therapies.

The people that will supervise how those therapies are administered. The people that will produce those therapists. Sam has helped us a lot not only by being a clinical network, but also being a clinical education network.

I’m looking forward to see our next generation starting to administer those therapies that we have spent years working on. Thank you. I think it was mentioned the sky is the limit. That’s really correct. When you talk about the drugs, you go 5,000, 10,000 compounds and you get eventually to one drug and post-marketing that may actually fall apart.

It may not work well. When you’ve been coming back to cell and gene therapy, again the sky’s the limit. You have the full capacity to change things, make it better. Be claimed from the first generation CAR T-cell to second to third to fourth. I don’t know how many generations we are now.

It’s just getting better.

If there is a problem, it’s solvable. That’s the difference between the chemical therapeutic versus the cellular therapeutic there that you can go make it better. Same things. Go back to clinic and make it work.

We went through the same thing 15 years ago and we have the first-generation CAR T-cells. It was working beautifully in the cell tissue in the cultures. I would adding tumors after tumors in challenging the same CAR T-cells with 10 times basically adding the tumors and each time with killing it. We put in a human, it didn’t work at all. We learned why we need co-stimulation molecule.

We need the second generations. Now we’re just getting better and better and better and the idea of cure for the first time. Whatever we did so far with management, the idea of a cure for the first time is that what makes this so appealing and so exciting there. Again, looking forward for the future, I think we’ll see a lot of applications.

We’re not going to treat the common cold with the cell therapy.

But there’s many, many other things that we can do and again, I think we’re all excited about that. We might cure the common cold [LAUGHTER] if a patient has severe combined immunodeficiency. [LAUGHTER] Two things really quick and closing. One statement, which is that I think we are here as the bellwether for the country in the world. For how regenerative medicine therapeutics gets done and I believe that.

The second is a challenge and the challenge to build on what Leo said is, how do we truly reach sincerely the communities that we serve? That’s not just with next-generation therapeutic cell therapy, gene therapy, etc. It’s also with basic health care and education which our communities often lack. I think for all of us that is the challenge. I’ll leave that.

Thank you so much. Sheila ad Sean if you have a quick word. Well, I’ll just add one point and that is, I’m cautiously optimistic and I think we should be celebrating our achievements. But to this point about education, I think it’s really important that we educate the public on the patients that’s going to be needed so that we retain their trust and support. Sam has obviously shown itself a real important partner with patient groups to be a broker for this.

Quick closing comments. First of all, hats off to all down for clinics, I think you have set the benchmark not only for the US, but as Maria mentioned globally. Keep moving mountains. You guys had phenomenal ideas and continued to support everything you guys do so hats off to you. Thank you, everybody.

This concludes our panel and I’ll leave it up to our leader, Dr. Jamieson, who want to close the session and maybe take questions. Thank you. Thank you so much, Maria and everybody on the panel, and specifically Sandra, for reminding us why this matters and how personal it is and to our friends on the panel.

I mean, these are heroic scientists who apply science to the clinic in real-time.

JT thank you again for being here. I’m just wondering if there’s anybody here that would like to ask any questions. We’ve got Maria who’s got plenty of energy. Jim Bright Meyer has a question slash comment. I’d like to provide a short testimonial to this group and our internal therapeutics that I have the honor of running has been vitally connected with Sherman with the Alpha clinics now for several years.

We’re developing a very important roar one antibody that was discovered here at UC San Diego in a project that CAT was involved in in the very early days under a certain grant. A phase 1 study was underway when nocturnal licensed the portfolio from the university under a CRM grant and was published in a cell stem cell so that we had a very nice and credible way to describe what the project was doing to patients and other physicians.

A key phase 1-2 clinical trial which is now essentially complete, was conducted with major support from a CRM grant and a 1/3 of the clinical sites where Alpha clinic sites, Davis and City of Hope, and a major contribution from UC San Diego. Where we are with all that, we have had the pleasure of moving on into phase 3. We’re opening our pivotal registration study right now.

We’ve been asked to consider working with CRM again on that trial. We’re talking to people in the CRM office right now. We’re delighted to have the interest in the potential to work with CRM together again and we’re moving into what all of you are vitally interested in a cellular therapy, a CAR T, again, using science discovered here at UC San Diego to direct CAR T-cells to kill tumor cells expressing or R1.

That’s something that again, could also be something that we could collaborate with CRM again. None of what they’ve been saying is theory, it’s real, it is in motion and it’s worked in the past and we’re looking forward to seeing it work again and continue to work in the future.

Thank you, Dr. bright Meyer. I think that sums it up awfully nicely. We have friends in high places. Thank you.


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