Oncothon: Pediatric Cancer Research with Ricardo Garcia and Ben Rayner

Oncothon is a telethon spanning 24 hours, dedicated to gathering donations for childhood cancer research.

In today’s episode our guests will talk about clinical trials in the pediatric oncology field, discuss pediatric cancer research support, and share their personal journeys in this field.

Speakers

Ricardo García is the Co-Founder, Chief Executive Officer, and a Member of the Board of Directors at Oncoheroes Biosciences. Ricardo has a diverse background in computer engineering, management, and business administration. His journey into entrepreneurship began early, leading to the successful sale of his first company when he was just 22 years old. This led Ricardo to start and grow multiple tech-focused companies, all driven by a passion for innovation.

In 2011, his son Richi was diagnosed with a brain tumor. This challenging experience prompted Ricardo to uproot his life and relocate to Boston to support his son’s treatment journey. During this period Ricardo discovered significant gaps and challenges in the support system for childhood cancer patients and their families.

Motivated by his journey, Ricardo founded the Richi Childhood Cancer Foundation, a non-profit organization with a global outreach. The foundation aims to address the unmet needs in childhood cancer care and support, leveraging Ricardo’s passion for making a meaningful difference in the lives of children and families affected by cancer.

Benjamin Rayner is a Senior Scientist in the Brain Tumours Group at the Children’s Cancer Institute. Previously, he worked at the Heart Research Institute, where his research focused on developing novel therapies for atherosclerosis and heart failure.

In his current role with the Brain Tumours team, Ben is dedicated to identifying and validating new targets for treating diffuse intrinsic pontine glioma (DIPG), an especially aggressive brain tumor that predominantly affects young children. His responsibilities include evaluating compounds that show activity against DIPG, elucidating their mechanisms of action, and discovering additional potential treatment options for the disease.

Looking ahead, Ben aims to develop and apply proteomic approaches to enhance our understanding of DIPG and the various mechanisms involved in its development.

Moderator

Shushan Hovsepyan is a pediatric oncologist and adjunct assistant professor at the Yerevan State Medical University. Currently, she is the Editor-in-Chief of OncoDaily Medical Journal.

She completed her clinical at the National Institute of Cancer in Milan, Italy and at the St. Anna Children’s Research Hospital in Austria Furthermore, she held a research fellowship position at the European Organisation for Research and Treatment of Cancer in Brussels, Belgium.

Dr. Hovsepyan also completed the Postgraduate Harvard Medical School’s Effective Writing for Healthcare program. She is a former co-chair of the SIOP Global Health Network Education and Training Working Group.

00:00 Introduction
1:37 Ricardo Garcia
6:21 Benjamin Rayner
7:51 How Oncothon will bridge future clinical trials in pediatric cancer
8:52 Challenges when you have clinical trials all around the world
9:53 Importance of pediatric cancer research support
14:22 How Benjamin Rayner decided to become a scientist
15:48 Collaboration of Oncoheroes Biosciences with others
20:39 About sites and involved countries
22:35 About ultrasound bubbles

The Transcript of Oncothon: Pediatric Cancer Research with Ricardo Garcia and Ben Rayner

Shushan Hovsepyan: Hello everyone. Today we unite from across the world with a singular mission: to accelerate cancer research and let’s inspire each other, let’s challenge each other. I’m Shushan Hovsepyan, a pediatric oncologist from Yeolyan Hematology and Oncology Center, and I’m very happy and pleased to lead this session with two renowned specialists.

Ricardo Garcia, we already met him, is the CEO of Oncoheroes Bioscience. He’s a wonderful person, who used his personal experience and put it into action. And Ben Rayner from Australia, from the Children’s Cancer Institute, who is a senior scientist in the brain tumors group. I’m very happy to welcome you both. Hello.

Ricardo Garcia: Hello. Thank you, Shushan.

Shushan Hovsepyan: Thank you for being here, and I’m very excited for this opportunity,  because this is a unique and first-ever initiative to help children and raise funds.

So, today I would like to speak with both of you a little bit about the Oncoheroes Bioscience and also about the trial that we are going to launch after the Oncothon. The first question is for Ricardo. Can you share a little bit about what was the inspiration behind launching the first-ever biotech company which is 100% dedicated to pediatric cancer research?

Ricardo Garcia: Well, thank you again for having us here today. The reason that made me decide to move forward and launch a company specialized on developing drugs for cancer was the lack of interest from the industry. As you all know, my son was diagnosed with brain cancer. Everything was new to me, and I started by creating a foundation when I moved to the US to save his life for treatment.

Then everything started by creating a foundation, and I realized the needs of pediatric cancer, the lack of support from the industry. It was very traumatic to me to learn also that there was a lack of interest from the industry to support pediatric cancer, because it is a rare disease.

After talking with a lot of foundations, a lot of pediatric oncologists, a lot of experts from academia, parents, and organizations. I realized there was a lot of frustration. You think about the effort that the community has been put in for so many years to support research, but unfortunately, the few new drugs that were specifically approved for pediatric cancer is unfair. 

Then I was wondering what the problem was, so what we could do, and I realized that the problem was mainly because you need a company at the end of the tunnel to really get a drug approved, to go through all the regulatory processes, and eventually bring the drug to patients. When I was discussing with the pharma industry and asking them why they were not interested in saving lives, their answer was clear to me.

They were more focused in bigger markets because, of course these are private companies, they have to cover a lot of costs, and it’s not justified to invest so much money for such a small market, because you know, there’s supposed to be not-so-big profits on that.

Another person, a mother from another foundation in the US, thought we needed to meet each other because we both had the same vision. He also wanted to launch a biotech company. We were introduced, and it was a perfect match: a serial entrepreneur and a scientist with more than 20 years of experience in the pharma industry. Here we are, and that was a good combination. I’m very excited about what we have today.

Shushan Hovsepyan: Thank you very much for sharing that. It’s truly an inspirational story that you created a company only dedicated to pediatric cancer. We all know that oncology research is very fast-growing in the adult oncology field, but for pediatrics, it takes too long for approvals and regulatory processes.

Thank you for sharing your perspective. It’s really inspirational, but I also want to highlight the challenges not only from the industry part but also from the scientific part. Could you please share, Ben, a little bit about the challenges you face in the scientific part?

Ben Rayner: I guess Ricardo summed it up. The pharmaceutical industry is not really interested in pediatrics. The Children’s Cancer Institute and the Zero Program have made significant progress in identifying molecular characteristics, particularly in DMG and DIPGs, but also other pediatric cancers. They are identifying the molecular targets that we can treat these cancers with. However, getting the drugs from the pharmaceutical industry is very difficult.

That’s the biggest challenge. With DMG in particular, each individual is different, so finding drugs to treat them across the board is very difficult. With PLK1, for example, it is one of the targets that stands out.

When looking through a high throughput screen, many DMGs are susceptible because they have high expression of PLK1. Drugs like VP will treat them across the board. Finding that target and a company that can supply the drugs to actually get into the kids is the biggest challenge

Shushan Hovsepyan: How do you envision that the Oncothon in bridging the funding gap for the Vaser to clinical trial, and how will this be applied to future projects and clinical trials with this drug?

Ben Rayner: Well, definitely the first step is to get into kids and see if it works. The fact that you’re targeting multiple pediatric cancers with this program is very important, especially with PLK1 being highly expressed across cancers. The work that Heidelberg is doing with medulloblastoma and M-driven cancers, and what we’re doing with DIPG

Shushan Hovsepyan: I think it’s very important. It’s actually answering multiple questions at the same time when we have multiple entities with just one drug. So, it’s a very great design to use in this setting. Also, you are connecting from Sydney, Ricardo is from the US right now.

How is the challenge of having a clinical trial across the world? What are the main obstacles that you face? The practical ones for clinicians also?

Ben Rayner: I’m not sure. I mean, I’m not a clinician myself. I’m actually a lab-based scientist working on the preclinical side of the research. But just as far as I can say, it’s actually gotten a lot easier since COVID. I have to say, it’s actually gotten a lot easier because people are used to doing things over Zoom, and you know, we connect regularly with the people in Heidelberg to get updates. So, yeah, I think the challenge is easier now in this day and age. But really, finding people to share information with and to actually drive research from a collaborative standpoint, it’s really good.

Shushan Hovsepyan: Thank you for that. Another question for Ricardo. For those watching today who have never been personally touched by childhood cancer, what message would you like to convey about the importance of supporting pediatric cancer research and initiatives like the Oncothon?

Ricardo Garcia: Well, I think for the ones that have not been touched by child cancer, the ones, you know, I would say, well, child cancer is a disease that is killing, as far as we know, at least, you know, the data we have is more than 990,000 kids every year.

So, I would say we’ve been mentioning this for the whole session, is we’re talking about 250 kids and adolescents who will be gone by the end of the day because there are no specific drugs developed for them. So, I think what we’re trying to prove by building a company specialized in pediatric cancer is that there is a way that we can really change the situation.

And you mentioned something before at the very beginning of the session, is like you mentioned about, well, it’s going to take, which is true, you know, literally, it’s taking forever to develop drugs for pediatric cancer, and it is very slow compared to adult medication.

And it’s true, but it’s slow, it is slow, and it’s taking forever not because it’s complicated, but it’s because of the lack of funding, purely. This is, full stop, there’s no other reason. So, because the truth is that developing drugs for pediatric cancer, there’s a lot of misconception about this. I mean, it is not so complicated as it sounds.

Normally, you know, we have so many different types of regulatory pathways that allow us to get our drugs for diseases like cancer approved faster, you know, they’re different types of mechanisms. There are many incentives for companies developing drugs for pediatric cancers to help us just make a profit and loss our balance more profitable and get more resources to develop more drugs.

But also, I would say, you know, there is also the opportunity to run trials or build clinical trials with not so much money compared to adult medication.

I mean, if you would like to set up a trial for an adult cancer drug, you would probably need millions of dollars, and probably you would need to enroll hundreds of thousands of patients, right, and go up to phase three, while in pediatric cancer, it’s different. You can run a trial with a few numbers of patients.

Well, unfortunately, because there are not so many options and because it’s a very rare disease, right, but we’re talking about enrolling patients, you know, in the number of 50, 60, 70, 100, for instance, you know, the clinical trial we’re talking about right now where we’re planning to test Vaser-T.

So, I believe we’re talking about enrolling maximum 100, 110 patients to test five different types of pediatric indications. So, for each indication, we’ll be having not so much patients, like the total.

So, I think that reduces the cost of a clinical trial and literally makes a very affordable adventure that could be done. It’s just a matter of willingness and, you know, and support from different institutions to provide the necessary funds that we need to run these trials.

But it is easier and cheaper, way cheaper, to run a trial for a pediatric indication. And with just a tiny fraction of what is dedicated to support and the FDA for adult medication, you can get a drug approval to beat cancer faster, cheaper, and quicker.

Shushan Hovsepyan: I always encounter the feeling from regulatory institutions and also some parts of the industry that when you speak about pediatric cancer, they say there are very few patients, so they are not interested. But these are patients, people with families.

It’s important to highlight that even if we have a low number of patients, we can answer many questions and cover the whole pediatric cancer spectrum. It’s really important to emphasize that. Another kind of motivational question for Benjamin: how did you decide to become a scientist and work in the scientific field?

Ben Rayner: That’s a long question. I’m getting a bit old for that. Um, the look, I’ve always been interested in science, and what drew me to pediatric brain cancer in particular is this so much that’s not known.

Um, and it’s that that makes it so much, I mean, it’s purely from a scientist’s point of view, like, it makes it a very interesting field to study, and the fact that you can identify something completely new and investigate things that just are unknown, that’s what sort of inspiring.

Shushan Hovsepyan: Yeah, that’s correct. I remember that I always, when I was writing some motivational letters to apply for some programs, I always wrote that I would like to know as much pathophysiology, biology as cancer cells do because they are really clever and they know how to invade a lot of mechanisms. So, just working in the scientific field is very interesting, and also, you always have the inspiration and the enthusiasm to understand okay, what this cell wants from me. We want to understand.

Another question that I wanted to ask Ricardo regarding the collaboration with Heidelberg and what from the industry part, how is the collaboration built across the board. So you are the industry for people who just are not in the pediatric oncology field or not in the industry field, they want to know how you collaborate with the hospitals and how is that collaboration?

Ricardo Garcia: Well, in that case, we connected with Heidelberg and the entire group, including, of course, Australia because they were aware that we licensed the rights of Volasertib, which is a drug that was initially developed by a big pharma company in Boehringer Ingelheim in Hunt for leukemia.

So during several years, the drug was studied by different groups, and it showed an interesting potential for treating different types of pediatric cancer. So when our company in Oncoheroes got light, you know, got the control on the track, we’ve been approached by Heidelberg because they showed a lot of interest based on preclinical data that would justify the launch of a trial specifically to test Volasertib, you know, the compound in different indications. So because this is our mission, we were very excited.

So, you know, I think this is one of the best calls that we can ever have, you know, the experts, the clinicians calling us and saying, you know, we are interested in your drug because we think your drug could be used for different types of pediatric cancer.

So I guess because this is 100% our mission, so on the collaboration, I think it’s very, and it was very easy and it’s been very easy from the very beginning. So we wanted to, we said yes from the very beginning. We wanted to get on board and we wanted to do our best to support the effort.

So our company will provide the drug at no cost, so that’s kind of our main commitment, and we want to raise additional funds because we need to cover the cost of the shipping and labeling, and also we need to provide some funds to the sites so that they can just cover the cost of the trial.

So we’re doing as much as we’re doing different efforts to raise these funds. One of those is the Oncothon because we’re going to get the funding ready to initiate the trial as soon as possible. But I think the collaboration is so, so easy.

I mean, for me, it’s the first time in the industry because I come from another industry, but I never expected that it would be so easy to work with the academics and the experts. In a way, we are both in the same boat. We’re both motivated and we just move by the same mission.

So I mean, it would be probably different with a company that is thinking about other types of markets, but in our case, I mean, there’s nothing else we’re doing here except for focusing on pediatric cancer.

Shushan Hovsepyan: Just a quick question: Where does Volasertib come from?

Ricardo Garcia: Comes from, again, I think I mentioned before is this is a drug compound that was developed by Boehringer Ingelheim, a German pharmaceutical company. They were developing Volasertib for adult leukemia and it went up to phase three. But the company decided to finally discontinue the compound for strategic reasons.

But during the time that Boehringer was developing the compound for adult indication, there were different groups of pediatric cancer experts from all around the world working and testing the compound for different types of pediatric indications, and they generated very interesting preclinical data that suggested that the drug could be a potential new therapy for different types of sarcomas and brain cancer mainly.

Unfortunately, because we’re talking about a pediatric indication, so by then, Boehringer was not interested in pediatric cancer indication. So the drug was shelved and the program was shut down for a period of time until, you know, there was a company out there specialized in pediatric cancer, you know, was Oncoheroes, and then we were connected to Boehringer, and it was a very smooth conversation, to be honest.

And then they were very open and very willing to help, and to provide the drug to Oncoheroes, so that we could just move forward and try to get this drug to pediatric patients. So that was the story of how we ended up bringing Volasertib into our portfolio.

Shushan Hovsepyan: That’s very nice and could you share also a little bit information about the sites, the countries that are going to be involved, how many sites we have, how many countries?

Ricardo Garcia: We’re talking about, uh, 20 sites approximately, uh, 20 sites in different countries in Europe, UK and Australia, and we’re talking about, uh, the size of the trial will be, we’re talking about five different ARS, we’re talking about 100 patients, more or less, so maybe Ben you have more information and you want to just jump in, uh, jump in because you guys are on on on the scientific side.

Ben Rayner: Yeah, I mean obviously one of the sides run by Professor David Zigler, who’s my boss here, specifically looking at DIPG and effective Volsertib with radiotherapy which is a standard for DIPG and unfortunately radiotherapy by itself it doesn’t cure DIPG and only prolongs a little bit because the tumors do bounce back and so the one thing that radiotherapy does.

Which is why we’re interested in Volsertib is because it opens out the blood brain barrier for that period of time so that the drugs can get into the brain and you know getting a decent concentration which I think is the second arm of the study which is more to do with the Heidelberg group and medulloblastoma.

But they’re using it with vinan which is the partner drug and the vinbin is an is compound as well because it can actually open out the blood brain barrier further and with DMG or DIPG that’s particularly important because we know that DRPG actually closes off the blood brain barrier so having a a partner drug there where you can not only Target the tumor but you can also open out the blood MBR for further drugs to get in there.

It just it it’s an added benefit to us and so that’s that’s why we’re interested in you know doing the clinical trial with with DMG.

Shushan Hovsepyan: I was wondering what do you think about also an ultrasound bubbles that also open the blood brain barrier also the setting.

Ben Rayner: No, it’s actually a focus of another researcher within our group to actually get that into the clinic as well. Yeah, it’s a possibility and we’re looking at disrupting the blood brain barrier in other ways using other compounds that Target the barrier and see if we can actually increase penetration and that’s the key with DMG.

Shushan Hovsepyan: Yeah, actually, it’s a very devastating disease, and we need action. We should act very fast because we don’t have time, especially in DIPG for patients.

Ben Rayner: That’s one thing we actually get feedback from parents, that the fact that they’ve got a child who suddenly gets DIPG and there’s not a long time to do anything. But everything takes so long; just the fact that you’ve got to do the analysis, you’ve got to do the workup, you’ve got to do, ‘Okay, this drug’s good for you, let’s get it into you,’ and by that stage, it’s just a lot of the time, it’s just too late, you know? So yeah, timing is imperative.

Shushan Hovsepyan: Yeah, apart from the research perspective, also if you are working in a low-middle-income country, you don’t have access to the basic medications, the essential ones, and it also delays the treatment. So we need action, and we should work fast.

So I think that the Oncothon is one step forward, so that after one quarter, when we raise funds, we can launch the trial and we can have a lot of answers to our questions. I wanted to thank you both for this session, thank you for being here, thank you Ricardo, and thank you Ben. Thank you for your inspirational thoughts and your insights, your perspectives.

I just want to remind the audience that if you have questions, you can put it in the chat, and also you can write down from which country are you connected. Do you have some connections with childhood cancer? And we can answer those questions as well.

And yeah, again, would like to challenge everyone, let’s make a difference together, and let’s make this event an extraordinary one, so that afterwards we can save a lot of lives in pediatric oncology. Thank you very much both, thank you.