Podcast: Building the future of childhood cancer medicines
In this episode, journalist Dylan Kissane of Labiotech and Tony Hickson, a member of the C-Further Joint Steering Committee, discuss the critical challenges of developing drugs for childhood cancer. Tune in to this 10-minute discussion to understand what prevents innovative science from becoming tangible medicines for young patients, and how emerging science, collaborative models and child-first approaches could help break the cycle.
Building the future of childhood cancer medicines
Credit: Dylan Kissane. & Labiotech
Journalist Dylan Kissane (Labiotech) and Tony Hickson (C-Further JSC member) discuss the critical challenges of developing drugs for childhood cancer. Tune in to this 10-minute discussion to understand what hinders innovative science from becoming tangible medicines for young patients, and learn about emerging science, collaborative models, and child-first approaches that are working together to break the cycle.
00:00:00 Dylan Kissane
The last few years, last few decades, we've only had a handful of drugs that are approved specifically for childhood cancer.
00:00:08 Dylan Kissane
Why has this just been neglected commercially?
00:00:10 Dylan Kissane
Is it just that rational decision making on the part of pharma companies?
00:00:15 Tony Hickson
Well, yeah, I think it is.
00:00:16 Tony Hickson
I mean, I know the data is in the last 20 years, there's only been 9 approvals exclusively for children from the EMA and the FDA in the USA.
00:00:26 Tony Hickson
So compared to 60
00:00:29 Tony Hickson
adult indications alone in a single year in 2024.
00:00:33 Tony Hickson
So there is a real dearth of new medicines being developed for paediatric child and young people's cancers.
00:00:41 Tony Hickson
And the reason for that is that they are generally small patient groups.
00:00:46 Tony Hickson
they're very difficult to actually drug.
00:00:50 Tony Hickson
Much of the biology here is developmental.
00:00:52 Tony Hickson
I mean, children are not small adults, they are still developing.
00:00:56 Tony Hickson
So their biology is different and a lot of it is very complex.
00:01:00 Tony Hickson
There's fusion proteins and transcription factors and all sorts of things involved that are traditionally difficult things to hit with a drug.
00:01:08 Tony Hickson
And then of course there's just the market size type thing.
00:01:11 Tony Hickson
Many of these conditions are also by definition rare conditions.
00:01:16 Tony Hickson
Now, sometimes you see some great successes like the day one sort of story where there is an adult follow on to that.
00:01:24 Tony Hickson
So you start into pediatric cancer and then you can progress into adults.
00:01:27 Tony Hickson
And there's a commercial case to be made for that.
00:01:29 Tony Hickson
It's a sort of orphan drug story.
00:01:31 Tony Hickson
But sometimes there are tragically a number of childhood cancer conditions that just don't have an adult follow on.
00:01:37 Tony Hickson
They will only ever be in that market.
00:01:39 Tony Hickson
And I think that's where we're very interested as a charity in trying to help sort of
00:01:44 Tony Hickson
correct this broken market problem.
00:01:48 Dylan Kissane
I want to ask a little bit more about one of these companies, which is SeeFurther.
00:01:52 Dylan Kissane
They've got two therapeutic programs at the moment, CF012 and CF033.
00:01:58 Dylan Kissane
What are these programs trying to do?
00:02:00 Dylan Kissane
What are they focused on?
00:02:04 Tony Hickson
Yeah, so just to be clear on that, SeeFurther is not a company.
00:02:07 Tony Hickson
It's a joint venture between Astels, Cancer Research Horizons, COUK,
00:02:14 Tony Hickson
Life Arc is another charity in the UK and they're focused on rare diseases and Great Ormond Street Hospitals charity, which clearly is focused on pediatrics.
00:02:25 Tony Hickson
So we've all come together and put some money on the table and some support and people and teams to try and move forward some ideas in this space and really put a bit of pressure on the system because you end up caught in a cycle where because there is no market for them,
00:02:43 Tony Hickson
there's no impetus to start development of them and then nothing happens.
00:02:47 Tony Hickson
So we're trying to put some pressure on the system at the start to say, well, actually, we are developing some molecules here.
00:02:52 Tony Hickson
They're novel and we need to find a route forward for them.
00:02:55 Tony Hickson
So let's sort of embrace that challenge and move forward with that.
00:02:57 Tony Hickson
So CFurther is that joint development.
00:03:02 Tony Hickson
It has raised about $50 million so far.
00:03:06 Tony Hickson
and announced the funding of two programmes, but we have several more that we're currently sort of homing in on and hope to, and almost certainly will announce more before the end of this kind of year.
00:03:19 Tony Hickson
The first two programmes, one is developing a drug against Ewing sarcoma, which is a sort of bone and soft tissue disease primarily in children.
00:03:28 Tony Hickson
It's with ourselves, the University of Virginia Cancer Center, Dana Farber and Mass Brigham in Boston.
00:03:36 Tony Hickson
And we're trying to develop a new sort of small molecule, first in class small molecule for that.
00:03:42 Tony Hickson
And what we offer we see further is it's not just a here's a grant, go and do it.
00:03:47 Tony Hickson
We're offering our drug discovery capabilities alongside the funding, alongside the biology capabilities within those universities.
00:03:54 Tony Hickson
So it's really trying to bring it all together.
00:03:57 Tony Hickson
The other programme is slightly different.
00:03:59 Tony Hickson
It addresses several different types of childhood cancer and it's with actually Mink Therapeutics and the University of Southampton working together and it's an off-the-shelf cell therapy format.
00:04:12 Tony Hickson
So we're trying a very different type of approach, looking at sort of trying to address some of the limitations in that therapy area.
00:04:19 Dylan Kissane
Is it more difficult to find the academic researchers in the 1st place who are interested in childhood cancers just because?
00:04:27 Dylan Kissane
of this commercial, this lack of commercial appeal.
00:04:35 Tony Hickson
Yeah, I mean, I think, yeah, there are, the pediatric oncology community is a very tight knit and very well networked community who do an amazing job.
00:04:45 Tony Hickson
They're incredibly good at engaging with patient advocates and they're highly impassioned people who really want to fix this market broken problem here.
00:04:54 Tony Hickson
So they're all looking for different solutions and certainly
00:04:57 Tony Hickson
we feel very embraced by that community and bringing new ideas, expressions of interest to us and trying to move things forward.
00:05:04 Tony Hickson
But I think they would admit along with us that that community is still relatively small in the grand scheme of things.
00:05:11 Tony Hickson
And that means there's only a select number of new start points for therapeutic medicines for us to sort of harvest and mine and move forward.
00:05:21 Tony Hickson
And there's lots of challenges in developing those medicines.
00:05:24 Tony Hickson
So I think it's important that we
00:05:26 Tony Hickson
focus both on the academic community as a source of new ideas, but also on industry, on biotechs and pharmaceutical companies who may have stored or shelved assets.
00:05:38 Tony Hickson
So maybe for a pharmaceutical company, they stop development or something because they don't consider the market to be big enough, but we could help them move it forward.
00:05:47 Tony Hickson
Or it may be for a small biotech company that their venture capital funders are focusing all of their efforts on their lead molecule in a certain indication.
00:05:56 Tony Hickson
And then number two and number three, which could be great in childhood cancer, are being sort of neglected a bit.
00:06:02 Tony Hickson
Again, could we help get out, structure a deal, move it forward?
00:06:07 Tony Hickson
So I think that whilst it can be challenging to find some of these ideas, I think that there is still plenty of scope for us to move forward a number of exciting programs.
00:06:17 Dylan Kissane
You've described See Further in the past as a child-first approach to discovery and development rather than just adapting adult science.
00:06:25 Dylan Kissane
And you mentioned children are not just small versions of adult, they're very different biology.
00:06:30 Dylan Kissane
What does that actually mean in terms of mindset and what sort of practical impact does that have on the science?
00:06:38 Tony Hickson
So I mean, I think there's child first and child only and See Further will do both.
00:06:44 Tony Hickson
But what we won't do is adult only.
00:06:46 Tony Hickson
So we are child first is of, well, child only is of particular interest to us because as a charity, we are uniquely positioned, you know, and as a group of charities, we've got and LifeArc to take on the risk of moving these things forward and find novel ways of getting them funded, novel access to clinical trial structures and things like that.
00:07:08 Tony Hickson
And frankly, it's very unlikely that industry or venture capital will ever take forward some of those sort of niche conditions.
00:07:15 Tony Hickson
Child first is also of interest as well because the incentives that have been created by governments, which are great to see, have not always worked in terms of getting the biopharmaceutical industry to run pediatric trials either alongside or shortly after the adult ones.
00:07:33 Tony Hickson
There's a very large delay, historic delay between starting a pediatric study and the adult drug reaching the market.
00:07:40 Tony Hickson
Most of the time the adult drug actually reaches the market well before they even start the first pediatric
00:07:45 Tony Hickson
Some recent legislation changes in the US have helped start to create that, but there's still sort of more to be done there.
00:07:52 Tony Hickson
So I think we are very interested in that child first model.
00:07:56 Tony Hickson
in terms of building a portfolio alongside the child only model.
00:07:59 Tony Hickson
And you want a mixed portfolio of both really, because that maximizes the chance of seeing success.
00:08:05 Tony Hickson
And it maximizes the chance of us leveraging more money for Sea Further in the future.
00:08:10 Tony Hickson
Because we're going to need more than $50 million.
00:08:12 Tony Hickson
We're going to have to raise money against this.
00:08:13 Tony Hickson
We need philanthropy and industry and other people to gather around it and sort of believe in the cause we're trying to create.
00:08:20 Dylan Kissane
So what does success look like then for Sea Further if we're thinking five years ahead, 10 years ahead?
00:08:27 Tony Hickson
I mean, I think we want a science, we want to see a science-led portfolio.
00:08:31 Tony Hickson
So the first task is to create a portfolio because of this attrition problem we've spoken about before.
00:08:37 Tony Hickson
We need a significant portfolio of assets all being developed all at different stages.
00:08:42 Tony Hickson
So early preclinical, late preclinical, in the clinic and so on to really show the model is moving and we're moving a number of things forward.
00:08:49 Tony Hickson
And we'd like to see that a mixed bag of child first and child only indications within that covering a broad depth of diverse cancers.
00:08:57 Tony Hickson
We don't want to all focus on certain types of cancer.
00:09:01 Tony Hickson
And then I think we're also thinking about the commercial model.
00:09:04 Tony Hickson
So how do we move these ideas forward?
00:09:07 Tony Hickson
There'll be a combination of licensing some to industry, some there won't be any sort of industry take-up.
00:09:15 Tony Hickson
There may be the risk is considered higher, so we're going to have to take it further into the clinic before we'll get industry engagement.
00:09:22 Tony Hickson
And then the third category is there will never be industry engagement.
00:09:26 Tony Hickson
And they're the ones we're really going to have to think about novel models with.
00:09:30 Tony Hickson
And I guess the final part is we want to become a voice in this market.
00:09:34 Tony Hickson
We want to shout from the rooftops.
00:09:36 Tony Hickson
This market is broken.
00:09:38 Tony Hickson
It has a problem.
00:09:39 Tony Hickson
We need collective solutions here.
00:09:40 Tony Hickson
We need everyone to crowd in and work with us or us work with others to help solve this.
00:09:45 Tony Hickson
So we'll probably put out some white papers and some policy influencing things as well alongside the developing of the drugs.
Hear the full podcast here: https://podcast.labiotech.eu/1995493/episodes/19602865-how-cancer-research-horizons-is-building-the-future-of-childhood-cancer-medicines