August 5, 2025 • 35 mins
Children’s Tumor Foundation CEO Annette Bakker discusses what non-profit organizations uniquely bring to clinical research and new financial models sustaining their contribution. Plus, host Deborah Borfitz rounds up the latest news in clinical research: new funding approaches for ALS clinical trials, spatial biology to match patients to trials, a bio map of lung cancer tumor changes, improving diabetes treatment outcomes for patients in China, and more.
Show Notes
News Roundup
Fund of Adaptive Royalties
- Research article in PLOS One
TACIT algorithm matching patients to trials
- Article in Nature Communications
Mapping biomarkers linked to treatment resistance
- Press release on the City of Hope website
Pragmatic diabetes trial in China
- Paper about China-specific genetic risk score in Diabetologia
- News of trial launch on the University of Exeter website
Cough medicine to slow dementia?
- Study in JAMA Neurology
Beating the EARL hurdles in the UK
- Study in JAMA Network Open
Guest
Annette Bakker, Ph.D., CEO of the Children's Tumor Foundation
- Article in Clinical Research News
The Scope of Things podcast explores clinical research and its possibilities, promise, and pitfalls. Clinical Research News senior writer, Deborah Borfitz, welcomes guests who are visionaries closest to the topics, but who can still see past their piece of the puzzle. Focusing on game-changing trends and out-of-the-box operational approaches in the clinical research field, the Scope of Things podcast is your no-nonsense, insider’s look at clinical research today.
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Transcript
Episode Transcript
Available transcripts are automatically generated. Complete accuracy is not guaranteed.
Deborah Borfitz (00:01):
Hello and welcome to the Scope of Things
podcast, a no-nonsense look atthe promise and problems of
clinical research, based on asweep of the latest news and
emerging trends in the field andwhat I think is worthy of your
30 or so minutes of time.
I'm Deborah Borfitz, SeniorScience Writer for Clinical
Research News, which means Ispend a lot of time with my ear
(00:22):
to the ground on your behalf anda lot of hours every week
speaking to top experts fromaround the world.
Please consider making thisyour trusted go-to channel for
staying current on things thatmatter, whether they give us
hope or cause for pause.
In another five minutes or so,I'll be speaking with Annette
Bakker, CEO of the Children'sTumor Foundation, about what
(00:43):
nonprofits uniquely bring toclinical research and new
financial models for sustainingtheir contribution.
But first the latest news,including a new approach to
funding clinical trials for ALS,an algorithm using spatial
biology biomarkers to matchpatients to trials, development
of a biomap of lung cancer tumorchanges, improving diabetes
(01:04):
treatment outcomes for patientsin China, the possibility of
slowing Parkinson's-relateddementia with cough medicine,
and how the UK is uniquelyovercoming some of the
timeline-related Earl hurdles inEurope.
A new financing model has beenproposed for developing drugs
for amyotrophic lateralsclerosis, or ALS, to bridge the
(01:27):
notorious valley of deathwhereby promising scientific
discoveries often fail totranslate into viable therapies.
The so-called Fund of AdaptiveRoyalties approach merges the
efficiencies of adaptiveplatform trials, which allow
multiple drug candidates to betested simultaneously under a
single master protocol, with afund that rewards investors with
(01:50):
future royalties fromsuccessful drugs that emerge
from the trials.
A simulated fund of this typegenerated an estimated return of
28%, with a 22% probability oftotal loss, making it
potentially attractive to hedgefunds, sovereign wealth funds,
family offices andphilanthropists.
Returns more palatable tomainstream investors might be
(02:11):
generated by funding multipleplatform trials simultaneously
and employing financial toolsthat bundle future income from
assets like loans or royaltiesinto investment products.
Researchers at VirginiaCommonwealth University have
been working with principalinvestigators across campus to
include spatial biologybiomarkers in their cancer
(02:31):
clinical trials, to predictpatient responses prior to
enrollment and, when individualscan't be part of a trial, point
them toward already approveddrugs that could help.
The guidance is provided byTACIT, short for Threshold-Based
Assignment of Cell Types fromMultiplexed Imaging Data, an
algorithm that assigns cellidentities based on cell marker
(02:52):
expression profiles and, thanksto artificial intelligence, does
the job in minutes rather thanweeks.
Tacit distinguishes cells usingdata from over 5 million cells
across major body systems likethe brain, gut and oral glands.
In a new study, it was found tooutperform existing
unsupervised methods in accuracyand scalability, while
(03:12):
revealing new cellularassociations, and the results
strongly agreed with genetic andprotein datasets.
I'm writing a piece on this forDiagnostics World News, so be
on the lookout for that to learnmore.
City of Hope was recentlyawarded close to $24 million
from the federal government tocreate a biomap of tumor changes
that cause immunotherapyresistance in advanced or
(03:34):
metastatic non-small cell lungcancer.
As part of a national precisioncancer therapy program, it will
be conducting a six-year535-patient clinical trial
designed to adjust treatment asresistance arises, with the goal
of increasing progression-freesurvival by 50% in at least one
patient group.
Investigators will monitortumor trajectory and patients'
(03:57):
response to treatment usingtechniques like liquid biopsies,
single-cell sequencing andradioimaging.
They are also funding thedevelopment, testing and
matching of new biomarkers withtherapeutic options currently
available or in a clinical trial.
Trial enrollment begins within12 months.
A large-scale pragmaticclinical trial has launched in
(04:18):
hospitals across China, designedto improve treatment outcomes
for patients diagnosed withdiabetes by using a
Chinese-specific version of thegenetic risk score to
distinguish between people witha type 1 and type 2 disease
variety, since patients in theregion often don't fit the
classic Asian weight profilewith which the diseases are
diagnosed, and about 25% ofindividuals with newly diagnosed
(04:41):
type 1 diabetes lack detectableislet autoantibodies measured
by early detection tests.
Misdiagnosis of the type 1adult onset variety, whereby the
body's own immune systemattacks insulin-producing beta
cells in the pancreas, meansdelays in getting daily insulin
injections to avoidcomplications.
Type 2 diabetes, which could betriggered by high body mass
(05:04):
index, has several treatmentsand nearly half of patients with
a short disease duration maysucceed in achieving disease
remission.
A landmark study in Canada hasfound that a cough medicine used
safely for decades in Europecould potentially slow
Parkinson's-related dementia.
In the year-long clinical trialinvolving 55 participants with
(05:25):
Parkinson's disease dementia,ambroxol was found to be safe,
well-tolerated and reachtherapeutic levels in the brain.
Moreover, in the treatmentgroup, psychiatric symptoms
remained stable, as did a bloodmarker of brain cell damage, and
participants with high-riskGBA1 gene variants showed
improved cognitive performance.
(05:45):
Researchers plan to start afollow-up clinical trial focused
specifically on cognition laterthis year.
And finally, investigators haveidentified major ethical,
administrative, regulatory andlogistical barriers, or
so-called URL hurdles, formultinational clinical trials in
19 European countries, based onan analysis of data from an
(06:07):
international platform trialtesting multiple drugs for the
treatment of pneumonia between2016 and 2023.
The analysis was focused onthree key metrics time to
complete site contracts, time toregulatory and ethical approval
and time to first patientenrollment and pronounced
differences were found instudies conducted in the UK
(06:28):
versus non-UK countries.
Notably, the UK achieveddramatic gains on all three
timeline-related measures duringthe pandemic, far outpacing
that of the other geographieswhich researchers credit to the
country's established researchnetworks and emergency trial
frameworks.
As a reminder, links to all thearticles, studies and press
(06:51):
releases covered in this month'snews segment are only a click
away in the show notes.
It is now time to bring inAnnette Bakker, head of the
Children's Tumor Foundation, totalk about the evolving role of
nonprofits in the ever-time andcost-challenged R&D ecosystem.
Welcome to the show, annette.
Thank you so much, deb.
This is so interesting.
Thank you, you are so welcome.
(07:12):
When we last spoke a few monthsago, one of the things that
stuck with me was yourmotivation for pivoting from
industry to the nonprofit sector, where there is a shared, as
you said, urgency with patientsto get to answers rather than to
get published or keep thingssecret.
I was hoping you could start usoff by sharing that tale with
(07:34):
our listeners, because I thinkit gets to the heart of the work
the Children's Tumor Foundationis doing, in this case for
patients with neurofibromatosisor NF.
Can you oblige?
Annette Bakker (07:45):
Sure, absolutely Very happy to do so.
So, just historically, I wasabout for about a good 10 years
in academia, followed by a good15 years in industry, and what I
noted is that the industrysector has excellent drugs and
the academia has excellentmodels, and the models are
(08:07):
anything from cell models toanimal models to anything.
You need to bring the goodtreatments to the patients, and
so if these two come togetherthe drugs and the models then
you can start thinking aboutbetter treatments for patients.
Unfortunately, that is not thecase.
Very often the models are let'snow say for the lack of a
(08:29):
better word stuck in academia,because in the academic
enterprise, people are driven bycuriosity and it's a lot about
publications and it's a lotabout making sure that you keep
the funding for your lab.
That is all dependent on whatyou discover.
On the other hand, the industrypeople they are very laser
(08:51):
focused on drug discovery.
However they are there, let'ssay, modus operandi is that they
patent their drugs and need tokeep their data and their
information secret as long aspossible.
And on top of that, in theindustry you as an individual
(09:12):
don't per se exist, it's more ateam sport.
So when I was working in theearly 2000s in Italy in a
biotech in Italy in theglioblastoma space, I discovered
that in fact, the models thatwere used to do drug selection
and that were accessible to usas pharma people were not good
models, whereas the good models,the good animal and cell models
, were actually in academia andthere was this constant blaming
(09:34):
back and forth between academiaand industry where, in fact, at
the end of the day, the patientwas the one who was really
suffering, because of course, ifwe screen on the wrong, however
accessible models, we areobviously going to select the
wrong drugs and we will not getthe right drugs to the patient.
(09:56):
So, being a little bitfrustrated about that ecosystem
and about the fact that thesetwo kind of stayed in their
comfort zone, the opportunitycame up for me to join the
Children's Tumor Foundation asthe then chief scientific
officer and what I very earlylearned is that, as a
non-for-profit coming frompharma, you know the disease
(10:19):
pretty well, you know theecosystem pretty well and you
can really add a different valuethan patient support.
Nothing wrong with patientsupport, but I think you can
really become a partner in theR&D ecosystem as, let's say, a
nexus between, on the one hand,the patients and, on the other
hand, all the stakeholders,right from academia to industry
(10:41):
to regulators, so you reallydon't have any conflict and can
push everybody a little bit outof their comfort zone to make
sure that we're selecting theright drugs to go to the clinic.
Deborah Borfitz (10:53):
Got it and I'd love to revisit specifically
that what you would call theshelved asset problem, whereby a
lot of great drugs areeffectively being trashed
because they don't meet acompany's commercial or
strategic business goals, orbecause they buy a basket of
assets from biotech and focus ononly one of them and sort of
(11:14):
ditch the rest.
Please share your views on howa nonprofit like the Children's
Tumor Foundation can step intothat gap to become a true R&D
partner with a pharma company.
Annette Bakker (11:25):
Yeah, thanks for that question.
I think the shelved assets is avery interesting problem is
that pharmaceutical companies alot of pharmaceutical companies
are working on very similarmechanisms and very similar
molecules and so when there isthe observation that maybe
another pharmaceutical companyis ahead or is closer to
(11:45):
approval, the big pharmaceuticalcompanies and the small ones
actually shelve that asset foranything from commercial to
strategic reasons.
So for me, a shelved asset isnot an asset that is shelved
because it's toxic, right.
Those are the assets thatshould never come anywhere close
to the clinic.
For me, the definition of ashelved asset is an asset that
(12:07):
still has IP, that still can begreat for any other disease than
for which it was beingdeveloped, that still has a
commercial value, so that thereis a real justification to get
these assets back into, let'ssay, active development.
The challenge is that for apharmaceutical company,
(12:30):
especially focusing on the rarediseases, that is a really hard
place for pharmaceuticalcompanies.
Why?
Because, first of all, there isthousands of rare diseases.
The second thing is that thecompanies for them, this
ecosystem of that specific raredisease, is really unknown.
There is very few researchers,there are very few models and
(12:53):
once an asset is shelved, theproject team within the company
is, let's say, reassigned toother projects, leaves the
company.
I mean there is a wholereshuffle of these experts that
were working on these shelvedassets that in fact there is not
really anyone left to careabout these assets.
And that is where I could seean organization like the
(13:16):
Children's Tumor Foundation comein, or another nonprofit,
obviously, to really act as anR&D partner for these companies
in a couple of ways.
One, we, for example,specifically at CTF, we do what
we call target and drug scouting, which means that we looked at
what has been published, Then welook at for what has been
(13:38):
published as new molecularmechanisms.
Are there any drugs that existthat could potentially benefit
our patients?
We know the disease really well, but we also really know well
the community, and so we havebuilt what I would call an NF
therapeutic accelerator that iscomposed of two elements.
One, there is a preclinical huband we have a dedicated staff
(14:01):
member who runs the preclinicalhub that if a drug gets
identified, she exactly knows inwhich models they should be
tested, which models we can haveaccess to, because a lot of the
agreements have already been,let's say, mapped out with the
academic institutions.
So that is a way to reallyaccelerate the preclinical,
(14:23):
let's say, de-risking of thatmolecule and then couple that to
also what you just mentionedthe platform trials in ALS.
We have also a multipleplatform trials now for NF.
So the idea is can we make surethat we kill this drug fast?
Right that if a drug doesn'tmake sense, it's not a good drug
(14:44):
for our patients.
Let's make sure that we getthat drug as quickly as possible
out of the door, because thereis one thing that we have in
common with the pharmaceuticalcompanies is that we want to be
fast, just like what companieswant, and we want treatments for
our patients, and so those twowishes align really well.
(15:04):
And I know that the academicinstitutions are also
interesting in making sure thattheir science actually
translates into bettertreatments.
So it's a way of bringing theworld together and stop having
people locked up in their littleboxes, but really bring that
human factor as well into themix, with the patients at the
(15:27):
table and no longer on the table.
Deborah Borfitz (15:30):
And on a very related note, I'd like to turn
to the idea of creating acentralized drug vault where
companies might be incentivizedto deposit their shelved assets
rather than letting all theaccumulated data quietly gather
dust.
I know this wasn't your idea,but could you perhaps explain
the premise and why you think itmight work, wasn't?
Annette Bakker (15:52):
your idea, but could you perhaps explain the
premise and why you think itmight work?
Yeah, so, first of all, my veryfirst experiment was there was
actually a drug that was atPfizer in the, let's say, 2015,
2016,.
That was a drug that wouldreally be able to help our NF
patients, a drug that wouldreally be able to help our NF
(16:13):
patients.
That drug was shelved by Pfizerand we wanted to prove that it
was possible to get a drug thatwas actually shelved, to get
that back into commercial.
So we work literally directlywith, at that time, the head of
Pfizer Cures, lara Sullivan, andFrida Lewis-Hall, who was the
chief medical officer of Pfizerat that time, to make sure that
(16:34):
we assemble a team of peoplethat would make sure that they
get the data they were lookingat, getting the necessary
investors and making sure thatthat drug was not forgotten.
And the reason why weidentified that drug is that we
already had generated, withinthe academic institutions, good
clinical data for that molecule,so that molecule was really
(16:57):
well, let's say, de-risked forNF and so, as of, let's say, as
a case study, we were able tohelp these people to make sure
that that drug got out of Pfizerinto a new company, springworks
, and then the Springworks teamdid an excellent job getting
(17:17):
that drug all the way toapproval on February 11, 2025.
Now this company, springworks,was just acquired by Merck, the
German Merck, for $3.5 billion.
Merck the.
German Merck for $3.5 billion.
So the reason why I'm tellingyou this story is that this is
the story that, yes, you can getthe drug out of a
(17:39):
pharmaceutical company, yes, youcan create a new company around
it and yes, you can get itapproved and, yes, you can make
money.
The challenge, and what makesthis so difficult, is that this
the way we did this, let's saycase study is not scalable.
To give you an idea, frida andLaura managed to get 200
(18:02):
volunteers at Pfizer, who workedday and night on their weekends
to make sure that the necessarydata on these shelved assets
were collected, organized andusable to spin out a new company
.
So you're dependent on 200volunteers, which, of course, is
a very unique thing, but is nota very scalable thing.
(18:22):
So that is where we then cameup with the idea and where, in
fact, now we're working togetherwith Professor Andrew Lowe, dr
Frida-Louise Hall, dr TanishaCarino and myself to figure out
can we create a vault so thatthe shelved assets can be
together in one organized waythat we don't have to redo the
(18:48):
200 volunteer experience everytime we have a shelved asset?
Do the 200 volunteer experienceevery time we have a shelved
asset?
The other challenge that we'retrying to solve here is that it
is very difficult to know whichassets were shelved in a
pharmaceutical company or not.
So it is also.
I think the number of shelvedassets that people are talking
about are highly underestimated.
(19:09):
I think they're way more so theideas.
We're working now on thisproject to create the vault.
Dr Lowe is looking at buildinga business model to also get a
return on investment.
Tanisha is focusing on thepolicy side of the world.
Can we do something?
Frida is the one who did it, soshe is really our brain trust
(19:33):
to make sure that we are gettingthese assets to something that
is valuable and then I see whereCTF and other foundations can
really participate is to act nowas the riskers and build these
R&D accelerators to make surethat companies don't, between
(19:53):
brackets, waste time on agreeingand making sure that we get the
necessary paperwork in place toeven start doing the drug
testing right.
Deborah Borfitz (20:02):
Yeah yeah, yeah , definitely keep me posted on
progress.
That is super exciting and veryinnovative.
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Deborah Borfitz (20:26):
Meanwhile, the Children's Tumor Foundation has,
in fact, some active R&Daccelerators.
You just referenced one amoment ago.
Can you talk a little bit moreabout that?
And I think there's a greatdeal of emphasis on
patient-centric platform trialsin particular.
Yeah, yeah, the value of allthat.
Annette Bakker (20:44):
Well, the value of the preclinical hub is really
to make sure that we get a drugvery quickly from we don't know
what it's doing to it's workingor it's not working, because,
especially for the biotics, theyare on very short runways,
specifically in the currentinvestment environment that we
(21:08):
are in, the faster they can know.
Whether a drug has I always sayhas legs, yes or no, right,
could make it through the clinicor not is something that you
need to know faster, soonerrather than later.
The problem with a lot of theseis the agreements, in getting
the necessary legal agreementsand lawyers in the room to agree
(21:30):
on conditions and on everythingthat actually delays the onset
of the trial scares, especiallythe small biotech.
So they will just not do itbecause it takes too long.
So we have that is, in fact,the reason why we've built this
preclinical hub.
That is, in fact, a network ofboth public and private partners
(21:51):
.
Both academic labs and CROs aremembers of that preclinical hub
.
The hub is constantly expanding, but we have that one call it
expert in preclinicaldevelopment who really acts as a
tour guide right to thepharmaceutical companies.
There is this drug and they andthat person will be able to say
(22:11):
go do this, don't do this, etc.
Etc.
So that is, in fact, the reasonof the preclinical hub.
On the clinical side, we havetwo platform trials up and
running one for NF2-relatedschwannomatosis and one for pain
in schwannomatosis.
And then there is one thatwe're now building in Europe,
(22:32):
which we hope to get off theground within this year, which
was funded by the Europeancommunity, called EU Pearl, and
where we really would like tobuild a same platform trial
platform basket trial actuallywhere you can look at multiple
indications with multipledifferent drugs in a master
(22:53):
protocol that is up there formany years.
Okay, the patient-centric.
I just want to give you onething on the patient-centricity.
Yes, I think it is sointeresting that we get asked
from the morning to the eveningwhether we prefer peanut butter
or strawberry jam, but when itcomes to healthcare, nobody asks
(23:16):
.
You get told what is going tohappen to you, and no, and the
patients don't ask the questionsand the clinicians are not
always used to communicate withthe patients about their trials,
and so what we have done isreally figure out a way to make
clinical trials morepatient-centric and not just
(23:37):
have a patient in the room as atoken, but really have a patient
or a parent in the room whoactually participates in the
design.
And I'm going to give you avery simple example.
If, for example, your child hasa glioma, a three-year-old child
has a glioma and the clinicaltrial requires to do a MRI.
(24:00):
Just giving you this examplenow, right, mri every three
months.
That means that your child ofthree-year-olds will have to be
in anesthesia every three months.
Parents may say, no, I'm notdoing that, that's too much for
my three-year-old.
What if you get a conversationand you agree with the
clinicians that you would do anevery six-month MRI and then the
(24:24):
parents are like, okay, twice ayear, every six months, I can
live with that.
Now you have a patient-centrictrial and now you get the trial
that opens and your patientrecruitment will not be a
problem.
And I think this is soimportant to get these kinds of
conversations going, because 30%of the clinical trials in rare
(24:46):
disease fail because thecompanies or the sponsors cannot
recruit their patients in time.
Deborah Borfitz (24:54):
Yeah, very, very good points.
And besides thesepatient-centric platform trials,
I know the NF Clinical TrialsConsortium is another way NF
trials are getting efficientlyconducted and, as we speak, I
also know that, being acongressionally directed medical
research program, it ispotentially subject to funding
cuts.
But, that aside, tell us aboutwhy this consortium matters and
(25:19):
deserves financial support.
Annette Bakker (25:21):
I think the most important thing to understand
in rare disease is that not onesingle center sees enough
patients to run a clinical trialby itself.
None of them.
If you would do a breast cancertrial, you could do that in one
center, but an NF trial cannever be done in one center for
the simple reason that there isnot enough patients in any of
(25:44):
the centers.
So you need multiple centers.
And the way to make sure thatthese clinical trials are run in
an efficient way and in aneffective way you need to set up
a consortium of clinical trialcenters that actually do
collaborate, that they allmeasure the endpoints in the
(26:04):
exact same way that walkingthree minutes or anything that
needs to be done at the clinicaltrial measuring hearing,
measuring movement, measuringanything that that is done and
agreed upon by the clinicians tobe done in the same way.
So that led, in fact, to thebirth of this NF Clinical Trials
(26:25):
Consortium over 10 years agonow, where today there's about
20 clinical trial centers thatare working together if it's not
more than 20, that are actuallydiscussing clinical trials,
agreeing on the clinical trialdesign, sharing the data in one
place, having the operations inone place.
(26:46):
So this is just a superefficient way to run clinical
trials that otherwise you wouldhave to go and you would have to
assemble all these differenttrial centers to be able to run
to agree every time you run anew trial.
So it is a fantasticinfrastructure that has been
(27:07):
built with money from thecongressionally directed medical
research programs, from thespecific NFCDMRP program and,
yes, it got cut.
We are heavily advocating tomake sure that we can, that this
program would get back up,because, if this government is
about efficiency, this isefficient.
(27:28):
This is very efficient.
I can tell you that a typicalclinical trial runs anywhere
from $5 to $10 to $15 millionper trial.
This clinical trial consortiumran, I think, 29 clinical trials
with about $15 million.
So now here you go.
You're about speaking aboutmaximum $1 million per trial to
(27:51):
do these trials.
And why is it so cheap?
Because, honestly, theinstitutions have put in a lot
of volunteer time.
These clinicians are amazing.
They put in a lot of volunteertime institutions co-fund.
So it's really an ecosystemthat is super efficient in
(28:11):
making sure that we run clinicaltrials together and it's a
prototype for rare disease, ifyou ask me.
So there is the platform trialson the one hand, the clinical
trials consortium.
On the other hand, I think withthose two we will be able to
run the clinical trials and Iwill keep advocating to make
sure that this program doesn'tget cut.
(28:34):
You have a strong case there,annette, I think so I think so,
yeah, it's just about efficiency, right, and it's about building
prototype and prototypes incase studies for rare disease,
and the reason why this clinicaltrials consortium is so
important is because there arecertain studies that are not of
(28:55):
interest to the pharmaceuticalcompanies A drug that was
already approved for many years,a drug that is off patent,
certain things that don't have,per se, a commercial value, but
have a huge patient value, andso this is where I think the
government should be investingtheir money in things that
matter to patients but don'talways lead to return on
(29:18):
investments.
Deborah Borfitz (29:19):
Yeah, yeah, yeah Makes total sense to me.
I love your thinking and, Ihave to say, I also love another
idea that you previously sharedwith me about how to better
inject capital into early stagebiotechs a biotech focused on
rare disease by building I thinkyou called it a financial
continuum to reward theirprogress and that of their
(29:39):
nonprofit partners, step by step.
I think I'd like to end today'schat with that hopeful vision.
Does that sound good to you?
Sounds perfect.
Annette Bakker (29:50):
So I say this also with some of my friends who
are in the nonprofit friendsCEOs, michael Hunt, mark
Reutemeyer, from the Alzheimer'sDrug Discovery Foundation we
are in fact together thinkingabout ways of being part of also
a financial continuum, and letme explain what that means.
(30:10):
So we, for example, as afoundation, we are not wealthy
wealthy, but we can put enoughmoney in there to get an idea
from a non-investable brilliantidea into an investable
brilliant idea, and we have donethat for many, many years.
When it comes to the academicresearchers, small grants to be
(30:36):
able to create the preliminarydata set that they need in order
to then get access to thebigger funds from the NIH, cdmrp
, any of the bigger funding.
And I was thinking in thebiotech space that we're, in
fact, exactly at the same pointwhere some biotechs have
brilliant ideas but they don'thave the conviction, they don't
(31:00):
have the, let's say, datapackage that they need to
convince relatively risk-averseinvestors these days to put in
money and to help make that ideahappen.
So what if the R&D foundationsreally come in early and a lot
of us are doing this right,giving money to this
(31:22):
non-investable brilliant ideasfrom the biotechs and get them
to a point where we then createa financial continuum with the
investment community when we cansay okay, mr and Mrs Investor,
you have more money than me, butI can de-risk it.
And can we create so that we canpass these great de-risk ideas
(31:47):
on to you who have more money,and then you pass it on to one
who has even more money?
So really creating thatfinancial continuum, just like
we create a research continuumright, it's like building
pipelines that's, in fact, mybig dream.
But we would need theinvestment community to come
(32:07):
together and to partner with usand say we are with you, we are
willing to inject some capitalto help you de-risk, because my
real dream is to becomeautosufficient, that in fact we
make enough money that comesback to the foundation, that in
(32:27):
fact this becomes a kind offlywheel of research pipeline
and financial pipeline and thatwe can bring these treatments to
the patients that so deservethe treatments that we're
developing.
Deborah Borfitz (32:39):
Well, I absolutely love your vision of
this new kind of ecosystem,annette, and it has been an
absolute delight having you as aguest on today's show.
Thanks for taking the time toshare your passion for finding
answers for patients with NF, aswell as pointing to a way
forward where nonprofitsfunction more proactively and
sustainably in the clinicaltrials enterprise.
(33:02):
I hope we can speak again withnews of a cure or at least
better ways to manage the manysymptoms and complications of
this perplexing disease, andreally just everything that you
are doing for the larger worldof clinical research and
patients in general.
Annette Bakker (33:18):
Thank you, thank you, thank you so much.
I just wanted to close with aninvitation for my companies and
the investors to really look forthose foundations that are more
R&D and patient-driven butscience foundations and let's
work together.
We can do it.
We are playing a very uniquerole in the R&D ecosystem from,
(33:42):
on the one hand, being with thepatients and caring for the
patients and bringing in thathuman factor, that it's about
people, it's not about ascientific problem, and then, on
the other hand, kind of reallyconnecting that to the
investment in the pharma sector.
Let's do it.
Deborah Borfitz (34:01):
Let's do it, okay.
Thank you again, Annette, and,as always, a big thank you to
everyone out there for listeningin.
If you're not subscribed tothis podcast yet, please
consider going to Apple Podcastsand doing so right now, so you
don't miss your monthly dose ofnews and perspectives.
You'll be hard-pressed to findanywhere else and if you're up
(34:21):
for it, I'd also be so verygrateful if you'd leave a rating
and review while you're there.
One more thing before we go, ifyou'd like today's conversation
.
It is only a glimpse of whatyou can expect from SCOPE Europe
.
Presenters and panelists,please plan to join us October
14th and 15th in Barcelona whenclinical operations executives
(34:41):
will be exploring the latesttrends in clinical trial
innovation, planning andoperations.
Save an additional 10% off anycurrent rate by using the code
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For more information, visitscopesummiteuropecom.
Bye for now.
Hello and welcome to the Scope of Things
podcast, a no-nonsense look atthe promise and problems of
clinical research, based on asweep of the latest news and
emerging trends in the field andwhat I think is worthy of your
30 or so minutes of time.
I'm Deborah Borfitz, SeniorScience Writer for Clinical
Research News, which means Ispend a lot of time with my ear
(00:22):
to the ground on your behalf anda lot of hours every week
speaking to top experts fromaround the world.
Please consider making thisyour trusted go-to channel for
staying current on things thatmatter, whether they give us
hope or cause for pause.
In another five minutes or so,I'll be speaking with Annette
Bakker, CEO of the Children'sTumor Foundation, about what
(00:43):
nonprofits uniquely bring toclinical research and new
financial models for sustainingtheir contribution.
But first the latest news,including a new approach to
funding clinical trials for ALS,an algorithm using spatial
biology biomarkers to matchpatients to trials, development
of a biomap of lung cancer tumorchanges, improving diabetes
(01:04):
treatment outcomes for patientsin China, the possibility of
slowing Parkinson's-relateddementia with cough medicine,
and how the UK is uniquelyovercoming some of the
timeline-related Earl hurdles inEurope.
A new financing model has beenproposed for developing drugs
for amyotrophic lateralsclerosis, or ALS, to bridge the
(01:27):
notorious valley of deathwhereby promising scientific
discoveries often fail totranslate into viable therapies.
The so-called Fund of AdaptiveRoyalties approach merges the
efficiencies of adaptiveplatform trials, which allow
multiple drug candidates to betested simultaneously under a
single master protocol, with afund that rewards investors with
(01:50):
future royalties fromsuccessful drugs that emerge
from the trials.
A simulated fund of this typegenerated an estimated return of
28%, with a 22% probability oftotal loss, making it
potentially attractive to hedgefunds, sovereign wealth funds,
family offices andphilanthropists.
Returns more palatable tomainstream investors might be
(02:11):
generated by funding multipleplatform trials simultaneously
and employing financial toolsthat bundle future income from
assets like loans or royaltiesinto investment products.
Researchers at VirginiaCommonwealth University have
been working with principalinvestigators across campus to
include spatial biologybiomarkers in their cancer
(02:31):
clinical trials, to predictpatient responses prior to
enrollment and, when individualscan't be part of a trial, point
them toward already approveddrugs that could help.
The guidance is provided byTACIT, short for Threshold-Based
Assignment of Cell Types fromMultiplexed Imaging Data, an
algorithm that assigns cellidentities based on cell marker
(02:52):
expression profiles and, thanksto artificial intelligence, does
the job in minutes rather thanweeks.
Tacit distinguishes cells usingdata from over 5 million cells
across major body systems likethe brain, gut and oral glands.
In a new study, it was found tooutperform existing
unsupervised methods in accuracyand scalability, while
(03:12):
revealing new cellularassociations, and the results
strongly agreed with genetic andprotein datasets.
I'm writing a piece on this forDiagnostics World News, so be
on the lookout for that to learnmore.
City of Hope was recentlyawarded close to $24 million
from the federal government tocreate a biomap of tumor changes
that cause immunotherapyresistance in advanced or
(03:34):
metastatic non-small cell lungcancer.
As part of a national precisioncancer therapy program, it will
be conducting a six-year535-patient clinical trial
designed to adjust treatment asresistance arises, with the goal
of increasing progression-freesurvival by 50% in at least one
patient group.
Investigators will monitortumor trajectory and patients'
(03:57):
response to treatment usingtechniques like liquid biopsies,
single-cell sequencing andradioimaging.
They are also funding thedevelopment, testing and
matching of new biomarkers withtherapeutic options currently
available or in a clinical trial.
Trial enrollment begins within12 months.
A large-scale pragmaticclinical trial has launched in
(04:18):
hospitals across China, designedto improve treatment outcomes
for patients diagnosed withdiabetes by using a
Chinese-specific version of thegenetic risk score to
distinguish between people witha type 1 and type 2 disease
variety, since patients in theregion often don't fit the
classic Asian weight profilewith which the diseases are
diagnosed, and about 25% ofindividuals with newly diagnosed
(04:41):
type 1 diabetes lack detectableislet autoantibodies measured
by early detection tests.
Misdiagnosis of the type 1adult onset variety, whereby the
body's own immune systemattacks insulin-producing beta
cells in the pancreas, meansdelays in getting daily insulin
injections to avoidcomplications.
Type 2 diabetes, which could betriggered by high body mass
(05:04):
index, has several treatmentsand nearly half of patients with
a short disease duration maysucceed in achieving disease
remission.
A landmark study in Canada hasfound that a cough medicine used
safely for decades in Europecould potentially slow
Parkinson's-related dementia.
In the year-long clinical trialinvolving 55 participants with
(05:25):
Parkinson's disease dementia,ambroxol was found to be safe,
well-tolerated and reachtherapeutic levels in the brain.
Moreover, in the treatmentgroup, psychiatric symptoms
remained stable, as did a bloodmarker of brain cell damage, and
participants with high-riskGBA1 gene variants showed
improved cognitive performance.
(05:45):
Researchers plan to start afollow-up clinical trial focused
specifically on cognition laterthis year.
And finally, investigators haveidentified major ethical,
administrative, regulatory andlogistical barriers, or
so-called URL hurdles, formultinational clinical trials in
19 European countries, based onan analysis of data from an
(06:07):
international platform trialtesting multiple drugs for the
treatment of pneumonia between2016 and 2023.
The analysis was focused onthree key metrics time to
complete site contracts, time toregulatory and ethical approval
and time to first patientenrollment and pronounced
differences were found instudies conducted in the UK
(06:28):
versus non-UK countries.
Notably, the UK achieveddramatic gains on all three
timeline-related measures duringthe pandemic, far outpacing
that of the other geographieswhich researchers credit to the
country's established researchnetworks and emergency trial
frameworks.
As a reminder, links to all thearticles, studies and press
(06:51):
releases covered in this month'snews segment are only a click
away in the show notes.
It is now time to bring inAnnette Bakker, head of the
Children's Tumor Foundation, totalk about the evolving role of
nonprofits in the ever-time andcost-challenged R&D ecosystem.
Welcome to the show, annette.
Thank you so much, deb.
This is so interesting.
Thank you, you are so welcome.
(07:12):
When we last spoke a few monthsago, one of the things that
stuck with me was yourmotivation for pivoting from
industry to the nonprofit sector, where there is a shared, as
you said, urgency with patientsto get to answers rather than to
get published or keep thingssecret.
I was hoping you could start usoff by sharing that tale with
(07:34):
our listeners, because I thinkit gets to the heart of the work
the Children's Tumor Foundationis doing, in this case for
patients with neurofibromatosisor NF.
Can you oblige?
Annette Bakker (07:45):
Sure, absolutely Very happy to do so.
So, just historically, I wasabout for about a good 10 years
in academia, followed by a good15 years in industry, and what I
noted is that the industrysector has excellent drugs and
the academia has excellentmodels, and the models are
(08:07):
anything from cell models toanimal models to anything.
You need to bring the goodtreatments to the patients, and
so if these two come togetherthe drugs and the models then
you can start thinking aboutbetter treatments for patients.
Unfortunately, that is not thecase.
Very often the models are let'snow say for the lack of a
(08:29):
better word stuck in academia,because in the academic
enterprise, people are driven bycuriosity and it's a lot about
publications and it's a lotabout making sure that you keep
the funding for your lab.
That is all dependent on whatyou discover.
On the other hand, the industrypeople they are very laser
(08:51):
focused on drug discovery.
However they are there, let'ssay, modus operandi is that they
patent their drugs and need tokeep their data and their
information secret as long aspossible.
And on top of that, in theindustry you as an individual
(09:12):
don't per se exist, it's more ateam sport.
So when I was working in theearly 2000s in Italy in a
biotech in Italy in theglioblastoma space, I discovered
that in fact, the models thatwere used to do drug selection
and that were accessible to usas pharma people were not good
models, whereas the good models,the good animal and cell models
, were actually in academia andthere was this constant blaming
(09:34):
back and forth between academiaand industry where, in fact, at
the end of the day, the patientwas the one who was really
suffering, because of course, ifwe screen on the wrong, however
accessible models, we areobviously going to select the
wrong drugs and we will not getthe right drugs to the patient.
(09:56):
So, being a little bitfrustrated about that ecosystem
and about the fact that thesetwo kind of stayed in their
comfort zone, the opportunitycame up for me to join the
Children's Tumor Foundation asthe then chief scientific
officer and what I very earlylearned is that, as a
non-for-profit coming frompharma, you know the disease
(10:19):
pretty well, you know theecosystem pretty well and you
can really add a different valuethan patient support.
Nothing wrong with patientsupport, but I think you can
really become a partner in theR&D ecosystem as, let's say, a
nexus between, on the one hand,the patients and, on the other
hand, all the stakeholders,right from academia to industry
(10:41):
to regulators, so you reallydon't have any conflict and can
push everybody a little bit outof their comfort zone to make
sure that we're selecting theright drugs to go to the clinic.
Deborah Borfitz (10:53):
Got it and I'd love to revisit specifically
that what you would call theshelved asset problem, whereby a
lot of great drugs areeffectively being trashed
because they don't meet acompany's commercial or
strategic business goals, orbecause they buy a basket of
assets from biotech and focus ononly one of them and sort of
(11:14):
ditch the rest.
Please share your views on howa nonprofit like the Children's
Tumor Foundation can step intothat gap to become a true R&D
partner with a pharma company.
Annette Bakker (11:25):
Yeah, thanks for that question.
I think the shelved assets is avery interesting problem is
that pharmaceutical companies alot of pharmaceutical companies
are working on very similarmechanisms and very similar
molecules and so when there isthe observation that maybe
another pharmaceutical companyis ahead or is closer to
(11:45):
approval, the big pharmaceuticalcompanies and the small ones
actually shelve that asset foranything from commercial to
strategic reasons.
So for me, a shelved asset isnot an asset that is shelved
because it's toxic, right.
Those are the assets thatshould never come anywhere close
to the clinic.
For me, the definition of ashelved asset is an asset that
(12:07):
still has IP, that still can begreat for any other disease than
for which it was beingdeveloped, that still has a
commercial value, so that thereis a real justification to get
these assets back into, let'ssay, active development.
The challenge is that for apharmaceutical company,
(12:30):
especially focusing on the rarediseases, that is a really hard
place for pharmaceuticalcompanies.
Why?
Because, first of all, there isthousands of rare diseases.
The second thing is that thecompanies for them, this
ecosystem of that specific raredisease, is really unknown.
There is very few researchers,there are very few models and
(12:53):
once an asset is shelved, theproject team within the company
is, let's say, reassigned toother projects, leaves the
company.
I mean there is a wholereshuffle of these experts that
were working on these shelvedassets that in fact there is not
really anyone left to careabout these assets.
And that is where I could seean organization like the
(13:16):
Children's Tumor Foundation comein, or another nonprofit,
obviously, to really act as anR&D partner for these companies
in a couple of ways.
One, we, for example,specifically at CTF, we do what
we call target and drug scouting, which means that we looked at
what has been published, Then welook at for what has been
(13:38):
published as new molecularmechanisms.
Are there any drugs that existthat could potentially benefit
our patients?
We know the disease really well, but we also really know well
the community, and so we havebuilt what I would call an NF
therapeutic accelerator that iscomposed of two elements.
One, there is a preclinical huband we have a dedicated staff
(14:01):
member who runs the preclinicalhub that if a drug gets
identified, she exactly knows inwhich models they should be
tested, which models we can haveaccess to, because a lot of the
agreements have already been,let's say, mapped out with the
academic institutions.
So that is a way to reallyaccelerate the preclinical,
(14:23):
let's say, de-risking of thatmolecule and then couple that to
also what you just mentionedthe platform trials in ALS.
We have also a multipleplatform trials now for NF.
So the idea is can we make surethat we kill this drug fast?
Right that if a drug doesn'tmake sense, it's not a good drug
(14:44):
for our patients.
Let's make sure that we getthat drug as quickly as possible
out of the door, because thereis one thing that we have in
common with the pharmaceuticalcompanies is that we want to be
fast, just like what companieswant, and we want treatments for
our patients, and so those twowishes align really well.
(15:04):
And I know that the academicinstitutions are also
interesting in making sure thattheir science actually
translates into bettertreatments.
So it's a way of bringing theworld together and stop having
people locked up in their littleboxes, but really bring that
human factor as well into themix, with the patients at the
(15:27):
table and no longer on the table.
Deborah Borfitz (15:30):
And on a very related note, I'd like to turn
to the idea of creating acentralized drug vault where
companies might be incentivizedto deposit their shelved assets
rather than letting all theaccumulated data quietly gather
dust.
I know this wasn't your idea,but could you perhaps explain
the premise and why you think itmight work, wasn't?
Annette Bakker (15:52):
your idea, but could you perhaps explain the
premise and why you think itmight work?
Yeah, so, first of all, my veryfirst experiment was there was
actually a drug that was atPfizer in the, let's say, 2015,
2016,.
That was a drug that wouldreally be able to help our NF
patients, a drug that wouldreally be able to help our NF
(16:13):
patients.
That drug was shelved by Pfizerand we wanted to prove that it
was possible to get a drug thatwas actually shelved, to get
that back into commercial.
So we work literally directlywith, at that time, the head of
Pfizer Cures, lara Sullivan, andFrida Lewis-Hall, who was the
chief medical officer of Pfizerat that time, to make sure that
(16:34):
we assemble a team of peoplethat would make sure that they
get the data they were lookingat, getting the necessary
investors and making sure thatthat drug was not forgotten.
And the reason why weidentified that drug is that we
already had generated, withinthe academic institutions, good
clinical data for that molecule,so that molecule was really
(16:57):
well, let's say, de-risked forNF and so, as of, let's say, as
a case study, we were able tohelp these people to make sure
that that drug got out of Pfizerinto a new company, springworks
, and then the Springworks teamdid an excellent job getting
(17:17):
that drug all the way toapproval on February 11, 2025.
Now this company, springworks,was just acquired by Merck, the
German Merck, for $3.5 billion.
Merck the.
German Merck for $3.5 billion.
So the reason why I'm tellingyou this story is that this is
the story that, yes, you can getthe drug out of a
(17:39):
pharmaceutical company, yes, youcan create a new company around
it and yes, you can get itapproved and, yes, you can make
money.
The challenge, and what makesthis so difficult, is that this
the way we did this, let's saycase study is not scalable.
To give you an idea, frida andLaura managed to get 200
(18:02):
volunteers at Pfizer, who workedday and night on their weekends
to make sure that the necessarydata on these shelved assets
were collected, organized andusable to spin out a new company
.
So you're dependent on 200volunteers, which, of course, is
a very unique thing, but is nota very scalable thing.
(18:22):
So that is where we then cameup with the idea and where, in
fact, now we're working togetherwith Professor Andrew Lowe, dr
Frida-Louise Hall, dr TanishaCarino and myself to figure out
can we create a vault so thatthe shelved assets can be
together in one organized waythat we don't have to redo the
(18:48):
200 volunteer experience everytime we have a shelved asset?
Do the 200 volunteer experienceevery time we have a shelved
asset?
The other challenge that we'retrying to solve here is that it
is very difficult to know whichassets were shelved in a
pharmaceutical company or not.
So it is also.
I think the number of shelvedassets that people are talking
about are highly underestimated.
(19:09):
I think they're way more so theideas.
We're working now on thisproject to create the vault.
Dr Lowe is looking at buildinga business model to also get a
return on investment.
Tanisha is focusing on thepolicy side of the world.
Can we do something?
Frida is the one who did it, soshe is really our brain trust
(19:33):
to make sure that we are gettingthese assets to something that
is valuable and then I see whereCTF and other foundations can
really participate is to act nowas the riskers and build these
R&D accelerators to make surethat companies don't, between
(19:53):
brackets, waste time on agreeingand making sure that we get the
necessary paperwork in place toeven start doing the drug
testing right.
Deborah Borfitz (20:02):
Yeah yeah, yeah , definitely keep me posted on
progress.
That is super exciting and veryinnovative.
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Deborah Borfitz (20:26):
Meanwhile, the Children's Tumor Foundation has,
in fact, some active R&Daccelerators.
You just referenced one amoment ago.
Can you talk a little bit moreabout that?
And I think there's a greatdeal of emphasis on
patient-centric platform trialsin particular.
Yeah, yeah, the value of allthat.
Annette Bakker (20:44):
Well, the value of the preclinical hub is really
to make sure that we get a drugvery quickly from we don't know
what it's doing to it's workingor it's not working, because,
especially for the biotics, theyare on very short runways,
specifically in the currentinvestment environment that we
(21:08):
are in, the faster they can know.
Whether a drug has I always sayhas legs, yes or no, right,
could make it through the clinicor not is something that you
need to know faster, soonerrather than later.
The problem with a lot of theseis the agreements, in getting
the necessary legal agreementsand lawyers in the room to agree
(21:30):
on conditions and on everythingthat actually delays the onset
of the trial scares, especiallythe small biotech.
So they will just not do itbecause it takes too long.
So we have that is, in fact,the reason why we've built this
preclinical hub.
That is, in fact, a network ofboth public and private partners
(21:51):
.
Both academic labs and CROs aremembers of that preclinical hub
.
The hub is constantly expanding, but we have that one call it
expert in preclinicaldevelopment who really acts as a
tour guide right to thepharmaceutical companies.
There is this drug and they andthat person will be able to say
(22:11):
go do this, don't do this, etc.
Etc.
So that is, in fact, the reasonof the preclinical hub.
On the clinical side, we havetwo platform trials up and
running one for NF2-relatedschwannomatosis and one for pain
in schwannomatosis.
And then there is one thatwe're now building in Europe,
(22:32):
which we hope to get off theground within this year, which
was funded by the Europeancommunity, called EU Pearl, and
where we really would like tobuild a same platform trial
platform basket trial actuallywhere you can look at multiple
indications with multipledifferent drugs in a master
(22:53):
protocol that is up there formany years.
Okay, the patient-centric.
I just want to give you onething on the patient-centricity.
Yes, I think it is sointeresting that we get asked
from the morning to the eveningwhether we prefer peanut butter
or strawberry jam, but when itcomes to healthcare, nobody asks
(23:16):
.
You get told what is going tohappen to you, and no, and the
patients don't ask the questionsand the clinicians are not
always used to communicate withthe patients about their trials,
and so what we have done isreally figure out a way to make
clinical trials morepatient-centric and not just
(23:37):
have a patient in the room as atoken, but really have a patient
or a parent in the room whoactually participates in the
design.
And I'm going to give you avery simple example.
If, for example, your child hasa glioma, a three-year-old child
has a glioma and the clinicaltrial requires to do a MRI.
(24:00):
Just giving you this examplenow, right, mri every three
months.
That means that your child ofthree-year-olds will have to be
in anesthesia every three months.
Parents may say, no, I'm notdoing that, that's too much for
my three-year-old.
What if you get a conversationand you agree with the
clinicians that you would do anevery six-month MRI and then the
(24:24):
parents are like, okay, twice ayear, every six months, I can
live with that.
Now you have a patient-centrictrial and now you get the trial
that opens and your patientrecruitment will not be a
problem.
And I think this is soimportant to get these kinds of
conversations going, because 30%of the clinical trials in rare
(24:46):
disease fail because thecompanies or the sponsors cannot
recruit their patients in time.
Deborah Borfitz (24:54):
Yeah, very, very good points.
And besides thesepatient-centric platform trials,
I know the NF Clinical TrialsConsortium is another way NF
trials are getting efficientlyconducted and, as we speak, I
also know that, being acongressionally directed medical
research program, it ispotentially subject to funding
cuts.
But, that aside, tell us aboutwhy this consortium matters and
(25:19):
deserves financial support.
Annette Bakker (25:21):
I think the most important thing to understand
in rare disease is that not onesingle center sees enough
patients to run a clinical trialby itself.
None of them.
If you would do a breast cancertrial, you could do that in one
center, but an NF trial cannever be done in one center for
the simple reason that there isnot enough patients in any of
(25:44):
the centers.
So you need multiple centers.
And the way to make sure thatthese clinical trials are run in
an efficient way and in aneffective way you need to set up
a consortium of clinical trialcenters that actually do
collaborate, that they allmeasure the endpoints in the
(26:04):
exact same way that walkingthree minutes or anything that
needs to be done at the clinicaltrial measuring hearing,
measuring movement, measuringanything that that is done and
agreed upon by the clinicians tobe done in the same way.
So that led, in fact, to thebirth of this NF Clinical Trials
(26:25):
Consortium over 10 years agonow, where today there's about
20 clinical trial centers thatare working together if it's not
more than 20, that are actuallydiscussing clinical trials,
agreeing on the clinical trialdesign, sharing the data in one
place, having the operations inone place.
(26:46):
So this is just a superefficient way to run clinical
trials that otherwise you wouldhave to go and you would have to
assemble all these differenttrial centers to be able to run
to agree every time you run anew trial.
So it is a fantasticinfrastructure that has been
(27:07):
built with money from thecongressionally directed medical
research programs, from thespecific NFCDMRP program and,
yes, it got cut.
We are heavily advocating tomake sure that we can, that this
program would get back up,because, if this government is
about efficiency, this isefficient.
(27:28):
This is very efficient.
I can tell you that a typicalclinical trial runs anywhere
from $5 to $10 to $15 millionper trial.
This clinical trial consortiumran, I think, 29 clinical trials
with about $15 million.
So now here you go.
You're about speaking aboutmaximum $1 million per trial to
(27:51):
do these trials.
And why is it so cheap?
Because, honestly, theinstitutions have put in a lot
of volunteer time.
These clinicians are amazing.
They put in a lot of volunteertime institutions co-fund.
So it's really an ecosystemthat is super efficient in
(28:11):
making sure that we run clinicaltrials together and it's a
prototype for rare disease, ifyou ask me.
So there is the platform trialson the one hand, the clinical
trials consortium.
On the other hand, I think withthose two we will be able to
run the clinical trials and Iwill keep advocating to make
sure that this program doesn'tget cut.
(28:34):
You have a strong case there,annette, I think so I think so,
yeah, it's just about efficiency, right, and it's about building
prototype and prototypes incase studies for rare disease,
and the reason why this clinicaltrials consortium is so
important is because there arecertain studies that are not of
(28:55):
interest to the pharmaceuticalcompanies A drug that was
already approved for many years,a drug that is off patent,
certain things that don't have,per se, a commercial value, but
have a huge patient value, andso this is where I think the
government should be investingtheir money in things that
matter to patients but don'talways lead to return on
(29:18):
investments.
Deborah Borfitz (29:19):
Yeah, yeah, yeah Makes total sense to me.
I love your thinking and, Ihave to say, I also love another
idea that you previously sharedwith me about how to better
inject capital into early stagebiotechs a biotech focused on
rare disease by building I thinkyou called it a financial
continuum to reward theirprogress and that of their
(29:39):
nonprofit partners, step by step.
I think I'd like to end today'schat with that hopeful vision.
Does that sound good to you?
Sounds perfect.
Annette Bakker (29:50):
So I say this also with some of my friends who
are in the nonprofit friendsCEOs, michael Hunt, mark
Reutemeyer, from the Alzheimer'sDrug Discovery Foundation we
are in fact together thinkingabout ways of being part of also
a financial continuum, and letme explain what that means.
(30:10):
So we, for example, as afoundation, we are not wealthy
wealthy, but we can put enoughmoney in there to get an idea
from a non-investable brilliantidea into an investable
brilliant idea, and we have donethat for many, many years.
When it comes to the academicresearchers, small grants to be
(30:36):
able to create the preliminarydata set that they need in order
to then get access to thebigger funds from the NIH, cdmrp
, any of the bigger funding.
And I was thinking in thebiotech space that we're, in
fact, exactly at the same pointwhere some biotechs have
brilliant ideas but they don'thave the conviction, they don't
(31:00):
have the, let's say, datapackage that they need to
convince relatively risk-averseinvestors these days to put in
money and to help make that ideahappen.
So what if the R&D foundationsreally come in early and a lot
of us are doing this right,giving money to this
(31:22):
non-investable brilliant ideasfrom the biotechs and get them
to a point where we then createa financial continuum with the
investment community when we cansay okay, mr and Mrs Investor,
you have more money than me, butI can de-risk it.
And can we create so that we canpass these great de-risk ideas
(31:47):
on to you who have more money,and then you pass it on to one
who has even more money?
So really creating thatfinancial continuum, just like
we create a research continuumright, it's like building
pipelines that's, in fact, mybig dream.
But we would need theinvestment community to come
(32:07):
together and to partner with usand say we are with you, we are
willing to inject some capitalto help you de-risk, because my
real dream is to becomeautosufficient, that in fact we
make enough money that comesback to the foundation, that in
(32:27):
fact this becomes a kind offlywheel of research pipeline
and financial pipeline and thatwe can bring these treatments to
the patients that so deservethe treatments that we're
developing.
Deborah Borfitz (32:39):
Well, I absolutely love your vision of
this new kind of ecosystem,annette, and it has been an
absolute delight having you as aguest on today's show.
Thanks for taking the time toshare your passion for finding
answers for patients with NF, aswell as pointing to a way
forward where nonprofitsfunction more proactively and
sustainably in the clinicaltrials enterprise.
(33:02):
I hope we can speak again withnews of a cure or at least
better ways to manage the manysymptoms and complications of
this perplexing disease, andreally just everything that you
are doing for the larger worldof clinical research and
patients in general.
Annette Bakker (33:18):
Thank you, thank you, thank you so much.
I just wanted to close with aninvitation for my companies and
the investors to really look forthose foundations that are more
R&D and patient-driven butscience foundations and let's
work together.
We can do it.
We are playing a very uniquerole in the R&D ecosystem from,
(33:42):
on the one hand, being with thepatients and caring for the
patients and bringing in thathuman factor, that it's about
people, it's not about ascientific problem, and then, on
the other hand, kind of reallyconnecting that to the
investment in the pharma sector.
Let's do it.
Deborah Borfitz (34:01):
Let's do it, okay.
Thank you again, Annette, and,as always, a big thank you to
everyone out there for listeningin.
If you're not subscribed tothis podcast yet, please
consider going to Apple Podcastsand doing so right now, so you
don't miss your monthly dose ofnews and perspectives.
You'll be hard-pressed to findanywhere else and if you're up
(34:21):
for it, I'd also be so verygrateful if you'd leave a rating
and review while you're there.
One more thing before we go, ifyou'd like today's conversation
.
It is only a glimpse of whatyou can expect from SCOPE Europe
.
Presenters and panelists,please plan to join us October
14th and 15th in Barcelona whenclinical operations executives
(34:41):
will be exploring the latesttrends in clinical trial
innovation, planning andoperations.
Save an additional 10% off anycurrent rate by using the code
SOT10.
For more information, visitscopesummiteuropecom.
Bye for now.
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