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- Hello. Thank you so much for tuning in
- to the Becker's Oncology Leadership Podcast.
- I'm Elizabeth Gregersen, a reporter here at Becker's.
- And today, I'm talking to Jody Skiles,
- doctor Jody Skiles,
- medical director of the pediatric stem cell transplant
- program at Riley Hospital for Children in Indianapolis
- and the FACT Accreditation medical director for both
- pediatric and adult stem cell transplant programs at
- Indiana University.
- Doctor Skiles, thank you so much for taking
- the time to speak with me today.
- Of course. Thank you for the opportunity to
- be here.
- Absolutely. And before we dive into our conversation,
- because it's a good one for our listeners,
- I would love if you could share just
- a bit about your background and your role
- at Riley Hospital for Children.
- Sure.
- So I have been on staff at Riley
- since 2012 when I graduated from fellowship. So
- my background training, I was internal medicine and
- pediatrics trained
- for residency,
- at Indiana University,
- and then I stayed on for pediatric hematology
- oncology fellowship just on the pediatric side for
- oncology.
- And then I became a stem cell transplant
- physician, and I have been here since 2012.
- And I've been the medical director of the
- pediatric program since 2018 and the FACT accreditation
- director since 2023.
- Perfect.
- And I'm excited to share the reason that
- you're on the podcast today.
- In February of this year,
- a 14 year old patient at Riley Hospital
- for Children became the first
- pediatric patient in the world to receive a
- bone marrow stem cell transplant
- using cells from a deceased
- donor.
- Doctor Styles, can you walk
- our audience through what made this case different
- and why this approach matters
- for the industry.
- Sure. It really is very exciting.
- So
- our patient is a 14 year old who
- was originally diagnosed with acute myeloid leukemia in
- the fall of twenty twenty five, And
- he received his upfront chemotherapy,
- but, unfortunately, was
- refractory to chemotherapy. His disease did not get
- into remission as quickly as what we expected,
- and that made him eligible to receive a
- stem cell transplant.
- So we proceeded to his stem cell transplant
- according to sort of standard of care.
- We
- found an unrelated donor through the NMDP
- and received those donor cells, and his original
- transplant,
- his first transplant
- happened in
- late December, early January.
- Unfortunately, that donor cells did not grow.
- His
- the patient's immune system rejected those stem cells
- from coming in,
- And so that forced us into a situation
- where we had to be
- very nimble in trying to find a new
- donor for him. And, really, when you're in
- that situation,
- it really is life and death scenario where
- if a patient's stem cells fail to engraft
- with a transplant,
- you really need new cells in very quickly
- in order to not allow that patient to
- succumb to infections.
- Primarily viral infections, can really become a big
- problem when you don't have an immune system.
- And so we were in a situation where
- we were looking for a backup donor and
- looking to move very quickly.
- Because we know that this is something that
- can happen in transplant, we always enter into
- a stem cell transplant
- with a plan a and at least a
- plan b, hopefully, also a plan c and
- d.
- And in this patient situation,
- his plan b that we went to mobilize
- and activate, that plan fell through.
- For a variety of reasons, that that plan
- did not work out.
- And, unfortunately,
- this patient did not have a plan c
- or d. And so we found ourselves in
- a situation where we were reaching out to
- NMDP to say, can you please help us
- mobilize a replacement donor very quickly?
- And they were very willing, but the best
- that they could do was, like, a four
- to six week timeline
- in order to get a backup donor mobilized.
- And we knew that if we waited for
- that timeline to happen, that our patient would
- not survive.
- And so we had to put on our
- thinking caps and become innovative, and we were
- aware of a company called Osteom,
- that they had been working to bring forward
- the option for deceased donor stem cell transplants,
- through a clinical trial process.
- And
- we had been in conversation with them about
- potentially opening that trial, but we had not
- activated things for that trial yet. And so
- I reached out to Osteem and asked, hey.
- We have an urgent need. We have a
- patient's life on the line. Can you help
- us? And they were very gracious in saying,
- yes. We absolutely can.
- Let's get to work. And so from the
- time that I reached out to them until
- the time that we had cells available for
- this patient, it was about a week,
- to get through the process. And, and being
- totally transparent, it was a crazy week. It
- was lots of work.
- I would not
- advocate for doing, a deceased donor transplant under
- that kind of stress and pressure.
- Being proactive is always better, but, we were
- able to get it done with a lot
- of support from Oceum.
- And,
- and as a result, the patient survived and
- is doing great.
- Amazing. Thank you for,
- kind of
- giving us the overview, I know, as
- comprehensively as you can.
- I remember when the news broke.
- It says four thousand children a year in
- The US are diagnosed with blood cancers that
- may need a transplant.
- And the process you just mentioned obviously highlights
- how finding a living living donor is still
- one of the biggest bottlenecks, and like you
- said, is a life or death situation
- without the four to six weeks that you
- can wait. Right?
- How big of a dent do you think
- deceased donor stem cell banking can realistically
- make
- in
- this gap over the next five years? And,
- you know, what would need to be true
- for this type of process to scale beyond
- just kind of a small number
- of transplant centers or unique patients where it
- just works out, just like you said, after
- a crazy week.
- What does that look like?
- Yeah. I think it's a really good question.
- And I think the the important
- point is that, it's not
- this this type of donor is not just
- for children. It's also for adults.
- Our patient was the first pediatric patient in
- the world, but there now to date have
- been 27 other patients
- who have received a deceased donor transplant.
- But you're a 100% correct that identifying a
- donor
- is,
- by far the biggest bottleneck in getting a
- patient successfully to transplant.
- So there are about ten thousand patients who
- undergo allo transplant in The United States every
- year, and every single one of them needs
- a donor. And so the NMDP,
- they're they're really
- outstanding
- at finding a match for nearly every patient.
- The problem is that finding a match is
- only half of it. Right?
- So just because you have a match in
- the registry does not necessarily
- mean that that translates to an available donor
- who's willing to donate on the timeline that
- the patient needs it. And I think that's
- why it becomes such a big barrier is
- because it's very
- dependent on willingness, availability
- of the donor, but also willingness and availability
- of the donor center.
- So you can find a donor somewhere out
- there in the world through the registry, but
- whether or not the timeline aligns with the
- patient's needs
- is very questionable,
- and often is not as fast as what
- we would like for it to be.
- And there are patients who die every year
- because they can't get a donor on the
- timeline that's needed.
- So we are working to fill the gap
- from a number of different fronts. But when
- when you're looking specifically about the option for
- deceased donor transplants, I do think that there
- is a gap that needs to be filled.
- And I do think that deceased donor transplant
- has huge potential
- for not only helping to fill that gap,
- but I would even go so far as
- to say that this could be the preferred
- way that we do transplants in the future
- once we have enough data to say data
- and longevity of data to say that this
- really is a viable alternative source of cells,
- and it might even be preferable
- and more cost effective at at the end
- of the day.
- So
- right at this moment in time, you know,
- Oceum has been building their deceased donor bank
- for several years.
- I don't know exactly how many deceased donors
- that they have available
- at any given time, but the number is
- growing every day.
- So for con for, you know, context, the
- NMDP
- has
- somewhere
- near or around the ballpark of 20,000,000,
- donors that are part of the registry.
- I think ASEAN, the last time that I
- talked with them, had somewhere north of a
- thousand,
- deceased donors that are banked. And so right
- at this moment in time, there's not an
- outstanding chance that you would search the registry
- and find a perfect match for a patient,
- but you can search the registry and find
- an acceptable mass match for a patient.
- One of the things that we have
- been working toward in the field for probably
- the last ten years is utilization
- of donors that are not a perfect match
- that we know we can overcome
- a slight mismatch from a patient,
- with the use of additional chemotherapy.
- The problem with that approach, it works very
- well for getting stem cells to grow. However,
- the downside is that it's associated with more
- toxicity, both in terms of side effects from
- the extra chemotherapy
- and side effects from the degree of mismatch
- between the patient and the donor.
- So
- for my patient,
- the the donor that we received from Osteem
- was not a perfect match, but it was
- an acceptable match, and that's how we were
- able to move forward.
- And I think one of the real benefits
- in addition to the timeliness and availability of
- getting a donor that's a deceased donor is
- also the fact that on the front end,
- the day that you order
- the mobilization of the cord from Osium to
- your center, you know the minute that you
- place that order what the cell dose is
- going to be. And that's just not true.
- When you have an unrelated donor,
- you get whatever cell dose you get And
- by the you know, there are things that
- you can do to stack the duck in
- your favor, but you never know what you're
- gonna get until the cells show up to
- your to your site. And
- that was, I think, part of the contributing
- problem for
- my patient whose first transplant failed is that
- the cell dose was not as much as
- what we needed it to be for the
- condition that we were treating.
- And when I ordered the donor from Osteem,
- I knew the minute that I asked them
- to ship it that I was gonna have
- enough cells. And and in fact, I had
- enough sales and some sales left over in
- the bank that if he he should ever
- need a boost of sales in the future,
- we still have some leftover in the freezer,
- which is incredible.
- Absolutely. And, you know, you kind of alluded
- it to
- what it took to be operationally
- ready
- for this.
- If, you know, this or if slash when
- this becomes more common
- to, you know, have a patient from no
- donor match to transplant in days instead of
- the weeks or months that we talked about
- previously.
- For health system leaders that are listening
- to this podcast and thinking about
- what this might do for their own programs,
- What did it take operationally to be ready
- to act, and what should leaders be building
- to make sure that they're ready should they
- find themselves in a similar situation?
- Yeah. It's a really good question. So what
- it took to be ready,
- is
- there was a lot of frankly, there was
- a lot of paperwork that needed to happen.
- So number one, there needed to be contracts
- established with ACM through our health care organization.
- We had to set up contracts for, you
- know, materials transfer, those sort of things.
- And then the other thing that, was a
- lot of work in getting set up was,
- because deceased donor stem cells are not considered
- an they're not considered an investigational product by
- the FDA,
- but because there there isn't a lot of
- data around it, many institutions will still want
- any infusion of deceased donor stem cells to
- go through
- their institutional review board process
- so that it's reviewed and has a formal
- stamp of approval that this is, you know,
- to make sure that we're communicating properly to
- patients, that this is still considered something that
- is a new therapy,
- and that we don't really have long term
- data on the outcomes. But what we do
- have for early data is very promising. They
- wanna make sure that we're communicating that properly
- to patients and that we're tracking
- outcomes, that we don't just do a deceased
- donor stem cell transplant and then pray that
- everything is fine, and it's not until you
- do 50 of them that you realize that
- there's a problem. Right? The only way that
- that happens in a systematic way is through
- the process of of having an IRB
- approval
- for tracking patients. And so our institution, like
- many others, really wanted this to go through
- the IRB approval pathway.
- So getting a institutional approval to proceed
- was part of it and then writing the
- consent documents to explain all that I just
- explained to you to a patient,
- and having them sign all of those documents
- and having everything squared away for that, those
- were the things that took a lot of
- hours in that one week of time from
- the time that we knew that we had
- a potential donor to the time that we
- were ready to move and ship the cells.
- I think Aseem would have been more than
- willing to put the cells bring the cells
- to us literally the next day. But from
- the institutional side of things, we still needed
- to get our ducks in a row. So
- I think when we think about what it
- could look like for another institution
- to mobilize access to this type of donor
- source,
- if you really wanted to be proactive so
- that you don't have a really rough week
- like I had and, like, my patient waiting,
- it would be to reach out to Oceum
- in advance and say, what can we do
- to be prepared
- for
- being able to offer cells
- if we end up in a similar situation
- like this? Or if you wanted to be
- super proactive, you can actually sign up to
- be part of the clinical trials.
- So you could reach out and say, Assiim
- is running formal clinical trials that are more
- than just an expanded access program,
- and they are recruiting sites now. So you
- could reach out to them and say, we
- would like to be a site. Can we
- get onboarded for being a clinical trial site?
- Perfect.
- I'm gonna shift gears a little bit as
- we wrapped up the conversation.
- We've kind of zoomed in on this, you
- know, specific case, but I'd love to just
- pick your brain
- for a kind of broader perspective
- on
- pediatric oncology, transplant programs,
- the pressures that they're facing right now.
- As you've got an audience of health system
- leaders, is there anything
- you feel
- is not getting enough attention, whether that's, you
- know, access pressure, funding, staffing?
- And through the same veins, is there anything
- you see in the future that leaders might
- need to tackle when it comes to closing
- care gaps within pediatric oncology and transplant programs
- like this?
- That is a heavyweight question. One. Yeah.
- So
- I'll start with, you know, pressures in the
- field right now and challenges that we're facing.
- Really, in the field of stem cell transplant,
- obviously, we've talked about donor access. But I
- think you can't talk about,
- challenges and shortfalls in stem cell transplant without
- talking about what it takes to pull off
- a transplant successfully.
- And, really, that starts with having health care
- team members that have experience.
- We the nursing shortage is profound
- across all health care systems.
- And then when you're
- trying to deliver these these highly advanced therapies
- that are highly toxic,
- having an experienced skill set of nursing
- and nurse practitioners and physicians and coordinators,
- all of those people
- not being new
- is really critical. And so I think,
- health care systems valuing the experience that they
- have and their existing team members and working
- to retain. I know everybody wants to retain
- their nurses,
- but, and everybody is working on the short
- fallings that we have in staffing. But in
- a field like this, where experience is sometimes
- the difference between life and death, we have
- to prioritize
- retaining experience. So I think that's probably one
- really big thing.
- I think
- in terms of, you know,
- other system challenges, I think, you know,
- the cost of these type of therapies of
- advanced therapeutics, like stem cell transplant, CAR t
- cell therapy, gene therapy,
- both inside and outside of cancer care require
- really innovative thinking and budgetary planning.
- And I think, you know, we're in the
- midst of these conversations with our own health
- care system right now of, you know, health
- care as a whole is really struggling financially
- right now. And there are lots of reasons
- for that, but I think,
- being strategic in what we invest in
- and,
- being,
- you know, thoughtful about
- where we place our dollars and cents,
- in order to provide care to patients
- and,
- specifically, to provide equitable access to care is
- another really big piece of things.
- And that requires having
- proactive conversations
- with public payers specifically,
- in order to ensure continued equitable access to
- care.
- I think in terms of
- what we're, like, looking at in the field
- and closing gaps in
- care as they currently exist of,
- where the science is versus where we wanna
- be. When we really think about what therapeutics
- are available to us, I think we have
- amazing tools in our toolbox
- and working to move those tools a little
- earlier in care so that patients don't come
- to us
- with, you
- know, existing organ damage or existing toxicity that
- makes their care more complicated
- would be beneficial
- for patient care. The rub and the challenge
- in that is that those advanced therapeutics are
- more expensive.
- So trying to balance, you know, who really
- needs these therapies and how can we integrate
- them in a way that financially makes sense
- both for patients and health care systems is
- it's a big challenge if I'm being really
- honest.
- And then within the science of where it
- currently exists within I'll speak specifically to cancer
- because that's what I do.
- Mhmm. You know, we've gotten pretty good at
- using advanced therapeutics,
- like cell therapy and gene therapy
- for
- hematologic malignancies, so leukemias, lymphomas, that sort of
- thing. But there are still very large challenges
- that exist in the realm of solid tumors,
- which are actually much greater in number than
- hematologic malignancies.
- So,
- we're working to we're working with our bench
- scientists to advance to next generation CAR T
- cell therapies that can
- address some of those shortcomings.
- And then the other thing that we really
- have to figure out is how to deliver
- this care
- less expensively.
- Mhmm. So,
- we are you know, that's another area where
- science is working so that we can do
- this therapy and not break the bank in
- doing it,
- because there will be ways to streamline this
- therapy moving forward
- that will be less
- costly and less labor intensive,
- and that will help provide equitable access to
- care as well.
- So
- those are just some of the musings off
- the you know? Yeah. Off of what I
- see every day, but I think,
- that there are gaps to be filled is
- not a question.
- Absolutely. And, you know, that's what I love
- about having these conversations on the podcast because
- you are able to share, you know,
- your musings based on what you're seeing, and
- I know that it resonates with, you know,
- other oncology leaders across the country. So I'm
- so grateful, doctor Scowles, that you took the
- time to come on the podcast to share
- more details behind this case, but also just
- your insights. Overall, I hope we can connect
- again soon.
- Absolutely. Thank you for having me.
- And thank you to our listeners for tuning
- in to Becker's Oncology Leadership Podcast.
- I invite you all to check out even
- more Becker's Healthcare Podcasts at breccarspodcasts.com.
- Thank you again to doctor Styles for joining
- me, and thank you to our listeners for
- tuning in. Have a good day.