Latest / Becker’s Healthcare Podcast / Jodi Skiles, MD, Medical Director of the Pediatric Stem Cell Transplant Program at Riley Hospital for Children
Transcript
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- 0:52- Hello. Thank you so much for tuning in
- 0:54- to the Becker's Oncology Leadership Podcast.
- 0:57- I'm Elizabeth Gregersen, a reporter here at Becker's.
- 1:00- And today, I'm talking to Jody Skiles,
- 1:03- doctor Jody Skiles,
- 1:05- medical director of the pediatric stem cell transplant
- 1:08- program at Riley Hospital for Children in Indianapolis
- 1:12- and the FACT Accreditation medical director for both
- 1:15- pediatric and adult stem cell transplant programs at
- 1:18- Indiana University.
- 1:19- Doctor Skiles, thank you so much for taking
- 1:21- the time to speak with me today.
- 1:23- Of course. Thank you for the opportunity to
- 1:25- be here.
- 1:26- Absolutely. And before we dive into our conversation,
- 1:28- because it's a good one for our listeners,
- 1:30- I would love if you could share just
- 1:32- a bit about your background and your role
- 1:34- at Riley Hospital for Children.
- 1:36- Sure.
- 1:37- So I have been on staff at Riley
- 1:41- since 2012 when I graduated from fellowship. So
- 1:44- my background training, I was internal medicine and
- 1:47- pediatrics trained
- 1:49- for residency,
- 1:50- at Indiana University,
- 1:52- and then I stayed on for pediatric hematology
- 1:55- oncology fellowship just on the pediatric side for
- 1:58- oncology.
- 1:59- And then I became a stem cell transplant
- 2:01- physician, and I have been here since 2012.
- 2:04- And I've been the medical director of the
- 2:05- pediatric program since 2018 and the FACT accreditation
- 2:08- director since 2023.
- 2:11- Perfect.
- 2:12- And I'm excited to share the reason that
- 2:14- you're on the podcast today.
- 2:16- In February of this year,
- 2:18- a 14 year old patient at Riley Hospital
- 2:21- for Children became the first
- 2:24- pediatric patient in the world to receive a
- 2:26- bone marrow stem cell transplant
- 2:29- using cells from a deceased
- 2:31- donor.
- 2:33- Doctor Styles, can you walk
- 2:35- our audience through what made this case different
- 2:37- and why this approach matters
- 2:40- for the industry.
- 2:43- Sure. It really is very exciting.
- 2:47- So
- 2:47- our patient is a 14 year old who
- 2:50- was originally diagnosed with acute myeloid leukemia in
- 2:53- the fall of twenty twenty five, And
- 2:56- he received his upfront chemotherapy,
- 2:58- but, unfortunately, was
- 3:00- refractory to chemotherapy. His disease did not get
- 3:03- into remission as quickly as what we expected,
- 3:05- and that made him eligible to receive a
- 3:07- stem cell transplant.
- 3:09- So we proceeded to his stem cell transplant
- 3:12- according to sort of standard of care.
- 3:15- We
- 3:16- found an unrelated donor through the NMDP
- 3:20- and received those donor cells, and his original
- 3:23- transplant,
- 3:25- his first transplant
- 3:26- happened in
- 3:28- late December, early January.
- 3:30- Unfortunately, that donor cells did not grow.
- 3:34- His
- 3:35- the patient's immune system rejected those stem cells
- 3:38- from coming in,
- 3:39- And so that forced us into a situation
- 3:41- where we had to be
- 3:44- very nimble in trying to find a new
- 3:46- donor for him. And, really, when you're in
- 3:48- that situation,
- 3:49- it really is life and death scenario where
- 3:53- if a patient's stem cells fail to engraft
- 3:55- with a transplant,
- 3:56- you really need new cells in very quickly
- 3:59- in order to not allow that patient to
- 4:01- succumb to infections.
- 4:04- Primarily viral infections, can really become a big
- 4:06- problem when you don't have an immune system.
- 4:08- And so we were in a situation where
- 4:10- we were looking for a backup donor and
- 4:12- looking to move very quickly.
- 4:15- Because we know that this is something that
- 4:16- can happen in transplant, we always enter into
- 4:19- a stem cell transplant
- 4:20- with a plan a and at least a
- 4:22- plan b, hopefully, also a plan c and
- 4:24- d.
- 4:25- And in this patient situation,
- 4:28- his plan b that we went to mobilize
- 4:30- and activate, that plan fell through.
- 4:33- For a variety of reasons, that that plan
- 4:36- did not work out.
- 4:37- And, unfortunately,
- 4:38- this patient did not have a plan c
- 4:40- or d. And so we found ourselves in
- 4:43- a situation where we were reaching out to
- 4:44- NMDP to say, can you please help us
- 4:46- mobilize a replacement donor very quickly?
- 4:49- And they were very willing, but the best
- 4:51- that they could do was, like, a four
- 4:53- to six week timeline
- 4:54- in order to get a backup donor mobilized.
- 4:58- And we knew that if we waited for
- 5:00- that timeline to happen, that our patient would
- 5:02- not survive.
- 5:03- And so we had to put on our
- 5:05- thinking caps and become innovative, and we were
- 5:08- aware of a company called Osteom,
- 5:11- that they had been working to bring forward
- 5:14- the option for deceased donor stem cell transplants,
- 5:18- through a clinical trial process.
- 5:20- And
- 5:21- we had been in conversation with them about
- 5:23- potentially opening that trial, but we had not
- 5:25- activated things for that trial yet. And so
- 5:28- I reached out to Osteem and asked, hey.
- 5:30- We have an urgent need. We have a
- 5:32- patient's life on the line. Can you help
- 5:34- us? And they were very gracious in saying,
- 5:37- yes. We absolutely can.
- 5:39- Let's get to work. And so from the
- 5:41- time that I reached out to them until
- 5:43- the time that we had cells available for
- 5:45- this patient, it was about a week,
- 5:47- to get through the process. And, and being
- 5:50- totally transparent, it was a crazy week. It
- 5:52- was lots of work.
- 5:54- I would not
- 5:55- advocate for doing, a deceased donor transplant under
- 5:58- that kind of stress and pressure.
- 6:00- Being proactive is always better, but, we were
- 6:03- able to get it done with a lot
- 6:05- of support from Oceum.
- 6:06- And,
- 6:08- and as a result, the patient survived and
- 6:10- is doing great.
- 6:12- Amazing. Thank you for,
- 6:14- kind of
- 6:16- giving us the overview, I know, as
- 6:18- comprehensively as you can.
- 6:21- I remember when the news broke.
- 6:24- It says four thousand children a year in
- 6:26- The US are diagnosed with blood cancers that
- 6:29- may need a transplant.
- 6:31- And the process you just mentioned obviously highlights
- 6:34- how finding a living living donor is still
- 6:37- one of the biggest bottlenecks, and like you
- 6:39- said, is a life or death situation
- 6:42- without the four to six weeks that you
- 6:44- can wait. Right?
- 6:46- How big of a dent do you think
- 6:49- deceased donor stem cell banking can realistically
- 6:53- make
- 6:54- in
- 6:55- this gap over the next five years? And,
- 6:58- you know, what would need to be true
- 7:01- for this type of process to scale beyond
- 7:03- just kind of a small number
- 7:05- of transplant centers or unique patients where it
- 7:08- just works out, just like you said, after
- 7:10- a crazy week.
- 7:11- What does that look like?
- 7:14- Yeah. I think it's a really good question.
- 7:16- And I think the the important
- 7:18- point is that, it's not
- 7:21- this this type of donor is not just
- 7:23- for children. It's also for adults.
- 7:25- Our patient was the first pediatric patient in
- 7:28- the world, but there now to date have
- 7:30- been 27 other patients
- 7:32- who have received a deceased donor transplant.
- 7:35- But you're a 100% correct that identifying a
- 7:38- donor
- 7:39- is,
- 7:40- by far the biggest bottleneck in getting a
- 7:43- patient successfully to transplant.
- 7:45- So there are about ten thousand patients who
- 7:47- undergo allo transplant in The United States every
- 7:49- year, and every single one of them needs
- 7:51- a donor. And so the NMDP,
- 7:55- they're they're really
- 7:57- outstanding
- 7:59- at finding a match for nearly every patient.
- 8:02- The problem is that finding a match is
- 8:05- only half of it. Right?
- 8:07- So just because you have a match in
- 8:09- the registry does not necessarily
- 8:11- mean that that translates to an available donor
- 8:15- who's willing to donate on the timeline that
- 8:17- the patient needs it. And I think that's
- 8:19- why it becomes such a big barrier is
- 8:21- because it's very
- 8:23- dependent on willingness, availability
- 8:26- of the donor, but also willingness and availability
- 8:28- of the donor center.
- 8:30- So you can find a donor somewhere out
- 8:32- there in the world through the registry, but
- 8:34- whether or not the timeline aligns with the
- 8:36- patient's needs
- 8:38- is very questionable,
- 8:40- and often is not as fast as what
- 8:42- we would like for it to be.
- 8:44- And there are patients who die every year
- 8:45- because they can't get a donor on the
- 8:47- timeline that's needed.
- 8:50- So we are working to fill the gap
- 8:51- from a number of different fronts. But when
- 8:54- when you're looking specifically about the option for
- 8:57- deceased donor transplants, I do think that there
- 8:59- is a gap that needs to be filled.
- 9:01- And I do think that deceased donor transplant
- 9:04- has huge potential
- 9:06- for not only helping to fill that gap,
- 9:08- but I would even go so far as
- 9:10- to say that this could be the preferred
- 9:12- way that we do transplants in the future
- 9:14- once we have enough data to say data
- 9:17- and longevity of data to say that this
- 9:19- really is a viable alternative source of cells,
- 9:22- and it might even be preferable
- 9:24- and more cost effective at at the end
- 9:26- of the day.
- 9:28- So
- 9:29- right at this moment in time, you know,
- 9:31- Oceum has been building their deceased donor bank
- 9:34- for several years.
- 9:36- I don't know exactly how many deceased donors
- 9:38- that they have available
- 9:40- at any given time, but the number is
- 9:42- growing every day.
- 9:44- So for con for, you know, context, the
- 9:47- NMDP
- 9:48- has
- 9:49- somewhere
- 9:50- near or around the ballpark of 20,000,000,
- 9:54- donors that are part of the registry.
- 9:57- I think ASEAN, the last time that I
- 9:59- talked with them, had somewhere north of a
- 10:02- thousand,
- 10:03- deceased donors that are banked. And so right
- 10:06- at this moment in time, there's not an
- 10:08- outstanding chance that you would search the registry
- 10:11- and find a perfect match for a patient,
- 10:13- but you can search the registry and find
- 10:15- an acceptable mass match for a patient.
- 10:17- One of the things that we have
- 10:20- been working toward in the field for probably
- 10:22- the last ten years is utilization
- 10:24- of donors that are not a perfect match
- 10:26- that we know we can overcome
- 10:28- a slight mismatch from a patient,
- 10:32- with the use of additional chemotherapy.
- 10:34- The problem with that approach, it works very
- 10:36- well for getting stem cells to grow. However,
- 10:39- the downside is that it's associated with more
- 10:42- toxicity, both in terms of side effects from
- 10:44- the extra chemotherapy
- 10:46- and side effects from the degree of mismatch
- 10:48- between the patient and the donor.
- 10:50- So
- 10:52- for my patient,
- 10:53- the the donor that we received from Osteem
- 10:56- was not a perfect match, but it was
- 10:58- an acceptable match, and that's how we were
- 11:00- able to move forward.
- 11:02- And I think one of the real benefits
- 11:05- in addition to the timeliness and availability of
- 11:08- getting a donor that's a deceased donor is
- 11:10- also the fact that on the front end,
- 11:12- the day that you order
- 11:14- the mobilization of the cord from Osium to
- 11:17- your center, you know the minute that you
- 11:20- place that order what the cell dose is
- 11:21- going to be. And that's just not true.
- 11:24- When you have an unrelated donor,
- 11:26- you get whatever cell dose you get And
- 11:28- by the you know, there are things that
- 11:30- you can do to stack the duck in
- 11:31- your favor, but you never know what you're
- 11:32- gonna get until the cells show up to
- 11:34- your to your site. And
- 11:36- that was, I think, part of the contributing
- 11:39- problem for
- 11:40- my patient whose first transplant failed is that
- 11:43- the cell dose was not as much as
- 11:44- what we needed it to be for the
- 11:46- condition that we were treating.
- 11:48- And when I ordered the donor from Osteem,
- 11:51- I knew the minute that I asked them
- 11:53- to ship it that I was gonna have
- 11:54- enough cells. And and in fact, I had
- 11:56- enough sales and some sales left over in
- 11:58- the bank that if he he should ever
- 11:59- need a boost of sales in the future,
- 12:01- we still have some leftover in the freezer,
- 12:03- which is incredible.
- 12:06- Absolutely. And, you know, you kind of alluded
- 12:08- it to
- 12:10- what it took to be operationally
- 12:12- ready
- 12:13- for this.
- 12:14- If, you know, this or if slash when
- 12:17- this becomes more common
- 12:19- to, you know, have a patient from no
- 12:22- donor match to transplant in days instead of
- 12:25- the weeks or months that we talked about
- 12:27- previously.
- 12:28- For health system leaders that are listening
- 12:31- to this podcast and thinking about
- 12:34- what this might do for their own programs,
- 12:37- What did it take operationally to be ready
- 12:40- to act, and what should leaders be building
- 12:43- to make sure that they're ready should they
- 12:45- find themselves in a similar situation?
- 12:48- Yeah. It's a really good question. So what
- 12:50- it took to be ready,
- 12:52- is
- 12:54- there was a lot of frankly, there was
- 12:55- a lot of paperwork that needed to happen.
- 12:57- So number one, there needed to be contracts
- 12:59- established with ACM through our health care organization.
- 13:03- We had to set up contracts for, you
- 13:05- know, materials transfer, those sort of things.
- 13:08- And then the other thing that, was a
- 13:10- lot of work in getting set up was,
- 13:14- because deceased donor stem cells are not considered
- 13:17- an they're not considered an investigational product by
- 13:20- the FDA,
- 13:22- but because there there isn't a lot of
- 13:24- data around it, many institutions will still want
- 13:27- any infusion of deceased donor stem cells to
- 13:29- go through
- 13:30- their institutional review board process
- 13:33- so that it's reviewed and has a formal
- 13:35- stamp of approval that this is, you know,
- 13:38- to make sure that we're communicating properly to
- 13:40- patients, that this is still considered something that
- 13:43- is a new therapy,
- 13:44- and that we don't really have long term
- 13:46- data on the outcomes. But what we do
- 13:48- have for early data is very promising. They
- 13:51- wanna make sure that we're communicating that properly
- 13:53- to patients and that we're tracking
- 13:55- outcomes, that we don't just do a deceased
- 13:57- donor stem cell transplant and then pray that
- 13:59- everything is fine, and it's not until you
- 14:01- do 50 of them that you realize that
- 14:02- there's a problem. Right? The only way that
- 14:05- that happens in a systematic way is through
- 14:07- the process of of having an IRB
- 14:10- approval
- 14:11- for tracking patients. And so our institution, like
- 14:14- many others, really wanted this to go through
- 14:16- the IRB approval pathway.
- 14:18- So getting a institutional approval to proceed
- 14:21- was part of it and then writing the
- 14:23- consent documents to explain all that I just
- 14:25- explained to you to a patient,
- 14:28- and having them sign all of those documents
- 14:29- and having everything squared away for that, those
- 14:32- were the things that took a lot of
- 14:34- hours in that one week of time from
- 14:36- the time that we knew that we had
- 14:37- a potential donor to the time that we
- 14:39- were ready to move and ship the cells.
- 14:41- I think Aseem would have been more than
- 14:43- willing to put the cells bring the cells
- 14:45- to us literally the next day. But from
- 14:48- the institutional side of things, we still needed
- 14:50- to get our ducks in a row. So
- 14:52- I think when we think about what it
- 14:54- could look like for another institution
- 14:56- to mobilize access to this type of donor
- 14:59- source,
- 15:01- if you really wanted to be proactive so
- 15:02- that you don't have a really rough week
- 15:04- like I had and, like, my patient waiting,
- 15:07- it would be to reach out to Oceum
- 15:08- in advance and say, what can we do
- 15:10- to be prepared
- 15:12- for
- 15:13- being able to offer cells
- 15:16- if we end up in a similar situation
- 15:17- like this? Or if you wanted to be
- 15:19- super proactive, you can actually sign up to
- 15:21- be part of the clinical trials.
- 15:23- So you could reach out and say, Assiim
- 15:25- is running formal clinical trials that are more
- 15:29- than just an expanded access program,
- 15:31- and they are recruiting sites now. So you
- 15:33- could reach out to them and say, we
- 15:35- would like to be a site. Can we
- 15:37- get onboarded for being a clinical trial site?
- 15:42- Perfect.
- 15:43- I'm gonna shift gears a little bit as
- 15:45- we wrapped up the conversation.
- 15:47- We've kind of zoomed in on this, you
- 15:49- know, specific case, but I'd love to just
- 15:53- pick your brain
- 15:54- for a kind of broader perspective
- 15:57- on
- 15:58- pediatric oncology, transplant programs,
- 16:01- the pressures that they're facing right now.
- 16:04- As you've got an audience of health system
- 16:06- leaders, is there anything
- 16:08- you feel
- 16:10- is not getting enough attention, whether that's, you
- 16:13- know, access pressure, funding, staffing?
- 16:16- And through the same veins, is there anything
- 16:18- you see in the future that leaders might
- 16:20- need to tackle when it comes to closing
- 16:22- care gaps within pediatric oncology and transplant programs
- 16:26- like this?
- 16:29- That is a heavyweight question. One. Yeah.
- 16:32- So
- 16:33- I'll start with, you know, pressures in the
- 16:35- field right now and challenges that we're facing.
- 16:39- Really, in the field of stem cell transplant,
- 16:41- obviously, we've talked about donor access. But I
- 16:44- think you can't talk about,
- 16:47- challenges and shortfalls in stem cell transplant without
- 16:49- talking about what it takes to pull off
- 16:51- a transplant successfully.
- 16:53- And, really, that starts with having health care
- 16:56- team members that have experience.
- 16:59- We the nursing shortage is profound
- 17:02- across all health care systems.
- 17:04- And then when you're
- 17:06- trying to deliver these these highly advanced therapies
- 17:10- that are highly toxic,
- 17:12- having an experienced skill set of nursing
- 17:15- and nurse practitioners and physicians and coordinators,
- 17:18- all of those people
- 17:20- not being new
- 17:22- is really critical. And so I think,
- 17:25- health care systems valuing the experience that they
- 17:28- have and their existing team members and working
- 17:30- to retain. I know everybody wants to retain
- 17:33- their nurses,
- 17:34- but, and everybody is working on the short
- 17:36- fallings that we have in staffing. But in
- 17:39- a field like this, where experience is sometimes
- 17:42- the difference between life and death, we have
- 17:45- to prioritize
- 17:46- retaining experience. So I think that's probably one
- 17:49- really big thing.
- 17:51- I think
- 17:52- in terms of, you know,
- 17:55- other system challenges, I think, you know,
- 17:58- the cost of these type of therapies of
- 18:00- advanced therapeutics, like stem cell transplant, CAR t
- 18:03- cell therapy, gene therapy,
- 18:06- both inside and outside of cancer care require
- 18:09- really innovative thinking and budgetary planning.
- 18:12- And I think, you know, we're in the
- 18:13- midst of these conversations with our own health
- 18:15- care system right now of, you know, health
- 18:18- care as a whole is really struggling financially
- 18:20- right now. And there are lots of reasons
- 18:22- for that, but I think,
- 18:25- being strategic in what we invest in
- 18:29- and,
- 18:30- being,
- 18:31- you know, thoughtful about
- 18:34- where we place our dollars and cents,
- 18:37- in order to provide care to patients
- 18:40- and,
- 18:41- specifically, to provide equitable access to care is
- 18:44- another really big piece of things.
- 18:48- And that requires having
- 18:50- proactive conversations
- 18:52- with public payers specifically,
- 18:55- in order to ensure continued equitable access to
- 18:57- care.
- 18:59- I think in terms of
- 19:01- what we're, like, looking at in the field
- 19:03- and closing gaps in
- 19:06- care as they currently exist of,
- 19:09- where the science is versus where we wanna
- 19:11- be. When we really think about what therapeutics
- 19:14- are available to us, I think we have
- 19:16- amazing tools in our toolbox
- 19:18- and working to move those tools a little
- 19:21- earlier in care so that patients don't come
- 19:23- to us
- 19:24- with, you
- 19:26- know, existing organ damage or existing toxicity that
- 19:30- makes their care more complicated
- 19:32- would be beneficial
- 19:33- for patient care. The rub and the challenge
- 19:35- in that is that those advanced therapeutics are
- 19:37- more expensive.
- 19:39- So trying to balance, you know, who really
- 19:41- needs these therapies and how can we integrate
- 19:43- them in a way that financially makes sense
- 19:46- both for patients and health care systems is
- 19:49- it's a big challenge if I'm being really
- 19:50- honest.
- 19:53- And then within the science of where it
- 19:55- currently exists within I'll speak specifically to cancer
- 19:58- because that's what I do.
- 20:00- Mhmm. You know, we've gotten pretty good at
- 20:03- using advanced therapeutics,
- 20:04- like cell therapy and gene therapy
- 20:07- for
- 20:09- hematologic malignancies, so leukemias, lymphomas, that sort of
- 20:12- thing. But there are still very large challenges
- 20:15- that exist in the realm of solid tumors,
- 20:18- which are actually much greater in number than
- 20:20- hematologic malignancies.
- 20:22- So,
- 20:23- we're working to we're working with our bench
- 20:26- scientists to advance to next generation CAR T
- 20:29- cell therapies that can
- 20:30- address some of those shortcomings.
- 20:33- And then the other thing that we really
- 20:35- have to figure out is how to deliver
- 20:36- this care
- 20:38- less expensively.
- 20:39- Mhmm. So,
- 20:41- we are you know, that's another area where
- 20:43- science is working so that we can do
- 20:45- this therapy and not break the bank in
- 20:47- doing it,
- 20:49- because there will be ways to streamline this
- 20:51- therapy moving forward
- 20:53- that will be less
- 20:55- costly and less labor intensive,
- 20:58- and that will help provide equitable access to
- 21:00- care as well.
- 21:02- So
- 21:03- those are just some of the musings off
- 21:04- the you know? Yeah. Off of what I
- 21:06- see every day, but I think,
- 21:08- that there are gaps to be filled is
- 21:10- not a question.
- 21:11- Absolutely. And, you know, that's what I love
- 21:14- about having these conversations on the podcast because
- 21:16- you are able to share, you know,
- 21:18- your musings based on what you're seeing, and
- 21:20- I know that it resonates with, you know,
- 21:22- other oncology leaders across the country. So I'm
- 21:26- so grateful, doctor Scowles, that you took the
- 21:28- time to come on the podcast to share
- 21:31- more details behind this case, but also just
- 21:33- your insights. Overall, I hope we can connect
- 21:35- again soon.
- 21:37- Absolutely. Thank you for having me.
- 21:39- And thank you to our listeners for tuning
- 21:41- in to Becker's Oncology Leadership Podcast.
- 21:44- I invite you all to check out even
- 21:45- more Becker's Healthcare Podcasts at breccarspodcasts.com.
- 21:50- Thank you again to doctor Styles for joining
- 21:52- me, and thank you to our listeners for
- 21:53- tuning in. Have a good day.