Care of Sickle Cell Disease and Cancer Patients

Transcript

00:00 --> 00:01Funding for Yale Cancer Answers
00:01 --> 00:03is provided by Smilow Cancer
00:03 --> 00:05Hospital and AstraZeneca.
00:07 --> 00:09Welcome to Yale Cancer Answers with
00:09 --> 00:12your host doctor Anees Chagpar.
00:12 --> 00:14Yale Cancer Answers features the
00:14 --> 00:16latest information on cancer care by
00:16 --> 00:18welcoming oncologists and specialists
00:18 --> 00:20who are on the forefront of the
00:20 --> 00:22battle to fight cancer. This week,
00:22 --> 00:24it's a conversation about sickle
00:24 --> 00:26cell disease and cancer in pediatric
00:26 --> 00:28patients with doctor Farzana Pashankar.
00:28 --> 00:30Dr Pashankar is an associate
00:30 --> 00:32professor of Pediatrics in hematology
00:32 --> 00:35oncology at the Yale School of Medicine,
00:35 --> 00:38where Doctor Chagpar is a
00:38 --> 00:40professor of surgical oncology.
00:40 --> 00:43Maybe we can start off by you telling
00:43 --> 00:45us a little bit about yourself and
00:45 --> 00:48how you got involved in doing what
00:48 --> 00:50you do and what exactly do you do?
00:53 --> 00:57Essentially I had a really long,
00:57 --> 00:59circuitous career journey,
00:59 --> 01:03but I got involved in doing pediatric
01:03 --> 01:07oncology when I was training in England,
01:07 --> 01:10after which I did a pediatric hematology
01:10 --> 01:12oncology fellowship in Canada.
01:12 --> 01:14And after fellowship,
01:14 --> 01:18the two areas that I really loved
01:18 --> 01:21and wanted to focus my career
01:21 --> 01:23on were sickle cell disease
01:23 --> 01:25and solid tumors
01:25 --> 01:27and development of clinical
01:27 --> 01:30trials and improving care for children
01:30 --> 01:33with sickle cell disease and cancer,
01:33 --> 01:34particularly solid tumors.
01:34 --> 01:38So those are the two areas that
01:38 --> 01:40I have focused on in
01:40 --> 01:44my career for the last
01:44 --> 01:47about 17 to 20 years and my
01:47 --> 01:51passion primarily has been to focus on
01:51 --> 01:54development of clinical trials for children for
01:54 --> 01:57certain rare types of solid tumors,
01:57 --> 01:59and also in bringing new and
01:59 --> 02:01innovative therapies for sickle cell
02:01 --> 02:03disease to our patient population.
02:03 --> 02:04So maybe we
02:04 --> 02:06can start with that.
02:06 --> 02:08Is there much overlap between sickle
02:08 --> 02:10cell disease and pediatric cancers?
02:10 --> 02:14I mean, do children get sickle cell disease?
02:14 --> 02:16Does sickle cell kind of lead to
02:16 --> 02:19cancer or are these just two separate
02:19 --> 02:21passions of yours that happen to
02:22 --> 02:24coincide in the same individual?
02:25 --> 02:28These are two separate passions,
02:28 --> 02:31and because in Pediatrics we
02:31 --> 02:34train in both hematology and oncology,
02:34 --> 02:36these are two passions which
02:38 --> 02:40developed during my training,
02:40 --> 02:44but it is not connected in any way in terms
02:44 --> 02:47of children with sickle cell disease being
02:47 --> 02:49more prone to getting cancer or
02:49 --> 02:51children with cancer more prone to
02:51 --> 02:54having any issues with sickle cell.
02:56 --> 02:59Let's talk about each of the two in turn
02:59 --> 03:02and let's start maybe with
03:02 --> 03:04talking about pediatric cancers.
03:06 --> 03:09Any time we hear about children getting cancer,
03:09 --> 03:10the uniform emotion that
03:10 --> 03:12people feel is heartbreak.
03:12 --> 03:15So tell us a little bit more
03:15 --> 03:17about how you get involved.
03:17 --> 03:20I know so many medical
03:20 --> 03:23students come up to me and they say,
03:23 --> 03:26how can you possibly dedicate your career
03:26 --> 03:29to doing something that is so heartbreaking,
03:31 --> 03:34but honestly after doing this for over 20 years
03:34 --> 03:36this is such a rewarding journey.
03:36 --> 03:39It is the time that a lot of
03:39 --> 03:41families are going through
03:41 --> 03:43probably the most intense and difficult
03:43 --> 03:46time of their life and to be able
03:46 --> 03:49to be a part of it and to help them
03:49 --> 03:52navigate and think about the treatment
03:52 --> 03:54decisions for their child and to be
03:54 --> 03:57able to treat their child effectively,
03:57 --> 03:59honestly, I don't think
03:59 --> 04:01there's a substitute for that.
04:01 --> 04:03I think it's so emotionally rewarding.
04:03 --> 04:06It is also heartbreaking at times.
04:06 --> 04:09I mean, we do have children who could
04:09 --> 04:11have a recurrence and it is
04:11 --> 04:14a lot of intense time thinking about
04:14 --> 04:17not only the management but also
04:17 --> 04:20supporting these families through that.
04:20 --> 04:22But the relationships I've built with
04:23 --> 04:25even the children that we've lost,
04:25 --> 04:26the relationships
04:26 --> 04:28built with those parents
04:28 --> 04:29is just unbelievable.
04:29 --> 04:31And after losing the child,
04:31 --> 04:33they still think of
04:33 --> 04:36us as being part of their family.
04:36 --> 04:39And I think that bond is
04:39 --> 04:41so valuable and precious.
04:41 --> 04:44So yes, it can be heartbreaking at times,
04:44 --> 04:46but it's also extremely rewarding.
04:46 --> 04:48And today I would say that we cure
04:48 --> 04:50about 85%
04:50 --> 04:52of children with cancer very successfully.
04:52 --> 04:54So clearly we have done a really
04:54 --> 04:57good job at trying to make advances
04:57 --> 04:59and improve the life of these
04:59 --> 05:01children diagnosed with cancer.
05:03 --> 05:06And I think that's such a key point is that
05:06 --> 05:08whereas many people will
05:08 --> 05:11think of cancer as a death sentence,
05:11 --> 05:13now more and more what we're
05:13 --> 05:15finding out in a variety of cancers
05:15 --> 05:18is that really we're beginning to
05:18 --> 05:20discover that many of these cancers
05:20 --> 05:23are treatable and with good outcomes,
05:23 --> 05:25but you're interested in solid tumors,
05:25 --> 05:28so tell us more about the solid tumors
05:28 --> 05:32that occur in Pediatrics and what kind
05:32 --> 05:35of treatments we have to offer these kids.
05:35 --> 05:36What the prognosis is,
05:36 --> 05:38and the other thing that
05:38 --> 05:40I'm always curious about
05:40 --> 05:43on this show, we spend so much time
05:43 --> 05:45talking about personalized medicine.
05:45 --> 05:47The fact that now
05:47 --> 05:50we've begun to really unlock the
05:50 --> 05:52genomic abnormalities that
05:52 --> 05:54occur in cancers we're able to
05:54 --> 05:56better target these abnormalities.
05:56 --> 06:00Can we do the same thing in kids and
06:00 --> 06:04is that resulting in higher cure rates?
06:04 --> 06:08Great question and a lot to unpack.
06:08 --> 06:12In terms of solid tumors, they
06:15 --> 06:19really change across the age spectrum,
06:19 --> 06:21so the solid tumors that we see
06:21 --> 06:25in the much younger child are
06:25 --> 06:27tumors such as neuroblastoma,
06:27 --> 06:29Wilms tumors, retinoblastoma so
06:29 --> 06:32much more embryonal based tumors,
06:32 --> 06:34and then as you gradually
06:34 --> 06:37advance and you're coming to the
06:37 --> 06:38prepubertal young adolescence,
06:38 --> 06:41we start seeing more tumors
06:41 --> 06:44such as the sarcomas, so the osteosarcoma
06:44 --> 06:47the soft tissue sarcomas
06:47 --> 06:49which have an overlap
06:49 --> 06:52with the adult population as well,
06:52 --> 06:54and in addition, we see,
06:54 --> 06:55besides these sarcomas,
06:55 --> 06:58of course we see Rhabdomyosarcoma
06:58 --> 07:00which occurs across the age
07:00 --> 07:02spectrum from childhood onto the
07:02 --> 07:05adolescent young adult population.
07:05 --> 07:07So in terms of solid tumors,
07:07 --> 07:09really the main areas or the
07:09 --> 07:12main types of solid tumors we see
07:12 --> 07:14would be the embryonal tumors.
07:14 --> 07:16As I already mentioned and
07:16 --> 07:18then sort of the sarcomas
07:18 --> 07:21and the bone sarcomas.
07:21 --> 07:23Those are the two big groups
07:23 --> 07:25of solid tumors that we see.
07:25 --> 07:28We also see interestingly a lot of rare
07:28 --> 07:31tumors and one of my particular area
07:31 --> 07:34of interest has been in rare tumors,
07:34 --> 07:36and I've been very involved
07:36 --> 07:38in developing clinical trials
07:38 --> 07:40for these children with rare tumors
07:40 --> 07:42through the Children's oncology group,
07:42 --> 07:45so the rare tumors that we see are
07:45 --> 07:47things like nasopharyngeal carcinoma,
07:47 --> 07:49adrenocortical carcinoma, thyroid cancer,
07:49 --> 07:52which of course can occur in adults
07:52 --> 07:55but also starts in young adolescence.
07:55 --> 07:58So we see several of those patients,
07:58 --> 08:00and now we've started seeing some
08:00 --> 08:03of the tumors that are adult tumors
08:04 --> 08:06earlier in Pediatrics,
08:06 --> 08:08such as even colorectal carcinoma.
08:08 --> 08:11So that's sort of the spectrum of
08:11 --> 08:14tumors we see in pediatric solid tumors.
08:14 --> 08:17I've not included brain tumors because
08:17 --> 08:19we almost separate brain tumors,
08:19 --> 08:21just like we do leukemia and lymphomas.
08:21 --> 08:25And I don't treat brain tumors.
08:25 --> 08:28I focus on the extracranial solid tumors,
08:28 --> 08:31so those are the ones I've just mentioned
08:31 --> 08:33with regards to the treatment and
08:33 --> 08:36the role of personalized medicine or
08:36 --> 08:39immunotherapy in treating these cancers.
08:41 --> 08:43Again, the role of personalized medicine
08:44 --> 08:47is very well known in the adult oncologic world.
08:48 --> 08:51In Pediatrics we still do profile
08:51 --> 08:54most of our patients with solid tumors,
08:54 --> 08:56and there have been tumors
08:56 --> 08:58which have happened recently and
08:58 --> 09:00there's a lot of excitement on
09:00 --> 09:02tumors where there's a specific
09:02 --> 09:05targeted drug that is available,
09:05 --> 09:09and one classic example of this is the
09:09 --> 09:09TRK fusion cancers
09:09 --> 09:12where there is a specific drug
09:14 --> 09:17that has been developed with
09:17 --> 09:19excellent outstanding results.
09:19 --> 09:23So TRK fusion cancers can occur
09:23 --> 09:26from infants where you
09:26 --> 09:29have infantile fibrosarcoma's that occur in
09:29 --> 09:32the first year of life,
09:32 --> 09:34and then TRK Fusion
09:34 --> 09:36sarcomas are also seen in older
09:36 --> 09:39adolescents and young adults,
09:39 --> 09:40so in specific situations
09:40 --> 09:44we do also use what the adults
09:44 --> 09:46use much more frequently,
09:46 --> 09:49which is a very targeted therapy based on
09:49 --> 09:52tumor profiling.
09:55 --> 09:57How does prognosis vary
09:57 --> 09:58amongst the pediatric cancers?
09:58 --> 10:00Because you've kind of mentioned
10:00 --> 10:02this whole spectrum,
10:02 --> 10:05we have the leukemia lymphoma
10:05 --> 10:07as one separate group and brain
10:07 --> 10:09tumors as another separate group.
10:09 --> 10:12But even within the non cranial solid
10:12 --> 10:16tumors in pediatric populations
10:16 --> 10:19we're looking at everything from eye tumors,
10:19 --> 10:20retinoblastoma's to kidney
10:20 --> 10:23tumors like Wilms tumor
10:23 --> 10:24to sarcomas.
10:24 --> 10:29So how do these vary in terms of prognosis,
10:29 --> 10:33and have we seen a shift in terms of
10:34 --> 10:36moving towards being able to treat
10:36 --> 10:39these children better with new therapies?
10:41 --> 10:44Yeah, so it is a whole spectrum.
10:44 --> 10:47As you've already mentioned,
10:47 --> 10:50I think we've done really well in
10:50 --> 10:53some of these tumors.
10:53 --> 10:56For example, in patients with retinoblastoma
10:56 --> 10:58you have an excellent outcome,
10:58 --> 11:01particularly now with intra arterial
11:01 --> 11:03chemotherapy delivering very focused
11:03 --> 11:05chemotherapy.
11:05 --> 11:08We've also reduced the issue with long
11:08 --> 11:12term side effects giving systemic therapy.
11:14 --> 11:17Treatment has evolved
11:17 --> 11:20significantly over the last maybe 10-15
11:20 --> 11:22years with the development of
11:22 --> 11:25an antibody called dinutuximab
11:25 --> 11:27which focuses on the GD2
11:27 --> 11:31which is expressed by neuroblastoma cells.
11:31 --> 11:34So now we have this multi modality therapy
11:34 --> 11:37that we do in addition to chemotherapy,
11:37 --> 11:38surgery, and radiation.
11:38 --> 11:42We also have this immunotherapy that is done
11:42 --> 11:43in combination
11:43 --> 11:45particularly for those who have high
11:45 --> 11:48risk neuroblastoma in Wilms tumor,
11:48 --> 11:50our outcomes have always been excellent,
11:50 --> 11:52and we're continuing to improve
11:52 --> 11:53those outcomes.
11:53 --> 11:55And similarly I didn't
11:55 --> 11:57mention germ cell tumors,
11:57 --> 12:00which honestly, is
12:00 --> 12:02a really strong interest of mine,
12:02 --> 12:06so we do very well in germ cell tumors.
12:06 --> 12:08And in all these four categories,
12:08 --> 12:11I would say we have excellent outcomes.
12:11 --> 12:12In sarcomas,
12:12 --> 12:15I think we still have challenges.
12:15 --> 12:17And the challenge really depends on
12:17 --> 12:21the time of presentation,
12:21 --> 12:23what the staging is, and
12:23 --> 12:25for patients who present
12:25 --> 12:27with metastatic sarcomas,
12:27 --> 12:29be it Rhabdomyosarcoma or osteosarcoma,
12:29 --> 12:33we still are challenged in terms
12:33 --> 12:36of long term outcomes at times,
12:36 --> 12:39and we have numerous clinical trials
12:39 --> 12:43looking at different options which
12:46 --> 12:48this is where we are
12:48 --> 12:50looking to improve our outcomes
12:50 --> 12:51by newer therapies.
12:51 --> 12:54And as you mentioned, personalized therapies are
12:55 --> 12:57so important to really try to get
12:57 --> 13:00people involved in clinical trials
13:00 --> 13:02to really move those therapies forward,
13:02 --> 13:05but it's really great to hear that
13:05 --> 13:08we're moving in the right direction,
13:08 --> 13:11at least for the majority of solid tumors in kids.
13:11 --> 13:14We're going to take a short
13:14 --> 13:17break for medical minute and then learn
13:17 --> 13:19more not only about pediatric cancer,
13:19 --> 13:22but also delve into your interest in
13:22 --> 13:25sickle cell disease right after this break.
13:25 --> 13:27Please stay tuned for more
13:27 --> 13:29with my guest Doctor Farzana Pashankar.
13:29 --> 13:32Funding for Yale Cancer
13:32 --> 13:34Answers comes from AstraZeneca, working
13:34 --> 13:38to eliminate cancer as a cause of death.
13:38 --> 13:41Learn more at astrazeneca-us.com.
13:41 --> 13:44Genetic testing can be useful for
13:44 --> 13:46people with certain types of cancer
13:46 --> 13:48that seem to run in their families.
13:48 --> 13:51Genetic counseling is a process that
13:51 --> 13:53includes collecting a detailed personal
13:53 --> 13:55and family history or risk assessment and
13:55 --> 13:58a discussion of genetic testing options.
13:58 --> 14:01Only about 5 to 10% of all cancers
14:01 --> 14:02are inherited, and genetic testing
14:02 --> 14:04is not recommended for everyone.
14:04 --> 14:07Individuals who have a personal and
14:07 --> 14:08or family history that includes
14:08 --> 14:10cancer at unusually early ages,
14:10 --> 14:11multiple relatives
14:11 --> 14:14on the same side of the
14:14 --> 14:16family with the same cancer,
14:16 --> 14:18more than one diagnosis of
14:18 --> 14:20cancer in the same individual,
14:20 --> 14:23rare cancers or family history of a
14:23 --> 14:25known altered cancer predisposing gene
14:25 --> 14:27could be candidates for genetic testing.
14:27 --> 14:30Resources for genetic counseling and
14:30 --> 14:32testing are available at federally
14:32 --> 14:33designated comprehensive cancer
14:33 --> 14:35centers such as Yale Cancer Center
14:35 --> 14:37and at Smilow Cancer Hospital.
14:37 --> 14:40More information is available at
14:40 --> 14:41yalecancercenter.org. You're listening
14:41 --> 14:43to Connecticut Public Radio.
14:43 --> 14:43Welcome
14:43 --> 14:45back to Yale Cancer Answers.
14:45 --> 14:47This is doctor Anees Chagpar
14:47 --> 14:49and I'm joined tonight by my
14:49 --> 14:51guest Doctor Farzana Pashankar.
14:51 --> 14:53We're talking about sickle cell
14:53 --> 14:55disease and cancer in pediatric
14:55 --> 14:57patients. Before the break for
14:57 --> 14:59any of you who missed it,
14:59 --> 15:01there is no connection between sickle
15:01 --> 15:03cell disease and pediatric cancers,
15:03 --> 15:05except that our guest happens
15:05 --> 15:07to be an expert in both.
15:07 --> 15:10Right before the break we were
15:10 --> 15:13talking about pediatric cancers and the fact
15:13 --> 15:17that some kids get solid tumors.
15:17 --> 15:20This must not be very common, right?
15:20 --> 15:24How common are pediatric cancers?
15:24 --> 15:26Especially the non hematologic cancers?
15:28 --> 15:31I think of course each one of those
15:31 --> 15:33cancers is overall pretty rare
15:33 --> 15:36and even leukemias, which are the
15:36 --> 15:38most common pediatric cancer we say
15:38 --> 15:41happens one in a million.
15:41 --> 15:43So the solid tumors are much rarer
15:44 --> 15:47and each one has a different frequency,
15:47 --> 15:50so it's hard to give a
15:50 --> 15:53number for all of them combined.
15:55 --> 15:57This is very interesting
15:57 --> 15:59because as you may know,
15:59 --> 16:03there's a lot of interest in rare cancers,
16:03 --> 16:06and the NIH was looking at developing a
16:06 --> 16:10rare Cancer Institute in order to try and
16:10 --> 16:13improve the outcomes in these rare cancers.
16:13 --> 16:16And when we were looking at
16:16 --> 16:18defining what rare cancers is,
16:18 --> 16:21it's very clear up front that every
16:21 --> 16:24pediatric cancer is rare in that sense,
16:24 --> 16:26but the solid tumors,
16:26 --> 16:27particularly,
16:27 --> 16:30many of the tumors we discussed are
16:33 --> 16:35even much rarer than leukemia,
16:35 --> 16:38which is already
16:38 --> 16:38pretty uncommon.
16:39 --> 16:41And I'm sure that every parent
16:41 --> 16:43out there thinks that their
16:43 --> 16:45child is one in a million,
16:45 --> 16:46but really wouldn't want their
16:46 --> 16:49child to be one in a million in
16:49 --> 16:50this particular circumstance.
16:50 --> 16:53And one of the
16:53 --> 16:54questions that comes up and
16:54 --> 16:56you mentioned that you had an
16:56 --> 16:58interest in clinical trials,
16:58 --> 17:00especially in rare tumors,
17:00 --> 17:03is that so much of the data that
17:03 --> 17:06we get that leads to best practice
17:06 --> 17:09that dictates how we treat cancer
17:09 --> 17:10comes from clinical trials.
17:10 --> 17:13And when you have these tumors that
17:13 --> 17:17are so rare that are one in a million,
17:17 --> 17:19how on Earth do we get the data
17:19 --> 17:22to actually know what's best
17:22 --> 17:24practice to treat our children,
17:24 --> 17:27and for every parent going through this,
17:27 --> 17:29I mean that is their deepest anxiety.
17:31 --> 17:34That's a very good point
17:34 --> 17:38and I think what I must say is that in
17:38 --> 17:41pediatric oncology we have honestly and I
17:41 --> 17:44am not taking all the any credit for this,
17:44 --> 17:47but we have done an amazing job at
17:47 --> 17:50being able to conduct clinical trials
17:50 --> 17:53and the way we've done this is through
17:53 --> 17:55the development of a consortium
17:55 --> 17:57called the Children's Oncology Group,
17:57 --> 17:59which really has about 230
17:59 --> 18:01institutions across the United States,
18:01 --> 18:04Australia, New Zealand and Canada
18:04 --> 18:08and the beauty of this is that
18:08 --> 18:10as a group then we can,
18:10 --> 18:12because each individual
18:12 --> 18:14institution will only have
18:14 --> 18:17a patient very rarely with a
18:17 --> 18:18particular type of cancer,
18:18 --> 18:21we can bring all of us together,
18:21 --> 18:23and we can then get the numbers to
18:23 --> 18:26be able to conduct a clinical
18:26 --> 18:28trial and more importantly,
18:28 --> 18:30conduct some randomized clinical trials
18:30 --> 18:33to be able to answer the question of
18:33 --> 18:35which treatment is the best and most
18:35 --> 18:38appropriate for these rare cancers.
18:38 --> 18:40So the children's Oncology Group has
18:40 --> 18:43existed for a while and we
18:43 --> 18:45have designed clinical trials
18:45 --> 18:47on each type of pediatric cancer,
18:47 --> 18:49but more recently what is happening
18:49 --> 18:51that I am very
18:52 --> 18:55happy to be involved with is that we are now
18:55 --> 18:57looking at international collaborations.
18:57 --> 19:00So for example in germ cell tumors
19:00 --> 19:02because germ cell tumors are again so
19:02 --> 19:05rare even in the US and Canada and
19:05 --> 19:08Australia we cannot have the appropriate
19:08 --> 19:10numbers to do a randomized trial.
19:10 --> 19:13So currently we are conducting two trials,
19:13 --> 19:15one for low risk and
19:15 --> 19:16intermediate risk,
19:16 --> 19:18and one for high risk.
19:20 --> 19:22So we've collaborated with the
19:22 --> 19:24UK with India with Australia,
19:24 --> 19:26New Zealand and we are all
19:26 --> 19:27running the same trials,
19:27 --> 19:30so that again we can bring all this
19:30 --> 19:33information together and be able to
19:33 --> 19:35make advances for future patients.
19:35 --> 19:36I think that's so critical.
19:38 --> 19:40You know one of the issues that we
19:40 --> 19:43face in adult tumors, however, is,
19:43 --> 19:46although all of us know that clinical trials
19:46 --> 19:48are the drivers of improved care
19:48 --> 19:50it's how we make practice
19:50 --> 19:52changing discovery, is that still there is
19:52 --> 19:55a reluctance on the part of some patients
19:55 --> 19:57to participate in clinical trials.
19:57 --> 20:00So if you look across the board,
20:00 --> 20:02our rate of clinical trial
20:02 --> 20:05accrual is somewhere South of 5%,
20:05 --> 20:08and with children I mean I can imagine
20:08 --> 20:10that parents have obvious anxiety when
20:10 --> 20:14you talk about clinical trials,
20:14 --> 20:17but I understand that the rate
20:17 --> 20:18is much higher for accrual
20:18 --> 20:20to these clinical trials.
20:20 --> 20:22Honestly in Pediatrics,
20:22 --> 20:24the rate is significantly higher,
20:24 --> 20:27and I think part of the reason
20:27 --> 20:30at least at Yale,
20:30 --> 20:33of all the patients eligible for a trial,
20:33 --> 20:34because sometimes,
20:34 --> 20:37of course a trial may not be available
20:37 --> 20:40for that particular type of tumor.
20:40 --> 20:41But for any eligible patient,
20:41 --> 20:45we enroll up to 80% of the children
20:45 --> 20:47who are eligible for a trial.
20:47 --> 20:50When you're taking care of
20:50 --> 20:51your child, who has cancer
20:51 --> 20:54I think the motivation from the parents
20:54 --> 20:56is very different than maybe
20:56 --> 20:58the motivation for yourself.
20:58 --> 20:59I'm not sure,
20:59 --> 21:01but clearly we all do go
21:01 --> 21:04above and beyond for our kids.
21:04 --> 21:06Then we probably even do
21:06 --> 21:07for ourselves.
21:07 --> 21:09And I think that that desire
21:09 --> 21:12to figure out the best treatment,
21:12 --> 21:14especially when we're talking
21:14 --> 21:17about rare diseases is so important.
21:17 --> 21:20And I think the other piece is that
21:20 --> 21:22parents sometimes have trepidation
21:22 --> 21:25about what is the
21:25 --> 21:27right answer to treat my child,
21:27 --> 21:29especially when all of these cancers
21:29 --> 21:32are so rare and clinical trials
21:32 --> 21:35gives you some modicum of this
21:35 --> 21:37actually might be best practice because,
21:37 --> 21:38as you say,
21:38 --> 21:40all of these professionals get
21:40 --> 21:42together in designing these trials,
21:42 --> 21:46so they've put in that brain trust of,
21:46 --> 21:49you know this is potentially best
21:49 --> 21:51practice or best practice versus
21:51 --> 21:54what best practice will be and we want to see
21:54 --> 21:57which is best for patients who are
21:57 --> 21:59not candidates for a clinical trial
21:59 --> 22:02where there still may be
22:02 --> 22:05questions about what is best practice.
22:05 --> 22:08How do you reassure patients and parents
22:08 --> 22:11that this really is
22:11 --> 22:13the way to go?
22:14 --> 22:16Are there still collaborations where you
22:16 --> 22:19get together with a consensus,
22:19 --> 22:21either nationally or internationally,
22:21 --> 22:24to figure out what might be best
22:24 --> 22:26practice for these patients?
22:26 --> 22:29Absolutely. I think one thing
22:29 --> 22:32is that the best practice is obviously
22:32 --> 22:35the standard of care in many cases.
22:35 --> 22:38But in many cases there is
22:38 --> 22:40no proper standard of care,
22:40 --> 22:43but the beauty again of having these
22:43 --> 22:45close collaborations working together
22:45 --> 22:48on trials means that we have a
22:48 --> 22:50really great phenomenal community of
22:50 --> 22:52oncologists that you can call upon to
22:52 --> 22:54discuss and get guidance on in
22:54 --> 22:56really rare cases.
22:56 --> 22:59So I think that is a really
22:59 --> 23:02fulfilling part of being able to
23:02 --> 23:05connect with friends and colleagues across
23:05 --> 23:07the country, across the world to be
23:07 --> 23:09able to discuss some difficult cases.
23:09 --> 23:12What is really fun is
23:12 --> 23:15we've now developed these virtual
23:15 --> 23:16International tumor boards
23:16 --> 23:19for some of these really rare cancers,
23:19 --> 23:21so we have an international tumor board
23:21 --> 23:22for patients with hepatoblastoma,
23:22 --> 23:24where experts from across the
23:24 --> 23:27country meet once a month and you
23:27 --> 23:29can put in a case and they will
23:29 --> 23:31review everything and discuss it,
23:31 --> 23:34just like we do at a local tumor board.
23:34 --> 23:35Similarly,
23:35 --> 23:37we have a rare tumor board
23:37 --> 23:39which is across the country,
23:39 --> 23:40so again,
23:40 --> 23:43people do go above and beyond to try and
23:43 --> 23:46put in their time and effort to bring their
23:46 --> 23:48thoughts and their experience to help
23:48 --> 23:50kids across the country and across
23:50 --> 23:53the world.
23:53 --> 23:56I love the fact that there is such humility
23:56 --> 23:59among pediatric oncologists to
23:59 --> 24:01really collaborate with each other and to
24:01 --> 24:04figure out what's the best for this child.
24:04 --> 24:06Which is so important and so
24:06 --> 24:09heartening for parents going through this.
24:09 --> 24:12Now I did promise that we'd
24:12 --> 24:14spend at least a few minutes
24:14 --> 24:17talking about your other passion,
24:17 --> 24:19which is sickle cell disease and
24:19 --> 24:22sickle cell disease is still rare,
24:22 --> 24:23but presumably less rare
24:23 --> 24:24than pediatric cancers.
24:24 --> 24:27Is that right?
24:27 --> 24:30I think it is rarer than pediatric cancers,
24:30 --> 24:33and in the US now with
24:33 --> 24:35also a changing demographic,
24:35 --> 24:38we have patients of many
24:38 --> 24:40different ethnicities who can
24:40 --> 24:44also have sickle cell disease so
24:44 --> 24:47it's definitely something that we
24:47 --> 24:50in Connecticut see 24 to 26 new
24:50 --> 24:53diagnoses of sickle cell disease
24:53 --> 24:56each year and about 600 new patients
24:57 --> 25:00with sickle cell trait per year.
25:07 --> 25:09Talk a little
25:09 --> 25:11bit about sickle cell disease and
25:11 --> 25:14the problems that people can run into.
25:14 --> 25:16I mean, when people think about cancer,
25:16 --> 25:19you really don't need to say anything
25:19 --> 25:21more than cancer for it to strike
25:21 --> 25:23the fear of God into some people.
25:23 --> 25:26But what problems do people with
25:26 --> 25:28sickle cell disease run into that
25:28 --> 25:30are problematic and talk a
25:30 --> 25:32little bit about some of the new
25:32 --> 25:34therapies that are out now?
25:37 --> 25:39So sickle cell disease
25:39 --> 25:42interestingly, is the first single
25:42 --> 25:44gene disorder that was described
25:44 --> 25:48over 120 years ago.
25:49 --> 25:52It is a lifelong chronic disease that
25:52 --> 25:54obviously you inherit from your parents
25:54 --> 25:58and the hallmarks of sickle cell disease
25:58 --> 26:01are these painful crises,
26:01 --> 26:04which really mean that patients
26:04 --> 26:05with sickle cell disease
26:05 --> 26:09can come into the hospital or have pain
26:09 --> 26:12at home several times a year.
26:12 --> 26:15These chronic VS occlusive crises can
26:15 --> 26:17also lead to multiple complications,
26:17 --> 26:20including stroke
26:20 --> 26:23and acute chest syndrome.
26:28 --> 26:32You can also have a lot of long term chronic
26:32 --> 26:35morbidity because of this ongoing
26:35 --> 26:37microvascular occlusion that happens in
26:37 --> 26:38all your organ systems.
26:38 --> 26:41So patients with sickle cell disease can
26:41 --> 26:44have long term problems with their kidneys,
26:44 --> 26:46leading to sickle nephropathy.
26:46 --> 26:48They can have problems with their
26:48 --> 26:51liver leading to sickle hepatopathy.
26:51 --> 26:53They can have sickle retinopathy,
26:53 --> 26:55so it's a disease which has
26:55 --> 26:57acute complications which brings
26:57 --> 26:59someone to the hospital.
26:59 --> 27:02But also has ongoing long term chronic
27:02 --> 27:05disease burden which continues to affect
27:05 --> 27:09pretty much every organ system in their body.
27:09 --> 27:13So it is a disease
27:13 --> 27:17where you have to pay attention to
27:17 --> 27:20obviously the acute management during pain,
27:20 --> 27:21crisis, stroke,
27:21 --> 27:23acute chest syndrome, etc.
27:23 --> 27:26but you also have to take care of these
27:26 --> 27:30adults and children for preventative care.
27:30 --> 27:32To make sure that you are monitoring
27:32 --> 27:35for these long term complications and
27:35 --> 27:38you are intervening when feasible.
27:38 --> 27:41But the good part about sickle
27:41 --> 27:44cell disease or the exciting part
27:44 --> 27:48currently is that we have a lot of new
27:48 --> 27:50therapies which have come about in
27:50 --> 27:53order to improve not only the pain crises,
27:54 --> 27:56the FDA has now approved several
27:56 --> 27:58new drugs besides hydroxyurea,
27:58 --> 28:00which was the only drug available for
28:00 --> 28:03a long time to
28:03 --> 28:06modify sickle cell disease and the
28:06 --> 28:08most exciting thing really is the
28:08 --> 28:10advent of bone marrow transplant,
28:10 --> 28:12which is currently the only curative
28:12 --> 28:14option for sickle cell disease but
28:14 --> 28:17also gene therapy and many of you
28:17 --> 28:19might have seen data on gene therapy,
28:20 --> 28:22some case reports of gene therapy for
28:22 --> 28:24sickle cell disease which is exciting
28:24 --> 28:27and we are looking forward to that
28:27 --> 28:29becoming more streamlined in the next
28:29 --> 28:31few years.
28:31 --> 28:33Dr. Pashankar is an associate professor of
28:33 --> 28:34Pediatrics in hematology oncology
28:34 --> 28:37at the Yale School of Medicine.
28:37 --> 28:38If you have questions
28:38 --> 28:40the address is canceranswers@yale.edu
28:40 --> 28:42and past editions of the program
28:42 --> 28:44are available in audio and written
28:44 --> 28:45form at yalecancercenter.org.
28:45 --> 28:48We hope you'll join us next week to
28:48 --> 28:50learn more about the fight against
28:50 --> 28:52cancer here on Connecticut Public
28:52 --> 28:55radio. Funding for Yale Cancer
28:55 --> 28:57Answers is provided by Smilow
28:57 --> 29:00Cancer Hospital and AstraZeneca.

Information

Care of Sickle Cell Disease and Cancer Patients with guest Dr. Farzana Pashankar July 25, 2021 Yale Cancer Center visit: http://www.yalecancercenter.org email: canceranswers@yale.edu call: 203-785-4095