Latest / Get Real with Jim Weiss / A Mother’s Mission to Cure Rett Syndrome with Monica Coenraads

Transcript

A Mother’s Mission to Cure Rett Syndrome with Monica Coenraads · Get Real with Jim Weiss

0:03
Speaker 1: Welcome to get real. There's much to learn about ret
0:05
Speaker 1: syndrome and the Research Trust that you really got going here.
0:10
Speaker 1: I like to start with the origin stories anyway, and
0:13
Speaker 1: what got you here today. Before founding ret Syndrome Research Trust,
0:18
Speaker 1: you worked in the family business operating restaurants. What did
0:22
Speaker 1: that teach you about the grit required to be a founder?
0:25
Speaker 1: I often say that that's one of the best places
0:28
Speaker 1: to get that grit at a restaurant.
0:30
Speaker 2: The restaurant business is not easy. So my dad, who
0:34
Speaker 2: was an engineer, decided later in life to buy a restaurant.
0:39
Speaker 2: My mom, my brother, and I really tried to talk
0:42
Speaker 2: him out of it. We did not think it was
0:43
Speaker 2: a good idea, and he knew nothing about running restaurants.
0:46
Speaker 2: But he did it anyway, and then he got sick.
0:50
Speaker 2: He needed a liver transplant. He was in a hospital
0:53
Speaker 2: away from home for about a year. I had just
0:55
Speaker 2: gotten an MBA, my brother had just gotten his bachelor's
0:59
Speaker 2: and we quit our jobs to kind of work at
1:02
Speaker 2: the restaurant, kind of rescue this restaurant that had nobody
1:05
Speaker 2: to run it. We ended up spending ten years there
1:08
Speaker 2: and we built it up over the years. It taught
1:10
Speaker 2: me a lot. It taught me. I think probably the
1:13
Speaker 2: most important thing is just self confidence. It taught me
1:17
Speaker 2: how to manage people, how to manage customers, employees, scheduling, accounting, payroll,
1:23
Speaker 2: you know, you name it. I had my hands and everything.
1:25
Speaker 2: It was a small restaurant and you had to be
1:27
Speaker 2: an owner operator.
1:28
Speaker 3: Where was it in Stamford?
1:30
Speaker 2: Right in Stamford, Connecticut?
1:32
Speaker 3: Yep? Is that where you are? Now? Where are you
1:34
Speaker 3: based now?
1:35
Speaker 2: I'm about half an hour from there. I'm in Trumble, Connecticut.
1:38
Speaker 1: All right, so you're still in the same zone as
1:40
Speaker 1: the restaurant still going or how did that end?
1:43
Speaker 2: So we sold it and the people that bought it
1:47
Speaker 2: ran it for a couple of years and then didn't
1:49
Speaker 2: do so well. And then it's a Peruvian restaurant.
1:52
Speaker 3: Now interesting, what was your food like?
1:54
Speaker 2: It was a pasta restaurant. It was a diner when
1:57
Speaker 2: we bought it, and then we changed. We made all
1:59
Speaker 2: our her own fresh pasta wow from scratch, and tons
2:04
Speaker 2: of specials every day. And yeah, it was a hopping place.
2:07
Speaker 3: Oh that's cool.
2:08
Speaker 2: My brother's still in the restaurant business. He owns a
2:11
Speaker 2: restaurant in Newtown. What's that called it's called sal e
2:15
Speaker 2: peppe salt and pepper.
2:16
Speaker 3: Oh, it sounds great.
2:17
Speaker 1: I got to hit that and is that how you
2:19
Speaker 1: know Barbara Palmer our mutual friend from that part of
2:23
Speaker 1: the world.
2:23
Speaker 2: There now no I know Barbara. We have a mutual friend,
2:27
Speaker 2: Rachel and Jason Rothschild. They're very close with Rachel. Rachel
2:31
Speaker 2: has a daughter with Reht syndrome. She used to live
2:33
Speaker 2: in Stamford. I met her through the diagnosis of Bret syndrome.
2:37
Speaker 1: And your daughter, Chelsea has rhet syndrome. What exactly is
2:41
Speaker 1: rhet syndrome and when was it identified?
2:44
Speaker 2: So rehet syndrome was initially well, it's been around forever,
2:48
Speaker 2: of course, right before people knew it was REHD syndrome.
2:51
Speaker 2: The first paper that came out about Reht syndrome was
2:54
Speaker 2: written in the sixties in a very obscure medical journal
2:58
Speaker 2: in German and not too many people read it. And
3:01
Speaker 2: then in the eighties there was a paper written from
3:05
Speaker 2: another neurologist and that's when it started to become better
3:09
Speaker 2: known and people started getting diagnosed with rhet syndrome based
3:12
Speaker 2: on a clinical presentation. So what your clinical history was
3:17
Speaker 2: and the symptoms that you presented we didn't know the cause.
3:20
Speaker 2: And then in nineteen ninety nine the cause was identified,
3:24
Speaker 2: so mutations in a particular gene and that was discovered
3:28
Speaker 2: by a physician scientist in Houston called Huda Zogby, and
3:32
Speaker 2: then we knew what the cause was. We had something
3:35
Speaker 2: to sink our teeth into, and I think the research
3:38
Speaker 2: really took off from there. From nineteen ninety nine.
3:40
Speaker 3: Cow old Chelsea, I mean, you know, when did you get.
3:44
Speaker 2: Chelsea's turning thirty this year?
3:46
Speaker 3: Oh wow, hard to believe.
3:47
Speaker 2: Yeah, she was diagnosed clinically at the age of two,
3:51
Speaker 2: and then a year later, in ninety nine, the gene
3:54
Speaker 2: mutations were identified and she was one of the first
3:58
Speaker 2: kids to be tested and tested positive for a mutation
4:02
Speaker 2: in this gene. This is a gene that all of
4:04
Speaker 2: us have, but if you have mutations in it, it
4:07
Speaker 2: leads to red syndrome.
4:08
Speaker 1: Take us through the whole What was it like, you know,
4:11
Speaker 1: your two year old kids, I mean, and then learning
4:13
Speaker 1: about this.
4:14
Speaker 2: We had the restaurant for ten years and I was
4:17
Speaker 2: working crazy hours, one hundred hours a week and it
4:19
Speaker 2: was very physical, and we put off having kids until
4:22
Speaker 2: we sold the restaurant. Got pregnant right away and had
4:25
Speaker 2: a great pregnancy, and she was the first grandchild on
4:27
Speaker 2: both sides of the family, so everybody was ecstatic and
4:31
Speaker 2: waiting impatiently for the birth. First year was pretty normal,
4:35
Speaker 2: and then around a year I started becoming concerned. She
4:39
Speaker 2: wasn't learning new things. She wasn't pulling up to standing,
4:43
Speaker 2: she was not crawling. She had a funny way of crawling.
4:48
Speaker 2: She seemed disinterested in her environment. Things that she had
4:52
Speaker 2: learned how to do, like wait, bye bye, play patty Cake,
4:55
Speaker 2: she stopped doing. She started crossing her eyes. You know,
4:58
Speaker 2: she just semed very autistic, stop smiling, stopped looking at us.
5:03
Speaker 2: First we go to the pediatrician, and then we went
5:05
Speaker 2: to a developmental pediatrician, and then we went to a geneticist,
5:08
Speaker 2: and then we went to a neurologist. And every test
5:11
Speaker 2: we did came back normal. Through serendipity, which you know
5:15
Speaker 2: often happens in life. I belonged to what back then
5:21
Speaker 2: in ninety eight ninety nine was called a LISTSERP. So
5:23
Speaker 2: I belonged to this email list of a bunch of
5:25
Speaker 2: other moms who we had all had kids October of
5:28
Speaker 2: nineteen ninety six, and we were communicating with each other
5:32
Speaker 2: and not my kids doing this, and my kid's doing that,
5:34
Speaker 2: and you know, I had the kid that wasn't learning stuff.
5:37
Speaker 2: One of the moms reached out to me and said,
5:40
Speaker 2: my next door neighbor has a daughter with rehet syndrome.
5:43
Speaker 3: Look it up. So I did.
5:44
Speaker 2: I looked it up and started doing this mental you know,
5:49
Speaker 2: checking off. Yep, she does this, she does that. Oh
5:51
Speaker 2: my god, you know, I think this might be it.
5:54
Speaker 2: Every doctor I spoke to saying I think Chelsea has
5:57
Speaker 2: red syndrome, They're like, oh, it's so rare. I don't
5:59
Speaker 2: think so. I don't think so. But at the time
6:01
Speaker 2: we were living in Virginia. In Baltimore, there was a
6:05
Speaker 2: rehet syndrome clinic so at Kennedy Johns Hopkins. So we
6:10
Speaker 2: took her there. Wow, and they weren't used to seeing
6:12
Speaker 2: kids that young at the time. She was like fifteen
6:16
Speaker 2: sixteen months old, and we had to go back a
6:18
Speaker 2: couple of times, and then they gave her a diagnosis
6:22
Speaker 2: of rhet and then a few months later the genetic
6:25
Speaker 2: cause was identified and we were able to do a
6:28
Speaker 2: blood test. At that point, her blood was in all
6:31
Speaker 2: the labs of the doctors that were trying to find
6:33
Speaker 2: the cause for ret syndrome. They got back to me immediately,
6:36
Speaker 2: and so it was devastating, of course, to kind of
6:42
Speaker 2: slowly realize over time, right over a matter of say,
6:47
Speaker 2: six months or so, that our daughter was going to
6:49
Speaker 2: be severely disabled. And then I tried to turn that
6:55
Speaker 2: those emotions and really feeling helpless, tried to say, right,
6:58
Speaker 2: what can I do about it? At first it was
7:00
Speaker 2: like why us?
7:01
Speaker 3: Why me?
7:01
Speaker 2: Why her? And then it was like, okay, why not us,
7:04
Speaker 2: Let's try to do something about it, And That's what
7:07
Speaker 2: I've been doing ever since.
7:09
Speaker 1: And there really wasn't anything out there. You broke the ground.
7:13
Speaker 1: Chelsea really broke a lot.
7:14
Speaker 3: Of the ground.
7:15
Speaker 2: Up until that point, the effort had been to try
7:18
Speaker 2: to find the genetic cause of red syndrome, and fortunately
7:21
Speaker 2: that happened shortly after Chelsea was diagnosed clinically, so it
7:25
Speaker 2: was a great time to get involved. So yes, I mean,
7:28
Speaker 2: there was nobody trying to develop a treatment or genetic medicine.
7:33
Speaker 2: Biotechs weren't interested, they'd never heard of it. It was
7:36
Speaker 2: an uphill back.
7:37
Speaker 1: Well, you just had the blood test, you know, there'd
7:39
Speaker 1: be no way yet that anyone was going to jump
7:42
Speaker 1: on that, and then they really didn't. And we can
7:44
Speaker 1: get into that in a minute. But her primary caretaker.
7:47
Speaker 1: I'm also familiar with caretaking, but nothing like that. I mean,
7:51
Speaker 1: I had a wife with cancer and I've talked a
7:53
Speaker 1: lot about it.
7:54
Speaker 3: Let's hear what it's like caring for I.
7:57
Speaker 1: I mean, I also have friends who have kids with
7:59
Speaker 1: rare asses and IQ stories and we've had some people
8:03
Speaker 1: on the show here, what do you have to do?
8:05
Speaker 1: What are some of the things did you have to
8:07
Speaker 1: like build a new house or things in the house
8:10
Speaker 1: or all the things?
8:11
Speaker 3: I mean, I'm sure, yes.
8:12
Speaker 2: We had to make some modifications to the house. So,
8:17
Speaker 2: first of all, Chelsea is nonverbal. Okay, she does not walk.
8:22
Speaker 2: She could walk when she was younger with assistance. She
8:25
Speaker 2: never had balance, but you could hold her hands and
8:28
Speaker 2: she could take some steps. Over the years, she's lost
8:31
Speaker 2: that ability, so she can't stand. She can't help you
8:34
Speaker 2: transfer weight or anything like that. So completely unable to
8:38
Speaker 2: stand or assist in any kind of transfer. She cannot
8:41
Speaker 2: use her hands at all, so she can't hold anything,
8:44
Speaker 2: she can't reach out for anything, she can't point. She
8:48
Speaker 2: is fed through a feeding tube. For many years, she
8:51
Speaker 2: had a feeding tube that fed into her stomach. We
8:53
Speaker 2: had to recently transition to a tube that feeds into
8:56
Speaker 2: her intestine because she was having lots of reflex She
9:00
Speaker 2: has really bad seizures. They started at five. She's so
9:04
Speaker 2: she's been having seizures for twenty five years. We've tried
9:06
Speaker 2: medications and the ketogenic diet we've we've not been able
9:09
Speaker 2: to get them under control. She has orthopedic issues, scoliosis
9:14
Speaker 2: and contractors and trouble sleeping and a host of other things.
9:19
Speaker 2: But a point I want to stress is that she's
9:21
Speaker 2: so much more laundry list of symptoms. She's got the
9:24
Speaker 2: most amazing personality, even though communication is difficult. She's always smiling,
9:30
Speaker 2: she's patient, she's loving, she's affection that she's got a
9:34
Speaker 2: sense of humor, and we get her out every day.
9:36
Speaker 2: Wherever I go, she goes. And I do have a
9:39
Speaker 2: caregiver that comes nine to five during the week so
9:42
Speaker 2: that I can work. She comes to the house. Chelsea
9:44
Speaker 2: lives at home with us. You asked about modifications to
9:47
Speaker 2: the house. We turned our living room, which we really
9:51
Speaker 2: never used, was really large. We turned it into a
9:53
Speaker 2: bedroom and bathroom for her. On the first floor. We
9:56
Speaker 2: have a ramp in the garage and.
10:00
Speaker 3: We have a wheelchair Vand Okay, it's going to say
10:02
Speaker 3: how you get her around? Yeah, so I guess that's it. Yeah.
10:05
Speaker 1: I mean you say she doesn't verbalize, but you're talking
10:08
Speaker 1: about all these wonderful traits.
10:10
Speaker 3: How does she express all that.
10:12
Speaker 2: She's very engaged, she has really good eye contact. You
10:16
Speaker 2: call her name, she looks at you right away.
10:18
Speaker 3: So she's really there.
10:19
Speaker 1: I mean, it's not like you've lost her, right, I mean,
10:22
Speaker 1: it's not that kind.
10:23
Speaker 3: Of grief right now.
10:24
Speaker 2: She's there.
10:25
Speaker 3: I'm sure there's grief, but it's different.
10:27
Speaker 2: The grief changes over time. In the early days was
10:31
Speaker 2: like you'd been kicked in the stomach, like it physically hurts.
10:35
Speaker 2: A period of I don't know, two or three weeks
10:37
Speaker 2: when I first realized how severe things were going to be,
10:40
Speaker 2: where it was difficult to get.
10:41
Speaker 3: Up in the morning.
10:42
Speaker 2: You'd wake up, you'd be okay for two seconds, then
10:45
Speaker 2: you'd remember, and then it was like a crushing feeling
10:49
Speaker 2: on your chest, like just crushing. You know, you can't
10:52
Speaker 2: exist and keep going with that kind of emotion. So
10:55
Speaker 2: it does get better, but it still hits you. You
11:00
Speaker 2: heard school friends that she's had over the years, going
11:05
Speaker 2: to college and getting married and having careers and her
11:09
Speaker 2: brothers moving on with their lives. It's always a little bittersweet.
11:12
Speaker 3: Yeah, and they don't have this any trace of it nothing.
11:17
Speaker 2: Now. For the majority of cases, it does not run
11:20
Speaker 2: in families. It's a sporadic mutation that happens, and the
11:24
Speaker 2: vast majority of families have never heard of red syndrome
11:26
Speaker 2: and it doesn't run in their family. There's a few rare,
11:31
Speaker 2: unusual cases of familial red syndrome, and then if Chelsea
11:35
Speaker 2: was to have a baby, she'd have a fifty percent
11:37
Speaker 2: chance of passing it on. But kids with red syndrome
11:40
Speaker 2: don't have kids, so it stops there. If we cured
11:43
Speaker 2: it and these kids would go on to have children,
11:46
Speaker 2: you could do pore implantation testing to make sure that
11:51
Speaker 2: you're in you do in vitro when you inplant an
11:53
Speaker 2: embryo that doesn't have the mutation, so there'd be a
11:56
Speaker 2: way around it.
11:57
Speaker 3: Right.
11:57
Speaker 1: So you've channeled that feeling I feel it just talking
12:02
Speaker 1: to you about it, you know, I just feel it.
12:04
Speaker 1: You've channeled that into launching and building an organization that
12:08
Speaker 1: is designed to tackle clinical trials and treatments.
12:11
Speaker 3: So how has it gone? Where are we I mean,
12:14
Speaker 3: and what's the journey been like?
12:16
Speaker 2: Yeah, so in the beginning it was really focused on
12:20
Speaker 2: basic science. Right, we didn't know much about this gene.
12:22
Speaker 2: What does it do? Where is it in the body,
12:25
Speaker 2: what does the protein do, how do the mutations affect
12:27
Speaker 2: the protein?
12:28
Speaker 1: And how did it even get triggered? She was fine
12:31
Speaker 1: for right, or we weren't sure.
12:33
Speaker 2: Well, yes, that's because when a baby's born there's very
12:37
Speaker 2: low levels of this protein. It's not really needed. As
12:40
Speaker 2: a baby matures and becomes a toddler, the amount of
12:43
Speaker 2: protein in the brain gets just naturally. You get more
12:47
Speaker 2: and more of it as it's needed and it's involved
12:50
Speaker 2: for more, it's involved with you know, learning and more
12:53
Speaker 2: advanced skills. And when it's needed and it's mutated and
12:57
Speaker 2: not doing its job, then the problems show up. But
13:00
Speaker 2: so in the beginning, I would say, the first you know,
13:03
Speaker 2: seven or eight years, we tried to get academic researchers
13:09
Speaker 2: interested in the disorder. So we were raising money and
13:12
Speaker 2: we were giving out smaller grants, say fifty thousand dollars
13:16
Speaker 2: a year for two or three years, to lots of
13:19
Speaker 2: different labs trying.
13:20
Speaker 3: Was that at Johns Hopkins, mostly because you said they
13:23
Speaker 3: had a clinic.
13:24
Speaker 2: No, so we weren't really funding clinicians. We were funding
13:28
Speaker 2: basic scientists, molecular geneticists, neurobiologists, and so it was more
13:33
Speaker 2: PhDs not mds in the beginning, just trying to understand
13:38
Speaker 2: the function, what does this protein do and where does
13:42
Speaker 2: where does it live? And we did that for a
13:44
Speaker 2: number of years and then there was a landmark paper
13:49
Speaker 2: that we funded that really kind of changed everything. And
13:53
Speaker 2: this was a paper that was came out of the
13:55
Speaker 2: lab of Adrian Bird at the University of Edinburgh and
13:59
Speaker 2: Skull and he did an experiment where he genetically engineered
14:06
Speaker 2: these mice models so that they were missing the gene
14:10
Speaker 2: that's mutated in red syndrome, and they developed red symptoms
14:14
Speaker 2: as much as a mouse can have red symptoms. But
14:16
Speaker 2: then they were able to modify this gene that was kept.
14:22
Speaker 2: It's not that it didn't have the gene, but the
14:24
Speaker 2: gene was modified so that it wouldn't make the protein.
14:27
Speaker 2: But then at will by doing something else we don't
14:30
Speaker 2: have to get into the details, you can turn the
14:33
Speaker 2: gene back on and then the gene starts to make
14:36
Speaker 2: its protein. And the question he was trying to answer
14:39
Speaker 2: is if we can restore levels of the protein, so
14:42
Speaker 2: genes make proteins. If you have a mutated gene, you're
14:45
Speaker 2: going to have a mutated protein. So the question was,
14:48
Speaker 2: if we turn this gene back on and it starts
14:51
Speaker 2: to make normal protein, what happens to the symptoms? Is
14:54
Speaker 2: it reversible? Are they reversible? Is there a window of
14:57
Speaker 2: opportunity within which you have to act, or can you
15:01
Speaker 2: take an old mouse that's never had the protein, turn
15:04
Speaker 2: it back on, turn the gene back on, it makes
15:06
Speaker 2: the protein and the symptoms go away. So that's what
15:09
Speaker 2: he was trying to answer, and the unexpected answer was
15:12
Speaker 2: that the symptoms are all reversible, complete, almost completely reversible,
15:17
Speaker 2: and the age didn't matter. You could do it in
15:20
Speaker 2: a young mouse, an old mouse. The symptoms go away.
15:24
Speaker 2: And the other thing he did was you can take
15:26
Speaker 2: a normal adult mouse and take the protein away and
15:30
Speaker 2: that mouse develops red symptoms.
15:32
Speaker 1: And you can't synthetically you know, because obviously a lot
15:35
Speaker 1: of our world is based on protein drugs that replace
15:40
Speaker 1: or replenish that which you.
15:42
Speaker 3: Don't produce or you lose.
15:44
Speaker 1: Is that one of the approaches or do you have
15:46
Speaker 1: to turn the gene back on.
15:48
Speaker 2: We are advancing a number of genetic medicines. So after
15:52
Speaker 2: that paper came out, it really changed the focus of
15:56
Speaker 2: what I wanted to do. At that point, I was like, right,
15:58
Speaker 2: we're going full on genetic mass medicines, not small molecule drugs,
16:03
Speaker 2: because this is a complicated gene that does a lot
16:05
Speaker 2: of things, and a drug may act on one pathway,
16:08
Speaker 2: but you know you're leaving a bunch of other pathways untouched.
16:11
Speaker 2: So let's go after the root cause of the disease.
16:14
Speaker 2: The one genetic medicine modality that everybody's familiar with is
16:18
Speaker 2: gene therapy, right, You're basically supplementing a gene to compensate
16:23
Speaker 2: for a mutated gene. So we started funding gene therapy,
16:26
Speaker 2: and then as new technologies were discovered, like for example,
16:30
Speaker 2: base editing you can now fix a particular kind of
16:33
Speaker 2: mutation or at RNA transplacing. As these new technologies became available,
16:38
Speaker 2: we added them to our portfolio. So we're now advancing
16:42
Speaker 2: a number of modalities, some of which should work for
16:46
Speaker 2: all mutations. Some of the modalities work for only some
16:49
Speaker 2: of the mutations. We're constantly, you know, looking at the
16:53
Speaker 2: science and making decisions about what do we advance, what
16:56
Speaker 2: do we prune, what new things are there. And there's
16:59
Speaker 2: two gene therapy trials now that are underway. These were
17:04
Speaker 2: gene therapy programs that started at the ret Syndrome Research Trust.
17:07
Speaker 2: We funded these investigators for years and years and years.
17:11
Speaker 2: They generated the data that got these companies intrigued. They
17:15
Speaker 2: licensed the programs, and now they're advancing them. So we
17:18
Speaker 2: have two gene therapy trials ongoing, and then we're advancing
17:21
Speaker 2: a number of other modalities.
17:23
Speaker 3: How do you raise money?
17:23
Speaker 1: How did you get people interested to help you raise
17:26
Speaker 1: the money you needed to get all this gone? How
17:28
Speaker 1: much does it cost? I know what it takes to
17:31
Speaker 1: bring a drug to market. It's like crazy.
17:34
Speaker 2: It is crazy. We don't have to raise all the money. Well,
17:37
Speaker 2: first of all, to answer your first question, our money
17:39
Speaker 2: comes from efforts of families like mine that have a
17:43
Speaker 2: child with red syndrome.
17:45
Speaker 3: How many is that approximately? Do you think.
17:48
Speaker 2: Fifteen thousand in the US? Only a small number actually fundraised. Unfortunately,
17:53
Speaker 2: that's the case for free disease. It's always a small
17:55
Speaker 2: subset of people. So those families donate personally and importantly
18:00
Speaker 2: reach out to their networks. So we've been raising somewhere
18:04
Speaker 2: between seven and nine million dollars a year. That's incredible,
18:07
Speaker 2: and we've raised I think the latest number was like
18:10
Speaker 2: one hundred and twenty million since I started. And we
18:12
Speaker 2: don't have to raise all the money to get something commercialized.
18:16
Speaker 2: We have to raise enough to generate the preliminary data
18:20
Speaker 2: to then get a biotech company interested and then they
18:24
Speaker 2: move it ahead with their own money.
18:26
Speaker 3: So we've got some biotechs involved. Now.
18:28
Speaker 2: Yeah, there's actually quite a few involved. Two gene therapy
18:32
Speaker 2: companies that have clinical trials. There's other companies that are
18:37
Speaker 2: in the gene therapy space that haven't yet gone public
18:41
Speaker 2: with their programs. There's RNA editing companies, so there we're
18:46
Speaker 2: not acting on the gene but we're actually trying to
18:48
Speaker 2: fix the RNA. There's a company that we're funding in
18:51
Speaker 2: the Netherlands called Procure, and there's a couple of other
18:55
Speaker 2: companies here in the US. The difference is if you
18:57
Speaker 2: have a common disease like Alzheimer or something that's very
19:01
Speaker 2: well known, companies are going to pursue those on their
19:04
Speaker 2: own for a rare disease companies are not going to
19:08
Speaker 2: start from scratch their programs for rare diseases, so they
19:13
Speaker 2: really have to count on an organization like ours to
19:16
Speaker 2: generate the preliminary data, get that data going, get those
19:20
Speaker 2: programs going, and then put it on a silver platter
19:23
Speaker 2: and say here are you interested? But they're not going
19:25
Speaker 2: to start from scratch. So that's the difference. No.
19:28
Speaker 1: I know, We've had Kathy Juicy on to talk about
19:30
Speaker 1: multiple Maloma Foundation, and I've been involved with the Leukemian
19:34
Speaker 1: and Foma Foundation, and back in the day when I
19:37
Speaker 1: was at genen Tech with a lot of the growth
19:39
Speaker 1: hormone and growth disorders as well as I mean, there's
19:43
Speaker 1: the list goes on and on. As you know, do
19:46
Speaker 1: you ever feel like a hero or I mean, it's
19:49
Speaker 1: kind of amazing what you're doing now?
19:52
Speaker 2: Quite the opposite. I'm reminded every single day that despite
19:58
Speaker 2: the funds raised and all the efforts, and I'm a workaholic,
20:02
Speaker 2: so a lot of work over the past twenty seven years,
20:05
Speaker 2: my daughter has absolutely nothing to show for it yet,
20:08
Speaker 2: and neither does any other child with ret.
20:11
Speaker 3: She knows you're at it, though, somehow doesn't.
20:13
Speaker 2: She Yes, she absolutely does, and it's the only reason
20:17
Speaker 2: that I'm able to look her in the eye every day.
20:19
Speaker 2: Once she has something to show for it, and other
20:22
Speaker 2: individuals as well, then we'll take a step back and say, Okay, job,
20:26
Speaker 2: well done, but we're not there yet.
20:28
Speaker 1: But qualifying as a rare disease, just you mentioned the
20:30
Speaker 1: big diseases, what is that? Just remind us that you
20:34
Speaker 1: can develop treatments under that umbrella. I mean, do we
20:37
Speaker 1: still have all the special rules that I remember we
20:40
Speaker 1: were able to create out of genon tech.
20:42
Speaker 2: Yeah, there are some And the definition of a rare
20:44
Speaker 2: disease is that it needs to affect under two hundred
20:47
Speaker 2: thousand people in the US. So we're definitely a rare disease.
20:51
Speaker 2: In the world of rare diseases, we're a fairly common
20:53
Speaker 2: one actually, because there's ultra rare and that's even tougher.
20:58
Speaker 3: Right.
20:59
Speaker 1: Yeah, there's been a lot of protesting and marches on DC.
21:02
Speaker 1: I saw one, you know at the FDA, Well there
21:05
Speaker 1: were two in a row. Are you guys part of
21:07
Speaker 1: that activism or not.
21:10
Speaker 3: No.
21:11
Speaker 2: We're a small organization with limited bandwidth, and we just
21:15
Speaker 2: focus on what we think we do best. There's other
21:19
Speaker 2: people out there that do lobbying and we don't, so
21:22
Speaker 2: we leave that up to others that can do it
21:25
Speaker 2: better than we do.
21:25
Speaker 1: That's not our field, and you partner with them, right,
21:28
Speaker 1: I mean what else can you do? I mean, are
21:30
Speaker 1: there a bunch of orgs or like, there's not that many.
21:32
Speaker 1: It's a handful, right, focused on red?
21:35
Speaker 2: Oh, focused on REHT, there's a couple, and then of
21:37
Speaker 2: course there's every like in Europe for example, every country
21:41
Speaker 2: has at least one, some have four or five. But
21:44
Speaker 2: we're the only ones that are focused on genetic medicines.
21:47
Speaker 1: So yeah, let's talk about that. What's the state of
21:49
Speaker 1: deed therapy that seems to be the for red syndrome?
21:53
Speaker 1: Is that the furthest along in the things that you're
21:56
Speaker 1: looking at.
21:56
Speaker 2: That's the furthest A long. It's the only genetic medica
22:00
Speaker 2: that is in clinical trials. The things that we're advancing
22:03
Speaker 2: are still in the lab haven't moved to clinical trials yet.
22:06
Speaker 2: So there's two companies and they both are in what's
22:09
Speaker 2: called pivotal trials or registrational trials. They fully enrolled trials
22:14
Speaker 2: where the FDA is expected to be able to make
22:17
Speaker 2: a decision on approval or not. Companies are sharing some
22:20
Speaker 2: information on efficacy, but more information shared during the year.
22:26
Speaker 2: With something like rehet syndrome, you're not going to get
22:28
Speaker 2: an immediate you know, you have gene therapy today and
22:31
Speaker 2: tomorrow you're better. It's going to take some time. And
22:35
Speaker 2: what we still don't know is there may be some
22:38
Speaker 2: modest improvements in year one, Will there be more improvements
22:41
Speaker 2: in year two? Will they continue to build? And maybe
22:45
Speaker 2: five years out you've got dramatic improvements. You know, we
22:47
Speaker 2: don't know that yet, but you can imagine not having
22:51
Speaker 2: all these symptoms and not having had the possibility to speak,
22:55
Speaker 2: and maybe the seizures are so bad you've missed a
22:57
Speaker 2: ton of school and all these other things like that's
23:00
Speaker 2: going to take time to really learn skills. So I
23:03
Speaker 2: think it's still early days.
23:04
Speaker 3: Well, this leads to a whole ton of other questions.
23:07
Speaker 1: If we do get good signs that it's helpful in
23:11
Speaker 1: that short term and then but you have to stay
23:14
Speaker 1: on it, keep getting more and more benefit. It's not
23:17
Speaker 1: a one and done treatment, is it, Or it is
23:20
Speaker 1: and then you watch and see what happens.
23:22
Speaker 3: Right, Yeah, it's.
23:23
Speaker 2: A one and done And the other thing to mention
23:26
Speaker 2: because it's very critical for families, and so gene therapy
23:31
Speaker 2: or gene supplementation right where you're adding healthy genes into
23:34
Speaker 2: the body that has to be delivered. That's the cargo, right,
23:38
Speaker 2: The gene is the cargo, and then you need delivery vehicle,
23:41
Speaker 2: and the delivery vehicle in most cases is a virus.
23:46
Speaker 2: You can only get a virus one time because basically
23:50
Speaker 2: you're inoculated against it, so if you give it again,
23:53
Speaker 2: you're going to have a very bad immune response that
23:56
Speaker 2: could kill you. So you can only get gene therapy
23:59
Speaker 2: delivered with the virus one time. And so direct development
24:03
Speaker 2: and genetic medicines also, it's an iterative process. The first
24:08
Speaker 2: gene therapy that's going to be approved is unlikely to
24:12
Speaker 2: be the last or the best. It's the first step.
24:15
Speaker 2: There may be other better gene therapies that come along,
24:18
Speaker 2: but if they're delivered virally and you've already had the
24:22
Speaker 2: first one, you're not going to be to get the
24:24
Speaker 2: other one. So this is something that is tough for
24:26
Speaker 2: families to figure out. Do I jump in now into
24:29
Speaker 2: a trial or when it's commercial wized, do I wait
24:32
Speaker 2: for something better? Yeah?
24:33
Speaker 3: Tough decisions, how many patients are in the trials.
24:36
Speaker 2: The trials are small. They've been like fifteen people something
24:39
Speaker 2: like that, So the trials are small. Gene therapy trials
24:42
Speaker 2: were always small. It's very expensive to make gene therapies
24:46
Speaker 2: and they're always small. So once it's commercialized, yeah, families,
24:50
Speaker 2: and we're assuming they'll be commercialized and families will have
24:53
Speaker 2: to make that decision.
24:55
Speaker 1: Well, that's the question. So you know, you mentioned it's
24:58
Speaker 1: very expensive. What's the cost for patient for treatment and
25:01
Speaker 1: how much is Obviously insurance does not cover this.
25:05
Speaker 2: Now other gene therapies that for other diseases insurance does cover.
25:09
Speaker 3: It does cover, but not yet not for rets.
25:13
Speaker 2: No, but there's nothing commercialized yet. So I'm sure the
25:16
Speaker 2: companies they're going to assume that they're going to get commercialized,
25:20
Speaker 2: and so I'm sure efforts are underway where they're already
25:22
Speaker 2: talking to insurance companies and medicaid medicare mostly Medicaid, but
25:27
Speaker 2: to get it covered because these are going to be
25:29
Speaker 2: multimillion dollar therapeutics and there is no business model if
25:34
Speaker 2: patients have to pay, because how many patients would be
25:37
Speaker 2: able to afford I don't know, two to four million
25:40
Speaker 2: dollars not many.
25:41
Speaker 1: So would you start raising money for that then, I
25:44
Speaker 1: guess probably to some degree, but then that would be hard.
25:47
Speaker 2: Well, I don't think we could raise enough to cover everybody.
25:51
Speaker 2: It really needs to be the companies need to be
25:55
Speaker 2: contracting and coming to agreements with insurance companies and medicaid
26:00
Speaker 2: to cover it. So, for example, spinal muscular atrophy has
26:04
Speaker 2: a gene therapy calls OLDGENSMA and it's available in I
26:09
Speaker 2: don't know fifty countries or more by now, and either
26:13
Speaker 2: governments pay if they're socialized medicine or private insurance or
26:17
Speaker 2: Medicaid pays in the US, and that's been the case
26:19
Speaker 2: for quite a number of years now. So it can
26:22
Speaker 2: be done for something that's transformative.
26:26
Speaker 1: I mean that's the key, right, You made that point
26:28
Speaker 1: that you won't now maybe for a few years, how transformative.
26:33
Speaker 1: But we always make the argument that we're saving costs
26:37
Speaker 1: of these fifteen thousand patients in other ways and not
26:42
Speaker 1: for an insurer could be very helpful because I imagine
26:46
Speaker 1: or you're tapping insurance to keep Chelsea where she needs
26:49
Speaker 1: to be.
26:50
Speaker 2: Yeah, and it's not just medical costs. So I mean
26:53
Speaker 2: for a lot of these kids. Medicaid is paying for
26:57
Speaker 2: help in the house or to modify the house, or
26:59
Speaker 2: feeding tubes, and the formula at Chelsea's formula alone is
27:03
Speaker 2: twelve hundred dollars a month. Medications and hospitalizations. They often
27:08
Speaker 2: have to have surgeries and they often get pneumonias and
27:11
Speaker 2: then so but how to pay for these very expensive
27:15
Speaker 2: medications is a whole other topic that really I'm not
27:19
Speaker 2: an expert on it, but people have to figure this
27:22
Speaker 2: out because when there's only a handful of gene therapies,
27:27
Speaker 2: any ensure payer is only going to have a few
27:30
Speaker 2: individuals in their policies, right. But when you've got one
27:34
Speaker 2: hundred gene therapies and there's going to be more and
27:36
Speaker 2: more and more every year, they're going to start bulking, Well,
27:39
Speaker 2: we can't afford it. So there needs to be the
27:42
Speaker 2: economics of it and the business model people are going
27:44
Speaker 2: to have to figure out, because what a shame it
27:46
Speaker 2: would be if we have transformative treatments and we just
27:49
Speaker 2: can't figure out how to pay for them. That would
27:51
Speaker 2: be an incredible tragedy.
27:53
Speaker 3: Well, that's a conundrum.
27:54
Speaker 1: But obviously, if it's a cure, you know, the better
27:57
Speaker 1: it works, the better chance right, because if you get
28:00
Speaker 1: what you pay for in that I think that example
28:04
Speaker 1: you gave before is a good one because there's real advance,
28:08
Speaker 1: you know, you really see change and difference. And I
28:12
Speaker 1: mean some of the gene therapies are delivering incremental change.
28:16
Speaker 1: That's a little harder potentially to justify than completely getting
28:21
Speaker 1: somebody back into life.
28:22
Speaker 2: And in time, there are drugs that are out there,
28:25
Speaker 2: for example, for muscular dystrophy that nobody really knows if
28:29
Speaker 2: they work or not, and yet they're out there and
28:32
Speaker 2: payers are paying for it. But eventually something better will
28:35
Speaker 2: come along. And when something better comes along, everybody will
28:38
Speaker 2: know it, and then no one's going to go after
28:41
Speaker 2: the drugs that are incremental, right.
28:43
Speaker 1: We have to do it as a society. We have
28:46
Speaker 1: to allow it or we'll never get there. Now we
28:49
Speaker 1: completely accept GLP ones, but for many many years that
28:53
Speaker 1: was not ubiquitous, and then we started with a very
28:56
Speaker 1: high cost and now it's low. And that's true for apath,
29:00
Speaker 1: which lowers your cluster all dramatically. I mean, I think
29:03
Speaker 1: we're going to see if we're at the cutting edge.
29:05
Speaker 1: I mean, with limited resources and a smaller team, how
29:08
Speaker 1: do you decide what to push forward. Obviously, you have
29:11
Speaker 1: to decide I'm going to worry about that payment thing later.
29:16
Speaker 1: Right now, I need to worry about getting this thing
29:19
Speaker 1: to work.
29:20
Speaker 2: Yes, and you've kind of nailed what my philosophy has
29:23
Speaker 2: always been, like, we're going to tackle one big problem
29:25
Speaker 2: at a time, and we're not going to look down
29:26
Speaker 2: the road at ten steps from now what that big
29:29
Speaker 2: problem is going to be. Well, for one thing, we've
29:31
Speaker 2: decided to focus on genetic medicines. It was a thoughtful
29:34
Speaker 2: decision to say we're not going to go after small
29:36
Speaker 2: molecule drugs, Like we can't do everything. We're also not
29:39
Speaker 2: a family support organization. We're not an awareness organization. We're
29:43
Speaker 2: research and we're focused on solely on genetic medicines. That
29:47
Speaker 2: decision was made so that we can have the bandwidth
29:50
Speaker 2: that we need to really focus on what we do best,
29:52
Speaker 2: and then everything else is data driven. Kind of data
29:55
Speaker 2: driven these projects start off. We don't take on a
29:58
Speaker 2: project that's going to be twenty million dollars right off
30:00
Speaker 2: the bat. We'll start off with the project that needs
30:02
Speaker 2: you two million to get it going, and then we
30:04
Speaker 2: see what happens with the data, and we generate the
30:07
Speaker 2: data and does it look good and not we prune
30:09
Speaker 2: things we've stopped projects. And then, for example with base
30:13
Speaker 2: editing or RNA editing, if it turns out that an
30:16
Speaker 2: approach that works for all mutations gives us the kind
30:19
Speaker 2: of results that we need, well then we don't need
30:21
Speaker 2: a base editing or an RNA editing, you know, and
30:23
Speaker 2: then we'll prune those. So it's always kind of looking
30:26
Speaker 2: at what you have and figuring out what are the
30:28
Speaker 2: go no go decisions? And it's iterative.
30:31
Speaker 1: Does AI help us? I mean, everybody says AI, it's
30:34
Speaker 1: the great savior? Is that going to be good that
30:36
Speaker 1: we have these new texts to help?
30:39
Speaker 2: Yes, the companies that we work with are some of
30:42
Speaker 2: them are definitely using AI. So for example, with one
30:46
Speaker 2: of our base editing projects, we've partnered with an AI
30:51
Speaker 2: company called Profluent, who uses AI to develop editors that
30:56
Speaker 2: are small and can fit into the cargo space that
31:01
Speaker 2: we have available to us. With a viral delivery, for example,
31:04
Speaker 2: there are companies that are using AI to engineer viruses
31:10
Speaker 2: so that they can get into the brain better. You know,
31:12
Speaker 2: they're using AI for all sorts of things.
31:15
Speaker 1: And are you collaborating with some of the ones who
31:17
Speaker 1: have been successful in getting these gene therapies to market.
31:22
Speaker 1: I mean you're learning from it, of course, but are
31:24
Speaker 1: you collaborating with some of those companies and scientists and people.
31:29
Speaker 2: I wouldn't call it so much as collaborating as lessons
31:33
Speaker 2: learned what can we learn from them? But honestly, there
31:35
Speaker 2: haven't been that many gene therapies of the kind that
31:40
Speaker 2: we need. Sol Gentleman is one of them, the SMA,
31:43
Speaker 2: So there's been a lot of lessons learned there.
31:46
Speaker 1: That gives you hope. How do you feel where are we?
31:48
Speaker 1: Are you feeling optimistic? I guess you have to.
31:51
Speaker 2: You can't be in this line of work if you're
31:54
Speaker 2: not optimistic. Like, yes, I'm an eternal optimist, and the
31:58
Speaker 2: more I learn, the more optimistic I am. I hope
32:01
Speaker 2: that when we have something that's transformative that Chelsea can
32:03
Speaker 2: still benefit from it. And I've met so many families
32:07
Speaker 2: over the years, and I'm motivated by their kids as well.
32:11
Speaker 2: But yes, I'm very optimistic.
32:13
Speaker 3: Does life ever feel normal?
32:15
Speaker 2: I have a pretty abnormal life. I would say life
32:18
Speaker 2: with Chelsea is, I mean, compared to lots of other people,
32:22
Speaker 2: it's not normal. We can't travel with her. We don't
32:25
Speaker 2: go on vacations because I won't leave her home. We
32:27
Speaker 2: can't really be spontaneous. It's difficult to be spontaneous. She
32:31
Speaker 2: has seizures often like wherever we go, so we have
32:35
Speaker 2: to be able to Like, I wouldn't go out on
32:37
Speaker 2: a boat with her because what if she has a
32:38
Speaker 2: really bad seizure and I need to get her to
32:40
Speaker 2: a hospital or something like that. So we're careful about
32:43
Speaker 2: what we do. We always kind of have an exit strategy.
32:46
Speaker 2: If we're going somewhere where my husband or I one
32:48
Speaker 2: of us has to stay, we might go with two cars,
32:51
Speaker 2: that kind of thing. And then I guess the other
32:53
Speaker 2: reason life is not normal is more of a decision
32:56
Speaker 2: on my part. But I pretty much work seven days
32:58
Speaker 2: a week.
32:59
Speaker 3: Have you got left?
33:00
Speaker 2: He occasionally for work very short one two three day trips,
33:04
Speaker 2: very very short.
33:05
Speaker 1: That's it, and your husband or the caregiver or somebody
33:08
Speaker 1: watches her.
33:10
Speaker 2: It really takes two people to lift Chelsea now, so
33:13
Speaker 2: I can't leave her with one person. I have to
33:15
Speaker 2: leave her with two people. So if I have to
33:18
Speaker 2: go somewhere, then my husband has to be here to
33:20
Speaker 2: be with the caregiver.
33:22
Speaker 1: How'd you handle the boys graduation and all that stuff.
33:26
Speaker 3: You know.
33:26
Speaker 2: Well, my older son graduated during COVID, so there was
33:30
Speaker 2: no graduation, so that took care.
33:32
Speaker 3: Of that, all right. That was the one silver lining
33:35
Speaker 3: of COVID.
33:35
Speaker 2: I guess my other son, he was in New Hampshire
33:39
Speaker 2: and we took her with our van. My husband came
33:42
Speaker 2: up with a very cool idea. We have a wheelchair
33:45
Speaker 2: van at ramp off the back and two captain chairs
33:49
Speaker 2: or whatever you call them behind the driver and passenger,
33:52
Speaker 2: and she fits in the middle with her wheelchair. So
33:54
Speaker 2: he came up with this idea of we can take
33:56
Speaker 2: the wheelchair out. We have a piece of ply and
34:00
Speaker 2: a crib mattress that we lay on top of the
34:03
Speaker 2: two chairs. We can lift her, he lifts her, walks
34:07
Speaker 2: her in laser down. We can change her, let her stretch,
34:10
Speaker 2: so you know, if we're driving a long distance, we
34:13
Speaker 2: stop somewhere and get her out and change her and
34:16
Speaker 2: let her stretch and let her take a nap whatever,
34:18
Speaker 2: and then we'll just pick up and keep going.
34:21
Speaker 3: So we do that great. I mean, I guess that's
34:23
Speaker 3: sort of a vacation or not really.
34:26
Speaker 2: You know, at home we have all of the comforts
34:28
Speaker 2: that make it easier to take care of it. We
34:30
Speaker 2: need to bring a lot of stuff with us.
34:33
Speaker 1: Right, So there's an album that's a soundtrack to your life?
34:36
Speaker 1: What is that?
34:37
Speaker 3: Oh?
34:37
Speaker 2: God, I don't know. I'd have to think about that.
34:40
Speaker 3: Does she respond to music?
34:42
Speaker 2: Yes, she loves music, and she listens to a lot
34:45
Speaker 2: of seventies music because that's what i'd like. Well, when
34:48
Speaker 2: her brothers are here, she'll listen to others. She'll listen
34:51
Speaker 2: to anything. She's very easy going. She does love music.
34:54
Speaker 3: Do you see a different response on her when that's happening.
34:57
Speaker 2: Yeah, takes attention and she'd perks up.
35:00
Speaker 3: And yeah that's great. Yeah.
35:02
Speaker 1: I mean, I'm working on a program. I'm gonna we
35:05
Speaker 1: just st at about to launch a podcasts about music
35:08
Speaker 1: and the mind and music and medicine and how important
35:11
Speaker 1: that is in all kinds of diseases. But I would
35:14
Speaker 1: imagine in these sort of rare diseases even more so
35:18
Speaker 1: in some ways because it really engages you.
35:22
Speaker 2: And I know there's studies on if you listen to
35:24
Speaker 2: music when you're having like painful procedures done or anxiety
35:29
Speaker 2: provoking procedures that can help.
35:32
Speaker 1: So, what's something that you're getting real about right now,
35:36
Speaker 1: besides what we just got real about.
35:39
Speaker 2: Trying to carve out some me time. I'm bad at that.
35:43
Speaker 3: Yeah, sounds like you could use that.
35:45
Speaker 2: Yeah, So trying to get real about starting to work
35:49
Speaker 2: out and just a little little me time. Not sure
35:52
Speaker 2: if I'll be bestful.
35:54
Speaker 1: Well do you think that would be good for her?
35:56
Speaker 1: I mean you've dedicated a lot of your life to her.
35:59
Speaker 1: Somehow you managed to raise two other boys that sound
36:02
Speaker 1: pretty normal and healthy. Do they have Are they all
36:05
Speaker 1: for it? Or is there resentment? I mean, is there
36:08
Speaker 1: any they're all in?
36:09
Speaker 2: Oh? No, no resentment. They love her to pieces and
36:12
Speaker 2: yeah no.
36:13
Speaker 1: No, yeah, and just have help and support it and
36:16
Speaker 1: the cause we're all part of it. Did anyone come
36:19
Speaker 1: into the family business?
36:20
Speaker 3: Are they in it? I mean are they in the foundation?
36:23
Speaker 3: In the trust?
36:23
Speaker 2: We're not big enough for that, Like we're how big?
36:27
Speaker 3: Yeah? How many people are involved?
36:29
Speaker 2: So besides myself, we have four people on our research team.
36:33
Speaker 2: We have a somebody with decades of genetic medicine background.
36:38
Speaker 2: We have someone that has deep, deep experience in red
36:41
Speaker 2: syndrome that worked in a red lab. We've got a
36:43
Speaker 2: chief medical officer, we have kind of a research manager,
36:47
Speaker 2: chief development officer, and an administrative officer. And then we
36:50
Speaker 2: have some part time folks or some contractors. So you
36:53
Speaker 2: know we're under ten people. Yeah we're lean.
36:55
Speaker 1: Yeah, you run lean and mean for my audience, let's
36:59
Speaker 1: ask this question and see what you say.
37:01
Speaker 3: What do you need from us?
37:03
Speaker 1: What do you need from us the community, from everyone
37:06
Speaker 1: to move this to the next phase and step.
37:09
Speaker 2: If anyone listens to this and is moved to make
37:12
Speaker 2: a donation. Of course, we would love that you could
37:15
Speaker 2: google RET Syndrome Research Trust. If you know somebody that
37:19
Speaker 2: has red syndrome, let them know about our organization. I mean, really,
37:23
Speaker 2: awareness is always important. And maybe people know somebody that
37:28
Speaker 2: sits on a foundation that funds whether it's women's disorders
37:32
Speaker 2: or you know, children diseases, or maybe they have a
37:36
Speaker 2: philanthropic family member. Maybe they want to volunteer some time,
37:40
Speaker 2: maybe they whatever it is, we're only in email away.
37:43
Speaker 1: Is there anything everybody needs to know about RET syndrome
37:46
Speaker 1: that you think they're misunderstanding?
37:48
Speaker 2: Well, I mean, one thing that doesn't just apply to
37:52
Speaker 2: REHT applies to everyone that's kind of nonverbal. Just because
37:56
Speaker 2: they can't speak doesn't mean they have nothing to say,
37:58
Speaker 2: and it doesn't mean that they can under stand what
38:00
Speaker 2: you're saying. So I'm always disappointed when we talk to
38:05
Speaker 2: people and they ignore Chelsea. It's like, say hello, say something.
38:08
Speaker 2: She's thirty years old, she's not a child. Acknowledge her.
38:12
Speaker 2: I would say, acknowledge people whether they can speak or not.
38:15
Speaker 2: A lot of people do, and it's wonderful when they do,
38:18
Speaker 2: but a lot of people don't. They don't think of it,
38:20
Speaker 2: or they feel awkward, they don't know what to say,
38:22
Speaker 2: and so it's just easier or yeah, but that always
38:25
Speaker 2: hurts a little bit. And I don't know what she
38:27
Speaker 2: thinks about it, but it doesn't take much to just
38:30
Speaker 2: say hello and comment about something. I'd love your hair,
38:34
Speaker 2: it's great what you're wearing, or what a cool wheelchair?
38:37
Speaker 2: Something you know, say something.
38:38
Speaker 1: Look what she made happen. She made her mom do this,
38:43
Speaker 1: leaving pasta making to drug making and possibly changing the
38:48
Speaker 1: world in a big way.
38:51
Speaker 3: So that that's huge. Yeah.
38:53
Speaker 2: I tell her that, without ever saying a word, she's
38:57
Speaker 2: had a big impact, and I hope a bigger impact
39:00
Speaker 2: to come.
39:01
Speaker 1: You're changing the world and not we just want to
39:03
Speaker 1: say thanks and our audience. You know hopefully we'll get
39:07
Speaker 1: more active and involved as a result.
39:09
Speaker 2: Well, thank you, Jim, I appreciate that.
39:11
Speaker 1: Great to finally meet you, and we'll be sharing this
39:14
Speaker 1: far and live as far as wide as I can
39:17
Speaker 1: get it awesome.
39:18
Speaker 2: I appreciate that.
39:19
Speaker 3: Thank you, thanks for being here. Hey everyone, thanks for watching.
39:23
Speaker 1: I really appreciate that you take the time to listen
39:26
Speaker 1: to this and tune in, so please subscribe, follow I've
39:31
Speaker 1: got a lot of amazing guests. You're going to learn
39:33
Speaker 1: a lot, and you're going to feel a lot better
39:35
Speaker 1: because you did it.