Documentary about a family’s journey to Minnesota for gene therapy premieres in Minneapolis
Celia Grace Hamlett was 4-years-old when she traveled with her parents from Alabama to Minneapolis to receive life-saving treatment for a rare genetic disease, Metachromatic Leukodystrophy or MLD.
Courtesy Lindsey Seavert
The Minneapolis St. Paul International Film Festival is currently underway, and Monday night a documentary called “Sequencing Hope” will premiere.
The film, directed by Lindsey Seavert and Maribeth Romslo, follows an Alabama family who came to Minnesota to get their young daughter life-saving gene therapy for a rare disease called metachromatic leukodystrophy.
Celia Grace Hamlett was 4 years old when she came to M Health Fairview Masonic Children’s Hospital in 2021 and became the first person in the U.S. to undergo the experimental gene therapy.
Her family is in town for the film’s premiere. Celia Grace’s dad, Gary spoke with MPR News host Nina Moini along with their doctor, Dr. Paul Orchard.
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Audio transcript
NINA MOINI: The Minneapolis Saint Paul International Film Festival is currently under way. And tonight, a documentary will premiere called "Sequencing Hope." The film is directed by Lindsey Seavert and Maribeth Romslo. It follows an Alabama family who came to Minnesota to get their young daughter life-saving gene therapy for a rare disease. Let's listen to a clip from the trailer.
CELIA GRACE HAMLETT: Can you hold my hand?
[CHUCKLES]
They give me the medicine.
SUBJECT 1: There is lots of other gene therapy research on the horizon.
SUBJECT 2: That's got far-reaching consequences to move medicine forward.
SUBJECT 3: In my heart, I feel that the good Lord has something in store for Celia Grace.
SUBJECT 4: We just pray, Lord, for a miracle. We pray for a healing for Celia Grace.
SUBJECT 5: You know, when it comes to your kids, you're going to do whatever it takes to protect them.
SUBJECT 6: If it saves one child, then I feel like we have accomplished something.
NINA MOINI: Celia Grace Hamlett was four-years-old when she came to M Health Fairview Masonic Children's Hospital in 2021 and became the first person in the US to undergo the experimental gene therapy. Her family's in town for the film's premiere tonight and Celia Grace's dad, Gary, joins us now along with their doctor, Doctor Paul Orchard. Thank you both for being here.
GARY HAMLETT: Thank you.
PAUL ORCHARD: Thank you, Nina.
NINA MOINI: Yeah, and Gary, let me start with you if I might. Tell us about your daughter, Celia Grace. She's seven-years-old now and I understand she was diagnosed with this rare and often fatal genetic disorder, MLD, when she was diagnosed at three-years-old. What were her options at that point?
GARY HAMLETT: Well, at that point had one or had two options. One was bone marrow transplant or the other was gene therapy that was only being done in Milan, Italy.
NINA MOINI: Wow. Doctor Orchard, can you tell us all what MLD is?
PAUL ORCHARD: Certainly. Appreciate the opportunity to speak with you today. So metachromatic leukodystrophy is a rare inherited disorder. It's what we call a lysosomal disease. The lysosome is an organelle within cells that help break down materials that the cell is attempting to get rid of.
And there's a number of enzymes that are present in the lysosome that help accomplish that. Arylsulfatase A. is one of those. And in this circumstance, if you are unlucky enough to receive a mutation within the arylsulfatase gene from both mom and from dad, then you're affected with the disease.
But both parents who have one normal copy of the gene are absolutely fine. There's nothing to suggest that they have any sort of problem, but again, if you receive an abnormal copy from both parents then you see the disease. And in this situation, it's primarily a neurologic disorder. It occurs in kids as young as one or so in terms of manifestations of the disease, but it's progressive and lethal if there's no therapy.
NINA MOINI: Wow, that's just so much to take in, Gary. And you know, you mentioned having to maybe think about treatment over in Milan. How did you hear about the treatment for MLD that was right here in Minnesota?
GARY HAMLETT: Our doctor neurologist in Alabama, Doctor Matt, is the one that contacted us and said, what would y'all think if I told y'all y'all's daughter was going to make history books? At that point we said, what do you mean? And she said, well, your daughter may be the first child in the United States to receive gene therapy for MLD and it will be done at the Masonic Children's Hospital in Minneapolis under the care of Doctor Paul Orchard.
NINA MOINI: And then what did you think? I mean, were you going to have to pay for that?
GARY HAMLETT: Yes. At that point we didn't really care what it cost us being able to save our daughter's life. So our community started doing fundraisers to try to raise money to pay for this.
NINA MOINI: Wow, yeah. Doctor Orchard, can you explain how the gene therapy works and is it accessible to most people or is it just too costly?
PAUL ORCHARD: Well, the gene therapy clinical trials occurred in Europe, as Gary was alluding to, and the data was sufficiently positive that it was approved as therapy in the EU, essentially. So it's been licensed therapy there for several years, but none of the clinical trials have been done here in the US.
And because of the promise of this new therapy, we were gearing up to being able to offer this regardless, but there was the opportunity in this situation from Celia Grace's diagnosis to be able to intervene. So it's just become licensed therapy in the last month or so, as March 18th.
But prior to that and for Celia Grace, we had to petition the FDA to allow us to use it because it's still considered experimental therapy, and get all the approvals from all the various regulatory groups to be able to do that. So it took some time, but it opened the doors. And now we've treated a total of five patients with compassionate use therapy.
NINA MOINI: All right. Is it still pretty pricey, though? I understand it's among some of the priciest treatments.
PAUL ORCHARD: Yes, it is very expensive. So for the compassionate use treatment as an experimental therapy, the company actually donated the cell product, but it's millions of dollars now as licensed therapy.
NINA MOINI: Yeah. So still working to make it more accessible. Gary, you said something that really struck me in the trailer for the film. You said that you take care of people for a living. I understand you work in law enforcement, but you couldn't fix this for your daughter. And it seems like this film is really an exploration of your family's journey. Tell me how did that feel to feel sort of helpless in the moment, but then to see her go through this journey and be, I mean, cured?
GARY HAMLETT: We just felt very helpless, not knowing the outcome of it, how sick Grace was. Just thinking that we were going to lose our daughter. Possibly by the age of five-years-old.
NINA MOINI: Yeah.
GARY HAMLETT: And seeing her now as a normal seven-year-old, running, playing, is going to graduate in kindergarten, and it's just an amazing feeling.
NINA MOINI: Yeah, I'm sure. And so how is she doing? Tell us a little bit just about how she's getting around fine, and she's feeling well.
GARY HAMLETT: Oh, she is rambunctious, non-stop playing, running, doing her schoolwork. She is just like a typical seven-year-old little girl.
NINA MOINI: Yeah, and I understand some more patients are going to be undergoing that same treatment as Celia Grace, which is great news, Doctor Orchard.
PAUL ORCHARD: Yes, I hope it's going to be widely available. As you mentioned, the cost is going to be significant and attempting to determine how we're going to do this. The vast majority of these patients that we treat are obviously not from Minnesota.
And so being able to get insurance that's going to work across state lines and going to be sufficient for this is going to be a challenge. But that's one of the things that we're currently working on.
NINA MOINI: OK, and Gary, I'll leave the last question for you here. What do you hope people will take away from watching your family's story in this documentary?
GARY HAMLETT: The struggles of not knowing the outcome of your child. The struggles of possibly knowing that you will only have a few years with your child. And then knowing that there are people out there willing to help and willing to do anything possible to save your daughter or your son. I just can never repay everybody that along this journey for what they have done for my child.
NINA MOINI: Yeah, and it really sounds like it's some of the best parts of humanity and also some of the hardest struggles that anyone will go through. Thank you so much for sharing that journey and for being here, Gary. And to you as well, Doctor Orchard, thank you, and congratulations on this film reaching an audience today.
PAUL ORCHARD: Thank you.
GARY HAMLETT: Thank you so much.
NINA MOINI: Gary Hamlett is the father of Celia Grace Hamlett and Doctor Orchard is a pediatric blood and marrow transplant physician at M. Health Fairview. Both are featured in the documentary, "Sequencing Hope," which is premiering tonight at 7:00 PM. We'll have that information on our website, mprnews.org.
CELIA GRACE HAMLETT: Can you hold my hand?
[CHUCKLES]
They give me the medicine.
SUBJECT 1: There is lots of other gene therapy research on the horizon.
SUBJECT 2: That's got far-reaching consequences to move medicine forward.
SUBJECT 3: In my heart, I feel that the good Lord has something in store for Celia Grace.
SUBJECT 4: We just pray, Lord, for a miracle. We pray for a healing for Celia Grace.
SUBJECT 5: You know, when it comes to your kids, you're going to do whatever it takes to protect them.
SUBJECT 6: If it saves one child, then I feel like we have accomplished something.
NINA MOINI: Celia Grace Hamlett was four-years-old when she came to M Health Fairview Masonic Children's Hospital in 2021 and became the first person in the US to undergo the experimental gene therapy. Her family's in town for the film's premiere tonight and Celia Grace's dad, Gary, joins us now along with their doctor, Doctor Paul Orchard. Thank you both for being here.
GARY HAMLETT: Thank you.
PAUL ORCHARD: Thank you, Nina.
NINA MOINI: Yeah, and Gary, let me start with you if I might. Tell us about your daughter, Celia Grace. She's seven-years-old now and I understand she was diagnosed with this rare and often fatal genetic disorder, MLD, when she was diagnosed at three-years-old. What were her options at that point?
GARY HAMLETT: Well, at that point had one or had two options. One was bone marrow transplant or the other was gene therapy that was only being done in Milan, Italy.
NINA MOINI: Wow. Doctor Orchard, can you tell us all what MLD is?
PAUL ORCHARD: Certainly. Appreciate the opportunity to speak with you today. So metachromatic leukodystrophy is a rare inherited disorder. It's what we call a lysosomal disease. The lysosome is an organelle within cells that help break down materials that the cell is attempting to get rid of.
And there's a number of enzymes that are present in the lysosome that help accomplish that. Arylsulfatase A. is one of those. And in this circumstance, if you are unlucky enough to receive a mutation within the arylsulfatase gene from both mom and from dad, then you're affected with the disease.
But both parents who have one normal copy of the gene are absolutely fine. There's nothing to suggest that they have any sort of problem, but again, if you receive an abnormal copy from both parents then you see the disease. And in this situation, it's primarily a neurologic disorder. It occurs in kids as young as one or so in terms of manifestations of the disease, but it's progressive and lethal if there's no therapy.
NINA MOINI: Wow, that's just so much to take in, Gary. And you know, you mentioned having to maybe think about treatment over in Milan. How did you hear about the treatment for MLD that was right here in Minnesota?
GARY HAMLETT: Our doctor neurologist in Alabama, Doctor Matt, is the one that contacted us and said, what would y'all think if I told y'all y'all's daughter was going to make history books? At that point we said, what do you mean? And she said, well, your daughter may be the first child in the United States to receive gene therapy for MLD and it will be done at the Masonic Children's Hospital in Minneapolis under the care of Doctor Paul Orchard.
NINA MOINI: And then what did you think? I mean, were you going to have to pay for that?
GARY HAMLETT: Yes. At that point we didn't really care what it cost us being able to save our daughter's life. So our community started doing fundraisers to try to raise money to pay for this.
NINA MOINI: Wow, yeah. Doctor Orchard, can you explain how the gene therapy works and is it accessible to most people or is it just too costly?
PAUL ORCHARD: Well, the gene therapy clinical trials occurred in Europe, as Gary was alluding to, and the data was sufficiently positive that it was approved as therapy in the EU, essentially. So it's been licensed therapy there for several years, but none of the clinical trials have been done here in the US.
And because of the promise of this new therapy, we were gearing up to being able to offer this regardless, but there was the opportunity in this situation from Celia Grace's diagnosis to be able to intervene. So it's just become licensed therapy in the last month or so, as March 18th.
But prior to that and for Celia Grace, we had to petition the FDA to allow us to use it because it's still considered experimental therapy, and get all the approvals from all the various regulatory groups to be able to do that. So it took some time, but it opened the doors. And now we've treated a total of five patients with compassionate use therapy.
NINA MOINI: All right. Is it still pretty pricey, though? I understand it's among some of the priciest treatments.
PAUL ORCHARD: Yes, it is very expensive. So for the compassionate use treatment as an experimental therapy, the company actually donated the cell product, but it's millions of dollars now as licensed therapy.
NINA MOINI: Yeah. So still working to make it more accessible. Gary, you said something that really struck me in the trailer for the film. You said that you take care of people for a living. I understand you work in law enforcement, but you couldn't fix this for your daughter. And it seems like this film is really an exploration of your family's journey. Tell me how did that feel to feel sort of helpless in the moment, but then to see her go through this journey and be, I mean, cured?
GARY HAMLETT: We just felt very helpless, not knowing the outcome of it, how sick Grace was. Just thinking that we were going to lose our daughter. Possibly by the age of five-years-old.
NINA MOINI: Yeah.
GARY HAMLETT: And seeing her now as a normal seven-year-old, running, playing, is going to graduate in kindergarten, and it's just an amazing feeling.
NINA MOINI: Yeah, I'm sure. And so how is she doing? Tell us a little bit just about how she's getting around fine, and she's feeling well.
GARY HAMLETT: Oh, she is rambunctious, non-stop playing, running, doing her schoolwork. She is just like a typical seven-year-old little girl.
NINA MOINI: Yeah, and I understand some more patients are going to be undergoing that same treatment as Celia Grace, which is great news, Doctor Orchard.
PAUL ORCHARD: Yes, I hope it's going to be widely available. As you mentioned, the cost is going to be significant and attempting to determine how we're going to do this. The vast majority of these patients that we treat are obviously not from Minnesota.
And so being able to get insurance that's going to work across state lines and going to be sufficient for this is going to be a challenge. But that's one of the things that we're currently working on.
NINA MOINI: OK, and Gary, I'll leave the last question for you here. What do you hope people will take away from watching your family's story in this documentary?
GARY HAMLETT: The struggles of not knowing the outcome of your child. The struggles of possibly knowing that you will only have a few years with your child. And then knowing that there are people out there willing to help and willing to do anything possible to save your daughter or your son. I just can never repay everybody that along this journey for what they have done for my child.
NINA MOINI: Yeah, and it really sounds like it's some of the best parts of humanity and also some of the hardest struggles that anyone will go through. Thank you so much for sharing that journey and for being here, Gary. And to you as well, Doctor Orchard, thank you, and congratulations on this film reaching an audience today.
PAUL ORCHARD: Thank you.
GARY HAMLETT: Thank you so much.
NINA MOINI: Gary Hamlett is the father of Celia Grace Hamlett and Doctor Orchard is a pediatric blood and marrow transplant physician at M. Health Fairview. Both are featured in the documentary, "Sequencing Hope," which is premiering tonight at 7:00 PM. We'll have that information on our website, mprnews.org.
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