A Mother’s Gift: The Foundation for a Lifelong Passion in Writing
Every mother has her unique way of guiding and teaching her children. My mother was no exception. She was instrumental in teaching me to read and write even before I started my formal education. Her nurturing and guidance have made me who I am today: a passionate writer and the proud author of the L.A. Loft Blog and Entar.com. As we celebrate Mother’s Day, I want to share my story as a tribute to my mother and all the amazing moms out there who shape their children’s lives in profound ways.
The Wise Woman and Her Free-Range Approach
My mother was not the type to constantly instruct me on what to do or not to do. Instead, she was more of a free-range mom who believed in giving me space to learn and grow at my own pace. She offered valuable advice when needed, and time has revealed that she was, indeed, a wise woman. Her approach allowed me to develop a strong sense of independence and curiosity, which later translated into my passion for reading and writing.
The California Home
The Gift of Reading and Writing
Before I even set foot in my first grade classroom, my mother had already taught me to read and write at a third-grade level. She recognized the importance of a strong foundation in literacy and spent countless hours nurturing my abilities. This early start in my education not only made me feel confident and ready for school but also sparked a love for reading and writing that has stayed with me throughout my life.
The L.A. Loft Blog: A Testament to a Mother’s Love
In addition to Loft Blog readers, friends and clients, my mother’s guidance and support led to my success. Her belief in me and her dedication to my education laid the groundwork for my passion for writing. This Mother’s Day, I want to acknowledge her impact on my life and express my gratitude for her unwavering love and support.
A Gift for All Mothers
This Mother’s Day, let’s celebrate the wisdom, love, and dedication of all mothers, both present and those who are no longer with us. Each mother has her unique way of shaping her children’s lives, and their influence lasts a lifetime. So here’s a heartfelt gift to all moms out there: Buy a home in May, get $5,000 cash from your broker, the Corey Chambers Team, at closing. Happy Mother’s Day!
Though my mother is no longer here, her legacy lives on in my writing and my love for reading. Her wisdom and love have made a lasting impact on my life, and I am forever grateful. As we celebrate Mother’s Day, let’s remember to honor and appreciate the incredible women who have made us who we are today. Happy Mother’s Day to all the wise, loving, and dedicated mothers out there. Your impact is immeasurable, and your love knows no bounds.
Corey Chambers Team raising $25,000 for CHLA
Supporting Moms at Children’s Hospital: How Your Real Estate Referrals Can Help Families in Need
There are many ways to make a positive impact on the lives of families with sick children. At Children’s Hospital Los Angeles, the dedicated staff goes above and beyond to support mothers whose children are fighting for their lives. As we approach Mother’s Day, it’s important to remember that many moms are by their child’s bedside, focusing on their well-being rather than on their own special day. One way you can help these moms and their children is through your real estate referrals. Read on to learn how your referrals can make a difference in the lives of these families.
The Mission: Raising $25,000 for Children’s Hospital Los Angeles
Our team is on a mission to raise $25,000 for Children’s Hospital Los Angeles. The funds raised will support the Children’s Recovery Center, where kids battling cancer and other debilitating diseases receive life-saving care. The Recovery Center relies on sponsorships and donations to operate, and your real estate referrals can help ensure that more children have access to this vital resource.
Children receiving care at the Children’s Recovery Center are 300% more likely to enter remission when they can access its services. With your help, we can make a difference in the lives of these young patients and their families.
How Your Referrals Help the Kids
When you refer someone to our real estate sales team, not only do they benefit from our award-winning service, but we also donate a substantial portion of our income from every home sale to Children’s Hospital of Los Angeles. This means that your referrals directly contribute to the well-being of children in need.
How to Make a Referral
Referring someone is easy. Just visit www.ReferralsHelpKids.com or call us directly at 213-880-9910. You can rest assured that your referrals will receive excellent service, as well as our exclusive guarantees:
Home Sellers: We will sell your home at your price, or we’ll buy it ourselves.*
Home Buyers: If you are not completely satisfied with your home within 24 months of purchase, we will buy it back or sell it for free, your choice.*
Why Your Referrals Matter
Your referrals not only help us provide top-notch real estate services, but they also support a worthy cause. Children’s Hospital Los Angeles relies on the generosity of people like you to continue its life-saving work.
As we honor mothers this month, let’s not forget the moms who are fighting for their children’s lives. Your referrals can make a difference for these families and help Children’s Hospital continue its vital mission.
*Conditions apply. Please inquire for details.
A Lifelong Connection: Why I Support Children’s Hospital Los Angeles
Children’s Hospital Los Angeles (CHLA) is a beacon of hope for countless families in need of specialized care for their children. As a native of the Greater Los Angeles Area, I have always felt a deep connection to this incredible institution and its mission. In this article, I will share my personal story of why I support Children’s Hospital Los Angeles and how my team and I work together to contribute to their cause.
A Personal Connection to Children’s Hospital Los Angeles
We are grateful for your support in our effort to raise $25,000 for Children’s Hospital Los Angeles. By referring friends, family, and associates to our real estate sales team, you’re not only helping them find their dream home, but you’re also giving back to a meaningful cause. Together, we can make a difference in the lives of children and their families. Visit www.ReferralsHelpKids.com or call us at 213-880-9910 to make a referral today.
Growing up in the Greater Los Angeles Area, I was born in Los Angeles County at St. Francis Hospital. My connection to Children’s Hospital Los Angeles began when a young person close to our family suffered from a severe illness and received treatment at CHLA. This experience opened my eyes to the vital work carried out by the dedicated healthcare professionals at the hospital. As a result, I felt compelled to contribute to their mission in any way possible.
The Common Cause: Healing Young Lives
Children’s Hospital Los Angeles brings together hard-working healthcare professionals from the Los Angeles area, united by a common cause – to help young people overcome the health challenges life sometimes presents. As a native of the area, I take immense pride in supporting the incredible work carried out by the CHLA team. My team and I have made it our annual goal to raise money and donate a portion of our income to help CHLA in their quest to heal young people when they need it the most.
Our Commitment to Supporting CHLA
My team and I are dedicated to providing outstanding results for buyers and sellers referred to us by our past clients. We have found that Children’s Hospital Los Angeles shares a similar commitment to their patients. Since their services rely on sponsorships and donations, we are delighted to contribute and proud to support their life-changing work.
Children’s Hospital Los Angeles is an institution that has touched the lives of countless families in the Greater Los Angeles Area. My personal connection to CHLA has inspired me and my team to support their mission in any way we can. By raising funds and donating a portion of our income, we aim to contribute to the incredible work they do to heal young lives. Together, we can make a difference and help CHLA continue to provide hope and healing to those who need it the most.
A 67-Year-Old Donor’s ‘Sliver of Liver’ Saves a Baby’s Life
Selena’s successful liver transplant highlights the viability of older donors and the work of CHLA’s living donor program, one of the busiest in the nation.
“How Selena Met Mark” is unusual and random, and yet a story that ultimately comes around to make great sense—and good science.
In their first encounter, Selena met only a piece of Mark—his liver, specifically, and only a small slice of it. “A sliver of my liver,” Mark likes to say.
Mark Scotch was, and still very much is, a living donor who matched with Selena through the Children’s Hospital Los Angeles living donor liver transplant program, one of the few and busiest programs of its kind in the U.S. One-third of transplanted donor livers at CHLA come from living donors.
So what was so unusual about how the two connected? First, Scotch was not family. “It happens, but it’s uncommon,” says CHLA surgeon Kambiz Etesami, MD, Director of Abdominal Transplantation and Surgical Director of the Liver Transplant Program, who assisted in performing Selena’s transplant.
And random? Scotch was an altruistic liver donor—now more commonly called “non-directed,” Dr. Etesami says, meaning his liver could go to whoever needed it. It didn’t take long before it found Selena.
Liver failure caused by biliary atresia
Selena was born with biliary atresia, a disorder that inflames and scars the bile ducts, preventing bile—a green-yellow fluid—from flowing out of the liver and through the ducts and eventually into the small intestine. Because of the blockage, bile gets stuck in the liver, damaging the organ and, eventually, causing it to fail.
“It’s the most rapidly progressive fibrotic disease of the liver, period,” CHLA hepatologist and gastroenterologist Keith Hazleton, MD, says. “It causes incredibly fast scarring of the liver.”
Though a congenital disease, biliary atresia can’t be detected before birth, but it’s typically identified early, since its symptoms—a yellowing of the skin and eyes—are apparent to anyone. And when caught early, an intervention called the Kasai procedure establishes bile flow to the intestines through a direct connection to the liver.
The surgery is ideally performed shortly after birth, but Selena’s case wasn’t caught until she was 6 months of age. Dr. Hazleton says that can happen because jaundice has other causes that are more common and harmless, including what’s called breast milk jaundice, where substances in breast milk disrupt the liver’s ability to process bilirubin.
A biopsy of Selena’s liver confirmed the diagnosis—a blockage in the bile ducts. Since she was not eligible for the Kasai procedure, the only option available was a transplant, and at 6 months old she was already a quarter of the way through the two-year range for her survival if her liver went untreated.
“Unfortunately,” Dr. Hazleton says, “this condition is fatal without treatment by two years of age—100% of the time.”
Since birth, E.J.’s oxygen saturation level had been consistently low, once dipping down to 33%, far below the desired 95% to 100%. Before the Glenn Procedure, his level averaged about 60% to 70%. Now it began rising. “He was very active, with a lot of energy,” Marie says.
A living donor emerges
To increase Selena’s chances at getting a liver transplant, doctors advised her mother, Liliana, to take Selena to Children’s Hospital Los Angeles so she could be a candidate for live donor liver transplantation, as the local pediatric center in Arizona could not offer that to her.
After being evaluated at CHLA in early spring of 2023, she was placed on the waiting list for liver transplantation, and the process of evaluating potential live donors began.
Meanwhile, Selena’s condition declined. “Her belly started getting round and hard, and her skin went completely yellow,” Liliana says. “Her eyes were a green-yellow. She was getting worse, not just on her labs and tests. You could see it happening.”
In late May 2023, Selena’s wait for a donor ended after a brief two months when she matched with Scotch, who three years earlier was introduced to organ donation after an idle chat with a man in a Louisiana bar ended in Scotch’s volunteering to give the man his kidney.
He has since become a vigorous advocate, riding his bike across the U.S. along “The Organ Trail,” as he named it, to bring attention to the dire need for donors, traveling the same routes taken by his donated organs. His first ride took him from his home in Madison, Wisconsin to Baton Rouge, Louisiana. Though his kidney ended up going to someone in New York because it didn’t match with the guy in the bar, the ride commemorated their fateful meeting.
To get his message out, Scotch pitches media outlets to cover his journeys. “An old guy riding a bike with one kidney,” he says. “Isn’t that an interesting story?”
The “Today” show and PBS, among others, certainly thought so. Additionally, transplant centers have joined with Scotch in promoting organ donorship as he rides through their town.
Scotch came into Selena’s life as the result of appearing at one such event at longstanding CHLA partner Keck Medical Center of USC, where he was encouraged to get an evaluation at the center’s Living-Donor Liver Program. He was rejected because doctors would need to remove too much of his liver to allow it to still function for Scotch. The portion they could safely take was not enough to give to an adult.
“I thought, well, I’m off the hook,” Scotch says.
Not so fast, he found out. His liver was just fine for donating to a child, who needed a much smaller cut of it. His name was placed on the living donor registry for liver in October 2022. Months later it matched with Selena.
Dual surgeries were performed May 25, 2023, timed so that the segment of Scotch’s liver was removed at Keck Hospital, transported to CHLA, and implanted into Selena within a matter of hours.
Doubts about an older donor
What distinguished Selena’s transplant was the age of the donor. At the time of the donation, Scotch was 67. That’s far past the age limit most hospitals apply to living donors, and on first consideration, seems illogical.
“Most centers would say less than 50 years old for the donor,” Dr. Etesami says. “He was probably one of the oldest donors in the country ever, if not the oldest.”
The idea of a 67-year-old man’s liver being suitable for an infant challenges common sense. One would figure that a liver depreciates like an automobile—the more usage, the more wear and tear on it, the less value it has.
“You’re thinking, the younger the donor, the more longevity there is in the organ,” Dr. Etesami says. “While generally true, an exact cutoff age is not really based on concrete science. There’s no 1-to-1 correlation between the age of the person and the age of their organ.”
“There’s no 1-to-1 correlation between the age of the person and the age of their organ.” — CHLA transplant surgeon Kambiz Etesami, MD
He explains that the liver, unique among all organs, renews itself. Only about 20% of an adult liver—the left lateral lobe—is taken and transplanted into an infant. The portion that was left alone will soon regrow almost in full, with new cells generating and original tissue enlarging. Additionally, since only a small piece of liver is removed, the surgery presents far less risk to the donor than if a larger segment was being removed for transplant into an adult.
Dr. Etesami notes that the health of the donor is more telling than the age. Scotch, who has competed in several ultra-endurance cycling events—“I’ve done 160-mile races in the middle of winter, 20 below zero,” he says—is in superior shape.
“If you have a perfectly healthy 60-something-year-old who wants to donate a small portion of the liver,” Dr. Etesami says, “although historically this hasn’t been done routinely, cases like Selena’s help to demonstrate that maybe they merit consideration.”
After the liver transplant
Following the same-day surgeries, Scotch had a note sent to Selena’s family to see if they would be interested in meeting him. Liliana responded that they were, and a week later both sides met at CHLA.
“I didn’t even know what to say,” Liliana says. “I did thank him. That’s the first thing I did. I thanked him and I hugged him.”
They keep up with each other’s lives over the phone. Selena, now 2 ½ and thriving, recognizes Scotch, even if she doesn’t know who he is yet.
“We show her pictures,” Liliana says. “I talked to Mark the other day and she heard his voice and she ran to the phone to say hello.
“We see him like a family friend. We reach out to him and tell him how grateful we are, especially on Selena’s birthday or holidays, or when milestones come around that we know we wouldn’t have reached without his generosity.”
A little over a year ago, Scotch visited Liliana’s family at their home. He left with a gift that he now keeps in his office.
“They made a couple of little posters for me,” he says. “One says, ‘Because of you, she lives.’ I’m looking at it right now. It’s a heart, a red heart.”
Mark Scotch joined CHLA and Keck Hospital of USC on April 4 at events celebrating National Donate Life Month. Afterward, Scotch began his bike ride back home to Wisconsin, retracing the route his liver took to get to Selena.
Best outcomes for pediatric liver transplants in the country
One-third of donated livers come from living donors
Tied for third-most pediatric liver transplants performed
How You Can Help
Anyone you know who might be making a move — refer them to the Corey Chambers real estate team. Not only will they benefit from our award winning service, but this very worthy cause will benefit as well. Corey Chambers 213-880-9910 helpkids@coreychambers.com www.ReferralsHelpKids.com
I am busy helping people find their new homes, thinking of how grateful I am for the value you bring to my real estate business. While looking around out in the world, we see blue skies emerging, and the recent cold days will soon be only a memory. It looks like Spring has sprung. For many across the country, it’s still cold, grey, and snowy! It very well could hail and snow more in the foothills around LA. But one thing is for sure, this winter season is coming to an end. It does every year without fail.
Just like the weather seasons come and seasons go, so do the seasons of life. I’m sure you have noticed as I have the older I get the faster they move by. These “seasons of life” go by so fast, my hope is that you enjoy each one. Yes, some will be hot and others will be cold highs and lows. The lows we want to move by quickly, the highs we want to stay in forever sometimes.
Corey Chambers, Broker
Please know that my team and I are eager to help anyone you know wanting to make a move. So much so that we are willing to make an offer that your referrals will LOVE – AND – the Kids at Children’s Hospital Los Angeles will love too.
Your referrals help the kids!
Go Serve Big!!! Investing In Our Southern Californian Kids
If you or a friend are thinking about selling, make sure to choose a real estate company you can trust! A real estate company with experience, proven results, and a give-back philosophy!
AND REMEMBER… Your referrals help the Kids…
We are on a mission to raise $25,000 for CHLA. We do this by donating a portion of our income from homes we sell. As you know, Children’s Hospital of Los Angeles does great work in helping kids fight through and survive serious life-threatening diseases like cancer, Non-Hodgkin’s lymphoma, leukemia and others. They also lead the way in helping kids come back from spinal cord injuries as well as early diagnosis of autism. Last year alone, Children’s helped over 1,000,000 kids right here in Los Angeles. BUT, Children’s relies on sponsorships and donations to provide their elite level of care, and to keep families’ expenses to a minimum. So YOUR REFERRALS REALLY DO HELP THE KIDS…
Who do you know considering buying or selling a home you could refer to my real estate sales team?
Not only will they benefit from our award-winning service, but you can rest assured we are also donating to a very worthy cause.
Go Serve Big!!! Investing in the Children of Los Angeles.
A Real Estate Company that Gives Back!
Children’s Hospital LA leads the way in serving kids one patient at a time.
We are still boldly on a mission to raise $25,000 for Children’s Hospital of Los Angeles, and we are making progress! We do this by donating to them a portion of our income from homes we sell. As you know, CHLA does AMAZING work in helping kids fight through and survive nasty diseases like cancer, Non-Hodgkin’s lymphoma, leukemia, and others. They also lead the way in many other fields.
They can provide this care and keep patient costs to a minimum due to donations and sponsorships. We are proud to support the Children’s Hospital of Los Angeles!
As in the attached story, Children’s Hospital of Los Angeles provides the best pediatric medical care available anywhere in the country. To do that, CHLA needs donations to continue its leading-edge care. We proudly donate a portion of our income from real estate sales to CHLA to help them continue serving the needs of those who most need it in our Los Angeles!
Who do you know considering buying or selling a home you could refer to our real estate sales team? Not only will they benefit from our award-winning real estate service, but a very worthy cause will also benefit as well. To refer anyone considering buying or selling a home just give me a call or pass on my number. 213-880-9910.
Thank you in advance for your referrals!
You and your referrals mean more than ever to my team and me. As we move forward thru this winter, please know we are extremely thankful for you and you being a special part of our business.
Go Serve Big!!! — Corey Chambers
Entar® Real Estate and Investment Technologies!
P.S. I copied and pasted the story below from the CHLA website. It better tells the story of the work they are doing.
MAKING A DIFFERENCE
As a leading charitable hospital, CHLA depends on sponsorships and donations to continue its leading-edge service. We proudly donate a portion of our income from real estate sales to CHLA to help them continue serving the needs of those who most need it in Los Angeles!
A real estate company with experience, proven results, and a give-back philosophy!
Over the years of helping many families sell their homes and/or buy another, we have met some wonderful, loving, caring people. People like you! So your referrals can rest assured that, not only will they get the award-winning service we are known for and the guarantee to back it up, but that a solid portion of the income we receive will go toward helping the kids.
Refer your friends, neighbors, associates or family members considering making a move:
You can go to www.ReferralsHelpKids.com and enter their contact info online or forward the link to someone you know considering a move.
Of course you can always call me direct as well at 213-880-9910
Why I support Childrenʼs Hospital Los Angeles
I grew up right here in the Greater Los Angeles Area, born in Los Angeles County at St. Francis Hospital. I remember when I first heard about a young person close to our family suffering from a nasty disease and getting treated for that at Children’s Hospital Los Angeles. It was then that I began to pay closer attention to the work they do at that hospital. Since then, I have learned that it is a collection of hard-working health care professionals, most making their home right here in the Los Angeles area, all coming together for a common cause. That cause is to help young people overcome unfortunate health issues that life sometimes throws our way. Being a Los Angeles Area California native, I take pride in supporting in a way that I can do the good work these people do at Children’s. My team rallies around our annual goal of raising money and donating portions of our income to help Children’s in their quest to heal young people when they need healing. My team and I are committed to providing outstanding results for buyers and sellers referred to us by our past clients. I have discovered that Children’s Hospital Los Angeles shares a similar commitment to their patients. And since their services survive on sponsorships and donations, we are happy to contribute and proud to support them.
Sincerely,
Corey Chambers
“Meaningful Hope:” Elevidys Gene Therapy Helps Eliot Move With Ease
At 4 years old, Eliot was the first patient at CHLA to receive Elevidys, a groundbreaking new gene therapy for Duchenne muscular dystrophy. The treatment has been a game changer for his health. | by Caitlin Kryl
Elizabeth was stunned.
She’d just watched her 4-year-old son, Eliot, jump fluidly up and down on his pirate ship (read: bed).
This might sound like the typical activity of a preschool-aged kid. For Eliot, though, it was extraordinary.
Throughout his early life, Eliot couldn’t properly jump, despite attempting often. He’d get winded walking down the block and had difficulty climbing stairs. These were the early manifestations of a genetic mutation linked to a severe degenerative neuromuscular disease, Duchenne muscular dystrophy (DMD).
Today, however, Elizabeth saw Eliot’s reality shift:
One month earlier, he had received a groundbreaking gene therapy called Elevidys at Children’s Hospital Los Angeles. After watching Eliot jump, she even called the neurologist who developed Elevidys to check if it were possible the therapy was already working. “I was very conscious of the placebo effect,” she says. “But no, I was seeing the therapy’s effect.”
Deep inside Eliot’s body, Elevidys was helping his muscle cells get healthy. Eliot’s body was restoring itself.
Life-improving Elevidys
Today, Eliot is a rambunctious 5-year-old who walks, runs, jumps, and climbs with relative ease—and who actually has to be reminded to slow down.
While Eliot may always have DMD, Elevidys is designed to significantly slow neuromuscular degeneration, and in turn, profoundly improve patients’ quality of life.
Elevidys gene therapy works through a one-time IV infusion. Doctors inject a non-disease-causing virus, called a viral vector, into the patient’s body. That vector’s job is to find the person’s muscle cells. Then, the vector delivers a modified gene that makes a shortened version of dystrophin, a critical protein that patients with DMD have trouble producing.
Early days
Eliot was diagnosed with DMD early and unexpectedly. At 5 months old, he spent five days in the hospital with a bout of RSV. Doctors mentioned noticing abnormal liver function values, which aren’t uncommon for viruses but can also indicate a neuromuscular problem.
When Elizabeth took Eliot to his follow-up appointment at CHLA a week later, his pediatrician, Eyal Ben-Isaac, MD, recommended further monitoring. Dr. Ben-Isaac referred Eliot to Leigh Maria Ramos-Platt, MD, Medical Director of CHLA’s Neuromuscular Disorders Center, for evaluation and genetic testing.
The diagnosis
In January 2020, Dr. Ramos-Platt called Elizabeth to confirm their worst nightmare. Eliot had DMD.
“There are no words to describe that feeling. It’s beyond sadness and grief,” Elizabeth reflects. “I remember saying, ‘I just need to know he’s going to be OK,’ and Dr. Ramos-Platt responding, ‘I think he’s going to be OK.’”
The words Dr. Ramos-Platt offered Elizabeth that January day weren’t platitudes. As the two spoke, researchers were trialing game-changing new treatments for DMD. CHLA had already sent children to participate in some of these clinical trials.
“At the time, her words didn’t give me immediate comfort,” says Elizabeth, “but it wasn’t the conversation I think she would be having with patients 10 years ago.”
“Watching the treatment landscape for DMD shift, I was very optimistic,” says Dr. Ramos-Platt. “It was so different from when I was a medical resident.” Until very recently, people with DMD had few treatment options aside from corticosteroids and physical therapy to reduce inflammation and prolong muscle function. Without more effective treatments, the disease could often lead to loss of motor function and ultimately total immobility, difficulty breathing, and shortened life expectancy.
“I remember saying, ‘I just need to know he’s going to be OK,’ and Dr. Ramos-Platt responding, ‘I think he’s going to be OK.’”Elizabeth
The wait
By March 2020, the COVID-19 pandemic had shut down businesses across the globe, slowing progress toward new gene therapies to a crawl. Both Elizabeth and Dr. Ramos-Platt kept close tabs on which trials were progressing, monitoring for any indication from the FDA that a drug would be approved within the next few years.
“We just had to watch and wait. And it was horrible,” Elizabeth says. “Eliot was too young for alternative treatments like steroids. He was so little. We just sat with this terrible information.”
The text
Shortly after Eliot’s fourth birthday, on Jun. 22, 2023, Elizabeth received the text from Dr. Ramos-Platt she’d been dreaming of for three years. Elevidys was officially FDA-approved for 4- and 5-year-olds.
Dr. Ramos-Platt and the Neurological Institute team were ready to spring into action, having conducted several test runs with the drug maker, Sarepta, a month prior. “We’d been building our plan since 2021 based on our experience administering the gene therapy Zolgensmafor spinal muscular atrophy,” Dr. Ramos-Platt recalls. “We didn’t have a concrete process yet for Elevidys—and as the first patient to receive the treatment at CHLA, Eliot’s journey would become our blueprint for patient No. 2, No. 3, and so on.”
Dr. Ramos-Platt introduced Elizabeth to treatment coordinators at Sarepta, and soon began an odyssey of approvals, consents, and back-and-forths with insurance. Eliot’s blood also needed to be tested for any viral antibodies that could interfere with the vector’s ability to deliver the gene therapy.
Once it was confirmed that Eliot was free of any conflicting antibodies, Elizabeth says the family lived their lives “in a bubble,” while staying in constant contact with Dr. Ramos-Platt.
Treatment day: “Like watching someone go to the moon”
To an objective observer, Eliot’s treatment might have looked like any number of routine IV infusions. Except, one day earlier, Eliot’s dose of Elevidys arrived at CHLA with its very own security detail, stored inside a massive, armored, refrigerated box set to exactly minus-76 degrees Fahrenheit.
On Aug. 29, 2023, Eliot, Elizabeth, and Eliot’s father Richard arrived at the CHLA Infusion Center as the pharmacy began thawing and preparing the medicine.
“It was the most extraordinary moment. Still, there were so many things about it that seemed ordinary,” Elizabeth reflects. Eliot was treated in a typical infusion room, sitting on a hospital bed with his stuffed animals like any other child. Inside Eliot’s room, Dr. Ramos-Platt introduced them to Susie Tatoy, RN, who has worked in CHLA’s Infusion Center for more than 40 years. Dr. Ramos-Platt says Tatoy is exactly whom you want by your side on a life-changing treatment day.
“I think almost the entire Neurology team showed up,” Elizabeth recalled. “Then I remember Susie saying, ‘All right, it started.’ And it’s just like … I don’t know … like watching someone go to the moon.”
“It was such an ordinary moment in some ways. And yet what’s happening is they’re transferring a gene to my son to give him something his body can’t produce. And he’s one of, I don’t know, a hundred or so kids who this happened to in the entire world,” says Elizabeth.
Eliot sat there quietly and patiently for the hour-long process and subsequent hours of observation. “It was mind-blowing,” Elizabeth adds. “It moves me every time I go back there in my memory.”
“It was such an ordinary moment in some ways. And yet what’s happening is they’re transferring a gene to my son to give him something his body can’t produce. And he’s one of, I don’t know, a hundred or so kids who this happened to in the entire world.” — Elizabeth
Recovery and milestones
For the next 16 weeks, Eliot and Dr. Ramos-Platt needed to be nearly inseparable. “I become the patient’s best friend,” she often tells families, as patients must undergo weekly tests to ensure their body is responding positively to the therapy. Eliot was a bit shy at first, but eventually, he and Dr. Ramos-Platt bonded over their mutual love of unique animals—especially guinea pigs.
Dr. Ramos-Platt explains that in the first week, patients tend to feel a bit of digestive discomfort, which might persist through the end of the month. She also notes that the care team needs to avoid confusing any early newfound energy with the effects of extra steroids. “But about a month later, they’re doing things that make me say, ‘OK, you didn’t do that before!’”
Before being treated, Eliot scored 22 out of 34 on the North Star Ambulatory Assessment (NSAA), a test used to assess motor function—which indicated that the disease had already begun to impair his movement considerably.
Six weeks after his treatment, he scored a perfect 34 out of 34.
“The physical therapist couldn’t believe her eyes,” Dr. Ramos-Platt recalls. They decided to have Eliot come back another day and conduct the test one more time after he’d stopped taking the extra steroids.
Once again: perfect score.
‘Eliot Day’
Aug. 29, 2024, marked the one-year anniversary of Eliot’s treatment—a day Elizabeth has coined “Eliot Day.”
The family acknowledges Eliot Day not with a cake or a celebration, but with quiet reverence.
“He could tell that things were changing,” says Elizabeth when asked how Eliot processed receiving what she calls “the magic medicine” as a preschooler. “He’s so aware of the seriousness of what happened, and I think it’s probably very overwhelming for someone so young.”
“Internally, as the parent of a child with something so serious, it’s one of those days that is just special,” Elizabeth says. “I think a lot about that day, I talk about it with my husband, and it’s more solemn than celebratory. I don’t mean that negatively—it was just such a profound experience. There is no party or celebration that could capture what gene therapy did for my son.”
I think a lot about that day, I talk about it with my husband, and it’s more solemn than celebratory. I don’t mean that negatively—it was just such a profound experience. There is no party or celebration that could capture what gene therapy did for my son. — Elizabeth
Not fantastical hope, but meaningful hope
Elizabeth encourages other families grappling with a DMD diagnosis to acknowledge that today’s treatment for DMD is “a whole new world and trajectory” than even five years ago.
“Someone once said to me, ‘The best treatment is the one in front of you.’ Do I think that there may be better treatments in 10 years? That is a possibility. But right now, we have a treatment that could be life changing for many of these kids.”
“It’s important to believe the science—to believe there is not fantastical hope, but meaningful hope. If Eliot is any poster child, gene therapy has been a game changer for him.”
Learn more about gene therapy at Children’s Hospital Los Angeles.
— Story and photos courtesy Children’s Hospital Los Angeles
How You Can Help
Refer your friends, neighbors, associates, or family members considering making a move: www.ReferralsHelpKids.com or call Corey at 213-880-9910
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