Cord Blood Used to Treat Child with Hurler Syndrome, Genetic Disorder

Cord Blood Used to Treat Child with Hurler Syndrome, Genetic Disorder

Meet Lyla Edgington. Lyla was first diagnosed with Hurler Syndrome (MPS Type I) on April 4th, 2016 when she was just 7 months old.  Her doctors said she needed a stem cell transplant and that cord blood would give her the best chances at overcoming this life-threatening genetic disorder.

Lyla’s doctors and parents began to worry when their pediatrician, Dr. Katherine Lichtsinn at Scottsdale Children’s Group noticed a heart murmur during her 4 month check-up. Returning for another check-up at 6 months, Helen, Lyla’s mother, pointed out a sharp curve in Lyla’s spine that she had noticed. It was then that her doctor suspected a possible genetic disorder and referred them to a specialist at Phoenix Children’s Hospital.

In Phoenix, Lyla underwent numerous blood tests hoping to rule out MPS. Unfortunately, this would not be the case. Not only were their fears confirmed but the medical team concluded that Lyla had the most severe form of the disease, MPS Type 1, better known as Hurler Syndrome or Hurler’s disease.

Our world came crashing down that day, but luckily the weeks to follow were full of good news. To say life was like a storm on April 4th, 2016 on the day we received Lyla’s diagnosis, is an understatement…..the tears flowed like a monsoon, my body shook like an earthquake, and the life I envisioned for my daughter was torn to the ground. I truly thought our world had ended.Helen Edgington, Lyla's Mother

Helen was devastated, and worse, she felt responsible even though there was nothing she could have done differently.  She and her husband, Steve, had given birth to a beautiful little girl. She was perfect in every way. Hundreds of questions came pouring through the emotions as they wondered “Why us?”

MPS I (Hurler, Hurler-Scheie, Scheie syndrome)
MPS I, along with six other MPS diseases is a mucopolysaccharide disease that is relentlessly progressive and potentially fatal. MPS I has also been called Hurler, Hurler-Scheie and Scheie syndrome . Hurler takes its name from Gertrude Hurler, the doctor who described a boy and girl with the condition in 1919. In 1962, Dr. Scheie, a consultant ophthalmologist, wrote about some of his patients who were more mildly affected. Individuals who seem not to fit clearly in either the severe or the mild end of the disease were said to have Hurler/Scheie. The specific disease names have been replaced with the designations attenuated (diminished severity) and severe MPS I. There is no cure for MPS diseases, but there are ways of managing and treating the problems they cause.

(Source: National MPS Society, )

Child with Hurlers Syndrome treated with Cord Blood

Lyla, diagnosed at 7 months with MPS Type 1 (Hurler Syndrome)

Helen still remembers those initial hours, “As a parent your duty is to protect your child and I had failed within the first moments she was created. I passed on a defective gene that would cripple her body, deteriorate her brain, and end her precious life after it had barely begun. I held my beautiful 7 month old daughter so tight. I sobbed because I couldn’t bear to watch her smile fade away and her energy disappear day by day over the next couple years. It wasn’t possible. Lyla was too perfect, she had too much personality, she was too smart, and she could already throw a tennis ball so well. But at that moment… Lyla was still Lyla and I couldn’t help but smile when she did and enjoy every moment with her. Everything had changed but nothing had changed.”

Right away, Dr. Kristin Lindstrom at the Phoenix Children’s Hospital recommended a stem cell transplant. Initially, they thought this meant a bone marrow transplant and Dr. Lindstrom referred them to Dr. Paul Orchard at the University of Minnesota. Dr. Orchard explained that transplants for MPS patients have greatly improved over the last several years. He explained that cord blood could be the best choice for Lyla and give her the best chances for a successful transplant.

The goal was not simple. In order to be successful, the transplant needed to rid Lyla’s body of her own cells (because they were genetically faulty) and replace her cells with healthy new ones. Although the actual transplant lasts only a few minutes, the whole healing process would be quite long and span several months.

Helen and Steve were encouraged by this possible treatment and agreed that this was their best option for young Lyla. The wheels were put in motion. They would go to Minnesota for treatment. Lyla’s parents received approval from insurance and doctors began to search for a donor. It was a race against the clock as the disease was constantly working against them, deteriorating brain cells. Luckily, a match was found and it was a perfect 6 out of 6 match! The transplant date was set for July 25th, 2016.

The procedure lasted 25 minutes and was much like a typical blood transfusion. In accordance with donor rules, Lyla’s parents were not told where the donor was born or the identity of the generous family. They only know that the donated cord blood came from a little girl born in May 2013. She could be from anywhere in the world.

Looking back the reality of the situation, it should have been much scarier than it was. But, we were just so happy and thankful Lyla had the option of a stem cell transplant and that she had a perfect 6 out of 6 cord blood match. We knew there was a long road ahead, but we were very optimistic!Helen Edgington, Lyla's Mother
Hurlers Syndrome Treated with Cord Blood

A cord blood donation gave a new life to Lyla.

Hurler Syndrome is a progressive genetic disease. If you don’t treat it, it will gradually cause severe physical and mental damage. Lyla’s parents knew this and this is why timing was so crucial in her case. Luckily, Lyla was diagnosed early and did not have too much damage prior to the transplant. This meant her chances were good. However, because of the nature of the disorder, Lyla’s parents and doctors were not expecting to see major improvements necessarily in her mental and physical functions but what they hoped was that the progression of the disorder would stop. The goal was to stop the disorder from becoming worse. Questions remained as to if some damage would continue to occur following transplant or would the transplant be effective immediately?

The first few weeks after the transplant were really difficult because of the impact of the chemotherapy. Lyla suffered from low energy and clearly her spirits were down. However, after the first month, doctors confirmed that the transplant had fully engrafted (the donor cells had been accepted by her body). Success!  From there it is was smooth sailing. . . she returned to being a happy girl most of the time while living under constant medical supervision (remember, she was not even a year old at the time).

When discussing stem cell transplants, doctors often refer to the “first 100 days.” Although not always applicable to every case, this milestone often indicates whether a transplant has been “successful” or not. In Lyla’s case, she was doing great and around day +95 her parents started preparing for their flight home. Then, the phone rang. Lyla’s blood tests came back with issues.

Lyla’s parents explained, “The transplant worked but for some unknown reason the new white cells were creating antibodies against the new platelets and red blood cells. Lyla’s immune system needed to be wiped out again to kill the antibodies and we had to hope the new ones wouldn’t do the same thing. This was a huge bump in the road for us and really tore down our spirits. It took another very long 5 months of Lyla on high dose steriods, tons of infusions, very little sleep, and at a huge risk for infection but luckily it worked and the bad antibodies didn’t return! Overall, it took a full 9 months after transplant to know that it was an overwhelming success.”

Post transplant Lyla is developing normally.

Post-transplant, Lyla has been blossoming!

Success. What a magic word! Success. This word meant hope to the Edgingtons. Success, meant their daughter might beat the odds. Success meant life and quality of life!

Today, the Edgingtons are just starting to turn the corner on this battle but they have been very encouraged by the results. The transplant worked and despite some complications around day 95, it appears that the transplant stopped damage caused by the disorder almost immediately. Based on various tests, Lyla did not experience any further damage post-transplant.

“She is developmentally on track,
so we feel confident the transplant
stopped damage to her brain,”
says Lyla’s mother.

Her parents realize how lucky they are. The stars aligned for Lyla. She had excellent doctors. She had a 6 out 6 stem cell match, thanks to cord blood. She had pro-active parents and grandparents who did everything they could to help her. In the end, it literally took everyone but they have been fortunate in many ways.

Looking to the future, we asked Helen what advice she has for parents, health professionals and even students interested in pursuing STEM careers with an interest in cord blood.

For Parents:

My advice for parents is to never ignore anything that seems odd about your baby. It took me a month or so to mention the curve in Lyla’s spine because I thought it could have been a normal baby thing or something she would grow out of. 

For Health Professionals:

My advice for health professionals, is to continue to do the amazing work you’re doing and saving so many lives! I have so much appreciation for the medical field and doctors who devote their life to finding cures to all the horrible diseases out there. It’s truly incredible!!

For STEM Students:

For students, I encourage you to use your social media skills and networks to help raise awareness for cord blood donation and it’s amazing benefits!

In honor of Lyla’s donor and to help build awareness for MPS / Hurler Syndrome as well as the need for cord blood donors, Helen and Steve have created a beautiful video detailing Lyla’s story. Watch it here:

Make a difference in your community.
Join us for World Cord Blood Day on November 15th, 2017!
Learn more, click here.



A Father’s Story: Cord Blood Saved His Son’s Life

A Father’s Story: Cord Blood Saved His Son’s Life

Erik Praskins spoke about his son’s battle with leukemia at the Arizona Cord Blood Conference 2017 held in Phoenix, Arizona. His story is not unique yet, it is hard for many of us to imagine. Watch Erik tell his story below. . .

Children are sometimes described as “angels among us.” Indeed, every parent has felt that emotional moment of amazement looking into their child’s eyes or watching them discover something new in the world. Everytime they walk in the room, our perspective on the world changes. In their innocence, we are reminded of all that is good in the world. Perhaps, this is why we are always so shocked and saddened to hear about children fighting cancer? It seems so unjust (and it is).

According to the American Cancer Society (source:, leukemia is the most common cancer in children and teens. Within the leukemia category, the most common are acute lymphocytic leukemia (ALL) and acute myeloid leukemia (AML). Although 5-year survival rates have increased, each year more than 2,000 children under age 19 die from cancer in the United States alone (source:

In a recent talk at the Arizona Cord Blood Conference, Erik Praskins spoke openly about how his son’s life was saved thanks to a generous cord blood donation from a family he will probably never meet. His son, Dylan, was only 2 months old when he was diagnosed with leukemia, specifically ALL. His case was very serious. He was considered high-risk and required a stem cell transplant immediately. When a bone marrow donor could not be found, doctors turned to cord blood. Cord blood is more easily matched than bone marrow. It is also readily available if stored at birth. Recent studies have also suggested that a cord blood transplant for high-risk patients results in fewer cases of relapse (source:

Dylan was lucky in so many ways. His parents and doctors were quick to identify the cancer. They worked tirelessly to find an appropriate treatment for him. Little did they know on day one of this battle, that it would be the birth of another child somewhere in the world who would give Dylan a second chance. That child’s family literally gave life twice by donating their child’s cord blood instead of simply throwing it away. For this, Dylan’s family is eternally grateful. Just watch Erik’s face at the end of this video and you will see the gratitude spill over.

Perhaps their are angels among us?

Learn more about Dylan’s story here.


Save the Cord Foundation is proud to work with and support public cord blood donation programs around the world. We are especially proud of our work with the Arizona Public Cord Blood Program in our home state.
Find out how you can donate to this amazing public program
that helps patients, like Dylan, in Arizona and across the globe.


Cord Blood Experts Discuss Educational Approaches for Parents, Health Professionals & Students

Cord Blood Experts Discuss Educational Approaches for Parents, Health Professionals & Students

cord blood world europe 2017 educational approaches roundtableSave the Cord Foundation recently had the honor of attending Cord Blood World Europe 2017, part of the World Advanced Therapies & Regenerative Medicine Congress and World Precision Medicine Congress, held in London. This was a fantastic opportunity to hear excellent speakers such as Dr. Joanne Kurtzberg, Dr. Colleen Delaney, Dr. Paul Veys, Dr. Guy Sauvageau and many others speak about cord blood research and current uses for cord blood in the medical world. In addition, topics went beyond cord blood at times as experts discussed processing and uses for cord tissue as well as other perinatal stem cells. Regenerative medicine was a recurring theme throughout.

A great emphasis was placed on interacting with the expert presenters via lively roundtables and open panel discussions. Save the Cord Foundation was invited to host a roundtable discussion called “Educational Approaches.” We worked with attendees on new educational approaches addressing a variety of audiences, namely health professionals, expectant parents and our next generation of STEM professionals. Several of the participants in this roundtable discussion agreed to share their thoughts with the Save the Cord Foundation community. Below is a quick summary based on some of the most talked about presentations:

One of the most exciting presentations was made by Dr. Colleen Delaney of Nohla Therapeutics and Fred Hutchinson Cancer Research Center. Dr. Delaney presented “Ex-vivo Expanded Hematopoietic Stem/Progenitor Cell Therapies: The Road from a Patient Specific to Universal Donor Approach.” Participants in our roundtable commented that there were several key points made in this presentation that health professionals and parents needed to know about, for example:

After hearing Dr. Delaney’s presentation, I feel parents and health professionals need to realize why cord blood expansion techniques and the concept of the ‘universal donor’ which Dr. Delaney is exploring are so necessary. In particular, compared to patients of Caucasian descent, if a patient of mixed ethnicity is trying to find a match today the odds go down drastically. It is important for parents to know why this cord blood expansion technology led by Dr. Delaney is important and that happily it is perhaps just a few years away from being market ready. That potential is very encouraging.Mara Lucato (Create Cord Blood Bank Canada -

Another interesting presentation was made by Chiranya (Anjie) Prachaseri of Cryoviva in South East Asia, called “Perinatal Stem Cell Banking- Uses and Future Opportunities – A South East Asian Perspective.” This presentation emphasized the on-going problem with press and media coverage of cord blood which can often mislead parents and doctors. Prachaseri also discussed the fact that many parents do not know about the various accreditation groups who play a very important role in the industry.

Although the presentation focused mainly on South East Asia, Dr. Mareike Uhlmann from Stemlab, who participated in our roundtable, agreed it is crucial. . .

to ensure that the bank you want to use for storage is properly accredited and certified to guarantee that the parent’s hopes won’t be shattered in case a treatment is needed.Dr. Mareike Uhlmann (Stemlab -

Andre Gomes, also from Stemlab, stated how important it is to address the misinformation that is being given to our youth – the next generation of scientists, doctors, policy makers, etc. He applauded Prachaseri’s efforts of speaking to university students about cord blood and stem cell applications.

This has to be explained. More information must be given to young people otherwise we will always be fighting misinformation and confusing press. We need to move from the idea that ‘stem cells are bad’ to a discussion about the good and bad reasons for stem cell storage.Andre Gomes (Stemlab -

Finally, another presentation that prompted interesting discussion was made by Joana Correia of Exogenus Therapeutics. Correia presented “Umbilical Cord Blood Supply for the Development of Exo-Wound, an Exosome-based Product for Chronic Wounds.” Based on this presentation, roundtable participants saw opportunity for future STEM professionals who want to explore cellular expansion, delivery mechanisms, exosome extraction or even the shifts between translational and clinical applications. While these opportunities cannot be denied, several roundtable participants expressed caution reminding the group that cord blood stem cells are truly precious and should be used wisely.

Our collective responsibility, as scientists and educators, is to ensure that parents understand the true value of their newborn’s stem cells.Dr. Ayub Lulat (Create Cord Blood Bank Canada - )
Challenges in cord blood education

Experts discuss challenges in cord blood education

We, at Save the Cord Foundation, could not agree more. Cord blood is rich in stem cells. If collected and stored properly, these stem cells could be currently used to treat more than 80 different life-threatening diseases including many blood cancers. Compared to bone marrow, it is much easier to match and carries less risk of Graft versus Host Disease. Impressive research is also underway around the globe to test cord blood’s potential in regenerative medicine. For example, there is ground-breaking research to potentially treat spinal cord injury, traumatic brain injury, hearing loss and diabetes all using cord blood.

This is why we tell every parent. . . do everything you can to save your baby’s cord blood. If you can donate it, then donate it. If you prefer to privately store it (for eventual use by your child or family member), then do it. Be proactive and plan ahead. Please don’t throw this valuable medical resource away. Save the cord.

Meet another cord blood expert: Dr. Curtis Cetrulo, Sr.

Learn about the current uses of cord blood and perinatal stem cells. . . click here.



About Save the Cord Foundation

Save the Cord Foundation, a 501c3 non-profit organization, was established to promote awareness of the life-saving benefits of cord blood based on unbiased and factual information. The Foundation educates parents, health professionals and the general public about the need to preserve this valuable medical resource while providing information on both public cord blood donation programs and family cord blood banks worldwide.

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