‘His legacy lives on’: Grandmother who helped create newborn screening law tells history of bill – News-Leader
By daniellenierenberg
Buy Photo
Two-year-old Regann Moore lights up as she watches videos on her iPad at home on Thursday, Feb. 20, 2020. Moore has a rare disease known as Krabbe Disease and received a life-saving stem cell donation less than a month after being born.(Photo: Nathan Papes/Springfield News-Leader)
Soon after the News-Leader published a story about 2-year-old Regann Moore,a Springfield child whose life was saved thanks to a newborn screening test, someone tweeted the story toMissouri State Rep. Becky Ruth.
"I bawled my eyes out," Ruth said. "I just cried."
She cried because she knew Regann is alive thanks to the death of Ruth's grandson, Brady.
"I cry and smile when I see these children," Ruth said. "We are always so thankful. For us, we see Brady's death wasn't in vain. His legacy lives on by helping save the lives of other children."
More: Springfield child with rare, deadly disease continues to amaze doctors, family
Regann, who is 2 now, was diagnosed right after she was born withKrabbe Disease, a rare metabolic disorder that must be diagnosed at birth and treated as soon as possible with a stem cell donation.
The newborn screening is important because babies with Krabbe Disease appear healthy at birth. Signs something is wrong usually don't appear until it's too late for treatment to be effective.
That is what happened to Brady in 2009. He wasn't diagnosed with the disease until he was 4.5 months old too late for treatment.
Brady died 10 days before his first birthday.
Brady Cunningham died of Krabbe Disease just before his first birthday.(Photo: Courtesy of the Cunningham family)
That's why Ruth and her family fought to get lawmakers on board with making sure all newborns in Missouri are screened for Krabbe Disease.
TheBrady Alan Cunningham Newborn Screening Act was passed in 2009 and screening began in 2012. Ruthsaid her family was OK with the three-year lag because they realized the lab needed time to become equipped to test for the disease.
Missouri is one of just a few states that do the newborn screening.
Brady's law also includes screening for Pompe, Fabry, Gauche and Niemann-Pick diseases. Since then, SCID, MPS I, MPS II and SMA diseases are screened, as well.
Ruth became a state representative in 2015and said newborn screening is her passion.
Her experience with getting Brady's law passed is what led her to seek office.
"It showed me what just a regular everyday person can do and what a differenceyou can make," Ruth said. "People a lot of times complain about politicians and the legislature, but we also do very good things here."
Ruth said her family knows of another child with Krabbe Disease who was saved thanks to newborn screening and a stem cell transplant.
That child is now 4. Ruth said her family and that child's family have a "strong connection."Ruth said shehopes to someday meet Regann's family.
Brady Cunningham was born in 2008. His family is from Campbell in southeast Missouri.
Bradyappeared healthy at birth and was not tested for Krabbe Disease.
Ruth said he started having health problems after about a month and a half. Brady went through "a myriad of diagnoses," Ruth recalled, including acid reflux and seizures.
"Finally my daughter took him to Children's Hospital in St. Louis," she said. "They promised her he wouldn't leave without a diagnosis."
Missouri State Rep. Becky Ruth was moved to tears after reading about Regann Moore, a Springfield child whose life was saved thanks to newborn screening for Krabbe Disease. Ruth and her family encouraged Missouri lawmakers to make sure all Missouri babies are tested for the deadly disease after her grandson, Brady, died from it.(Photo: Submitted by Becky Ruth)
Three weeks later, Brady was diagnosed with Krabbe Disease, which rapidly destroys the nervous system.
"We were told there was nothing they could do," she said. "It was one of the worst days of all of our lives."
Brady was 4.5 months old when he was diagnosed. In order for a stem cell donation to have any chance of being effective, babies must have the transplant within the first month of their life.
Regann, the Springfield child, was given a stem cell donation thanks to an umbilical cord donation.
Thediseaseaffects about one in every 100,000 people in the United States.
"They are missing an enzyme that helps keep their nervous system intact," said Dr. Shalini Shenoy, Regann's transplant doctor. "Because this is missing, they have degeneration of the brain and nervous system. And if you let it progress, it is fatal very early."
Without the stem cell donation, babies die within the first few months, Shenoy said.
"You can't change someone's genetic makeup," Shenoy said. "But when you put stem cells into their bone marrow from somebody else who is normal, some of these cells migrate into their brain and into their nervous system and supply what they are lacking themselves."
It takes some time for the transplant to begin working for the transplanted cells to "settle down" and begin making the missing enzyme, Shenoy said.
"Because of that, the earlier you transplant a Krabbe patient, the more you will be able to rescue them," she said. "You want to catch them before too much damage is done. Once there's a lot of nerve damage, it's not reversible. If I saw a Krabbe patient two months after they were born or four months after they were born when they already had major problems, it's unlikely I'd be able to rescue them too much."
Since the screening and the stem cell transplant treatment are both relatively recent medical advancements, Shenoy said it's anybody's guess what the future will hold for children who, like Regann, were successfully treated with a stem cell transplant early on.
Ferrell Moore holds his two-year-old daughter Regann Moore at their home on Thursday, Feb. 20, 2020. Regann has a rare disease known as Krabbe Disease and received a life-saving stem cell donation less than a month after being born.(Photo: Nathan Papes/Springfield News-Leader)
Regann can't stand on her own or walk yet. But her family is determined to make that happen. She cannot talk but is learning sign language to communicate.
She has regular visits with speech and occupational therapists.
Regann's dad Ferrell Moore got to take her to the circus recently, something the little girl seemed to enjoy.
"She is the joy of my life," Ferrell Moore said. "When I come home, it couldn't be any better to see her and how happy she is to see me."
Read or Share this story: https://www.news-leader.com/story/news/local/ozarks/2020/03/05/grandma-who-helped-pass-newborn-screening-law-tells-story-bill/4954655002/
- Rejuvenating the immune system by depleting certain stem cells - National Institutes of Health (NIH) (.gov) - April 19th, 2024
- New gene therapy eliminates need for bone marrow transplant. Here's how it works. - CBS News - April 19th, 2024
- New gene therapy eliminates need for bone marrow transplant. Here's how it works. - MSN - April 19th, 2024
- Long Island boy with rare blood disorder undergoes gene therapy - MSN - April 19th, 2024
- Philadelphia Wings player, Connecticut man will be forever bonded by bone marrow donation: "He's my hero" - CBS Philly - April 10th, 2024
- VRD versus VCD as induction therapy before autologous stem cell transplantation in multiple myeloma: a nationwide ... - Nature.com - April 10th, 2024
- Register as a bone marrow donor today and save lives - The Citizen - April 10th, 2024
- Resilient anatomy and local plasticity of naive and stress haematopoiesis - Nature.com - March 26th, 2024
- A Deeper Depth of Response After Salvage Therapy Improves Outcomes of Autologous Stem Cell Transplantation in ... - Cureus - March 26th, 2024
- Iron restriction keeps blood stem cells young, researchers find - Phys.org - March 18th, 2024
- Blood drive, bone marrow testing to be held in local woman's memory - The Winchester Star - March 18th, 2024
- Signal of Benefit for Stem Cell Therapy in Progressive MS - Medscape - March 10th, 2024
- Woman, 22, With Leukemia Recalls Symptoms And New Treatment She Received: EXCLUSIVE - TODAY - March 10th, 2024
- This Swedish startup wants to reduce the cost, and controversy, around stem cell production - TechCrunch - March 10th, 2024
- Outcomes and prognosis of haploidentical haematopoietic stem cell transplantation in children with FLT3-ITD mutated ... - Nature.com - March 10th, 2024
- Harmonizing definitions for hematopoietic recovery, graft rejection, graft failure, poor graft function, and donor ... - Nature.com - March 10th, 2024
- Hematopoietic cell transplantation and cell therapy activity landscape survey in the Kingdom of Saudi Arabia; a report ... - Nature.com - March 10th, 2024
- How an MS friendship led to HSCT and a love of running - Multiple Sclerosis News Today - March 10th, 2024
- Iron Limitation Preserves Youthfulness of Blood Stem Cells - Mirage News - March 10th, 2024
- Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the ... - Nature.com - March 10th, 2024
- AJMC in the Press, February 23, 2024 - AJMC.com Managed Markets Network - February 24th, 2024
- Orca Bio Presents Promising Data on Orca-T in Two Oral Presentations at the 2024 Tandem Meetings of ASTCT and ... - Yahoo Finance - February 24th, 2024
- New approaches to live-track the production of different types of blood cells in mice - Medical Xpress - February 24th, 2024
- If Other Treatments Aren't Working -- Stem Cell Transplant May Be A Good Option In CLL - SurvivorNet - February 24th, 2024
- Expanding the Horizons of Cell and Gene Therapy - RegMedNet - February 24th, 2024
- The strangers who saved each others lives - BBC - February 24th, 2024
- City of Hope Research Featuring the Successful Treatment of the Oldest Patient to Achieve Remission for Leukemia ... - StreetInsider.com - February 15th, 2024
- 3D printing and material processing combined to create artificial bone - Optics.org - February 15th, 2024
- Man, 63, is in remission from HIV five years after receiving groundbreaking stem cell transplant... - The Sun - February 15th, 2024
- Team demonstrates fabrication method to construct 3D structures that mimic bone microstructure - Phys.org - February 15th, 2024
- Hematopoietic Stem Cells and Their Role in Development and Disease Therapy - The Scientist - February 15th, 2024
- Blood cell family trees trace how production changes with aging - MIT News - February 7th, 2024
- New study on promising stem cell-based therapy for Crohn's disease - Medical Xpress - January 30th, 2024
- Second haploidentical bone marrow transplantation with antithymocyte antibody-containing conditioning regimen for ... - Nature.com - January 30th, 2024
- Stem cell study shows how gene activity modulates the amount of immune cell production in mice - Medical Xpress - January 30th, 2024
- Global Stem Cell Therapy Industry Outlook to 2028, Driven by Therapeutic Innovations and Clinical Advancements ... - Yahoo Finance - January 30th, 2024
- 1st-of-its-kind therapy blocks immune attack after stem-cell transplant - Livescience.com - January 22nd, 2024
- Individualized dose of anti-thymocyte globulin based on weight and pre-transplantation lymphocyte counts in pediatric ... - Nature.com - January 22nd, 2024
- Implications of stress-induced gene expression for hematopoietic stem cell aging studies - Nature.com - January 22nd, 2024
- LVHN announces opening of new stem cell transplant center. Here's what that means for the Lehigh Valley - The Morning Call - January 22nd, 2024
- Fast Five Quiz: Chronic GVHD Risk Factors and Prevention - Medscape Reference - January 22nd, 2024
- Could Treatments for HIV and Sickle Cell Open the Gene Therapy Floodgates? - BioSpace - January 22nd, 2024
- Effects of fine particulate matter on bone marrow-conserved hematopoietic and mesenchymal stem cells: a systematic ... - Nature.com - January 14th, 2024
- Donating Bone Marrow and Stem Cells: The Process and What To Expect - On Cancer - Memorial Sloan Kettering - January 14th, 2024
- No, Rep. Steve Scalise Didn't Vote Against Stem Cell Research From Which He Is Now Benefiting - Yahoo News - January 14th, 2024
- Hematopoietic Stem Cell Transplantation Market to Grow Rapidly During the Study Period (2019-2032), Evaluates ... - PR Newswire - January 14th, 2024
- Life-saving donation from Philly athlete saves life: 'Feeling so strong, I owe that all to him' - AOL - January 14th, 2024
- The Key to Creating Blood Stem Cells May Lie in Your Own Blood - ScienceAlert - January 14th, 2024
- Dr Phillips on the Rationale for the GLOBRYTE Trial in Relapsed/Refractory MCL - OncLive - January 14th, 2024
- COVID-19 and HSCT Recipients: Risk Factors and Prevention Measures - Medriva - January 14th, 2024
- Bone Marrow Transplant: Heres What You Need To Know About This Therapy - Times Now - January 5th, 2024
- New insights about the development of hematopoietic stem cells - Drug Target Review - December 28th, 2023
- Bone Marrow Transplantation | Johns Hopkins Medicine - December 20th, 2023
- Stem Cell or Bone Marrow Transplant | American Cancer Society - December 20th, 2023
- Embryonic-stem-cell-derived mesenchymal stem cells relieve experimental contact urticaria by regulating the functions ... - Nature.com - December 20th, 2023
- Researchers discover crucial step in creating blood stem cells - Phys.org - December 20th, 2023
- A niche topic: understanding the development of hematopoietic stem cells - Fred Hutchinson Cancer Center - December 20th, 2023
- Vertex developed a CRISPR cure. Its already on the hunt for something better. - MIT Technology Review - December 20th, 2023
- FDA approves cure for sickle cell disease, the first treatment to use gene-editing tool CRISPR - NBC News - December 12th, 2023
- First therapy using CRISPR technology will treat sickle cell disease - Morning Brew - December 12th, 2023
- 7 medical breakthroughs that gave us hope in 2023 - National Geographic - December 12th, 2023
- Understanding Chronic Myeloid Leukemia: Causes, Symptoms, and Treatment - Everyday Health - December 12th, 2023
- Mansour bin Zayed witnesses inauguration of ADSCC Bone Marrow Transplant & Cellular Therapy Congress 2023 - ZAWYA - November 26th, 2023
- ADSCC Bone Marrow Transplant and Cellular Therapy Congress 2023 to take place in Abu Dhabi - ZAWYA - November 18th, 2023
- Orchard Therapeutics Reports First Quarter 2023 Financial Results and Announces Initiation of Rolling Submission for Biologics License Application of... - May 16th, 2023
- Family of 7-month-old in need of bone marrow transplant hosting donor registration event - CBS Pittsburgh - May 8th, 2023
- Anika Continues to Expand Addressable Market for Tactoset Injectable Bone Substitute with Additional 510(k) Clearance from FDA - Marketscreener.com - April 5th, 2023
- MorphoSys Completes Enrollment of Phase 3 MANIFEST-2 Study of Pelabresib in Myelofibrosis with Topline Results Expected by End of 2023 -... - April 5th, 2023
- VOR BIOPHARMA INC. Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-K) - Marketscreener.com - March 25th, 2023
- BioRestorative Therapies to Seek FDA Approval to Expand the Clinical Application of BRTX-100 - Marketscreener.com - March 17th, 2023
- BioSenic delivers a new post-hoc analysis of its Phase III JTA-004 trial on knee osteo-arthritis with positive action on the most severely affected... - March 17th, 2023
- JASPER THERAPEUTICS, INC. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS (form 10-K) - Marketscreener.com - March 9th, 2023
- For a range of unmet medical needs, India offers a fantastic opportunity to push cell and gene therapies: B .. - ETHealthWorld - March 9th, 2023
- NGM BIOPHARMACEUTICALS INC Management's Discussion and Analysis of Financial Condition and Results of Operations. (form 10-K) - Marketscreener.com - March 1st, 2023
- Bone health: Tips to keep your bones healthy - Mayo Clinic - January 27th, 2023
- Bone marrow drive held for military wife with cancer - January 27th, 2023
- Bone cancer - Symptoms and causes - Mayo Clinic - January 27th, 2023
- Bone | Definition, Anatomy, & Composition | Britannica - January 19th, 2023
- Bone Definition & Meaning - Merriam-Webster - January 19th, 2023
- What Is Bone? | NIH Osteoporosis and Related Bone Diseases National ... - January 19th, 2023