In every visible way, Marley Gaskins is an average 12-year-old she enjoys painting, playing online games like Roblox with her friends and taking ukulele lessons. But until recently, her life was far from normal.
Marley was born with a one-in-a-million genetic disorder called leukocyte adhesion deficiency-1, or LAD-1, which cripples the immune system and results in recurring infections, coupled with slow wound healing.
She started getting what looked like ant bites on her skin when she turned 1, said Marleys mother, Tamara Hogue. When she was 3, she got a really big skin abscess on her stomach that landed her in the hospital for five weeks because it was just a massive infection they couldnt control. The infections, Tamara said, continued to get worse and more frequent, and Marley eventually needed round-the-clock attention.
Due to a defective gene, Marley was missing a protein that enables white blood cells to stick to blood vessel walls a crucial step these cells take before moving outside the vessel walls and into tissues to fight infections. Most kids with Marleys disorder, if untreated, die before the age of 2. The fact that she survived well past that mark without a diagnosis is remarkable in itself.
When Marley was officially diagnosed at age 8, after years of uncertainty and hospital stays, doctors in her home state of Florida said the only possible cure would be a bone marrow transplant from a matched donor. Tamara said Marleys doctors couldnt even provide a survival rate for LAD-1 patients who undergo bone marrow transplants because so few people are diagnosed with the disorder.
I just felt like there had to be another option, and that's when I started asking for a second opinion, Tamara said.
A new hope: Gene therapy at UCLA
Tamaras search for other treatment options led her to Dr. Donald Kohn, a physician-scientist and member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.
He was leading a new clinical trial sponsored by Rocket Pharmaceuticals and the California Institute for Regenerative Medicine, the states stem cell agency for children with LAD-1 in which doctors collect blood-forming stem cells with the defective gene from each child, add in a healthy copy of the gene in the lab and then return the corrected cells into the childs body. The therapy works by promptingthe childs body to create a continuous supply of healthy white blood cells capable of fighting infection. And because the corrected cells are the patients own, there is no risk of rejection, making the treatment far less risky than a bone marrow transplant.
By the time a patient gets a bone marrow transplant, theres a lot of infection and they may have already had lung problems or other complications, said Kohn, a distinguished professor of microbiology, immunology and molecular genetics in the UCLA College and of pediatrics and molecular and medical pharmacology at theDavid Geffen School of Medicine at UCLA. They have manyskin lesions and those tend to be the sites where the donor cells attack the recipients body in whats known as graft-versus-host disease.
To avoid the rejection risks of a bone marrow transplant, Marley became the first LAD-1 patient ever to receive the stem cell gene therapy.
I didnt hesitate in letting her be a participant in the trial because I knew in my heart that this would give her a chance at having a normal life, Tamara said.
Treatment and recovery
In July 2019, Tamara and Marley traveled from their hometown Live Oak, Florida, to UCLA. There, doctors gave the then 9-year-old Marley two medications to move the stem cells from her bone marrow into her bloodstream so that they could be collected. Her harvested stem cells were then sent to a Rocket Pharmaceuticalslab where a healthy copy of the defective gene was delivered into them.
A month later, mother and daughter returned to Los Angeles so that Marley could undergo chemotherapy. This step was necessary to clear Marleys bone marrow of the remaining defective stem cells and make space for the new transplanted ones to grow. Next, the genetically corrected stem cells were injected back into her bloodstream to replicate and make fully functioning new white blood cells.
One month after receiving the transplant, she was already feeling pretty well and her immune system was working great, Kohn said.
Last week, Kohn reported at the American Society of Hematology Annual Meeting and Exposition that Marley and five other children who received the gene therapy at UCLA,including three siblings,remain healthy and disease-free.Doctors expect that the one-time therapy will keep LAD-1 patients healthy for life.
None of the six kids have experienced new infections or inflammatory skin lesions since receiving treatment, Kohn said. He added that patients treated with the same type of gene therapy for more than a dozen other blood diseases have also shown very good recovery.
Marley and her mom went into this as the first ones ever, Kohn said. What they did was an act of bravery, so its great that weve been able to really help.
A string of firsts
More than two years out of treatment and no longer limited by the severe infections that used to keep her in the hospital for months, Marley is experiencing a lot of firsts: first time camping, first time getting her ears pierced and first time going to what she calls big school this year.
She tells me that shes thankful she has a story that makes her unique, Tamara said. Now she shares her journey even with kids at school to give others courage and hope.
For Tamara, who quit her job in banking to homeschool Marley from pre-kindergarten through fifth grade, the gene therapys success has also opened up new possibilities for her own future.
Its a rebirth for the patients and the parents, Kohn said. Its life-changing, from a life of worry to a life of health.
See the rest here:
- Phenotype, allele and genotype distribution of ABO and RhD | JBM - Dove Medical Press - January 5th, 2022
- Importance of stem cells-Past, present and future - Express Healthcare - December 23rd, 2021
- The Worldwide Cord Blood Banking Services Industry is Expected to Reach $6+ Billion by 2026 - GlobeNewswire - December 17th, 2021
- LifeCell - India's First and Largest Stem Cell Bank - December 15th, 2021
- Stem Cell Banking Outsourcing Market Analysis and Demand with Forecast Overview To 2026 - Northwest Diamond Notes - December 12th, 2021
- Cell Banking Outsourcing Market 2021 Analysis by Business Share, Industry Growth Statistics, Prominent Players Strategies, Investment Opportunities,... - December 11th, 2021
- Stem Cell Banking Market Size 2021, Latest Business Opportunities, Growth Statistics, Industry Share, Upcoming Trends, Covid-19 Impact, Demand... - November 30th, 2021
- Drs. Malik and McDuffee join Erlanger Hematology and Oncology and more Chattanooga-area career moves - Chattanooga Times Free Press - November 15th, 2021
- Global Cord Blood (CO) gains 0.46% in Active Trading on November 4 - Equities.com - November 7th, 2021
- Umbilical Cord Blood Banking, Stem Cell Banking In India ... - October 30th, 2021
- Umbilical Cord Blood Banking Market Size to Expand Significantly by the End of 2025 - BioSpace - October 28th, 2021
- Stem Cell Banking Market Detailed Analysis Of Current Industry Figures With Forecasts Growth By 2028 IMIESA - IMIESA - October 28th, 2021
- Stem Cell Banking: A New Approach towards Saving Lives ... - October 19th, 2021
- Harvesting dental stem cells - Overview - October 19th, 2021
- Cord blood banking: What are the options? - Mayo Clinic - October 19th, 2021
- Stem cell banking: A UK perspective on a global challenge ... - October 2nd, 2021
- Stem Cell/Cord Blood Banking Global Market Report 2021: - October 2nd, 2021
- How to decide Stem Cell Bank for babys cord preservation ... - September 27th, 2021
- Cell banking outsourcing market is expected to grow at a CAGR of 14%-18% by 2026 An exclusive market research report by Lucintel - Digital Journal - September 22nd, 2021
- Cheshire father with blood cancer still searching for stem cell match that could save his life - The Chester Standard - September 22nd, 2021
- Stem Cell Banking Market Report with Detailed Analysis of Current Revenue and Future Scope - BioSpace - September 22nd, 2021
- LifeCells Mesocel gets nod for clinical trial from DCGI; Therapy to treat patients with moderate to severe COVID 19 - APN News - September 18th, 2021
- Stem Cell Banking Market Report | Exclusive Trends and Untapped Growth Opportunities Analysis - BioSpace - September 18th, 2021
- $26.12 Billion Opportunity by 2028 in the Cell Separation Technology Market: Global Key Vendors GE, BD, Terumo, Merck, Thermo Fisher, Bio-Rad Lab -... - September 18th, 2021
- Global Cord Stem Cell Banking Market Growth Factors, New Top Players, Competitive Analysis by regions from 2021 to 2028||Viacord, SMART CELLS PLUS.,... - September 8th, 2021
- Artificial Intelligence And Regenerative Medicine: What The Future Of Medicine Holds? - TheHealthSite - September 7th, 2021
- Cord Blood Banking Services Market Poised to Expand at a Robust Pace The Manomet Current - The Manomet Current - September 7th, 2021
- Stem Cells Banking: Pros & Cons - Being The Parent - September 4th, 2021
- Mothercell | WORLD'S BIGGEST DENTAL STEM CELL BANK - September 4th, 2021
- Cord Blood Banking | ACOG - September 4th, 2021
- The Ultimate Choice in Stem Cell Banking Services Medeze ... - September 4th, 2021
- Cord Blood Banking: Legit Or A Scam? | American Council on ... - September 4th, 2021
- Stem Cell Banking for a better future - StemCells21 - September 2nd, 2021
- Cord Stem Cell Banking Process - StemCord - September 2nd, 2021
- Stem Cell Banking Market SWOT Analysis, Key Indicators, Forecast 2027 : Cord Blood Registry (CBR) Systems, Cordlife Group Limited, Cryo-Cell... - September 2nd, 2021
- STEMCELL Technologies Launches Human Pluripotent Stem Cell Characterization and Banking Services in Partnership with WiCell - Business Wire - September 2nd, 2021
- Global Cord Blood Corporation Reports Financial Results for the First Quarter of Fiscal 2022 - PRNewswire - September 2nd, 2021
- Cryo-Cell International Announces Uplisting to The Nasdaq Capital Market - GlobeNewswire - September 2nd, 2021
- Vita 34 again achieves double-digit growth in revenues and earnings in the first half of the year - PharmiWeb.com - September 2nd, 2021
- Acute Exacerbation of CRS Treatment Market is Expected to Reach US$ 7.06 Bn by 2031 - BioSpace - August 23rd, 2021
- 22nd Century Group Appoints Accomplished Life Sciences and Biotechnology Executive Anthony Johnson to Board of Directors - Yahoo Finance - August 20th, 2021
- Wearable Technology Market 2021-2026: Global Size, Share, Trends and Forecast Report - The Market Writeuo - The Market Writeuo - August 20th, 2021
- Stem Cell Banking Market Revenue, Statistics, Industry Growth and Demand Analysis Research Report by 2028 - The Manomet Current - August 14th, 2021
- Melatonin Supplements Market 2021-2026: Global Size, Share, Trends and Forecast Report - The Market Writeuo - The Market Writeuo - August 14th, 2021
- High therapeutic potential of stem cells for treating genetic diseases to aid in growth of Stem Cell Banking Market - The Manomet Current - August 12th, 2021
- Cryo-Cell International to Present at the Q3 Virtual Investor Summit - GlobeNewswire - August 12th, 2021
- Cell Banking Outsourcing Market Expectation Surges with Rising Demand and Changing Trends The Manomet Current - The Manomet Current - August 12th, 2021
- High Demand Owing To the Increasing Expansion of Cell Banks and the Usage of Cell Therapy in the World - The Manomet Current - August 12th, 2021
- Actinic Keratosis Treatment Market Trends, Scope, Demand, Opportunity and Forecast by 2026 - The Market Writeuo - The Market Writeuo - August 12th, 2021
- Principles of seed banks and the emergence of complexity from dormancy - Nature.com - August 12th, 2021
- Global $5.57 Billion Cell Therapy Technologies Markets, 2015-2019, 2023F, 2025F, 2030F: Cell Processing; Cell Preservation, Distribution, and... - August 12th, 2021
- CryoCord | Umbilical Cord Stem Cell Banking - August 8th, 2021
- Netcells | Stem Cell Banking | South Africa's Leading Stem ... - August 8th, 2021
- Home - Lifebank Stem Cell Banking - August 8th, 2021
- Umbilical Cord Blood Banking Australia and Stem Cell Storage - August 8th, 2021
- Stem Cell Banking for Pets | Gallant - August 8th, 2021
- Striking the right c(h)ord - The Tribune India - August 8th, 2021
- Cord Blood Banking Services Market Giants Spending Is Going To Boom - The Market Gossip - The Market Gossip - August 8th, 2021
- Banking Cell Stem Market Scope and Forecast By 2021-2027 I Top key players-Cord Blood Registry (CBR) Systems (US), Cordlife Group Limited (Singapore),... - August 8th, 2021
- Global Stem Cell Banking Market to Reach US$11.3 Billion by the Year 2027 - Yahoo Finance - August 6th, 2021
- Global Cord Blood (CO) gains 0.98% in Light Trading on August 4 - Equities.com - August 6th, 2021
- Global Stem Cell Banking Market Size And Forecast to 2021 2027 analysis with key players : Cord Blood Registry (CBR) Systems, Cordlife Group Limited,... - August 4th, 2021
- Global Cord Blood Corporation Files Annual Report on Form 20-F - PRNewswire - August 2nd, 2021
- Stem cell company asked to compensate couple - Times of India - July 31st, 2021
- Cell Banking Outsourcing Market Production, Consumption, Worldwide Business, Revenue, Price Trend by Product Type, Analysis by 2027 The Manomet... - July 31st, 2021
- LifeCell International appoints Mullen Lintas as their creative partner - ETBrandEquity.com - July 31st, 2021
- Worldwide Stem Cell Banking Industry to 2026 - Featuring CBR Systems, Cordlife and Lifecell Among Others - ResearchAndMarkets.com - Business Wire - July 17th, 2021
- Stem Cells Market Key Players, Competitive Landscape, Growth, Statistics, Revenue and Industry Analysis Report by 2027 The Manomet Current - The... - July 13th, 2021
- Shawn Johnson East is among influencers promoting cord blood banking. What is it? - Yahoo News - July 9th, 2021
- Bringing the clinic home - Harvard School of Engineering and Applied Sciences - July 9th, 2021
- Stem Cell Banking Market to Witness Robust Expansion Throughout the Forecast Period 2019 2026 Rust Wire - Rust Wire - July 9th, 2021
- Global Cord Blood Banking Market Demand Analysis and Projected huge Growth By 2027 The Manomet Current - The Manomet Current - July 3rd, 2021
- Need-based innovations happen overnight in India: Padmashri Dr. Manjula Anagani - ETHealthworld.com - July 1st, 2021
- Global Cord Stem Cell Banking Market by Technology, Application & Geography Analysis & Forecast to 2028 ||CBR Systems, Inc Cordlife Cells4Life... - June 29th, 2021
- Stem Cells Banking Market Estimated to Boost in Near Future 2027 with Top Key Players KSU | The Sentinel Newspaper - KSU | The Sentinel Newspaper - June 29th, 2021
- Global Cord Blood Corporation Reports Financial Results for the Fourth Quarter and Full Year of Fiscal 2021 - PRNewswire - June 29th, 2021
- Cell Banking Outsourcing Market is Projected to Witness an Upward Growth Over 2027: SGS Life Sciences, Lonza, CCBC, Vcanbio The Manomet Current - The... - June 24th, 2021
- Global Cord Blood Corporation to Report Fourth Quarter and Full Year Fiscal 2021 Financial Results - Yahoo Finance - June 22nd, 2021
- Cord Stem Cell Banking Market 2021-Competitive Insights: CBR Systems, Inc., Cordlife, Cells4Life Group LLP, Cryo-Cell International, Inc., Cryo-Save... - June 22nd, 2021
- COVID-19 Impact on Cancer Stem Cells Market to Witness Comprehensive Growth by 2026 The Manomet Current - The Manomet Current - June 16th, 2021