When a person's immune system is impaired by a genetic disease, a bone-marrow transplant can be a powerful therapeutic tool, but with a major downside: during the first few months the recipient's defenses against viruses are severely weakened. The slightest infection can lead to a hospital trip.
A still-experimental type of treatment known as T-cell therapy aims to assist during this vulnerable period -- the months during which the body is rebuilding its natural defenses. After two decades of clinical trials, the technology has been refined, and is being used to treat more and more patients, many of them children.
A boy named Johan is one of them.
Today he is a mischievous, smiling toddler with a thick shock of light-brown hair, who never tires, playfully tormenting the family's puppy, Henry. There is no sign of the three-year-long medical and emotional roller-coaster ride he and his family, who live in an affluent Washington suburb, have been on.
The first traumatic surprise came with the results of a pregnancy test: Johan was not planned.
"That was a huge shock. I cried," said his mother, 39-year-old Maren Chamorro.
She had known since childhood that she carried a gene that can be fatal in a child's first 10 years, chronic granulomatous disease (CGD). Her brother died of it at the age of seven. The inexorable laws of genetics meant that Maren had a one in four chance of transmitting it to her child.
For their first children, she and her husband Ricardo had chosen in-vitro fertilization, allowing the embryos to be genetically tested before implantation.
Their twins Thomas and Joanna were born -- both disease-free -- seven and a half years ago. But in Johan's case, a post-birth genetic test quickly confirmed the worst: he had CGD.
After conferring with experts at Children's National Hospital in Washington, the couple took one of the most important decisions of their lives: Johan would receive a bone-marrow transplant, a risky procedure but one that would give him a chance of a cure.
"Obviously, the fact that Maren had lost a sibling at a young age from the disease played a big role," Ricardo confided.
Bone marrow, the spongy tissue inside bones, serves as the body's "factory" for the production of blood cells -- both red and white.
Johan's white blood cells were incapable of fighting off bacteria and fungal infections. A simple bacterial infection, of negligible concern in a healthy child, could spread out of control in his young body.
Luckily, Johan's brother Thomas, six years old at the time, was a perfect match. In April 2018, doctors first "cleansed" Johan's marrow using chemotherapy. They then took a small amount of marrow from Thomas's hip bones using a long, thin needle.
From that sample they extracted "supercells," as Thomas calls them -- stem cells, which they reinjected into Johan's veins. Those cells would eventually settle in his bone marrow -- and begin producing normal white blood cells.
The second step was preventive cell therapy, under an experimental program led by immunologist Michael Keller at Children's National Hospital.
The part of the immune system that protects against bacteria can be rebuilt in only a matter of weeks; but for viruses, the natural process takes at least three months.
From Thomas's blood, doctors extracted specialized white blood cells -- T-cells -- that had already encountered six viruses.
Keller grew them for 10 days in an incubator, creating an army of hundreds of millions of those specialized T-cells. The result: a fluffy white substance contained in a small glass vial.
Those T-cells were then injected into Johan's veins, immediately conferring protection against the six viruses.
"He has his brother's immune system," said Keller, an assistant professor at Children's National.
Johan's mother confirmed as much: today, when Thomas and Johan catch a cold, they have the same symptoms, and for nearly the same amount of time.
"I think it's pretty cool to have immunity from your big brother," Maren Chamorro said.
This therapeutic approach -- boosting the body's immune system using cells from a donor or one's own genetically modified cells -- is known as immunotherapy.
Its main use so far has been against cancer, but Keller hopes it will soon become available against viruses for patients, like Johan, who suffer from depressed immune systems.
The chief obstacles to that happening are the complexity of the process and the costs, which can run to many thousands of dollars. These factors currently restrict the procedure to some 30 medical centers in the United States.
For Johan, a year and a half after his bone marrow transplant, everything points to a complete success.
"It's neat to see him processing things, and especially play outside in the mud," his mother said. "You know, what a gift!"
Her only concern now is the same as any mother would have -- that when her son does fall ill, others in the family might catch the same bug.
Read this article:
The 'supercells' that cured an infant's grave genetic illness - Japan Today
- City of Hope to buy Cancer Treatment Centers of America for $390M - Modern Healthcare - December 9th, 2021
- The lung game: is hyperbaric oxygen therapy any good? - Financial Times - December 9th, 2021
- Cell Culture Media Market is predicted to grow from US$ 2.6 Bn in 2020 to over US$ 5.6 Bn by 2031 - PRNewswire - December 9th, 2021
- It is imperative to reduce the cost of cancer treatment: Ramesh Ramadurai, MD, 3M India - ETHealthworld.com - December 9th, 2021
- COVID: How bad is the omicron variant? Q&A on what new research is telling us - The Denver Post - December 9th, 2021
- What Are New Medical Solutions That Can Help Treat Patients? - iLounge - November 22nd, 2021
- Slidell mom with months to live, couldn't afford to fight 'very treatable' cancer - WWLTV.com - November 22nd, 2021
- Cell and Gene Therapy Market to reach US$ 47,095.2 Mn by end of 2028, Says Coherent Market Insights - PRNewswire - November 22nd, 2021
- FMQs: Senior Glasgow doctor warns claims doctors act in secret or conceal information could damage public confidence as Andrew Slorance widow claims... - November 22nd, 2021
- Former Tranmere player Gary Stevens' son dies after battle with leukaemia - The Chester Standard - November 22nd, 2021
- Braeden Lichti - Investing in Precision Medicine to Yield New Treatments for Neurodegenerative Diseases - PRNewswire - November 22nd, 2021
- Global Cancer Therapy Market Expected to Reach $268 Billion In 2026, With A CAGR Of 9.15% - PRNewswire - November 22nd, 2021
- Indiana's top doctors frustrated about the state's COVID-19 vaccination rate - 953mnc.com - August 31st, 2021
- Should You Get a COVID Booster Shot? Our Doctor Explains - Articles and Videos, COVID-19, Featured, Health Topics - Hackensack Meridian Health - August 31st, 2021
- OpRegen: tackling the leading cause of blindness with cell replacement - Clinical Trials Arena - August 31st, 2021
- I blamed stress for my three-month period but it turned out to be much more sinister... - The Sun - August 31st, 2021
- New fault lines emerge in Car-T therapy - Vantage - August 31st, 2021
- When aid-in-dying means you have to go before youre ready - Monterey Herald - August 31st, 2021
- Dr. Death Season 1 Review: Truth continues to be stranger than fiction. - Times of India - August 31st, 2021
- Circulating Tumor Cells (CTC) Diagnostics Market is expected to Account for Over US$ 9.5 Million in - PharmiWeb.com - August 31st, 2021
- COVID-19 in children with cancer: Severe disease and disrupted treatment - Markets Insider - August 31st, 2021
- Sophomore saves sister's life with stem cell donation - University of Miami - August 5th, 2021
- Myelodysplastic Syndrome Treatment Options - Healthline - August 5th, 2021
- Gold River Productions, Inc. (GRPS) Appoints Michael Berkowitz to spearhead the Rejuvenation Division with StemSpa - Yahoo Finance - August 5th, 2021
- Colin Jackson health: Im in constant pain Athlete to undergo stem cell therapy to help - Express - August 5th, 2021
- The Basics of MDS: Diagnosis and Staging - Curetoday.com - August 5th, 2021
- Blood donation clinic will honour life of Jocelyn McGlynn - Chatham Daily News - August 5th, 2021
- Pharming Group reports financial results for the first half of 2021 - PRNewswire - August 5th, 2021
- Symptoms of seizures: Early signs, post-seizure, and more - Medical News Today - August 5th, 2021
- Lymph nodes: Purpose, location, and disease warning signs - Medical News Today - June 8th, 2021
- Teesside baby will be third in country to have ground breaking complex cancer treatment - Teesside Live - June 8th, 2021
- Brave Nathaniel Nabena, 9, all smiles as he has life-saving procedure - thanks to you - The Mirror - June 8th, 2021
- Local veterinary clinics offer integrated care options for pets - williamsonherald.com - June 8th, 2021
- Blood cancer and leukaemia: UAE oncologist urges public to spot the symptoms - The National - June 8th, 2021
- Replotting the human: the thorny ethics of growing babies outside the womb - Prospect - June 8th, 2021
- Crossroads of life: Jordan Reed's journey through concussions, injuries and post-NFL healing - The Athletic - June 8th, 2021
- B-cell acute lymphoblastic leukemia: What to know - Medical News Today - May 28th, 2021
- Chronic lymphocytic leukemia and stomach pain: What is the link? - Medical News Today - May 28th, 2021
- Top 12 Disruptive Gene and Cell Therapy Technologies Announced - GlobeNewswire - May 28th, 2021
- BlueRock and Senti Collaborate to Build Smarter, Disease-Fighting Cell Therapies - BioSpace - May 28th, 2021
- Living with blood cancer - Lara Said - Times of Malta - May 28th, 2021
- 3D Printed Stem Cells Could Revolutionize Neuroscience, Aid Treatment and Understanding of Neural Diseases - Science Times - May 28th, 2021
- In the War on Cancer, Science Is Winning - The Wall Street Journal - May 28th, 2021
- Whats Causing Black Fungus? Here Is What The Doctors Have To Say - The Logical Indian - May 28th, 2021
- University of Pittsburgh Won't Explain its Planned Parenthood Ties | Opinion - Newsweek - May 28th, 2021
- Trial treatment with a mythologically-inspired name saves life of young man diagnosed with multiple myeloma - Neos Kosmos - May 28th, 2021
- An unusual addition to Californias 2022 ballot - CalMatters - May 28th, 2021
- Stem Cell Therapy Los Angeles - Darrow Stem Cell Institute - February 14th, 2021
- Stem Cell Transplants: A Lifesaving Treatment for Cancer ... - February 14th, 2021
- Smart Stem Cells Made From Fat Have the Power to Heal - Freethink - February 14th, 2021
- Are Covid-19 vaccines safe for cancer patients? Here is what cancer experts say - CNN - February 14th, 2021
- The race to treat a rare, fatal syndrome may help others with common disorders like diabetes - Science Magazine - February 14th, 2021
- Arlo's Army needs stem cell donor as mum begs for help to save three-year-old's life - Glasgow Live - February 14th, 2021
- Arlo's Army: mum opens up on heartbreaking decision to make 3-year-old sick to save his life - Glasgow Live - February 14th, 2021
- Bone marrow transplant shows signs of curing brave little boy with one in a million condition - Shields Gazette - February 14th, 2021
- Canada's blood supply has a diversity problem and people are dying because of it - CBC.ca - February 2nd, 2021
- State-of-the-Art Treatments Garner University Foot and Ankle Institute Multiple Top Doctor Awards - Yahoo Finance - February 2nd, 2021
- Dean Tracy Johnson seeks to diversify the pipeline of future scientists and doctors - UCLA Newsroom - February 2nd, 2021
- Transforming optimism: finding new ways to treat rare cancers - Cancer Research UK - Science Blog - February 2nd, 2021
- Family of Belfast woman Eimear Gooderham (25) share memories and dealing with grief in special UTV programme - Belfast Telegraph - February 2nd, 2021
- Doctor: COVID-19 cases will continue to slowly go down, but were not out of the woods - Yahoo Money - January 23rd, 2021
- Global Precision Medicine Market With COVID 19 Impact Analysis| Leading Players In-depth Analysis Research Report Foresight to 2027 - KSU | The... - January 23rd, 2021
- Leading Urologist Doubles Down on CaverStem Regenerative Stem Cell Procedure for Treatment of Erectile Dysfunction in Men - PRNewswire - January 22nd, 2021
- Sisters who organised an Ilkley fundraising ball are to be honoured - Wharfedale Observer - January 22nd, 2021
- Medical Doctor: Roes Overlooking of the Consensus of Societal Morality and Science Reverberates to This Day - National Catholic Register - January 22nd, 2021
- DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) Becomes the First FDA-Approved Treatment for Patients with Newly Diagnosed Light Chain (AL)... - January 22nd, 2021
- Seattle researchers find clues for treatments that could eliminate HIV in infected patients - GeekWire - January 14th, 2021
- Flow Cytometry Market is Projected to Reach a Value of US$8100 Mn by the End of 2025 - KSU | The Sentinel Newspaper - January 14th, 2021
- Doctors diagnosed a Suffolk teen with a rare condition. Now, his mom is part of the solution - 13newsnow.com WVEC - January 12th, 2021
- Cassville club assists 2 families as they recover from illness - telegraphherald.com - January 12th, 2021
- The 26 billion-dollar startups to watch that are revolutionizing healthcare in 2021 - Business Insider - January 12th, 2021
- Global Precision Medicine Market 2020 Overview By Size, Share, Trends, Growth Factors and Leading Players With Detailed Analysis of Industry Structure... - January 12th, 2021
- More than 250 articles and a book later, the Valley Doctor bids adieu - San Lorenzo Valley Press-Banner - January 12th, 2021
- Groundbreaking Treatment for Severe COVID-19 Using Stem Cells It's Like Smart Bomb Technology in the Lung - SciTechDaily - January 5th, 2021
- New combo therapy offered against refractory T-cell lymphoma - Korea Biomedical Review - January 5th, 2021
- Hair Growth Treatment Secrets Bollywood Will Never Tell You About - Times of India - January 5th, 2021
- Are metabolic hormones the next frontier in cancer treatment? - MedCity News - January 5th, 2021
- Limerick toddler to wait six months for trial cancer treatment after family raises over 400,000 - Extra.ie - January 5th, 2021
- West Lothian mum fighting for her life after back pain turned out to be terminal blood cancer - Edinburgh Live - January 5th, 2021
- MorphoSys and Incyte Announce the Acceptance of the Swissmedic Marketing Authorization Application for Tafasitamab - Yahoo Finance UK - January 5th, 2021