Posted: Aug. 23, 2017 8:00 am Updated: Aug. 23, 2017 12:30 pm
UTRECHT, Netherlands (AP) Els van der Heijden, who has cystic fibrosis, was finding it ever harder to breathe as her lungs filled with thick, sticky mucus. Despite taking more than a dozen pills and inhalers a day, the 53-year-old had to stop working and scale back doing the thing she loved best, horseback riding.
Doctors saw no sense in trying an expensive new drug because it hasn't been proven to work in people with the rare type of cystic fibrosis that van der Heijden had.
Instead, they scraped a few cells from van der Heijden and used them to grow a mini version of her large intestine in a petri dish. When van der Heijden's "mini gut" responded to treatment, doctors knew it would help her too.
"I really felt, physically, like a different person," van der Heijden said after taking a drug and getting back in the saddle.
This experiment to help people with rare forms of cystic fibrosis in the Netherlands aims to grow mini intestines for every Dutch patient with the disease to figure out, in part, what treatment might work for them. It's an early application of a technique now being worked on in labs all over the world, as researchers learn to grow organs outside of the body for treatment and maybe someday for transplants.
So far, doctors have grown mini guts just the size of a pencil point for 450 of the Netherlands' roughly 1,500 cystic fibrosis patients.
"The mini guts are small, but they are complete," said Dr. Hans Clevers of the Hubrecht Institute, who pioneered the technique. Except for muscles and blood vessels, the tiny organs "have everything you would expect to see in a real gut, only on a really small scale."
These so-called organoids mimic features of full-size organs, but don't function the same way. Although many of the tiny replicas are closer to undeveloped organs found in an embryo than adult ones, they are helping scientists unravel how organs mature and providing clues on how certain diseases might be treated.
In Australia, mini kidneys are being grown that could be used to test drugs. Researchers in the U.S. are experimenting with tiny bits of livers that might be used to boost failing organs. At Cambridge University in England, scientists have created hundreds of mini brains to study how neurons form and better understand disorders like autism. During the height of the Zika epidemic last year, mini brains were used to show the virus causes malformed brains in babies.
In the Netherlands, the mini guts are used as a stand-in for cystic fibrosis patients to see if those with rare mutations might benefit from a number of pricey drugs, including Orkambi. Made by Vertex Pharmaceuticals, Orkambi costs about 100,000 euros per patient every year in some parts of Europe, and it's more than double that in the U.S., which approved the drug in 2015. Despite being initially rejected by the Dutch government for being too expensive, negotiations with Vertex were reopened in July.
Making a single mini gut and testing whether the patient would benefit from certain drugs costs a couple of thousand euros. The program is paid for by groups including health insurance companies, patient foundations and the government. The idea is to find a possible treatment for patients, and avoid putting them on expensive drugs that wouldn't work for them.
About 50 to 60 patients across the Netherlands have been treated after drugs were tested on organoids using their cells, said Dr. Kors van der Ent, a cystic fibrosis specialist at the Wilhelmina Children's Hospital, who leads the research.
Clevers made a discovery about a decade ago that got researchers on their way. They found pockets of stem cells, which can turn into many types of other cells, in the gut. They then homed in a growing environment in the lab that spurred these cells to reproduce rapidly and develop.
"To our surprise, the stem cells started building a mini version of the gut," Clevers recalled.
Cystic fibrosis is caused by mutations in a single gene that produces a protein called CFTR, responsible for balancing the salt content of cells lining the lungs and other organs.
To see if certain drugs might help cystic fibrosis patients, the medicines are given to their custom-made organoids in the lab. If the mini organs puff up, it's a sign the cells are now correctly balancing salt and water. That means the drugs are working, and could help the patient from whom the mini gut was made.
Researchers are also using the mini guts to try another approach they hope will someday work in people using a gene editing technique to repair the faulty cystic fibrosis gene in the organoid cells.
Other experiments are underway in the Netherlands and the U.S. to test whether organoids might help pinpoint treatments for cancers involving lungs, ovaries and pancreas.
While the idea sounds promising, some scientists said there are obstacles to using mini organs to study cancer.
Growing a mini cancer tumor, for example, would be far more challenging because scientists have found it difficult to make tumors in the lab that behave like in real life, said Mathew Garnett of the Wellcome Trust Sanger Institute, who has studied cancer in mini organs but is not connected to Clevers' research.
Also, growing the cells and testing them must happen faster for cancer patients who might not have much time to live, he said.
Meanwhile, Clevers wants to one day make organs that are not so mini.
"My dream would be to be able to custom-make organs," he said, imagining a future where doctors might have a "freezer full of livers" to choose from when sick patients arrive.
Others said while such a vision is theoretically possible, huge hurdles remain.
"There are still enormous challenges in tissue engineering with regards to the size of the structure we're able to grow," said Jim Wells, a pediatrics professor at the Cincinnati Children's Hospital Medical Center. He said the mini organs are far smaller than what would be needed to transplant into people and it's unclear if scientists can make a working, life-sized organ in the lab.
There are other limitations to growing miniature organs in a dish, said Madeline Lancaster at Cambridge University.
"We can study physical changes and try to generate drugs that could prevent detrimental effects of disease, but we can't look at the complex interplay between organs and the body," she said.
For patients like van der Heijden, who was diagnosed with cystic fibrosis as a toddler, the research has helped her regain her strength. Vertex agreed to supply her with the drug.
"It was like somebody opened the curtains and said, 'Sunshine, here I am, please come out and play.'" she said. "It's strange to think this is all linked to some of my cells in a lab."
___
This Associated Press series was produced in partnership with the Howard Hughes Medical Institute's Department of Science Education. The AP is solely responsible for all content.
View original post here:
Lab-made "mini organs" helping doctors treat cystic fibrosis - New Jersey Herald
- Latest Insights on Stem Cell Assay Market Business Strategies, Acquisitions, Revenue Analysis Share, Forecast to 2028 | by Perkinelmer Inc, Promega... - October 13th, 2022
- S.I. school hosts dance-a-thon to support 9-year-old student with sickle cell disease - SILive.com - October 13th, 2022
- Cord blood bank - Wikipedia - October 4th, 2022
- Cell Reprogramming Market is Expected to Record the Massive Growth, with Prominent Key Players Allele Biotechnology, ALSTEM, Applied Biological... - September 16th, 2022
- Pleco Therapeutics Announces Final Close of its Series A Financing Raising 17.3m to Progress its Novel Plecoid Product in Acute Myeloid Leukaemia to... - September 8th, 2022
- Century Therapeutics Reports Second Quarter 2022 Financial Results and Provides Business Updates - KULR-TV - August 14th, 2022
- Cell Expansion Market Size, Scope, Growth Opportunities, Trends by Manufacturers And Forecast to 2029 Shanghaiist - Shanghaiist - August 5th, 2022
- 3D Bioprinting for Tissue and Organ Regeneration Market worth $424.3 Million by 2030 - Exclusive Report by InsightAce Analytic - Yahoo Finance - July 27th, 2022
- After conquering sickle cell and multiple sclerosis, it was COVID-19 that claimed her - Lynchburg News and Advance - July 11th, 2022
- Stem Cell Therapy Market to Observe Exponential Growth By 2022 to 2030 | Smith, Nephew (UK), MEDIPOST Co Ltd. (South Korea) - Digital Journal - June 22nd, 2022
- Teclison Announces Publication of Phase 1 Data Demonstrating the Safety and Therapeutic Potential of Tirapazamine Chemoembolization for Patients with... - June 22nd, 2022
- This Key Protein Is Essential for Brain Cell Longevity and Growth - SciTechDaily - June 13th, 2022
- Stem Cell Cartilage Regeneration Market to Witness Massive Growth from 2022 to 2030 | Central members covered as a piece of this study incorporate... - June 13th, 2022
- Stem Cell Therapy Market Size, Scope and Forecast | Osiris Therapeutics Medipost Co. Ltd., Anterogen Co. Ltd., Pharmicell Co. Ltd.,... - June 4th, 2022
- Advanced Therapy Medicinal Products (ATMP) Service Providers Market worth $34.59 Billion by 2030 - Exclusive Report by InsightAce Analytic - Yahoo... - June 4th, 2022
- Gene Therapy Market Size, Scope and Forecast | Novartis AG, Biogen, Gilead Sciences MolMed SpA, Spark Therapeutics Orchard Therapeutics plc., SIBIONO,... - June 4th, 2022
- Cell Isolation/Cell Separation Market Size, Scope and Forecast | Beckman Coulter (Subsidiary of Danaher Corporation), Becton, Dickinson and Company,... - June 4th, 2022
- 8 Players Leading the 3D Printed Meat Revolution - 3DPrint.com - May 2nd, 2022
- Penn researchers discover new cell type in human lung with regenerative properties - EurekAlert - April 6th, 2022
- FDA Grants Fast Track Designation to CYNK-001 for AML Treatment - Targeted Oncology - January 5th, 2022
- Stem Cell Therapy: a Look at Current Research, Regulations ... - December 24th, 2021
- Stem Cells Market to Witness Gigantic Growth by 2026 LSMedia - LSMedia - December 24th, 2021
- Peaceful Protest at Cherry Hill Women's Center Warning Public of Increase in Late Term Abortions In New Jersey. - InsiderNJ - October 16th, 2021
- Time to Go Sushi With Cellular Salmon; When Pet Owners Tire of Their Minions - The SandPaper - August 18th, 2021
- Avalon GloboCare, UPMC Hillman and University of Pittsburgh to develop new cancer immunotherapy - Proactive Investors USA & Canada - August 5th, 2021
- John Theurer Cancer Center Investigators Participated in ZUMA-7 Study Showing Value of CAR T-Cell Therapy as Second-Line Treatment for Relapsed Large... - July 21st, 2021
- ExoFlo From Direct Biologics Fulfills Urgent Medical Need in COVID-19 Treatment - PRNewswire - July 21st, 2021
- GXGX Merger: The SPAC News That Has GXGX Stock Soaring - InvestorPlace - July 21st, 2021
- This Startup is Changing the Way Spinal Cord Injury Is Treated Around the World - Entrepreneur - June 23rd, 2021
- Genmab Announces that Janssen has Received European - GlobeNewswire - June 23rd, 2021
- Gene therapy offers potential cure to children born without an immune system - UCLA Newsroom - May 13th, 2021
- Commencement stories from Rowan paint a picture of opportunities near and far - NJ.com - May 13th, 2021
- Vitalant to Hold Blood Donation Event on December 5 in Freehold - TAPinto.net - December 1st, 2020
- Catalent and BrainStorm Cell Therapeutics Announce Partnership for the Manufacture of Mesenchymal Stem Cell Platform Therapy NurOwn - GlobeNewswire - October 23rd, 2020
- Genmab Announces IFM, HOVON and Janssen Achieve Positive Topline Results in Second Part of Phase 3 CASSIOPEIA Study of Daratumumab in Multiple Myeloma... - October 23rd, 2020
- Novel Strategies for Targeting the Guardian of the Genome Emerge - OncLive - October 23rd, 2020
- CRISPR Market to See Massive Growth by 2026 | GE Healthcare Dharmacon, CRISPR Therapeutics, Thermo Fisher Scientific - Aerospace Journal - October 23rd, 2020
- Smith's 2005 stem cell law to be reauthorized by House - InsiderNJ - October 4th, 2020
- Oakland 24-year-old seeking multiethnic bone marrow donor - The Jewish News of Northern California - September 21st, 2020
- 99-million-year-old fight between hell ant and its prey preserved in amber - BBC Focus Magazine - August 12th, 2020
- Vitalant to Hold Blood Donation Drive on August 11 in Atlantic Highlands - TAPinto.net - August 12th, 2020
- Stem Cells Sales Market Growth By Manufacturers, Type And Application, Forecast To 2026 - 3rd Watch News - July 7th, 2020
- Mesenchymal Stem Cells Market Growth By Manufacturers, Type And Application, Forecast To 2026 - 3rd Watch News - July 7th, 2020
- Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) Market 2019 Break Down by Top Companies, Countries, Applications, Challenges,... - June 10th, 2020
- Stem Cells Market 2019 Break Down by Top Companies, Countries, Applications, Challenges, Opportunities and Forecast 2026 - Cole of Duty - June 10th, 2020
- Genmab Announces European Marketing Authorization for the Subcutaneous Formulation of DARZALEX (daratumumab) for the Treatment of Patients with... - June 10th, 2020
- CO2 Incubators Market is Expected to Witness a Steady Growth by 2025 - Farmers Ledger - June 4th, 2020
- Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma GmbH Enter into Strategic Clinical and Commercial Manufacturing Agreements with... - May 13th, 2020
- Genmab Announces U.S. FDA Approval of Subcutaneous Formulation of Daratumumab, DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), for the Treatment... - May 6th, 2020
- CO2 Incubators Market Update: Uncover the Business Strategies of Leaders & Laggards of the Market - 3rd Watch News - March 6th, 2020
- SpaceX set to launch Falcon 9 rocket and Dragon capsule from Cape Canaveral this week - Florida Today - March 4th, 2020
- Are stem cells really the key to making humans live longer? - Wired.co.uk - February 16th, 2020
- Musicians and community coming together in Asbury Park to help Point Pleasant 3-year old - Asbury Park Press - February 12th, 2020
- TV Guy: A return to history with Auschwitz Untold - New Jersey Herald - January 27th, 2020
- Europe's guardian of stem cells and hopes, real and unrealistic - FRANCE 24 - January 20th, 2020
- Horizon Discovery to Provide Access to Novel Base Editing Technology - Yahoo Finance - January 15th, 2020
- John Theurer Cancer Center Announces Appointment of Five New Physicians - Newswise - January 15th, 2020
- Horizon to provide access to novel base editing technology - SelectScience - January 15th, 2020
- Reviewing Phibro Animal Health (NASDAQ:PAHC) & AgeX Therapeutics (NASDAQ:AGE) - Riverton Roll - January 15th, 2020
- Stemming the Tide Confronting Anti-Semitism in the 21st Century - The Times of Israel - January 5th, 2020
- Weekly pick of Neuroscience news from around the world - Brain Tumour Research - November 30th, 2019
- Comparing of Rigel Pharmaceuticals Inc. (RIGL) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) - The Broch Herald - November 28th, 2019
- Celgene to Present New and Updated Data on Key Hematology Pipeline Therapies at American Society of Hematology (ASH) 2019 Annual Meeting - Business... - November 6th, 2019
- Reviewing Surface Oncology Inc. (SURF)'s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)'s results - FinanceMercury - November 6th, 2019
- Reviewing Magenta Therapeutics Inc. (MGTA)'s and PTC Therapeutics Inc. (NASDAQ:PTCT)'s results - FinanceMercury - November 6th, 2019
- Reviewing Capricor Therapeutics Inc. (CAPR)'s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)'s results - FinanceMercury - November 6th, 2019
- Live Cell Imaging Consumables Market Evolving to a Next-Generation Strategy with New Partnerships, Technologies and Targets by 2023 key players LCC... - November 6th, 2019
- Contrasting of Spero Therapeutics Inc. (SPRO) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) - FinanceMercury - November 6th, 2019
- PTC Therapeutics Inc. (PTCT) and Jounce Therapeutics Inc. (NASDAQ:JNCE) Comparing side by side - FinanceMercury - November 6th, 2019
- Contrasting of Brainstorm Cell Therapeutics Inc. (BCLI) and HOOKIPA Pharma Inc. (NASDAQ:HOOK) - MS Wkly - November 2nd, 2019
- Reviewing Brainstorm Cell Therapeutics Inc. (BCLI)'s and Pieris Pharmaceuticals Inc. (NASDAQ:PIRS)'s results - MS Wkly - November 2nd, 2019
- Reviewing ImmunoGen Inc. (IMGN)'s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)'s results - MS Wkly - November 2nd, 2019
- On Stage: Allegaeon ready to rock Philly - The Unionville Times - November 2nd, 2019
- Comparison of Merus N.V. (MRUS) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) - MS Wkly - October 14th, 2019
- Reviewing Curis Inc. (CRIS)'s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)'s results - MS Wkly - October 14th, 2019
- Comparing of NewLink Genetics Corporation (NLNK) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) - MS Wkly - October 14th, 2019
- Reviewing Morphic Holding Inc. (MORF)'s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)'s results - MS Wkly - October 14th, 2019
- Reviewing PTC Therapeutics Inc. (PTCT)'s and Inovio Pharmaceuticals Inc. (NASDAQ:INO)'s results - MS Wkly - October 14th, 2019
- Comparing of Osmotica Pharmaceuticals plc (OSMT) and VistaGen Therapeutics Inc. (NASDAQ:VTGN) - MS Wkly - October 14th, 2019
- PTC Therapeutics Inc. (PTCT) and MacroGenics Inc. (NASDAQ:MGNX) Comparing side by side - MS Wkly - October 14th, 2019