Newswise LA JOLLA, CALIF. October 11 Researchers from Sanford Burnham Prebys have discovered a new source of stem cells just outside the liver that could help treat people living with Alagille syndrome, a rare, incurable genetic disorder in which the bile ducts of the liver are absent, leading to severe liver damage and death. The findings, published recently in the journal Hepatology,have extensive biomedical implications for Alagille syndrome and for liver disease in general, including cancer.
Weve been aware of the regenerative power of the liver for a long time, possibly even going back to the ancient Greek myth of Prometheus, says lead authorDuc Dong, Ph.D., an associate professor in theHuman Genetics Programat Sanford Burnham Prebys. But the existence and nature of liver stem cells remains an intensely debated topic.
The new study suggests that the reason these cells have been so hard to find may be that researchers have been looking in the wrong place.
The stem cells that we found are actually outside the liver, not within it, which may have made their discovery difficult, adds Dong. We think these outside the box liver stem cells act more like reserves, only traveling into the liver when all other options are exhausted. It only requires a few of these cells to enter the liver and multiply to repopulate all of the cells lost to the disease.
Over 4 thousand babies each year are born with Alagille syndrome, which is caused by a mutation that prevents duct cells from forming in the liver. And while the syndrome can occasionally resolve naturally, and there are treatments available to manage the symptoms, the disease is incurable, carrying a 75% mortality rate by late adolescence for those without a liver transplant.
"We have known and supported Dr. Dong for years and we feel the work he and his team have done on this disease to date is extraordinary," says Cher Bork, Executive Director of the Alagille Syndrome Alliance. "Hope can be difficult to come by for families dealing with any incurable disease, and discoveries like this help give that hope back to families living with this life-dominating condition."
Using zebrafish, which have many of the same genes and cellular pathways as humans, Dongs research team were able to create a model of Alagille syndrome by selectively deactivating genes associated with Alagille syndrome. These genes encode for chemical messengers from the Notch pathway, a signaling system found in most animals that is involved in embryonic development and adult cell maintenance.
Our work suggests that there is potential for liver regeneration in Alagille patients, but because this signaling pathway is mutated, the regenerative cells fail to fully mature into functioning liver duct cells, says Dong.
In further animal studies, the team showed that by genetically restoring this signaling pathway, the regenerative cells could remobilize to form liver ducts, restoring the function of the liver and improving survival. The researchers are now leveraging their discovery to develop new therapies for Alagille syndrome.
Weve shown not just that regeneration is possible in models of Alagille syndrome, but, importantly, how it can be enhanced, says Dong. These missing duct cells can regenerate if Jagged/Notch is restored, and our lab has developed the first drug that can boost this pathway.
While the new drug requires further studies to advance into clinical trials, the team has already found that it could enhance regeneration and survival in animal models and can trigger the Notch pathway in cells from Alagille patients. These results will be published in separate studies.
Were hopeful that this drug will restore the regenerative potential of the liver in Alagille patients, to be more like the liver of Prometheus, adds Dong.
About Sanford Burnham Prebys Medical Research Institute
Sanford Burnham Prebys is a preeminent, independent biomedical research institute dedicated to understanding human biology and disease and advancing scientific discoveries to profoundly impact human health. For more than 40 years, our research has produced breakthroughs in cancer, neuroscience, immunology and childrens diseases, and is anchored by our NCI-designated Cancer Center and advanced drug discovery capabilities. For more information, visit us atSBPdiscovery.orgor on Facebookfacebook.com/SBPdiscoveryand on Twitter@SBPdiscovery.
- COVID-19: Researchers warn against overhyping early-stage therapies - Medical News Today - October 26th, 2021
- OpRegen Data Update to Be Featured at 2021 American Academy of Ophthalmology Annual Meeting in Presentation by Michael S. Ip, M.D. - Yahoo Finance - October 26th, 2021
- Insights & Outcomes: Place cells, planarians, and 'prewet' proteins - Yale News - October 26th, 2021
- IMAC Holdings The Back Space Announces the Completion of Two Additional Retail Spinal Care Locations - Yahoo Finance - October 26th, 2021
- JEFFERSON PARISH PARTNERS WITH LOUISIANA DEPARTMENT OF HEALTH TO ADMINISTER FREE FLU SHOTS AND COVID-19 VACCINES ON NOVEMBER 6, 2021 - 10-25-YYYY -... - October 26th, 2021
- Male Infertility Could Be Treated With Monkey Cells; New Study Shows How These Animals Could Help Address the Condition - Science Times - October 26th, 2021
- Citius Pharmaceuticals to Host Key Opinion Leader Webcast on the Compelling Need to Salvage Central Venous Catheters in CLABSI Patients - Yahoo... - October 26th, 2021
- New findings may help widen therapeutic interventions for B-cell acute lymphoblastic leukemia - News-Medical.Net - October 25th, 2021
- Getting to Know Kate Roth at Beacon Integrative Medical Center in Rexburg - East Idaho News - October 25th, 2021
- Global Cell Therapy Market Report 2021-2028 - Growing Adoption of Regenerative Medicine & Introduction of Novel Platforms and Technologies -... - October 25th, 2021
- Vertex reports positive results for first patient in Type 1 diabetes trial - BetaBoston - October 23rd, 2021
- Morris receives grant for stem cell research - The Source - Washington University Record - October 23rd, 2021
- Vertex moves ahead with its bid to develop a curative treatment for type 1 diabetes - PMLiVE - October 23rd, 2021
- McLaughlin Research Institute receives $5 million gift to support neurology studies - Great Falls Tribune - October 23rd, 2021
- AML medication and treatment: What to know - Medical News Today - October 23rd, 2021
- U S Stem Cell : USRM to Focus on Animal Health and Lead Pet-Care Innovation - Marketscreener.com - October 19th, 2021
- Opinion: Why Colin Powell's death should not be framed as the death of a vaccinated person - Poynter - October 19th, 2021
- Quris combines AI with patient on a chip to speed drug development and reduce animal testing - TechCrunch - October 19th, 2021
- Dr. Helen Heslop elected to the National Academy of Medicine - Baylor College of Medicine News - October 19th, 2021
- Researchers report long-lasting effects of gene therapy in children with deadly immune disorder - News-Medical.Net - October 19th, 2021
- Coronavirus: How does misinformation spread, and how can we stop it? - The Jerusalem Post - October 17th, 2021
- Dr. Kahl on the Potential Utility of Frontline CAR T-Cell Therapy in MCL - OncLive - October 17th, 2021
- FDA Approves Genentech's Tecentriq as Adjuvant Treatment for Certain People With Early Non-Small Cell Lung Cancer - Business Wire - October 17th, 2021
- Global Stem Cell and Primary Cell Culture Medium Market 2021 Trends and Leading Players Analysis 2027 Merck, STEMCELL Technologies, Irvinesci, Cell... - October 15th, 2021
- Dr. Erba on the Evolution of Treatment in MCL - OncLive - October 15th, 2021
- After no stem cell donors stepped up to saved her son, mom continues to help others facing the same challenge - Chicago Tribune - October 15th, 2021
- International ZIM Network: SmartMed - Regenerative Solutions for the Therapies of Tomorrow - Business Wire - October 15th, 2021
- Are 'robot massages' the future of muscle repair? - Medical News Today - October 15th, 2021
- What the FDA Panel's Moderna Booster Shot Vote Means and What's Next - NBC Chicago - October 15th, 2021
- Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in... - October 15th, 2021
- R3 Stem Cell & Exosome Therapy: Joint & Back Pain, Avoid ... - October 13th, 2021
- Stem Cell PhD Program | Institute for Stem Cell Biology ... - October 13th, 2021
- Repurposed Drug Reverses Signs of Alzheimers in Mice, Human Cells - The Scientist - October 13th, 2021
- Bone Therapeutics appoints Scientific Advisory Board for iMSC cell and gene therapy platform development - StreetInsider.com - October 13th, 2021
- What the latest COVID research says about breakthrough cases and transmission : Shots - Health News - NPR - October 13th, 2021
- HNF collaborates with Rarebase on a Drug Discovery Platform to develop treatments for Charcot-Marie-Tooth (CMT) - PRNewswire - October 13th, 2021
- Autologous HSCT Continues to Show Sustained Benefit in MCL, But Targeted Therapies May Challenge its Role as Standard Frontline Treatment -... - October 13th, 2021
- Bartolo Coln, Winner Of The Cy Young, Wins Lawsuit In Case Of Stem Cell Treatment To Which He Was Subjected Before His Return To The Major Leagues. -... - October 11th, 2021
- The Impact Of Market Restrictions On The US Stem Cell Biomaterials Market - Med Device Online - October 11th, 2021
- Vitro Biopharma Inc. July 31st 2021; 3rd Quarter ended Financial Results of Operations and Shareholder Letter. - Yahoo Finance - October 11th, 2021
- Top US Leukemia Experts from Academia, Industry and Public Sector Coming Together at Acute Myeloid Leukemia Conference on October 22 - OncLive - October 11th, 2021
- Precision medicine data dive shows water pill may be viable to test as Alzheimer's treatment - National Institutes of Health - October 11th, 2021
- Treating Early Relapse in Follicular Lymphoma - Targeted Oncology - October 11th, 2021
- Humans and Chimpanzees Use a Part of Their DNA in Different Ways - Technology Networks - October 11th, 2021
- A stem cell treatment for MS offers some patients hope. But is it hope that will last? - WHYY - October 9th, 2021
- CAR T Offer New Treatment Option for Non-Hodgkin Lymphoma - Targeted Oncology - October 9th, 2021
- FDA Advisory Committee Recommends Maribavir to Treat Cytomegalovirus CMV Infection and Disease - Contagionlive.com - October 9th, 2021
- What surgeons are saying about regenerative medicine in 2021 - Becker's Orthopedic & Spine - October 9th, 2021
- Study illuminates how BRD4 protein directs the spatial organization of DNA within cell nucleus - News-Medical.net - October 9th, 2021
- Cellular Therapy Strategies Offer the Prospect of Deep Remissions for Multiple Myeloma - OncLive - October 9th, 2021
- Anti-CD47 Therapy and Other New Approaches to the Treatment of Myelodysplastic - Targeted Oncology - October 9th, 2021
- ViaCyte to Present at Alliance for Regenerative Medicine Cell & Gene Meeting on the Mesa - PRNewswire - October 7th, 2021
- Seattle Stem Cell Therapy Clinic - Seattle Sports Medicine ... - October 7th, 2021
- StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa - PRNewswire - October 7th, 2021
- New Stem Cell Approach Through Using Wavelength Laser Might have Discovered Why Humans Lose Hair - Tech Times - October 5th, 2021
- Faster healing of wounds can decrease pain and suffering and save lives - WMTV - NBC15 - October 5th, 2021
- Losing Your Hair? You Might Blame the Great Stem Cell Escape. - The New York Times - October 5th, 2021
- Northwesterns SPORE has made advances in understanding the genetic basis of glioblastoma - News-Medical.Net - October 5th, 2021
- NextCure Announces New Appointments to its Board of Directors - GlobeNewswire - October 5th, 2021
- Therapy and Prevention Strategies for Myocardial Infarction | IJN - Dove Medical Press - October 3rd, 2021
- Lack of awareness about blood stem cell donation is one of the leading causes for low number of donors in In.. - ETHealthworld.com - October 3rd, 2021
- Advanced technique offers new insights about the dynamics of gliomas - News-Medical.net - October 3rd, 2021
- "Stem cell-based therapeutics poised to become mainstream option - BSA bureau - October 2nd, 2021
- The stem cell revolution isnt what you think it is - New Scientist News - October 2nd, 2021
- Stem cell agency gives out $2 million a minute - Capitol Weekly - October 2nd, 2021
- Researching the Safety and Effectiveness of Stem Cells to Treat 'Long Covid' - Entrepreneur - October 2nd, 2021
- United States Regenerative Medicine Markets to 2026: Focus on Cell Therapies, Gene Therapies, Progenitor & Stem Cell Therapies, Tissue Engineered... - October 2nd, 2021
- U.S. FDA Approves Kite's Tecartus as the First and Only Car T for Adults With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia - Business... - October 2nd, 2021
- Omeros Provides Regulatory Update on Biologics License Application for Narsoplimab in the Treatment of HSCT-TMA - Business Wire - October 2nd, 2021
- Stem Cell Umbilical Cord Blood Market in North America 2021 Sees Growth Due to Rising Spending Capacity and Suitable Payment Plans - PRNewswire - October 2nd, 2021
- BrainStorm to Present Phase 2 Progressive MS Study Results in Oral Presentation at 37th Congress of ECTRIMS - BioSpace - October 2nd, 2021
- Macomics Announces New Hires that Expands its Macrophage-based Drug Discovery R&D Team - BioSpace - October 2nd, 2021
- Stanford neuroscientist's 'assembloids' pave the way for innovative brain research - Scope - October 2nd, 2021
- At the crossroads of reproductive and regenerative medicine: new opportunities for preserving and restoring fertility - ESHRE - September 25th, 2021
- Doctors Debate: Do CAR T Cells and Transplantation Both Have a Place in Aggressive B-Cell Lymphoma Treatment? - Targeted Oncology - September 25th, 2021
- Global Stem Cell Alopecia Treatment Market 2021 Development Status,Industry Insights and Forecast Research Report 2027 Stillwater Current -... - September 25th, 2021
- AML vs. ALL: Differences in symptoms, diagnosis, and survival - Medical News Today - September 25th, 2021
- New gene therapies may soon treat dozens of rare diseases - American Society for Biochemistry and Molecular Biology - September 25th, 2021
- BioRestorative Therapies Receives Notice of Allowance for Patent Application in Israel for its Metabolic Program - GlobeNewswire - September 25th, 2021
- Shifts Away From R-CHOP Treatment Shows Improved EFS and OS Benefit in MCL - Targeted Oncology - September 25th, 2021