Här kommer mitt utlovade inlägg om aktuell Reg Med-info.Det blir att axplock av alla de nyheter som strömmar in.Jag börjar med att saxa från en nypublicerad krönika skriven av en prisbelönad vetenskapsjournalist med fokus Reg Med och stamcellsforskning, Eve Herold.(Om inte länken till krönikan funkar klicka här.)
Progress in the lab has addressed previous concerns. Back in the early 2000s, some of the most fervent controversy centered around somatic cell nuclear transfer (SCNT), the process used by scientists to produce Dolly. There was fear that this technique could be used in humans, with possibly adverse effects, considering the many medical problems of the animals who had been cloned.
But today, scientists have discovered better approaches with fewer risks. Pioneers in the field are embracing new possibilities for cellular reprogramming, 3D organ printing, AI collaboration, and even growing organs in space. It could bring a new era of personalized medicine for longer, healthier lives - while potentially sparking new controversies.
In the context of medical history, the field of regenerative medicine is progressing at a dizzying speed. But for those living with aggressive or chronic illnesses, it can seem that the wheels of medical progress grind slowly.
The ethical debate has been dialed back and, in the last few decades, the field has produced important innovations, spurring the development of whole new FDA processes and categories, says Anthony Atala, a bioengineer and director of the Wake Forest Institute for Regenerative Medicine. Atala and a large team of researchers have pioneered many of the first applications of 3D printed tissues and organs using cells developed from patients or those obtained from amniotic fluid or placentas.
His lab, considered to be the largest devoted to translational regenerative medicine, is currently working with 40 different engineered human tissues. Sixteen of them have been transplanted into patients. That includes skin, bladders, urethras, muscles, kidneys and vaginal organs, to name just a few.
These achievements are made possible by converging disciplines and technologies, such as cell therapies, bioengineering, gene editing, nanotechnology and 3D printing, to create living tissues and organs for human transplants. Atala is currently overseeing clinical trials to test the safety of tissues and organs engineered in the Wake Forest lab, a significant step toward FDA approval.
Fotnot : Professor Anthony Atala leder konsortiumet (ReMDO) PHI ingår i.
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| PHI:s CEO Peter Egelberg & WFIRM:s director Anthony Atala. |
Jeanne Loring, another pioneer in the field and director of the Center for Regenerative Medicine at Scripps Research Institute in San Diego, says that investment in regenerative medicine is not only paying off, but is leading to truly personalized medicine, one of the holy grails of modern science.
This is because a patient’s own skin cells can be reprogrammed to become replacements for various malfunctioning cells causing incurable diseases, such as diabetes, heart disease, macular degeneration and Parkinson’s. If the cells are obtained from a source other than the patient, they can be rejected by the immune system. This means that patients need lifelong immunosuppression, which isn’t ideal. “With Covid,” says Loring, “I became acutely aware of the dangers of immunosuppression.” Using the patient’s own cells eliminates that problem.
First time bioprinted solid tissue constructs to be sent to the International Space Station and induced pluripotent stem cells to be manufactured in space.
Microgravity allows researchers to study the behavior of cells and tissues in a unique environment, which can lead to new insights and medical breakthroughs in combating disease. For the Ax-2 mission, Axiom Space is working with the University of Connecticut, Eascra Biotech, Cedars-Sinai, and the Wake Forest Institute for Regenerative Medicine (WFIRM) to learn more about how microgravity affects stem cells and thick tissue constructs. Their research will provide valuable insights into detecting diseases and developing therapies for people on Earth. Among the experiments flying on Ax-2 are bioengineered liver and kidney tissue constructs, which will assess the impact of microgravity on the vascularization of thick tissues, which could help create a solution for patients in need of organ transplants.
Space Tissue and Regeneration
In collaboration with WFIRM and the RegenMed Development Organization (ReMDO)
During the Ax-2 mission, WFIRM will make history when sending the first bioprinted solid tissue constructs to the International Space Station (ISS). The Ax-2 crew will evaluate the vascularization of thick tissue in microgravity and the effectiveness of this platform technology for other tissue types.
γδ-Enriched CAR-T cell therapy for bone metastatic castrate-resistant prostate cancer
Abstract
Licensavtal mellan CBMG och Janssen för två CAR T-terapier
Under gårdagen annonserades det att Cellular Biomedicine Group Inc. (CBMG) har ingått ett globalt samarbets- och licensavtal med Janssen Biotech för utveckling och kommersialisering av C-CAR039 och C-CAR066, två CAR T-terapier för behandling av non-Hodgkin lymfom (NHL). FDA har beviljat C-CAR039 Fast Track and Regenerative Medicine Advanced Therapy (RMAT) klassificering för återfallande/terapiresistent inom diffust stort B-cellslymfom (r/r DLBCL). Enligt villkoren i avtalet kommer Janssen att få en exklusiv licens att utveckla och kommersialisera C-CAR039 och C-CAR066, Kina exkluderas ur avtalet. CBMG kommer att erhålla en engångsbetalning på 245 miljoner USD och milstolpesbetalningar samt royalties i kommersialiseringsfas, vilka inte offentliggörs i det pressmeddelandet.
En fas Ib-studie i USA som utvärderar C-CAR039 vid behandling av patienter med r/r DLBCL pågår. När det gäller C-CAR066 är en fas Ib-studie planerad på patienter med r/r DLBCL och den förväntas påbörjas under andra halvan av 2023. Både C-CAR039 och C-CAR066 har redan visat lovande kliniska resultat i fas I-studier på patienter med r/r non-Hodgkins lymfom i Kina.
Betydelsen av avtalet för Elicera
Avtalet har en tydlig relevans för Elicera. För det första omfattar det samma område som för den egna iTANK-förstärkta CAR T-cellterapin (ELC-301) inom B-cellslymfom. För det andra rör det sig om samma måltavlor – ELC-301 är riktad mot målstrukturen CD20. För CMBG och de två CAR T-cellterapierna i avtalet är måltavlorna CD19 och CD20 för C-CAR039 och enbart CD20 när det gäller C-CAR066. Vidare rör det sig om projekt som är i tidiga utvecklingsstadium – kliniska fas I-studier.
Detta avtal styrker vår tes om att intresset från större läkemedelsbolag inom immunonkologi är stort, i synnerhet för projekt i tidigt utvecklingsstadium, och framför allt är det ett tecken på att nya CAR T-cellterapier är särskilt intressanta. Det totala avtalsvärdet är inte annonserat, men engångsersättningen ligger över genomsnittet i tidigare genomförda affärer.
Bristol Myers Squibb boosts CAR T therapy manufacturing capabilities
1 May 2023
Viral vector production for CAR T therapies
Specifically, additional production capability will enable BMS to “manufacture current and next-generation vector technology, and increase future capacity,” stated Karin Shanahan, Executive Vice President of Global Product Development & Supply at Bristol Myers Squibb.
The manufacture of cell therapies is a complex operation, as they are produced as personalised therapies for individual patients. Viral vectors are a key part of engineering T cells for infusion back to a patient.
Cell therapies are rapidly changing how different cancers are treated. Therefore, demand for viral vector is growing significantly, according to BMS. A dual-sourcing vector strategy supports reliable delivery of its CAR T-cell therapies to help meet this demand.
Berömda Stanford University med egen Reg Med fakultet
har även de hoppat på T-cells tåget. I en aktuell artikel berättar forskare därifrån att deras T-cell forskning kommit långt.
Stanford Medicine scientists transform cancer cells into weapons against cancer
Utöver T-cell forskning har det under veckan kommit info att sjukdomen ALS (minns hockeyikonen Börje Salming) närmar sig bot. 2 olika studier har uppmärksammats.
Regenerative Medicine: A New Path for ALS Treatment
A first-of-its-kind stem cell therapy for ALS passes a critical safety benchmark, advancing the search to slow down, reverse and prevent the disease. In a parallel study, investigators are growing patient-derived stem cells to model ALS, hoping to uncover its mechanisms and classify it with more specificity. Can the cure to this degenerative condition lie in the endlessly regenerative power of stem cells?
Biogen’s Qalsody ‘sets a precedent for personalized treatment in ALS’
Last week, the US Food and Drug Administration (FDA) approved Qalsody (tofersen) to treat amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS).
Utöver Stanford University ska nämnas att än mer klassiska sätet Harvard University bedriver Reg Med forskning.
Ett exempel på vad de lyckats åstadkomma :
Restoring hearing loss through regenerative medicine
De nya regelverken beträffande Reg Med har börjat implimenterats i befintlig lagstiftning världen över.
Pharma responds to proposed EU pharmaceutical legislation reform
28 April, 2023
FDA releases draft guidance for decentralised clinical trials
Recommendations on the conduct of remote clinical trial visits are included in new US Food and Drug Administration (FDA) draft guidance.
2 May, 2023
The US Food and Drug Administration (FDA) has released a new draft guidance to further support the use of decentralised clinical trials (DCTs) for drugs, biologics and devices.
The publication includes recommendations for sponsors, investigators and other stakeholders regarding the implementation of DCTs to advance medical product development and research.
In DCTs, some or all the clinical trial-related activities occur at locations other than traditional clinical trial sites. Examples of these activities include obtaining laboratory tests at a local facility rather than a research medical centre or conducting a clinical follow-up visit in the trial participant’s home using telemedicine.
Alliance for REGENERATIVE MEDICINE har sammanfattat nuläge och framtid i en rapport titulerad:
What stood out in 2022?
The past year can be characterized by momentous regulatory approvals: THREE NEW GENE THERAPIES APPROVED FOR RARE DISEASES, A NEW RECORD FOR THE SECTOR! THREE CAR-T CELL THERAPIES WERE EXPANDED AS TREATMENT OPTIONS AGAINST DIFFERENT CANCERS, WITH TWO APPROVED AS EARLIER-LINE TREATMENTS. THE FIRST-EVER ‘OFF-THE-SHELF’ T-CELL THERAPY APPROVED GLOBALLY But 2022 wasn’t without challenge. After two recordbreaking years of investment, economic headwinds produced a difficult financing environment. Despite these obstacles, the sector is poised to make unprecedented progress this year
What can we anticipate for the rest of 2023?
The next wave of cell and gene therapies is arriving fast. In the United States (US) alone, we are looking in the range of 10 regulatory decisions this year on cell and gene therapies. This includes decisions on: SEVERAL GENE THERAPIES FOR RARE DISEASES, INCLUDING TWO FOR SICKLE CELL DISEASE – AND THE FIRST-EVER CRISPR GENE EDITING THERAPY. THE FIRST-EVER ADOPTIVE CELL THERAPY FOR A SOLID TUMOR. THE FIRST GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY One thing is clear – a larger patient population is on the horizon, and we need to collectively act to ensure that healthcare systems are ready to deliver the future of medicine.
Och slutligen har bloggen under förmiddagen fått mejl från 2 phi,are om en ny forskningsrapport (från Korea) som är inom just stamcellsområdet. Tack till Jörgen och Oscar.
Eftersom detta inlägg redan är långt går jag inte in på vad denna nya aktuella studie innehåller. Ni får traggla er igenom den själva. Dock lägger jag ut forskarnas användande av HoloMonitor.
SOCS3 Protein Mediates the Therapeutic Efficacy of Mesenchymal Stem Cells against Acute Lung Injury
4.4. Quantification of Morphometric Changes in Alveolar Macrophages
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| Representative images of RAW264.7 alveolar macrophages,taken at 100× magnification using HoloMonitor cell imaging system (bottom panel; bar 100 µm). The color bar indicates cell height (thickness); higher is red, lower is blue. |
Sååå,efter X antal koppar kaffe 😎 fick jag äntligen ihop detta inlägg.Bland all aktuell Reg Med info valde jag ut ovanstående.Jag kunde ha inkluderat att Kanada är på G med sin Reg Med forskning,men utrymmet räcker inte för att ta med allt som händer inom området. Behöver jag ytterligare poängtera att Reg Med är snubblande nära sitt definitiva genombrott? Nää,trodde inte det heller. Har man läst igenom inlägget noggrannt listar man ut det själv.Jag låter ändå kritiska U.S. Government Accountability Office ge sin syn på läget från en aktuell rapport: The field of regenerative medicine and advanced therapies—including cell, gene, and tissue-based therapies—is evolving and interdisciplinary. Practitioners believe these therapies have the potential to revolutionize patient care and improve lives. The promise of such therapies to ameliorate, or cure, previously untreatable diseases and conditions depends, in part, on the existence of a robust, well-trained workforce.
Mvh the99
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