How far are we from a new generation of stem cell therapy?

Turn from . . . The mystery of medical mission life lies in how a fertilized egg develops into a complex organism.
in this process, stem cells that can multiply and differentiate indefinitely play an extremely important role, it can be said that stem cells are the origin of human life.
more than 60 years, scientists have never stopped studying stem cells.
the research results in this field, has been ranked in the "20th century in the world's top ten scientific and technological achievements" in time of time, also won the Nobel Prize in physiology or medicine in 2012.
, stem cell therapy remains a major focus, with a proliferation of new pharmaceutical companies such as Bayer, Novo Nordisk and Takeda, which are also involved in the development and clinical application of the technology.
Today, we'll take readers to see how potential stem cells and the therapies behind them are. More than 60 years
, the discovery of a new generation of stem cells dates back to the 1960s.
this is a class of cells with infinite or eternal self-renewal capabilities that produce at least one type of highly differentiated child cell.
this characteristic opens up a broad prospect for its application in the field of medicine.
With the continuous research and exploration of human beings, more than ten first-generation stem cell therapy products have been approved for clinical use worldwide, in addition to stem cell transplantation.
, for example, FCB-Pharmicell's Hearticell-AMI, was approved in South Korea in 2011 for the treatment of acute myocardial infarction, and Prochymal, developed by Osiris, was approved in Canada in 2012 for the treatment of childhood transplant anti-host disease (GVHD).
A new generation of stem cell therapies based on genetic and cell modification are emerging, according to a review published in May this year by Nature Reviews Drug Discovery.
these treatments are expected to improve the effectiveness and specificity of stem cell therapies and to expand the range of diseases treated to a wider range of areas.
screenshot source: Nature reviews drug discovery three applications of the current, stem cell for disease treatment application prospects mainly include the following aspects: First, for the development of cell therapy.
, hematopoietic stem cell transplantation (also known as bone marrow transplantation) is one of the most mature stem cell therapies, which reconstructs the patient's normal hematopoietic and immune system by intravenous injection of hematopoietic stem and protoblastation.
, the treatment has been approved in several countries for the treatment of malignant blood diseases such as leukemia.
the development of "universal" cell therapy using induced pluripotent stem cells (iPSC) in newly developed stem cell therapies.
this type of therapy can induce stem cells to differentiate into specific tissue cells, treating a wide range of diseases, such as inflammation, autoimmune diseases, heart failure, and stroke.
, Bayer, Takeda and other companies are promoting the clinical development of such products.
, stem cells can be designed for tissue repair, genetic diseases, etc. the second
is as a carrier of gene therapy.
, such as OTL-101, a gene therapy for hematopoietic stem cells developed by Orchard, isolates hematopoietic stem cells from patients, introduces the ADA gene that expresses adenosine deaminase through a slow-virus vector, and then sends them back to the patient.
the treatment of patients with severe combined immunodeficiency caused by adenosine deaminase deficiency has achieved positive results, with 20 subjects still maintaining a 100% overall survival rate after two years of treatment. the third
is for tissue/organ replacement therapy.
this is the scope of regenerative medicine, in short, using stem cell technology to replace the body's "unhealthy" parts.
, important progress has been made in early research in this area.
, for example, using stem cells to regenerate myocardial muscles is expected to help repair the injured heart; to create human skin, promising revolutionary treatments for those suffering from hair loss, scarring or inherited skin disease; and even potential to promote brain tissue regeneration, potentially helping patients with brain injuries such as ischemic stroke or concussion regain function.
it is worth noting that, in addition to stem cell transplantation, stem cell therapies currently being developed in China are part of clinical trials.
global pharmaceutical companies have dabbled in the broad prospects of stem cells in the field of disease treatment, in addition to the favor of capital, a number of national regulatory authorities have also issued relevant policies to promote the development of this technology.
this context, including Novo Nordisk, Bayer, Takeda and other global pharmaceutical companies have been involved in this field.
Novo Nordisk: The company has been studying the differentiation of pluripotent stem cells into insulin-producing beta cells for more than 20 years, and has achieved preclinical proof-of-concept and, through collaboration, began to develop stem cell therapies that can be used for Parkinson's disease.
2018, Novo Nordisk announced an exclusive partnership with the University of California, San Francisco (UCSF) to develop stem cell therapy to treat type 1 diabetes and other serious chronic diseases.
based on the agreement, Novo Nordisk is granted a technical license to produce a range of human embryonic stem cells (hESC) that comply with good manufacturing practices (GMP) and to further develop these products into future regenerative medicine therapies.
Bayer: In 2019, Bayer will build its cell therapy pipeline by acquiring BlueRock, which specializes in the development of stem cell therapies using the iPSC platform.
BlueRock's iPSC technology platform can recode healthy skin cells, blood cells, or other cells to differentiate them into any cells that need to be regenerated, such as nerve cells or liver cells.
BlueRock iPSCs platform diagram (Photo: BlueRock official website): In 2019, Takeda and Kyoto University announced that a CAR-T therapy developed using "induced pluripotent stem cell technology" has moved from academia to industry for clinical development by Takeda.
the treatment uses an induced pluripotent stem cell bank to create a "universal" CAR-T therapy that can be fine-tuned to the clinical needs of different patients.
it is expected to be treated through a major cell pool that produces a large number of homologous cells.
the next generation of stem cell therapy developers in addition to the world's leading large pharmaceutical companies, but also a number of new companies have targeted the field of stem cell therapy, with cutting-edge technology to develop a new generation of stem cell therapy.
below shows the progress of only a few innovative companies in this area for readers to understand.
Elp Regenerative Medicine: Founded in 2016, is a Chinese biotechnology company focused on drug-incurable degenerative and functionally impaired diseases, cell therapy product development and commercial applications, with iPSC technology at its core.
has established a product line that includes ischemic heart failure, dilated cardiomyopathy, stroke, pediatric cerebral palsy, Parkinson's disease, etc.
it is understood that the company has successfully established more than 90 iPSC cell lines, including ALS, in cooperation with a number of scientific research units.
Hodd Biology: A team of neuroandal and stem cell scientists at Johns Hopkins University in the United States was founded in Hangzhou in January 2017.
, relying on its strengths in the neurodifferentiation and cell engineering of human iPSCs/ESCs, completed tens of millions of dollars in Round A financing in 2018.
according to the company's official website, there are currently three products under development, are in the preclinical stage, the proposed development of indications including: spinal cord injury, stroke, brain injury, autism and so on.
Fate Therapeutics: Founded in 2007, it is a company focused on cancer immunotherapy.
it is understood that the company's strategy for developing "ready-to-use" CAR-T therapy is to genetically engineer iPSC to screen and preserve iPSC cell lines that can amplify and differentiate into T-cells and NK cells.
April, the company partnered with Janssen globally to develop new CAR-NK and CAR-T cell candidates using its iPSC platform and Jansen's proprietary antigen binding domain.
Gamida Cell: An Israeli-based immunotherapy company focused on cancer and rare genetic diseases.
the company has exclusive NAM technology that uses the epigenetic function of small molecule NAM (Niacinamide) to improve the amplification of their in vitro culturewhile while preserving cell characteristics and functions.
may, the company announced that its cord blood stem cell therapy, omidubicel, has reached the end of a Phase 3 trial to treat patients with high-risk malignant blood cancers who need a bone marrow transplant.
this is the first bone marrow transplant product to be recognized by the FDA for breakthrough therapy, and has also been eligible for orphan drugs in the United States and the European Union.
NAM Platform Technology (Photo: Gamida Cell) Mogrify: Mogrify, founded in 2016 at Cambridge Science Park, is committed to the systematic development of new cell therapies in areas such as regenerative medicine.
the company has established proprietary iPSCs and embryonic stem cell (ESCs) technology that transforms any human cell type into any other human cell type.
October 2019, the company completed its first $16 million round A financing.
April, the company partnered with Sangamo Therapeutics to develop ready-to-use CAR-Treg therapy in combination with stem cell and zinc finger protein genetic engineering to treat inflammatory and autoimmune diseases.
Notch Therapeutics: Co-founded by two leaders in iPSC and T cell differentiation at the University of Toronto, Dr. Juan Carlos Z?iga-Pfl?cker and Dr. Peter Zandstra.
the eTN platform established by the company, iPSCs differentiate into mature and functional immune cells by building a cell culture environment that simulates thymus microenvironments.
the platform can amplify iPSCs in large quantities and differentiate into immunotherapy cells that are highly consistent and can be genetically engineered.
Allogene Therapeutic: Allogene, founded in 2018, focuses on the development of allogeneic CAR-T therapy.
the company's founders are Dr. Arie Belldegrun and Dr. David Chang.
the company has previously partnered with pfizer, Cellectis and Servier, a number of well-known biopharmaceutical companies.
last November, Allogene spent a partnership with Notch to jointly develop a new generation of ready-to-use CAR-T therapy, derived from iPSC, which can be used to treat blood cancers such as multiple myeloma, leukemia and non-Hodgkin's lymphoma.
concluding recently, some of the cell therapies under development have been positive in late clinical terms, which adds confidence to the industry.
, Mesoblast's heterogeneous intergene-insist stem cell candidate remestemcel-L filed a biologics licensing application (BLA) in the United States in April this year, and Gamida Cell's umbilical cord blood stem cell therapy omidubicel treatment of high-risk malignant blood cancer patients, the third phase of clinical treatment, also received positive results in May this year.
While stem cell therapy still faces many challenges, we believe that as the new generation of stem cell therapies moves closer to clinical use, the clearer the understanding of the challenges that need to be addressed."
we expect these new stem cell therapies to change patients' treatment and make their diseases better controlled.
Source: 1 Orchard Therapeutics Presents Two-Year Follow-Up Data Versus Historical Control from Registration Al Trial of OTL-101 for Treatment of ADA-SCID. Retrieved February 22, 2019, from Bayer Acquires Blue Rock Therapeutics to build position in cell therapy, Retrieved August 8, 2019, from first iPSC-Derived CAR T-Cell The CreatIng by Kyoto University CiRA and Takeda Therapis sgheaud y Sangamo announce collaboration and license agreement for Mogrify's iPSC- and ESC-derived t c.