Category Archives: Cell Therapy

An International Biomedical Research Alliance Committed to Curing Disease

Nova Southeastern University (NSU) is at the forefront of conducting pioneering biomedical research with the launch of its soon-to-be-completed, 215,000 sq. ft. Center for Collaborative Research (CCR), one of the largest and most advanced research facilities in Florida with state-of-the-art laboratories. The CCR will be home to a diverse group of accomplished physician-scientists and basic science researchers, representing many interdisciplinary sciences. As part of its bold new research mission, the university has launched the NSU Cell Therapy Institute, a unique collaboration with leading medical research scientists from Swedens world-renowned Karolinska Institutet (KI). KI is globally recognized for its Nobel Assembly, which awards the Nobel Prize in Physiology or Medicine annually.

Mission:The NSU Cell Therapy Institute is dedicated to the discovery and development of innovative translational biomedical research, focused on the potential of cell-based therapies to prevent, treat and cure life-threatening and debilitating diseases. The Institute is accelerating the advancement of next-generation approaches to precision medicine such as targeted immunotherapy and regenerative medicine with an initial focus on treating cancers, heart disease and disorders causing blindness. The Institute is prioritizing establishing collaborations with other world-class universities, hospitals, research institutions and corporate partners.

With an overriding mission to contribute to the improvement of human health through research and education, Karolinska Institutet provides more than 40% of the medical academic research conducted in Sweden and offers the country s broadest range of education in medicine and health sciences. Many of the discoveries made at Karolinska Institutet have been of great significance, including the pacemaker, the gamma knife, the sedimentation reaction, the Seldinger technique and the preparation of chemically pure insulin. Since 1901, the Nobel Assembly at Karolinska Institutet has selected the Nobel laureates in Physiology or Medicine.

Research Team: Evren Alici, M.D., Ph.D.; Adil Duru, Ph.D.; Carin Dahlberg, Ph.D. Summary: Natural killer cells (NK-cells) are cytotoxic lymphocytes critical to the innate immune system. Discovered at Karolinska Institutet in 1974, NK-cells hold the potential to be programmed to selectively bind to and destroy tumor cells with very few side-effects. Preliminary human studies are encouraging in targeting multiple myeloma, a deadly form of blood cancer. New studies will be advanced at the NSU Cell Therapy Institute, including investigating novel approaches harnessing the potency of NK-cells against a broad range of difficult-to-treat cancers.

Research Team: Andreas Lundqvist, Ph.D.; Shannon Murray, Ph.D. Summary: Cancer can employ multiple defense mechanisms to suppress the immune system and avoid detection. For example, myeloid-derived suppressor cells (MDSC) can be used by cancer cells to down-regulate the immune system and diminish the cancer killing capabilities of T-cells, NK-cells, dendritic cells, macrophages and other immune responses. The NSU Cell Therapy Institute is advancing novel anti-MDSC approaches to overcome suppression defenses while recruiting targeted immune responses by both NK-cells and T-cells, as potential monotherapy or in combination with other anti-cancer treatments for malignant melanoma and kidney cancer.

Research Team: Richard Jove, Ph.D.; Thomas Temple, M.D. Summary: While standard cancer therapies such as chemotherapy and radiation can destroy many kinds of cancers, cancer stem cells possess the ability to escape, survive and metastasize to distant sites in the body. These cells typically develop into new tumors, destroy normal tissues such as bone and are often resistant to most therapies. The NSU Cell Therapy Institute is advancing novel approaches to modulating signaling pathways, such as JAK/STAT, which are critical to cancer stem cell survival and regeneration of normal tissue. By inhibiting cancer stem cell pathways, cancer recurrence may be eliminated, resulting in durable remissions. Conversely, activating these same pathways can enhance regeneration of damaged normal tissues including bone.

Research Team: Karl-Henrik Grinnemo, M.D., Ph.D.; Vladimir Beljanski, Ph.D. Summary: There are currently no available options to repair vital heart muscle and tissue following a heart attack, and heart transplantation is a highly limited option. The NSU Cell Therapy Institute is developing novel culturing systems to produce pure populations of mesenchymal stromal cells (MSCs) and at the same time activate cardiac progenitor cell differentiation. These activated cardiac MSCs can be differentiated into three types of cardiac cells: cardiomyocytes; endothelial cells; and smooth muscle cells. The ability of these cells to regenerate the damaged myocardium, while at the same time possessing traits that prevent immune rejection and inflammatory response, makes this a highly promising approach to restoring healthy heart function.

Research Team: Cecilia Osterholm Corbascio, PhD; Vladimir Beljanski, Ph.D. Summary: Valvular heart disease (VHD) is a major cause of death and disability worldwide, yet the mechanisms of this process remain largely unknown. Furthermore, the properties of currently used bioprosthetic valves lead to valve degeneration and subsequent heart failure. A regenerative approach would allow for the generation of new heart valves by combining the patients own stem cells with a biological matrix not prone to immunologically mediated deterioration. The focus of this research at NSU will be to study valvular heart disease from decoding mechanistic pathways to creating new valves from the patients own stem cells.

Research Team: Outi Hovata, M.D., Ph.D.; Ben Josey, Ph.D.Summary: Macular Degeneration (MD - dry & wet forms) is the leading cause of vision loss in Americans aged 60 or older. The dry form of the disease affects 90-95% of sufferers and causes vision loss in the center of a patients field of vision due to deterioration of the macula which is in the center of the retina. The NSU Cell Therapy Institute is developing a novel approach to cure dry MD by transplanting human stem cell-derived retinal pigment epithelial cell (RPE cells). This approach targets dry MD by replacing the degenerating, damaged cells in the eye with healthy RPE cells to restore full function and vision. Immune rejection has been a major impediment in such transplant techniques, so this program is developing differentiated RPE cells combined with technology that induces transplant tolerance, eliminating the need for chronic immunosuppression.

The NSU Cell Therapy Institute offers cutting-edge molecular and cellular profiling analyses to support its research programs, and those of its collaborative partners, with the highest quality of comprehensive scientific resources, facilities and staff, including in the areas of:

Philanthropy, sponsored research and other key collaborative partnerships play a critical role in the NSU Cell Therapy Institutes mission to impact the understanding, prevention, and treatment of a broad range of life-threatening and debilitating diseases. The NSU Cell Therapy Institute offers many kinds of sponsorship and partnering options for individual donors, foundations, and corporations in order to accelerate next-generation cures to patients in urgent need.

For more information about the NSU Cell Therapy Institute, please contact:

Douglas W. Calder Director of Development & Strategic Planning NSU Cell Therapy Institute 3301 College Avenue Ft. Lauderdale, FL 33314 Phone: (954) 262-3382 Mobile: (772) 418-6302 Email: dcalder@nova.edu

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Cell Therapy Institute - Nova Southeastern University NSU

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Founded in 2009 by Nobel prize winner Professor Sir Martin Evans and Ajan Reginald, former Global Head of Emerging Technologies at Roche, CTL develops life-saving and life altering regenerative medicines. CTLs team of scientists, physicians, and experienced management have discovered and developed a pipeline of world-class regenerative medicines.

Sir Martin Evans' unique expertise in discovering rare stem cells led to CTLs innovative drug discovery engine that can uniquely isolate very rare and potent tissue specific stem cells. This exceptional class of cells is then engineered into unique disease-specific cellular regenerative medicines. Each medicine is disease specific and forms part of CTLs world-class portfolio of four off the shelf blockbuster medicines all scheduled for launch before 2020.

The products in late stage clinical trials include Heartcel which regenerates the damaged heart of adults with coronary artery malformations and children with Kawasaki Disease and Bland White Garland Syndrome. In 2014, Heartcel reported unprecedented heart regeneration clinical trial results and is scheduled to launch in 2018 to treat ~400,000 patients worldwide. Myocardion is in Phase II/III trials and treats mild-moderate heart failure affecting 10 million patients worldwide. Tendoncel is the worlds first topical regenerative medicine, for early intervention of severe tendon injuries, and has completed Phase II trials. It is designed to treat the >1 million severe tendon injuries each year in the US and Europe. Skincel is for skin regeneration, and is due to complete Phase II trials in 2015. It is designed to address ulceration and wrinkles.

CTL combines world-class science and management expertise to bring life-saving regenerative medicines to market.

European Society of Gene and Cell Therapy Congress, 17-20 September 2015, Helsinki,Finland (ESGCT 2015)

4th International Conference and Exhibition on Cell & Gene Therapy, August 10-12, 2015, London (CGT 2015)

The International Society for Stem Cell Research Annual Meeting, 24th-27th June 2015, Stockholm, Sweden (ISSCR 2015)

British Society for Gene and Cell Therapy Annual Conference, 9th-11th June 2015, Strathclyde, Glasgow (BSGCT 2015)

Original post:
Cell Therapy Ltd

Gene therapy and cell therapy are overlapping fields of biomedical research with the goals of repairing the direct cause of genetic diseases in the DNA or cellular population, respectively. These powerful strategies are also being focused on modulating specific genes and cell subpopulations in acquired diseases in order to reestablish the normal equilibrium. In many diseases, gene and cell therapy are combined in the development of promising therapies.

In addition, these two fields have helped provide reagents, concepts, and techniques that are elucidating the finer points of gene regulation, stem cell lineage, cell-cell interactions, feedback loops, amplification loops, regenerative capacity, and remodeling.

Gene therapy is defined as a set of strategies that modify the expression of an individuals genes or that correct abnormal genes. Each strategy involves the administration of a specific DNA (or RNA).

Cell therapy is defined as the administration of live whole cells or maturation of a specific cell population in a patient for the treatment of a disease.

Gene therapy: Historically, the discovery of recombinant DNA technology in the 1970s provided the tools to efficiently develop gene therapy. Scientists used these techniques to readily manipulate viral genomes, isolate genes, identify mutations involved in human diseases, characterize and regulate gene expression, and engineer various viral vectors and non-viral vectors. Many vectors, regulatory elements, and means of transfer into animals have been tried. Taken together, the data show that each vector and set of regulatory elements provides specific expression levels and duration of expression. They exhibit an inherent tendency to bind and enter specific types of cells as well as spread into adjacent cells. The effect of the vectors and regulatory elements are able to be reproduced on adjacent genes. The effect also has a predictable survival length in the host. Although the route of administration modulates the immune response to the vector, each vector has a relatively inherent ability, whether low, medium or high, to induce an immune response to the transduced cells and the new gene products.

The development of suitable gene therapy treatments for many genetic diseases and some acquired diseases has encountered many challenges and uncovered new insights into gene interactions and regulation. Further development often involves uncovering basic scientific knowledge of the affected tissues, cells, and genes, as well as redesigning vectors, formulations, and regulatory cassettes for the genes.

While effective long-term treatments for anemias, hemophilia, cystic fibrosis, muscular dystrophy, Gauschers disease, lysosomal storage diseases, cardiovascular diseases, diabetes, and diseases of the bones and joints are elusive today, some success is being observed in the treatment of several types of immunodeficiency diseases, cancer, and eye disorders. Further details on the status of development of gene therapy for specific diseases are summarized here.

Cell therapy: Historically, blood transfusions were the first type of cell therapy and are now considered routine. Bone marrow transplantation has also become a well-established protocol. Bone marrow transplantation is the treatment of choice for many kinds of blood disorders, including anemias, leukemias, lymphomas, and rare immunodeficiency diseases. The key to successful bone marrow transplantation is the identification of a good "immunologically matched" donor, who is usually a close relative, such as a sibling. After finding a good match between the donors and recipients cells, the bone marrow cells of the patient (recipient) are destroyed by chemotherapy or radiation to provide room in the bone marrow for the new cells to reside. After the bone marrow cells from the matched donor are infused, the self-renewing stem cells find their way to the bone marrow and begin to replicate. They also begin to produce cells that mature into the various types of blood cells. Normal numbers of donor-derived blood cells usually appear in the circulation of the patient within a few weeks. Unfortunately, not all patients have a good immunological matched donor. Furthermore, bone marrow grafts may fail to fully repopulate the bone marrow in as many as one third of patients, and the destruction of the host bone marrow can be lethal, particularly in very ill patients. These requirements and risks restrict the utility of bone marrow transplantation to some patients.

Cell therapy is expanding its repertoire of cell types for administration. Cell therapy treatment strategies include isolation and transfer of specific stem cell populations, administration of effector cells, induction of mature cells to become pluripotent cells, and reprogramming of mature cells. Administration of large numbers of effector cells has benefited cancer patients, transplant patients with unresolved infections, and patients with chemically destroyed stem cells in the eye. For example, a few transplant patients cant resolve adenovirus and cytomegalovirus infections. A recent phase I trial administered a large number of T cells that could kill virally-infected cells to these patients. Many of these patients resolved their infections and retained immunity against these viruses. As a second example, chemical exposure can damage or cause atrophy of the limbal epithelial stem cells of the eye. Their death causes pain, light sensitivity, and cloudy vision. Transplantation of limbal epithelial stem cells for treatment of this deficiency is the first cell therapy for ocular diseases in clinical practice.

Several diseases benefit most from treatments that combine the technologies of gene and cell therapy. For example, some patients have a severe combined immunodeficiency disease (SCID) but unfortunately, do not have a suitable donor of bone marrow. Scientists have identified that patients with SCID are deficient in adenosine deaminase gene (ADA-SCID), or the common gamma chain located on the X chromosome (X-linked SCID). Several dozen patients have been treated with a combined gene and cell therapy approach. Each individuals hematopoietic stem cells were treated with a viral vector that expressed a copy of the relevant normal gene. After selection and expansion, these corrected stem cells were returned to the patients. Many patients improved and required less exogenous enzymes. However, some serious adverse events did occur and their incidence is prompting development of theoretically safer vectors and protocols. The combined approach also is pursued in several cancer therapies.

Further information on the progress and status of gene therapy and cell therapy on various diseases is listed here.

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Gene Therapy and Cell Therapy Defined | ASGCT - American ...

On July 1, 2014, the United States Food and Drug Administration granted 'breakthrough therapy' designation to CTL019, the anti-CD19 chimeric antigen receptor T-cell therapy developed at the University of Pennsylvania. This is the first personalized cellular therapy for cancer to be so designated and occurred 25 years after the first publication describing genetic redirection of T cells to a surface antigen of choice. The peer-reviewed literature currently contains the outcomes of more than 100 patients treated on clinical trials of anti-CD19 redirected T cells, and preliminary results on many more patients have been presented. At last count almost 30 clinical trials targeting CD19 were actively recruiting patients in North America, Europe, and Asia. Patients with high-risk B-cell malignancies therefore represent the first beneficiaries of an exciting and potent new treatment modality that harnesses the power of the immune system as never before. A handful of trials are targeting non-CD19 hematological and solid malignancies and represent the vanguard of enormous preclinical efforts to develop CAR T-cell therapy beyond B-cell malignancies. In this review, we explain the concept of chimeric antigen receptor gene-modified T cells, describe the extant results in hematologic malignancies, and share our outlook on where this modality is likely to head in the near future.

2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

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Going viral: chimeric antigen receptor T-cell therapy for ...

Hello, Public Anonymous User! Our Story Hello and thank you for your support of US Preventive Medicine (USPM).

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Lack of sleep affects your immune system. Mother knows best at least it appears that way when it comes to lack of sleep. It turns out that lack of sleep really may make us more prone to catching colds and the flu

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Slide: 1 / of 1 . Caption: With several companies on the verge of mass-marketing genetic tests that claim to read agings cellular clock, some researchers say the science isnt yet ready for prime-time use. The tests measure telomeres, or protein sheaths that prevent the tips of chromosomes from fraying.

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A small study suggests that stem cells from a stranger may be as good as a patients own in undoing the damage after a heart attack has weakened the hearts ability to pump blood.

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Study says stem cells ? even a stranger's ? may repair heart attack damage

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Newswise Fat progenitor cells may contribute to cancer growth by fortifying the vessels that provide needed blood to tumors, according to preclinical research findings by investigators at The University of Texas Health Science Center at Houston (UTHealth). The results were reported in Cancer Research, a journal of the American Association for Cancer Research. Studies of groups of people have demonstrated a link between obesity and certain cancers; however, the physiological causes have not been identified

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