Category Archives: Gene therapy

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NEW YORK(CBSNewYork) It has been called the cancer breakthrough of the year by a major scientific journal.

Therapy that eradicates cancer using a patients own cells has already saved a number of terminal leukemia patients, CBS 2s Dr. Max Gomez reported.

It has been the Holy Grail of cancer therapy and it harnesses the patients own immune system to attack cancer.

Now, a major new study has shown how to do that when treating leukemia. It involves using gene therapy to convert a patients white blood cells into killers.

Ive had several doctors tell me there is nothing else that can be done, leukemia patient Paolo Cavalli said, It is difficult with a new family to think about those things.

After six years of chemotherapy, stem cell transplants, and multiple relapses Cavalli was out of options for his leukemia.

I dont think I had many days left, he said.

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Dr. Max Gomez: Gene Therapy Could Be Lifesaver For Cancer Patients

By combining a synthetic scaffolding material with gene delivery techniques, researchers at Duke University are getting closer to being able to generate replacement cartilage where it's needed in the body.

Performing tissue repair with stem cells typically requires applying copious amounts of growth factor proteins -- a task that is very expensive and becomes challenging once the developing material is implanted within a body. In a new study, however, Duke researchers found a way around this limitation by genetically altering the stem cells to make the necessary growth factors all on their own.

They incorporated viruses used to deliver gene therapy to the stem cells into a synthetic material that serves as a template for tissue growth. The resulting material is like a computer; the scaffold provides the hardware and the virus provides the software that programs the stem cells to produce the desired tissue.

The study appears online the week of Feb. 17 in the Proceedings of the National Academy of Sciences.

Farshid Guilak, director of orthopaedic research at Duke University Medical Center, has spent years developing biodegradable synthetic scaffolding that mimics the mechanical properties of cartilage. One challenge he and all biomedical researchers face is getting stem cells to form cartilage within and around the scaffolding, especially after it is implanted into a living being.

The traditional approach has been to introduce growth factor proteins, which signal the stem cells to differentiate into cartilage. Once the process is under way, the growing cartilage can be implanted where needed.

"But a major limitation in engineering tissue replacements has been the difficulty in delivering growth factors to the stem cells once they are implanted in the body," said Guilak, who is also a professor in Duke's Department of Biomedical Engineering. "There's a limited amount of growth factor that you can put into the scaffolding, and once it's released, it's all gone. We need a method for long-term delivery of growth factors, and that's where the gene therapy comes in."

For ideas on how to solve this problem, Guilak turned to his colleague Charles Gersbach, an assistant professor of biomedical engineering and an expert in gene therapy. Gersbach proposed introducing new genes into the stem cells so that they produce the necessary growth factors themselves.

But the conventional methods for gene therapy are complex and difficult to translate into a strategy that would be feasible as a commercial product.

This type of gene therapy generally requires gathering stem cells, modifying them with a virus that transfers the new genes, culturing the resulting genetically altered stem cells until they reach a critical mass, applying them to the synthetic cartilage scaffolding and, finally, implanting it into the body.

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Regenerating orthopedic tissues within the human body

Duke researchers use gene therapy to direct stem cells into becoming new cartilage on a synthetic scaffold even after implantation into a living body

By Ken Kingery

By combining a synthetic scaffolding material with gene delivery techniques, researchers at Duke University are getting closer to being able to generate replacement cartilage where it's needed in the body.

Performing tissue repair with stem cells typically requires applying copious amounts of growth factor proteinsa task that is very expensive and becomes challenging once the developing material is implanted within a body. In a new study, however, Duke researchers found a way around this limitation by genetically altering the stem cells to make the necessary growth factors all on their own.

They incorporated viruses used to deliver gene therapy to the stem cells into a synthetic material that serves as a template for tissue growth. The resulting material is like a computer; the scaffold provides the hardware and the virus provides the software that programs the stem cells to produce the desired tissue.

The study appears online the week of Feb. 17 in the Proceedings of the National Academy of Sciences.

An artistic rendering of human stem cells on the polymer scaffolds. Photo courtesy of Charles Gersbach and Farshid Guilak, Duke University

The traditional approach has been to introduce growth factor proteins, which signal the stem cells to differentiate into cartilage. Once the process is under way, the growing cartilage can be implanted where needed.

But a major limitation in engineering tissue replacements has been the difficulty in delivering growth factors to the stem cells once they are implanted in the body, said Guilak, who is also a professor in Dukes Department of Biomedical Engineering. Theres a limited amount of growth factor that you can put into the scaffolding, and once its released, its all gone. We need a method for long-term delivery of growth factors, and thats where the gene therapy comes in.

A microscopic view using electron microscopy of human stem cells and viral gene carriers adhering to the fibers of a polymer scaffold. Photo courtesy of Charles Gersbach and Farshid Guilak, Duke University

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Gene Therapy Might Grow Replacement Tissue Inside the Body

Stamford, CT (PRWEB) February 06, 2014

Alliance for Cancer Gene Therapy (ACGT) the nations only non-profit dedicated exclusively to cell and gene therapies for cancer is redoubling its efforts to treat and combat cancers in the New Year, and announces $1 million in recent grants.

The funding spread across three grants will support basic and clinical science at premier institutions in and outside the United States, with ACGTs mission top-of-mind: uncovering effective, innovative cancer treatments that supersede radiation, chemotherapy and surgery.

This January, ACGTs President and Co-Founder Barbara Netter has announced two Young Investigator Grants that provide promising researchers with $250,000 each for two- to three-year studies.

Young Investigator Fan Yang, PhD Assistant Professor of Orthopedic Surgery and Bioengineering at Stanford University will use the funds to research new treatment options for pediatric brain cancer, the leading cause of death from childhood cancer. Dr. Yangs study will deploy adult-derived stem cells to target solid brain tumor cells, which are often highly invasive and difficult to treat.

Arnob Banerjee, MD, PhD Assistant Professor of Hematology and Oncology at the University of Maryland will use ACGTs funding to further develop the long-term effectiveness of immune-mediated treatments, the most advanced form of gene therapy.

It is imperative that the best and brightest young scientists like Fan Yang and Arnob Banerjee have the funds necessary to study and treat cancer, Netter said. This was my husband Edwards vision in 2001, when gene cell therapy was a fledgling science. We are proud to continue his pioneering foresight today. Partnerships with Dr. Yang, a former fellow at MIT, and Dr. Banerjee, a former fellow and instructor at the University of Pennsylvania, dovetail with ACGTs record of funding outstanding researchers and physicians with the capability to make unprecedented breakthroughs.

The Young Investigator grants come on the heels of a $500,000 Investigators Award to John Bell, PhD, Senior Research Scientist and Professor of Medicine at the Ottawa Hospital Research Institute in Canada. Dr. Bell has worked extensively with oncolytic viruses man-made viruses that target only cancer cells, and spare patients the harrowing side-effects of chemotherapy, radiation or surgery and has discovered the enormous promise they offer in the war on cancer.

The research and trials funded by ACGTs grant have the potential to treat metastatic and recurrent brain cancer, extend patients survival timeline, and vastly improve patients quality of life during treatment, Dr. Bell said.

ACGT has served as a major funding engine in the fight against cancer since its formation in 2001, and has provided nearly $25 million in grants to date. ACGT was created by Barbara and Edward Netter after the loss of their daughter-in-law to breast cancer. Since Edwards passing in 2011, Barbara Netter has led the foundation as President and Co-Founder, continuing her husbands vision.

Link:
Alliance for Cancer Gene Therapy (ACGT) Targets Brain, Pediatric Cancers with $1 Million in New Grants

Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome -- an often fatal condition that causes organ damage and other medical complications.

Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and Stroke (NINDS) report their unique strategy for treating the disease the week of Feb. 3-7 in Proceedings of the National Academy of Sciences (PNAS).

Researchers were able to genetically insert into the cells a gene that produces a critical lysosomal enzyme (called IDUA) and then inject the engineered cells into mice to treat the disorder. Follow up tests showed the treatment resulted in a complete metabolic correction of the disease, according to the authors.

"Our findings demonstrate a unique and somewhat surprising delivery pathway for lysosomal enzymes," said Dao Pan, PhD, corresponding author and researcher in the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's. "We show proof of concept that platelets and megakaryocytes are capable of generating and storing fully functional lysosomal enzymes, which can lead to their targeted and efficient delivery to vital tissues where they are needed."

The mice tested in the study modeled human Hurler syndrome, a subset of disease known as mucopolysaccharidosis type I (MPS I), one of the most common types of lysosomal storage diseases. MPS I is a lysosomal storage disease in which people do not make an enzyme called lysosomal alpha-L-iduronidase (IDUA).

IDUA helps break down sugar molecules found throughout the body, often in mucus and fluids around joints, according to the National Library of Medicine/National Institutes of Health. Without IDUA, sugar molecules build up and cause organ damage. Depending on severity, the syndrome can also cause deafness, abnormal bone growth, heart valve problems, joint disease, intellectual disabilities and death.

Enzyme replacement therapy can be used to treat the disease, but it is only temporary and not curative. Bone marrow transplant using hematopoietic stem cells also has been tested on some patients with mixed results. The transplant procedure can carry severe risks and does not always work.

Pan and her colleagues -- including Roscoe O. Brady, MD, a researcher at NINDS -- report that using platelets and megakaryocytes for gene therapy is effective and could reduce the risk of activating cancer-causing oncogenes in hematopoietic stem cells.

The authors said tests showed that human megakaryocytic cells were capable of overexpressing IDUA, revealing their capacity for potential therapeutic benefit. While engineering megakaryocytes and platelets for infusion into their mouse models of Hurler, the scientists report they were able to release IDUA directly into amply sized extracellular spaces or inside micro-particles as the cells matured or activated. The cells were able to produce and package large amounts of functional IDUA and retained the capacity to cross-correct patient cells.

After infusing mouse models of Hurler with the genetically modified cells, researchers said this led to long-term normalization of IDUA levels in the animal's blood with versatile delivery routes and on-target preferential distribution to the liver and spleen. The treatment led to a complete metabolic correction of MPS I in most peripheral organs of the mice.

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Gene therapy may be possible cure for Hurler syndrome: Mouse Study

PUBLIC RELEASE DATE:

4-Feb-2014

Contact: Nick Miller nicholas.miller@cchmc.org 513-803-6035 Cincinnati Children's Hospital Medical Center

CINCINNATI Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome an often fatal condition that causes organ damage and other medical complications.

Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and Stroke (NINDS) report their unique strategy for treating the disease the week of Feb. 3-7 in Proceedings of the National Academy of Sciences (PNAS).

Researchers were able to genetically insert into the cells a gene that produces a critical lysosomal enzyme (called IDUA) and then inject the engineered cells into mice to treat the disorder. Follow up tests showed the treatment resulted in a complete metabolic correction of the disease, according to the authors.

"Our findings demonstrate a unique and somewhat surprising delivery pathway for lysosomal enzymes," said Dao Pan, PhD, corresponding author and researcher in the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's. "We show proof of concept that platelets and megakaryocytes are capable of generating and storing fully functional lysosomal enzymes, which can lead to their targeted and efficient delivery to vital tissues where they are needed."

The mice tested in the study modeled human Hurler syndrome, a subset of disease known as mucopolysaccharidosis type I (MPS I), one of the most common types of lysosomal storage diseases. MPS I is a lysosomal storage disease in which people do not make an enzyme called lysosomal alpha-L-iduronidase (IDUA).

IDUA helps break down sugar molecules found throughout the body, often in mucus and fluids around joints, according to the National Library of Medicine/National Institutes of Health. Without IDUA, sugar molecules build up and cause organ damage. Depending on severity, the syndrome can also cause deafness, abnormal bone growth, heart valve problems, joint disease, intellectual disabilities and death.

Enzyme replacement therapy can be used to treat the disease, but it is only temporary and not curative. Bone marrow transplant using hematopoietic stem cells also has been tested on some patients with mixed results. The transplant procedure can carry severe risks and does not always work.

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Mouse study shows gene therapy may be possible cure for Hurler syndrome

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one.

Although the technology is still in its infancy, it has been used with some success.

Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.

In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene.

A carrier called a vector must be used to deliver the therapeutic gene to the patient's target cells.

Currently, the most common type of vectors are viruses that have been genetically altered to carry normal human DNA.

Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner.

Scientists have tried to harness this ability by manipulating the viral genome to remove disease-causing genes and insert therapeutic ones. Target cells such as the patient's liver or lung cells are infected with the vector.

The vector then unloads its genetic material containing the therapeutic human gene into the target cell.

The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state. In theory it is possible to transform either somatic cells (most cells of the body) or cells of the germline (such as sperm cells, ova, and their stem cell precursors).

Excerpt from:
Gene therapy - Science Daily

Two adults with a rare disease that causes gradual loss of eyesight had their vision improved after being treated with a new gene therapy, according to preliminary results from a new study.

The study involved six patients ages 35 to 63 with choroideremia, an inherited condition with no cure that causes vision problems early in life, and eventually leads to blindness. Patients have a mutation in a gene called CHM, which causes light-sensitive cells in the eye to slowly stop working.

The goal behind the new gene therapy is to use a safe virus to deliver a working copy of the gene to the right part of the eye to prevent the cells from degenerating. [7 Diseases You Can Learn About From a Genetic Test]

The new study was an early test of the therapy in which the researchers aimed to carry out the treatment without causing damage to the eye. (Patients must have an eye surgery so that the virus can be injected under the retina with a fine needle).

The result showed that the treatment did not cause harm, and in fact, improved vision in a few of the patients.

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Six months after the treatment, four patients recovered the visual acuity (clearness or acuteness of vision) that they had before the surgery, and developed increased sensitivity to light. And two patients had improvements in vision: They were able to read two to four more lines on a sight chart.

"We did not expect to see such dramatic improvements in visual acuity," study researcher Robert MacLaren, of the Nuffield Laboratory of Ophthalmology at the University of Oxford in the U.K., said in a statement. It is still too early to know if the improvements will last, but they have so far been maintained for as long as two years, MacLaren said.

The study is the first to test gene therapy in patients before they'd experienced significant thinning of the retinal cells, MacLaren said.

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Gene therapy improves vision for some with rare disease

PUBLIC RELEASE DATE:

23-Dec-2013

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

In a recent study published in the Neural Regeneration Research (Vol. 8, No. 33, 2013), Prof. Feng Li and team from Zhongshan School of Medicine, Sun Yat-sen University in China, synthesized a 19-nt oligonucleotide targeting BACE1, the key enzyme in amyloid beta protein (A) production, and introduced it into the pSilenCircle vector to construct a short hairpin (shRNA) expression plasmid against the BACE1 gene. Researhcers transfected this vector into C17.2 neural stem cells and primary neural stem cells, resulting in downregulation of the BACE1 gene, which in turn induced a considerable reduction in reducing A protein production. This technique combining cell transplantation and gene therapy will open up novel therapeutic avenues for Alzheimer's disease, particularly because it can be used to simultaneously target several pathogenetic changes in the disease.

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Article: " Targeting -secretase with RNAi in neural stem cells for Alzheimer's disease therapy " by Zhonghua Liu, Shengliang Li, Zibin Liang, Yan Zhao, Yulin Zhang, Yaqi Yang, Minjuan Wang, Feng Li (Department of Neurobiology and Anatomy, Zhongshan School of Medicine, Sun Yat-sen University, Guangzhou 510080, Guangdong Province, China)

Liu ZH, Li SL, Liang ZB, Zhao Y, Zhang YL, Yang YQ, Wang MJ, Li F. Targeting -secretase with RNAi in neural stem cells for Alzheimer's disease therapy. Neural Regen Res. 2013;8(33):3095-3106.

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

Full text: http://www.sjzsyj.org/CN/article/downloadArticleFile.do?attachType=PDF&id=783

Excerpt from:
Combination of cell transplantation and gene therapy for Alzheimer's disease

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The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight.

Cellular therapy products include cellular immunotherapies, and other types of both autologous and allogeneic cells for certain therapeutic indications, including adult and embryonic stem cells. Human gene therapy refers to products that introduce genetic material into a persons DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition.

Although some cellular therapy products have been approved, CBER has not yet approved any human gene therapy product for sale. However, the amount of cellular and gene therapy-related research and development occurring in the United States continues to grow at a fast rate. CBER has received many requests from medical researchers and manufacturers to study cellular and gene therapies and to develop cellular and gene therapy products. In addition to regulatory oversight of clinical studies, CBER provides proactive scientific and regulatory advice to medical researchers and manufacturers in the area of novel product development.

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Cellular & Gene Therapy Products - Food and Drug Administration