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Blind Mice Get Experimental Stem Cell Treatment For Blindness

§ October 2nd, 2012 § Filed under Nano Medicine Comments Off on Blind Mice Get Experimental Stem Cell Treatment For Blindness

April Flowers for redOrbit.com Your Universe Online

Columbia University ophthalmologists and stem cell researchers have developed an experimental treatment for blindness using the patients skin cells, which has improved the vision of blind mice in testing.

The findings of this research, published online in the journal Molecular Medicine, suggest that induced pluripotent stem cells (iPS) could soon be used to improve vision in people with macular degeneration and other eye retina diseases. iPS cells are derived from adult human skin cells but have embryonic qualities.

With eye diseases, I think were getting close to a scenario where a patients own skin cells are used to replace retina cells destroyed by disease or degeneration, says Stephen Tsang, MD, PhD, associate professor of ophthalmology and pathology & cell biology. Its often said that iPS transplantation will be important in the practice of medicine in some distant future, but our paper suggests the future is almost here.

Scientists were very excited by the advent of human iPS cells when they were discovered in 2007, as they provide a way to avoid the ethical complications of embryonic stem cells. Another advantage is that the iPS cells are created from the patients own skin, eliminating the need for anti-rejection medications. Like the ethically challenged embryonic cells, iPS cells can develop into any type of cell. To-date, no iPS cells have been implanted into people, but many ophthalmologists say that the eye would prove to be ideal testing ground for iPS therapies.

The eye is a transparent and accessible part of the central nervous system, and thats a big advantage. We can put cells into the eye and monitor them every day with routine non-invasive clinical exams, Tsang said. And in the event of serious complications, removing the eye is not a life-threatening event.

Professor Tsang is running a new preclinical iPS study using human iPS cells derived from the skin cells of a 53-year-old donor. The cells were first transformed with a cocktail of growth factors into cells in the retina that lie underneath the eyes light-sensing cells.

Retina cells nourish the light-sensing cells and protect the fragile cells from excess light, heat and cellular debris. In macular degeneration and retinitis pigmentosa, retina cells die, which allows the photoreceptor cells to degenerate causing the patient to lose their vision. It is estimated that 30 percent of people will have some form of macular degeneration by the time they are 75 years old, as it is the leading cause of vision loss in the elderly. Currently, it affects 7 million Americans and that is expected to double by 2020.

The Columbia research team injected the iPS-derived retina cells into the right eyes of 34 mice that had a genetic mutation that caused their retina cells to degenerate. In many of the mice, the iPS cells assimilated into the retina without disruption and functioned as normal retina cells well into the animals old age. Mice in the control group, who received injections of saline or inactive cells, showed no improvement in retina tests.

Our findings provide the first evidence of life-long neuronal recovery in a preclinical model of retinal degeneration, using stem cell transplant, with vision improvement persisting through the lifespan, Tsang says. And importantly, we saw no tumors in any of the mice, which should allay one of the biggest fears people have about stem cell transplants: that they will generate tumors.

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Stem cells improve visual function in blind mice

§ October 2nd, 2012 § Filed under Nano Medicine Comments Off on Stem cells improve visual function in blind mice

ScienceDaily (Oct. 1, 2012) An experimental treatment for blindness, developed from a patient’s skin cells, improved the vision of blind mice in a study conducted by Columbia ophthalmologists and stem cell researchers.

The findings suggest that induced pluripotent stem (iPS) cells — which are derived from adult human skin cells but have embryonic properties — could soon be used to restore vision in people with macular degeneration and other diseases that affect the eye’s retina.

“With eye diseases, I think we’re getting close to a scenario where a patient’s own skin cells are used to replace retina cells destroyed by disease or degeneration,” says the study’s principal investigator, Stephen Tsang, MD, PhD, associate professor of ophthalmology and pathology & cell biology. “It’s often said that iPS transplantation will be important in the practice of medicine in some distant future, but our paper suggests the future is almost here.”

The advent of human iPS cells in 2007 was greeted with excitement from scientists who hailed the development as a way to avoid the ethical complications of embryonic stem cells and create patient-specific stem cells. Like embryonic stem cells, iPS cells can develop into any type of cell. Thousands of different iPS cell lines from patients and healthy donors have been created in the last few years, but they are almost always used in research or drug screening.

No iPS cells have been transplanted into people, but many ophthalmologists say the eye is the ideal testing ground for iPS therapies.

“The eye is a transparent and accessible part of the central nervous system, and that’s a big advantage. We can put cells into the eye and monitor them every day with routine non-invasive clinical exams,” Tsang says. “And in the event of serious complications, removing the eye is not a life-threatening event.”

In Tsang’s new preclinical iPS study, human iPS cells — derived from the skin cells of a 53-year-old donor — were first transformed with a cocktail of growth factors into cells in the retina that lie underneath the eye’s light-sensing cells.

The primary job of the retina cells is to nourish the light-sensing cells and protect the fragile cells from excess light, heat, and cellular debris. If the retina cells die — which happens in macular degeneration and retinitis pigmentosa — the photoreceptor cells degenerate and the patient loses vision. Macular degeneration is a leading cause of vision loss in the elderly, and it is estimated that 30 percent of people will have some form of macular degeneration by age 75. Macular degeneration currently affects 7 million Americans and its incidence is expected to double by 2020.

In their study, the researchers injected the iPS-derived retina cells into the right eyes of 34 mice that had a genetic mutation that caused their retina cells to degenerate.

In many animals, the human cells assimilated into mouse retina without disruption and functioned as normal retina cells well into the animals’ old age. Control mice that got injections of saline or inactive cells showed no improvement in retina tests.

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International Stem Cell Corp Granted Key Patent for Liver Disease Program

§ October 2nd, 2012 § Filed under Nano Medicine Comments Off on International Stem Cell Corp Granted Key Patent for Liver Disease Program

CARLSBAD, CA–(Marketwire – Sep 25, 2012) – International Stem Cell Corporation ( OTCQB : ISCO ) (www.internationalstemcell.com) (“ISCO” or “the Company”) a California-based biotechnology company, today announced that the United States Patent and Trademark Office (USPTO) has granted the Company a patent for a method of creating pure populations of definitive endoderm, precursor cells to liver and pancreas cells, from human pluripotent stem cells.This patent is a key element of ISCO’s metabolic liver disease program and allows the Company to produce the necessary quantities of precursor cells in a more efficient and cost effective manner.

The patent, 8,268,621, adds to the Company’s growing portfolio of proprietary technologies relating to the development of potential treatments for incurable diseases using human parthenogenetic Stem Cells (hpSC).Human parthenogenetic stem cells are unique pluripotent stem cells that offer the possibility to reduce the cost of health care while avoiding the ethical issues that surround the use of fertilized human embryos.Aside from the Company’s current liver disease program, this new patented method can be used as a route to create pancreatic and endocrine cells that could be used in future studies of diabetes and other metabolic disorders.

ISCO currently has the largest collection of hpSC including cell lines which immune match the donor, as is the case with induced pluripotent stem cells (iPS), and cell lines which immune-match millions of individuals and potentially reduce tissue rejection issues.The Company is focusing its therapeutic development efforts on three clinical applications where cell and tissue therapy is already proven but where there currently is an insufficient supply of safe and efficacious cells: Parkinson’s disease, inherited/metabolic liver diseases and corneal blindness.

About International Stem Cell Corporation

International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products.ISCO’s core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs) hence avoiding ethical issues associated with the use or destruction of viable human embryos.ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at http://www.internationalstemcell.com.

To receive ongoing corporate communications via email, visit: http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0

To like our Facebook page or follow us on Twitter for company updates and industry related news, visit: http://www.facebook.com/InternationalStemCellCorporation and http://www.twitter.com/intlstemcell

Safe harbor statement

Statements pertaining to anticipated developments, the potential use of technologies to develop therapeutic products and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as “will,” “believes,” “plans,” “anticipates,” “expects” or “estimates”) should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products and the management of collaborations, regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company’s business, particularly those mentioned in the cautionary statements found in the company’s Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.

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Global Human Embryonic Stem Cell (hESC) Research Industry

§ October 2nd, 2012 § Filed under Nano Medicine Comments Off on Global Human Embryonic Stem Cell (hESC) Research Industry

NEW YORK, Sept. 3, 2012 /PRNewswire/ — Reportlinker.com announces that a new market research report is available in its catalogue:

Global Human Embryonic Stem Cell (hESC) Research Industry

http://www.reportlinker.com/p0960425/Global-Human-Embryonic-Stem-Cell-hESC-Research-Industry.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Biological_Therapy

This report analyzes the worldwide markets for Human Embryonic Stem Cell (hESC) Research in US$ Million. The report provides separate comprehensive analytics for the US, Europe, and Rest of World. Annual estimates and forecasts are provided for the period 2009 through 2018. The report profiles 26 companies including many key and niche players such as Advanced Cell Technology, Inc., BD Biosciences, BioTime, Inc., Cell Cure Neurosciences Ltd., Cellartis AB, GE Healthcare, Millipore Corporation, Molecular Transfer, Inc., PerkinElmer, Inc., Pfizer, Inc., Research & Diagnostics Systems, Inc., Reliance Life Sciences Ltd., Stem Cell Network, Stemina Biomarker Discovery, Inc., UK Stem Cell Bank, and ViaCyte, Inc. Market data and analytics are derived from primary and secondary research. Company profiles are primarily based upon search engine sources in the public domain.

I. INTRODUCTION, METHODOLOGY & PRODUCT DEFINITIONS

Study Reliability and Reporting Limitations I-1

Disclaimers I-2

Data Interpretation & Reporting Level I-2

Quantitative Techniques & Analytics I-3

Product Definitions and Scope of Study I-3

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Bestellen Sie den kostenlosen Newsletter von 4investors:

§ October 2nd, 2012 § Filed under Nano Medicine Comments Off on Bestellen Sie den kostenlosen Newsletter von 4investors:

PRESS RELEASE: MagForce to radically enforce market set up strategy with new management team

MagForce AG / MagForce to radically enforce market set up strategy with new management team . Processed and transmitted by Thomson Reuters ONE. The issuer is solely responsible for the content of this announcement.

* Focus on most important value drivers in the short-term

* Experienced senior executives added to the management board

* Core functions will be centralized in Munich

* Million Euro cost savings expected from resturcturing measures

Berlin, Germany, October 1, 2012 – MagForce AG (Frankfurt, XETRA: MF6), a leading medical device company in the field of nanomedicine with a focus on oncology, today announced additions to the management board and further measures to enforce commercialization of NanoTherm therapy. Thus, focussing on establishing the NanoTherm therapy in the oncology market as well as on its commercialization for near-term value generation.

In the short to mid term, MagForce will dedicate its financial resources to the post marketing trial in glioblastoma, which is expected to start in early 2013 and is supposed to make a significant contribution to its strategic set up. Simultaneously, the Company will also focus on the commercialization of its NanoTherm therapy with its distribution partners including DELRUS and TekGrup, development partners such as the Mayo Clinic for gastro-intestinal cancer and the Department of Urology at Duesseldorf University for prostate cancer, as well as the production of nanoparticles.

In line with this strategy, the Company’s core functions, including clinical and business development, medical affairs plus legal & IP will be concentrated at the MagForce site in Munich where these teams are already located. Production of nanoparticles, NanoActivators((TM)), software engineering as well as the finance department will remain at the Company’s registered office in Berlin. All early research activities will be reduced to a minimum and will be outsourced as deemed neccessary.

In this respect experienced senior executives are added to the management board. Prof Dr Hoda Tawfik, currently VP Clinical Development and Medical Affairs, will be appointed COO. Christian von Volkmann, currently acting CFO, will assume the position of CFO, both effective immediately. They will jointly lead the Company as co-CEOs going forward. The founder and long-term Board member Dr Andreas Jordan will step down from the management board of MagForce to pursue new projects, but will continue to serve the Company as an advisor.

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PRESS RELEASE: MagForce publishes half-year report

§ October 2nd, 2012 § Filed under Nano Medicine Comments Off on PRESS RELEASE: MagForce publishes half-year report

MagForce AG / MagForce publishes half-year report . Processed and transmitted by Thomson Reuters ONE. The issuer is solely responsible for the content of this announcement.

* Comprehensive restructuring introduced

* Integration of medical opinion leaders into post-marketing trial to increase acceptance of NanoTherm therapy

* Strategic and distribution partnerships established

* Cost savings expected to amount to million Euros due to a package of measures

* Loss for the period reduced by 22% to EUR3.6 million

Berlin, Germany, October 1, 2012 – MagForce AG (Frankfurt, Xetra: MF6), a leading medical device company in the field of nanomedicine with a focus on oncology, today announced the financial results for the first half of the 2012 fiscal year, ending June 30, 2012. Simultaneously, the company released details of a package of measures (see today’s press release “MagForce to radically enforce market setup strategy with new management team”). With this restructuring, MagForce is completing a period of important strategic refocus.

“In the first half of the year, we laid out the basic requirements for the acceptance and future commercialization of our NanoTherm therapy, and for the future development of our company. Today we are concluding this phase of strategic focus with the introduction of vital measures and additions to the management team. Over the short term, we are concentrating all our efforts and financial resources on establishing NanoTherm therapy in the area of oncology and consequentially on the post-marketing clinical trial in glioblastoma. Together with strategic and distribution partners, we want to reinforce the commercialization of NanoTherm therapy, ensuring sufficient funding.” commented Christian von Volkmann, CFO.

In the first half of the year, MagForce was able to successfully finalize distribution partnerships with DELRUS (Russia) and Tek Grup (Turkey), as well as a development partnership with the Mayo Clinic (USA).

During the reporting period, the company recorded a loss of EUR3.6 million (prior year period: EUR4.6 million). The decline is essentially a result of the restructuring measures introduced at the end of 2011 and the accompanying strategic change. Personnel expenses decreased due to the reduction in staff, particularly in the second management level, as well as in commercial functions.

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MagForce publishes half-year report

§ October 2nd, 2012 § Filed under Nano Medicine Comments Off on MagForce publishes half-year report

Comprehensive restructuring introduced

Integration of medical opinion leaders into post-marketing trial to increase acceptance of NanoTherm therapy

Strategic and distribution partnerships established

Cost savings expected to amount to million Euros due to a package of measures

Loss for the period reduced by 22% to 3.6 million

Berlin, Germany, October 1, 2012 – MagForce AG (Frankfurt, Xetra: MF6), a leading medical device company in the field of nanomedicine with a focus on oncology, today announced the financial results for the first half of the 2012 fiscal year, ending June 30, 2012. Simultaneously, the company released details of a package of measures (see today`s press release “MagForce to radically enforce market setup strategy with new management team”). With this restructuring, MagForce is completing a period of important strategic refocus.

“In the first half of the year, we laid out the basic requirements for the acceptance and future commercialization of our NanoTherm therapy, and for the future development of our company. Today we are concluding this phase of strategic focus with the introduction of vital measures and additions to the management team. Over the short term, we are concentrating all our efforts and financial resources on establishing NanoTherm therapy in the area of oncology and consequentially on the post-marketing clinical trial in glioblastoma. Together with strategic and distribution partners, we want to reinforce the commercialization of NanoTherm therapy, ensuring sufficient funding.” commented Christian von Volkmann, CFO.

In the first half of the year, MagForce was able to successfully finalize distribution partnerships with DELRUS (Russia) and Tek Grup (Turkey), as well as a development partnership with the Mayo Clinic (USA).

During the reporting period, the company recorded a loss of 3.6 million (prior year period: 4.6million). The decline is essentially a result of the restructuring measures introduced at the end of 2011 and the accompanying strategic change. Personnel expenses decreased due to the reduction in staff, particularly in the second management level, as well as in commercial functions.

In the first half of the year, the company successfully raised 4.5 million by issuing new shares. After the reporting period end, two further capital increases took place with gross proceeds amounting to 665,000. The capital measures serve to safeguard liquidity and maintain business operations, as well as financing further business growth.

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MagForce to radically enforce market set up strategy with new management team

§ October 2nd, 2012 § Filed under Nano Medicine Comments Off on MagForce to radically enforce market set up strategy with new management team

Focus on most important value drivers in the short-term

Experienced senior executives added to the management board

Core functions will be centralized in Munich

Million Euro cost savings expected from resturcturing measures

Berlin, Germany, October 1, 2012 – MagForce AG (Frankfurt, XETRA: MF6), a leading medical device company in the field of nanomedicine with a focus on oncology, today announced additions to the management board and further measures to enforce commercialization of NanoTherm therapy. Thus, focussing on establishing the NanoTherm therapy in the oncology market as well as on its commercialization for near-term value generation.

In the short to mid term, MagForce will dedicate its financial resources to the post marketing trial in glioblastoma, which is expected to start in early 2013 and is supposed to make a significant contribution to its strategic set up. Simultaneously, the Company will also focus on the commercialization of its NanoTherm therapy with its distribution partners including DELRUS and TekGrup, development partners such as the Mayo Clinic for gastro-intestinal cancer and the Department of Urology at Duesseldorf University for prostate cancer, as well as the production of nanoparticles.

In line with this strategy, the Company`s core functions, including clinical and business development, medical affairs plus legal & IP will be concentrated at the MagForce site in Munich where these teams are already located. Production of nanoparticles, NanoActivators(TM), software engineering as well as the finance department will remain at the Company`s registered office in Berlin. All early research activities will be reduced to a minimum and will be outsourced as deemed neccessary.

In this respect experienced senior executives are added to the management board. Prof Dr Hoda Tawfik, currently VP Clinical Development and Medical Affairs, will be appointed COO. Christian von Volkmann, currently acting CFO, will assume the position of CFO, both effective immediately. They will jointly lead the Company as co-CEOs going forward. The founder and long-term Board member DrAndreas Jordan will step down from the management board of MagForce to pursue new projects, but will continue to serve the Company as an advisor.

Along with these measures the headcount of MagForce will be reduced from 27 to 12. These measures are meant to annually save the Company a seven digit Euro amount.

“With this restructuring MagForce is completing a period of important strategic refocus. In the short-term, we have to concentrate all our efforts and financial resources on the development of strategic partnerships as well as on establishing NanoTherm therapy in the area of oncology. Thus, concentrating on the post-marketing clinical study planned at different sites in Germany, which is critical to achieving acceptance of our NanoTherm therapy in the German medical community. We are encouraged about the progress we are making and need to ensure that we translate this momentum into commercial success,” commented Prof Dr Hoda Tawfik, COO of MagForce AG.

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Power of the Dream Ventures Acquires Genetic Immunity

§ October 1st, 2012 § Filed under Nano Medicine Comments Off on Power of the Dream Ventures Acquires Genetic Immunity

BUDAPEST, HUNGARY–(Marketwire – Sep 28, 2012) – Power of the Dream Ventures, Inc. ( OTCBB : PWRV ) is pleased to announce the acquisition of Genetic Immunity, Inc., a Phase III clinical stage biotechnology company with experimental nanomedicines that will lead to the next generation of immunotherapies, in a market that is projected to reach $11.00 billion by 2018.

Genetic Immunity’s lead product candidate is an immune boosting drug for HIV, which is now only treated by antiretroviral drugs that decrease the ability of the immune system to fight with the virus. DermaVir HIV-specific Immunotherapy is the first of a new line of curative nanomedicine products developed for the treatment and eradication of HIV. In addition, Genetic Immunity has implemented a Predictive Genomic Biomarker as companion diagnostics to accurately predict potential responder patients to DermaVir treatment. Such innovations towards personalized medicine increase the treatment effect and reduce the cost of pivotal trials in full compliance with the FDA’s initiatives to improve products for patients (Driving Biomedical Innovation, 2011). In addition, following a successful DermaVir trial on HIV-infected adults, the US government is sponsoring a Phase II pediatric clinical trial.

DermaVir is the first therapeutic vaccine that consistently boosts broadly directed central memory T-cells in human subjects. This immune response has been correlated with containment of viremia in Elite Controllers. The Phase II randomized, multicenter, placebo controlled trial conducted in Germany established the optimal DermaVir dose and provided data that demonstrates the killing of HIV-infected cells. Therefore, the eradication of HIV or the conversion of progressors to Elite Controllers via DermaVir immunization became a testable hypothesis.

“This acquisition milestone is the result of our collaboration for a common goal to sell stock in Genetic Immunity to the public. The acquisition of a private company by a public one corresponds to a novel IPO, and offers tremendous upside potential for all the shareholders of Genetic Immunity and PWRV. Starting today, financial market participants will have an opportunity to determine the price of our business. We are eager, because comparable technology companies trade at over half a billion dollar valuation. On a more personal note, I believe that Genetic Immunity’s platform technology is a once in a lifetime opportunity. For the first time we are truly in reach of eradicating a highly infectious disease. We are proud to be a part of the process whereby the innovations presented by Genetic Immunity can become publicly available,” commented Viktor Rozsnyay, CEO of Power of the Dream Ventures.

“Through this highly innovative financial transaction, Genetic Immunity achieves its corporate objective to become a publicly traded company and to retain the control over the business. The financial and technological synergy between the two Companies provides for substantial growth opportunity and high return on investment to our shareholders,” said Dr. Julianna Lisziewicz, CEO of Genetic Immunity.

With the acquisition Genetic Immunity becomes a 100% wholly owned subsidiary of Power of the Dream Ventures, Inc.

About PDV Power of the Dream Ventures, Inc. is a leading technology holding company. We identify and harness the unique technological prowess of Hungary’s high-tech industry, turning promising ideas and ready to market products/technologies into global industry leaders. We focus on developing, acquiring, or co-developing technologies that originate exclusively in Hungary. For more information, please visit http://www.powerofthedream.com

About Genetic Immunity Genetic Immunity is a clinical stage technology company committed to discovering, developing, manufacturing and commercializing a new class of immunotherapeutic biologic drugs for the treatment of viral infections, cancer and allergies. The Company’s two distinguished technology platforms will revolutionize the treatment of these chronic diseases. Our Langerhans’ cell targeting nanomedicines are exceptional in both safety and immune modulating activity boosting specific Th1-type central memory T cells. Such immune responses differ from antibodies induced by vaccines. These are essential to eliminate infected cells or cancerous cells, and balance the immune reactivity in response to allergens. Our IT team generated a complex algorithm to match the mechanism of action of our drugs with clinical efficacy. In the future, we will predict the clinical and immunological benefits of our drugs based on the patient’s disease and genomic background. The unique mixture of our technologies represents the next generation of personalized but not individualized medicines ensuring a longer and higher economic return.

Genetic Immunity’s primary focus is the development of DermaVir that acts to boost the immune system of HIV-infected people to eliminate infected cells that remain in the reservoirs after successful antiretroviral treatment. Three clinical trials conducted in the EU and US showed that DermaVir immunizations were as safe as placebo and only four sequential patch treatments required to reduce the HIV infected cells in the blood within 24 weeks.

In 1988 Drs. Lisziewicz and Lori founded Genetic Immunity in the US after they described the 1st patient whose immune system was boosted to control HIV after treatment interruption (Lisziewicz et al. New England Journal of Medicine 1999) that lead to the invention of DermaVir. The Company’s innovative technology team directed by Dr. Lisziewicz, a champion of immune busting therapies, is now headquartered in Budapest, Hungary. For more information please visit http://www.geneticimmunity.com

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Research and Markets: Nanomedicine, Vol 508. Cancer, Diabetes, and Cardiovascular, Central Nervous System, Pulmonary …

§ October 1st, 2012 § Filed under Nano Medicine Comments Off on Research and Markets: Nanomedicine, Vol 508. Cancer, Diabetes, and Cardiovascular, Central Nervous System, Pulmonary …

DUBLIN–(BUSINESS WIRE)–

Research and Markets (http://www.researchandmarkets.com/research/9zfhfp/nanomedicine_vol) has announced the addition of Elsevier Science and Technology’s new book “Nanomedicine, Vol 508. Cancer, Diabetes, and Cardiovascular, Central Nervous System, Pulmonary and Inflammatory Diseases” to their offering.

This volume in the Methods in Enzymology series comprehensively covers Cancer, Cardiovascular and the central nervous system of Nanomedicine. With an international board of authors, this volume is split into sections that cover subjects such as Diabetes and nanotechnology as potential therapy, Nanomedicines for inflammatory diseases, and Development and use of ceramide nanoliposomes in cancer.

Comprehensively covers Cancer, Cardiovascular and the central nervous system of Nanomedicine. An international board of authors. Split into sections that cover subjects such as Diabetes and nanotechnology as potential therapy, Nanomedicines for inflammatory diseases, and Development and use of ceramide nanoliposomes in cancer.

Key Topics Covered:

– Preparation, characterization, and cellular associations of silicon logic-embedded vectors

– Post-formulation peptide drug loading of nanostructures

– Application of mesoporous silica nanoparticles in intracellular drug delivery

– Multifunctional anticancer nanomedicine based on a magnetically responsive cyanoacrylate polymer

– Development and Use of Ceramide Nanoliposomes in Cancer

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Anticipated short-term cell therapy industry clinical milestones

§ September 30th, 2012 § Filed under Nano Medicine Comments Off on Anticipated short-term cell therapy industry clinical milestones

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What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.  


There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.


CellCoTec (http://www.cellcotec.com)
  • Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application.  The CE mark application is under review and they anticipate a response in October.  
  • This device and the potential emergence of Sanofi’s MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.



ERYtech Parma (http://www.erytech.com)

  • Their ‘pivotal’ phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2012. 


GamidaCell (http://www.gamidacell.com)

  • Their ‘pivotal’ phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012.  They have not been shy about the fact they expect to be in the market in 2013.


Innovacell (http://www.innovacell.com)

  • They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed.  Status unknown.


Miltenyi Biotec (www.miltenyibiotec.com)

  • Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.


NovaRx (http://www.novarx.com)

  • Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next ‘interim analysis’ in February.


NuVasive (http://www.nuvasive.com)

  • They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.


Sanofi’s Genzyme (http://www.genzyme.com)

  • Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.


Hope that’s helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.  



–Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

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The cost of clinical trial data bias/loss, FDA’s new job and the need for bold leadership.

§ September 30th, 2012 § Filed under Nano Medicine Comments Off on The cost of clinical trial data bias/loss, FDA’s new job and the need for bold leadership.

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The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.


Before you right this post off as the stuff of conspiracy theories, fear-mongering, and ‘alternative world views’ consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.


Here’s the underlying premise as succinctly described by author Ben Goldacre:

“Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.

When trials throw up results that companies don’t like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug’s true effects. Regulators see most of the trial data, but only from early on in a drug’s life, and even then they don’t give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion.”

Authors M. Todwin and J. Abramson summarize it thusly:

“Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge.”

Here is a great list of very recent resources that may convince you of the merits of this concern:

Yesterday, the US Secretary of Health and Human Services announced (in an FR notice) that the FDA was now charged with ensuring all organizations comply with the heretofore enacted but relatively unenforced  requirement to submit all relevant clinical trial data to http://www.clinicaltrials.gov

For further commentary on this move see the following reports from:
What is abundantly clear to me is that the FDA is left almost powerless – and if not powerless than certainly without sufficient resources – to successfully enforce its new power.  This requires collective industry leadership.  Bold, industry-initiated standards, infrastructure and old-fashioned peer pressure.

Here’s what I wish.  

I wish that as a cell therapy industry we – through organizations like ISSCR, ARM, ISCT, etc and leading publishers of some of our leading journals like Regenerative Medicine, Cytotherapy, Cell Stem Cell, Stem Cells, etc – would take a leadership position on an issue like this.

I believe that as a relatively small and nascent sector of the biopharma industry we are more likely capable of collaborating on something important like this than larger, more established [entrenched] and diverse sectors.  Of course it requires the political will and cajones.

The payoff from our sector in taking a leadership role on this issue could potentially be enormous in terms of providing our sector with truly transparent and useful data.  Perhaps even more important would be the public profile such leadership would provide the sector.  Such a move requires bold leadership, pain, and cost but this is the kind of stuff that moves the needle and goes down as critical pivot points in history. 

Just my thought for the day…

–Lee

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Size matters: Researchers warn nano-polyphenols may have reversed effects

§ September 17th, 2012 § Filed under Nano Medicine Comments Off on Size matters: Researchers warn nano-polyphenols may have reversed effects

The beneficial effects attributed to antioxidants such as polyphenols could be reversed when they are used in nano-forms, according to new research.

The UK-based researchers have suggest the antioxidant effects of green tea polyphenols could be reversed when the compounds are used in nano-formulations after research published in Nanomedicine compared the effects of different polyphenol forms on white blood cells.

Led by Professor Diana Anderson from the University of Bradford, UK, the researchers revealed that the known antioxidant properties of polyphenols such as epigallocatechin gallate(EGCG) and theaflavins can be reversed when they are used in their nanoparticle form.

The findings showed that when used in bulk form, these polyphenols exhibited their anticipated antioxidant responses, but the nanoform at higher concentrations had the reverse effect and exhibited statistically significant pro-oxidant effects, which can cause increased DNA damage.

Both the compounds in the bulk form produced statistically significant concentration-dependent reductions in DNA damage, revealed the authors. In contrast, when used in the nano-particle form both theaflavin and EGCG, although initially causing a reduction, produced a concentration-dependent statistically significant increase in DNA damage.

“We didn’t expect these changes,” explained Anderson. “When my PhD student came to me with the results, she assumed she’d made a mistake. But it struck me that I’d seen this happen before – in a study we published in 1994 describing a dose-related switch of properties in Vitamin C in the presence of hydrogen peroxide.

At the time I didn’t think much about it, but this is the first time I’ve seen this happen with the nano-form of a compound,” explained Anderson.

Study details

During the research, Anderson and her colleagues used white blood cells (lymphocytes) from healthy volunteers and cancer patients to assess the potential anti-cancer effects of tea polyphenols. The polyphenols were presented in both bulk and nano- forms and compared against of anti-cancer drugs to measure their protection against DNA damage.

The authors explained that many previous studies have suggested polyphenols to be increasingly useful as anti-cancer compounds, and may also offer beneficial effects to healthy, non-cancer cells due to their antioxidant properties that can quench free radical species.

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Two lessons I learned this week.

§ September 16th, 2012 § Filed under Nano Medicine Comments Off on Two lessons I learned this week.

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I learned two valuable things this week I thought I’d pass on in a Friday afternoon post.  Actually strictly speaking these are likely things I’ve learned before but needed to re-learn or to be ‘reminded’ of their importance.
Please pardon a little stroll away from the typically strict focus on cell therapy — but in a way that’s the theme of today’s post.
1.  Take time each week to read something from outside your specific profession or job focus.  
I’m not talking here about the importance of escaping in the evening with a fiction novel (also very important) but rather reading something professional but from well outside your area of focus.  Here are my examples.

I always read WIRED magazine.  Aside from GEN it’s the only magazine I read.  Just reading something outside of cell therapy or biotech often infuses me with an idea that otherwise would have never occurred to me like the need for a cell therapy X Prize or cellular aggregates as microcarriers or tissue-engineered memory and processing devices or even just the conviction to better represent cell therapy to the broader world out there of scientists, engineers, journalists, policy-makers, or perhaps people with too much money looking to be inspired and wanting to make a difference.

Similarly, on a flight this week I reached into the seat pocket in front of me and discovered a recent copy of the Journal of the American Medical Association.  I read a fascinating article that has me excited about an idea for how we as a cell therapy industry might lead the way in addressing clinical trial and data transparency that would put our sector in a leadership position, lend the industry a much-needed spotlight, and has the potential to facilitate the kind of meta-analysis and data-mining that could only be done through data aggregation.  I believe the concept has the potential to be disproportionately significant for a sector defined by so many small, under-powered trials.
The idea may never see the light of day but the point is the source of the inspiration.  In order to ‘think’ outside the box one typically has to ‘be’ outside the box.  Lesson?  Spend some time outside your box.
2. It often takes something very small to make a disproportionately significant impact on someone.  
I was reminded recently through an exchange of simple kindnesses just how little it sometimes takes to make a big difference in someone’s life.  For you what might be so easy to give might be of unparalleled value to someone for whom that is so unattainable.  
Lesson?  When the opportunity knocks for you to give something small or simple, take it.  This kind of charity almost always has the potential to be mre impactful than you might ever imagine.
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ClassOne Equipment Donation Supports the Nanotechnology Mission at Georgia Tech

§ September 6th, 2012 § Filed under Nano Medicine Comments Off on ClassOne Equipment Donation Supports the Nanotechnology Mission at Georgia Tech

ATLANTA, GA–(Marketwire -09/06/12)- The applications of nanotechnology are vast and have potential to revolutionize medicine, environmental protection methods, and lead to the development of new and innovative systems and devices based on nano materials and processes.

On April 24th, 2009, the Georgia Institute of Technology dedicated the new Marcus Nanotechnology Building; named after its prime sponsor, Mr. Bernie Marcus of the Marcus Foundation. The building is dedicated to exploring new fields of science, technology, and engineering for the benefit of humankind. It is also the headquarters for the Georgia Tech Institute of Electronics and Nanotechnology (IEN).

This signature facility embodies Georgia Tech’s dedication to improving the human condition through advanced science and engineering.

In support of this mission, ClassOne Equipment, a leading supplier of high quality refurbished equipment to the semiconductor, MEMS, LED, wireless, and emerging technology markets, has made a significant contribution of key process equipment.

“We appreciate this significant contribution of equipment that is now contained within the Marcus Organic and Inorganic Cleanroom Laboratories,” said Mark Allen, executive director of the IEN.

ClassOne has core expertise in Suss Microtec, EVG, SPTS, Oxford, Plasmatherm, Semitool, and KLA-Tencor equipment. They can provide a turn-key solution which includes full refurbishment to original specifications, 6-month warranty, and full installation and training by experienced factory trained technicians. ClassOne currently has 40 full-time employees. ClassOne engineers and technicians have worked in technical positions at Suss Microtec, EVG, Semitool, STS, and KLA-Tencor. Since its founding in 2002, ClassOne has refurbished and sold over 2,000 pieces of equipment to more than 500 satisfied customers around the world, including some of the best-known institutes and semiconductor industry labs. In addition to its headquarters in Atlanta, GA, ClassOne has offices in California, Germany, UK, and China.

The IEN is a Georgia Tech interdisciplinary research center designed to enhance support for rapidly growing research programs spanning biomedicine, materials, electronics and nanotechnology.

The IEN is comprised of multiple Electronics and Nanotechnology research units, each offering a unique intellectual focus ranging from basic discovery and innovation to systems realization for academic, industry and government sponsors. Faculty leadership within the IEN centers includes global experts, several of whom are Eminent Scholars and National Academy of Engineering members. IEN faculty and researchers are capable of providing a broad spectrum of research and development activities ranging from basic discovery to systems prototypes.

These research programs are enabled by the IEN Nano, Micro, and Bio Cleanroom Laboratories valued in excess of $400M. These open-user, fee based laboratories are available to global academic, industry, and government clientele, offering a unique and comprehensive laboratory and teaming environment. For more information about IEN please visit: http://www.ien.gatech.edu/

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Research and Markets: Nanomedicine: Technologies and Applications

§ September 6th, 2012 § Filed under Nano Medicine Comments Off on Research and Markets: Nanomedicine: Technologies and Applications

DUBLIN–(BUSINESS WIRE)–

Research and Markets (http://www.researchandmarkets.com/research/svn5wb/nanomedicine_tech) has announced the addition of Woodhead Publishing Ltd’s new book “Nanomedicine: Technologies and applications” to their offering.

Nanotechnology is at the forefront of advances in medicine. Nanomedicine: Technologies and applications provides an important review of this exciting technology and its growing range of applications.

After an introduction to nanomedicine, part one discusses key materials and their properties, including nanocrystalline metals and alloys, nanoporous gold and hydroxyapatite coatings. Part two goes on to review nanomedicine for therapeutics and imaging, before nanomedicine for soft tissue engineering is discussed in part three, including organ regeneration, skin grafts, nanotubes and self-assembled nanomaterials. Finally, nanomedicine for bone and cartilage tissue engineering is the focus of part four, with electrically active biocomposites for smart scaffolds investigated alongside cartilage and bone tissue engineering, regeneration and replacement.

With its distinguished editor and international team of expert contributors, Nanomedicine: Technologies and applications is an indispensible guide for all those involved in the research, development and application of this exciting technology, whilst providing a comprehensive introduction for students and academics interested in this field.

Key Topics Covered:

PART 1 MATERIALS, PROPERTIES AND CONSIDERATIONS

Introduction to nanomedicine

Trends in nanomedicine

Biomedical nanocrystalline metals and alloys: structure, properties and applications

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Product classification in Southeast Asia

§ September 2nd, 2012 § Filed under Nano Medicine Comments Off on Product classification in Southeast Asia

By Wai Mun Poon, Regional Regulatory Manager, EAS Strategic Advice, http://www.eas.asia

As scientific evidence on the relationship between foods, physiological function and disease has grown, so has the functional/fortified food and food supplement market in Southeast Asia. Along with this, many regulatory authorities in Southeast Asian countries have started to pay attention to the controls of these products on the market. The challenge when regulating functional/fortified food products is in classifying these products, as they often fall into the grey zone between medicinal products and foods. While across the region national authorities take varying approaches to controlling these products, in general, the common factors taken into consideration for classification are:

  • Product presentation forms
  • Intended use
  • Ingredients/composition

Product presentation forms

Functional/fortified food products presented in a conventional food forms, such as yogurts, fruit juices and tea are generally regulated under the food regulations in South East Asia. Food supplements presented in pharmaceutical dosage forms such as capsules and tablets, on the other hand, present a very different picture. There is no common approach for regulating these products. Therefore, the same product may be regulated as a food product in one country, but falling under medicinal law in another. In Malaysia, for example, food supplements are regulated according to the Sales of Drug Act and other relevant regulations, whereas in Thailand they fall under the food act [1,2]

Intended use

The intended use of the product is generally considered to be the most important factor in determining the classification of functional/fortified food and food supplement products. The intended use may include recommended amounts, duration of use, targeted groups of users and product claims. If the product does not have a defined duration of use; is suitable for the general population, and has no limits to the amount consumed, it will most likely be considered as a food product in Southeast Asia. If there are limits on any of these however, the product may be classified as a food supplement or in the medicinal product category.

Because food supplements have defined dosages just like medicinal products – for example two capsules per day – and may be targeted at specific groups, product claims play an important role in the determination of whether the product should be a food supplement or medicinal product. Across Southeast Asia there are broadly common requirements on the use of claims on food supplement products: in general these must not bear claims that imply the treatment or prevention of disease. In Malaysia and Singapore, for example, to help companies determine whether a claim is considered medicinal or not, the relevant authorities have published a list of diseases and conditions for which food supplement products are not permitted to bear claims. Examples include cardiovascular and joint diseases.[3] In general, claims relating to diseases and conditions are also not permitted for functional/fortified foods, which tend to be regarded as food products in most Southeast Asian countries.

Ingredients/composition

The choice of ingredients and product composition is also important in the determination of the product category, but there is no common approach to the requirements for the functional/fortified food and food supplement ingredients and compositions across Southeast Asia. Again, some ingredients are allowed for food supplements and functional/fortified foods in some countries, but regarded as medicinal in others. Ingredients most likely to fall into the medicinal product category are botanical ingredients, since many botanicals used as food are known to possess medicinal properties, for example rosemary. To help companies determine the classification of the ingredients, some countries have compiled lists of permitted list of food or food supplement ingredients. In Singapore, for example, the Agri-Food and Veterinary Authority has issued a list of ‘Chinese Medicinal Material commonly use in Food’ to guide companies in the selection of botanical ingredients for their products [4]. Some ingredients in this list include Angelica sinensis, Citrus reticulata and Ginkgo biloba. In Thailand, the Food and Drug Administration has also established list of permitted botanicals and other bioactive substances in food, which includes Garcinia cambogia, Aloe Vera (gel), coenzyme Q10 and royal jelly. In Thailand, products are likely be regulated as medicinal products if these ingredients exceed the permitted levels. However, in some cases companies may apply for special permission to use ingredients not on the list, by submitting safety information to the Thai FDA for approval.

Another approach taken to guide companies in classifying their products based on ingredients and product composition, is through a decision tree. Malaysia follows this approach. According to the decision tree, the following is controlled by Malaysia’s Drug Control Authority:

  • Single and/or combination of bioactive substances, such as dietary fibres, vitamins, minerals and plant stanols
  • Substances that are used strictly for therapeutic purpose, such as pearl powder and gypsum fibrosum
  • Natural ingredients that are not traditionally used as food and have medicinal properties, such as alfafa

However, if the product is presented in a food matrix with more than 80 percent of food ingredients and less than 20 percent of the above three categories of active ingredients, it can be reviewed by the food control authority in terms of classification.[5]

Besides botanicals and other bioactive substances, differences also exist in the requirements for other commonly used functional/fortified food and food supplement ingredients, such as vitamins and minerals, especially when it comes to permitted levels of use. For example, the maximum levels for vitamins and minerals in food supplements in the Philippines and Thailand are based on the recommended daily intake of the nutrients, while most other Southeast Asia countries base them on the levels derived from safety assessments.

It is important, therefore, for manufacturers to consider all of the determination factors: product presentation, intended use, and choice of ingredient and product composition, when determining the appropriate classification of a product. Also noteworthy is that the classification of the product will vary among the different Southeast Asia countries due to different requirements in each of the three consideration factors. Reviewing each country’s regulatory environment and understanding the regulatory boundaries is imperative before planning for product launch and marketing. Companies also need to be clear on the intended usage of product during the product development stage, because if the product cannot be classified, it may not be permitted.

The differences in requirements for functional/fortified food and food supplements cause significant trade challenges. Therefore the Southeast Asian countries are working to harmonise regulatory requirements for various products. One of the product working groups active in the development of common regulatory requirements is the “Traditional Medicine and Health Supplement Product Working Group”, which aims to have common regulatory requirements ready by the end of 2012 to be fully implemented across the Southeast Asian countries by 2015. A key area for harmonisation is maximum levels of vitamins and minerals, which is being developed using a risk-based approach rather than recommended daily intake. It means that in the near future, food supplement products currently classified as medicinal products in some countries due to vitamin and mineral levels higher than the recommended daily intake could be regulated as food supplements across the Southeast Asia region. [6]

References

  1. Ministry of Health Malaysia. National Pharmaceutical Control Bureau. Drug Registration Guidance Document (May 2012). http://portal.bpfk.gov.my/
  2. Thailand Food and Drug Administration. Ministry of Public Health Notification No. 209 (A.D. 2007) Dietary supplement.
  3. Health Sciences Authority Singapore. Guidelines for Health Supplement (March 2012).http://www.hsa.gov.sg/
  4. Agri-Food & Veterinary Authority of Singapore. List of Chinese Medicinal Material (CMM) commonly used in food. Download
  5. Ministry of Health Malaysia. Guide to Classification of Food-Drug Interface Products (June 2011). Download
  6. Associations of South East Asia Nations. Harmonization of Standards and Technical Requirements in ASEAN. http://www.aseansec.org

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Are some cell counts too good to be true? Why some companies’ product data may mislead.

§ September 2nd, 2012 § Filed under Nano Medicine Comments Off on Are some cell counts too good to be true? Why some companies’ product data may mislead.

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This is a cautionary tale about the need for robust product characterization and release specifications for all cell therapy products.
Background
While our food often has a list of ingredients, our drugs don’t.  We rely on our regulatory agencies to rule on the safety of our drugs.  These agencies require drug manufacturers to submit to them the composition of their therapeutic compounds and then to comply with the product specifications.  It is this composition and these specifications which formed the basis of the clinical data evaluated by the agency and upon which the marketing approval is based.  Any deviation from those specifications requires a submission to the regulatory agency for review. Any deviation without such a submission is punishable.   
At the manufacturing site, as products come off the line they are subjected to a panel of product release tests to ensure each batch complies with the product specifications.
Specification compliance is a direct function of the consistency of the raw and ancillary materials, equipment, and operating procedures used in the manufacturing process.



Cell therapies present unique challenges when complying with this paradigm for several reasons only two of which I will mention here.  Firstly, it is not possible to achieve the level of product purification as one might with other therapeutic products.  Secondly, the product characterization is at a cellular rather than molecular level.

Autologous cell therapies present another set of unique challenge in this paradigm because of the notable patient-to-patient variability where the patient is also the donor of the raw material.  This often means there is a wider tolerance of heterogeneity in the product but it still must be within what has been proven to the regulatory agency as a safe and effective range.  


In cases where an autologous cell therapy is centrally manufactured, they are most often subjected to product release testing similar to that described above.  One notable difference, particularly for fresh products, is that the products may be shipped to the clinic and even administered before the full panel of test results are obtained.  This wold be considered highly unusual (if ever acceptable) with other types of products but is tolerated because of the time-sensitivity of these products and their high safety profile.


In the case of autologous cell therapy products produced at the bedside there is often not the same kind of product release discipline.  Often the regulatory agencies deal with the product consistency and specification compliance issue by ensuring that the cell processing device used point-of-care is validated to ensure the cellular product output is always within a specified range shown to be clinically safe and effective.


The Varying Degree of Product Characterization/Specification of Autologous GTP Cell Therapy Products


However – and now I get to the point of this blog post – for cell-based products, procedures and/or devices/kits which are not mandated to be formally approved by a regulatory agency before they can be commercially marketed, there is no product specification rigor.  Compliance with the Good Tissue Practice regulations and guidance is deemed to ensure safety.  In the United States, cell-based products which are deemed to be “minimally manipulated” and intended for “homologous use” are typically allowed to go straight to market with no formal approval.  Safety and clinical data is not required but is practically necessary to support physician adoption and, where applicable, reimbursement.  


This means that for these products there is a great deal of variability in terms of how much rigor companies apply in characterizing their product and then ensuring that each batch complies with the specifications they themselves have determined to be safe and effective. Again, where such products are manufactured in a centralized facility the likelihood of some release testing is greater.  However, those companies relying on a point-of-care processing kit or device business model that has not been deemed to require formal market approval, rarely (if ever) include product release testing.


The common criticism of these companies is that they simply do not know what they are injecting into patients because of the combination of the patient-to-patient donor variability, the lack of any disciplined product characterization or dosing studies, and the absence of any product release testing.  


This criticism is not equally levied at all autologous GTP products or companies – even those relying on point-of-care processing.  Of course some companies care and do a lot to try to ensure their product is well-characterized and that each batch complies with product specifications. This may involve the use of product release tests but can also involve the combination of pre-market research into the product characterization, safety, and dosing along with validation of the device/kit output.  In this way a company can say that within a very small margin, the output will be within the product specifications the company knows is safe and efficacious.


However, in a rush to get their device/kit to market some companies appear to care very little about the cell product characterization, validation of the output of their device/kit, or tying this data to optimal dose.


More concerning are those companies that appear to provide such data but it is wrong or meaningless.  What follows appears to potentially be a case study of precisely this problem. 


The INCELL Study 


This week I came across a fascinating white paper from Incell Corporation analyzing the output of adipose tissue processing kits of MediVet-America apparently demonstrating the inaccuracy of their cell counts (a common type of cell therapy product characterization) and calling into the question the cell count claims of Intellicell Biosciences (New York, NY) and Adistem (Hong Kong).


At the heart of the critique is the claim that the cell counting (product characterization) techniques employed by these companies counts as cells things (namely acellular micelles) which are not cells.

I encourage you to read the white paper in its entirety.  They corresponding author told me to watch for one or more papers which they are preparing for submission to peer-reviewed publications shortly.  Presumably these will rely on a larger data set and perhaps test other methodologies or technologies.


For the purposes of this blog, I’ve pulled what I believe are the most salient excerpts below:

Intrigued by the high cell numbers  (5 to 20 million cells/gram)  reported by kit/device manufacturers such as MediVet-America (Lexington, KY), Intellicell  Biosciences (New York, NY), and Adistem, Ltd. (Hong Kong) in adipose stem cell therapy compared to other methods (e.g., 
Chung,Vidal, and Yoshimura), INCELL staff conducted a research study to  investigate the high apparent yield of stem cells.  This initial work was focused  on SVF cells from the MediVet Kit, which is marketed to isolate adiposederived canine SVF and stem cells.

The cell yields reported for the Medivet Kits are five to more than ten times higher than the yields routinely obtained by INCELL from freshly harvested human or animal adipose tissue using our adipose tissue processing methods.  These yields are also tenfold or higher than those reported in the literature by most academic researchers (Chung-canine, Vidal–equine, Yoshimura–human).  Since these  cell counts are used to support stem cell dosing recommendations and cell banking, it is important to better understand why the cell numbers are higher.

A comparative analytical study of three dog donors of adipose tissue was designed to evaluate the cell yields using the MediVet Kit as an example of this class of isolation system. All  kit procedures were followed as per the instructions provided.  A brief overview of the different cell counting methods used, and the resultant cell counts, observations and explanations of the results observed, are described below

.

This study shows that incorrect counting of adipose derived SVF cells and the subset of regenerative stem cells can subsequently result in inaccurate dosing, both in direct therapeutic applications and in cryostorage of cells for future use.  The DAPI-hemocytometer cell count (manual) was considered the most accurate, but there are various sources of technical difficulties that  can lead to incorrect  cell numbers. The nature of adipose tissue itself with variability in dissociation by enzymatic digestion can all contribute to the outcomes. Fat tissue has a propensity to form acellular micelles and oils upon tissue disruption. Processing methods or reagents (e.g., Solution E or lecithins) can generate micelles that may be  erroneously  counted as cells. Autofluorescence and dye trapping or uptake by the micelles can lead to very high inaccurate cell counts when automated cell counting is used. 


In this study the most inaccurate counting came from the Cellometer. When used according to kitrecommended guidelines and on-site training provided by Nexelcom for counting  cells by the MediVet procedure, the Cellometer overstated the DAPI-hemocytometer cell count by up to 20X or more. The Coulter Counter protocols also led to incorrect, high cell numbers. Although the cell counts were still a bit high, the authors recommend the NucleoCounter, or similar equipment, as more acceptable for automated counting.  The manual hemocytometer-DAPI method is the most accurate, but requires a highly experienced cell biologist or technician to make accurate counts and  is not suitable for routine clinical use. 

Other companies also have claims of very high cell numbers when their processes are used. Adistem, like MediVet, states they add an emulsifying agent to their kits to assist in cell release, and they also use a light activation system. Their kits were not tested in this study but it is possible that the high cell numbers reported by Adistem are also incorrect and result from the same problems highlighted in this paper for the MediVet procedure. Ultrasonic energy, which is commonly used to manufacture micellular  liposome  structures and to disrupt and lyse cells, is  another potentially problematic procedure for counting and verifying viable, regenerative cells.  Intellicell 3uses ultrasonic energy to release cells from adipose tissue, and it is possible that resultant micelles or cell fragments contribute to the higher than expected cell numbers.  This assumption could be verified with additional studies.  

In summary, the authors caution that great care must be taken when using kits and automated cell counting for stem cell dosing and cryobanking of cells intended for clinical use. Overestimated  cell numbers would be a major confounding source of variation when efficacy of stem cells injected are compared as doses based on cell number and when cryostored cells are aliquoted for use based on 

specific cell numbers as a treatment dose.  Hopefully, this study will lead to more  reproducible counting and processing methods being reported in the literature, more inter-study comparability of cell doses to clinical outcomes,  more industry diligence to support claims, and more accurate counting for dosing stem cell therapies to patients.

Chung D, Hayashi K, Toupadakis A, et al.  Osteogenic proliferation and differentiation of canine bone marrow and adipose tissue derived mesenchymal stromal cells and the influence of hypoxia.  Res Vet Sci, 2010; 92(1):66-75. Vidal MA, Kilroy GE, Lopez MJ, Johnson JR, Moore RM, Gimble JM. Characterization of equine adipose tissue-derived stromal cells: adipogenic and osteogenic capacity and comparison with bone marrow-derived mesenchymal stromal cells. Vet Surg, 2007; 36:613–622.  Yoshimura K, Shigeura T, Matsumoto D, et al:  Characterization of freshly isolated and cultured cells derived from the fatty and fluid portions of liposuction aspirate.  J Cell Phys, 2006; 205:64-76.

 In Conclusion

Despite some of their other challenges, Intellicell, MediVet-America, and AdiStem (and others) have scored credibility points with some of my colleagues who have been impressed by the fact that they have incorporated product release criterion and testing technologies into their business model where their peer companies have not bothered.  This credibility may be quickly eroded if it turns out the results of their cell counts have been misleading.  For now it is a word of caution to do your own due diligence and/or not to fall into a similar product development/characterization trap.  Meanwhile, we will watch for the peer-reviewed papers.

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Researchers pioneer world's first HIV/AIDS nanomedicines

§ August 30th, 2012 § Filed under Nano Medicine Comments Off on Researchers pioneer world's first HIV/AIDS nanomedicines

Public release date: 29-Aug-2012 [ | E-mail | Share ]

Contact: Sarah Stamper sarah.stamper@liv.ac.uk 01-517-943-044 University of Liverpool

Scientists at the University of Liverpool are leading a 1.65 million project to produce and test the first nanomedicines for treating HIV/AIDS.

The research project, funded by the Engineering and Physical Sciences Research Council (EPSRC), aims to produce cheaper, more effective medicines which have fewer side effects and are easier to give to newborns and children.

The new therapy options were generated by modifying existing HIV treatments, called antiretrovirals (ARVs). The University has recently produced ARV drug particles at the nanoscale which potentially reduce the toxicity and variability in the response different patients have to therapies. Drug nanoparticles have been shown to allow smaller doses in other disease areas which opens up possibilities to reduce drug side-effects and the risk of drug resistance. Nanoscale objects are less than one micron in size a human hair is approximately 80 microns in diameter.

Professor Steve Rannard, from the University’s Department of Chemistry, said: “Nanomedicines are being used daily to treat a range of conditions around the world. There are, however, no current nanoparticle HIV therapies that are providing this kind of patient benefit. This project is the first step towards taking the nanomedicine options that we have developed out of our labs and into the clinic, representing a significant milestone in the development of new HIV treatments.

“If we can demonstrate real potential from our planned clinical work with healthy volunteers at the Royal Liverpool University Hospital, then our collaboration partner, IOTA NanoSolutions, will take forward the further development and clinical validation of the ARV drug particles in HIV patients. We also aim to test new formulations for children in developing countries, offering HIV patients around the world the prospect of safer, more effective treatments.”

Professor Andrew Owen, from the University’s Department of Molecular and Clinical Pharmacology, added: “We have integrated an assessment of pharmacology and safety early in the research and this has allowed us to rapidly progress lead options for clinical trials. The work has been conducted with the Medical Research Council (MRC) Centre for Drug Safety Science also based at the University.”

“Our data so far looks really exciting, offering the potential to reduce the doses required to control the HIV virus. This work builds on initiatives by Mdecins Sans Frontires and other groups to seek ways to improve ARV therapy and could have real benefits for the safety of ARVs globally. Importantly we also hope to reduce the costs of therapy for resource-limited countries where the burden of disease is highest.”

HIV continues to increase in prevalence, with 34 million people currently infected worldwide. The new HIV therapies offer particular hope for treating children with HIV which affects 3.4 million children under the age of 15 years in Sub Saharan Africa. About 90% of infected infants acquire the virus through mother-to-child transmission. Without treatment one third of children die within their first year of life.

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Researchers pioneer world's first HIV/AIDS nanomedicines

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Fifth edition of Bangalore Nano on December 6, 7

§ August 29th, 2012 § Filed under Nano Medicine Comments Off on Fifth edition of Bangalore Nano on December 6, 7

It is important to focus on nanotechnology field

The fifth edition of Bangalore Nano, the annual nanotechnology conference-cum-trade show organised by the Karnataka government, will be held on December 6 and 7.

C.N.R. Rao, scientific adviser to the Prime Minister, said that it was a matter of pride that his city, Bangalore, was at the forefront of leading and nurturing innovation in the field of nanotechnology. He spoke about his visit to Israel where he met a young researcher who wanted to use exhaled air and analyse molecules to detect cancer. Years later, Prof. Rao got to know that the researcher came up with a product called the nano nose that helps detect cancer. Such are the possibilities of nanotechnology, and this is why it is important to focus on this emerging field, he said.

Criticising the resistance among academia to collaborate on research work, Prof. Rao said that working together is imperative to move forward.

Nano, a game changer

Chief Secretary S.V. Ranganath said that Karnataka has taken an early lead in science and technology, and compared the nanotechnology scene now to what IT was two decades ago. Nano is going to be a game changer, and it presents a unique challenge as it applies across disciplines. He said that Karnataka has 396 research and development organisations and over 2,100 IT companies, and that over 40 per cent of software exports come from here.

The two-day event includes several plenary sessions on healthcare and medicine, aerospace and defence, electronics, food and agriculture, energy and environment, water management solutions and advanced materials.

The event organisers claim that over 100 leading international and domestic companies are slated to participate. A poster presentation session will display at least 120 posters, and 450 graduate level students will attend the event. As part of the conference, the Research Industry Collaboration Hub will be organised.

The pre-event schedule includes a pre-conference tutorial session on December 5 for delegates.

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Fifth edition of Bangalore Nano on December 6, 7

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