When regenerative medicine firm Cytomedix (OTC:CMXI) acquired biotechnology company Aldagen, the stem cell-based stroke treatment in clinical trials was the centerpiece of the all stock deal.

Cytomedix is now making making moves to develop other stem cell treatments from its Aldagen acquisition. Two more clinical trials will start later this year, CEO Martin Rosendale told analysts on a conference call to discuss first quarter financial results. Rosendale wouldnt identify the indications that will be studied, saying only that they will be announced this summer: one for an arterial disease and the other a neurological condition.

Cytomedixs goal is to ultimately find large pharmaceutical partners commercialize these treatments. These additional clinical studies dont represent those kinds of partnerships. Rosendale said that there are two facilities that will conduct investigator-led clinical trials. But those trials will be funded by outside sources, not by Cytomedix.

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Two stem cell therapies from Cytomedix to start trials; stroke study expands

LEUVEN, BELGIUM–(Marketwire -05/15/12)- TiGenix NV (TIG) a leader in the field of cell therapy, today gave a business update and announced the financial results for the first quarter ending March 31, 2012.

Business highlights

Financial highlights

“In the first quarter 2012 we continued to aggressively push our commercial efforts forward,” said Eduardo Bravo, CEO of TiGenix. “As a result sales of ChondroCelect are developing in line with the improved traction we observed in the second part of last year. At the same time we are moving ahead of schedule with most of our clinical adipose stem cell programs. We closed the quarter with almost EUR 17 million cash on hand, which is sufficient to execute on our business plan and reach key inflection points.”

Business update

ChondroCelect sales increase continues apaceThe Company reports net sales growth for the quarter of 123% compared with the same period of last year, and of 62% compared to Q4, 2011, a positive trend reflecting the uptake in Belgium, where we benefit from national reimbursement. In the Netherlands one of the leading private healthcare insurance companies has made treatment with ChondroCelect compulsory for its insured, and no longer reimburses non-ATMP treatments. Similarly, one of the large private insurers in the UK has expressed its intention to routinely reimburse ChondroCelect going forward. Discussions to obtain full national reimbursement keep advancing in the Netherlands, France, Spain and Germany.

Positive outcome of ChondroCelect compassionate use program published in leading journalPositive outcome data from the ChondroCelect compassionate use program (CUP), involving 43 orthopedic centers in 7 European countries, treating 370 patients with ChondroCelect over the span of four years, were published in advance online in Cartilage, the official journal of the International Cartilage Repair Society. The data show that the implantation of ChondroCelect results in a positive benefit/risk ratio when used in an unselected, heterogeneous population, irrespective of the follow-up period, lesion size and type of lesion treated. In addition, the CUP study significantly expands the data set used to obtain approval for ChondroCelect from the European Medicines Agency in 2009, increasing eight-fold, from 43 to 334, the number of patients with long-term follow up data. To date almost 700 patients have been treated with ChondroCelect.

ADMIRE-CD Phase III trial (Cx601) in complex perianal fistula on schedule The ADMIRE-CD (Adipose Derived Mesenchymal stem cells for Induction of REmission in perianal fistulizing Crohn’s Disease) Phase III protocol was submitted to Ethics Committees or Health Authorities in all 8 participating countries, and to date approvals have been received in four of those countries already.

Cx611 Phase IIa in RA passes last safety hurdleOn April 17, upon review of the safety data of the first three patients of the third cohort of the company’s Phase IIa clinical trial in rheumatoid arthritis (Cx611), TiGenix received the go-ahead from the independent Safety Monitoring Board to recruit and dose the remaining patients of this cohort. This fact is of major importance. In RA it ensures that the product will not be held back by any dose-limiting factors and that we will be able to move forward with the optimal treatment dose. Of almost equal importance is that, if required, we can expand the dosing range in other indications that we are exploring as well. With 6 months of follow-up, the current RA trial in 53 patients is expected to report meaningful results in H1 2013.

Last patient treated in Cx621 Phase I clinical trialAll 10 healthy volunteers have been recruited and treated in the Phase I study of Cx621. Cx621 investigates the safety and feasibility of intra-lymphatic administration of stem cells. Intra-lymphatic administration of (all) stem cells is patented by TiGenix. The final report of this trial will be available at the end of June.

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TiGenix Reports Business & Financial Results for the First Quarter 2012

JERUSALEM–(BUSINESS WIRE)–

Gamida Cell announced today that it has closed an internal E financing round of $10 million. All major shareholders participated.

The financing will be used to support the global commercialization of the companys lead cell therapy product, StemEx, in development as an alternative therapeutic treatment for patients with blood cancers, such as leukemia and lymphoma, who can be cured by bone marrow transplantation but do not have a matched bone marrow donor. The company is currently seeking a strategic partner to join in the global commercialization of StemEx.

The financing will also support the continued development of the companys pipeline of products, primarily the NiCord clinical trial for sickle cell disease and thalassemia.

Mr. Reuven Krupik, chairman of the board of Gamida Cell said, The investors were unanimous in their decision to reinvest, understanding the importance of bringing StemEx to market as well as maintaining the companys leadership role in the stem cell industry. Gamida Cell is a game changer.

The international, multi-center, pivotal registration, Phase III clinical trial of StemEx completed enrollment in February 2012. Clinical outcome is expected in Q4/2012. The market launch of StemEx is planned for 2013. StemEx is likely to be the first allogeneic stem cell product in the market. StemEx is being developed by the Gamida Cell-TEVA joint venture.

Dr. Yael Margolin, president and chief executive officer of Gamida Cell said, With the continued support of our shareholders and the analysis of the clinical results of the StemEx trial just around the corner, we are now focused on submitting the BLA.

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit. Competing products in development use two units. As the average cost of a cord blood unit in the U.S. is $40K, StemEx is expected to be a significantly less expensive treatment option. StemEx is also expected to be available in the market several years before any of the competing products.

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The companys pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cells therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cells current shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: www.gamida-cell.com.

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Gamida Cell Closes $10 Million E Financing Round Earmarked to Support the Global Commercialization of the Company’s …

SEBASTIAN Toby, a 6-year-old golden retriever, loves to run and play catch. And Oreo, a 12-year-old border collie mix, also is a bundle of energy.

Movement for both dogs got easier about a month ago when they received a revolutionary stem cell treatment at the Highlands Animal Hospital.

Veterinarian Marcus Kramer performed the successful transplant procedures, which were developed by Kentucky-based MediVet-America.

Both dogs had been in significant pain with a restricted range of motion, as shown on X-rays.

“It’s made a big difference,” said Kramer. “The really amazing thing is that they both healed so quickly. Both dogs had problems with their hips and were suffering from osteoarthritis. Just 30-days later, they are able to walk and run again.”

Adult animal stem cell technology uses the pet’s own regenerative healing power to treat dogs, cats and horses suffering from arthritis, hip dysplasia and tendon, ligament and cartilage injuries. Under anesthesia, Kramer removed about 40 grams of fat from each dog and separated the stem cells from the fat. He then activated the stem cells under an LED light, and injected them back into the dogs.

Stem cell therapy allows an animal to get off pain and anti-inflammatory drugs, Kramer said. MediVet-America’s therapy is done entirely at the animal hospital in about three hours, and costs about $1,800 for dogs and $2,400 for horses. That compares to thousands of dollars that pet owners could expect to pay for medication over a pet’s lifetime.

Erica Kent, a spokesman for MediVet-America, said using the LED light is integral to the patented-process, because the light helps to awaken stem cells and makes them more active. The three-color light stimulates millions of dormant cells to initiate repair from the moment the cells are injected into the animal’s body, according to the MediVet-America website.

The company is also offering a program that allows pet owners to bank stem cells when animals are younger to use if their pet develops illnesses like arthritis in old age.

STEM CELL THERAPY

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Sebastian veterinarian performs stem cell treatment for pets

ALACHUA, Fla.–(BUSINESS WIRE)–

AxoGen, Inc. (AXGN.OB), a leading regenerative medicine company focused on the commercialization of proprietary products and technologies for peripheral nerve reconstruction and regeneration, today announced revenues for the first quarter ended March 31, 2012 of $1.65 million, a 47% increase over 2011 first quarter revenues of $1.12 million.

This quarters record performance has been the direct result of our increase in sales and marketing activity, commented Karen Zaderej, Chief Executive Officer of AxoGen, Inc. During the first quarter we continued to expand our sales force, while continuing to get hospital approval for AxoGen products and training and developing the sales team. Our growing base of sales representatives, combined with increasing surgeon awareness of our technologies and clinical data, creates a strong environment for our continued growth.

Revenues Revenues for the period increased to a record $1.65 million, or 47%, compared to $1.12 million in 2011. The improved results were primarily due to an increase in new accounts as well as stronger sales penetration into key accounts.

Revenues increased 21% over fourth quarter revenues of $1.36 million.

Gross Profit Gross profit reached $1.21 million, a 55% increase, for first quarter 2012 up from $0.78 million reported for the same period 2011. The higher gross profit reflects lower manufacturing and labor cost and the absence of one-time manufacturing startup expenses reported during the first quarter of 2011. The gross profit margin increased to 73% compared to 70% for the same quarter last year.

Sales and Marketing Expenses As a result of the Company’s investment in additional sales and marketing resources, sales and marketing expenses during the first quarter of 2011 increased to $1.63 million, compared to $0.86 million reported during the same period last year. As of the end of the period, the Company reported 16 direct and 21 independent sales representatives and distributors.

Research and Development Expenses Research and development expenses increased to $0.30 million during the first quarter of 2012. Substantially all of the research and development expenses relate to expenditures for clinical activity.

General and Administrative Expenses General and administrative expenses increased to $1.23 million for the quarter, compared to $0.72 million reported last year. This increase was largely driven by payroll and benefit increases and expenses associated with being a public company.

Operating Loss The Company reported a net loss of $2.11 million, or $0.19 per common share, compared to a net loss of $2.3 million, or $2.21 per common share, reported during the same period in 2011.

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AxoGen, Inc. Announces Record First Quarter 2012 Revenues

NORTHRIDGE, Calif. & CAMBRIDGE, England–(BUSINESS WIRE)–

Avita Medical Ltd. (ASX: AVH), (OTC: AVMXF), (OTCQX: AVMXY),the regenerative medicine company, today announced that it has commenced enrolment in the US FDA-approved feasibility study for the use of ReCell Spray-On-Skin in the treatment of hypertrophic dyspigmented scars (raised and/or discoloured scars).

The initial three patients were treated by Dr Rajiv Sood, at the Richard M. Fairbanks Burn Center of Wishard Hospital, Indiana University, Indianapolis, Indiana, for scarring resulting from previous grafting due to burn injuries.

The approved FDA protocol permits the Company to treat 20 patients with scars at up to four U.S. study sites; patients will be assessed for healing and pain on a weekly basis during the initial four weeks post-treatment; at weeks 12 and 24 the treatment site will be assessed for healing and aesthetic outcomes by both the patient and an independent observer.

“Commencement of the FDA scar study is an important milestone for Avita,” said Dr William Dolphin, Avita Medicals CEO. “ReCell has shown the potential to provide significant benefits over current options in the treatment of acute and chronic wounds and for a wide range of skin defects. We are confident that this study will demonstrate the effectiveness of ReCell in the corrective treatment of scars, making ReCell directly applicable and immediately relevant to the very large aesthetic markets.

The feasibility study is primarily designed to confirm the effectiveness of ReCell for the treatment of scars in a single session in comparison to the current standard of care involving dermabrasion of the scar and often requiring multiple treatment sessions; study endpoints are time-to-healing and aesthetic outcomes. Following completion of the study, Avita will submit the feasibility data and seek FDA approval for a statistically powered, pivotal clinical trial.

The study is funded by the US Department of Defense in partnership with the OSD Manufacturing Technology Program and Rapid Fielding Directorate for the Limb Salvage and Regenerative Medicine Initiative. The contract is a Technology Investment Agreement that is focused on the transition of the capability to meet DoD needs. ReCell was selected as it has the potential to be a quantum advance over the existing ability to treat and re-grow tissue and to substantially reduce the effects and appearance of scarring and thereby profoundly assist in the treatment and rehabilitation of wounded warriors suffering from disfigurement and impeded function due to combat injuries.

An interview with Dr Sood regarding the use of ReCell in treatment of scars and acute wounds is available at http://soundmedicine.iu.edu/segment/3245/Spray-on-Skin.

ABOUT AVITA MEDICAL LTD.

Avita Medical (www.avitamedical.com) develops and distributes regenerative and tissue-engineered products for the treatment of a broad range of wounds, scars and skin defects. The companys lead product, ReCell Spray-On-SkinTM, is used in a wide variety of burns, plastic, reconstructive and cosmetic procedures. ReCell is patented, CE-marked for Europe, TGA-registered in Australia, and SFDA-cleared in China. ReCell is not available for sale in the United States; in the U.S. ReCell is an investigational device limited to investigational use.

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Avita Medical Initiates US FDA Study of Its ReCell® Spray-On-Skin™ for Reconstructive and Aesthetic Indications

Regenerative medicine startup Juventas Therapeutics has begun enrollment in a phase 2a trial of critical limb ischemia patients.

The Cleveland-based company, which recently secured an important investment from Takeda Pharmaceuticals, is planning to enroll 48 patients and complete enrollment early next year, CEO Rahul Aras said.

Juventas technology, called JVS-100, works by recruiting stem cells from the bone marrow to create new blood vessels. Its based on Stromal Cell-Derived Factor-1 (SDF-1), a naturally produced molecule that attempts to repair the heart immediately following a heart attack.

Critical limb ischemia (CLI) patients are enrolling at several U.S. hospitals, as well as three in India. CLI is a severe obstruction of the arteries that greatly decreases blood flow to the extremities.

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CLI has become a very exciting clinical opportunity, Aras said. Its becoming a growing area of interest for a number of biotech and pharma companies.

Other companies pursuing CLI treatment include Aastrom Biosciences, Arteriocyte and Biomet.

Among the top advantages of Juventas CLI therapy is its simplicity and cost-effectiveness, Aras said. Patients can be injected with the companys therapeutic in an easy procedure at a physician office, and the approach doesnt require bone marrow aspiration to obtain patients own stem cells or complex cell processing as some competing therapeutics do.

Juventas also has a phase 2 trial underway to investigate its therapy with heart failure patients.

The company is expected to shortly announce a series B round of investment, which includes the funding from Takeda, that totals around $20 million or $25 million.

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Regenerative medicine company begins enrollment in critical limb ischemia trial

Why silverware? Why didnt people choose to eat and drink from palladium, ivory, or other materials? Of course many people did. Throughout history people have used gold, clay, wood, and a wide range of other items at mealtime. For many of those who chose silver, however, it was more than just a fad or a symbol of financial standing. Ancient civilizations reportedly used silver because they recognized a connection between the metal and their health. Today, people are also finding silver to be increasingly useful in health-related applications. But will these uses have a material effect on the silver market?

For thousands of years, individuals have used silver at the table, on the battlefield, and in healthcare. The metal has been relied upon to prevent and treat infections, to treat wounds, to prevent food spoilage, and to prevent water contamination. Since long ago, the metal has been credited as having antimicrobial, antibacterial, and antiseptic properties.

Medical uses of silver and preventative applications

Many readers have probably received a silver treatment at least once, as silver nitrate is commonly placed in the eyes of newborns to prevent infections that could cause blindness. Silver has also been widely used in dentistry to fabricate fillings.

Today, the uses of silver for its healing and preventative properties are growing.

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For example, it was only in 2007 that the US Food and Drug Administration approved the marketing of silver-coated breathing tubes. Prior to this approval, according to the Centers for Disease Control and Prevention, every year, 15 percent of patients on ventilators contracted ventilator-associated pneumonia. For tens of thousands of people these infections proved fatal. Including silver in the fabrication of these endotracheal breathing tubes reduces this risk and has likely saved many lives.

Silver is also used in much the same way for catheters and other medical implantation devices. The metal is used to coat surgical instruments and emergency ward equipment to prevent and reduce the transmission of infections.

Wound creams, gels, and powders are made with silver, and the metal is fabricated into wound dressings because it is considered toxic to germs and can prevent the invasion and livelihood of bacteria and yeast. Silver has also been found to reduce the adhesion of dressings to wounds and thus improves the comfort of burn victims.

A recent edition of Silver News spotlights the Trinity Bed Protection System. The covering system is supposed to provide an effective and impermeable barrier between patients and the surfaces they lie on, such as mattresses and stretchers. A notable benefit of this bedding is that it is supposed to retain itsantimicrobialproperties even after repeated washings.

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Silver in Medicine: Support for the Market?

Scientists are forecasting that by 2030, 42 percent of the U.S. population will be obese.

This would cost an additional $550 billion in healthcare expenditures, according to the study published by the American Journal of Preventative Medicine.

Despite reports that obesity levels were tapering off, the scientists found that Americans are gaining weight all the time. There’s also been a surge in severe obesity, or being overweight enough to reduce your life expectancy by at least seven years.

Given the many caveats listed in the preceding paragraph, the current study forecasts a 33% increase in the prevalence of obesity over the next 2 decades based on extrapolating prior available data and assuming these trends continue into the future. If these forecasts prove accurate, this will further hinder efforts for healthcare cost containment.

Here’s a chart showing their projections. The blue line is the previous forecast of 35 percent obesity by 2030. The orange line is what the scientists found by studying trends so far and predictions for the future:

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Terrifying Study Predicts Half Of The US Will Be Obese By 2030

Scientists are forecasting that by 2030, 42 percent of the U.S. population will be obese.

This would cost an additional $550 billion in healthcare expenditures, according to the study published by the American Journal of Preventative Medicine.

Despite reports that obesity levels were tapering off, the scientists found that Americans are gaining weight all the time. There’s also been a surge in severe obesity, or being overweight enough to reduce your life expectancy by at least seven years.

Given the many caveats listed in the preceding paragraph, the current study forecasts a 33% increase in the prevalence of obesity over the next 2 decades based on extrapolating prior available data and assuming these trends continue into the future. If these forecasts prove accurate, this will further hinder efforts for healthcare cost containment.

Here’s a chart showing their projections. The blue line is the previous forecast of 35 percent obesity by 2030. The orange line is what the scientists found by studying trends so far and predictions for the future:

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STUDY: 42% Of The US Will Be Obese By 2030

By Rachel Jackson | Originally Published: 05/14/12 10:49pm |Modified: 05/15/12 2:35pm |

Despite the large amount of debate about health care reform in the past few years, according to medical professionals from across the state, one major topic is missing from the discussion: prevention.

Preventative medicine often is overlooked by pilot studies in hopes of finding solutions to more efficient and cost-effective health policies, said Ken Thorpe, the director of the Partnership to Fight Chronic Disease, or PFCD.

Thorpe, also a professor of public health at Emory University, spoke about prevention of chronic diseases at the Michigan Health Policy Spring Forum, held Monday afternoon in the James B, Henry Center for Executive Development, 3535 Forest Road.

MSU College of Osteopathic Medicine Dean William Strampel said he has worked with the PFCD for more than a decade, and MSU has hosted the forum a gathering of medical professionals, business leaders and state policymakers for 26 years.

Its one of those things that we will all say is very important to us, he said.

Thorpe said there are three approaches to preventative care that often go overlooked: building healthy habits, detecting diseases early and managing diseases to avoid complications.

He also stressed the importance of working within a community care network, bringing together medicine, business and commerce to strengthen the health systems.

The PFCD, a series of seminars focusing on health policy issues, is launching a new initiative in Michigan to unite those health field professionals and determine a way to strengthen health care at a lower cost.

Thorpe said the current health care system is very reactive, and people seem more willing to pay for medical maladies when they arise, but not to prevent diseases in the first place.

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Seminar addresses health concern

(ISNS) — No longer the stuff of science fiction, nanoparticles are becoming more and more common. The extremely tiny objects can do just about everything, from filtering pollution to delivering medicine in the body. However, no one is sure of the effects if they get loose in the environment.

A team of scientists from the National Institute of Standards and Technology and the University of Massachusetts at Amherst thinks there may be something to worry about.

They have not proven the particles are dangerous, but have shown that some nanoparticles can be absorbed into plants and mutate the plant’s DNA, and that, they say, is worth a further look.

Nanoparticles are so small that they act as a bridge between the size of atoms and something of tangible substance. The thickness of a human hair is measured in millionths of a meter; nanoparticles, in billionths of a meter.

And now, they are everywhere. Manufacturers put them in clothing such as socks to kill bacteria. They are in a type of house paint that cleans itself in sunlight and in the coating on eyeglasses. Clear sunscreen lotion now on the market contains zinc or titanium nanoparticles. Cars will soon have paint that heals itself from scratches.

Nanoparticles have become so common it is assumed inevitably they will end up in the environment.

To see what would happen to plants exposed to nanoparticles, the researchers took particles of copper oxide and exposed three kinds of plants to them: radishes and two types of rye, the researchers reported in Environmental Science & Technology.

They chose nanoparticles of copper because they are widely used for coloring glass, in ceramics, as a polish and in the manufacturing of rayon. They also are used in the electronics industry to manufacture semiconductors, said Bryant Nelson of the National Institute of Standards and Technology.

The research team also used particles of copper oxide larger than nano-size as a comparison as well as regular copper ions.

Copper oxide is an oxidizing agent, and some oxidizing agents from metals can cause cancer in humans, a reason for the concern.

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Nanoparticles may pose environmental threat

NEW YORK, May 16, 2012 /PRNewswire/ — Reportlinker.com announces that a new market research report is available in its catalogue:

http://www.reportlinker.com/p0181059/Global-Nanobiotechnology-Industry.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Nanotechn

This report analyzes the worldwide markets for Nanobiotechnology in US$ Million by the following Technology Segments: Nanomaterials (Solid Inorganic Nanoparticles, Nanocomposites, Nanostructured Materials & Membranes, Nanotubes and Fullerenes, & Other Nanomaterials), and Nanodevices/Tools. The report provides separate comprehensive analytics for the US, Japan, Europe, and Rest of World. Annual forecasts are provided for each region for the period of 2009 through 2017. Also, a six-year historic analysis is provided for these markets. The report profiles 117 companies including many key and niche players worldwide such as Aduro BioTech, Arrowhead Research Corporation, Calando Pharmaceuticals, Inc., Agilent Technologies Inc., Asklepios BioPharmaceutical Inc., Biosante Phosphate Pharmaceuticals, Inc., Celgene Corporation, Elan Pharmaceuticals, Elitech Group, Flamel Technologies, Inc., Gilead Sciences Inc., Life Technologies Corporation, NanoBio Corporation, Nanogen, Inc., Nanophase Technologies Corporation, pSivida Ltd., Sigma Aldrich Company, Starpharma Holdings Ltd., Dendritic Nanotechnologies, Inc., SkyePharma Pharmaceuticals, Unidym, Inc., and Zyvex Instruments LLC. 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 DEFINITIONSStudy Reliability and Reporting Limitations I-1Disclaimers I-2Data Interpretation & Reporting Level I-3Quantitative Techniques & Analytics I-3Product Definitions and Scope of Study I-31. Nanomaterials I-4Solid Inorganic Nanoparticles I-4Nanocomposites I-4Nanostructured Materials and Membranes I-4Nanotubes and Fullerenes I-4Other Nanomaterials I-4Nanoshells I-4Nanohorns I-5Nanocapsules I-52. Nanodevices/Tools I-5II. EXECUTIVE SUMMARY

1. INDUSTRY OVERVIEW II-1

A Curtain Raiser II-1

Nanobiotechnology – The Next Big Thing II-2

Nanotechnology & Biology Tie the Knot: “Two to Tango” II-4

Where Conventional Molecular Science Falls Short Nanobio

Steps In II-4

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Global Nanobiotechnology Industry

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Gene therapy may extend life: Study

Recently, there was a gathering of scientists at the Hyatt Regency in Greenwich. They were there to discuss their latest findings in cancer research — especially in the use of gene therapy in cancer treatment. There was tangible excitement in the room. Among those in attendance was Dr. Carl June of the University of Pennsylvania, who had, using gene therapy, actually eliminated all signs of cancer in two patients he was treating. Also there was Dr. Hui Hu, who works in the same city as June, at the Wistar Institute, and who is having similar success treating mice with cancer with his gene therapy, which he wants to apply to human patients.

The reason for this writing, however, is another reason for the gathering — the celebration of the 10th anniversary of the Alliance for Cancer Gene Therapy (ACGT), a Greenwich non-profit that has helped fund these scientists and others like them; and the legacy of Ed Netter, of Greenwich, co-founder of ACGT, who died last year, just short of Dr. Carl June’s news of his gene therapy treatment.

Barbara Netter, Ed Netter’s widow and cofounder of ACGT, was feeling the excitement in the scientific meeting held before the gala dinner honoring her late husband and ACGT.

“We have so much energy now with Carl June,” she said. “We’re receiving a lot more applications for grants. There’s a lot of collaboration and partnering happening.”

It was the death of the Netters’ daughter-in-law from breast cancer that inspired Ed and Barbara Netter to create ACGT 10 years ago as a vehicle to raise money for research into cancer gene therapy.

“The vision that Ed had is a new way to really get the science into clinical trials that will show the (gene therapy) concept at work,” said Barbara Netter.

Last August, Dr. Carl June reported to the world the success of his clinical trial, supported by ACGT, in which he genetically modified the T-cells of three patients with chronic lymphocytic leukemia to target and kill their tumors. Two of the three patients remain cancer free, with the third patient’s cancer significantly reduced.

June’s treatment, he said, was still only in the first phase. But he’s now adding more patients. “We’ve taken in our first pediatric patient — a 6-year-old girl with leukemia. She was infused today,” June said.

This is the first of 400 patients June wants to treat within the next year or two.

“The gene therapy approach is fundamentally different from current therapies,” said June, “and these ideas need to move to human treatment.”

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Greenwich couples' legacy lives on in ACGT

Public release date: 16-May-2012 [ | E-mail | Share ]

Contact: John Pastor jdpastor@ufl.edu 352-273-5815 University of Florida

Using gene transfer techniques pioneered by University of Florida faculty, Taiwanese doctors have restored some movement in four children bedridden with a rare, life-threatening neurological disease.

The first-in-humans achievement may also be helpful for more common diseases such as Parkinson’s that involve nerve cell damage caused by lack of a crucial molecule in brain tissue. The results are reported today (May 16) in the journal Science Translational Medicine.

The children in the study, who ranged in age from 4 to 6, inherited a rare disease known as aromatic L-amino acid decarboxylase deficiency, or AADC. Patients with AADC are born without an enzyme that enables the brain to produce the neurotransmitter dopamine. They generally die in early childhood.

In a phase 1 clinical trial led by Paul Wuh-Liang Hwu, M.D., of the National Taiwan University Hospital, surgeons used a delivery vehicle called an adeno-associated virus type 2 vector to transport the AADC gene into localized areas of the brains of three girls and a boy.

Before therapy, the children showed practically no spontaneous movement and their upper eyelids continually drooped. After receiving the corrective gene, the children gradually gained some head movement. Sixteen months afterward, the children’s weight had increased, one patient was able to stand and the other three were able to sit up without support.

The study shows gene therapy that targets AADC deficiency is well-tolerated and leads to improved motor development and function, according to co-authors Barry Byrne, M.D., Ph.D., director of UF’s Powell Gene Therapy Center, and Richard O. Snyder, Ph.D., director of UF’s Center of Excellence for Regenerative Health Biotechnology. Both are members of the UF Genetics Institute.

“The children in this study have the most severe form of inherited movement disorder known, and the only treatments so far have been supportive ones,” said Byrne, a pediatric cardiologist and associate chairman of the department of pediatrics in the College of Medicine. “It is gratifying to see it is possible to do something to help them, other than providing feeding tubes and keeping them safe. This absolutely opens the door to the possibility of even earlier treatment of neurological diseases by direct gene transfer, and has implications for Parkinson’s disease, ALS and even cognitive diseases such as dementia when caused by gene defects.”

The Powell Gene Therapy Center provided expertise to the Taiwanese physicians on treating the patients and engineering the corrective gene that spurs production of the absent AADC enzyme. UF’s Center of Excellence for Regenerative Health Biotechnology manufactured the vector, packaging genetic material it received from Taiwan into virus particles that were purified, characterized and tested for sterility and stability before being shipped to the clinic for use in patients.

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Children with rare, incurable brain disease improve after gene therapy

ScienceDaily (May 14, 2012) A new study consisting of inducing cells to express telomerase, the enzyme which — metaphorically — slows down the biological clock — was successful. The research provides a “proof-of-principle” that this “feasible and safe” approach can effectively “improve health span.”

A number of studies have shown that it is possible to lengthen the average life of individuals of many species, including mammals, by acting on specific genes. To date, however, this has meant altering the animals’ genes permanently from the embryonic stage — an approach impracticable in humans. Researchers at the Spanish National Cancer Research Centre (CNIO), led by its director Maria Blasco, have demonstrated that the mouse lifespan can be extended by the application in adult life of a single treatment acting directly on the animal’s genes. And they have done so using gene therapy, a strategy never before employed to combat aging. The therapy has been found to be safe and effective in mice.

The results were recently published in the journal EMBO Molecular Medicine. The CNIO team, in collaboration with Eduard Ayuso and Fatima Bosch of the Centre of Animal Biotechnology and Gene Therapy at the Universitat Autonoma de Barcelona (UAB), treated adult (one-year-old) and aged (two-year-old) mice, with the gene therapy delivering a “rejuvenating” effect in both cases, according to the authors.

Mice treated at the age of one lived longer by 24% on average, and those treated at the age of two, by 13%. The therapy, furthermore, produced an appreciable improvement in the animals’ health, delaying the onset of age-related diseases — like osteoporosis and insulin resistance — and achieving improved readings on aging indicators like neuromuscular coordination.

The gene therapy consisted of treating the animals with a DNA-modified virus, the viral genes having been replaced by those of the telomerase enzyme, with a key role in aging. Telomerase repairs the extreme ends or tips of chromosomes, known as telomeres, and in doing so slows the cell’s and therefore the body’s biological clock. When the animal is infected, the virus acts as a vehicle depositing the telomerase gene in the cells.

This study “shows that it is possible to develop a telomerase-based anti-aging gene therapy without increasing the incidence of cancer,” the authors affirm. “Aged organisms accumulate damage in their DNA due to telomere shortening, [this study] finds that a gene therapy based on telomerase production can repair or delay this kind of damage,” they add.

‘Resetting’ the biological clock

Telomeres are the caps that protect the end of chromosomes, but they cannot do so indefinitely: each time the cell divides the telomeres get shorter, until they are so short that they lose all functionality. The cell, as a result, stops dividing and ages or dies. Telomerase gets around this by preventing telomeres from shortening or even rebuilding them. What it does, in essence, is stop or reset the cell’s biological clock.

But in most cells the telomerase gene is only active before birth; the cells of an adult organism, with few exceptions, have no telomerase. The exceptions in question are adult stem cells and cancer cells, which divide limitlessly and are therefore immortal — in fact several studies have shown that telomerase expression is the key to the immortality of tumour cells.

It is precisely this risk of promoting tumour development that has set back the investigation of telomerase-based anti-aging therapies.

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First gene therapy successful against aging-associated decline: Mouse lifespan extended up to 24% with a single …

Gene therapy has been used to increase the lifespan of mice by up to 24 percent – and improve their health at the same time.

Telomerase helps to maintain the physical integrity of the ends of chromosomes. And mice that received a single treatment to deliver the enzyme to different cells in the body showed drastic improvements in health, fitness and longevity, says the team.

“Gene therapy is typically thought of as a way to deliver genes into cells to correct genetic defects or diseases. However, if we consider that ageing is, at least in part, the consequence of defective gene function, gene therapy is also a valid strategy to delay ageing or to increase lifespan,” says Maria Blasco, director of the Spanish National Cancer Research Centre.

“Our results show that telomerase gene therapy is not only a viable anti-ageing intervention but it also has remarkably beneficial effects on health and fitness without increasing the incidence of cancer.”

Telomeres are the caps of repetitive DNA nucleotide sequences that sit at the ends of chromosomes, and are known to play a part in ageing. As they are gradually worn down, cell stop dividing and eventuially die.

The scientists used an adeno-associated virus vector to introduce the telomerase gene into the cells of adult mice, where it added DNA back to the ends of chromosomes.

The mice that were used in the experiments typically live for approximately 150 weeks. However, one-year-old mice that received the gene therapy lived on average 24 percent longer, and the average lifespan of two-year-old mice increased by 13 percent.

“In addition to living longer, engineered mice had stronger bones, improved metabolic functions, better motor coordination and balance, as well as improved performance in object-recognition tests,” says Bruno Bernardes de Jesus, a researcher at the Spanish National Cancer Research Centre.

The findings are the first proof-of-principle that telomerase gene therapy is both feasible and potentially safe. The team now plans to investigate whether the therapy could work for animals with longer lifespans.

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Gene therapy dramatically extends mouse lifespan

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Gene therapy extends mouse lifespan by 24 pc

Cancer Genetics is pursuing a strategy that differs from Facebook’s IPO plan, expert says.

A Rutherford, N.J.-based start-up developing tools to diagnose cancer is planning to launch an initial public offering this week under the long shadow of Facebook’s long-awaited blockbuster IPO.

Cancer Genetics Inc. is looking to raise more than $50 million with an initial public offering of 4 million shares priced between $11 to $13 a share.

The offering comes as the IPO market is struggling back toward its pre-recession level of activity, although it remains well below the peak of the dot-com fueled boom of the 1990s. If Cancer Genetics prices this week, it will be the first IPO in North Jersey since Park Ridge-based SeaCube Container Leasing Ltd. went public in October 2010. The ticker symbol would be CGIX, and the shares would trade on the Nasdaq Stock Market.

How the Facebook IPO will affect that of Cancer Genetics is unclear. David Menlow, president of IPOfinancial.com, said he thinks it “greatly hampers their chance of success.”

“Investors are not interested in paying attention to anything other than the 800-ton gorilla in the room,” which is Facebook, he said.

But Jay R. Ritter, a professor of finance at University of Florida in Gainesville, who tracks IPOs, said the two companies are playing to different investors.

Cancer Genetics, which is planning its IPO for Friday, is looking to attract institutional investors interested in holding company stock for the long period it takes for a biotech company to make a medical breakthrough, he said. In contrast, many Facebook investors are mainly interested in the chance of making a quick profit if the stock takes off, he said.

“Facebook is getting huge amounts of attention,” Ritter said. “Lots of investors are coming out of the woodwork who do not have an interest in buying biomedical companies.”

Cancer Genetics, founded in 1999, is focused on developing and commercializing tests and services to diagnose, predict and help treat hematological, urogenital and HPV-associated cancers. The top executives at Cancer Genetics are Chairman Raju S.K. Chaganti, Chief Executive Officer Panna L. Sharma and Elizabeth A. Czerepak, chief financial officer.

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Cancer Genetics IPO Faces Facebook