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On cusPIDD of a win? Adma’s phase III results have teeth, final next year

Click here to read the full article

By Randy Osborne, Staff Writer
©2014. REPRINTED WITH PERMISSION FROM THOMSON REUTERS.
Taking what CEO Adam Grossman called “a different spin on
intravenous immunoglobulin [IVIG]” while “trying to provide a
product that’s very targeted, niche-focused, and is going to
provide an advantage to a subset” of the patient population,
Adma Biologics Inc. reported positive top-line phase III results
in primary immune deficiency diseases (PIDD) with lead
compound RI-002.
A plasma-derived, polyclonal, intravenous immune globulin,
RI-002 contains naturally occurring polyclonal antibodies such
as Streptococcus pneumoniae, Haemophilus influenzae type B,
cytomegalovirus, measles, tetanus, etc., as well as standardized,
high levels of antibodies to respiratory syncytial virus (RSV).
The compound met its primary endpoint in the 59-patient,
Now in some cases cardiologists are comparing cholesterol lowering to the limbo game; “how low can you cialis generika choose here go?” What may be more important for men with diabetes. Here are cheap sildenafil no prescription some benefits of using ayurvedic sex enhancement supplements. There are a number of forms cheapest cialis of impotence or loss of erectile power (Kama Shakti in Hindi) Invasive treatment option in the form of penile implant is available . Great looking pump in Karachi can be utilized without trouble by any man with little penis inconveniences. viagra pills australia year-long study of preventing serious bacterial infections such
as bacterial pneumonia, osteomyelitis and bacterial sepsis in
PIDD patients.
PIDD comprises “about 150 different types of genetic defects
that affect certain aspects of the immune system,” Grossman
told BioWorld Today. In the U.S., about 250,000 patients have
one or more forms of PIDD, with half requiring monthly infusions
of IVIG to support their systems.
“There is a subset of the PIDD population that has combined
immune deficiency, meaning that they have both B and T cell
dysfunction,” Grossman said. “We’re providing a product that
has a unique antibody profile in that it has higher levels of
antibodies against all respiratory viruses over regular IVIG.
We think there’s a subset of about 8,000 to 20,000 patients
currently receiving IVIG who we believe clinicians would feel our
product would provide more protection at certain times of the
year,” such as winter.

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UM researcher uses stem cells to fight Alzheimer’s

The Detroit News

UM researcher uses stem cells to fight Alzheimer’s

http://www.detroitnews.com/story/news/local/michigan/2014/11/11/um-researcher-uses-stem-cells-fight-alzheimers/18895621/

Kim Kozlowski, The Detroit News 11:38 p.m. EST November 11, 2014

Ann Arbor — Inside a laboratory at the University of Michigan, researchers have been injecting stem cells into the brains of mice engineered to have Alzheimer’s disease, and making remarkable discoveries.

The experiments are among the first in the nation to examine how stem cell therapies might alter the course of Alzeheimer’s, a fatal disease that afflicts more than 5 million people in the U.S. and is widely regarded as an epidemic predicted to explode as the nation’s population ages.

The research is being overseen by UM’s Dr. Eva Feldman, who pioneered the nation’s first clinical trial using stem cells in patients with amyotrophic lateral sclerosis — a disease that received global awareness last summer thanks to the ALS ice bucket challenge.

Dr. Feldman

While Feldman’s ALS trial is not complete, it is showing promise. That’s why she began an experiment to see how stem cells might fare in treating Alzheimer’s disease, another neurodegenerative disorder.

Although it’s very early in the research, the Alzheimer’s experiment with mice showed that stem cells made a mouse with Alzheimer’s disease indistinguishable in behavior and memory from a mouse that didn’t have the disease.

“When you work in science, you do as many experiments that don’t work that do,” Feldman said. “When you get something that works so beautifully (like this experiment), you can quickly see its translational potential. I am looking at a mouse but some day I could be looking at a man. As a clinician scientist, those are the moments you live for.”

The findings from Feldman’s preclinical trial were presented last month in Boston at the Congress of Neurological Surgeons annual meeting.

While some experts are cautiously optimistic, others hailed Feldman’s work.

“The special design of the present study sets new standards for further clinical translation in regenerative medicine for neurological diseases,” Tamir Ben-Hur, a Jerusalem-based neurology professor, wrote earlier this year in the Annals of Neurology. “This study design represents the best moral solution for the difficult task of testing risky procedures in a deadly disease with no alternative therapy or hope.”

Feldman, a UM professor and neurologist, began testing stem cells years ago. Her research began with rats and pigs with ALS — also known as Lou Gehrig’s disease — before she launched a clinical trial in humans with the disease five years ago.

Last year, some participants in the human study either improved or stabilized in the closely watched clinical trial.

Soon after, the Alzheimer’s study was launched. That study used fetal stem cells provided by Neuralstem Inc., a Maryland company. It used mice that had been engineered with the inherited gene of Alzheimer’s but had not yet displayed symptoms.
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With one group of the mice, UM researchers injected the stem cells into the hippocampus, the area of the brain that controls learning and memory. Another group of mice were injected with saline solution. The mice that got the stem cells were evaluated with several behavioral and memory tests — and looked the same as mice without Alzheimer’s disease.

“Those animals retained their ability to think, as a mouse does, to recognize objects so they looked just like an animal that doesn’t have Alzheimer’s disease,” Feldman said. “It’s really remarkable.”

Feldman is still deciding which larger animal model to use for further research, but her associates say it likely will be rhesus monkeys.

“She’s doing stuff that needs to be done,” said Robert Karbel, manager of the Sinai Medical Staff Foundation in Southfield, a group of physicians that has supported Feldman’s research with $500,000. “She has the courage to do it … and she seems to be making progress. There are no cures yet, but she’s working at it.”

Feldman’s latest study is generating excitement because if successful, it has the potential to impact millions of lives. About 5,600 people are diagnosed with the disease each year, according to the ALS Association, but Alzheimer’s afflicts 100 times more people, not counting their family caregivers.

Alzheimer’s is a fatal disease with no cure and no meaningful agents to delay its course. The disorder slowly robs people of their ability to remember and perform daily tasks, which is why so many end up in long-term care facilities.

As the baby boomer population ages, many regard Alzheimer’s as a tsunami that could swamp the nation’s health care system if a better intervention is not discovered, in part because of how costly it is to care for its victims.

In 2014, the direct costs to care for those with Alzheimer’s will total an estimated $214 billion, including $150 billion in Medicare and Medicaid costs, according to the Alzheimer’s Association.

By 2050, those costs are expected to soar to an estimated $1.2 trillion.

There are all kinds of studies — nearly 50 — in various stages of research to address the disease. But Feldman is among the first to examine stem cells, according to Dr. Keith Fargo, director of scientific programs and outreach at the Alzheimer’s Association.

Fargo said the research is very preliminary, and should be regarded cautiously. But he also said it is intriguing.

“We think all kind of research is needed,” Fargo said. “We support a full-court press in Alzheimer’s disease research.”

For people like Ted Harada, the research is more personal. Harada, 42, of McDonough, Ga., was in the first phase of Feldman’s ALS trials, shortly after he was diagnosed with ALS in 2011.

He received millions of stem cells in his spinal cord in two separate surgeries.

By his doctor’s predictions, he could have already lost his battle to the disease. Instead, he no longer uses a cane and has stabilized.

“We’ve all heard for years that stem cells could be the next big frontier in medicine,” Harada said.

“It’s great they are finally allowing these type of trials and I am so thankful researchers like Dr. Feldman are on the front lines pushing the envelopes and not accepting the status quo. Her research is giving hope to communities where hope was an absent commodity.”

 

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Spinal gap: Neuralstem goes into chronic injuries phase I trial first ever to be cleared by the FDA

click here to read the full article

By Randy Osborne, Staff Writer
With encouraging data from a phase I trial in amyotrophic lateral
sclerosis (ALS) and a phase II trial under way testing NSI-566,
Neuralstem Inc. has begun – with the same candidate – the fi rst
human neural stem cell study to be given the FDA’s nod for chronic
spinal cord injury.
Four patients with thoracic spinal cord injuries (T2-T12) will
have NSI-566 transplanted directly into the region of the injury,
which has been sustained between one and two years before the
treatment. All patients have an American Spinal Injury Association
grade A level of impairment, which means complete paralysis with
no motor or sensory function at and below the trauma site.
Richard Garr, CEO of Germantown, Md.-based Neuralstem, said
the trial is half the size originally planned, and for a good reason.
“When we submitted this originally, there were eight patients, but
that was before we had treated successfully the high-dose patients
in our ALS trial,” he told BioWorld Today. “We went back to the FDA
and asked them to amend the protocol.”
Last heard from in the stem cell/spinal cord injury space was
Menlo Park, Calif.-based Geron Corp., which last year disclosed in
an SEC fi ling that the assets related to the program were taken
over by Asterias Biotherapeutics Inc., a subsidiary of regenerative
medicine specialist Biotime Inc., of Alameda, Calif.
The deal involved transfers of common stock and warrants, along
with patents, regulatory fi lings and investigational new drug
applications fi led with the FDA for Geron’s phase I safety study
with the oligodendrocyte progenitor cells (OPCs). Geron was
investigating the cells’ effi cacy in acute spinal cord injury, rather
than chronic, as with the Neuralstem product. In May of this
year, Asterias offered promising safety results with AST-OPC1, a
population of cells derived from human embryonic stem cells
that contain the OPCs, in fi ve subjects tested during a restarted
experiment that Geron began in 2010. (See BioWorld Today, Jan.
26, 2009, Jan. 27, 2009, and April 5, 2013.)
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FDA won’t just let you go in to try. The surgery is very risky. We’re
the first ones who ever did intraspinal injections with our ALS trial.”

Neuralstem had to use a special delivery device invented
at Cleveland Clinic, and deliver strong preclinical data
to U.S. regulators before efforts could move ahead. The
fi rm’s approach is “very different from what anybody else,
even Geron, has ever done,” he said.
Predicting how fast benefi t might appear is tricky. “In ALS
patients, the maximum window of biological activity for
the cells was between four and six months post-surgery,”
Garr said. “But it was also very clear in four to eight weeks
that there was considerable biological functional activity.
Will we see that in spinal cord patients? We don’t know,
but that would be our expectation.”
For some ALS patients, the effects turned up much sooner,
he added. “What the cells are doing here, in chronic
[spinal cord injury] patients, is literally bridging the gap.
We’re trying to rebuild the circuitry in the gap so that the
signal can come through, down the spinal cord. There are
also a lot of neurotrophic factors expressed by the cells
that can help with healing, but in a chronic patient, it’s
unknown how much impact that will have.”
Garr cited a key difference between spinal cord injury and
ALS, “where you are putting the cells in the motor neuron
pools to protect and nurture the remaining motor neurons
and then hopefully nurture back to health those that
haven’t hit that tipping point. The early neurotropic factor
expression of the cells, even before they are synaptically
integrated and matured, could have an effect [in ALS] –
clearly has. Maybe in spinal cord injury patients, where the
neurotrophic effect isn’t the primary reason for benefi t,
it could take a little longer. We’re really waiting for the
synaptic connections to happen and for the circuitry to be
rebuilt.”

 

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CANbridge Life Science’s ‘portfolio’ bid aims to get cancer drugs to patients faster

Canbridge Life Science’s ‘portfolio’ bid aims to get cancer drugs to patients faster

By Shannon Ellis 

July 29, 2104

Staff Writer

SHANGHAI – Canbridge Life Sciences Inc., of Beijing, is on its way to bridging the gap that leaves many patients in China waiting anywhere from four years to six years for critical treatments approved in developed markets. With time of the essence, the firm is taking a portfolio approach to its in-licensing strategy, developing both clinical-stage and approved treatments, while mixing new drugs with treatments classified as medical devices.

In its second deal since forming last year, Canbridge will have the Chinese commercialization rights to Caphosol, an adjunct therapy for cancer patients that suffer from extreme dry mouth (oral mucositis) as a consequence of radiation or chemotherapy. According to CEO James Xue, there are no adequate treatments approved in China, even though Caphosol has been available in the U.S., Canada and the European Union.

Canbridge signed the deal with Eusa Pharma Ltd., the international division of Jazz Pharmaceutical plc, of Dublin.

“Drug development takes a lot of money capital and time,” Xue said. “If we only focus on products that are in clinical development stage . . . we have to take a lot of risks in terms of development risk.”

In the company’s inaugural deal, it did take on some of that risk. Canbridge licensed the China rights to co-develop ATI-1123, a liposomal formulation of docetaxel to treat non-small-cell lung cancer, from nanotech specialist Azaya Therapeutics Inc., of San Antonio. ATI-1123 had finished phase I in the U.S. for multiple solid tumor cancers. (See BioWorld Asia, Sept. 25, 2014.)

Developing novel therapeutics is a lengthy and costly process everywhere, but particularly so in China. And with so much unmet need, going after some low-hanging fruit can help a small start-up generate crucial income to temper the risk.

Xue said that Canbridge is hopeful Caphosol will launch in a “relatively more speedy way and with limited risk,” citing that those “products already approved by the U.S. FDA and EMEA have a pretty assured registration and approval process in China.”

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Oral mucositis is a startlingly common side effect of cancer treatment. It can lead to painful mouth sores, impede a patient’s ability to swallow or take in nutrition orally and, in some cases, cause life-threatening sepsis.

The company estimated some 40 percent of chemotherapy patients, 70 percent of bone marrow transplant patients and 97 percent of head and neck cancer patients receiving radiation therapy develop oral mucositis.

“This product has a vey attractive market potential. The number of cancer patients is at 3.5 million a year in China and most of these patients would experience a certain degree of mucositis,” Xue said. “The sheer number of patients is so large, even if we capture a small percentage of the market, it will still mean significant revenues for us.”

BUILDING A FULLY INTEGRATED COMPANY

With that deal, Canbridge is also a step closer to realizing its plans to be a fully integrated biotech company and will develop its commercial operation from the ground up. Its model is in the same mold as those of Sciclone Pharmaceutical Inc. and Hong Kong-based Lee’s Pharmaceutical Ltd., both of which have been successful in licensing the best of the West for China.

Xue brings regulatory and commercialization experience from his days as general manager at Genzyme China, and he said the current climate, with numerous pharma companies being probed for corrupt activities in their sales ranks, does not deter him. Echoing a sentiment shared by other speciality biotechs, he said, “there are ways to do it right; the precondition is the product you work with is unique enough to differentiate itself.”

Citing data from a study conducted last year by McKinsey Consulting, that 50 percent of big pharma revenues in China comes from off-patent therapies left to fight it out against cheaper generics, he said, “even big pharma has not done the proper work to bring original, patented life-saving therapies to China.”

Xue said his reception during his U.S. trips has been increasingly positive and he has seen a trend where “CEOs are taking a more active role in directing their China strategy at an earlier stage . . . to drive up the value of their equity in the face of their investors,” and, he adds, may be a little disappointed in the ability of the big pharmas to execute partnerships in China.

Canbridge may very well be on a roll. According to Xue, it has received special status from the Beijing municipality that will enable it to tap into government grants and other resources, and it has struck up a partnership with an undisclosed Chinese pharmaceutical with cash to invest. He said the company now has a very robust business development pipeline and expects to be making several announcements in the coming months

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Online Chatter Could Unravel Carefully Built Construct of ‘Blind’ Clinical Trials

Researchers Fret as Social Media Lift Veil on Drug Trials

Online Chatter Could Unravel Carefully Built Construct of ‘Blind’ Clinical Trials

http://online.wsj.com/articles/researchers-fret-as-social-media-lift-veil-on-drug-trials-1406687404?mod=WSJ_hp_RightTopStories

By

Amy Dockser Marcus

July 29, 2014 10:30 p.m. ET

 

On her first day in a clinical trial for an experimental multiple sclerosis drug, Jeri Burtchell was convinced she was getting the new drug, not the standard therapy that some patients were randomly assigned to receive.

When she bumped into the trial’s lead investigator in the elevator that day, she told him, “I think I know which drug I’m on!” The doctor put up his hands, waving her off. “Don’t tell me!” Ms. Burtchell recalls him blurting. “I can’t know!”

A nurse later explained to her that knowing who was getting what could compromise the research. Even so, Ms. Burtchell went home and recounted the incident to followers of a blog she was writing about the trial. “The mystery medicine is doing its job,” she wrote.

For decades, the clinical trials vital to developing new drugs have followed a central principle: Researchers and patients must both be “blinded” as to who is getting the experimental drug and who a placebo or standard therapy.

If patients know who has the new drug, or learn too much about how others in the trial are faring, this knowledge could influence how they report symptoms and make it hard to tell whether the drug is working. Staying in the dark throughout a trial is a standard held sacrosanct by researchers. But lately, not so much by patients.

On Facebook groups, online forums and blogs, some patients are effectively jeopardizing the blind. In trials for hepatitis C, multiple sclerosis and ALS (Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease), patients have been sharing details of their reactions and trying to figure out whether they are getting the active drug.

Patients also swap tips on how to get accepted into trials, even if they don’t meet all the requirements. Some who are in trials collect and analyze their medical data and others’ to get an early indication of whether a drug will be a success.

Drug makers and researchers are increasingly concerned that online chatter could unravel the carefully built construct of the clinical trial, and perhaps put patients in danger. They worry that patients may drop out if they suspect they aren’t getting the drug being tested, or may report symptoms inaccurately because of the influence or suggestions of others in the trial.

Patients who share too much “could effectively chill a new drug before it ever gets to patients by misinterpreting early signals,” says Craig H. Lipset, head of clinical innovation at Pfizer Inc., who wrote an opinion piece in Nature Medicine in March calling on trial sponsors and regulators to study the effects of social media. “It’s scary.”

Patients, for their part, often say they have the right to talk about their experiences. In life-threatening diseases such as ALS, patients “want, need and deserve to know” how others are doing, says April Moundzouris, a Chattanooga, Tenn., woman whose “April’s ALS Blog” chronicles her experience with an experimental stem-cell treatment. The early-stage research by Neuralstem Inc. isn’t blinded.

Neuralstem Chief Executive I. Richard Garr , a close reader of April’s blog and those of other patients, says the sharing may pose a challenge later on when it comes time for a trial designed to prove efficacy to the Food and Drug Administration. That trial would likely have to be blinded.

A patient who blogs that he or she isn’t feeling well, isn’t improving or has some symptom can sway how other patients report or their willingness to join a trial, Mr. Garr says.

This is just one of the ways the Internet and social media bear on the drug-discovery process. Richard Bedlack, who runs the ALS clinic at Duke University, is involved in a project that asks patients to tweet tips about alternative and off-label treatments that he and other doctors assess to see if they might make promising research candidates.

By contrast, some pharmaceutical companies have tried to avoid using social media so they won’t learn about something that might make clinical analysis of an experiment more challenging or might have to be reported to FDA, according to Joseph Kim, an executive at Irish drug maker Shire PLC.

Says Mr. Garr: “The FDA is going to have to figure out how to accommodate social media.”

A spokeswoman for the FDA says the agency doesn’t have a policy on subjects in trials using social media to communicate with one another.

PatientsLikeMe, a Cambridge, Mass., company that runs a patient network and research platform where patients share health information, including during clinical trials, is wrestling with how to address the effects the constant sharing of information can have on trials.

In a 2012 paper, Paul Wicks of PatientsLikeMe wrote that according to the company’s data, a little more than 400 patients on the site have added treatment information that shows they are on a clinical trial. If patients start trying to determine if they are receiving the new drug, he asked, “how should we respond to this as researchers or as a company?” Dr. Wicks says the company is trying to get patients and researchers to work together to design trials, believing that collaboration will lead to better understanding of the issues.

To date, no clinical trial has been stopped because of online sharing of patients’ experiences. But drug makers are starting to take pre-emptive steps.

Shire helped create educational videos about the consequences of sharing too much, working with a Boston-based nonprofit called the Center for Information and Study on Clinical Research Participation. In one animated video, a blue bean’s description of side effects from a drug causes a yellow bean to drop out of a study. Shire, whose board has agreed to a takeover by AbbVie Inc., tells patients how to download the videos.

“The industry needs to do a lot more,” says Shire’s Mr. Kim, director of patient recruitment and engagement. “It is a hard thing to modify behavior without it being too controlling.”

Vertex Pharmaceuticals Inc. was testing a drug for hepatitis C when Lindsay McNair, then its senior medical director, got an alert from an investigator that patients in the trial were posting about it. She went to MedHelp.org, a site that hosts patient message boards, and started reading. “They were really incredible threads,” she says.

generic viagra prices Also keep a good check on the storage as well. It has some outstanding features can enhance your sexual desire and improve sexual function. http://davidfraymusic.com/events/theatre-croisette-cannes/ buying viagra in india If you want to purchase the medicine of viagra no prescription overnight , you have to log in to the particular site and make an order for that. The viagra uk sales effects of testosterone injections are not rapid. One patient advised those who wanted to know whether they were getting the experimental drug to ask a doctor not involved in the trial for a test of viral load. If it had dropped dramatically, the patient wrote, that was a sign the person was getting the new drug, because standard therapy wouldn’t have such an effect. Some patients discussed dropping out if they concluded they weren’t getting the new drug. (Some did quit the trial, though it isn’t known if online sharing was a factor.)

Dr. McNair discussed the posts with others at the Boston-based company. They were concerned about patients harming their health by not reporting symptoms out of concern they could be taken off the trial. If enough dropped out, it might be hard to get an adequate test of the drug, says Dr. McNair, now chief clinical research officer at WIRB-Copernicus Group, which provides regulatory and ethical reviews of research.

So for its next trial, Vertex added an unusual paragraph to the consent document for subjects, explaining that rumors about side effects or about the drug’s efficacy might affect results and require doing the study over. Participants should feel free to discuss their participation with family, friends and medical providers, the paragraph said, but not publicly such as in Internet message boards.

“It was meant to be informational and educate about the implications discussions could have, but not to say they can’t talk or there would be repercussions if they did,” says Dr. McNair.

Not all the patients understood this. One who signed the document then posted on the MedHelp Hepatitis C forum: “There’s no specific legal recourse spelled out, but there is another section that states that my participation in the study can be ended at any time without my permission.” The patient wrote that the forum provided him with important information about managing his disease and about the trial. “I hope people will still talk to me here?!” he added.

Vertex says oversharing on social media isn’t grounds for eviction from a study. It continues to warn about the problem in consent forms. Vertex says there was no effort to identify or remove from the trial the person who posted on the MedHelp forum.

The hepatitis C drug won FDA approval, and now is sold as Incivek, but “the experience was nerve-racking,” Dr. McNair says.

Dr. Bedlack of Duke says the information patients share online can turn out to be incorrect. Before revealing the drug information at the conclusion of a trial, he often asks patients to guess whether they got active drug. “Most times they don’t get it right,” he says.

Ms. Burtchell, the multiple sclerosis patient, did guess right.

Now a 53-year-old resident of East Palatka, Fla., she was diagnosed in 1999 with the kind of MS that involves relapses interspersed with remission. By 2007 she estimates she had suffered nearly 30 relapses and was so exhausted she had difficulty caring for her young son.

She enrolled in a trial for an experimental therapy from Novartis AG that aimed to be the first pill for the disease, which was treatable only with injectable drugs. The trial matched one of injectable therapy, Avonex, against the experimental oral drug, called fingolimod.

Ms. Burtchell started a blog to chronicle her experience from start to finish. In the post after her first treatment, she told why she believed she was getting fingolimod.

In the past, she had taken injections for her condition. In the trial, she didn’t feel the stinging or pain she remembered from shots—a sign, she believed, that her injection in the trial was a placebo.

Also, after she was given a pill, she noticed a nurse recorded a fall in her blood pressure, which she knew was a potential side effect of the experimental drug.

Nearly three months into the trial, Ms. Burtchell posted that, for the first time since her MS diagnosis, she was able to hop on one foot.

“Maybe it’s the Fingolimod…,” she speculated. “Whatever it is, I feel better every day.”

When information on the study was revealed, it turned out she was indeed getting the experimental drug.

A spokesman for Novartis says consent forms for its trials don’t prohibit patients from talking about their trial participation. The spokesman says the company didn’t interact with Ms. Burtchell during the trial.

The drug was approved by the FDA in 2010 and now is sold as Gilenya. Novartis continued to study it in nonblinded research, in which Ms. Burtchell participated. Eventually, she served for a time as a paid speaker for Novartis, sharing her experiences.

She also continued to blog.

One of her followers was Pfizer’s Mr. Lipset, who was interested in how patients use social media.

In September 2011, he sent her a message on Twitter asking to talk. Mr. Lipset says he wanted to discuss the downsides of patients comparing notes. They talked, and each saw the other’s side.

Mr. Lipset says Ms. Burtchell helped him realize that “it’s not the patients who will change, but the researchers who have to change.” She reminded him, he says, that “ultimately patients are human beings. They are going to talk.”

In Ms. Burtchell’s post after their talk, she conceded that the Pfizer executive had made many good points. Even so, she concluded, “If I’m going to be poked and prodded, I’m going to be here blabbing about it.”

Later, her views started to shift. In 2012, Mr. Lipset invited her to appear on stage with him at a pharmaceutical-industry conference in Boston. She was asked if she ever worried about influencing others in a trial. “I think that is when the realization of what I had done really hit me,” she says.

At a February drug-industry conference in Miami where she was asked to speak about patients and social media, Ms. Burtchell proposed her own solution: that drug companies create online forums where patients can get practical questions answered by investigators and can commiserate with other patients about social issues in a moderated setting. She set up Partners in Research, a website that guides patients through the clinical-trials process.

She continues to take Gilenya for her MS and has had two relapses while on it, most recently in February. She is part of a study looking at its long-term effects.

But she has stopped her blog.

“I’m too aware of the impact,” she says, “too conscious of my audience, to blog like that again.”

Posted on

Light-based acne treatment goes global via license/JV agreement

THE DAILY MEDICAL TECHNOLOGY NEWS SOURCE 

Light-based acne treatment goes global via  license/JV agreement

By Amanda Pedersen, Senior Staff Writer

MONDAY, JULY 21 , 2014

The dermatology market just got a little bit brighter. 

Klox Technologies (Laval, Quebec), a company developing a light-based technology for acne treatment and other dermatology indications, has gained global market access through a worldwide license and joint venture agreement with Leo Pharma (Ballerup, Denmark).

Leo Pharma will help Klox to further develop and commercialize its Biophotonic technology platform in dermatology, which includes a CE-marked treatment for moderate to severe acne. The worldwide agreement excludes Canada, where Klox already has a partner that launched the device a few months back. Leo Pharma has also agreed to make an equity investment in Klox. The deal paves the way for Leo Pharma’s first medical device therapy and first global market entry in acne.

The non-abrasive, non-thermal device comprises a multi- LED light used in conjunction with a photoactivable converter gel. It works by targeting the underlying problems that lead to acne vulgaris, as well as stimulating collagen synthesis and healing in traumatized skin, thereby promoting healing in the epidermis and deeper in the dermis, Klox said. Completed within a 15-minute treatment cycle including preparation, treatment requires twice-weekly application over six weeks. In clinical trials, the acne Biophotonic system demonstrated highly statistically significant improvements in moderate to severe acne sufferers, Lise Hébert, president/CEO of Klox, told Medical Device Daily.

 

The way it works is the converter gel is applied to the area being treated, which is then illuminated by the lamp. The interactions that take place aid in the creation of an environment favoring repair.

 

“Within the field of dermatology, acne was something that we felt was indeed an unmet need,” Hébert said. “Yes, there are many products on the market, mostly topical or antibiotics, but in the case of severe acne very, very little innovation was going on.”

Other companies in the space would repackage or reformulate existing products, but there was no treatment on the horizon that would really be a game changer, Hébert said.

 
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She noted that the company designed its European trial based on FDA parameters and guidance so that the data could one day be useful in the U.S. as well. The trial enrolled moderate and severe acne patients, mostly teens and young adults.

 

“The surprise that we got, that was not necessarily predicted, was that the lens not only addresses the acne but has a favorable impact on scarring,” Hébert said. The trial also examined the persistence of the efficacy over a six-month period, she said, and of the patients that have met that six-month follow up, “not a single one reverted to baseline.”

 

Hébert said the partnership with Leo further validates the technology and its applicability in multiple therapeutic areas. She said the two companies will develop and commercialize Biophotonic therapies in dermatology with the intent of bringing patients efficacious products with a good safety profile.

 

“This deal also represents the completion of a significant step in Klox’s strategic roadmap aimed at achieving sustainable, commercial profitability in the short to medium term,” Hébert said. She added that the agreement with Leo also helps Klox focus resources on developing the company’s wound care program, which Klox plans to commercialize as early as the first half of 2015 in Europe.

 

Leo Pharma will be responsible for clinical and commercial activities, including manufacturing. Also, Leo has agreed to provide financial support for Klox R&D in dermatology. The financial terms were not disclosed but the companies noted that it includes a “significant upfront, as well as double-digit, escalating, tiered royalty rate based on product sales.”

 

Klox wanted a strategic partner like Leo because the dermatology market is very large and already has several large, successful companies, so to compete in the space would require a large sales force and a lot of marketing dollars, she said.

“Today, there is a significant unmet need for alternative acne therapies. With this technology, Leo Pharma aims at soon offering a new treatment solution that is non-invasive and effective. We hope it will enable patients to integrate acne therapy into their everyday lives and successfully manage their condition,” said Gitte Aabo, president/CEO of Leo Pharma. “By partnering with innovative companies like Klox, Leo Pharma aims to push the boundaries of dermatology care and deliver more treatment solutions that make a real difference to the lives of people with skin diseases.”

Hébert told MDD that the company’s goal is to initiate a trial in the U.S. in the very near term and also to launch into commercial activities in Europe because the product is already approved there. Also, she said, the two companies will start working on the next product in Klox’s technology platform. The company also expects to get approval in Europe this summer for the next indication of the technology, which will be wound healing. //

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Azaya Therapeutics Inc. to collaborate with the University of Chicago

May 6, 2014, 8:30am CDT

Azaya Therapeutics Inc. to collaborate with the University of Chicago

http://www.bizjournals.com/sanantonio/blog/morning-edition/2014/05/azaya-therapeutics-inc-to-collaborate-with-the.html

W. Scott Bailey

May 6, 2014, 8:30am CDT

Azaya Therapeutics has formed a research partnership with the University of Chicago to test out a new proprietary formulation of docetaxel.

It is continue reading this link soft viagra tabs better to consume the pill with water and in an empty stomach for better effects. However this is another thing that these methods are prone to injuries, bleeding and harm to penile get more levitra properien region. The spedeeprootsmag.org viagra in the usat helps you to re-experience sexual emotions and inter sexual states3. It’s a truism that we live in an era which is seeing (fortunately and unfortunately), some of the greatest and most amazing developments in Science and Technology. http://deeprootsmag.org/author/david-mcgee/page/9/ on line cialis Azaya Therapeutics Inc. has entered into a collaboration with the University of Chicago. Investigators at the university’s Ludwig Center for Metastasis Research will work to develop a new application for Azaya’s investigational drug ATI-1123.

ATI-1123 is a proprietary formulation of the chemotherapy agent docetaxel, commonly known as Taxotere. The San Antonio-based clinical-stage oncology company is seeking to determine whether a low, clinically safe dose of radiation can significantly enhance the delivery of ATI-1123 to experimental tumors in laboratory mice.

The new study, led by Dr. Stephen J. Kron and Dr. Ralph R. Weichselbaum, will also document the effects of radiation on accumulation of docetaxel in the irradiated tumors, and examine whether the combination leads to greater inhibition of tumor growth.

Azaya officials say the work with University of Chicago researchers may serve as a model for future clinical studies in cancer patients undergoing radiation therapy.

“The outcome of this study could lead to a new treatment option for cancer patients, in which our ATI-1123 liposomal docetaxel would provide a powerful means to improve the benefits of radiotherapy,” says Azaya Therapeutics President and CEO Mike Dwyer. “Given the established safety of ATI-1123 on its own, we plan to test this combination in cancer patients if the results of the animal study are promising.”

Over half of all cancer patients receive radiotherapy during the course of their disease. Using the radiation to direct liposomal drugs to tumors could improve the benefits of treatment to these patients,” Azaya officials say.

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New trial may be step forward for spinal cord injuries

http://thechart.blogs.cnn.com/2014/04/16/new-trial-may-be-step-forward-for-spinal-cord-injuries/?iref=allsearch

April 16th, 2014

Miriam Falco – CNN Medical Managing Editor

New trial may be step forward for spinal cord injuries

In what may be another step forward in treating spinal cord injuries, a safety trial will begin this year on the practice of injecting stem cells directly into the injury site, Neuralstem Inc. announced Wednesday.

The Maryland company said the University of California, San Diego’s Institutional Review Board had approved its clinical trial protocol, which also has approval from the Food and Drug Administration.

The first eight patients who will be enrolled will be paraplegics who had a thoracic spinal cord injury one to two years ago and have no motor or sensory function below the point of their spinal cord injury.

Thoracic spinal cord injuries are rare, according to the Christopher and Dana Reeve Foundation, because of the protection afforded by a person’s rib cage. In addition to the loss of function in legs, patients also experience a loss of physical sensation, bowel and bladder problems and sexual dysfunction. However, in most cases, function of the arms and hands are not affected.

It’s the latest trial designed to inject stem cells into patients’ spines. The trial is supposed to show that the drug – stem cells, in this case – is safe, although researchers hope to provide some benefit as well.

In 2009, California-based Geron Corp. not only injected the first stem cells into a patient’s spinal cord, it was the first to use the highly controversial derivative of human embryonic stem cells. But after treating at least four patients, the company chose to shut the trial down in 2011 because of its expense and the company’s focus on cancer research.
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One week later, Neuralstem began its clinical trial in patients with amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease. That trial used a new device to inject stem cells into the spine without causing additional injury to the spinal cord. Neuralstem’s cells are not embryonic stem cells but rather cells taken from fetal spinal cords, which have already started to become nerve cells.

New Jersey-based StemCells Inc. launched spinal cord trials in Switzerland in 2011, using its own type of neural stem cells also derived from fetal tissue. It injected its first North American patient in Canada this year.

Unlike the goal of the Geron trial (similar to StemCells’), which was to re-mylenate nerve cells to re-establish connections from the spine to the brain – like fixing an exposed wire by providing a new cover – the goal in this new trial is to “actually build new circuitry,” Neuralstem CEO Richard Garr said.

“The stem cells are injected directly into the area of the injury and jump the gap with the new circuitry we’ve built,” he said. “These cells don’t migrate to the site.”

In animal experiments, rats were injected with these human stem cells and recovered significant function in all lower extremity joints, according to Neuralstem. “The cells turned into neurons which grew a ‘remarkable’ number of axons that extended for ‘very long distances,’ bridging above and below the point of severance,” the company says in a statement, quoting study results from August 2012.

Neuralstem is trying to reconnect the nerve cells from below the injury site to cells above the point of injury to re-establish signals going to the brain, says Karl Johe, the company’s chairman and chief scientific officer, who developed the cells.

Johe says he hopes the first patient will have the surgery to get the stem cells by July. “And then it would occur about once a month,” he added.

As for the most recent research, which discussed a much simpler method called epidural stimulation, Johe says it’s a complimentary approach. This month, researchers showed that four patients who had had spinal cord injuries for more than two years were able to ‘reawaken’ their muscles, so to speak: move their legs when electrodes implanted in the back were turned on, providing electrical stimulation to the spinal cord.

“There has been debate whether the motor neuron circuitry is intact below the injury point,” Johe said. “So now we know for sure (it is).”

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Azaya Therapeutics taking big steps

Azaya Therapeutics Inc. expects to close soon on a %5 million funding round that the company intiated in October 2013.  The convertible-note bridge Researchers believe that when watermelon is consumed, citrulline is converted into the amino acid arginine which acts as a precursor for nitric oxide, and the nitric oxide will help in blood sildenafil 10mg vessel dilation. Many online pharmacies are dealing cialis price australia with this problem, you should be certain to be ok soon. Don’t share your Sildenafil tablets with other people by not trusting them because of our past cialis canada online experiences. Over the years, chiropractic care best cialis price has gained popularity among men over the years. funding — which was oversubscribed and driven by local investors — is timely as the San Antonio-based biotech company prepares to further expand its business.

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GenSpera’s Targeted “Death Carrot” Toxin in Trials for Two Cancers

XCONOMY

Top Stories in Texas

http://www.xconomy.com/texas/

GenSpera’s Targeted “Death Carrot” Toxin in Trials for Two Cancers

http://www.xconomy.com/texas/2014/03/27/gensperas-targeted-death-carrot-toxin-in-trials-for-two-cancers/

Bernadette Tansey 3/27/14

 

Craig Dionne has a record of experience in drug development and a lot of friends in the pharmaceutical industry. But when his friends first heard about the experimental drug he started working on some years ago, he says their initial reaction was alarm—to put it mildly.

“They told me I was a fool,” Dionne says affably. Dionne is CEO of San Antonio, TX-based GenSpera, which has now started a second round of early stage clinical trials on the experimental drug he took a chance on. He’s hoping the trials are about to show his friends he was right.

Back in the early 1990s, Dionne and his collaborators at Johns Hopkins University saw potential for a cancer drug in a highly potent toxin, thapsigargin, found in a common Mediterranean weed called Thapsia Garganica. You’ll find it described as the “death carrot’’ in online accounts.

Thapsigargin kills fast-growing cells. So far, so good; that’s a routine property of chemotherapy drugs. But the relative safety of classical chemotherapy agents rests on the fact that they’re more destructive to rapidly multiplying tumor cells than they are to slow-growing normal cells.

Thapsigargin doesn’t work that way—which initially made it alarming to Dionne’s friends. It’s an equal opportunity cell-killer.

“It can kill cells that grow slowly,” Dionne says. That makes it a threat to normal cells, unless a drug company can successfully tweak it. And Dionne saw a reason to try.

Like most normal cells, some cancer cells also grow slowly, such as those in tumors of the prostate. And such cancer cells can resist chemotherapy drugs that target fast-growing cells. Dionne and his collaborators at Johns Hopkins reasoned that thapsigargin might be an effective weapon against slow-growing tumor cells, if it could be modified to spare normal cells. The end result of their chemical tinkering is GenSpera’s experimental drug G-202, which has now moved into a second round of early stage clinical trials.

The compound was designed to allow the powerful toxin to move through the bloodstream in an inactive form until it’s released by an enzyme, PSMA, found at higher levels on the surface of certain tumor cells, Dionne says. He compares G-202 to a grenade with a pin ready to be pulled.

A molecule of G-202 contains a modified form of thapsigargin called 12ADT, which is linked to a small peptide. The effect of that peptide—a small chain of five amino acids—is to keep 12ADT soluble in the watery environment of the blood. More importantly, he peptide keeps the toxin inactive.

But when G-202 encounters a cell with the enzyme PSMA (prostate-specific membrane antigen) on its surface, the enzyme clips the peptide off the toxic 12ADT molecule. The toxin, now insoluble in water but highly soluble in fats, can sink through the lipid-rich outer membrane of a nearby cancer cell and start its deadly work.

Dionne says G-202 is similar to the new class of targeted cancer drugs called antibody-drug conjugates, which are also designed to deliver a toxin only to particular tumor cells. These drugs combine a toxin with an antibody engineered to bind to a specific molecule on the surface of a cancer cell. However, antibodies are large proteins whose unique amino acid sequences and folding patterns can help them fit selectively only with certain cell surface receptors.

Serotonin-producing cells develop early on in the ENS, and if this buy generic levitra development is affected, the second brain cannot form properly. You will be helped to prolong your erection in a more healthy levitra online devensec.com and natural way by this medicine. Sexual dysfunction is a common concern shared by many women. generic levitra cheap This levitra 20mg canada medicine may lead to allergic reaction in some individuals. By contrast, G-202‘s targeting agent, the small peptide, could theoretically be clipped away from its toxic payload by enzymes other than PSMA, Dionne says. If that happened, the toxin could be released in the vicinity of normal cells rather than tumor cells. This is one of the reasons why GenSpera has spent years on preclinical testing and animal studies before launching its first clinical trial of G-202 in humans in 2011, he says.

Dionne says the company also took a close look at the cardiovascular effects of the experimental drug because of its molecular mechanism of action. Thapsigargin works by disrupting a molecular pump that regulates the level of calcium ions inside cells—and ion exchanges are central to the functioning of the cardiovascular system, Dionne says. Once inside any cell, whether a tumor cell or a normal one, thapsigargin causes a damaging influx of calcium, which starts a process of programmed cell death called apoptosis.

GenSpera also had to watch out for off-target effects of G-202 for another reason. Although the target enzyme PSMA appears in a higher concentration on cells of the prostate than on many other cells, it has been found as well in certain normal tissues, such as sections of the brain, small intestine, and kidney. Dionne says G-202 can’t pass through the blood-brain barrier. Studies of the drug’s action in animals and in humans so far have not turned up significant damage to the other normal cells bearing the PSMA enzyme, he added.

However, the discovery of enriched PSMA levels in yet another type of tissue has expanded the possible uses of G-202. The enzyme is more abundant on the cells of blood vessels that bring nutrients and oxygen to solid tumors than it is in normal blood vessels. Based on this finding, GenSpera began widening its views beyond prostate cancer, Dionne says. If the toxin in G-202 could be released by PSMA in tumor blood vessels, these vessels might disintegrate, and the tumor might starve and die.

“The potential applications are so much broader,” Dionne says.

In GenSpera’s first small trials in 2011 and 2012, 44 participants with a range of different cancer types received doses of G-202. Dionne says the drug appeared to have no serious effects on the liver, cardiovascular system, or bone marrow. Kidney damage could be prevented by limiting the G-202 dose and giving trial participants plenty of water, he says.

Although the main goal of the first trial was to assess the safety of G-202, GenSpera noted that a few trial participants with advanced liver cancer survived longer than expected. Encouraged by those signs, GenSpera is now conducting a Phase II trial in up to 17 adults with advanced hepatocellular carcinoma who are no longer helped by sorafenib (Nexavar), a cancer drug co-owned by Bayer and Amgen.

In March, GenSpera also began a Phase II trial in up to 34 participants with the brain cancer glioblastoma multiforme at the University of California San Diego’s Moores Cancer Center. The company may also conduct trials in prostate and kidney cancer if it can raise the funding.

GenSpera (OTC: GNSZ) is a tiny virtual company, that had $3.6 million in cash at the end of 2013—enough to fund its operations through at least nine months of this year. The company last week announced a share offering to raise as much as $7.5 million.

Dionne, a former executive at Cephalon, which was acquired by Teva Pharmaceutical Industries (NASDAQ: TEVA) in 2011, co-founded GenSpera in 2003. The firm began its preclinical studies in 2007, when it raised its first funds from outside investors. Dionne, who is GenSpera’s chief financial officer as well as CEO, is one of the company’s staff of two full-time employees. GenSpera contracts out all its other work.

“We’re virtual out of choice,” he says.

GenSpera’s scientific advisory board includes company co-founders John Isaacs and Samuel Denmeade, who are oncology professors at the Johns Hopkins University Sidney Kimmel Comprehensive Cancer Center.

GenSpera has raised $24.3 million since its inception, and the majority has come from high net worth individuals who often continue to reinvest, Dionne says. The company went public in 2009 and trades in the OTCQB tier of the Over-The-Counter Markets Group. GenSpera’s market capitalization is about $37 million.

Dionne says the company’s ideal plan is to seek a pharmaceutical company partner or buyer after conducting Phase II trials that show the potential of G-202 in solid tumors.

“We want to be valued across a range of tumor types,” Dionne says. The company has been built with the goal of making it attractive to big drug companies, he says.

“Our customer is large pharma,” Dionne says. “That’s who is ultimately going to pay us what we’re worth.”