Osiris Therapeutics inc said on Thursday that Canadian health regulators have approved its treatment for acute graft-versus host disease in children, making it the first stem cell drug to be approved for a systemic disease anywhere in the world.

Osiris shares rose 14 percent to $6.00 in extended trading after the news was announced.

Graft versus host disease (GvHD) is a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient’s body. Symptoms range from abdominal pain and skin rash to hair loss, hepatitis, lung and digestive tract disorders, jaundice and vomiting.

The disease kills up to 80 percent of children affected, Osiris said. To date there have been no approved treatments for the disease. Canadian authorities approved the therapy, Prochymal, for use in children who have failed to respond to steroids.

Prochymal was approved with the condition that Osiris carry out further testing after it reaches the market. C. Randal Mills, the company’s chief executive, said in an interview that could take three to four years.

Some investment analysts have been skeptical about Prochymal’s future. in 2009, two late-stage clinical trials failed to show the drug was more effective overall than a placebo in treating the disease, though it showed promise in certain subgroups of patients.

Since then, the company has mined data from all its clinical trials to show that in patients with severe refractory acute GvHD — those who have more or less failed all other therapies — Prochymal demonstrated a clinically meaningful response at 28 days after therapy began in 61-64 percent of patients.

In addition, treatment with Prochymal resulted in a statistically significant improvement in survival when compared with a historical control population of pediatric patients with refractory GvHD.

The Canadian authorities approved the drug on the basis of that data, the company said.

FDA submission this year

Osiris, which is based in Columbia, Maryland, plans to apply for marketing authorization with the U.S. Food and Drug Administration by the end of this year, including the newly-analyzed information. Mills said that if the FDA were to approve the drug, he would expect it to be on similarly conditional terms as outlined by the Canadian authorities.

In general, the FDA does not approve drugs based on subset analyses.

Prochymal is made up of bone marrow stem cells derived from an adult donor and is designed to control inflammation, promote tissue regeneration and prevent scar formation. It is not entirely clear how it works to help patients with GvHD, of which there are between 3,500 to 4,000 worldwide, Mills said.

Osiris said it will receive at least eight years of exclusivity in the Canadian market. but competition is heating up elsewhere.

Athersys inc said last month it had met with the FDA to discuss the results of a recently completed clinical trial of its MultiStem stem cell treatment to prevent GvHD in patients being treated for leukemia or other conditions that place them at risk of the complication.

Dozens of adult stem cell therapies are moving through clinical trials, and Canada’s approval of Osiris’s drug will likely boost optimism in the sector. Stem cells derived from adult tissue such as fat or bone marrow circumvent the ethical concerns raised by the use of cells derived from embryos.

In 2008, Genzyme Corp, which is now owned by Sanofi SA, paid $130 million to Osiris and agreed to pay up to $1.25 billion more if Prochymal and another Osiris drug, Chondrogen, designed to repair tissue in damaged knees, reached the market and achieved certain sales levels.

In February, Sanofi said it had discontinued its project with Prochymal. Osiris said the statement was issued without its consultation and that it had not received notice from Sanofi regarding the discontinuation. however, the company said that the agreement between the two companies provides that in this instance all rights to Prochymal revert to Osiris without compensation to Sanofi.

Tulane University’s Dr. Son Nguyen is hoping to raise hepatitis awareness locally by coordinating two West Bank testing events this weekend. Free screenings for hepatitis B and C, as well as blood pressure, blood glucose, and body mass index, will be offered.

The Saturday event will be held from 9 a.m. to 5 p.m. at Saint Agnes Le Thi Thanh Church, 6851 Saint Le Thi Thanh St., Marrero. the second event, on Sunday, runs from 9 a.m. to 4 p.m. at Duc Me Len Troi Catholic Church/Assumption of Mary Church, 171 Noel Dr., Avondale. Organizers hope more than 600 will attend.

Inflammation caused by hepatitis can result in liver disease, including cancer and cirrhosis. many people with chronic hepatitis B have no symptoms; those who do often mistake them for the flu — with fatigue, nausea, and stomach pain commonly reported. Vietnamese-Americans are disproportionately impacted by chronic hepatitis B.

According to organizer Chantelle Jabbia, a similar testing event held in January in eastern new Orleans reached 392 people. Of these, 39 were diagnosed with hepatitis B and referred for treatment.

Back to other news for April 2012

On April 24, in response to multiple bite reports, Pender County Sheriff’s Office Animal Control removed a pet monkey named Elvis from the home of Nancy Browning, 68 Camellia Drive.

Under orders from State Veterinarian Dr. David Marshall, Animal Control Officer Lt. Keith Ramsey and several deputies removed the male macaque monkey from the home. The animal had reportedly bitten one of Browning’s neighbors for the second time in two months.

According to Ramsey, the macaque had bitten the woman once before and her son twice. The first bite occurred on Feb. 3 and was reported Feb. 6. The second bite was reported April 21. Ramsey said another bite occurred sometime between the dates of the first two bites but was unreported by the victim.

“this monkey has bitten three times in two months,” said Ramsey. “It had obviously become a health hazard.”

After the first incident, the macaque had been quarantined in the Browning home for 30 days for observation. After an investigation and the quarantine, Ramsey said all appeared to be acceptable with the monkey. then, a few days before the removal, the sheriff’s office received the report of another bite.

At that point, state authorities stepped in and ordered the euthanization of the monkey and testing for rabies.

“The health director received a letter from the state vet authorizing to euthanize. It’s an unfortunate situation that vaccination and containment could have prevented,” Ramsey said.

Though it is believed that Elvis did not have rabies, a bite could have still led to infection with Hepatitis B. A previously published report by the Centers for Disease Control specifically outlined the risk of B-virus infection from pet macaque monkeys.

“B-virus from Pet Macaque Monkeys: an Emerging Threat in the United States?” states, “Of primary concern when evaluating Macaque bites are bacterial and B-virus infections. B-virus infection is highly prevalent (80 percent to 90 percent) in adult Macaques. The virus must be assumed to be a potential health hazard in Macaque bite wounds; this risk makes Macaques unsuitable as pets.”

The CDC report said macaque bites may cause potentially fatal infections of the brain and central nervous system in humans.

The report supports the actions of animal control and the state vet, stating, “both veterinary specialists and breeders of nonhuman primates agree that as a rule, all these animals bite. Biting incidents eventually bring the animals to the attention of animal control authorities. Most state health departments can require that any biting non-domestic animal be euthanized and the brain be submitted for rabies testing.”

The close bond formed by owners and pet macaques can lead to serious health hazards, according to the CDC.

The report continues, “Owners of pet Macaques are often reluctant to report bite injuries from their pets, even to their medical care providers, and may fail to appreciate that the premonitory headache and flu-like symptoms could be associated with healed, often minor, bite wounds dating back more than a month.”

Ramsey could not speculate as to what, if any, medical treatment the victims sought for their wounds. The victims’ names were not provided.

Numerous attempts were made to contact Browning for this story. she was unavailable for comment.

Pender County currently has no regulations regarding the keeping of exotic animals, according to Pender Health Director Carolyn Moser.

The responsibility for the establishment of regulations on exotic pets would fall to county commissioners.

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"The choice is yours; either get tested for thalassaemia before tying the knot or suffer the regrets and life-long traumas," Thalassaemia Awareness and Prevention in Pakistan’s President Ayesha Abid.

ISLAMABAD: 

There is a dearth of treatment facilities for haemophilia patients in public hospitals, said health experts here on World Haemophilia Day (Tuesday).

In the government sector, there is only one haemophilia centre based in Pakistan Institute of Medical Sciences (Pims) in the capital. The treatment being expensive, remains inaccessible especially to low income groups.

Haemophilia is a genetic bleeding disorder, in which the blood takes a long time to clot. it is caused when clotting factors VIII or IX are missing, which results in high chances of bleeding. Since the disorder occurs in X chromosome, the chance that women will have two defective chromosomes are minimal. so they generally serve as carriers and pass them on to male childs.

Pims Haemophilia Centre Senior Medical Officer Dr Tazeen Anwar said that patients are primarily treated by injecting factor VIII, if not available then by transfusing plasma. “The injection is not available here and patients are asked to get it from outside. one injection costs over Rs10,000 and patients can require more than one doze depending on the severity of the bleeding,” she added.

She explained that previously, patients received financial assistance from Baitul Mal, but that has not been the case for the past five months due to financial constraints. Plasma is also not available around the clock in the hospital. she said currently there are 560 cases of haemophilia registered with Pims and it is estimated that every year about 400 new cases of this disease emerge in the country but remain unreported.

Pakistan Haemophilia Society Rawalpindi-Islamabad Chapter President Dr Tahira Zafar, said currently there are 4,000 registered haemophilia patients in Pakistan. she said in Pakistan only factor VIII injections are available. however, there is a need to have injections of other factors such as VII, IX, XII among others.

The society through international organisations has arranged these missing injections for patients in case of an emergency. she said repeated plasma infusions can make the patient vulnerable to hepatitis C, as many of the process of blood transfusion or screening is not safe in most blood banks.

While sharing the symptoms, she said when a baby is born with haemophilia, there are blue bruises on his body, bleeding from nose, gums, there is an excessive bleeding when umbilical cord is cut and in males when circumcision is done.

Health Awareness Society Director Dr Samia Baber stressed a need to create awareness about this disease among general public. whenever there is an excessive bleeding on minor cuts, one should consult the doctor and check for haemophilia. Dentists and surgeons should also be informed about the disease.

A family having history of this disease should avoid inter-family marriages. “There is a need to educate people on haemophilia, as it does not spread by shaking hands, secretions, touching or having food with a patient,” she said.

Published in The Express Tribune, April 18th, 2012.

This week, at the American College of Cardiology annual conference, new research was presented that linked soy products with reductions in blood pressure. The study was presented by lead researcher Safiya Richardson, who analyzed the diets of over 5-thousand patients in a survey related to cardiovascular diseases.Richardson found that those who had a daily intake of isoflavones had the lowest blood pressure. Isoflavone compounds are commonly found in soy milk, tofu, green tea, and even peanuts. Experts agree that the unique study will be fruitful to those looking to avoid heart disease and hypertension, but recommend that a more active lifestyle along with a healthier diet will prove most effective. The Korean Financial Supervisory Service has released a preliminary report on delinquincy rates on domestic bank loans. they found that households last month had the highest rate of late payments and defaulting loans in over three years. The financial regulatory agency stated that delinquincy rates were at zero.eight-five percent compared to just under half a percent in 2009 The report also stated that corporate delinquicy rates have been steady at 1.three-eight percent.The rising household loan delinquincy rates were attributed to the higher cost of goods and a larger number of families taking out loans and mortgages… According to recent statistics released by the Korea Centers for Disease Control and Prevention, the number of patients with hepatitis rose to over 4,300 last year.Hepatitis C is spread through blood and bodily fluids. Symptoms include chronic fatigue, nausea, vomiting, and muscle ache. unlike hepatitis B, hep-C cannot be prevented through vaccinations, but symptoms can be treated with various drug therapies. The CDC recommends paying attention to personal hygiene especially when it comes to piercings and tattoos. Paul Yi, Arirang NewsMAR 31, 2012   Reporter : arirangpaulyi@gmail.com  

While Hepatitis B and C have acquired almost an epidemic status in our country, Auto Immune Hepatitis is largely overlooked in the list of liver diseases. known to mostly affect women, AIH incidentally does not have any specific symptoms, making it a potential threat for Indian population.

World renowned couple Dr Giorgina Mieli-Vergani, emeritus professor of paediatric hepatology, King?s College Hospital, London and her husband

Dr Diego Vergani, emeritus

professor of liver immunopathology, King?s College Hospital London, said, ?AIH is

a progressive inflammatory

liver disorder affecting mainly females, characterised by the presence of auto antibodies. as a result, our body?s infection fighting mechanism cells start behaving just the opposite. By the time, it is diagnosed, much of the damage to the liver is already done.?

If left untreated, AIH generally progresses rapidly to cirrhosis and liver failure. the peak incidence of the disease is before puberty and 75 per cent of the patients are girls.

?In fact researches have established that patients of Hepatitis C are at greater risk of developing AIH and since India has a significant number of patients of Hepatitis C virus, therefore it assumes larger significance?, said Dr Giorgina.

Top identify AIH patients in this part of the country, already a team of experts at the department of Immunopathology, PGIMER is on board a project with the doctor couple. ?The only hindrance is about transportation of samples. We are working out on a modalities for the smooth continuation of the research to find out the pathogenesis of Indian patients with this condition,? said Dr Diego.

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Major breakthroughs in hepatitis C treatment are of no help to the estimated 500,000 UK residents who are unaware they are infected. A poor understanding of who is at risk for hepatitis C means many who could be treated are not, said Mark Wright, a consultant hepatologist at Southampton General Hospital. “Hepatitis C is a tricky virus which often results in liver damage before it causes any symptoms so, with most of the estimated half-a-million affected people in the UK unaware they have the disease, it is easy to see we are on dangerous ground,” said Wright. “Yet the tragedy of all this is that, while we are not seeing the large numbers of people we know are infected coming forward due to ongoing poor understanding of the risks, treatments continue to get better and stronger yet may, sadly, go to waste if we can’t get this vital message through,” Wright noted. “We run the risk of seeing an entire generation lose their chance to live a fit, long and healthy life, in the worst possible circumstances, when the treatment is there and available for those who recognize they are vulnerable.”Two new hepatitis C drugs, telaprevir and boceprevir, will be rolled out by the National Health Service later this year. Southampton is participating in a series of studies of the drugs at the Wellcome Trust Clinical Research Facility.

Back to other news for February 2012

Gilead Sciences inc.’s shares plunged Friday after the drugmaker said a promising hepatitis C treatment it acquired as part of the $11 billion Pharmasset purchase may need help from other drugs to effectively treat some patients.

The Foster City, Calif., company said some patients in a small portion of a midstage study relapsed after completing a treatment that combined the company’s potential drug, labeled GS-7977, and ribavirin, an older medicine. Gilead executives told analysts they may have to add additional antiviral drugs to that combination or extend the length of treatment.

Company shares had climbed 47 percent over the past two months from around $37 to top $54 as Gilead worked toward completing the Pharmasset inc. deal, which it announced in November. But the stock sank 14.3 percent, or $7.81, to close at $47 Friday while the Nasdaq exchange fell slightly.

Hepatitis C is a virus that can lead to life-threatening liver damage and is the main cause of liver transplants in the United States. the disease is spread through the blood, and that can happen through sharing intravenous drug needles or having sex with an infected person.

It can take years to manifest, and analysts see hepatitis treatments as potentially lucrative for drugmakers because they expect the virus to become a growing health problem as the U.S. baby boom generation ages.

Analysts see potential in GS-7977, a pill that could become a preferred option for care if it works without the injectable drug interferon, which can leave patients with flu-like symptoms that last for months. Gilead is running several middle- and late-stage studies of the drug.

In the study discussed Friday, Gilead tested the medicine in hepatitis C patients with genotype 1, the most prevalent subset of the disease in the Western world and the hardest to treat. the patients in this study had already been treated unsuccessfully with interferon.

Gilead said eight patients had undetectable levels of the virus in their blood after being treated with the oral GS-7977-ribavirin combination. But six had a virus relapse in the month after treatment ended. the other two showed no relapse in the two weeks after their treatment ended.

WBB Securities analyst Steve Brozak said drugs typically have unanticipated outcomes like this as they advance through clinical testing and even after they hit the market. He said Gilead knows the drug works, but now they need to retool their treatment approach.

"It isn’t so much that this happened," he said. "the critical key is how does Gilead respond to what just happened."

Gilead, known for the HIV drugs Atripla and Truvada, also acquired some other potential hepatitis treatments in the Pharmasset deal, which closed last month. even so, some analysts see the acquisition as risky, given the price and the fact that Pharmasset had no products on the market. Gilead agreed to pay $137 per share in cash for Pharmasset, a stock that had traded as low as $20.49 in the year before the deal was announced.

GS-7977, which previously went by a different name, was seen as Pharmasset’s most promising treatment.

Summer Street Research analyst Carol Werther said Friday’s announcement was the first disappointing news for a potential treatment that "looked like the holy grail" in hepatitis C. she thinks the stock market overreacted.

"the drug isn’t dead," she said.

The judge’s ruling ended more than a year of legal wrangling over the ability of the 62-year-old former gastroenterologist and Nevada state Board of Medical Examiners member to understand the charges against him and assist in his defense.

“The only impediment to competency asserted by the defendant is self-reported memory loss, secondary to two strokes, regarding facts relevant to his criminal charges,” the judge said. “Memory loss itself, even if true, is not a bar to prosecution of an otherwise competent defendant.”

Desai’s lawyer, Richard Wright, contends that Desai is incapacitated by his strokes and other physical and mental ailments, but lost a bid to get the Nevada Supreme Court to postpone trial. He did not immediately respond to messages Thursday.

The ruling cleared the way for Desai and two former employees to face a jury before another Clark County judge, Donald Mosley.

Trial is currently set for March and is expected to take several weeks. that schedule could change because Mosley is preparing to retire from the bench.

Court spokeswoman Mary Ann Price said Thursday that Mosley hasn’t announced a departure date.

Desai and nurse-anesthetists Keith Mathahs, 75, and Ronald Lakeman, 64, have pleaded not guilty to the felony charges against them, including racketeering, reckless disregard for and patient safety, insurance fraud, theft and obtaining money under false pretenses. Each remains free on bail pending trial.

The three men were accused in a 28-count indictment handed up in June 2010 of infecting at least seven patients with hepatitis C by reusing endoscopy scopes, bite plates and vials of injected anesthetic during outpatient procedures in 2007 at Desai’s Endoscopy Center of Southern Nevada and Desert Shadow Endoscopy Center.

Southern Nevada Health District officials began notifying more than 50,000 Desai patients in February 2008 to be tested for hepatitis and HIV. Authorities later determined that nine people contracted hepatitis C through the unsafe practices, and said cases involving another 105 patients might have been related.

The outbreak spawned dozens of civil lawsuits in Nevada by patients seeking damages for becoming infected with hepatitis C or for being put in fear that they had been infected.

Desai also faces federal conspiracy and fraud charges on a separate indictment handed up last April in U.S. District Court in Las Vegas. Wright pleaded not guilty on Desai’s behalf and argued in federal court that Desai is not competent for trial.

Published: Sunday, February 19, 2012 at 10:23 p.m. Last Modified: Sunday, February 19, 2012 at 10:23 p.m.

LAKELAND | Hope and cure are words some hepatitis C patients haven’t put much stock in, but the approval of two new drugs is causing that attitude to change.

Facts SUPPORT GROUP

The hepatitis C support group meets at 6:30 p.m. Tuesday in Room 201 of Lakeland Regional Medical Center, off the B lobby. A nurse practitioner from Vertex Pharmaceuticals, which markets Incivek, will speak. This is open to anyone with hepatitis C or is interested in the treatment for it. Register at 863-409-4719.

Victrelis and Incivek can make a significant difference, said Dr. James H. Johnson, a Lakeland gastroenterologist who treats those patients and offers a monthly support group.

“We can wipe the slate clean,” he said. “We can use the word ‘cure.’ “

Although not everyone will be cured, he said, clinical trials have shown major successes in reducing the level of the virus in patients and keeping it from going back up once treatment ends. the new drugs were approved in spring 2011.

Hepatitis C is a liver disease that results from infection with hepatitis C virus. it is spread through the blood.

About 3.2 million people in the United States have the disease. more than half don’t realize they’re infected, according to the federal Centers for Disease Control and Prevention.

For some, it’s become almost a life sentence.

Elaine Erb, 54, has been battling hepatitis C for half of her life. This latest round of treatment, Incivek combined with hepatitis medications previously in use, is her third attempt.

In the previous two, she responded well but the virus level came back up when treatment ended.

That makes her a relapser, someone who does well but isn’t able to totally eliminate the disease from her body.

“This is my third time and I’m also zero viral content,” said Erb, who worked in the medical field and at Piper Aircraft before becoming too sick to keep working.

“I just hope it stays this time.”

Ironically, being a previous relapser may make her a winner this time. Because she responded to the previous treatment, the East Polk resident has a better chance of responding favorably to this combination.

“Hepatitis C is a very complex thing,” Johnson said. “The medications have always had a lot of side effects. There are a lot of interactions.”

These new drugs work directly on the virus. They’re used in combination with existing anti-viral therapies like pegylated interferon and ribavirin that boost the immune system’s ability to fight the virus.

This is how the process is designed to work: the new drugs, direct-acting antivirals, prevent the virus from replicating itself. When the level of the virus shrinks, the interferon and ribavirin keep it in check.

Previous therapies alone had a clearance rate of about 40 percent, but combining a new drug with standard therapies had a clearance rate of more than 75 percent. For some patients, Johnson said, therapy will be cut in half from 48 weeks to 24 weeks.

At a January support meeting, patients shared their stories of how the illness and medications affect them.

The new therapy isn’t noticeably easier on them physically or mentally, they said.

Throughout their comments, however, ran a thread of hope and excitement.

“We’re thrilled with the new medications that are available and thrilled with the prospect of people being able to say they’re cured,” said Sue Clements, a nurse who works with Johnson.

“I love these guys,” said Tim Hardee, 57, who is undergoing hepatitis C treatment for the first time. “They are family.”

Hardee, describing hepatitis C as “a time bomb” said he saw the ugliness of end-stage hepatitis C/liver disease when he was in an Orlando jail and helped with palliative care for inmates.

“I had them drop off with hepatitis C in front of me,” the Kathleen resident said.

“I’ve seen them swell up this big and the state didn’t want to spend money to drain them off. … I didn’t want to go out that way.”

In addition to being complex, hepatitis treatment also is costly. Private companies that manufacture the prescription treatment he gets, Merck and Genetech, supply it free to him, Hardee said. He is on Victrelis.

The level of hepatitis C virus in his blood is “at zero detectability” now, Hardee said.

He believes he acquired hepatitis C as a former drug user.

Erb thinks hers came when she worked as a phleboto-mist.

About two-thirds of those who are infected are baby boomers, in large part stemming from IV drug use in the 1970s and 1980s, Johnson said. Needle sticks in the health-care profession are another common cause.

Treatment isn’t easy. Flu-like symptoms, fatigue, nausea, anemia, depression, irritability, sleep disturbances and anxiety occur.

Although the interferon helps the immune system fight the hepatitis C virus, it leaves patients more vulnerable to some other infections, Johnson said.

Erb’s husband, Gary, takes her to the movies but they go to matinees to avoid close contact with people who have colds.

“That’s nice,” another group member said. “That lets you be part of society.”

Getting together at the support group meeting helps boost the morale of people who have hepatitis C, Hardee said.

[ Robin Williams Adams can be reached at or 863-802-7558. Read her blog at robinsrx.blogs.theledger.com. Follow on Twitter at ledgerROBIN. ]