USFDA gives 21 grants for research on RARE DISEASES

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The U.S. Food and Drug Administration today announced that it has awarded 21 new clinical trial research grants totaling more than $23 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry with research spanning domestic and international clinical sites.

“We are proud of our 30-year track record of fostering and encouraging the development of safe and effective therapies for rare diseases through our clinical trials grant program,” said Gayatri R. Rao, M.D., J.D., director of FDA’s Office of Orphan Product Development, within the Office of Special Medical Programs. “The grants awarded this year will support much-needed research in 21 different rare diseases, many of which have little, or no, available treatment options.”

The FDA awards the grants through the Orphan Products Clinical Trials Grants Program to encourage clinical development of drugs, biologics, medical devices, or medical foods for use in rare diseases. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products.

Since its creation in 1983, the Orphan Products Clinical Trials Grants Program has provided more than $370 million to fund more than 590 new clinical studies and supported the marketing approval of more than 55 products. Five of the studies funded by this grants program supported product approvals in 2015 alone, including much needed treatments for neuroblastoma, lymphangioleiomyomatosis, hypoparathyroidism, and hypophosphatasia.

Consistent with the tenor set by Vice President Joe Biden’s National Cancer Moonshot Initiative to accelerate cancer research, 24 percent of the new grant awards fund studies enrolling patients with cancer; 40 percent of these studies target devastating forms of brain cancer, one of which recruits children with recurrent or progressive malignant brain tumors.

Forty-three percent of this year’s awards fund studies that enroll pediatric patients as young as newborns. Of these, two focus on research in transplantation and related issues.

In addition, one funded project is a medical device trial to develop a fully implantable neuroprosthesis for grasp, reach, and trunk function in individuals with spinal cord injury with the potential to enable these patients to use their hand, arm, and trunk more independently.

A total of 68 grant applications were received for this fiscal year, with a funding rate of 31 percent (21/68). The grant recipients for fiscal year 2016 include:


  • Chemigen, LLC (Zionsville, Indiana), Yansheng Du, Phase 1 Study of CC100 for the Treatment of Amyotrophic Lateral Sclerosis — about $243,000 for one year
  • Chemocentryx, Inc. (Mountain View, California), Petrus Bekker, Phase 2 Study of CCX168 for the Treatment of Anti-Neutrophil Cytoplasmic Auto-Antibodies Associated Vasculitis — $500,000 for one year
  • Columbia University Health Sciences (New York, New York), Elizabeth Shane, Phase 2B Study of Denosumab to Prevent Bone Loss in Idiopathic Osteoporosis in Premenopausal Women Treated with Terripatide — about $1.6 million over four years
  • DNATRIX, Inc. (Houston, Texas), Frank Tufaro, Phase 2 Study of DNX-2401 for the Treatment of Glioblastoma — $2 million over four years
  • Elorac, Inc. (Vernon Hills, Illinois), Scott Phillips, Phase 3 Study of Naloxone Lotion for the Treatment of Pruritus in Mycosis Fungoides — about $2 million over four years
  • Johns Hopkins University (Baltimore, Maryland), Pamela Zeitlin, Phase 1/2 Study of Glycerol Phenylbutyrate for the Treatment of Cystic Fibrosis — $750,000 over three years
  • Oncoceutics, Inc. (Hummelstown, Pennsylvania), Wolfgang Oster, Phase 1/2 Study of ONC201 for the Treatment of Multiple Myeloma — about $1.7 million over four years
  • Oregon Health and Science University (Portland, Oregon), Kevin Winthrop, Phase 2 Study of Clofazimine for the Treatment of Pulmonary Mycobacterium Avium Disease — about $1.8 million over four years
  • Santhera Pharmaceuticals (Liestal, Switzerland), Thomas Meier, Phase 1 Study of Omigapil for the Treatment of Congenital Muscular Dystrophy — $246,000 for one year
  • Scioderm, Inc. (Durham, North Carolina), Jay Barth, Phase 3 Study of SD101 for the Treatment of Epidermolysis Bullosa — $500,000 for one year
  • Seattle Children’s Research Institute (Seattle, Washington), Leslie Kean, Phase 2 Study of Abatacept Combined with Calcineurin Inhibition and Methotrexate for Prophylaxis of Graft Vs Host Disease — $99,630 for one year
  • Sloan-Kettering Institute Cancer Research (New York, New York), Katharine Hsu, Phase 1 Study of Humanized 3F8 MoAb and NK cells for the Treatment of Neuroblastoma — about $750,000 over three years
  • Taimed Biologics USA Corp (Irvine, California), Stanley Lewis, Phase 3 Study of Ibalizumab for the Treatment of Patients with Multidrug Resistant HIV — $500,000 for one year
  • University of Alabama (Birmingham, Alabama), Gregory Friedman, Phase 1 Study of HSV G207 & Radiation for the Treatment of Pediatric Brain Tumors — about $750,000 over three years
  • University of California, San Diego (La Jolla, California), Donald Durden, Phase 1 Study of PI-3 Kinase/BRD4 Inhibitor SF1126 for the Treatment of Hepatocellular Carcinoma — $750,000 over three years
  • University of Florida (Gainesville, Florida), Peter Stacpoole, Phase 3 Study of Dichloroacetate for the Treatment of Pyruvate Dehyrugenase Complex Deficiency — about $2 million over four years
  • University of Michigan (Ann Arbor, Michigan), Kathleen Stringer, Phase 2 Study of Inhaled Activase for the Treatment of Acute Plastic Bronchitis — $2 million over four years
  • University of North Carolina Chapel Hill (Chapel Hill, North Carolina), Matthew Laughon, Phase 2 Study of Furosemide for the Prevention of Bronchopulmonary Dysplasia in Premature Infants — about $1.4 million over four years
  • Vanderbilt University Medical Center (Nashville, Tennessee), Cyndya Shibao, Phase 2 Study of Atomoxetine for the Treatment of Multiple System Atrophy — about $1.6 million over four years
  • Wilson Wolf Manufacturing Corporation (New Brighton, Minnesota), Sunitha Kakarla, Phase 1 Study of Viralym-A for the Treatment of Adenovirus Disease — about $750,000 over three years

Medical Devices:

  • Case Western Reserve University (Cleveland, Ohio), Kevin Kilgore, Phase 2 Study of a Networked Neuroprosthesis for Grasp, Reach, and Trunk Function in Cervical Spinal Cord Injury — about $2 million over four years

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The measles vaccine has saved more than 20 million lives since 2000, report finds

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A new report from the World Health Organisation (WHO) estimates that 20.3 million deaths have been prevented by the measles vaccine between 2000 and 2015.

It’s an incredible achievement for public health. In fact, thanks to vaccines, many of us have forgotten how deadly measles can be.

But not everyone is so lucky – despite all the progress in the developed world, the report also revealed that almost 400 children still die from measles every single day around the world. And the WHO says global immunisation progress has slowed since 2010.

To figure out the number of lives saved, the study compared the number of people who died of the disease before the WHO started its global push for vaccination in 2000, to now.

Between 2000 and 2015, it calculated that there was a 75 percent decline in reported measles incidents, and a 79 percent drop in measles deaths.

That means that roughly 20.3 million lives were saved thanks to the vaccine.

Screen Shot 2016-11-14 at 12.35.22 pmWHO

But despite all this progress, we still have a long way to go. As recent outbreaks in California and Germany have shown, the airborne measles virus is just as contagious, and just as deadly as ever.

As a reminder of how dangerous measles can be, in 1954, before the invention of a vaccine, the virus was so common that infection with measles was talked about as being “as inevitable as death and taxes“.

As recently as 1980, before widespread use of the measles, mumps, and rubella (MMR) vaccine, 2.6 million people worldwide were dying from measles every year. 

The vaccine has been so successful, in fact, that the whole of the Americas has now been declared measles free, thanks to vaccine campaigns. But progress has been uneven.

Just last year, 134,200 children were killed by measles, and 75 percent of them lived in the Democratic Republic of the Congo, Ethiopia, India, Indonesia, Nigeria, and Pakistan.

“It is not acceptable that millions of children miss their vaccines every year. We have a safe and highly effective vaccine to stop the spread of measles and save lives,” said Jean-Marie Okwo-Bele, the director of WHO’s Department of Immunisation, Vaccines, and Biologicals.

“This year, the Region of the Americas was declared free of measles – proof that elimination is possible. Now, we must stop measles in the rest of the world. It starts with vaccination.” 

In the latest report, the WHO admitted it had fallen short of all three goals it set itself for 2015 back in 2010.

Those were to:

  • Increase the number of one-year-olds who have been given the first dose of a measles vaccine to more than 90 percent in each country, and more than 80 percent in each region
  • Reduce global annual measles incidences to less than 5 cases per 1 million people
  • Reduce global measles mortality by 95 percent from the 2000 estimate.

Although significant progress has been made, and mortality rates are now almost 80 percent lower than they were in 2000, we’ve still got more to do.

Local governments around the world are now fighting to keep their immunisation rates high, with strategies such as cutting child and healthcare benefits for parents who refuse to vaccinate their children for non-medical reasons.

And the WHO is putting more money into regions with low vaccination coverage.

“We need to close these gaps, ensure that commitments for adequate human and financial resources are kept and used well to reach every child, detect and respond to every case of measles, and prevent further spread,” said Rebecca Martin, director of the US Centres for Disease Control and Prevention Centre for Global Health.

“These efforts will protect all children so that they can become the next generation of leaders.”

As a reminder, this is what measles really looks like:

enhanced-24237-1422308822-6Julien Harneis/Flickr

Let’s hope one day, all countries have the luxury of forgetting just how terrifying this disease really is.

You can read the full study over at Morbidity and Mortality Weekly Report.

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TB epidemic in India larger than what was previously estimated: WHO

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Tuberculosis epidemic in India is “larger” than what was previously estimated, the World Health Organisation (WHO) today said while asserting that the country was one of six nations which accounted for 60 per cent of the new cases in 2015.

The Global Tuberculosis Report 2016 which was released today, however, said the number of TB deaths and incidences rate continue to fall globally as well as in India.

“The TB epidemic is larger than previously estimated, reflecting new surveillance and survey data from India. However, the number of TB deaths and the TB incidence rate continue to fall globally and in India,” WHO said.

It said that in 2015, there were an estimated 10.4 million new (incident) TB cases worldwide, of which 5.9 million (56 per cent) were among men, 3.5 million (34 per cent) among women and 1.0 million (10 per cent) among children while people living with HIV accounted for 1.2 million (11 per cent) of all new TB cases.

“Six countries accounted for 60 per cent of the new cases – India, Indonesia, China, Nigeria, Pakistan and South Africa. Global progress depends on major advances in TB prevention and care in these countries,” the global health body said in its report.

It said worldwide, the rate of decline in TB incidence remained at only 1.5 per cent from 2014 to 2015 and stressed the need for it to accelerate to a 4–5 per cent annual decline by 2020 to reach the first milestones of the End TB Strategy.

“In 2015, there were an estimated 480000 new cases of multidrug-resistant TB (MDR-TB) and an additional 100000 people with rifampicin-resistant TB (RR-TB) who were also newly eligible for MDR-TB treatment.

“India, China and the Russian Federation accounted for 45 per cent of the combined total of 580000 cases. There were an estimated 1.4 million TB deaths in 2015, and an additional 0.4 million deaths resulting from TB disease among people living with HIV. Although the number of TB deaths fell by 22 per cent between 2000 and 2015, TB remained one of the top 10 causes of death worldwide in 2015,” it said.

WHO said that in 2015, 6.1 million new TB cases were notified to national authorities and reported to WHO while notified TB cases increased from 2013–2015, mostly due to a 34 per cent increase in notifications in India.

“However, globally there was a 4. 3 million gap between incident and notified cases, with India, Indonesia and Nigeria accounting for almost half of this gap. The crisis of MDR-TB detection and treatment continues.

“In 2015, of the estimated 580000 people newly eligible for MDR-TB treatment, only 125000 (20 per cent) were enrolled.

Five countries accounted for more than 60 per cent of the gap – India, China, the Russian Federation, Indonesia and Nigeria,” it said.

WHO said that the upward revisions to estimates of the burden of TB disease in India for the period 2000–2015 follow accumulating evidence that previous estimates were “too low”.

“This evidence includes household surveys, a state-wide TB prevalence survey, studies of anti-TB drug sales in the private sector, notification data and new analysis of mortality data.

“Since India accounts for more than one quarter of the world’s TB cases and deaths, these revisions have had a major impact on global estimates. Estimates for India are considered interim, pending a national TB prevalence survey scheduled for 2017/2018,” WHO said.

It said that in the African Region where the burden of HIV-associated TB is highest, 81 per cent of notified TB patients had a documented HIV test result.

“The proportion of known HIV-positive TB patients on ART was above 90 per cent in India, Kenya, Malawi, Mozambique, Namibia and Swaziland,” it said.

Giving out details about India, WHO said mortality (HIV+TB only) was 37 per thousand while incidence (HIV+TB only) was 113 per thousand.

Estimated TB incidence in India in 2015 in the age group of 0-14 years for both males and females was 255 per thousand.

Commenting on the report and the disease in the South East Asian Region which also includes India, WHO Regional Director (SEARO) Poonam Khetrapal Singh said TB remains a serious problem across the region and requires the fullest attention and strongest commitment of governments.

“As outlined in WHO’s new global report on TB, a number of countries in the region are among the world’s highest TB burden countries, while revised estimates based on increased case-reporting and enhanced surveillance show that the TB caseload is higher than previously projected.

“TB is the single largest cause of death of any infectious disease in the region, and remains responsible for incalculable suffering, premature mortality, impoverishment and foregone development,” she said.

She said though countries have been making efforts to end TB and the number of TB deaths and its incidence rate continues to fall, at the current trend the region would not be able to achieve the SDG targets.

“A newer and bolder approach is needed to bend the curve faster and sharper to achieve the global targets. This means intensifying measures to ensure early diagnosis and treatment, such as active case-finding and enhancing access to cutting-edge diagnostic tools.

“Adopting newer approaches of case diagnosis, community based treatment and treatment of latent infection. It means integrating TB programmes with existing health systems, thereby amplifying the effect these interventions have,” she said.

Singh also said that funds must be allocated accordingly, while political commitment must be fortified and both must occur at national and international levels.

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WHO to visit India – Asses Drug Regulatory Market for Vaccine Products

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In the run up to the World Health Organisation (WHO)’s National Regulatory Authority (NRA) Assessment of Vaccines in February 2017, a delegation of WHO is scheduled to visit India for conducting an observed audit in select vaccine manufacturing facilities from October 17 to 21, 2016 where observers from WHO shall assess the performance of the GMP inspectors of CDSCO and SLAs during the entire inspection process.

This will be a preparation visit which will decide the agenda of WHO delegation’s visit to be held in February next year. In February visit, WHO experts will take a final decision on whether National Regulatory Authority of India is capable to meet international standards for vaccine regulations.

“WHO will assess the overall capability of drug regulator that reflects how the vaccine manufacturers are being regulated in terms of safety, quality and effectiveness of vaccines produced in the country. The drug regulator is assessed for compliance against the revised WHO NRA indicators. In addition to the general framework for the system, the regulatory functions which will be evaluated include marketing authorization and licensing; post-marketing surveillance, including for adverse events following immunization; lot release by the national regulatory authority; laboratory access; regulatory inspections of manufacturing sites and distribution channels; and authorization and monitoring of clinical trials. National drug regulator capability is assessed by WHO every three years and the last assessment happened in 2013. It is due in 2016,” said Drug Controller General of India (DCGI) Dr GN Singh.

In the run up to WHO delegation’s visit this month, DCGI held a meeting with vaccine manufacturers on September 26 to educate them about quality management system including post marketing surveillance, GMP, GLP compliance etc. The visiting WHO team will assess certificates issued to Indian vaccine manufacturers by CDSCO to meet International criteria of WHO.

Thirty representatives from vaccine manufacturers including Cadila Pharmaceuticals, Chiron Behring Vaccines, Haffkine Bio Pharmaceuticals, Panacea Biotec, Serum Institute of India, Bharat Biotech, Indian Immunologicals, Shantha (Sanofi) Biotechnics, GreenSignal Bio Pharma, Biological E Limited attended the meet.

If the WHO team concludes that the NRA for vaccines is functional, Indian vaccine manufacturers will become eligible to apply for prequalification status of their products. Prequalification status indicates that a vaccine meets international standards. Prequalification status is mandatory for vaccine manufacturers exporting their products to other nations through United Nations procuring agencies.

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America becomes MEASLES FREE!

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More than 50 years since a vaccine became available, the World Health Organisation (WHO) has officially declared measles eliminated from the Americas – not just the US, but the land mass stretching from Canada to Chile, and all the countries in between.

It’s the first region in the world to have rid itself of all local cases of the viral disease, and a pretty huge deal for public health.

“This is a historic day for our region and indeed the world,” said Carissa Etienne, director of the Pan American Health Organisation, who made the announcement with the WHO.

“It is proof of the remarkable success that can be achieved when countries work together in solidarity towards a common goal.”

Of course, even though the disease has been eliminated within the Americas, people can still catch the virus in the region – there have been 54 cases this year in the US alone – but these infections all originate overseas and are brought in by travellers.

The last home-grown outbreak in the Americas was Venezuela in 2002. The US was declared free of measles way back in 2000.

Unfortunately, eliminating a viral disease from one region doesn’t mean we’ve eradicated it completely – that would be when an infectious disease has been removed from the entire planet.

To date, humans have only managed to successfully eradicate one disease from the planet. Thanks to the successful smallpox vaccine developed by Jonas Salk in 1796, the last case of smallpox was reported in 1977, and the disease was announced eradicated in 1980.

But we’re getting very close to eradicating a second disease, too. Guinea worm disease, caused by the parasitic Guinea worm, now only exists in four countries, with its territory shrinking thanks to water filtration devices.

None of this news means we can get slack with vaccinating though. Measles has only been eliminated through something known as ‘herd immunity‘ – which means that roughly 90 to 95 percent of a population is immunised against a disease, so random outbreaks won’t be able to take hold.

That level of protection safeguards the small percentage of people who can’t be vaccinated for health reasons, newborn babies, and those for whom vaccines aren’t effective.

Once immunisation rates drop below that herd immunity level, though, we see imported outbreaks become more sustained, and the disease spreading within local populations again – which happened in 2014 in California.

And given the fact that measles is still widespread in many parts of the world, it’s worth staying vigilant.

“I would like to emphasise that our work on this front is not yet done,” said Etienne. “We cannot become complacent with this achievement but must rather protect it carefully.”

So stay protected, and stay educated, but also take a minute if you’re in the Americas to give yourself a pat on the back for helping to eliminate a viral disease that still kills more than 100,000 people around the world each year.

Hopefully, one day, we’ll be able to announce that the disease is eradicated altogether. That’ll be a really good day.

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