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USFDA approves first drug to treat tardive dyskinesia

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The U.S. Food and Drug Administration approved Ingrezza (valbenazine) capsules to treat adults with tardive dyskinesia. This is the first drug approved by the FDA for this condition.

Tardive dyskinesia is a neurological disorder characterized by repetitive involuntary movements, usually of the jaw, lips and tongue, such as grimacing, sticking out the tongue and smacking the lips. Some affected people also experience involuntary movement of the extremities or difficulty breathing.

“Tardive dyskinesia can be disabling and can further stigmatize patients with mental illness,” said Mitchell Mathis, M.D., director of the Division of Psychiatry Products in the FDA’s Center for Drug Evaluation and Research. “Approving the first drug for the treatment of tardive dyskinesia is an important advance for patients suffering with this condition.”

Tardive dyskinesia is a serious side effect sometimes seen in patients who have been treated with antipsychotic medications, especially the older medications, for long periods to treat chronic conditions, such as schizophrenia and bipolar disorder. Tardive dyskinesia can also occur in patients taking antipsychotic medications for depression and certain medications for gastrointestinal disorders and other conditions. It is unclear why some people who take these medications develop tardive dyskinesia yet others do not.

The efficacy of Ingrezza was shown in a clinical trial of 234 participants that compared Ingrezza to placebo. After six weeks, participants who received Ingrezza had improvement in the severity of abnormal involuntary movements compared to those who received placebo.

Ingrezza may cause serious side effects including sleepiness and heart rhythm problems (QT prolongation). Its use should be avoided in patients with congenital long QT syndrome or with abnormal heartbeats associated with a prolonged QT interval. Those taking Ingrezza should not drive or operate heavy machinery or do other dangerous activities until it is known how the drug affects them.

The FDA granted this application Fast Track, Priority Review and Breakthrough Therapy designations.

The FDA granted approval of Ingrezza to Neurocrine Biosciences, Inc.

In the Future, Cells Reprogrammed Inside Our Bodies Will Fight Cancer for Us

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The Emperor of All Maladies

Cancer is when an aberrant mutation in a cell leads it to prolifically divide, causing abnormal cell growth that can potentially spread to other parts of the body if untreated. Cancer is the second leading cause of death globally, with one in every six deaths caused by a type of cancer in 2015, leading to 8.8 million deaths.

Because different mutations can cause cancer, and cancerous cells can develop in many different parts of the body, doctors are continually coming up with different strategies for treating the disease. With each passing year, research on the topic continues to progress. From personalized vaccines to mecha-suit sperm, we’ve been looking in every possible nook and cranny in hopes of finding an effective therapy that can work better than what we have today.

Currently, we use a host of methods to treat cancer, including surgery, radiation therapy, chemotherapy, immunotherapy, targeted therapy, hormone therapy, stem cell transplant, and precision medicine. While some cancer patients receive only one treatment, these treatments are normally used in conjunction to increase the rate of success.

Since the 1960s, our efforts in tackling cancer have progressed significantly. Patients back in the day had a five-year survival rate of around 50 percent. With the advent of these new therapies, some the most commonly diagnosed cancers in the U.S. have 5-year-survival rates at around 75 percent.

Many are hopeful that these numbers will soon improve again, with studies concluding that, of all new cancer therapies that make it into randomized controlled trials, at least 25 to 50 percent will improve current therapies. In fact, this might be the case for a new potential cancer treatment that uses nanoparticles.

Nanoparticles to The Rescue

The next step in cancer therapies might be quietly waiting for its time in the limelight in Seattle, Washington, at the Fred Hutchinson Cancer Research Center, where scientists have constructed biodegradable nanoparticles that can genetically program immune cells while inside the body to target cancer cells. The study was published on April 17 in Nature Nanotechnology and focused on the effect that nanoparticle-programmed immune T cells had on leukemia in mouse models.

The nanoparticles carried genes that code for chimeric antigen receptors (CARs), which are proteins designed by scientists to help immune cells target and destroy cancer. Once the immune cells undergo this molecular modification, they turn into an army of cancer serial killers.

*4* In the Future, We’ll Fight Cancer by Reprogramming Our Cells

This new method can eliminate expensive and time-consuming steps that lag previous T cell cancer therapies. The current protocol is that the T cells are removed from the patient, genetically altered, regrown, and infused back into the patient. The biodegradable nanoparticles will eliminate the removal, regrowth, and infusion steps by accomplishing the reprogramming step over a time span of 24 to 48 hours while the T cells are in the body.

When the researchers compared the nanoparticle-based method to current immunotherapy methods that require the T cells to be removed, researchers noticed that leukemia-induced mice lived an additional 58 days on average when compared to the mice that received the current treatment.

While these results are exciting, the researchers are looking to make the process safer before they move into human trials. But if this new technique is approved for humans, it could have many more applications. Scientists are looking to adapt the method for diseases like hepatitis, HIV, or even solid tumors.

By quickly arming patients’ immune cells to fed off disease, this new treatment could lower healthcare costs and improve the quality of patients’ lives.

CDSCO to audit manufacturing units globally

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As a step towards ensuring that India retains its identity as the pharmacy of the world, the Central Drugs Standard Control Organisation (CDSCO) is planning to audit manufacturing units in both developed and developing markets globally to ensure that India gets quality drugs and ingredients from the importing countries like the US, Europe and China.

“The audits on manufacturing units are not restricted to developed economies alone as CDSCO plans to expand it to other markets from where India gets medicines to ensure drug quality and compliance,”  informed Drug Controller General of India (DCGI) Dr G N Singh.

At the same time CDSCO, as part of its larger commitment towards harmonisation of global regulatory standards, is on its way to increase audits in the manufacturing units across the country towards compliance with GMP as per the requirements of the respective regulated markets.

This comes at a time when Department of Pharmaceuticals (DoP) is also working on Pharmaceutical Technology Upgradation Assistance (PTUA) scheme for specific manufacturing units in the country. PTUA envisages support to medium scale manufacturing units in bulk drugs and formulation manufacturing sectors for their upgradation from Schedule M compliance to WHO GMP compliance.

According to industry leaders, PTUAS should not be restricted to simply upgradation to WHO-GMP as aspiring industries are willing to upgrade their current WHO-GMP facility and also for advancement to EDQM, MHRA, US FDA and other regulated markets to boost exports.

Regulators globally during their audit visits at Indian sites have issued in total 19 Form 483 last year as part of their observations on data integrity. Several Indian drug makers have come under the scanner of the global drug regulatory authorities in the recent years over a range of issues like data integrity, including production quality, sanitation standards and alleged data manipulation.

Any product which does not comply with good manufacturing practices of overseas markets is being considered as adulterated and hence has the chance of losing the market, a senior CDSCO official said emphasizing the purpose of the inspection in the interest of patient safety at large.

GMPs is based on a criteria involving factors such as sanitation and hygiene, qualification and validation, self-inspection, quality audits, suppliers’ audits, prevention of cross-contamination and bacterial contamination during production, training employees and personnel. Global GMP violations have hurt India’s image as an inexpensive and reliable supplier of generic drugs in international markets.

Over the past decade, pharma industry has invested heavily in upgrading its manufacturing plants to match international standards. Approximately 1400 manufacturing units in India are WHO GMP certified, and over 800 are UK MHRA approved. India continues to have the highest number of US FDA registered manufacturing facilities outside the US. The industry has strong capabilities in product development. Also, Indian pharmaceutical companies have the largest share of DMFs and ANDAs outside the US.

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Experimental Nasal Spray Could Deliver Drugs Across the Blood-Brain Barrier

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Life-saving medicines could soon be sent directly to the brain with just a sniff, thanks to a new nasal spray that uses gold nanoparticles to cross through the blood-brain barrier.

This protective barrier keeps our brains safe from damaging toxins, but also makes it hard for beneficial drugs to be effective – so this nasal mode of delivery could enable all kinds of new treatments.

The spray has been developed by a team from Washington University in St. Louis, who tested it on the antennae of locusts – insects with blood-brain barriers and olfactory networks that are anatomically similar to those in humans.

Based on these tests, the nasal spray method could deliver drugs to the brain in as little as 30 to 60 minutes, according to researchers.

“The shortest and possibly the easiest path to the brain is through your nose,” says one of the researchers, Barani Raman.

“Your nose, the olfactory bulb and then olfactory cortex: two relays and you’ve reached the cortex.”

nasal-spray-2WUSTL

The researchers developed an aerosol spray made up of gold nanoparticles small enough to pass through the blood-brain barrier. Fluorescent markers were added to track the movement of the nanoparticles.

After exposing locusts to the spray, the nanoparticles moved through the antennas, olfactory nerves, and blood-brain barrier in just a few minutes, before spreading through the brain.

What’s more, no noticeable changes were noticed in the olfactory neurons of the locusts after the treatment, suggesting minimal disruption to brain function.

The next stages are to adapt the technique so the gold nanoparticles can carry different types of medicines – and of course to see if the same approach could work with humans in addition to locusts.

If it does, it could be our best option yet for drug delivery to the brain. Pills aren’t precise and the medications in them struggle to get through the blood-brain barrier, while injections into the brain are invasive and risk damaging tissue.

The nasal spray is the latest example of how nanotechnology could help improve our health: whether it’s fighting cancer or stopping allergies, we’re seeing tiny nanoparticles show a lot of potential for treating areas of the body that other drugs struggle to reach.

Thanks to their microscopic size, nanoparticles can be very carefully targeted – as in the case of chemotherapy drugs designed to hit cancer without affecting healthy tissue.

The scientists behind this new study suggest their work could lead to improved treatments for several health problems, including brain tumours, which could be targeted using a combination of a nasal spray and ultrasound.

“This is only a beginning of a cool set of studies that can be performed to make nanoparticle-based drug delivery approaches more principled,” says Raman.

The findings have been published in Scientific Reports.

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DBT soon to begin research on monogenic disorders

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With an aim to control the onset of various monogenic disorders such as the metabolic disorders, endocrine disorders, hemoglobin disorders and others, the Department of Biotechnology (DBT) will soon begin research on monogenic disorders.

With this aim, the DBT has now invited investigators working in these areas to submit concept notes in collaboration with clinicians for the molecular analysis of the most prevalent monogenic disorder from different zones of the country viz. North-Eastern zone, Eastern Zone, Central zone, Northern zone, North-Western zone, Western zone, South-Western zone, Southern zone, South-Eastern zone and Andaman-Nicobar Island zone.

For this programme, Concept notes will only help in identifying investigators (both clinicians and scientists) who have relevant expertise in molecular analysis of monogenic disorders. A networking meeting of all the selected participants would then be facilitated by DBT to develop multi-approach, multi-disciplinary programme towards an identified goal with both short term and long term objectives clearly defining the scientific approaches.

Scientists working in Universities/Academic Institutions/National Laboratories/Industries [Department of Scientific & Industrial Research (DSIR)-Recognized R&D Centre] & Non-Profit Organizations with necessary facilities and strong scientific background in the proposed area are eligible to participate in this prgramme. Collaborative projects with clinicians would only be considered for funding.

The DBT’s initiative in this regard is significant as monogenic disorders are an important cause of neonatal mortality both in developed and developing countries. Millions suffer from inherited genetic diseases and the one-sixth of the world’s population living in India has never been systematically studied. It is not only a leading cause of fetal loss, but also contributes significantly to preterm birth, childhood and adult morbidity along with considerable repercussion on the mothers and their families. Worldwide surveys have shown the birth prevalence of monogenic disorders varies due to social, racial, economical and ecological influences. In order to control the onset of various monogenic disorders (metabolic disorders, endocrine disorders, hemoglobin disorders and others), it is pertinent to understand the epidemiology and molecular basis of these disorders.

Funding priority will be placed on those projects that will lead to a better understanding of molecular mechanism of the most important inherited single-gene disorder prevalent in the particular zone with the listing of overall prevalence of other monogenic disorders in the concerned zone. Individual project should represent all the regions of the concerned zone.

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