The U.S. Food and Drug Administration granted accelerated approval to Aliqopa (copanlisib) for the treatment of adults with relapsed follicular lymphoma who have received at least two prior treatments known as systemic therapies.
“For patients with relapsed follicular lymphoma, the cancer often comes back even after multiple treatments,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Options are limited for these patients and today’s approval provides an additional choice for treatment, filling an unmet need for them.”
Follicular lymphoma is a slow-growing type of non-Hodgkin lymphoma, a cancer of the lymph system. The lymph system is part of the body’s immune system and is made up of lymph tissue, lymph nodes, the spleen, thymus, tonsils and bone marrow. The National Cancer Institute at the National Insititutes of Health estimates that approximately 72,240 people in the United States will be diagnosed with some form of non-Hodgkin lymphoma this year; approximately 20,140 patients with non-Hodgkin lymphoma will die from the disease in 2017.
Aliqopa is a kinase inhibitor that works by blocking several enzymes that promote cell growth.
Aliqopa received an Accelerated Approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need using clinical trial data that is thought to predict a clinical benefit to patients. Further clinical trials are required to confirm Aliqopa’s clinical benefit and the sponsor is currently conducting these studies.
Today’s approval of Aliqopa was based on data from a single-arm trial that included 104 patients with follicular B-cell non-Hodgkin lymphoma who had relapsed disease following at least two prior treatments. The trial measured how many patients experienced complete or partial shrinkage of their tumors after treatment (overall response rate). In the trial, 59 percent of patients had a complete or partial response for a median 12.2 months.
Common side effects of Aliqopa include high blood sugar levels (hyperglycemia), diarrhea, decreased general strength and energy, high blood pressure (hypertension), low levels of certain white blood cells (leukopenia, neutropenia), nausea, lower respiratory tract infections, and low levels of blood platelets (thrombocytopenia).
Serious side effects include infections, high blood sugar levels (hyperglycemia), high blood pressure (hypertension), inflammation of the lung tissue (non-infectious pneumonitis), low levels of certain white blood cells (neutropenia), and severe skin reactions. Women who are pregnant or breastfeeding should not take Aliqopa because it may cause harm to a developing fetus or newborn baby.
Aliqopa was granted Priority Review designation, under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition.
Aliqopa also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The FDA granted the approval of Aliqopa to Bayer Healthcare Pharmaceuticals, Inc
A novel form of fabrication on a microscale level could mean we could one day receive a single vaccination that wouldn’t require ongoing boosters.
Researchers have recently constructed tiny containers from a polymer that can be set to degrade inside the body at a specific time, allowing a drug or vaccine to be delivered in intervals over an extended period.
Described by their inventors as “tiny coffee cups“, the 400 micrometre pods are made from poly lactic-co-glycolic acid (PLGA), a polymer already approved by the FDA as a potential drug-delivery material.
MIT engineers developed the production technique as part of a project funded by the Bill and Melinda Gates foundation with the aim of addressing the problem of vaccinating people in parts of the world where it’s hard to deliver regular medical attention.
“This could have a significant impact on patients everywhere, especially in the developing world where patient compliance is particularly poor,” says senior research Robert Langer from MIT.
Using PLGA to deliver controlled releases of medication isn’t new.
Most existing technologies revolve around the particles disintegrating slowly, freeing the pharmaceuticals embedded in the polymer’s matrix.
But by filling small containers with a substance, larger doses can be released in one hit.
The engineers developed a fabrication method called stamped assembly of polymer layers (or SEAL if you like your acronyms), which is more or less like making tiny soft-centre chocolates in the world’s smallest silicon moulds.
In principle the technique could work with just about any thermoplastic material, opening the way to developing microstructures of just about any complex shape.
Once the cup has been moulded and filled, a tiny lid is added and heat-sealed in place.
Tweaking the properties of the PLGA changes the time it takes for the cup to fall apart, meaning it’s possible to create a variety of containers that disintegrate at different stages on cue.
“We are very excited about this work because, for the first time, we can create a library of tiny, encased vaccine particles, each programmed to release at a precise, predictable time, so that people could potentially receive a single injection that, in effect, would have multiple boosters already built into it,” says Langer.
The researchers showed in mice the particles successfully held their contents without leaking, and then released them in four stages between 9 and 41 days after injection.
Technically this could have practical uses in any process that requires the delivery of a substance in measured doses over a long period of time.
“The SEAL technique could provide a new platform that can create nearly any tiny, fillable object with nearly any material, which could provide unprecedented opportunities in manufacturing in medicine and other areas,” says Langer.
But the big win would be in vaccines that require more than one hit.
Vaccinations work by exposing the body to chemical samples of a disease agent called antigens, allowing specialised white blood cells to make the biochemical equivalent of a mug-shot.
Unfortunately those tiny antigen wanted posters get lost over time as immune cells die, requiring regular reminders.
For pathogens such as tetanus, a booster vaccination is required roughly every decade.
Keeping up to date with regular booster vaccinations might be sound medical advice, but it’s not something we all make time for.
In some of the poorer parts of the world it also isn’t practical, where vaccines can spoil without adequate storage and experienced medical staff are harder to come by.
The challenge now is to prolong the life of these particles to last years rather than days or months.
The team have produced particles that could feasibly disintegrate after 100 days. But they still have a way to go.
It’s an exciting first step in any case, one that could make a world of difference where preventable diseases claim too many lives every year, all for want of a simple jab.
This research was published in Science.
In order to arrive at a policy for regulating online pharmacy in the country in an effective way, the Union health ministry is currently examining technical feasibility and modalities involved in it. This is in line with the government’s much awaited plan to launch a centralised portal which will make use of information technology to make medicines available in a transparent manner across the country. The move follows Drug Consultative Committee’s (DCC) acceptance of recommendations by a select group of state drug regulators on online pharmacy.
“Government is examining technical aspects related to creating an online platform to be able to facilitate online sale of medicines in a transparent and regulated manner. The sole intention is to make best use of information technology for such kind of online systems,” says Drug Controller General of India Dr G N Singh.
The online portal system which is currently being developed by Central Drugs Standard Control Organisation (CDSCO) in consultation with Department of Pharmaceuticals (DoP), Law Ministry and Commerce Ministry will involve manufacturers and supply chain managers.
DCC had last year deliberated on aspects related to information technology which can be aligned with the drug regulations existing currently to suit patient needs among other major points.
Based on the recommendations and agreement with all the concerned stakeholders, the union health ministry is currently on the final phase to frame a policy on the use of information technology in online pharmacy.
Guidelines on the same were also submitted last year by the sub-committee set up by the 48th DCC under the chairmanship of former Maharashtra FDA Commissioner Dr Harshdeep Kamble.
The sub-committee had taken representations and views from the concerned stakeholders for a comprehensive online policy and subsequent release of a set of guidelines on the use of information technology in online pharmacy and authorise its legal validity.
Online pharmacy is currently governed by Information Technology (IT) Act, 2000 and Drugs and Cosmetics Act, 1940 but recommendations from all stakeholder had been sought on the broad contours of a policy that will also ensure a level playing field for online pharmacies vis-a-vis traditional retailers.
DCC met on November 6, 2016 and accepted the much awaited recommendations of a select group of state drug regulators on online pharmacy under the chairmanship of former Maharashtra Food and Drug Administration (FDA) Commissioner Dr Harshadeep Kamble.
News on the emergence of the mosquito-borne Zika virus in recent years has been full of heart-breaking images of infants born with a confronting neurological disorder caused by the pathogen’s effects on certain stem cells.
Now researchers are proposing a daring use for the virus’s talent for destroying these kinds of brain tissues; to turn it loose on an aggressive form of cancer called a glioblastoma.
The study, conducted by scientists from Washington University School of Medicine and the University of California, San Diego, has demonstrated Zika virus can preferentially infect and kill glioblastoma stem cells cultured outside the body.
In addition, mice which had an adapted strain of the virus injected into their tumours lived longer as a result.
Both sets of results provide some hope of a novel form of treatment for a highly malignant type of cancer that is difficult to destroy.
Glioblastomas form as the brain’s so-called ‘glue cells’, or glia, grow out of control. The tumours that result are usually removed surgically, with chemotherapy and radiotherapy prescribed to mop up any leftover cancerous cells.
Unfortunately their branching structures and the diverse types of cells that make up the tumours make these cancers difficult to treat.
Less than a third of patients with more aggressive forms of the disease live to see another two years, making it a rather bleak outlook for those receiving the diagnosis.
This new approach to treating glioblastoma makes use of the properties of a microbe that has a reputation for causing a very different kind of disease.
Surprisingly, this isn’t the first instance of a pathogen being commissioned to fight this kind of brain tumour.
Earlier this year researchers reported their success in treating glioblastomas in rats with engineered strains of the bacterium Salmonella.
Zika virus has been on epidemiologist’s radar since the mid-20th century, but first rose to global notoriety in 2015 following an outbreak in Brazil, where it was blamed for a significant rise in newborns with a condition called microcephaly.
The most striking feature of this disorder is the lack of brain growth that gives rise to a smaller skull, developmental delays, feeding difficulties, and hearing loss.
In adults the virus is usually produces symptoms that are far less severe, limited to fever, rash, headache, joint pain, and in some rare cases, inflammation of the nervous system’s covering, or meninges.
Recent research on the Asian lineage of the virus responsible for the 2015 outbreak found it stopped the growth of stem cell tissues in the brain and prevented them from turning into nerve cells.
This preference for interfering with the development of certain lines of stem cells is disastrous for developing embryos, but it could also be the perfect silver bullet to polish off glioblastomas.
“We showed that Zika virus can kill the kind of glioblastoma cells that tend to be resistant to current treatments and lead to death,” says researcher Michael S. Diamond from Washington University School of Medicine.
Chemotherapy and surgery can remove the bulk of the cancerous tissue, but tends to leave stray stem cells that continue to proliferate and give rise to a new mass near the previous tumour site.
Adding the virus to the mix could be the perfect one-two-three punch for cleaning up the scraps.
“We see Zika one day being used in combination with current therapies to eradicate the whole tumour,” says researcher Milan G. Chheda, also from Washington University School of Medicine.
The team tested the virus on 18 mice with the equivalent of glioblastomas, comparing them against 15 that were injected with a saltwater control. Two weeks after treatment, the tumours were significantly smaller in the Zika-infected mice.
You can be forgiven for any apprehensions over injecting a virus known for causing brain damage directly into patients’ brains.
But while there is plenty of research to be done before the virus could even be considered a viable option for cancer treatment, the fact it only targets cells that are rare in adult nervous systems makes them a relatively safe bet.
The researchers also induced mutations that weakened the virus’s ability to defend itself against the immune system. While it also made it less potent, the virus still managed to kill stem cells.
“We’re going to introduce additional mutations to sensitise the virus even more to the innate immune response and prevent the infection from spreading,” says Diamond.
It’s certainly a somewhat unconventional approach to treating brain cancer. But then again, glioblastoma isn’t your average cancer.
This research was published in the Journal of Experimental Medicine.
Aiming to advance research and provide innovations in various issues related to ophthalmic disorders, the Department of Biotechnology (DBT) will soon begin research on nano-intervention in management of ophthalmic diseases.
DBT’s initiative to begin research on nano-intervention in management of ophthalmic diseases holds significance as nanobiotechnology holds considerable promise to address various issues related to ophthalmic disorders such as glaucoma, diabetic retinopathy, age related macular degeneration and other such diseases especially those affecting posterior segment of an eye. Nano enabled system and devices can find novel therapeutic solutions for crossing blood retina barrier and make sustained availability of drugs in desired therapeutic concertation in different compartments of eye.
The DBT has invited proposals built upon innovative ideas in the areas of “Nano Enabled System and Devices for Ocular diseases” by undertaking studies including but not limited to the two areas. First, a design and development of nano-enabled system for delivery of drugs in chronic diseases of eye for sustained delivery of drugs for chronic conditions–age related Macular Degeneration, Diabetic Retinopathy, Glaucoma; tissue specific targeting of drugs in ciliary body, trabecular network, bruch’s membrane/retinal pigment epithelial complex (sealing agents); crossing blood retina barrier; non-invasive nano formulations of existing drugs; delivery of gene/s for child blindness like Leber Congenital Amaurosis and Retinitis Pigmentosa; and cell based therapy/regeneration.
Second, design and development of nano-enabled novel implant system for sustain release of Steroid; sustain release of non-steroidal anti inflammatory agents; anti-angiogenic implant with conventional (ANTI-VEGF) and novel targets; microneedle based systems/refillable drug delivery system; anterior segment implant ; microenvironment responsive/tunable drug release system; and post-surgery biodegradable artificial vitreous substitutes.
Interested applicants can submit full proposal in DBT format online through e-Promis http://www.dbtepromis.nic.in before 15th September, 2017.