FDA approves new treatment that uses engineered genes to treat aggressive leukemia, adding a ‘superweapon’ to the existing armamentarium against cancer


The Food and Drug Administration (FDA) agency has recently approved a revolutionary new treatment for one of the most aggressive forms of leukemia, effectively implementing the first ever gene-therapy to treat cancer in the United States.

The brand new treatment is a Chimeric Antigen Receptor (CAR) T-cell therapy created by Swiss pharma company Novartis and commercialized as Kymriah. The therapy has been approved to treat pediatric acute lymphoblastic leukemia (ALL) in patients up to the age of 25 with B-cell precursor ALL that is refractory or in second or later relapse.

ALL is a type of aggressive cancer of the white blood cells and bone marrow. The disease progresses rapidly, causing overproduction of immature white blood cells (lymphocytes), which inhibits the production of mature cells. Death will occur without quick treatment, but complete remission in children is also a typical outcome.

Kymriah uses genetically-modified cells to target a specific cancer cells in the receptor.

The process involves retrieving cells from the patient and sending them to a facility where they are genetically altered to include a new protein (CAR). The modified cells are then sent back and injected into the patient. The new cells stimulate the receptor’s immune system to target specific leukemia cells that contain the CD19 antigen.
Each single treatment is effectively ‘customized’ to every patient. Kymriah has shown astonishingly positive results in clinical trials, where 83 percent of patients treated with it achieved remission within three months.

Kymriah is not without its downsides. Its cost is rather high at present, for example. A single treatment costs $475,000, which may seem incredibly high, but it’s actually well below market expectations of around $700,000 per vial. And that price tag is for the treatment alone, it does not include hospitalization costs, or any other associated monetary outlays. On this regard, Novartis is working with health centres that provide Medicaid and Medicare facilities to iron out financial arrangements for those patients who undergo this treatment.

The new therapy may also cause severe neurological side effects in some cohorts, and the activation of CAR cells in the receptor may trigger a cytokine release syndrome (CRS). Both side effects may be fatal, but are treatable.

Despite these issues, Kymriah has been hailed as a quantum leap forward in the fight against cancer, particularly in the treatment of ALL. Scientists are now open to new avenues of research, and may consider applying similar therapies for the treatment of other types of leukemias and solid tumours.

Scientists develop new process to induce death of cancer cells


The fight against cancer has scored a major victory today, after researchers develop a brand new process to induce the death of cancerous cells.

The new method, known as Caspase Independent Cell Death (CICD), has achieved total eradication of tumours in experimental models.

Current standard treatments for cancer patients include chemotherapy and radiotherapy, which kill off cancer cells via apoptosis.

Apoptosis is a sort of ‘programmed cell death’, where a cell is effectively induced to kill itself. This process involves proteins called caspases, which kick off the apoptosis process by breaking down the essential components needed for cell survival. The cells shrink, and as they do, they send out distress signals which are picked up by the human immune system. Macrophages (white blood cells) are then dispatched to consume the dying cell, essentially cleaning up the body. Apoptosis is often neat and leads no trace of the cell.

Despite its efficacy, apoptosis often fails to kill off all targeted cells, and crucially, the remaining cancerous cells fail to trigger an immune response, which is the reason why some types of cancer tend to reoccur.

CICD triggers cell death in such a way that the dying cell alerts the human immune system via the release of inflammatory proteins. The body responds and kills off the cancerous cells that escaped treatment.

CICD has shown great potential by inducing complete tumour regression in experimental models, and the results suggest new ways of treating cancer more effectively in the near future.

New weapon in oncology armamentarium against ovarian cancer deemed ‘biggest breakthrough in a decade’

A new experimental drug has shown extremely promising results in the treatment of metastasized ovarian cancer, with trial doctors going as far as saying that it is the ‘biggest breakthrough ten years.’

Ovarian cancer may cause few or no symptoms when it first develops, so it is commonly detected at an already advanced stage, making treatment options difficult. About a fifth of cases actually present with distant metastases, with most of these being terminal and requiring supportive or palliative care. Ovarian cancer kills an average of 4,000 people a year in the UK alone.

A new drug, ONX-0801, is currently being tested in a phase one clinical trial conducted at the Royal Marsden cancer hospital in London. The compound has shown extremely positive results so far, after seven out of fifteen women who were administered the trial drug experienced substantial tumour shrinkage.

Notably, ONX-0801 was only being tested for safety, but the unexpectedly beneficial therapeutic results encouraged the investigators to quickly move to further trials.

ONX-0801 was administered to women who had poor or negligible therapeutic response to standard chemotherapy treatment.

The drug is an alpha-folate receptor (aFR)-mediated inhibitor of thymidylate synthase. Administered intravenously, it selectively targets and binds to tumour cells where aFR expression is higher. Healthy cells remain relatively unaffected as aFR expression is significantly lower. Once bound, ONX-0801 inhibits both DNA synthesis and cell division, inducing cell apoptosis (death of the cell.)

Though initial results regarding the therapeutic outcomes of ONX-0801, further research and trials are needed to confirm its viability in the fight against ovarian cancer.

Irish research team leads the way in possible breakthrough in the fight against aggressive breast cancer


An Irish research team, Breast-Predict, is confident that it has achieved a breakthrough in the fight against Triple-negative Breast Cancer (TNBC). The team is based in St. Vincent’s Hospital in Dublin.

TNBC accounts for about 15% of all breast cancer diagnoses, but has the highest mortality rate due to a lack of truly effective treatment.

The team believes that compound APR-246 can be used to treat TNBC effectively.

TNBC differs from other subtypes in that it does not express estrogen receptor (ER), progesterone receptor (PR) or the amplification of Her2/neu. Since most chemotherapy drugs target one of these three molecular markers, the medical armamentarium in the fight against aggressive breast cancer is severely limited.

Patients diagnosed with TNBC usually undergo chemotherapy, but the disease does not respond well to treatment in many cases, since the targeted receptors are missing. As a result, most patients face a poor outcome.

Crucially, the vast majority of TNBC cases feature a mutated P53 gene, which makes it a target of interest for treatment.

A mutation in P53 renders it ineffective in enabling the DNA damage response pathway, which allows the survival of incipient tumour cells.

APR-246’s mechanism of action targets the aberrant P53 gene, ‘correcting’ its mutation and thus inhibiting tumour progression.

The compound will undergo clinical trials to determine its long-term viability.

Dr Robert O’Connor, Head of Research at the Irish Cancer Society, has welcomed the development and said that “These research programmes focus on finding new ways to prevent as many cancers as we can, ensuring the most advanced personalised treatment options are available and that as many patients as possible thrive after their treatment.”

“The number of people with cancer in Ireland is expected to double by 2040, and more research is vital if to tackle this growing epidemic of cancer.”

Recent clinical trials reveal groundbreaking possibilities for successful cancer treatment


An experimental combination therapy recently put to the test in human clinical trials has shown outstanding results in progression-free survival rates for terminally ill cancer patients.

Current chemotherapy drugs may buy a terminally ill patient a few months to live, at best, and in many cases mere weeks.

But recent trials have tested immunotherapy drugs that have proved strikingly effective against aggressive forms of melanoma and lung cancer.

Immunotherapy has been hailed as the most exciting development in cancer treatment in recent times, and it has been postulated that it will replace chemotherapy as standard treatment in the near future.

Immunotherapy works by boosting the human body’s own immune system to fight disease, and though it is commonly used in other therapeutic areas, cancer research made little use of it until now.

Recent trials were conducted on 945 patients with advanced melanoma. They were treated with a combination therapy of the drugs ipilimumab and nivolumab, and at the end of the trial, the therapy achieved a tumour reduction of over 50%, which is an outstanding result.

Dr Alan Worsley, Cancer Research UK’s senior science information officer, has said: “This research suggests that we could give a powerful one-two punch against advanced melanoma by combining immunotherapy treatments.

“Together these drugs could release the brakes on the immune system while blocking cancer’s ability to hide from it.

“But combining these treatments also increases the likelihood of potentially quite severe side effects. Identifying which patients are most likely to benefit will be key to bringing our best weapons to bear against the disease.”

The trials have so far been restricted to two types of cancer, but these therapies may soon be extended to other common cancers.

It has been claimed that the evidence in favor of this groundbreaking new treatment is so overwhelmingly positive that tens of thousands of lives may be saved in the UK alone within a decade.