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FDA grants accelerated approval to fam-trastuzumab deruxtecan-nxki for HER2-mutant non-small cell lung cancer

On August 11, 2022, the Food and Drug Administration granted accelerated approval to fam-trastuzumab deruxtecan-nxki (Enhertu, Daiichi Sankyo, Inc.) for adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating human epidermal growth factor receptor 2 HER2 (ERBB2) mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy. This is the first drug approved for HER2-mutant NSCLC.

One Step Forward to the human’s dream: Youngness Elixir

For a long time, children with the ultrarare genetic disease progeria, which causes rapid aging, didn’t have an FDA-approved therapy. But that’s about to change.

Friday, the FDA approved Zokinvy, or lonafarnib, to treat Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies in patients ages 1 and above.
Zokinvy isn’t a cure. The drug, a farnesyltransferase inhibitor, targets the toxic buildup of progerin or progerin-like farnesylated proteins.
you may think that this found is not very important due to the rare of the disease but if this drug works to prevent rapid aging in children it may prevent rapid aging for all. which was always humans dream: youngness elixir!

Party drug’ could help to treat PTSD

Post-traumatic stress disorder (PTSD), as the name suggests, is an anxiety disorder that occurs following a major trauma.

Affecting up to 17.1 percent of veterans and up to one third of first responders, the symptoms include intrusive memories of said stressful event, flashbacks, nightmares, and intense distress.

To date, finding effective treatments for PTSD has proven difficult; psychotherapy and other talking therapies are normally the first port of call, but they do not work for everyone.

Medications such as antidepressants and antipsychotics are typically used, but, again, they do not work for everyone, and side effects can be significant.

Though researchers are keenly investigating ways to improve treatment, running effective studies can also be challenging; there are often high drop-out rates.

Often this is due to the nature of PTSD’s symptoms, but sometimes it is because the condition has worsened, and perhaps the person has been hospitalized.

Also, some believe that patient characteristics might play a role, as might homelessness and drug use disorders.

These difficulties are mirrored in real-life situations; people with PTSD often drop out of therapy, limiting how useful it can be.


In an attempt to get around these problems, scientists are approaching the treatment of PTSD from innovative directions. Recently, a study funded by the Multidisciplinary Association for Psychedelic Studies in Santa Cruz, CA, investigated the potential use of MDMA — the active ingredient in the controversial party drug, ecstasy.

This might, at first, seem to be an odd choice, but this is not the first time that MDMA has been used in this way.

MDMA was synthesized for the first time in 1912 by scientists looking for drugs to stop bleeding, but no significant use was made of it for many years. But, from the 1970s onward, MDMA was tested for use in depression, relationship problems, substance abuse, premenstrual syndrome, and autism, among others.

The latest study involved just 26 service personnel (22 veterans, three firefighters, and one police officer). All had experienced a traumatic experience and developed PTSD no less than 6 months earlier. All participants had failed to respond to earlier medical or psychological treatments.

They were split into three treatment groups, each receiving different quantities of MDMA: 30 milligrams, 75 milligrams, or 125 milligrams. There was no placebo group.

The researchers wanted to explore whether taking MDMA alongside psychotherapy could increase its effectiveness. Because the drop-out rates from therapy are so high for individuals with PTSD, maximizing every session is key.

Initially, the participants went through three 90-minute psychotherapy sessions without MDMA, so that the therapists could establish a working relationship and prepare them for the experience. Then, they received the MDMA dose during an 8-hour session of tailored psychotherapy.

After the experimental session, the participants stayed the night and were followed for 7 days by telephone contact. Lastly, there was a final 90-minute psychotherapy session. The findings were recently published in The Lancet Psychiatry.

Positive early findings

Although this was a small-scale study with no placebo group, the results offer hope. One month after the final session, 58 percent of the participants in the 125-milligram group no longer met the criteria to be classed as having PTSD, compared with 86 percent in the 75-milligram group and 29 percent in the 30-milligram group.

These findings will, of course, need to be confirmed in large phase III studies. It is also worth noting that more than three quarters of the participants relayed adverse events — including, most frequently, “anxiety, headache, fatigue, muscle tension, and insomnia.”

The researchers believe that, in the right setting, MDMA could be useful in the treatment of PTSD.

[O]ur study suggests that MDMA might help augment the psychotherapeutic experiences and may have a role to play in the future treatment of PTSD.

  • Study author Dr. Allison Feduccia

But, Dr. Feduccia is quick to remind us that this must be conducted under strict supervision. “[W]e would certainly not recommend that individuals try these drugs for the treatment of psychiatric disorders without the support from trained psychotherapists.”

This project builds on earlier studies, including one that compared MDMA-assisted psychotherapy in 12 participants with eight people in a placebo group, and another that compared two doses of MDMA using 12 participants.

In total, six phase II trials provided positive results, which led the Food and Drug Administration (FDA) to declare MDMA-assisted psychotherapy as a “Breakthrough Therapy.”

While this title does not necessarily mean that there is high-quality evidence to back up MDMA’s efficacy, it does mean that the FDA will give priority to MDMA research.

This means that, hopefully, more large-scale, in-depth studies will follow soon, finally confirming whether MDMA truly can help in the treatment of PTSD.

Colorectal cancer: Treatment looks set for human clinical trials

In a study paper published in the journal Cancer Immunology Research, researchers at Thomas Jefferson University in Philadelphia, PA, report how they tested the treatment, which is a type of immunotherapy known as chimeric antigen receptor (CAR) T-cell therapy, in mice that were implanted with human colorectal cancer tumors.

The treatment killed colorectal cancer tumors and prevented them spreading.

Successful completion of this last preclinical stage means that the next step would be a phase I clinical trial in human patients.

The progress is significant because there are few treatment options for colorectal cancer once it has advanced.

“The concept of moving [CAR T-cell] therapy to colorectal cancer is a major breakthrough,” states Dr. Karen Knudsen, who is director of the Sidney Kimmel Cancer Center at Thomas Jefferson University, “and could address a major unmet clinical need.”

Advanced colorectal cancer

Although colorectal cancer is the “third most common” cancer to affect both men and women in the United States, it is the second main cause of cancer deaths.

Estimates suggest that there were 139,992 new cases of colorectal cancer and 51,651 deaths to the disease in the U.S. in 2014, the latest year for official figures.

As with most cancers, most deaths in colorectal cancer occur in patients with advanced disease, which begins when the primary tumor starts to spread.

The tumor can spread either locally into the neighboring tissue, or through metastasis, a process in which cells escape the primary tumor and migrate to other parts of the body where they can set up new, secondary tumors.

Not all cancer cells that escape a primary tumor succeed in forming secondary tumors. The process is complex and has many steps — from breaking away to migrating, evading the immune system, and setting up camp — and it can fail at any step.

The cells that eventually succeed may no longer resemble the cells of the primary tumor. This is one of the reasons that metastatic cancer is harder to treat.

CAR T-cell therapy ‘reprograms’ T cells

CAR T-cell therapy is a type of immunotherapy in which clinicians reprogram genes in “patients’ own immune cells to attack cancer cells.”

To do this, immune system T cells are taken from the patient, genetically reprogrammed in the laboratory, multiplied to vastly increase their number, and then infused back into the patient.

The reprogramming of T cells restores their ability to find and attack cancer cells that had previously been very successful at suppressing the attacks.

However, in order for the T cells to find and kill only the target cancer cells, there has to be a way to identify them uniquely to the T cells. This is where the genetic reprogramming comes in — it makes the T cell seek out a unique marker, called a tumor antigen, on the cells.

The study used GUCY2C tumor antigen

In the case of the new study, the tumor antigen that they used was GUCY2C, whose potential had previously been identified by senior author Adam E. Snook, who is an assistant professor in the Department of Pharmacology and Experimental Therapeutics at Thomas Jefferson University.

Initially, the scientists tested the therapy on laboratory-cultured cancer cells. They showed that it targeted and killed only those cancer cells that expressed the GUCY2C marker; cancer cells without GUCY2C were spared.

Prof. Snook and colleagues then showed that the CAR T-cell therapy using the GUCY2C tumor antigen successfully treated mice implanted with human colorectal cancer tumors.

All the treated mice survived for the whole of the study’s observation time, which amounted to 75 days. Mice treated with a control therapy survived for an average of 30 days.

In another set of experiments, the researchers used mice that had developed their own “murine” colorectal cancer tumors but that had been genetically altered to “express human GUCY2C.”

When they treated those mice with T cells programmed to find GUCY2C-tagged cancer cells, the researchers found that they “provided long-term protection against lung metastases.”

The lung is a common site for secondary tumors in colorectal cancer in humans.

The mice that received CAR T-cell therapy lived for another 100 days with no secondary tumors, whereas the mice that received a control treatment only lived an average of 20 days after treatment.

No ‘off-target’ side effects

Although this study did not test for any side effects that may have arisen from the engineered T cells going “off-target,” the researchers had previously shown, using a mouse version of the therapy, that there were “no off-target effects.”

Prof. Snook acknowledges the “major concern” about safety with using CAR T-cell therapy. “In other cancers,” he notes, “the field has observed lethal autoimmune responses.”

He says that there are ongoing efforts to create fast-acting antidotes to these off-target responses, but he and his colleagues believe that their study shows that GUCY2C CAR T-cell therapy “may be very effective and safe in cancer patients.”

They also see wider applications of the therapy in other hard-to-treat cancers that also express the GUCY2C tumor antigen.

“The antigen we target for colorectal cancer,” explains Prof. Snook, “is one that is shared across several high-mortality cancers including esophageal and pancreatic cancer.”

Taken together, 25 percent of people who die from cancer could potentially be treated with this therapy.

-Prof. Adam E. Snook

Seasonal affective disorder: Why brown-eyed women are at risk

Seasonal affective disorder: Why brown-eyed women are at risk

Seasonal affective disorder (SAD), a psychiatric condition, is often characterized by feelings of hopelessness and acute sadness that occur during the fall and winter months.

A form of depression, SAD is estimated to affect 5 percent of the United States population. And of these, women are thought to be at a higher risk.

In fact, 4 in 5 people living with the condition are thought to be women.

Previously, researchers found that the strong prevalence of SAD among women is independent of social or lifestyle factors, suggesting that perhaps there are biological sex-specific differences that account for the predisposition.

Recent research confirms that women are more prone to the condition, but it adds an interesting element to the mix: eye color.

Additionally, the two new studies provide intriguing novel explanations for why sex and eye color can influence the risk of SAD.

The team’s findings were presented at the annual conference of the British Psychological Society in Nottingham, United Kingdom, by Lance Workman, who is a professor at the University of South Wales, also in the U.K.

Why ‘blue eyes keep the blues away’

The first study to be presented by Prof. Workman — aptly titled ‘Blue eyes keep the blues away: the relationship between SAD, lateralized emotions, and eye color’ — surveyed 175 students from the University of South Wales and the Girne American University in North Cyprus.

The results of the questionnaires revealed that participants with brown eyes were significantly more likely to experience shifts in mood compared with blue-eyed participants.

Prof. Workman has an interesting explanation for this. He says, “We know that light entering the brain causes a decrease in levels of melatonin.”


As blue eyes allow more light into the brain, it may be that this leads to a greater reduction in melatonin during the day and this is why people with lighter eyes are less prone to SAD.

-Prof. Lance Workman

“Individuals with blue eyes appear to have a degree of resilience to SAD,” explain the authors.

“This,” they add, “may be taken as suggestive that the blue eye mutation was selected as a protective factor from SAD as sub-populations of humans migrated to northern latitudes.”

People with SAD use their right brain

The team also asked the participants with SAD to take part in an additional test that examined how their two brain hemispheres responded when they were trying to recognize different emotional expressions on other people’s faces.

This test revealed that people with SAD tended to use their left visual field when recognizing facial expressions and use their brain hemisphere to “decode” these expressions.

As Prof. Workman explains, “This tendency to use the left visual field and right side of the brain for identifying facial expressions is present in the general population, whether they [live with] SAD or not.

“But,” he continues, “people who [have] more conventional forms of depression generally lose this right hemisphere advantage.”

“In the case of SAD, we found this left visual field advantage was actually increased. This suggests SAD has different causes than, say, bipolar depression,” adds Prof. Workman.

Why women may be at a higher risk

The second study presented at the conference surveyed a much larger sample of 2,031 people. Of these, 8 percent had a chronic form of SAD, while 21 percent had a milder form of the illness.

Women were at a particularly high risk — in fact, they were 40 percent more likely to develop the condition than men. The study also points out that SAD is more severe when women are of reproductive age.

This made Prof. Workman venture another possible evolutionary explanation for the findings. He speculates that the disorder is nothing but an energy-preserving mechanism gone haywire.

During a woman’s reproductive years, he says, the mother would have to conserve energy to ensure survival of both her and her offspring, particularly during the winter months.

This seems to be supported by the fact that symptoms of SAD also include a craving for carbs, and putting on weight during the winter months may have also helped our ancestors to cope with the cold, the researcher says.

What causes mucus strands in the eyes?

What causes mucus strands in the eyes?

FARMALISA’s 8 Best selling medicines in 2020

FARMALISA’s 8 Best selling medicines in 2020:

1. Opdivo (BMS)

2. Sevorane (ABBVIE)

3. Perjeta (ROCHE)

4. Survanta (ABBVIE)

5. Thiotixene (MYLAN)

6. Keytruda (MSD)

7. Imbruvica (J&J)

8. Kadcyla (ROCHE)