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‘Remarkable’ breakthrough in cancer treatment saves Leicester girl Alyssa, 13

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A 13-year-old girl who relapsed with leukaemia is now completely cancer-free after a world-first use of what scientists have described as the most sophisticated cell engineering technique ever. Alyssa, who lives in Leicester, would have been receiving end-of-life care now if it weren’t for the treatment, scientists said.

The teenager, the daughter of TV presenter Gok Wan’s cousin, was diagnosed with T-cell acute lymphoblastic leukaemia last year and was given all the conventional treatments including chemotherapy and a bone marrow transplant but the disease returned. The new treatment involved T-cells from a healthy donor that went around her body killing the cancerous T-cells.

T-cells are a type of white blood cell that we all have, which usually move around the body to find and destroy defective cells, helping us stay healthy and fight infections. But in patients like Alyssa they go wrong, attacking the body.

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After Alyssa was diagnosed last year, Gok Wan cried on This Morning as he told Holly Willoughby and Phillip Schofield about her struggle. He had his head shaved on the show, raising more than £53,000 for the charity Young Lives vs Cancer.

He said: “There are so many kids and young people going through this right now so I said I would do something to support her. Alyssa wants to make it very clear that short hair can be cool. They’ve just changed her chemo so she’s a little poorly.”

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In her treatment at Great Ormond Street Hospital for Children (GOSH) in London, Alyssa was given “base-edited” T cells from a donor. She said she had no hesitation about taking part in the clinical trial of the new treatment: “Once I do it, people will know what they need to do, one way or another, so doing this will help people – of course I’m going to do it.”

The trial she was part of, funded by the Medical Research Council, saw her being given edited T-cells that had been pre-manufactured from a healthy volunteer donor in May this year. The researchers chemically converted parts of the cells’ DNA code which carry instructions.

The edited T-cells can be injected into a patient so that they quickly find and destroy T-cells in the body, including cancerous ones, after which the person can have a bone marrow transplant to restore their depleted immune system. Just 28 days after being given the treatment, Alyssa was in remission and was able to have a second bone marrow transplant.

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Great Ormond Street Hospital for Children has been treating Alyssa
(Image: PA Media)

She is now recovering at home and continues with follow-up monitoring at the children’s hospital. It is hoped the research, due to be presented for the first time at the American Society of Haematology annual meeting in New Orleans in the US this weekend, could lead to new treatments and better chances for sick children.

Alyssa’s mother Kiona said the family were “on a strange cloud nine” and that it was “amazing to be home” with Alyssa again. She added: “Hopefully this can prove the research works and they can offer it to more children – all of this needs to have been for something.”

Scientists aim to recruit up to 10 other patients who have T-cell leukaemia and have exhausted all conventional options for the clinical trial into the new treatment. Medics at GOSH hope that if it is successful it can be offered to children earlier in their treatment when they are less sick and that it can be used for other types of leukaemia in future.

Potential patients for trials will be referred by NHS specialists. Professor Waseem Qasim, consultant immunologist at GOSH, said: “This is a great demonstration of how, with expert teams and infrastructure, we can link cutting-edge technologies in the lab with real results in the hospital for patients.

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Alyssa, 13, receiving treatment in hospital
(Image: PA Media)

“It’s our most sophisticated cell engineering so far and paves the way for other new treatments and ultimately better futures for sick children. We have a unique and special environment here at GOSH that allows us to rapidly scale up new technologies and we’re looking forward to continuing our research and bringing it to the patients who need it most.”

Dr Robert Chiesa, a consultant in bone marrow transplant and CAR T-cell therapy at GOSH, said: “Since Alyssa got sick with her leukaemia in May last year, she never achieved a complete remission – not with chemotherapy and not after her first bone marrow transplant. Only after she received her CD7 CAR-T cell therapy and a second bone marrow transplant in GOSH she has become leukaemia-free.”

He described the outcome as “quite remarkable” but cautioned that she must be monitored over the next few months.

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ER closure for Seaforth's emergency department due to COVID-19 outbreak – CTV News London

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Starting on Feb. 1, Seaforth’s emergency department will be closed in the overnight hours.

The Huron Perth Healthcare Alliance said due to “sudden health human resource shortages related to COVID-19,” the Seaforth Community Hospital’s emergency department will be closed from 5 p.m. to 7 a.m., from Feb. 1st to Feb. 6, when regular hours are expected to resume.

On Jan. 28, a COVID-19 outbreak was declared in Seaforth’s inpatient unit, closing all admissions to the unit. On Tuesday, a COVID-19 outbreak was declared at the Clinton General Hospital’s inpatient unit, also closing it to admissions.

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In total, 10 people are in Huron-Perth hospitals dealing with COVID-19.

Two long-term care homes in the region are also dealing with COVID-19 outbreaks at the moment. Since Jan. 1, eight Huron-Perth residents, most of them over the age of 75, have died due to COVID-19, according to the Huron Perth Health Unit.

“I extend my condolences to the loved ones of these individuals,” said Dr. Miriam Klassen, medical officer of health for the Huron Perth Health Unit.

She added, “COVID-19 remains a serious illness for some people, especially those who are older. While we are seeing signs of improvement, it is important to keep taking actions to protect those who are most vulnerable to severe outcomes from this virus.” 

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GLP-1 Agonists Protected Kidneys in T2D With Advanced DKD

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Researchers published the study covered in this summary on Research Square as a preprint that has not yet been peer reviewed.

Key Takeaways

  • In patients with advanced diabetic kidney disease (DKD; estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73m2), treatment with a glucagon-like peptide-1 (GLP-1) agonist had a neutral effect on cardiovascular outcomes but significantly linked with preservation of kidney function and improved survival in a propensity-score matched, retrospective analysis of observational data from more than 2000 people with type 2 diabetes in Taiwan.

Why This Matters

  • Cardiovascular disease is a leading cause of mortality in people with type 2 diabetes and among those with chronic kidney disease.
  • GLP-1 agonists reduce all-cause mortality and cardiovascular death in people with type 2 diabetes, but their role in patients with advanced DKD is controversial.
  • Research on the effect of GLP-1 agonists on cardiovascular outcomes in patients with advanced DKD is limited. Trials that have assessed GLP-1 agonists in people with type 2 diabetes have generally excluded those with advanced DKD and completely excluded those with end-stage kidney disease (eGFR < 30 mL/min/1.73m2).
  • Treatment with GLP-1 agonists has been associated with a significant reduction in composite cardiovascular outcomes in people with type 2 diabetes and relatively fair kidney function (eGFR > 30 mL/min/1.73m2), but among people with type 2 diabetes and lower levels of kidney function, research has shown neutral composite cardiovascular outcomes levels. However, limitations of previous studies include being mainly based on subgroup analysis or including a limited sample of patients.

Study Design

  • Retrospective analysis of observational data from nearly 9000 people in Taiwan with type 2 diabetes and an eGFR < 30 mL/min/1.73m2 who received a first prescription for a GLP-1 agonist or dipeptidyl peptidase 4 (DPP-4) inhibitor in 2012-2021 and had the data necessary for this analysis in their records.
  • The data came from the largest multi-institutional electronic medical record database in Taiwan, which includes two medical centers and five general hospitals and information on more than 11 million patients, from 2001 to 2019.
  • Researchers used propensity scoring to match 602 people treated with a GLP-1 agonist with 1479 people treated with a DPP-4 inhibitor.

Key Results

  • During a mean follow-up of 2.1 years, the rate of the composite cardiovascular outcome (cardiovascular death, myocardial infarction, and ischemic stroke) did not significantly differ between the GLP-1 agonist and DPP-4 inhibitor groups, with incidence rates of 13.0% and 13.8%, respectively, and a nonsignificant hazard ratio of 0.88. Rates of each of the three components of the composite endpoint also did not significantly differ between the two groups.
  • Progression to end-stage kidney disease with dialysis was significantly lower in those treated with a GLP-1 agonist compared with a DPP-4 inhibitor, with incidence rates of 23.4% and 27.5%, respectively, and a significant hazard ratio of 0.72.
  • The incidence of a greater than 50% drop in eGFR from baseline was 32.2% with GLP-1 agonist treatment compared to 35.9% with a DPP-4 inhibitor, with a significant hazard ratio of 0.74.
  • Median time until patients needed new-onset dialysis was 1.9 years with GLP-1 agonist treatment and 1.3 years with DPP-4 inhibitor treatment, which was a significant difference.
  • The rate of all-cause death was 18.4% with GLP-1 agonist treatment compared with 25.1% with DPP-4 inhibitor treatment, a hazard ratio of 0.71 that was significant.

Limitations

  • Because the study was a retrospective analysis of observational data it cannot prove causality.
  • The study could be subject to residual confounding despite propensity-score matching.
  • The data came from health records that could have included coding errors.
  • Treatment compliance was unknown.

Disclosures

This is a summary of a preprint research study, “The cardiovascular and renal effects of glucagon-like peptide 1 receptor agonists in patients with advanced diabetic kidney disease,” by researchers in Taiwan on Research Square and provided to you by Medscape. This study has not yet been peer reviewed. The full text of the study can be found on researchsquare.com.

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Research by UBC professor lays groundwork for life-saving breast cancer treatment

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A drug originally designed to prevent osteoporosis is now expected to save and improve the lives of millions of people with breast cancer, thanks in part to decades of foundational research by Dr. Josef Penninger, a professor in UBC’s Faculty of Medicine and director of the Life Sciences Institute.

The achievement highlights how UBC scientists are developing effective new treatments — and unlocking the full potential of existing drugs – through research into the fundamental biological principles behind disease. By advancing scientific discoveries from the lab to the clinic, UBC researchers are bringing life-changing treatments to patients everywhere.

The drug, called Denosumab, was recently shown in a long-term Phase 3 clinical trial to improve survival among postmenopausal women with hormone receptor-positive early breast cancer receiving aromatase inhibitor treatment. Moreover, the drug markedly improved patients’ quality of life by reducing broken bones by 50 per cent, a common side effect of breast cancer treatment. The results of the trial were recently reported in The New England Journal of Medicine.

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Denosumab is a monoclonal antibody developed by American biopharmaceutical company Amgen to prevent bone loss. In the early 2000s, research by Dr. Penninger and his team revealed the therapeutic potential of Denosumab, as well as the drug’s surprising connections with breast cancer.

“More than two decades ago we started the experimental groundwork that revealed Donosumab’s potential as a treatment for breast cancer patients,” says Dr. Penninger. “These results are incredibly exciting and will help improve the lives of millions of patients. I am very proud of all the people in my lab over the years who did that work and helped pave the way for this achievement.”

Discovering the link between osteoporosis and breast cancer

Denosumab works by binding to and inhibiting the activity of a protein called RANKL, which plays a key role in bone-resorbing cells called osteoclasts. By blocking RANKL, denosumab reduces the activity of osteoclasts and slows down bone resorption, helping to increase bone density and preventing osteoporosis.

Dr. Josef Penninger

Dr. Josef Penninger

Dr. Penninger and his team began to draw the connection between osteoporosis and HR-positive breast cancer when they generated the first RANKL “knock-out” mice in the late 1990s.

A knockout mouse is a laboratory mouse that has been genetically engineered to have certain genes deactivated, or “knocked-out”. Dr. Penninger’s team engineered mice that lacked the genes necessary to produce the RANKL protein in an effort to study the protein’s essential function in bone metabolism.

However, to the researchers’ surprise, they discovered that the RANKL-deficient mice failed to develop a lactating mammary gland in pregnancy – a process that depends on sex hormones.

“This proved an evolutionary link: showing how bone loss is regulated by sex hormones, and how pregnant mammals activate RANKL to form breast tissue for lactation among other functions,” says Dr. Penninger.

Based on this initial finding, Dr. Penninger’s team went on to show that RANKL played a key role in progestin-driven breast cancer, as well as breast cancer driven by BRCA1 mutations.

“Further researcher revealed how RANKL controls the stem cells in the breast that respond to sex hormones and thereby drives growth of the breast tissue at every menstruation cycle and in particular in pregnancy and lactation,” adds Dr. Penninger.

In the case of breast cancer, RANKL spurs mammary epithelial cells to divide, and helps to maintain the stem cells that give rise to breast tumours.

A dual benefit drug

One in eight Canadian women will be diagnosed with breast cancer in their lifetime according to the Canadian Breast Cancer Network. An estimated 70 to 80 per cent of these breast cancers are hormone receptor-positive (HR-positive), making it the most prevalent breast cancer subtype.

The current standard treatment for HR-positive breast cancer involves surgery and radiation, followed by treatment with aromatase inhibitors for 5 to 7 years. While aromatase inhibitors diminish sex hormones that drive new cancer growth, they can have serious adverse effects on bone health, including increased risk of osteoporosis and fractures.

The now-published clinical trial, led by the Austrian Breast and Colorectal Cancer Study Group, was conducted to see if Denosumab could help in two ways: by reducing these negative effects on bone health, while also improving breast cancer survival outcomes.

“These results are incredibly exciting and will help improve the lives of millions of patients.”
Dr. Josef Penninger

The results reveal that 6 mg of Denosumab every six months — the recommended treatment level for osteoporosis — improved disease-free survival, bone metastasis-free survival, and overall survival among participants. It also effectively reduced bone fractures over the long term.

“Blocking RANKL in breast cancer patients reduces broken bones by 50 per cent, massively improving their quality of life, and even at a very low treatment dose,” says Dr. Penninger. “We now know that RANKL drives breast cancer cell growth, is the critical mechanism behind bone loss, and has also an effect on anti-cancer immunity and immunological rewiring in pregnancy. These clinical results in patients show how blocking RANKL could save the lives of 50,000 women among one million women with the diagnosis of breast cancer.”

Based on the data, the researchers behind the trials are recommending that Denosumab be considered for routine clinical use in postmenopausal breast cancer patients receiving aromatase inhibitor therapy.

These trials were largely based on the foundational research published by the Penninger laboratory, including Kong et al. Nature 1999, Fata et al. Cell 2000, Jones Nature 2006, Schramek et al. Nature 2010, Sigl et al. Cell Research 2016, and Paolino et al. Nature 2021.

Dr. Penninger is now part of a large international prevention trial evaluating Denosumab in young women who carry BRCA1 mutations.

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