Category Archives: Stem Cell Treatment

Europe’s guardian of stem cells and hopes, real and unrealistic – INQUIRER.net

Submerged in liquid nitrogen vapor at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukemias and lymphomas, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industrys leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States, a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

Health insurance

Mum-of-two Teresa Przeborowska has firsthand experience.

At five years old, her son Michal was diagnosed with lymphoblastic leukemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sisters stem cells into Michals bloodstream.

It was not quite enough for Michals needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, is now flourishing, both intellectually and physically, his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, each container holds up to 10,000 blood bags Safe and secure, they wait to be used in the future, its head, Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the blood will be ready to use without the whole process of looking for a compatible donor and running blood tests, the biologist told AFP.

For families who have paid an initial nearly 600 euros (around P34,000) and then an annual 120 euros (around P7,000) to have the blood taken from their newborns umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

Beauty products

Hematologist Wieslaw Jedrzejczak, a bone marrow pioneer in Poland, describes promoters of the treatment as sellers of hope, who make promises that are either impossible to realize in the near future or downright impossible to realize at all for biological reasons.

He compares them to makers of beauty products who swear their cream will rejuvenate the client by 20 years.

Various research is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

There is a list of almost 80 diseases for which stem cells could prove beneficial, U.S. hematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood program Vitalant in New Jersey, told AFP.

But given the present state of medicine, they are effective only for around a dozen of them, like leukemia or cerebral palsy, he said.

Its not true, as its written sometimes, that we can already use them to fight Parkinsons disease or Alzheimers disease or diabetes.

EuroStemCell also cautions against private blood banks that advertise services to parents suggesting they should pay to freeze their childs cord blood in case its needed later in life.

Studies show it is highly unlikely that the cord blood will ever be used for their child, the network said.

It also pointed out that there could be a risk of the childs cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most E.U. countries however permit it while imposing strict controls.

Rapid growth

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of 2,500 euros (around P140,000) upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with 2 million zlotys (around P26 million), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35% of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed 63 million euros to the firm, PBKMs chief executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.IB/JB

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Europe's guardian of stem cells and hopes, real and unrealistic - INQUIRER.net

Diane Francis: Treating aging like a disease is the next big thing for science – Financial Post

LOS ANGELES Extending everyones life in a healthy fashion is one of many goals held by Peter Diamandis, a space, technology, aeronautics and medicine pioneer. But the new field known as longevity is of interest to everyone.

One hundred will be the new 60, he told his Abundance360 conference recently. The average human health span will increase by 10+ years this decade.

He, like others in Silicon Valley, believe that aging is a disease and the result of planned obsolescence, or the wearing down of, or damage to, certain critical mechanisms, sensors and functions within our bodies. Longevity research is about identifying the core problems to mitigate or reverse them.

The average human health span will increase by 10+ years this decade

Peter Diamandis

The exponential technologies of artificial intelligence, machine learning and computational heft have been harnessed, and have resulted in breakthroughs and clinical trials that are just a handful of years away from deployment on human patients. The main areas of research include: Stem cell supply restoration, regenerative medicine to regrow damaged cartilage, ligaments, tendons, bone, spinal cords and neural nerves; vaccine research against chronic diseases such as Alzheimers; and United Therapeutics that is developing technology to tackle the organ shortage for humans by genetically engineering organs grown in pigs.

New tools are accelerating the development of new, tailor-made medicines at a fraction of todays costs. Alex Zhavoronkov of Insilico Medicine told the conference that drugs take 10 years and cost $3 billion to research and 90 per cent fail. But his company can test in 46 days using human tissue, then model, design and produce in weeks with the help of advanced computing.

In regenerative medicine, advances appear to be arriving relatively soon. For instance, Diamandis asked the audience if anyone was awaiting a knee replacement operation and suggested that they might be better off postponing these until 2021 when regenerative medicine innovator, Samumed LLC in San Diego, is expected to complete phase three clinical trials of cartilage regeneration.

Samumeds founder, Osman Kibar, said his company has successfully injected a protein that activates nearby stem cells into producing new cartilage in a knee or a new disc in a spine. Preliminary success has also occurred to regenerate muscle and neural cells, retinal cells, skin and hair. Not surprisingly, the private company just raised US$15.5 billion to continue research and product development.

Another hot area of early stage research is called epigenetic reprogramming or identifying how to reverse deficiencies in proteins, stem cells, chromosomes, genes that repair DNA and damaged cells. A leader in this field is David Sinclair, professor of genetics at the Harvard Medical School, whose new book Lifespan: Why We Age and Why We Dont Have To explains the science and offers advice.

Aging is a disease, and that disease is treatable, he said. As research progresses toward actual corrections or cures, there are also lifestyle habits that can slow down the aging process, or avert damage. For instance, he said humans should replicate some behaviour that their bodies were designed for. Obviously, exercising and sleep are necessary but so is eating less often. You should feel hungry regularly, he said.

Another condition that is useful to emulate is hormesis, a scientific term for what Neitzsche posited which was that that which does not kill us makes us stronger. Sinclair recommends stressing our bodies with temperature changes such as going from a hot sauna to rolling in the snow. This invigorates the bodys processes and cells.

Theres also xenohormesis or gaining benefits from eating plants that have been environmentally stressed, therefore contain more beneficial nutrients. For instance, drought-stressed or wild strawberries have better flavour but they also are enhanced with additional antioxidant capacity and phenol content.

The age of 100 is easily in sight now, said Diamandis. And kids born today can expect to live to 105.

Financial Post

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Diane Francis: Treating aging like a disease is the next big thing for science - Financial Post

OncoImmune Announces Presentation of CD24Fc Phase IIa Data and Phase III Clinical Trial Design at the 2020 Transplantation & Cellular Therapy…

OncoImmune, Inc. announced today that clinical data from its Phase IIa clinical trial of CD24Fc are being presented at the 2020 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Meeting, taking place in Orlando, Florida in February. The Phase IIa data will be presented by the studys Principle Investigator, Dr. John Magenau of the University of Michigans Department of Medicine, at 11:15 am on February 21. Dr. Pan Zheng, the Chief Medical Officer of OncoImmune, Inc., will present Phase III clinical trial design in a poster session on February 19-20th.

CD24Fc is OncoImmunes first-in class fusion protein that selectively represses inflammation induced by tissue injury while preserving innate immune response to pathogens. The Phase IIa study is a randomized, double blind, placebo controlled, multi-center study to investigate adding CD24Fc to standard of care tacrolimus and methotrexate in acute graft-versus host disease (GVHD) prophylaxis for allogeneic hematopoietic stem cell transplantation (HCT) with matched unrelated donors in treatment of leukemia and myelodysplastic syndrome. The trial included three CD24Fc dose cohorts: 240 mg at day -1, 480 mg at day -1, and the multi-dose cohort of 480-240-240 mg at day -1, day 14 and day 28. CD24Fc has received orphan drug designation from both the US FDA and European Medical Agency (EMA) for GVHD prophylaxis.

The presentation, entitled, "Mitigating Damage Response with CD24 Fusion Protein for Prevention of Acute Graft-Versus Host Disease," compares safety and efficacy data of CD24Fc when used in combination with standard of care GVHD prophylaxis compared to placebo and historical controls. The results demonstrate that CD24Fc was safe and well tolerated in the patient population. More importantly, patients receiving CD24Fc performed significantly better than placebo and historical controls in 180 day grade III-IV GVHD-free survival, the planned primary endpoint for the Phase III trial. These data thus provided strong support for the primary endpoint and dosing regimen of the upcoming phase III clinical trial. Moreover, significantly better relapse free survival (RFS) was observed over placebo control and historical controls. Overall survival (OS) was also significantly improved when compared with a matched historical control. Furthermore, a significant, dose-dependent reduction of mucositis was observed.

"We are very excited by the data observed in the Phase IIa clinical trial. In addition, we have completed enrollment of an open label Phase II expansion study where the drug continues to perform very well with clear signs of clinical efficacy," said Dr. Pan Zheng. "HCT is a curative therapy for refractory leukemia patients but hampered by GVHD, leukemia relapse and conditioning toxicity. As suggested by our preliminary data, CD24Fc shows significant promise in all three of these outcomes and would likely be transformative for the HCT field," she continued.

About OncoImmune, Inc.

OncoImmune (www.oncoimmune.com) is a privately-held, clinical-stage biopharmaceutical company that is actively engaged in the discovery and development of novel immunotherapies for cancer, inflammation and autoimmune diseases.

In addition to the pivotal trial of CD24Fc for HCT indications, OncoImmune is also launching multiple clinical trials testing its efficacy in immunotherapy-related adverse events, mucositis, and sequalae of chronic inflammation of patients infected with human immunodeficiency virus.

More recently, OncoImmune received FDA approval for the first-in-human clinical trial, testing the safety and efficacy of ONC-392, the only CTLA-4 targeting agent that preserves CTLA-4 recycling and thus the potential to simultaneously improve safety and efficacy of cancer immunotherapy.

OncoImmune is based in Rockville, Maryland.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200127005538/en/

Contacts

Martin Devenport OncoImmune, Inc. mdevenport@oncoimmune.com

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OncoImmune Announces Presentation of CD24Fc Phase IIa Data and Phase III Clinical Trial Design at the 2020 Transplantation & Cellular Therapy...

Rochester nonprofit offers a helping hand to patients affected by blood cancers – Med City Beat

When Woodbury resident Tracy McGarry was diagnosed with multiple myeloma, she and her husband Mike turned to a city 90 miles south for answers. Rochester soon became a second home of sorts not by choice, but by necessity.

We were referred to Mayo Clinic by a myeloma specialist in the [Twin] Cities, recalled Mike. Every time wed be down there, we were there for seven days at a time. Tracy was gearing up for a stem cell transplant in the fall of 2017, so we were down there quite a bit.

Blood cancers like multiple myeloma and leukemia bring many families like the McGarrys to Rochester every year. They seek treatment that may not be available at their hometown hospital, all while trying to navigate a city thats entirely new to them. The whole experience can be disorienting, and Rochester can start to feel cold to outsiders.

Enter Kristina Wright-Peterson and Red Drop Resources. You may know this organization by their previous name, Med City Foundation, but for their five-year anniversary, Wright-Peterson decided it was time for a change.

She said the new name better reflects what the organization provides.

The term resources really speaks to everything we provide patients, said Wright-Peterson. We dont just provide financial assistance. We dont just provide assistance with finding a place to stay. We start every conversation with patients in terms of, what do you need help with? We dont tell them what we help with; we ask them what they need.

A majority of the time, families dealing with blood cancers have not had much time to prepare for a sudden move to Rochester. That means they need income support and a place to stay fast.

The blood cancer treatment regimen requires people to stay for 6-8 weeks, said Wright-Peterson. That means the patient, plus a caregiver normally their spouse are stuck in Rochester, trying to pay for things here with no money coming in.

Wright-Peterson founded Red Drop Resources in 2014 in honor of her late father, who died in 1995 after a battle with leukemia. She says the nonprofit fills a need that her family had nearly 20 years ago, and working with her mother, Virginia, has been a benefit for both of them.

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Rochester nonprofit offers a helping hand to patients affected by blood cancers - Med City Beat

Cardiff researchers on brink of ‘one size fits all’ cancer therapy – Active Quote

Monday, January 27, 2020

Cancer patients could be treated with a one-size-fits-all therapy, following the discovery of an immune cell which kills all forms of the disease.

Researchers at Cardiff University have found a new type of killer T-cell, capable of recognising and destroying most human cancers while preserving healthy cells. The scientists discovered a method of killing prostate, breast, lung and other cancers in lab tests and say there is enormous potential for immunotherapies not previously thought to be possible.

Cardiff Universitys cancer findings came from scientists looking for unconventional ways in which the immune system naturally attacks tumours. They found, inside human blood, a T-cell that can scan the body for a threat, such as cancerous cells, and eliminate the danger while leaving healthy cells alone. The team described the work as at an early stage, but exciting.

T-cell cancer therapies are where immune cells are removed, modified and returned to the patients blood to seek and destroy cancer cells. The most widely-used, known as CAR-T, is personalised to the patient but combats only a handful of cancers and has not been successful in eliminating solid tumours - which account for the vast majority of cancers.

The Cardiff teams discovery involves a new type of T-cell receptor (TCR), which recognises a molecule present on the surface of a wide range of cancer cells as well as in many of the bodys normal cells and is, remarkably, able to distinguish between the two. In tests, T-cells equipped with the new TCR killed lung, skin, blood, colon, breast, bone, prostate, ovarian, kidney and cervical cancer cells.

Professor Andrew Sewell, the lead author on the study and an expert in T-cells from Cardiff Universitys School of Medicine, said it was highly unusual to find a TCR with such broad cancer specificity, raising the prospect of universal cancer therapy.

Prof Sewell said: We hope this new TCR may provide us with a different route to target and destroy a wide range of cancers in all individuals. Current TCR-based therapies can only be used in a minority of patients with a minority of cancers.

Cancer-targeting via MR1-restricted T-cells is an exciting new frontier - it raises the prospect of a one-size-fits-all cancer treatment; a single type of T-cell that could be capable of destroying many different types of cancers across the population. Previously nobody believed this could be possible.

Further experiments and safety testing are now underway, with the hope of trialling this new approach in patients towards the end of 2020. Prof Sewell added: There are plenty of hurdles to overcome; however, if this testing is successful, then I would hope this new treatment could be in use in patients in a few years time.

Cancer is the leading cause of all avoidable deaths in the UK. Breast cancer is the most common, followed jointly by prostate and lung cancer and then by bowel cancer. Obesity is now a bigger cause than smoking of some cancers, namely bowel, kidney, liver and ovarian cancer.

According to financial information business Defaqto*, 38 out of 51 health insurance products include cancer cover, with benefits ranging from breakthrough treatment not otherwise available on the NHS to hormone therapy, reconstructive surgery and stem cell therapy. To find the right cancer cover for your family, use our online comparison tool or speak with our team on 0800 862 0373.

Photo:Cardiff Universitys Professor Andrew Sewell, left, with Research Fellow Garry Dolton.

Credit: Cardiff University

* Data sourced on January 2, 2020.

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Scientists Think They Know How Stress Causes Gray Hair – Healthline

Sorry Mom and Dad: It turns out you might not have been exaggerating when you told us your children made your hair turn gray.

Stress may play a key role in just how quickly hair goes from colored to ashen, a study published this past week in the journal Nature suggests.

Scientists have long understood some link is possible between stress and gray hair, but this new research from Harvard University in Massachusetts more deeply probes the exact mechanisms at play.

The researchers initial tests looked closely at cortisol, the stress hormone that surges in the body when a person experiences a fight or flight response.

Its an important bodily function, but the long-term presence of heightened cortisol is linked to a host of negative health outcomes.

But the culprit ended up being a different part of the bodys fight or flight response the sympathetic nervous system.

These nerves are all over the body, including making inroads to each hair follicle, the researchers reported.

Chemicals released during the stress response specifically norepinephrine causes pigment producing stem cells to activate prematurely, depleting the hairs reserves of color.

The detrimental impact of stress that we discovered was beyond what I imagined, Ya-Chieh Hsu, PhD, a lead study author and an associate professor of stem cell and regenerative biology at Harvard, said in a press release. After just a few days, all of the pigment-regenerating stem cells were lost. Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.

But stress isnt the only or even the primary reason that most people get gray hair.

In most cases, its simple genetics.

Gray hair is caused by loss of melanocytes (pigment cells) in the hair follicle. This happens as we age and, unfortunately, there is no treatment that can restore these cells and the pigment they produce, melanin, Dr. Lindsey A. Bordone, a dermatologist at ColumbiaDoctors and an assistant professor of dermatology at Columbia University Medical Center in New York, told Healthline. Genetic factors determine when you go gray. There is nothing that can be done medically to prevent this from happening when it is genetically predetermined to happen.

That doesnt mean environmental factors such as stress dont play a role.

Smoking, for instance, is a known risk factor for premature graying, according to a 2013 study. So kick the habit if you want to keep that color a little longer.

Other contributing factors to premature graying include deficiencies in protein, vitamin B-12, copper, and iron as well as aging due in part to an accumulation of oxidative stress.

That stress is prompted by an imbalance between free radicals and antioxidants in your body that can damage tissue, proteins, and DNA, Kasey Nichols, NMD, an Arizona physician and a health expert at Rave Reviews, told Healthline.

And some degree of oxidative stress is a natural part of life.

We would expect increasing gray hair as we advance in age, and we see about a 10 percent increase in the chance of developing gray hair for every decade after age 30, Nichols said.

Changes you can pursue to delay premature grays include eating a diet high in omega-3 fatty acids such as walnuts and fatty fish, not spending too much time in the skin-damaging and hair-damaging ultraviolet light of the sun, and taking vitamin B-12 and vitamin B-6 supplements.

That said, if you are going gray prematurely, it wouldnt hurt to go have a checkup just in case natural genetic factors arent the sole culprit.

The new Harvard research is only a mouse study, so replicating the same results in a human study would be necessary to strengthen the findings.

But the Harvard research has implications far beyond graying hair, with the hair color change merely one obvious sign of other internal changes as a result of prolonged stress.

By understanding precisely how stress affects stem cells that regenerate pigment, weve laid the groundwork for understanding how stress affects other tissues and organs in the body, said Hsu. Understanding how our tissues change under stress is the first critical step towards eventual treatment that can halt or revert the detrimental impact of stress.

Might that also mean someday halting and reverting the march of premature gray hair? Its too soon to tell.

We still have a lot to learn in this area, Hsu said.

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Scientists Think They Know How Stress Causes Gray Hair - Healthline

Revving the Engine – Harvard Medical School

The hearts ability to beat normally over a lifetime is predicated on the synchronized work of proteins embedded in the cells of the heart muscle.

Like a fleet of molecular motors that get turned on and off, these proteins cause the heart cells to contract, then force them to relax, beat after life-sustaining beat.

Now a study led by researchers at Harvard Medical School, Brigham and Womens Hospital and the University of Oxford shows that when too many of the hearts molecular motor units get switched on and too few remain off, the heart muscle begins to contract excessively and fails to relax normally, leading to its gradual overexertion, thickening and failure.

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Results of the work, published Jan. 27 inCirculation,reveal that this balancing act is an evolutionary mechanism conserved across species to regulate heart muscle contraction by controlling the activity of a protein called myosin, the main contractile protein of the heart muscle.

The findingsbased on experiments with human, mouse and squirrel heart cellsalso demonstrate that when this mechanism goes awry it sets off a molecular cascade that leads to cardiac muscle over-exertion and culminates in the development of hypertrophic cardiomyopathy (HCM), the mostcommon genetic diseaseof the heartand aleading causeof sudden cardiac death in young people and athletes.

Our findings offer a unifying explanation for the heart muscle pathology seen in hypertrophic cardiomyopathy that leads to heart muscle dysfunction and, eventually, causes the most common clinical manifestations of the condition, said senior authorChristine Seidman, professor of genetics in the Blavatnik Institute at Harvard Medical School, a cardiologist at Brigham and Womens Hospital and a Howard Hughes Medical InstituteInvestigator.

Importantly, the experiments showed that treatment with an experimental small-molecule drug restored the balance of myosin arrangements and normalized the contraction and relaxation of both human and mouse cardiac cells that carried the two most common gene mutations responsible for nearly half of all HCM cases worldwide.

If confirmed in further experiments, the results can inform the design of therapies that halt disease progression and prevent complications.

Correcting the underlying molecular defect and normalizing the function of heart muscle cells could transform treatment options, which are currently limited to alleviating symptoms and preventing worst-case scenarios such as life-threatening rhythm disturbances and heart failure, said study first authorChristopher Toepfer,who performed the work as a postdoctoral researcher in Seidmans lab and is now a joint fellow in the Radcliffe Department of Medicine at the University of Oxford.

Some of the current therapies used for HCM include medications to relieve symptoms, surgery to shave the enlarged heart muscle or the implantation of cardioverter defibrillators that shock the heart back into rhythm if its electrical activity ceases or goes haywire. None of these therapies address the underlying cause of the disease.

Imbalance in the motor fleet

Myosin initiates contraction by cross-linking with other proteins to propel the cell into motion. In the current study, the researchers traced the epicenter of mischief down to an imbalance in the ratio of myosin molecule arrangements inside heart cells. Cells containing HCM mutations had too many molecules ready to spring into action and too few myosin molecules idling standby, resulting in stronger contractions and poor relaxation of the cells.

An earlier study by the same team found that under normal conditions, the ratio between on and off myosin molecules in mouse heart cells is around 2-to-3. However, the new study shows that this ratio is off balance in heart cells that harbor HCM mutations, with disproportionately more molecules in active versus inactive states.

In an initial set of experiments, the investigators analyzed heart cells obtained from a breed of hibernating squirrel as a model to reflect extremes in physiologic demands during normal activity and hibernation. Cells obtained from squirrels in hibernationwhen their heart rate slows down to about six beats per minutecontained 10 percent more off myosin molecules than the heart cells of active squirrels, whose heart rate averages 340 beats per minute.

We believe this is one example of natures elegant way of conserving cardiac muscle energy in mammals during dormancy and periods of deficient resources, Toepfer said.

Next, researchers looked at cardiac muscle cells from mice harboring the two most common gene defects seen in HCM. As expected, these cells had altered ratios of on and off myosin reserves.The researchers also analyzed myosin ratios in two types of human heart cells: Stem cell-derived human heart cells engineered in the lab to carry HCM mutations and cells obtained from the excised cardiac muscle tissue of patients with HCM. Both had out-of-balance ratios in their active and inactive myosin molecules.

Further experiments showed that this imbalance perturbed the cells normal contraction and relaxation cycle. Cells harboring HCM mutations contained too many on myosin molecules and contracted more forcefully but relaxed poorly. In the process, the study showed, these cells gobbled up excessive amounts of ATP, the cellular fuel that sustains the work of each cell in our body. And because oxygen is necessary for ATP production, the mutated cells also devoured more oxygen than normal cells, the study showed. To sustain their energy demands, these cells turned to breaking down sugar molecules and fatty acids, which is a sign of altered metabolism, the researchers said.

Taken together, our findings map out the molecular mechanisms that give rise to the cardinal features of the disease, Seidman said. They can help explain how chronically overexerted heart cells with high energy consumption in a state of metabolic stress can, over time,lead to a thickened heart muscle that contracts and relaxes abnormally and eventually becomes prone to arrhythmias, dysfunction and failure.

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Revving the Engine - Harvard Medical School

Researchers Say They Have Clue to Early-Onset Parkinson Disease – AJMC.com Managed Markets Network

Patients with early-onset Parkinson disease may have been born with disordered brain cells that mishandled dopamine for decades, according to a study released Monday.

To perform the study, researchers at Cedars-Sinai Medical Center and UCLA generated special stem cells, known as induced pluripotent stem cells (iPSCs), from cells of 3 patients with young-onset Parkinson disease; the patients were aged 30-39 and hadno known familial history of PD or PD mutations.

The process involved taking adult blood cells back to a primitive embryonic state. These iPSCs can then produce any cell type of the human body, all genetically identical to the patient's own cells. The team used the iPSCs to produce dopamine neurons from each patient and then cultured them in a dish and analyzed the neurons' functions.

The researchers detected 2 key abnormalities in the dopamine neurons:

The investigators also used their model to test a number of drugs that might reverse the abnormalities. One drug, PEP005, or ingenol mebutate gel, used for basal cell carcinoma, reduced the elevated levels of alpha-synuclein in both the dopamine neurons in the dish and in laboratory mice.

The drug also countered another abnormality they found in the patients' dopamine neuronselevated levels of an active version of an enzyme called protein kinase C. However, the role of this enzyme version in Parkinson disease is unknown.

In Parkinson, brain neurons that make dopamine, a substance that helps coordinate muscle movement, become impaired or die. The disease is diagnosed in at least 500,000 people in the United States each year, and the incidence is rising. About 10% are aged 21 to 50 years.

Michele Tagliati, MD, director of the Movement Disorders Program, vice chair and professor in the Department of Neurology at Cedars-Sinai, said the team plans to investigate how PEP005 might be delivered to the brain to potentially treat or prevent young-onset Parkinson. The team also plans more research to determine whether the abnormalities the study found in neurons of young-onset Parkinson's patients also exist in other forms of the disease.

Reference

Laperle AH, Sances S, Yucer N, et al.iPSC modeling of young-onset Parkinsons disease reveals a molecular signature of disease and novel therapeutic candidates [published online January 27, 2020].Nat Med.doi: 10.1038/s41591-019-0739-1.

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Researchers Say They Have Clue to Early-Onset Parkinson Disease - AJMC.com Managed Markets Network

Divorce as Seen Through the Eyes of a Child – SWAAY

With so many groundbreaking medical advances being revealed to the world every single day, you would imagine there would be some advancement on the plethora of many female-prevalent diseases (think female cancers, Alzheimer's, depression, heart conditions etc.) that women are fighting every single day.

For Anna Villarreal and her team, there frankly wasn't enough being done. In turn, she developed a method that diagnoses these diseases earlier than traditional methods, using a pretty untraditional method in itself: through your menstrual blood.

Getting from point A to point B wasn't so easy though. Villarreal was battling a disease herself and through that experience. I wondered if there was a way to test menstrual blood for female specific diseases," she says. "Perhaps my situation could have been prevented or at least better managed. This led me to begin researching menstrual blood as a diagnostic source. For reasons the scientific and medical community do not fully understand, certain diseases impact women differently than men. The research shows that clinical trials have a disproportionate focus on male research subjects despite clear evidence that many diseases impact more women than men."

There's also no denying that gap in women's healthcare in clinical research involving female subjects - which is exactly what inspired Villarreal to launch her company, LifeStory Health. She says that, with my personal experience everything was brought full circle."

There is a challenge and a need in the medical community for more sex-specific research. I believe the omission of females as research subjects is putting women's health at risk and we need to fuel a conversation that will improve women's healthcare.,"

-Anna Villarreal

Her brand new biotech company is committed to changing the women's healthcare market through technology, innovation and vocalization and through extensive research and testing. She is working to develop the first ever, non-invasive, menstrual blood diagnostic and has partnered with a top Boston-area University on research and has won awards from The International Society for Pharmaceutical Engineering and Northeastern University's RISE.

How does it work exactly? Proteins are discovered in menstrual blood that can quickly and easily detect, manage and track diseases in women, resulting in diseases that can be earlier detected, treated and even prevented in the first place. The menstrual blood is easy to collect and since it's a relatively unexplored diagnostic it's honestly a really revolutionary concept, too.

So far, the reactions of this innovative research has been nothing but excitement. The reactions have been incredibly positive." she shares with SWAAY. Currently, menstrual blood is discarded as bio waste, but it could carry the potential for new breakthroughs in diagnosis. When I educate women on the lack of female subjects used in research and clinical trials, they are surprised and very excited at the prospect that LifeStory Health may provide a solution and the key to early detection."

To give a doctor's input, and a little bit more of an explanation as to why this really works, Dr. Pat Salber, MD, and Founder of The Doctor Weighs In comments: researchers have been studying stem cells derived from menstrual blood for more than a decade. Stem cells are cells that have the capability of differentiating into different types of tissues. There are two major types of stem cells, embryonic and adult. Adult stem cells have a more limited differentiation potential, but avoid the ethical issues that have surrounded research with embryonic stem cells. Stem cells from menstrual blood are adult stem cells."

These stem cells are so important when it comes to new findings. Stem cells serve as the backbone of research in the field of regenerative medicine the focus which is to grow tissues, such as skin, to repair burn and other types of serious skin wounds.

A certain type of stem cell, known as mesenchymal stem cells (MenSCs) derived from menstrual blood has been found to both grow well in the lab and have the capability to differentiate in various cell types, including skin. In addition to being used to grow tissues, their properties can be studied that will elucidate many different aspects of cell function," Dr. Salber explains.

To show the outpour of support for her efforts and this major girl power research, Villarreal remarks, women are volunteering their samples happily report the arrival of their periods by giving samples to our lab announcing de-identified sample number XXX arrived today!" It's a far cry from the stereotype of when it's that time of the month."

How are these collections being done? Although it might sound odd to collect menstrual blood, plastic cups have been developed to use in the collection process. This is similar to menstrual products, called menstrual cups, that have been on the market for many years," Dr. Salber says.

Equally shocking and innovative, this might be something that becomes more common practice in the future. And according to Dr. Salber, women may be able to not only use the menstrual blood for early detection, but be able to store the stem cells from it to help treat future diseases. Companies are working to commercialize the use of menstrual blood stem cells. One company, for example, is offering a patented service to store menstrual blood stem cells for use in tissue generation if the need arises."

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Divorce as Seen Through the Eyes of a Child - SWAAY

Diabetes-related proteins examined for the first time at high resolution – Drug Target Review

A key receptor has been examined for the first time at high resolution which could lead to better treatments for conditions such as type 2 diabetes.

Scientists have examined a key receptor for the first time at high resolution which, they say, broadens understanding of how it might function and opens the door to future improvements in treating conditions such as type 2 diabetes.

The scientists were led by experts at the University of Birmingham, UK and the Max Planck Institute for Medical Research, Germany.

Glucagon-like peptide-1 receptors (GLP1R) are found on insulin-producing beta cells of the pancreas and neurons in the brain. The receptor encourages the pancreas to release more insulin, stops the liver from producing too much glucose and reduces appetite. This combination of effects can help to control blood sugar levels.

Therefore, GLP1R has become a significant target for the treatment of type 2 diabetesand a range of drugs are now available that are based on it. But much remains unknown about GLP1R function because its small size makes it difficult to visualise.

Our research allows us to visualise this key receptor in much more detail than before, David Hodson, Professor of Cellular Metabolism at the University of Birmingham. Think about watching a movie in standard definition versus 4k, thats how big the difference is. We believe this breakthrough will give us a much greater understanding of GLP1R distribution and function. Whilst this will not immediately change treatment for patients, it might influence how we design drugs in the future.

GLP1R visualized in insulin-secreting beta cells at super-resolution (credit: University of Birmingham).

The researchers used a number of techniques to conduct a detailed examination of the receptor in living cells including synthesis of marker compounds, immunostaining, super-resolution microscopy, as well as in vivo examination of mice.

Our experiments, made possible by combining expertise in chemistry and cell biology, will improve our understanding of GLP1R in the pancreas and the brain. Our new tools have been used in stem cells and in the living animal to visualise this important receptor and we provide the first super-resolution characterisation of a class B GPCR. Importantly, our results suggest a degree of complexity not readily appreciated with previous approaches, added Johannes Broichhagen, Departmental Group Leader of the Max-Planck Institute for Medical Research.

The findings were published in Nature Communications.

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Diabetes-related proteins examined for the first time at high resolution - Drug Target Review