Category Archives: Stem Cell Medicine

Genfit throws in the towel on NASH, exiting a tumultuous field full of bright market prospects and lethal R&D setbacks – Endpoints News

Bob Nelsen had been quietly wondering how to eradicate viruses for years, before one day in 2015, he welcomed a pair of immunologists into the ARCH Venture Partners offices on the 34th floor of Seattles Wells Fargo Building.

Louis Picker and Klaus Frh, professors at Oregon Health & Science University, had by then spent 5 years running around the country in search of funding for their startup, TomegaVax, and Frh, at least, was nearing wits end. The Gates Foundation was interested but told them they needed other investors. Investors told them to come back with more data, pharmaceutical executives said theyre in the wrong game too little money to be made fighting infectious disease. Still, a well-connected board member named Bob More landed them a meeting with the coveted venture capitalist, and so, in a narrow conference room overlooking the Puget Sound, Picker prepared to again explain the idea he had spent 15 years on: re-engineering a benign microbe into the first vaccines for HIV and better ones for hepatitis and tuberculosis.

This lightbulb went on his head, Picker recalled in a recent interview. Most of them just didnt get it. And Bobs hit.

By that point, Nelsen was more than just a venture capitalist. Scraggly and greying but no less opinionated at 52, he was mobbed at biotech conferences, having earned a reputation for crass wisdom and uncanny foresight, for making big bets on big ideas that changed medicine. Those ideas included DNA sequencing, which he first cut a check for in the 90s, and leveraging the immune system to tackle cancer. He earned millions making billion-dollar companies.

Yet for years he had harbored an almost singular obsession: I hate viruses, he told Forbes in 2016. He told me he was pissed off at them. The obsession drove him to his first biotech investment in 1993, for an inhalable flu vaccine approved a decade later and still in use. And it drove him to invest in CAR-T as a potential cure for HIV, years before it proved a wildly effective treatment for some cancers.

Now, listening to Picker talk about T cells and antibodies and the curious biology of cytomegalovirus, Nelsen began wondering if it was time for another bet. Pickers technology was not only promising, he reasoned, it could be the basis of a company that changed how researchers approached viruses. Instead of trying to come up with an antidote for every pathogen, you could do what cancer researchers had learned to do, and harness the immune system to do the work for you.

This wasnt a popular opinion at the time. Its like the least trendy idea in the world, Nelsen told me. People would say, Why the hell are you going into infectious disease?

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Genfit throws in the towel on NASH, exiting a tumultuous field full of bright market prospects and lethal R&D setbacks - Endpoints News

Stem Cell & Regenerative Medicine Specialist – Greenville …

What is regenerative medicine?

Regenerative medicine consists of cutting-edge therapies that concentrate on healing substances found in the human body. Research suggests that regenerative medicine may help your body repair damaged tissues. The two main types of regenerative medicine are PRP serums and MSC therapies.

Your blood is filled with tiny plate-shaped cell fragments called platelets. Platelets are the type of cell your body uses to coagulate your blood to stop a wound from bleeding and to help rebuild tissue as you heal. Platelets are among the solids that make up your blood.

The liquid part of your blood is called plasma. When your licensed medical expert makes a PRP serum, they start by taking a small donation of blood from your arm. They spin that in a sterile centrifuge to separate the platelets from the plasma.

Your medical expert mixes all of the healing platelets into a much smaller amount of your plasma. You then have a concentration of healing platelets thats about 5-10 times stronger than your blood.

At the Carolinas Center for Advanced Management of Pain, MSC therapy is created with donated, pre-approved, and pre-screened stem cells derived from the umbilical cords of healthy, full-term, live births. Umbilical cords are rich in mesenchymal stem cells but are usually discarded.

MSCs are blank slate cells that have the ability to transform themselves into other cells. When injected into an area of injury, MSCs have the potential to become tendon cells, bone cells, or skin cells, among others.

If youre getting PRP, your licensed medical practitioner first uses a syringe to withdraw blood and create the serum. The entire process takes about 15 minutes.

When your PRP or MSC serum is ready, your expert injects it directly into your area of pain or damage. The healing platelets or MSCs are thought to accelerate your own bodys healing process, helping it to rebuild tissues with new proteins and cells. Research has shown that as your body heals the underlying injury, your pain may be reduced, too.

To find out if regenerative medicine is the right therapy for you, contact the Carolinas Center for Advanced Management of Pain office nearest you. Phone the friendly staff or use the convenient online scheduler.

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Stem Cell & Regenerative Medicine Specialist - Greenville ...

Another Voice: Terminally ill New Yorkers need a peaceful dying option – Buffalo News

The other one of us, Deborah Stern, suffers from myelodysplastic syndrome/chronic myelomonocytic leukemia. I want very much to live as long as I can and to be there for my family and friends. I am on weekly chemo to try to give myself more time. Ultimately, the only possibility of a cure for me would be a stem cell transplant and so far there have been no donor matches for me located in the world registries.

I dont believe God wants people to suffer. Knowing I have a peaceful dying option would enable me to live the rest of my life with a much better quality of life, taking the worry of increased suffering off the table.

It gave us great comfort that Gov. Andrew M. Cuomo recognized our reality when he announced his support for the Medical Aid in Dying Act last year: Pass the bill the older we get and the better medicine gets, the more weve seen people suffer for too, too long, he said. But we need our Assembly and Senate representatives to act before its too late for us.

Most New Yorkers, including doctors, agree. They also support medical aid in dying, according to recent polling by Quinnipiac University and Medscape-WebMD, respectively.

Washington, D.C., and nine states allow terminally ill adults to have the option of medical aid in dying, including New Yorks neighbors in Maine, New Jersey and Vermont.

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Another Voice: Terminally ill New Yorkers need a peaceful dying option - Buffalo News

Cleave Therapeutics Announces Commencement of a Phase 1 Clinical Study of CB-5339, A Valosin-Containing Protein (VCP)/p97 Inhibitor, in Patients with…

Details Category: Small Molecules Published on Tuesday, 28 July 2020 17:27 Hits: 114

SAN FRANCISCO, CA, USA I July 28, 2020 I Cleave Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on VCP/p97 as a novel target in oncology, announced today that the first patient has been dosed with CB-5339 in a Phase 1 clinical trial of patients with relapsed/refractory acute myeloid leukemia (AML) or relapsed/refractory intermediate or high-risk myelodysplastic syndrome (MDS). CB-5339 is a potent and selective, second-generation, oral small molecule inhibitor of VCP/p97.

We are pleased to begin patient enrollment of our clinical study in AML and MDS, which marks an important milestone in evaluating the initial safety and potential therapeutic benefit of VCP inhibitors for patients with cancer, said Amy Burroughs, president and chief executive officer of Cleave. This clinical program stems from more than a decade of research by scientists who have identified VCP/p97 as a pan-cancer core fitness target that is essential for cancer cell growth and survival.

The primary objectives of the Phase 1 study are to characterize the safety and tolerability and determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose of CB-5339. Additional objectives are to examine the pharmacokinetic parameters and estimate the anti-tumor effects of CB-5339. CB-5339 is administered orally for four days, followed by a three-day treatment-free period weekly in successive 28-day cycles. The trial is expected to enroll 50 to 60 patients in the United States and Australia and does not select or exclude patients based on tumor genetic profile.

Despite several new drug approvals in the last several years, there remains an urgent need for well-tolerated, effective therapies for patients with myeloid malignancies, said Courtney DiNardo, MD, MSCE, associate professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. We are eager to study CB-5339 as a unique oral therapy that could benefit our AML and MDS patients perhaps even regardless of their mutational status - by disrupting stress pathways that are critical to cancer cells.

In addition to the AML/MDS trial, the National Cancer Institute (NCI), part of the National Institutes of Health, is sponsoring a Phase 1 clinical trial with CB-5339 in solid tumors and lymphomas. For more information on both of these trials, please visit the AML/MDS study or the Solid Tumors/Lymphomas study at http://www.clinicaltrials.gov.

About Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)

Acute myeloid leukemia is a type of cancer of the blood and bone marrow, caused by mutations in the genetic material (DNA) of myeloid stem cells which result in the formation of leukemic cells. These cells, also referred to as AML cells, cannot mature into fully functional blood cells, and they multiply uncontrollably. Nearly 20,000 newly diagnosed acute myeloid leukemia patients and 12,000 deaths are expected from AML in the U.S. this year. Myelodysplastic syndromes are a group of diverse bone marrow disorders in which the bone marrow does not produce enough healthy blood cells. Nearly 15,000 newly diagnosed cases of MDS are expected per year.

About Cleave Therapeutics

Cleave Therapeutics is a clinical-stage biopharmaceutical company focused on VCP/p97 as a novel target in protein homeostasis and cellular stress pathways for therapeutic use in cancer. The privately held company, based in San Francisco, is studying CB-5339, its second-generation, small molecule VCP/p97 inhibitor, in a Phase 1 clinical trial in acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), while the National Cancer Institute (NCI) is sponsoring and evaluating CB-5339 in a Phase 1 clinical trial of patients with solid tumors and lymphomas. Cleave investors include 5AM Ventures, Bristol-Myers Squibb, Orbimed, U.S. Venture Partners (USVP), Arcus Ventures, Astellas Venture Management, and Osage University Partners. For additional information, visit http://www.cleavetherapeutics.com.

SOURCE: Cleave Therapeutics

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Cleave Therapeutics Announces Commencement of a Phase 1 Clinical Study of CB-5339, A Valosin-Containing Protein (VCP)/p97 Inhibitor, in Patients with...

Trump looks to hammer down drug prices with 4 executive orders – Endpoints News

President Donald Trump signed 4 executive orders targeting the pharmaceutical industry Friday afternoon, four months before voters head to the polls.

The presidential directives are part of Trumps long-standing goal to reduce prescription drug prices in the US. As the calendar turns toward November, the president has shifted to executive action over working with Congress to reach this objective.

Whether or not such action will result in any meaningful changes before the election remains to be seen, as thepharma industry continues to be a lobbying force on Capitol Hill. The executive orders are also likely to be challenged in court.

The most controversial order reportedlyattempts to cap some prices Medicare pays to the price in foreign countries, which are usually significantly lower. The text wasnt immediately available.

These most-favored nations clauses are akin to price controls in pharma circles, and Trump threatened drug companies by imposing this order within a month if the companies didnt lower prices on their own. How Trump can enact the clause without Congress remains unclear.

One of pharmas most influential lobbyist groups, PhRMA, unsurprisingly reacted negatively to the developments. Heres PhRMA president Stephen Ubl:

In his 2020 State of the Union Address, President Trump declared that we will never let socialism destroy American health care.

Yet, in the middle of a global pandemic, when nearly 145,000 Americans have lost their lives and millions of others have suffered untold economic hardships, this administration has decided to pursue a radical and dangerous policy to set prices based on rates paid in countries that he has labeled as socialist, which will harm patients today and into the future.

The research-based biopharmaceutical industry has been working around the clock to develop therapeutics and vaccines to treat and prevent COVID-19. The administrations proposal today is a reckless distraction that impedes our ability to respond to the current pandemic and those we could face in the future. It jeopardizes American leadership that rewards risk-taking and innovation and threatens the hope of patients who need better treatments and cures.

The presidents attempt to open our country up to socialized health care sets America, our economic recovery and scientific progress back at a time when we need them most.

BIOs chief Michelle McMurry-Heath also slammed the move, saying she was dumbfounded that the Trump administration would seek to cripple the industry trying to end the COVID-19 pandemic.

During the press conference Trump said top pharma executives will meet with administration officials next week.

A second order is an extension of a plan introduced late last year that would allow states, drug wholesalers and pharmacies to re-import drugs from Canada at a lower cost. Pharma lobbyists claim that such plans would not be able to guarantee the safety of the US drug supply chain, a stance that has been met with skepticism, while Canada isnt particularly interested in acting as a proxy between drug companies and states.

The other two orders target insulin pricing and the rebates drug wholesalers and pharmaceutical benefit managers receive when selling to insurers.

The lobbyist group PCMA has already come out in opposition to the latter measure, with president JC Scott saying in a statement, This policy does nothing to address drug prices, it only serves to create uncertainty and raise premiums for seniors while imposing nearly $400 billion in additional taxpayer costs, all at a time when taxpayers are already footing additional costs to counter the pandemic and the national debt is at record levels.

Drug prices have been a pet issue for Trump, and the president reportedly expressed his frustration with HHS secretary Alex Azar in January saying the Democratic Party will kill us on the issue. The COVID-19 pandemic has further complicated the issue, as Trumps approval regarding how hes handling the coronavirus is deep in the red, and the White House has been funneling cash into the industry to fuel the research and development of potential vaccines.

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Trump looks to hammer down drug prices with 4 executive orders - Endpoints News

Therapeutic Solutions International Files Patent on Ability of QuadraMune Ingredient Pterostilbene to Reduce Inflammation while Stimulating Brain…

OCEANSIDE, Calif., July 28, 2020 /PRNewswire/ --Therapeutics Solution International, Inc., (OTC Markets: TSOI), reported new data demonstrating that the blueberry derived compound pterostilbene possesses numerous brain protective and potentially brain regenerative activities. Pterostilbene is one of four ingredients which comprise QuadraMune, a nutraceutical being developed by the Company which is currently in a clinical trial for prevention of COVID-19. Information on the clinical trial can be found on the Government Clinical Trials website https://clinicaltrials.gov/ct2/show/NCT04421391.

"These new data confirm previous findings published on pterostilbene and also reveal novel therapeutic properties of this naturally occurring nutrient," said Dr. James Veltmeyer, Chief Medical Officer of the Company and Voted Top Doctor of San Diego. "By continually investigating mechanisms of action of our products, our ambition is to ensure we have support of the top medical and scientific leaders. In my opinion, many times, it does not matter how good your product is, what matters is that you have scientific basis for why it works. This is especially important in the current age of molecular medicine."

The data disclosed by the Company indicates:

a) pterostilbene suppresses inflammatory cytokines TNF-alpha, IL-1 beta and IL-6;

b) pterostilbene inhibits death of neurons caused by inflammatory mediators;

c) pterostilbene stimulates production of regenerative factors from cells in the brain such as BDNF, NGF, FGF-1, and FGF-2; and

d) pterostilbene allows/enhances proliferation of endogenous brain stem cells.

"The importance of possessing a deep scientific understanding of your product can be seen in the example of nutraceuticals which ended up obtaining regulatory clearance as drugs," said Timothy Dixon, President and CEO of Therapeutic Solutions International. "For example, Lovaza and Vascepa are both fish oil based products which have been developed scientifically and FDA approved through clinical trials. Lovaza, which is sold by GSK, had sales of approximately 1 billion per year and is used for reduction of triglycerides.1 Vascepa, sold by Amarin, had sales in 2019 of 410 million and is used to prevent heart attacks.2 We believe that similar developmental pathways may be possible with several of our existing products."

The Company is currently pursuing FDA clearance for its cell therapy produce StemVacs, for which an IND number was previously granted. The Company's in-licensed stem cell, the JadiCell has FDA clearance for other indications.

About Therapeutic Solutions International, Inc.Therapeutic Solutions International is focused on immune modulation for the treatment of several specific diseases. The Company's corporate website is http://www.therapeuticsolutionsint.com, our public forum is https://board.therapeuticsolutionsint.com/.

1http://www.pmlive.com/pharma_news/teva_launches_first_generic_of_gsks_lovaza_in_us_559273 2https://www.fool.com/investing/2020/01/07/amarin-reports-preliminary-vascepa-sales-projects.aspx

Therapeutic Solutions International, Inc. [emailprotected]

SOURCE Therapeutic Solutions International

therapeuticsolutionsint.com

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Therapeutic Solutions International Files Patent on Ability of QuadraMune Ingredient Pterostilbene to Reduce Inflammation while Stimulating Brain...

Duvelisib May Reduce Lung Inflammation in Severe COVID-19 Cases – OncLive

An Emory University-led phase 2 study will evaluate whether the the PI3K-gamma/delta inhibitor duvelisib (Copiktra) is effective at reducing lung inflammation in patients with severe novel coronavirus 2019 (COVID-19), thereby decreasing incidences of mechanical ventilation and death in these patients.1,2

Investigators theorize that the agent will not only work to reduce inflammation and pulmonary edema, but could also potentially decrease duration of viremia and prompt a response from immunoglobulin G (IgG) and immunoglobulin M (IgM) antibodies that could in turn respond to SARS-Cov-2 spike proteins.

The increase in proteins can lead to a cytokine storm, resulting in severe pneumonitis and acute respiratory distress syndrome (ARDS), and can be fatal.1 Investigators hope to reduce the incidence rate of death or mechanical ventilation intervention, which is 40%, to 15% with the use of duvelisib.

Duvelisibs use in COVID-19 comes not from its anti-cancer properties, but its activity in the immune system. Particularly, we found that it can enhance the growth and expansion of T-cells that retain anti-cancer activity, principal study investigator Edmund K. Waller, MD, PhD, a professor in the Departments of Medicine, Pathology, and Hematology and Medical Oncology at Emory University School of Medicine, as well as the medical director of the Center for Stem Cell Processing and Apheresis Emory University Hospital, said in an interview with OncLive. Results from clinical trials in patients with lymphoma show that these patients had lower levels of cytokinesthe inflammatory proteins we believe are a part of COVID-19 pathologywhen theyve been treated with duvelisib compared with other anticancer therapies.

In September 2018, the FDA has approved duvelisib for the treatment of patients with relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or relapsed/refractory follicular lymphoma.

Waller added that duvelisib may help T cells grow and potentially fight COVID-19, while simultaneously modulating the inflammation that could lead to fluid accumulation in patients lungs.

In the study, duvelisib will be administered to patients with COVID-19 over the course of 2 weeks, and investigators will evaluate the agents efficacy in not only reducing the need for mechanical ventilation and the risk of death within 30 days of treatment.

Additional end points include the need for high-flow oxygen devices, supplemental oxygen, and hospitalization with or without ongoing medical care. The targeted enrollment is 80 adults whose ages are 18 years and older who have been confirmed to have severe COVID-19 symptoms, such as pneumonia and difficulty breathing. Patients will then be randomized to either duvelisib (n = 40) or placebo (n = 40). Waller said he hopes to determine whether or not the agent will be effective in managing the virus to prevent patients from being admitted to the intensive care unit. Duvelisib, he added, could provide a valuable third line of defense after the infection has been established.

In a preclinical pharmacology setting, duvelisib was shown to be highly potent, with investigators noting a whole blood IC50 of 0.36 nM for delta subunit inhibition and whole blood IC50 of 19.6nM for gamma subunit inhibition. The agent was shown to double T cells in both patients with CLL, as well as healthy volunteers from 40 nM to 0.4 mM over 9 to 14 days.

Should the study results be positive, Waller said that the data warrant a need for a larger, multicenter study to further confirm the findings. Even in the event of a smaller, but still significant reduction by one-third or more of patients, the research could be expanded to include hundreds of patients with severe COVID-19, he said.

This is a challenging time, concluded Waller. There are more [factors about this virus] that we dont know than we do know. We have to be cautious as new data become available [to ensure] that its rigorously looked at in order to change medical practices based on solid clinical data. Our study is small, and I wont say that it will change clinical practice, but hopefully its a step towards finding better ways to treat these patients.

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A breakthrough: Stem cell therapy cures Covid-19 The Manila Times – The Manila Times

SIX patients who were suffering from the acute coronavirus disease 2019 (Covid-19) were cured through the stem cell technology using blood from preserved umbilical cords.

The revolutionary method of curing the dreaded virus, which originated in China and has spread worldwide, was announced by The Medical City (TMC) hospital in Pasig City.

Health Secretary Francisco Duque 3rd was informed of the breakthrough in treating the pandemic illness through a letter sent by Dr. Eugenio F. Ramos, TMC president and chief executive officer, on Thursday, July 23.

Our government, particularly the Department of Health, can take pride in the fact that the Philippines is not behind at all in innovative practice in medicine, said Ramos in his letter to Duque.

The TMC has been experimenting with stem cell therapy since 2015 through its Institute of Personalized Molecular Medicines stem cell program.

The stem cell experiment was made on seven Covid-19 patients who were so ill they were about to be intubated.

Intubation means inserting a tube into the patients mouth that reaches the trachea to allow him/her to breathe through a lung machine.

Six of the seven patients had recovered, the TMC said; one was so ill he died during the procedure.

As I told you this morning, we would have wanted to complete at least 10 Covid-19 cases before informing you, but it might be taken against us if we kept this knowledge from the public too long while patients are dying and no single drug seems to work, Dr. Ramos told Duque.

Ramos simplified for the public how the stem cell therapy works:

Stem cells are very young cells from the body that can be differentiated into whatever cell lines (lung, heart, kidney, liver, skin, etc.) that the body needs when it needs them, e.g., when an organ is injured or deteriorates. Stem cells can be triggered or infused to do [repair] work.

In severe Covid-19 cases, what triggers a cascade of inflammatory cells (called cytokines) is the injury caused by the virus. The lungs, in particular, become inflamed and the respiratory function rapidly deteriorates leading to death.

Before the cascade happens in which case the patient is in a severe condition probably requiring intubation the infusion of mesenchymal stem cells collected [from] the umbilical cord and grown in [the regenerative medicine laboratory of TMC into the patients veins, takes place.

A total of four infusions are given over a short period of a few days. These messenchymal stem cells rally the cells in the lungs to fight the cytokines.

In short, the stem cells from the umbilical cord produce new cells to replace those damaged by the virus in the lungs, leading to the patients recovery.

The umbilical cord is rich in nutrients to rebuild destroyed tissues, according to Ramos.

TMCs stem cell program also makes use of bone marrow taken from a patients spine to cure cancer.

But the bone marrow stem cell is also used to delay the aging process in elderly patients.

TMC had the most number of bone marrow transplantation for blood cancers, with more than 90 percent survival rate, said Dr. Ramos.

Since 2015, a total of 112 patients have enrolled in TMCs regenerative medicine program.

Of the total, 57 percent were cancer patients and 43 percent were non-cancer patients, said TMCs Dr. Michelle Joy Baldorado-de Vera.

Oncologists (doctors who specialize in cancer) are part of the regenerative medicine program Alan Olavere, Marina Chua-Tan and Josephine Tolentino.

Other members are hematologists (blood doctors) Norma Ona and Alma Calavera, neurologists John Tiongson and Marc Joseph Buensalido, and immunologist Michelle de Vera.

Dr. Sam Bernal, a Filipino American who grew up in the United States, is a consultant to the program.

Bernal is a molecular scientist and an oncologist who has been into stem cell technology for decades.

He was part of the foreign group that was tapped by the government for technology transfer.

Former Health secretary Alfredo Bengzon, who is a part owner of TMC, engaged Bernal to pioneer stem cell therapy in the country.

TMC has had foreign patients who underwent stem cell therapy, but refuses to disclose their number.

As is the norm among Filipinos who have the crab mentality, TMCs stem cell program has been criticized by fellow doctors.

Weve been defending the program from the outset, said Dr. Ramos.

But its not only TMC that has been into stem cell therapy; the other hospitals are the Makati Medical Center and the governments National Kidney and Transplant Institute.

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A breakthrough: Stem cell therapy cures Covid-19 The Manila Times - The Manila Times

UAE hails success of first bone marrow transplant – The National

The UAE is celebrating the success of its first bone marrow transplant.

Typically used to treat patients with blood and cancer disorders , the landmark procedure was carried out on a patient with multiple myeloma, a form of blood cancer.

While cancer is the third-highest cause of death across the Emirates, many Emirati citizens and residents have sought treatment abroad for cell therapy and regenerative medicine.

News of the successful transplant, which was the result of collaboration between Abu Dhabi Stem Cells Centre and Sheikh Khalifa Medical City, represents a major breakthrough for cancer patients living in the UAE who can now seek treatment closer to home to remain with family.

Researchers work to develop medication that can be used to treat Covid-19 patients, at Abu Dhabi Stem Cell Centre. Wam Twitter

Researchers work to develop medication that can be used to treat Covid-19 patients, at Abu Dhabi Stem Cell Centre. Wam Twitter

Researchers work to develop medication that can be used to treat Covid-19 patients, at Abu Dhabi Stem Cell Centre. Wam Twitter

Researchers work to develop medication that can be used to treat Covid-19 patients, at Abu Dhabi Stem Cell Centre. Wam Twitter

The doctors and researchers at Abu Dhabi Stem Cell Centre who were involved in the development of a potential Covid-19 treatment. CourtesyAbu Dhabi Stem Cell Centre

The milestone announcement comes as the centre launched the Abu Dhabi Bone Marrow Transplant programme on Monday.

"It fills us with great pride to be part of a first for the country and to make such a life-saving treatment available and accessible to those who need it here," said Dr Fatima Alkaabi, executive Director of the Abu Dhabi Bone Marrow Transplant programme.

"We are very grateful for the support and collaboration of SKMC in making this opportunity possible."

Bone marrow transplants are one of the most established stem cell-based therapies in the treatment of cancer, particularly blood cancers.

The unique procedure developed by the centre involved harvesting peripheral blood stem cells from the patient, who then underwent high-dose chemotherapy to eradicate all cancerous cells and most of the bone marrow.

The harvested stem cells were then infused back into the bloodstream, where they restore the destroyed cells and over the course of two weeks restart the production of healthy non-malignant blood cells.

"The patient is basically without an immune system while waiting for the transfused cells to take effect and must remain in isolation under the strictest infection control guidelines," said Dr Yendry Ventura, general manager of the stem cell centre and Abu Dhabi bone marrow transplant programme director.

"Since we are still in the midst of a global pandemic, we took additional precautions to ensure the best outcome possible for all involved."

Abu Dhabi Stem Cells Centre has been a driving force behind medical research in the country, including playing a key role in the ongoing fight against Covid-19.

Earlier last month, the team announced the results of the trial of its treatment for Covid-19 patients, branded UAECell19.

After opening in December, the centre has already received international press coverage over to its research into a treatment for the virus.

Their groundbreaking work has involved taking stem cells from a patients blood and returning them, via a nebuliser, as a fine mist to the lungs.

There they help regenerate lung cells and improve the body's immune response by preventing an overreaction to the infection that can damage healthy cells.

Updated: July 27, 2020 05:14 PM

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UAE hails success of first bone marrow transplant - The National

Drug target for aggressive breast cancer found by team of UK and US scientists – iNews

The scientists said they were excited by their findings (Photo: ERIC PIERMONT/AFP/Getty)

A team of British and American scientists have discovered a way to slow the growth of breast cancer stem cells in the lab, which could eventually lead to combination drug therapies on previously untreatable forms of the disease.

Around three quarters of women who have breast cancer have what are known as oestrogen receptor positive tumours. Some breast cancer cells have receptors that bind to the hormone oestrogen and depend on it to grow. Though anti-oestrogen drugs such as tamoxifen and fulvestrant are able to prevent reoccurrence in most of these breast cancers, tumours reoccur in 1 out of 4 cases.

Many of the women relapse after several years, because some of the cancer cells remain after treatment. The cells, called cancer stem cells, lay dormant in the body and cannot be targeted by anti-oestrogen therapies.

Scientists have now found that cancer stem cells resistant to anti-oestrogen drugs express an immune system-related receptor, called interleukin 1 receptor. They also found that Anakinra, a drug used to treat rheumatoid arthritis, was able to reduce the ability of the cancer stem cells to grow.

However, the team said further research in animals and humans is needed before they can confirm if drugs targeting interleukin 1 receptor could be used as an effective treatment.

Scientists from the Universities of Michigan, Liverpool and Edinburgh were closely involved in the study led by Dr Bruno Simes and Professor Rob Clarke from The University of Manchester

Dr Simes said: Resistance to anti-oestrogen therapies in breast cancer patients is a major problem and one which cancer scientists have been trying to address for many years. Many drugs have been investigated by scientists as a possible combination therapy taken with anti-oestrogen therapies and we are still searching for a solution for these patients.

However, most of these drugs target actively dividing cells whereas we are trying to also target cells that are in a dormant state. So we hope that these women who have increased numbers of cancer stem cells and do not respond to currents treatment could one day benefit from combination therapy.

Theres a very long way to go before we can say if this approach will help these women but we are excited by our findings.

The study is published in the journal Stem Cell Reports.

Justin Stebbing, professor of cancer medicine and medical oncology at Imperial College London, said: Stem cells represent a very important target to understand better and eradicate, because they are responsible for some of the most worrying aspects of cancer, such as its ability to regrow, or be resistant to treatment. They are also a rare population but this laboratory research shows that cancer stem cells have on their surface a protein that we have a drug for interestingly one that is being trialled in Covid-19 too.

Hopefully, we can in the future link this laboratory work to research in women in the clinic, helping to reduce recurrence rates by eradicating rare stem cell populations that contribute to recurrence.

Baroness Delyth Morgan, chief executive at Breast Cancer Now, which helped fund the study, said: Its promising that targeting this protein [interleukin-1 receptor] may help reduce the growth of breast cancer stem cells, which are thought to be central to the disease spreading around the body, where it becomes incurable.

The development of resistance to hormone therapies remains a major challenge in the clinic. With around 11,500 women still dying from breast cancer each year in the UK, we urgently need to find new ways to prevent the disease spreading and to treat it more effectively when it does.

This exciting discovery highlights the importance of research to understand the molecular detail of how breast cancer spreads, and of investigating the potential of existing drugs to try to stop it. In particular, these findings offer further evidence that arthritis drugs such as Anakinra may be useful additions in treating breast cancer, and we hope further research can now help us fully understand their potential and whether and how they might work as a cancer treatment.

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Drug target for aggressive breast cancer found by team of UK and US scientists - iNews