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Category Archives: Stem Cell Therapy

Hemostemix Announces the Appointment of Dr. Renzo Cecere, MD, FRCSC to Its Scientific Advisory Board – Yahoo Finance

Calgary, Alberta--(Newsfile Corp. - September 14, 2022) - Hemostemix Inc. (TSXV: HEM) (OTCQB: HMTXF) ("Hemostemix" or the "Company") is pleased to announce the appointment of Dr. Renzo Cecere, MD, FRCSC, to its Scientific Advisory Board.

Dr. Cecere is the McGill University Chief of Cardiac Surgery, Surgical Director of the Heart Failure and Heart Transplantation Program, and Director of the Mechanical Circulatory Support Program. He is also Associate Member of the McGill University Department of Mechanical Engineering, and a Director and Principal Investigator of the Research Institute of the MUHC Myocardial Regeneration Laboratory.

For over a decade, Dr. Cecere's lab has been investigating novel methods to strengthen the stem-cell induced regeneration of infarcted heart tissue. Dr. Cecere has utilized placenta-derived stem cells and investigated their regenerative potential in different animal models of myocardial infarction ("MI"). More recently, Dr. Cecere's lab is actively involved in a project to create a platform to generate patient-specific cardiomyocytes from the blood of patients with heart failure. In Dr. Cecere's recent project (under review, Journal of Tissue Engineering and Regenerative Medicine), the team encapsulated placenta derived stem cells in a hydrogel scaffold and implanted it in a rat MI model. The stem cell/scaffold composite enhanced several parameters of cardiac function, including ejection fraction and fractional shortening, while also reducing fibrosis and increasing angiogenesis. In fact, Dr. Cecere's lab recently published a systematic review and meta-analysis that demonstrated that stem cells combined with bioactive scaffolds provide enhanced tissue regeneration in animal models of MI, compared to stem cells injected alone. This study paves the way for future research and clinical trials, supporting the use of ACP-01-based bioactive scaffolds to improve the stem cell-induced repair after a MI.

"I have worked in the field of heart-based stem cell science for more than a decade, and I find ACP-01's unique properties, safety profile and statistically significant preliminary intramyocardial efficacy results to be very promising" said Dr. Cecere. I look forward to collaborating with management to create the best product to repair hearts before an infarct or following an infarct, and designing a clinical trial of ACP-01 that proves its efficacy" said Dr. Cecere.

"Hemostemix is delighted to welcome Dr. Cecere to our team. His appointment to the SAB is the first of many areas of collaboration. As one of Canada's most well-regarded stem cell focused heart transplant surgeons, Dr. Cecere and his team enable Hemostemix to fast-track product development and clinical trials. We very much look forward to his counsel and his teams' collaboration to trial ACP-01 based bioactive scaffolds to improve stem cell-induced repair of the heart," stated Thomas Smeenk, CEO.

ABOUT HEMOSTEMIX

Hemostemix is an autologous stem cell therapy company, founded in 2003. A winner of the World Economic Forum Technology Pioneer Award, the Company has developed, patented, and is scaling a patient's blood-based stem cell therapeutics platform that includes angiogenic cell precursors, neuronal cell precursor and cardiomyocyte cell precursors. Seven studies including 260 ACP-01 recipients define its safety and efficacy profile as a treatment for heart diseases such as Dilated and Ischemic Cardiomyopathy, Angina, and diseases of Ischemia such as Critical Limb Ischemia. The Company owns 91 patents across five patent families. For more information, please visit http://www.hemostemix.com.

For further information, please contact: Thomas Smeenk, President, CEO & Co-FounderEM: tsmeenk@hemostemix.com PH: 905-580-4170

Neither the TSX Venture Exchange nor its Regulation Service Provider (as that term is defined under the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Forward-Looking Information: This news release contains "forward-looking information" within the meaning of applicable Canadian securities legislation. All statements, other than statements of historical fact, included herein are forward-looking information. In particular, this news release contains forward-looking information in relation to: the lead product ACP-01, future studies of ACP-01 in bioactive scaffolds to improve the stem cell-induced repair after an infarct, the company's Clinical Trial results, and the results of the retrospective study of ischemic and dilated cardiomyopathy, and the commercialization of ACP-01. There can be no assurance that such forward-looking information will prove to be accurate. Actual results and future events could differ materially from those anticipated in such forward-looking information. This forward-looking information reflects Hemostemix's current beliefs and is based on information currently available to Hemostemix and on assumptions Hemostemix believes are reasonable. These assumptions include, but are not limited to: the underlying value of Hemostemix and its Common Shares; the successful resolution of the litigation that Hemostemix is pursuing or defending (the "Litigation"); the results of ACP-01 research, trials, studies and analyses, including the analysis being equivalent to or better than previous research, trials or studies; the receipt of all required regulatory approvals for research, trials or studies; the level of activity, market acceptance and market trends in the healthcare sector; the economy generally; consumer interest in Hemostemix's services and products; competition and Hemostemix's competitive advantages; and Hemostemix obtaining satisfactory financing to fund Hemostemix's operations including any research, trials or studies, and any Litigation. Forward-looking information is Subject to known and unknown risks, uncertainties and other factors that may cause the actual results, level of activity, performance or achievements of Hemostemix to be materially different from those expressed or implied by such forward-looking information. Such risks and other factors may include, but are not limited to: the ability of Hemostemix to complete clinical trials, complete a satisfactory analyses and file the results of such analyses to gain regulatory approval of a phase II or phase III clinical trial of ACP-01; potential litigation Hemostemis mayface; general business, economic, competitive, political and social uncertainties; general capital market conditions and market prices for securities; delay or failure to receive board or regulatory approvals; the actual results of future operations including the actual results of future research, trials or studies; competition; changes in legislation affecting Hemostemix; the timing and availability of external financing on acceptable terms; long-term capital requirements and future developments in Hemostemix's markets and the markets in which it expects to compete; lack of qualified, skilled labour or loss of key individuals; and risks related to the COVID-19 pandemic including various recommendations, orders and measures of governmental authorities to try to limit the pandemic, including travel restrictions, border closures, non-essential business closures service disruptions, quarantines, self-isolations, shelters-in-place and social distancing, disruptions to markets, disruptions to economic activity and financings, disruptions to supply chains and sales channels, and a deterioration of general economic conditions including a possible national or global recession or depression;the potential impact that the COVID-19 pandemic may have on Hemostemix which may include a decreased demand for the services that Hemostemix offers; and a deterioration of financial markets that could limit Hemostemix's ability to obtain external financing. A description of additional risk factors that may cause actual results to differ materially from forward-looking information can be found in Hemostemix's disclosure documents on the SEDAR website at http://www.sedar.com. Although Hemostemix has attempted to identify important factors that could cause actual results to differ materially from those contained in forward-looking information, there may be other factors that cause results not to be as anticipated, estimated or intended. Readers are cautioned that the foregoing list of factors is not exhaustive. Readers are further cautioned not to place undue reliance on forward-looking information as there can be no assurance that the plans, intentions or expectations upon which they are placed will occur. Forward-looking information contained in this news release is expressly qualified by this cautionary statement. The forward-looking information contained in this news release represents the expectations of Hemostemix as of the date of this news release and, accordingly, it is Subject to change after such date. However, Hemostemix expressly disclaims any intention or obligation to update or revise any forward-looking information, whether as a result of new information, future events or otherwise, except as expressly required by applicable securities law.

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/137137

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Hemostemix Announces the Appointment of Dr. Renzo Cecere, MD, FRCSC to Its Scientific Advisory Board - Yahoo Finance

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NIH researchers develop gene therapy for rare ciliopathy – National Institutes of Health (.gov)

News Release

Thursday, September 8, 2022

Gene augmentation rescues cilia defects in light-sensing cells derived from patients with blinding disease.

Researchers from the National Eye Institute (NEI) have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood. Using patient-derived retina organoids (also known as retinas-in-a-dish), the researchers discovered that a type of LCA caused by mutations in the NPHP5 (also called IQCB1) gene leads to severe defects in the primary cilium, a structure found in nearly all cells of the body. The findings not only shed light on the function of NPHP5 protein in the primary cilium, but also led to a potential treatment for this blinding condition. NEI is part of the National Institutes of Health.

Its so sad to see little kids going blind from early onset LCA. NPHP5 deficiency causes early blindness in its milder form, and in more severe forms, many patients also exhibit kidney disease along with retinal degeneration, said the studys lead investigator, Anand Swaroop, Ph.D., senior investigator at the NEI Neurobiology Neurodegeneration and Repair Laboratory. Weve designed a gene therapy approach that could help prevent blindness in children with this disease and one that, with additional research, could perhaps even help treat other effects of the disease.

LCA is a rare genetic disease that leads to degeneration of the light-sensing retina at the back of the eye. Defects in at least 25 different genes can cause LCA. While there is an available gene therapy treatment for one form of LCA, all other forms of the disease have no treatment. The type of LCA caused by mutations in NPHP5 is relatively rare. It causes blindness in all cases, and in many cases it can also lead to failure of the kidneys, a condition called Senior-Lken Syndrome.

Three post-doctoral fellows, Kamil Kruczek, Ph.D., Zepeng Qu, Ph.D., and Emily Welby, Ph.D., together with other members in the research team collected stem cell samples from two patients with NPHP5 deficiency at the NIH Clinical Center. These stem cell samples were used to generate retinal organoids, cultured tissue clusters that possess many of the structural and functional features of actual, native retina. Patient-derived retinal organoids are particularly valuable because they closely mimic the genotype and retinal disease presentation in actual patients and provide a human-like tissue environment for testing therapeutic interventions, including gene therapies. As in the patients, these retinal organoids showed defects in the photoreceptors, including loss of the portion of the photoreceptor called outer segments.

In a healthy retina, photoreceptor outer segments contain light-sensing molecules called opsins. When the outer segment is exposed to light, the photoreceptor initiates a nerve signal that travels to the brain and mediates vision. The photoreceptor outer segment is a special type of primary cilium, an ancient structure found in nearly all animal cells.

In a healthy eye, NPHP5 protein is believed to sit at a gate-like structure at the base of the primary cilium that helps filter proteins that enter the cilium. Previous studies in mice have shown that NPHP5 is involved in the cilium, but researchers dont yet know the exact role of NPHP5 in the photoreceptor cilium, nor is it clear exactly how mutations affect the proteins function.

In the present study, researchers found reduced levels of NPHP5 protein within the patient-derived retinal organoid cells, as well as reduced levels of another protein called CEP-290, which interacts with NPHP5 and forms the primary cilium gate. (Mutations in CEP-290 constitute the most common cause of LCA.) In addition, photoreceptor outer segments in the retinal organoids were completely missing and the opsin protein that should have been localized to the outer segments was instead found elsewhere in the photoreceptor cell body.

When the researchers introduced an adeno-associated viral (AAV) vector containing a functional version of NPHP5 as a gene therapy vehicle, the retinal organoids showed a significant restoration of opsin protein concentrated in the proper location in outer segments. The findings also suggest that functional NPHP5 may have stabilized the primary cilium gate.

The study was funded by the NEI Intramural program. Patient samples were collected at the NIH Clinical Center.

NEI leads the federal governments efforts to eliminate vision loss and improve quality of life through vision researchdriving innovation, fostering collaboration, expanding the vision workforce, and educating the public and key stakeholders. NEI supports basic and clinical science programs to develop sight-saving treatments and to broaden opportunities for people with vision impairment. For more information, visit https://www.nei.nih.gov.

About the NIH Clinical Center:The NIH Clinical Center is the worlds largest hospital entirely devoted to clinical research. It is a national resource that makes it possible to rapidly translate scientific observations and laboratory discoveries into new approaches for diagnosing, treating, and preventing disease. Over 1,600 clinical research studies are conducted at the NIH Clinical Center, including those focused on cancer, infectious diseases, blood disorders, heart disease, lung disease, alcoholism and drug abuse. For more information about the Clinical Center, visit:https://www.cc.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Kruczek K, Qu Z, Welby E, et al. In vitro modeling and rescue of ciliopathy associated with IQCB1/NPHP5 mutations using patient-derived cells. Stem Cell Reports. Sept 8, 2022.

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Be The Match and William G. Pomeroy Foundation Announce $500k Gift and Matching Campaign – PR Newswire

The Foundation's donation and fundraising efforts will help diversify the Be The Match Registry

MINNEAPOLIS, Sept. 13, 2022 /PRNewswire/ -- In support of enhanced accessibility to life-saving stem cell transplants through the Be The Match Registry, the William G. Pomeroy Foundation has made a $500,000 grant to Be The Match that will further the organization's partnerships at historically Black colleges and universities (HBCUs).

"Patients are most likely to match a donor of their own ethnic background," said Bill Pomeroy

The $500,000 gift will enable Be The Match to grow its partnerships at HBCUs with focused recruitment efforts, education, awareness and internship opportunities for students; all in pursuit of increased stem cell transplant accessibility for Black and African American patients. Six new campus partnerships have already been established as a result of this gift.

"This generous gift from the Pomeroy Foundation is going to have a lasting impact on our ability to reach Black and African American communities and will make a difference for diverse patients in need of a donor," said Amy Ronneberg, Chief Executive Officer of Be The Match.

In addition, the Pomeroy Foundation donation sponsors a gift matching campaign that will help to diversify the donor registry. The matching opportunity coincides with this year's observance of World Marrow Donor Day on Saturday, Sept. 17. Donations to Be The Match throughout the month of September will be matched dollar for dollar, up to $500,000. Visit this link to donate and have your gift matched. The doubled donation will give patients a greater chance of finding a match and a second chance at life.

"Patients are most likely to match a donor of their own ethnic background," said Bill Pomeroy, Founder and Trustee of the Pomeroy Foundation. "Improving the ethnic diversity of the registry improves all patients' odds of finding a life-saving match."

Currently, the chance of having a matched, available donor on the registry ranges from 29-79%, with people of color less likely to find a match because fewer potential donors with diverse backgrounds are registered.

In 2004, Bill Pomeroy was diagnosed with an aggressive form of leukemia. He received a life-saving transplant from a fully matched donor found on the Be The Match Registry in 2005. Bill's transplant experience served as a catalyst for the creation of the Pomeroy Foundation, which works closely with Be The Match to expand recruitment efforts, especially for ethnically diverse donors. Bill is Trustee Emeritus for the Be The Match Foundation.

To double your donation to Be The Match during this month's matching gift campaign and to learn more about diversifying the Be The Match Registry, visit: bethematch.org.

About Be The Match:

Be The Match is the leading global partner working to save lives through cellular therapy. With more than 30 years of experience managing the most diverse registry of potential unrelated blood stem cell donors and cord blood units in the world, Be The Match is a proven partner in providing cures to patients with life-threatening blood and marrow cancers and diseases. Through their global network, they connect centers and patients to their best cell therapy options, from blood stem cell transplant to next-generation therapy, and collaborate with cell and gene therapy companies to support therapy development and delivery through Be The Match BioTherapies. Learn more at bethematch.org.

About the Pomeroy Foundation

The William G. Pomeroy Foundationis committed to supporting the celebration and preservation of community history; and working to improve the probability of finding appropriate donor matches or other life-saving treatments for blood cancer patients. Established by Trustee Bill Pomeroy in 2005 to bring together his two greatest passions, the Pomeroy Foundation is a private, philanthropic organization located in Syracuse, N.Y. As the nation's leading funder of historic roadside markers, the Pomeroy Foundation has awarded nearly 2,000 grants for markers and bronze plaques in 46 states and Washington, D.C. To learn more about the Pomeroy Foundation, visit wgpfoundation.org.

Media Contact: Erica SevillaPhone Number: 763-406-8758Email: [emailprotected]Website: bethematch.org

Media Contact: Steve BodnarCommunications ConsultantWilliam G. Pomeroy Foundation[emailprotected]315-913-4068

SOURCE Be The Match; William G. Pomeroy Foundation

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Be The Match and William G. Pomeroy Foundation Announce $500k Gift and Matching Campaign - PR Newswire

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A history of blood cancer treatment – – pharmaphorum

Despite being one of the most common forms of cancer, awareness of blood cancer pales in comparison to other types of the disease. In fact, according to Blood Cancer UK research, more than half of UK adults cannot name a single symptom of blood cancer.

Over the past two centuries, researchers have identified more than 100 different types of blood cancer, while most patients may be familiar with the big three (leukaemia, lymphoma, and melanoma). However, myelodysplastic syndromes and myeloproliferative neoplasms are also prominent types of blood cancer.

Thanks to the dedicated efforts of doctors, patients, carers, and healthcare professionals, people diagnosed with blood cancer are now living longer, with a steady stream of more effective treatments entering the market each year. However, there is still much to be done to achieve a vision wherein all those diagnosed with blood cancer survive.

As we enter Blood Cancer Awareness month, a global event dedicated to spotlighting and supporting efforts to improve awareness, detection, and treatment of blood cancer, we take a look back in celebration of the achievements and breakthroughs that paved the way for todays innovations.

1832 Discovery of Hodgkins and non-Hodgkins lymphoma

Although early accounts of an illness akin to leukaemia can be traced back to Ancient Greece, the first official description of blood cancer didnt appear until 1832, when British pathologist and pioneer of preventative medicine Thomas Hodgkin used the controversial concept of micrology to identify the abnormalities in the lymphatic system.

During his time working in the pathology museum at Guys Hospital in London, Hodgkin studied several preserved specimens of human organs affected by disease. Noticing a pattern in the lymph nodes and spleen that indicated the appearance of disease, he published his findings in a paper entitled, On Some Morbid Appearances of the Absorbent Glands and Spleen.

At the time, his hypothesis appeared to fall on deaf ears, and it would take a further three decades before Hodgkins discovery was recognised.

1844 First reported case of multiple myeloma

The first well-documented case of multiple myeloma was reported in 1844 by renowned British surgeon Samuel Solly. In 39-year-old patient Sarah Newbury, Solly observed the appearance of fatigue and bone pain resulting from multiple fractures. Only four years after the patient first showed symptoms, she died, and an autopsy revealed abnormalities in the bone marrow that closely matched the autopsy findings of 45-year-old Thomas Alexander McBean.

McBeans case is perhaps the most well-known account of multiple myeloma. Similar to Newbury, McBean known to be a highly respected tradesman developed fatigue and severe pain from weak and easily broken bones. After attempts to treat McBeans symptoms through cupping, applying leeches for maintenance therapy, and therapeutic phlebotomy proved unsuccessful, his physician, Dr Thomas Watson, prescribed steel and quinine, while a sample of his urine was sent to chemical pathologist Henry Bence Jones.

Following his death in 1846, histologic examination of McBeans bone marrow revealed a red gelatiniform substance consisting of nucleated cells, some twice the size of an average blood cell.

1847 Virchow links tumours and white blood cells

By the 1840s, histology (the study of microscopic anatomy) was a recognised discipline in the scientific community. Building upon early descriptions of leukaemia by French anatomist and surgeon Alfred-Armand-Louis-Marie Velpeau, in 1847, the father of modern pathology Dr Rudolf Virchow and English physician John Hughes Bennett independently observed abnormal increases in white blood cells in patients.

Virchow was the first to argue that cancer derives from changes in normal cells. Crucially, he observed a connection between certain tumours and inflammation, noting that neoplastic tissues were often covered with leukocytes of the immune system.

As with Hodgkins discovery, Virchows theory went almost unnoticed until the 20th century.

1907 The magic bullet of immunotherapy

In the early 1900s, researchers uncovered the existence of several types of blood cancer. However, effective treatments were not available at the time. During this period, Nobel prize-winning German scientist Paul Ehrlich developed his lock-key hypothesis of molecules that specifically bind to cell receptors.

Further research led Ehrlich to develop his side-chain theory, that antibodies produced by white blood cells act as receptors on the cell membrane. For his contribution, in 1908, Ehrlich received the Nobel Prize for Medicine in the field of immunology, together with the father of innate immunity, Ilia Metschnikow, whose discovery of phagocytosis formed the foundation of cell-mediated immunity.

While they may not have known it at the time, through their work Ehrlich and Metschnikow formed the cornerstone of modern immunology, including chemoreceptor and chemotherapy concepts that revolutionised blood cancer treatment over the following century.

1942 Chemotherapy moves from trenches to treatment

In the aftermath of World War I, medical researchers noticed that the mustard gas used to make chemical weapons for the battlefield also destroyed lymphatic tissue. Early experiments showed that topically applying nitrogen mustard caused tumours to shrink in mice.

Research into the medical potential of mustard gas stagnated until 1942, when two assistant professors at Yale, Louis S Goodman and Alfred Gilman, began to study the effects of nitrogen mustard on lymphoma. Although clinical trials proved that chemicals could be used to treat cancer, the results of the study remained a closely guarded military secret until 1946.

1956 The rise of bone marrow transplants

In a milestone achievement for blood cancer research and treatment, Dr E Donnall Thomas performed the first successful bone marrow transplant in 1956. The procedure involved transplanting bone marrow between identical twins, with tissue taken from the healthy twin given to the other who had leukaemia.

In 1968, the first bone marrow transplant using a matched donor took place at the University of Minnesota. Using a blood test developed by Dr Fritz Bach, Dr Robert Good determined that the patient, a baby with a severe immune deficiency, was a human leukocyte antigen match with his nine-year-old sister.

The ground-breaking approach to donor selection paved the way for future bone marrow transplants, including the first successful bone marrow transplant with unrelated patients in 1973.

Before the birth of bone marrow transplants, patients were often treated using chemotherapy, which could be used to kill cancer cells. However, this also presented a problem: chemotherapy does not discriminate between healthy and cancer cells, meaning that if patients were given sufficient doses to kill the disease, normal cells would also be harmed. With the advent of bone marrow transplantation, these healthy cells could be replaced with donor cells, allowing for higher doses of chemotherapy in treatment.

1980s Emergence of cord blood transplants

Another source of haematological stem cells emerged in the late 80s cord blood stem cells. The remaining blood found within the umbilical cord and placenta after birth is rich in blood-producing stem cells. Cord blood collection has rarely changed since the first successful procedure occurred in 1988.

Stem cells extracted from a donated cord can be frozen for a number of years and quickly accessed when needed. Once the transplant is complete, the cells will travel into the patients bone marrow, where they will begin to grow into normal blood cells.

Recognising the need to identify and match potential donors with patients, in 1989 the Bone Marrow Donors Worldwide programme was established.

Today, the bone marrow donor registry comprises more than 39,527,166 donors and 804,246 cord blood units.

2001 FDA green lights revolutionary treatments

Innovation in blood cancer treatments ushered in a new generation of targeted and precision treatments. One such therapy was Imatinib (also known as Gleevec or Glivec), a first-generation tyrosine kinase inhibitor dubbed a magical bullet, designed to specifically target BCR-ABL tyrosine kinase.

Just over a decade after it was developed by biochemist Nicholas Lyndon, Imatinib received US Food and Drug Administration (FDA) approval in 2001. Since then, it has transformed the treatment of chronic myeloid leukaemia and non-Hodgkins lymphoma.

The following year, the regulator also approved Rituximab, a monoclonal antibody targeting CD-20 positive B-cells, as a companion treatment of chemotherapy in older diffuse large B-cell lymphoma patients.

2002Emergence of CAR-T therapy

Building on the success of cytokine-based immunotherapies, scientists continued to seek other areas where the immune system could be leveraged against tumours. Throughout the 90s, Dr James Allison spearheaded research into T-cell engineering, a revolutionary technique that formed the foundation of chimeric antigen receptor (CAR) T-cell therapy.

Dr Allisons research into the function and application of T-cells in cancer treatment greatly broadened scientific understanding of the immune system. However, the first generation of CAR T-cells proved to be clinically ineffective.

It wasnt until 2002, when Memorial Sloane Kettering Cancer Center scientists Michel Sadelain, Renier Brentjens, and Isabelle Rivire opted to push the boundaries of research, by genetically engineering T-cells with a CAR, that the technique achieved successful results.

This research paved the way for the first successful treatment of a patient with acute lymphoblastic leukaemia in 2011.

2012 The 100,000 Genomics Project begins

Unlocking the secrets of the human genome has intrigued investigators for centuries. However, the technology needed to analyse genomic and long-term clinical data is a relatively recent development. With the launch of the 100,000 Genomes Project in 2012, an international team of researchers studied the role that genes play in health and disease.

For the first time, researchers demonstrated that whole genome sequencing could be used to uncover new diagnoses across the broadest range of rare diseases. This was an entirely new approach to DNA research. Previously, DNA would be segmented into short sections, which would then be read and sequenced separately.

The 100,000 Genomes Project sparked a new wave of research exploring the clinical potential of sequencing long strands of individual DNA without cutting them into sections. With this technique, it is hoped that researchers will gain previously inaccessible insights into cancer, revealing more accurate diagnoses and treatment pathways for patients.

20162022 New treatments enter the market

Over the past few years, the number of treatments approved for blood cancer has skyrocketed. Johnson & Johnsons Darzalex (daratumumab) was a notable development for the sector. The monoclonal antibody first received FDA approval in November 2015 as a monotherapy for patients with multiple myeloma, marking it as the first CD38-directed antibody to receive regulatory approval to treat the disease. It has since gone on to receive numerous approvals for multiple myeloma designations.

As of 2022, more than 800 new cell therapies are being developed for five blood cancers, with the market for oncology cell therapies expected to exceed $37 billion in value globally by 2028.

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Kochi Scientists Win Patent for Novel Nanomedicine that Enables Early Liver Tumour, Cirrhosis Detection & Treatment – The Weather Channel

Representative image.

Researchers from the School of Nanosciences and Molecular Medicine at Amrita Vishwa Vidyapeetham here have won a patent in the US and Australia for a novel nanomedicine that holds great promise for early detection and treatment of liver cirrhosis and liver tumour.

The invention is the outcome of a research project funded by the Nanobiotechnology Task Force of the Department of Biotechnology of the Government of India.

Shantikumar V. Nair and Manzoor Koyakutty from Amrita School of Nanosciences and Molecular Medicine in Kochi led a team to create a special type of nanomedicine that responds to radio wave signals sent from outside the body.

Once the nanoparticles are injected into a tumour, they can be heated up using external, medically approved radio waves. Doctors can visualise the tumour using an MRI machine and burn it off in a controlled manner.

Amrita researchers have demonstrated that the novel technology can be used for early detection of liver cirrhosis and liver tumour, early-stage image-guided treatment of liver tumours using radio-frequency ablation therapy as well as labelling and tracking the movement of stem cells inside the body after stem-cell transplantation to assess how effective the therapy has been.

Koyakutty said they had made a unique nanomedicine that can be used for medical imaging and drug delivery.

"Its particles are made of synthetically prepared calcium phosphate, a biomineral present in our bones. Generally, chemically prepared inorganic nanoparticles cause safety issues when used as nanomedicines. However, as a biomineral, calcium phosphate is biocompatible and biodegradable, hence totally safe for human use," said Koyakutty.

Shantikumar V. Nair said that they are currently investigating the cancer-immunotherapy application potential of these nanoparticles with the support of the Biotechnology Industry Research Assistance Council (BIRAC) and an Indo-Swiss collaboration with Ludwig Cancer Research Institute, University of Lausanne, and the University of Geneva, Switzerland.

"We are now testing regulatory safety studies in large animal models. We expect to conduct human trials of the nanomedicine within the next year," Nair said.

**

The above article has been published from a wire agency with minimal modifications to the headline and text.

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Kochi Scientists Win Patent for Novel Nanomedicine that Enables Early Liver Tumour, Cirrhosis Detection & Treatment - The Weather Channel

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Early Timing, Optimal Conditioning Maximize Outcomes of Allo-SCT in MDSs and MPNs – AJMC.com Managed Markets Network

A review published in Frontiers in Oncology explored the application of allo-SCT for myelodysplastic syndromes and myeloproliferative neoplasms.

Myelodysplastic syndromes (MDSs) and myeloproliferative neoplasms (MPNs) are difficult to treat due to their varied clinical characteristics and prognostic heterogeneity. Allogeneic stem cell transplantation (allo-SCT) is the only potentially curative treatment thus far, and a recent review in Frontiers in Oncology explored the application of and challenges associated with allo-SCT in this disease setting.

MDSs and MPNs are neoplastic hematological disorders, the most common of which is chronic myelomonocytic leukemia (CMML), a type of MPN. Both disorders lack specific molecular signatures and tend to show myelodysplastic and myeloproliferative features. Thus, these conditions are complicated to treat, and there is a lack of clear treatment guidelines or effective therapies to target them.

For patients with CMML, overall survival (OS) ranges from a few months to several years after diagnosis. Although hydroxyurea has shown effectiveness against proliferative features and treatment with a hypomethylating agent can benefit patients with dysplastic features, these therapy options do not foster long-term remission or prevent disease progression to acute leukemia.

Currently, allo-SCT is the only potentially curative treatment across MDS/MPN types, but significant morbidity and mortality limit more widespread use of allo-SCT. It is typically not recommended for patients who are of an older age or who have comorbidities; but when possible, allo-SCT should be a go-to approach, the authors wrote.

The only treatment with curative potential remains allo-SCT, which should be provided to all patients with prognostically unfavorable, rapidly evolving, or symptomatic CMML, or other MDSs/MPNs, who have an available stem cell donor, the authors wrote. Ideally, this should be performed early, before disease progression, because in the latter case, nonrelapse mortality and relapse rate are higher and worse than those in [acute myeloid leukemia] evolved from classical MDS.

The authors highlight a selection of studies aiming to show the value of allo-SCT for patients who have MPNs and MDSs. A report from Mayo Clinic including 406 patients, 70 of whom underwent allo-SCT, found that median leukemia-free survival was significantly better in patients who underwent allo-SCT vs the nontransplanted cohort (40 vs 20 months). OS was also superior in the transplanted group (40 vs 21 months).

In another multicenter analysis including 261 patients, 119 of whom underwent allo-SCT, patients who underwent transplant had superior median OS (4.3 vs 2.3 years) compared with those who did not after a median follow-up of 6 years.

Several factors have been shown to have an impact on allo-SCT prognosis in patients who have CMML. Early transplantation is an important aspect of success in MPNs and MDSs, and patients Hematopoietic Cell Transplantation-Specific Comorbidity Index scores have also shown prognostic value in several studies. The percentage of bone marrow blasts, pre-transplant cytogenetics, pre-transplant hematocrit, and age have also been shown to impact survival.

The most challenging decision for the treating physician concerns patients between 60 and 70 years and few fit patients older than 70 years, the authors wrote. For this age range, the physician needs to discriminate higher-risk features that have been well characterized and described. A disease mutational profile can greatly help in any case but particularly for patients of the seventh decade of their life.

Helping patients achieve the best possible disease status ahead of allo-SCT is the most crucial aspect of successful implementation, the authors noted. The treatment armamentarium includes traditional therapies such as hypomethylating agents and novel options, but choosing the best strategy for each individual can be challenging.

Establishing the most appropriate drug combinations in each individual patient is a long way, which could be delineated through the use of these combinations either as a bridging treatment before transplantation or by incorporating appropriate drugs in the preparatory conditioning regimens, the authors wrote. All of these potential new directions could only be substantiated through prospective multicenter randomized trials.

Reference

Symeonidis A, Chondropoulos S, Verigou E, Lazaris V, Kourakli A, Tsirigotis P. Allogeneic hematopoietic stem cell transplantation for mixed or overlap myelodysplastic/myeloproliferative disorders.Front Oncol. Published online August 5, 2022. doi:10.3389/fonc.2022.884723

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