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ORION CORPORATION STOCK EXCHANGE RELEASE 21 OCTOBER 2020 at 6.40 p.m. EEST
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Composition of the Nomination Committee of Orion Corporation
OTTAWA, Oct. 21, 2020 (GLOBE NEWSWIRE) -- Cardiovascular disease (CVD) is the country’s leading cause of premature death in women. The Canadian Women’s Heart Health Alliance (CWHHA), a national network of women’s heart health experts and advocates powered by the Canadian Women’s Heart Health Centre (CWHHC) at the University of Ottawa Heart Institute (UOHI), released eye-opening research this week revealing the scope of the problem.
Originally posted here:
National Alliance reveals shocking truths about the world’s leading killer of women
In acute lymphoblastic leukemia (ALL), the standard curative approaches are intensive chemotherapy (IC) and allogeneic stem cell transplantation (allo-SCT). In children, the cure rate of IC alone is an encouraging 80% to 90%. Allo-SCT is mostly applied in adult, high-risk patients and when transplanted in first complete remission (CR1), the cure rate is about 50%.
However, promising cure rates should not be the sole focus as there are toxicities to consider. Intensive chemotherapy is associated with death in CR (complete remission) in up to 3% in children and 10% or higher in adults. Allo-SCT is even more toxic, with a treatment-related mortality of 10% to 20%. In addition, after intensive chemotherapy and particularly after SCT, there are a variety of long-term sequelae that decrease the quality of life. A substantial need exists for new treatments to avoid or reduce this toxicity.
There are several indications for the use of allo-SCT in adults (FIGURE 1). With the implementation of targeted therapies, these are continuously changing. Thus, the dogma that only allo-SCT is curative in patients with relapsed/ refractory disease should be revised to include CAR (chimeric antigen receptor) T cells, which are associated with a cure rate of about 50%.
Substantial progress in the treatment of adult ALL has been made in the last decade by the introduction of new targeted therapies, either with tyrosine kinase inhibitors (TKIs) or by immunotherapeutic approaches only allo-SCT is curative in patients with relapsed/refractory disease should be revised to include CAR (chimeric antigen receptor) T cells, which are associated with a cure rate of about 50% (FIGURE 2).
Patients with Philadelphia (Ph)-positive ALL constitute approximately 25% of adult B-lineage ALL, with an incidence increasing to about 50% among older patients.
In the pre-imatinib (Gleevec) era, CR rates were 60% to 70%, the survival rates associated with chemotherapy about 10%, and after patients underwent allo-SCT, about 30%. With the first-generation TKI imatinib, CR rates increased to 80% to 90%, the rate of BCR-ABL negativity from 5% to 50%, and the 5- to 10-year OS (overall survival) improved to 50% to 70%.1
Patients achieve faster and deeper molecular responses with second-generation TKIs such as dasatinib (Sprycel) and nilotinib (Tasigna), which translates into a survival benefit. The third-generation TKI ponatinib (Iclusig) targets resistant mutations, particularly T315I.2
Treating adult Ph-positive ALL with an allogeneic SCT in CR1 is still a good treatment option for patients with a 5-year OS of 60% to 70%. In older patients, when low-intensity chemotherapy was combined with dasatinib or nilotinib, the CR rate was greater than 90%, but many patients relapsed.3,4 In the next step, combining low-intensity chemotherapy with a TKI and adding an immunotherapy with inotuzumab ozogamicin (Besponsa), demonstrated a CR rate that surpassed 90% and the OS substantially improved.5 Here, the relapse rate was low and only several patients needed an SCT.
The newest approach in frontline Ph-positive ALL is a chemotherapy-free regimen that includes dexamethasone, the TKI dasatinib, and the bispecific antibody blinatumomab (Blincyto). A CR rate of 98% and the OS and disease-free survival (DFS) of 95% and 88%, respectively, for 2 years were observed.6 Interestingly, blinatumomab has been shown to eliminate Ph leukemic cells with resistant mutations.
Treatments with monoclonal antibodies or activated T cells are currently changing the treatment paradigm of ALL. The prerequisite is that B-lineage blast cells express a variety of specific antigens, such as CD19, CD20, and CD22 (TABLE ON PAGE 100), which are targetable with a wide variety of monoclonal antibodies.7 A new treatment principle is the activation of the patients T cells to destroy their CD19-positive leukemic blasts.
Anti-CD20/Rituximab
Rituximab (Rituxan) is a chimeric monoclonal antibody that binds to CD20-positive cells, resulting in cell death. Rituximab is now included in most B-lineage ALL regimens and is administered at the usual dose of 375 mg/m2 at day -1 before chemotherapy, typically for 8 cycles. This leads to a significant increase in the CR rate and MRD negativity rate of about 90% and improved survival of about 70% in standard-risk patients. Previously, rituximab was administered only to patients with more than 20% CD20 expression but is now administered to all patients because corticosteroids upregulate CD20 expression.8
It is difficult to extract the effect of allo-SCT in the context of rituximab-containing regimens with wide variation from 20% to 69%, according to study protocols. It is even more debatable whether the addition of rituximab improves outcomes after SCT.9-11 In one study, investigators examined the newer anti-CD20 antibody ofatumumab (Arzerra) in elderly patients with promising results.12
The anti-CD20 monoclonal body rituximab has substantially increased the cure rate of patients with de novo Burkitt leukemia/lymphoma. With repeated short cycles of intensive chemotherapy, combined with 8 doses of rituximab, the OS has increased to greater than 80%.13
Inotuzumab is an antibody-drug conjugate against CD22 and is linked to a cytotoxic agent from the class of calicheamicins called ozogamicin, which after internalization induces DNA strand breaks. CD22 is expressed in nearly all patients with adult ALL. Clinicians have explored inotuzumab in patients with relapsed/refractory ALL and it is now included in frontline regimens.
In an international, randomized multicenter trial (INO-VATE; NCT01564784), inotuzumab was compared with standard of care (SOC). Investigators reported that the CR rate was significantly higher in the inotuzumab group, 81% compared with 29% in SOC.14 Similarly, the inotuzumab group achieved a higher MRD- negativity rate of 78% versus 28% in the SoC group. This resulted in a transplant rate of 41% with the inotuzumab group compared with 11% in the SOC. Interestingly, this indicates that the achievement of a much higher MRD negativity by inotuzumab resulted in a higher rate for allo-SCT in CR1.
Inotuzumab has now moved to frontline therapy based on the results from a study involving older patients (60-97 years old) when combined with a reduced chemotherapy regimen of hyper-CVAD (cyclophosphamide, vincristine, doxorubicin [Adriamycin], and dexamethasone). Investigators reported a CR rate of 81% in this patient population, an MRD negativity of 100%, a DFS of 87%, and an OS of 70%.15 Taking the age of this patient group into consideration, the allo-SCT rate was only 6%, but the OS may also indicate that a substantial proportion of patients no longer need SCT.
Inotuzumab is a highly effective drug for achieving a fast tumor reduction in frontline therapy. If this combination of immunotherapies is considered, inotuzumab may be the first to reduce the tumor load and could be followed by blinatumomab to reduce remaining MRD.
Targeting CD19 is of great interest, as this antigen is highly expressed in all B-lineage cells, most likely including early lymphoid precursor cells. In contrast to the other antibodies, CD19-directed therapies act via T-cell activation to kill leukemic cells.
Blinatumomab
A new, promising approach is the bispecific antibody blinatumomab, which combines single chain antibodies to CD19 and CD3. Blinatumomab has been extensively explored in the MRD setting, which means that patients remained MRD positive after induction or with a molecular relapse.
In a pilot study evaluating 21 patients, the conversion rate to MRD negativity was 80%; 40% of these MRD-negative patients received an SCT.16 Interestingly, a fraction of patients also survived without undergoing an allo-SCT.
In an international, confirmatory, single-arm study (BLAST; NCT01207388) evaluating 116 patients in hematological remission, the rate of conversion to MRD negativity was high, with 78% achieving a complete MRD response with blinatumomab. In this setting, 40% of patients received an allo-SCT.17 Further, there was a fraction of patients who did not receive an allo-SCT.
Chimeric Antigen Receptor (CAR) T cells
The adoptive transfer of CAR-modified T cells directed against CD19 is another new promising approach for the treatment of CD19-positive disease in pediatric or adult ALL.18 In the first of 3 larger studies in adults with relapsed/refractory ALL, the CR rate ranged from 67% to 91%, with MRD negativity in 60% to 81% of patients who experienced a CR. OS was reported as 50% or greater at 2 years or more, which is remarkable for those heavily pretreated patients. It is noteworthy that CAR T cells are also effective in CNS leukemia and other extramedullary manifestations. Furthermore, CAR T-cell therapies are moving to frontline therapies.
CAR T-cell therapy in relapsed/refractory ALL was first considered as a bridge to allo- SCT and was applied in 10% to 50% of patients. Currently, however, the role of allo-SCT after CAR T-cell therapy remains unclear. Whereas in some institutions the therapy is always considered as a bridge to SCT, others have explored this treatment in different populations, such as in patients with high tumor burden, insufficient expansion of CAR T cells, or loss of MRD negativity.19
CD19-negative relapses after CAR T-cell therapy or blinatumomab because of CD19 escape are a relevant obstacle. To overcome this, bispecific antibodies targeting CD19/CD22 and other antigens are under development.
ALL immunotherapies are associated with toxicities, such as hepatotoxicity observed after the administration of inotuzumab, or cytokine release syndrome (CRS) and neurotoxicity after the administration of blinatumomab or CAR T cells. But despite these hurdles, death in CR is nearly zero, and this is the promise for further immunotherapies.
References:
1. Bassan R, Hoelzer D. Modern therapy of acute lymphoblastic leukemia. J Clin Oncol. 2011;29(5):532-543. doi:10.1200/JCO.2010.30.1382
2. Sasaki K, Jabbour EJ, Ravandi F, et al. Hyper-CVAD plus ponatinib versus hyper-CVAD plus dasatinib as frontline therapy for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: a propensity score analysis. Cancer. 2016;122(23):3650-3656. doi:10.1002/ cncr.30231
3. Rousselot P, Coud MM, Gokbuget N, et al; European Working Group on Adult ALL (EWALL) group. Dasatinib and low-intensity chemotherapy in elderly patients with Philadelphia chromosome-positive ALL. Blood. 2016;128(6):774-782. doi:10.1182/blood-2016-02-700153
4. Ottman OG, Pfeifer H, Cayuela JM, et al. Nilotinib (Tasigna) and low intensity chemotherapy for first-line treatment of elderly patients with BCR-ABL1-positive acute lymphoblastic leukemia: final results of a prospective multicenter trial (EWALL-PH02). Blood. 2018;132(suppl 1):31. doi:10.1182/blood-2018-99-114552
5. Jabbour E, Ravandi F, Kebriaei P, et al. Salvage chemoimmunotherapy with inotuzumab ozogamicin combined with mini-hyper-CVD for patients with relapsed or refractory Philadelphia chromosome-negative acute lymphoblastic leukemia: a phase 2 clinical trial. JAMA Oncol. 2018;4(2):230- 234. doi:10.1001/jamaoncol.2017.2380
6. Chiaretti S, Bassan R, Vitale A, et al. Dasatinib-blinatumomab combination for the front-line treatment of adult Ph+ ALL patients. Updated results of the Gimema LAL2116 D-Alba trial. Blood. 2019;134(suppl 1):740. doi:10.1182/blood-2019-128759
7. Hoelzer D. Novel antibody-based therapies for acute lymphoblastic leukemia. Hematology Am Soc Hematol Educ Program. 2011;2011(1):243- 249. doi:10.1182/asheducation-2011.1.243
8. Dworzak MN, Gaipa G, Schumich A, et al. Modulation of antigen expression in B-cell precursor acute lymphoblastic leukemia during induction therapy is partly transient: evidence for a drug-induced regulatory phenomenon. Results of the AIEOP-BFM-ALL-FLOW-MRD-Study Group. Cytometry B Clin Cytom. 2010;78(3):147-153. doi:10.1002/cyto.b.20516
9. Thomas DA, OBrien S, Faderl S, et al. Chemoimmunotherapy with a modified hyper-CVAD and rituximab regimen improves outcome in de novo Philadelphia chromosome-negative precursor B-lineage acute lymphoblastic leukemia. J Clin Oncol. 2010;28(24):3880-3889. doi:10.1200/ JCO.2009.26.9456
10. Hoelzer D, Huettmann A, Kaul F, et al. Immunochemotherapy with rituximab improves molecular CR rate and outcome in CD20+ B-lineage standard and high risk patients; results of 263 CD20+ patients studied prospectively in GMALL study 07/2003. Blood. 2010;116(21):170. doi:10.1182/blood.V116.21.170.170
11. Maury S, Chevret S, Thomas X, et al; for GRAALL. Rituximab in B-lineage adult acute lymphoblastic leukemia. N Engl J Med. 2016;375(11):1044- 1053. doi:10.1056/NEJMoa1605085
12. Richard-Carpentier G, Kantarjian HM, Konopleva MY, et al. Phase II study of the hyper-CVAD regimen in combination with ofatumumab (HCVAD-O) as frontline therapy for adult patients (pts) with CD20-positive B-cell acute lymphoblastic leukemia (B-ALL). Blood. 2019;134(suppl 1):2577. doi:10.1182/blood-2019-129884
13. Hoelzer D, Walewski J, Dhner H, et al; German Multicenter Study Group for Adult Acute Lymphoblastic Leukemia. Improved outcome of adult Burkitt lymphoma/leukemia with rituximab and chemotherapy: report of a large prospective multicenter trial. Blood. 2014;124(26):3870- 3879. doi:10.1182/blood-2014-03-563627
14. Kantarjian HM, DeAngelo DJ, Stelljes M, et al. Inotuzumab ozogamicin versus standard therapy for acute lymphoblastic leukemia. N Engl J Med. 2016;375(8):740-753. doi:10.1056/NEJMoa1509277
15. Jabbour E, OBrien S, Sasaki K, et al. Frontline inotuzumab ozogamicin in combination with low-intensity chemotherapy (mini-hyper- CVD) for older patients with acute lymphoblastic leukemia (ALL). Blood. 2015;126(23):83. doi:10.1182/blood.V126.23.83.83
16. Topp MS, Kufer P, Gkbuget N, et al. Targeted therapy with the T-cell-engaging antibody blinatumomab of chemotherapy-refractory minimal residual disease in B-lineage acute lymphoblastic leukemia patients results in high response rate and prolonged leukemia-free survival. J Clin Oncol. 2011;29(18):2493-2498. doi:10.1200/JCO.2010.32.7270
17. Gkbuget N, Dombret H, Bonifacio M, et al. Blinatumomab for minimal residual disease in adults with B-cell precursor acute lymphoblastic leukemia. Blood. 2018;131(14):1522-1531. doi:10.1182/ blood-2017-08-798322
18. Park JH, Geyer MB, Brentjens RJ. CD19-targeted CAR T-cell therapeutics for hematologic malignancies: interpreting clinical outcomes to date. Blood. 2016;127(26):3312-3320. doi:10.1182/ blood-2016-02-629063
19. Gou L, Gao J, Yang H, Gao C. The landscape of CAR T-cell therapy in the United States and China: a comparative analysis. Int J Cancer. 2019;144(8):2043-2050. doi:10.1002/ijc.31924
More than 2 million people have registered to become stem cell donors the UK, new figures released today reveal. The UK stem cell register had an immensely successful year in 2019/20, with 326,756 new donors added over 100,000 more than the previous year.
The UK stem cell register is known as the Anthony Nolan and NHS Stem Cell Registry and is made up of donors recruited by NHS Blood and Transplant, the Welsh Blood Service, DKMS and Anthony Nolan. The UK donor registers are urging young men, and people from black, Asian and minority ethnic backgrounds to register and ensure that all patients in need of a stem cell transplant can find a, potentially, lifesaving match.
If a patient has a condition that affects their bone marrow or blood, then a stem cell transplant may be their best chance of survival. Doctors will give new, healthy stem cells to the patient via their bloodstream, where they begin to grow and create healthy red blood cells, white blood cells and platelets.
In 2019/20 62 per cent of people who donated stem cells or bone marrow to patients in the UK were men under 30. They are the demographic most likely to be chosen to donate, but make up just 19 per cent of the UK stem cell register.
The percentage of all donors from minority ethnic backgrounds has remained steady at 13 per cent in 2019/20, highlighting the importance of raising awareness of their lifesaving potential amongst this group. Patients from Black, Asian or other minority backgrounds have a 20 per cent chance of finding the best possible stem cell match from an unrelated donor, compared to 69 per cent for northern European backgrounds.
Henny Braund, Chief Executive of Anthony Nolan, said:
Nobody could have foreseen the challenges this year would bring to building a healthy, diverse stem cell register. But weve adapted and weve innovated as patients cant wait and were thrilled that in 2020, weve collectively recruited two million donors onto the stem cell register. Each donor represents hope, a potential cure for blood cancer.
I thank colleagues and partners for their commitment helping us reach this point. I am also immensely grateful for the two million selfless individuals who signed up to the registry, making themselves available whenever they are needed.
The two million milestone means increased chances for many of finding an unrelated donor match. But were still far from our goal of finding a match for everyone who needs one.
I would urge anyone thinking of joining the stem cell register, especially young men, who are the most likely to be chosen, to do so today. You could be someones lifesaver, without you there is no cure.
Christopher Harvey, Head of the Welsh Bone Marrow Registry, said:
Its incredibly heart-warming to know there are two million people in the UK who are willing to donate stem cells should they be the match for someone in need of their potentially lifesaving donation.
We see in our roles the difference stem cells make, for lots of patients receiving stem cells is the final treatment option.
Despite this great news we still have more to do. Unfortunately, there are still patients who are unable to find a match. Thats why were committed to ensuring every patient has the best possible chance of finding that one lifesaving donor in their time of need.
Guy Parkes, Head of Stem Cell Donation & Transplantation at NHS Blood and Transplant, said:
We want all patients in need of a transplant to be able to find a lifesaving match. Each time a person joins this register it brings fresh hope to patients of a match.
This register is used by hospitals across the UK to find suitable matches for patients and it has helped to save and improve the lives of thousands of people since its creation 33 years ago its amazing that we now have over 2 million people on the register, putting the chances of matching donors to patients at a record high.
Donating stem cells is an altruistic, lifesaving act and its an amazing thing to do. We will continue to expand the UK register to help patients in need. We particularly need more young men to join.
Jonathan Pearce, CEO of DKMS UK said:
Were delighted to have reached such an amazing milestone and are grateful to those two million people who are actively registered and waiting to help give someone living with blood cancer or a blood disorder a second chance of life.
At any one time there are around 2,000 people in the UK in need of a blood stem cell transplant, so whilst we recognise this achievement it goes without saying that we need to continue to encourage everyone that can register to do so. This will help to grow the numbers and diversify the registry further in order to improve the odds for those who currently have less chance of finding a matching donor.
Excerpt from:
The Anthony Nolan and NHS Stem Cell Registry - The Hippocratic Post
Careful, man, theres a beloved actor here.
Jeff Bridges revealed that he has lymphoma, which is the most common type of blood cancer. And this sobering news has spurred celebrities and fans to send their best wishes to the star best known for playing the Dude, the White Russiandrinking bowler and casual-wear icon from the Coen brothers 1998 cult classic, The Big Lebowski.
But the Dude abides, and Bridges suggested that his outlook looks just as promising.
As the Dude would say.. New S**T has come to light, tweeted Bridges, 70, on Monday. I have been diagnosed with Lymphoma. Although it is a serious disease, I feel fortunate that I have a great team of doctors and the prognosis is good.
Celebrities such as Cary Elwes, John Lithgow, Patricia Arquette and George Takei posted encouraging words and prayers to Bridges, who is the son of Lloyd and Dorothy Bridges, and has starred in more than 70 films including Starman, True Grit and The Last Picture Show. He won an Academy Award in 2010 for Crazy Heart, and was honored with the Cecil B. DeMille lifetime-achievement award during the 2019 Golden Globes.
And he is now one of the most high-profile cases of lymphoma, a cancer of the bodys infection-fighting lymphatic system that affects the blood and bone marrow. And more than 85,000 new cases of lymphoma are expected to be diagnosed in the U.S. this year, according to American Cancer Society data shared by the Leukemia & Lymphoma Society, with some 791,550 people currently living with lymphoma or in remission from the disease in the U.S.
Many different types of lymphoma exist, and Bridges did not share any more details about his diagnosis or treatment. But his disclosure is an opportunity to share more information about lymphoma, the risk factors and symptoms to be aware of, as well as treatment options.
What is lymphoma?
Lymphoma is a type of cancer that starts in cells that are part of the bodys immune system, specifically the lymphocytes, which are a type of white blood cell that fights germs. So these cancers can affect the blood and bone marrow, as well as the other tissues and organs that produce, store and carry white blood cells including the spleen.
Doctors still dont know what specifically causes lymphoma, but at some point a lymphocyte mutates and begins to reproduce rapidly. The mutated, abnormal cells live longer than the normal cells would, and in time, the diseased and ineffective lymphocytes outnumber the healthy cells, which causes the lymph nodes, liver and spleen to swell.
There are two main types of lymphoma, the CDC explains, including:
Hodgkin lymphoma (HL), which spreads in an orderly manner from one group of lymph nodes to another.
Non-Hodgkin lymphoma (NHL), which spreads through the lymphatic system in a non-orderly manner.
What are the symptoms?
Signs and symptoms of lymphoma may include:
These symptoms can be signs of other health conditions, of course, so its recommended that anyone experiencing them should see a doctor to determine the cause.
How is it treated?
There are many different types of lymphoma including 90 different types of non-Hodgkin lymphoma and treatment varies depending on the type and severity. Generally, lymphoma treatment involves chemotherapy, radiation therapy and immunotherapy medication. The Mayo Clinic, which is an international authority on lymphoma research, explains that the goal of treatment is to destroy as many cancer cells as possible to bring the disease into remission. A bone marrow or stem cell transplant may be performed in some cases to help rebuild healthy bone marrow after chemo and radiation has suppressed the diseased bone marrow.
Bridges didnt specify his own treatment, only saying that he is beginning treatment and will keep the public posted on his recovery.
Treatment can be very expensive, however, with almost 60% of patients covered by Medicare telling the Leukemia & Lymphoma Society in a 2019 study that they decided to delay or forego treatment, largely due to steep out-of-pocket costs. It noted that some traditional Medicare lymphoma patients getting anti-cancer therapy though infusions experienced out-of-pocket costs of more than $19,000 in their first year. And costs can extend two or three years beyond a blood cancer diagnosis.
Who is most at risk?
While children, teens and adults can all develop lymphoma, some types are more common in certain age groups. The CDC notes that rates of Hodgkin lymphoma are highest among teens and young adults (ages 15 to 39) as well as among older adults (ages 75 and older). But non-Hodgkin lymphoma becomes more common as people get older.
Men are also slightly more likely to develop lymphoma than women, the CDC adds, and white people are more likely than Black people to develop non-Hodgkin lymphoma.
Cases have also been more common in people who are immunocompromised, including those who take drugs to suppress their immune systems. And some infections such as HIV and the Epstein-Barr virus are also associated with an increased lymphoma risk.
And like many other cancers, family history has been linked with a higher risk of Hodgkin lymphoma.
What is the survival rate?
The good news is, Hodgkin lymphoma is now considered to be one of the most curable forms of cancer, according to the Leukemia & Lymphoma Society, with a five-year survival rate of 94.4% among patients younger than 45 at diagnosis. And the five-year relative survival rate for those with Hodgkin lymphoma more than doubled from 40% in whites in 1960 to 1963 (the only data available) to 88.5% for all races from 2009 to 2015.
And the five-year relative survival rate for people with non-Hodgkin lymphoma rose from 31% in whites from 1960 to 1963 (the only data available) to 74.7% for all races from 2009 to 2015.
Still, an estimated 20,910 Americans are expected to die from lymphoma this year, including 19,940 with non-Hodgkin lymphoma and 970 with Hodgkin lymphoma.
How does COVID-19 complicate things?
While the medical community is still learning about COVID-19, the general consensus is that people with cancer, who are in active cancer treatment or have previously been treated for cancer, may be at higher risk of severe illness and death if they get the coronavirus. So its important that these folks lower their risk of exposure to COVID-19 by avoiding large crowds and non-essential travel; working from home, if possible; staying at least six feet away from people outside their household; wearing a face mask when they cant socially distance; as well as washing their hands frequently, and not touching their eyes, nose or mouth.
Where can I find more information or support?
Visit the CDC and American Cancer Society pages on lymphoma.
The Mayo Clinic also outlines its lymphoma research and treatment strategies on its website.
The Leukemia & Lymphoma Society and the Lymphoma Research Foundation also provide valuable information and support.
Original post:
Jeff Bridges is one of the 85,000-plus lymphoma cases expected in the U.S. this year - MarketWatch
DENVER A baby broken, inside the womb.
Most doctors gave little unborn Payton Calvillo any hope she would survive. But through strong faith and the help of a team of medical experts, she is thriving today.
"She's a complete miracle baby," said Payton's mother, Ahna Calvillo.
When Ahna was just five months pregnant, she was told her unborn baby would probably not survive birth.
"It was pretty much a death sentence from the beginning."
Payton's bones were breaking and bending inside the womb.
"She likely had a problem where she couldn't make alkaline phosphatase properly," explained Dr. Sunil Nayak, a pediatric endocrinologist at Rocky Mountain Hospital for Children.
Alkaline phosphatase is needed for bones to grow and strengthen and there was little anyone could do.
Nineteen different specialists were on hand for the C-section delivery
"They even asked us the question that morning, how far do you want us to go?" Ahna remembered. "'Do you want a ventilator on her?', you know, 'How far do you want us to prolong her life?' Our ultimate hope and goal was that she would come out and breathe on her own."
"She just came out screaming," said Ahna. "She came out crying. She breathed on her own right away. She was perfect."
Payton was diagnosed with hypophosphatasia, a disorder that weakens bones and was immediately placed on a new FDA-approved medicine.
"Here we are just one year later at one year of age and you see a dramatic difference in the shape," said Dr. Jared Riley, a pediatric orthopedic surgeon at Rocky Mountain Hospital.
Before the medicine, 75% of all patients died by the age of five.
Now there is a 97% chance Payton will live a normal life.
"My baby was broken and that's what I needed God to do was a miracle," said Ahna.
One was also treated with bone fragments and cultured osteoblasts, which are bone-forming cells.
"Cultured" refers to cells that are grown under specific conditions outside of the natural environment (the body) and within a laboratory.
Both patients showed significant, but incomplete improvement, although no more formal studies have been conducted.
Then, the drug teriparatide (parathyroid hormone 1-34) has been given "off-label" to several adults with HPP with metatarsal stress fractures or femoral pseudo fractures, resulting in healing.
The drug is not permitted for use in children.
More research is necessary to determine the long-term safety and effectiveness of teriparatide in the treatment of HPP.
Every year eight million babies are born with genetic disorders passed down from generation to generation.
Payton will stay on the new medication for the next few years and then doctors will re-evaluate whether she needs to continue.
Payton's family didn't even know they carried the problematic HPP gene until an ultrasound revealed it in their unborn baby.
After being genetically tested, Payton's mother and grandfather are positive.
Neither one has ever suffered from weak or broken bones.
If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens atjim.mertens@wqad.comor Marjorie Bekaert Thomas atmthomas@ivanhoe.com.
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YOUR HEALTH: Saving an unborn baby breaking apart in the womb - WQAD.com
"Young people today are often drivers of social change movements and we look forward to engaging them."
South African youth aged 16 and 17 will be able to make history, alongside their peers in the UK, as the worlds youngest bone marrow donors.
The South African Bone Marrow Registry (SABMR) received the nod from its Clinical Governance Committee and board members, as well as the National Health Department to allow 16 and 17-year-old teens to become bone marrow, stem-cell donors.
Recent changes in legislation and advances in stem cell donation have allowed registries to reduce the age limit of donors. South Africa now joins the UK in this move. The latter became one of the first countries to do so.
Dr Charlotte Ingram, Medical Director of the SA Bone Marrow Registry (SABMR) the largest registry in the country says it's a landmark moment as the change in joining policy will contribute to saving more lives.
"In general, young people make better donors. Research shows that younger donors are associated with better survival rates for patients following a stem cell transplant."
"It's a step towards further enhancing the registry towards a younger and more ethnically diverse pool for blood cancer patients and others in need of a bone marrow transplant."
Previously, teenagers had to wait until they were 18 to join the SA Bone Marrow Registry. Now they can join by following the same procedure as others would.
While it is not required, it is important for the SABMR to involve parents and address any questions or concerns they may have re the procedure and what it entails.
Once youth have applied online, they will be contacted to discuss the easiest way of dispatching and collecting swab kits. The only initial sample that is required is a cheek swab.
Currently, 18-25-year olds only account for 6.8% of the SABMR registry but with increased awareness of bone marrow donation among young people, the figure should increase substantially.
Read:Knowledge is key: What you need to know about the most common childhood cancer in SA
"Studies tell us that generation WE (aged 14-20) and generation Z (21-25) are a lot more self-aware, socially-responsible and globally-minded than previous generations. They are more concerned about tackling social issues and want to roll up their sleeves and make a difference. Young people today are often drivers of social change movements and we look forward to engaging them."
She says there is no greater way to help another than to potentially save a life.
"So many lives are lost if there is a delay in finding a donor match. While we have 74 000 donors on our registry, we often discover that many older donors can no longer donate stem cells as they have developed hypertension, heart disease or diabetes."
"When this happens, we have to start the search process all over again, which prolongs the agonising wait for a patient, who doesn't have time to waste."
"By opening up the donor pool to a younger audience, means doctors and donors can choose the healthiest matches that substantially increases a patients chance of survival."
For now, social media will serve as the primary channel to create awareness among youth, but physical donor drives at schools and other initiatives, which encourage collaboration between learners, peers and patients are in the pipeline for 2021.
If you are between the ages of 16 and 45 and want to become a donor, contact the SABMR on 021 447 8638 or email: donors@sabmr.co.za.
Financial donations can also be made via http://www.sabmr.co.za/donate.
Submitted to Parent24 by theSA Bone Marrow Registry
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SA becomes 2nd country to allow 16 and 17-year-olds to donate bone marrow - News24
Orgenesis completes acquisition of Koligo Therapeutics and announces additional acquisition of Icellator(R) Technology from Tissue Genesis in related transaction
Icellator(R) commercially available for lipotransfer in first two countries with more approvals expected
Germantown, MD, US October 19th, 2020 Orgenesis Inc. (NASDAQ: ORGS)(Orgenesis or the Company), a global biotech company working to unlock the full potential of celland gene therapies, today announced completion of thepreviously announcedacquisition ofKoligo Therapeutics, Inc.(Koligo), a regenerative medicine company. Additionally, the Company announced that it has acquired substantially all of the assets ofTissue Genesis, LLC(Tissue Genesis), adding to a growing list of POCare technology resources.
The acquisition of Tissue Genesis was initially undertaken via Koligo Therapeutics, Inc., and became part of the Koligo acquisition transaction. Orgenesis will now own the entire inventory of Tissue GenesisIcellator(R) devices, related kits and reagents, a broad patent portfolio to protect the technology, registered trademarks, clinical data, and existing business relationships for commercial and development stage use of the Icellator technology.
The Icellator device is a point-of-care cell isolation technology that rapidly recovers high yields of stromal and vascular cells (SVF) from adipose tissue (fat) to be used therapeutically. Adipose tissue is recognized as a superior source for adult stem cells found abundantly in the micro-vasculature and stroma of human fat. Further details include:
With the Koligo acquisition closed, we believe that we are making rapid progress on a number of fronts, said Vered Caplan, CEO of Orgenesis. Specifically, we plan to leverage the therapies and technologies from Koligo and Tissue Genesis across our POCare Platform. One of our first goals is to accelerate the commercial scaleup of KYSLECEL throughout the United States and, subject to regulatory and logistical considerations, in international markets as well. Subject to FDA review and clearance of our IND application, we also look forward to commencing patient recruitment for a phase 2 randomized clinical trial of KT-PC-301, an autologous clinical development stage cell therapy candidate for COVID-19-related Acute Respiratory Distress Syndrome, or ARDS. We plan to use the Icellator device to support scalable, cost-effective production of KT-PC-301. Additionally, Koligos development stage 3D-V bioprinting technology for the vascularization of autologous cells to create biodegradable and shelf-stable three-dimensional cell and tissue implants will be explored for diabetes and pancreatitis applications, with longer term applications for neural, liver, and other cell/tissue transplants also potentially explored.
The Icellator system is highly complementary to our POCare technology systems, as well as Koligos 3D-V bioprinting technology. Technologies such as these support our mission of improving the efficacy and lowering the costs of cell and gene therapies by delivering autologous cell therapies at the point of care through our global network of hospitals and healthcare institutions, concluded Caplan.
Under the terms of the Koligo merger agreement, Orgenesis acquired all of the outstanding stock of Koligo from its shareholders for approximately USD 14.5 million in shares of Orgenesis common stock valued at USD7.00 per share (with certain non-accredited investors paid approximately USD 20,000 solely in cash) and an assumption of USD 1.9 million in liabilities, estimated to be substantially all of Koligos liabilities. Orgenesis acquired substantially all the assets of Tissue Genesis for an additional consideration of USD 500,000 in closing cash and future royalties. Additional details of the transactions will be available in the Companys Form 8-K, which will be filed with the Securities and Exchange Commission, and will be available atwww.sec.gov.
Pearl Cohen Zedek Latzer Baratz LLP and KPMG advised Orgenesis on the Koligo Transaction. Maxim Group LLC acted as a finder and Nelson Mullins Riley & Scarborough, LLP advised Koligo on the Transaction.
About Orgenesis
Orgenesis is a global biotech company working to unlock the full potential of celland gene therapies (CGTs) in an affordable and accessible format at the point of care. The Orgenesis POCarePlatform is comprised of three enabling components: a pipeline of licensedPOCare Therapeuticsthat are processed and produced in closed, automatedPOCare Technologysystems across a collaborativePOCare Network. Orgenesisidentifies promising new therapies and leverages its POCare Platform to provide a rapid, globally harmonized pathway for these therapies to reach and treat large numbers of patients at lowered costs through efficient, scalable, and decentralized production. The Network brings together patients, doctors, industry partners, research institutes and hospitals worldwide to achieve harmonized, regulated clinical development and production of the therapies. Learn more about the work Orgenesis is doing atwww.orgenesis.com.
Notice Regarding Forward-Looking Statements
The information in this release is as of October 19, 2020. Orgenesis assumes no obligation to update forward-looking statements contained in this release as a result of new information or future events or developments. This release contains forward looking statements about Orgenesis, Koligo, Koligos technology, and potential development and business opportunities of Koligo and Orgenesis following the closing of the Transaction, each of which involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of the Companys products; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any such regulatory authorities may approved the Companys development products, and, if approved, whether such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of the Companys products; uncertainties regarding the impact of COVID-19 on the Companys business, operations and financial results and competitive developments.
A further description of risks and uncertainties can be found in the Companys Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available atwww.sec.gov.
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Orgenesis completes acquisition of Koligo Therapeutics and announces additional acquisition of Icellator(R) Technology from Tissue Genesis in related...
Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.
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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.
It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.
Global Stem Cell Therapy Market: Overview
Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.
Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.
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Global Stem Cell Therapy Market: Key Trends
The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.
On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.
Global Stem Cell Therapy Market: Market Potential
A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.
In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.
Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.
Global Stem Cell Therapy Market: Regional Outlook
The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.
Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.
Global Stem Cell Therapy Market: Competitive Analysis
Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.
Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.
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Stem Cell Therapy Market to Witness Steady Expansion During 2025 KYT24 - KYT24