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TOKYO & BASKING RIDGE, N.J.--(BUSINESS WIRE)--Positive results from the global pivotal QuANTUM-First phase 3 trial of Daiichi Sankyos (TSE:5468) quizartinib combined with standard induction and consolidation chemotherapy and then continued as a single agent demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) in adult patients aged 18-75 with newly diagnosed FLT3-ITD positive acute myeloid leukemia (AML) compared to standard chemotherapy alone. The data were featured as part of the press program and presented during the Presidential Symposium (#S100) at the European Hematology Association (#EHA2022) Congress.

AML is one of the most common leukemias in adults with an estimated five-year survival rate of approximately 30.5%.1,2 Of all newly diagnosed cases of AML, 25% carry the FLT3-ITD gene mutation, which is associated with particularly unfavorable prognosis including increased risk of relapse and shorter overall survival.3

Quizartinib combined with standard induction and consolidation chemotherapy and then continued as a single agent demonstrated a 22.4% reduction in the risk of death compared to standard chemotherapy alone (HR = 0.776 [95% CI: 0.615-0.979; 2-sided p=.0324]) in patients with newly diagnosed FLT3-ITD positive AML. After a median follow-up of 39.2 months, median OS was more than double at 31.9 months for patients receiving quizartinib (95% CI: 21.0-NE) compared to 15.1 months for patients receiving chemotherapy (95% CI: 13.2-26.2).

The safety of quizartinib combined with intensive chemotherapy and as continuation monotherapy in QuANTUM-First was generally manageable, with no new safety signals observed. Rates of grade 3 or higher treatment emergent adverse events (TEAEs) were similar for both study groups and the most common grade 3 or higher TEAEs occurring in 10% of patients were febrile neutropenia (43.4% quizartinib; 41.0% placebo), neutropenia (18% quizartinib; 8.6% placebo), hypokalemia (18.9% quizartinib; 16.4% placebo) and pneumonia (11.7% quizartinib; 12.7% placebo). Rates of TEAEs associated with fatal outcomes were 11.3% for quizartinib versus 9.7% for chemotherapy alone and were mainly due to infections.

QTcF > 500 ms occurred in 2.3% of patients receiving quizartinib and 0.8% of patients discontinued quizartinib due to QT prolongation. Ventricular arrhythmia events with quizartinib were uncommon. Two (0.8%) patients experienced cardiac arrest with recorded ventricular fibrillation on ECG (one with fatal outcome) both in the setting of severe hypokalemia.

The QuANTUM-First results show that adding quizartinib to standard chemotherapy significantly improved overall survival in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia, said Harry P. Erba, MD, PhD, Instructor, Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke Cancer Institute. There is great interest in the increased use of targeted therapies to improve outcomes for patients with AML, particularly those with the FLT3-ITD subtype, which is one of the most common, aggressive and difficult-to-treat.

We are proud that another one of our medicines has demonstrated a significant survival advantage, as our goal is to leverage innovative science to change the way cancer is treated, said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. Adding targeted treatment with quizartinib, a potent and selective FLT3 inhibitor, to standard chemotherapy resulted in a doubling of median overall survival in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia compared to standard chemotherapy alone. Based on these positive QuANTUM-First results, we have initiated global regulatory filings in order to bring quizartinib to patients as quickly as possible.

The OS improvement with quizartinib was also supported by a sensitivity analysis censoring for the effect of allogenic hematopoietic stem cell transplant (HSCT) (HR = 0.752; [95% CI: 0.562-1.008]).

Additional secondary and exploratory analyses provide further understanding and some supporting evidence for improved OS in patients receiving quizartinib combined with chemotherapy in the trial.

The primary event-free survival (EFS) analysis (with induction treatment failure (ITF) defined as not achieving complete remission (CR) by day 42 of the last induction cycle), did not show a statistically significant difference between the two study arms; two pre-specified sensitivity analyses on EFS (the first one defining ITF as not achieving CR by the end of induction; the second one defining ITF as having not achieved composite complete remission (CRc) by the end of induction) showed HR = 0.818 [95% CI: 0.669, 0.999] and HR = 0.729 [95% CI: 0.592-0.897], respectively.

The CRc rate was numerically higher for patients receiving quizartinib compared to chemotherapy alone (71.6% versus 64.9%), and rates of CR were similar for the two study arms (54.9% and 55.4%). The median duration of CR was 38.6 months for quizartinib (95% CI: 21.9-NE) and 12.4 months for chemotherapy (95% CI: 8.8-22.7).

The median relapse-free survival (RFS) for patients who achieved CR was 39.3 months for quizartinib and 13.6 months for placebo, representing a 38.7% relative risk reduction of relapse or death (HR = 0.613 [95% CI: 0.444-0.845]).

Summary of QuANTUM-First Results for OS

Efficacy Measure*

Quizartinib +ChemotherapyN=268

Placebo +ChemotherapyN=271

HR(95% CI)

OS Primary Analysis

Median OS (95% CI)

31.9 months

(21.0 NE)

15.1 months

(13.2 26.2)

HR = 0.776

(0.615 0.979)

2-sided p = 0.0324

*A hierarchical testing procedure was used to test the primary endpoint OS, followed by EFS, CR and CRc. Formal statistical testing was stopped after EFS as its result was not statistically significant.Data cut-off: August 13, 2021Abbreviations: HR = Hazard ratio; NE = not estimable; OS = overall survival

About QuANTUM-First

QuANTUM-First is a randomized, double-blind, placebo-controlled global phase 3 study evaluating quizartinib in combination with standard induction and consolidation chemotherapy and then as continued single agent therapy in adult patients (aged 18-75) with newly diagnosed FLT3-ITD positive AML. Patients were randomized 1:1 into two treatment groups to receive quizartinib or placebo combined with anthracycline- and cytarabine-based regimens. Eligible patients, including those who underwent allogeneic HSCT, continued with single agent quizartinib or placebo for up to 36 cycles.

The primary study endpoint was OS. Secondary endpoints include EFS, post-induction rates of CR and CRc, and the percentage of patients who achieve CR or CRc with FLT3-ITD minimal residual disease negativity. Safety and pharmacokinetics, along with exploratory efficacy and biomarker endpoints, also were evaluated. QuANTUM-First enrolled 539 patients at 193 study sites across Asia, Europe, North America, Oceania and South America. For more information, visit ClinicalTrials.gov.

About Acute Myeloid Leukemia (AML)

More than 474,500 new cases of leukemia were reported globally in 2020 with more than 311,500 deaths.4 AML is one of the most common types of leukemia in adults, representing about one-third of all cases, and the average age of diagnosis is 68 years old.1 The five-year survival rate for AML is 30.5%, the lowest by far among the major leukemia subtypes, and is 9.4% for patients aged 65 and older.5,6,7 The conventional treatment for newly diagnosed AML is intensive induction and consolidation chemotherapy with HSCT for eligible patients.8 The introduction of new targeted therapies in recent years has added to the standard of care and improved outcomes for some patients with molecularly defined AML subtypes.9

About FLT3-ITD

FLT3 (FMS-like tyrosine kinase 3) is a tyrosine kinase receptor protein normally expressed by hematopoietic stem cells that plays an important role in cell development, promoting cell survival, growth and differentiation through various signaling pathways.3 Mutations of the FLT3 gene, which occur in approximately 30% of AML patients, can drive oncogenic signaling.3 FLT3-ITD (internal tandem duplication) is the most common type of FLT3 mutation in AML, occurring in about 25% of all newly diagnosed patients, and is associated with increased risk of relapse and shorter overall survival.3

About Quizartinib

Quizartinib is an oral, highly potent and selective type II FLT3 inhibitor currently in clinical development for the treatment of FLT3-ITD positive AML.3 In addition to QuANTUM-First, the quizartinib development program includes a phase 1/2 trial in pediatric and young adult patients with relapsed/refractory FLT3-ITD AML in Europe and North America. Several phase 1/2 combination studies with quizartinib are also underway at The University of Texas MD Anderson Cancer Center as part of a strategic research collaboration focused on accelerating development of Daiichi Sankyo pipeline therapies for AML.

Quizartinib has received Fast Track Designation from the U.S. Food and Drug Administration for the treatment of adult patients with newly diagnosed AML that is FLT3-ITD positive, in combination with standard cytarabine and anthracycline induction and cytarabine consolidation. Orphan Drug Designation has been granted to quizartinib for the treatment of AML in Europe, Japan and the U.S.

Quizartinib is currently approved for use in Japan under the brand name VANFLYTA for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, as detected by an approved test. Quizartinib is an investigational medicine in all countries outside of Japan.

About Daiichi Sankyo

Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose to contribute to the enrichment of quality of life around the world. In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an Innovative Global Healthcare Company Contributing to the Sustainable Development of Society. For more information, please visit http://www.daiichisankyo.com.

References

1 American Cancer Society. Key Statistics for Acute Myeloid Leukemia. Updated January 2020.2 National Cancer Institute SEER Program. Cancer Stat Facts: Acute Myeloid Leukemia 3 Daver N et al. Leukemia. (2019) 33:299312.4 Global Cancer Observatory. Population Fact Sheet: World. Updated November 2020.5 National Cancer Institute SEER Program. Cancer Stat Facts: Acute Myeloid Leukemia: 5-Year Survival Rates 6 Leukemia and Lymphoma Society. Facts and Statistics. Leukemia: Survival 7 National Cancer Institute SEER Explorer. Acute Myeloid Leukemia: 5-Year Survival Rates by Age 8 Daver et al. Blood Cancer J (2020) 10(10):1079 Short et al. Cancer Discov. (2020);10:50625.

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Quizartinib Plus Chemotherapy Significantly Improved Overall Survival Compared to Chemotherapy in Patients with Newly Diagnosed FLT3-ITD Positive...

A Texas family said they faced a fight for their childs life after their daughter's health plan provider denied coverage of a medication known as the most expensive drug in the world.

The family said doctors told them the one-time infusion that costs millions of dollars was their daughters best hope.

Read on to learn more about their story and how an "army" of strangers stepped in.

At 4-months old, every move Aniya Porter makes is a miracle to her parents.

Aniya Porter was born with a rare genetic disease called spinal muscular atrophy or SMA.

You go day-by-day. Is my daughter going to stop breathing? Is she going to stop attempting to stand up? Is she going to stop putting her head up? Will Porter said.

Aniya was born with a rare genetic disease called spinal muscular atrophy (SMA).

SMA progressively kills motor neurons, the nerve cells in the brain stem and spinal cord that control essential functions like talking, walking, swallowing and breathing.

We live every day wondering and hoping that nothing else happens to her, that she doesn't lose any more of her motor neurons, said Hailey Weihs, Aniyas mother.

There is treatment.

In 2019, the FDA approved a drug called Zolgensma which carries a price tag of $2.1 million. The one-time gene therapy, given through an IV, is designed to replace the function of a missing or non-working SMN1 gene in a patient to stop the progression of SMA preserving motor neurons before theyre gone.

It doesn't give back what she has already lost. That's why it's so time-sensitive, explained Weihs.

When Aniyas doctor prescribed Zolgensma, Weihs and Porter said their daughters insurance, a Medicaid provider in Texas, denied coverage of the drug.

She got the denial from her insurance and that was devastating for us, said Weihs.

As they appealed, they said Aniya started to show signs she may lose control of her muscles.

She started to stop having reflexes in her legs and then her tongue started twitching, said Porter.

Racing against time, Weihs connected with other families of kids with SMA and a lawyer who agreed to represent Aniya for free.

Aniyas attorney, Eamon Kelly, told NBC 5 Responds, All the doctors agree that Aniya Porter should receive this treatment.

Kelly, who is based in Chicago, said Aniya is the seventh child with SMA hes represented in insurance coverage battles.

In Aniyas case, he said the health plan provider said the treatment was not medically necessary because doctors believe Aniya has four copies of the SMN2 gene, also known as a backup gene, which can indicate a milder illness.

Kelly argued backup genes, along with other available therapies, would not be enough to keep Aniya from losing muscle function. However, he said cutting-edge gene therapy could help.

We have a treatment that will take a little girl that is going to have a degenerative disease that threatens her life, that will put her in a wheelchair and as long as we get it to her before she's 2-years-old and before she loses her motor neurons, she'll walk, she'll dance, she'll live a full life. Its like science fiction, Kelly said.

The first child with SMA Kelly represented is Maisie Forrest, who received Zolgensma in 2019 when she was 20 months old.

Ciji Green, Maisies mom, said Maisie was on a ventilator 22 hours a day before getting the drug.

We met with her pulmonologist and I just wept, Green recalled. I told him she's not going to make it to 2 and he didn't offer any words of hope because he knew Maisie was on the decline as well.

Maisie is now 4-years-old.

She touches my face and it's absolutely beautiful, said Green.

Maisie is playing, talking and crawling.

Something that I have now that I didn't have was hope, hope that I will get to see her continue to meet milestones that she was never supposed to meet, Green added.

Maisies mom and a team of volunteers known as Maisies Army introduced Aniyas parents to Eamon Kelly. Last month, Kelly represented Aniya at a Medicaid State Fair Hearing.

Aniyas family requested the hearing from Texas Health and Human Services.

A week after the hearing and a few days after NBC 5 reached out to Aniyas health plan provider, Superior HealthPlan, Superior told Aniyas family Zolgensma would be covered for Aniya.

We have fought for four months. Those have been the hardest four months of our lives, said Weihs.

On April 27, Aniyas family made the trip from their home in Abilene to Cook Childrens Medical Center in Fort Worth where Aniya received the drug.

She's going to sit up on her own, she is going to walk one day, she's going to feed herself with a spoon, she's going to walk down and she's going to get her diploma, Weihs said.

NBC 5 Responds reached out to Superior HealthPlan by phone and email. We didnt hear back.

We asked Texas Health and Human Services about options for families denied coverage for Zolgensma.

It told us, in part, Medicaid covers medically necessary services including medications, and those services are delivered through managed care organizations. If the prior authorization is denied, the provider or the member can appeal the decision and MCOs have flexibility to make medically necessary decisions. Members also have the right to access the State Fair Hearing process with or without an External Medical Review (EMR). The EMR is conducted by a third-party Independent Review Organization.

It also shared, Medicaid covers alternative therapies for spinal muscular atrophy treatment, including Spinraza (nusinersen) and Evrysdi (risdiplam). Also, some manufacturers offer patient assistance programs.

A spokesperson for Novartis, which makes Zolgensma, told NBC 5, in part, Zolgensma (onasemnogene abeparvovec) is a transformative and highly innovative gene therapy for a devastating, progressive genetic disease. This one-time gene therapy is priced based on the value it provides to patients, caregivers and health systems.

It also said, Novartis is working in partnership with governments and health care systems worldwide to identify and define new sustainable access models.

Aniyas parents said she will still see a doctor and be monitored after getting the gene therapy treatment. Theyre hopeful about her prognosis.

We just knew we couldnt give up, Weihs said.

Weihs told NBC 5 Responds the family is now focused on helping other kids get access to a drug they believe is priceless.

I don't care how expensive it is. It's a child's life. Every baby deserves a chance, said Porter.

Novartis said more than 1,800 patients have been treated with Zolgensma worldwide.

Texas newborn screening program began screening for SMA last June. The states health and human services website reports SMA is among the leading genetic causes of death among infants and toddlers.

NBC 5 Responds is committed to researching your concerns and recovering your money. Our goal is to get you answers and, if possible, solutions and a resolution. Call us at 844-5RESPND (844-573-7763) orfill out our customer complaint form.

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Texas Family Fights to Access $2.1 Million Treatment for Baby - NBC 5 Dallas-Fort Worth

There are few things more grueling than running a marathon.

One of those is battling cancer.

A La Conner native knows all about the former, having previously run the Denver Colfax Marathon. Now shes helping bring greater awareness to the latter by raising funds for the Leukemia & Lymphoma Society as she trains for the Chicago Marathon in October.

Morgan Harlan, a 2020 Baylor University grad now teaching kindergarten in Denver, is hoping to raise $4,000 for LLS by running the urban Chicago course with two friends this fall. The Chicago Marathon is typically viewed by more than a million spectators.

I hope to raise as much money as I possibly can for such a worthy cause that is so important to my family, she told the Weekly News on Friday.

Her family has seen first-hand the life-saving potential of bone marrow and blood stem cell transplants, and is committed to helping find cures and ensure access to treatments for all blood cancer patients.

Harlans grandfather, longtime La Conner resident and Dunlap Towing retiree Mit Harlan, waged a successful battle against leukemia over a decade ago.

My grandfather, said Harlan, is alive because of a stem cell transplant.

While a student at Baylor, where she was a journalism/public relations major and played club soccer, Harlan signed up for Be the Match, which connects patients with transplant donors.

As a college student with a family member who had experienced cancer, said Harlan, I thought I was doing my due diligence by signing up for the registry.

Last December, four years after joining Be the Match, Harlan flew to Seattle to donate her stem cells.

Her patient was a 65-year-old male with leukemia the same age her grandfather was when he received his transplant.

When Be the Match called to inform me that I was the match and asked me if I would be willing to donate my stem cells, Harlan added, my response was: Absolutely. I hope I can give another little girl or boy more time with their grandpa like I was given.

Harlan has not stopped there. She has taken on fundraising for the cause, doing so in a way that shows she is in it for the long run.

She has enlisted a coach, La Conner alum Carlee Daub, to help her train for Chicago. Daub is an owner of Wahoo Running, an online platform that provides coaching to runners throughout the nation.

My first marathon, Harlan recalled, I was focused on completion. I wanted to prove to myself that I had the physical and mental grit to get through 26.2 miles. The Chicago Marathon will be focused more on speed and race strategy.

As Harlan has lowered her running times, her fundraising numbers have increased.

My fundraising has gone really well because of the wonderful people around me, she said. I am very thankful to have generous family members, friends, and community members.

My original goal was to raise $2,000, Harlan explained, which I was able to raise in the first week. I have since raised my goal to $4,000.

Committing to run the Chicago Marathon on behalf of LLS is a big step for Harlan. After graduating from Burlington-Edison High School, having competed in soccer and track there, Harlan chose to go out of state for college.

I wanted to travel outside of Washington for my four years of college and live somewhere new, she said. Baylor had a great mix of academic strength, athletics success and extracurriculars.

While on the Waco, Texas campus, Harlan regularly wrote for the student newspaper, the Baylor Lariat.

Now, as she preps for the Chicago Marathon and generates support for LLS, Harlan is making rather than reporting the news.

For her, its a story whose headliner is her grandfather.

Hes one of the best humans I know, said Harlan. Growing up, he never missed a soccer match (of mine), including a tournament in Spain. Hes very giving with his time and money, especially towards charities like LLS.

Harlan, daughter of Mike and Jennifer Harlan, of Landing Road, southeast of La Conner, said the best ways to donate are through either her donation page: (https://pages.lls.org.tnt/rm.chicago22/MHarlan) or Facebook.

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La Conner native raising funds to cure blood cancer - La Conner Weekly News

In a press release issued earlier today by Affimed N.V. (Nasdaq: AFMD), please note that the headline named an incorrect organization. It should have mentioned the annual meeting of the American Society of Clinical Oncology (ASCO), not the American Association for Cancer Research. The corrected release follows:

Affimed Announces Clinical Update and Trial in Progress Posters at the Annual Meeting of the American Society of Clinical Oncology (ASCO)

HEIDELBERG, Germany, April 27, 2022 (GLOBE NEWSWIRE) -- Affimed N.V. (Nasdaq: AFMD), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, today announced that four abstracts with clinical trial designs and clinical data of its innate cell engagers (ICE) have been accepted for presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting, taking place June 3-7, 2022 in Chicago, IL.

The events include an oral presentation by Yago Nieto, M.D., Ph.D., professor of Stem Cell Transplantation and Cellular Therapy at The University of Texas MD Anderson Cancer Center with an update of the study that evaluates AFM13 pre-complexed with NK cells in patients with relapsed/refractory CD30-positive lymphomas. In addition, three Trial in Progress posters will be presented to provide background information and introduce the study design of the three ongoing AFM24 studies in which patients with EGFR-positive solid tumors are treated with AFM24 monotherapy or combinations with either Roches checkpoint inhibitor atezolizumab or NKGen Biotechs NK cell product SNK01.

Oral presentation details:

Title: Innate cell engager (ICE) AFM13 combined with preactivated and expanded cord blood (CB)-derived NK cells for patients with refractory/relapsed CD30+ lymphoma

Authors: Yago Nieto, Pinaki Banerjee, Indreshpal Kaur, Roland Bassett, Lucila Kerbauy, Rafet Basar, Mecit Kaplan, Lori Griffin, Daniel Esqueda, Christina Ganesh, Melissa Barnett, Amin Alousi, Chitra Hosing, Jeremy Ramdial, Neeraj Saini, Samer Srour, Sairah Ahmed, Swaminathan Iyer, Hun Lee, Ranjit Nair, Raphael Steiner, Karenza Alexis, Andreas Harstrick, Elizabeth J Shpall, Katayoun Rezvani

Oral session: Hematologic Malignancies Lymphoma and Chronic Lymphocytic Leukemia, Friday, June 3, 2022, 1:00 - 4.00 p.m. CDT

Poster details:

Title: A phase 1/2a open label, multicenter study to assess the safety, tolerability, pharmacokinetics, and efficacy of AFM24 in patients with advanced solid cancers: Study design and rationale.

Authors: Omar Saavedra Santa Gadea, Elena Garralda, Juanita Suzanne Lopez, Mark M. Awad, Jacob Stephen Thomas, Crescens Diane Tiu, Daniela Morales-Espinosa, Christa Raab, Bettina Rehbein, Gabriele Hintzen, Kerstin Pietzko, Paulien Ravenstijn, Michael Emig, Anthony B. El-Khoueiry

Poster details:

Title: AFM24 in combination with atezolizumab in patients with advanced EGFR-expressing solid tumors: Phase 1/2a study design and rationale.

Authors: Omar Saavedra Santa Gadea, Eric Christenson, Anthony B. El-Khoueiry, Andres Cervantes, Christa Raab, Ulrike Gaertner, Kerstin Pietzko, Gabriele Hintzen, Paulien Ravenstijn, Daniela Morales-Espinosa, Juanita Suzanne Lopez

Poster details:

Title: The combination of CD16A/EGFR innate cell engager, AFM24, with SNK01 autologous natural killer cells in patients with advanced solid tumors.

Authors: Anthony B. El-Khoueiry, Paul Y. Song, Jennifer Rubel, Dorna Y. Pourang, Christa Raab, Gabriele Hintzen, Michael Emig, Pilar Nava-Parada

Poster session for all posters: Developmental Therapeutics Immunotherapy, Sunday, June 5, 2022, 8:00 11:00 a.m. CDT

Abstract release: The full abstracts will become public at 5:00 p.m. EDT on Friday, May 26.

More details about the programs for the ASCO Annual Meetings are available online at http://www.asco.org

About AFM13

AFM13 is a first-in-class innate cell engager (ICE) that uniquely activates the innate immune system to destroy CD30-positive hematologic tumors. AFM13 induces specific and selective killing of CD30-positive tumor cells, leveraging the power of the innate immune system by engaging and activating natural killer (NK) cells and macrophages. AFM13 is Affimeds most advanced ICE clinical program and is currently being evaluated as a monotherapy in a registration-directed trial in patients with relapsed/refractory peripheral T-cell lymphoma (REDIRECT, NCT04101331).

In addition, The University of Texas MD Anderson Cancer Center is studying AFM13 in an investigator-sponsored phase 1/2 trial in combination with cord blood-derived allogeneic NK cells in patients with relapsed/refractory CD30-positive lymphomas (NCT04074746).

About AFM24

AFM24 is a tetravalent, bispecific innate cell engager (ICE) that activates the innate immune system by binding to CD16A on innate immune cells and EGFR, a protein widely expressed on solid tumors, to kill cancer cells. Generated by Affimeds fit-for-purpose ROCK platform, AFM24 represents a distinctive mechanism of action that uses EGFR as a docking site to engage innate immune cells for tumor cell killing through antibody-dependent cellular cytotoxicity and antibody-dependent cellular phagocytosis.

Affimed is evaluating AFM24 in patients with advanced EGFR-expressing solid malignancies whose disease has progressed after treatment with previous anticancer therapies as monotherapy and in combinations with other cancer treatments. AFM24-101, a monotherapy, first-in-human phase 1/2a open-label, is a non-randomized, multi-center, multiple ascending dose escalation and expansion study. Additional details may be found at http://www.clinicaltrials.gov using the identifier NCT04259450. Furthermore, AFM24 is being evaluated in a phase 1/2a study in combination with Roches anti-PD-L1 checkpoint inhibitor atezolizumab (AFM24-102, NCT05109442). Affimed and NKGen Biotech have initiated a phase 1/2a study (AFM24-103), investigating AFM24 in combination with SNK01, NKGen Biotechs NK cell product (NCT05099549).

About Affimed N.V.

Affimed (Nasdaq: AFMD) is a clinical-stage immuno-oncology company committed to give patients back their innate ability to fight cancer by actualizing the untapped potential of the innate immune system. The companys proprietary ROCK platform enables a tumor-targeted approach to recognize and kill a range of hematologic and solid tumors, enabling a broad pipeline of wholly-owned and partnered single agent and combination therapy programs. The ROCK platform predictably generates customized innate cell engager (ICE) molecules, which use patients immune cells to destroy tumor cells. This innovative approach enabled Affimed to become the first company with a clinical-stage ICE. Headquartered in Heidelberg, Germany, with offices in New York, NY, Affimed is led by an experienced team of biotechnology and pharmaceutical leaders united by a bold vision to stop cancer from ever derailing patients lives. For more about the companys people, pipeline and partners, please visit: http://www.affimed.com.

Investor Relations Contact

Alexander FudukidisDirector, Investor RelationsE-Mail: a.fudukidis@affimed.comTel.: +1 (917) 436-8102

Media Contact

Mary Beth Sandin Vice President, Marketing and CommunicationsE-Mail: m.sandin@affimed.com Tel.: +1 (484) 888-8195

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CORRECTING and REPLACING -- Affimed NV - GlobeNewswire

Heres a look at other notable health studies, innovations and research from around the world. Links will take you to press releases or journal entries.

Researchers Identify Specific Genes that Play Key Role in SchizophreniaSchizophrenia is a serious psychiatric disorder that starts in late adolescence or early adulthood and affects around 1 in 300 people worldwide, according to the World Health Organization.

Inflammasome activation in infected macrophages drives COVID-19 pathologySevere COVID-19 is characterized by persistent lung inflammation, inflammatory cytokine production, viral RNA, and sustained interferon (IFN) response all of which are recapitulated and required for pathology in the SARS-CoV-2 infected MISTRG6-hACE2 humanized mouse model of COVID-19 with a human immune system.

Cancer cells use self-inflicted DNA breaks to evade growth limits imposed by genotoxic stressGenotoxic treatments, such as radiation and some chemotherapy drugs, are a mainstay of cancer therapy, but they often fail to fully destroy tumor cells.

Unlocked Enzyme Structure Shows How Strigolactone Hormone Controls Plant GrowthAs sessile organisms, plants have to continually adapt their growth and architecture to the ever-changing environment.

P-wave and S-wave response of coal rock containing gas-water with different saturation: an experimental perspectiveThe acoustic response of gas and/or water saturated coal rock is fundamental for establishing the correspondence between the physical properties of the coal reservoir and the characteristics of the well-logging response, which is the technology essential for the geophysical exploration of coalbed methane (CBM).

Lipofilling procedure improves pain and function in finger osteoarthritisFor patients with painful finger osteoarthritis, a nonsurgical procedure called lipofilling in which fat obtained from another part of the body is transferred into the arthritic joints produces lasting improvements in hand function and especially pain, suggests a study in the May issue ofPlastic and Reconstructive Surgery,the official medical journal of theAmerican Society of Plastic Surgeons(ASPS).

Neural pathway key to sensation of pleasant touch identifiedStudying mice, scientists at Washington University School of Medicine in St. Louis have identified a neural circuit and a neuropeptide a chemical messenger that carries signals between nerve cells that transmit the sensation known as pleasant touch from the skin to the brain.

Engineers at UBC get under the skin of ionic skinIn the quest to build smart skin that mimics the sensing capabilities of natural skin, ionic skins have shown significant advantages.

Higher COVID-19 Death Rates in the Southern U.S. Due to Behavior DifferencesDuring the pre-Omicron phases of the COVID-19 pandemic, regions of the U.S. had markedly different mortality rates, primarily due to differences in mask use, school attendance, social distancing and other behaviors.

Rabies shows how scale of transmission can enable acute infections to persist at low prevalenceRabies is a deadly zoonotic disease that causes tens of thousands of deaths every year, mainly among African and Asian children.

Predictive metabolic networks reveal sex- andAPOEgenotype-specific metabolic signatures and drivers for precision medicine in Alzheimers diseaseLate-onset Alzheimers disease (LOAD) is a complex neurodegenerative disease characterized by multiple progressive stages, glucose metabolic dysregulation, Alzheimers disease (AD) pathology, and inexorable cognitive decline.

High-Frequency Spinal Cord Stimulation Shows Improved Longer Lasting Pain ReliefSpinal cord stimulation (SCS) for chronic pain involves delivering low levels of electricity directly into the spinal cord using an implanted device, which modifies or blocks nerve activity to minimize the sensation of pain reaching the brain.

Nemours Childrens Health Researchers Awarded $10.9 Million NIH COBRE Grant Supporting Work Well Beyond MedicineThe National Institutes of Health (NIH) recently awarded a Phase 1, 5-year $10.9 million Center of Biomedical Research Excellence (COBRE) award, entitled Research Expanding Access to Child Health (REACH) Center to Anne Kazak, PhD, Enterprise Director, and Melissa Alderfer, PhD, Director of the Center for Healthcare and Delivery Science (CHDS) at Nemours Childrens Health.

Understanding Black griefThe average life span for Black Americans is 78 years six years shorter than it is for white Americans. Compared with white Americans, Black Americans are twice as likely to die of heart disease, 50% more likely to have high blood pressure and are likelier to die at earlier ages of all causes.

Viewing a Microcosm Through a Physics LensWhat can physics offer biology? This was howAlison Patteson, assistant professor in the College of Arts and Sciences physics department and also a faculty member in theBioInspired Institute, began the explanation of why her physics lab was studying bacteria.

CAR T drives acute myeloid leukemia into submission in pre-clinical studiesMassachusetts General Hospital (MGH) researchers have developed a novel treatment strategy that has the potential to bring the life-saving benefits of chimeric antigen receptor T-cell therapy (CAR T) to patients with acute myeloid leukemia (AML) the most common form of leukemia in adults.

Tufts Researchers Discover New Function Performed by Nearly Half of Brain CellsResearchers at Tufts University School of Medicinehave discovered a previously unknown function performed by a type of cell that comprises nearly half of all cells in the brain.

Study exhibits sleep deprivation impairs stem cells within the corneaSleep deprivation, which implies getting too little high-quality sleep, is a critical well being downside.

Historic Redlining and Contemporary Behavioral Health Workforce DisparitiesAs the nation continues to confront the lasting legacy of Jim Crowera structural racism, attention is increasingly turning to the association between historical redlining policies and contemporary racial disparities in access to health care, including behavioral health.

Gut microbiome could alter response to most cancers remedySince historic instances, our intestine microbiome, dwelling to an unlimited variety of micro organism, viruses, fungi, and different microorganisms, has been thought to affect many facets of human well being.

Researchers Share Insights about Mechanisms of Human Embryo and Create Method to Develop Transcriptionally Similar Cells in Tissue CultureBlood-forming stem cells found in bone marrow are the life-saving component used in bone marrow transplants.

Seven hours of sleep is optimal in middle and old age, say researchersSeven hours is the ideal amount of sleep for people in their middle age and upwards, with too little or too much little sleep associated with poorer cognitive performance and mental health, say researchers from the University of Cambridge and Fudan University.

Stanford scientists found that free-living runners default to an energy-saving speed, no matter the distanceStanford University scientists have found that when recreational runners are left to their own devices and outfitted with a wearable fitness tracker, they prefer to run at the same calorie-saving pace, regardless of the distance ran contrary to the explicit goals of competitive racing.

Historic Redlining and Contemporary Behavioral Health Workforce DisparitiesAs the nation continues to confront the lasting legacy of Jim Crowera structural racism, attention is increasingly turning to the association between historical redlining policies and contemporary racial disparities in access to health care, including behavioral health.

New Article Outlines the Characteristics of a Longevity DietExamining a range of nutrition research from studies in laboratory animals to epidemiological research in human populations provides a clearer picture of the best diet for a longer, healthier life, said USC Leonard Davis School of Gerontology professorValter Longo.

Study tracks COVID-19 infection dynamics in adultsA team led by scientists at the University of Illinois Urbana-Champaign tracked the rise and fall of SARS-CoV-2 in the saliva and nasal cavities of people newly infected with the virus.

Humans run at the most energy-efficient speed, regardless of distanceAs race season approaches, many runners have the same goal: go faster. But in a study publishing April 28 in the journalCurrent Biology, researchers show that speeding up might require defying our natural biology.

Not All Dietary Fibers Are Equal: Heres WhyThats according to anew studyTrusted Sourcein which researchers found that the benefits of fiber can depend on the type of fiber, the amount of fiber, and the individual consuming the fiber.

Combination of weak muscles and abdominal obesity can be an early sign of functional decline in menA study conducted at the Federal University of So Carlos (UFSCar) in Brazil suggests that early detection of functional decline the dwindling capacity to perform everyday tasks independently is possible by observing patients as they engage in simple actions such as sitting down and getting up from a chair, standing still, and walking a short distance.

Changing Guidelines for Treating Mild Chronic Hypertension in PregnancyA study published this month in theNew England Journal of Medicineproves through a large clinical trial that treating high blood pressureeven mild casesduring pregnancy is safe and beneficial for both mother and developing baby.

New report calls on bioethics to take a stand against anti-black racismA new Hastings Center special report calls on the field of bioethics to take the lead in efforts to remedy racial injustice and health inequities in the United States.

Mother and child vulnerable to endocrine disruptor exposureThey can be found in cosmetics, plastic containers, furniture, toys, or baby bottles. Endocrine disruptors, molecules disrupting our hormones, are everywhere in our daily lives.

Supporting school-community collaboration for the implementation of a multi-tiered school mental health program: The Behavioral Health Team modelInvesting in school mental health programs has the potential to improve youths access to mental health services.

Inclusive Design and Research Methods Will Lead To More Innovative, Intelligent TechnologyThat observation is at the heart of her latest research exploring how older Black adults in lower income environments feel about asking health questions, how they pose those questions verbally, and whether voice assistant devices respond as expected.

From Blurry to Bright: AI Tech Helps Researchers Peer into the Brains of MiceJohns Hopkins biomedical engineers have developed an artificial intelligence (AI) training strategy to capture images of mouse brain cells in action.

MD Anderson and Community Health Network announce partnership to create fully integrated cancer programThe University of Texas MD Anderson Cancer Centerand Indianapolis-based Community Health Networktoday announced a partnership agreement to create Community Health Network MD Anderson Cancer Center.

Improving mental health in Multiple Sclerosis with an interpersonal emotion regulation intervention: A prospective, randomized controlled trialOver a third of people with Multiple Sclerosis (PwMS) struggle with poor mental health, which exacerbates physical symptoms and complicates clinical treatment.

Vaccination campaign messages often prove ineffectiveA study in eight European countries shows that information on the benefits of vaccines can even reduce the willingness to get immunized.

Online health and wellbeing program using singing techniques can improve quality of life and breathlessness after COVID-19There are few evidence-based interventions for long COVID; however, holistic approaches supporting recovery are advocated.

A complete ban on all smoking would not improve healthy life expectancy for 40 yearsThe negative impact of smoking on health inequalities in the UK means even if smoking stopped tomorrow, the full health benefits would not be seen until 40 years down the line.

UTHealth Houstons UTMOVE program receives distinguished Edmond J. Safra Fellowship in Movement DisordersUTHealth Houstons Movement Disorders and Neurodegenerative Diseases Fellowship Training Program (UTMOVEfellowship program) has been chosen byThe Michael J. Fox Foundation for Parkinsons Research(MJFF) as one of eight international academic centers to train a new movement disorder clinician-researcher a neurologist with additional training and expertise in diagnosing and treating Parkinsons and related diseases as part of the Edmond J. Safra Fellowship in Movement Disorders Class of 2025.

More than a million smokers likely to quit after U.S. bans menthol cigarettesA new study projects that a U.S. ban on menthol cigarettes, proposed by the U.S. Food and Drug Administration, will lead more than 1.3 million smokers to quit. Among them, Black smokers will see the greatest impact.

Pediatric transplant patients may skip adult appointmentsYoung adults who received organ transplants as childrenmay not be regularly attending their doctor appointments after leaving their pediatric providers.

Large-Scale Social Media Analysis Reveals Emotions Associated with Nonmedical Prescription Drug UseThe behaviors and emotions associated with and reasons for nonmedical prescription drug use (NMPDU) are not well-captured through traditional instruments such as surveys and insurance claims.

New insight in patient response to surgical disruption in life-saving hormonesCardiac surgery patients may experience different levels of disruption to their body producing life-saving hormones during their operations, a new study reveals.

Radiologists, AI Systems Show Differences in Breast-Cancer Screenings, New Case Study FindsRadiologists and artificial intelligence systems yield significant differences in breast-cancer screenings, revealing the potential value of using both human and AI methods in making medical diagnoses.

Montefiore Einstein Cancer Center Finds CAR-T Therapy Effective in Black and Hispanic PatientsCAR-T therapy, a form of immunotherapy that revs up T-cells to recognize and destroy cancer cells, has revolutionized the treatment of blood cancers, including certain leukemias, lymphomas, and most recently, multiple myeloma.

Tackling the Consequences of Long CovidA research team at the University of Zurich has helped people affected by Long Covid identify the problems they most urgently want scientists to tackle, through a collaborative citizen science approach.

Self-sampling for cervical screening offered at the point of invitation: A cross-sectional study of preferences in EnglandThis study assessed preferences for human papillomavirus (HPV) self-sampling if offered as an alternative to clinician-based screening at the point of invitation for cervical screening.

New study identifies genetic changes in patients who progress to esophageal cancerLed by researchers at Fred Hutchinson Cancer Research Center, a scientific team that studies a precancerous condition of the esophagus (called Barretts esophagus or BE) are working to answer this question.

Risk Factors for Severe COVID-19 in Hospitalized Adults Differ by AgeA just-published study provides previously unknown answers about which hospitalized COVID-19 patients are most likely to need mechanical ventilation or to die.

Hypoxia-activated neuropeptide Y/Y5 receptor/RhoA pathway triggers chromosomal instability and bone metastasis in Ewing sarcomaAdverse prognosis in Ewing sarcoma (ES) is associated with the presence of metastases, particularly in bone, tumor hypoxia and chromosomal instability (CIN).

Unravelling the origins of the human spineEMBL Barcelona scientists have recapitulated for the first time in the laboratory how the cellular structures that give rise to our spinal column form sequentially.

New Study Finds Climate Change Could Spark the Next PandemicAs the Earths climate continues to warm, researchers predict wild animals will be forced to relocate their habitats likely to regions with large human populations dramatically increasing the risk of a viral jump to humans that could lead to the next pandemic.

How brains form visual mapsMaps have played an important role in scientific progress. Claudius Ptolemaeus transformed our understanding of the world with his map of Earth and Tycho Brahe our understanding of the Universe with his map of the stars.

Efficient dendritic learning as an alternative to synaptic plasticity hypothesisSynaptic plasticity is a long-lasting core hypothesis of brain learning that suggests local adaptation between two connecting neurons and forms the foundation of machine learning.

More Relaxation and Less Stress Through Combined Yoga TechniquesYoga is often equated with acrobatic stretching exercises that are supposed to induce relaxation and a better body awareness.

Carrier for Chemotherapy Medications CreatedA group of scientists at Ural Federal University has proposed the use of polyoxometallate nanoclusters as a carrier for chemotherapy medication.

Free Fundamental Biology of Endocrine, Metabolic & Resistant conditionsThereference Free Radical Biology of Endocrine, Metabolic & Immune disorders uniquely explores the science of signalling mechanisms associated with diseases like endocrine, metabolic, and immune disorders which are linked to oxidative stress-mediated disease mechanisms.

All Cells Are Important: A Roadmap to Characterize Lymphoma StromaLymphomas are blood cancers that often start from lymph nodes. Lymph nodes contain not only hematopoietic cells, mainly B- and T-lymphoid cells, but also non-hematopoietic cells (NHCs), also called stromal cells.

Machine learning can help address stigma of substance abuse in developing countriesNow, a research team is using machine learning and anonymized data to get a clearer picture of the underlying factors that influence tendencies to abuse drugs and alcohol.

Gene mutations that contribute to head and neck cancer also provide precision treatment targetsAbout one-fifth of often deadly head and neck cancers harbor genetic mutations in a pathway that is key to normal cell growth, and scientists report those mutations, which enable abnormal cancer cell growth, can also make the cancer vulnerable.

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StudyFinds Blotter: Other Notable Health Research From April 28, 2022 - Study Finds

Sung takes over at San Antonio cancer institute

Patrick Sung, a professor, interim department chair and associate dean for research at the University of Texas Health Science Center at San Antonio's Long School of Medicine, has taken on another leadership role there. On March 1, he became the new director of the Greehey Children's Cancer Research Institute. He succeeds interim director Manjeet Rao and former director Peter Houghton.

Patrick Sung

The institute, founded in 2004, is a group of 18 labs doing research focused on topics related to pediatric cancers, including cancer genomics, DNA repair, tumor biology and drug development. Research in Sungs lab, which is part of the institute, focuses on DNA damage repair. He studies homologous recombination as a mechanism for repairing double-stranded DNA breaks, focusing on the mechanism of the recombinase Rad51. His lab is known for recapitulating double-stranded DNA repair in vitro. Failure of such repair can lead to chromosomal rearrangements that drive the development of cancer; at the same time, cancer cells are unusually adept at repairing DNA damage. Several years ago, Sungs lab found that Rad51 interacts with the well-known BRCA tumor suppressor proteins, suggesting new insights into how BRCA proteins suppress tumor formation.

Sung earned his Doctor of Philosophy degree in biochemistry at the University of Oxford in 1985. He came to the U.S. for a postdoc at the University of Rochester. After starting his faculty career at the University of Texas Medical Branch in Galveston, he worked as an associate professor at UT Health San Antonio before taking a position at Yale in 2003, in the department of molecular biophysics and biochemistry, which he later chaired. He was recruited back to the University of Texas in 2019 as a professor.

Sung has been an associate editor of the Journal of Biological Chemistry since 2014. He also is on the editorial board of the journal Genes and Development and formerly served on the editorial board of the journal Molecular & Cellular Biology.

Adriana Bankston, a legislative analyst for the University of California, has received a 2022 fellowship from Advancing Research Impact in Society, or ARIS, a program supported by the National Science Foundation.

Adriana Bankston

This award, shared with the University of California, Irvine's Harinder Singh, will support a program for training in science policy. The project, titled "Developing the next generation workforce through science policy as a bridge between science and society," will use insights from a course that Bankston and Singh taught at Irvine to develop an educational toolkit for universities and to build a community of practice in science policy and advocacy.

Bankston received her Ph.D. in biochemistry and cell and developmental biology at Emory University and was a postdoctoral researcher at the University of Louisville before becoming a policy and advocacy fellow at the Society for Neuroscience. Today, in addition to her position at UC, she works on numerous initiatives as chief executive officer and managing publisher of the Journal of Science Policy and Governance, and as a research investigator with the STEM Advocacy Institute. In February, she was part of a panel discussion hosted by the National Academies of Science, Engineering and Medicine's strategic council for research excellence, integrity and trust. She is also an ASBMB Today contributor.

The Center for Advancing Research Impact in Society is a project to improve public engagement with science and diversify the research workforce. Its fellows, selected annually, work on projects that synthesize research to help scientists achieve these goals.

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Sung to lead cancer institute; Bankston wins fellowship - ASBMB Today

Houston teen first in Texas to become own donor to reverse sickle cell anemia

Here's a huge advancement in treating sickle cell disease! A teen patient at Texas Children's Cancer and Hematology Center in Houston is the first patient in Texas to become her own donor to reverse her rare blood disorder.

HOUSTON - 17-year-old Helen Ndku has been suffering severe pain from sickle cell disease her whole life. The first major crisis, when she was only one year old.

"She actually died in my hands, and we called the ambulance," remembers Helen's mother Enita Odofin. "She came back to life, we went to the hospital, but she died there again, before they brought her back to life."

RELATED: Teen cowboy dies on horse but is brought back to life

Helen's life was spared, but her pain wasn't.

"When I was little, I remember times where I'd be in the middle of class, and I just started crying because I was in so much pain. I remember not being in school very often. Going home a lot," explains Helen. "I just remember crying, I cried really hard. It's a pain that goes everywhere. It's kind of like needles, maybe bricks, just stacked on top of you squeezing sometimes pulling? I don't know. Yes, it just feels like your body's decaying in the worst ways possible."

Sickle cell anemia can affect anyone; however, it mostly affects African Americans, followed by Hispanics.

"There's a single genetic mutation that causes the red blood cells, the cells in the body to deliver oxygen to become inflexible," says Dr. Tami John. "Under stress, they will stick like create kind of a sickle C shape, and those themselves then get stuck in small blood vessels throughout the body and cause things like the significant pain. They cause organ damage."

Dr. John is a Pediatric Hematologist-Oncologist who specializes in stem cell transplants at Texas Children's Cancer and Hematology Center, as well as an Assistant Professor at Baylor College of Medicine. She's heading the clinical trial for sickle cell.

Until now, there was only one treatment option for children with sickle cell, but it only helped manage the condition and still often required monthly blood transfusions.

But now, a possible cure is on the horizon and many families and researchers are watching and waiting to see how it works for Helen.

"She's the very first patient at the Texas Medical Center to receive gene modification therapy. There have been others and on this particular clinical trial. There are about 50 other people with sickle cell disease who have received this therapy. She is the first in the Texas Medical Center and the first in Texas, as well as one of the youngest patients," says Dr. John.

RELATED: Toddler paralyzed by rare Guillain-Barr Syndrome still recovering

Helen had her doubts at first. "I didn't have hope for it to be honest. I thought it was a lost cause," explains Helen.

However, she agreed to go for it!

Siblings often act as a stem cell donor, but that wasn't an option for Helen.

"What we were able to do was take stem cells from Helen and take those to a lab where a scientist modified those cells with a new gene, we inserted a new gene and then we were able to give those stem cells back to her and watch them grow and they produced new hemoglobin," explains Dr. John.

It takes one year to find out the results, and it has now been a year for Helen.

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"From everything we've seen, she has no evidence of sickle cell disease. She clinically has changed. She's been able to stop all of her chronic pain medications and really start to engage in everyday activities that for years and years, she just wasn't able to do. The concept of a cure is still under discussion in the medical community about gene therapies. Clinically, her symptoms have resolved," exclaims Dr. John.

Because she feels well for the first time ever, Helen just learned how to ride a bike. She can finally sit up straight for longer than an hour, plus she can walk and exercise for the first time.

Helen has more hope than ever but is still adjusting to what it feels like to be pain-free.

"I think it's strange. It's not it's not normal to me to like, not be sick all the time, so it's like foreign land, I don't know how to conquer it yet, but I'm working on it," says Helen. "She's amazing, it's night and day for me," says her smiling mom.

While about 50 other patients have also undergone this procedure, it's still in clinical trials, but doctors believe this could brighten the future of many patients with sickle cell.

"This is a huge breakthrough to be able to discuss and share with patients I think it can be life changing, as we've seen, and the hope is we see it become more and more available," says Dr. John.

RELATED: Couple from Alabama seeks life-saving care for twins at Texas Childrens Hospital

Helen never imagined she'd be well enough to go to college. Now she's doing so well, she may even go out-of-state to her favorite school of choice.

Click here for more information on Texas Children's Sickle Cell Program.

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Houston teen first in Texas to become own donor to reverse sickle cell anemia - FOX 26 Houston

Summary: Oleic acid produced in the brain is an essential regulator of processes that enable memory, learning, and mood regulation. Oleic acid, which is abundant in olive oil, also promoted neurogenesis and increases cell proliferation.

Source: Baylor College of Medicine

Many people dread experiencing the cognitive and mood declines that often accompany reaching an advanced age, including memory disorders such as Alzheimers disease and mood conditions like depression.

While searching for new ways to prevent or treat these and other related conditions, a team at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Childrens Hospital identified a missing piece of the puzzle of how memory and mood are sustained and regulated in the brain.

Their study, published in theProceedings of the National Academy of Sciences, reveals that oleic acid produced in thebrainis an essential regulator of the process that enables learning and memory and supports proper mood regulation. The finding has paved the path to discovering potential new therapeutic strategies to counteract cognitive and mood decline in patients with neurological disorders.

Years ago, scientists thought that the adult mammalian brain was not able to repair and regenerate. But research has shown that somebrain regionshave the capacity of generating new neurons, a process called neurogenesis.

The hippocampus region of the adult mammalian brain has the ongoing capacity to form new neurons, to repair and regenerate itself, enabling learning and memory and mood regulation during the adult life, said co-corresponding author Dr. Mirjana Maletic-Savatic, associate professor of pediatrics-neurology at Baylor and Texas Childrens and an investigator at the Duncan NRI.

Ever since neurogenesis was discovered, it has been envisioned as the fountain of youth. But, with increasing age, in certain diseases or after exposure to certain drugs or insults, neurogenesis decreases and this has been associated withcognitive declineand depression.

In this study, the team searched for a way to tap into the fountain of youth, to reignite the process of neurogenesis to prevent its decline or restore it.

We knew that neurogenesis has a master regulator, a protein withinneural stem cellscalled TLX that is a major player in the birth of new neurons. We however did not know what stimulated TLX to do that. Nobody knew how to activate TLX, said co-corresponding author Dr. Damian Young, associate professor of pharmacology andchemical biologyand of pathology at Baylor and Texas Childrens and member of Baylors Dan L Duncan Comprehensive Cancer Center.

We discovered that a common fatty acid called oleic acid binds to TLX and this increasescell proliferationand neurogenesis in the hippocampus of both young and old mice, said co-first author Dr. Prasanna Kandel, who was in the graduate program of Integrative Molecular and Biomedical Sciences at Baylor while working on this project.

This oleic acid is produced within the neural stem cells in order to activate TLX.

While oleic acid is also the major component inolive oil, however, this would not be an effective source of oleic acid because it would likely not reach the brain, the researchers explained. It must be produced by the cells themselves.

The finding thatoleic acidregulates TLX activation has major therapeutic implications.

TLX has become a druggable target, meaning that knowing how it is activated naturally in the brain helps us to develop drugs capable of entering the brain and stimulating neurogenesis, Young said.

This strategy could potentially be used to treat diseases such as major depressive disorders and Alzheimers disease. This is incredibly exciting because it provides a new way of treating these debilitating diseases in need of effective treatments.

Beside the scientific progress, I am hopeful that the current findings and ongoing related work will have real impact on people who are in need of improved and effective therapies, like my mother who suffers from clinical depression, Kandel said.

Author: Press OfficeSource: Baylor College of MedicineContact: Press Office Baylor College of MedicineImage: The image is in the public domain

Original Research: The findings will appear in PNAS

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Oleic Acid, a Key to Activating the Brains Fountain of Youth - Neuroscience News

TUESDAY, Nov. 16, 2021 (HealthDay News) -- Heart failure patients who fit a specific profile can benefit from injection of stem cells delivered directly into their heart muscle, a new study finds.

Patients with mild or moderate heart failure who have high levels of inflammation responded well to the stem cell injections, and experienced a decline in their risk of heart attacks, strokes and heart-related death, clinical trial results show.

Stem cells injected into targeted areas of a failing heart become activated by inflammation and start pumping out beneficial biochemicals, explained lead researcher Dr. Emerson Perin, medical director of the Texas Heart Institute in Houston.

"These cells are little factories of different proteins, cytokines and other products that then have an effect locally on the heart muscle cells," Perin said, adding that the cells also help improve the health of blood vessels both large and small.

For this clinical trial, Perin and his colleagues recruited 537 people suffering from advanced heart failure with reduced ejection fraction, which is when the main pumping chamber in the left side of the heart is significantly weakened.

Half of the patients chosen at random received 150 million stem cells into targeted areas of still-working heart muscle, delivered though 15 to 20 injections in a single procedure, Perin said.

The areas were selected using a mapping system that found places in the heart where electrical activity still occurred but might be hampered by inflammation.

The therapy did not significantly reduce the number of hospitalizations caused by heart failure, but the researchers found that it did improve participants' heart health in other ways during an average 30 months of follow-up:

These results show that a personalized approach with stem cell therapy can help some with heart failure, Perin said.

Doctors have the ability to single out patients with high levels of inflammation and either mild or moderate heart failure, "and then we're using a very precise way of delivering these cells in each individual patient exactly where they need to be placed," Perin said.

The findings were presented Sunday at the American Heart Association's online annual meeting. Research presented at medical meetings is considered preliminary until published in a peer-reviewed journal.

This new clinical trial "is really a promising study to provide further insights into potential subgroups of patients who may hopefully benefit from stem cell therapy," said Dr. Biykem Bozkurt, director of the Winters Center for Heart Failure at the Baylor College of Medicine in Houston.

Bozkurt said future studies should be able to further refine and identify exactly who would most benefit from stem cell therapy among heart failure patients.

"There's always definitely a need to do more research," Bozkurt said.

More information

The Cleveland Clinic has more about heart failure.

SOURCES: Emerson Perin, MD, PhD, medical director, Texas Heart Institute, Houston; Biykem Bozkurt, MD, PhD, director, Winters Center for Heart Failure, Baylor College of Medicine, Houston; American Heart Association annual meeting, Nov. 14, 2021, online presentation

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Stem Cell Therapy Boosts Outcomes for Some Heart Failure Patients - HealthDay

TOKYO & MUNICH & BASKING RIDGE, N.J.--(BUSINESS WIRE)--Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) today announced positive topline results from the global pivotal QuANTUM-First phase 3 trial evaluating quizartinib, a highly potent and selective FLT3 inhibitor, in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia (AML).1

QuANTUM-First met its primary endpoint, demonstrating that patients who received quizartinib in combination with standard induction and consolidation chemotherapy and then continued with single agent quizartinib had a statistically significant and clinically meaningful improvement in overall survival (OS) compared to those who received standard treatment alone. The safety of quizartinib was shown to be manageable and consistent with the known safety profile.

AML is one of the most common forms of leukemia in adults, representing about one-third of all cases.2 The five-year survival rate of AML is about 29%, and patients with FLT3-ITD positive AML have a particularly unfavorable prognosis, including an increased risk of relapse and shorter overall survival.1,3 There remains a high unmet need to improve survival for the majority of patients with AML.4

The results of the phase 3 QuANTUM-First trial showed that adding quizartinib, a potent and selective FLT3 inhibitor, to chemotherapy significantly prolonged overall survival in patients with newly diagnosed FLT3-ITD positive AML, said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. We look forward to sharing the QuANTUM-First data with the hematology community and will initiate discussions with global regulatory authorities.

Data from QuANTUM-First will be presented at an upcoming medical meeting and shared with regulatory authorities globally.

About QuANTUM-First

QuANTUM-First is a randomized, double-blind, placebo-controlled, multi-center global phase 3 study evaluating quizartinib in combination with standard induction and consolidation chemotherapy and then as continued single agent therapy in adult patients (age 18 75) with newly diagnosed FLT3-ITD positive AML.

Patients were randomized 1:1 into two treatment groups to receive quizartinib or placebo in combination with standard anthracycline and cytarabine-based induction and consolidation regimens. Eligible patients, including those who underwent allogenic hematopoietic stem cell transplant (HSCT), continued with single agent quizartinib or placebo for up to 36 cycles.

The primary study endpoint is OS. Secondary endpoints include event-free survival (EFS), post-induction rates of complete remission (CR) and composite complete remission (CRc), and the percentage of patients who achieve CR or CRc with FLT3-ITD minimal residual disease negativity. Safety and pharmacokinetics, along with exploratory efficacy and biomarker endpoints, were also evaluated.

QuANTUM-First enrolled 539 patients at approximately 200 study sites worldwide including in Asia, Europe, North America, Oceania and South America. For more information, visit ClinicalTrials.gov.

About Acute Myeloid Leukemia (AML)

More than 474,500 new cases of leukemia were reported globally in 2020 with more than 311,500 deaths.5 AML is one of the most common types of leukemia in adults, representing about one-third of all cases.2 A heterogenous blood cancer, AML is characterized by a five-year survival rate of about 29%, the lowest by far among the major leukemia subtypes.6,7

Treatment guidelines for patients with newly diagnosed AML recommend a cytarabine-based chemotherapy regimen with or without a targeted therapy as determined by the presence of genetic mutations, age and other factors.8 Patients with newly diagnosed FLT3 mutated AML may receive a FLT3 inhibitor as part of their initial treatment regimen and/or subsequent regimens.8 While intensive chemotherapy and/or HSCT can improve chances for sustained remission in eligible patients, a substantial proportion of patients are not suitable for either intervention, and cure rates are particularly low for older patients.1,6 In recent years, new targeted treatments have increased options and improved outcomes for some patients with molecularly defined AML subtypes.6

About FLT3-ITD

FLT3 (FMS-like tyrosine kinase 3) is a transmembrane receptor tyrosine kinase protein normally expressed by hematopoietic stem cells; FLT3 plays an important role in cell development by promoting cell survival, growth and differentiation through various signaling pathways.1 Mutations of the FLT3 gene, which occur in approximately 30% of patients with AML, can drive oncogenic signaling.1 The most common type of FLT3 mutation is the FLT3-ITD (internal tandem duplication), which is present in about 25% of all AML patients and contributes to cancer cell proliferation.1 Patients with FLT3-ITD mutations have a particularly unfavorable prognosis, including an increased risk of relapse and shorter overall survival.1

About Quizartinib

Quizartinib, an oral, highly potent and selective type II FLT3 inhibitor, is in phase 1/2 clinical development in pediatric and young adult patients with relapsed/refractory FLT3-ITD AML in Europe and North America.1 Several phase 1/2 combination studies with quizartinib are also underway at The University of Texas MD Anderson Cancer Center as part of a strategic research collaboration focused on accelerating development of Daiichi Sankyo pipeline therapies for AML.

Quizartinib is currently approved for use in Japan under the brand name VANFLYTA for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, as detected by an approved test. Quizartinib is an investigational medicine in all countries outside of Japan.

About Daiichi Sankyo Oncology

The oncology portfolio of Daiichi Sankyo is powered by our team of world-class scientists that push beyond traditional thinking to create transformative medicines for people with cancer. Anchored by our DXd antibody drug conjugate (ADC) technology, our research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in the U.S. We also work alongside leading academic and business collaborators to further advance the understanding of cancer as Daiichi Sankyo builds towards our ambitious goal of becoming a global leader in oncology by 2025.

About Daiichi Sankyo

Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose to contribute to the enrichment of quality of life around the world. In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an Innovative Global Healthcare Company Contributing to the Sustainable Development of Society. For more information, please visit http://www.daiichisankyo.com.

References

1 Daver N et al. Leukemia. (2019) 33:299312.2 American Cancer Society. Key Statistics for Acute Myeloid Leukemia. Updated January 2020.3 Leukemia and Lymphoma Society. Facts and Statistics. Leukemia: Survival (SEER Data for 2009-2015).4 Daver N et al. Blood Cancer Journal. (2020) 10:107.5 Global Cancer Observatory. Population Fact Sheet: World. Updated November 2020.6 Short et al. Cancer Discov. (2020);10:50625.7 Leukemia and Lymphoma Society. Facts and Statistics. Leukemia: Survival (SEER Data for 2009-2015).8 NCCN Practice Guidelines for Oncology. Acute Myeloid Leukemia. Version 3.2021 (March 2, 2021).

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Quizartinib Added to Chemotherapy Demonstrates Superior Overall Survival Compared to Chemotherapy Alone in Adult Patients with Newly Diagnosed...