Opinion: Primate research is cruel, expensive, unnecessary and happening here – OregonLive.com

David Gomberg

Gomberg, D-Central Coast, represents House District 10 in the Oregon Legislature.

Behind tall fences and security gates, on a pristine wooded campus at the edge of Hillsboro lies the Oregon National Primate Research Center. School groups are invited to visit where they will see thousands of non-human primates in large airy cages or walled corrals happily socializing, playing and eating. What they will not see, a few steps away, are rows of buildings where these magnificent creatures macaques, baboons and monkeys are the subject of medical research, simply because they share 93 percent of the same DNA as we human primates.

Last summer I asked to see the research rooms with my legislative staff. Inside we found stacks of small, 3-by-3-foot cages. Residents, we were told, stayed in those cages, inside those windowless rooms, for up to three years. As we entered, one baby primate cowered behind her mother. Dont worry, the technician reassured her. Im not here to take your baby. Not today, I thought.

The primate research center is now being sued by the People for the Ethical Treatment of Animals to release videos of maternal nutrition research. According to PETA, in these experiments, pregnant monkeys are fed special diets. Their babies are later separated and deliberately frightened to test their response to stress. In a 2010 project, $750,000 was spent on similar research where technicians either stared at the infants to intimidate them or used a Mr. Potato Head doll to frighten them, PETA contends.

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U.S. Department of Agriculture inspection reports document ongoing problems: stress-based hair loss, burns from electric heating pads, incorrect injections, cages too small to sit up in, accidental strangulation by chains in the enclosure, death from being trapped by PVC pipes, deaths from stress induced riots", death from anesthesia mistakes, death from dehydration, death from being given a toxic substance and death from inattention during childbirth. Some of these mistakes resulted in fines exceeding $10,000, but curiously, these penalties are not listed among reports posted to the centers web page.

Since 2017, the research center has been cited at least 10 times for Animal Welfare Act violations, the inspection reports show. That is substantially more than any other such center in the United States during this same period, according to the animal rights group Stop Animal Exploitation Now.

Center administrators told me that, even during normal research, about 500 primates are killed annually in terminal protocols examining aging, AIDS, depression, infectious diseases, substance abuse and obesity. The sad fact is that one in 10 primates in Hillsboro will not survive the year.

What I saw inside the primate research center were young animals trained to extend their arms from cages for easy examination.

The scientists I met at the center are proud of their work. But not so proud it seems that they want to tell people what they do. Instead, they requested legislation to keep their names from the public. These protections are so broad that even the identity of the company delivering monkey chow to the center cannot be released.

These same scientists will argue they are doing important work that will improve or save lives. Perhaps so. But who measures the results? And why dont we Oregonians know more?

An analysis from the Legislative Fiscal Office details that the center, a unit of Oregon Health & Science University, receives no state money but over $50 million annually in federal grants and funds. We should all be asking if the money is well spent.

Is research on non-human primates relevant to human primates? Is research done here in Oregon duplicative of work done at one of the other six national primate centers? Are non-primate research technologies like computer modeling less expensive and more accurate? We wont learn the answer to these questions because the primate research industry is a multi-billion-dollar enterprise funded by and fueled by people invested in doing things tomorrow the same way they did yesterday.

Ethologists, geneticists, and other scientists have taught us that primates share many physiological and emotional characteristics with human beings. They have feelings like us, they suffer like us, and they have social relationships that are important to them just as our relationships are to us. As a nation, weve ended the use of chimpanzees in invasive medical experiments. Its time we extend that same policy of no-testing to all primates.

For those of us who believe primate research is unnecessary and unproductive, the answer is not passionate protests at the center gates. We can only end these cruel practices by pulling the federal financial plug. The time has come for fundamental changes in our research practices and the treatment of our primate cousins.

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Opinion: Primate research is cruel, expensive, unnecessary and happening here - OregonLive.com

Into the Future With CAR-T Cell Therapy – Curetoday.com

An early clinical trial participant likens his journey into the unknown to a space mission.

For my wife and me, part of that involved the decision to discuss starting a family. After all, my father, my uncle and I were the only men in our small family it was left to me to keep our name going.

Of course, it wasnt ideal timing for starting a family. It was eight years after losing my father, and I was facing liver failure, kidney failure, a feeding tube strapped to my face and significant weight loss 55 pounds, to be exact. I was in immense pain, both physical and emotional, and felt anything but myself.

Just before I received my first treatment of chemotherapy R-CHOP, short for Rituxan (rituximab), cyclophosphamide, Adriamycin (doxorubicin), Oncovin (vincristine) and prednisone my doctors asked if my wife and I planned to ever have a family of our own. If so, we would need to bank some sperm immediately. Considering my physical condition at the time, I was in no mood to fulfill this obligation to secure a future with children in it. Dont misunderstand me I wanted nothing more than to have a family with my wife but in that moment, I didnt think it would be possible. I thought I was dying.

With just minutes to spare, due to the distanceof the fertility clinic from where I was in the city, we took a chance on having a future. I couldnt have done any of this without the unconditional support of my wife, Rachel, who carried us both when I was unable to stand on my own.

Later that night, I received my first chemotherapy treatment. Three hours into it we got the call from our fertility doctor, telling us that she could definitely get us pregnant. I never cry, but Idid then. The cancer and the looming possibility of death didnt exist in the moment that we learned we still might have a chance to become parents. It was exactly what we needed to hear to get through that tough first night of chemo.

The journey wasnt easy. I endured two years of treatments that included chemotherapy, immunotherapy, autologous stem cell transplant, more chemotherapy and various medical trials that I kept failing. Through it all, I adopted the mindset of an astronaut. Like an astronaut, I had an obligation to my fellow man to discover the unknown in hopes of finding something that would save not just my life but also the lives of the patients after me. Like an astronaut, I didnt know if being launched would makeme a spectacle of fireworks or perhaps get me to my destination with no way to return. Or would I have the rare opportunity to complete my mission and get home safely, delivering something that would drive us all forward to a better life?

Thats the mission I set for myself during my treatment. It was a job, one I took passionately and wanted nothing more than to do well so that, as a result, all who followed would have a better chance at life. This was especially true becauseI was the fifth person in the world to be treated with chimeric antigen receptor-T cell therapy, more commonly referred to as CAR-T cell therapy. I was part of the JCAR017 clinical trial.

In the months leading up to the trial, not knowing what my fate would be, my wife and I decided to live for today and take the next steps toward becoming parents. I didnt truly think I would ever see the birth of my child. That was a very sad thought, but this was bigger than me; I was leaving a piece of me behind for my wife.Thankfully, the CAR-T cell therapy was a success and got me into remission. Shortly after that, my wife and I welcomed our daughter, Julia, into the world.

People always wonder, How should I interact with someone I care about who has cancer?

I think the answers easy: Give them a future. Give them something to look forward to. Plan things with them, because we all need a future to survive.

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Into the Future With CAR-T Cell Therapy - Curetoday.com

Walking in His Shoes – Curetoday.com

A man who received chimeric antigen receptor-T cell therapy shares his firsthand account.

Backer received a diagnosis of diffuse large B-cell lymphoma (DLBCL). It is the most common type of non-Hodgkin lymphoma, which affects nearly 75,000 people mostly men in the United States each year.

Despite multiple rounds of chemotherapy and a stem cell transplant, Backers disease kept relapsing. Then he participated in a clinical trial involving chimeric antigen receptor (CAR)-T cell therapy, which has left him cancer-free for almost three years. With this type of immunotherapy, a patients T cells are removed, altered in a lab and then reinfused in the hope that they will attack cancer cells.

Dr. Frederick L. Locke, a medical oncologist at Moffitt Cancer Center in Tampa, Florida, isco-lead investigator of the pivotal ZUMA-1 trial, which led to the Food and Drug Administration (FDA) approval of the second available CAR-T cell therapy, Yescarta (axicabtagene ciloleucel). During the National Comprehensive Cancer Network 2019 Annual Conference, Locke, Backer and Alix Beaupierre, a transplant nurse coordinator, took a 360-degree look at CAR-T cell therapy.

LIMITED OPTIONSOutcomes in refractory aggressive non-Hodgkin lymphoma are poor, Locke explained. Patientsare often treated upfront with combination chemotherapy, as Backer was. He initially wenton a chemotherapy regimen commonly known as R-CHOP Rituxan (rituximab), cyclophospha- mide, Adriamycin (doxorubicin), Oncovin (vincris- tine) and prednisone. He achieved a complete remission and went back to work.

We can cure up to about 60% of patients with initial chemotherapy, and thats pretty remarkable, Locke said. Unfortunately, 40% of patients either dont respond to chemotherapy or progress.

Backer was among that 40%. He relapsed about a year later and started on a new chemotherapy regimen with a planned autologous stem cell transplant, which would involve removing his own stem cells and later putting them back into his body to help fight the cancer. This treatment plan cures only about 5% of patients, Locke said. Prior to CAR-T cell therapy, more chemotherapy would have been next.

In the United States, two CAR-T therapies are available to patients with certain types of cancer. The first, Kymriah (tisagenlecleucel), was approved in August 2017 for patients up to 25 years old who have acute lymphoblastic leukemia that relapsed or did not go into remission with other treatments. Two months later, the FDA approved Yescarta to treat adult patients with certain types of large B-cell lymphoma who have not responded to or relapsed after at least two other kinds of treatment.In patients with DLBCL, durable responses the length of time that a partial or complete response is observed because of treatment have been seen in 40% of patients who received CAR-T cell therapy. We think we can cure about 15% of people, Locke said. We need these patients referred and referred early.

A NUCLEAR BOMBBacker first read about CAR-T cell therapy on clinicaltrials.gov, an online registry of all clinical trials that anyone can access to see what might be enrolling participants. Im the typeof person who needs a plan A and a plan B, Backer said.I received a plan A, but the plan B in the case of the transplant failing was not encouraging.

He reached out to Moffitt Cancer Center Tampa is not far from where he lived in Orlando, Florida to see if he was eligible. He wasnt. In December 2015, Backer went ahead with the stem cell transplant. A few months later,he again relapsed with growths all over his body butthis made his participation in the ZUMA-1 clinical trial possible. Being a participant in a clinical trial is scary and daunting at the same time, he said. I remember sitting there with the transplant coordinator and they handed me a 27-page consent form, and I could barely read page one. Ijust wanted to sign. I was ready to sign anything right then and there.

Although potentially curative, CAR-T cell therapy is not without risk. Patients can develop two serious side effects. Cytokine release syndrome, caused by a large, rapid release of cytokines (small proteins importantin cell signaling) into the blood from immune cells affected by the immunotherapy, can be life-threatening. Neurological events, such as confusion, tremor and seizures, can also occur.

Despite lengthy discussions with his medical teamat Moffitt and reading the consent form, Backer said,he wasnt fully prepared for the coming side effects and recovery when he went forward with CAR-T therapy in June 2016. Thats when the nuclear bomb set off, he said. Within 12 hours of receiving the infusion, he experienced severe chills, violent shaking and a high fever, and also felt certain he was experiencing side effects that were affecting his brain.

About two days after Backer received the reinfusedT cells, the infectious disease team rushed him in for a CT scan. They found no infection and no signs of the cancer.

For me, it was a miracle treatment, Backer said.

Locke has been following patients enrolled in ZUMA-1 for more than two years and said that half are still alive.

BEING PREPAREDBacker admitted that going in with a full understanding of CAR-T cell therapy would have made the experience dramatically different.

After seeing patients and their loved ones in distress, care providers at Moffitt Cancer Center learned to smooth the process, Beaupierre said. For instance, the 27-page document has been broken down into one-page educational handouts and shorter consent forms. The team also created a flow sheet and that grew to a detailed patient calendar. All CAR-T therapy recipients also now have a dedicated nurse and social worker.

In addition to those resources, Backer said, peer-to-peer support would be helpful.

As CAR-T cell therapy continues to evolve and be explored in the treatment of other cancer types, experts are learning more about how it works and how to improve the process.

For Backer, quality of life has changed a bit. Initially, he received blood and platelet transfusions every two weeks for several months following CAR-T cell therapy. Although he is back to work full time, he runs the risk of being exposed to other diseases and viruses. Its always at the back of my mind, he said. I wear a mask and goggles at work but still get sick.

He spends his free time hiking and fishing and feels blessed to still be alive. It worked out for me, and here we are 33 months into this thing, Backer said.

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Walking in His Shoes - Curetoday.com

American CryoStem Expands Investigator Team with Sub-Investigators for Post Concussive Syndrome IND – GuruFocus.com

The Company and the Principal Investigator announce the addition of sub-investigators to the clinical trial team with extensive experience assessing and treating athletes and military personnel suffering from concussion injury, traumatic brain injury and Post Concussion Syndrome

EATONTOWN, NJ / ACCESSWIRE / October 3, 2019 / American CryoStem Corporation (OTC PINK:CRYO) a leading strategic developer, marketer and global licensor of patented adipose tissue-based cellular products and technologies for the Regenerative and Personalized Medicine industries, today announced the selection of three new Investigators to assist the Principal Investigator with CRYO's Phase I clinical study of ATcell("Investigational Drug") as part of a single center study under a protocol entitled: ATcell Expanded Autologous, Adipose-Derived Mesenchymal Stem Cells Deployed via Intravenous Infusion for the Treatment of Post Concussion Syndrome (PCS) in Retired Military and Athletes.

Dr. Tal David, a Sport Medicine specialist and former NFL Head Team Physician for the San Diego Chargers, is going to be the Co-Principal investigator for the study. Sub-Investigators, Dr. Jason Bailie, PhD, Senior Clinical Research Director at the Defense and Brain Injury Center (DVBIC) at the Naval Hospital Camp Pendleton, CA and Dr. Ettenhofer, Director of Research Operations, Defense and Veterans Brain Injury Center (DVBIC) Naval medical Center, San Diego, CA were the key designers of the evaluation aspect of the study. This included advising on the pre-treatment evaluations to rule out complex psychological co-pathology as well as the outcome measures to follow the efficacy of the treatments, since the chronic injuries seen in athletes mirrors those issues found in the military with multiple concussions and blast injuries.

Under the direction of Dr. Hanson and Dr. David, the sub-investigators will be responsible for assessing and screening all applicants for participation and completing the ongoing assessment of each participant. These evaluations include patient physical and neuropsychological assessment, testing and screening, preparation of source documentation and collection of assessment results, and assistance with completion of the final study reports and publications. The surgical tissue collection, ATcell treatments and follow up clinical visits will be conducted at BioSolutions Clinical Research Center facility in Le Masa, CA. The Company is pleased to present a world class team with significant experience in chronic concussion syndromes.

PRINCIPAL INVESTIGATOR: Dr. Peter Hanson, MD, as previously announced on September 11, 2019, has been appointed as the Principal Investigator, for the Company's study. Dr. Hanson is also Medical Director of BioSolutions Clinical Research Center the clinical research facility engaged by the Company to conduct the Study. During Dr. Hanson's career he has participated in approximately 41 clinical studies of which he led 24 as principal investigator. His clinical studies have been sponsored by many of the biggest names in the pharmaceutical and biotech industry such as Pfizer, Sanofi, Bristol Meyers Squibb, Regeneron, Cytori and InGeneron.

CO-PRINCIPAL INVESTIGATOR: Dr. Tal David, M.D. is a board-certified orthopedic surgeon specializing in arthroscopic and sports medicine surgery, is certified in both Orthopedic Surgery and Orthopedic Sports Medicine. He practices in San Diego, CA as part of the Synergy Specialists Medical Group and the San Diego Stem Cell Treatment Center. He is a clinical faculty member of Orthopedic Surgery at UC San Diego and a faculty member of the San Diego Arthroscopy and Sports Medicine Fellowship. He has cared for injured athletes for 15 years and has served on the medical staff of various professional sports teams including more than 11 years as an NFL team physician. In addition to his private practice, he served as the former Head Team Physician for the San Diego Chargers NFL football team and is Medical Director for the San Diego Gulls, AHL hockey team.

SUB-INVESTIGATOR: Jason M. Bailie, Ph.D, is a neuropsychologist who serves as Senior Clinical Research Director of the Defense and Veterans Brain Injury Center (DVBIC), Naval Hospital in Camp Pendleton, CA. Dr. Baille currently directs research, clinical and educational activities and supervises research staff. He is responsible for clinical research program development, experimental design, human-subject recruitment, experimental procedural implementation, data acquisition, and statistical analysis for local and multi-site research studies. Additional duties include oversight of research compliance with Institutional Review Board for the Department of Navy, Department of Defense clinical research and human subject research policies; and facilitating grant acquisitions and management. He supervises a clinical staff of social workers and psychologists, educational outreach personnel and provides educational presentations and grand rounds to providers and service members in collaboration with DVBIC Regional Education Coordinator.

SUB-INVESTIGATOR Mark L. Ettenhofer, Ph.D is a neuropsychologist and Director of Research Operations American Hospital Services Group (AHSG), Defense and Veterans Brain Injury Center (DVBIC), Naval Medical Center, San Diego (NMCSD): San Diego, CA.Dr. Ettenhofer's primary research focuses on the development and evaluation of novel approaches for neurocognitive assessment and rehabilitation of traumatic brain injury (TBI) for the US Navy. Dr. Ettenhofer currently directs DVBIC supported traumatic brain injury (TBI) research at NMCSD, including 8 approved protocols evaluating novel technologies for TBI assessment, methods for remediation of TBI-related impairment, natural history of TBI, and guidelines for progressive return to activity after injury. Additionally he provides programmatic review for DVBIC national research portfolio, supervises and mentors eight full-time staff members in research activities and is well published.

Overall, it is estimated that the cost of traumatic brain injuries (TBI) in the United States weighs in at $48.3 billion annually of which $31.7 billion is spent on hospitalization costs; an additional $16.6 billion is associated with costs attributed to fatalities. According to the Center for Disease Control, acute care and rehabilitation of brain injury patients in the United States costs about $9 billion to $10 billion per year. This does not include indirect costs to society and family caretakers due to lost productivity, work time and earnings, as well as costs linked to providing social services.

Brain Injury Cost

The Company stated that "The successful assembly of this expert team of investigators who are currently treating athletes and military personnel suffering from Concussive and Traumatic Brain Injury is a major step towards properly implementing, monitoring and effectively evaluating the treatment of the participants with ATcell. Working with this expert team will expand the Company's knowledge for treating concussion injury, traumatic brain injury and Post Concussion Syndrome and accelerate its ability to complete this Phase 1 study and provide the necessary input to continue our work toward final FDA approval of ATcell for Post Concussion Syndrome.

For further detailed Corporate or Regenerative Medicine information please visit:

http://www.americancryostem.com, request by email at [emailprotected] or gathering phone 732-747-1007

This press release may contain forward-looking statements, including information about management's view of American CryoStem Corporation's ("the Company") future expectations, plans and prospects. In particular, when used in the preceding discussion, the words "believes," "expects," "intends," "plans," "anticipates," or "may," and similar conditional expressions are intended to identify forward-looking statements. Any statements made in this press release other than those of historical fact, about an action, event or development, are forward-looking statements. These statements involve known and unknown risks, uncertainties and other factors, which may cause the results of the Company, its subsidiaries and concepts to be materially different than those expressed or implied in such statements. Unknown or unpredictable factors also could have material adverse effects on the Company's future results. The forward-looking statements included in this press release are made only as of the date hereof. The Company cannot guarantee future results, levels of activity, performance or achievements. Accordingly, you should not place undue reliance on these forward-looking statements. Finally, the Company undertakes no obligation to update these statements after the date of this release, except as required by law, and also takes no obligation to update or correct information prepared by third parties that are not paid for by American CryoStem Corporation.

SOURCE: American CryoStem Corporation

View source version on accesswire.com: https://www.accesswire.com/561800/American-CryoStem-Expands-Investigator-Team-with-Sub-Investigators-for-Post-Concussive-Syndrome-IND

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American CryoStem Expands Investigator Team with Sub-Investigators for Post Concussive Syndrome IND - GuruFocus.com

Oncternal Therapeutics Announces Opening of Phase 1b Expansion Cohort of Clinical Trial of Cirmtuzumab in Combination with Ibrutinib in Patients with…

SAN DIEGO--(BUSINESS WIRE)--Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that it has opened for enrollment a Phase 1b expansion cohort of its Phase 1/2 clinical trial of cirmtuzumab, a ROR1-targeted monoclonal antibody, combined with ibrutinib, in patients with mantle cell lymphoma (MCL). The decision to open an expansion cohort in MCL of the ongoing Phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial was based on favorable interim results from the dose-finding cohort of the trial, including that the combination was well-tolerated and that complete responses were observed in two heavily pre-treated patients who had received and failed multiple chemotherapy regimens and an autologous transplant, as well as either an allotransplant or CAR-T therapy, prior to participating in this clinical trial.

In June, the Company presented interim data at the American Society of Clinical Oncology (ASCO) annual meeting, including the preliminary results from the first six patients with MCL treated in the CIRLL clinical trial. One patient with MCL, who had relapsed following an allogeneic stem cell transplant, experienced a confirmed complete response (CR) after three months of cirmtuzumab plus ibrutinib treatment, including complete resolution of a large mediastinal mass. This CR appears to be sustained and has been confirmed to be ongoing after completing 12 months of cirmtuzumab plus ibrutinib treatment. Following ASCO, a second confirmed CR occurred in a patient who had progressive disease after failing several different chemotherapy regimens, autologous transplant and CAR-T therapy. Additional data from this clinical trial will be presented at a future medical conference.

It is encouraging to see that the drug has been well tolerated as well as the early signal of efficacy of cirmtuzumab with ibrutinib in MCL, particularly the rapid and durable complete responses of the heavily pre-treated patients after three months of therapy, which is an unusually fast response in this patient population, said Hun Lee, M.D., Assistant Professor of Medicine in the Department of Lymphoma & Myeloma at the University of Texas MD Anderson Cancer Center, who is an investigator on the CIRLL clinical trial.

The CIRLL clinical trial is supported by a grant from the California Institute for Regenerative Medicine (CIRM) and is being conducted in collaboration with the University of California San Diego (UC San Diego).

We are pleased to be opening the expansion cohort portion of the CIRLL clinical trial for patients with MCL, and continue to be encouraged by the interim results from this study for both patients with MCL and patients with chronic lymphocytic leukemia, for whom a randomized Phase 2 portion of the trial was opened in August, said James Breitmeyer, M.D., Ph.D., Oncternals President and CEO.

About the CIRLL Clinical Trial

The CIRLL clinical trial (CIRM-0001) is a Phase 1/2 trial evaluating cirmtuzumab in combination with ibrutinib in separate groups of patients with chronic lymphocytic leukemia (CLL) or mantle cell lymphoma (MCL). Part 1 of the clinical trial was a Phase 1 dose-finding portion designed to determine the Phase 2 dose, or recommended dosing regimen (RDR). Part 2 is a Phase 1b expansion cohort to confirm the RDR. Additional information about the CIRM-0001 clinical trial and other clinical trials of cirmtuzumab may be accessed at ClinicalTrials.gov.

About Cirmtuzumab

Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of CLL and MCL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine (CIRM). In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with metastatic breast cancer is being conducted at the UC San Diego School of Medicine. CIRM has also provided funding to support development programs for cirmtuzumab and a CAR-T therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors.

ROR1 is a potentially attractive target for cancer therapy because it is an oncofetal antigen a protein that confers a survival and fitness advantage when reactivated and expressed by tumor cells. When expressed by hematologic malignancies such as CLL and MCL, ROR1 acts as a receptor for the tumor growth factor Wnt5a. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to inhibiting Wnt5a activation, specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Preclinical data showed that when cirmtuzumab bound to ROR1, it blocked Wnt5a signaling, inhibited tumor cell proliferation, migration and survival, and induced differentiation of the tumor cells. Cirmtuzumab is in clinical development and has not been approved by the U.S. Food and Drug Administration for any indication.

About Oncternal Therapeutics

Oncternal Therapeutics is a clinical-stage biopharmaceutical company focused on developing product candidates for the treatment of cancers with critical unmet medical need. Oncternal focuses drug development on promising yet untapped biological pathways implicated in cancer generation or progression. The pipeline includes cirmtuzumab, an investigational monoclonal antibody designed to inhibit the ROR1 receptor, a type I tyrosine kinase-like orphan receptor, that is being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), and TK216, an investigational small-molecule compound that is designed to inhibit E26 transformation specific (ETS) family oncoproteins, that is being evaluated in a Phase 1 clinical trial for patients with Ewing sarcoma alone and in combination with vincristine chemotherapy. In addition, Oncternal has a program to develop a CAR-T therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors. More information is available at http://www.oncternal.com.

Forward-Looking Information

Oncternal cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as may, will, should, expect, plan, anticipate, could, intend, target, project, contemplates, believes, estimates, predicts, potential or continue or the negatives of these terms or other similar expressions. These statements are based on the Companys current beliefs and expectations. Forward looking statements include statements regarding: Oncternals plans to present additional data from its ongoing Phase 1/2 clinical trial of cirmtuzumab; the expectation that Oncternal will be able to enroll patients into the Phase 1b expansion cohort; and Oncternals belief that favorable outcomes from the ongoing Phase 1 portion of the clinical trial support opening the Phase 1b portion. The inclusion of forward-looking statements should not be regarded as a representation by Oncternal that any of its plans will be achieved. Actual results may differ from those set forth in this release due to the risks and uncertainties inherent in Oncternals business, including, without limitation: uncertainties associated with the clinical development and process for obtaining regulatory approval of cirmtuzumab and Oncternals other product candidates, including potential delays in the commencement, enrollment and completion of clinical trials; the Companys dependence on the success of cirmtuzumab and its other product development programs; the risk that interim results of a clinical trial do not necessarily predict final results and that one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data become available; the risk that unforeseen adverse reactions or side effects may occur in the course of developing and testing product candidates such as cirmtuzumab and Oncternals other product candidates; the Companys limited operating history and that fact that it has incurred significant losses, and expects to continue to incur significant losses for the foreseeable future; risks related to the inability of Oncternal to obtain sufficient additional capital to continue to advance the development of cirmtuzumab and its other product candidates; and other risks described in the Companys prior press releases as well as in public periodic filings with the U.S. Securities & Exchange Commission. All forward-looking statements in this press release are current only as of the date hereof and, except as required by applicable law, Oncternal undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

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Oncternal Therapeutics Announces Opening of Phase 1b Expansion Cohort of Clinical Trial of Cirmtuzumab in Combination with Ibrutinib in Patients with...

March to fight multiple myeloma scheduled this weekend – BlackburnNews.com

http://www.lasalle.ca By Adelle LoiselleSeptember 28, 2019 6:00am

Among the people taking part in this weekends Myeloma March in LaSalle, is Jennifer Radjenovich. She is doing it for her father.

Guido Vetorello was given just three years to live after he was diagnosed with multiple myeloma, but four years later he continues to do what he loves; play with his grandchildren, watch baseball, and travel with his wife.

Trouble started in 2015 when he was treated for anemia when he could not shake unusual fatigue. Unfortunately, the treatments did not work, and his family feared something else was wrong with him.

They were right.

Blood tests and a bone marrow biopsy came back positive for the life-threatening blood cancer affecting plasma cells.

Multiple myeloma or just myeloma is the second most common form of blood cancer. A release from the organizers of this years march said eight Canadians are diagnosed with it every day, and as of yet, doctors do not know what causes it, and there is no cure.

Like Guido, those with the disease can experience fatigue, but also excessive thirst, bone pain, numbness in the legs, nausea, and weight loss.

Unfortunately, Guido did not qualify for a stem cell transplant. Doctors decided it was too risky, but a new drug treatment looked promising, and they were right.

These days, Guido is relatively stable and has exceeded his doctors expectations. Although he undergoes chemotherapy treatments weekly, he is still around to watch his grandchildren grow.

My dad looks forward to going to his treatments every week because he knows they are giving him more time, said Jennifer Vettorello. In the four years since he was diagnosed, the research has been incredible with so many new treatment options available to patients. It gives me real hope that will be with us for some time.

The Vettorello family will be at the Vollmer Culture and Recreation Complex in LaSalle Sunday morning when the ninth annual march gets underway at 9 a.m.

Fundraising, which supports research, has helped almost double the lives of patients in the past 15 years.

Dr Sindu Kanjeekal, the Multiple Myeloma Clinical and Research Lead at the Windsor Regional Cancer Centre, said life expectancies are continuing on an upward trend.

Were now seeing incredibly promising treatment options that are helping us to stay ahead of the disease, he said. For the first time, we can actually say that were getting closer to finding a cure. Investing in research is critical, which is why raising funds is more important than ever.

Windsor-Essex is one of 28 communities across the country taking part in this years march.

The goal this year is $40,000, but across the country, marches are trying to raise $550,000.

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March to fight multiple myeloma scheduled this weekend - BlackburnNews.com

Taz Believes CM Punk Will Be At SmackDown On Fox Thinks Edge Could Pop Up On AEW Or SmackDown – Fightful

Taz has theories.

CM Punk has been in the headlines for the past week, beginning with a report on Sept. 27 that he had tested on-camera for WWE's upcoming studio show on Fox Sports 1; WWE Backstage. Punk was back in the headlines on Sept. 29 when news broke that he and Colt Cabana had settled their lawsuit.

With so much smoke surrounding CM Punk, former WWE announcer Taz believes a fire is about start.

"I think CM Punk is coming back to WWE," stated Taz on The Taz Show. "I know there's been some scuttlebutt about that and I believe when there's smoke there's fire. I believe he's gonna debut on Fox, without a doubt, on Friday. I believe that. Straight up. They're gonna go full-throttle with Fox."

For what it's worth, Booker T, who is set to co-host WWE Backstage alongside Renee Young, said he knows nothing about Punk testing on-camera for the show.

That wasn't the only theory Taz dropped on his show. On the latest edition of the Edge & Christian Pod of Awesomeness, Edge revealed that he would be stepping away from the podcast for the time being. Taz believes there's more to the story.

"If Edge can still go out there and do it at his age, he looks to be in great shape. If he can still get after it in the ring, I think that's great. This is something I gotta say. I know Edge has been looking into stem cell treatment because it would help his neck," said Taz. "I've heard stuff, that he wasn't medically cleared and apparently WWE's doctors aren't clearing him. But yet, they allowed him to do a spear. I'm a little confused on that. I got a feeling that Edge stopping his podcast, my conspiracy theory is, I think he's cleared by WWE. Or he ends up in another company. We've got a couple of big shows debuting this week. I've got a feeling The Rated R Superstar is gonna pop up somewhere. He pops up in WWE, I do think it would be SmackDown. Or AEW. You never know, he could pop up there."

Edge surprised many in the wrestling world when he not only made an appearance at WWE SummerSlam, but delivered a Spear to Elias. Following the appearance, Edge stated on his podcast that he's not as limited as people may think despite his neck injury.

While Edge and CM Punk are not confirmed for SmackDown on Fox, The Rock announced that he will return home on Friday when he appears on SmackDown.

If you use any of the quotes above please credit Taz Show with a h/t to Fightful for the transcription.

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Taz Believes CM Punk Will Be At SmackDown On Fox Thinks Edge Could Pop Up On AEW Or SmackDown - Fightful

Middlesbrough blood donor meets man she saved in Germany – The Northern Echo

A TEESSIDE woman, who three years ago donated blood stem cells to a stranger in Germany, met the man she saved for the very first time as he made the thousand-mile round trip to thank her in person.

Husband, father and grandfather Siegfried Siggi Wahl, 71, from Hattersheim in Germany, was diagnosed with chronic myeloid leukaemia in 2016.

After initial treatment proved unsuccessful, doctors told him his best chance of survival was a blood stem cell donation from a person with identical tissue type.

Luckily for him, just six months earlier his genetic twin, 50-year-old Jackie Wray, who runs a wedding venue in Great Ayton, signed up with donor charity DKMS in 2015 after seeing an appeal.

Shortly after being identified as Siggis perfect match, Jackie began the process of attending medicals and then donating her blood stem cells.

However, back in Germany, Siggis problems were not over. To prepare Siggi for the transfusion, his doctors began to administer a strong dose of chemotherapy to destroy his existing blood stem cells, ready to welcome the new ones, and if he did not receive Jackies healthy blood stem cells quickly, he was unlikely to survive.

As a courier arrived to escort Jackies blood stem cells from London to Frankfurt, news broke of the 2016 terrorist attack in France, and the courier was not able to leave the UK.

After an agonising wait, Jackies blood stem cells finally arrived in Frankfurt, and the transplant successfully went ahead.

UK law states whilst donors and their patients can communicate following the transplant, it must remain anonymous for the first two years. After this, if both parties are in agreement, their identities can be revealed and they can make arrangements to meet.

I loved receiving cards from Siggi, said Jackie. It used to make me cry every time. I would think thank god hes still alive.'

All I knew of Siggi is that he had a son and daughter my age, and a grandson and he used to play ice hockey when he was a young man. Its one of the reasons I was so excited to meet him, to hear his life story.

When he and his wife arrived at my house, there were lots of tears and lots of cuddling. They had made me a photo album with pictures of Siggi during his treatment and they bought me a little angel necklace because they say Im his angel. It was just so lovely and I will treasure it forever.

Siggi added: "It was beautiful. We hugged, and we both cried. It was like a fairy tale.

Jackie will come and visit me and my family in Germany next year to celebrate my birthday. She is just a good hearted person.

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Kevin Mwachiro: Keeper of Stories – Roads and Kingdoms

This week on The Trip podcast: talking African stories, LGBTQ rights in Kenya, and surviving cancer with Kevin Mwachiro.

One of the things, one of the unnerving things, that you first realize as a foreigner visiting Nairobi, is that many of your cohortthose other foreigners landing at Jomo Kenyatta airportseem to be looking right past the people, past the humans of Kenya. They are searching for animals. Nairobi is the worlds busiest transit hub for safari-goers. Theres even a national park inside the city limits, a park with wild warthogs and zebras and giraffes and the rest of the cast from The Lion King.

I am resolutely and proudly not here for a safari. It just feels, well, a little premature, a little colonial, like Id need to be wearing a pith helmet and khaki knee breeches. Maybe on my fifth visit I would go for a walkabout. But Nairobis wildlife will not be ignored. In Karen, the district Im staying innamed after Karen Blixen, who wrote Out of Africa nearbythe birds bring the safari to you. Kestrels and crakes and bee-eaters and bustards, sooty falcons and jacksons widowbirds all circle and sing and their song to me, Im sure, roughly translates as fuck you if you think youre too good for us.

But I stand firm. Itspeople Im most interested in, and if Ive found anything in two decades as a foreign correspondent and daydrinker, its that humans are endlessly fascinating, and occasionally even delightful. Case in point, this episodes guest, Kevin Mwachiroan openly gay athlete in a country where gay relationships are illegal, a survivor of a rare cancer who is also the sunniest person Ive met in ages. Kevin was a guest on one of the last episodes of Parts Unknown, when Bourdain and W. Kamau Bell came to Kenya, and it is my distinct human pleasure to have him, like Tony and Kamau before him, on this show.

Here is an edited and condensed transcript from my conversation with Joshua. Subscribers canlisten to the full episode here. If youre not on Luminary yet, subscribe and listen (and get a 1-month free trial) by signing uphere.

Nathan Thornburgh: All right, so this coffee is great. This is exciting. And I remember Colombians, God love them, make incredible coffee, Juan Valdez on down, but the national passion was instant coffee, and it was always very confusing. But its one of those things where you have this really valuable commodity, easy to export, and somehow I think its true in a lot of places that make the best coffees in the world where the average person is like, Well, Im not going to compete in the global market for these high quality beans. Im going to just be happy with the instant.

But what is Kenyan coffee drinking culture like? Is there some proportion of the population that recognizes that Kenya makes some amazing coffee and rolls with that?

Kevin Mwachiro: The thing with Java House, they make Kenyan coffee accessible to Kenyans, so to speak. Id say maybe middle-class Kenyans. Before that, we were getting high on instant coffee. You were paying a pretty penny for coffee. Then Java opened up and they had baristas there making coffee, good food as well, and youre like, Yeah, its not too bad.

Thornburgh: Yeah.

Mwachiro: But we are a tea-drinking nation.

Thornburgh: So coffee is still the number two?

Mwachiro: Coffee is number two. You go anywhere, people will offer you tea first. And not just teabag tea, but brewed tea with milk and that is it. Coffee, a smaller proportion of people drink coffee, and especially black coffee.

Thornburgh: So, I have been internet stalking you in your various talks and interviews. You have a very entertaining and interesting media profile, I guess you would say.

Mwachiro: Yeah. Entertaining is an interesting word to use.

Thornburgh: A lot of it is the kind of stuff that would give me pause: Getting up on stage, single person, telling a story, trying to hold an audience, and you do it really well, and Im interested in how you got into that. How did you decide, or maybe it just sort of fell into place, that this is what you were going to do for a living?

Mwachiro: I recently took on that label of a storyteller. I think this comes from my time at the BBC, and Ive told people this, I consider myself a custodian of peoples stories, even as a journalist. People gave me their stories and I told them to the wider world. I love hearing peoples stories, so I think through everything as a journalist, as an activist, as a podcaster, as a cancer survivor, and telling my own story, I think life handed me what Id been avoiding for a very long time, and now find myself doing it and people actually say I tell stories pretty well.

Thornburgh: Why had you been avoiding it?

Mwachiro: I love and I dont love being on stage. I dont like the attention, but I know once Im on stage I become a whole new, confident person, and I feel comfortable. And people, every time Im up on stage, were like, You are comfortable there. I used to act once up on a time and yeah, the stage is also home and Im now doing this. I think over time, its a question of valuing yourself, its been a journey to actually get here, and valuing my own story and realizing that I have a story to tell. And was it two, three years ago I gave a talk on, its like a Ted version of Kenya called Engage, and I spoke about finding my voice and that, I think, was a turning point for me. I used to moderate quite a bit before that, professionally, but now being on stage and telling people my own story and actually saying, This is me finding my voice, and sharing it and not feeling ashamed about whatever I have gone through has landed me now here with you.

Thornburgh: Yeah. Lets not make than an end point. This is a humble detour.

Mwachiro: And I realized I like sitting in front of a mic. When I used to work at the BBC, one of the requirements of the work as a journalist was to perform in front of a microphone and on camera. And I realized this is cool stuff, man. I do like cams, I do like microphones, I love audio, I love sound.

I believe in the spoken word.

Thornburgh: Well, lets talk about that. Obviously this is a medium that weve jumped into with great enthusiasm in podcasting. What is the state of podcasting in Kenya? Is it a word that people recognize or is your audience both kind of local and international? How do you look at podcasting in particular?

Mwachiro: Its very interesting you should say that. As I was buying coffee, I met another podcaster, a guy called Armani, and he

Thornburgh: Thats a very Brooklyn scene right there.

Mwachiro: Yeah.

Thornburgh: Here we are at the coffee shop, just a couple podcasters. All right.

Mwachiro: And he recently got into podcasting as well. He wants to bring other podcasters together. I would say its a very Nairobi-centric thing, and very middle class.

I wanted to get back into radio at one time. Id missed audio. I wanted to come up with content, spoken word, and when I talked to people about it, they were like, You should go into podcasting. Im like, No, I want to go back into mainstream radio. And then after some time I figured this might be my avenue, going into mainstream radio. I believe in the spoken word Whats the term we used to use? Anyway, spoken word radio. Im a big fan of that, NPR, BBC.

And thats my background. And I just like storytelling. I figured this is a way of getting Kenyans to listen, but I want to go mainstream. But after some time I realized, speaking to other podcasters, that this just might be an avenue to explore.

Thornburgh: Right.

Mwachiro: I saw it as very niche. As you know, everyones podcasting in the States, in Europe, and its not quite herethe audiences still listen to radio.

Thornburgh: Yeah.

Mwachiro: And commercial radio. And I love public service radio. I really do. And I wanted to go back into that, but that didnt quite happen. Then I started listening to podcasts, started meeting other Kenyan forerunners, forerunner podcasters, and liked what I was doing. I realized this is something that I could do. I started buying kits, good kits, listening to stuff on YouTube, tutorials, I rented quite a number of those, and came up with content. Hence, Nipe Story. Initially I wanted to have full podcasts, then after just trying I realized this is a lot of work, so I scaled down to Nipe Story, which is my podcast. But in general its a very urban thing here in Nairobi. People are beginning to recognize what it has and coming up with a lot of niche content.

Thornburgh: Yeah. Thats the thing about the promise of podcasting. You can find your audience and it doesnt have to be that big, but they can be with you deeply because they can find something thats just specific for their taste. I also, from my short time here in Nairobi, I see tremendous opportunity in podcasting because its all about cars and traffic

Mwachiro: Absolutely.

Thornburgh: and the commute.

Mwachiro: Yeah.

Thornburgh: So, you have so many human hours in the car.

Mwachiro: You have already an audience sitting in Nairobi traffic waiting to listen to stuff.

Thornburgh: Waiting for Nipe Story. So tell me about that show, what is it trying to do and how are you getting that done?

Mwachiro: Nipe Story is just trying to get people into listening to stories, Kenyan stories, African short story fiction. I love stories. I love reading fiction and yet again, I love listening to spoken word and this was my way of just getting involved in that. It started in, I think, December or November 2017.

Mwachiro: So I said, This is amazing, and its a simple goal, mate. Just to make people love listening to stories. And theres a lot of African creative writing going out there and Im hoping to provide a platform for that, and also for a lot of queer writing that doesnt get a platform, especially in this form. So I just want to do that with Nipe Story. I would love Nipe Story to have a Pan-African feel.

Mwachiro: First of all.

Thornburgh: So they could go across the continent and not just be for Kenyans.

Mwachiro: Absolutely. And sometimes when I look at the stats you get people listening in South Africa. But the thing that surprised me, theres a huge North America audience and a British audience as well, that surprises me.

Thornburgh: Yeah. I dont know. I could see the appeal, especially because one, theres a huge diaspora and two, it is a different Ive gotten to listen to some of the episodes and its beautiful in the way that the concerns are just different, the dialect, the accents are different, its transporting in some way. If these were presented from a local perspective in the States, you could have the same mission, the same mandate, but its just very different in the way that it plays and sounds and listens, and you can lose yourself in it.

Mwachiro: Thank you.

Thornburgh: And its interesting, I remember you did an interview in Berlin where you were talking about the flip side of thatparticularly talking about the context of, I think, queer filmwhere you were saying there are some really great films that you saw at the Teddys, where you were a judge, but that they didnt necessarily speak to you. It was just a different experience, because these are European or American filmmakers. That was the sense that I got from that, and there is a way where your experience just does have a local identity to it, right? I mean, its a very Kenyan thing, even though you have common cause with people who are trying to do fiction podcasts, with queer activists, with people in different countries, but your experience is going to be your own, and fairly specific here. So, Im interested in getting a sense from you, taking your temperature on where Kenya is at right now in that particular realm, in queer activism. It seems like a tough game right now.

Mwachiro: People say it is. I dont consider it a really tough game, because weve been here a long time, so were used to this. I had drinks with a friend yesterday whos visiting also from the States and he said, It must be tough being gay, and its a question these days I dont know how to answer. Because Im just doing my thing.

Its waking up Kenya to the reality that queer people are here and theres nothing you can do about it and were as Kenyan as you are.

Thornburgh: Youve always been here.

Mwachiro: Yeah, Ive always been here. Some of my girlfriends are like, Who comes up with single guys? I look at my phone book and Im like, I dont know single guys and if theres any single guys here, theyre dicks and I dont want to introduce you to them. My phone book is full of queer men and women. And Ive normalized that existence here.

Thornburgh: Yeah.

Mwachiro: But the fact that we lost the case in court that was trying to decriminalize gay sex. That was hurtful, that was painful.

Thornburgh: Yeah. I spoke with Wanuri about this too and I think especially for, its a three-year legal battle, but even longer than that, just an entire life of wishing this to be true.

Mwachiro: But the fact that we were actually in courtyou have to look at the positives. I remember some of the comments that I got on my page: Were not coming to Kenya. Were going to boycott. Im like, Why? Weve been in this space for quite a while and to find that 10, 12 years ago we never even thought that we would be in court.

Thornburgh: Yeah.

Mwachiro: But the fact that on the 24th of May, people showed up in court from all over, didnt care about the media glare, didnt care what people thought, but we were in court, mate.

Thornburgh: Yeah.

Mwachiro: That was powerful. And even before that, the first time they postponed it, we had activists come in from Uganda, Tanzania, and Nigeria. Such was the magnitude of what we were doing. And I dont think it will stop us. The fact that now people know that queer people do exist in Kenya. Theyve always known, but the fact that we are coming out strong, were waving the flag next to the Kenyan flag, and we are your brothers, your sisters, your sons, your daughters, your fathers, your husbands, your wives. Its sort of waking up Kenya to the reality that queer people are here and theres nothing you can do about it and were as Kenyan as you are.

So I think the space now and being involved with the Gay and Lesbian Coalition of Kenya and talking to people, its now wanting to move now to dialogues and getting Kenyans to come out in support of us. I feel weve been preaching to the choir for quite a while and its now engaging other members of society and saying, Yeah, you know, this is who we are. What do you want to know about us? How can you help? So that when we do go back to court, wed have other people speaking for us as well and not just ourselves.

Thornburgh: Yeah.

Mwachiro: That would be great. I mean if you look at the case, Botswana luckily won theirs and people were making direct comparisons and you cant look at it that way. This is the jealous coming up, its a very simplistic way of looking at life. That story is not that simple.

But the fact that I was in Botswana last year and the deputy mayor, whos a man living with albinism, came and opened the largest Pan-African LGBT conference that had happened on the continent. The fact that he was there was a huge thing. And I remember sitting there, like would we even get someone from the city coming to attend one of these?

Thornburgh: Yeah, you dont think Mike Sonkos showing up?

Mwachiro: No, I dont think so. I dont think so.

Thornburgh: Your entertaining rapper turned [governor] of Nairobi.

Mwachiro: No, lets not go there. He vexes my spirit, man.

Thornburgh: Mike Money.

Mwachiro: Thats how low we can go as a country.

Thornburgh: So yeah, that idea of building some sort of bridges to other parts of society, so youre not out here fighting alone.

Mwachiro: Absolutely. And then the fact that the President of Botswana gave out very positive statements about homophobia, saying that we cant be in this space and other people have spoken. We have a president who still describes us as non-issues.

Thornburgh: Right.

Mwachiro: So we need to get them to move from non-issues, but I keep on saying almost everything in Kenya is a non-issue. Thats why we are the way we are. So weve just been lumped with everything else. Fight corruption, not an issue. You know?

What do I have to lose now? My shit is out there. I think thats the way you take power from people.

Thornburgh: So many amazing, pressing issues that are actually non-issues.

Mwachiro: Exactly, so when that happened, and then I talked, and people were like, Oh, arent you annoyed? I said, No, Im not annoyed. The truth is, a lot of things that should be dealt with in the country that weve made non-issues, and we have been put into that category of non-issues with everything else.

Thornburgh: So you have joined the mainstream then by becoming a non-issue.

Mwachiro: We have, exactly that. So thats where the space is at. Im just hoping that the ruling has just made us as a movement stronger.

Thornburgh: Yeah.

Mwachiro: Will make us be very introspective in how our strategy going forward will be. And for me personally, I know its made me bolder, totally unapologetic about what I feel and the gay shit Im putting out there on my face.

Thornburgh: The Supreme Court has unleashed the wave of gay shit from Kevin.

Mwachiro: I wrote an article thats going up on this platform called Yellephant. Theyve been very kind in giving us an opportunity to put queer stories out there, two of my articles there, and Im like, Yes, we need it. And as a journalist Im taking I used to be slightly apologetic about it and Im like, Im just going to put stuff, Im just going to put the good journalist I think I am into this area where my energies are and where my life is involved in.

Thornburgh: Yeah.

Mwachiro: And come up with good journalism talking about queer Kenyans.

Thornburgh: Yeah.

Mwachiro: And what it is to be a queer Kenyan in Kenya.

Thornburgh: When you, I think it was a film festival or some sort of forum here that you were involved in that had the tag line Shame is a luxury we cant afford.

Mwachiro: Absolutely.

Thornburgh: But thats that kind of thing. Because certain things are stacked against you, you actually have to be bolder and brighter and kind of more out there. That seems to be your perspective on it.

Mwachiro: I was telling someone, what do I have to lose now? My shit is out there. I think thats the way you take power from people. Ive hung up my dirty laundry. I dont think its there, I just use that expression. I hang it there. So you can see Ive taken the power away from you.

Thornburgh: Right. Right. This sort of constant, ongoing This is the experience I think that gay people have had for a long time in the States. Its like this daily blackmail.

Mwachiro: Exactly. Exactly. Ive gone through a process and a journey, Nathan, where I didnt like myself pretty much, I didnt think I was worth something to a point where I know what Im worth and Im happy about who I am in this space. Life has dealt me numbers, thrown lemons at me and Ive made lemonade and Im dealing with that and moving on. I was telling a friend yesterday, You only have one life.

Thornburgh: Right. How are you going to spend it, huh?

Mwachiro: How are you going to spend it? And if this life is going to be used trying to make it easier for other queer individuals in Kenya, so be it, mate. And not just that, but also just trying to make a world, the world a better place for other folk, man.

Thornburgh: Yeah. I mean this is the thing also that I feel people understand very little of. I mean, clearly by some of the conversations were having in the United States, rights for LGBT, justice or rights for minority groups, its not even about them. Its about you and what kind of country youre going to be. And Im sure Botswana is looking at it holistically as well as all these goals they have as a nation, which include development, increased tourism, equal standing on the world stage, all of these things are hurt when they diminish the rights of some large percentage of their population. We have that same conversation in the States. Its not about being nice to gay people. Its about your quality of country.

Mwachiro: Absolutely. I like that. And just being nice to all people. Being nice to women. I think we as a country could do a much better job in being nicer to our women. Slight digression here, we still havent fulfilled that constitutional quota that requires 25% representation of women.

Thornburgh: Wow. Which isnt a big number.

Mwachiro: Its not a number, but

Thornburgh: Its not totally proportionate, but yeah, even that is beyond reach.

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Kevin Mwachiro: Keeper of Stories - Roads and Kingdoms

Genome editing to be tested in kidney organoids – UW Medicine Newsroom

Gene editing will be tested in UW Medicine labs on kidney organoids tiny, kidney-like structures grown from stem cells as part of a federally funded effort to develop safe, effective genome editing technologies and therapies.

The National Institutes of Health today, Oct. 1, announced the next set of grant awards for the Somatic Cell Genome Editing consortium, created in 2018. Somatic cells make up the bodys tissues and organs, such as the lungs or blood, in contrast to reproductive cells, like fertilized eggs. Alterations made to somatic cell DNA are not passed down to the next generation.

In the latest round of SCGE funding, twenty-four grants, totaling about $89 million over four years, been awarded across the country. They will fund studies to address the promises and challenges of genome editing in the search for new treatment or cures for a number of genetic disorders.

The human genome contains thousands of genes responsible for making proteins. In many inherited disorders, a variation in the DNA code means that an important protein is not made, or is not made correctly. The missing or faulty protein could result in serious health problems. Genetic editing would aim to change the DNA to enable cells to make a sufficient amount of the proper protein.

For one of the new SCGE projects, collaborative research will take place between the University of Washington School of Medicine lab of kidney disease researcher Benjamin Beno Freedman, assistant professor of medicine, Division of Nephrology, and the University of California Berkeley lab of Jennifer Doudna, professor of molecular and cellular biology.

As a group, Freedman and his fellow researchers bring together expertise in kidney organoids, kidney cell biology, and kidney diseases. Their collaborators at UC Berkeley are leaders in the field of genome editing, including CRISPR-Cas9 gene editing technology to cut and paste portions of DNA in living cells.

Freedmans lab at the UW Medicine Institute for Stem Cell and Regenerative Medicine grow stem cell-derived organoids to study how kidney diseases begin and how they might be treated. Human kidney organoids and kidney-on-a-chip technologies (in which some functions of kidneys are simulated with living cells in tiny chambers) are providing useful medical information. For example, researchers have found new molecules that can reduce the signs of disease in these laboratory models.

Human kidney organoid showing podocytes (red) and proximal tubules (green) developed in the Freedman lab

Freedman explains the importance of exploring responsible gene-editing therapies for inherited kidney diseases: Genetic kidney diseases impact more than half a million people in the United States alone. If we can learn to safely repair the mutation that causes the disease, we can offer a way to treat patients that is much more effective than any current intervention.

Freedman emphasizes that dialysis and transplants two of the most common treatments for kidney diseases are expensive and hard on patients. Kidney transplants are in short supply; donor organs become available to less than 20 % of the patients who need them each year.

The shortcomings of dialysis and transplants make gene therapy an appealing area of research because it might get to the root of the problem.

One of the primary aims of the NIH-funded somatic cell genome editing explorations are to reduce the chances that gene editing produces unintended side effects that do more harm than good. In their collaborative project with UCBerkeley, the UW Medicine team will screen different gene therapies for their effects on normal kidney function and for risks of renal cancer or autoimmune disease.

Our hypothesis is that gene editing will cause adverse effects, but that these effects are predictable and controllable, says Freedman. Our goal is to prove this using laboratory models like organoids and kidneys on chips so we know the approach is safe before we ever involve a human patient.

Freedmans lab is in the Division of Nephrology, Department of Medicine, at the UW School of Medicine, and his lab is also part of the Kidney Research Institute, a collaboration between Northwest Kidney Centers and UW Medicine.

Joining Freedman on the UW Medicine research team are Institute for Stem Cell and Regenerative Medicine colleagues Hannele Ruohola-Baker, professor in biochemistry, and Julie Mathieu, assistant professor of comparative medicine, both at the UW School of Medicine.

Ruohola-Baker will investigate how genome-editing therapies affect cell metabolism. Mathieu adds CRISPR expertise to the UW research team. Several faculty members from other departments are also on the team.

How broad are the implications of developing responsible genome-editing methods?

This is a new paradigm for therapy development, says Freedman. Were looking at the kidney. But the liver, heart, and lungs all have similar challenges. Our hope is to create a model for doing this work in human organoids, which are faster and more humane than animal models, and can be more directly compared to human patients.

Genome editing has extraordinary potential to alter the treatment landscape for common and rare diseases, said Christopher P. Austin, director of the National Center for Advancing Translational Sciences and SCGE Program Working Group chair. The field is still in its infancy, and these newly funded projects promise to improve strategies to address a number of challenges, such as how best to deliver the right genes to the correct places in the genome efficiently and effectively. Together, the projects will help advance the translation of genome-editing technologies into patient care.

Nearly 40 million Americans have chronic kidney disease, a family of progressive conditions that can come with widespread health complications, including a higher risk for heart disease. When kidneys fail, the primary interventions, dialysis and kidney transplants, are not cures. These treatments come with significant side effects and a heavy economic burden. Medicare costs average $114 billion a year total for the care of the nations patients with kidney failure. Altogether, kidney disease is the ninth leading cause of death in the United States.

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Thatcher Heldring of the Institute for Stem Cell and Regenerative Medicine contributed to this news report.

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Genome editing to be tested in kidney organoids - UW Medicine Newsroom