Stem Cell Clinic

Experts see blood stem cell transplant as a curative treatment for thalassemia – India Today

Baby Bhushra Naqeeb, an 11-year-old girl who has suffered from thalassemia since the age of 6 months, recently suggested an allogeneic stem cell transplant as her only chance of survival after over 10 years of blood transfusion. Finally, with the support from the DKMS-BMST thalassemia programme, she underwent stem cell transplantation in October 2021 under Dr. Sachin Jadhav, HCG (HeathCare Global Enterprises Ltd.) group of hospitals. Post the transplant, Bhushra is able to lead a normal life as she also attends regular school.

India is also known as the Thalassemia capital of the world. The country has the largest number of children with thalassemia major with over 10,000 children born with it every year. According to the World Health Organisation (WHO), over four million Indians are thalassemia carriers and more than 1,00,000 are patients. Parents, who are usually asymptomatic, are the carriers of this disease and have a 25% chance of passing this disease to their children.

Patients suffering from thalassemia are required to undergo lifelong regular blood transfusions to maintain their hemoglobin levels. However, it can now be cured with stem cell transplantation.

THE ONLY CURE

Dr. Sunil Bhat, Director and Clinical Lead, Pediatric Hematology, Oncology and Blood & Marrow Transplantation, Narayana Health said, Thalassemia patients are mostly children who go through painful blood transfusion for several years in their life. Blood transfusions have their own challenges and risks for the patients. A stem cell transplantation is currently the only curative option available for this condition. Recent data shows a more than 90% success rate of stem cell transplantation in patients who have HLA (Human Leukocyte Antigen) matched stem cell donors.

In a blood stem cell transplant, stem cells are collected from the blood of the donors and transplanted into the thalassemia patient after their bone marrow has been destroyed. Only 30% of patients who need transplants have a fully HLA-matched donor in their family, while the rest of them depend on an unrelated donor.

Shobha Tuli, the founder of the NGO - Thalassemics India, President-Federation of Indian Thalassemia Associations, says At present, stem cell transplant is the only curative treatment available for Thalassemia patients. If not cured on time, such patients are dependent on blood transfusions all throughout their lives along with other expensive treatments & regular investigations. Thalassemia Bal Sewa Yojna is a unique project initiated by the Ministry of Health & Family Welfare, Govt. of India. Under this scheme, a patient can get financial aid up to Rs. 10 lakhs from Coal India Ltd. DKMS has offered us an opportunity to get free HLA tests done in the country. This enables the patients to check if they have a matched sibling.

LOW AWARENESS

There are around 270 million Thalassemia patients in the world but awareness about the disease is alarmingly less. Symptoms of Thalassemia usually start at 4 to 6 months of age and usually present with lethargy, poor feeding, progressive pallor, poor weight gain and sometimes vomiting and diarrhoea. On examination, the babies are pale and have liver and spleen enlargement.

Patrick Paul, CEO, DKMS BMST Foundation India, says, It is our mission to support blood cancer, and other blood disorders such as Thalassemia patients in India, for which we have initiated the DKMS-BMST Thalassemia programme. Under this programme, DKMS-BMST collaborates with local NGOs and transplantation clinics to organise camps where paediatric thalassemia patients and their siblings travelling from afar places in India to give buccal swab samples for free HLA typing. Samples from the camps are analysed in the DKMS laboratory based out of Germany and clinical matching reports of the same are provided. In cases where there is no matching sibling for a sick child, we also support unrelated donor searches for patients. Since the inception of the programme, so far, 7,162 HLA typings have been facilitated by DKMS-BMST.

HOW YOU CAN HELP

Dr. Bhat also added, A successful blood stem cell transplant depends on a perfect HLA tissue match. Patients and donors of Indian origin have unique HLA characteristics that are severely under-represented in the global database, which makes the probability of finding a suitable donor even more difficult. Indian patients mainly require an Indian tissue match. This calls for increased awareness and the need to encourage many more people in India to register as potential blood stem cell donors.

To register as a potential blood stem cell donor, healthy individuals between 18-50 years of age can register at: dkms-bmst.org/register

All it takes is five minutes of your time and a simple 3-step process:

Step 1: Visit the site, fill in an online form and you will receive a DIY swab kit at home.

Step 2: Once you receive the swab kit, fill out the consent form and take a tissue sample from the inside of your cheeks with 3 cotton swabs provided in the kit.

Step 3: Send back your swab sample in the pre-paid envelope provided.

The DKMS laboratory will then analyse your tissue type and your details will be available in the global search for blood stem cell donors. If you do come up as a suitable donor, DKMS-BMST will get in touch with you straight away. Once you come up as a match, blood stem cells will be obtained from the bloodstream using a procedure called Peripheral Blood Stem Cell Collection, which is similar to a blood donation wherein only your stem cells are taken. This is a safe, non-surgical outpatient procedure.

READ MORE | JNU biologists identify a new way to better treat kala-azar

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Experts see blood stem cell transplant as a curative treatment for thalassemia - India Today

Sleep deprivation may increase the risk of eye disease – Medical News Today

While scientists know that sleep deprivation has a negative effect on the body, they are still researching how it affects different organs.

Researchers from China and the U.S. recently published a study in Stem Cell Reports on how poor sleep can affect the eyes. They found that sleep deprivation can affect both stem cells in the cornea and the tear film surface of the cornea.

Getting a good nights sleep on a regular basis is important, but according to the Centers for Disease Control and Prevention (CDC), more than one-third of adults dont get enough sleep. Doctors recommend that adults get at least 7 hours of sleep per night.

The CDC reports that not getting enough sleep can cause a myriad of health issues, such as having a higher risk for obesity, developing diabetes, and high blood pressure.

Part of the issue contributing to so many people not getting enough rest is having a sleep disorder. The National Institutes of Health reports that around 40 million Americans have a sleep disorder.

Some sleep disorders include:

For those who are not affected by a sleep disorder, the best way to regularly get enough sleep is to practice good sleep hygiene. People can accomplish this by going to bed at the same time every night, avoiding screens 1-2 hours before bed, and not drinking alcohol before bed.

The researchers in this study used mice to learn more about how sleep deprivation affects the eyes.

According to the authors, the cornea is the clear front surface of the eye. They also write that the cornea has an overlying tear film that helps keep the eyes comfortable and offers protection against infection.

The researchers were interested to find out to what degree sleep deprivation may affect stem cells in the cornea.

As Dr. Neil Neimark, a board certified family physician in functional medicine who applies stem cell therapy in his practice, noted in a TEDx Talks podcast, stem cells have healing power and all tissue repair in the body is initiated by stem cells.

The researchers of the current study assessed gene expression in the mice after 2 days of sleep deprivation and then after 10 days of sleep deprivation.

At the 2-day point, the researchers found that 287 genes were significantly upregulated and 88 were downregulated in corneas. At the 10-day point, they saw 272 significantly upregulated genes and 150 downregulated genes.

The authors tested the mice after 1 and 2 months of further sleep deprivation and found that the transparency of the cornea was reduced and the ocular surface was rough.

While stem cells were upregulated in the sleep-deprived mice early on, it eventually led to what the authors referred to as an early manifestation of limbal stem cell deficiency. After being upregulated for so long, the stem cells became depleted.

Short-term consequences of insufficient sleep or delayed sleep cause ocular discomfort, including dryness, pain, pruritus, and hyperemia of the eye, the authors note.

Despite these issues, the authors observed that treating the mice with damaged corneas with eye drops containing antioxidants helped restore the eye health of the mice.

Dr. Howard R. Krauss, a surgical neuro-ophthalmologist and director of Neuro-Ophthalmology for the Pacific Neuroscience Institute at Providence Saint Johns Health Center in Santa Monica, CA, who was not involved in the study, spoke to Medical News Today about the findings.

The design of the study was to study chemical and cellular changes in the ocular surface of sleep-deprived mice, revealing indeed that there are damaging effects, which shed light on mechanisms which may be at play in human symptoms and disease, Dr. Krauss said.

While Dr. Krauss thought the study was helpful at showing how sleep deprivation can potentially affect humans, he noted a limitation.

A weakness of the study is the methodology by which sleep deprivation is induced in mice, who are in cages, perched on sticks to remain above a water-filled bottom when the mouse falls asleep, it falls into the water, immediately wakes up, and climbs back up onto the stick, Dr. Krauss explained.

Dr. Krauss said the method used to induce sleep deprivation raises the question of how much of the chemical and cellular change they observed was purely secondary to sleep deprivation and how much may be a stress reaction to the means by which sleep deprivation has been engineered.

Nonetheless, the study refocuses our attention on sleep deprivation and leads us to think that the scope of damaging effects of sleep deprivation may be far [broader] than we realize, he said.

As such, the human need for sleep for maintenance of good health becomes more obvious every day. Dr. Howard R. Krauss

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Sleep deprivation may increase the risk of eye disease - Medical News Today

Aileron Therapeutics Announces Late-Breaking Oral Presentation of Non-Clinical Data Demonstrating ALRN-6924 Protected Human Hair Follicles and Their…

Aileron Therapeutics, Inc.

Taxanes, such as paclitaxel and docetaxel, cause severe and often permanent chemotherapy-induced hair loss (alopecia)

New non-clinical data demonstrate proof of principle that ALRN-6924 can temporarily arrest the cell cycle in human scalp hair follicles and their stem cells

ALRN-6924-induced cell cycle arrest protected hair follicles from paclitaxel-induced toxicity and irreversible stem cell damage

Ailerons precision medicine-based approach is designed to selectively protect normal, healthy cells from chemotherapy while ensuring chemotherapy cannot protect cancer cells

Ailerons ongoing non-small cell lung cancer (NSCLC) clinical trial and upcoming breast cancer clinical trial will evaluate ALRN-6924s protection against chemotherapy-induced bone marrow toxicities and other side effects, including alopecia

BOSTON, May 10, 2022 (GLOBE NEWSWIRE) -- Aileron Therapeutics (Nasdaq: ALRN), a chemoprotection oncology company that aspires to make chemotherapy safer and thereby more effective to save more patients lives, today announced a late-breaking oral presentation at the upcoming Society for Investigative Dermatology (SID) Annual Meeting, which will be held May 18 21, 2022 in Portland, Oregon. The presentation will highlight new non-clinical data developed in collaboration with Professor Ralf Paus, M.D., DSc, FRSB and his colleagues at the Dr. Phillip Frost Department of Dermatology & Cutaneous Surgery at the University of Miami Miller School of Medicine. This collaboration has generated promising ex vivo data demonstrating that ALRN-6924 protected human hair follicles and their stem cells from chemotherapy-induced acute and permanent damage. Details of the presentation are as follows:

Title:

ALRN-6924, a dual inhibitor of MDMX and MDM2, protects human scalp hair follicles and their epithelial stem cells from paclitaxel-induced toxicity (LB1018)

Presenter:

Jennifer Gherardini, Ph.D.; Paus Laboratory, University of Miami Miller School of Medicine

Date:

Thursday, May 19th

Time:

8:45 AM 11:15 AM PT

Session:

Late-Breaking Abstract Concurrent Session

Chemotherapy-induced toxicities range from severe and life-threatening to those that impact and diminish patients quality of life, sometimes long after chemotherapy has been completed. These toxicities occur because chemotherapy destroys normal, healthy cells while simultaneously destroying cancer cells, said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer at Aileron. Previously, we showed chemoprotection against severe bone marrow toxicities in small cell lung cancer patients receiving topotecan and demonstrated in healthy volunteers the mechanism of action cell cycle arrest underlying this chemoprotection benefit. We are excited to now present new data that may suggest ALRN-6924s ability to also protect against chemotherapy-induced hair loss, another devastating chemotherapy-induced side effect for millions of cancer patients.

Dr. Paus commented, These results got us quite excited as they directly follow in the footsteps of our prior work that showed arresting the cell cycle can have a strong protective effect against taxane-induced hair follicle damage. Until our research with ALRN-6924, we had not come across a cell cycle arrest-inducing drug that is in clinical testing for protection of normal cells without protecting cancer cells. Thus, ALRN-6924 invites a very promising and completely novel selective protection approach. In addition, we found that ALRN-6924 may exert some additional benefits that could reduce the risk of long-term damage of human hair follicle stem cells by taxanes.

Story continues

Aileron is currently developing ALRN-6924, a first-in-class MDM2/MDMX dual inhibitor, to selectively protect healthy cells in patients with cancers that harbor p53 mutations to reduce or eliminate chemotherapy-induced side effects while preserving chemotherapys attack on cancer cells. ALRN-6924 is designed to activate p53 in normal cells, which in turn upregulates p21, which pauses cell cycle in normal cells but not in p53-mutated cancer cells. The companys vision is to bring chemoprotection to all patients with p53-mutated cancer regardless of the type of cancer or chemotherapy.

About the Findings

Taxanes, such as paclitaxel and docetaxel, are known to cause severe and often permanent chemotherapy-induced alopecia. Over 90% of patients treated with this chemotherapy class experience alopecia, and approximately 10% (paclitaxel) to 25% (docetaxel) of patients experience permanent alopecia. Dr. Paus and his team previously demonstrated that paclitaxel damages human scalp hair follicles by inducing massive mitotic defects and apoptosis in hair matrix keratinocytes as well as bulge stem cell DNA damage, and that pharmacological induction of transient cell cycle arrest can protect hair follicles and stem cells (Purba et al. EMBO Molecular Medicine 2019). Aileron previously conducted in vitro studies showing that ALRN-6924 protected human fibroblasts in cell culture from multiple chemotherapies, but not p53-mutant breast cancer cells.

In the new non-clinical findings to be presented at the SID meeting, when organ-cultured anagen (i.e., active growth phase) scalp hair follicles from two human donors were pre-treated with ALRN-6924 or vehicle (i.e., placebo), followed by paclitaxel or vehicle, ALRN-6924 significantly increased the number of p21-positive hair matrix keratinocytes and bulge stem cells compared to vehicle or paclitaxel alone, confirming cell cycle arrest ex vivo. Further, pretreatment of paclitaxel-treated human hair follicles with ALRN-6924, led to a reduction in the number of melanin clumps, a marker of hair follicle cytotoxicity and dystrophy, as well as a reduction in apoptosis, pathological mitosis, and DNA damage. Aileron believes that these findings support clinical investigation of ALRN-6924 to prevent both acute and permanent chemotherapy-induced alopecia, in addition to its ongoing evaluation of ALRN-6924 to protect against chemotherapy-induced bone marrow and other toxicities.

About Ailerons Clinical Trials of ALRN-6924

Aileron is on track to initiate a Phase 1b randomized, controlled trial of ALRN-6924 in patients with p53-mutated ER+/HER2- neoadjuvant breast cancer in 2Q 2022. The planned breast cancer trial will evaluate ALRN-6924s protection against chemotherapy-induced bone marrow toxicities, as well as other toxicities, including alopecia, in patients with p53-mutated ER+/HER2- breast cancer treated with a doxorubicin plus cyclophosphamide and docetaxel chemotherapy regimen.

The company is currently enrolling patients in a Phase 1b randomized, double-blind, placebo-controlled trial evaluating ALRN-6924s protection against chemotherapy-induced bone marrow and other toxicities in patients with advanced p53-mutated non-small cell lung cancer undergoing treatment with first-line carboplatin plus pemetrexed with or without immunotherapy. While patients in this trial are monitored for alopecia, historically, only a small percentage of patients treated with carboplatin plus pemetrexed experience acute alopecia. Aileron is on track to report interim results on the first 20 patients enrolled in the NSCLC trial in June 2022 and topline results on 60 patients in 4Q 2022.

About Aileron Therapeutics

Aileron is a clinical stage chemoprotection oncology company that aspires to make chemotherapy safer and thereby more effective to save more patients lives. ALRN-6924, our first-in-class MDM2/MDMX dual inhibitor, is designed to activate p53, which in turn upregulates p21, a known inhibitor of the cell replication cycle. ALRN-6924 is the only reported chemoprotective agent in clinical development to employ a biomarker strategy, in which we exclusively focus on treating patients with p53-mutated cancers. Our targeted strategy is designed to selectively protect multiple healthy cell types throughout the body from chemotherapy without protecting cancer cells. As a result, healthy cells are spared from chemotherapeutic destruction while chemotherapy continues to kill cancer cells. By reducing or eliminating multiple chemotherapy-induced side effects, ALRN-6924 may improve patients quality of life and help them better tolerate chemotherapy. Enhanced tolerability may result in fewer dose reductions or delays of chemotherapy and the potential for improved efficacy.

Our vision is to bring chemoprotection to all patients with p53-mutated cancers, which represent approximately 50% of cancer patients, regardless of type of cancer or chemotherapy. Visit us at aileronrx.com to learn more.

Forward-Looking Statements

Statements in this press release about Ailerons future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements about the potential of ALRN-6924 as a chemoprotective agent, including its ability to prevent both acute and permanent chemotherapy-induced alopecia, and the Companys strategy and clinical development plans. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether Ailerons cash resources will be sufficient to fund its continuing operations for the periods anticipated or with respect to the matters anticipated; whether initial results of clinical trials will be indicative of final results of those trials or results obtained in future clinical trials, including trials in different indications; whether ALRN-6924 will advance through the clinical trial process on a timely basis, or at all; whether the results of such trials will be accepted by and warrant submission for approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether ALRN-6924 will receive approval from regulatory agencies on a timely basis or at all or in which territories or indications ALRN-6924 may receive approval; whether, if ALRN-6924 obtains approval, it will be successfully distributed and marketed; what impact the coronavirus pandemic may have on the timing of our clinical development, clinical supply and our operations; and other factors discussed in the Risk Factors section of Ailerons annual report on Form 10-K for the year ended December 31, 2021, filed on March 28, 2022, and risks described in other filings that Aileron may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Aileron specifically disclaims any obligation to update any forward-looking statement, whether because of new information, future events or otherwise.

Investor Contact: Stern Investor Relations Alexander Lobo alex.lobo@sternir.com

Media Contact: Liz Melone 617-256-6622

Original post:
Aileron Therapeutics Announces Late-Breaking Oral Presentation of Non-Clinical Data Demonstrating ALRN-6924 Protected Human Hair Follicles and Their...

Aspen Neuroscience Announces $147.5 Million Series B Financing, Led by GV, LYFE Capital and Revelation Partners – PR Newswire

Aspen Neuroscience is preparing to study its first autologous cell replacement candidate in Parkinson's disease

GV, LYFE Capital and Revelation Partners co-led the investment round with participation from additional new investors Newton Investment Management, Singapore-based global investor EDBI, LifeForce Capital, Medical Excellence Capital Partners, Mirae Asset Capital, NS Investment and others. As part of the financing, Doug Fisher of Revelation Partners will join the board of directors.

The raise included significant investments from Series A/Seed investment teams including OrbiMed, ARCH Venture Partners, Frazier Life Sciences, Section32 and Alexandria Venture Investments.

"As the leader in autologous cell therapy development, Aspen Neuroscience is preparing to study its first cell replacement candidate in Parkinson's disease," said Damien McDevitt, Ph.D., president and chief executive officer. "This company was founded by a visionary team of scientists and patient advocates, who envisioned a day when personalized cell replacement could be used to target neurodegenerative diseases. We are happy to be aligned with such a prestigious group of founders and investors, having raised more than $220 million since the company's founding, towards our mission to develop transformational medicines for patients with limited treatment options."

The Series B funding will support the planned studies of the company'slead product candidate for Parkinson's disease, ANPD001, including its patient Screening Cohort study and the upcoming Phase 1/2a clinical trial,post-IND submission to FDA.

"This is an important milestone in the field of cell therapy for neurodegenerative diseases and we are immensely proud of this innovative team, who are working to transform the field and to modify the course of Parkinson's disease," said Faheem Hasnain, Aspen Neuroscience chairman.

Aspen is the leading company developing a personalized cell replacement using a patient's own cells, eliminating the need for immunosuppressive therapy. Developed from a simple skin biopsy, each patient's stem cell-derived dopamine neurons will be evaluated for potential effectiveness using proprietary AI-based genomics tools, before being transplanted for clinical use. Aspen Neuroscience's process allows for a reduction in the time and cost of the manufacturing process required to produce a safe, reproducible, and personalized autologous cell therapy. To date, Aspen has performed in vitro and in vivo studies demonstrating that the protocol reliably produces dopamine-releasing neurons when examined both physiologically and functionally.

Dr. McDevitt, continued, "We combine stem cell biology with innovative AI/bioinformatic and manufacturing tools to develop patient-specific approaches. To that end, the Series B financing will also support our R&D pipeline, which includes autologous gene-corrected cells and programs that explore the treatment of other neurological diseases."

About Parkinson's DiseaseParkinson's Disease is the second most common neurodegenerative disease, affecting around one million Americans and greater than ten million people worldwide, with 60,000 people newly diagnosed every year. Even with the current standard of care therapy, patients eventually develop debilitating motor complications due to loss of dopamine neurons in the brain; approximately 50% are lost even before diagnosis. Cell replacement therapy of dopamine neurons has the potential to release dopamine and reconstruct neural networks.

About Aspen NeuroscienceHeadquartered in San Diego, Aspen Neuroscience, Inc. is a development stage, private biotechnology company focused on personalized (autologous) cell therapies. The company is developing induced-pluripotent stem cells (iPSCs) to address diseases with high unmet medical need, beginning with autologous neuron replacement for both sporadic and genetic forms of Parkinson's disease (PD) and extending across the brain and affected organs.

A leading iPSC platform company, Aspen combines stem cell biology with the latest artificial intelligence and genomic approaches to investigate patient-specific, restorative treatments. The company has developed a best-in-class platform to create and characterize pluripotent-derived cell medicines, which includes in-house bioinformatics, manufacturing and QC. For more information and important updates,please visit http://www.aspenneuroscience.com.

SOURCE Aspen Neuroscience , Inc.

Continued here:
Aspen Neuroscience Announces $147.5 Million Series B Financing, Led by GV, LYFE Capital and Revelation Partners - PR Newswire

Stem Cell Technology Helps Identify a Potential Casual Mechanism in Schizophrenia That Could Be Targeted – Brain & Behavior Research Foundation |

New research by a team that included four BBRF grantees has demonstrated the value of studying the causes of complex psychiatric illness by analyzing neurons grown in the laboratory, using stem-cell technology.

In experiments reported in Nature Communications, 2014 BBRF Young Investigator Ian Maze, Ph.D., a Howard Hughes Medical Institute Investigator at the Icahn School of Medicine at Mount Sinai, and 2019 BBRF Young Investigator Lorna A. Farrelly, Ph.D., also at Mount Sinai, and colleagues, used stem cell technology to identify a protein involved in the regulation of gene expression whose inhibition may help to ameliorate abnormalities in neurons that are associated with early pathology in schizophrenia. Adding to the interest of the finding, there is a known drug that can inhibit the protein in question, suggesting a potential future approach to treat or even prevent schizophrenia in some instances.

Developed in the second decade of the 2000s by researchers including BBRF Scientific Council members Ronald McKay, Ph.D., Stewart Anderson, M.D., Fred Gage, Ph.D. and Kristen Brennand, Ph.D., "human induced pluripotent stem cell" (hiPSC) technology involves harmlessly sampling cells (usually skin cells) from an individual and then genetically reprograming these cells to re-develop as other cell types. When reprogrammed as neural cells, these baby cells are grown in culture dishes, and can be brought together with other developing neurons to form "organoids," in which neurons and other cells found in the living brain wire together and form functional networks.

Not only does stem-cell technology enable researchers to generate virtually limitless quantities of live human neurons, overcoming the problem of having to rely on postmortem brain tissue to study brain tissue from psychiatric patients. Just as important, every cell perfectly represents the complex genetics of the patient whose donated skin cells are the basis of the organoid.

This makes hiPSC technology uniquely valuable in the study of illnesses like schizophrenia, in which genetic factors are strongly involved in causation, and pathology in many cases is hypothesized to have its origins in the early development of the braina phase that organoids can recapitulate in the lab.

The team, which included BBRF Scientific Council members Dr. Brennand (who is 2018 BBRF Maltz Prize winner, 2016 Independent Investigator and 2012 Young Investigator) and 2011 BBRF Lieber Prize winner and 2010 and 1998 BBRF Distinguished Investigator Carol A. Tamminga, M.D., induced skin cells sampled from individuals with schizophrenia to re-develop as neurons of the type found in the forebrain. Cells in the forebrain process information from the senses and are involved in thinking, perceiving, producing, and understanding language, as well as controlling motor function.

The team made an important observation in reprogrammed neurons derived from patient samples as these cells were maturing: they detected aberrant patterns of epigenetic activity. Epigenetics refers to molecular processes that affect the way specific genes are expressed in a cell. Every human cell contains an individual's entire genome, and gene-regulating factors, including epigenetic factors, determine when and where in the body or in an organ like the brain specific genes will be activated or repressed, depending upon the biological context.

The abnormal epigenetic pattern the team found is called hyperacetylation. Acetyl molecules are among the epigenetic factors that attach to DNA packaging proteins to encourage or repress gene activation. Hyperacetylation means that there are too many acetyl molecules attaching to bundles of DNA called histones. This causes one or more genes to be abnormally regulated.

The team also identified a specific protein, called BRD4, which, when blocked or repressed in its activity of "reading" the epigenetic state of a given histone, can restore or alleviate the severity of aberrant gene expression caused by hyperacetylation. Experiments indicated that a BRD4 inhibitor might specifically alleviate a kind of aberrant gene expression linked with schizophrenia.

This is exciting because a drug called JQ1 has been shown, in other research, to prevent interactions between proteins like BRD4 and bundled DNA. The drug has been tested in anti-cancer applications, but as the team notes, "the potential of using such inhibitors [of BRD4 and related proteins] to alleviate schizophrenia-related gene expression has remained unexplored."

The team proposes that treatments with JQ1 might "partially rescue" irregularities in gene expression associated with schizophrenia. They say their results warrant further experimental investigation of this possibility.

In addition to Drs. Brennand and Maze, Haitao Li, Ph.D. of Tsinghua University, PRC, was also a senior member of the team.

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Stem Cell Technology Helps Identify a Potential Casual Mechanism in Schizophrenia That Could Be Targeted - Brain & Behavior Research Foundation |

INFOBAE.COM: Houston doctors and researchers that are making the biggest international impact according to the KOLs list – Most of them are women – PR…

SANTA BARBARA, Calif., May 11, 2022 /PRNewswire/ --The work of Houston doctors and researchers is getting international attention. The most researched areas in Houston after COVID are Oncology, Cardiology, and Endocrinology.

According to the KOLs list, the leading institutions are The University Of Texas MD Anderson, Houston Methodist, Harris Health System, Heart Institute, and Baylor College Of Medicine.

Gender diversity among the top Houston researchers

Ana Gannon, Director of technology of the firm Key Opinion Leaders, commented on the issue "It is not common to see the level of gender diversity we see in Houston in terms of researchers that are creating international impact with their work and findings."

"To give an example, within the group of Houston researchers working on next-generation cancer therapies, such as CAR-T, and whose work is having an international projection, the vast majority of them are women," Mrs. Gannon added.

The analysis prepared by the firm Key Opinion Leaders and available at keyopinionleaders.comquantifies the level of impact of researchers around the world for specific medical concepts like medical conditions, medications, active ingredients, treatments, devices, and more.

What is a "Key Opinion Leader"?

A key opinion leader (KOL) is an expert, thought leader, or influencer who has earned the trust of their peers for an area of knowledge. In healthcare, KOLs play an essential role in shaping the discourse around key issues and helping to drive change within the health system. Patients, physicians, and sometimes even regulatory agencies accept their input while making decisions.

Top Houston Researchers working on next-generation Cancer therapies

According to the KOLs list, some of the Houston researchers whose work on next-generation cancer therapies is getting the most international attention are:

... Please click here to see the other research categories and the full KOLs list on infobae.com.

Amy Mcquade [emailprotected] https://www.youtube.com/watch?v=t32izA3MWrwHow-to-guide for finding KOLs

SOURCE Key Opinion Leaders, LLC

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INFOBAE.COM: Houston doctors and researchers that are making the biggest international impact according to the KOLs list - Most of them are women - PR...