Category Archives: Genetic medicine

Damian Adams grew up knowing that his parents had used an anonymous sperm donor to conceive him, and as a teen, he was even proud of this identity. He considered donating to help other families have children. Becoming a father himself, however, changed everything. When his daughter was born 18 years ago, he cradled her in his arms, and he instantly saw himself in her and her in himself. He felt a biological connection so powerful that it made him reconsider his entire life up until then. What Id had there with my daughter, he says, was one thing I had been missing in my life. He felt the need to know where he came from.

Adams, a biologist in Australia, would spend years searching for his biological father, running into one dead end after another. Meanwhile, he also began campaigning to end donor anonymity for others like him. In 2016, he and fellow activists pushed the state of Victoria to retroactively abolish anonymity for all sperm donors. (A previous law had already banned it from 1998 onward.) Donor-conceived people in the United Kingdom have also successfully campaigned to ban anonymous sperm donation. In the United States, where anonymous donation is still technically offered, some donor-conceived people are asserting a right to know their genetic origins and even to contact their biological parents, who may or may not welcome the surprise.

All of this was unimaginable a few decades ago. Doctors used to routinely advise parents to keep the use of a sperm donor secreteven from their own childrenand this silence reinforced a sense of shame about the practice. Today, parents are strongly encouraged to tell the truth; moreover, DNA tests mean they couldnt hide it even if they wanted to. As more people find out they are donor-conceived, they are in turn finding one another: They are gathering in online communities such as We Are Donor Conceived and other Facebook support groups catering to a mix of donors, parents, donor-conceived people, and others who have learned that their parents are not who they thought they were. There are also several podcasts, at least two magazines, and even training courses for therapists who work with people in this situation. The shared identity that connects this online community is small by proportion but large in raw numbers. An estimated 30,000 to 60,000 children conceived with donor sperm are born in the U.S. every year, though that statistic may well be an underestimate. The fertility industry doesnt have to keep records, so the true number is unknown.

In learning more about their own conception, some donor-conceived people have been shocked by the lack of transparency in the industry that created them. They have been disturbed to find, in some cases, that they have dozens of half siblings from the same donor, that doctors have secretly impregnated patients with their own sperm, or that donors have lied about themselves to sperm banksall at least partially because donation was anonymous. Now donor-conceived people like Adams are questioning the need for any secrecy at all. In a forthcoming book called Uprooted, Peter Boni, who learned he was donor-conceived at age 49, lays out a Donor-Conceived Bill of Rights that demands, first and foremost, the end of anonymous donations and includes access to a donors medical records, limits on the number of offspring per donor, and consequences for outright fertility fraud. Can you point to any federal law, Boni asked me rhetorically, that protects the rights of the donor-conceived child?

Indeed, the agreements around sperm donations were originally forged among donors, parents, and doctors. Fast-forward a few decades and the childrennow adultsare trying to change a fertility industry that sees them as neither its customers nor its patients, even though it is directly responsible for their existence. The issues raised might apply to both sperm and egg donation alike, though historically, the focus has been on sperm donation because its much more common. As donor-conceived people advocate for new rights today, they are also forcing society to confront a more fundamental question, one left perhaps inadequately answered all those years ago: How much do biological ties really matter?

In a recent newsletter, the journalist Alison Motluk, who covers assisted reproduction, highlighted a discussion comparing three scenarios in which people were cut off from a biological parent:

One had a biological parent die before they were born. A second had a biological parent abscond before they were born. A third was conceived using an anonymous donor. Why do we acknowledge loss and feel empathy in the first two scenarios but not in the third?

I liked the way the question was framed, in terms of empathy rather than the harder-edged language of rights. Because when donor-conceived people ask for regulations on the exchange of sperm, they are also asking for acknowledgment that this biological exchange matters, that biological origins matter. We naturally recognize this in situations where a child has never met a parent owing to tragic life circumstancesso how does the situation then differ when the bond is severed by biotechnology instead? In my conversations with donor-conceived people, they often grappled with comparisons to more familiar scenarios. They are asking for rights, but they are also asking for compassion.

Tiffany Gardner, now 39 and an attorney in Atlanta, told me she had lost her father, Ken, to colon cancer when she was 4. Although Gardner later became close to her stepfather, she continued to treasure the few things she knew about Kenhe had been good at baseball and horseback riding, and she gravitated toward the same sports to maintain some kind of connection. At age 35, Gardner learned that her parents had actually used a sperm donor, and when she eventually found him, via a DNA test, parts of her identity suddenly clicked into place. She saw a photo of the donors son and it was, she told me, my face popping up as a dude. Her biological father had been an art teacher, and Gardner immediately linked that detail to her own longing to go to art school when she was younger. These connections made it all the more painful when the donors family objected to his having a relationship with her, and he abruptly cut her off.

To Gardner, her desire to connect with her biological fatherher actual biological father, this timewas not so different from her desire to stay connected with Ken. Her immediate family has been supportive, she said, but shes faced some pushback from acquaintances and older family members. Ive had a couple people that are sort of like, I dont get it. Those people arent really your family. Why do you want anything to do with this? she told me. But to Gardner, genetic ties clearly matteredif not, why did she spend her first 35 years trying to connect with a dead father she never really knew?

Adams began asking these same questions when he became a father. Not long after his daughters birth, it occurred to him that if he died right there and then, people would acknowledge the tragedy of his daughter never getting to know him. They would all recognize I was her father, he told me. Or, he went on, lets say, for example, a one-night stand. People recognize that as a tragedy, when that person is not in that childs life. Or in adoption, we recognize that as being problematic. The adoption world has in fact moved toward more and more open adoptions.

Isnt the difference, I asked, that sperm donation never promises, even implicitly, a relationship, and in fact in most cases explicitly disavows one before conception? To Adams, that disavowal made the situation even worsethat society would intentionally create people cut off from their biological parents. The genetic connection was, to him, immutable. If the connection really meant nothing, Adams said, why do many couples who use an egg or sperm donor still choose to have the child be related to one parent? It cant be that the connection is meaningful in one case but not in the other.

Not every donor-sperm-conceived personthey are, after all, a large groupfeels the same about the strength of genetic connection. In a 2020 survey conducted by We Are Donor Conceived in its own Facebook group and another called the Worldwide Donor Conceived People Network, 67 percent of respondents wanted the donors identity to be known from birth. Only 33 percent, though, felt that donors needed to be available for a relationship with the child from birth.

But its also impossible to survey the donor-conceived people who never join these groups, for whom this fact about their birth might feel unremarkable. Anecdotally, at least, this is common. Many of the donor-conceived people Ive talked with mentioned half siblings who never responded to messages through 23andMe or AncestryDNA, or who responded politely once and stopped there. Some people might feel very little curiosity about their biological origins, says Brianne Kirkpatrick, a genetic counselor who has helped many people through DNA discoveries. Some might feel that any curiosity would be betraying their family, and some are simply more interested in donor siblings than the donor. Age or stage of life may play into how people react differently, Kirkpatrick suggests, but theres not much research on why. What donor-conceived people want is a question few people asked until recently.

Sperm donation has already changed dramatically in the past three to four decades, and some of the changes that donor-conceived people are pushing have indeed happened, even if they are not necessarily enshrined in law. The secrecy around the use of donor sperm, for example, has dissipated as more single women and lesbian couples have openly used sperm banks. The ethics committee of the American Society of Reproductive Medicine (ASRM) now strongly encourages parents to tell their children if they were donor-conceived. Fertility doctors have also gone from procuring live sperm samples themselvescommonly from medical studentsto ordering frozen sperm from banks that can more thoroughly, if not always perfectly, screen donors. ASRM guidelines suggest that banks voluntarily limit the number of births per donor to 25 in a given population of 800,000 people. And sperm banks are more likely to offer open-ID donations, in which the donor can be contacted by his biological children. Some banks still have anonymous donations, in which they dont share any donor information. But these days, I think someone would have to be intentionally ignorant to not realize anonymity is not possible, Kirkpatrick says. Even without more formal regulation of sperm donation, widespread DNA testing means theres no more hiding in the passage of time.

These shifts have left plenty of gaps, though: Sperm banks, for example, do not have to keep track of births or coordinate with one another, so one donor could still be a prolific biological father. (There are donors with more than 100 biological children.) And in practice, anonymous donation still leaves the job of identifying a biological parent to the donor-conceived person. This could be easy enough if they are lucky and get a close DNA match such as a half sibling or first cousin, but it may be impossible or near-impossible if not. Some donor-conceived people have spent years searching.

Even so, the changing landscape of sperm donation is impossible to ignore, and donor-conceived children have grown up into adults who have their own opinions. At the ASRMs annual meeting in Baltimore next week, a panel is scheduled to discuss Open Identity Gamete Donation: What Are the Children Saying? (Gamete is the scientific term for egg or sperm.) But Erin Jackson, the founder of We Are Donor Conceived, pointed out to me that, despite the panels name, it features four experts and the mother of a donor-conceived sonbut not any children who are donor-conceived. To her, this just revealed how the fertility industry continues to ignore the people it helped create.

When I inquired with the ASRM, the organizations chief advocacy and policy officer, Sean Tipton, underplayed the importance of a single panel. So does ASRM have any plans to engage with the donor-conceived community? I asked. We very well might, Tipton said, without specifying what that would entail. He also questioned whether they were in fact the right people to engage: Were including patients a lot more in our organizations deliberations but, again, these are not patients. Furthermore, he added, a 30-year-old, donor-conceived person in 2021is that the right person to consult about somebody whos going through the process now? After all, none of us has a say in the circumstances of our conception, natural or otherwise. If donor-conceived people have the right to know about their genetic parentage, we should also consider how that right extends to other aspects of parent-child relationships and in other situations, says Judith Daar, the dean of Northern Kentucky Universitys law school and the former chair of the ASRM ethics committee that drafted guidelines for disclosure about donor conception in 2018. For example, she says, theres a debate in the infertility community whether a child had a right to know they were conceived through IVF, which may reveal aspects of the parents private reproductive history. And what about naturally conceived children, Daar askswhat do parents owe them about their sex lives?

Most people dont want to know how they were conceived, you know? Jackson said. Its not something you want to picture. For her, its medical, though. And, more important when donors and sperm banks are involved, its commercial. Money changes hands. Contracts are signed. The feeling of commodification is very real, she told me. The fertility industry is responsible for the creation of human beings; it is also a multibillion-dollar industry that is growing every year. Donors, doctors, and parents today, Jackson said, should be thinking more clearly about the futurebeyond holding a baby in their arms to what that baby will want when they grow up. She sees inspiration in the campaigns to abolish donor anonymity in the U.K. and parts of Australia, and she hopes to turn We Are Donor Conceived into a nonprofit, so that it might more forcefully advocate for changes.

Adams, in Australia, did eventually find his biological father, after multiple DNA tests and investigative work spanning years. (Though he got a donor number from medical records, the number didnt match anyone.) They look like spitting images of each other. Anybody whos seen the photos of the two of us can spot it instantaneously, he said. Theyve met in person and speak frequently on the phone. He finally found the connection he had been searching for, and now he wants others to find it too.

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Do We Have a Right to Know Our Biological Parents? - The Atlantic

A researcher at the OU College of Medicine has weighed in on a large genetics and COVID-19 study that was recently completed.

The study found 13 genes which could play a role in contracting the virus and how severe someone's battle with COVID-19 could be.

"Some people get COVID and nothing happens, said Dr. Dharambir Sanghera, Professor of Pediatric Genetics at the OU College of Medicine. "And they are asymptomatic. Some people, the disease gets so aggravated, and they get so severely sick they end up in hospitals and sometimes after hospitals people end up dying."

The answer as to why, Sanghera said, could lie in genetics.

"This is the first time we are seeing this association," Dr. Sanghera said.

Some of the genes found could slightly protect people from getting infected, such as having Type O blood, which has been predicted before.

However, 13 were found to predispose some more than others to more severe cases of covid-19, especially genes like obesity

"All the diabetes is also linked to severity of COVID," Dr. Sanghera said.

She said Oklahoma's poor health track record has played a role in the outcomes in the state and in the U.S.

"We are ranked second in chronic respiratory diseases and ranked four in cancer diabetes and obesity," Dr. Sanghera said.

Dr. Sanghera also said more genetic studies need to be completed to understand who is at the highest risk.

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OU College Of Medicine Researcher Sheds Light On Connection Between Genes, COVID-19 - News On 6

FARGO, N.D., August 30, 2021--(BUSINESS WIRE)--Agathos Biologics, a biotechnology company developing transformational science within a strong ethical and moral framework, today announced the company has been awarded $900,000 from the North Dakota Department of Agriculture Bioscience Innovation Grant (BIG) Program. Agriculture Commissioner Doug Goehring announced that nine grants have been awarded totaling $4.9 million to foster the growth of the bioscience industry in North Dakota. "Advances in bioscience have already transformed many sectors including agriculture and medicine," Goehring said. "These grants will help North Dakota stay on the forefront of bioscience innovation."

Agathos Biologics project funded by ND BIG will focus on challenges that limit patient access to advanced genetic medicines that can significantly impact quality of lifecost, availability, and ethical concerns. Company scientists will create new materials and methods for research and biomanufacturing and use them for drug development, which will address unmet medical needs and increase the availability of genetic medicines to more patients. The company will make these products and services available to the broader biotechnology industry through direct sales and licensing, partnerships, and collaborations.

"We are honored to receive this support from the State of North Dakota and thank the Commissioner and the Committee for their work on behalf of the citizens of the state," said James Brown, Chief Executive Officer of Agathos Biologics. "We founded the company in North Dakota because its business-friendly environment, skilled workforce, and growing biotechnology ecosystem make it an ideal place to expand the company and achieve our goal to develop genetic medicine products and services that positively impact human health and are ethically acceptable to all."

About Agathos Biologics

Agathos Biologics is a biotechnology company pursuing transformational science in biomanufacturing, biologic payload delivery, and cell and gene therapy. Discoveries in bioprocessing and genetic characterization and control have created an abundance of scientific possibilities that can help us all lead better lives. Our mission as the good science company is to create breakthrough products and services within a strong ethical and moral framework that benefits everyone. We believe in science that serves and have a relentless focus on serving our clients, employees, and society. For more information, please visit http://www.agathos.bio.

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Agathos Biologics Receives $900,000 from the North Dakota Bioscience Innovation Grant Program - Yahoo Finance

The treatment landscape for advanced ovarian cancer has evolved from where it was ten years ago, when at that stage it was very much a standard approach of surgery, chemotherapy, and then watch and wait to one where patients receive individualized care based on the unique features of their cancer, said Warner Huh, MD, FACOG, FACS, chair of the Department of Obstetrics and Gynecology at the University of Alabama at Birmingham School of Medicine. I dont think we have ever seen so many options to help improve clinical outcomes in the modern management of ovarian cancer than we have with the introduction of personalized medicine. To say its been transformative would be an understatement.

Identifying the Right Patients for Personalized MedicineThe introduction of PARP inhibitors for the treatment of certain women with advanced ovarian cancer is one recent example of how personalized medicine is transforming the way the disease is treated. Research has demonstrated that targeted treatment with PARP inhibitor-based regimens shows greater clinical benefit in women with homologous recombination deficient tumors as HRD is associated with an increased sensitivity to PARP inhibition. This group of patients accounts for approximately 50 percent of the advanced ovarian cancer population.

HRD is the functional impairment in a key DNA damage repair pathway that hinders a cell from fixing damaged DNA. Identifying HRD may suggest that a patient is a candidate for an appropriate PARP inhibitor-based regimen, which further blocks remaining DNA repair pathways causing the cancer cells to die. Given the relationship with HRD-positivity and PARP inhibitor regimens, HRD genomic instability testing is an important component of advanced ovarian cancer management.

Advanced ovarian cancer is a tough disease. When I see a patient is HRD-positive, I make sure she understands the results. I use it as an opportunity to share what it may mean for her care, said Dr. Huh. This conversation can also serve as a way to discuss the full treatment path ahead and set expectations for whats to come during different phases of her treatment journey.

Certain mutations that signal the presence of HRD, such as BRCA1/2 mutations, and other markers of genomic instability are identified through comprehensive biomarker testing.

In todays treatment landscape where cancer therapy is becoming increasingly personalized and biomarker-based, every woman diagnosed with advanced ovarian cancer should receive HRD genomic instability testing. Knowing that roughly half of our patients are HRD-positive, this testing can identify important information about her tumor which may better inform her treatment plan than testing for a BRCA mutation alone which is present in approximately just 25% of women with the disease, said Dr. Huh.

This puts into perspective the importance of HRD genomic instability testing. Without this test, we are not providing optimal information for informed decision-making about treatment, which is even more crucial given recent advances in medicine have brought us further than before as we aim to offer our patients hope.

However, given the various testing options across tumor types and the overall ongoing evolution in tumor-specific biomarker and genetic testing, gaps in knowledge about HRD genomic instability are complicating the testing landscape and hindering oncologists from consistently conducting the right tests immediately following diagnosis.

Elevating the Standard of Care by Addressing Gaps in KnowledgeAccording to a recent US survey 1 of 230 oncologists conducted by IntegraConnect and sponsored by AstraZeneca, many clinicians often underestimate the prevalence of HRD and the role it plays in treating cancer. The survey, which sought to better understand HRD genomic instability testing habits, barriers to testing and current knowledge gaps, showed that two-thirds of oncologists surveyed thought that fewer than 40 percent of women with advanced ovarian cancer are HRD-positive though the prevalence is much higher at 50 percent.2

The survey also revealed a common misunderstanding about the role of disease markers in ovarian cancer in this rapidly evolving space. Although three-quarters of respondents said that they were extremely or very familiar with HRD testing, 73% incorrectly said that homologous recombination repair (HRR) gene panels found in next-generation sequencing (NGS) tests help identify markers of genomic instability, when in fact, HRD genomic instability and BRCA are the only actionable biomarkers for use of PARP inhibition-based regimens known today.

HRR and HRD are both important features in DNA damage response, but the two terms are often confused. HRR is not a marker of genomic instability and is not a predictive biomarker for who may respond to PARP inhibition so an HRRm gene panel should not be used in place of an HRD test to help inform treatment decisions, said Dr. Huh.

Integrating HRD Genomic Instability Testing into the Full Treatment PlanNow, with a deeper understanding of the underlying biology of advanced ovarian cancer, oncologists have the tools to match the right treatment with the right patient.

Available tests for identifying HRD genomic instability in advanced ovarian cancer include: myChoice CDx from Myriad Genetics; FoundationOne CDx from Foundation Medicine; Caris Molecular Intelligence Comprehensive Genomic Profiling Plus (CGP+) from Caris Life Sciences; and Tempus xT from Tempus. However, 21% of oncologists surveyed did not select any of these HRD testing partners when presented with a list of 12 choices.3

These gaps demonstrate there is still more work to be done to ensure patients receive the right test as soon as possible following diagnosis to inform treatment decisions. One way to approach this is by adding a literal checklist to your clinical routine. In my practice, this checklist includes HRD genomic instability testing and germline testing at the earliest opportunity to ensure we are providing the optimal information to our patients. Simple things can make a big difference and the earlier you conduct HRD genomic instability testing, the better.

Were only at the tip of the iceberg in expanding the clinical utility of precision medicine. We can go even further to improve the treatment of advanced ovarian cancer particularly for the estimated one in two patients with HRD-positive tumors, said Dr. Huh. To sustain progress, the medical community needs to move beyond BRCA testing alone and conduct HRD genomic instability testing at the first opportunity. Without this information, physicians may not be able to optimize treatment decisions based on the patients individuals type of disease.

As we aim to work towards a cure for this devastating disease, we can enhance our care discussions by educating about comprehensive biomarker testing and ensuring that we order the right tests tests at the right time.

Learn more about incorporating HRD testing into clinical practice.

Dr. Warner Huh was compensated for his time associated with this article.

Original post:
Precision medicine in advanced ovarian cancer: Incorporating homologous recombination deficiency (HRD) genomic instability testing to transform care -...

In the United States, one in 9 males will get prostate most cancers and greater than 4 million males are at the moment dwelling with the illness.

Today, on the eve of Prostate Cancer Awareness Month, researchers from Johns Hopkins University and the University of Washington School of Medicine introduced theyre collaborating on a first-of-its-kind long-term observational examine to find out how genetic variations can have an effect on affected person outcomes. Dubbed PROMISE (Prostate Cancer Registry of Outcomes and Germline Mutations for Improved Survival and Treatment Effectiveness), this analysis will study how explicit genetic profiles can:

PROMISE researchers are looking for prostate most cancers sufferers nationwide, ages 18+ with particular inherited genetic elements utilizing a saliva DNA take a look at. PROMISE will entry every affected persons medical info by way of their doctor each six months. PROMISE will even survey taking part sufferers each six months about their remedy expertise.

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While genetic info has contributed to advances in remedy of illnesses resembling breast most cancers, it has not been extensively utilized in prostate most cancers. Researchers anticipate that the PROMISE examine will result in necessary new discoveries, new analysis and new remedy therapies.

If we want to better understand prostate cancer, we have to better understand genes, defined Dr. Heather Cheng, PhD, affiliate professor of medical oncology at the University of Washington School of Medicine; director of the Prostate Cancer Genetics Clinic at Seattle Cancer Care Alliance; college member at Fred Hutchinson Cancer Research Center; and co- lead investigator of PROMISE. This information is the next step in our collective fight against the disease.

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Dr. Cheng is working intently with Dr. Channing Paller, affiliate professor of oncology and urology at Johns Hopkins University, Sidney Kimmel Comprehensive Cancer Center; affiliate director for oncology of the Johns Hopkins Clinical Research Network; and co-lead investigator of PROMISE. They know that prostate most cancers could also be written in some maless genes, however so are directions for locating new therapies and understanding household danger. Together, Dr. Cheng and Dr. Paller will convey extra genetic info to the battle towards prostate most cancers.

How PROMISE works

PROMISE is totally free. Participants proceed with their present healthcare supplier and dont want to go away residence to take part. Registration will be executed on-line and PROMISE will ship a easy, home-based DNA take a look at equipment. Participants present a saliva pattern and return it by way of pre-paid U.S. mail. The equipment will display screen for 30 most cancers danger genes. PROMISE will ship the outcomes and will present a licensed genetic counselor to assist individuals perceive their outcomes. Participants will be taught if theyve any gene mutations that may have an effect on their care plan. The outcomes could inform sufferers of obtainable remedy choices and beforehand unknown dangers of members of the family growing most cancers.

Benefits of becoming a member of the PROMISE examine.

Prostate most cancers sufferers who be a part of PROMISE will:

PROMISE is supported by a gaggle of mission-driven nonprofit organizations. Study administration is offered by The Prostate Cancer Clinical Trials Consortium (PCCTC). No pharmaceutical corporations or any business pursuits are supporting this analysis. To be taught extra, go to prostatecancerPROMISE.org.

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First-of-its-Kind Study Looks at Link Between Genes and Prostate - News Chant USA

Gene therapy is a promising new method for treating non-small cell lung cancer (NSCLC). It allows doctors to target specific genes to prevent cancerous cells from growing and spreading.

NSCLC is a common form of cancer. It causes cancerous cells to form in the tissues of the lung. NSCLC is a serious condition. However, many people receive treatment and survive for years.

Treating NSCLC typically involves interacting with multiple specialists and receiving a combination of therapies. Specific treatment plans depend on factors that include the tumor size, type of NSCLC, and the extent of its spread to other organs.

Surgery, radiation or chemotherapy, and immunotherapy are examples of key techniques that doctors use to treat NSCLC.

Gene therapy is another promising treatment for NSCLC, which targets genes that contribute to the tumor.

There are two main approaches to using gene therapy to treat cancer:

This article focuses on the second approach to NSCLC gene therapy. Read on to learn more.

Getting genes into cells requires making vectors, which are vehicles that scientists engineer to deliver genetic materials. For example, viruses have a natural ability to deliver genetic material into cells and can act as vectors.

Scientists can deactivate parts of the virus that cause infectious diseases. They can then modify the virus to carry genetic material into cancerous cells.

One type of gene therapy for NSCLC targets tumor-suppressor genes, which are the most common gene mutation that contributes to the disease. Another approach involves restoring specific proteins to prevent disease progression.

Other possible applications include inserting genes that:

NSCLC gene therapy is a new form of treatment. However, early results are promising.

A 2017 review suggests that restoring a functional tumor-suppressing gene could slow the growth of cancer cells. Clinical trials have found that inserting tumor-suppressing genes into people who had not responded to other treatments reduced tumor size by up to 50%.

Another review in 2016 suggests that the treatment is more effective when combining NSCLC gene therapy with other therapies, such as chemotherapy or immunotherapy.

According to the American Cancer Society, doctors typically use gene therapy for advanced cancer cases.

NSCLC gene therapy is a new technique. However, it still has to meet rigorous Food and Drug Administration (FDA) standards for safety and effectiveness before a doctor can recommend it.

Gene therapies that the FDA approves are safe. However, they can have side effects, such as:

According to the FDA, gene therapies can transform medicine and provide options for people with illnesses that were previously without a cure. However, every treatment has limitations to its effectiveness.

Some limitations to gene therapy include:

Doctors will typically develop a treatment plan with people who have NSCLC depending on their health, age, and other relevant factors. Some common forms of NSCLC treatment include:

Doctors may combine these treatments to maximize their effectiveness. This will involve undergoing multiple treatments at once or back-to-back treatments, or both.

For example, doctors may use a therapy to treat cancer in one part of the body and another therapy to treat where it is spreading.

Doctors typically describe the outlook for people with cancer using the percentage of people alive at least 5 years after their diagnosis. This is the 5-year survival rate. They may further break down 5-year survival rates according to specific NSCLC diagnoses.

According to the American Cancer Society, the 5-year survival rate for people with NSCLC are:

NSCLC is a common form of lung cancer in the United States. Gene therapy for people with NSCLC is a promising new treatment that targets specific genes that contribute to disease progression. There is evidence that gene therapy can slow the growth of tumors in people with NSCLC.

Gene therapy is new, but has the potential to change the way doctors can treat cancer. Scientists and doctors must first overcome limitations, including finding reliable methods to deliver gene therapy.

Read more:
NSCLC gene therapy: Success rate, other options, and more - Medical News Today

PALO ALTO, Calif. & SHANGHAI & PRINCETON, N.J.--(BUSINESS WIRE)--LianBio, a biotechnology company dedicated to bringing paradigm-shifting medicines to patients in China and other major Asian markets, and BridgeBio Pharma, Inc. (Nasdaq: BBIO) today announced the first patient has been treated in a Phase 2a clinical trial of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with fibroblast growth factor receptor-2 (FGFR2) gene amplification and other advanced solid tumors with FGFR genomic alterations.

Infigratinib is a potent and selective FGFR inhibitor that has demonstrated compelling clinical activity across multiple tumor types with FGFR alterations, said Yizhe Wang, Ph.D., chief executive officer of LianBio. Given the disproportionately high prevalence rate of gastric cancer in China, LianBio is pursuing a region-specific development strategy focused on this area of great unmet need. This study marks LianBios first trial initiation and demonstrates our continued progress in delivering potentially transformational medicines to patients in Asia.

TRUSELTIQ (infigratinib) is an oral selective inhibitor of FGFR1-3 that is approved in the United States for the treatment of patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangement as detected by an FDA-approved test. It is also being further evaluated in clinical trials based on demonstration of clinical activity in patients with advanced urothelial carcinoma with FGFR3 genomic alterations. LianBio in-licensed rights from BridgeBio for infigratinib for development and commercialization in Mainland China, Hong Kong and Macau.

The Phase 2a trial is a multicenter, open-label, single-arm study in China designed to evaluate the safety and efficacy of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with FGFR2 gene amplification and other advanced solid tumors with FGFR alterations. The primary endpoint is objective response rate (ORR). Secondary endpoints include duration of response, safety, disease control rate, progression-free survival and overall survival.

Preclinical data have demonstrated the potential infigratinib may have for patients with gastric cancer. These results, published in Cancer Discovery, demonstrated tumor regression in multiple in vivo FGFR2 amplified gastric models.1

We believe that infigratinib could have a meaningful impact for people living with gastric cancer as well as many other cancers with FGFR alterations, and are pleased LianBio is initiating this clinical trial in China where more therapeutic options are needed to match the growing diagnosis rate, said BridgeBio founder and chief executive officer Neil Kumar, Ph.D. On the heels of TRUSELTIQ recently obtaining accelerated approval in the United States, we are hopeful that this trial will yield pivotal results in another subset of cancer patients as we continue to build our portfolio of oncology indications with the aim of reaching as many people in need as possible.

About TRUSELTIQ (infigratinib)

TRUSELTIQ (infigratinib) is an orally administered, ATP-competitive, tyrosine kinase inhibitor of fibroblast growth factor receptor (FGFR) that received accelerated approval from the FDA in the United States for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. TRUSELTIQ targets the FGFR protein, blocking downstream activity. In clinical studies, TRUSELTIQ demonstrated a clinically meaningful rate of tumor shrinkage (overall response rate) and duration of response. TRUSELTIQ is not FDA-approved for any other indication in the United States and is not approved for use by any other health authority, including any Chinese or other Asian health authority. It is currently being evaluated in clinical studies for first-line cholangiocarcinoma, urothelial carcinoma (bladder cancer), locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma, and other advanced solid tumors with FGFR genomic alterations.

About BridgeBio Pharma, Inc.

BridgeBio is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBios pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the companys first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com.

About LianBio

LianBios mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and other major Asian markets, through partnerships that provide access to innovative therapeutic discoveries with a strong scientific basis and compelling clinical data. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a broad and clinically validated pipeline with the potential to impact patients with unmet medical needs. For more information, please visit http://www.lianbio.com.

About the LianBio and BridgeBio Pharma, Inc. Strategic Alliance

In August 2020, LianBio entered into a strategic alliance with BridgeBio, a commercial-stage biopharmaceutical company focused on genetic diseases and cancers with clear genetic drivers, to develop and commercialize BridgeBios programs in China and other major Asian markets. This strategic relationship initially focuses on two of BridgeBios targeted oncology drug candidates: FGFR inhibitor infigratinib, for the treatment of FGFR-driven tumors, and SHP2 inhibitor BBP-398, in development for tumors driven by MAPK pathway mutations. The agreement also provides LianBio with preferential future access in China and certain other major Asian markets to more than 20 drug development candidates currently owned or controlled by BridgeBio. This collaboration is designed to advance and accelerate BridgeBios programs in China and other major Asian markets, allowing BridgeBio and LianBio to potentially bring innovation to large numbers of patients with high unmet need.

BridgeBio Pharma, Inc. Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as anticipates, believes, estimates, expects, intends, may, plans, projects, seeks, should, will, and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act, and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to: the timing and success of the Phase 2a clinical trial of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with fibroblast growth factor receptor-2 (FGFR2) gene amplification, and other advanced solid tumors with FGFR genomic alterations; the planned approval of infigratinib by foreign regulatory authorities in China and the necessary clinical trial results, and timing and completion of regulatory submissions related thereto; and the competitive environment and clinical and therapeutic potential of infigratinib; reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation: the safety, tolerability and efficacy profile of infigratinib observed to date may change adversely in ex-U.S. clinical trials, ongoing analyses of trial data or subsequent to commercialization; foreign regulatory agencies may not agree with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; the continuing success of the BridgeBio and LianBio strategic alliance; and potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; as well as those set forth in the Risk Factors section of BridgeBio Pharma, Inc.s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent SEC filings, which are available on the SECs website at http://www.sec.gov. Except as required by law, each of BridgeBio and QED disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. Moreover, BridgeBio and QED operate in a very competitive environment in which new risks emerge from time to time. These forward-looking statements are based on each of BridgeBios and QEDs current expectations, and speak only as of the date hereof.

1 Guagnano, V., Kauffman, A., Wrle, S., et al. FGFR Genetic Alterations Predict for Sensitivity to NVP-BGJ398, a Selective Pan-FGFR Inhibitor. Cancer Discovery 2 (2012): 1118-1133.

Read more:
BridgeBio Pharma and LianBio Announce First Patient Treated in Phase 2a Trial of Infigratinib in Patients with Gastric Cancer and Other Advanced Solid...

The height of a childs biological parents can be a good indicator of how tall a child will be, as genetics play a prominent role in determining height. However, this can vary, and siblings with the same parents may all reach different heights. Other factors, such as biological sex, overall health, nutrition, sleep, and exercise, during developmental years all factor into height and growth.

The height a person reaches by adulthood can depend on the genes they inherit from their biological parents, although some factors may mean a child does not reach their full potential height.

Nutrition and overall health during childhood and adolescence also affect human growth and height. Over hundreds of years, the average human height has increased due to improved nourishment in children and a reduction in illness and infections.

This article explores methods people may use to predict height, factors that affect growth in children and adolescents, and when to speak with a doctor if growth becomes a concern.

A combination of genetics and external factors can affect how tall a child will grow.

Health experts believe that 80% of a persons height is genetic. This means the height of biological parents can be an indicator of a childs height, although this is not always a reliable predictor.

Siblings with the same parents can vary in height, and one child in the family may be taller or shorter compared to the rest of the family.

Other factors, such as nutrition, illness, or premature birth, can also play a part in height and growth and may prevent a child from reaching their full potential height.

Learn more about which factors can influence a persons height.

According to Johns Hopkins Medicine, people may wish to try the following formula for predicting how tall a child will be:

However, note that predictive methods such as these are not concrete, and a childs adult height could change depending on different factors.

Learn how to measure height accurately.

According to the Society for Endocrinology, people can usually expect the following average growth patterns in children and adolescents:

Learn about the signs and stages of puberty.

Most females will have a growth spurt in the year before they start their first menstrual period. Their feet and hands will likely increase in size first, followed by the rest of the body.

Female growth slows down after their first menstrual period, but females will usually still grow 12 in after this time.

According to health experts, males usually have a growth spurt in puberty 2 years after most females. The peak time of growth is before sperm develops, and males will grow about 9 cm a year. Males also usually have longer growth spurts than females.

According to the Society for Endocrinology, there is no set age for when males and females will stop growing. Once a person has gone through all the stages of puberty to reach adult development, their growth will slow down and stop.

Additionally, growth plates in the bones fuse together at this stage, meaning individuals will not grow any taller.

Learn about when male children might stop growing.

Learn about when female children might stop growing.

Adolescents go through puberty at different stages, so variations in growth spurts can be normal. In some cases, going through puberty at a slower rate can be due to an inherited pattern, known as constitutional delay.

If there is too much variation, such as a growth spurt not occurring or females not menstruating by the age of 16 years, then it is important to speak with a doctor for a checkup.

In some cases, unusual growth or development in a young person may be the result of an underlying medical condition, such as:

Treating the underlying condition may help improve growth. In the case of a growth hormone deficiency, people may need treatment with artificial growth hormone.

Although genetics largely determine a persons height, proper nutrition is an important factor in healthy growth and development for children and adolescents.

According to the Academy of Nutrition and Dietetics, a balanced and nutritious diet for healthy growth and development includes:

According to a 2018 study, both exercise and good sleep can help increase height, as they elevate the release of growth hormones.

Sometimes, children or adolescents may face peer pressure or bullying for being a different height than those around them. This can lead to feelings of inadequacy, anxiety, or depression.

The American Academy of Child and Adolescent Psychiatry provides the following advice for parents and caregivers to help young people deal with peer pressure:

Learn more about teen anxiety and depression.

How tall a child or teenager will grow largely depends on the genes they inherit from their biological parents.

Good nutrition, exercise, and sleep also all play an important role in healthy growth and development. Illness, infection, or premature birth may all affect whether a child reaches their potential full height or not.

In most cases, young people will not be able to have much impact on their height other than living a healthy and balanced lifestyle. In some cases, children or adolescents may have a deficiency in growth hormone, which may require medical treatment to resolve.

If a child or adolescent is not growing or developing as expected for their age and biological sex, misses a growth spurt, or females do not have their first period by 16 years, then individuals can consult with their doctor for a checkup.

Excerpt from:
How tall will I be? A guide for parents and children - Medical News Today

Precision NanoSystems, Inc. 50 655 West Kent Ave North Vancouver, BC Canada V6P 6T7 1-888-618-0031

VANCOUVER, BC, Aug. 11, 2021 /PRNewswire/ - Precision NanoSystems (PNI), a global leader in innovative solutions for genetic medicine development, will expand its global headquarters with a 75,000-square-foot facility currently under construction. Slated for completion in Q4 2022, the new site will be located at 1055 Vernon Drive, in the Evolution Block building, in the False Creek Flats area of Vancouver. PNI's new genetic medicine GMP Biomanufacturing Centre will also be housed at the site.

PNI intends to expand capabilities to include the clinical manufacturing of RNA vaccines and therapeutics.

PNI's rapidly expanding team of more than 180 life science professionals will work from the site. James Taylor, General Manager, Precision NanoSystems, says: "This is a huge leap forward in our mission to accelerate the creation of transformative medicines that significantly impact human well-being. We are proud to contribute to the Canadian biotechnology industry by enabling them to develop and manufacture the next generation of medicines."

The Biomanufacturing Centre will support PNI's client base of leading drug developers through clinical development, as well as Canada's efforts for future pandemic preparedness through the manufacture of RNA vaccines.

PNI continues to rapidly expand its clinical manufacturing organization. The Biomanufacturing Centre is led by Elaine Copsey, VP of Biomanufacturing Operations, and Lloyd Jeffs, Senior Director of Biomanufacturing Services. Hiring is ongoing to find scientists, engineers, and other skilled professionals.

Elaine Copsey says: "PNI is excited to build on our successful preclinical services to include clinical and GMP production. We are growing our expertise as one of the industry leaders and will be hiring professionals with expertise in GMP operations including RNA and lipid nanoparticles manufacturing, process development, analytical testing, quality control, sterility, and quality assurance. PNI is looking forward to working with our clients to support all stages of their drug development process resulting in expedited therapeutic batches for clinical trials and routine production."

The Biomanufacturing Centre has been partially funded with CAD 25.1 million dollars from Canada's Strategic Innovation Fund (SIF).

The Honourable Franois-Philippe Champagne, Minister of Innovation, Science and Industry, says: "We are continuing to safely restart our economyby investing in companies like PNI that will help create well-paying jobs and ensure Canada is more prepared for future pandemics. By accelerating the creation of drugs and therapeutics, PNI will contribute to the strengthening of Canada's biomanufacturing sector and to building a stronger and healthier country for all of us."

PC Urban Properties is overseeing the construction of the new site. Brent Sawchyn, CEO, PC Urban Properties, says: "PNI's new lease solidifies the False Creek Flats area of Vancouver as a growing biotech and health science hub. We're excited to welcome PNI toEvolution Block, a pioneering stacked industrial development that will help transform one of the city's oldest industrial neighborhoods into its reimagined future."

About PNIPNI is a global leader in ushering in the next wave of genetic medicines in infectious diseases, cancer and rare diseases. We work with the world's leading drug developers to understand disease and create the therapeutics and vaccines that will define the future of medicine. PNI offers proprietary technology platforms and comprehensive expertise to enable researchers to translate disease biology insights into non-viral genetic medicines.

SOURCE Precision Nanosystems

http://www.precisionnanosystems.com

Read more from the original source:
Precision NanoSystems sets time and place to open new Genetic Medicine GMP Biomanufacturing Centre in Vancouver, BC - PRNewswire

Should doctors take particular care to talk to patients about the potential dangers of COVID-19 if those patients have a problematic relationship with pot?

New research from Washington University in St. Louis suggests perhaps they should.

Diabetes, obesity and a history of smoking cigarettes are all considered risk factors for poorer COVID-19 outcomes. Warnings and tailored information are targeted to people with these conditions, and doctors are acutely aware of the elevated risks they pose.

Findings from the lab of Ryan Bogdan, associate professor in the Department of Psychological & Brain Sciences in Arts & Sciences, suggests cannabis use disorder (CUD) should be added to the list because the genetic predisposition to CUD is overrepresented in people with poor COVID-19 outcomes. More work is needed to determine if there is direct causation.

The research is in press in the journal Biological Psychiatry: Global Open Science.

As sociocultural attitudes and laws surrounding cannabis use become increasingly permissive, and COVID-19 continues to spread, we need to better understand how cannabis use as well as heavy and problematic forms of use are associated with COVID outcomes, Bogdan said.

First author Alexander S. Hatoum, a postdoctoral researcher in the Washington University School of Medicine, used genetic epidemiological models to determine that genetic predisposition to CUD is related to risk for a severe reaction to COVID-19 (i.e., being hospitalized with COVID-19).

Hatoum combined existing datasets to test whether being at higher genetic risk for cannabis use disorder was correlated to the risk of COVID hospitalization.One set of data involved 357,806 people, including 14,080 with CUD; the other involved 1,206,629 people, including 9,373 who were hospitalized with COVID. He also looked at 7 million genetic variants to assess the association between CUD and severe COVID.

Having genetic variants does not mean a person has CUD or that the person has used cannabis.

In comparing people with the variants to their COVID outcomes, the researchers found genetic liability for CUD accounted for up to 40% of genetically influenced risk factors, such as body mass index (BMI) and diabetes, for a severe COVID-19 presentation.This association suggested that heavy and problematic cannabis use may represent a modifiable pathway to minimize severe COVID-19 presentations.

The results of this study point to two possible outcomes: That a predisposition to CUD and severe COVID-19 are due to a common biological mechanism, like inflammatory conditions causing individuals to develop worse symptoms of COVID-19 and/or dependence on cannabis; or that they are associated because of a causal process.

If we know the genes that predispose individuals to cannabis use disorder, and if cannabis use disorder is a risk factor for COVID-19 hospitalization, you will see the genes influencing cannabis use disorder as predictors of severe COVID-19 cases, Hatoum said. We found that a persons genetic risk for cannabis use disorder is correlated with their risk for COVID-19, without having to ask directly about illegal substance use.

The genetic association between CUD and COVID-19 severity was similar in size to genetic correlations between COVID-19 severity and BMI, a well-known risk factor for severe COVID-19 presentations. Moreover, it was present even when accounting for genetic liability to BMI as well as other risk factors for a severe reaction to COVID-19, including metabolic traits (e.g., fasting glucose, hypertension); respiration traits (e.g., forced expiratory volume, COPD); socioeconomic status; alcohol and tobacco use; and indices of impulsivity.

That the genetic relationship between CUD and COVID-19 is independent of these factors raises the intriguing possibility that heavy and problematic cannabis use may contribute to severe COVID-19 presentations. As such, it is possible that combating heavy and problematic cannabis use may help mitigate the impact of COVID-19, Hatoum said.

This information needs to be incorporated into any strategy to defeat this disease, Hatoum said.

These data suggest that heavy cannabis users may have a more adverse reaction to COVID-19 and that, much like quitting tobacco smoking or reducing BMI, reducing and/or stopping heavy cannabis use may protect against severe COVID-19 reactions.

Julia Strait contributed to this story.

See more here:
Cannabis use disorder: another COVID risk factor - Washington University in St. Louis Newsroom