Stem Cell Clinic

Navigating the Clinical Trial Maze – Curetoday.com

Cutting-edge treatment is not out of reach, thanks to a little guidance from the Cancer Research Institute.

Currently, 4,000 active clinical trials involve immunotherapy drugs, which are examining T cell targets, cell therapy, vaccines and other immunomodulators. CRI works to help fund and advance this research.

Founded in 1953 by Helen Coley Nauts, the daughterof Dr. William B. Coley more commonly known as the Father of Cancer Immunotherapy and her friend Oliver R. Grace Sr., CRI has conducted research in this space for 65 years. Immunotherapy treats cancer by using the bodys immune system to prevent, target, control and eliminate the disease.

Decades of research have led to breakthrough treatments that mobilize our own immune systems to hunt down and eliminate cancer cells, wherever they are in the body, Jill ODonnell-Tormey, who holds a doctorate in philosophy, cell biology and anatomy, and serves as CEO and director of scientific affairs at CRI, said in an interview with CURE. Its a more sophisticated, dynamic and durable approach than other cancer treatments and may prove to be the best way to keep pace with cancers that evolve over time. Immunotherapies have also been proven to synergize with other forms of cancer treatment, increasing response rates while minimizing negative side effects and reducing risk of recurrence in some cancers.

The Food and Drug Administration has approved seven checkpoint inhibitors, a specific type of immunotherapy, to treat different cancers: Bavencio (avelumab), Imfinzi (durvalumab), Keytruda (pembrolizumab), Libtayo (cemiplimab), Opdivo (nivolumab), Tecentriq (atezoli- zumab) and Yervoy (ipilimumab).

A lot of research must still be done, according to ODonnell-Tormey. She encourages patients and their caregivers to be open to clinical trials, which give patients an opportunity to receive cutting-edge treatments under the watchful care of an expert team.

Many patients who participate in clinical trials describe the experience as very positive, often feeling part of the research team while cognizant that they are contributing to science and potentially helping future patients with cancer, she said. They can expect to receive lots of information about the study and the rationale supporting it, along with around-the-clock access to a clinical team that can answer any questions patients might have. While the treatments being tested are investigational, its important for patients to understand that new drugs or drug combinations undergo extensive laboratory testing before they are given to humans.

Often, the most challenging part of the process is finding a clinical trial. Not all health care providers discuss these studies with patients, and navigating some websitescan be difficult. CRI offers a Clinical Trial Finder to help patients learn about the basics and what to consider before enrolling, as well as connect with navigators who will walk them through the process.

Patients are asked to fill out a disease-specific questionnaire. The navigator needs to know the primary diagnosis, including tumor type; stage or extent of disease, such as if it has spread; and any treatment history. Then the navigator searches a national database of immunotherapy trials to find the best match for the patient.

Beyond locating trials and connecting to trial sites, our navigation is really customized education that varies by patient, said Caroline Melendez, director of client services at EmergingMed, the company that runs CRIs Clinical Trial Finder. Some people are very familiar with their diagnosis and treatment history, but those who have very little information are offered guidance about what to ask their health care team. Once a patient has identified trial matches, the navigator can help explain the differences between the types of treatment modalities and phases of trials being offered in their match results.

Enrollment time varies by trial and site, and patientsgo through an informed consent process, Melendez said. Next, they learn about the specific trial and undergo an examination to determine eligibility. The process can take several days, a few weeks or longer, she said.

We encourage every patient to ask this question: Is there an interesting clinical trial available to me today? Melendez said. The decision to enroll in a clinical trial is entirely dependent upon whether a compelling new therapy is in development. Everyones situation is unique, and patient interest in clinical trials should be informed by discussions with their medical team each time they have to make a treatment decision.

Several myths surround clinical trials, such as that they are a last resort or that patients are treated like guinea pigs. Another common misconception is that being on a trial means theres a chance youll receive placebo rather than treatment, ODonnell-Tormey said. This is not true except in cases where there are no existing treatments proven to improve patient outcomes. Generally, patients will receive some form of therapy, whether its standard of care, a new drug or a combination of the two.

Patients should investigate all options, educate them- selves and then make treatment decisions. Trials open and close all the time, but generally speaking, there are trialsfor patients at almost every phase of the journey, newly diagnosed to relapsed, Melendez said. Its never too early to get educated and connected to a clinical trial navigator, but it can be too late.

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Navigating the Clinical Trial Maze - Curetoday.com

The nanoengineer working to mend broken hearts – UNSW Newsroom

When Hossein Tavassoli first told his mother that he was researching heart disease, she responded with But, youre an engineer...

She was right, of course Hosseins undergraduate degree was in material engineering but at the time, he found it difficult to explain the intersections between biology and engineering. Now, not only is he a nanoengineer working in a tissue regeneration lab, but he is also an avid science communicator.

Its very interdisciplinary, says Hossein, who is completing the biology component of his PhD at UNSW Medicine and the engineering component at Swinburne University of Technology.

Hossein began working with the heart several years ago after finding himself interested in the heart-blood development and regeneration research of UNSW biologists Dr Vashe Chandrakanthan and Professor John Pimanda. Fortunately, they were intrigued by the technology Hossein was working on, and a collaboration began.

The heart is the most important organ, with heart disease the number one killer in the world, Hossein says.

My goal is to find a way for mending broken hearts or, in other words, to find a solution for cardiac problems.

During their first seven days of life, baby mice can self-repair broken heart tissue.

They develop this ability while in the embryo, which although impossible for scientists to view under a microscope could hold the clue to repairing heart damage in humans.

We cannot put a mouse embryo under the microscope, but what I do is model what is going on in the embryonic heart in a microchip, at a microscale.

The microchip that Hossein nanoengineered has been embedded with mice heart cells. It was built to mimic the embryonic heart cells natural environment, enabling the researchers to study how the regenerative cells communicate with each other. Knowledge of how this works could be applied to repairing human heart tissue, although Hossein speculates this technology is at least a decade away. In order to expand their research, additional project funding is needed.

Hossein sees many benefits to using nanotechnology in biomedicine.

First of all, its cheap. You dont need to have lots of plastic, petri dishes, culture media all those things we need for the cell culture. Secondly, you dont need too many animals or cells or patients. Instead of 10-100 million cells, what I need in that tiny family is like 100 or 1000 cells.

Or, in a simple way, instead of needing a hundred mice for each series of experiments, I just need one or two.

Hossein observes the cells interacting via a microscope connected to a supercomputer. Visually, it looks like a microchip. It has channels, it has valves it is like a simplified model of a heart.

When the cells interact in just the right way, it starts to pulse just like a heartbeat.

Hossein is lost for words when remembering the first time he saw the cells start beating.

His work, he explains, differs from many other PhD students. Instead of working from 9-5, he needs to visit the lab at all hours sometimes as late as midnight to feed the cells. One particular lab visit stands out to him.

I came to the lab on Friday to add culture media, which is food for the cells. The day after, I was at Coogee Beach with some friends and said, I should go, I need to check on my cells.

His friends were confused, but it was normal routine for Hossein. It was then he realised that the cells were effectively his babies.

Only when he arrived, they werent beating.

I thought, Oh no, something is wrong! He quickly added the culture media, put them in the incubator, and waited.

A few hours later, they started beating again.

It was incredible, Hossein remembers.

For Hossein, when the cells are beating, they are a symbol of hope and life.

"Everything in our world starts with that beating. And everything finishes with that beating. When that line becomes straight I've seen this in my life, with my family you see that everything is gone. As long as there is beating, there is hope."

Everything in our world starts with that beating. And everything finishes with that beating. When that line becomes straight I've seen this in my life, with my family you see that everything is gone.

As long as there is beating, there is hope.

Hossein knew that his family and friends were proud of his achievements but was frustrated at his inability to properly explain his research to them.

He remembers the specific moment when that changed.

Hossein was taking an Uber to university one night (to feed the cells, he recalls) when the driver asked what he studied. Knowing that the answer was convoluted, Hossein kept his response simple: Im an engineer, he said.

An engineer? the driver responded. Then why am I dropping you off at the medicine building?

Hossein then had a realisation: if he could explain his work to an Uber driver, then perhaps he could also explain it to his mum.

From that moment, he tried to articulate his research to any driver who asked. He tested new ways of describing his work and kept the ones that worked best.

Soon, something surprising happened Hossein developed a love for science communication.

I realised that it is important to explain what I'm doing, but in simple words, so that anyone can understand.

The practice paid off. Earlier this year, Hossein was a finalist in FameLab Australia (a live annual science communication competition), a shortlisted speaker at TEDx Melbournes Open Mic, and last year was a finalist in AMP Amplify Ignite (a national PhD pitch competition). He now shares his research via Twitter.

Science communication is an art, he says. For me, informing and educating is more important than doing just research all the time.

And, he adds, I now get five-star ratings on Uber!

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The nanoengineer working to mend broken hearts - UNSW Newsroom

OncLive Presents State of the Science Summit on Non-Small Cell Lung Cancer – Business Wire

CRANBURY, N.J.--(BUSINESS WIRE)--OncLive, the nations leading multimedia resource focused on providing oncology professionals with the most current and insightful information they need to offer the best patient care, will host its latest State of the Science Summit on Non-Small Cell Lung Cancer (NSCLC) on Thursday, Oct. 3, from 5-9 p.m. at the DoubleTree by Hilton Hotel Cleveland, in Ohio. The chair for the summit will be Nathan Pennell, M.D., Ph.D., director of the lung cancer medical oncology program and assistant professor of medicine at Cleveland Clinic.

This interactive and educational meeting will analyze and discuss novel treatments for patients with NSCLC. The expert presenters will explore a wide variety of informative topics surrounding NSCLC, such as updates in immunotherapy in stage 4 squamous NSCLC, epidermal growth factor receptor-positive NSCLC, screening for NSCLC, anaplastic lymphoma kinase-positive and ROS1 NSCLC, targetable biomarkers in NSCLC, new standard of care for stage III NSCLC, new immunotherapy combinations in stage 4 nonsquamous NSCLC and updates in the management of small-cell lung cancer. The presenters will also engage in a peer exchange and address audience questions.

The presenters for the summit include the following:

State of the Science Summit is a premier conference series hosted by OncLive that features medical experts from across the nation discussing treatment options. Each summit integrates academic and community-based physicians and health care professionals across key disciplines, from medical and surgical oncology to hematology.

Registration is free and open to all health care professionals, and food and beverages will be served. For more information and to register, visit https://www.onclive.com/meetings/soss or contact Kayla Collins at kcollins@onclive.com.

About OncLive

A digital platform of resources for practicing oncologists, OncLive offers oncology professionals information they can use to help provide the best patient care. OncLive is a brand of MJH Life Sciences, the largest privately held, independent, full-service medical media company in the U.S. dedicated to delivering trusted health care news across multiple channels.

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OncLive Presents State of the Science Summit on Non-Small Cell Lung Cancer - Business Wire

Greenleaf Health Expands Services to Support Cell & Gene Therapy – Business Wire

WASHINGTON--(BUSINESS WIRE)--Greenleaf Health, Inc., a leading Food and Drug Administration (FDA) regulatory consulting firm, today announces that the firm has expanded its portfolio of services to guide companies developing cell and gene therapy products. Greenleafs team of regulatory experts will be led by Karen Midthun, M.D., former Director of the FDAs Center for Biologics Evaluation and Research (CBER), and John Taylor, former FDA Counselor to the Commissioner and Principal Deputy Commissioner.

REGULATORY LANDSCAPE: Cell & Gene Therapy

The rapidly evolving fields of cell and gene therapy offer the possibility of novel treatments, and perhaps ultimately cures, for devastating and intractable illnesses. In response to what the FDA has called a "turning point in the development of these technologies and their application to human health, new policies have been introduced to address the development of safe and effective cell and gene therapies.

With innovation often comes uncertainty. In the case of cell and gene therapy products, the FDA has raised concerns about developers operating outside of the existing regulatory paradigm. To prevent this, the FDA has clarified the regulatory framework for regenerative medicine products and announced near-term enforcement actions aimed at ensuring compliance by companies developing and manufacturing cell and gene therapies.

Greenleafs expanded services support companies striving to introduce cell and gene therapy products to patients. The firms team of experts has a robust blend of technical skill and FDA institutional knowledge that spans all therapeutic areas and quality, manufacturing, and compliance systems. By working cross-functionally, Greenleaf ensures that clients have the comprehensive, specialized support needed to understand and navigate the complex regulatory landscape for cell and gene therapies.

FULL-SERVICE SUPPORT

Members of Greenleafs Drug and Biological Products Team work together with the firms Product Quality, Manufacturing, and Compliance Team to deliver guidance on cell and gene therapy products.

Product Development & Review

With the expert direction of Karen Midthun, M.D., Greenleafs team of advisors assists sponsors of cell and gene therapies by optimizing FDA interactions and submissions to support development and regulatory review. Greenleaf also helps sponsors understand and respond to the FDA requirements applicable to various cellular products, and provides guidance to sponsors of cell and gene therapies to treat rare and ultra-rare diseases on ways to maximize trial design using appropriate clinical endpoints and natural history study data to aid efficient product development.

Quality, Manufacturing & Compliance

Greenleafs Product Quality, Manufacturing, and Compliance Team, led by John Taylor and supported by the firms network of independent compliance experts, offers credible, informed guidance to help manufacturers of cell and gene therapies comply with the FDAs multiple current GXP regulations. Greenleaf experts provide strategic and technical support for establishing manufacturing and quality controls; pre- and postapproval inspection readiness; compliance assessments; evaluating and responding to FDA regulatory correspondence; and engaging with CBERs Advanced Technologies Team.

UNMATCHED EXPERTISE

Greenleaf is comprised of experts with a combined total of more than 250 years of FDA experience. The firms team of advisors demonstrates unmatched levels of skill in its specialties of drug and biological products and product quality, manufacturing, and compliance. Greenleafs Cell and Gene Therapy Team, led by Dr. Karen Midthun and John Taylor, is guided by decades of regulatory experience in senior FDA positions, global public health organizations, academia, and industry.

Karen Midthun, M.D.Principal, Drug & Biological Products

Dr. Midthun contributes specialized insight informed by her regulatory, research, and clinical experience to FDA-regulated entities developing cell and gene therapies. Dr. Midthun joined Greenleaf following a distinguished 28-year career in public health, of which 22 years were dedicated to the FDA.

An infectious disease physician by training, Dr. Midthun most recently served as the Director of the FDAs Center for Biologics Evaluation and Research (CBER). During her FDA tenure, Dr. Midthun played a critical role in facilitating policy and technology development in the areas of cell, tissue, and gene therapies, blood products, and vaccines.

John Taylor, J.D.President, Greenleaf Health, and Principal, Compliance & Regulatory Affairs

Taylor has held many high-profile positions at the FDA, as well as senior leadership roles within industry. Taylors wealth of regulatory experience, robust technical skills, and unique strategic perspective are unmatched. Clients working with Greenleafs Product Quality, Manufacturing, and Compliance Team benefit from Taylors vast FDA institutional knowledge.

Taylor joined Greenleaf following a distinguished 20-year career at the FDA, where he served in multiple leadership positions, including as the FDAs Acting Deputy Principal Commissioner, FDA Counselor to the Commissioner, Acting Deputy Commissioner for Global Regulatory Operations and Policy, and Associate Commissioner for Regulatory Affairs.

ABOUT GREENLEAF

Greenleaf Health provides strategic and technical guidance to pharmaceutical, biotechnology, and medical device companies researching, developing, and manufacturing innovative solutions to pressing global public health challenges.

The firm includes former leaders and regulatory professionals from the FDA, Capitol Hill, top global pharmaceutical and medical device companies, leading law firms, and the top U.S. biotechnology trade organization. Greenleafs blend of former FDA officials and industry experts provides a unique set of capabilities when advising entities regulated by the FDA.

For more information on Greenleafs cell and gene therapy services and Greenleaf Health, visit greenleafhealth.com.

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Greenleaf Health Expands Services to Support Cell & Gene Therapy - Business Wire

Emmaus withdraws European application for sickle cell drug that got US approval – MedCity News

A U.S. biotech company has withdrawn its application seeking approval for a drug to treat sickle cell disease in Europe following a negative review by a body of the E.U.s drug regulation authority.

Torrance, California-based Emmaus Life Sciences said Thursday that it was withdrawing its marketing authorization application with the European Medicines Agency for Xyndari (glutamine), which it markets as Endari in the U.S., after a negative opinion by the EMAs Committee for Medicinal Products for Human Use, or CHMP. The CHMP stated that the data in the application did not show the drug was effective at reducing the number of sickle cell disease crises or hospital visits. It had previouslyissueda negative opinion in May.

Shares of Emmaus were down 9 percent Friday morning on the over-the-counter market and were down more than 20 percent when markets closed.

The drug, marketed as Endari in the U.S., has had Food and Drug Administration approval to treat sickle cell disease in patients aged 5 and older since 2017. Earlier his month, the FDA accepted an application for accelerated approval for Global Blood Therapeutics voxelotor, another drug to treat sickle cell disease.

Concerns expressed in the CHMPs opinion in May included a higher discontinuation rate among patients taking the drug than among those taking placebo, and data on how the drug worked for them was not available, with the committee concluding that the way their data were dealt with was not appropriate. Many patients in a supportive study also dropped out early, and the higher number of patients in the Xyndari arm than in the placebo arm who had received another drug, hydroxyurea, may have influenced the results.

According to data from the Phase III study on the ClinicalTrials.gov database, the trial enrolled 230 patients, of whom 152 were randomized to the Endari/Xyndari arm and 78 of whom were randomized to placebo. Of those in the treatment arm, 55 did not complete the study, compared with 19 of those in the placebo arm, yielding respective discontinuation rates of about 36 percent and 24 percent.

One sickle cell disease expert noted that the drug seems to have been approved in the U.S. because of the limited treatment options for sickle cell disease and there being few problems with safety, but it still raised other issues.

There were multiple methodological concerns about the pivotal study of [Endari] to prevent acute complications of sickle cell anemia, wrote Dr. John Strouse, a hematologist and associate professor of medicine at Duke University, in an email.

The FDAs Oncologic Drugs Advisory Committee had expressed similar concerns to those of CHMP about the higher discontinuation rate in the treatment arm. ODAC nevertheless voted to support the drugs overall efficacy and safety profile.

According to the Phase III datathat led to FDA approval, patients in the Endari arm experienced a median three sickle cell crises, two hospitalizations and 6.5 days in the hospital, compared with a respective median four crises, three hospitalizations and 11 days in the hospital among those receiving placebo. The Endari arm also showed an increase in the number of days before a sickle cell crisis occurred and a lower number of patients experiencing acute chest syndrome.

Because we have demonstrated the efficacy of Xyndari, as supported by the data from the trials conducted, we are disappointed by the CHMPs position, Emmaus CEO Yutaka Niihara said in a statement Thursday. We remain committed to the patients who suffer from sickle cell disease and will continue to endeavor to broaden our global patient base while identifying new clinical uses for L-glutamine, obtaining additional patients and distribution partners, and through ongoing community and physician outreach.

Niihara said the company was seriously considering a decentralized approval procedure on a country-by-country basis, though he was not available to elaborate further.

Photo: virusowy, Getty Images

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Emmaus withdraws European application for sickle cell drug that got US approval - MedCity News

Disabling one protein might one day lead to a cure for the common cold – Science News

An uncommon way of thinkingmay be bringing scientists one step closer to a cure for the common cold.

Researchers have identifieda key protein in humans that some viruses use to multiply inside of human cells.Disabling that protein, instead of attacking the virus itself, may prevent infections from spreading. In mice and human cells engineered to lack thisprotein, the viruses couldnt replicate, Jan Carette, a microbiologist at Stanford University School of Medicine,and colleagues report September 16 in Nature Microbiology.

Its not quite a cure forthe common cold, but its an interesting step forward, says Ellen Foxman, animmunologist at Yale School of Medicine who was not involved in the study.

Colds are the most common infectious disease in humans. On average, adults catch a cold two or three times each year, while children get the sniffles even more often (SN: 2/12/09). Any one of a few hundred viruses, including rhinoviruses, can cause these infections. That fact and because these viruses can mutate quickly to become resistant to drugs makes it difficult to find a cure.

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So researchers at Stanford and the University of California, SanFrancisco focused on the human host rather than the virus. Viruses hijack cells and rely on humans own cellularmachinery to make more virus and sicken their host. The team wanted to see if itcould identify human genes that make the proteins that many viruses hijack inorder to replicate.

Using the gene-editing tool CRISPR,Carette and colleagues systematically deleted chunks of DNA to build a libraryof human cells, each missing one gene and therefore unable to make that genescorresponding protein. The researchers then infected the cells with two typesof viruses, one that causes colds and another that has been linked toneurological diseases.

Using different viralproteins like hooks, the scientists pulled out human proteins that werephysically attached to a viral protein. That let the team identify which humanproteins were interacting with viral ones an indication that the virus was using that proteinto hijack the cell.

One human protein wasrepeatedly fished out of the cells: SETD3. And experiments indicated that theviruses needed SETD3 to take over the cell. Scientists knew this protein couldaffect actin proteins, which help muscles contract. But its role in viralinfections came as a surprise.

When the researchersinjected viruses into mice engineered to lack a working version of the SETD3 gene, the mice didnt get sick. Humanlung cells that also lacked the gene remained healthy. (Lung cells are oftenused in these types of studies because they are especially susceptible to manyrhinoviruses that cause colds.)

Repeating those experimentswith similar but potentially more serious viruses suggested the approach may beeffective against more than just the common cold. Engineered human cells didntbecome infected when they were exposed to viruses that cause hand, foot and mouthdisease and a polio-like spinal cord disease called acute flaccid myelitis. Andwhen mice were exposed to these viruses, the rodents that didnt have a functioningversion of SETD3 were much morelikely to survive than those that had the working gene.

We have identified an excellent target, says Carette, of SETD3. But its unclear whetherdisabling that gene and its protein could cause other problems. While theengineered mice survived and were healthy and fertile, they werent able to push their pups out of the wombduring birth, he says, which might be related to the proteins role in musclecontractions.

Scientists dont fully understand what this gene does in the human body, and getting rid of it completely could have unknown effects, says Vincent Racaniello, a virologist at Columbia University who wasnt involved in the work. The authors show that mice lacking the gene for SETD3 are viable and resistant to infection. However, this observation does not mean that SETD3 in humans is dispensable, he wrote in an e-mail.

Instead, the researchers think their best bet is to search for a drug that blocks the human protein and its viral counterparts from interacting, or one that destroys the human protein only when it is interacting with viral ones. But those types of drugs are still a long way off. The question is always When can I buy it over the counter? Carette says. Drug development takes time.

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Disabling one protein might one day lead to a cure for the common cold - Science News