My Third Stem Cell Treatment for MS – Everyday Health

Since 2014, I have had three stem cell treatments, each time hoping it would help fight off the progression of my multiple sclerosis (MS) symptoms. Because the procedures Ive undergone do not have a very long history of use or many studies to support them, Ive basically made myself a guinea pig by trying them.

So far, Ive experienced failure and success, but overall, the positives have been life-changing for me. I am continuing down this path of healing because there is currently no cure for MS, and now that Im 61, time is not on my side for a cure to be discovered!

In 2014, I had a procedure in which adult stem cells were isolated from my fat tissue, grown in a lab, and reinfused into my body. Initially I had great results, but they were short-lived, and within three months, all my MS symptoms had returned.

In 2018, I had a different type of procedure, in which 300 million stem cells derived from umbilical cord tissue were infused into my arm. I wrote about my experience in a blog just two months after that infusion. At the time, I was having a positive response, but I was also skeptical, since I had seen similar early improvements in 2014.

Two weeks after my 2018 treatment, my left-side drop foot was gone, I could jump off the floor, and I had regained some feeling in my left arm. But I needed to give this new stem cell treatment some more time before I could positively state that this one had worked. So I waited and kept working out in the gym as I have always done, pushing myself harder as time went by.

I found my body getting stronger and stronger as the months passed, and I even filmed myself squatting 500 pounds and posted it in our MS Fitness Challenge GYM Facebook group to show the community that I was not just imagining the results.

Months after the treatment, my left leg was almost as good as my right one, which has never been affected by MS, and my overall strength was increasing.

By the eighth month or so, I was running on the treadmill, and at approximately nine months post-infusion, I was jumping rope. I had not run or jumped rope in almost 14 years.

I was and am extremely happy with what my legs are now capable of, but I only got partial feeling back in my left arm and hand, and I want that back the same way I have my left leg back.

With that goal in mind, I reached out to the doctor who had performed my stem cell procedures and asked if I was eligible for more cells. I also asked if he thought more cells would bring back the feeling in my left arm and hand.

His response was that no one had predicted what results I would get from the 2018 treatment, and here I was running, jumping rope, and squatting like a champion. So we had nothing to lose by transfusing another 300 million cells into my body. We both thought that since my leg no longer needed the stem cells, maybe they would find their way to my arm!

Theoretically, stem cells go where theyre needed to repair the damage.It seemed worth a shot, so I booked my flight to the Cayman Islands for a January 2020 infusion. And now I am home.

Its only been a few weeks, but I can already feel hot and cold in my left hand, which I have not been able to do since 2006, when I was diagnosed.

Again, I am cautiously optimistic that I will get similar results in the areas I need them now. But only time will tell. All I can say is that this therapy has changed my life, and I am hopeful that an ongoing clinical trial of the stem cell treatment I received will provide evidence that it will also be helpful to others with MS.

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JDRF Reaches New Milestones in Work to Drive Cures for Type 1 Diabetes and Improve Lives – Yahoo Finance

Annual Report showcases year of advances, driven by $121.5 million in direct support and nearly $300 million in additional funding for T1D research

NEW YORK, Feb. 14, 2020 /PRNewswire/ -- JDRF, the leading global organization funding type 1 diabetes (T1D) research, funded $121.5 million directly and helped generate more than $400 million in total T1D research funds from nonprofits, government, and industry to propel a year of remarkable breakthroughs in 2019, JDRF announced today. JDRF research funding increased by 10 percent over the previous year.

"I am more excited about our progress than ever before, given the tremendous breakthroughs made in 2019," said Aaron J. Kowalski, Ph.D., president and CEO of JDRF. "We could not be driving this work forward without the support of so many dedicated individuals, partners and organizations all helping us deliver on our mission to cure T1D and improve lives."

JDRF funding supported more than 180 active T1D research grants and is funding about 70 clinical trials for drugs, biologic and devices to prevent or cure T1D. The organization also supported new investment through the JDRF T1D Fund in 15 T1D companies, including 11 focused on cures.

"We are focused on beta cell therapies and immune therapies to deliver cures for T1D," Kowalski said. "At the same time, we continue accelerating work in glucose control and complication therapies to improve the lives of those living with type 1 diabetes today."

Highlights of breakthroughs outlined in the 2019 Annual Report are:

Progress Toward Cures:

Improving Lives:

Reducing Complications:

JDRF's Annual Report also cites progress in advocacy and community engagement, two areas aimed at both supporting research and the T1D community. "The combined strengths of research, advocacy and community engagement are helping us support more members of the T1D community and enabling more people to support our mission," Dr. Kowalski said. "JDRF works to build partnerships that advance research and build and sustain critical support for type 1 diabetes (T1D) research funded by the Federal Government."

Expanding Access to Coverage:

Learn more about JDRF's research and advocacy priorities at jdrf.org and read the health insurance guide JDRF has complied to help the T1D community navigate their healthcare and health insurance here.

About JDRF

JDRF is the leading global organization funding type 1 diabetes (T1D) research. Our mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.2 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact and uniting on a national stage to pool resources, passion and energy. We collaborate with academic institutions, policymakers and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter: @JDRF.

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JDRF Reaches New Milestones in Work to Drive Cures for Type 1 Diabetes and Improve Lives - Yahoo Finance

‘Genetic rewiring’ by a microRNA gene drives cancer’s drug resistance – The Institute of Cancer Research

Image:MicroRNA and mRNA visualisation in differentiating C1C12 cells. Credit: Ryan Jeffs, License:CC BY-SA 3.0

A tiny molecule of RNA known as microRNA plays a key role in rewiring cancer cells so they can resist the effects of chemotherapy, a new study reveals.

The discovery opens up the possibility of creating new cancer drugs that target this microRNA molecule, rather than more conventional treatments that block the action of proteins.

Scientists at The Institute of Cancer Research, London, found that a microRNA gene known as MIR1249 plays a key role in allowing bile duct cancers to resist chemotherapy.

Whereas most genes are translated from RNA molecules into proteins, microRNA genes remain functional at the RNA level and seem to be crucial for controlling the signalling networks within cells.

The researchers hope that MIR1249 could be a potential target for new drugs in bile duct cancer that could make chemotherapy much more effective. Bile duct cancer is very hard to treat and there is an urgent need to develop new therapies.

The researchers also believe that this mechanism driving resistance may be shared with other cancer types raising the possibility that drugs to counteract it could have wider benefits.

The study is published in the journal Hepatology and released today on Bile Duct Cancer Awareness Day. It was funded by The Institute of Cancer Research (ICR) as well as the European Union, Cancer Research UK, Pancreatic Cancer Actionand the Italian Foundation for Cancer Research AIRC.

We are building a new state-of-the-art drug discovery centre to develop a new generation of drugs that will make the difference to the lives of millions of people with cancer. Find out more about the Centre and about how you can help us finish it.

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The study reveals that MIR1249 is able to rewire the so-called WNT signalling network, which plays an important role in some healthy cells, and can be co-opted by cancer cells.

The WNT network is involved in the upkeep of stem cells cells with the ability to self-renew and develop into many different cell types.

But when rewired, it seems to give cancer cells the features of stem cells, allowing them to become more resistant to treatment and able to survive the onslaught of chemotherapy.

The study, which involved mice and human tissue samples, revealed that by blocking MIR1249 activity, cancer cells became more sensitive to chemotherapy and responded better to treatment.

The researchers also found that 41 per cent of bile duct cancers had increased levels of MIR1249, suggesting it could be playing an important role within these tumours. In support of this idea, MIR1249 was also statistically associated with poorer survival outcome.

The next step will be to create drugs that could act against MIR1249, with the aim of preventing cancers from developing drug resistance and resensitising them to chemotherapy.

The ICR a charity and research institute is focused on understanding and combating cancers ability to evolve and become drug resistant. It has less than 10 million still to raise for a new Centre for Cancer Drug Discovery, which will house an ambitious Darwinian drug discovery programme to create new anti-evolution treatments.

Study leader Dr Chiara Braconi, who carried out the research as Clinician Scientist at The Institute of Cancer Research, London, and is now Lord Kelvin Adam Smith Reader in the Institute of Cancer Sciencesat the University of Glasgow, said:

Our study shows the crucial role played by a piece of microRNA in rewiring the network of signals within cancer cells and helping them to resist the effects of chemotherapy. It identifies MIR1249 as a potential drug target in bile duct cancers and possibly other tumour types, and opens up what could be an exciting new avenue of treatment.

Its remarkable how such a tiny piece of RNA can play such a significant role in rewiring cancer cells so that they can resist chemotherapy. There is growing interest in the idea of developing drugs against RNA rather than against proteins, as studies like ours show the important role of microRNA in cell signalling.

Study co-author Professor Paul Workman, Chief Executive of The Institute of Cancer Research, London, said:

Bile duct cancer is becoming increasingly common around the world and survival rates are very poor, so there is an urgent need to develop better therapies for people with advanced disease whose treatment stops working. This new study shows the potential of targeting molecules called microRNAs as a new form of treatment for drug-resistant cancers.

At the ICR, we believe overcoming cancer evolution and drug resistance is the biggest challenge we face today in the field of cancer research. Through our new Centre for Cancer Drug Discovery, we are aiming to find new anti-evolution treatments that can offer long-term control or cure even for advanced cancers.

Sal Cheema, 42, from Uxbridge, who was diagnosed with stage 4 bile duct cancer in 2018, said:

I had chemotherapy for eight months which kept my cancer at bay. But within six weeks, I was devastated to learn that the cancer had spread to my lymph nodes.

Bile duct cancer is rare, yet the incidence is increasing and its not known why. This cancer is also affecting younger people more and more, so its imperative that this specific research continues not only for bile duct but other cancers too.

There are very few treatment options for bile duct cancer patients, so Im elated to hear of this research and discovery as it could open up a whole new way of targeting the cancer. More targeted treatments with fewer side effects are vital these findings are very promising for people like me.

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'Genetic rewiring' by a microRNA gene drives cancer's drug resistance - The Institute of Cancer Research

Unproven Stem Cell Treatments Offer Hope & Risks

The injections of stem cells into his spine were supposed to help Jim Gass, 66, recover from a stroke he had six years ago.

Gass traveled to clinics in Mexico, China, and Argentina to undergo these unproven procedures. Including travel, he spent close to $300,000, according to a story in The New York Times.

After the final round of shots, he was able to walk better. But his hope for a full recovery was cut short. While on vacation in Thailand six months after his treatments, he developed low back pain and difficulty walking and standing.

Back in Boston, doctors at Brigham and Womens Hospital did an MRI scan of his spine, and found a large mass filling the entire lower part of his spinal column.

Genetic testing revealed that the abnormal, primitive cells of the mass did not come from Gass, but from stem cells injected into his spine.

Radiation treatments seemed to slow the growth of the mass and improve Gass symptoms. But another scan done later in San Diego showed that the mass was growing again.

The doctors involved wrote about his case in a letter published June 22 in the New England Journal of Medicine.

Despite the outcome of this case, experts familiar with this kind of stem cell tourism say that some good may still come of it.

It is a really sad case, but its good that its causing discussion around both the potential harm of these therapies and the lack of evidence regarding the benefits, Timothy Caulfield, research director of the Health Law Institute at the University of Alberta, who wrote a recent commentary on stem cell hype, told Healthline.

Read more: Get the facts on stem cell research

This is not the first time that stem cell treatments have led to bad outcomes such as tumors or lesions.

There have been other reports of adverse events as a result of these kinds of therapies, said Caulfield. There have even been reports of adverse events when the procedure is less extreme such as people getting stem cell therapy for anti-aging, anti-wrinkle procedures.

Caulfield is quick to point out that therapy should be in quotes because with the exception of a few approved treatments the use of stem cells to treat illnesses has not reached the point where it is ready for widespread use in clinics.

There are very few stem cell therapies that have been proven, at this point, to be efficacious, said Caulfield. Lots of exciting work is going on theyre in clinical trials right now but for most conditions we simply arent there yet.

Although there are a few documented cases like Gass, many more may go unreported, resulting from treatments at unregulated stem cell clinics around the world.

We dont know exactly how many people are having these procedures, Dr. Jaime Imitola, a neurologist and stem cell researcher at The Ohio State University Wexner Medical Center, who has written about the dangers of stem cell tourism and how to counsel patients, told Healthline.

There are so many diseases that these clinics are often treating for from diabetes to ALS and some of these treatments may involve more risk than others, Imitola said.

Theres a big difference in risk between taking cells from your own body and putting them back in your blood, and injecting foreign cells into your spine, as was done in Gass case.

Also, these clinics are not part of a clinical research program, so there are a lot of unknowns about what happens during the procedures.

Are they actually using stem cells? How are they getting the stem cells into people? said Caulfield. Those are all open questions, because it is such an unregulated field.

While Gass traveled outside the United States for injections, unproven stem cell therapies show up much closer to home.

A paper published online Thursday in the journal Cell Stem Cell found that at least 351 businesses in the United States are marketing stem cell therapies that have not gone through the rigorous clinical trial process, or been approved by the Food and Drug Administration (FDA).

These businesses marketed stem cells as treatment for a wide range of conditions from spinal cord injuries and immune system problems to heart disease or even cosmetic fixes.

Read more: Stem cell treatments offering hope for MS patients

With few treatments available for many diseases, stem cell clinics step in to fill the void, many overhyping the actual research being done in this area.

[Clinics] are leveraging excitement around legitimate stem cell research and the pop culture footprint Ill put it that way of stem cells, said Caulfield.

Some of this hype has been generated when high-profile athletes undergo stem cell therapy and see improvements, like Peyton Manning did in Germany for a neck injury.

The company that Gass contacted had been involved in the treatment of former NFL quarterback John Brodie.

These remarkable success stories offer people hope. But because they happened outside a clinical trial, its impossible to know if the athletes health would have improved on their own.

Imitola compares this to using acupuncture alongside proven treatments.

If I give you acupuncture after a stem cell treatment, I cannot make the distinction whether what happens is a result of the acupuncture or the treatment, said Imitola, because this is not a clinical trial.

Researchers, universities, and the media also have a hand in stem cell hype. The time element, in particular, can be misrepresented.

"I think that the scientific community really needs to be careful how they talk about stem cell research, said Caulfield. We did a study that showed, for example, that the time from doing basic research to getting into the clinic is often exaggerated when people talk about stem cell research. Our study found that it was often portrayed as if the research was going to be in the clinic in 5 to 10 years, or sooner, which is really, really fast. It creates unrealistic expectations."

Read more: Stem cells as a possible treatment for rheumatoid arthritis

Patients with spinal cord injuries or diseases are often anxious for new treatments to be approved quickly. But stem cell researchers have good reason to be cautious.

One characteristic that stem cells share with cancer cells is that they both multiply rapidly. This is why stem cell researchers have long been concerned that stem cells could form tumors.

Thats why there are so many years of testing in the lab, in animal models, and finally in clinical trials.

It is unethical to offer a procedure or a drug that is unproven, said Imitola.

When clinics skip ahead and offer treatments that have not been properly tested, they may end up hurting people instead of helping them.

Its interesting because [Gass] case, and others, is generating a new disease, a new complication, an iatrogenic tumor, said Imitola.

Of course, bad outcomes can happen during a clinical trial. But these are tracked, and clinical trials can be shut down if unforeseen side effects happen.

A recent stem cell clinical trial in Japan was stopped, because when the researchers looked at whether the cells were clean from a genetic point of view, the cells had some problems, some changes, said Imitola, So the researchers said, We cant do that, we cant inject the cells.

Imitola recently co-authored a paper in JAMA Neurology calling on doctors to educate patients with neurological diseases about stem cell tourism.

But he admits that cases like Gass can serve as an even more effective warning.

This patient, in particular, is important because he put a human face to this tragedy, said Imitola. We need more patients to come forward. Most likely, this is not an isolated case.

Read more: Stem cell treatment for COPD

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Unproven Stem Cell Treatments Offer Hope & Risks

Update on stem cell treatment cost for 2018 from ongoing poll …

I get asked many questions about stem cell therapies, but one of the most common over the years has been about the stem cell treatment cost. For instance, a reporter might ask, How much does a stem cell treatment for MS cost? and a patient might ask me, How much is a fair cost for a stem cell therapy for arthritis? Or, patients will voluntarily tell me what they paid or mention it in the comments. We hear various numbers thrown around about costs so I decided to do a poll on this. I even did an early update on the results of this poll, voicing my skepticism that the costs paid were worth it.

But the poll has gotten well over 500 responses now so I thought I would revisit it and what it might mean.

You can see a screenshot of the images. Its fair to say, as much as Internet polls arent considered particularly accurate, that this one largely fits with what is reported out in the field.

(On a side note, I wish there was such a thing as going out into the field for stem cell scientists as Ive always been a bit jealous of scientists who really do go out in the field. What do we do, go out in the wild and catch wild or feral stem cells in the bush?)

Patients self-reported most often paying between $2,500 and $7,500 for their stem cell therapy so if we take the average of those we get that $5,000 figure that is what I hear most often from others. Yes, not necessarily very rigorous, but the result makes good sense. Not far behind though were responses in the $7,500-20,000 range.

About 1 in 10 respondents reported paying $20,000 or more, including some beyond $100,000. Thats a whopping stem cell treatment cost, especially for something most often unproven and unapproved by the FDA.

If we consider these responses, the average cost may be more like $7,500-$10,000.

Notably, about 1/16 respondents indicated their stem cells were free. Im not sure what that means in terms of how that came to be.

Interestingly, most respondents who also went on to answer a 2nd poll in that post about where they got the treatment indicate it was at a stem cell clinic (scroll down in that Oct. 2017 post and youll see the 2nd poll). This 2nd poll has about 200 responses.

So today buying a simple stem cell treatment, most often unproven and non-FDA approved, is often not so different in cost than buying a 10-year old used car, while less often it is similar to buy various new cars including at the high end of stem cell therapy cost, some very expensive new cars. This cost and the risks involved are why I have suggested to patients in the past to be assertive when considering a stem cell treatment, ask questions, dont just accept too good to be true kinds of answers, etc. In short, be at least (or ideally much more) rigorous about unproven stem cell treatments as you are about buying a car.

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Hematology society recognizes four School of Medicine researchers Washington University School of Medicine in St. Louis – Washington University…

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ASH Scholar Awards support fellows to junior faculty in hematology

The American Society of Hematology has recognized four Washington University researchers with ASH Scholar Awards, supporting early-career faculty whose research is focused on understanding and treating blood disorders. Clockwise from top left: Amanda Smith, PhD, Julia Warren, MD, PhD, Stephanie Luff, PhD, and Karolyn Oetjen, MD, PhD.

Four early-career researchers at Washington University School of Medicine in St. Louis have been recognized by the American Society of Hematology (ASH) for their dedication to the field of hematology. The ASH Scholar Awards provide financial support to fellows and junior faculty who have dedicated their careers to the study of blood disorders.

One of ASHs most prestigious research award programs, the Scholar Awards provide funding to young scientists as they make the transition from training programs to careers leading independent labs.

Karolyn A. Oetjen, MD, PhD, Amanda M. Smith, PhD, Stephanie Luff, PhD, and Julia T. Warren, MD, PhD, are among the 39 recipients of the 2020 ASH Scholar Awards. Depending on whether the researcher is a fellow or junior faculty member, the awards provide $100,000 to $150,000 over a two- to three-year period to support basic, translational and clinical research seeking to understand and improve the treatment of blood disorders.

Oetjen, an instructor in medicine, uses single-cell sequencing and imaging techniques to understand how cancer cells evolve and adapt to chemotherapy, allowing the recurrence of blood cancers including acute myeloid leukemia (AML) and myelodysplastic syndromes.

Smith, an instructor in medicine, studies the origins of AML in the lab of Timothy J. Ley, MD, the Lewis T. and Rosalind B. Apple Professor of Oncology. She is interested in understanding the events that lead a normal cell to start showing the early signs of becoming cancerous and, from there, identifying the triggers that lead to full-blown AML. The goal is to identify strategies to block this process.

Luff is a postdoctoral research scholar in the lab of Christopher M. Sturgeon, PhD, an assistant professor of medicine. She studies blood-forming stem cells in the early embryo in an effort to understand how they develop.

Warren is a fellow in pediatric hematology/oncology, conducting postdoctoral research in the lab of Daniel C. Link, MD, the Alan A. and Edith L. Wolff Professor of Medicine. Her work is focused on understanding the genes and metabolic processes that regulate formation of granulocytes, a type of white blood cell. Understanding these processes could help develop new treatments for patients with diseases that affect granulocytes and for cancer patients whose treatment causes low numbers of white blood cells, increasing the risk of infection.

The American Society of Hematology is the worlds largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment and prevention of disorders affecting the blood.

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Hematology society recognizes four School of Medicine researchers Washington University School of Medicine in St. Louis - Washington University...

The Art of Science Cutting edge science and captivating images – The – The Institute of Cancer Research

Image: The winning image by PhD student Sumana Shrestha, showing differentiating brain cancer cells used to study glioblastoma

PhD student Sumana Shrestha at The Institute of Cancer Research, London, has won the ICRs Science and Medical imaging competition with a colourful image of neural stem cells to study glioblastoma, as well as the public vote, with 212 votes for her detailed image of microparticles.

Researchers from the ICR and The Royal Marsdensubmitted some fantastic images to this years competition, with a wide array of images that tell vivid stories about our science.

Entries to the competition have all been created in the course of our pioneering cancer research but at times they can look like a piece of modern art, and they each tell a compelling story about our research.

The most engaging and exciting image selected by our panel of judges is named the winner of the ICR Science and Medical Imaging Competition.

Our followers on social media picked the supporters' favourite through a public vote, with over 800 votes submitted across Facebook, Instagram and Twitter.

The winning image was taken using a technique called confocal microscopy and shows neural stem cells from mice which are being used to study glioblastoma, an aggressive type of brain cancer. The cells were genetically modified and exposed to a virus to develop glioblastoma, before mice were treated with microRNA repressors and activators to change how the cells grow in the brain and specialise.

A process called immunostaining shows up a garden of stem cells growing and specialising into specific types of cell, to carry out jobs in the brain. Star shaped cells called astrocytes are marked in green, which perform a range of functions in the brain, while neurons are marked in red, cells in the nervous system that help transmit information.

MicroRNAs are proteins in cells that help regulate gene expression, and in cancer they can be severely malfunctioning. Modulating microRNA levels in brain cancer cells could have a major impact on how the tumour cells develop, which could help to make the disease less aggressive and easier to treat.

The judges picked this as their wining image noting the striking use of colours in the image, and how Sumana has used her image to illustrate how her research is helping to tackle cancer.

Catherine Graham, Head of Brand and Creative at the ICR, said:

"Sumanas image is really eye-catching and it is a great example of the technical skill and creative flair of our researchers, and the visual impact that can be captured even in a disease like cancer.

Choosing the winning image from all of those submitted to the competition this year was challenging, but it was a pleasure to see the variety of our researchers work and to learn more about their science.

PhD student Sumana Shrestha said:

This is very unexpected news for me as the competition was so strong, but I am over the moon to find that my image has won! Many thanks to the judges for taking their time to go through the images. I am very excited for the announcement, and again, overjoyed about the results.

The image shows microparticles, tiny beads about the width of a human hair, taken using scanning electron microscopy. To image the microparticles, they are first coated in a layer of gold just nanometres thick to make them conductive. The particles have been etched using an antibiotic called fusidic acid to create dimples on their surface like a golf-ball.

Microparticles can be used to deliver drugs in the body, but studies have shown that the textured surface of microparticles can also have an effect on cell behaviour.

Cells are influenced by the topographical features of their surroundings, and the 'dimples' on the treated microparticles provide an environment that can be used to control proliferation or differentiation of stem cells. Manipulating fundamental cellular processes using microparticles could have huge implications in cancer, as well as applications in regenerative medicine.

This image received a total of 212 across the ICRs social media channels to be crowned our supporters favourite, with an ICR follower on social media thinking the microparticles looked like pollen grains.

Science Communications Officer Graham Shaw, who organised the competition, said: Its surprising that two submissions from one researcher have won both prizes in the competition, but this black and white image clearly captured the imaginations of our supporters on social media.

The detail on the microparticles is amazing, and their potential to alter cells as a way to treat cancer is a really powerful idea. This image helps to convey how this has been implemented in cancer research.

Eight of the most highly rated and vibrant images from this year's competition were voted on by our supporters for their favourite image. Learn more about the other amazing images that made it onto our shortlist.

The eye of the Storm: the tumultuous yet spectacular nature of cancer tumours by Dr Lisa Pickard and Louise Howell

The eye of the storm: the tumultuous, dramatic nature of cancer tumours. Lab grown mini-tumours are better at capturing the unique characteristics of tumours in patients, and can be used to understand how they might respond to cancer drugs.

Melanoma on a Chip by Professor Chris Bakal and Nick Moser at Imperial College, London

Melanoma on a chip: part of a melanoma cell has been blasted away using a technique called ion beam milling. This process helps researchers look inside cells in unprecedented detail, to help them understand the processes happening inside.

Melanoma cell invasion in 3D by Vicky Bousgouni, Professor Chris Bakal and David Robertson

Invading melanoma cell in 3D. A cell from aggressive skin cancer is growing into and around fibres of collagen, mimicking how cancer invades tissue. Cancer remodels its environment to spread around the body, so understanding this process could make it easier to treat.

Super-resolution microscopy image of cell focal adhesions by PhD Student Ian Jones, Dr Lucas Dent, and Professor Chris Bakal

The first ever super-resolution microscopy image taken of focal adhesions molecules inside cellular structures which help cancer cells move and spread around the body.

The Art of Deception by Dr Jarama Clucas

The Art of Deception. These aggressive breast cancer cells look like structures normally seen in healthy breast tissue. Cancer use deception to hide from the immune system and avoid treatment, but growing samples in the lab can be used to test treatments that could help our body fight the disease.

Forward and reverse translation by PhD Student Somaieh Hedayat and Louise Howell

Tumour organoids are mini replicas of a patients tumours, and could be used to rapidly test a patients cancer against a range of cancer drugs to see which ones may be most effective.

These eye-catching images illustrate just some of the cutting-edge research being carried out at the ICR, taken using sophisticated equipment purchased thanks to generous donations from our supporters.

From images like these our researchers are gaining unprecedented insights into the mechanisms that drive cancer, and new ways to target the disease to help treat patients.

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The Art of Science Cutting edge science and captivating images - The - The Institute of Cancer Research

After pursuing him two years ago, the Padres are betting on Tommy Pham – The Athletic

PEORIA, Ariz. Two summers ago, members of the Padres front office trekked to Cooperstown, N.Y., to attend the enshrinement of one of their own. They watched as Trevor Hoffman, franchise icon and senior advisor, was inducted into the National Baseball Hall of Fame. They toasted and they celebrated. And, in between festivities, they tapped away at their phones.

In a wooded, secluded region of Central New York, team executives passed the final hours before the July 2018 trade deadline. Less than two weeks earlier, the Padres had sent relievers Brad Hand and Adam Cimber to Cleveland for catching prospect Francisco Meja. Now they were attempting, among other things, to acquire a St. Louis Cardinals outfielder named Tommy Pham.

We were going back and forth, general manager A.J. Preller said.

At the time, the Padres were slogging through the third year of a rebuild. They had signed first baseman Eric Hosmer to a nine-figure contract, but not six...

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After pursuing him two years ago, the Padres are betting on Tommy Pham - The Athletic

Carl June on CRISPR, CART and how the Vietnam War dropped him into medicine – Endpoints News

In August of 2011, Carl June and his team published a landmark paper showing their CART treatment had cleared a patient of cancer. A year-to-the-month later, Jennifer Doudna made an even bigger splash when she published the first major CRISPR paper, setting off a decade of intense research and sometimes even more intense public debate over the ethics of what the gene-editing tool could do.

Last week, June, whose CART work was eventually developed by Novartis into Kymriah, published in Sciencethe first US paper showing how the two could be brought together. It was not only one of the first time scientists have combined the groundbreaking tools, but the first peer-reviewed American paper showing how CRISPR could be used in patients.

June used CRISPR to edit the cells of three patients with advanced blood cancer, deleting the traditional T cell receptor and then erasing the PD1 gene, a move designed to unleash the immune cells. The therapy didnt cure the patients, but the cells remained in the body for a median of 9 months, a major hurdle for the therapy.

Endpoints caught up with June about the long road both he and the field took to get here, if the treatment will ever scale up, and where CRISPR and other advancements can lead it.

The interview has been condensed and edited.

Youve spoken in the past about howyou started working in this field in the mid-90s after your wife passed away from cancer. What were some of those early efforts? How did you start?

Well, I graduated from high school and had a low draft number [for the Vietnam War] and was going to go to study engineering at Stanford, but I was drafted and went into the Naval Academy in 1971, and I did that so I wouldnt have to go to the rice fields.

The war ended in 73, 74, so when I graduated in 1975, I was allowed to go to medical school, and then I had a long term commitment to the Navy because they paid for the Acadamy and Medical school. And I was interested in research and at the time, what the Navy cared about was a small scale nuclear disaster like in a submarine, and like what happened at Chernobyl and Fukushima. So they sent me to the Fred Hutchinson Cancer Center where I got trained in cancer, as a medical oncologist. I was going to open a bone marrow transplant center in Bethesda because the Navy wanted one in the event of a nuclear catastrophe.

And then in 1989, the Berlin Wall came down and there was no more Cold War. I had gone back to the Navy in 86 for the transplant center, which never happened, so then I had to work in the lab full time. But in the Navy, all the research has to be about combat and casualty. They care about HIV, so my first papers were on malaria and infectious disease. And the first CAR-T trials were on HIV in the mid-90s.

In 96, my wife got diagnosed with ovarian cancer and she was in remission for 3-4 years. I moved to the University of Pennsylvania in 1999 and started working on cancer because I wasnt allowed to do that with the Navy. My wife was obviously a lot of motivation to do that. She passed away in 2001. Then I started working with David Porter on adoptive transfer T cells.

I got my first grant to do CAR-T cells on HIV in 2004, and I learned a whole lot. I was lucky to have worked on HIV because we did the first trials using lentiviruses, which is an engineered HIV virus.

I was trained in oncology, and then because of the Navy forced to work on HIV. It was actually a blessing in disguise.

So if you hadnt been drafted, you wouldve become an engineer?

Yes. Thats what I was fully intending. My dad was a chemical engineer, my brother is an engineer. Thats what I thought I was going to do. No one in my family was ever a physician. Its one of those many quirks of fate.

Back then, we didnt have these aptitude tests. It was just haphazard. I applied to three schools Berkeley, Stanford and Caltech and I got into all three. It was just luck, fate.

And it turned out when I went to the Naval Academy, they had added a pre-med thing onto the curriculum the year before, so thats what I did when I started, I did chemistry.

I wouldve [otherwise] been in nuclear submarines. The most interesting thing in the Navy then was the nuclear sub technology.

You talked about doing the first CAR-T trials on HIV patients because thats where the funding was. Was it always in your head that this was eventually going to be something for cancer?

So I got out of the Navy in 99 and moved to Penn. I started in 98 working on treating leukemia, and then once I got to Penn, I continued working one day a week on HIV.

Its kind of a Back-to-the-Future thing because now cancer has paved out a path to show that CART cells can work and put down the manufacturing and its going to be a lot cheaper making it for HIV. I still think thats going to happen.

Jim Riley, who used to be a postdoc in my lab, has some spectacular results in monkeys with HIV models. They have a large NIH and NIAID research program.

So were going to see more and more of that. The CAR technology is going to move outside of cancer, and into autoimmune and chronic infections.

I want to jump over to cytotoxic release syndrome (CRS)because a big part of the CRISPR study was that it didnt provoke this potentially deadly adverse effect. When did you first become aware that CRS was going to be a problem?

I mean we saw it in the very first patient we treated but in all honesty, we missed it. Im an MD, but I dont see the patient and David Porter tookcare of the first three patients and our first pediatric patient,Emily Whitehead.

In our first patients, 2 out of 3, had complete remission and there were fevers and it was CRS but we thought it was just an infection, and we treated with antibiotics for 3 weeks and[eventually] it went away. And sort of miraculously he was in remission and is still in remission, 9 years later.

And then when we treated Emily. She was at a 106-degree fever over three days, and there was no infection.

Ive told this story before. My daughter has rheumatoid arthritis, and I had been president of the Clinical Immunologists Society from 2009 to 2010, and the first good drug for juvenile rheumatoid arthritisthat came out. I was invited to give the Japanese scientist Tadamitsu Kishimoto the presidential award for inventing the drug.

Then in 2012, Emily Whitehead was literally dying from CRS, she had multiple organ failures. And her labs came back and IL-6 levels were 1000x normal. It turns out the drug I was looking at for my daughter, it blocks IL-6 levels. I called the physician and I said, listen theres something actionable here, since its in your formulary to give it to her off-label.

And she gave her the appropriate dose for rheumatoid arthritis. It was miraculous. She woke up very rapidly.

Now its co-labeled. When the FDA approvedKymriah, it was co-labeled. It kind of saved the field.

How were you feeling during this time? Did you have any idea what was happening to her?

No, not until we got the cytokine levels, and then it was really clear. The cytokine levels go up and it exactly coincided. Then we retroactively checked out adults and they had adverse reactions and it easy to see. We hadnt been on the lookout because it wasnt in our mouse models.

And it appeared with those who got cured. Its one of the first on-target toxicities seen in cancer, a toxicity that happens when you get better. All the toxicities from chemotherapy are off-target: like leukopenia or hair loss.

I had a physician who had a fever of 106, I saw him on a fever when he was starting to get CRS. When the nurse came in and it said 106, they thought the thermometer must be broken. On Monday, I saw him, and said how are you feeling and he said fine. And I looked at the thermometer and histemperature was still 102.

People will willingly tolerate on-target toxicity thats very different from chemotherapy if they know it helps get them better. Thats a new principle in cancer therapy.

You had these early CART results almost at the same time that Doudna publishes the first CRISPR papers, then still in bacteria. When did you first start thinking about combining the two?

Yeah, it was published inSciencein 2012 and thats when Emily Whitehead got treated. Its an amazing thing.

Thats something so orthogonal. You think how in the heck can that ever benefit CART cells? but my lab had done the first edited cells in patients, published in 2012. And we used zinc-fingered nucleases, which were the predecessors to CRISPR. It knocked out one gene at a time, but we showed it was safe.

I was already into gene editing because it could make T cells resistant to HIV. So it was pretty obvious that there were candidates in T cells that you can knock out. And almost every lab started working on some with CRISPR, cause it was much easier.

We were the first to get full approval by the FDA, so we worked on it from 2012, had all the preclinical data by 2016, and then it takes a while to develop a lot of new assays for this as we were very cautious to optimize safety and it took longer than we wanted, but in the end, we learned a tremendous amount.

So what did we learn?

First of all our patients had advanced metastatic cancer and had had a lot of chemotherapy. The first patient had had 3 bone marrow transplants.

One thing is feasibility: could you really do all the complex engineering? So we found out we could. feasibility was passed.

Another was the fact that cas9 came out of bacteria, forms of strep and staph. Everyone has pre-existing immunity to Cas9 and we had experience from the first trial with Sangamo[with zinc-finger nucleases] where some patients had a very high fever. In that case, we had used adenoviruses, and it turned out our patients had very high levels of baseline immune response to adenoviruses, so we were worried that would happen with CRISPR, and it did not happen.

It did not have any toxicity. If it had, it would have really set the field back. If there was animmune response to cas9 and CRISPR, there couldve been a real barrier to the field.

And then, the cells survived in the patients. The furthest on, it was 9 months. The cells had a very high level of survival. In the previous trials, the cells survived less than 7 days. In our case, the half-life was 85 days. We dont know the mechanism yet.

And we found very big precision in the molecular scissors, and that was a good thing for the field. You could cut 3 different genes on 3 different chromosomes and have such high fidelity.

It [CRISPR] is living up to the hype. Its going to fix all these diseases.

Whats the potential in CAR-T, specifically?

Well theres many many genes that you can add. There are many genes that knocking outwill make the cells work better. We started with the cell receptor. There are many, I think, academics and biotechs doing this now and it should make the cells more potent and less toxic.

And more broadly, what else are you looking at for the future of CART? The week before your paper, there were the results from MD Anderson on natural killer cells.

Different cell types, natural killer cells, stem cells putting CAR molecules into stem cells, macrophages. One of my graduate students started a company to do CAR macrophages and macrophages actually eat tumor cells, as opposed to T cells that punch holes in them.

There will be different cell types and there will be many more ways to edit cells. The prime editing and base editing. All different new variations.

Youve talked about how people used to think the immuno-oncology, if it ever worked, would nevertheless be a boutique treatment. Despite all the advancements, Novartis and Gilead still have not met the sales they once hoped to grab from their CART treatments. Are you confident CART will ever be widely accessible?

Oh yeah, Novartis sales are going up. They had a hiccup launching.

Back in 96 or 97, when Genentech launched Herceptin, their commercial antibody, they couldnt meet the demand either and then they scaled up and learned how to do better cultures. So right now Novartis is using tech invented in my lab in the 1990s culture tech thats complex and requires a lot of labor, so the most expensive part is human labor. A lot can be made robotic. The scale problem will be much easier.

Thats an engineering problem that will become a thing of the past. The manufacturing problem will get a lot cheaper. Here in the US, we have a huge problem with how drugs are priced. We have a problem with pricing. Thats a political issue.

But in cell therapy, its just kind of the growth things you see in a new industry. Itll get worked out.

This article has been updated to reflect that Jim Riley conducted work on CAR in HIV.

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Carl June on CRISPR, CART and how the Vietnam War dropped him into medicine - Endpoints News