University of Minnesota bioethicist takes on clinics touting stem-cell studies – Minneapolis Star Tribune

U of M professor Leigh Turner is taking huge flack from this "stem sell" industry for a paper he wrote analyzing how many of the clinics are using the government website clinicaltrials.gov to advertise their services by disguising them as legitimate clinical trials, which they are not. Photographed at the University...

Renee Jones Schneider, Star Tribune

By Joe Carlson , Star Tribune August 05, 2017 - 6:46 PM

The clinics offer futuristic-sounding treatments for everything from eye problems to osteoarthritis.

Listed on a government website, they present the opportunity to participate in clinical trials to test the potential of one of the most promising tools in medicine the bodys own stem cells. Its an attractive pitch for many patients, even though some of the clinics charge $6,000 and up to participate.

Now, with a national debate raging over the future of one of the hottest frontiers in 21st-century medicine, a University of Minnesota bioethicist has taken center stage in questioning whether many of these services are legitimate.

You have these businesses that dont have meaningful clinical research going on, the Us Leigh Turner said in an interview. There is a risk for fraud, in that people may be charged thousands of dollars to get an intervention that has no chance of working.

Turner has emerged as a major critic of the clinics, some of which he says have flawed procedures that allow bias to distort the results of treatment studies. He also says allowing clinics to list studies can imply government approval, lending false legitimacy to marketing pitches.

My concern is that you basically take clinicaltrials.gov and transform it into a marketing platform, Turner said.

In the past, Turner has moved beyond academic criticism, reporting several clinics he considered questionable to regulators at the U.S. Food and Drug Administration.

One of them, Celltex Therapeutics Corp., received a warning letter from the FDA for its practices in 2012. The company eventually moved its stem-cell infusion operations to Mexico, but it pushed back against Turner in a complaint lodged with U President Eric Kaler.

Clinics have also fired back at Turners latest critical article, which appeared last month in the medical journal Regenerative Medicine.

Research scientist Duncan Ross of Florida-based Kimera Labs, which was identified in Turners July 19 article as advertising an undisclosed pay-for-participation stem-cell study on clinicaltrials.gov, has threatened legal action.

I encourage you to amend your publication or I am going to bring suit against the institution for defamation or slander, Ross wrote to Turner. I am going to lobby the journal for the retraction of this publication. I followed the letter of the FDA as it exists at this time and I am not going to have my name disparaged because of your lack of interest in due diligence.

Turner said no litigation has materialized, and an editor at Regenerative Medicine said no request for a retraction has been made.

Beverly Hills cosmetic surgeon Dr. Mark Berman who is co-medical director of the national Cell Surgical Network, another clinic group named in Turners article said academic researchers like Turner are misguided and out of touch with real-world medical needs.

We are not taking public funding and using it to our benefit while pursuing scientific excellence were actually trying to help our patients while learning about the treatments and the disease they have, Berman said in an e-mail to the Star Tribune. Frankly, I think this is much more ethical than a major university with billions of endowment dollars taking millions of dollars of taxpayer money so they can build new offices and laboratories to further the study of stem cells.

Stem cells are the undifferentiated raw cells in the body that have the ability to quickly produce copies of themselves and also change into other kinds of cells like bone, muscle and blood cells.

Hospitals have safely used stem-cell transplant procedures to treat cancers for decades, but the FDA has approved just one commercial stem-cell product to date, which is made from infant cord blood and can only be used to produce more blood cells.

One of the most common sources of stem cells in pay-to-participate studies is body fat, often obtained via liposuction and known as adipose-derived stem cells. The fact that a byproduct of liposuction can be turned into a potentially therapeutic substance helps explain why smaller clinics are often affiliated with or run by plastic surgeons and cosmetic surgery centers.

Some critics have called for more oversight by the FDA. FDA officials, for their part, have said they share the excitement over the theoretical promise of stem cells to treat or cure disease by converting into cell types needed by the patient. But the agency has sounded a cautionary tone over the profusion of stem-cell clinics and studies popping up around the country.

Studies so far have not reliably demonstrated the effectiveness of stem-cell treatments, even in some of the most systematically studied conditions, FDA officials wrote in the New England Journal of Medicine in March. This lack of evidence is worrisome.

Turner said that many of the studies advertised on clinicaltrials.gov dont seem geared to produce high-quality data that will be medically useful, especially when they involve open-label study designs, where doctors and patients know what treatments are given, and patients are paying out of their own pockets.

In most clinical trials, study subjects are not charged fees to participate. In contrast, individuals enrolled in what are often called pay-to-participate studies are charged thousands or tens of thousands of dollars, Turners report says. Pay-to-participate studies also risk amplifying placebo effects as a result of the sizable fees companies often charge research participants and the hyperbole used to promote such studies.

Allowing participants to know what treatments are given departs from a typical randomized, blind study in which the intervention is kept secret for a period of time. That practice reduces the risk of a placebo effect, which can run high in medical studies.

A 2010 analysis of studies of irritable bowel syndrome treatments found that nearly 40 percent of 8,400 patients experienced an improvement in their symptoms even though they didnt receive the drug being studied.

Tennessee resident Doug Oliver, a nationally known advocate for stem-cell research who says he was legally blind before his eyes were treated with stem cells, agreed that placebo-controlled trials are the best way to ensure that patients are really being helped by a treatment.

But he argues that the coming age of cellular medicine will also require a wider understanding of how medical evidence is generated.

Oliver said most stem-cell clinics are trying to do the right thing, and many people feel that it wasnt even possible to get true FDA oversight of a stem-cell clinical trial before the signing of the 21st Century Cures Act last December. But even Oliver acknowledges that some clinics have exploited clinicaltrials.gov and disregarded any form of regulation thus far.

You have a group of clinics, which I think is a minority, maybe 20 percent, who are ill-intended, unqualified, or there is a personal and cultural aversion to doing anything that even smacks of following a regulation, he said. There are a number of clinics out there like that, and they are hurting people and they should be shut down.

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University of Minnesota bioethicist takes on clinics touting stem-cell studies - Minneapolis Star Tribune

Medical Mystery: Golda Meir’s midnight doctor visits – Philly.com

Golda Meir, Israels first and only femaleprime minister, once was voted the most admired woman in America. Her life took her from Ukraine to Milwaukee to Palestine before she fought for Israels autonomy, signed its declaration of independence, and led it through tumultuous times.

Her power was so legendary that the American feminist movement in 1970 used her image on a poster bearing the caption, But Can She Type?, swiping at sexist assumptions that used to send women with all kinds of qualifications to work in the typing pool.

But behind that image of strength was a woman with serious health problems, which she worked hard to conceal, trying to save her new nation from additional worries.

Meir was born GoldieMabovitch in Kiev, present-day Ukraine, on May 3, 1898. She emigrated as a child with her mother and two sisters in 1906 from pogrom-torn Pinsk, Ukraine, to meet her father, who was by then a storekeeper in Milwaukee. As a teen, she became a Zionist, and graduated from teachers college at what is now the University of Wisconsin-Milwaukee.

She married Morris Meyerson, a committed socialist, and the couple quit their jobs to move to a kibbutz in Palestine in 1921. She thrived on the hard farm work of kibbutz life, and her leadership skills soon became clear. Starting as a kibbutz leader, Meir rose swiftly.

By 1938, she was the Jewish observer from Palestine at the conference called by President Franklin Roosevelt to consider the fate of Jews fleeing the Nazis. To her despair, most nations expressed only sympathy, rather than admitting refugees. Later, she told reporters, There is only one thing I hope to see before I die and that is that my people should not need expressions of sympathy anymore.

After Israels 1948 War ofIndependence, Meir became a member of the Knesset, the nations legislature. She then served as minister of labor (1949-1956) and then foreign minister in 1956 under prime minister David Ben-Gurion. Asked to adopt a Hebrew surname, she chose Meir, which means illuminator.

In March 1969, after Prime Minister Levi Eshkol died in office, Meir was chosen to take his place temporarily. Later that year, she was elected Israels first female prime minister, leading a coalition party. She remained in office until she resigned on June 3, 1974.

But her health had started to fail her years before then. She had lost consciousness at least twice while serving in the foreign ministry, once during a daylong political party meeting in Israel, and again during a bus journey in southern Africa. She even retired temporarily in 1966, citing ill health and exhaustion.

By the time she resigned as prime minister, she had served more than four years despite a continuing and lingering secret illness. She felt progressively weaker, and kept midnight appointments at Israels Hadassah Hospital, where a white blood cell abnormality was uncovered.

What was wrong with Meir, and why was she so secretive about her condition?

Golda Meir led her young nation through years of turmoil. In 1972, at the Munich Olympics, 11 Israeli Olympic team members taken hostage and killed. Then came the Yom Kippur War, which Meir described as her greatest crisis, when Egypt and Syria attacked and almost defeated Israel in October 1973. Many critics said that Meirs and Israels defenses were caught sleeping on the Day of Atonement, the holiest day on the Jewish calendar. Still, her party won the next election.

But on April 10, 1974, Meir retired, stating, It is beyond my strength to continue carrying this burden.

Four years later, in 1978, she died in Jerusalems Hadassah Hospital at the age of 80. For 15 years, she and her staff had concealed that she suffered from lymphoma, a cancer that starts in the bodys infection-fighting white blood cells, known as lymphocytes.

Symptoms and signs of lymphoma include swollen glands (lymph nodes), anemia, neutropenia (low white blood cell count), fatigue, weakness, gastrointestinal symptoms, weight loss, nausea, vomiting, diarrhea, neurological numbness, and even heart failure.

Lymphoma is divided into two categories: Hodgkin Lymphoma, which is one of the most treatable cancers, and non-Hodgkin Lymphoma, a diverse group of diseases, some of which are slow growing.

Modern treatment options for lymphoma include radiation, chemotherapy, immunotherapy, and stem cell transplants.

Allan B. Schwartz, M.D., is a professor of medicine in the Division of Nephrology & Hypertension at Drexel University College of Medicine.

Published: August 6, 2017 3:01 AM EDT

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Medical Mystery: Golda Meir's midnight doctor visits - Philly.com

Israeli Scientists Develop First Haploid Human Stem Cells – NoCamels – Israeli Innovation News (press release) (blog)

Israeli scientists have developed the first haploid human stem cells, a discovery that will change our understanding of human genetics and medical research.

Already being used to predict whether people are resistant to chemotherapy drugs, the finding earned Igo Sagi, a PhD student at the Hebrew University of Jerusalem, the 2017 Kaye Innovation Award.

The long-sought haploid

Most of the cells in our body are diploid, which means they carry two sets of chromosomes (the structure in which DNA is contained) one chromosome from each parent. Haploid cells, in contrast, contain only a single set of chromosomes.

Scientists have long been trying to develop haploid stem cells. It is an important area of research, as embryonic stem cells are able to grow into any cell in the human body; this makes them extremely useful for treatment of diseases.

Haploid cells in particular are a powerful discovery, as they allow for a much better understanding of the human genetic makeup. For example, in diploid cells, it is difficult to identify the effects of mutations in one chromosome because the other copy is normal and provides a backup. Haploid cells dont have this limitation.

SEE ALSO: Five Israeli Biotech Companies Using Stem Cells To Change The Face Of Medicine

Up until now, scientists have only succeeded in creating haploid embryonic stem cells in animals such as mice, rats, and monkeys. The research conducted by Igo Sagi was the first time anyone was able to successfully isolate and maintain human haploid embryonic stem cells. These haploid stem cells were able to turn into many other cell types, such as brain, heart, and pancreas, while still retaining a single set of chromosomes.

The benefits are immense. Professor Nissim Benvenisty, who worked with Sagi on the research, explained: It will aid our understanding of human development for example, why we reproduce sexually instead of from a single parent. It will make genetic screening easier and more precise, by allowing the examination of single sets of chromosomes. And it is already enabling the study of resistance to chemotherapy drugs, with implications for cancer therapy.

SEE ALSO: Biological Breakthrough: Researchers Succeed In Creating Human Egg and Sperm Cells In Lab

Haploid Human Embryonic Stem Cells

Diagnosis of Chemotherapy Resistance

Based on this research, Yissum, the Technology Transfer arm of the Hebrew University, launched the company NewStem. The company is currently developing a diagnostic kit that can predict resistance to chemotherapy drugs. The large library of human haploid stem cells they are amassing will allow them to provide therapeutic and reproductive products, as well as personalized medication.

The haploid stem cells were developing have the potential to change the face of medical research as they hold a pivotal role in regenerative medicine, disease therapy and cancer research, revealed CEO of NewStem, Ayelet Dilion-Mashiah.

The research was conducted by Igo Sagi, a doctoral student at the Hebrew University of Jerusalem, along with Professor Nissim Benvenisty, Director of the Azrieli Center for Stem Cells and Genetic Research at the Hebrew University. The Kaye Innovation Awards at the Hebrew University of Jerusalem have been awarded annually since 1994 with the goal of encouraging academics to develop innovative methods and inventions with good commercial potential.

Photo:Azrieli Center for Stem Cells and Genetic Research at Hebrew University

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Israeli Scientists Develop First Haploid Human Stem Cells - NoCamels - Israeli Innovation News (press release) (blog)

Early gene-editing holds promise for preventing inherited diseases – The Jerusalem Post

The secret to healing what ails you lies within your own DNA. (photo credit:DREAMSTIME)

Scientists have, for the first time, corrected a disease-causing mutation in early-stage human embryos using gene editing.

The technique, which uses the CRISPR- Cas9 system, corrected the mutation for a heart condition at the earliest stage of embryonic development so that the defect would not be passed on to future generations.

It could pave the way for improved in vitro fertilization outcomes as well as eventual cures for some thousands of diseases caused by mutations in single genes.

The breakthrough and accomplishment by American and Korean scientists, was recently explained in the journal Nature. Its a collaboration between the Salk Institute, Oregon Health and Science University and South Koreas Institute for Basic Science.

Thanks to advances in stem cell technologies and gene editing, we are finally starting to address disease-causing mutations that impact potentially millions of people, said Prof. Juan Carlos Izpisua Belmonte of Salks gene expression lab and a corresponding author of the paper. Gene editing is still in its infancy, so even though this preliminary effort was found to be safe and effective, it is crucial that we continue to proceed with the utmost caution, paying the highest attention to ethical considerations.

Though gene-editing tools have the power to potentially cure a number of diseases, scientists have proceeded cautiously partly to avoid introducing unintended mutations into the germ line (cells that become eggs or sperm).

Izpisua Belmonte is uniquely qualified to speak on the ethics of genome editing because, as a member of the Committee on Human Gene Editing at the US National Academies of Sciences, Engineering and Medicine, he helped author the 2016 roadmap Human Genome Editing: Science, Ethics and Governance.

Hypertrophic cardiomyopathy is the most common cause of sudden death in otherwise healthy young athletes, and affects approximately one in 500 people. It is caused by a dominant mutation in the MYBPC3 gene, but often goes undetected until it is too late. Since people with a mutant copy of the MYBPC3 gene have a 50% chance of passing it on to their own children, being able to correct the mutation in embryos would prevent the disease not only in affected children but also in their descendants.

The researchers generated induced pluripotent stem cells from a skin biopsy donated by a male with Hypertrophic cardiomyopathy and developed a gene-editing strategy based on CRISPR-Cas9 that would specifically target the mutated copy of the MYBPC3 gene for repair. The targeted mutated MYBPC3 gene was cut by the Cas9 enzyme, allowing the donors cells own DNA -repair mechanisms to fix the mutation during the next round of cell division by using either a synthetic DNA sequence or the non-mutated copy of MYBPC3 gene as a template.

Using IVF techniques, the researchers injected the best-performing gene-editing components into healthy donor eggs that are newly fertilized with donors sperm. All the cells in the early embryos are then analyzed at single-cell resolution to see how effectively the mutation was repaired.

They were surprised by the safety and efficiency of the method. Not only were a high percentage of embryonic cells get fixed, but also gene correction didnt induce any detectable off-target mutations and genome instability major concerns for gene editing.

The researchers also developed an effective strategy to ensure the repair occurred consistently in all the cells of the embryo, as incomplete repairs can lead to some cells continuing to carry the mutation.

Even though the success rate in patient cells cultured in a dish was low, we saw that the gene correction seems to be very robust in embryos of which one copy of the MYBPC3 gene is mutated, said Jun Wu, a Salk staff scientist and one of the authors.

This was in part because, after CRISPR- Cas9 mediated enzymatic cutting of the mutated gene copy, the embryo initiated its own repairs. Instead of using the provided synthetic DNA template, the team surprisingly found that the embryo preferentially used the available healthy copy of the gene to repair the mutated part.

Our technology successfully repairs the disease-causing gene mutation by taking advantage of a DNA repair response unique to early embryos, said Wu.

The authors emphasized that although promising, these are very preliminary results and more research will need to be done to ensure no unintended effects occur.

Our results demonstrate the great potential of embryonic gene editing, but we must continue to realistically assess the risks as well as the benefits, they added.

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Early gene-editing holds promise for preventing inherited diseases - The Jerusalem Post

For Redskins Coach Jay Gruden, in some cases, less is more – The … – Washington Post

RICHMOND Here at Camp Svelte, the Washington Redskins boast many fitness feats. Trent Williams, the star left tackle, went (somewhat) vegan and lost his jolly cushioning. Junior Galette, the snakebit linebacker, dropped 24 pounds after recovering from two Achilles tendon tears that robbed him of the past two seasons. Rob Kelley, the running back wrongfully nicknamed Fat Rob as a child, now looks like he ought to go by Non-Fat Rob.

In the background stands Coach Jay Gruden, celebrating his players commitment and flashing an aw-shucks grin when hes asked about his own transformation. Gruden is quietly disappearing, too. You see it in the cheekbones resurfacing on his face. You see it in the absence of his tummy, which must have been shipped to the Bermuda Triangle.

At the end of last season, Gruden weighed 241 pounds. On Monday, he reported proudly that hes down to 218. Its the lightest he has been since 1991, when he was a 24-year-old, 215-pound quarterback starting his Arena Football League career with the Tampa Bay Storm.

You want to know how I did it? Gruden said, smiling. Youre going to have to pay extra for that, man. Youre going to have to YouTube my video, and its going to cost you $39.95.

[After Redskins chaos and Super Bowl collapse, Kyle Shanahan is ready to fix the 49ers]

Gruden will get to the how later. Its the why that matters most to him.

While his players alter their bodies to compete in a game full of world-class athletes, Gruden has a more relatable motivation. He was aching because of the extra weight on his 6-foot-2 frame. He blood pressure was high. He was a little embarrassed. He turned 50 in March, and he was tired of making excuses. He needed to do something or risk slipping from husky to obese.

I just didnt feel healthy, Gruden said. I hit 50 years old, and maybe it was part midlife crisis. I dont know. Who knows? You start looking around, and people are walking the streets, and you see people that are your age, and they look better and younger and healthier. And youre like, [Expletive], I shouldnt be this big.

Two years ago, CBS Sports radio host Scott Ferrall called Gruden a fat ass on the air. After learning of the cheap shot, Gruden referenced it during his next news conference. The comical coach tried to have fun with it, but the words bothered him.

I really dislike the guy that called me a fat ass, Gruden said then with a laugh. That really ticked me off. I dont mind you critiquing my coaching style, but to make fun of my weight, thats unfair. Im only 225.

As he recalled the exchange last week, Gruden was still upset.

Ive never considered myself, like, fat, he said. I know Im thick and a little heavy at times.

(Lee Powell/The Washington Post)

A more persuasive comment came at the end of last season. Anthony Lanier, a young defensive end who needs to add weight and strength to realize his potential, was honest with Gruden.

Man, Coach, youre too big right now, Lanier said.

The coach and player made a bet. Gruden vowed to lose as much weight, if not more, than Lanier gained. Gruden won. Hes down 23 pounds. Lanier is up 22.

But hes not paying me, Gruden said, jokingly cursing at the player. I told him Ill take the money when he gets his next contract.

[If Colin Kaepernick played basketball, the NBA would embrace him]

Gruden wont charge $39.95 for his weight-loss secrets because he didnt really do anything special. Over the past few months, he has paid more attention to what hes eating and begun exercising. Thats it, basically. He started by supplementing his breakfast with a shot of apple cider vinegar in the morning. That helped him lose four pounds in the first week or so. Since then, he has been committed to a balanced diet and regular exercise. He has been amazed at the results.

I did it because I was sore all over, Gruden said. My joints were sore. My ankle, my knee. Ive had shoulder issues. And Ive used that as an excuse for not working out or exercising. Then I lost an initial four pounds. I felt a lot better, and then I kept going. I started eating better, watching portion control, not eating late at night. And then the more I lost, the better my joints felt, so I was able to exercise more. Its amazing.

For years, Gruden thought football multiple injuries, multiple surgeries was to blame for his aches. He would wake up some mornings and struggle to walk to the bathroom because his Achilles tendon was sore. He had platelet-rich plasma injections in his knees. He took anti-inflammatory medication to get through the daily grind of coaching. But now that he is almost back to his playing weight, he feels good.

It sounds easy, but in a reality all too familiar to many people (myself included), its easier to remain unaware and mindlessly punish your body by indulging in the wrong foods and save the workouts for another day. Despite being a former high-level athlete, Gruden succumbed to bad habits.

Food is always available at the teams practice facility in Ashburn, and Gruden munched often. His job is active for about two hours of practice, but the bulk of his day is spent in meetings and sitting in rooms watching film late into the night.

The choices that you have at lunch and dinner are pretty big at our place, Gruden said. I would go out and have a sensible lunch, and then wed always have pizza there, and Id take a couple of pieces of pizza, and Id take a cookie upstairs.

Now I just have a small plate and I get the hell out of there, as much as I want to eat the pizza because its so damn good.

During training camp, Gruden used to bike 3 miles from the hotel in Richmond to the teams site. This year, he started walking every morning. Then he advanced to jogging and walking. On Monday, he ran the entire way for the first time. Earlier in camp, he played quarterback and danced in the pocket for the entirety of a long pass rush drill. Hes not a coach who has to stand back and evaluate anymore.

Last year, I couldnt do any of that, Gruden said. I didnt do any of that hardly because I was sore. Last year, I couldnt even walk from the hotel to work. I never even tried walking it. Now I ran the whole way, and I didnt stop. Thats how good my joints feel.

Gruden looked away and said softly, Lets hope I dont put it back on because its easy to put back on.

Acknowledging the threat is the first step to resistance. Besides, at Camp Svelte, there is ample peer pressure to keep Skinny Gruden or, better yet, Healthy Gruden motivated.

For more by Jerry Brewer, visit washingtonpost.com/brewer.

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For Redskins Coach Jay Gruden, in some cases, less is more - The ... - Washington Post

Proton Therapy: How It Could Change The Outcome of Paediatric Cancer – HuffPost

Today, cancer is no longer on the list of incurable diseases, thanks to medical developments in the last few decades. Yet, it is hard to ignore the fact that cancer does alter the lives of patients. We know of many adults that have undergone not just physical but an emotional overhaul during their cancer journey - some of them returning to life with an altered perspective and unending empathy for people around them after having been through the trials and trauma of chemotherapy and radiation.

For children, it might be a totally different story. For many of them, the effects of cancer treatment do not begin until later in life resulting in other health concerns. In fact, studying these late effects is the research focus of many medical researchers in the field of pediatric cancer. Many of these effects are the result of how chemotherapy works - in order to kills the cancerous cells, it destroys a good number of healthy cells as collateral damage.

I recently spoke with Dr. Ramesh Rengan, medical director of Seattle Cancer Care Alliance (SCCA) Proton Therapy Center in Seattle, Washington, and a long-term researcher in the field of novel radiation approaches with immune therapies in the treatment of cancer. Dr. Rengan is an active voice for proton therapy emerging as an increasingly effective and less harmful way to treat specific types of cancers, particularly pediatric cancers and those that occur close to vital organs.

Here are a few excerpts from our interview:

The SCCA Proton center works on an alternative treatment method for cancer. Can you tell us more about it?

At the SCCA Proton Therapy Center, we use highly targeted proton beam radiation to treat cancer. By precisely focusing on the tumor, proton therapy minimizes radiation exposure to surrounding healthy tissue, thereby reducing the risk of short- and long-term radiation-related side effects. Our center is currently the only proton therapy center in the Pacific Northwest.

How does proton therapy work?

The fundamental concept underlying all cancer treatment is to deliver a lethally effective treatment to the cancer and only the cancer, be it chemotherapy, surgery or radiation. The challenge for the oncologist is to design a way to deliver this treatment with specificity to the tumor while minimizing the damage to the surrounding organs. What really dictates the long-term clinical outcome for the patient is not just the number of cancer cells that we kill but the balance between that and the number of healthy cells that are injured in the course of treatment.

Standard radiation uses X-ray beams that enter and pass through the patient to the tumor and exit the other side. However, because X-rays penetrate so well, all tissue that lies in front of and beyond the tumor is exposed to radiation, which can be potentially harmful to the patient.

Protons, due to their mass, stop within the patient and do not continue to pass through the body. We can therefore calibrate the proton beam to stop within the tumor itself. Additionally, due to their positive charge, protons deposit most of their radiation at the point where they stop, rather than near the point of entry into the patient as X-rays do. As a result of these properties, protons concentrate the radiation dose delivery to the tumor itself. This more targeted form of radiation is especially useful for pediatric cancers, where any excess radiation exposure to healthy, developing organs can be potentially harmful.

What are the benefits of proton therapy over other types of cancer treatments?

Protons allow radiation to be delivered to the tumor while significantly minimizing collateral radiation exposure to surrounding healthy tissue. That benefit pays dividends in the near term because it reduces exposure, and it drives additional future benefits as patients continue with their lives after treatment, such as fewer secondary cancers. The precision that protons allow is particularly beneficial for patients whose tumors are near critical organs or structures, such as the brain, bladder, rectum, heart or spinal cord; patients whose cancers have recurred after initial radiation treatment; and patients whose organs are particularly sensitive to radiation exposure, such as children and adults with certain genetic syndromes.

For what kind of cancers is the proton therapy most effective ?

Although protons can be used to treat most cancers that require radiation, this treatment has been best established for tumors that lie in close proximity to radiosensitive vital organs, such as central nervous system tumors and eye tumors. For pediatric patients, protons have become an indispensable tool, as children are particularly vulnerable to the negative effects of radiation. Therefore, proton treatment for many pediatric tumors is a well-established standard of care, despite the limited access to proton therapy centers worldwide. Today, we also treat tumors in the head and neck, lung, breast, esophagus and gastrointestinal system, as these tumors are often situated next to radiosensitive vital organs. As such, the role of protons is being actively investigated in ongoing clinical trials in these disease locations.

Are protons a stand-alone treatment, or is it best used in conjunction with other therapies?

Proton therapy can often be used in combination with chemotherapy and surgery, similar to standard radiation treatment.

How is proton therapy improving?

Access to clinics, lowered cost, optimized technology to treat a broader range of tumors and use of a combination of approaches to treatment are the biggest recent leaps we have made with proton treatment.

Its important to understand that protons initially were postulated in the treatment of cancer in a paper published in 1946. Protons are not new as a modality, having been around since the 1950s. It has taken a long time for the technology to become relatively cost-efficient and be feasibly delivered in a hospital-based or stand-alone outpatient clinical setting such as the Seattle Cancer Care Alliance Proton Therapy Center.

Growth has been deliberate and incremental: the first hospital-based proton center opened in the 90s. Today there are 20 proton centers in North America, but the real promise of proton treatment is being realized now. During the next 10 years, we expect to see that number triple or quadruple, as the cost of building a center has been rapidly decreasing while proton beam technology has been simultaneously improving.

Are protons the best choice for everyone?

Protons are just as effective as X-rays in killing cancer cells but generally require less exposure to surrounding healthy tissues in order to deliver this treatment to the cancer, so they are a potential option whenever radiation is called for in the curative treatment of cancer. However, protons are only a single tool in the ever-increasing arsenal against cancer. As such, protons are the best treatment for some patients, but not for others. The decision regarding whether protons are the right choice for a given patient is made by a proton-experienced radiation oncologist in conjunction with members of the multidisciplinary cancer care team and the patient.

What do you believe are the next big revolutions in cancer treatments?

The big revolution in cancer treatment will come from our increasing ability to deliver effective therapies to the tumor with absolute specificity. For example, that is the founding principle behind cancer immunotherapy, namely, to turn our immune system against the cancer. Similarly, the greater our ability to focus our radiation beam or our scalpels at purely the cancer and minimize the impact to the surrounding organs, the greater the benefit to our patients.

In the past few years we have made fundamental paradigm shifts in the way we treat cancer. Today we attack cancer by exploiting the unique genetic characteristics of the tumor to design drugs that attack the cancer by homing in on their gene signature. Even with these modern advancements, it must be mentioned that cancer is not just treated with one method alone; a combination of weapons is used to effectively cure most cancers. One might say that it takes a village to effectively treat a cancer patient.

Much of the early history of cancer treatment had focused on maximizing the lethality of our weapons against the cancer, either by increasing the radiation or chemotherapy dose or by performing larger and more radical surgery to remove the tumor. We now recognize that the impact of these treatments on healthy tissue can have an even greater negative effect on the clinical outcome than the positive benefit of killing the tumor. In short, we dont need a bigger gun or bomb, but rather a smarter bomb that equally emphasizes supporting the health of the patient and the destruction of the cancer. Protons are ideally suited to this evolution in cancer care.

Devishobhais the founder ofKidskintha,a happy place to jumpstart conversations around family and millennial parenting, living in India. You can find her voiceon the Huffington Post,LifeHack, Parent.co,Addicted2Success, Inc.com, Entrepreneur, Tiny Buddha,SivanaEast and others on a range of topics. You can get yourself equipped for happy parenting with one hack a week for an entire year (each one backed by science).

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Proton Therapy: How It Could Change The Outcome of Paediatric Cancer - HuffPost

Wilson’s Disease Market: Unmet Needs of Patient Population to Inspire Players for Improved Treatment Options – Edition Truth

The global Wilsons disease market is driven by the development of gene therapy as a potential cure for this disease. As per the European Association, approximately 6 to 12 percent of the total patient population suffering from liver failure is affected by Wilsons disease, and this in turn will drive a demand for their treatment. It has also been found that females with liver failure are more prone to Wilsons disease. In addition to this, the risk of Wilsons disease is high among patients with low hemoglobin, jaundice, and low cholinesterase.

As patients suffering from Wilsons Disease are unable to discharge copper at a normal rate from their liver on account of the mutation of the ATP7B gene, its treatment becomes essential. One of the key challenges of this market is the under-diagnosis of this disease. A lack of safety in the treatment offered is also another factor challenging the market. The current treatments show poor compliance and this is posing another challenge. However, the unmet need for efficient treatment for Wilsons disease can be viewed by market players as a scope for vigorous improvement and newer discoveries can be made by doubling their efforts in research and development.

The report segments the global Wilsons disease market on the basis of treatment, end-user, geography, and indication. By treatment, the market is segmented into penicillamine, zinc, and trientine. Of these, penicillamine continues to enjoy highest popularity as treatment option. On the basis of indication, the global Wilsons disease market can be segmented into psychiatric, hepatic, ophthalmic, and neurological. On the basis of end-user, the market can be segmented into specialty clinics, diagnostic laboratories, and hospitals.

On the basis of geography, the market is segmented into Asia Pacific, Europe, Latin America, North America, and the Middle East and Africa. Of these, North America is leading in the Wilsons disease market on account of growing patient awareness and increasing demand for various treatment options. Europe is anticipated to trail next. The increasing population and growing demand for quick diagnosis will boost the market in the region. The demand for improved and efficient treatment will also help the market players to strive for better discoveries and developments. The growing medical tourism in developing nations of Asia Pacific will also help the market to grow in the region.

Some of the leading players operating in the global Wilsons disease market are Meda Pharmaceuticals, Inc., Valeant Pharmaceuticals International, Inc., Teva Pharmaceuticals, Ipsen, Taj Pharmaceutical Limited, and Wilson Therapeutics.

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Wilson's Disease Market: Unmet Needs of Patient Population to Inspire Players for Improved Treatment Options - Edition Truth

John Theurer Cancer Center and MedStar Georgetown University … – Business Wire (press release)

WASHINGTON & HACKENSACK, N.J.--(BUSINESS WIRE)--MedStar Georgetown University Hospital in Washington, D.C. in collaboration with John Theurer Cancer Center, part ofHackensack Meridian Health, Hackensack University Medical Center in Hackensack, N.J., announce the 100th blood stem cell transplant performed since the BMT programs first patient was treated in September, 2013.

The patient, a woman from Arlington, Virginia, received her blood stem cell transplant at MedStar Georgetown as a treatment for multiple myeloma diagnosed in December 2016.

The BMT program at MedStar Georgetown is a joint effort with specialists from Hackensack John Theurer Cancer Center and a key component of the Lombardi Comprehensive Cancer Center, the only cancer program in the Washington, D.C. region designated by the National Cancer Institute (NCI) as a comprehensive cancer center.

Once considered experimental, BMT is todays established gold standard for treating patients with a number of malignant and other non-malignant diseases of the immune system, blood, and bone marrow, including multiple myeloma, lymphoma, and acute and chronic leukemia. For some conditions, blood stem cell transplant can provide a cure in patients who have failed conventional therapies, says Scott Rowley, MD, chief of the BMT program at MedStar Georgetown as well as a member of the John Theurer Cancer Centers Blood and Marrow Stem Cell Transplantation. For some conditions, it can actually be a cure; for others, it prolongs survival and improves quality of life. Having performed 100 BMTs at MedStar Georgetown including allogenic transplantation illustrates the strength and maturity of our program achieved in rather short time.

MedStar Georgetowns program is also the only comprehensive BMT center within Washington, D.C. and southern Maryland with accreditation from the Foundation for the Accreditation of Cellular Therapy (FACT) for adult autologous procedures, where the patient donates his or her own cells.

The BMT program at JTCC is one of the top 10 transplant programs in the United States, with more than 400 transplants performed annually.

A BMT involves a two-step process: first, collecting bone marrow stem cells from the patient and storing them for future use. Then, a week or so later, patients receive high dose chemotherapy to eliminate their disease. The previously stored cells are reinfused back into the bloodstream, where after reaching the bone marrow, they begin repopulating and allow the patient to recover their blood counts over the following 2 weeks.

Even though BMT is considered standard therapy for myeloma worldwide, in the United States fewer than 50 percent of the patients who could benefit from BMT are referred for evaluation, says David H. Vesole, MD, PhD, Co- Chief and Director of Research of John Theurer Cancer Centers Multiple Myeloma division and director of MedStar Georgetowns Multiple Myeloma Program.

Thats mostly due to physicians concerns that a patient is too old or compromised from other health conditions like diabetes, cardiac disease or renal failure. But new techniques and better supportive care have improved both patient outcomes and the entire transplant process, extending BMT to more patients than ever before.

The MedStar Georgetown/Georgetown Lombardi Blood and Marrow Stem Cell Transplant Program is part of a collaborative cancer research agenda and multi-year plan to form an NCI-recognized cancer consortium. This recognition would support the scientific excellence of the two centers and highlight their capability to integrate multi-disciplinary, collaborative research approaches to focus on all the aspects of cancer.

The research areas include expansion of clinical bone marrow transplant research; clinical study of haplo transplants use of half-matched stem cell donor cells; re-engineering the function and focus of key immune cells; and the investigation of immune checkpoint blocking antibodies that unleash a sustained immune response against cancer cells.

In this partnership, weve combined John Theurers strength in clinical care with Georgetown Lombardis strong research base that significantly contributes to clinical excellence at MedStar Georgetown. By working together, we have broadened our cancer research to offer more effective treatment options for tomorrows patients, says Andrew Pecora, MD, FACP, CPE, president of the Physician Enterprise and chief innovations officer, Hackensack Meridian Health. This is one of many clinical and research areas that have been enhanced by this affiliation.

Our teams are pursuing specific joint research projects we feel are of the utmost importance and significance in oncology particularly around immuno-oncology as well as precision medicine, says Andr Goy, MD, MS, chairman of the John Theurer Cancer Center and director of the division chief of Lymphoma; chief science officer and director of Research and Innovation, RCCA; professor of medicine, Georgetown University. Together our institutions have a tremendous opportunity to transform the delivery of cancer care for our patient populations and beyond.

ABOUT THE JOHN THEURER CANCER CENTER AT HACKENSACK UNIVERSITY MEDICAL CENTER

John Theurer Cancer Center at Hackensack University Medical Center is New Jerseys largest and most comprehensive center dedicated to the diagnosis, treatment, management, research, screenings, and preventive care as well as survivorship of patients with all types of cancers. The 14 specialized divisions covering the complete spectrum of cancer care have developed a close-knit team of medical, research, nursing, and support staff with specialized expertise that translates into more advanced, focused care for all patients. Each year, more people in the New Jersey/New York metropolitan area turn to the John Theurer Cancer Center for cancer care than to any other facility in New Jersey. Housed within a 775-bed not-for-profit teaching, tertiary care, and research hospital, the John Theurer Cancer Center provides state-of-the-art technological advances, compassionate care, research innovations, medical expertise, and a full range of aftercare services that distinguish the John Theurer Cancer Center from other facilities.www.jtcancercenter.org.

MedStar Georgetown University Hospital is a not-for-profit, acute-care teaching and research hospital with 609 beds located in Northwest Washington, D.C. Founded in the Jesuit principle of cura personaliscaring for the whole personMedStar Georgetown is committed to offering a variety of innovative diagnostic and treatment options within a trusting and compassionate environment.

MedStar Georgetowns centers of excellence include neurosciences, transplant, cancer and gastroenterology. Along with Magnet nurses, internationally recognized physicians, advanced research and cutting-edge technologies, MedStar Georgetowns healthcare professionals have a reputation for medical excellence and leadership.

For more information please visit: medstargeorgetown.org/bmsct

About Hackensack Meridian Health Hackensack University Medical Center

Hackensack Meridian Health Hackensack University Medical Center, a 775-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the countys first hospital, it is now part of one of the largest networks in the state comprised of 28,000 team members and more than 6,000 physicians. Hackensack University Medical Center was listed as the number one hospital in New Jersey in U.S. News & World Reports 2016-17 Best Hospital rankings - maintaining its place atop the NJ rankings since the rating system was introduced. It was also named one of the top four New York Metro Area hospitals. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades Americas 50 Best Hospitals Award for five or more years in a row. Beckers Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2017. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 25 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its fifth consecutive designation in 2014. Hackensack University Medical Center has created an entire campus of award-winning care, including: the John Theurer Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Womens and Childrens Pavilion, which houses the Joseph M. Sanzari Childrens Hospital and Donna A. Sanzari Womens Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guides list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to The Northern Trust PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Childrens Cancer Institute housed at the Joseph M. Sanzari Childrens Hospital. To learn more, visit http://www.HackensackUMC.org.

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John Theurer Cancer Center and MedStar Georgetown University ... - Business Wire (press release)

Lung fibrosis? Stem cell therapy holds promise – The Hindu

A team of scientists from the UNC School of Medicine and North Carolina State University (NCSU), U.S. have developed promising research towards possible stem cell treatment for several lung conditions, such as idiopathic pulmonary fibrosis (IPF), chronic obstructive pulmonary disease (COPD), and cystic fibrosis, all of which are known to be fatal conditions. In the journal Respiratory Research, the scientists demonstrated that they could harvest lung stem cells from people using a relatively non-invasive, doctors office technique. They were then able to multiply the harvested lung cells in the lab to yield enough cells sufficient for human therapy.

In a second study, published in the journal Stem Cells Translational Medicine, the team showed that in rodents they could use the same type of lung cell to successfully treat a model of IPF a chronic, irreversible, and ultimately fatal disease characterised by a progressive decline in lung function. These diseases of the lung involve the build-up of fibrous, scar-like tissue, typically due to chronic lung inflammation. As this fibrous tissue replaces working lung tissue, the lungs become less able to transfer oxygen to the blood. Patients ultimately are at risk of early death from respiratory failure. In the case of IPF, which has been linked to smoking, most patients live for fewer than five years after diagnosis.

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Lung fibrosis? Stem cell therapy holds promise - The Hindu

From one cell to billions: Juan Carlos Izpisa Belmonte studies all stages of life – The San Diego Union-Tribune

Salk Institute researcher Juan Carlos Izpisa Belmonte appears to be everywhere in the field of stem cells and regenerative medicine.

He has helped to invent a new kind of stem cell, performed groundbreaking research in growing human organs in animals and demonstrated in mice how to reverse signs of aging.

In addition, his research has produced mini-kidney organoids from stem cells, pointed to a novel way of preventing mitochondrial disease and most recently, helped to prove that a genetic defect can be efficiently repaired in human embryos. The production of mini-kidneys was chosen by Science magazine in 2013 as a runner-up for its Breakthrough of the Year honor.

Izpisa Belmonte tackles the most difficult problems from conception to old age, including aging itself. Its an interest carried from his youth in Spain, when he became fascinated by the regenerative powers of some animals, powers he would like to confer on humanity.

I am a basic developmental biologist at heart, he said by email from Spain, where he performs research in addition to his Salk lab in La Jolla.

My passion is to study and understand the development of an organism the process by which, after conception, a single cell divides and generates billions of cells and a human being is created. This includes the study of degeneration and regeneration of our cells and organs.

Evan Snyder, a prominent stem cell researcher at the Sanford Burnham Prebys Medical Discovery Institute who has known Izpisa Belmonte since 2003, said: He knows what will be the important questions to answer. The very fact that he can answer them so quickly after theyve been posed means hes been thinking about them already.

Izpisa Belmonte maintains a huge variety of projects in collaboration with colleagues in Europe, the United States and Asia. Snyder and other peers said he succeeds by harnessing imagination with good organizational skills and persistence.

And somehow, while succeeding in the highly competitive field of science, Izpisa Belmonte maintains a courtly, Old World grace and charm, and even humility.

I would say Juan Carlos is one of the most gentle, self-effacing, collaborative, genuinely nice people Ive ever met, Snyder said.

In one instance, Snyder recalled witnessing Izpisa Belmonte encounter a scientist he had beaten to the punch with a discovery. He apologized for having done that and simply said the work just went quicker than expected, Snyder said. It was one of the sweetest things Ive ever seen.

Jun Wu, a Salk researcher in Izpisa Belmontes lab, co-authored the recent study on repairing genetic defects in human embryos. Wu said his first meeting with Izpisa Belmonte was memorable because of that tact and consideration, along with helpful advice.

Hes a good listener, Wu said. He listened to what I had to say about my project. And then he gave me some suggestions. He gave me a lot of freedom to think in my own way, but at the same time he gave me the proper guidance to where I should go.

In other words, Izpisa Belmonte maintains an open-mindedness as a person and scientist, Wu said. Many scientists specialize in particular areas, using a fixed pattern of investigation. Izpisa Belmonte encourages his researchers to explore different paths, and if that path turns out to be wrong, to think about it and learn from that experience.

As scientists, we need to be critical of ourselves, while at the same time we need to be open to the possibility that other people criticizing or saying negative things about us could be right, Wu said.

Izpisa Belmonte was born in 1960 in the small town of Helln in southwestern Spain.

I come from a modest, poor family, he said. My grandfather was a baker, my father left home when I was little and my mother worked in whatever she could to feed me and my brothers.

I left school very early to help earn money for my family by picking almonds, grapes, etc., during harvest time and working as a bellboy before I was even 14, the legal age at that time in my country to start working.

His chief role model was his mother, who taught me the spirit of hard work and fairness, he said.

I remember I used to read a lot, first comics and later on more serious things, mostly classical philosophy. I always remember having a special interest and curiosity to know more about the meaning of life.

But while reading philosophy, Izpisa Belmonte also entertained more worldly fantasies, particularly about being a soccer player.

I even managed to play on a professional soccer team, he said. However, just when I thought that I had a bright future as a player, the coach sat me on the substitutes bench for several months and I realized that perhaps I was not good enough. I then decided that my footballing dream was over and I decided to go back to school. I think the soccer coach that didnt let me play was very decisive for my scientific career.

For that career, Izpisa Belmonte traveled to the big city, Valencia, to enroll in college. Uncertain of his academic path, he chanced on pharmacology.

I am very satisfied with my decision, he said. What I learned there, especially in the subjects of biology and chemistry, was key for me to dedicate my life afterward to try to understand how life, a living organism, is generated.

He earned a series of degrees, including a Ph.D. from the University of Valencia and the University of Bologna in Italy. He did postdoctoral work in Germany and at UCLA.

As a young scientist, he focused on his first big question: How is an embryo formed from one cell, and how does it develop into a complete organism? From that question came early discoveries, including key genes and mechanisms for the formation of organs and limbs, and how they are arranged in the body.

Izpisa Belmonte arrived at the Salk Institute for Biological Studies in 1993, and is now a professor in the Gene Expression Lab. In 2011, he was named as the first holder of the Roger Guillemin Chair, endowed with $3 million by San Diego philanthropists Irwin and Joan Jacobs.

Guillemin, also a Salk professor, received the 1977 Nobel Prize for physiology or medicine.

Guillemin recalls that when he met Izpisa Belmonte, he was struck by the mans purposeful demeanor.

Immediately I knew that this was a very serious young man, that he knew what he was talking about, Guillemin said. From what I understand of the science, hes at the very frontier of whats possible in molecular biology.

In the most recent research, Izpisa Belmonte was one of five senior authors of a study that demonstrated in human embryos that a genetic defect can be corrected. The defect causes a heart disease called hypertrophic cardiomyopathy. It can cause sudden death in apparently healthy people, such as young athletes.

While the embryos werent allowed to develop for more than several days, the study provided proof of principle that such defects can be fixed right around the time of conception.

This is the type of problem that modern molecular biology can look into and become proficient at affecting, Guillemin said.

Last year, Izpisa Belmonte received a grant of at least $2.5 million over five years to study how to generate functional primate organs in pigs, using stem cell technologies he and colleagues developed.

The grant was a Pioneer Award from the National Institutes of Health, given to individual scientists of exceptional creativity who propose pioneering and highly innovative approaches with the potential to produce an unusually high impact on biomedical or behavioral research.

Getting organs of one species to grow in another is difficult, but Izpisa Belmonte said if tissues are implanted at the right time and under the right conditions, a host animals body could provide the proper signaling needed to direct organ development.

Early this year, a team including Izpisa Belmonte reported success in creating a rat-mouse mixture that grew functional organs. They did this by disabling a single gene in single-cell mouse embryos that was needed for development of a specific organ. They then added rat stem cells, which took the place of the vacant mouse organ. This method yielded functional rat eye, heart and pancreas tissue in the mouse.

They tried a somewhat similar experiment in pigs, which proved more difficult. Pigs gestate for about three months and three weeks, while human pregnancies extend for nine months. By experimenting with different kinds of human stem cells, the team finally produced human-pig embryos that grew for about three weeks before they were killed.

While both human and pig cells were present in the embryos, the rate of human cell survival was low. The research team is trying to overcome this challenge by selectively disabling genes in the pig embryos, as they did in the mouse embryos.

Along with the technical challenges, ethical considerations must be met.

For example, allowing human brain tissue to develop in a pig might produce an animal with a partially human consciousness. Thats not likely in the pig-human experiments, because there was no sign that the human cells ever got into the pig brains to begin with.

Izpisa Belmonte downplays his role in the various groundbreaking projects, preferring to give most of the credit to those in his lab.

My role is perhaps the least important of all in our team, he said. I am very fortunate to have many talented junior and senior research scientists working with me. They all have different backgrounds and expertise, not just scientifically, but also culturally, since many of them come from abroad. This has enabled us to think about a problem from different angles and see things more comprehensively.

Equally important, he said, is the scientific and administrative support at the Salk Institute, a true scientific paradise.

I have visited many other institutions and I feel that there is no place that can compare. Most of our best ideas and projects come through interactions with my faculty colleagues and learning from the incredible discoveries and conceptual advances they produce.

Most of his friends come from the world of science, Izpisa Belmonte said, expressing some regret that he let science interfere with some of his relationships.

I had a very close friend who died very young, and I didnt have many other friends in my youth, he said. Friendships are like plants; they need to be fertilized from time to time to grow well, and unfortunately, I did not dedicate time to this.

It was also difficult because I moved from country to country very often during my scientific pursuits. Now I have some colleagues that, after working with them, have become good friends.

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From one cell to billions: Juan Carlos Izpisa Belmonte studies all stages of life - The San Diego Union-Tribune