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Clinic helps women have babies after cancer – WNDU-TV

A specialized team of doctors is working to help cancer patients preserve their fertility and get treatment.

Ten months ago, the Seattle Cancer Care Alliance opened its clinic, staffed with experts in fertility and cancer, all in the same place.

Chenault is concealing her identity to protect her family's privacy.

She was diagnosed with Hodgkin's lymphoma but says maintaining her ability to have children was the most emotional part of her treatment.

"I had never really thought of a life where I wouldn't be able to have children and have a family. It's been important to me for a really long time," Chenault says.

Dr. Genevieve Neal-Perry runs the Oncoreproduction Clinic, a place where newly diagnosed women can come to freeze eggs, freeze embryos, even get Lupron to put them into temporary menopause until after chemo.

"You have a select population, and you can address their select needs and make sure that we can really provide them very direct and focused care," Neal-Perry explains.

The Livestrong Foundation offers financial aid, since insurance often won't cover fertility preservation.

Chenault chose to freeze two sets of embryos and get Lupron.

She just finished chemo and hopes to start talking about babies in six months to a year.

"I can't imagine how much harder this would have been without this response," Chenault says.

Both she and Dr. Neal-Perry say this gives patients some control in a situation where they may feel they have none.

"It really does give patients kind of a license to kind of fight, really move through the treatment and feel positive about the end of the tunnel," Neal-Perry says.

Cancer patients considering fertility preservation should ask a lot of questions about possible treatments and resources.

Both Livestrong and Walgreen's offer financial aid for meds.

MEDICAL BREAKTHROUGHS RESEARCH SUMMARY

TOPIC: MAKING BABIES AFTER CANCER

REPORT: MB #4641

BACKGROUND: Cancer treatments are important for your future health, but they may harm reproductive organs and glands that control fertility. Changes to fertility may be temporary or permanent. Chemotherapy (especially alkylating agents) can affect the ovaries, causing them to stop releasing eggs and estrogen. Radiation therapy to or near the abdomen, pelvis, or spine can harm nearby reproductive organs. Some organs, such as the ovaries, can often be protected by ovarian shielding or by oophoropexya procedure that surgically moves the ovaries away from the radiation area. Surgery for cancers of the reproductive system and for cancers in the pelvis region can harm nearby reproductive tissues and cause scarring, which can affect your fertility. Hormone therapy (also called endocrine therapy) used to treat cancer can disrupt the menstrual cycle, which may affect fertility. Side effects depend on the specific hormones used and may include hot flashes, night sweats, and vaginal dryness. Bone marrow transplants, peripheral blood stem cell transplants, and other stem cell transplants involve receiving high doses of chemotherapy and/or radiation. These treatments can damage the ovaries and may cause infertility. (Source: https://www.cancer.gov/about-cancer/treatment/side-effects/fertility-women)

SYMPTOMS: Effects to fertility may not be obvious right away. Some symptoms that may indicate infertility are irregular menstrual cycles, hot flashes, painful sex, inability to get pregnant or having several miscarriages. Patients who experience these symptoms should contact their doctor. (Source: https://www.livestrong.org/we-can-help/fertility-services/cancer-and-fertility-risks-women)

CHALLENGES: Genevieve S. Neal-Perry, MD, PhD from SCCA-UW Medicine talked about the struggles with treatment and maintaining fertility, "in many young people, having a full life means being able to be a parent and have children. As a result, some patients have pushback to say, no, my fertility is important. And as a result, doctors, physicians, scientists are listening and trying to identify what will that mean for their cancer? And several studies have shown that delaying treatment doesn't necessarily change the outcome of their cancer so that we're able to now, in addition to the advancement of science, freeze eggs, freeze embryos and give these young couples the opportunity to be parents later in life."(Source: Genevieve S. Neal-Perry, MD, PhD)

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Clinic helps women have babies after cancer - WNDU-TV

Fit and healthy teacher dies 20 days after discovering he had leukaemia at 33 – The Sun

WHEN teacher Matt Meads started suffering with stomach pains, night sweats and tiredness - he quickly dismissed it as end of school year fatigue.

He was fit and healthy, and avoided drinking and smoking.

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But just three weeks after first falling ill, Matt died - aged just 33 - and after just three chemo sessions.

He and his wife Abi were devastated when they discovered his symptoms were actually a sign ofleukaemia.

Heartbroken Abi, 27, from Nottingham, is now sharing his story to raise awareness and to urge others to be vigilant for signs of acute lymphoblastic leukaemia, a rare and aggressive form of blood cancer.

"I've got so many people around me offering help and support but I still feel really lonely because I've lost my best mate, my husband, my soulmate," Abi said.

It comes as blood cancer charity Bloodwise warns thousands of people are dying of the disease because it's diagnosed too late.

Matt and Abi first suspected something was wrong on July 6, when he began feeling more and more fatigued.

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And when he started vomiting to the point of being unable to keep ice cubes down, Abi urged him to go to the doctor.

At first he was told by his GP he had gastroenteritis or constipation but he soon returned to the hospital for a second time where they did a blood test.

Abi, who works as a teacher too, said: "Obviously I wish it was spotted sooner.

"I don't feel any anger towards to the hospital.

"I genuinely believe they did everything they could for him.

"He would say he was feeling sick."He would send a text saying he wasn't feeling well so was going to bed. I was out with some friends.

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"He said something about feeling hot as well, but we didn't put that down to anything because it was the middle of summer and everyone was feeling hot.

"He was sleeping a lot, particularly at weekends which he would spend mostly asleep.

"But we're both teachers, it was coming up to the end of the school year and we had both got a lot of work on.

"We put it down to the job, and just tried to keep going because we had five weeks off soon.

"We thought it was the usual fatigue that we feel at the end of the year.

"There were sickness bugs going around at both of our schools so it wasn't anything out of the ordinary.

"He was referred to A&E for the second time thinking it was gallstones.

I've got so many people around me offering help and support but I still feel really lonely because I've lost my best mate, my husband, my soulmate

"They did some blood tests on him, sent him for a CT scan. The doctor came back and basically said that it was leukaemia."

Despite the devastating diagnosis, Abi said her brave husband remained positive as he came to terms with what was really happening.

She added: "Matt was a really positive person and was always somebody who believed what would be would be, it is what it is and all that stuff.

"So when the doctor told him he was quite composed.

"He didn't really give anything away about what he was feeling.

"It was obviously a massive shock for him but he didn't really respond in the way I would have done. He was listening to the doctor.

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"The doctor actually stopped at one point because he was explaining what would happen next.

"He actually stopped at one point to ask him if he was okay, it was really big news and is he taking it all in?

"Matt's response was, 'yes, but there's nothing I can do about it. It is what it is'.

"He was definitely really brave."

And Abi says the diagnosis came as even more of a shock given how healthy he was.

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She was forced to watch her husband go from happy and sporty to intensive care within days, before he passed away from a pulmonary embolism after just three chemotherapy sessions.

She said: "We knew he was poorly, but maybe not quite how poorly he was.

"I certainly wasn't expecting a phone call from the hospital.

"I don't know how I am now. It's hard. I don't think I really started to grieve until after the funeral.

"It's hard to think ahead for anything. It's a case of taking everything one day at a time.

"Some days are better than others. Some days are horrific and I don't want to get out of bed.

What is Acute Lymphoblastic Leukaemia?

Acute lymphoblastic leukaemia (ALL) is a type of blood cancer that starts from young white blood cells called lymphocytes in the bone marrow.

Adults and children can get it but it is most often diagnosed in younger people.

It'svery rare, with around 650 people diagnosed with the condition each year in the UK.

Many symptoms of ALL are vague and non specific. It may feel like the flu as symptoms are caused by too many abnormal white blood cells and not enough normal white cells, red cells and platelets.

Symptoms can include:

Recently blood cancer charity Bloodwise warned thousands of patients in England could be unnecessarily dying from blood cancer because they are diagnosed too late.

Experts analysed NHSdata and found 28 per cent of patients are told they have the disease after needing emergency treatment for their symptoms.

Figures show there are around 40,000 cases of blood cancer - a group of diseases including leukaemia, lymphoma and myeloma - each year in the UK.

Around 77 per cent of patients will survive for three years if they are diagnosed after visiting their GP, Bloodwise says.

In contrast, the same survival rate plummets to just 40 per cent for patients who are diagnosed as an emergency.

This is because symptoms develop over a few weeks and become more severe as the number of immature white blood cells increases.

"Matt was a really happy person. He was really positive.

"He was kind, caring, loving, wicked sense of humour. He would make a joke about anything and was very quick-witted.

"As a teacher he would have done anything for his students. He would have done anything for his family.

"He was just a really positive person who would have done anything for anybody.

"He loved his sport. He would go to the gym, he loved being outside and walking. He liked cycling. He was careful about what he ate.

"Everything the doctors warn you about, he didn't do. He didn't drink, he didn't smoke, he had a good diet, he exercised.

If you've got any of the symptoms which are lasting or you can't explain why you've got them, you need to go to the doctor and get checked out and be persistent in asking for a blood test

"He always put sun-cream on because he was paranoid he might catch skin cancer or something.

"He did everything he could to try and prevent anything from happening to him.

"As the doctors said there was nothing he could have done to prevent this."

Abi is now speaking out to urge others to get checked out and insist for a blood test if they have persistent symptoms of blood cancer.

She said: "If you've got any of the symptoms which are lasting or you can't explain why you've got them, you need to go to the doctor and get checked out and be persistent in asking for a blood test.

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"You know your own body. It's as simple as having a blood test.

"If you've got it for days and it's not getting any better, if you're in any doubt, get it checked.

"We didn't know what the symptoms were.

"The only one I knew was bruising, but Matt didn't have any bruises until he was in hospital. So the one thing I knew wasn't relevant.

"I didn't realise about the night sweats, fatigue or heavy breathing.

"We never expected it would be that.

"We had thought worst case scenario it was gallstones or an impacted bowel, so when he came and said leukaemia it was just unexpected.

"When you're poorly you have all these possibilities going through your head but you never think it's going to be that.

Warning

terror vision I heard a pop and lost my eye when I dried my face with a towel

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"I had a really good chat with Matt's consultant where I questioned whether I should have done more, if I had spotted things sooner, whether if I had been more pushy in getting him to hospital.

"But the symptoms are vague and it can come on within days. It doesn't have to be something that has been happening for weeks or months."

You can donate on Abi's fundraising page in memory of Matt here.

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Fit and healthy teacher dies 20 days after discovering he had leukaemia at 33 - The Sun

New universe of miniproteins is upending cell biology and genetics – Science Magazine

By Mitch LeslieOct. 17, 2019 , 2:00 PM

Mice put human runners to shame. Despite taking puny strides, the rodents can log 10 kilometers or more per night on an exercise wheel. But the mice that muscle biologist Eric Olson of the University of Texas Southwestern Medical Center in Dallas and colleagues unveiled in 2015 stood out. On a treadmill, the mice could scurry up a steep 10% grade for about 90 minutes before faltering, 31% longer than other rodents. Those iron mice differed from counterparts in just one small waythe researchers had genetically altered the animals to lack one muscle protein. That was enough to unleash superior muscle performance. "It's like you've taken the brakes off," Olson says.

Just as startling was the nature of the crucial protein. Muscles house some gargantuan proteins. Dystrophin, a structural protein whose gene can carry mutations that cause muscular dystrophy, has more than 3600 amino acids. Titin, which acts like a spring to give muscles elasticity, is the biggest known protein, with more than 34,000 amino acids. The protein disabled in the mice has a paltry 46. Although researchers have probed how muscles work for more than 150 years, they had completely missed the huge impact this tiny protein, called myoregulin, has on muscle function.

Olson and his colleagues weren't the only ones to be blindsided by Lilliputian proteins. As scientists now realize, their initial rules for analyzing genomes discriminated against identifying those pint-size molecules. Now, broader criteria and better detection methods are uncovering minuscule proteins by the thousands, not just in mice, but in many other species, including humans. "For the first time, we are about to explore this universe of new proteins," says biochemist Jonathan Weissman of the University of California, San Francisco.

Biologists are just beginning to delve into the functions of those molecules, called microproteins, micropeptides, or miniproteins. But their small size seems to allow them to jam the intricate workings of larger proteins, inhibiting some cellular processes while unleashing others. Early findings suggest microproteins bolster the immune system, control destruction of faulty RNA molecules, protect bacteria from heat and cold, dictate when plants flower, and provide the toxic punch for many types of venom. "There's probably going to be small [proteins] involved in all biological processes. We just haven't looked for them before," says biochemist Alan Saghatelian of the Salk Institute for Biological Studies in San Diego, California.

The venom of this predatory water bug has more than a dozen small proteins.

Small proteins also promise to revise the current understanding of the genome. Many appear to be encoded in stretches of DNAand RNAthat were not thought to help build proteins of any sort. Some researchers speculate that the short stretches of DNA could be newborn genes, on their way to evolving into larger genes that make full-size proteins. Thanks in part to small proteins, "We need to rethink what genes are," says microbiologist and molecular biologist Gisela Storz of the National Institute of Child Health and Human Development in Bethesda, Maryland.

Despite the remaining mysteries, scientists are already testing potential uses for the molecules. One company sells insecticides derived from small proteins in the poison of an Australian funnel-web spider. And a clinical trial is evaluating an imaging agent based on another minute protein in scorpion venom, designed to highlight the borders of tumors so that surgeons can remove them more precisely. Many drug companies are now searching for small proteins with medical potential, says biochemist Glenn King of the University of Queensland in St. Lucia, Australia. "It's one of the most rapidly growing areas."

Other short amino acidchains, often called peptides or polypeptides, abound in cells, but they are pared-down remnants of bigger predecessors. Myoregulin and its diminutive brethren, in contrast, are born small. How tiny they can be remains unclear. Fruit flies rely on a microprotein with 11 amino acids to grow normal legs, and some microbes may crank out proteins less than 10 amino acids long, notes microbial genomicist Ami Bhatt of Stanford University in Palo Alto, California. But even the largest small proteins don't measure up to average-size proteins such as alpha amylase, a 496amino-acid enzyme in our saliva that breaks down starch.

Few small proteins came to light until recently because of a criterion for identifying genes set about 20 years ago. When scientists analyze an organism's genome, they often scan for open reading frames (ORFs), which are DNA sequences demarcated by signals that tell the cell's ribosomes, its proteinmaking assembly lines, where to start and stop. In part to avoid a data deluge, past researchers typically excluded any ORF that would yield a protein smaller than 100 amino acids in eukaryotes or 50 amino acids in bacteria. In yeast, for example, that cutoff limited the list of ORFs to about 6000.

Relaxing that criterion reveals that cells carry vastly more ORFs. Earlier this year, Stanford postdoc Hila Sberro Livnat, Bhatt, and colleagues trawled genome fragments from the microbes that inhabit four parts of the human body, including the gut and skin. By searching for small ORFs that could encode proteins between five and 50 amino acids long, the researchers identified about 4000 families of potential microproteins. Almost half resemble no known proteins, but the sequence for one small ORF suggested that a corresponding protein resides in ribosomesa hint that it could play some fundamental role. "It's not just genes with esoteric functions that have been missed" when scientists overlooked small ORFs, Bhatt says. "It's genes with core functions."

For the first time, we are about to explore this universe of new proteins.

Other cells also house huge numbers of short ORFsyeast could make more than 260,000 molecules with between two and 99 amino acids, for example. But cells almost certainly don't use all those ORFs, and some of the amino acid strings they produce may not be functional. In 2011, after finding more than 600,000 short ORFs in the fruit fly genome, developmental geneticist Juan Pablo Couso of the University of Sussex in Brighton, U.K., and colleagues tried to whittle down the number. They reasoned that if a particular ORF had an identical or near-identical copy in a related species, it was less likely to be genomic trash. After searching another fruit fly's genome and analyzing other evidence that the sequences were being translated, the group ended up with a more manageable figure of 401 short ORFs likely to yield microproteins. That would still represent a significant fraction of the insects' protein repertoirethey harbor about 22,000 full-size proteins.

Weissman and colleagues found microproteins a second way, through a method they invented to broadly determine which proteins cells are making. To fashion any protein, a cell first copies a gene into messenger RNA. Then ribosomes read the mRNA and string together amino acids in the order it specifies. By sequencing mRNAs attached to ribosomes, Weissman and his team pinpoint which ones cells are actually turning into proteins and where on the RNAs a ribosome starts to read. In a 2011Cellstudy, he and his team applied that ribosome profiling method, also called Ribo-seq, to mouse embryonic stem cells and discovered the cells were making thousands of unexpected proteins, including many that would fall below the 100amino-acid cutoff. "It was quite clear that the standard understanding had ignored a large universe of proteins, many of which were short," Weissman says.

Saghatelian and his colleagues adopted a third approach to discover a trove of microproteins in our own cells. The researchers used mass spectrometry, which involves breaking up proteins into pieces that are sorted by mass to produce a distinctive spectrum for each protein. Saghatelian, his then-postdoc Sarah Slavoff, and colleagues applied the method to protein mixtures from human cells and then subtracted the signatures of known proteins. That approach revealed spectra for 86 previously undiscovered tiny proteins, the smallest just 18 amino acids long, the researchers reported in 2013 inNature Chemical Biology.

Being small limitsa protein's capabilities. Larger proteins fold into complex shapes suited for a particular function, such as catalyzing chemical reactions. Proteins smaller than about 50 to 60 amino acids probably don't fold, says chemist Julio Camarero of the University of Southern California in Los Angeles. So they probably aren't suited to be enzymes or structural proteins.

However, their diminutive size also opens up opportunities. "They are tiny enough to fit into nooks and crannies of larger proteins that function as channels and receptors," Olson says. Small proteins often share short stretches of amino acids with their larger partners and can therefore bind to and alter the activity of those proteins. Bound microproteins can also shepherd bigger molecules to new locationshelping them slip into cell membranes, for instance.

A microprotein in the poison of the deathstalker scorpion has been fused to a fluorescent dye to make tumors emit near-infrared light. (1) A tumor seen in visible light (2)Same tumor in visible and near-infrared light

Because of their attraction to larger proteins, small proteins may give cells a reversible way to switch larger proteins on or off. In a 2016 study inPLOS Genetics, plant developmental biologist Stephan Wenkel of the University of Copenhagen and colleagues genetically alteredArabidopsisplants to produce extra amounts of two small proteins. The plants normally burst into flower when the days are long enough, but when they overproduced the two microproteins, their flowering was postponed. The small proteins caused that delay by blocking a hefty protein called CONSTANS that triggers flowering. They tether CONSTANS to other inhibitory proteins that shut it down. "A cell uses things that help it survive. If a short protein does the job, that's fine," Saghatelian says.

Those jobs include other key tasks. In 2016, Slavoff, Saghatelian, and colleagues revealed that human cells manufacture a 68amino-acid protein they named NoBody that may help manage destruction of faulty or unneeded mRNA molecules. NoBody's name reflects its role in preventing formation of processing bodies (P-bodies), mysterious clusters in the cytoplasm where RNA breakdown may occur. When the protein is missing, more P-bodies form, thus boosting RNA destruction and altering the cell's internal structure. "It shows that small proteins can have massive effects in the cell," Slavoff says.

Muscles appear to depend on a variety of microproteins. During embryonic development, individual muscle cells merge into fibers that power contraction. The 84amino-acid protein myomixer teams up with a larger protein to bring the cells together, Olson's team reported in 2017 inScience. Without it, embryonic mice can't form muscles and are almost transparent.

Later in life, myoregulin steps in to help regulate muscle activity. When a muscle receives a stimulus, cellular storage depots spill calcium, triggering the fibers to contract and generate force. An ion pump called SERCA then starts to return the calcium to storage, allowing the muscle fibers to relax. Myoregulin binds to and inhibits SERCA, Olson's team found. The effect limits how often a mouse's muscles can contractperhaps ensuring that the animal has muscle power in reserve for an emergency, such as escaping a predator. Another small protein, DWORF, has the opposite effect, unleashing SERCA and enabling the muscle to contract repeatedly.

Even extensively studied organisms such as the intestinal bacteriumEscherichia coliharbor unexpected small proteins that have important functions. Storz and her team reported in 2012 that a previously undiscovered 49amino-acid protein called AcrZ helps the microbe survive some antibiotics by stimulating a pump that expels the drugs.

And the venom produced by a variety of organismsincluding spiders, centipedes, scorpions, and poisonous mollusksteems with tiny proteins. Many venom components disable or kill by blocking the channels for sodium or other ions that are necessary for transmission of nerve impulses. Small proteins "hit these ion channels with amazing specificity and potency," King says. "They are the major components of venoms and are responsible for most of the pharmacological and biological effects."

Australia's giant fish-killing water bug, for instance, doesn't just rely on sharp claws and lancelike mouthparts to subdue prey. It injects its victims with a brew of more than 130 proteins, 15 of which have fewer than 100 amino acids, King and colleagues reported last year.

Unlike hulking proteinssuch as antibodies, microproteins delivered by pill or injection may be able to slip into cells and alter their functions. Captopril, the first of a class of drugs for high blood pressure known as angiotensin-converting enzyme inhibitors was developed from a small protein in the venom of a Brazilian pit viper. But the drug, which the Food and Drug Administration approved for sale in the United States in 1981, was discovered by chance, before scientists recognized small proteins as a distinct group. So far, only a few microproteins have reached the market or clinical trials.

Cancer researchers are trying to capitalize on a microprotein in the poison of the deathstalker scorpion (Leiurus quinquestriatus) of Africa and the Middle East. The molecule has a mysterious attraction to tumors. By fusing it to a fluorescent dye, scientists hope to illuminate the borders of brain tumors so that surgeons can safely cut out the cancerous tissue. "It lights up the tumor. You can see the margins and if there are any metastases," King says. A clinical trial is now evaluating whether the dual molecule can help surgeons remove brain tumors in children.

How important small proteins will be for medicine is still unknown, but they have already upended several biological assumptions. Geneticist Norbert Hbner of the Max Delbrck Center for Molecular Medicine in Berlin and colleagues found dozens of new microproteins in human heart cells. The group traced them to an unexpected source: short sequences within long noncoding RNAs, a variety that was thought not to produce proteins. After identifying 169 long noncoding RNAs that were probably being read by ribosomes, Hbner and his team used a type of mass spectrometry to confirm that more than half of them yielded microproteins in heart cells, a result reported earlier this year inCell.

Bacteria such as Escherichia coli also churn out many microproteins, although their functions remain unclear in many cases.

The DNA sequences for other tiny proteins also occur in unconventional locations. For example, some lie near the ORFs for bigger proteins. Researchers previously thought those sequences helped manage the production of the larger proteins, but rarely gave rise to proteins themselves. Some coding sequences for recently discovered microproteins are even nested within sequences that encode other, longer proteins.

Those genomic surprises could illuminate how new genes arise, says evolutionary systems biologist Anne-Ruxandra Carvunis of the University of Pittsburgh in Pennsylvania. Researchers had thought most new genes emerge when existing genes duplicate or fuse, or when species swap DNA. But to Carvunis, microproteins suggest protogenes can form when mutations create new start and stop signals in a noncoding portion of the genome. If the resulting ORF produces a beneficial protein, the novel sequences would remain in the genome and undergo natural selection, eventually evolving into larger genes that code for more complex proteins.

In a 2012 study, Carvunis, who was then a postdoc in the lab of Marc Vidal at the Dana-Farber Cancer Institute in Boston, and colleagues found that yeast translate more than 1000 short ORFs into proteins, implying that these sequences are protogenes. In a new study, Carvunis and her team tested whether young ORFs can be advantageous for cells. They genetically altered yeast to boost output of 285 recently evolved ORFs, most of which code for molecules that are smaller than the standard protein cutoff or just over it. For almost 10% of the proteins, increasing their levels enhanced cell growth in at least one environment. The results, posted on the preprint server bioRxiv, suggest these sequences could be on their way to becoming full-fledged genes, Carvunis says.

Slavoff still recalls being astonished when, during her interview for a postdoc position with Saghatelian, he asked whether she would be willing to go hunting for small proteins. "I had never thought that there could be this whole size of proteins that was dark to us until then."

But the bet paid offshe now runs her own lab that is searching for microproteins. Recently, she unleashed some of her postdocs and graduate students on one of the most studied organisms, the K12 strain ofE. coli.The team soon uncovered five new microproteins. "We are probably only scratching the surface," she says.

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New universe of miniproteins is upending cell biology and genetics - Science Magazine

Drug Treats Inflammation Related to Genetic Heart Disease – Technology Networks

When young athletes experiences sudden cardiac death as they run down the playing field, it's usually due to arrhythmogenic cardiomyopathy (ACM), an inherited heart disease. Now, Johns Hopkins researchers have shed new light on the role of the immune system in the progression of ACM and, in the process, discovered a new drug that might help prevent ACM disease symptoms and progression to heart failure in some patients.

"We realized that heart muscle inflammation in ACM is much more complicated than we thought, but also might provide a therapeutic strategy," saysStephen Chelko, Ph.D., assistant professor of medicine at the Johns Hopkins University School of Medicine and senior author of the new paper, inSept. inCirculation.

In ACM, patients often harbor mutations in any of the five genes that make up the cardiac desmosome -- the gluelike material that holds heart cells together and helps coordinate mechanical and electrical synchronization of heart cells. Because of this, it's often called "a disease of the cardiac desmosome." In patients with ACM, heart cells pull apart over time, and these cells are replaced with damaged and inflamed scar tissue. These scars can increase risk of instances of irregular heart rhythms and lead to sudden cardiac death if the scar tissue causes the heart wall to stiffen and renders it unable to pump.

If a person is aware they carry an ACM-causing genetic mutation, doctors help them avoid cardiac death through lifestyle changes, such as exercise restriction, and medications that keep their heart rate low. However, there are currently no drugs that treat the underlying structural defects of the desmosome. People who live for many years with ACM still accumulate scar tissue and inflammation in their hearts, leading to chronic heart disease.

"We tended in the past to view ACM as something that kills due to a sudden arrhythmic event," said Chelko. "But now we're starting to also see it as a chronic inflammatory disease that can progress more slowly over time, leading to heart failure."

Chelko and his colleagues wanted to determine the molecular cause of inflammation in the hearts of people with ACM. So they studied mice with an ACM-causing mutation, as well as heart muscle cells generated from stem cells isolated from an ACM patient. They found that the inflammation associated with the disease arose from two separate causes. First, they noticed high levels of macrophages, a type of immune cell that's normally found at sites of inflammation, such as around cuts or scrapes that are healing.

"Macrophages are usually the good guys who help heal a wound and then leave," said Chelko. "But in ACM they're permanently setting up shop in the heart, which, over time, reduces its function."

Chelko's team also found that in ACM, the heart cells themselves are triggered by a protein known as nuclear factor kappa-light-chain-enhancer of activated B cells (NF-B) to produce chemicals called cytokines, which act as homing beacons for other inflammatory cells and molecules. When the researchers treated mice or isolated cells with a drug blocking NF-B, heart cells stopped producing many of these cytokines, leading to decreased inflammation and infiltration of inflammatory cells. In mouse models of ACM, animals treated with the NF-B-blocking drug Bay-11-7082 had a twofold increase in heart function, measured by how much blood their hearts could pump over time compared with untreated ACM animals. They also had a twofold reduction of damaged and inflammatory scar tissue in the heart.

More than one-third of patients with ACM who die of sudden cardiac death have no previous cardiac symptoms, so wouldn't ever know to seek treatment. However, for relatives of these people who discover that they carry a genetic mutation causing ACM -- or those who discover the mutation for other reasons -- a drug could help stave off long-term heart disease, Chelko said.

While the Bay-11-7082 drug is currently only used in the lab for experimental purposes, the U.S. Food and Drug Administration has approved canakinumab, a drug that targets the same inflammatory pathway, for use in juvenile arthritis and a collection of rare auto-inflammatory syndromes. Canakinumab is also being studied for use in coronary artery disease. Chelko's group is now investigating whether this drug would have the same effect as Bay-11-7082 in ACM.

"We're very excited to have found an FDA-approved drug that can reduce heart inflammation in ACM, and we're eager to do more research to ultimately help those who carry these genetic mutations," said Chelko.

Reference:Chelko, et al. (2019) Therapeutic Modulation of the Immune Response in Arrhythmogenic Cardiomyopathy. Circulation. DOI:https://doi.org/10.1161/CIRCULATIONAHA.119.040676

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Drug Treats Inflammation Related to Genetic Heart Disease - Technology Networks

AASCP Delegates Meet With The Governor General Of The Bahamas – Anti Aging News

Delegates from the American Academy of Stem Cell Physicians recently traveled to the Bahamas to meet with the Governor General Sir Cornelius Alvin Smith to discus medical industry safety standards.

While in the Bahamas the delegates attended a 3 day regenerative health summit for educators, experts, professionals and learners where in doctors from all over the world arrived to discuss the future of regenerative medicine and stem cell therapy.

Dr. Krutchkoff announced the launch of the AASCPs Research Consortium dedicated to supporting research to validate therapies and promote compliance within the industry. During the HEALinc Summit Dr. Farschian and Dr. Krutchoff, the American Academy of Stem Cell Physicians delegates, held a workshop to teach physicians about a pathway to compliance when utilizing cell therapies in their practices.

The AASCP was created to advance research and the development of therapeutics in regenerative medicine, and aims to serve as an educational resource for scientists, physicians, and the public in diseases that can be caused by physiological dysfunction that are ameliorable to medical treatment.

The HEALinc Summit was organized by Dr. Desiree Cox, CEO of the HEALinc Future of Health Innovation Summit and The HEALinc Innovation Incubator which is a movement based organization with a mandate to champion a future health paradigm where medical therapy is driven by personal health and regenerative medicine by incorporating ongoing, real world capture, predictive analytics, and education.

Dr. Cox currently chairs the National Stem Cell Ethics Committee for the Bahamas, and has been leading health innovation and the development of the stem cell and regenerative industry since 2015 in the Bahamas which is a fast track test bed for real world studies in stem cell, regenerative medicine, and medical devices among others.

During the HEALinc Summit leading international experts spoke on hot topics in stem cell therapy, longevity and vitality, regeneration, resilience and adaptability, precision health, epigenetic and nutrition, personal wellness and optimal health, regenerative ventures/investment and much more.

"Within the Bahamas, health innovations in cellular and gene therapies, regenerative medicine, precision medicine and digital health can be tested ethically and safely in a regulated environment, so that the data obtained from the research can be used in later applications for FDA or EMA approval, says Dr. Cox.

Dr. AJ Farshchian, spokesperson for AASCP said, "It was a great honor meeting the Governor General in the Bahamas. We discussed safety standards currently practiced in the industry and how to improve the industry moving forward."

Dr. Adam Shwani, a member of the board for the American Academy of Stem Cell Physicians, added, "It is so important to bring industry partners together from all over the world to develop a standard that promotes patient safety and encourages growth in the field of regenerative medicine.

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AASCP Delegates Meet With The Governor General Of The Bahamas - Anti Aging News

He Was Unbearably Itchy, but the Problem Wasnt in His Skin – The New York Times

When the patient was dressed, Iammatteo returned to the examination room. She didnt think this was an allergy, she told him. But it could be a parasite. She thought it was more likely toxocara, given his recent exposures to dogs and cats. Toxocara is a type of parasite called a nematode or roundworm. It lives in the gastrointestinal tract of dogs and cats. Until the end of the 20th century, visceral toxocariasis could be diagnosed only by the symptoms it caused when it invaded the organs of the body the liver, the lungs, the brain or the eyes. These were serious infections causing everything from wheezing and shortness of breath to blindness or, rarely, death. It wasnt until a diagnostic blood test was developed that other manifestations of the disease were identified. In whats called common toxocariasis, patients have gastrointestinal symptoms as well as an itchy rash. In covert toxocariasis, the only symptom is an itchy rash. These infections often resolve on their own over time, but they can also be treated with a medicine. This patient could have covert toxocariasis.

Iammatteo said she would test for both toxocariasis and strongyloidiasis. She would also refer him to a hematologist to look for a malignancy or other trigger that might have caused his overproliferation of white blood cells. There were other causes of his rash and eosinophilia, but these were the most likely and a good place to start.

A few days later, she got a possible answer and called the patient. You probably have toxocariasis, she told him. The blood test came back positive, but she explained there was a caveat. The test measures whether the immune system has responded to this particular parasite ever. The fact that it was positive meant that the patient had been exposed to the parasite, but it couldnt determine when the exposure occurred. Toxocara infections are most common in children. But the test will still be positive even if the infection is long gone. The only way to know for certain that the toxocara was causing the itch, she told him, was to treat him and see how he responded. She referred him to an infectious-disease doctor who prescribed the recommended five days of Albendazole.

Because he couldnt know for sure if this was the right diagnosis, the patient kept his appointment with the hematologist. That doctor sent off more blood to look for signs that the overabundance of these cells could be caused by an eosinophil gone wild.

But well before those tests results came back negative, the patient felt that he had his answer. Within days of completing his treatment with Albendazole, the itching resolved. And by the time he went back to see Iammatteo two weeks later, even the rash had mostly disappeared.

Why had Iammatteo been able to figure this out when other doctors couldnt? the patient asked when he saw her for a follow-up visit. She explained that shed gone to Albert Einstein College of Medicine in the Bronx, and one professor there was an expert in parasitology. She took her class, and what she learned stuck with her. Different medical schools have different strengths, she told me later. Parasites were one of theirs.

And, she added, doctors are taught that toxocara infection is rare. But now shes not so sure. Since making this patients diagnosis last spring, she told me she has diagnosed nearly a dozen cases of toxocariasis in patients whom she might not have thought to test for the parasite if not for this older man and his rash. I know Ive been successfully diagnosing more of it because its on my mind.

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He Was Unbearably Itchy, but the Problem Wasnt in His Skin - The New York Times

ArsenalBio Launches With $85 Million Series A Financing to Advance New Paradigm to Discover and Develop Immune Cell Therapies – Business Wire

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--ArsenalBio makes its debut today backed by $85 million Series A financing to build a programmable cell therapy company to create highly effective and accessible immune cell therapies. The company will integrate technologies such as CRISPR-based genome engineering, scaled and high throughput target identification, synthetic biology, and machine learning to advance a new paradigm to discover and develop immune cell therapies, initially for cancer. ArsenalBios foundation stems from the contributions of scientific leaders from a consortium of academic medical and research institutions.

Investors include Westlake Village BioPartners, the Parker Institute for Cancer Immunotherapy (PICI), Kleiner Perkins, the University of California, San Francisco (UCSF) Foundation Investment Company, Euclidean Capital, and Osage Venture Partners.

Our goal is to address the unmet need and suffering of patients with cancer, and ultimately other diseases, by developing and advancing a new paradigm of human immune cell therapy design and treatment, said Ken Drazan, MD, ArsenalBios founding Chief Executive Officer. The integrated technology approach were embarking upon will create a new arsenal of tools and medicines for researchers, patients and their physicians to reduce cancer morbidity and mortality.

Todays commercialized, first-generation T cell therapies are designed and manufactured with the goal of inserting into T cells a single cell-targeting transgene, a chimeric antigen receptor or a new T cell receptor through viral delivery. ArsenalBio seeks to exponentially advance this process by precisely inserting, without viral vectors, significantly larger DNA payloads, designed with proprietary tools and encoding a broader set of biological software instructions to enable immune cells to effectively target and destroy solid organ and hematologic cancers. ArsenalBios approach will move beyond the current model of tumor-targeting strategies to enable the rewiring of immune cell circuitry through computationally driven design. The company aspires to evolve critical metrics of success for immune cell therapies, including enhanced and broader efficacy, increased patient safety, reduced provider costs, and expanded market access.

The technology ArsenalBio is developing represents a significant advance in how cancer could be treated. The experience leaders such as Jane Grogan, Michael Kalos and Tarjei Mikkelsen bring, combined with Dr. Drazans results-oriented management approach, will help rapidly advance this transformational platform to benefit patients, added Beth Seidenberg, MD, co-founding Managing Director of Westlake Village BioPartners, a Los Angeles area-based venture capital firm focused on incubating and building life sciences companies.

ArsenalBio is taking different approaches to gene editing, target selection, cell circuit engineering, and computation to reimagine dosing, delivery, persistence, and affordability of cell therapy. The networks of pharma, science, and talent relationships of PICI, Westlake and Kleiner Perkins is a booster to ArsenalBios remarkable team and R&D progress, said Brook Byers, Founding Partner of Kleiner Perkins of Menlo Park, CA.

ArsenalBio allows us to rewrite vast stretches of code to give T cells dramatic new functions--that means they can be made to be more effective at killing cancer and a broad spectrum of other diseases, said Sean Parker, founder and Chairman of PICI. Its also very rewarding to see ArsenalBio born from the deep collaboration of PICI investigators---who worked together across research centers, hospitals and universities on the science behind these technologies. The companys very existence demonstrates how much faster and better we can get therapies from bench to bedside when we collaborate and put patients first.

Experienced Management Team

ArsenalBios management team includes seasoned industry executives who bring immuno-oncology, cell therapy and genomic expertise:

Board of Directors with Proven Track Record

ArsenalBios board brings together industry leaders who have proven track records building successful companies:

Scientific Founders

The companys scientific founders comprise a bi-coastal, multi-disciplinary consortium of leading academic researchers:

About ArsenalBio

ArsenalBio is building a programmable cell therapy company to create highly effective and accessible immune cell therapies to impact outcomes for a much broader number of patients, initially those with cancer. Founded in 2019, ArsenalBio is focused on integrating technologies such as CRISPR-based genome engineering, scaled and high throughput target identification, synthetic biology, and machine learning to advance a new paradigm to discover and develop in immune cell therapies. With its programmable and computationally driven approach, ArsenalBio aspires to evolve critical metrics of success for immune cell therapies, including enhanced and broader efficacy, increased patient safety, reduced provider costs, and expanded market access. Visit http://www.arsenalbio.com to learn more.

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ArsenalBio Launches With $85 Million Series A Financing to Advance New Paradigm to Discover and Develop Immune Cell Therapies - Business Wire

Immunomic Therapeutics to Present at Precision Lung Cancer World R&D Summit – BioSpace

Oct. 17, 2019 20:05 UTC

BOSTON--(BUSINESS WIRE)-- Immunomic Therapeutics, Inc. announced today a presentation at the Precision Lung Cancer World R&D Summit in Boston. On Tuesday, October 22, Business Development Advisor at Immunomic, Yan Su, will present a talk entitled Current & Future Application of the UNITE Platform in Cancer Immunotherapy, highlighting Immunomics investigational UNiversal Intracellular Targeted Expression (UNITE) platform and its application in immuno-oncology.

Immunomics UNITE technology platform has the potential to utilize the bodys natural biochemistry to develop a broad immune response, including antibody production, cytokine release and critical immunological memory, a method that could put UNITE at the crossroads of immunotherapies in a number of illnesses, including cancer. The investigational UNITE technology is currently being applied as a cancer immunotherapy in a Phase II clinical trial targeting glioblastoma multiforme (GBM). Sus presentation will explore the broader potential of UNITE through discussion of the LAMP-based immunotherapy technology, VAC2. VAC2 is an allogeneic cancer immunotherapy being developed by Lineage Cell Therapeutics, Inc. in collaboration with Cancer Research UK and is currently in a Phase 1 clinical trial for treatment of non-small cell lung cancer. In addition, the presentation will discuss the potential application of the UNITE Platform in Cancer targeting shared neoantigens.

Who: Yan Su, Business Development Advisor at Immunomic Therapeutics, Inc.

What: Presentation on Current & Future Application of the UNITE Platform in Cancer Immunotherapy

When: Tuesday, October 22 at 4:00 p.m. ET

Where: Wyndham Boston Beacon Hill, 5 Blossom St., Boston, MA 02114

About UNITE

ITIs investigational UNITE platform, or UNiversal Intracellular Targeted Expression, works by fusing pathogenic antigens with the Lysosomal Associated Membrane Protein, an endogenous protein in humans, for immune processing and MHC-II presentation to helper T-cells. In this way, ITIs vaccines (DNA or RNA) have the potential to utilize the bodys natural biochemistry to develop a broad immune response including antibody production, cytokine release and critical immunological memory. This approach could put UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy and infectious diseases. UNITE is currently being employed in Phase II clinical trials as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.

About Immunomic Therapeutics, Inc.

Immunomic Therapeutics, Inc. (ITI) is a privately-held, clinical stage biotechnology company pioneering the development of vaccines through its proprietary technology platform, UNiversal Intracellular Targeted Expression (UNITE), which is designed to utilize the bodys natural biochemistry to develop vaccines that generate broad immune responses. ITIs UNITE platform could potentially have broad therapeutic applications in oncology, including viral antigens, cancer antigens, neoantigens and antigen-derived antibodies as biologics and ITI has built a large pipeline from UNITE with six oncology programs and two allergy programs. ITI has entered into a significant allergy partnership with Astellas Pharma and has formed several academic collaborations with leading Immuno-oncology researchers at Fred Hutchinson Cancer Research Institute, Johns Hopkins University of Medicine, and Duke University. ITI maintains its headquarters in Rockville, Maryland. For more information, please visit http://www.immunomix.com.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform, Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191017005872/en/

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Immunomic Therapeutics to Present at Precision Lung Cancer World R&D Summit - BioSpace

Canadian breakthrough that became the world’s most expensive drug, then vanished, gets second chance – CBC.ca

A made-in-Canada medical breakthrough that disappeared from the market because it wasn't profitable is being revived by the National Research Council of Canada (NRC).

It's the latest chapter in the saga of Glybera, the world's first approved gene therapy, which also became the world's most expensive drug after it was licensed toa Dutch company and priced at $1 million for a one-time dose.

Glybera treats arare and potentially deadly genetic disorder called lipoprotein lipase deficiency, or LPLD.Canada has the world's largest population of LPLD patients clustered in the Saguenay region of Quebec, where an ancestor with the genetic mutation settled several hundred years ago.

People with LPLD lack a critical enzyme that helps their bodies process the fat from food. There is currently no available treatment and no cure. Those with LPLDmust avoid most dietary fat to try to prevent painful and dangerous attacks of pancreatitis.

The decision to re-develop a Canadian version of Glybera is the result of a serendipitous series of events, beginning when the NRC'sdirector of research and development for translational bioscience happened to be watching CBC'sThe Nationallast November.

Dr. Danica Stanimirovic was in the process of selecting the first project for a new federally funded program aimed at bringing rare gene and cell therapies to Canadians at an affordable price. Thenshe sawCBC's feature report telling the story ofhow Glybera was pulled from the Europeanmarket after only one commercial sale. The drug was never offered for sale in Canada or the U.S.

"That really sparked some thinking," she said."We really have the abilityto advance that."

So she picked up the phone and called Dr. Michael Hayden in Vancouver.He's thescientist at the University of British Columbiaand the BC Children's Hospital whose team developed Glybera.Hayden said he was happy to get the call.

"I was thrilled because this represented a unique response to solve a big Canadian problem, particularly for families in Quebec.And I was just thrilled that we could do something as a national effort to achieve this."

The Glybera story started at UBC in the early 1990s, when Hayden and his teamdiscovered the first genetic mutations that caused LPLD. The researchers then developed a method to fix the malfunctioning gene and allow patients to live a nearly normal life.

After doing the preliminaryresearch, the Canadian discovery was licensed to a Dutch companycalled uniQure, which took Glybera through the rigorousclinical trialandapproval process.

When the treatment was approved by the European Medicines Agency in 2012, it made headlines as the world's firstgene therapy the first treatment that could repair a faulty gene.

When it went on sale in Europe in 2015,Glybera quickly made headlines again, this time as the "world's most expensive drug,"priced at $1 millionfor the one-time dose.

Dr. Sander van Deventer,uniQure's chief scientific officer, told CBC News last year that the price was a business calculation based on the price of other drugs that treat rare diseases. Many of those drugs cost more than $300,000 per patient per year.Because Glybera is a one-time treatment thatkeeps working for years, the $1-million price seemed reasonable, he said.

Less than twoyears later, the drug was pulled from the market after only one commercial sale. uniQure has no plans to revive the therapy.

Although Hayden discovered the gene mutation and developed the early phase of the treatment, he had no role in the commercialization of his discovery. And that meant he also had no control over the price.

"You don't determine the outcome, you don't determine its costs," he said."I'd say what went wrong is that it was very hard to be able to make sure that this got to patients at a reasonable cost."

Stanimirovic said the fact that Canada has such a large population of LPLD patients was an important factor in deciding to give Glybera a second chance.

"This gene mutation is very prevalent in Canada compared to other places in the world," she said. "For us, it was almost calling us to do something on the manufacturing side for this particular gene therapy."

LPLD is rare, affecting one or two out of every million people around the world. But inthe Saguenay region of Quebec, where the gene mutationhas been passed down through generations,the numbers are 30 times higher.Up to one in 50 people in some communities are carrying the gene mutation. Both parents must have the mutation for a child to inherit the disease.

The ultimate goal of gene therapy is to fix a genetic problem by giving the patient a new gene. Specially engineered viruses are used to deliver therepair gene to the patient's cells. The cost of manufacturing those virusesis often cited as one reason for the high price of therapies. The need to generate pharmaceutical shareholder profits is another factor.

"[Gene therapies] areusually targeted to very smallpatient populations," Stanimirovicsaid. "It's hard to make them in a typical pharma-driven model because it drives theprice of these therapies to astronomical levels."

At its facility in Montreal, theNRChas already developed expertise in producing viral vectors thatact as the delivery system for gene therapy. Because the scientistswill be re-engineeringGlyberausing new viral vectors,and improving the therapy, any remainingpatentswill not be an obstacle,Stanimirovicsaid.

The ultimate plan is to developpublic sector manufacturing capacity to create not just an affordable version of Glybera but other gene and cell therapies as well. The total federal funding for six projects including Glyberais estimated at about $80 million over seven years.

"Our goal is to create new partnership models that will create therapies that are more accessible and more affordable," said Stanimirovic. "We hope we can do that through public partnership or public/private partnerships. So the end goal is to really, through this project, develop Canadian capacity to take on subsequent gene therapies."

Hayden called the plan a "beautiful Canadian story."

"Now we have to translate this into something that will truly be effective forpatients in a limited time frame and I'm so excited to do this."

For patients suffering from LPLD, the wait is frustrating.

Felix Lapointe, a 10-year-old from Repentigny, Que.,was fiveweeks old when his mother learned the terrible news that her son had thepotentially deadly genetic disease.

Because there is no treatment available right now, he'smanaging the disease through a strict diet to reduce the risk of dangerous pancreatic attacks. He will have to wait another five years for the first clinical trials of the re-inventedGlybera.

"We'd like it to happen tomorrow morning," said Brenda Potter, Felix's mother. "Still, we're a little used to this. We'vebeen fighting for 10 years with doors closed. The possibility that something is comingis encouraging, but yes, it's long."

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Canadian breakthrough that became the world's most expensive drug, then vanished, gets second chance - CBC.ca

Dr. Raj & Stem Cell Therapy Innovation – LATF USA

For anyone who has had hip replacement surgery, Im sure they will agree that it is better to get hit by a bus than to undergo another one. Last year after several years of suffering, I decided to take the leap and go for the hip replacement that my specialist recommended. I was told that it was a common surgery and that it was the best solution for me. Between us; it was probably the most painful thing I have ever gone through. So much so, that at the time, I just wanted to die. Not only did the pain persist for several weeks after the operation, but I was on painkillers for days, which eventually added to my suffering. I had to use a walker for the first 2 weeks and then depended on a cane for over 2 months before I could walk on my own.

My entire demeanor changed, as well as the way I dealt with what once were minor things in life. I feared slipping in the shower, going down the stairs or walking my dogs. No one had prepared me for this. Ive had my share of surgeries including a double mastectomy when I was diagnosed with breast cancer but pain wise; this one was by far the worse. I was hoping after a very long recovery that I would never have to face this situation again. Unfortunately, a year later, I am starting to feel pain on the other side and dread the re-experience of my nightmare.

Although, I heard about Stem Cell, I did not know much about it. So I started to investigate for myself, speak to people, enquire about the procedure and look for a doctor in my area who specialized in Stem Cell. I was willing to do just about anything before considering another hip replacement. After extensive research, I came across Dr. Raj, a Double-Board Certified Orthopedic doctor in Beverly Hills, CA. Going to his website; I learned that he has been in private practice for 10 years. He has been named as one of Americas Top Orthopedists, been featured on the Best of LA and has received numerous other accolades and awards as one of the Top Orthopedic doctors. Providing the ultimate in state-of-the-art orthopedic care, Dr. Rajs practice is always on the cutting-edge of surgical and nonsurgical technologies, such as PRP (Platelet Rich Plasma) injections, stem cell injections for tendinitis and arthritis, minimally invasive surgery and more.

He is Board Certified as a Medical Legal Specialist in America, as well as, Canada and Dubai (Trial, Testimony, Deposition, IME) with a Subspecialty in Hip and Knee Surgery in Los Angeles, including Sports Surgeries.

He is also an Undergraduate from Dalhousie University in Halifax and Canada. He pursued his medical education at Memorial University PGME, before doing his internship and residency in the Department of Orthopedic Surgery. Now that I had found Dr. Raj, all I needed was to get myself educated. So lets start by what are stem cells? This is what I read: Mesenchymal stem cells (MSCs), commonly called stem cells, are precursor cells that havent decided yet what they are going to be in the body. They can differentiate into multiple forms including bone, cartilage, fat and other connective tissues. They play a significant role in the reparative processes throughout the human body.

Where do we find stem cells?

They may be harnessed from fat tissue, bone marrow, synovial tissue or umbilical cord tissue. While stem cell therapy is a promising technology, there is much we are still learning about the causes and pathways that lead to symptomatic osteoarthritis. We have not optimized the factors found in stem cell therapies. To be sure, only the good cells and growth factors are injected into a specific joint. And that is why further research is necessary before being approved by the FDA.

My next move would be to consult with Dr. Raj who would tell me the medical truth, beginning with this question:

What is the current state of Stem Cells and its success rate?

It's relatively new. It's been popular for about 20 years, internationally. In areas like Germany and Korea, it was utilized a lot more. It became popular here when athletes like Kobe Bryant started going to Germany for modified versions of PRP, which led on to regenerative technologies. We have a stigma correlating stem cells with abortions and issues like that. This in itself is completely different. We are not utilizing amniotic stem cells or placenta stem cells. We're utilizing your own stem cells. For issues such as a hip replacement, the most powerful stem cells are the ones in your body. Bone marrow stem cells work well on joints. Joints have zero blood supply. So, if God or the higher power created us where we had blood supply going through our joints, like a cut in our skin - we would constantly replenish or repair. A break in our bone would repair. If you get stem cells and you're in decent enough shape, you will heal no matter what because these stem cells will deposit. Will you heal straight? Probably not - that's where we come into play.

The reason why joints; hips, knees and shoulders degenerate is because there is no blood supply. So, if you have a cut or a loss of cartilage, it stays like that and accumulates overtime. The only way you can control it is externally. You get stronger, you lose weight and you increase your range of motion. But you can't control anything internally.

So regenerative technology is basically utilizing these cells to regenerate cartilage and repair. These are the same cells that flow through our body - and upon signal of an injury will heal skin to skin, bone to bone, tendon to tendon, muscle to muscle. Our joints are just an alcove of joint fluid and no blood supply. The whole concept is - throughout the years, we did steroid injections - they're like band aids. Basically they mask pain. What does masking pain do? It propagates injury. Because we put the band aid on, we don't feel it and we do more. We take this little cut or loss of cartilage and we make it even more over time.

Why is it that specialists do not recommend seeing a surgeon at a certain stage?

There are a lot of people who think one way and everyone is entitled to their own opinions. You can't change opinions.

Are people afraid of stem cells?

Some people are afraid because of stem cells causing cancer. But that's embryonic stem cells.

What is the process?

Bone marrow stem cells are the best because there is a higher chance of live stem cells. Less manipulation, meaning that - in a Mayo Clinic study 4 or 5 years ago, which has a two year follow through on people who are ready to get replacements for joint or knee - they had an 80% success rate where they didn't need it. I do replacements and I do stem cells.

How do you determine what's better for the patient?

My knowledge and years of experience. Also, my knowledge with fitness and being athletic myself. Understanding at a certain point, someone is mechanically compromised. Bone on bone is a term that's been used for years. There are a lot of people who think they are 'bone on bone." Coming from Canada, the US is notorious for doing unnecessary surgeries and replacements. It's the highest rate of replacements in the world. I do not like the term 'bone on bone' because a surgeon will look at an x-ray and say you're bone on bone because that's all they do: replacements. They become a 7-11 or 99 Cents store, lining up 21 people a day. That's not the right way to do things. You don't want to be one of those 21 people getting a replacement because you're not getting that surgeon's full attention. The reality is - you have a PA or an old plastic surgeon who's doing most of your surgery and there is more likelihood of issues. Amongst every specialty there is a lot of ignorance. The whole concept is - you preserve what you have for as long as you can. You have beauty on the outside; you need beauty on the inside too. What's beauty on the inside? Feeling good, you're less inflamed and your joints are healthy.

How does it work with a stem cell procedure?

I extract bone marrow from your pelvis. Take approximately 6 ccs. Under slight sedation, it takes about 5 minutes to take it. Then we separate it via an FDA approved technique. Per FDA, we cannot add anything to it, nor would I want to. We cannot harvest it because the longer it's outside of the body, the better it is. Basically, we then inject those pure cells right away into the joint. It's a four month process for an 80% of regeneration. So, it's not just reduction of inflammation, it's regeneration. It will be a year for a 100% effect. I've had probably about 20% of patients who have taken 6 months+. I've had over a 95% success rate with this technology.

Are you one of the only doctors doing this in LA?

I'm one of them. There are some family and pain management doctors who are doing it. I'm the only Orthopedic surgeon doing it. I'm sure different practitioners are starting to.

Dr. Raj and patient Paula Abdul

How often do you do the stem cell procedure?

You do it one time. It's a powerful injection and there are people Ihave 6 years out who are doing well.

Does it hurt after the fact?

No, not at all. You can walk and move. For example, with your hip - I would combine it with physical therapy to increase your range of motion. Once you have the anti-inflammatory effect, you have to take advantage of it. If you don't increase your range of motion - what happens is - you're walking on one nail vs. 100 nails. You want to dissipate the force over a greater area so that there's a higher chance of external success. Then you strengthen the muscles.

Are there people who are not good candidates for it?

Yes, when it's too far gone. Like I said, people are told they're bone on bone when they're not. They show you different views. It's a marketing gimmick. That person is lined up and ready to sell. Age is relative. There's physiologic age. It really depends on the person. Hypothetically, if you're an inflamed mess, a drinker and abusive to your body, then nothing is going to work. If you take care of yourself and you're motivated with the right protoplasm, then it's going to work.

What about the skeptics or the ones who think it's bad for you?

Don't get me wrong; amniotic stem cells are good for certain situations. Embryonic is bad. It means that it's too far gone. You want live stem cells in an area that does not have blood supply. The data is out there. How can you argue against a Mayo Clinic study with an 80% success rate? How can you argue against the hospitals for special surgery in New York that's doing it, or the Steadman Hawkins Clinic, I'm doing it. Top facilities in the world are doing it and a number of top athletes who are getting it done with success rates. Who's ignorant? Is it that one surgeon or everyone else?

Does insurance cover it?

No, not yet. Insurances are very backwards in terms of their understanding. They would rather cover a replacement.

Is it expensive?

If you break it down par and par and avoid a replacement, not really. On average, you're talking about $7,000, versus hospital, surgeon, facility fees+++,which can be about $25,000.

You're very progressive.

There are a lot of things that I do to try and reduce pain significantly.When I use screws, I use screws that are made out of calcium so they dissolve in your body. Some of my colleagues use tourniquet, I don't use one. I control bleeding and do it in less than an hour. The whole concept is, you don't have atourniquetsqueezing your leg and toxins causing significant pain.

And there you have it. Everything is a risk in life, we do not know if we will wake up tomorrow or if you will get hit by a car and so on so why not try this procedure. I believe that I am lucky enough to have met Dr. Raj. I have taken the decision to undergo the stem cells therapy FDA approved or not, anything before going under the knife one more time. Stay tuned, I will give you a report on the progress.

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Dr. Raj & Stem Cell Therapy Innovation - LATF USA