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Bilirakis Secures Better Treatment for Blood Cancer Patients – The – The Floridian

Posted: January 5, 2020 at 3:41 am

Last year,Florida Rep. Gus Bilirakis (R)successfully contributed to passing important bills that strengthened the sunshine state and its residents.

This year, hes hitting the ground running by announcing that he has co-authored a bipartisan bill that has been signed into law.

With the new bill, Rep. Bilirakis has ensured that patients in need of bone marrow and stem cell transplants have access to lifesaving treatments.

Specifically, as reported in a press release,the Patient Access to Cellular Transplant Actallows older Americans with blood cancers like leukemia, lymphoma, and Myelodysplastic Syndromes to have better treatment options than they currently have had.

Sorry. No data so far.

In a statement regarding the importance of the bill, Bilirakis commented that medicare policy simply hadnt kept pace with technological advances; and tragically, this disparity threatened the lives of older Americans with blood cancers often stripping them of hope for a cure.

Furthermore, the Florida Rep. added that whereas physicians once had very few options to treat patients, today, blood transplants offer a cure.

So, these treatments can be the difference between life and death, and he expressed great joy at being able to pass a new law that fixes the problem and ensures access.

This actually marks the 8th piece of legislation that Rep. Bilirakis authored to become law in 2019.

Although Republicans are currently in the minority in the House, Rep. Bilirakis asserts that this does not discourage him. Speaking on the matter, he explained that he considers himself a consensus builder, regardless of which party was in power or what distractions may exist.

As well, when it comes to doing the right thing for patients and for our nations heroes, he declared that he would continue to reach across the aisle and get things done for my constituents and my community.

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Why microneedling facials really work to revive 40+ skin – harpersbazaar.com

Posted: January 5, 2020 at 3:41 am

I call it peak bleak: its right about now when all of us beyond our thirties are really thinking that our skin looks particularly knackered. Its central heating, its illness, its being overtired, over worked and over partied and it makes for a combination of low-level dryness and dullness that no illuminating make-up seems to ameliorate (highlighter on a dehydrated cheekbone is never flattering). Hydrating sheet masks, richer moisturisers and glycolic peels make some strides to improve exhausted skin, but the thing Ive found to make the single biggest difference is microneedling.

Im not referring to deep derma rolling treatments here (brilliant as they are for long term rejuvenation, they do entail some down-time) but rather facials - and at-home facial treatments - that incorporate a level of gentle needling. What gives these facials the edge on less than young skin is twofold: firstly, as leading facialist Sarah Chapman explains, microneedling is electronic precision engineering, creating thousands of needle columns into the skin, each one penetrating into the dermis layer to rejuvenate your skin by supercharging collagen production, which in turn reduces the appearance of wrinkles, fine lines and improves the overall texture of your skin. Which goes to say that it gets right to the root cause of a bleak complexion and directly revs it up.

Secondly, needling is astoundingly effective at aiding absorption of serums applied both during and after treatment (thanks to those tiny channels that Chapman described) and, quite frankly, the more hydrating serum you can get your skin to suck up, the better in terms of improving its plumpness and luminosity in both the short and long term.

Treatment wise, the best facial that incorporates needling is Chapmans Stem Cell Collagen Therapy treatment, 210. Chapman calls it the ultimate youth-boosting facial, a punchy claim that I must say its hard to dispute. The needling itself feels like nothing more than an electric toothbrush being whisked over the skin as it pushes in concentrated doses of botanical stem cells and peptides, while the finishing Dermalux red-light therapy adds to the impressive post-treatment glow. Whether you're looking for a facial that really delivers pre- or post-party, or simply want a fix to rid you of lacklustre skin, this is the facial to book.

At home, I like to needle every other day with a gentle manual 0.2-0.3mm roller: freshly rolled skin sucks in serum incredibly satisfyingly, and the increased microcirculation it induces adds to the don't you look well effect. Environs CIT Roller, 59, and Nannette de Gaspes Art of Noir Roller Noir, 35, both manage to be effective yet gentle. Do not be tempted to buy a cheap roller on Amazon or eBay; the needles are often hooked, which can rip the skin leading to redness and inflammation.

Roller Noir

35.00

Skinesis Intense Hydrating Booster

64.00

B-Hydra Intensive Hydration Serum

40.00

Peptide Veil

115.00

Rolling can be done on bare skin, but I find it more effective and comfortable to apply a thick layer of hydrating serum first, slathering on three times the amount Id usually apply of either Skinesis Intense Hydrating Booster, 64, or Drunk Elephant B-Hydra Intensive Hydrating Serum, 44. Start at the forehead and roll over each area three or four times horizontally, three or four times vertically, then diagonally in each direction, before moving onto the cheeks and finally chin and neck. Finish with a thick veil of cream (Im loving Decree Peptide Emollient Veil, 115) and youll wake up to skin that is anything but bleak.

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Research – Florida Stem Cell Research

Posted: January 3, 2020 at 5:49 pm

ResearchAdministrator2018-10-04T19:36:47-05:00

Stem cells technology has been around for decades. It has been subject of ethical concerns and sanctioned (highly regulated) by the government. However, in the past few decades, it was found that umbilical derived stem cells are multi-potent (capable of forming different cell types) almost like the embryonic stem cells (ESC) derived from the inner cell mass (ICM), 5-day-old embryos. Unlike the ESC, stem cells harvested from donated umbilical cord, including the umbilical cord blood cells, amnion/placenta, umbilical cord vein or umbilical cord matrix are readily available and not subject to government regulation.

One of the characteristic of stem cells is that they renew the tissues that they populate, and throughout the body, they continue to populate (rejuvenate) and replace old dying cells. The body loses this ability to regenerate damaged tissue with advanced age.

Bone marrow derived mesenchymal stem cells (bmMSC) have been used in the past 40 years to treat adult with bone marrow deficiency, but their capacity to expand is limited, specially bmMSC derived from adults.

Umbilical cord stem cells are mesenchymal stem cells-like cells that are robust, easily harvested, isolated and extensively expanded in culture.

Mesenchymal stem cells (MSC) offers a large array of alternative treatment to physicians for their patients. MSC treatment gives the body the ability to heal itself by generating new tissues and growth factors that can promote healing and recovering from injury a lot faster.

MSC can be used to treat an array of debilitating conditions:

Advantages to using MSC:

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Florida Stem Cell Treatment Center

Posted: January 3, 2020 at 5:49 pm

Florida stem cell treatment centers often have patients seeking to avoid knee surgery. Are you one of the millions upon millions of people who have knee pain? Thinking about getting stem cell therapy as an option? Good news, even if you have bone-on-bone causing your knee pain, Regenerative Medicine of North Florida stem cell shots may be your best option because our stem cell injections for patients with knee pain are getting people tremendous positive results.

FACT: Smart people just like you, are looking towards stem cell treatments to help relieve chronic joint pain such as hip pain, knee pains, and back or shoulder pains. Its really exciting when you look at the incredible medical advancements for a variety of these conditions.

But before we go any further lets talk about the elephant in the room. Where does Regenerative Medicine of North Florida get the stem cells that will be used to treat you?

We want to be very clear. We do NOT use embryonic cells. Thats illegal. Plus thats not something that we would do.

Keep reading to discover exactly how we get the powerful stem cells we use to help our patients and if you like what you see, then hopefully youll be like the large growing number of our patients who are now enjoying the quality of life that they deserve.

Lets start with another fact.

FACT: Using stem cells from aborted babies is illegal in the USA.

Besides, we would NEVER do that as we stated before.

So lets start with some easy ways to understand some medical terms. Autologous simply means you have a surgical procedure and they use your own stem cells. This definitely works. It is very expensive, and the number of stem cells that you have, REDUCE by the millions and millions the older you are. Those are only two of the big reasons that we do not use these types of stem cells to get the powerful results that weve seen at Regenerative Medicine of North Florida.

Regenerative Medicine of North Florida uses a process known as HUCT, which basically means we use stem cells from the umbilical cord of healthy mothers who donate their umbilical cord. So just like people donate organs to help people have a better quality of life, these healthy mothers who delivered healthy happy babies donate the umbilical cord because they know the umbilical cord has an astonishing amount of stem cells. Specifically, they are referred to as MSC, which stands formesenchymal stem cells.

Long story short, these are the extremely potent stem cells we use that are responsible for the amazing results associated with stem cell therapy.

Now for a little more technical information for the people who love details.

As a direct result of medical advancements, there are a number of different ways to obtain stem cells, but the one that is very effective and most cost-effective is a mesenchymal stem cell (MSC) obtained from umbilical cords.

In addition to no surgery required and the reduced costs (Many times our stem cell injections are less than half the cost of your other options for stem cell treatment), our Regenerative Medicine of North Florida doctors selected this option because it will allow for a plentiful, and an ethical option to help our patients.

When you get MSC stem cells here at Regenerative Medicine of North Florida then youll know the benefits of MSC cells have been clinically researched.

MSCs afford several advantages for clinical use, such as availability and ease of harvesting; multilineal differentiation potential; potent immunosuppressive effects; safety without any possibility of malignant transformation after infusion of allogeneic cells, which is common in the case of ESCs and iPSCs; and the lack of ethical issues that occur with the application of human ESCs. One of the most promising benefits of MSCs for cell-based therapy is their availability and ease of harvesting.

FACT: The older someone becomes, the lower the count of stem cells available to be extracted and use. Think of it like this. When you were younger you healed much faster. Thats because of the number of stem cells available to do the natural regenerative repairs. The older you get then you have fewer stem cells of this nature. And when you try to extract the smaller amounts of stem cells it requires a surgical procedure. Most often the surgical process to obtain the autologous stem cells is performed using a very large needle that is plunged deep enough into your hip bone in order to reach your bone marrow, and the marrow is sucked out of your hip for use. Additionally, as stated before, extracting stem cells from the hip, is much more expensive. It is also more painful and the recovery period is significantly longer. This is particularly true when compared to a shot from Regenerative Medicine of North Florida which we have many patients say that the flu shot is more uncomfortable than getting Regenerative Medicine of North Florida therapy.

Lets be honest. There are a number of different stem cell treatment options available, each with their own marketed benefits, but none of them can 100% guarantee you the results that youll be cured, or that your pain will be completely gone. But when you take a look at how stem cells are helping people with cancer it logically makes sense that stem cell therapy is a very powerful option. Specifically, stem cells have been researched in their effectiveness for cancer treatment, and both autologous and allogeneic options have their own benefits to consider.

Advantage of autologous stem cell transplant is that the patient will be getting his/her own cells back. An advantage of allogeneic stem cell transplant is that the donor stem cells make their own immune cells, which may help destroy any cancer cells that may remain after high-dose treatment.

Now, lets be clear that stem cell therapy is not covered by most insurance. And the notion that just because insurance does not cover it means that stem cell therapy is not a good option is frankly just poor thinking.

FACT: Insurance did not cover bone marrow transplants when it first got started, but bone marrow transplants were clearly saving lives. Insurance will always try to pay for the least amount of options that they can while charging the most premium that they can. Thats their business.

So when youre digging into the details of whether you should get stem cell shots in Ohio, then, there is the cost that is a big consideration. A Regenerative Medicine of North Florida stem cell shot costs just on average of $4,000 per shot. And we have finance options that allow you to make easy monthly payments. This is significantly lower than most other options. Autologous treatment, meanwhile, is $7,000 to $20,000 or more. Yes, that is really expensive and in most cases, it means people have to pay that money before they can get treatment. This means they get a loan or get the treatment financed.

Regenerative Medicine of North Florida has a price structure that gets you top-notch quality care at an affordable price. Yes, people do choose some of our finance options too. But many just pay upfront because Regenerative Medicine of North Florida stem cell shots cost almost half the cost of many of your other options

American clinics charge approximately $10,000 per treatment. Notably, many patients get more than one of these non-FDA approved treatments and must pay each time of course. Some clinics have reduced prices to the $7,000-$8,000 range. Interestingly, costs for treatments outside of the US are usually far higher than in the US, charging anywhere from $20,000 all the way up to $100,000. These clinics still generally have Americans as clientele.

If youre still reading then weve covered a lot of information. So lets talk about the other elephant in the room when it comes to stem cell therapy.

FACT: Stem cell therapy is not FDA approved. These procedures, just like a skin graft, is not FDA approved. The regenerative cells we use come from a tissue bank that is overseen by the American Association of Tissue Banks.So that lets you know the cells are really highly monitored, and the FDA is getting really more involved in this, that is theyre going to the manufacturers. Theyre going to the places that take the cells from the umbilical cord, manufacture them and send them to our offices to use. Theyre really monitoring that process. So the FDA is really standardizing that, making sure all the safety qualifications are up where they need to be.

And thats why weve vetted labs to make sure that we use the best labs. The ones that are actually working with the FDA to make sure that they have the highest manufacturing standards possible, so you get the best cells. And that its the safest cell that you can get as well.

Just to be clear again, the American Association of Tissue Banks, or the AATB, set up guidelines for these labs that they need to follow. If they dont have these safe rooms and clean rooms and different things that are necessary to do, theyd never be able to do this. Its just like any other tissue donation process.

So think about someone who donates a kidney or a liver, those types of things. Its going to go through a lot of screening. That person, first of all, their history. Theyre gonna screen the tissue itself. Theyre gonna make sure there are no diseases, to make sure its pure so they can use it.

Where the FDAs focus is right now, its actually called the FDA INTERACT program to help and make sure they standardize the manufacturing process so that you do get the best cells, the safest and most effective cells available on the market.

For most of our clients cost is more important than the FDA approval because smart people realize that the FDA approves drugs every year that end up having side effects, including death, and in many cases, after they approve a drug, they will then, (after the drug companies make massive profits,) then essentially say: OOPS, lets take that one off the market.

So, when you really think about it, the FDA does not always get things right. Just look up Celebrexs FDA history.

Are we saying the FDA is bad? NO. The FDA serves a vital part in public health.

Yet, to think that the multi-billion dollar pharmaceutical companies who DO NOT WANT YOU TAKING NATURAL, non-drug options, in the place of their multi-billion dollar pills, MAY have the ability to lobby the decision-makers to impact whether the FDA says Yes or No to a treatment, then you must still be living under a rock somewhere and youd be a good candidate to buy some swampland in Florida.

Enough said there.

Lets wrap this up.

Anyone looking to have stem cell treatment should consider the pros and cons of all the different options. Our Regenerative Medicine of North Florida team believes that the closer you look at us vs your other options then the better we will look to you. That being said, Regenerative Medicine of North Florida.com (HUCT) stem cell therapy is quickly becoming the most popular option and for good reason.

Listen to what the National Institute for Health (a government website) has to say in one of their medical publications:

Human umbilical cord blood (HUCB) is being increasingly used as an alternative source of stem cells for cell-based therapy for malignant and nonmalignant diseases. HUCB is preferred to bone marrow because of its easy availability, low potential for graft-versus-host disease and tumorigenicity as well as infectious complications. Furthermore, no immunosuppression is required.https://www.ncbi.nlm.nih.gov/pubmed/20528762

To date, various studies have shown the effectiveness of this type of treatment, and we have real people who have real results who you can talk to when you come to one of our Free Seminars.

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Advances In Treatment Of Soft Tissue Injuries (Video) – South Florida Reporter

Posted: January 3, 2020 at 5:49 pm

Soft tissue injuries in muscles, tendons and ligaments, andosteoarthritis, can make moving around painful and limit your physical activity. ButDr. James Presley,a Mayo Clinic physical medicine specialist, says two specialized treatments are growing more common and can help you heal faster.

Platelet-rich plasmais a specialized treatment that Dr. Presley says can bring relief for many patients dealing with soft tissue injuries.

Platelet-rich plasma is a way of trying to harness the bodys immune system or the bodys own ability to heal tissues, Dr. Presley says. [We] concentrate it and then spot-shoot it into the area of injury.

Dr. Presley says the process involves taking blood from your arm, processing it to concentrate the platelets, then injecting it directly into the affected area.

These treatments seem to be helpful in helping the healing process move along when it comes to tendon and ligament injuries, and potentially to help decrease pain and improve function in a joint that has some arthritis, Dr. Presley says.

The second treatment is calledbone marrow aspirate concentrateand involves extracting cells, including stem cells, from bone marrow in the pelvis; processing them into a solution; and injecting them into a painful joint.

The studies that have been done with this have shown patients have decreased pain and thereby improved function of a joint with mild to moderate osteoarthritis, Dr. Presley says.

But he says the best thing you can do is protect your muscles, tendons, and ligaments from injury by finding a happy medium between staying active and avoiding overuse.

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Advances In Treatment Of Soft Tissue Injuries (Video) - South Florida Reporter

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What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it – FOX 10 News Phoenix

Posted: January 3, 2020 at 5:47 pm

This undated screen grab shows the cell-division of two fertilized human embryos during the first 24 hours of embryonic development following IVF treatment at a private clinic in London. ( Jim Dyson/Getty Images )

LOS ANGELES - Some of the scientific advancements of the 2010s have been truly mind-blowing, and perhaps none more so than the leaps and bounds weve made in the realm of reproduction.

This was not only the decade in which the first three-parent baby was born, it was the era when a rogue scientist chose to make edits to a set of twin girls DNA, making real the long-imagined scenario of genetically altering human beings while simultaneously thrusting the deeply complicated ethical discussions surrounding this practice into the limelight.

These are the five most life-altering breakthroughs in reproduction from the past decade.

In 2018, Chinese biophysics researcher He Jiankui announced that he had used the gene-editing tool CRISPR to modify the genes of two twin girls before birth. He and his team said that their goal was to make the girls immune to infection by HIV through the elimination of a gene called CCR5.

When the news broke, many mainstream scientists criticized the attempt, calling it too unsafe to try. Where some people saw the potential for a new kind of medical treatment capable of eradicating genetic disease, others saw a window into a dystopian future filled with designer babies and framed by a new kind of eugenics.

At the time, Dr. Kiran Musunuru, a University of Pennsylvania gene-editing expert, said Hes work was unconscionable... an experiment on human beings that is not morally or ethically defensible.

Other experts believe Hes work could propel the field of gene editing forward.

The twins, known as Lulu and Nana, have continued to make headlines since their birth. The gene modification that He claims to have carried out may have caused some unintended mutations in other parts of the genome, which could have unpredictable consequences for their health long term something many scientists who argue against Hes work cite as a reason to hold off on using gene-editing technology on humans.

Only time will tell what will happen to Lulu and Nana and if the edits to their DNA ultimately help or hurt them, but their story pushed the topic of human gene-editing and the ethics surrounding it to the forefront of the global scientific community.

In 2016, a technique called mitochondrial transfer was used successfully for the first time to create a three-parent baby grown from a fathers sperm, a mothers cell nucleus and a third donors egg that had the nucleus removed.

This technique was developed to prevent the transmission of certain genetic disorders through the mothers mitochondria. The majority of a three-parent babys DNA would come from his parents in the form of nuclear DNA, and only a small portion would come from the donor in the form of mitochondrial DNA.

A team led by physician John Zhang at the New Hope Fertility Center in New York City facilitated the birth of the first three-parent baby in April 2016.

Using human pluripotent stem cells, researchers were able to make the precursors of human sperm or eggs. In other words, they reprogrammed skin and blood stem cells to become an early-state version of what would eventually become either sperm or an egg.

"The creation of primordial germ cells is one of the earliest events during early mammalian development," Dr. Naoko Irie, first author of the paper from the Wellcome Trust/Cancer Research UK Gurdon Institute at the University of Cambridge told Science Daily. "It's a stage we've managed to recreate using stem cells from mice and rats, but until now few researches have done this systematically using human stem cells. It has highlighted important differences between embryo development in humans and rodents that may mean findings in mice and rats may not be directly extrapolated to humans."

A 2018 study showed that gene editing can allow two same-sex mice to conceive pups, and two female mice were able to successfully create healthy pups that then went on to reproduce themselves.

A team of researchers at the Chinese Academy of Sciences in Beijing, led by developmental biologist Qi Zhou, were able to use gene editing to produce 29 living mice from two females, seven of which went on to have their own pups. They were able to produce 12 pups from two male parents, but those offspring were not able to live more than two days.Whether or not the method can one day be used in same-sex human reproduction is still up for debate.

For the first time ever, Chinese scientists were able to clone two primates using the technique that produced Dolly the sheep, the first mammal to be cloned from an adult somatic cell via nuclear transfer.

The two cloned female macaques were named Zhong Zhong and Hua Hua, and their successful birth opened up the possibility of using the same cloning method to one day clone humans.

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Diomics Corporation and University of California, Irvine Collaborate to Enable Cell Therapy Clinical Trial for Type 1 Diabetes – BioSpace

Posted: January 3, 2020 at 5:44 pm

SAN DIEGO, CA, Jan. 02, 2020 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE --Diomics Corporation, a leader in forensic, diagnostic, and therapeutic science since 2009, and the laboratory of Dr. Jonathan Lakey, Professor of Surgery and BiomedicalResearchand Director of the Clinical Islet Program at the University of California, Irvine (UCI), today announced a Sponsored Research Agreement to ultimately improve islet transplantation for patients living with type 1 diabetes.

The current method for islet transplantation requires invasive, difficult, and time consuming surgeries that create stress and risk for both the patients and the islets. To circumvent these issues, cell encapsulation has been proposed as the next treatment option. Biomaterials can protect the transplanted islets from destruction from the body. Polycaprolactone (PCL) polymer has been used in cell replacement therapy, however, the PCL polymer degrade too slowly and exhibit poor cell adhesion qualities for optimal cell replacement therapy. Diomics technology overcomes these issues for improved cell adhesion.

Leading the Diomics Sponsored Research Agreementresearch at UCI is Dr. Jonathan Lakey, a world-class subject matter expert on cell therapies including pancreatic islets and stem cells. Dr. Lakey has pioneered the development of novel methods for implantation of pancreatic islets for patients with diabetes. I am thrilled for the opportunity to work with Diomics and examine this novel and important proprietary biomaterial, said Dr Lakey. I am most excited about the potential variety of applications for this novel material.

Diomics recently filed provisional patents with claims broadly covering its proprietary polymer technology, Diomat, for applications in cell therapy, transdermal and related drug delivery methods. The Diomics and UCI research will support the development of key data that can be leveraged in clinical trials for improved islet transplantation therapy. Improved islet transplantation can restore natural insulin production for type 1 diabetes patients.

In this sponsored research project, Diomat foams will be used to characterize the Diomics material and examine encapsulated pancreatic islets and stem cells for improved islet transplantation therapy. This data will provide the key results to proceed with clinical trials using Diomat foam-encapsulated products.

Diomics is committed to providing innovative solutions through its materials and technologies that will lead the way to remarkable life science discoveries, said Diomics Chairman of the Board, Kirk Avery. We are honored to collaborate with Dr. Lakey and UCI.

ABOUT DIOMICS CORPORATION

Diomics Corporation creates highly efficient hydrophilic materials, based on patented Diomat technology, that improve the speed, sensitivity, and accuracy in the capture and detection of nucleic acids, proteins, and similar compounds. Our technology has broad applicability in a multitude of nanoscale settings in biomedical engineering, genomics, proteomics, and stem cell research. Diomics has filed a total of 20 patents and has 12 issued patents. For more information, visit:www.Diomics.com

ABOUT DR. LAKEY AT UCI

Dr. Jonathan Lakey is Professor of Surgery and Biomedical Engineering at the University of California, Irvine, and a world-class subject matter expert on cell therapies including pancreatic islets and stem cells. Dr. Lakey has over 395 publications and authored 45 book chapters and has pioneered the development of novel methods for implantation of pancreatic islets for patients with diabetes.

Diomics Contact:

Eric J. Mathur, CEO

Diomics Corporation

cell: 760.889.8929

emathur@Diomics.com

http://www.Diomics.com

Investor Relations Contact:

Jeff Ramson, Founder & CEO

PCG Advisory, Inc.

phone: 646-863-6893

jramson@pcgadvisory.com

http://www.pcgadvisory.com

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Diomics Corporation and University of California, Irvine Collaborate to Enable Cell Therapy Clinical Trial for Type 1 Diabetes - BioSpace

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BrainStorm Cell Therapeutics Wins 2020 ‘Buzz of BIO’ Award for ALS Investigational Therapy – ALS News Today

Posted: January 3, 2020 at 5:44 pm

For its promising investigational therapeutic approach to neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics is theBuzz of BIO 2020 winnerin the Public Therapeutic Biotech category.

The Buzz of BIO contest identifies U.S. companies with groundbreaking, early-stage potential to improve lives. The event also is anopportunity to make investor connections that could take products to the next phase.

Ten biotechnology companies are nominated in each of the three categories ofBuzz of BIO: Public Therapeutic Biotech, Private Therapeutic Biotech, and Diagnostics and Beyond. In the Public Therapeutic Biotech category that BrainStorm won, nominated companies must be actively developing a publicly traded human treatment intended for review by theU.S. Food and Drug Administration (FDA).

As a developer of autologous cellular therapies treatments that use a patients own cells and tissues for debilitating neurodegenerative diseases, BrainStorm is now testing its NurOwn therapy for safety and effectiveness. The treatment involves extracting, from human bone, marrow-derived mesenchymal stem cells (MSCs), which are capable of differentiating into other cell types. The MSCs are then matured into a specific cell type that produces neurotrophic factors compounds that promote nervous tissue growth and survival. They are then reintroduced to the body via injection into muscles and/or the spinal canal.

Backed by a California Institute for Regenerative Medicine grant, Brainstorm has fully enrolledits randomized, double-blind, placebo-controlled Phase 3 clinical trial (NCT03280056) at six U.S. sites in California, Massachusetts, and Minnesota. Some 200 ALS patients are participating. A secondary safety analysis by the trials independent Data Safety Monitoring Board (DSMB) revealed no new concerns. Every two months, study subjects will be given three injections into the spinal canal of either NurOwn or placebo.

The trial is expected to conclude late this year. Results will be announced shortly afterward.

In a Phase 2 study (NCT02017912), which included individuals with rapidly progressing ALS, NurOwn demonstrated a positive safety profile as well as prospective efficacy.

The use of autologous MSC cells to potentially treat ALS was given orphan drug status by both the FDA and the European Medicines Agency.

Thanks to everyone who voted for BrainStorm during the Buzz of BIO competition,Chaim Lebovits, BrainStorm president and CEO, said in a press release. The entire management team at BrainStorm was very pleased with the results of this competition, and we look forward to presenting to an audience of accredited investors who may benefit from the companys story. We thank the BIO[Biotechnology Innovation Organization] team for singling out BrainStorms NurOwn as a key technology with the potential to improve lives.

As a contest winner, BrainStorm is invited to givea presentation at theBio CEO & Investor Conference, to be held Feb. 1011 in New York City, along with exposure to multiple industry elites and potential investors.

NurOwn cells also are being tested in a Phase 2 clinical study (NCT03799718) in patients with progressive multiple sclerosis.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

Total Posts: 6

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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BrainStorm Cell Therapeutics Wins 2020 'Buzz of BIO' Award for ALS Investigational Therapy - ALS News Today

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Gene Therapies Make It to Clinical Trials – Discover Magazine

Posted: January 3, 2020 at 5:44 pm

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

Theres been a convergence of the science getting better, the manufacturing getting much better, and money being available for these kinds of studies, says Cynthia Dunbar, a senior investigator at the National Heart, Lung, and Blood Institute. Its truly come of age.

CRISPR formally known as CRISPR-Cas9 has been touted as an improvement over conventional gene therapy because of its potential precision. CRISPR (clustered regularly interspaced short palindromic repeats) is a genetic code that, contained in a strand of RNA and paired with the enzyme Cas9, acts like molecular scissors that can target and snip out specific genes. Add a template for a healthy gene, and CRISPRs cut can allow the cell to replace a defective gene with a healthy one.

In April, scientists at the University of Pennsylvania announced they had begun using CRISPR for cancer treatments. The first two patients one with multiple myeloma, the other with sarcoma had cells from their immune systems removed. Researchers used CRISPR to genetically edit the cells in the lab, and then returned them back into their bodies.

On the other side of the country, Mark Walters, a blood and bone marrow transplant specialist at the University of California, San Francisco, Benioff Childrens Hospital in Oakland, is gearing up for trials that will use CRISPR to repair the defective gene that causes sickle cell disease. With CRISPR, once youve made that type of correction, [that cell] is 100 percent healthy, says Walters.

Another team is tackling the same disease using a type of hemoglobin, a protein in red blood cells, thats normally made only in fetuses and newborn babies. Researchers found that some adults continue to produce these proteins throughout their lives, and when those adults also have sickle cell disease, their symptoms are mild. So the international team used CRISPR to disable the gene that interferes with production of this hemoglobin, resuming its production and protecting the adult patients against sickle cell disease.

Several other CRISPR studies are in the works to treat a range of inherited disorders, including hemophilia and SCID-X1 (also known as X-linked severe combined immunodeficiency, the so-called bubble boy disease in which babies are born without a functioning immune system).

At St. Jude Childrens Research Hospital, a gene therapy trial cured Gael Jesus Pino Alva (pictured with his mother, Giannina) of SCID-X1, the bubble boy disease. (Credit: St. Jude Children's Research Hospital/Peter Barta)

The past year also saw success in a handful of experiments on conventional gene therapy. Instead of using CRISPR to repair disease-causing genes, these treatments use hollowed-out viruses to ferry healthy versions of genes into cells. Millions of these altered cells are released into the bloodstream or bone marrow in hopes that enough will land in the right places. But because scientists cant predict where the circulating genes may end up, this shotgun approach has had unintended, sometimes fatal, consequences including, in an earlier study, inadvertently activating leukemia-causing genes in patients treated for SCID-X1.

But in 2019, researchers learned that using a different type of virus one related to HIV to transport the genes may prevent these side effects. In an April study, researchers at St. Jude Childrens Research Hospital in Memphis, Tennessee, and UCSF Benioff Childrens Hospital in Oakland collected bone marrow from eight newborns with SCID-X1. They loaded corrective genes into the disabled HIV-related virus, which carried them into the patients bone marrow stem cells. The infants also received low doses of busulfan, a chemotherapy that gave the doctored stem cells room to grow. So far, we havent seen anything worrisome, says Ewelina Mamcarz, a pediatric oncologist at St. Jude who led the research team. The study recently added its 12th patient.

Gene therapy does have its momentum [back], says Mamcarz, reflecting on the fields setback after the earlier studys leukemia side effects. Theres so much that still needs to be done, and so many questions, she says. [But] this is how medicine evolves. We always want to be better than we were a week ago.

In the future, the hope is that gene therapy technologies will move beyond mending simple genetic mistakes and be used to combat big killers like diabetes or heart disease. [Those diseases are] more challenging, but a lot of them would benefit from knocking out a bad gene, says Dunbar.

For now, though, researchers are optimistic about the progress thats already been made. All of this has been very encouraging, says Dunbar. [And] for sickle cell in the U.S. and hemophilia in the developed world, these diseases may soon be solved.

[This story originally appeared in print as "Gene Therapy Gets Clinical."]

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Global Testosterone Replacement Therapy Market Expected To Feasible Growth , Recent Trends Over 2025 – Testifyandrecap

Posted: January 2, 2020 at 7:45 pm

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