Stem cell treatment for lethal STAT1 gene mutation produces mixed … – Medical Xpress

June 8, 2017 One example of STAT1 GOF Mutation phenotype. Credit: Hiroshima University

Researchers report the first-ever study assessing how patients with "gain of function" mutation of the STAT1 gene respond to stem cell transplantation. It involved 15 young patients from nine different countries, each suffering a range of complications caused by the gene's mutation.

Of these, only six survived a regime of stem cell transplantationwith five completely cured and disease free by the study's conclusion.

The study was carried out by Dr. Satoshi Okada (Hiroshima University), Professor Jennifer Leiding (University of Florida), Professor Tomohiro Morio (Tokyo Medical and Dental University), and Professor Troy Torgerson (University of Washington).

Dr. Okada, who first discovered the STAT1 gain of function mutation in 2011, says, "Overall, this result is disappointing but the fact that five patients were cured proves that treatment with stem cells can work, and we now need to learn from these 15 individual cases."

The STAT1 gene plays a vital role in the body's immune system. Rare mutations can lead to STAT1's over-activation (GOF) and autoimmunity.

While the majority of patients afflicted typically show mild to moderate symptoms involving fungal (mostly Candida), bacterial, and viral infectionsabout 10 percent of cases are severe and life threatening.

Until now, developing suitable treatments has been challenging; e.g. anti-fungal drugs temporarily treat the symptoms but not the source mutation, and immunosuppressive therapies often do more harm than good by knocking out already overburdened immune systems.

With only one confirmed case prior to this study of a sufferer being successfully cured using stem cell transplantation, researchers are keen to build an understanding of best practices in order to offer real hope for the typically young sufferers of this condition.

The 15 selected patients were sourced via an international appeal to transplant centers and consortiums. Their ages ranged from 13 months to 33 years at the time of treatment. Screening by HU researchers confirmed that each had the STAT1-GOF mutation, and that the mutation was the source of their ailments.

Treatment was carried out independently by centers around the world. It used chemotherapy to eradicate the host's bone marrowthe source of the damaging STAT1 mutation in these patients. Healthy stem cell cultures sourced from donors were then transplanted into the subjects with the aim of reconstituting their bone marrow to a mutation-free, disease-fighting state.

The researchers suspect three reasons for the low 40 percent success rate:

In response, the researchers have made several proposals for improving this treatment. Due to most of the patients having mild to moderate ailments, only those suffering from severe symptoms should undergo this treatment. In addition, the chemotherapy dosage should be reduced. Those who received low-dose chemotherapy reacted better.

However, a balance must be struck. Low-dose chemotherapy may not eradicate host bone marrow to the extent required for its reconditioning the chance of transplant rejection is thus increased. With this in mind, support treatment may be required to neutralize host antibodies and prevent attacks of introduced stem cells.

Finally, due to the relative success seen in younger patients, stem cell transplantation should occur at as early an age as possible. Due to recent advancements in STAT1-GOF diagnosis, early detection is now a very real possibility hopefully leading to greater success rates, and less suffering for those carrying this potentially devastating mutation.

Explore further: 'Smart' genetic library makes disease diagnosis easier

More information: Jennifer W. Leiding et al. Hematopoietic stem cell transplantation in patients with Gain of Function STAT1 Mutation, Journal of Allergy and Clinical Immunology (2017). DOI: 10.1016/j.jaci.2017.03.049

Researchers at Hiroshima University have developed a smart genetic reference library for locating and weeding out disease-causing mutations in populations.

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Texas is leading the charge on stem cell therapy ThinkProgress – ThinkProgress

In the last weeks of the legislative session, Texas lawmakers passed a historic bill that legalizes investigative stem cell treatments for chronically ill patients. Despite the prevalence of unregulated stem cell clinics in the United States, this could be the first time a state has authorized the treatment for chronic illnesses, if the states governor approves the legislation.

Youre watching an episode of In Session, a weekly series exploring interesting policy changes on the state level.

PHOEBE GAVIN, ThinkProgress: Youll never guess which state is on track to become the first to recognize stem cell therapy as a treatment for chronic illnesses. Ill give you a hint: its former governor and our former president once banned federal funding for research on embryonic stem cells

You guessed it: Its The Lone Star State!

In the last week of its legislative session, Texas lawmakers approved a bill that legalizes Investigational Stem Cell Treatment, which basically means treatments that have been evaluated by an institutional review board but have not yet been approved by the FDA.

Keep in mind, though: this isnt about embryonic stem cells. The cells will come from banked umbilical cords and the patients themselves, who will be allowed to pursue the treatment as a last resort. So people who suffer from debilitating chronic illnesses like Parkinsons, ALS, and multiple sclerosis could see relief.

For TX Rep. Springer, this is personal.

REP. DREW SPRINGER (R-TX68): I pray to God every time I go to mass, every time I close my eyes, that one day my wifeand not for my sake, but for her sakewould have the chance to have that opportunity again to be able to walk.

GAVIN: The bill almost missed the deadline until he came to the podium. After his plea, it unanimously passed the House and sailed through the Senate. It now awaits Gov. Greg Abbotts signature, and hes already tweeted his support for it.

But not everyone is on board. Two different stem-cell research organizations have vocalized their opposition, arguing that the lack of quote rigorous evidence of safety and efficacy would put vulnerable patients at risk.

But the reality is, patients have already been taking this risk without the states explicit authorization. There are hundreds of clinics across the country that have been operating under regulatory loopholesat least 71 in TX alone. Without regulation, these clinics are completely unchecked. And patients who seek help there have felt the consequences: theyve been blinded, developed tumors and even died after seeking unregulated stem cell treatment. But with this new bill, if something goes wrong, patients have the right to sue.

So there you goTexas, creating accountability for stem cell clinics for the first time in the US. An unprecedented act from a unpredictable state.

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Texas is leading the charge on stem cell therapy ThinkProgress - ThinkProgress

Bioquark Hopes to Bring Dead Back to Life Using Stem Cell Therapy – India West

A Philadelphia-based company along with an Indian orthopedic surgeon has said it will start a new type of stem cell therapy that claims it can bring people back from the dead.

Bioquark, led by chief executive Ira Pastor, said it will begin conducting trials of the therapy later in the year in an undisclosed country in Latin America, according to a New York Post report.

Pastor and Indian orthopedic surgeon Himanshu Bansal had initially hoped to run tests in India in 2016 but the Indian Council of Medical Research pulled the plug on their plans and asked them to take the trials elsewhere, the Post reported.

Most countries officially declare someone dead when there is irreversible loss of brain function. The Bioquark therapy boasts it can reboot the brain.

The company said it will begin testing on humans, with no plans to experiment on animals.

Scientists plan to examine individuals aged between 15 and 65 who have been declared brain dead from a traumatic brain injury, the report noted, citing a published study.

The three-stage process starts with harvesting stem cells from the patients own blood before injecting them back into their body. Next, the patient is given a dose of peptides injected into their spinal cord. The final step is a 15-day course of laser and median nerve stimulation while monitoring the patient with MRI scans.

Bansal practices in New Delhi.

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Bioquark Hopes to Bring Dead Back to Life Using Stem Cell Therapy - India West

Doctors Targets Stem Cell Therapy Launch – Bahamas Tribune

ByNEIL HARTNELL

Tribune Business Editor

nhartnell@tribunemedia.net

DOCTORS Hospital plans to launch stem cell therapy and enter the primary healthcare market during its current financial year, after profits for the year to end-January 2017 increased five-fold.

The BISX-listed healthcare provider said it planned to launch both initiatives at its Bahamas Medical Centre facility on Blake Road, having received the necessary approvals for one stem cell programme and another in its final stages.

Doctors Hospitals 2017 annual report did not identify the types of stem cell treatment involved, but said: It is envisioned that stem cell therapy will occur at the facility [Bahamas Medical Centre] in fiscal 2018, with one programme already receiving the necessary approvals and the second programme in its final stages of assessment and approval......

We anticipate that in fiscal 2018 we will launch one of our primary care centres at this location, supported by increased specialist services to best serve the neighbouring communities.

Joe Krukowski, Doctors Hospitals chairman, told shareholders via the annual report that the launch of primary care services will be a vital component in the continuum of care we provide.

We will seek to provide our customers with multiple entry points for this level of care, he added.

Doctors Hospitals stem cell initiatives, in particular, represent a potential boost to the Bahamas national effort to make greater inroads into the medical tourism market while also exploiting legislation passed by the former Christie administration.

The healthcare providers move into primary care will effectively create a fully-integrated model, combining with its core business in secondary and tertiary care provision to potentially make Doctors Hospital almost a one-stop shop for all medical needs.

The expansion comes after Doctors Hospital saw total comprehensive income for the year to end-January 2017 grow by 409 per cent or more than five-fold, from $702,790 to $3.578 million year-over-year.

The growth was driven entirely by the companys main Collins Avenue facility, where profits more than doubled, increasing by 157.4 per cent to $4.778 million compared to $1.856 million the year before. The Bahamas Medical Centres net loss increased slightly compared to the prior year, rising from $1.153 million to $1.2 million.

An improved top-line drove Doctors Hospitals improved profitability, with patient services revenue up $3.65 million or 7.4 per cent at $52.713 million.

Patient days increased by 6 per cent from the previous year, the annual report said of the main Collins Avenue hospital. Increases in the Intensive and Intermediary Care Units accounted for 37per centof the change, and the balance in medical surgical and maternity.

Total admissions to the facility were 4,114 in fiscal 2017 compared to 4,063 in fiscal 2016. The continued flat admission numbers and increased patient days are indicative of the trend toward a rising severity of illness. The average daily census increased to 33 patients per day from 31.2 in the previous year.

Doctors Hospitals total expenses grew by $818,452 or 1.7 per cent year-over-year, with salaries and benefits rising by $1.176 million or 5.6 per cent to $23.209 million. Due to the top-line growth, these fell as a percentage of patient net revenue from 44.3 per cent to 43.5 per cent.

At Bahamas Medical Centre, revenues rose by $28,015 or 1.9 per cent to $1.462 million. This slightly outpaced the increase in expenses, which jumped by 1.5 per cent or $43,479 to $2.819 million as a result of rising medical supplies costs.

Doctors Hospital is budgeting $7 million for capital spending projects in its financial year to end-January 2018, a sum more than double the prior years $3.1 million, as it bids to upgrade facilities and replace equipment.

Bad debt expense, as a percentage of patient service revenues, decreased to 2.6per centfor the year ended January 31, 2017, compared to 3.4per centthe previous year, Doctors Hospital said.This represented a decrease of $316,808, or 18.8per cent. This decrease is a result of a write-off of third-party receivables.

The number of days revenue in accounts receivable at year-end (AR Days) for fiscal 2017 stand at 51 compared with fiscal 2016 at 43 days, and net receivables as a percentage of net patient revenue increased to 14.1per centfrom 11.8per cent. These increases area result of high activity in the months of December and January, and payments not received until after year-end.

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Doctors Targets Stem Cell Therapy Launch - Bahamas Tribune

Column: Stem Cell Therapy A medical revolution – Current in Carmel

Commentary by Dmitry M. Arbuck, MD, President and Medical Director, Indiana Polyclinic

We are at a truly revolutionary time in health and medicine. The introduction of stem cell technology represents innovation on the same level as the development of antibiotics or the invention of modern imaging (MRIs, etc.). Stem cells are already changing the way medicine is delivered, increasing lifespans and saving countless lives.

Arbuck

Scientists and researchers have been studying the benefits of stem cells for more than 30 years. They have found that these special cells provide great benefits all over the body, from muscles and joints to chronic diseases, to growing new teeth. You may have read about athletes treated with stem cells to speed healing after an injury or about burn victims who use stem cell therapy to minimize scarring.

Stem cells used to be associated with embryos, but this is no longer the case. Today, live cells for treatment are either adult stem cells or umbilical cord blood stem cells. Adult stem cells are most likely extracted from tissue, like bone marrow or fat, which can be a painful and invasive process. Additionally, as we age, so do our stem cells, which become less potent and productive over time. Like every other tissue in our bodies, they are exposed to the toxins, radiation and other pollutants in the environment. Umbilical cord blood stem cells are collected from the donated cord blood and placenta of healthy newborns. The cells are then screened for disease and genetic problems. These umbilical stem cells are vibrant, vital and healthy.

When umbilical cord stem cells are infused, they carry a whole host of immune stabilizing factors throughout the body and work to repair the immune system. This is likely why stem cells are so helpful in the treatment of autoimmune diseases such as rheumatoid arthritis, Crohns disease, dermatitis and myasthenia gravis. Other things that may be successfully treated with this therapy include MS, lupus, graft vs. host disease and other immune conditions.

The future is today. For more, visit StemCellsIndy.com.

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Column: Stem Cell Therapy A medical revolution - Current in Carmel

Column: Stem Cell Therapy A medical revolution – Current in Westfield

Commentary by Dmitry M. Arbuck, MD, President and Medical Director, Indiana Polyclinic

We are at a truly revolutionary time in health and medicine. The introduction of stem cell technology represents innovation on the same level as the development of antibiotics or the invention of modern imaging (MRIs, etc.). Stem cells are already changing the way medicine is delivered, increasing lifespans and saving countless lives.

Arbuck

Scientists and researchers have been studying the benefits of stem cells for more than 30 years. They have found that these special cells provide great benefits all over the body, from muscles and joints to chronic diseases, to growing new teeth. You may have read about athletes treated with stem cells to speed healing after an injury or about burn victims who use stem cell therapy to minimize scarring.

Stem cells used to be associated with embryos, but this is no longer the case. Today, live cells for treatment are either adult stem cells or umbilical cord blood stem cells. Adult stem cells are most likely extracted from tissue, like bone marrow or fat, which can be a painful and invasive process. Additionally, as we age, so do our stem cells, which become less potent and productive over time. Like every other tissue in our bodies, they are exposed to the toxins, radiation and other pollutants in the environment. Umbilical cord blood stem cells are collected from the donated cord blood and placenta of healthy newborns. The cells are then screened for disease and genetic problems. These umbilical stem cells are vibrant, vital and healthy.

When umbilical cord stem cells are infused, they carry a whole host of immune stabilizing factors throughout the body and work to repair the immune system. This is likely why stem cells are so helpful in the treatment of autoimmune diseases such as rheumatoid arthritis, Crohns disease, dermatitis and myasthenia gravis. Other things that may be successfully treated with this therapy include MS, lupus, graft vs. host disease and other immune conditions.

The future is today. For more, visit StemCellsIndy.com.

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Column: Stem Cell Therapy A medical revolution - Current in Westfield

Stem cell-based spinal cord therapy expanded to more patients – The San Diego Union-Tribune

An experimental therapy to repair spinal cord injury with stem cell-derived tissue is progressing smoothly, according to a leader of that trial who spoke at a conference on stem cell therapy.

The Phase 1 safety trial is proceeding with no complications, said Dr. Joseph Ciacci, a University of California San Diego neurosurgeon. The conference was held last week at the Sanford Consortium for Regenerative Medicine in La Jolla.

With safety looking good, the green light has been given to treat more patients, Ciacci said. However, to produce effectiveness, more cells will need to be transplanted.

Four patients have been treated with neural stem cells, injected into the spinal cord. They had experienced complete loss of motor and sensor function below the injury. They had been injured between 1 and 2 years previously.

Moreover, the cells show signs of integrating with the surrounding tissue in animal studies, Ciacci said. If the preliminary evidence holds up, Ciacci and colleagues plan to submit a paper detailing the results.

Curing paralysis from spinal cord injury was a big selling point for those who successfully advocated Proposition 71, which authorized selling $6 billion in state bonds to establish and fund the California Institute for Regenerative Medicine, or CIRM. The institute got $3 billion, the remaining half is going for interest over the life of the bonds.

While CIRM has been under pressure to show results, doctors are taking great care to establish safety first in the spinal cord treatment, because of potential risks in the procedure.

We are now enrolling and recruiting for the second cohort, which is for chronic cervical spinal cord injuries, Ciacci said. They are medically classified as C5-C7 ASIA A Complete.

Chronic injuries need to have taken place more than 1 year before treatment. For this study, the injury must also be under two years old. The trial is being conducted at UCSD with Ciacci serving as the principal investigator.

For more information on the Phase I Chronic SCI study, contact Ciaccis research group at (619) 471-3698, nksidhu@ucsd.edu.

In addition, the researchers have been approved to start another spinal cord injury trial with a different set of cells. These oligodendrocyte progenitor cells, derived from embryonic stem cells, can turn into several different types of neural cells.

The trial, sponsored by Asterias, treats newly injured patients, between 14 and 30 days after injury.

For more information on the Asterias trial, contact the UCSD Alpha Stem Cell Clinic at 858-534-5932 alphastemcellclinic@ucsd.edu or visit http://www.scistar-study.com and j.mp/ucsdast.

Asterias acquired the technology from Geron, which had undertaken the work with a CIRM grant. Geron later canceled the work and refunded the money to CIRM. Asterias got funding from CIRM to continue the work.

The Asterias trial will use the same technique as used with the Chronic SCI trial, a technique which can improve safety, Ciacci said. The cells will be injected in a series of progressively larger amounts that may give evidence of the dose relates to effectiveness, although safety remains the main concern.

This cell line is cryopreserved, its sent to us as a single dose the day of surgery, Ciacci said. Were going to study different doses 2 million, 10 million, 20 million cells per injection. Its going to be a direct injection, just like what weve done before.

As in previous treatments, patients will also receive immune suppression to prevent rejection of the cells. Likewise, they will be monitored for many years after treatment.

Another trial coming to UCSD will test for efficacy in ALS, Ciacci said.

Ciacci said hes looking for qualified patients for these trials, and urged those in the audience to help find them.

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bradley.fikes@sduniontribune.com

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Stem cell-based spinal cord therapy expanded to more patients - The San Diego Union-Tribune

Stark County teen’s stem cell treatment ‘going in the right direction’ – Peoria Journal Star

Gary L. Smith of the Journal Star

TOULON A large benefit auction and dinner Friday evening will raise funds to help cover medical expenses for a Stark County teenager who is undergoing experimental stem cell treatment for a debilitating autoimmune disorder.

Proceeds of the event to be held 5 8:30 p.m. at the Orwig Auction and Event Center at the east edge of Toulon will benefit BrookeLynn Montgomery, 15, of rural Toulon, a Wethersfield High School student who was diagnosed last year with Postural Orthostatic Tachycardia Syndrome.

POTS symptoms, which arise from a malfunction of the autonomic nervous system, can include low blood volume, fiber neuropathy, dizziness, exercise intolerance, nausea, diminished concentration, fainting, and shortness of breath. They often are similar to those experienced in congestive heart failure and chronic obstructive pulmonary disease.

The teen received a two-stage stem cell treatment at a suburban Chicago clinic in April, and improvements seen since then indicate that things are going in the right direction, said her mother, Shelly Montgomery.

But the surgeon treating her believes that at least one more treatment probably will be necessary, though details of that will be determined at a follow-up appointment with Dr. Mark J. Holterman in Peoria next week, she added.

Weve had a little bit of improvement, Montgomery said. Shes not 100 percent yet, but she has more energy. She still has some symptoms, but they are getting better.

The out-patient treatments took place at the Mariam Clinic in Oakbrook Terrace, which specializes in regenerative medicine. It was co-founded by Holterman, according to its website.

Holterman diagnosed Brooke last year at Childrens Hospital of Illinois at OSF Saint Francis Medical Center of Peoria, where he also practices. He also is a professor of surgery and pediatrics at the University of Illinois College Of Medicine at Peoria.

He was really happy with the way the procedures went, said Montgomery. She did really well.

Fridays benefit has been organized by extended family members to help with the cost of the stem cell procedures, which are not covered by insurance, as well as accumulated costs of past medical care that was not covered or had extensive co-pays, Montgomery said.

At this point, we still have bills coming in from her being in the hospital last summer, she noted.

The items that have been donated for the auction range from Cubs and Cardinals tickets to Maui Jim sunglasses to a wide assortment of furniture to a whole hog and processing, ready to be picked up at a meat locker.

Its been overwhelming, Montgomery said. Theres been so many people who want to help people who dont even know Brooke.

Brooke is expected to be at the auction. Friends have been keeping in close touch with her, and she was even able to have a short visit to Matthiesen State Park recently, her mother said.

But she missed much of the school year and is still trying to finish her freshman year with visits from a tutor, Montgomery said. A key goal is for her to be able to return to school for her sophomore year.

Its going the right direction, she said. Thats all we can hope for at this point.

More information on the benefit is available at the Facebook page for the Brooke Montgomery Benefit, or by calling Ed Smith (her grandfather) at 883-8435. Donations can also be made to the Brooke Montgomery Medical Expenses account at State Bank of Toulon, 102 W. Main St., Toulon IL 61483.

Gary L. Smith can be reached at (800) 516-0389 or glsmith@mtco.com. Follow him on Twitter @Glsmithx.

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Stark County teen's stem cell treatment 'going in the right direction' - Peoria Journal Star

New Single Injection Stem Cell Treatment May be a "Cure" for Asthma and Allergies to Bees, Peanuts, Seafood – eMaxHealth

A mothers worse nightmare is hearing her child gasping for breath. Imagine a single injection treatment that could completely cure asthma and eliminate allergic responses for a lifetime by changing the gene within T cells which react to proteins in peanuts, seafood, and in bee venom.

In people who have allergies or asthma, the T cells have developed a kind of immune memory and have become resistant to treatment. Patients with asthma and allergies usually experience chronic respiratory problems and the potential of death due to anaphylaxis is very real. Allergic asthma affects approximately 235 million people worldwide.

Scientists working in gene therapy have taken blood stem cells and altered or wiped clean the memory within the T cell so they no longer respond to an allergen. As these new cells reproduce their cellular offspring are also free of the allergic memory response meaning the new healthy cells only produce healthy cells that are non-reactive.

When you are exposed to an allergy early in life you tend to be more likely to have an allergic response, says Associate Professor Ray Steptoe, Diamantina Institute, University of Queensland, and with each subsequent exposure the response gets bigger and bigger. What weve done is to interrupt that process and by altering the gene we can turn off that response. What that means is the disease is stopped in its tracks.

Associate Professor Ray Steptoe from The University of Queensland on Vimeo.

Current approaches to this disease really use drugs that limit side effects, limit acute symptoms, says Steptoe, but what we do is stop the underlying disease.

Steptoe believes the technology will revolutionize the treatment of severe allergies and prevent life-threatening allergic episodes by just getting one single shot of the altered blood stem cells. While this treatment has worked in mice it is not yet ready for use in humans and may take as long as 5 or 6 years before clinical trials in humans could begin and another 5 years of human trials.

One of the things that would really accelerate this research, adds Steptoe, would be to obtain additional research funding.

The study was published in JCI Institute June, 2, 2017.

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New Single Injection Stem Cell Treatment May be a "Cure" for Asthma and Allergies to Bees, Peanuts, Seafood - eMaxHealth

Custom Stem Cell Treatment Now Available for Orthopedic Pain – PR Web (press release)

NextStem logo

Tampa, FL (PRWEB) June 05, 2017

NextStem, a premier stem cell therapy service, is now offering a breakthrough treatment for pain management related to musculoskeletal injuries and joint conditions.

"Patients experiencing painful joints related to sports injuries, arthritis and other ailments now have an alternative treatment to surgery, says NextStem founder Rodolfo Gari, M.D., M.B.A. Its a new and better option for reclaiming a pain-free life.

Stem cell therapy is a process that uses unspecified cells to renew themselves and in some cases be turned into cells with specialized functions. Individual doctors partnering with NextStem have been using stem cell therapy since 2008, two years after researchers discovered that specialized adult cells could be genetically reprogrammed to assume a stem cell-like state.

Advantages of NextStem therapy are many:

Stem cell therapy may also involve injecting stem cells or blood products, known as plasma rich platelets, to relieve joint and muscle pain caused by injury, chronic conditions even previous surgeries. PRP treatments have been used by more than 70 professional athletes to relieve pain.

NextStem, based in Tampa, Fla., is currently available to patients in two locations in Texas and Florida, with plans to expand. In Texas, it is available at the Hurst Ambulatory Surgery Center, 1717 Precinct Line Road, Suite 101, (817) 605-9899; and in the Tampa Ambulatory Surgery Center, 4726 N. Habana Ave., Suite 100, (813) 769-8855.

For more information, call 855-957-3436 or visit http://www.nextstem.com

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Custom Stem Cell Treatment Now Available for Orthopedic Pain - PR Web (press release)