Author Archives: admin


Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says – KQED

A small stem cell trial in which patients with severe spinal injuriesappeared to make remarkable progress is still showing excellent results, according to the company conductingthe research.

One of the patients in the trial is 21-year-old Kris Boesen, from Bakersfield, California, whose story we reported on last year.A car crash had left theBakersfield, California native with three crushed vertebrae, almost no feeling below his neck, and a grimprognosis. Doctors believed he would live the rest of his life as a paraplegic.

Enter stem cell therapy. Most treatments for serious spinal injuries concentrate on physical therapy to expand the range of the patients remainingmotor skills and to limit further injury, not to reverse the actual damage. But last April, as part of an experimental phase 2 clinical trial called SCiStar, researchers injected Boesen with 10 million stem cells. By July, hehad recovered use of his hands to the point where he could use a wheelchair, a computer and a cellphone, and could take care of most of his daily living needs.In recent months his progress has continued, says his father.

Boesen is not the only patient to have improved in the trial, according toAsterias Biotherapeutics, which is conducting the research. Boesen is part of a cohort of six patients who were experiencing various levels of paralysis and were injected with the 10 million stem cell dose. In a Jan. 24update, the company saidfive of those patientshad improved either one or twolevels on a widely used scale to measuremotor function in spinal injury patients.

On Tuesday, Asterias issued a newupdate, announcingthat the sixth patient in the cohort has experienced a similar improvement.

While spontaneous recovery for spinal injury patients does occur,the likelihood of all six patients recovering to the degree they haveis less likely, researchers say.

This is as good as you could hope at this point, said Charles Liu, Boesens neurosurgeon and director of the USC Neurorestoration Center. So far all the evidence is pointing in the right direction.

To measure improvement in spinal injury patients, researchers use two yardsticks: the Upper Extremity Motor Scale, or UEMS, and the International Standards for Neurological Classification of Spinal Cord Injury, or ISNCSCI. On the UEMS scale,patients are scored from 0 to 5 on theirability to use five key muscles in the wrists, elbows and fingers. The ISNCSCI scale assesses where damage has occurred along the different levels of the cervical vertebrae, which generally determines the scope of impairment to the body and the level of care needed.

For instance, if a patient has sustained damage at the fourth cervical vertebra down, known as C-4, at the base of the neck, it generally means that person is paralyzed from the neck down, requiring round-the-clock care and a ventilator to breathe.A patient with a C-5 injury may not be able to move her arms or hands, requiring about 6 to 12 hours per day of assisted care; and at the C-6 level, better motor function mayallow a patient to take care of most of herdaily living needs on her own.

Which is all to say that even one level of recovery could substantially improve the daily life ofa spinal injury patient.

According to Asterias, all six patients in the 10million-cell cohort have improved their general UEMS scores, and jumped at least one motor level on the ISNCSCI scale on one or both sides of their body.

Two patients have improvedtwo motor levels on one side; and one patient,Boesen, has improved two motor levels on both sides.

Steve Cartt, president and CEO of Asterias, said anotherpatient, Jake Javier of Danville, California, has gonefrom partial paralysis to being able to use his hands well enough to considerpursuing a computer science career.

Throws Like a Regular Throw

In September, Boesens father, Rod Boesen, told us how excited he wasthat his son had regained some feeling in one of his feet. Last week, at11 months post-injection, the elder Boesensaid Kris has continued to improve.

Now he can move his toe and his knee together at the same time, Boesen said. Theyre about to give him a manual wheelchair now [instead of a motorized one]. He can grip with his hands enough to use a manual one.

Boesen said the movement in his sons arms and hands has greatlyimproved since September.Kris, a formerhigh school pitcher, had beenflinging a ball to his dog like people throw hand grenades, Boesen said. They kind of cradle them and thats how Kris would do it. But now he throws like a regular throw, tosses that ball down the hall, has that release point down, and just wings it.

Asterias is currently recruiting patients for a trial in which theyll receive 20 million stem cells, the optimal dose, according to company researchers. Two patients have already started the 20 million stem cell therapy, and six-month results from those patients will be released in the fall, Cartt said.

Patients who received 2 million stem cells in an earlier phase of the study have not shown much change in their condition, according to the Jan. 24 update.

Guarded Optimism

While Boesens father is impressed with the results, the optimism of researchers inside and outside the studyhas been guarded.The trial is still in its early stages, and the sample size is small, said Paul Knoepfler, a cell biology professor and stem cell researcher at UC Davis, who is not involved in the SCiStar study.

As a scientist, I still would want to wait for more data, Knoepfler said. Its certainly interesting, but its still early. Its a phase 2 trial.

To address the issue of small sample size, Asterias islooking at historical data to determinethe level of improvement for patients in similar circumstances who did not receive stem cell therapy. The company has said it found a meaningful difference in the recovery of its study patients compared to the norm.

Liu said one of the most importantresults is the lack of significant side effects or other negative outcomes resulting from the treatment to date.

Thats very significant to me, Liu said. Thats the first thing you look for, is anyone hurt from this therapy.

There was also a concern, he said, that some patients might regress over time, once the initial injection of stem cells wore off. Thathasyet to occur.

No one has lost anything theyve gained, Liu said. We were very happy to see that. This is all very promising.

The next step for the SCiStartrial will be to establish a control group, Cartt said.

Go here to see the original:
Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says - KQED

Caladrius: ‘Hitachi will unlock full potential of cell therapy CDMO PCT’ – BioPharma-Reporter.com

Hitachi Chemical Co. America will increase its presence in the cell therapy development and manufacturing space through the $75m acquisition of Caladrius Biosciences subsidiary PCT.

The US off-shoot of Japanese firm Hitachi Chemical has entered an agreement to up its position in cell therapy contract development and manufacturing organisation (CDMO) PCT by buying out majority shareholder Caladrius Biosciences for a total of $75m in a deal expected to close in May.

Im delighted to report the entry into an agreement for the acquisition of our remaining 80.1% interest in PCT by Hitachi Chemical, Caladrius CEO David Mazzo said on a conference call to discuss his firms Q4 results this week. This transaction has the potential to unlock the tremendous value of our PCT asset in a way that was unimaginable just a few years ago.

He explained Caladrius has been increasingly challenged by the tens of millions of dollars of additional capital investment need over the next several years for PCT to fully realise its cell therapy commercial manufacturing growth goals, and added Hitachi is in a position to deploy the capital and engineering capabilities needed to achieve these.

Caladrius will continue to use PCT for the manufacturing and development of its own cell therapy candidates including CLBS03, a T-regulatory (Treg) cell-based therapy for the treatment of type 1 diabetes mellitus without the burden of having to try to support and grow that business due to the very, very large capital needs necessary to remain competitive, he told stakeholders.

The deal will include the transfer to Hitachi of cGMP-compliant facilities in Allendale, New Jersey and Mountain View, California offering quality systems, streamlined technology transfer, storage and logistics, and cell and tissue processing services.

In a statement following the announced acquisition, PCT said: Our relationship with our clients does not change based on this announcement, nor will it change based on the finalization of this transaction.

Continue reading here:
Caladrius: 'Hitachi will unlock full potential of cell therapy CDMO PCT' - BioPharma-Reporter.com

Failed cell therapy study offers positives, raises new questions – ModernMedicine

Patients with a history of frequent anti-vascular endothelial growth factor (anti-VEGF) injections for the treatment of age-related macular degeneration (AMD) may not be the best potential candidates for encapsulated cell technology (ECT).

ECT contains human cell lines that are capable of producing a variety of proteins. After a single surgical implantation, they can produce proteins for up to two years or more, according to Szilard Kiss, MD, assistant professor of ophthalmology; director of clinical research at Weill Cornell Medical College, New York.

These cell lines survive even after being inside the human eye for six months, Dr. Kiss said.

A phase II study was initiated to compare the third generation of ECT (Neurotech, Cumberland, RI) to aflibercept (Eylea, Regeneron Pharmaceuticals). The study planned to enroll 90 subjects, with a primary outcome of non-inferiority to aflibercept alone after 108 weeks. Among the patient entry criteria, all patients had to have shown a response to aflibercept before randomization.

The patient profile was similar to other anti-VEGF treatment studies, with a best corrected visual acuity (BCVA) between 80 letters (20/25) and 35 letters (20/200) at baseline; limited pathology (such as fibrosis, scarring, or atrophy); good optical coherence tomography (OCT) response to injections; and having gone through at least three previous injections, with the last injection no more than four months before study enrollment.

The goal was to have fewer than 20% of patients needing a rescue injection during the follow-up period, Dr. Kiss said.

Study particulars

Unfortunately, there was not a significant difference between the implanted patients and those that received aflibercept. Those subjects that went on to 24 weeks may appear to have gained a little bit of vision, and one subject seemed to benefit, Dr. Kiss said. The OCT results show a saw-toothed pattern that we can see in patients who are treated with aflibercept every 8 weeks.

Although the responses look impressive, most subjects underwent rescue injections of aflibercept, Dr. Kiss said.

The time to rescue injections actually occurred as soon as 4 weeks after the implantation. And as such, there was early termination of the study, because it was not going to meet the primary endpoint of fewer than 20% needing injections, Dr. Kiss said.

However, the implantation itself was deemed successful, as the cells survived and produced the anti-VEGF molecule, but the amount that was produced was significantly below the 12 g/mL necessary.

There were two outlier subjects who had received an aflibercept injection alone about 10 days before the explant, and before measuring anti-VEGF activity. Dr. Kiss believes that to be the cause of the higher numbers compared to other subjects in the study.

None of the explants who did not receive aflibercept right before the explant produced enough anti-VEGF activity, he said.

Positive take-home

However, there had been indications the technology would be successful for this indicationa patient who had undergone six aflibercept injections during the previous year before enrollment did not need any injections (rescue therapy) during the 28 weeks before the AMD study terminated.

On the positive side, Dr. Kiss said the study results created new questions for the technologyincluding whether the approach could achieve better outcomes compared to real-world experience with other patient populations since the cell viability and stability was good.

The third generation ECT (NCT-503-3) did achieve the goal of improved VEGF-receptor production by at least 2-fold compared to double ECT (NT-503-2) implants. Further, while VEGF levels were not detectable, a complex formation was observed in the preliminary native gel.

While the AMD study has been halted, the company is investigating the technology in an ongoing phase II study in conjunction with the MacTel Project to investigate the long-term delivery of ciliary neurotrophic factor (CNTF) in people with macular telangiectasia (MacTel). A pilot study of neuro-enhancement in subjects with early visual impairment in collaboration with clinicians at Stanford University is also under way.

Most promising is that ECT is a unique and versatile drug delivery platform, with more than 1,000 patient years of safety data, Dr. Kiss added. The long-term continuous release of therapeutic proteins via ECT remains a viable and effective way to treat chronic ocular conditions.

Szilard Kiss, MD

E:[emailprotected]

This article is adapted from a presentation that Dr. Kiss presented at the Retina Subspecialty Day, prior to the 2016 American Academy of Ophthalmology meeting.

Continue reading here:
Failed cell therapy study offers positives, raises new questions - ModernMedicine

Want more stem cells and a higher pain tolerance? Try shocking yourself. – Men’s Fitness


Men's Fitness
Want more stem cells and a higher pain tolerance? Try shocking yourself.
Men's Fitness
In turn, the hypothalamus directs outgoing signals to stem cell niches resulting in their release, said study co-author Fletcher White, Ph.D., a neuroscientist at the Richard L. Roudebush VA Medical Center in Indianapolis. The researchers found that ...

Continue reading here:
Want more stem cells and a higher pain tolerance? Try shocking yourself. - Men's Fitness

Sunrise stem cell clinic behind blindness cases is largely unregulated – Sun Sentinel

U.S. Stem Cell Clinic is in the spotlight after three patients reportedly lost their eyesight following procedures here.

The Sunrise facility offers stem cell treatments for a range of diseases and chronic disorders and yet it has no medical facility license.

Heres what you might not know: It doesnt need one.

The facility falls under a regulatory loophole. Regulators with Floridas Agency for Health Care Administration, which licenses health care facilities like hospitals and rehabilitation clinics, say they have no authority over stem cell operations. Neither does the Florida Department of Health, which only has regulatory power over personnel like licensed doctors and nurses working in these facilities.

Both state agencies say that authority lies with the U.S. Food and Drug Administration. Yet even here, guidelines for adipose stem cells (harvested from the clients themselves) are unclear.

The FDA could not discuss whether U.S. Stem Cell has faced or could face a potential investigation, spokeswoman Andrea Fischer said. She said the agency is working on guidelines that will clarify how human cells, tissues and products based on them should be regulated. The agency also been posting consumer warnings for years alerting patients to ask if theyre going to be part of an FDA-regulated clinical study.

We really dont know what standards these [clinics] have to conform to, said Dr. Thomas Albini, an associate professor of clinical ophthalmology at the University of Miamis Bascom Palmer Eye Institute. He recently co-authored a report in the New England Journal of Medicine about the U.S. Stem Cell cases.

If someone were licensed, that license would be on the line, he said.

There were no sanctions against the private, for-profit clinic after three women, in their 70s and 80s, lost their sight in 2015 following procedures where they had fat cells liposuctioned out of their belly area and injected into both of their eyes. The women had macular degeneration, a common disorder which eventually leads to blindness. They each paid $5,000 for the procedure.

Two traveled from out of state, and one came from Floridas west coast. At least two went to U.S. Stem Cell because of clinical trials listed on clinicaltrials.gov, a database managed by the National Institutes of Health, said Albini, who along with a Bascom Palmer colleague treated two of the patients shortly after their clinic visits. Their complications included detached retinas, optic nerve damage and eye hemorrhages.

The clinicaltrials.gov posting now says the study was withdrawn prior to enrollment.

On its website, the Sunrise facility says its team of medical researchers and practitioners can draw stem cells from their clients own fat tissue and reinject them into their bodies. There, the cells regenerative power can beat back medical disorders like Parkinsons, congestive heart failure and rheumatoid arthritis, according to the company.

In a written statement, the company, originally called Bioheart, said neither the clinic nor its affiliate, U.S. Stem Cell Inc., is currently treating eye patients.

Since 2001, our clinics have successfully conducted more than 7,000 stem cell procedures with less than 0.01% adverse reactions reported, the statement said. We are unable to comment further on specific cases due to patient confidentiality or legal confidentiality obligations.

The company declined to produce published papers about its research or any trials it had conducted.

Albini questions whether a true trial ever existed. Typically, participants of a clinical trial dont pay for treatment and continue to be monitored through followup appointments. Neither was the case for the three women who went to U.S. Stem Cell Clinic, Albini said.

He also said no legitimate researcher would do an experimental procedure, with no clinical track record, on both of a patients eyes, risking blindness. These people were way out of their league, he said.

Two of the women sued for negligence, failure to warn, and allegations regarding how the product manufactured from their own bodies was defective. Both settled, and their cases were dismissed. Attorneys for U.S. Stem Cell argued that the cases, as filed, involved medical negligence and, as such, needed to be refiled to conform with state law, according to court records.

Attorney Benjamin Bedard, who handled both dismissal filings, could not be reached for comment.

Albini said the FDA had him speak at a workshop in September regarding its concerns about experimental, unlicensed stem cell clinics.

My understanding is its a billion-dollar industry already, he said. We dont have great treatments for people with these conditions. There are people who want tomorrows medicines today and are willing to pay for it.

Staff researcher Barbara Hijek contributed to this report.

dlade@sunsentinel.com or 954-356-4295

Go here to read the rest:
Sunrise stem cell clinic behind blindness cases is largely unregulated - Sun Sentinel

Medicine | Encyclopedia.com

hemoglobin. Wikimedia Commons (Reytan, GFDL)

nervous system. Wikimedia Commons (Public Domain)

heart. Wikimedia Commons (Ewen, CC)

The history of medicine can be traced back as far as Neolithic man, but our understanding of medicine began with the Sumerians. Our knowledge of anatomy began with the Greeks, but truly took hold during the Middle Ages. Modern medicine can be traced back to discoveries made in the 17th century. Thats when William Harvey demonstrated the circulation of blood in the body, quinine was used to treat ... Readmore

The genome project is also helping us understand disease and the human body on a genetic level and led to genetic engineering, which is used to develop pharmaceuticals and new approaches to cancer treatment.

Modern medicine also has given us pharmacology, allowing medical professionals to treat a host of diseases. From acetaminophen, which can treat a headache, to immunosuppressive drugs, which aid in organ transplantation, this growing field offers a multitude of new and effective drugs.

Psychology and psychiatry have also changed much since these sciences first began. People with disorders were often cast away from society or, later, placed in asylums and not treated. Today, psychiatry involves the treatment of many disorders and can involve both physiological and psychological treatments.

See more here:
Medicine | Encyclopedia.com

Plasma and stem cells: The future of regenerative medicine | WEAR – WEAR

Plasma and stem cells: The future of regenerative medicine

Blood platelet injections and stem cell treatments may sound like the future, but physicians at the Andrews Institute are already practicing these forms of regenerative medicine.

Weight lifting mixed with normal wear and tear left Howie Webber in constant pain.

"I probably felt it about four months ago," said Howie. "I did some stretching, thinking I could make it go away, but it just continued to get worse."

That's when Howie went to the doctor and found out he had two options: surgery or regenerative medicine; he picked the latter.

"I just added up the amount of time I'd be out of work and the cost of surgery, plus the copay and this whole thing just seemed like it would be a little faster and a little easier, and it ended up being just that," said Howie.

Physicians at the Andrew's Institute currently offer two different types of regenerative medicine, platelet rich plasma, or PRP and bone marrow aspirate concentrate, or BMAC.

With PRP, physicians take the patient's blood, separate the platelets and inject those platelets back into the patient at the site of injury. The idea is that platelets carry growth factors and molecules to stimulate the healing process.

BMAC utilizes platelets too, but also the patient's bone marrow harvested from the pelvis.

Both regenerative medicine methods have benefits, perhaps the biggest according to Dr. Brett Kindle, is avoiding invasive surgeries.

"If we need surgery, we need surgery, and that's what it is, but if we can avoid it, that often times is very beneficial from a financial standpoint, missing less work, etc.," said Dr. Kindle. "Also from a quality of life, to be able to get back to doing activities in a more timely manner."

The main difference between the two is price and neither are covered by insurance. BMAC costs upwards of $3,000, while PRP costs anywhere from $600 to $800. Howie opted for PRP.

"It hurt for about three days, then within a week I was pain free," said Howie. "Maybe a little discomfort that you would expect, but it wasn't near as bad as it was before."

Howie's issue was with his hamstrings, but Dr. Kindle said both PRP and BMAC can be used to treat a variety of aches and pains.

"Anything in the limbs," said Dr. Kindle. "Shoulders, elbows, hands, wrists, hips, knees, foot, ankle, all of those areas."

Recovery for both PRP and BMAC procedures is typically one to two weeks. Full effects of the injections don't usually kick in until six to eight weeks later. For more information about regenerative medicine or to schedule a consultation with an Andrews Institute physician, call (850) 916-8700.

More:
Plasma and stem cells: The future of regenerative medicine | WEAR - WEAR

Three-pronged approach is key to precision medicine – Medical Xpress

March 23, 2017 by Joseph Bonner

Combining genetic information from a patient's tumor cells with three-dimensional cell cultures grown from these tumors and rapidly screening approved drugs can identify the best treatment approaches in patients for whom multiple therapies have failed, according to a new study led by Weill Cornell Medicine investigators.

Published March 22 in Cancer Discovery, the study brings researchers one step closer to fulfilling the promise of precision medicine, which aims to provide targeted, individualized treatment based on each patient's genetic profile.

"Our goal is to use precision medicine to improve the way clinicians think about cancer therapies as opposed to selecting a therapy that may not be fitted to that patient's cancer," said senior author Dr. Mark Rubin, director of the Englander Institute for Precision Medicine and the Homer T. Hirst III Professor of Oncology in Pathology at Weill Cornell Medicine, and director of the joint precision medicine program at Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medical Center. "Combining genome sequencing with rapid drug screening enables us to nominate new therapies for patients, which we could not have predicted from the genomics alone."

Cancer genetics research has made great strides in the last 20 years, allowing investigators to now identify mutations in tumor cells that are susceptible to treatment. Still, a significant number of cases remain in which genetic mutations cannot indicate to clinicians exactly what drug will be effective in treating a patient's disease, particularly for those with advanced cancers that have failed multiple therapies.

To better treat these patients, Rubin and the research team including co-first authors Dr. Chantal Pauli, a former research fellow in Rubin's lab, and Benjamin Hopkins, a postdoctoral associate in co-author Dr. Lewis C. Cantley's lab developed an approach in which tumor cells taken from patients are grown into three-dimensional cell cultures called tumor-derived organoids. The researchers can then test more than 120 U.S. Food and Drug Administration-approved cancer drugs against the organoids to determine which drugs show the most promise. After identifying a potential treatment, they transplant the organoid into a mouse to assess how well the tumor responds to the drug. The organoid cell culture system, Rubin said, markedly accelerates the identification of potential therapies compared with current approaches, which depend on growing tumor cells in mice.

"With the organoid system, we can obtain answers in one to two months, compared to six months to a year with mice," Rubin said. "This is critically important for patients with advanced cancer. We need to strive to identify new and more effective therapies in a timely manner."

The video will load shortly

"Dramatic improvements in the ability to grow tumors as organoids outside the body is bringing precision medicine to the next level of precision," added Cantley, the Meyer Director of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine. "It is now possible to grow a wide variety of tumors outside of the body; this allows the precision medicine team to not only obtain full exome and RNA sequencing data, but also test a panel of approved drugs on the same tumor that is growing in the patient. Importantly, this can occur within a time scale that would allow one to make a clinical decision about the next therapy, should the tumor progress on standard of care.

"This approach could revolutionize how cancers are treated in the future," he said, "by providing oncologists with detailed information about the genetic aberrations, the gene expression profiles and the response to drugs of the same tumor that is growing in the patient."

For the study, researchers analyzed tumor-derived cells from four patients who came to the Englander Institute for care. Two of the patients had different forms of uterine cancer, while the other two had different forms of colon cancer. The scientists identified effective drugs and drug combinations many of which are medications approved for treating other cancers that they subsequently validated using organoid cultures and organoids transplanted into mice.

Specifically, the researchers discovered that the combination of two breast cancer drugs could treat one of the uterine cancer cases. For the other, one of the top treatments was a combination of a breast cancer drug and a lymphoma drug.

The investigators found that a colon cancer drug combined with one approved for metastatic melanoma could treat one of the colon cancer cases; they believe their results could establish a basis for a clinical trial. For the other colon cancer case, the scientists identified a therapy that combines a lung cancer drug with one approved for treating blood cancers such as lymphoma and multiple myeloma.

"At the moment, there is no standard to guide a clinician's decision of when to give a patient a drug that's not recommended by the FDA," Rubin said. "With this study, we're trying to establish a standard that uses a patient's cells to answer that question."

Explore further: Gene mutation found to drive prostate cancer subtype

More information: Chantal Pauli et al. PersonalizedandCancer Models to Guide Precision Medicine, Cancer Discovery (2017). DOI: 10.1158/2159-8290.CD-16-1154

A newly discovered genetic mutation that is found in a subtype of prostate cancer is integral to the disease's development and growth, according to research from Weill Cornell Medicine scientists. Their findings could pave ...

Much of precision medicine and cancer care focuses on targeting the genomes of specific tumors or metastases. A Weill Cornell Medical College research team has now shown that a more global look at the body using next-generation ...

The development of a new method to grow three-dimensional organoid cultures of pancreatic tumors directly from patients' surgical tissue offers a promising opportunity for testing targeted therapies and drug responses and ...

Mount Sinai researchers have discovered that a rheumatoid arthritis drug can block a metabolic pathway that occurs in tumors with a common cancer-causing gene mutation, offering a new possible therapy for aggressive cancers ...

Chemotherapy is indicated as the first line of treatment for advanced bladder cancer. New research by Weill Cornell Medicine and University of Trento scientists shows that while chemotherapy kills the most common type of ...

Research led by investigators at Memorial Sloan Kettering Cancer Center has shown for the first time that organoids derived from human prostate cancer tumors can be grown in the laboratory, giving researchers an exciting ...

Telomeres are regions of repetitive DNA at the end of human chromosomes, which protect the end of the chromosome from damage. Whilst shorter telomeres are hypothesized biological markers of older age and have been linked ...

Combining genetic information from a patient's tumor cells with three-dimensional cell cultures grown from these tumors and rapidly screening approved drugs can identify the best treatment approaches in patients for whom ...

(Medical Xpress)From the time it first comes online during development the nervous system begins to exact precise control over many biologic functions. In some cases, too much control. When it does, a little nerve-squelching ...

For decades, scientists and doctors have looked for ways to stop the damage that viruses cause to humans.

Discovery of the BRCA genetic mutation in the mid-90s represented a breakthrough in breast and ovarian cancer prevention. About 5-10% of breast cancer cases and 10-18% of ovarian cancer cases can be attributed to two BRCA ...

Faced with the negative quality-of-life effects from surgery and radiation treatments for prostate cancer, low risk patients may instead want to consider active surveillance with their physician, according to a study released ...

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Continue reading here:
Three-pronged approach is key to precision medicine - Medical Xpress

Childhood leukemia cured with genetically engineered T-cell therapy using donor cells – Genetic Literacy Project

Three months after the birth of a child, most parents are worried about getting the baby to sleep through the night or wondering when to start solid food. But Layla Richards parents had bigger concerns. Their daughter suddenly became ill and stopped eating. Thats when doctors discovered that Layla had acute lymphoblastic leukemia (ALL). None of the standard treatments worked. Even the heroic treatments couldnt quell her cancer. But parents Lisa and Ashleigh Richards refused to give up. From the Guardian:

Despite several rounds of intensive chemotherapy, Layla still had leukemia cells in her body when the transplant was performed, and seven weeks later the disease returned. Soon after, doctors told the family there were no other treatments that might cure Layla and suggested palliative care. But Laylas parents, Lisa and Ashleigh, insisted that the doctors keep trying. We didnt want to accept palliative care and give up on our daughter, so we asked the doctors to try anything for our daughter, even if it hadnt been tried before, Lisa said.

So doctors at Great Ormond Street Hospital in London gave Layla an experimental treatment that had only been tested in mice. They transfused Layla with genetically engineered immune cells from a female donor. Within 28 days she was cancer free. A second child was also treated a few months later. She also is cancer free. Kristen Brown at Gizmodo details the procedure:

The treatment entailed collecting blood from donors, isolating healthy immune T-cells, and then using a genetic engineering tool known as TALENs to deactivate certain T-cell genes that would normally cause them to be rejected once transplanted into a leukemia patient. The T-cells were also engineered to attack cancer cells directly.

This kind of engineered T-cell immunotherapy has been around for a few years. There are several, small human clinical trials ongoing. One trial was canceled earlier this year because of the deaths of three patients caused by side effects of the treatment. However, these therapies all rely on taking T-cells from the patient, engineering them to attack cancer, and reintroducing them into the patients system. The process is tedious and takes a relatively long time. But the girls in London got their engineered T-cells from a donor. And gene editing was used not just to put cancer-receptors on the T-cells, but also to erase the immunological signature that causes rejection when cells are transplanted from donor to patient. That significantly cuts the work, and potentially the costs, of this kind of treatment, writes Antonio Regalado at Technology Review:

The ready-made approach could pose a challenge to companies including Juno Therapeutics and Novartis, each of which has spent tens of millions of dollars pioneering treatments that require collecting a patients own blood cells, engineering them, and then re-infusing them.In the off-the-shelf approach, blood is collected from a donor and then turned into hundreds of doses that can then be stored frozen, says Smith. We estimate the cost to manufacture a dose would be about $4,000, she says. Thats compared to a cost of around $50,000 to alter a patients cells and return them.

The study also highlights an interesting paradox. T-cell immunotherapy is consideredone of the most advanced forms of precision medicine. Taking an individuals cells, engineering them to fight their specific cancer type then giving them back to the patient are all ways of matching genes to treatment. The safe use of donor cells used to cure these girls could be a back step for precision medicine. The more universal approach worked better in this case. And that begs the question, have we become overly focused on the individualized nature of precision medicine and missed easier, better and cheaper solutions along the way?

The treatment did have some side effects which the doctors described as limited. The second child who was treated developed graft versus host disease. The team did note that the first young patient did indeed have a bad immune reaction to the treatment two months after it took place, but after a round of steroids and some bone marrow transplantation, shes now doing well, IFL Science reported.

The girls will be closely monitoredfor signs that their cancers are returning or that there is some residual side effect of the T-cell treatment. But as of late January 2017, both children remained cancer-free 18 months and 12 months after the treatment.

Meredith Knight is a frequent contributor to the Genetic Literacy Project and freelance science and health writer based in Austin, Texas. Follow her @meremereknight.

For more background on the Genetic Literacy Project, read GLP on Wikipedia.

Continued here:
Childhood leukemia cured with genetically engineered T-cell therapy using donor cells - Genetic Literacy Project

American Academy of Ophthalmology Statement on Stem Cell … – PR Newswire (press release)

The Academy has called on both the National Institutes of Health and the U.S. Food and Drug Administration to take steps to better ensure public safety associated with treatments outside of FDA-approved clinical trials. Good research is being performed under FDA-approved trials by innovative clinician-scientists and tragedy associated with non-approved use of stem cells threatens the conduct and impact of this high-quality science.

The public and patients canlearn more about stem cells and eye careby visiting the Academy's EyeSmartpublic education website. EyeSmart is the leading online source for ophthalmic information. The content is written and approved by Academy member eye physicians and surgeons.

About the American Academy of OphthalmologyThe American Academy of Ophthalmology is the world's largest association of eye physicians and surgeons. A global community of 32,000 medical doctors, we protect sight and empower lives by setting the standards for ophthalmic education and advocating for our patients and the public. We innovate to advance our profession and to ensure the delivery of the highest-quality eye care. Our EyeSmart program provides the public with the most trusted information about eye health. For more information, visit aao.org.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/american-academy-of-ophthalmology-statement-on-stem-cell-therapy-for-treating-eye-disease-300426643.html

SOURCE American Academy of Ophthalmology

http://www.aao.org

Link:
American Academy of Ophthalmology Statement on Stem Cell ... - PR Newswire (press release)