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

Cell Therapy Manufacturing Market (2017-2027): Near-Term Demand Driven by Clincial Candidates – Research and … – PR Newswire (press release)

The Cell Therapy Manufacturing Market, 2017-2027 report provides an extensive study of the rapidly growing market of cell therapy manufacturing and focuses both on contract manufacturers and cell therapy developers with in-house manufacturing facilities. These therapies are anticipated to emerge as viable alternatives to conventional treatment options.

The scope of this report primarily includes manufacturing of advanced therapy medicinal products (ATMPs) that involve the use of immune cells such as T-cells, Tregs, dendritic cells, tumor cells and NK cells, and stem cells such as adult stem cells, human embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs).

Several players, including cell therapy developers, research institutes, contract manufacturing organizations, and government and non-profit organizations, are playing a critical role in the development and manufacturing of these cell therapies. In fact, a number of these players have made heavy investments to expand their existing capabilities and establish new facilities for cell therapy products in order to meet the increasing demand.

Additionally, stakeholders have received significant support from governments worldwide, in terms of funding and establishment of consortiums to accelerate the transition of these therapies from laboratories to clinics. It is important to highlight that companies that offer logistics and operational services have developed systems / tools for safer and quicker delivery of therapies from manufacturing sites to patients; this has been identified as one of the key challenges in the overall development process.

The near-term demand for manufacturing of cell-based therapies will primarily be driven by clinical candidates. In the longer term, the currently approved therapies and late-stage therapies (that are likely to get commercialized in future) will act as key drivers of the market. Our outlook is highly promising; we expect the market for cell therapy manufacturing to grow at an annualized growth rate of -42% over the course of next ten years and be worth over USD 4 billion in 2027.

Companies Mentioned

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. CELL THERAPY MANUFACTURING: INTRODUCTION

4. MARKET OVERVIEW

5. ROADMAPS: POTENTIAL STRATEGIES TO OVERCOME EXISTING CHALLENGES

6. CELL THERAPY MANUFACTURING: IN-HOUSE MANUFACTURERS

7. CELL THERAPY MANUFACTURING: INDUSTRY PLAYERS

8. CELL THERAPY MANUFACTURING: NON-INDUSTRY PLAYERS

9. ROLE OF NON-PROFIT ORGANIZATIONS

10. RECENT DEVELOPMENTS

11. MARKET SIZING AND FORECAST

12. SWOT ANALYSIS

13. CONCLUSION

14. SURVEY ANALYSIS

15. INTERVIEW TRANSCRIPT

For more information about this report visit http://www.researchandmarkets.com/research/z9w7mt/cell_therapy

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/cell-therapy-manufacturing-market-2017-2027-near-term-demand-driven-by-clincial-candidates---research-and-markets-300429018.html

SOURCE Research and Markets

http://www.researchandmarkets.com

Visit link:
Cell Therapy Manufacturing Market (2017-2027): Near-Term Demand Driven by Clincial Candidates - Research and ... - PR Newswire (press release)

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

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

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

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)

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