Category Archives: Cell Therapy

Dublin, April 06, 2017 (GLOBE NEWSWIRE) -- Research and Markets has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

The cell-based markets was analyzed for 2016, and projected to 2026.The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 305 of these are profiled in part II of the report along with tabulation of 291 alliances. Of these companies, 170 are involved in stem cells.

Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 64 Tables and 22 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

Key Topics Covered:

Part I: Technologies, Ethics & Regulations

Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit http://www.researchandmarkets.com/research/s5g673/cell_therapy

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Global 2017 Cell Therapy Technologies, Markets and Companies ... - Yahoo Finance

April 6, 2017 -- A stem cell treatment for autism shows promise, according to a new study, but the investigators and other experts emphasize that the therapy is still in the early stages and much more research is needed.

The Duke University study included 25 children, ages 2-6, with autism and assessed whether a transfusion of the youngsters' own umbilical cord blood containing rare stem cells would help treat their autism, CNN reported.

Behavioral improvements were reported in 70 percent of the patients, according to the study in the journal Stem Cells.

A second, larger trial is now underway and the researchers hope they will find a long-term treatment for autism, CNN reported.

Some experts say many unanswered questions remain and the study authors agree much more work needs to be done. This initial trial was a safety study, meaning doctors and the children's families knew the therapy was being administered and there was no comparison between treated and non-treated children.

"Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech," study author Dr. Joanne Kurtzberg, head of the Robertson Clinical and Translational Cell Therapy Program, told CNN.

"Many children were able to attend to play and have meaningful communication in a way that they weren't before. Some children had less repetitive behaviors than they did when they came onto the study," Kurtzberg said.

"The study was very encouraging. We did see positive results. However, it did not have a comparison group, which is very important in establishing whether a treatment is actually effective," study author Dr. Geraldine Dawson, director of the Duke Center for Autism and Brain Development, told CNN.

WebMD News from HealthDay

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Stem Cell Therapy for Autism Shows Promise - WebMD

In this photo, mouse ovarian cancer cells light up with different proteins targeted by the engineered T cells. Image by Kristin Anderson and Ingunn Stromnes / Greenberg Lab

For some patients, certain forms of immunotherapy are showing promise in treating previously difficult-to-treat cancers. In the case of T-cell therapies, though, most of the early experimental successes have been seen in blood cancers. Solid tumors, like breast, lung, ovarian and pancreatic cancers, pose a tougher nut to crack for this new wave of cancer therapies.

Fred Hutchinson Cancer Research Center immunotherapy researchers Drs. Kristin Anderson and Phil Greenberg and their colleagues are working on ways to tweak their teams early successes with T-cell therapy for leukemia to apply to solid tumors. In a presentation Tuesday at the annual meeting of the American Association of Cancer Research in Washington, D.C., Anderson described her preclinical results working toward T-cell therapy for ovarian tumors and the hurdles any clinical version of this therapy will need to overcome. To date, the therapies Anderson and her colleagues are developing have only been tested in mice and in mouse and human cells in the lab.

Developing T-cell therapy for solid tumors is more challenging than for leukemias and lymphomas, Anderson said, but many patients with these cancers are in desperate need of new treatment options. The top five cancer killers in the U.S. are all solid tumors, according to the American Cancer Society. Although ovarian cancer is less common in the U.S. than other solid cancers, its highly deadly it tends to be diagnosed at late stages, in part because it often doesnt cause obvious symptoms, and it has a high relapse rate, Anderson said.

All of these are huge problems, she said. An estimated 22,000 women in the U.S. are diagnosed per year with the disease, according to the ACS, and approximately 14,000 die of the cancer.

The challenges of T-cell therapy for cancers like ovarian cancer include the simple issue of access patients with leukemia or lymphoma can receive an infusion of engineered T cells directly into their bloodstream, but it can be more difficult to tweak the cells to traffic to a tumor tucked away in the body. Another major roadblock to adopting T-cell therapy to solid tumors is whats known as the tumor microenvironment, the local milieu of non-cancerous cells and molecules in and around the tumor.

Tumor microenvironment issues come hand-in-hand with working on solid tumors, said Anderson, who is one of 10 recipients of this years AACR Women in Cancer Research Scholar Awards, a travel award given to female early-career cancer researchers presenting at the meeting.

She and her colleagues have identified proteins overproduced by ovarian cancer cells, known as WT1 and mesothelin, and have found that T cells engineered to specifically recognize these proteins can kill both human and mouse ovarian cancer cells in the lab. Theyve also found that the T cells significantly extend survival in a mouse model of the cancer, but theres a ways to go before this therapy is ready for clinical trials in humans, Anderson said.

In her presentation, Anderson outlined three types of tumor microenvironment roadblocks to an effective ovarian cancer T-cell therapy and how the research team is working to overcome each. They are:

Immunosuppressive cells and proteins in the microenvironment that can signal the engineered T cells to shut down or ignore tumors. Existing checkpoint inhibitor drugs could circumvent this problem, Anderson said, and the Fred Hutch team is also exploring engineering the therapeutic T cells to block those immunosuppressive signals.

A death signal produced by both ovarian tumor cells and nearby blood vessels on their surfaces. This molecular signal causes T cells heading to the tumor from the bloodstream to commit suicide before they can fight the cancer. Dr. Shannon Oda in the Greenberg lab is working on a new type of fusion protein the engineered T cells will carry that will rewire their internal circuitry to instead boost their anti-tumor activity in response to the death signal.

The tumors low-sugar environment. Fast-growing ovarian cancer cells churn through the glucose in their environment the same energy source engineered T cells need to do their work. Researchers in the Greenberg lab are working to re-engineer the therapeutic T cells to process other sources of energy.

Although her current work focuses on ovarian cancer, a particularly difficult-to-treat solid tumor, Anderson hopes the work will shed light on new therapeutic avenues for other solid tumors as well.

If we can solve some of the issues that really plague us with these hard ones, then we can more readily apply [the solutions] to cancers that have fewer of these hurdles, she said.

The researchers are hoping to launch a clinical trial of the engineered T cells for patients with ovarian cancer in the next few years, Anderson said.

Paying it forward

For Anderson, the work is not just academic. Five years ago, while she was completing her doctorate research, Anderson was diagnosed with triple negative breast cancer when she was just 28. After her diagnosis, she learned she carried a mutation in the breast cancerlinked gene BRCA1, a mutation which also increases her risk for ovarian cancer.

An immunologist by training, Andersons own experience with cancer spurred her to look for research opportunities where she could one day have a direct impact on other cancer patients. She wasnt particularly looking to study breast or ovarian cancer, she said, but she was very interested in the burgeoning field of immunotherapy. It seemed a prime research area where she could use her background to make a difference.

When Anderson met with Greenberg, whos long been a leader in the field of T-cell therapy, to discuss research options for her postdoctoral fellowship, he proposed the ovarian cancer project to her. Anderson jumped at the chance.

Someone did a lot of research to come up with the drug that got rid of my cancer. Part of the reason that I wanted to go into cancer therapy was so I could pay it forward and do that for someone else, she said. It just so happened coincidentally to be [a cancer] that is close to my heart.

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T-cell Therapy for Ovarian Cancer Shows Promise | Technology ... - Technology Networks

Evotec and Sanofi have achieved preclinical proof-of-concept for a new beta cell therapy for diabetes, triggering a 3M milestone payment from Sanofi.

In 2015, Sanofi and the German CRO Evotecpartnered to jointly develop a beta cell replacement therapy for the treatment of diabetes in a deal that could reach more then 300Min potential milestone payments. Today, the first one arrived as Evotec received 3M for itsfirst preclinical success.

The new cell therapy will be based onfunctional beta cells, which are derived from human stem cells. These beta cells could not only serve as abeta cell replacement, but also ahigh-throughput drug screening platformto identify small molecules or biologicsbeneficial for beta cell activity.

Beta cells play a key role in pathogenesis of diabetes, an epidemic disease, which currently affects about 422M people worldwide.They reside within the pancreas and respond to elevated blood levels by secreting the glucose lowering hormone insulin.While in type 1 diabetes, beta cellsare destroyed by the patients own immune system, in type 2 diabetes these cells often become functionally impaired.

Given thepersistent need for new treatment opportunities and the incredible market opportunity, cell therapy for diabetes has become a popular field of research.Just recently, scientists from the Diabetes Research Institute (DRI) at the University of Miami have cured the first patient from diabetes after implantation of a bioengineered mini organ that mimics the native pancreas. The DRI Biohub is currently in Phase I/II studies.

Other companies such as British Islexa are working on reprogramming thepancreatic cells into functional islets for transplantation. Belgian Orgenesis uses the patients own liver cells and aims to convert these into functional insulin producing cells. The company has completed preclinical safety and efficacy studies and is currently planning to move into the clinic.

Eventually cell therapy approaches could obviatethe need to follow a life-long regimen of insulin injections, which often cannot fully prevent the long-term complications of high blood sugar.

However, Evotec and Sanofi are not only facing a crowded market, but the technology is also still in its infancy. In light of the high costs and safety risks of cell therapies, the new approaches will still have to prove that they can hold up to what they are promising.

You can read more about whats going on in the diabetes field in our recent review.

Images viaSyda ProductionsandDesignua / shutterstock.com

Continued here:
Evotec and Sanofi Hit the First Milestone for a Diabetes Cell Therapy - Labiotech.eu (blog)

No one wants surgery. Dr. Jeffrey Christianson is participating in a study that may provide relief for injured or immune-disordered pets without incisions.

Stem cells can morph into any type of cell needed. The cells are harvested from the animals fat, processed, and reinjected into the pet. These one-size-fits-all cells can be used to replace joint tissue, bone, or other tissues that have worn away or become injured.

Stem cell therapy is a branch of Restorative Medicine helping sick animals restore and improve function. Veterinary medicine has been utilizing Autologous (stem cells from the pet) therapy for well over a decade. It helps animals with arthritis, injuries, inflammatory bowel disease, and other immune-mediated disorders rehabilitate or regain some function.

Dr. Christiansons newest project is a research study to analyze the use of Allogeneic cells (cells from donor pets) in therapy. The clinic is taking part in a double-blinded placebo controlled research study. Half of the patients just get a saline control solution injected into the joint as opposed to a stem cell therapy. He will monitor the results over a six-month period. He wont know until the end of the study who got the control. Some of the pets get a stem cell treatment for free. We do blood work and x-rays, and its all covered by the study.

Its unknown if Allogeneic stem cell therapy is as safe and effective as Autologous therapy. Dr. Christianson noted, The goal of the study is that sick pets could receive stem cell therapy without the pain and discomfort of surgery.

Sick and injured animals recuperating at the Island Animal Clinic have a goodwill ambassador to their lift their spirits. Dr. Ballards dog, Jiminy Cricket (Jim) is a daily visitor. Jim was an injured client. Practice Manager Holly Davis explains, He came in with two broken legs, and it was too much for his owner to handle. Jim was in two casts for months and needed round-the-clock care. Jim now goes to work daily with Dr. Ballard. He runs around the hallways when Dr. Ballard is on break, providing comic relief for recuperating pet patients.

Veterinary medical discoveries are providing a better quality of life and extending the lives of our furry friends. Its exciting that a local animal hospital is at the forefront of these emerging trends. Its also comforting to know recovering pets have Jim for inspiration.

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Island Animal Hospital Offers Stem Cell Therapy - Beachside Resident

For about half a decade, its been something of an open secret in baseball that playerspitchers especiallyregularly undergo stem-cell therapy to stave off surgeries and lost playing time. Its a cutting-edge medical procedure, done by everyone from high-school standouts to major-league all-stars. Its rarely discussed by players, or by their coaches, parents, doctors, or employers.

So when the Los Angeles Angels went public in 2016 with the news that first Andrew Heaney and then Garrett Richards were undergoing stem-cell therapy for torn ulnar collateral ligaments (UCLs), it was both anticlimactic and a revelation. For the first time, baseball pitchers and their employers were openly admitting trying this novel procedure that, while fairly well-proven anecdotally, has yet to be validated by any well-designed scientific study.

By now, that so-called Tommy John surgery for a torn or damaged UCL has become a rite of passage for the top-flight professional baseball pitcher is a cliche of sports punditry. Every young arm that can fold and then unfold itself into tortuous patterns that facilitate throwing baseballs at 95 miles per hour or faster is bound for the knife, once those upper body contortions inevitably tear the tissue on the inside of their elbows connecting their upper and lower arms, the UCL.

The first Tommy John surgery (or more properly, UCL reconstruction) was performed in 1974 by the orthopedic surgeon Frank Jobe, then the team physician for the Los Angeles Dodgers, on the eponymous pitcher. It was a great success; Tommy John came back to pitch 14 more years in the pros, racking up 164 wins with four different teams.

TJ surgery is fairly straightforward: the connective tissue that makes up the UCL is either replaced with a tendon taken from elsewhere in the patients own body or from the donated tissue of a cadaver.

Nevertheless through the mid-1970s and into the 80s, TJ was something of a rarity; just a handful of baseball players underwent that particular knife. In the 1990s the numbers started to tick up, and then in the 2000s, they exploded. From 1995 to 2005, there was an average of 28 TJ surgeries per year across all levels of pro baseball; from 2005 to 2015, there was an average of 84 TJ surgeries per year.

Then something strange happened. In 2016, the total number of TJ surgeries performed dropped to 90, from 127 the year before, a 30% decline. Only one other year in Tommy John history, 2008, saw such a precipitous drop from the previous year. By 2009, TJ numbers were back to 2007 levels; obviously it remains to be seen whether 2017 will look more like 2015 or more like last year. But the data suggest that if TJ surgery numbers are in fact starting to trend downward, it might have something to do with the rise of stem-cell therapy.

What makes stem cells unique is that they are whats called undifferentiated; they can become other specialized cells depending on the bodys need at the time. There are two types of human stem cells, embryonic and adult. Embryonic stem cells come from a very early-stage embryo; these are what you likely think of when you hear the term stem cellstheyre at the center of one of most exciting fields of medical science research today. Embryonic stem cells are now used or are being studied for a shockingly wide range of applications, from Alzheimers and autism to vision impairment and infertility. However, thanks to the religious right-driven opposition to the harvesting, study, and use of embryonic stem cells, theyve been mired in controversy in the US.

On the other hand, the use of adult stem cellswhich can be harvested from bone marrow, fat, or blood of any person of any age (the name is a bit misleading)is widely accepted by both the medical community and politicians. They have less range, so to speak, than embryonic stem cells; they are primarily to repair and replace damaged tissue in the area they are found. That makes them just about perfect for repairing a torn UCL.

The first pro baseball player known to have undergone stem-cell therapy for a UCL weakness was Bartolo Colonand he was basically forced into talking about it. Following a long run of success culminating with a Cy Young Award season in 2005, Colon had four frustrating years racked with injury and ended up unsigned after 2009. He took a year off to recuperate and in spring of 2011, he was back, signed with the New York Yankees and feeling good. Serge Kovaleski, an investigative reporter with the New York Times, started digging into how Colon had made his comeback, and uncovered the name of Joseph Purita, an orthopedic surgeon and stem-cell therapy pioneer.

As Purita tells it, there was nothing illegal or nefarious about the work hed done on Colon; there was just never a plan to broadcast it, either. Then, he recalls, the Times called me up and said were going to write a story whether or not. So, Purita offered details. In April 2010, he told the paper, a team of Dominican doctors used stem-cell therapy to help repair Colons ligament damage and torn rotator cuff.

Colons recovery was a resounding success. Hes been an all-star twice, is the current active leader in major league wins, and, at age 44, is signed to a $12.5 million contract to be the Atlanta Braves number two starter for the 2017 season.

I cant give names but there are some professionalsBut instead of thrusting stem-cell therapy into the mainstream, the Colon incident forced it to stay underground. The treatment was not well understood at that point, and the circumstancesthat it was done offshore, that it was unearthed by investigative reporting, and that, in 2012, Colon was suspended for 50 games for testing positive for testosterone useclouded public opinion on it. Many were convinced Colon had gotten performance-enhancing drugs in the Dominican Republic. Purita denies this vociferously, and MLB inquiries back him up.

The upshot is that every doctor I spoke to who studies and performs stem-cell therapy for torn-ligament repair says some version of the same thing: I cant give names but there are some professionals who have come in for treatment, says Joshua Dines, an orthopedic surgeon at New Yorks Hospital for Special Surgery, and an assistant team physician for the New York Mets.

Purita says that since Colon, hes worked with some players that had team approvaland some just come on their own, but none wanted to go public about the procedure.

If use of your arm is mostly limited to spreadsheet jockeying and lifting forkfuls of pasta or salad from plate to maw, TJ is no big dealin that case, youre ready to go back to work in six weeks. But if you throw a ball at top speed past another pro athlete for a living, youre going to be out of commission for 18 months or more as you regain strength in your money arm.

And money is the (post) operative word. In 2016 alone, MLB teams lost nearly $60 million in player value because they had to fulfill dozens of contracts of players recovering from Tommy John. Thats nearly enough to field an entire pro teamdefinitely enough to roster a top-of-league pitching staff. And that $60 million doesnt come close to accounting for the losses suffered by players who had to undergo the knife during the last year of a contract, and found themselves released by their previous teams with no new offers on the table while they recovered.

There was never going to be a way to prevent the need for Tommy John surgeries. Baseball players throw far too hard, with far more breaking pitches, starting at far too young an age, to realistically stop UCLs from tearing (though all sports medicine experts do now warn coaches and parents to keep kids and teens at low pitch counts). The alternative was always going to be something that could cure ligament tearsbut better than TJ surgery, with a faster recovery time.

Everything weve seen in the past decade or so suggests stem-cell therapy is exactly that. At this point, platelet-rich plasma (PRP) injections are common first-line defenses against UCL injuries. The procedure entails harvesting PRP from the player and injecting it into the injured part of the body. PRP is dense with proteins specialized for injury repair.

You can think of these injections as a precursor to stem-cell therapy; both are considered biologic treatments and entail wielding the bodys own weapons against injury. Many of the doctors now doing stem-cell therapy started off with PRP procedures. When baseball players have a torn ligament, they typically try PRP first. If that fails, its Tommy John time.

Everyone in the field says that at this point PRP is last decades technologyExcept, everyone in the field says that at this point PRP is last decades technology, more than ready to be replaced by stem-cell therapy, which does much the same thing but better. Adult stem cells essentially are there for the very purpose of tissue repair. Why not take them from a part of the body thats all good, and send them to a region where reinforcements are desperately needed?

Dines says that in his own practice, hes been able to cut down the need for Tommy John surgery by about a third, thanks to his reliance on stem-cell therapy. He doesnt believe that the procedure will lower the number of players that have to have TJ, but it will limit the number of overall TJ surgeriesbecause at this point, many pitchers have to get the surgery twice in their career. Dines says stem-cell therapy can get 15- or 16-year-old pitchers through their first partial tear. They may still need to get a full TJ surgery by age 24, but avoiding that first one is still a huge victory. (A growing number of middle-age first-time TJ patients could also explain the overall drop in Tommy John surgeries.)

Purita is even more optimistic. While most orthopedic surgeons say that, right now, stem-cell therapy is effective on partial, but not full, ligament tears, Purita is confident his version can handle any UCL. He sent Quartz a photo showing a patientan MLB pitcher who wishes to remain anonymous, Purita sayswho had a full UCL tear in November 2011 and, after receiving stem-cell therapy at Puritas clinic, made a full recovery by February 2013.

You never say something replaces something else entirely, Purita says. Stem-cell therapy is not going to replace every case [of Tommy John], but it could probably replace the majority of cases.

Talk to anyone who knows the field and theyll rattle off the same reasons why stem-cell therapy for UCL tears isnt already the standard of care: One reason is that, relative to the population, the number of UCL tear patients is extremely small, which means theres only a tiny pool from which to draw potential study participants. Two, a trial for a new medical treatment is typically only considered well-designed if the subjects are blindthat is, they dont know if they are getting the real treatment or a placebo. But what kind of team or player is going to risk a million-dollar arm on a properly designed study where theres a 50% chance that the injury gets a placebo?

Thats not to say that this is some sort of back-alley procedure. Its performed by some of the most prestigious orthopedic surgeons and medical research centers in the US, and the US Food and Drug Administration approves its use: US doctors are allowed to harvest a persons stem cells and use those cells to treat that same person, as long as you dont manipulate (e.g. genetically modify) the cells.

Someone making $20 million a year is not going to do something he hasnt checked out wellThe lack of literature on the procedure hasnt exactly inspired the confidence of players and teams to go public with their decision to pursue it; nor does the fact that the procedure for years had, as Dines puts it, a bad rap[it] would get lumped in with things that were illegal. There was this specter of cheating. But Dines, and others, say thats changing.

The needle is moving towards this being a valid way of treating things, says Purita. People are starting to recognize that someone making [or risking] $20 million a year is not going to do something he hasnt checked out well.

Amadeus Mason, a sports medicine and biologics expert at Emory University, compares stem-cell therapy today to Tommy John in the 1980s. It was, Okay, were going to try this and see, says Mason, who trained with orthopedic surgeon James Andrews. (Andrews is the Michael Jordan of ligament repairhes saved the arms and careers of some of the greatest pitchers in major league baseball history.) There wasnt a big fanfare going in when players started with Tommy John surgeries, Mason says, but when players came back to pitch [there] was. Same thing here.

Mason thinks stem-cell therapy hasnt quite reached the inflection point, but it is near. Here, too, he sees a comparison with Tommy John: It took a while for them to perfect the procedure so that more and more doctors could do the surgery and reproduce the results well.

Right now, Mason says, there is a relatively small handful of doctors who can do stem-cell therapy for UCL tears, but that list is growing rapidly. For example, the annual conference of the Orthobiologic Institutea professional organization for regenerative medicine researchers and practitionersstarted in 2009 with 20 or so doctors; last years event had nearly 1,000.

Some players can throw faster after they have the surgeryThe Angels didnt want to talk to me about why they decided to go public with Heaney and Richards stem-cell therapies. Perhaps thats because Heaneys, on May 2, 2016, was unsuccessful. The 25-year-old former first-round draft pick underwent Tommy John surgery in July of that year after failing to regain strength in his left arm. Hell miss the entire 2017 season, setting back a promising young career.

Richards had his stem-cell procedure just 14 days after Heaney. So far, it seems to have worked. He didnt return to pitch in 2016, but in spring training this year, he was throwing nearly 100 miles per hour. Probably the Angels best starting pitcher, Richards will take the mound on April 5, and all eyes will be on his right throwing armand on his face, to see if it is registering any pain.

If Richards stays healthy this yearand next year, and the year after thathe could become something like the 21st-century Tommy John. Every team will have a stem-cell therapy expert on its medical staff, or at least one on speed dial. Careers will be saved, and so will millions of dollars.

But wider use of stem-cell therapy also will force the MLB to confront an interesting potential side effect of the procedure. Some players can throw faster after they have the surgery, says Purita. By definition, its making the performance better. Right now, major league baseball does not include stem-cell therapy in its list of banned performance enhancers (pdf). But what happens when a baseball player, perhaps a fringe pitching prospect in the low minors, feels some elbow pain one day and gets an MRI, and is diagnosed with nothingbut decides to get stem-cell therapy anyway, since it could give him an extra four miles per hour on his fastball?

The MLB will have a decision to make: To accept potential competitive imbalances to save young arms, or to seek to preserve a level playing field (or even just the fiction of one) at the cost of some of the games best players. The question is all but inevitable.

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Stem-cell therapy is poised to disrupt the Tommy John epidemic in baseball - Quartz

A stem cell treatment for heart failure patients is safe and shows early signs of effectiveness, according to a study published Wednesday.

The study was conducted by Japanese researchers in 27 patients, who received transplants of stem cells taken from their own thigh muscles. There were no major complications, and most patients showed considerable improvement in their symptoms.

The study was published in the open-access Journal of the American Heart Association. Dr Yoshiki Sawa of Osaka University Graduate School of Medicine was the senior author. It can be found at j.mp/stemheart.

However, two San Diego cardiologists who do stem cell research on heart disease cautioned that similar clinical trials have shown promise over the years, only to fail at the end for various reasons. There is no approved stem cell therapy for heart failure.

So while the trial itself appears to be well-conducted, the researchers are very far from actually proving their treatment is effective, said Dr. Richard Schatz of Scripps Health and Dr. Eric Adler of UC San Diego School of Medicine.

For one thing, the trial was small, they said, and larger trials are where the most rigorous scientific evaluations are made.

These early trials have looked beneficial in the past, Adler said. When we do the larger trials, the results are more equivocal.

Adler said the signs of efficacy in this trial are modest. For example, the change in ejection fraction, a measurement of efficiency in pumping blood, rose from 27 percent to 30 percent in 15 of the 27 patients. Their heart failure was associated with a lack of blood flow, or ischemia. The remaining non-ischemic patients actually had a slight decline.

The entire field of stem cell and regenerative therapy for heart disease has been a disappointment to date, Schatz said.

Weve been at it for 20 years now, and we dont have a product or a positive (late-stage) trial, so that tells you pretty much everything you need to know, he said. Its not for lack of trying or billions of dollars invested. Its just very, very difficult.

The cardiac field has had more success with other technologies, such as cardiac stents. Schatz is the co-inventor of the first stent.

In the study, the researchers acknowledge that previous attempts had only been modestly effective. They devised a method of producing sheets of muscle stem cells and attaching them to the inner layer of the sac that encloses the heart, a layer that rests directly on the heart surface.

The stem cell sheets stimulate healing by producing chemicals that stimulate cardiac regeneration, the study said. The cells themselves dont survive in the long term, but by the time they die they have served their purpose.

Loss of function

Heart failure is a progressive disease in which the heart gradually loses its ability to pump blood. This can be triggered by a heart attack or any other cause that damages the heart muscle.

When damaged heart muscle is replaced with scar tissue, as often happens, the heart loses pumping capacity. It becomes overstressed, and its output of blood declines. This limits the patients ability to engage in intensive physical activity. In advanced cases, patients may become bedridden.

Existing treatments include drugs and LVAD units, which take over some of the hearts function to relieve stress. Some drugs may help the heart work more efficiently, but none have been shown to improve heart failure by actually regenerating lost heart muscle.

Stem cell therapy is tested in patients who havent responded well to other treatments. Trials have been and are being conducted in San Diego area hospitals.

Scripps Health has been testing a cardiac stem cell therapy from Los Angeles-based Capricor. The cells, taken from donor hearts, are injected into the coronary artery, where they are expected to settle in the heart and encourage regrowth.

UC San Diego is testing a heart failure therapy from Teva Pharmaceutical Industries. It consists of bone marrow derived mesenchymal precursor cells. These can give rise to several different cell types, including muscle cells.

And many other trials are going on throughout the country and internationally.

Adler and Schatz said theres reason for optimism in the long run, as technologies improve.

Just because the other trials have been negative doesnt mean this technique wont be beneficial, Adler said. Its just too early to tell.

That said, Schatz emphasized that the nature of the three-phase clinical trial process means that the show-stoppers for a treatment typically appear late.

Tighter standards needed

Clean trials trials where we all agree that this is the patient population we want to look at, are needed, he said.

For example, heart failure comes in two types, he said. Ischemic heart failure is caused by heart attacks and blocked arteries, which impede blood flow. Non-ischemic heart failure can be caused by damage from diseases, such as a virus.

Non-ischemics can be younger people, in their 20s and 30s, while the ischemic patients are older. Mixing those patient groups in a single trial is a mistake, he said.

Theyre different animals, Schatz said.

Another pitfall is failing to screen carefully enough to enroll only patients likely to benefit, Schatz said.

You can have a patient who has chest pain, and coronary disease just incidentally, he said.

His shoulder or chest pain is from a virus. So he goes into the trial and gets a placebo injection in his arm of cortisone, and his arm pain goes away. And because hes in that placebo group, hes counted as a success the pain went away. It has nothing to do with his heart. Thats an extreme example, but we actually saw that happen.

In a failed gene therapy trial for heart disease, some patients apparently had received the injection in the wrong location, missing the heart muscle, Schatz said.

You assume they got the gene, but they didnt, Schatz said. The study was negative, and thats why I think it was negative.

Such errors dont show up in Phase 1 trials, Adler and Schatz said, because theyre focused on evaluating safety. And these early trials dont have many patients, there arent enough to comfortably determine the therapy is really effective.

By the last stage of the trial, these sources of error have often been identified and trial standards have tightened up. And thats when the faulty assumptions made early appear as the trial ends in failure.

Despite those forbidding hurdles, Adler said research should continue.

This disease is killing a lot of people. Theres not going to be enough hearts to go around for transplant. Theres six million Americans with heart failure, and theres 2,000 heart transplants a year. So coming up with novel regenerative cell-based therapy is something were still excited about.

bradley.fikes@sduniontribune.com

(619) 293-1020

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Study: heart failure stem cell therapy safe, shows early signs of effectiveness - The San Diego Union-Tribune

Summary

For patients with leukemia who relapse after chemotherapy, treatment options have traditionally been limited. At MSK, an experimental immunotherapy called CAR T therapy has expandedoptions for these patients. New research data presented at this years annual meeting of the American Association for Cancer Research (AACR) suggest that patients do better on the therapy when they are treated at the time of minimal residual disease.

Highlights

Like many patients who come to Memorial Sloan Kettering, Glen Blum, 31, had already received treatment at another hospital for a cancer that was proving stubbornly hard to beat.

His saga began several years ago, when lingering back pain led to a blood test, a biopsy, and eventually a diagnosis of acute lymphoblastic leukemia, or ALL. This aggressive cancer, which grows in the bone marrow, had already damaged several of his vertebrae. He received conventional treatment with both chemotherapy and radiation, which helped for a while. But as is often the case with ALL, the cancer came roaring back. And when it did, it was resistant to further treatment with conventional drugs.

Thats when Mr. Blums doctor recommended that he enroll in a clinical trial of an experimental immunotherapy treatment at MSK. The goal of that treatment, called chimeric antigen receptor (CAR) T cell therapy, would be to shrink his cancer down to a point where he would be eligible for a potentially life-saving bone marrow transplant.

The way they explained itto me is that the treatment would get my own immune cells to see the cancer cells as foreign and eliminate them, says Mr. Blum, who lives in East Harlem in New York City. Then the bone marrow transplant was a secondary step so that I wouldnt grow more cancer cells.

The treatment would get my own immune cells to see the cancer cells as foreign and eliminate them.

Historically, a bone marrow transplant is often a leukemia patients last, best hope for a cure once initial therapy has failed. But the procedure is not without significant risks. To receive new bone marrow, patients must first have their existing bone marrow destroyed with high-dose chemotherapy or radiation. Because the bone marrow is what produces blood cells including the white blood cells that make up the immune system patients are vulnerable to infections while the new bone marrow grows. There is also the risk that immune cells from the donor marrow will start to attack the bodys healthy cells, a dangerous complication called graft-versus-host disease.

But what if it were possible for patients to receive CAR T cell therapy earlier, before a relapse? Would outcomes for these patients be better? And might they be able to forgo a bone marrow transplant altogether?

On April 3, at the AACR annual meeting, MSK physician-scientist Jae Park presented research that speaks directly to these questions. Dr. Park and his colleagues took a retrospective look at all adult patients with relapsed or refractory ALL treated with CAR T cells at MSK 51 patients in all. They wanted to understand who benefits the most from this experimental treatment. For example, does the amount of leukemia a person has at the time of CAR T therapy influence how long that person remains free of disease or how severe the side effects are?

To get at these questions, the team divided those 51 patients into two groups: those with minimal residual disease (MRD, defined as less than 5% of cancer cells in the bone marrow at the time of CAR therapy) and those with obvious morphologic disease (MD, defined as 5% or greater cancer cells in the bone marrow). They then performed statistical analyses on the two groups to determine whether they differed in terms of length of survival and severity of side effects.

What they found was that, indeed, there was a significant difference in outcomes between the two groups. Although both groups initially experienced deep regressions leading to a high rate of complete responses, patients in the MRD group lived longerand hadless toxicity compared with those in the MD group. (After an average of 18 months of follow-up, most of the MRD patients werestill alive and free of disease, while the MD patients had a median survival of 17 months; the rate of a life-threatening side effect called cytokine release syndrome was 5% for MRD patients versus 42% for MD patients.)

According to Dr. Park, the results of this study provide strong support for administering CAR T cell therapy soon after initial chemotherapy, when a patient has minimal residual disease, rather than waiting until a patient relapses. The data from this study, he says, indicate that the CAR T cell therapy is likely to be both more effective and less toxic in the earlier setting. A prospective study to test this hypothesis is currently being planned.

Our ultimate goal is to cure the disease with as little therapy as possible and with the minimum of toxicity.

Another suggestive finding though one that needs to be interpreted with caution, given the small sample size was that receiving a BMT after the CAR T therapy did not seem to improve outcomes in either group of patients. This raises the possibility that CAR T therapy might serve as a final or destination therapy, rather than as a bridge to transplant as it is typically used.

Our ultimate goal is to cure the disease with as little therapy as possible and with the minimum of toxicity, Dr. Park says. A bone marrow transplant is currently the only proven curative treatment for patients with relapsed or refractory ALL. But at the same time, its a pretty toxic therapy, and carries a mortality rate anywhere from 15% to 25%. So if we could use CAR T cell therapy to treat the disease while sparing at least some patients the risks of transplant, that would be a big improvement.

Glen Blum and his fiance, Ashley

For Mr. Blum, there was never really a question that he would go for the transplant, though he admits there was a point after the CAR T cell therapy when he considered his options.

Hearing that your cancer is at zero feels like a victory, he said. It feels like, Oh great, Im done.But then when I thought about all I went through to get to that point all the rounds of chemo and radiation, the hospital stays, the experimental treatment to not go the last mile just didnt feel right.

Yet Mr. Blums experience with bone marrow transplantation indicates why doctors are eager get to a point at which they can safely avoid it. About a month after the transplant, he got an infection that led to a severe case of pneumonia.

I was in the ICU, and honestly, it was a really scary time, Mr. Blum says. The doctorstold my mother not to leave the hospital. They were worried I might not make it.

According to Dr. Park, the decision to recommend a BMT or not becomes a question of weighing different factors, including the number of previous treatments, the characteristics of the disease, the risks of the transplant, the risk of relapse, and the age of the patient.

These are the practical conversations were having with patients every day, he says. And while Im not suggesting by any means that weve answered the question definitively, this study raises the possibility that at least for some patients CAR therapy could be an end point.

In May, it will be one year since Mr. Blum had his bone marrow transplant. Though he still has some back pain, he says he is feeling much better. Hes since gotten engaged, and he and his fiance, Ashley, are planning a trip to Jamaica in June assuming doctors give him the all-clear before then.

He says he has no regrets about the treatment he received, despite the difficulties. He always felt very well cared for at MSK.

That hospital is a piece of heaven, Mr. Blum says. Everyone there, including Dr. Park, has a heart three times the size of normal.

Link:
#AACR17: Study Explores Best Time to Give CAR T Cell Therapy - Memorial Sloan Kettering Cancer Center (blog)

April 4, 2017

Although most patients with relapsed B-cell acute lymphoblastic leukemia (B-ALL) experienced complete response after treatment with a type of CAR T-cell immunotherapy, pretreatment disease burden impacted the durability of the responses and long-term survival, according to data from a clinical trial presented here at the AACR Annual Meeting 2017, April 1-5.

"Adult patients with relapsed or refractory ALL have extremely poor outcomes, with the five-year survival rate being less than 10 percent. Therefore, there is a clear need to develop effective therapy for these patients," said Jae Park, MD, assistant attending physician at Memorial Sloan Kettering Cancer Center (MSKCC) in New York.

"To this end, we and other groups have developed and tested CD19-specific CAR T-cell therapy [19-28z CAR T-cell therapy] and have reported encouraging results, with high initial complete response rates in patients with B-ALL. However, relapses are common, even after achieving seemingly deep remission, and severe toxicities have been observed in some patients," Park noted.

Park and colleagues, therefore, retrospectively analyzed data from a prospective clinical trial that tested 19-28z CAR T-cell therapy to identify patients who benefited the most from this therapy. All of the 51 adult patients in this trial had relapsed or refractory B-ALL after one or more conventional multiagent chemotherapy.

The researchers measured disease burden prior to CAR T-cell infusion in all patients and divided them into two cohorts those who had minimal residual disease (MRD) with less than 5 percent blast cells in bone marrow (20 patients), and those who had morphologic disease, with 5 percent or more blast cells in bone marrow (31 patients).

Complete response rates in the MRD cohort and morphologic disease cohort were 95 percent and 77 percent, respectively, which was not statistically different. After a median of 18 months of follow-up, median event-free survival and overall survival could not be computed for those in the MRD cohort (because most patients were still disease-free and alive), but they were 6.3 months and 17 months, respectively, for those in the morphologic disease cohort.

The study also found that long-term survival did not improve for patients in either cohort by having a hematopoietic stem cell transplant (HSCT) after CAR T-cell therapy.

"While more patients and longer follow-up will be needed to adequately address the significance of HSTC, the result of this analysis raises a question as to whether 19-28z CAR therapy can be considered as a definitive, curative therapy rather than a bridge to stem cell transplant, at least in a subset of patients," Park noted.

"Our data suggest that incorporation of 19-28z CAR T cells at the time of MRD following first-line chemotherapy will maximize the durability of CAR T-cell mediated remissions and survival and can potentially spare these high-risk patients from HSCT, rather than waiting until they relapse morphologically and then trying CAR T-cell therapy when it is less likely to achieve a durable long-term outcome," Park added.

Patients from the MRD cohort fared well in terms of side effects as well, compared with those in the morphologic disease cohort. Two of the major side effects associated with CAR T cells, cytokine release syndrome (CRS) and neurotoxicity, occurred in 42 percent and 58 percent of the patients, respectively, in the morphologic disease cohort, compared with 5 percent and 15 percent, respectively, in those from the MRD cohort. No case of cerebral edema was observed in either cohort of this study, Park noted.

A limitation of the study is that this is a retrospective analysis and the findings will need to be validated prospectively, Park said. Further, the analysis on the impact of post-CAR allogeneic HSCT was limited by a relatively small sample size in each cohort as the study was not designed to specifically answer or address that question.

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Durability of CAR T-cell therapy response may depend on ... - Medical Xpress

Bangladeshi activist and city-based medical centres collaborate to provide life-saving treatment for the three patients.

A Navi Mumbai-based treatment centre used stem cell therapy for the first time on three Bangladeshi patients suffering from an incurable muscular dystrophy.

The trio have been suffering from a rare disease called Duchenne Muscular Dystrophy, which causes progressive muscle degeneration, since birth. The disease limits the lifespan of the patient to just 30 years.

The doctors took stem cells from the patients bone marrow in the hip bone, processed it and injected it back into their bodies. The coordinator between the patients and the treating centre, Avantika Patil said, The patients are also undergoing physiotherapy and occupational therapy while we wait for the results of the procedure to show.

The treatment was made possible by the efforts of a Bangladeshi activist, Noor Khan, a Mumbai-based organisation, Meditourz, which worked in collaboration with a brain and spine institute from Navi Mumbai, NeuroGen.

It was NeuroGen which learnt about the three patients from an article in the international media and offered to treat their disease.

Of the three patients, the youngest, Shorab (8) has a mild disorder. This early medical intervention is expected to make his life less painful. However, the other two patients, Abdus (24) and Rahinul (14) are at a progressive stage.

Their father, Tofazzal Hossain, had even sought mercy killing from Bangladesh government for the duo as he could not afford the cost of their treatment. The travel arrangements were made in coordination with the Indian government. Free round-trip tickets were offered to six persons the patients and their caretakers who accompanied them to Mumbai from Kolkata, by Air India.

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Bangladeshi patients treated with stem cell therapy in city - Mumbai Mirror