By combining an experimental stem cell treatment with a nanoparticle delivery system, researchers may eventually stop MS and other autoimmune diseases.

An innovative stem cell therapy could change how we treat multiple sclerosis (MS), but are we any closer to a cure?

The work of Dr. Su Metcalfe, founder and chief scientific officer of the biotech company LIFNano, appears to be breathing new life into that hope.

Metcalfe and her team developed a way to fight MS by using the bodys own natural mechanisms but it hasnt been tested in humans yet.

MS is an inflammatory and neurodegenerative autoimmune disease that can result in an array of neurological symptoms including fatigue, muscle spasms, speech problems, and numbness. It is caused by the immune system attacking myelin, the insulating coating that runs along the outside of nerve cells. The result is damage to the brain and central nervous system.

The disease currently affects roughly 2.5 million people worldwide. About 200 new cases are diagnosed each week in the United States.

LIFNano uses a new treatment based on LIF a stem cell protein that forms naturally in the body to signal and regulate the immune systems response to myelin.

LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy, Metcalfe recently told Cambridge News.

In fact it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when its been damaged, she said.

Metcalfe has spent years studying LIF, but only recently realized its potential for treatment likening it to an on/off switch for the immune system.

However, once she discovered its potential, there were almost immediate problems in its application. One of the earliest was how quickly LIF breaks down once it is administered into the body.

If you try just to inject it into a patient, it dissipates or disappears in about 20 minutes, Olivier Jarry, CEO of LIFNano, told Healthline.

That makes it unusable in a clinic. You would have to have some kind of pump and inject it continually.

A breakthrough came for Metcalfe when she took findings from her studies of LIF and applied them to nanotechnology. The treatment she is now developing relies on nanospheres derived from a well-established medical polymer known as PLGA, which is already used in materials like stitches. And because it is biodegradable, it can be left to dissolve inside the body.

Storing LIF inside these PLGA nanospheres before administering them into the bloodstream allows for a sustained dose over the course of several days.

The process differs significantly from the current drugs used to treat MS. These treatments most often fall under the category of drugs known as immunosuppressors, which inhibit the bodys overall immune system response.

LIF is theoretically much more precise than immunosuppressors, and should keep the immune system functioning against harmful infections and disease.

Were not using any drugs, said Metcalfe. Were simply switching on the bodys own systems of self-tolerance and repair. There arent any side effects because all were doing is tipping the balance. Autoimmunity happens when that balance has gone awry slightly, and we simply reset that.

The team cautions that LIF therapy is still several years away.

While some outlets have run wild with Metcalfes research, announcing that a cure for MS is right around the corner, those headlines are speculative.

Some MS advocacy groups have even made public statements calling coverage of her work premature and irresponsible.

Jarry told Healthline that LIFNano is expecting to enter FDA phase I trials in 2020. This would be the first time that it is used in human subjects. But even if the treatment proves to be safe and effective, the soonest it could be on the market is 2023, he estimated.

The main focus of LIF therapy is now on MS. But it has potential for treating other autoimmune diseases including psoriasis and lupus.

We are optimistic in the sense that we may provide a long-term remission for patients with MS, said Jarry.

Is it a cure? Wed love at some point to use the term cure, but we are very cautious.

Continued here:
Stem Cell Therapy Offers Hope for Multiple Sclerosis Remission - Healthline

Power was forced to retire from inter-county hurling with the injury two years ago at the age of 29

RICHIE POWER hopes radical new stem-cell therapy in Croatia can fix his battered knee.

The eight-time All-Ireland medallist with Kilkenny retired in the wake of the 2015 Liam MacCarthy triumph at the age of just 29.

Power had three operations on his left knee that year alone and admits rushing back too soon ultimately ended his career.

The speedy attacker got back to Croke Park in February with AIB All-Ireland intermediate club champions Carrickshock.

But he has not played for them since and his quality of life has suffered with his knee causing him constant pain.

Ex-Kilkenny hurler David Byrne, who works with an American company linked to a hospital in Zagreb, put Power on the stem-cell trail.

Power said: The process is about regenerating cartilage in the left knee.

They take some good cartilage from the right knee, bring it into the lab and more or less clone it.

Then they inject it back into your left knee and you are hoping then that the blood will run to it and regenerate there.

From what I gather, youre not looking at a huge period of time out.

You have two or three months to help it and they bring you back over every three months for a check-up to see if its working.

The big issue is that I dont think theres anyone who has stood over it and said, Yeah, it definitely works, because they are waiting to see five, ten, 15 years down the line as to how it helps.

The feedback has been positive. Its worth a try.

The former Cats star is not looking to revive his county career though even if Kilkenny could do with him.

He is simply looking for a better quality of life and to extend his club career by a few more years.

Power added: If I dont get anything done in Croatia and if I decide to go back playing for another year or two and keep struggling through, then Im probably looking at a knee replacement by the time Im 40.

Im only 31 so its not something I want to face at such a young age.

It is just to try to give me an extra 15 or 20 years with my own knee.

If they can do that then great, it will be well worth it. If not, then I need to make a decision and maybe hang up the boots altogether.

See the article here:
Former Kilkenny star Richie Power hoping stem-cell therapy can fix his knee - The Irish Sun

"At StemGenex, we are committed to helping people achieve optimum health and better quality of life through the healing benefits of their own stem cells," said Alexander. "Specifically, we use adipose-derived adult stem cell therapy for patients battling conditions such as Multiple Sclerosis, Parkinson's disease, COPD, Rheumatoid Arthritis and Osteoarthritis. We are also committed to the science of stem cell therapy and sponsor five clinical outcome studiesregistered with theNational Institute of Health (NIH) for these diseases."

"What I personally witnessed before the start of StemGenex were patients who had exhausted conventional medical treatments but wanted to try alternative therapies. I was one of them, suffering from severe Rheumatoid Arthritis. Ihad only three options; I could seek a clinical trial, travel to outside of the U.S. to try alternative therapies such as stem cell treatment or petition the FDA for access to drugs under the agency's "expanded access," or "compassionate use" program. Now, new state laws like the one just passed in Texas, built on model legislation from the Goldwater Institute in Arizona, will allow doctors and patients to make their own informed decisions on treatments that have cleared the safety phase of FDA testing."

Last year, in a move that was seen by some as a response to "Right to Try" laws, the 21st Century Cures Act, a landmark piece of legislation focused on medical innovation and medical research, was signed into law by President Obama. This Act provides the FDA with the flexibility to accelerate how it evaluates regenerative medicine treatments, such as stem cell therapies, while maintaining its high standards of safety and efficacy.

"We're on the cusp of a major change on how patients can access stem cell therapy," saidAlexander. "Today, new treatments and advances in research are giving new hope to people affected by a wide range of autoimmune and degenerative illnesses," said Alexander. "StemGenex Medical Group is proud to offer the highest quality of care and to potentially help those with unmet clinical needs improve their quality of life."

ABOUT StemGenex Medical Group

StemGenex Medical Group is committed to helping people achieve optimum health and better quality of life through the healing benefits of their own stem cells. StemGenex provides stem cell therapy options for individuals suffering with inflammatory and degenerative illnesses. Committed to the science and innovation of stem cell treatment,StemGenex sponsors five clinical outcome studiesregistered with theNational Institutes of Health (NIH) for Multiple Sclerosis, Parkinson's Disease, Rheumatoid Arthritis, Chronic Obstructive Pulmonary Disease (COPD) and Osteoarthritis. These have been established to formally document and evaluate the quality of life changes in individuals following adipose-derived stem cell treatment.

Contact: Jamie Schubert, Director of Media & Community Relations jschubert@StemGenex.com, (858) 242-4243

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/stem-cell-therapy-becomes-law-in-texas-300472809.html

SOURCE StemGenex Medical Group

http://www.stemgenex.com

Excerpt from:
Stem Cell Therapy Becomes Law in Texas - PR Newswire (press release)

The year-old Novartis-Penn Center for Advanced Cellular Therapeutics in Philadelphia supplies cancer fighting T cells to multiple hospitals, visible out the window.

CANNONDESIGN

By Jennifer Couzin-FrankelJun. 13, 2017 , 3:15 PM

A transformative cancer therapy based on modified immune cells has lured doctors, companies, and patients alike, but many are hitting a frustrating roadblock: generating enough of these chimeric antigen receptor (CAR)-T cells to meet surging demand. The situation is fluid, with shortages cropping up in some places and easing in others. Doctors, meanwhile, are grappling with how best to distribute the experimental therapy among very sick patients in clinical trials.

How do I allocate the resource in a way thats fairest to everybody and that treats the most patients and potentially saves the most lives? asks Stephan Grupp, a pediatric oncologist at the Childrens Hospital of Philadelphia (CHOP) in Pennsylvania. Grupp has offered CAR-T therapy to more than 150 children with late-stage acute lymphoblastic leukemia (ALL)and worries that because of supply limitations, he cant help more.

CAR-T cell therapy took the cancer world by storm in the summer of 2010. It involves removing a patients immune cells, genetically modifying them to fight their particular cancer, then transfusing them back. The approach is riskysome have even died from itbut for blood cancers in particular, its been remarkable, saving patients at the 11th hour and keeping some in remission for years.

For patients, getting the most anticipated new treatments is never easy. Clinical trials are tightly controlled and not everyone is eligible. But for this personalized approach, the difficulties are multiplied. From the beginning, CAR-T cells were tough to produce. Unlike a drug, each batch is designed for a specific patient. Production involves genetic engineering and working with live cells, and it is still mostly done by hand, by highly trained technicians. Preparing cells for a single patient can take weeks and cost tens of thousands of dollars.

The demand took off when doctors began reporting impressive results in leukemia and, later, lymphoma, with response rates ranging from 40% to 50% in lymphoma to more than 90% in some leukemias. More researchers joined the fray, keen to test CAR-T therapy in patients with other cancers. Companies joined in, too, anticipating a burgeoning market. Dozens of trials are underway, and two CAR-T cell products, for childhood and young adult ALL and aggressive B-cell lymphoma, may be approved later this year by the U.S. Food and Drug Administration (FDA).

The promise of this stuff outpaced the typical approach to development, Grupp says. With about 50 scientists and technicians working furiously to produce cell therapies for trials throughout the University of Pennsylvania (UPenn), CHOP can treat about five children each month. That number rules my life, says Grupp, who receives two or three calls a week about yet another child whose parents hope to secure the therapy. He engages in a delicate dance, trying to keep some children stable while treating the sickest, before theyre too sick to benefit. So far, he believes, he has reached everyone referred from within the United States in time.

At the National Cancer Institute in Bethesda, Maryland, hematologist James Kochenderfers waiting list is driven by the two or three CAR-T cell products a month hes able to secure from the agencys facility. Thats not enough to accommodate all the adults eligible for the seven CAR-T trials hes running in blood cancers. He usually enrolls on a first-come, first-served basis.

Demand for the therapy is also spurring competition for the researchers and technicians who create the cells. Everyone is losing [people] to everyone else, says immunologist Bruce Levine of UPenn, who directs the cell production facility. He guards against the poaching of his staff by companies that can pay a higher salary, in part by stressing a connection with patients at the hospitals he can see out his window. We have patients come over on a regular basis who received cells made in UPenns facility. No company can offer that.

One center that says its keeping up with demand is Baylor College of Medicine in Houston, Texas, in part because of a stroke of luck. We kind of overbuilt, opening a vast facility in 2010, says Adrian Gee, who runs it. Seattle Childrens Hospital in Washington recently broke ground on a building that in a few years will triple or quadruple its cell therapy capacity, now about 10 batches of CAR-T cells a month.

Another way to generate cells for more patients is to shorten the time it takes to make them. Right now the time from vein to vein ranges from about 2 to 4 weeks, depending partly on the technique. Scientists are experimenting with more efficient approaches. Rebecca Gardner, a pediatric oncologist at Seattle Childrens, says the hospital is shifting to one that shaves a week or two off its 3- to 4-week time frame.

Ultimately, the supply problem can be solved with money, says Ronald Levy, a lymphoma specialist at Stanford University in Palo Alto, California. And no one has more money to funnel into CAR-T therapy than the companies. Novartis, for example, spent $43 million on a manufacturing facility in Morris Plains, New Jersey, and last week it released results from a lymphoma trial in which cells were frozen and flown to and from patients in 10 countries.

But some researchers wonder whether the companies will be ready to accommodate the surge in demand expected if FDA approves the first CAR-T therapies. The strain on supplies would increase if doctors want to offer CAR-T therapy to patients off-label, to those who fall outside the approved indication but might still benefit. Unlike a traditional drug, every order needs to be placed through the companiesand its not clear whether they and FDA will support off-label use. If Novartiss product is approved for leukemia patients up to 28 years old, say, and you have a 28.1-year-old, does that mean you cant treat them? asks David Maloney, an oncologist and immunotherapist at the Fred Hutchinson Cancer Research Center in Seattle. I dont know whats going to happen.

For now, scientists are pondering how to best allocate the therapy. At Seattle Childrens, pediatrician and bioethicist Douglas Diekema was drafted by colleagues to offer ethical guidance on what to do if the hospital cant make enough CAR-T cells for everyone in planned trials in brain and other solid tumors, as well as more leukemia trials. A year from now well probably have six to seven trials, up from three today, Gardner says.

Last month, Diekema and his colleagues published a paper online in the Journal of Medical Ethics describing a triage plan for selecting volunteers for CAR-T trials. They argued that, when possible, doctors should focus on the likelihood and magnitude of benefit, treating the sickest patients first. In a second paper still under review, the team will discuss how to allocate CAR-T therapy across clinical trials. There, the ethical calculus is different, including whether the disease affects many people versus just a few.

I did get a 10 p.m. phone call recently, saying we may need to implement the patient allocation strategy, Diekema says. But the hospital, in the end, managed to get the cells to everyone. We dodged it this time, Diekema says. Now, like many others, hes waiting for the next crunch.

More:
For experimental cancer therapy, a struggle to ensure supply keeps up with demand - Science Magazine