The Don Margolis Blog

LABELLE, Fla.- Two-year old Madeline Conner was born with the inability to hear. But new advances in medical science could offer hope in the form of a stem cell research study.

 

"I really wanted her in it. It was our one shot," said her mother, Stephanie Conner.

 

Conner heard about a new FDA-approved stem cell study for hearing loss.  She knew right away her little girl was the perfect candidate.

 

"It's a group of ten kids and she's the first one and the only one so far," she said.

 

The trial is a collaboration between Children's Memorial Hermann Hospital in Houston and the California-based Cord Blood Registry. 

 

"This is the first study FDA regulated looking at the safety and benefit of cord blood stem cells for treatment of acquired sensorineural hearing loss.  Which is loss that has to do with the damage of the inner ear and nerve fibers that go to the brain," said Principal Investigator, Dr. Fakhri.

 

Stem cells, saved from Madeline's own umbilical cord, were injected into her arm. 

 

"We expect that it will be safe. You are using your own blood stem cells as if it was your own transfusion," stated Dr. Fakhri.

 

"It was actually a one-time treatment, just one infusion.  Then we keep going,  We go four times total, just so they can check her and compare all the testing they did before hand to see if there has been any improvement," said her mom.

 

In theory, the treatment will adjust Madeline's immune system and will help her body repair itself.  In reality, researchers say they have no idea if it will work.

 

"We've definitely seen a lot of improvement. It's hard to say if it's 100 percent because of this or that.  It's just our observation," said Madeline's parents.

 

"We can not expect what the results will be, but potentially it can repair and restore normal hearing," Fakhri said.

 

Expensive stem cell treatments have gained negative attention for promising miraculous results.  The Conner family paid nothing to participate in this study and have been promised no specific improvements. 

Sitting in front of his occupational therapist last week at a Novi rehab facility, 9-year-old Kaden Strek had a choice to make: the green plastic turtle or the blue one.

 

"Where's the green?" asked his therapist, Sara Wasser.
 

After a slight pause, Kaden reached out and plucked the green turtle out of Wasser's hand. A big smile spread across his face.
 

"Good job!" cheered Wasser.
 

A year ago, Kaden, of St. Clair Shores, couldn't see most colors. Born several months premature, he suffered a severe brain bleed as an infant and was later diagnosed with spastic diplegic cerebral palsy and blindness. As a result, he could only see some shapes, colors and patterns.
 

Now, a year after receiving eight cord blood stem cell infusions in China, Kaden can see not only primary colors but pastels. His language has improved dramatically, and he's saying new words every day and speaking in some sentences, said his mother, Eddie (short for Elizabeth) Strek. Even his walking and balance have improved.

And his family believes stem cells have made all the difference.
 

"This has given him a whole new kind of independence and control over himself that he's never had," Eddie Strek said. "It's so exciting to see."
 

So happy with his improvement over the last year, the Streks are raising money for Kaden to go back to China for another round of stem cell therapy, hopefully next summer. They'll need about $38,000 for treatment and travel costs.

Stem cell therapy is available only for certain conditions in the United States, and cerebral palsy is not among them. The therapy has become a big business overseas, available everywhere from Mexico to Thailand.
 

Treatment programs are offered for a host of conditions, but they're often very expensive, and critics question their efficacy.
 

Beike Biotechnology, the firm that conducted Kaden's stem cell therapy, treats more than 200 international patients a month with stem cell therapy.
 

Researchers such as Larry Goldstein, director of the University of California San Diego's stem cell program, are very wary of overseas programs. They question where the stem cells come from and wonder about possible risks.
 

In a case such as Kaden's, Goldstein said it's hard to tell whether stem cells made a difference or if he improved on his own.
 

"That is what clinical trials are for," he said in an email last week. "Every disease fluctuates in symptoms, and kids with developmental diseases tend to continue developing albeit at different rates. How does one know whether the transplant caused improvement versus intensive physical therapy versus fluctuation versus normal developmental improvements that would have happened anyway?"
 

Eddie Strek disagrees. She said Kaden has received fairly extensive therapy his entire life. He now receives occupational therapy and speech therapy four days a week.
 

"There's just no other explanation" for his progress since the cord blood stem cell infusions, she said.

Eddie said she noticed a change in Kaden's vision within 48 hours after his first infusion in China last summer. She was talking with her husband, Tom, on Skype with Kaden next to her when Kaden wanted to get closer to the screen and was looking intently at his dad.
 

"He can see me!" said Tom.
 

Wasser, an occupational therapist with Children's Hospital of Michigan's Novi rehabilitation facility, said Kaden also is using the left side of his body now, something he didn't really do before stem cells. Kaden is the first patient Wasser has ever worked with who's had a stem cell transplant.
 

"I've been an OT for over 10 years, and this is the first patient I've ever had who has regained vision," Wasser said. "… For him, it's really teaching him to use something that he never had before."
 

That means teaching Kaden basic items such as cat, ball and bike. His mom said he knew what a cat said before, but didn't know what one looked like.
 

Working with Wasser during a recent therapy session, Kaden stared at an augmentative communication device the size of a laptop computer. The screen showed a grid with 15 squares. A bell popped up in one square. Kaden had to track it across the screen and tap it with a small pointer. Eventually, the grid got bigger and the bell got smaller, but he still found the bell.
 

Eddie Strek said it's frustrating that stem cell treatment largely isn't available in the United States, because "so many people could be helped."
 

"These are tangible results," Eddie Strek said. "Anyone that knew him before and sees him now, you can see the results. He's a different kid."

PS from Don Margolis: We featured Kaden last year as the family was preparing to go to China.  You can find that article by putting STREK in the search box above.

The Baldwin County doctor that treated former Alabama football players with adult stem cells also has treated at least two people diagnosed with amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.
 

One of the ALS patients, former NFL football player and college coach Frank Orgel, recently underwent a new stem cell reprogramming technique performed by Dr. Jason R. Williams at Precision StemCell in Gulf Shores.

Before the injections, Orgel’s health had declined. He could not move his left arm or leg. He couldn’t walk or stand on his own, he said.
 

Within a few days of having the stem cell treatment, Orgel’s constant muscle twitching diminished, said Bob Hubbard, director of stem cell therapy at the practice. Within weeks, he was able to walk in a pool of water and stand unassisted.
 

“I think it’s helped me,” said Orgel, who was a defensive coordinator at Auburn under former head coach Pat Dye. “I’m walking in the pool and I used to drag my feet. Now my left leg is picking up.”
 

ALS is a progressive neuro-degenerative disease that affects nerve cells in the brain and the spinal cord. The progressive degeneration of the motor neurons in ALS eventually leads to death, according to the ALS Association. 

Stem cells, sometimes called the body’s master cells, are precursor cells that develop into blood, bones and organs, according to the U.S. Food and Drug Administration, which regulates their use. Their promise in medicine, according to many scientists and doctors, is that the cells have the potential to help and regenerate other cells.
 

While Williams’ treatments are considered investigational, he has said, they meet FDA guidelines because the stem cells are collected from a patient’s fat tissue and administered back to that patient during the same procedure.
 

Orgel, 74, said Williams told him it would take between eight months to a year for his nerves to regrow. He is traveling to Gulf Shores from his home in Albany, Ga., this weekend for another stem cell treatment, Orgel said: “I need to get to where I can walk.”
 

In recent years, Orgel has gone to Mexico at least three times for different types of treatments, not sanctioned in the U.S. At least once, he said, he had placenta cells injected into his body. “That didn’t work,” Orgel said. “I didn’t feel any better.”
 

These days, he’s lifting weights and swimming twice a week as part of a physical therapy regimen.

 

Stem cell therapies
 

The technique performed on Orgel is called InVivo reprogramming, Hubbard said, which is described as reprograms adult stem cells into neural stem cells.
 

The procedure involves harvesting adult stem cells from the patient’s own fat, which Williams obtains through liposuction. Then, he uses image-guided therapy to insert the stem cells into the patient’s spine.
 

The patient is prescribed an oral medication that, as laboratory research has shown, causes stem cells to reprogram, converting them into neural stem cells, according to a written statement from the Gulf Shores medical practice.
 

Because of their experimental nature, stem cell injections to remedy conditions such as damaged knee joints or injured muscles are not covered by insurance. A typical stem cell therapy with Williams costs about $15,000. The collection of the cells through liposuction, he has said, makes up about half of the overall price.
 

Williams, a board-certified radiologist, said in a previous Press-Register interview that he spent about four years researching various stem cell therapies, including those collected from bone marrow. He said that the adult stem cells derived from fat tissue seemed to bring fewer chances for complications.
 

Harvesting stem cells from a patient’s own fat removes the need to culture cells, Williams said earlier this year, explaining that culturing stem cells can be a weeks-long process that may expose patients to risks such as infection.

In recent years, professional athletes such as Denver Broncos quarterback Peyton Manning and New York Yankees pitcher Bartolo Colon, among dozens of others, have acknowledged seeking stem cell injections outside the U.S. to try to help heal injuries.
 

FDA urges caution
 

Earlier this year, the FDA issued a consumer warning about claims regarding stem cells. In it, Stephanie Simek, deputy director of the FDA’s Office of Cellular, Tissue and Gene Therapies, said that stem cells from bone marrow or blood are routinely used in transplant procedures to treat cancer and disorders of the blood and immune system.
 

The document cautioned consumers, however, to make sure that stem cell therapy treatments have been approved by the FDA or are being studied under a clinical investigation allowed to proceed by the agency. “There is a potential safety risk when you put cells in an area where they are not performing the same biological function as they were when in their original location in the body,” Simek said.
 

Cells in a different environment may multiply, form tumors, or may migrate elsewhere in the body from the spot where they were placed, according to the FDA warning.
 

While several dozen clinical trials involving various forms of stem cell therapies are under way or have been announced around the world, few have included adult stem cells found in fat tissue.
 

Williams has said that universities and research groups have been slow to move forward because research funding tends to steer toward new drug therapies. He said that he is up front with his patients, telling them that results cannot be predicted.
 

“This new technique of InVivo reprogramming shows great promise for possibly repairing or regenerating nerve cells,“ Williams said in a written statement. “That means it may open up opportunities for treating several neural conditions such as spinal cord injury, stroke, Parkinson’s and Alzheimer’s disease.”
 

Williams said the new technique has been shown to help increase the number of neural stem cells that are transferred back into a patient.
 

“We are hopeful this will indeed help us heal or regrow nerve cells,” Williams said. “However, it is still too soon to really know.”  

How Standard Medicine tried its best to destroy a life and how a VERY smart lady decided to run away from the killers toward something else.
 

Please do not take this as a criticism of a doctor, but rather of a profiteering medical system which most MDs are forced to participate in.

By Eleanor Bradford

BBC Scotland Health Correspondent

 

Under the direction of Fabrice Chrétien, in collaboration with Shahragim Tajbakhsh, researchers from the Institut Pasteur, the Université de Versailles Saint-Quentin-en-Yvelines, the Paris Public Hospital Network (AP-HP), and the CNRS have shown for the first time in humans and mice, the capacity of stem cells to adopt a dormant state when their environment becomes hostile, including several days after death. This ability to significantly reduce metabolic activity enables them to preserve their potential for cellular division, even after extended periods post mortem. After isolation, they can then be used to repair damaged organs or tissues. This discovery could lead to new therapeutic avenues for treating numerous diseases. The study is being published in the journal Nature communications.
 

Remarkably, skeletal muscle stem cells can survive for seventeen days in humans and sixteen days in mice, post mortem well beyond the 1-2 days currently thought. This discovery was made by researchers from the Institut Pasteur, the Université de Versailles Saint-Quentin-en-Yvelines, the Paris Public Hospital Network (AP-HP), and the CNRS under the direction of Professor Fabrice Chrétien*, in collaboration with a team led by Professor Shahragim Tajbakhsh**. The scientists were also able to show that these stem cells, once back in culture, retained their capacity to differentiate into perfectly functioning muscle cells.
 

In light of this astonishing result, scientists then sought to characterize these cells to understand how they survive in such adverse conditions. They observed that these cells enter a deeper state of quiescence, drastically lowering their metabolism. This so-called "dormant" state is a result of cellular organization that is stripped to the bare minimum: fewer mitochondria (cellular power plants using oxygen to produce energy in cells) and diminished stores of energy.

“We can compare this to pathological conditions where cells are severely deficient in resources, before regaining a normal cell cycle for regenerating damaged tissues and organs, explains Fabrice Chrétien. When muscle is in the acute phase of a lesion, the distribution of oxygen is highly disrupted. We have even observed that muscle stem cells in anoxia (totally deprived of oxygen) at 4°C have a better survival rate than those regularly exposed to ambient levels of oxygen.”
 

The team of Fabrice Chrétien then wondered if these results were consistent with other cell types. Tests were then done on stem cells taken from bone marrow where blood cells are produced. These cells remained viable for four days in post mortem mice models, and more importantly, they retained their capacity to reconstitute tissue after a bone marrow transplant.
 

This discovery could form the basis of a new source, and more importantly new methods of conservation, for stem cells used to treat a number of pathologies. This is the case for leukemia, for example, which requires a bone marrow transplant to restore a patient's blood and immune cells destroyed by chemotherapy and radiation. By harvesting stem cells from the bone marrow of consenting donors post mortem, doctors could address to a certain extent the shortage of tissues and cells. Although highly promising, this approach in the realm of cellular therapy still requires more testing and validation before it can be used in clinical applications. Nevertheless, it paves the way to investigate the viability of stem cells from all tissues and organs post mortem.
 

Source: Institut Pasteur
 

From Don: "Scientists around the world are shocked, but they shouldn't be, because Repair Stem Cells are created to divide, grow and cure, no matter what the obstacles. There are many many obstacles which can slow or stop that repair, but we are constantly finding ways around them.  Now we know it will be possible in the future to collect stem cells (which are smart enough to go dormant) from a recently deceased body, test them, and use them to help another patient."

For all Stephanie Conner knew, her daughter would be born deaf, blind and with organ damage — the result of an often symptomless virus Stephanie had passed to her unborn child.

 

So when the girl, Madeline, was finally delivered at a Miami hospital, Stephanie and her husband considered it a major blessing that the girl’s biggest problem was her inability to hear.

 

Now, the LaBelle family hopes medical science will deliver another round of news verging on the miraculous: a stem cell cure (or partial cure) of Madeline’s hearing loss.

 

Madeline, who is nearly 2 years old, is the first person in the nation to take part in a new Food and Drug Administration-approved study of stem cell treatment of sensorineural hearing loss.

 

Children’s Memorial Hermann Hospital in Houston and California-based Cord Blood Registry will eventually enroll another nine children in this first phase of the study.

 

Unlike some advertised stem cell treatments that may cost patients tens of thousands of dollars — and, according to critics, are little more than quackery — the Conners paid nothing to take part and have been promised no specific results.

 

The process is simple: Doctors inject the children with stem cells from their own stored umbilical cords. Because it is the patients’ own blood, there is little chance the treatment will produce side effects.

 

In theory, the treatment will adjust patients’ immune systems to encourage their bodies to repair themselves. In truth, researchers have no idea if it will work.

 

Earlier Italian studies on mice concluded that stem cells may help the body repair damaged cells in the ear and restore some hearing in these types of cases, according to a 2008 published report in the University of South Florida journal Cell Transplantation.

 

The only other available treatments for Madeline’s kind of deafness are hearing aids and cochlear implants.

 

“I’m expecting really good results,” Stephanie Conner said. “I feel like God put this here in front of us for a reason.”

 

Madeline’s parents have a reason to be cautiously optimistic. Two of three preliminary tests after the treatment in January showed improvement in the girl’s hearing, the family said.

FAMILY TIME: Chris Walmsley at home with his parents Wilf and Carol. PHOTO: Mike Proudfoot MP120527

By Jackie Turley 

 

STEM cell therapy rocks!

 

That’s according to Chris Walmsley and his family who have seen amazing changes since Chris received the pioneering treatment in August 2011.

 

Chris, aged 33, was left quadriplegic after being hit by a car on Richmond Hill in 2006.

 

Thanks to the island’s support, some £27,000 was raised in three months so that Chris could undergo the treatment at a clinic in Germany.

 

His mum Carol said: ‘The changes we have seen in Chris are really amazing.

 

‘We knew it wasn’t a miracle cure but his reactions are quicker, his comprehension levels are really good and the right arm is really starting to function well.

 

‘As far as the family are concerned, stem cells rock!’

 

Carol has posted a video on YouTube which she says shows just how much progress Chris has made.

 

He is now able to touch his nose with his right hand.

 

‘I keep watching it over and over I am so thrilled with it,’ she said.

 

Carol said: ‘We haven’t had Chris assessed neurologically since the stem cell therapy but his neurologist is being kept informed of any progress we see and the video is on its way to him.

 

‘But before the therapy Chris could give you thumbs up and would look up and down, left and right but he held his right arm tight against his chest most of the time.

 

‘Now his arm relaxes a lot of the time, not all the time.

 

‘We do still have times when you need to use a crowbar to move it – not literally.’

 

She added: ‘Before the therapy there was no way he could have touched his nose. Yesterday he was touching his lips and his forehead too.’

 

Chris lives during the week at Abbotswood Nursing Home in Ballasalla.

 

But Carol said that after looking after Chris for a week in a caravan the family realised they could look after him at home, in Maine Road, Port Erin.

 

She said: ‘We realised we could look after him at home, not permanently because the house isn’t really wheelchair-friendly but we can get him into our lounge and study.

 

‘We bought him a bed – a fully profiling hospital bed with a pressure mattress.

 

‘And Chris now stays at home every Friday and Saturday night and we all absolutely love it.’

 

Chris now has physiotherapy weekly and carers are employed specifically to work with Chris, so he is starting to enjoy a social life.

 

Carol said his carers take him bowling to Sure Strike in Ramsey which he loves, the cinema and the Wildlife Park and he also went to see the Chinese Circus.

 

Last week saw Small Bear Records release Keep It Covered.

 

It is a digital album featuring 24 tracks of local artists covering songs by other local artists.

 

Proceeds from the album, costing £5, will go towards Chris’s ongoing care.

 

Download the album at www.smallbearrecords.com.

 

Meanwhile, Carol is raising money for Chris at a car boot sale at the Vintage Rally in Port St Mary on Sunday.

 

She said she would welcome any donations of items to sell – or anyone wanting to have a stall.

 

Contact Carol on 498403 or email carol.walmsley(at)yahoo.co.uk.

 

Money raised will help fund these sorts of activities, as well as equipment he needs. Chris’s physiotherapist has recommended that he has a £7,000 standing frame.

 

Carol said: ‘This will help get him up onto his feet, which is really important. It will help with kidney function and is important for the chest.’

 

Watch the video of Chris at www.youtube.com/watch?v=Ck-JFywrC3I&feature=youtube_gdata_player.

CHENNAI: For nearly three years, Sundar (26) was waiting to be told that he could to regain control of his bladder and move around. Sundar, who fell off a tree, injured his spine and was paralyzed below the navel. In 2011, when doctors at Laksha Hospital in Chennai told him there was hope in the stem cell therapy trials, he signed the consent form. 

 

Mumbai-based Dr B S Rajput, consultant orthopaedic and stem cell transplant surgeon at Breach Candy Hospital, told him he would inject stem cells drawn from his own body into him to repair the shredded network of nerves crisscrossing the spinal cord. At a press conference on Monday, Sundar, a farmer, walked with support, and smiled at flashing media cameras. "I tried everything before agreeing for stem cell therapy. I was told there is no hope of walking again and I was bed ridden. I chose stem cell therapy as I had no other option. Now, I have complete bladder and bowel control," he said, shaking hands with Dr Rajput. 

 

In 2008, Sundar suffered a serious spine injury after a fall from a tree. After initial treatment, doctors told him he had suffered post-traumatic paraplegia and that he would have to remain on bed. Sundar then met Laksha Hospital managing director Dr Senthil Kumar who told him about Dr Rajput and his experience in stem cell therapy. After the informed consent form was signed, Sundar was brought for trial on April 26, 2011. 

 

Dr Senthil Kumar and Dr Rajput hope Sundar's condition would further improve over time. They did a similar transplant on a 4-year-old boy with cerebral palsy. "It's a trial, but these tiny cells are helping us build big hopes," said Dr Senthil Kumar.

By the time Dr. Spencer Misner had carved away the dead and diseased flesh from Bobby Rice’s right foot last year, little remained other than bones and tendons.

 

“I couldn’t believe it. It didn’t look real. It looked like something out of a movie,” recalled Rice, a Whitfield County resident.

 

Today, the ankle has almost completely healed. It looks like Rice had simply scraped it. And Rice’s foot has largely healed, too. Misner credits cutting-edge stem cell treatments for saving Rice’s foot and leg.

 

Rice, who has diabetes, stepped on a piece of glass last fall and his foot quickly became infected. After trying a home remedy, Rice eventually went to Dalton’s Hamilton Medical Center emergency room, where doctors found he had a rapidly spreading necrotizing fasciitis, or in layman’s terms, flesh-eating bacteria.

 

Physicians treated the infection with antibiotics. However, Rice had one toe amputated. Doctors had to strip away much of the flesh from Rice’s foot and a great deal of flesh along his ankle.

 

“We did what we had to do,” Misner said. “We got the infection out. We saved his life. But what do you do next? We’d normally say all you can do now is cut of his leg so he can get on with his life.”

 

But Misner had another idea. He contacted Ed Fickey, a sales representative for Osiris Therapeutics and asked about using the company’s new stem cell technologies to rebuild the foot and ankle.

 

Stem cells can grow and differentiate into many different types of cells. Stem cell treatments introduce these cells into damaged or diseased organs to repair them.

 

“The problem is that Bobby is an indigent patient and didn’t have the financial resources. Ed spoke to the company, and they agreed to donate the products for free,” Misner said.

 

Osiris provided two products called Grafix and Ovation. Fickey said they are made from adult stem cells derived from donated placenta and do not come from embryos.

 

“They hadn’t had a case of this magnitude before,” Misner said. “Stem cells are starting to be used pretty extensively. They are used on burn patients to regenerate skin. But this involves muscle, bone, tendon, fat, skin, so many types of tissues.”

 

Misner said he asked the company for some guidelines.

 

“They basically said, ‘You let is know what the guidelines are because we don’t have a record of anything like this being done before.’ I did a literature search, and I couldn’t find anything like this before,” Misner said. “They did connect me with a doctor in Washington, D.C. who has used stem cells quite a bit. But when I sent him the pictures (of Rice’s foot and ankle), he basically said ‘Good luck.” He helped me with some general guidelines, but there was no recipe to follow.”

 

Misner started the treatments in November 2011, and Fickey recalls just how tricky it was.

 

“He (Misner) had a syringe, and he was looking for some tissue to push it into. But there was nothing but bone there,” he said. “Now, there’s a whole fleshy foot.”

 

Misner has applied the stem cell treatments nine times now, and he describes the foot as more than 90 percent healed. He had to perform the first treatments in an operating room at Hamilton Medical Center because of how complicated they were. But he has been able to do the last few treatments in his office. He expects to have to do at least three more before the foot has completely regenerated.

 

Fickey said Osiris has been watching “the Dalton foot” very closely.

 

“Each time we do an application, I send the latest pictures back, and they have always been very impressed. They wanted to see if there has been muscle growth and the answer has been yes. They wanted to see if there has been vascularization, blood flow, and there has been,” Fickey said. “The most impressive thing is that Bobby has feeling back. We were here a couple of weeks ago and the nurse tickled his foot and he kind of jumped. We didn’t expect that. Now, we are starting to have some other cases around and we can tell them ‘This is what we want to see.’”

 

Rice said he never dreamed he would regain his foot.

 

“I know what it looked like. I expected he would just have to take it off. To see where it is today is just amazing,” he said.