The Don Margolis Blog

Today, I introduce to you Karol LaBarrie, a normal looking 7 year old from Brooklyn, New York.  However, his life has been anything but normal after being stricken with Sickle Cell Anemia when he was 6 weeks old.  And he started feeling the sickle cell symptoms of swelling and pain when he was 3 months old.  All in all, he was hospitalized about 40 times.

However, his mother had a baby in 2004 and wisely saved the umbilical cord stem cells.  In 2006, Karol received cord blood stem cells from his brother Kequan.  And guess what happened next:

"If you look in his blood, there's no evidence, when you test it, it's all his brother's blood, so he is completely cured at this point," said Dr. Monica Bhatia.

His story is just one example. (And you can search this website for 100's of other examples- Don)

"There are, I believe, close to a hundred diseases that are treatable with stem cell transplantation today. Principally cancers and inherited diseases such as metabolic abnormalities, inherited anemias, acquired and inherited immune abnormalities," noted Dr. Robert Hariri of LifebankUSA. "We believe in the very near future in broader applications like treating neurologic injury, cardiac injury and so on."

Ahh- another happy adult stem cell story

Recently, I wrote about Osiris, the American stem cell (adult) company that had a couple products on the way to the market.  Their product- Prochymal is in Phase III of clinical trials in the United States to treat Crohn's Disease and Graft Vs Host Disease.  Prochymal can also apparently be used to treat Diabetes 1 and COPD, although not as far along as for Crohn's and GVHD.

Well, looks like Osiris struck it rich and they definitely deserve it for all their perseverance and hard work and money they have put in.  Genzyme is paying Osiris $130 million up front for the rights to sell Prochymal outside the United States and Canada in a deal that could be worth up to $1.25 Billion.

C. Randal Mills, who despite missing a first name still has become the CEO of Osiris said " This new arrangement really is about the fact that [Prochymal] we think is really a lifesaving drug," Mills said. "I'll visit little children we treat in hospitals, see a kid on a ventilator, skin peeling, intensive hemorrhaging, and three weeks later the kid's walking around the room and eating."

And the fact that this same drug can be used to treat not only GVHD, but also Crohn's and seemingly COPD and Diabetes is amazing as well.  I would be willing to bet that it is not limited to these 4 diseases either.

You did good C.  Looks like the world's first successful stem cell company is an ADULT stem cell company- go figure.

Today, I have a great interview with Dr. Desmond Schatz at the University of Florida.  Dr. Schatz is conducting a clinical trial that treats Diabetes Type 1 in children with  adult stem cells taken from their own blood.  This is a great story-  I especially like how this trial was initiated by a parent of a child with Diabetes and suggested it to Dr. Schatz:

Dr. Shatz: "It is a great story, actually. I got a phone call from a parent about four years ago and he called me up and said Dr. Schatz, I want to talk to you about a study he had recently seen in Nature and I asked which paper he was talking about. I read the paper and I saw that what they had done was that they had given bone marrow to animals that had been rendered diabetic and there was an improvement in the diabetes. He basically called me up and asked if I would give this drug to his child or would I give that drug, and I said no. He asked if I would I consider this and I said probably not. He said, "Well I want you to think about doing this. I have stored cord blood in my child. I believe it is safe. Is there anything to lose?"

We discussed it as a group and we looked at the rationale. We clearly thought that it was safe (it is the child's own stem cells- of course it is safe- Don) and we decided to write them a one person protocol and this person was infused with cord blood from that particular child and that child seemed to do better. As a result, we said if it is worked in this particular child, let's devise a study to recruit patients from over the country and the world to participate in this research study."

This is just the beginning.  This adult  stem cell research is moving in the right direction (although too slow as always) It is almost unfortunate that a father of a diabetic child had to provide the push to get this going. 

And all is not lost if you didn't store your child's cord blood.  They can now extract stem cells from the blood stream and bone marrow (I'm not sure if there is a minimum age regarding a bone marrow extraction). 

For more information on this trial, the article provides Dr. Schatz's contact info:

Desmond Schatz, MD
University of Florida
Gainesville, FL
Melanie Fridl Ross, Public Relations, (532) 273-5812
http://www.ufl.edu

And you can click here to read the whole interview for more information on the procedure

It is my pleasure to announce that one of the Repair Stem Cell Institute's Science Advisory Members- Dr. Zannos Grekos is now in Dubai where he is scheduled to be a featured speaker at the 2008 Dubai Congress on Anti-Aging and Aesthetic Medicine in Dubai, United Arab Emirates.

This is notable as Dr. Grekos will be showing clinical results of his heart disease and peripheral artery disease patients he has treated with adult stem cells.

 "We have literally saved the lives of patients who prior to the
development of adult stem cell therapy, had no option available to them
through medical science," stated Dr Grekos. "With a minimally invasive
treatment, the risks of which are equal to a cardiac catheterization,
we are more than doubling cardiac patients' ejection fraction,
improving kidney and pulmonary function, saving limbs from amputation
by creating new circulation pathways and most recently reversing the
life-threatening effects of metabolic heart disease."

If you happen to be in Dubai on Saturday, November 8,2008- go see Zannos speak.
You will be enthralled.

If you can't make it-
You can still see Dr. Grekos' Regenocyte website,
where he lists his current stem cell treatments :

• Congestive Heart Failure


• Coronary Artery Disease


• Ischemic Heart Disease


• Cardiomyopathy


• Peripheral Artery Disease


• Kidney Disease


• Severe Pulmonary Disease

Zannos has a private practice in Naples, Florida.
 You can also call his office at 1-866-216-5710.

Here is the whole stem cell press release

Since this is election day, I decided we all needed a good laugh. I received this in an email. I can't read the cartoonist's name. If somebody knows the person's name, please let me know. He/She deserves a Gold Medal for this one!

UPDATE: Here is a story on this cartoonist, Gary Varvel

Pastor Dave Castner is a man who doesn't take no for an answer.  Paralyzed by a spinal cord injury suffered in a horrific car accident, he still has hope.  And just like thousands of others, Pastor Dave found hope in Adult Stem Cell therapy.  While it is too soon to see if the adult stem cells will help Pastor Dave, this story is still one of hope- never giving up:

While clearly mentally strong, physically Pastor Dave, as he's known, has begun his own journey of hope.

"My whole thing was, if we don't try this, we'll always wonder, what if?" said Timory.Together, Dave and Timorie would travel halfway around the world to a clinic in Germany for a spinal cord repair treatment not available in the United States.

Dave made a video diary of the treatment, which takes stem cells from bone marrow in his own pelvis, then injects them into his spinal cord in hopes of repairing damage.

As a pastor, Castner said he had many discussions about this type of stem cell therapy he calls promising."There's no moral implications because it's my own tissue, Castner said.

Castner's surgery and travel in early October more than $11,000. While considered successful, Castner said it could be one to three months before he may seen any positive results, such as regaining some movement and strength.

"You have to trust, you have to have faith," he said.

Even with new technology around the corner, Dave said it's worth any risks knowing the pay off could mean doing more with his son sooner -- maybe, even walking again."If it doesn't happen, so be it,"

Pastor Dave said. "Anything I get back helps, it's about quality of life."

The article doesn't say, but from the description, it looks like Pastor Dave received stem cell treatment from the Xcell-Center in Germany.  The Xcell Center happens to be on our Repair Stem Cell Institute's list of top stem cell treatment centers.  Go check it out.

Pastor Dave is a source of inspiration.  Here's to hoping the adult stem cells give Pastor Dave a needed boost.  Keep on rolling Pastor Dave.

Today, I give you a fantastic story through the eyes of a bone marrow donor who saved the life of a young boy. Her story began with the  simple task of having someone taking a cotton swab to the inside of her cheek and collecting some cells.

This is great reading and brings all of you reading this- potential bone marrow donors into what is asked for and required by a bone marrow donor (essentially a stem cell donor)- not a lot and you get the gift of saving a life.

I urge you to read the full story- but here is a teaser:

April 19, 2005, 8 p.m., I heard the voice message that returned me promptly back to earth: “Hello, this is the Gift of Life bone marrow registry. You are a potential match. Please call us back.

A MATCH? WHAT?! OH MY GOSH! I immediately called my mom, who is also on the registry. I signed up in December 2001 because a friend's colleague was in need of a donation, and the best chance for a match was from someone of Eastern European descent. I thought it was the right thing to do, and it was so easy to register - a mere cotton swab of the inside of the cheek to collect some cells. My mom also encouraged me, “What is the worst thing that can happen? You will be a match and potentially save a life?

I love that last line.  It makes perfect sense, so after reading this story here, make sure you go to www.marrow.org here.

Not surprisingly this comes from Osiris, an American adult stem cell therapy that has been around for a long time- just waiting to pounce on the American market, but being hindered by the heavy FDA regulations and process.

However, now it looks like they have found their way with this:

The experimental drug is called Prochymal, which is a massive dose of mesenchymal cells given in a few doses through an IV.

“Prochymal is a formulation of a particular kind of cell that's found in the bone marrow of children and, to a lesser extent, adults, and the cell is a mesenchymal cell, said C. Randal Mills, president and CEO of Osiris. “The mesenchymal stem cell is the cell in our body that enables us to respond appropriately to connective tissue injury. So what does that mean? If you burn yourself, if you cut yourself, if you break a bone, if you get an ulcer, the way you heal from those is mediated by the mesenchymal stem cells.

When the mesenchymal cells realize there is a connective tissue injury, Mills said they come out of the bone marrow, travel to the injury and graft. If it is a broken bone, they will heal the bone, he said; if it is a burn, they will turn off the inflammation associated with the burn and heal the damage and any muscle damage.

“They're a rather universal cell for how our body responds to injuries naturally, Mills said. “So what we've

been able to do is isolate that one cell out of healthy, young adult donors between the ages of 18 and 30 years old, so there's no ethical or political controversy around it, and we can actually grow the cell so it will expand and multiply, so we have from a single donation, we can actually make 10,000 doses of this product.

The drug is being developed for several diseases, but two are in Stage III studies the final stage of safety and efficacy studies until it goes to the U.S. Food and Drug Administration for approval for use in the public. The graft-versus-host study is the furthest along, set for approval in the first half of 2009, Mills said, and the Crohn's disease study is a year or two behind.

Osiris sets out to solve critical unmet medical needs, Mills said, with no acceptable current therapeutic alternatives.

“Right now, Prochymal is set to if all goes well become the world's first stem cell product approved anywhere in the world for anything, Mills said. “And so certainly that is one of the goals we set out to do, is to bring to the market the world's first stem cell drug. But it's what we wanted to do that for that is important: To go in and solve an unmet medical disease like GVHD which literally kills little children, and they suffer a horrendous fate. It's going in and relieving that suffering and saving children's lives. It's the kind of thing we should be using stem cell technology for, and we feel very strongly about that.

Fantastic- "approved anywhere in the world for anything"!!!!!!  Imagine that.  They are also testing Prochymal in diabetes and COPD.  If you are interested in trying to get into one of their trials, you can- The company is still accepting volunteers for both the graft-versus-host study and the Crohn's study. For information or the possible participation, e-mail ibd@osiris.com.

Also, for all of you who say that the United States is falling behind in stem cell research (you know who you are-embryonic profiteers)- that isn't the case.  Actually the technology and research is here already: Adult Stem Cells.  The United States is just so slow to implement their use.  However, companies like Osiris are the future of medicine (and the future is now!)- and they didn't need a stem cell research bill passed to develop their product.  Hint hint Michigan!

Click here to read the full article

This one is so logical and makes so much sense- it is a no brainer. A new trial in England will inject the heart attack patient's own stem cells into his/her heart within 4 hours of having their heart attack. The goal is for the stem cells to go into the heart and help repair and limit the damage done by the heart attack-

Current treatments to unblock arteries immediately after a heart attack help reduce deaths from acute attacks, but the approach doesn't address the damage that has happened to heart tissue, and this can lead to heart failure later on, explained Prof Martin.

"Even though you open up the artery, there has been damage to the heart already," he said. "Our objective is to stop that damage happening and repair the little damage that has occurred by putting the stem cells down the artery as soon after the attack as possible, so they are going to the heart and they tell it how to repair itself."

The trial, which started in April, looks at the impact of taking stem cells from a patient's own bone marrow sample to deliver a "rescue force" to the heart. "When you come into my hospital in an ambulance and the diagnosis is made of heart attack, you will be asked if you want to enter our trial," said Prof Martin. "We believe if we can get the cells in early we can stop the damage. So we try to get them in within four hours."

Half of the 100 patients in the double-blind trial, funded by the UK Stem Cell Foundation and private donations, have their own stem cells delivered to their heart and the other half get water. Then they get follow-up tests on heart function and quality of life, he explained.

Stem cells can reduce heart-attack damage by one-third in animal models, although scientists are not sure how it works, he said. "I have been very much criticised by other scientists saying I shouldn't do this until I know what is happening. I say it's so complicated we might never know how it works."

I have no doubt you are being criticized. Criticized by scientists who would rather watch heart attack victims suffer from congestive heart failure than to possibly be helped by adult stem cells. Since this trial uses stem cells from the heart attack victim, they are his/her own cells so there are no side effects- there is no downside to this. And the best possible scenario is the stem cell treatment will help (which will surely be the case).

This trial will take two years to complete and then undoubtedly more trials will be called for- think of how many heart attack victims could be helped in this time span. However, taking a glass is half full approach, at least this is a step in the right direction.

Click here to read the full article

While Newsweek is blaring to the world that stem cells probably won't be helping Parkinson's Disease, much more quietly, to a much infinitely smaller audience, the Star Bulletin in Honolulu is following up on the first known American to be treated successfully for Parkinson's using adult stem cell therapy.  That is what we are up against in trying to make this stem cell therapy available/known everywhere to everyone.

Well, the first known American to be successfully treated with adult stem cell treatment for Parkinsons is Penny Thomas, approximately 2 years ago, in where else?  Yes, China:

Penny Thomas of Captain Cook said people from all over the world have contacted her since the Star-Bulletin published reports of her stem cell surgery in Beijing two years ago.

"I've had people come to visit me here to see if I'm for real," said the Big Island woman, who has been living the past year with her daughter, Cheyenne, in Pagosa Springs, Colo.

After hearing about the successful treatment with 3 million stem cells from a donor's retina deposited in her brain in May 2006, others followed her to the Tiantan Puhua Neurosurgical Hospital, she said. "I don't know what the numbers are up to now."

She said she is "doing quite well" and is active backpacking, camping, gathering wood for winter and riding her two horses. She is looking forward to snow for cross-country skiing.

Thomas, 55, takes about one-fourth of a tablet of Sinemet, used to increase dopamine levels in the brain, and amino acids five to six times a day "to remain strong and tremor-free." She also takes an herb known as macuna pruriens which makes dopamine naturally, and has found that curcumin (turmeric) is a great help. It acts as an anti-inflammatory for the nervous system, as well as other parts of the body, she said.

"I'm really happy with my life because I have a life now," she said. "I feel I can participate in life, and I'm grateful that I've replaced pretty much all the prescription drugs with herbs and amino acids, which I believe the body can handle much better."

Wouldn't this be a great story to see in the New York Times?  International Herald Tribune?  A press release from the Michael J Fox foundation?  Where is the coverage?  Sadly, the coverage seems to consist only of me and the Star Bulletin. I take off my hat to them for following up on this wonderful story.

See the full story here-