Treatment with Umbilical Cord Stem Cells Safe with Sustained Benefits for MS, Trial Shows

Image of GMP syringe prep lab at Stem Cell Institute clinic in Panama.

Stem cells being prepared for treatment.

March 20, 2018
Jose Marques Lopes, PhD
Link to Original Story at Multiple Sclerosis News Today

Treatment with umbilical cord [tissue-derived mesenchymal] stem cells was found to be safe and leads to sustained improvements in disability and brain lesions of multiple sclerosis (MS) patients, according to a clinical trial.

The study, “Clinical feasibility of umbilical cord tissue-derived mesenchymal stem cells in the treatment of multiple sclerosis,” was published in the Journal of Translational Medicine.

Although current treatments for MS are able to reduce the frequency of flare-ups and slow disease progression, they are not able to repair the damage to nerve cells or the myelin sheath, the protective layer around nerve fibers.

Mesenchymal stem cells (MSCs) are adult stem cells found in multiple tissues, such as umbilical cord, bone marrow, and fat. These cells are able to mature into bone, cartilage, muscle, and adipose tissue cells.

MSCs may inhibit immune-mediated alterations. In particular, MSCs derived from the umbilical cord have a high ability to grow and multiply, increase the production of growth factors, and possess superior therapeutic activity, compared with other MSCs.

Diverse clinical studies have shown that MSCs can safely treat certain immune and inflammatory conditions, including MS.

The research team had previously demonstrated that MSCs can also improve cognitive and motor function.

Recent results with placenta or umbilical cord MSCs showed few mild or moderate adverse events, as well improvements in patients’ level of disability.

Researchers at the Stem Cell Institute in Panama have now completed a one-year Phase 1/2 clinical study (NCT02034188) to test the effectiveness and safety of umbilical cord MSCs for the treatment of MS.

The trial included 20 MS patients with a mean age of 41 years, 60 percent of whom were women. Fifteen participants had relapsing-remitting MS, four had primary progressive MS, and one had secondary progressive MS. Patients’ disease duration was a mean of 7.7 years.

Participants received seven intravenous infusions of 20×106 umbilical cord MSCs over seven days. The treatment’s effectiveness was evaluated at the start, at one month, and at one year after treatment.

Assessments included evaluating brain lesions with magnetic resonance imaging (MRI) and disability based on the Kurtzke Expanded Disability Status Scale (EDSS), as well as validated MS tests for neurological function, hand function, mobility, and quality of life.

Patients did not report any serious adverse events. Most mild adverse events possibly related to treatment were headaches, which are common after MSC infusions, and fatigue, which is common in MS patients, the authors observed.

Improvements were most evident at one month after treatment, namely in the level of disability, nondominant hand function, and average walk time, as well as bladder, bowel, and sexual dysfunction. Patients also reported improved quality of life.

MRI scans at one year after treatment revealed inactive lesions in 15 of 18 evaluated patients. One patient showed almost complete elimination of lesions in the brain, which “is a particularly encouraging finding,” the researchers wrote.

At the one year point, improvements in disability levels were also still present, and could translate into improved ability to walk and work without assistance.

“The potential durable benefit of UCMSC [umbilical cord MSC] at 1 month, and sustained in some measures to 1 year, is in stark contrast to current MS drug therapies, which are required to be taken daily or weekly,” the researchers wrote.

The safety of the treatment is another advantage over available MS therapies, the team said.

They concluded that “treatment with UCMSC intravenous infusions for subjects with MS is safe, and potential therapeutic benefits should be further investigated.”

FDA poised to outlaw breast reconstruction for breast cancer survivors using their own fat tissue

FDA-NotApprovedStampThe U.S. Food and Drug Administration held public hearings for two days this week to allow for public commenting on proposed guidance relating to the regulation of human cells, tissues or tissue-based products.

In its current form, this guidance will classify a woman’s own fat tissue as a drug when used in breast reconstruction procedures. This is certainly bad news for the over 100,000 female cancer patients who seek this procedure each year.

According to the FDA, the sole purpose of a woman’s breast is to lactate. Of course that begs the question of what the purpose of a man’s breast might be but we will leave that for another day. Since fat from other parts of the body does not produce milk, it cannot be transferred into a woman’s breast.

Specifically, in its industry guidance entitled Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) from Adipose Tissue: Regulatory Considerations; Draft Guidance, the FDA states, “The basic function of breast tissue is to produce milk (lactation) after childbirth. Because this is not a basic function of adipose [fat] tissue, using HCT/Ps from adipose tissues for breast augmentation would generally be considered a non-homologous use.” “Homologous use” refers to a tissue’s ability to serve the same “basic function or functions” as the tissue into which it is being transplanted. So in this case, “non-homologous” use equals FDA-regulated “drug”.

That’s right ladies. Your fat tissue is an FDA-regulated drug if your doctor wishes to use it to help reconstruct your breasts following a mastectomy unless she goes through the FDA drug approval process, which can take a decade or two and cost upwards of 2 billion dollars. Needles to say, this is a financial burden that no doctor, clinic or hospital can bear. Once finalized, this new guidance will effectively shut the door on using a woman’s own fat tissue to help reconstruct her breasts.

And it doesn’t stop there. According to the FDA, in most cases, our own cells are drugs too. An FDA panel member even went so far as to state that our blood is an FDA-regulated drug.

For example, if your doctor wants to remove a small sample of bone marrow from your hip and inject part of it into your knee because she has read the literature and believes it can help you heal without surgery, the FDA says that’s verboten because you guessed it – it’s a drug that is subject to the full FDA drug approval process.

As the FDA becomes increasing intrusive in our lives, restricting the ability of licensed medical doctors to practice medicine, one has to wonder where it all will end? The answer seems to be that once the FDA has its way, our bodies, down to the very last cell, will be classified as drugs, and therefore subject to federal regulation by the FDA.

If you agree that the FDA has no business outlawing the use of your own fat tissue to reconstruct your breasts, please spread the word and ask your friends, family members and doctors to let the FDA know what you think before September 27th by commenting here: https://www.regulations.gov/comment?D=FDA-2014-D-1856-0061

You might also consider contacting your local congressman, congresswoman, and state senators.

2016-09-15T16:40:41+00:00 September 15th, 2016|Bone Marrow Stem Cells, Breast Cancer, Cancer, FDA, News|

FDA DRAFT STEM CELL GUIDANCE DOCUMENTS EXPOSED AS IMPROPER RULEMAKING, BAD SCIENCE AND HEARTLESS PUBLIC POLICY

Go to Original Post on RickJaffe.com

FDA-NotApprovedStampToday was a good day for people who want continued access stem cells outside of clinic trials, and also for people who want the FDA to allow faster access to this promising technology.

There was a wide spectrum of opinions. Some stem cell companies involved in clinical trials wanted the non-clinical trials clinics shut down. But at least there were representatives from some of these “unproven” clinics and interest groups who made some important points about the rights of patients and how the needs of patients are not being met by the current clinical trials model as it applies to stem cells. I heard a number like 250,000 people are not getting the stem cell treatments they need because of clogged research and regulatory hold-ups. There were numerous calls from very serious, highly credentialed people for the FDA loosen its death grip (my term) restricting access to these therapies, and the thrust of most of these presenters was that these draft guidance documents make thinks much worse.

The guidance documents are really bad and deny access for many

And that was the big takeaway for me; that the guidelines were much, much worse than even I thought. I understood that the guidelines would make illegal the 21 CFR 1271.15 exempt same surgical procedures provided by many of the 600 plus unregulated stem cell clinics.

But what I didn’t understand until Monday’s hearing is that the FDA intends to radically change the rules so that, for example, the most popular form of breast reconstruction surgery post mastectomy (flap something) would become illegal under the new guidelines. Many other popular and widely successful procedures in other areas like orthopedics would be eliminated (outside of clinical trials). We’re not talking unboarded docs with no relevant experience who take a weekend course and starts shooting people up with stem cells. We’re talking about big-time breast reconstructive surgeons, highly regarded orthopedists and other highly skilled and specialized physicians who have successfully worked with tens of thousands of patients. If the FDA gets its way, according to these folks, Poof! These best practices transplant procedures are gone.

Fortunately, there were some very smart professionals making presentations, including an extremely knowledgeable law professor from Boston College, Mary Ann Chirba. She and several other people with regulatory expertise made the case that this whole guidance exercise was an illegitimate attempt to pass new rules without complying with the rulemaking requirements under federal law. Works for me!

They and others honed in on the radical revisions to the two key preexisting terms/concepts used by the FDA to work its illegal magic: homologous use and more than minimal manipulation.

What’s a “main function?”

It was also pointed out that the guidance documents invented a new concept not existing in the statute or rule, namely the “main function” of a cell or HCT/P which is used as a way of forcing stem cell procedures from just registration under 362 into the IND/NDA drug approval path. It was argued persuasively by several regulatory experts that the creation of this new concept and its resulting transfer of many heretofore legal uses of stem cells into illegal new drug products turns the guidance documents into rulemaking without following federal administrative rulemaking procedures.

The FDA doesn’t understand what fat does

Another extremely cogent criticism made by a variety of people including Professor Chirba, other regulators and by both of the two top presenting stem cell researchers, Arnold Caplan and Keith March had to do with the FDA’s view of fat. According to the guidance documents, fat just has a structural function. But these presenters and especially March and Caplan showed that the FDA’s view was biologically unsound. Fat has definite, known and extremely important non-structural uses, starting with energy storage and continuing to assistance in the healing function. The FDA’s unscientific, unsubstantiated restriction on fat allows it to find most of the important uses of fat and fat stem cells illegal as either non-homologous or as a more than minimally manipulated product. The FDA was absolutely and repeatedly pummeled on this point by my count, at least a half dozen very, smart experts. I don’t see how even the FDA, which has a very particular agenda, is going to be able to hold on to its limitations/restrictions on fat/adipose tissue.

The Big Guys say regulations are holding back progress

The two big-time researchers (Caplan and March) also made the point that the regulatory climate is holding back research. Kaplan said that some bone marrow pioneers had observed that if they had the regulatory environment back then as what exists today, bone marrow transplants might never have taken off. Ouch!

Interestingly, Peter Rubin, the plastic surgeon who last Thursday presented the inspiring cases of reconstruction work from fat transfers, presented again. This time he was more critical of the FDA and stated that many of the most successful reconstructive plastic surgery procedures, including breast reconstruction would become illegal under the draft guidance documents. He and many other excoriated the draft homologous document which classifies fat tissue for breast reconstruction as non-homologous because the primary purpose of the breast is lactation. Several of the female presenters had some polite but pointed words to the FDA about that. Most of the day’s presenters agreed that regulation/regulatory expense was delaying bringing this technology to patients.

The 3 Billion Dollar Player Weighs-in

The biggest dollar player was the California Stem Cell Institute which has a 3 billion dollar budget and 12 research centers. Its director spoke, and his message was clear, concise and right on the money (and with 3 billion, it should be). The FDA has to loosen-up its grip and find an intermediate path between unregulated stem cell clinics and full-on clinical trials, because there is a desperate unsatisfied need and that need will be satisfied – just as water flowing down a hill will find a path – with or without the FDA’s help. He was very persuasive. Reminds me of an old TV ad: “When EF Hutton talks, people listen.”

Interestingly, no one picked up on what I though was the most egregious over reach in the draft guidelines, namely that the FDA tacitly incorporated or read the homologous and more than minimally manipulated requirements from registration facilities (1270.10) into the exemption for same surgical procedures (1271.15). Under the actual rule (1271.15) same day surgical procedures can do non-homologous and more than minimally manipulation. At least those two terms are not in that rule. Legal Method 101 says that if terms are in 1271.10 but not in 1271.15, then they’re in in 1271.15. (Maybe too technical. I’ll have more to say about that another time.)

Maybe there is a viable lawsuit

Something else I realized as a result of a couple of the astute presentations. I said in the last post that you can’t sue on a guidance document because it’s just the agency’s “current thinking.” However, if a guidance document is really disguised rulemaking without meeting the rule changing requirements, then maybe there is a lawsuit. Many presenters were clear about the fact that these guidance documents are disguised rule changes, so I’m now more optimistic about the chances of a legal challenge.

People are Mad and are going to do something about it

And speaking of possible legal challenges, while all of the presenters were very professional, very cordial, ostensibly courteous and complimentary to the FDA panel members on the dais, I sensed that quite a few, many in fact, were pretty upset by what the FDA is trying to do with the draft guidance documents.

So here is my prediction/wish/what I hope to make happen. There won’t be one lawsuit filed if the draft guidelines go into effect. There will many lawsuits. I don’t think these folks are going to go quietly. My sense is that the big players, sophisticated players, like Rubin, the fellow who started a society and has 5800 members, the guy with dozens of clinics, they have seen too many good results to give up their most effective tools. All these guys either run or are closely connected to prestigious professional societies and I predict that many of them are going to try to stop these guidance documents, in court or in Congress.

I hope for everyone’s sake the FDA really listened today, because people are mad as hell and there not going to take it. They want better and quicker access to this new technology, and my hope is they will get it.

Rick Jaffe, Esq.

www.rickjaffe.com

US FDA Green Lights Second Duchenne’s Muscular Dystrophy Patient To Receive Human Umbilical Cord Stem Cells In US

(PRWEB) MAY 26, 2016

Cell Dividing in SuspensionAfter several promising treatments in Panama using stem cell technology developed by Medistem Panama Inc. at the City of Knowledge in Panama, a 6 year-old Duchenne’s muscular dystrophy patient received his first umbilical cord tissue-derived mesenchymal stem cells in the US earlier this year following FDA approval of a second application for a single patient, investigational new drug (IND) for compassionate use.

Duchenne muscular dystrophy (DMD) is a rapidly progressive form of muscular dystrophy that occurs primarily in boys. It is caused by an alteration (mutation) in a gene, called the DMD gene, which causes the muscles to stop producing the protein dystrophin. Individuals who have DMD experience progressive loss of muscle function and weakness, which begins in the lower limbs and leads to progressively worsening disability. Death usually occurs by age 25, typically from lung disorders. There is no known cure for DMD.

This trial marks the second time the FDA has granted an investigational allogeneic stem cell IND for Duchenne’s in the United States.

Ryan Benton, the first DMD patient to be treated in the US with umbilical cord stem cells just celebrated his 30th birthday, a landmark age for any Duchenne’s patient. The FDA recently approved a request to increase Ryan’s treatments from two to three times per year. Since his treatments began in September 2014, Ryan’s condition has stabilized and there have not been any adverse side effects reported.

The new subject had traveled to the Stem Cell Institute in Panama several times for treatments similar to Ryan’s. Encouraging results and news of Ryan’s compassionate use trial prompted his parents to seek out a similar trial for him in the US, which was recently granted by the FDA.

Since 2007, The Stem Cell Institute has treated patients with human umbilical cord tissue-derived mesenchymal stem cells for autism, cerebral palsy, heart failure, multiple sclerosis, osteoarthritis, rheumatoid arthritis and spinal cord injury.

In Panama, the institute is currently providing clinical services for Translational Biosciences’ Institutional Review Board-approved phase 1/2 clinical trials for autism, MS, osteoarthritis, rheumatoid arthritis and spinal cord injury. It anticipates approvals for cerebral palsy and heart failure trials in the future. For more information about see: Translational Biosciences on ClinicalTrials.gov.

Renowned stem cell scientist Neil H. Riordan, PhD, developed the stem cell technology being utilized in this trial. Dr. Riordan is the founder and president of the Stem Cell Institute in Panama City, Panama, and Medistem Panama. Medistem Panama is providing cell harvesting and banking services for this trial.

The Aidan Foundation, a non-profit organization founded by Dr. Riordan in 2004 to provide financial assistance for researching unmet medical needs, is providing financial assistance for this trial.

About Stem Cell Institute Panama

Founded in 2007 on the principles of providing unbiased, scientifically sound treatment options; the Stem Cell Institute (SCI) has matured into the world’s leading adult stem cell therapy and research center. In close collaboration with universities and physicians world-wide, our comprehensive stem cell treatment protocols employ well-targeted combinations of autologous bone marrow stem cells and donor human umbilical cord stem cells to treat: autism, cerebral palsy, multiple sclerosis, spinal cord injury, osteoarthritis, rheumatoid arthritis, heart disease, and autoimmune diseases.

In partnership with Translational Biosciences, a subsidiary of Medistem Panama, SCI provides clinical services for ongoing clinical trials that are assessing safety and signs of efficacy for autism, multiple sclerosis, osteoarthritis, rheumatoid arthritis, and spinal cord injury using allogeneic umbilical cord tissue-derived mesenchymal stem cells (hUC-MSC) and hU-MSC-derived mesenchymal trophic factors (MTF). In the future, Translation Biosciences expects to expand its clinical trial portfolio to include heart disease and cerebral palsy.

For more information on stem cell therapy:

Stem Cell Institute Website: http://www.cellmedicine.com

Stem Cell Institute
Via Israel & Calle 66
Plaza Pacific Office #2A
Panama City, Panama

About Medistem Panama Inc.

Since opening its doors in 2007, Medistem Panama Inc. has developed adult stem cell-based products from human umbilical cord tissue and blood, adipose (fat) tissue and bone marrow. Medistem operates an 8000 sq. ft. ISO 9001-certified laboratory in the prestigious City of Knowledge. The laboratory is fully licensed by the Panamanian Ministry of Health and features 3 class 10000 clean rooms, class 100 laminar flow hoods, and class 100 incubators.

Medistem Panama Website: http://www.medistempanama.com

Medistem Panama Inc.
Ciudad del Saber, Edif. 221 / Clayton
Panama, Rep. of Panama
Phone: +507 306-2601
Fax: +507 306-2601

About Translational Biosciences

A subsidiary of Medistem Panama Inc., Translational Biosciences was founded solely to conduct clinical trials using adult stem cells and adult stem cell-derived products.

Translational Biosciences Web Site: http://www.translationalbiosciences.com

New article concludes US FDA restrictions hampering stem cell therapy progress

FDA-NotApprovedStampAn article published this month in Thieme Journal of Knee Surgery entitled, “The Use of Biologic Agents in Athletes with Knee Injuries” concluded that “Biologic agents… are becoming the mainstay of nonoperative therapy in the high-demand athletic population.” but “…Unfortunately, strict regulations by the FDA continue to restrict their application in clinical practice.”

The good news is they also believe, “As the volume and quality of evidence continue to grow, biologic agents are poised to become an integral component of comprehensive patient care throughout all orthopedic specialties.”

The article is authored by Michaela Kopka and James P. Bradley from the Department of Orthopaedic Surgery, University of Pittsburgh, Pittsburgh, Pennsylvania.

Abstract

Biologic agents are gaining popularity in the management of bony and soft tissue conditions about the knee. They are becoming the mainstay of nonoperative therapy in the high-demand athletic population.

The most well-studied agents include platelet-rich plasma (PRP) and stem cells—both of which have shown promise in the treatment of various conditions. Animal and clinical studies have demonstrated improved outcomes following PRP treatment in early osteoarthritis of the knee, as well as in chronic patellar tendinopathy. Early clinical evidence also lends support for PRP in the augmentation of anterior cruciate ligament (ACL) reconstruction. Research investigating the role of biologic agents in collateral ligament and meniscal injuries is ongoing.

Studies assessing the utility of stem cells have shown encouraging results in the setting of osteoarthritis.

Unfortunately, strict regulations by the FDA continue to restrict their application in clinical practice. A major limitation in the interpretation of current data is the significant variability in the harvesting and preparation of both PRP and stem cells.

As the volume and quality of evidence continue to grow, biologic agents are poised to become an integral component of comprehensive patient care throughout all orthopedic specialties.

View original here

First Duchenne’s Muscular Dystrophy Patient To Receive Umbilical Cord Stem Cell Therapy In US Turns 30

The first patient with Duchenne Muscular Dystrophy to be granted FDA approval for allogeneic adult stem cell therapy in the United States turned 30 this year, well surpassing his original life expectancy and paving the way for future patients, according to non-profit organization Coming Together For A Cure.

Duchenne Muscular Dystrophy Patient and Stem Cell Recipient, Ryan Benton

Ryan Benton

WICHITA, KANSAS (PRWEB) MAY 18, 2016 – Ryan Benton was diagnosed with Duchenne Muscular Dystrophy (DMD) at the age of three and given a life expectancy in the late teens to early twenties. DMD is a relatively common progressive genetic disorder, which causes aggressive deterioration of the muscles.

In 2009, at the age of 22, Benton’s condition was critical. He met with the founder of the Stem Cell Institute in Panama City, Panama and Medistem Panama, Neil H. Riordan, PhD. Research had shown that adult stem cell therapy might have the potential to reverse the progression of DMD.

Because of the laws restricting adult stem cell therapy in the United States, Benton was forced to travel to Panama to receive his first life-saving treatment. Ryan made seven trips to Panama to receive treatments from Dr. Riordan’s team of physicians at the Stem Cell Institute.

“Ryan has seen vast improvements in muscle mass and lung capacity as a result of his treatments…”

Ryan was assured at the start that there was no guarantee that we would find success but we knew it was his only hope in fighting the disease, especially since his health was at a critical point. Ryan could tell shortly after the first treatment that something was working. He found a renewed strength that he had never felt before and not once did he see any adverse side effects. He trusted Dr. Riordan and felt safe and eager to undergo additional treatments.

It took five years of hard work and successful treatments, but Benton became the first (and only) DMD patient granted FDA approval for this form of medical therapy inside the United States. An investigational new drug (IND) for compassionate use application was approved, allowing Benton to receive treatment in his hometown, Wichita, KS.

Approval from the FDA came with many stipulations, however. This form of treatment was to be used for only a single patient, twice a year for 3 years.

By all accounts, January 2016 was a major milestone. The FDA has recently granted an additional treatment per year, now allowing Ryan three total treatments per year, as well as approval for a second compassionate use IND for another patient. This second patient, a six-year-old boy, has also shown success from previous treatments in Panama. He received his first treatment in the United States this year .

Ryan and his family have been actively involved in the local muscular dystrophy community, and have personally known dozens of others with DMD who have passed away at far too young of an age. That number continues to grow each day, which only continues to frustrate Ryan and his family as they fight for this treatment to be more readily available for others suffering from the same disease. Ryan believes that if treated early enough, patients could have a strong chance to live a “normal” life. Ryan believes if he had been treated when he was six years old, it could be very likely that he would never have faced any of the diseases debilitating effects.

Ryan has seen vast improvements in muscle mass and lung capacity as a result of his treatments, but we believe additional treatments on a more frequent basis would help ensure maximum potential when it comes to reversing the progression of his disease. Immediately following each round of treatment, we see dramatic increases in his overall health, stamina, physical strength, and ease in ability to breathe. Unfortunately, we have found that on average, three to four months after each treatment, the effectiveness of the cells begins to decrease. We believe the FDA’s permission to increase the number of treatments per year will help safeguard Ryan’s ability to preserve his improvements and more effectively control his Duchenne’s Muscular Dystrophy. Video: Ryan Benton discusses stem cell therapy for DMD
For many families that have lived alongside, or suffered from this disease, this is very exciting news. Ryan and his family are continually heart stricken as they hear of another member of their md community has died far too young due to the disease. It’s their hope they can help provide other families the same opportunities that they were so fortunate to receive.

Coming Together for a Cure, (CTFAC) is a non-profit organization founded by Benton’s siblings, Lauren and Blake after Ryan’s first round of treatments in 2009. In the 7 years since the Bentons were given new hope, they’ve been hard at work raising awareness and support for adult stem cell research and therapy.

To find more information about their organization, their family, or to find out how you can help, please visit http://www.comingtogetherforacure.org

For all other inquiries, please email comingtogetherforacure(at)gmail(dot)com

Duchenne’s Patient Ryan Benton Discusses His Experience with Stem Cell Therapy

Ryan Benton is the first patient in the United States to receive human umbilical cord-derived mesenchymal stem cell therapy for Duchenne’s muscular dystrophy. The US FDA granted Ryan this trial under compassionate use. Ryan first began treatments at the Stem Cell Institute in Panama before being able to receive treatments in his hometown of Wichita, Kansas.

2015-02-17T00:59:43+00:00 February 17th, 2015|Duchenne's muscular dystrophy, News, Stem Cell Research|

After FDA Approval, Duchenne’s Muscular Dystrophy Patient Receives First Umbilical Cord Stem Cell Treatment in the United States

Ryan Benton, a 28 year-old Duchenne’s muscular dystrophy patient from Wichita, Kansas, received his first umbilical cord tissue-derived mesenchymal stem cell treatment yesterday at Asthma and Allergy Specialists of Wichita, KS following US FDA approval of his doctor’s application for a single patient, investigational new drug (IND) for compassionate use.

Wichita, KS (PRWEB) September 10, 2014

Picture of Ryan Benton

Ryan Benton

Ryan Benton, a 28 year-old Duchenne’s muscular dystrophy patient from Wichita, Kansas, received his first umbilical cord tissue-derived mesenchymal stem cell treatment yesterday following US FDA approval of his doctor’s application for a single patient, investigational new drug (IND) for compassionate use.

Duchenne muscular dystrophy (DMD) is a rapidly progressive form of muscular dystrophy that occurs primarily in boys. It is caused by an alteration (mutation) in a gene, called the DMD gene, which causes the muscles to stop producing the protein dystrophin. Individuals who have DMD experience progressive loss of muscle function and weakness, which begins in the lower limbs and leads to progressively worsening disability. Death usually occurs by age 25, typically from lung disorders. There is no known cure for DMD.

This trial, officially entitled “Allogeneic transplantation of human umbilical cord mesenchymal stem cells (UC-MSC) for a single male patient with Duchenne Muscular Dystrophy (DMD)” marks the first time the FDA has approved an investigational allogeneic stem cell treatment for Duchenne’s in the United States.

Ryan received his first intramuscular stem cell injections from allergy and immunology specialist, Van Strickland, M.D at Asthma and Allergy Specialists in Wichita, Kansas. He will receive 3 more treatments this week on consecutive days. Dr. Strickland will administer similar courses to Ryan every 6 months for a total of 3 years.

This is not the first time Ryan has undergone umbilical cord mesenchymal stem cell therapy. Since 2009, Ryan has been traveling to the Stem Cell Institute in Panama for similar treatments. Encouraging results from these treatments prompted Dr. Strickland to seek out a way to treat Ryan in the United States.

The stem cell technology being utilized in this trial was developed by renowned stem cell scientist Neil H. Riordan, PhD. Dr. Riordan is the founder and president of the Stem Cell Institute in Panama City, Panama and Medistem Panama. Medistem Panama is providing cell harvesting and banking services for their US-based cGMP laboratory partner.

Funding for this trial is being provided by the Aidan Foundation, a non-profit organization founded by Dr. Riordan in 2004 to provide financial assistance for alternative therapies to people like Ryan.

About Van Strickland, MD

Dr. Strickland came to Wichita in 1979 from his fellowship at the National Jewish Hospital in Denver. Since then he has spent one year in Wyoming, one year in Dallas, Texas and one year in Lee’s Summit Missouri before returning to full-time practice in Wichita, Kansas.

Dr. Strickland has been a clinical faculty member at The University of Kansas School of Medicine in Wichita in the department of Pediatrics and later in the department of internal medicine for most of his years in Wichita.

Dr. Strickland is certified by the American Board of Allergy and Immunology and the American Board of Pediatrics. He graduated from Baylor College of Medicine in Houston, Texas and served a full residency in Pediatrics at Baylor and a fellowship in Allergy and Immunology in Denver at National Jewish. He has trained in allergy and immunology at the University of Texas School of Medicine in Galveston as an elective while at Baylor and was a student on the team with Mary Ann South, MD and John Montgomery, MD who put baby David in the Bubble (Bubble Boy).

Dr. Strickland is a fellow of The American Academy of Allergy, Asthma and Immunology, The American College of Allergy, Asthma and Immunology, The American Association of Certified Allergists, The American Academy of Pediatrics, and The American College of Physicians. Dr Strickland has been recognized in the “Top Doctors in Wichita” listing several times.

Allergy & Asthma Consultants
MHV Strickland M.D.
10021 W. 21st St. Wichita, KS 67205

Phone: +1 (316) 722-4800
Toll-free: +1 (800) 347-4800
Fax: +1 (316) 722-5117

Web Site: http://www.stricklandallergy.com

About Neil Riordan, PhD

Neil Riordan PhD is the co-founder of the Riordan-McKenna Institute, a regenerative orthopedics clinic that will open its doors in Southlake, Texas in late 2014. RMI will offer non-surgical stem cell treatments and stem cell enhanced surgeries for orthopedic conditions. He is the founder and chairman of Medistem Panama, Inc., (MPI) a leading stem cell laboratory and research facility located in the Technology Park at the prestigious City of Knowledge in Panama City, Panama. Founded in 2007, MPI stands at the forefront of applied research on adult stem cells for several chronic diseases. MPI’s stem cell laboratory is ISO 9001 certified and fully licensed by the Panamanian Ministry of Health. Dr. Riordan is the founder of Stem Cell Institute (SCI) in Panama City, Panama (est. 2007).

Under the umbrella of MPI subsidiary Translational Biosciences, MPI and SCI are currently conducting seven IRB-approved clinical trials in Panama for autism, multiple sclerosis, rheumatoid arthritis and osteoarthritis using human umbilical cord-derived mesenchymal stem cells, mesenchymal trophic factors and stromal vascular fraction. Additional trials for spinal cord injury, and cerebral palsy are scheduled to commence in late 2014 upon IRB approval.

Dr. Riordan’s research team collaborates with a number of universities and institutions, including National Institutes of Health, Indiana University, University of California, San Diego, University of Utah, University of Western Ontario, and University of Nebraska.

Dr. Riordan has published over 60 scientific articles in international peer-reviewed journals and authored two book chapters on the use of non-controversial stem cells from placenta and umbilical cord. He is listed on more the 25 patent families, including 11 issued patents including a 2010 patent for a new cellular cancer vaccine.

In 2007, Dr. Riordan’s research team was the first to discover and document the existence of mesenchymal-like stem cells in menstrual blood. For this discovery, his team was honored with the “Medical Article of the Year Award” from Biomed Central.
Neil Riordan, PhD

Riordan-McKenna Institute
801 E. Southlake Bivd.
Southlake, TX 76092

Phone: +1 (817) 776-8155
Fax: +1 (817) 776-8154

Website: http://www.rmiclinic.com

Neil Riordan PhD – on opening a stem cell clinic in the United States

Stem Cell Pioneers featured Dr. Riordan in its February installment of “Ask the Doctor”, a monthly segment that features stem cell scientists and doctors answering questions from readers about stem cell therapy.

Over the next several days, we will share these questions and Dr. Riordan’s answers with our readers.

Question for Dr. Riordan: If the FDA loosens regulations in the U.S., do you have any plans to open a clinic here?

Dr. Riordan’s Answer: Unfortunately I don’t see FDA loosening regulations any time soon so I have no plans to do anything in the U.S. using umbilical cord MSCs or even autologous SVF in the near future.

It would be great if the U.S. would follow Japan’s lead. The Japanese parliament passed legislation in November of last year that essentially allows a company to market a cell product after the product has been demonstrated to be safe. Quoting from an Athersys press release: “Recently, Japan’s parliament enacted new legislation to promote the safe and accelerated development of treatments using stem cells. The new regenerative medicine law and revised pharmaceutical affairs law define products containing stem cells as regenerative medicine products and allow for the conditional approval of such products if safety has been confirmed in clinical trials, even if their efficacy has not been fully demonstrated.”

So you can guess where everyone is running to and isn’t the U.S. Here are press releases from Mesoblast and Athersys, respectively:


http://globenewswire.com/news-release/2013/11/25/592037/10059311/en/New-Japanese-Regenerative-Medicine-Legislation-and-Commercial-Opportunities-for-Stem-Cell-Products.html

http://finance.yahoo.com/news/athersys-announces-patents-japan-stem-120000430.html

Regarding plans for the U.S., I have thankfully partnered with Dr. Wade McKenna, who is Board Certified in Orthopedic surgery and Fellowship trained in Trauma and Trauma Reconstructive Surgery. Dr. McKenna has more experience using bone marrow concentrate for orthopedic conditions that anyone I know. We are opening a regenerative orthopedic center in the Dallas area hopefully by mid-April of this year. It will be in a new building and is being built out now. The center is called the Riordan McKenna Institute. It is located in Southlake, Texas, which is between Dallas and Ft. Worth, very near DFW airport.

Autologous Cell Therapies Do Not Represent a Public Health Risk and Should Not Be Regulated Like Drugs

SevOne Founder and Stem Cell Institute patient, Michael Phelan discusses what’s financially at stake for scientists, universities, drug companies, and the FDA who oppose autologous stem cell therapy and lobby for patients’ own stem cells to be regulated as drugs.

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Forbes interview with Michael Phelan from Feb 2013: One Man’s Reluctant Tour for Adult Stem Cells by John Farrell

Excerpt:

“I chose the Stem Cell Institute because they published their research in Translational Medicine. In addition, I corresponded with physicians and researchers experienced in Autologous Stem Cell treatments, including Roger Nocera, author of Healing Cells – Cells that heal us from cradle to grave, and I also listened to Arnold Caplan of Case Western.

So, at a Johns Hopkins managed hospital in Panama I had a mini-liposuction procedure. From my adipose-fat tissue they separated and expanded my cells, which took about a week then they gave to me in an IV.

I had visual problems for over a year before treatment, including double vision. After my first treatment in May of 2012, my vision problems resolved and I was able to continue driving. My mental and physical energy improved dramatically. A number of other problems improved. So, I was pleased with the outcome.”