Category Archives: Stem Cell Therapy

Contact Information

Available for logged-in reporters only

Newswise UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called Bubble Baby disease, a life-threatening condition that if left untreated can be fatal within the first year of life.

The groundbreaking treatment was developed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Donald Kohn, whose breakthrough was developed over three decades of research to create a gene therapy that safely restores immune systems in children with ADA-deficient SCID using the patients own cells with no side effects.

To date, 18 children with SCID have been cured of the disease after receiving the stem cell gene therapy in clinical trials at UCLA and the National Institutes of Health.

All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in Life Sciences.

To protect children born with SCID they are kept in isolation, in controlled environments because without an immune system they are extremely vulnerable to illness and infection that could be lethal.

Other current options for treating ADA-deficient SCID are not always optimal or feasible for many children, said Kohn. We can now, for the first time, offer these children and their families a cure, and the chance to live a full healthy life.

Defeating ADA-Deficient SCID: A Game-Changing Approach

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases, and in a child with ADA-deficient SCID even the common cold can prove fatal. The disease causes cells to not create an enzyme called ADA, which is critical for production of the healthy white blood cells that drive a normal, fully-functioning immune system. About 15 percent of all SCID patients are ADA-deficient.

See more here:
UCLA Stem Cell Researcher Pioneers Gene Therapy Cure for Children with "Bubble Baby" Disease

Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease.

The study results were presented today at a Breaking Basic Science presentation during the American Heart Association Scientific Sessions in Chicago. After laboratory mice with Duchenne muscular dystrophy were infused with cardiac stem cells, the mice showed steady, marked improvement in heart function and increased exercise capacity.

Duchenne muscular dystrophy, which affects 1 in 3,600 boys, is a neuromuscular disease caused by a shortage of a protein called dystrophin, leading to progressive muscle weakness. Most Duchenne patients lose their ability to walk by age 12. Average life expectancy is about 25. The cause of death often is heart failure because the dystrophin deficiency leads to cardiomyopathy, a weakness of the heart muscle that makes the heart less able to pump blood and maintain a regular rhythm.

"Most research into treatments for Duchenne muscular dystrophy patients has focused on the skeletal muscle aspects of the disease, but more often than not, the cause of death has been the heart failure that affects Duchenne patients," said Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute and study leader. "Currently, there is no treatment to address the loss of functional heart muscle in these patients."

During the past five years, the Cedars-Sinai Heart Institute has become a world leader in studying the use of stem cells to regenerate heart muscle in patients who have had heart attacks. In 2009, Marbn and his team completed the world's first procedure in which a patient's own heart tissue was used to grow specialized heart stem cells. The specialized cells were then injected back into the patient's heart in an effort to repair and regrow healthy muscle in a heart that had been injured by a heart attack. Results, published in The Lancet in 2012, showed that one year after receiving the experimental stem cell treatment, heart attack patients demonstrated a significant reduction in the size of the scar left on the heart muscle.

Earlier this year, Heart Institute researchers began a new study, called ALLSTAR, in which heart attack patients are being infused with allogeneic stem cells, which are derived from donor-quality hearts.

Recently, the Heart Institute opened the nation's first Regenerative Medicine Clinic, designed to match heart and vascular disease patients with appropriate stem cell clinical trials being conducted at Cedars-Sinai and other institutions.

"We are committed to thoroughly investigating whether stem cells could repair heart damage caused by Duchenne muscular dystrophy," Marbn said.

In the study, 78 lab mice were injected with cardiac stem cells. Over the next three months, the lab mice demonstrated improved pumping ability and exercise capacity in addition to a reduction in heart inflammation. The researchers also discovered that the stem cells work indirectly, by secreting tiny fat droplets called exosomes. The exosomes, when purified and administered alone, reproduce the key benefits of the cardiac stem cells.

Marbn said the procedure could be ready for testing in human clinical studies as soon as next year. The process to grow cardiac-derived stem cells was developed by Marbn when he was on the faculty of Johns Hopkins University. Johns Hopkins has filed for a patent on that intellectual property and has licensed it to Capricor, a company in which Cedars-Sinai and Marbn have a financial interest. Capricor is providing funds for the ALLSTAR clinical trial at Cedars-Sinai.

Read the original post:
Cardiac stem cell therapy may heal heart damage caused by Duchenne muscular dystrophy

By: Adam Feuerstein | 11/18/14 - 10:16 AM EST

Inject a cocktail of undifferentiated stem cellsinto a patient who has suffered a heart attack, and days or even weekslater, the stem cells transform into cardiac cells and rebuild the damaged heart muscle. Months later, the patient has a "new" healthy heart.It's a great story. But so far, the proof remains elusive though not for a lack of trying.

The latest company to fulfill this ambitious scenario is NeoStem (NBS) which presented disappointing (but not surprising) results from a small study of its proprietary cardiac stem-cell therapy NBS10 at the American Heart Association annual meeting Monday. NeoStem tried to put some positive spin on the bad news but shares are down 25% to $5.10.

NBS10, formerly known as AMR-001, is an autologous stem-cell therapy derived from a patient's own bone marrow. When injected back into patients following a heart attack, the stem cells are supposed torestore blood flow, rebuild damaged cardiac muscle and improve function.

Except in NeoStem's study, NBS10 fell short on two primary endpoints designed to assess the therapy's efficacy. The study used non-invasive imaging to assess blood flow through the heart, six months after a single infusion of NBS10 or a placebo. There was no difference between NBS and placebo, NeoStem said.

The study's other co-primary efficacy endpoint was a measurement of adverse cardiac "MACE" events --defined as cardiovascular death, a repeatheart attack, heart failure hospitalization and coronary revascularization. To date, 17% of patientstreated with NBS10 have suffered a MACE event compared to 19% of patients in the placebo arm -- a difference which was not statistically significant.

NeoStem said NBS10 therapy was safe relative to placebo and that no patients treated with the stem cells have died compared to three deaths in the placebo patients. But with only one year of follow up on a small number of patients, any claims about a mortality benefit are clinically meaningless.

Originally posted here:
NeoStem's Stem Cell Therapy Fails Mid-Stage Heart Attack Study

CBS Los Angeles (con't)

Affordable Care Act Updates: CBSLA.com/ACA

Health News & Information: CBSLA.com/Health

WESTWOOD (CBSLA.com) Doctors say a groundbreaking stem cell therapy treatment out of UCLA may have cured Bubble Baby syndrome once and for all.

KNX 1070s Brian Ping reports Dr. Donald Kohn has perfected a gene therapy that has now cured 18 children born without an immune system, known as ADA-deficient severe combined immunodeficiency (SCID).

Only weeks after giving birth to fraternal twins in 2012, Alysia and Christian Padilla-Vaccaro found out their daughter Evangelinas immune system was so deficient that she could have no exposure to the outside world.

After enrolling their daughter in Dr. Donald Kohns revolutionary stem cell gene therapy treatment which was nearly three decades in the making doctors extracted stem cells from the bone marrow in Evangelinas hip, then used a modified mouse virus to correct her faulty gene before replacing the marrow.

You hear the words mouse virus and you want to run the other way, said mom Alysia. But they modify it so that its teaching it to do something that they want it to do, which is put something in there that was missing.

Evangelinas new immune system developed without side effects and she is now living a healthy normal life.

Her mother Alysia said while the process was difficult for any mom to go through, it was all worth it.

Link:
UCLA Doctors Hail Potential Cure For Bubble Baby Syndrome

A large-scale trial conducted in India has shown that stem cell therapy does not work in stroke patientsREUTERS

A study conducted on 120 patients in India has shown that stem cell treatment is not effective in treating paralysis resulting from a stroke.

The research which is thefirst large-scale study conducted in Indiacompared outcomes in those treated with stem cells to others and found no difference, reports Down to Earth.

While 60 patients with some form of disability of limbs caused by a stroke were given conventional treatment, an equal number received bone marrow stem cells in addition. All had experienced a stroke 3-4 weeks before the trial.

"We found that at the end of the first month, patients with stem cells showed more improvement compared to the control group. But at the end of the third month and one year, there was no difference," said Kameshwar Prasad, head, Department of Neurology, All India Institute of Medical Sciences (AIIMS), who led the study.

On an average 280 million bone marrow cells were injected, of which blood forming stem cells were around 2.9 million per patient.

The average age of patients in the study was around 50.

The study, published in the current issue of American journal Stroke, was conducted at AIIMS in New Delhi and four other hospitals covering four cities.

The study comes when many others have been suggesting that stem cells could help treat paralysis in stroke patients. The earlier study was done on a small number of patients as compared to the AIIMs study.

More research needs to be done, before stem cells are used in therapy as in India, many private clinics are openly offering stem cell treatment for various diseases.

Link:
Stem Cells Treatment Not Useful In Stroke Patients Finds Indian Study

Beverly Hills, California (PRWEB) November 17, 2014

Veteran television and movie actor Darius McCrary has received a revolutionary stem cell procedure for his painful knee and ankle. The regenerative medicine procedure with stem cells was performed by Dr. Raj, a top orthopedic doctor in Beverly Hills and Los Angeles.

Darius McCrary is well known for his decade long stint on Family Matters as character Eddie Winslow. He won a Best Young Actor Award for this role along with movie roles in both Mississippi Burning and Big Shots. Currently, Darius appears along with Charlie Sheen in the show Anger Management.

While staying in tip top shape for his career, Darius has developed persistent pain in his right knee and ankle. Rather than seek a regular cortisone injection for pain relief or opt for surgery, he desired the ability to repair the joint damage and achieve pain relief. "I couldn't imagine being immobilized because of injury, so I opted for a stem cell procedure."

The procedures were performed by Dr. Raj, who is a prominent Beverly Hills orthopedic doctor with extensive experience in regenerative medicine. The procedure consisted of a combination of platelet rich plasma therapy along with amniotic derived stem cell therapy. Anecdotal studies are showing that the stem cell procedures for extremity joints allow patients to achieve pain relief and often avoid the need for potentially risky surgery.

Dr. Raj has performed over 100 stem cell procedures for patients who have degenerative arthritis or sports injuries. "Patients do extremely well with the procedures. Minimal risk and there's a huge potential upside!"

With an active acting career, Darius McCrary cannot afford to be distracted with chronic pain. "I'm looking forward to getting back in the gym and going hard without this pain," he stated excitedly. The procedure was filmed and can be seen on Dr. Raj's Facebook page.

To discuss stem cell procedures at Beverly Hills Orthopedic Institute and how they can benefit, call (310) 247-0466.

View original post here:
Veteran Actor Darius McCrary from Family Matters Receives Stem Cell Procedures with Dr. Raj in Beverly Hills

Posted Tuesday, November 18th 2014 @ 5am

Local orthopaedic doctors are using a new stem cell technology, which has been successfully used to speed the recovery of superstar athletes like Rafael Nadal and Peyton Manning, on weekend athletes which suffer from a wide range of relatively common ailments, Newsradio 1200 WOAI reports.

Dr. Christian Balldin at the San Antonio Orthopaedic Group says the stem cells help the body recovery more quickly from a range of aches and pains.

"We are starting to apply it in a multitude of areas, including sports injuries, whether it be into actual joints, or to allow for a quicker recovery of ligament sprains and also for low back pain," he said.

Current FDA policy, due to ethical concerns about stem cell use, only allow stem cells harvested from the patient to be injected directly into the site of the injury.

"Take that with a big syringe and we actually thin it down to get the proper concentration of the stem cells, in addition to all of the growth factors that are in the bone marrow, and then we inject them into the area of interest."

Some athletes have traveled to other countries where different types of stem cells in different mixtures can be used. Dr. Balldin says they usually take stem cells from the bone marrow of the patient.

He says stem cells prompt growth, and because they can rejuvenate muscles and ligaments, they help speed the recovery. Dr. Balldin says they only work following a successful surgical procedure, the stem cells themselves cannot cure tears and breaks.

But he says there are indications that this therapy can be successful on many sorts of routine injuries which are suffered by weekend warriors who are not Rafael Nadal or Peyton Manning.

View post:
Stem Cells Being Used to Boost Recovery Time for Local Sports Injury Patients

Why Stem Cells Aren #39;t Being Tested in the US
Stem cell treatment is restricted in the United States, and we discuss the reasons the FDA has been so restrictive about the game-changing research and thera...

By: TheLipTV

Read more here:
Why Stem Cells Aren't Being Tested in the US - Video