Stem cells from umbilical cord blood can be used by othe family members.

Should I have my baby’s stem cells stored? Could they really save her from serious disease or permanent injury at some point in the future? Or is stem cell therapy simply wishful thinking exploited by charlatans? YOU Pulse’s Elise-Marie Tancred investigates

The promise of stem cell therapy may seem like magic or science fiction. Paraplegics will walk; cancer, heart disease, diabetes, Alzheimer’s and other degenerative diseases will disappear; and we’ll all drink from the fountain of everlasting youth . . .

Diseased and aged cells or organs can be repaired or replaced with stem cell therapy. This means recovery without major surgery, transplants, antibiotics, chemotherapy or other drastic medical interventions that would otherwise put your life on hold for months.

The risks are minimal: the body heals itself. Sounds fantastic? Yes, but theoretically it’s possible.

More and more parents are asking, ‘‘Should I store the stem cell-rich blood from my baby’s umbilical cord in case it could later help treat a life-threatening disease for which there is no other cure?’’

Dozens of South Africans are already doing this. They pay about R12 000 to have their baby’s stem cells frozen and stored for 20 years in case they’re required. The stem cells could be used to save this child or a close family member such as the child’s brother, sister, mother, father or grandparent if one of them were to develop cancer, heart disease, or another degenerative illness.

‘‘Revolutionary’’ is the word often used to describe this kind of treatment. But has stem cell therapy really made the progress predicted by scientists a decade ago? Not yet – although many hospitals worldwide boast success stories with conditions ranging from type 1 diabetes to multiple sclerosis. In some cases, such as bone marrow cancer, stem cell therapy is now regarded as standard treatment.

More research is needed but it’s already clear stem cell therapy will bring about a revolution in medicine.

STEM CELL THERAPY: A real Life saver – or just a dream?

Stem cell treatment in South Africa

The first cord blood bank in South Africa opened its doors three years ago. Since then another laboratory has started offering the same service.

Stem cells collected from umbilical cord blood are used to treat more than 100 diseases, in particular certain kinds of cancer, Professor Michael Pepper says. He’s the managing director of the Netcare Institute of Cellular and Molecular Medicine and Netcell Therapeutics, a local cord blood bank with international accreditation.

Cord blood banks are increasing in popularity as people become more knowledgeable, says Harry Minnie, executive head of Lazaron Technologies, SA’s first cord blood bank.

‘‘About 2 000 parents have already stored their children’s cord blood with us,’’ Minnie says.

But one of Lazaron Technologies’ greatest challenges is to convince the medical profession, not parents, of the value of such a bank.

The marvel of a cord blood bank is that the stored stem cells can also be used for family members or even other relatives of the baby. Siblings and parents have a chance of one in four of benefitting, and grandparents a chance of one in eight.

Approximately 60 per cent of cord blood transplants are performed on other family members, not the baby.

Today bone marrow stem cell transplants are already standard procedure for people who have blood cancer because it is easy, cheap and has a high success rate, says Dr Yvonne Holt, medical director of Netcell Therapeutics.

But questions surrounding stem cell therapy remain. Dare I let such an opportunity slip through my fingers? Is it meaningful risk management for my family? Or is it a get-rich-quick scheme for people who want to exploit our insecurities?

Will science not find alternatives for umbilical cord stem cells? Charles and Sonica Marais of Johannesburg believe storing the blood from the umbilical cord of their daughter, Chanique, when she was born two years ago was the best thing they’ve ever done. They’re expecting twins in August and plan to also store the new babies’ cord blood. ‘‘We hope we’ll never have to use the stem cells but we see it as a kind of medical insurance that brings enormous peace of mind,’’ Charles says.

‘‘Who knows, maybe my children’s umbilical cord blood will be used one day to cure a disease. It’s without a doubt the best thing I’ve ever spent money on.”

Well-known South African singer Nianell also recently stored her triplets’ cord blood at Netcell as medical insurance for the future. But after becoming a father a psychiatrist (who prefers to remain anonymous for ethical reasons) carefully considered the matter and decided against storing his baby’s cord blood.

‘‘Certain events in our life are not in our own hands,’’ he says. ‘‘If we should ever need something such as stem cells to treat disease in our family we’ll cross that bridge when we come to it.’’

He says he can invest the same amount of money more successfully and use it to buy stem cells in future if necessary.

‘‘One also has to be sure the technicians at the cord blood banks know what they’re doing,’’ he warns. ‘‘The stem cells of a colleague’s child are now virtually useless because the right procedure wasn’t followed when the blood was delivered by courier.”

How are stem cells obtained and stored?

Most stem cell banks prefer that the prospective parents contact them about three months before the baby’s birth but the arrangements can also be made in a few hours if a woman decides during labour she wants to store her baby’s cord stem cells.

As soon as the baby is born the gynaecologist collects the blood from the umbilical cord. This takes about five to 10 minutes. The blood is then packed into a temperature-controlled container and sent by medical courier to the bank’s laboratory.

When it arrives the blood is stored immediately because the stem cells deteriorate during the trip and until they reach the correct low temperature in the lab.

Reliable stem cell banks have to comply with certain requirements. There are guidelines you can follow to make sure you’re not being taken for a ride.

Controversy and exploitation

As with any major new scientific breakthrough there are the pioneers, those who experiment – and those who take advantage of a patient’s desperation to get well. So it’s no surprise the field of stem cell therapy is fraught with controversy.

Until recently there was not even a whisper of legislation in South Africa to regulate stem cell research. Any Tom, Dick or Harry could run a stem cell laboratory and experiment.

Things changed after Stephen van Rooyen and his wife, Laura Brown, who owned the Cape Town-based company Advanced Cell Therapeutics (ACT), were accused in America of selling stem cell treatments that had never been tested in laboratories.

In 2006 the FBI listed 51 complaints lodged against Van Rooyen and Brown, all relating to fraudulent claims about stem cell therapy, on its website.

Only then did South Africa’s department of health take drastic steps to stop the medical cowboys in their tracks, although legislation drawn up to regulate the industry is not in force yet.

In essence the law says all intellectual property relating to stem cell research belongs to the state. This puts a stop to the plans of greedy individuals and private companies who’re in the stem cell business just to make money.

The law also doesn’t allow researchers to patent or sell their findings.

‘‘Such legislation is hampering research,’’ Professor Pepper says. ‘‘No one will invest money in a concern that doesn’t make them money. The result is that hardly any stem cell research is taking place in South Africa.

‘‘But in this country we first have to focus on primary health care and combating contagious diseases, not on breakthroughs in the field of stem cell research.’’

Even in the US stem cell research is progressing at a far slower rate than it could, mainly because President George W Bush’s conservative government doesn’t give a cent of America’s National Institutes of Health’s annual health research budget of $220-billion to stem cell research.

Pharmaceutical companies also don’t want to invest big money in stem cell research. Experts say it’s because they fear stem cell therapy will replace conventional medicine.

Renewed exploitation?

Dr Jeff Peimer, a South African doctor who used to work for the controversial stem cell company ACT, has now started a stem cell clinic, Regenecell, with his brother, Sid Peimer, in the Seychelles.

They claim type 2 diabetes, autism, multiple sclerosis, Parkinson’s disease, rheumatoid arthritis and stroke damage can all be treated with stem cells obtained from cord blood.

But according to medical experts consulted by YOU Pulse the treatment of many of these diseases is merely experimental and there’s no proof the stem cell therapy is successful.

Is this another case of pulling the wool over patients’ eyes?

Ethical experts are concerned that desperate patients are paying thousands of rands for experimental treatment that may not help.

According to Professor Lyn Horn, head of ethics in the faculty of health sciences at the University of Stellenbosch, a doctor is allowed to treat a terminally ill patient using experimental techniques only if it’s performed as a charitable deed.

‘‘The doctor’s reason must be to save the life of the patient, not make money. When patients are forking out large amounts of money for experimental treatment it could create a conflict of interest for the doctor who may have shares in such a facility.’’

Regenecell requires a prepayment of R134 000. We also know a few paralysed South Africans have paid thousands of rands for experimental stem cell therapy abroad.

The future

Scientists are expecting a major breakthrough soon. ‘‘Stem cell therapy will come into its own within the next five to 10 years,’’ Professor Pepper says. ‘‘There are great emotional expectations in this field. Sick people who’re dying at the moment could be saved in a decade or two.’’

If researchers could unravel the secret of how stem cells become specialised cells it would be possible to prevent serious genetic illnesses such as cancer and birth defects before they even start.

The magic of stem cells is that they single out damaged cells and change into new, healthy cells of the same kind. Organ donation will be unnecessary, because diseased and weak organs will be renewed with stem cell therapy. Stem cells can also be used to test new drugs.

Stem cell therapy sounds promising indeed – but major technical and ethical stumbling blocks have to be overcome before we can pick the fruits of this amazing treatment, Professor Horn adds.

‘‘Many of the positive results come from experimental treatment of a small group of patients with limited follow-up treatment. Only when we get positive results with extensive clinical tests and when possible side-effects such as immune system rejection have been ironed out dare we become excited.’’

Harry Minnie puts it in a nutshell: ‘‘When you try to look at the bigger picture each major breakthrough is a small step in the right direction. You could compare it to a jigsaw puzzle – each piece is important but the puzzle consists of thousands of pieces.’’

WHO HAS BEEN HELPED?

Helped

JOSÉ CARRERAS (61)
, world famous tenor. He was diagnosed with acute lymphoblastic leukaemia in 1987 at the age of 40. His chances of survival were slim but after gruelling cancer and bone marrow treatment he has recovered and still performs.

MATTHEW ROSS (10),
a young leukaemia sufferer from Durban, was recently treated with stem cells from his baby sister. The family had his sister’s cord blood stored at Netcell so it could be available for Matthew. He’s making good progress.

GERT SMIT (81) of Kuils River, Cape Town, celebrated his 80th birthday last year after Dr André Saaiman, cardiologist at the Kuils River Hospital, injected stem cells directly into his weakened heart. His heart function, a mere 20 per cent at the time, improved dramatically. But oom Smittie, as he’s known, is the only surviving member of a group of four who were suffering from heart disease and were experimentally treated with stem cells in 2005. This project has since been suspended because funding has dried up.

In vain

CHRISTOPHER REEVE of Superman fame, paralysed after a horseriding accident in 1995, didn’t benefit from stem cell therapy and died in 2004.

Too late

CHRIS BARNARD revolutionised medicine when he performed the world’s first human heart transplant. A revolution in treatment for arthritis or asthma, illnesses he suffered from, still lies many years in the future. Barnard died from asthma at the age of 78 in 2001.

Not yet

STEPHEN HAWKING (66), world famous physicist who suffers from motor neurone disease, commonly known as Lou Gehrig’s disease. Stem cell therapy has not helped him yet.

MICHAEL J FOX (47), actor, suffers from Parkinson’s disease and supports stem cell research through his foundation but has not benefited from the therapy yet.

HOW DO STEM CELLS WORK?

Stem cells have unique properties and the remarkable potential to change into other cells. They act as a kind of self-help recovery system for the body.

Stem cells are the master building blocks of the human body because of these three properties:

• Plasticity – they can change into any cell.


• Homing – like a homing torpedo they travel to where the diseased cells are.


• Engraftment – they become part of other cells.

For example, if someone has a nerve cell disease and receives stem cell therapy the stem cells travel from the damaged cells (homing), become part of the nerve cells (engraftment) and transform into nerve cells (plasticity).

The stem cells that didn’t engraft or migrate return to the bone marrow where they’re stored until they receive a signal indicating there is diseased tissue elsewhere in the body. This explains why the effect of stem cell therapy is visible often only after a few months.

Different types of stem cells are used: embryonic stem cells, bone marrow stem cells, stem cells derived from umbilical cord blood and stem cells from any other organ or tissue.

Embryonic stem cells are the most powerful kind of stem cell – and the most controversial. When these cells are harvested the embryo (the future baby) is destroyed in the process. Some see this as a kind of abortion.

The difference between embryonic and adult stem cells is that embryonic stem cells have the potential to transform into any type of cell. Adult stem cells have already differentiated and are programmed for a specific function. They also have a limited growth period.

An advantage of the use of adult stem cells is that the patient’s own cells are cultivated in a laboratory and re-injected. This almost eliminates the rejection of cells by the immune system – a fundamental risk in the case of embryonic stem cell transplants.

In a major breakthrough in November last year American scientists successfully reprogrammed human skin cells so they look and act exactly like embryonic stem cells. This success means the use of embryonic stem cells, which has been overshadowed by ethical and political issues, may become unnecessary.

Why are the stem cells derived from umbilical cord blood better than the stem cells from your own blood?

Cord blood cells are brand-new and therefore ‘‘naive’’ and more changeable than other kinds of stem cells, Professor Pepper explains. These stem cells are untouched by ageing – quite different to the stem cells of, for example, someone in their forties.

Worldwide there is an increase in the use of cord blood stem cells for bone marrow transplants, while the use of stem cells from bone marrow and blood is on the decrease.

The use of stem cells has its pros and cons. The advantage is that cord blood stem cells aren’t easily rejected by the receiver. The disadvantage is the number of stem cells in cord blood is so low it’s used mainly for children.

HOW MUCH DOES IT COST?

Netcell charges a once-off fee of R12 000 for 20 years’ storage. Lazaron Biotechnologies charges an initial fee of R7 923 and then an annual storage fee of R142,50 amounting to R10 773 over a 20-year period. Stem cells can be stored indefinitely.

Choosing a reliable stem cell bank

It’s important to select the stem cell bank that has the best chance of still being around in 20 years and will freeze and store your stem cells correctly. Here are the guidelines from the American Association of Blood Banks, which are seen as the norm for international guidelines:

• Make sure the bank is registered with FACT (Foundation for the Accreditation of Cellular Therapy). This ensures the harvesting and storage of stem cells comply with the required laboratory standards.
• The bank must count the stem cells to make sure the cells are alive before they are stored. This is called flow cytometry testing. It’s an expensive process and parents receive a certificate to indicate how many stem cells have been stored.


• The bank must use blood bags with two compartments – this simplifies future testing.

Successful stem cell therapy

For the following diseases stem cell treatment is the only therapy available – or it’s used if other treatments weren’t successful – and a good success rate has been achieved:

• Various kinds of acute and chronic forms of leukaemia (blood cancer)


• Pre-leukaemia


• Hodgkin’s lymphoma and non-Hodgkin lymphoma


• Congenital red cell abnormalities including sickle-cell anaemia and Fanconi anaemia


• A number of inherited immune system diseases


• Certain kinds of bone marrow cancer


• Cancers such as neuroblastoma (one of the most common types of cancer in children) and retinoblastoma

For the conditions below human testing is at an advanced stage. Treatment can delay the disease or in some cases cure it:

• Type1 diabetes and lupus (a deadly type of arthritis that affects the kidneys and is prevalent among coloured women in the Western Cape)


• Cerebral palsy and multiple sclerosis


• Inherited immune system or metabolic diseases (such as Tay-Sachs disease)


• Weakened heart muscles


• Breast cancer, Ewing’s sarcoma and kidney cancer

For a list of diseases visit Parent's Guide to Cord Blood Banks

YOU Pulse June 2008