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Vatican cell conference opens with focus on kids, rare disease

Vatican regenerative medicine conference opens with focus on kids, rare disease - EPA

Vatican regenerative medicine conference opens with focus on kids, rare disease - EPA

29/04/2016 06:49

(Vatican Radio)  For a child to be born sick is a “scandalous” problem for humanity.  That was one of Cardinal Gianfranco Ravasi’s reflections Thursday as he opened in the Vatican day one of the Third International Conference on the Progress of Regenerative Medicine and its Cultural Impact.  The President of the Pontifical Council for Culture partnered with the Stem For Life Foundation to organize what has been described as a “historic” three day event 28-30 April to look at the complex cultural and social framework of illnesses and at cutting edge research into cellular therapies.

In her opening remarks, the President of Stem For Life, Dr. Robin Smith, pointed to the growing range of therapies currently under study for the treatment of cancer, autoimmune disorders and rare diseases.  The first in the series of conferences was launched five years ago, she noted, to foster a dialogue about the importance of stem cell therapy.  Since then, the sector has progressed exponentially as scientists became increasingly aware of their ability to be “taught” to transform into a wide variety of tissue, cells and even organs.

Saving lives or playing God?

“Cellular cures are the light in front of us,” she said, but they need to be made more rapidly available to patients.  Super computers and ever-more powerful diagnostic tools are making it easier to identify the right treatment for the right patient at the right time.  The advances in cellular therapy are happening so quickly, she suggested, it will not be long before people begin to ask: can we design our own child?  Choose its hair and eye colour, its height and intelligence?  Can we turn back time and reverse aging?  Are we playing God?  The philosophical and ethical questions abound.

Smith invited us to have tissues at the ready for the heart-wrenching stories we were about to hear.  Stories like Good Morning America anchor Robin Roberts’ exhausting battle with breast cancer which evolved into any doctor’s worst nightmare: Mylodisplastic  Syndrome (MDS) or pre-Leukemia. She was told she had less than two years to live. But thanks to her sister, Sally, Robin received a perfect match for a bone marrow transplant that saved her life.

Transplants and “Reengineering” can transform lives

We heard that more than 70 disorders can be treated with bone marrow transplants.  Nearly half of the 50,000 such transplants performed around the world each year require a donor.

Though national registries have made matching up donors to patients easier in recent years, finding the right fit can take months. That, even though there are more than 20 million voluntary bone marrow donors worldwide.  Scientists are finding ways to train bone marrow cells to adapt to new hosts so they won’t be rejected by the body’s immune system.  They’re also finding promising new techniques by taking a patient’s own cells and re-programming them to fight off “bad” cells.  One such technique is called “chimeric antigen receptor T-cell therapy,” a revolutionary but experimental treatment which reengineers the patient’s cells to kill off all cancerous cells.

17 year old Nicholas Wilkins was diagnosed with the most common childhood cancer, Acute Lymphoblastic Leukemia, a cancer of the blood and bone marrow, at age 4.  After repeated relapses, he received a bone marrow transplant from his sister. But even that didn’t work. In 2013, his desperate parents enrolled Nicholas in a trial at the Children’s Hospital of Philadelphia where doctors reinfused reengineered T-cells back into his body to attack the cancer.  Three years later, he is cancer free and doctors are hopeful he will stay that way because the “good” T-cells are continuing to fight the cancer.

Researchers are hopeful this technique can be just as promising in the treatment of other diseases, such as rare and autoimmune disorders.

90% of kids with cancer die in developing nations

Georgetown University Health Care Ethics Professor Fr. Kevin Fitzgerald, sj told us that some 80-90% of children with cancer in industrialized countries are cured while 90% die in poor countries.  The moral imperative, then, is to ensure adequate medical care in developing countries: an invitation to policy makers, businesses, the pharmaceutical sector and medical and research communities to collaborate to make this a reality.  And, he reminded us that as the largest health care provider in the world, the Catholic Church is ready to partner with them.

Eugene Gasana Jr was 13 when he was diagnosed with Hodgkin lymphoma in 2011 and after intensive chemotherapy and radiation therapy in New York, he has been in remission. But Eugene wasn’t satisfied with just getting better himself.  He wanted kids in his home country of Rwanda to have access to similar, high quality medical care.  Thanks to a Foundation set up in his name and donors, his paediatric oncologist, Dr. Tanya Trippett of Memorial Sloan Kettering Cancer Center, is heading up a program to provide a hospital and cancer care for children in Kigali for the east African region.

According to Trippett, serving cancer patients in Rwanda and other parts of Africa is a challenge because of the lack of quality diagnostic equipment and in some cases, the absence of chemotherapy and cancer drugs.  The infrastructure is poor and oncologists are few.  Patients go hungry in hospitals which also struggle to provide follow-up care for families who live far away.  She wants to see more cooperation between Western hospitals and clinical professionals to provide training to Rwandan and other African doctors, nurses and hospital staff and greater access to funding.

Dr. Raphael Rousseau, Medical director of Genentech, a member of the Roche pharmaceutical group, would like to see more clinical trials in developing countries, using the same rigorous standards as Western trials.  He says he’s frustrated that drugs are not getting soon enough to children with cancer and appealed to drug companies to develop new therapies for cancer, especially in developing countries “where cancer is lethal.” This not an area of competition, he said, “we’re all in it for a good cause.”

Cord blood’s life-saving stem cells

Dr. Joanne Kurtzberg of Duke University Medical Center works with cord blood stem cells to find cures for brain diseases like cerebral palsy, or autism, and in some cases, with remarkable results.  Not long ago, after a woman gave birth, the placenta used to be thrown out in the trash, she said.  But now, the stem cell-rich material can be frozen and stored, perhaps for decades, in the some 700,000 public cord blood banks around the world until it is needed for therapy. Some four million banks preserve cord blood for private use. Cord blood can be an alternative source, she said, for patients who can’t find a matching donor.

Dr. Yong Zhao of Hackensack University Medical Center is finding encouraging results using cord blood cells for multiple autoimmune and inflammation-related diseases.

The rare disease challenge

The new treatments evolving are many: “nano technology,” “nano chips,” “gene therapy” and “gene editing” were some of the terms thrown out by the U.S. National Institute of Health’s Dr. Stephen Groft who said 4-8% of the population suffers from a rare disorder. Some 8,000 rare diseases have been identified, and most have a genetic origin, but more diseases are occurring and mutating. Multiply that by family and friends, he said, “and you have a big population affected by rare diseases.”  A lack of information on such disorders, misdiagnosis and lack of treatments are the real challenges facing patients with rare diseases.

But Dr. Groft is among a number of health experts worldwide who are compiling data bases of patients, doctors, symptoms, and treatment protocols so that the global health community can study these rare diseases and communicate with each other about them.  Social media plays a big part here, he said, as patients exchange their stories and search for clinical trials in which to participate and doctors looking for colleagues who have come across similar patient cases.

We heard about 14 year old Johnathan who suffers from a disorder known as “Butterfly disease,” a frightfully painful condition that makes his skin as fragile as powdery butterfly wings but has nothing to do with the beauty of the delicate creature.  Johnathan and his mom spend hours each day dressing him, bathing and changing the bandages covering the sores on much of his frail body. Here was one of the many times  I reached for a tissue on Thursday.   Johnathan knows he probably won’t survive past his mid- 20’s.

Then, there were the children with Batten disease, which one father described as a “thief” which comes in the night to steal away your small child’s vision, his brain, his ability to walk and talk.  And, the kids suffering childhood blindness who are receiving encouraging help with gene therapy.

Dr. Neil Warma of Opexa Therapeutics, is working with personalized T-cell vaccines to fit each individual’s patient’s profile to treat an array of autoimmune disorders including Multiple Schlerosis and NMO so the body can repair itself. New therapies are also evolving in the treatment of Type 1 Diabetes or juvenile diabetes giving fresh hope to patients suffering from this debilitating disorder too.

Tracey McClure

29/04/2016 06:49