A new stem cell research laboratory due to open in Dubai promises to accelerate the understanding of human genomics in the region and improve health outcomes from genetic diseases.
The Hortman Stem Cell Laboratory is due to open in May on The Palm Jumeirah’s Golden Mile, with laboratories and clean rooms purpose-built for stem cell isolation, culture and expansion to take the next step in preventive health care and research in Dubai.
As the UAE becomes a regional hub for life sciences, the first stem cell congress held in Dubai heard about the value of banking cord blood and placental tissue in preserving human health.
Stem cell research is an emerging area of healthcare, allowing parents to store cord blood from the placenta of newborns which could be used to treat cancers, immune deficiencies and genetic disorders.
Although average stem cell therapy is expensive — typically costing from $5,000 to $50,000 — the results can be life saving.
“We will begin clinical trials at the lab once it opens in May to take stem cells from cord tissue to primarily research heart and lung diseases,” said Dr Fatma Alhashimi, director of the Hortman Stem Cell Laboratory.
“The challenges we are facing in the UAE is having enough GMP [good manufacturing practices] labs for this kind of research and clinical trials.
“It is expensive and not always cost-effective but it saves lives and that is where the value is.”
The centre will also research metabolic disorders, sickle cell anaemia and Thalassemia, an inherited blood condition that is one of the most common genetic disorders in the UAE.
Value of gene therapies
Longevity science and the value of gene therapies in treating long-term disease and illness were discussed by specialists at the congress, while patient case studies revealed the value of therapies.
Linda Konsinski, a mother of three from the US, was diagnosed with leukaemia 15 years ago, at age 38.
A cord blood transplant from a two-year-old boy saved her life after a blood test revealed a dangerously high white blood cell count that indicated Philadelphia Chromosome positive acute lymphocytic leukaemia.
“Hearing the words ‘you have cancer’ were the scariest words ever said to me,” said Ms Konsinski.
“The day I heard them my life turned upside down.
“My doctors said I needed a bone-marrow transplant to survive long-term but neither of my sisters were a match so I had to search registries around the world.”
Unable to find a match, a cord blood transplant was considered instead.
Doctors at the City of Hope Medical Centre in California offered to help and a search began.
As cord blood was already in storage and any mismatches were more tolerated than bone marrow transplants, two potential donors were found.
Cord blood from a baby boy in New Jersey stored for 10 years and another from a young boy in Italy stored for six years were found to be suitable.
Ms Konsinski flew to California to begin the transplant process, staying for about 17 weeks for treatment and recovery.
“Each day got better and the cells began to graft,” she said.
“When I went home, it was the best day ever. I have been given more time with my family and been able to see so many things I thought I would never get to see.”
Diagnose early
Other developments discussed at the congress included gene replacement therapies for spinal muscular atrophy, a condition that affects about 1 in 10,000 newborns.
It is the second most common fatal autosomal recessive disorder after cystic fibrosis, with 60 per cent of those with Type 1 SMA experiencing rapid, irreversible motor neuron loss.
Gene therapy has helped reverse symptoms, allowing children to sit up, feed, swallow and walk with assistance.
“We know that children who are treated pre-symptomatically generally do better, so there is a need for neonatal screening,” said Dr Vivek Mundada, speaking at the congress as a consultant paediatric neurologist at the Medcare Women and Children Hospital, Dubai.
“Many countries are moving towards that, with Kuwait and Qatar having already started in the Middle East as have Belgium, Germany and Poland.
“If you treat and diagnose these children early, you can make a huge difference to their lives.”
