Emirati man with rare blood disorder benefits from new $2 million gene therapy in the US

The National

a day ago

An Emirati man with a rare inherited disorder has become only the second patient in the world to benefit from a pioneering $2 million gene therapy that eliminates the need for regular blood transfusions.
Khalifa Dawood, 22, from Abu Dhabi said he has been given a 'new lease on life' as he recovers from the novel gene treatment he received at Cleveland Clinic Children's hospital in Ohio, which recently won medical approval for the therapy.
The UAE has some of the regions highest rates of thalassaemia, also known as sickle cell disease, a condition that limits the body's ability to produce healthy red blood cells.
While some carriers show very limited symptoms, others experience bone deformities, shortness of breath, dizziness, organ damage and heart palpitations.
The condition can require regular blood transfusions, with research suggesting thalassaemia affects as many as 16.5 per cent of the population.
A new treatment used for the first time by the Cleveland Clinic Children's hospital has proven to modify a patient's blood forming cells, and can be delivered as a one-time treatment called Casgevy.
Mr Dawood said that he hoped the treatment would allow him to lead a normal life, without the need for regular hospital stays.
'I am hoping this gives me a new lease on life where I am energised to enjoy it,' he said.
Life-changing treatment
Since its approval by the US Food and Drug Administration in January, the therapy has been delivered to an American woman from Ohio and Mr Dawood.
I am hoping this gives me a new lease on life where I am energised to enjoy it
Khalifa Dawood
Although the treatment is expensive, it is life changing for those who receive it, according to Dr Rabi Hanna, chairman of the paediatric haematology-oncology department at Cleveland Clinic Children's hospital.
It is not uncommon for adults to receive specialist care at a children's centre, particularly for conditions they have had since birth, as is the case with Mr Dawood.
'Unlike regular medicines that people take for years, Casgevy is meant to be a one-time treatment that could last a lifetime,' he said.
'It uses CRISPR gene editing, which is a very advanced science. Editing cells safely and precisely takes a lot of time, technology, and testing.
'It is also custom-made, as Casgevy is made from a person's own cells, which are collected, sent to a lab, edited and then returned. That process is complex and individualised.'
The innovative treatment uses a gene editing technology called CRISPR/Cas9 to modify a patient's stem cells.
The edited cells are then transplanted back into the patient, where they produce more haemoglobin in new healthy red blood cells.
The FDA's approval was based on results from a single arm which showed 91 per cent of patients with beta thalassaemia did not need a blood transfusion for at least a year after having the treatment.
Costly medical care
Despite the therapy's success, it is likely to remain out of reach for the majority with costs of about $2.2 million.
Gene therapies are typically the most expensive medical treatments.
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of haemophilia B, a potentially life threatening disorder that prevents blood from clotting.
Priced at about $3.5 million, it was the world's most expensive drug until the arrival of Lenmeldy in March last year.
The gene treatment developed by Orchard Therapeutics costs about $4.2 million and is used to treat life threatening nerve disorders in young children.
High costs of certain drugs can be covered by insurers, government health systems or charitable foundations when there are no other treatment options.
Drug companies also occasionally offer financial support programmes for families in need.
The process of administering Casgevy begins by collecting stem cells from the patient's blood that are then sent to the lab to be edited using tiny, precise scissors.
By clipping off the DNA responsible for preventing the body making healthy haemoglobin, the repaired cells can then be returned to the body.
A course of chemotherapy is then required to clear out the old, sick marrow cells, allowing new healthy ones to grow in their place.
Recovery time in the hospital is about four weeks.
As the science behind gene editing improves, it should become faster, easier and cheaper to edit cells with higher accuracy, and fewer side effects.
'It is my hope and prediction that price will come down, but I am not sure how quickly,' said Dr Hanna.
'As the science of gene editing improves, it may become faster, easier, and cheaper to edit cells with higher accuracy and fewer side effects.
'Right now, Casgevy is the first gene editing therapy approved for sickle cell and thalassaemia.
'But more companies are developing similar treatments. When that happens, competition can help drive prices down.
'Also as companies learn how to make these therapies at scale, they may reduce production and delivery costs.
'Think of it like early computers or smartphones – they were expensive at first, but prices dropped as technology improved.'
Global health drive
Although patients like Mr Dawood must travel overseas for expensive gene therapy, the Gulf is also emerging as a go-to destination for emerging treatments.
The King Faisal Specialist Hospital & Research Centre in Saudi Arabia is emerging as a leader of regional clinical trials and delivered a breakthrough in haemophilia care, using one-time gene therapy to restore clotting function in eight patients.
In the UAE, at Medcare Royal Speciality Hospital Al Qusais, patients are receiving the latest care for rare genetic disorders like spinal muscular atrophy and Duchenne muscular dystrophy, two life-limiting growth conditions.
Doctors there said patients were travelling into Dubai from Turkey and elsewhere in the region.
Medcare Women and Children Hospital was one of the first private clinics outside the US to administer gene therapy for spinal muscular atrophy, and has since delivered 100 infusions.
American Hospital, Al Jalila Children's Hospital and Fakeeh University Hospital also offer gene therapy Zolgensma, which can cost about $2 million per dose.
Dr Vivek Mundada, a consultant paediatric neurologist at Medcare Royal Speciality Hospital, said the drug had delivered remarkable outcomes in managing complex genetic conditions.
'Dubai is becoming a centre of medical innovation, and Medcare is proud to be leading that movement, especially in the field of rare genetic diseases,' he said.
'Through our comprehensive, patient-centred care model, we are transforming outcomes for children who previously had limited treatment options.'