Health experts has emphasised the need for Nigerian Government to adopt gene therapy for treatment of sickle cell diseases, noting that an estimated four million Nigerians are living with the disease.
Prof. Jennifer Adair, Co-founder, Global Gene Therapy Initiative made the emphasis during a news conference at the 5th Global Sickle Cell Congress in Abuja.
According to her, gene therapy is a new curative treatment for sickle cell disease which uses the patient’s own bone marrow cells, negating the need for an unaffected donor.
She explained that gene therapy was the use of genetic material to prevent, treat and cure diseases, saying, If all components were manufactured locally, its cost would go down much further.
She therefore called on governments to adopt the treatment within the scale of its operating economy.
“The transformative potential of gene therapy comes from its targeting of underlying causes of disease, rather than treatment of symptoms.
“To date, no gene therapy clinical trial for sickle cell has been taken place in Africa where the majority of patients are,” Adair said.
She said the two days congress was necessary as Nigeria beared the largest proportion of patients living with sickle cell disease in the world.
She added that it was aimed at letting Nigerians, especially those warriors see the possibilities for curative therapies, such as gene therapy and also bone marrow transplant as possibilities for a better future for themselves.
She expressed optimism that the congress would address the global burden of sickle cell disease, while discussing gene therapy, bone marrow transplants, and other emerging treatments.
Also speaking, Dr Alexis Thompson, a Physician Scientist in the United States at the Children’s Hospital of Philadelphia, said some incremental steps had occurred in terms of improving outcomes for sickle cell disease.
Thompson said for the last five to ten years, they saw opportunity to apply science in a way that would allow patients to be their own donor.
“They will also conceivably have a long-term effect with controlling their sickle cell disease,” Thompson said, adding that the trials for both the approved products at her institution and some of the ones currently in clinical trial had been done.
“It is a field that we are far from over in terms of looking at innovations.
“But what is most extraordinary is the transformation in children and adult’s lives by giving the opportunity to undergo gene therapy and other curative treatments,” she said.
Mr Jimi Olaghere, a patient treated for sickle cell disease by gene therapy, said he had a comprehensive screening and care for sickle cell disease after enrolling in a gene therapy trail to find cure.
“Before I was born, my mom did not have access to prenatal screening and fortunately, she was able to fly to the U.S. and got that prenatal screening done.
“That prenatal screening confirmed that I would have sickle cell disease, so my mother decided to give birth to me there.
“I got fortunate to participate in a Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR-based) gene therapy that has completely changed my life 35 years after I was born.
“CRISPR-based gene therapy utilises CRISPR technology to precisely edit genes and correct genetic defects that cause diseases.
” So I am excited to come home, and show the community the impact of these gene therapies and just curative therapies across the board”, he said.(https://newsatlarge.ng)