India’s Medical Milestone: Gene Therapy Success for Haemophilia A

Indian scientists achieve a breakthrough in gene therapy for Haemophilia A, offering lasting relief to patients.

Highlights:

Integrated gene therapy offers long-term relief for Haemophilia A
Lentiviral vectors ensure safer treatment without immunosuppressants
Clinical trial success boosts India’s global gene therapy capabilities

Innovations in gene therapy are rewriting the future of rare disease treatment. In a recent breakthrough, integrated gene therapy has recently been successfully accomplished by a team of Indian scientists for Haemophilia A, a severe and rare blood clotting disorder. In a clinical trial conducted in Tamil Nadu by the Centre for Stem Cell Research (CSCR) at Christian Medical College (CMC), Vellore, five patients experienced no bleeding episodes for over 14 months after receiving this treatment ().

One-Time Treatment Offering Lasting Relief from Haemophilia A

Current approach to haemophilia treatment includes repeated injections of clotting factor VIII, which is expensive and time-consuming. Gene therapy however, provides a population-stable cure as compared with the limited life span of viral vectors. As this therapy involves the use of a functional gene wherein the lack or lower level of clotting factor results in bleeding episodes, the introduction of the gene helps in production of the clotting factor and sometimes eradicates these episodes.

Hemophilia A Patients With Inhibitors Benefit from Emicizumab
Hemophilia A patients experience traumatic bleeding. Emicizumab is an antibody that prevents bleeding and requires less frequent dosing.

‘Did You Know?
India ranks second in Haemophilia A prevalence, yet groundbreaking, gene therapy now offers lasting relief. #genetherapy #medindia’

Though it is estimated that just 1 in 10,000 people is a haemophilia https://www.medindia.net/news/hemophilia-may-now-be-treated-for-life-with-one-single-injection-179161-1.htm patient worldwide, India comes out second in haemophilia prevalence with 40,000-1,00,000 patients. Severe Haemophilia A that falls below 1% clotting factor VIII means constant medical attention and treatment; that is the rationale behind developing a gene therapy.

Innovative Gene Delivery Method

Delivering the targeted genes to the core of the cell at the right time is still one of the main challenges of gene therapy.

As opposed to the US-FDA approved commercial gene therapy, Roctavian which uses an adenoviral vector, the CMC trial used a lentiviral vector with stem cell incorporation. This method is regarded as safer because it does not require use of immunosuppressive drugs which would make the treatment accessible to children for example.

Novel Drug Brings New Hope for Hemophilia A
Investigational drug emicizumab shows promise in children with bleeding disorder, hemophilia A.

The made development has been referred to as “revolutionary” by experts in the field. In an article published in the New England Journal of Medicine (NEJM), Dr. Johny Mahlangu underscored that this success unveils India’s ability to do complex gene therapy trial. He also pointed to the possibility of cutting the treatment expenses and increasing its availability to patients around the world.

As research continues and there is possibility of localized production for this gene therapy, there is hope that patients with haemophilia will get a permanent cure and rely on other medical mechanisms in the event that the gene therapy fails to work permanently for them.

Gene Therapy Could Possibly Cure Hemophilia A!
Gene therapy may restore the low factor VIII levels in hemophilia A patients, thereby eliminating the painful bleeding episodes and the need for repeated injections.

Reference:

Hemophilia A Gene Therapy - Some Answers, More Questions - (https://www.nejm.org/doi/full/10.1056/NEJMe2212347)

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