The new gene editing treatments to treat thalassaemia cause the body to start making foetal haemoglobin, production of which normally ceases when a baby is a few months old. Getty Images
The new gene editing treatments to treat thalassaemia cause the body to start making foetal haemoglobin, production of which normally ceases when a baby is a few months old. Getty Images
The new gene editing treatments to treat thalassaemia cause the body to start making foetal haemoglobin, production of which normally ceases when a baby is a few months old. Getty Images
Prof Humaid Al Shamsi is the director of oncology services at Burjeel Holdings and president of the Emirates Cancer Society
December 23, 2022
The story of Alyssa, the cancer survivor who experienced a breakthrough from a new DNA-editing therapy, has inspired patients and doctors treating cancer worldwide. Alyssa, 13, has been battling acute lymphocytic leukaemia since May 2021. Although this cancer can affect both adults and children, cases in children are highly curable with chemotherapy. Still, unfortunately, several patients will have a recurrence and have low chances of survival due to the aggressive nature of this leukaemia.
While treatment options for this disease include bone marrow transplantation, in Alyssa's case, this also failed. When you reach this point, the treatment options are limited to palliative care that focuses on alleviating the symptoms of the patient who will eventually die from the disease. However, Alyssa and her family decided to try a new experimental treatment never used for such a case before.
They opted for the therapy that uses a new technology known as Clustered Regularly Interspaced Short Palindromic Repeats – commonly known as Crispr. This technology recognises the defect in the DNA. Then it repairs the codon, a sequence of three nucleotides forming a unit of genetic code in a DNA or RNA molecule, which controls or causes cancer. It can also modify the codon with the same technique as other cells so that they can fight against cancer. Many ongoing clinical trials are evaluating the efficacy of this gene-editing tool, which was introduced only about six years ago.
Researchers are studying how Crispr could be used to fight cancer. AP
Last year, a team of oncologists, including myself, published a scientific paper about this technique, as we believed it would have the potential to cure cancers in the future despite all the challenges the technique still has. In Alyssa's case, her T cells – usually a tool for the immune system to fight against invaders into the human body and protect from diseases – became the source of the cancer cells. These cancer cells started to attack her body and her normal cells. Despite chemotherapy and bone marrow transplant treatments, her T cells continued to grow out of control, causing her cancer to spread more aggressively. After her T cells were modified using the Crispr technique, the team at the Great Ormond Street Hospital in London infused them back into her body, unleashing a new army of healthy T cells that started to attack the cancerous T cells.
To the team's surprise, the newly engineered T cells were able to eliminate all the cancerous T cells. The next step was restoring her immune system through another bone marrow transplantation. Bone marrow transplantation carries a very high risk of infection-related complications, given that the body of a patient like Alyssa has no immunity against viruses or bacteria. Hence, she was isolated for almost three months from her brother, who was attending school, and was under stringent medical supervision to monitor her critical condition.
Alyssa eventually pulled through this difficult time and returned home with no signs of cancer and a fully functional immune system. Her journey was undoubtedly challenging and not easy, but she was brave enough to go through this experimental treatment without knowing its outcome. Alyssa's battle is not over, and she needs to continue to be under close follow-up as the risk of cancer recurrence is always there.
But her story is about the success of science and the courage of this brave teenager and her family. Our ongoing work to understand how cancer is growing and trying to target these pathways with novel therapies continues to advance as more breakthroughs are happening. Other promising technologies that are evolving, and used nowadays for cancer treatment, include blood-based DNA screening for cancer, such as the Galleri Test and CancerSEEK. Such tests have more public uptake and will make cancer screening easier by covering many cancers for which we previously didn't have screening. Another tool that we use in our clinic daily is measuring circulating tumour DNA, which helps detect cancer recurrence earlier than waiting for the cancer cells to grow and show on scans such as CT or MRI.
The Zayed Centre for Research into Rare Disease in Children at Great Ormond Street Hospital in London is helping to advance cancer treatment. Ministry of Presidential Affairs
Cancer, described for the first time in ancient Egypt more than 5,000 years ago, remains a significant health challenge and continues to be a global cause of death in both developing and developed countries. As we advance our tools to treat cancer, we must ensure that all patients from different backgrounds and economic statuses have access to treatment. Alyssa's story marks the beginning of a new era of gene-editing to try to cure cancer. Her story also highlights how important it is for patients with cancer to accept being treated in clinical trials. The participation and courage of patients and their families to confront uncertainties are commendable.
Precision medicine, through which patients are treated based on the genetic make-up of their disease, is becoming a new standard for a number of diseases, including cancer. Many approved drugs target specific mutations, but Alyssa's is the first successful case of human gene-editing for acute lymphocytic leukaemia.
There are also some setbacks with gene-editing, like off-targets wherein unintended modification of normal genes could cause side effects. Also, the effect of gene-editing might not be permanent and could be reversed, leading to the cancer cells getting active again. Such uncertainties are why more research and longer follow-ups for these cases after the gene-editing procedure are needed.
Ultimately, Alyssa's story sheds light on a new era of cancer research and potential cure. We are fortunate to witness such progress, and we are optimistic about more success stories using a number of new technologies at our disposal today, including Crispr.
Ten tax points to be aware of in 2026
1. Domestic VAT refund amendments: request your refund within five years
If a business does not apply for the refund on time, they lose their credit.
2. E-invoicing in the UAE
Businesses should continue preparing for the implementation of e-invoicing in the UAE, with 2026 a preparation and transition period ahead of phased mandatory adoption.
3. More tax audits
Tax authorities are increasingly using data already available across multiple filings to identify audit risks.
4. More beneficial VAT and excise tax penalty regime
Tax disputes are expected to become more frequent and more structured, with clearer administrative objection and appeal processes. The UAE has adopted a new penalty regime for VAT and excise disputes, which now mirrors the penalty regime for corporate tax.
5. Greater emphasis on statutory audit
There is a greater need for the accuracy of financial statements. The International Financial Reporting Standards standards need to be strictly adhered to and, as a result, the quality of the audits will need to increase.
6. Further transfer pricing enforcement
Transfer pricing enforcement, which refers to the practice of establishing prices for internal transactions between related entities, is expected to broaden in scope. The UAE will shortly open the possibility to negotiate advance pricing agreements, or essentially rulings for transfer pricing purposes.
7. Limited time periods for audits
Recent amendments also introduce a default five-year limitation period for tax audits and assessments, subject to specific statutory exceptions. While the standard audit and assessment period is five years, this may be extended to up to 15 years in cases involving fraud or tax evasion.
8. Pillar 2 implementation
Many multinational groups will begin to feel the practical effect of the Domestic Minimum Top-Up Tax (DMTT), the UAE's implementation of the OECD’s global minimum tax under Pillar 2. While the rules apply for financial years starting on or after January 1, 2025, it is 2026 that marks the transition to an operational phase.
9. Reduced compliance obligations for imported goods and services
Businesses that apply the reverse-charge mechanism for VAT purposes in the UAE may benefit from reduced compliance obligations.
10. Substance and CbC reporting focus
Tax authorities are expected to continue strengthening the enforcement of economic substance and Country-by-Country (CbC) reporting frameworks. In the UAE, these regimes are increasingly being used as risk-assessment tools, providing tax authorities with a comprehensive view of multinational groups’ global footprints and enabling them to assess whether profits are aligned with real economic activity.
Contributed by Thomas Vanhee and Hend Rashwan, Aurifer
Investors: Accel, Oaktree Capital (US); Chiratae Ventures, Epiq Capital, Innoven Capital, Kalaari Capital, Kotak Mahindra Bank, Piramal Group’s Anand Piramal, Pratithi Investment Trust, Ratan Tata (India); and Unilever Ventures (Unilever’s global venture capital arm)
The burning issue
The internal combustion engine is facing a watershed moment – major manufacturer Volvo is to stop producing petroleum-powered vehicles by 2021 and countries in Europe, including the UK, have vowed to ban their sale before 2040. The National takes a look at the story of one of the most successful technologies of the last 100 years and how it has impacted life in the UAE.
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