Recent scientific progress could soon mean that haematological diseases may be curable, starting with certain blood cancers. Meanwhile, every blood donation counts.
Blood, plasma and platelet transfusions save millions of lives every year. These donations are used notably to satisfy urgent hospital needs, such as haemorrhages. They are also indispensable for the treatment of almost 150 haematological and oncological diseases, such as sickle cell anaemia and leukaemia, which represent one third of childhood cancers.
Characterised by a reduction in the quantity of red blood cells and/or an alteration in their quality, anaemia remains the most common blood disorder. According to the World Health Organisation (WHO), 1.62 billion people are affected, representing almost one quarter of the world’s population.
An unusual discovery illustrates the relative lack of understanding of this topic however. Published in The Lancet1, research undertaken in the US surprised the international scientific community. Septicaemia is a serious blood infection which appears to have caused some 11 million deaths in 2017. This estimation, which is twice the level accepted by specialists, would correspond to one death in every 5 worldwide.
Genetic therapies are a veritable medical and technological innovation which have recently transformed the way in which certain blood cancers are treated. Two drugs are currently prescribed in the treatment of acute lymphoblastic B cell leukaemia and diffuse large B cell lymphoma, two rare but particularly aggressive forms of blood disorder. Know more commonly as CAR-T cells, their use is recommended for patients failing to react to traditional methods in a relapse situation.
According to available clinical data, the 12-month survival rate is quadrupled or even more. Several complete remissions have been observed as early as the first month of treatment. The first patient treated in the US about ten years ago is now fully cured. As a reflection of their immense curative potential, their prices have climbed to several hundred million dollars... for a single injection.
Genetic therapy may also prove its worth in treating haemoglobin disorders which affect more than 330,000 births per year, mainly in Africa. Among the most advanced projects, Vertex Pharmaceuticals and CRISPR Therapeutics are conducting promising clinical trials in beta thalassemia and sickle cell anaemia. The technique used enables the genetic defect to be identified, dissected, corrected and the modification to be inserted using molecular scissors. Presented at the American Haematology Society 62nd annual congress, the latest published results2 display a significant clinical improvement which led to a reduction in transfusion dependence for the 7 thalassemia patients and the disappearance of vaso-occlusive crises for the 3 sickle cell anaemia patients. This genetic therapy using genome editing, which is soon to be tested on a cohort of 90 people, embodies an improved chance of a cure for millions of patients3.
Several issues at stake
Despite the progress made in research, no drugs can replace human blood. According to the WHO, 118.5 million blood donations are collected annually. This level is considered “highly unsatisfactory” to respond to the increasing demand, exacerbated by the ageing population and also by the limited lifespan of blood products. Platelets last for 7 days, while red blood cells can be kept for 42 days and plasma for 360 days.
In order to combat this shortage, certain countries readily remunerate blood donors, including Switzerland, Austria, Russia and China. This strategy can sometimes prove efficient, such as in Germany, where one third of adults regularly give blood. Every US inhabitant can donate blood plasma 104 times per year, generating estimated earnings of 3,120 dollars. According to the New York Times, this “market” was valued at 21 billion dollars in 2017.
The principle of remunerating blood donations is contested and does not enjoy unanimous support. Behind the ethical questions, there are also questions over the traceability and security of donations. Led by the WHO, major international bodies recommend systematic screening for HIV, hepatitis B and C, and syphilis prior to any transfusion. They prefer building up reserves from voluntary unpaid donators, a group in which blood transmitted infections tend to be less prevalent in general. To achieve this goal, they invite the relevant authorities to launch awareness campaigns among populations, along with local blood collections and recruitment drives.
The search for artificial blood
Japanese researchers claim to have found the ultimate solution, asserting that they have created artificial blood from red blood cells and platelets. Published in the American magazine Transfusion4, this discovery has several tangible advantages. The blood substitute is universal as it has been developed without antibodies or antigens. It can therefore be transfused to all patients, whatever their blood group. It can also be conserved at normal temperatures for over a year. The “prototype” which has been developed in a laboratory, has been tested on rabbits affected by a haemorrhaging disease. The results are relatively conclusive: 6 of the 10 animals tested survived the experiment, which is a comparable success rate to natural blood transfusions. No side effects or coagulation problems were identified. The efficiency of the procedure in humans remains to be demonstrated and the manufacturing process needs to be industrialised.
The French biotech company EryPharma is also confronted with both of these challenges in its quest to produce red blood cells from cultures. EryPharma’s promising technique would enable the group to develop the equivalent de 100 blood donations from one single stem cell sample. The company is confident and claims to have proof of a robust concept and hopes to begin mass production by the end of the decade.
Investors clearly play a major role in the development of technological, therapeutic and logistical innovation. Candriam will continue to actively support the fight against hematological diseases in all their forms. Through its network of experts, we target the most promising projects and solutions in the stock markets.
- 3.4% of deaths in the under-fives are associated with haemoglobin disorders;
- 79% of transfused patients in high revenue countries are over 60 years old;
- 42% of blood donations are collected in high revenue countries which represent less than 19% of the global population;
- 54% of transfusions carried out in low revenue countries are intended for children under 5 years old;
- 100% of blood supplies are provided by unpaid voluntary donors in 62 countries.
World Blood Donor Day: contributing to solidarity
The first World Blood Donor Day was held on 14 June 2004 and this event has been held every year for the past 26 years on the same date. The event is organised by the WHO and promoted by the Red Cross and Red Crescent international federations, the Blood Donor Organisations International Federation and the International Blood Transfusion Society. This major annual solidarity event is held to thank and encourage donors and to increase public awareness, and also to alert and mobilise governments around the world. This year’s edition is running under the universal slogan “Give blood and keep the world beating”. In addition to the traditional messages, this year’s campaign is focusing more particularly on the role played by young people in guaranteeing a secure blood supply, ideally through voluntary unpaid donations. This year, the event will be centred in Italy. Many different events will be held around the country, notably in Rome.
(1) “A global accounting of sepsis”, The Lancet (January 2020).
(2) “CRISPR-Cas9 gene editing for sickle cell disease and β-Thalassemia”, New England Journal of Medicine (January 2021).
(3) 4 drugs treating sickle cell anaemia are currently authorised in the US. According to the FDA, this hereditary genetic disease affects 100,000 Americans and more than 20 million people worldwide.
(4) “Combination therapy using fibrinogen γ-chain peptide-coated, ADP-encapsulated liposomes and haemoglobin vesicles for trauma-induced massive haemorrhage in thrombocytopenic rabbits”, Transfusion (July 2019).