27 July 2015
ByAppeared in BioNews 812
BBC2, Tuesday 14 July 2015
This series follows the staff and families of children who are receiving treatment at Great Ormond Street Hospital, and the first episode focuses on their world-leading team dealing with congenital diseases.
We meet three families, all facing rare genetic immune diseases for which Great Ormond Street has been pioneering new treatments. The documentary witnesses the utter anguish the families go through and the heartbreaking decisions parents have to make. The documentary has a warning that it 'contains upsetting scenes' but this is an understatement; it is truly a harrowing series of stories.
Herb, aged five, suffers from a rare immune disease called NEMO, and is under the care of Professor Paul Veys. Herb has to have a bone-marrow transplant from his older brother Rufus, who is fortunately a match. However, the treatment comes with a ten percent chance of mortality. This is something Herb's mum finds difficult to read in a letter from the hospital. 'That is just a statistic. My son is not a statistic,' she says.
Herb has the treatment using Rufus's bone marrow. Previously, a happy and spirited child, Herb becomes moody and depressed and must be kept in isolation for several weeks. He gets very upset when he loses his hair as a result of the chemotherapy, which is used to wipe out his immune system before the transplant. Another consequence of this, which causes greater concern for his parents, is that Herb must be made sicker before he can be made better again. And, if the transplant fails, he will have no immune system, leaving him defenceless to infection.
When he develops a fever after the transplant, there is real worry that Herb has an infection, which would be extremely dangerous. However, it could also be a sign that the transfusion has worked. We, and his parents, must wait to find out which of these outcomes it is.
We also meet Keano, who is nine years' old and has congenital neutropenia. Doctors are struggling to find a bone-marrow donor for him because of his rare mixed-race background – his mother is of mixed Indian descent and his father of mixed African–Caribbean descent.
Keano has the option of a transplant from his mother, who is a mismatched donor and only 50 percent compatible. And, with the high odds of failure – at up to 30 percent – she faces a painful decision: for the chance to give Keano a future, she must run the risk of losing him now.
After agonising over what to do, Keano's mum decides to proceed with the transplant. However, when Keano's body rejects the transfusion, he is left dangerously ill.
Staff work to get another donor from the USA in a record time of two weeks. But Keano then develops a fungal infection that causes sores all over his body, and he is clearly in a lot of pain. Dr Kanchan Rao takes over his care, but she is doubtful that he can recover and says that it will be a miracle if he survives.
Finally, the show follows Teigan, 10, who suffers from SCID (Severe Combined Immunodeficiency). Seven years ago she was one of the first patients to undergo gene therapy, but the chemotherapy caused serious side effects so was halted. She has now been on weekly injections to increase her white-cell count, but these are painful and not a long-term solution. Her family and doctors have made the decision to try gene therapy again, which has not been given to a child of Teigan's age before, now that less aggressive chemotherapy is available.
While the documentary shows how hard it is for patients to go through these treatments, through Teigan we see a different side to the story: the disappointment when treatment cannot go ahead. Difficulties in getting enough stem cells from Teigan's bone marrow mean Dr Bobby Gaspar decides the treatment needs to be delayed as the risk is too high. She and her family are disconsolate as they must return home to Scotland and wait to attempt a cure again.
As well as portraying the lives of the families involved, during the documentary we also come to understand the motivations of the staff and why they are willing to take the sort of risks that many other doctors would be unwilling to. Professor Veys says that the thing he always regrets is taking treatment too far and causing too much pain. However, he says, once in a while there is one child who defies the odds and comes through, which spurs them to continue to take chances in the face of adversity.
Although the show highlighted the need for more bone-marrow donors, particularly for people with mixed-raced origins, this point could have been made more strongly. The need to go to USA to get a donor for Keano shows just how difficult it can be to find donors for people of mixed-race origins.
The show is an emotional journey, and you would have to be heartless not feel the pain and anguish the families go through. Although the documentary is essentially a big advert for Great Ormond Street Hospital, after the first 15 minutes you don't mind and are simply in awe of the strength of the patients, families and staff.
I challenge anyone to watch the documentary and not want to support the work of Great Ormond Street.