New Hope for Thalassemia Sufferers

The blood disorder, Thalassemia is very prevalent among the Kadazandusuns.

Last month I donated blood in support of a 30+ year old thalassemic girl who lives just 3 houses next to mine. She has had blood transfusions since small but since she had bone marrow transplant she had the joy of enjoying her thirties age. Hopefully she will enjoy many more, especially at this time when the promise of new technologies offers brighter hopes for a better cure.

In a 2008 study headed by a researcher from the Faculty of Medicine, University of Malaya, from 125 blood samples obtained from unrelated Kadazandusuns, α- and β-thalassemia were confirmed at 33.6% and 12.8%. In another study it was found that from all the ethnic groups studied in Sabah, Kadazandusuns showed the highest prevalence for this disease.

Thalassemia is an inherited blood disorder in which the body makes an abnormal form of hemoglobin, the protein molecule found in red blood cells. The disorder results in excessive destruction of red blood cells, which leads to anemia. It is caused by either a genetic mutation or a deletion of certain key gene fragments. There are two main forms of thalassemia which are serious: In alpha thalassemia, at least one of the alpha globin genes has a mutation or abnormality, and in beta thalassemia, the beta globin genes are affected.

People with thalassemia can get sick from an overload of iron in their bodies, either from the disease itself or from frequent blood transfusions. Young children, even with blood transfusions, are not expected to reach beyond their teen years because of this disease. Bone marrow transplantation—or in more detail, the blood-making stem cells (hematopoietic stem cells) found in bone marrow, but more of this later)?—may offer the only possibility (so far) of a cure in young people who have an HLA-matched donor. Success rates have been in the 80–90% range.

If you recalled, the root cause of thalassemia is the inability of the body to make either one of two proteins that makes haemoglobin, and that deficiency arose because the sufferer’s crucial deletion or mutation in his/her genome (genes that codes for the making of the protein(s). But, what if somehow those missing/bad genes can be corrected? What if it is possible, much like a software programmer can edit his code, to shift though the 3 billion plus of DNA code pairs in the human genome, zero in on the flawed part, delete it, and replace with the right and healthy one?

Enter CRISPR (Clustered regularly interspaced short palindromic repeats) technology, a new method of targeted gene modification discovered not more than 5 years ago. More specifically known as CRISPR/Cas9, this technology makes it possible to do genome editing – the precise and targeted modification of the genetic material of cells. Genome editing works by using an enzyme to make a cut at a particular sequence in the genome, followed by the deletion, repair or insertion of genetic material at the cut site.

Can CRISPR genome editing pave the way for a better and safer cure for thalassemia? Sci-fi wishful thinking? Think again.

Today (2nd August in the U.S.), it was reported in the Washington Post that scientists have successfully undertaken gene editing in the first human embryo CRISPR experiment in U.S. to corrects gene for an inherited heart disease, hypertrophic cardiomyopathy. This method of genome editing and repair has already been found to work in a mouse with the β-thalassemia defective gene, with “sustained elevation of blood haemoglobin levels into the normal range, reduced reticulocytosis, [and] reversal of splenomegaly “. In 2015 a team of Chinese scientists, using the same technology, reported that their success rate was one in four for β-thalassemia genetically abnormal human embryos although they did raised their concerns about off-target effects. Just last march, Intellia Therapuetics, a company dedicated to developing potentially curative genome editing treatments, together with healthcare global giant, Novartis, have announced that their hematopoietic stem cell transplant (read “thalassemia” here) program is planned to undertake the first human clinical study next year. A different company, CRISPR Therapeutic is also targeting human clinical trials for β-thalassemia treatments with about the same timeline.

Here’s praying for my neighbour. And for my cousin-in-law, who is a carrier of the thalassemia gene.

No New Shoes or No Legs

We rant. We moan. We bellyache.

Usually about inconsequential things: The 36-Channel Astro with nothing good to see; the clowns at Puzzle Palaces; the minimum-wage waitress who delivered the wrong food; the pesky mosquitoes; the frying pan that got scratched; the weather; the ugly driver who cut you off from the wrong side…the list goes own. Most of the time we complain at things we do not have control of, and we whine because whining is about whining, and anyway, it is of no consequence.

We cry because we have no new shoes…until we meet a person with no legs. Let me tell you about some people I know with “no legs”.

Just yesterday I went to a funeral of a girl whose mum and dad I knew well since young. The girl was born healthy, developed a fever (and meningitis probably) within a few days and her health deteriorated. Her doctors could not bring her health back and she became a severely handicapped baby. She died 34 years later four days ago. I can’t imagine the sadness and anguish, and the supreme efforts of both parents in taking in care of their child—bed ridden, unable to feed herself, and mentally handicapped—for 34 years.

Just 2 days ago, an old friend was traveling from Semporna to Lahad Datu as a front seat passenger when they had an accident. In an instant their Hilux Vigo lost the road, flipped and went wheels up among some oil palms. Thankfully he and the driver were not hurt—just some dented emotions. The Vigo was another story all together.

Life can cut off the legs from under you in the heartbeat moment that a moth flutters its wings.

Last week I went to visit another old friend at KPG Medical Center. He was admitted because of heart problems. Later I found from the daughter that his heart had already been zapped 3 times as the doctors tried to tame his really bad tachycardia. He was literally a heartbeat away from death.

As I finished my greetings, he looked up at me with regretful eyes and said, “I know. You had told me many times.” Indeed I did. He was referring to the numerous times I pestered him to try drag him to see my doctor about his hypertension.

There is no satisfaction in being right. Who wants to be right in a situation like this? I don’t even know I am right; maybe it is all one big nebulous genetic sweepstake. Some have teflon-coated arteries; some don’t. I am happy to know that after going to IJN, he had a defibrillator installed, and stents done, and he is on the road to recovery.

They say, to a bacteria a pin’s head is an Everest to climb. Perhaps rather than whining that the mountain should be lower, we should grow taller instead. I know I would like to. Maybe we should look more inwards, look for a place we find some solace, and apply a speed throttle to that dissatisfaction engine. If every thing fails, let us heed baseball great, Yogi Bera’s entreaty to “take it with a grin of salt.”

God bless.

Book: Anatomy of Terror by Ali Soufan

In my comments about Joby Warrick’s book, Black Flags: The Rise of ISIS, a 2016 Pulitzer winner for general non-fiction, I said this:

Good but incomplete/one-sided. This is told mostly from the perspective of those (mostly Western powers, and at that, mostly American) who oppose and fought (and still fighting) Zarqawi and later ISIS. If you want to talk about the rise of ISIS at least you attempt to explain or go into the antecedents and causes why, and motivations of thousands of men (and women) from all over the world (including from Malaysia) who went (and is still going) to Syria and Iraq to take up the cause and put themselves in harm’s and death’s way.

In “Anatomy of Terror,” author and former F.B.I. special agent Ali Soufan wrote this in his introduction:

“The key to a more constructive use of our imaginations is empathy – not in the colloquial sense of sharing another person’s perspective, but in the clinical sense of being able to see the world through another person’s eyes. Sadly, after fifteen years of the war on terrorism, we still do not really know our enemy in this deeper sense. In this book, by delving into the personalities of men who mean us harm, I aim not to create sympathy for them – far from it – but to help point the way to a deeper understanding of their worldview, their motivations, and how best to combat the destructive ideology they represent.”

To a certain extent Soufan has achieved his goal. Disappointingly though he has written only a short last chapter on the “how best to combat the destructive ideology they represent.” Even this is skewed towards a Western perspective. It is noteworthy to point out that thousands and thousands more Muslims (compared to Western victims) have died and suffered at and through the hands of ISIS and Al-Qaeda, and their many offshoots in Muslim countries like Iraq, Yemen, Syria, Pakistan and Afghanistan where they command actual real estate to operate and proliferate.

On this, I think Donald Trump, during his recent speech in Riyadh, touched on a very pertinent and important point when he said:

“It is a choice between two futures. And it is a choice America can not make for you. A better future is only possible if your nations drive out the terrorists and drive out the extremists…Drive them out. Drive them out of your places of worship. Drive them out of your communities. Drive them out of your holy lands. Drive them out of this earth.”

The Opposition’s Fight Club

If you recall the 1999 movie, The Fight Club—a cult Hollywood offering starring Brad Pitt and Ed Norton—the first rule was, “Don’t talk about the Fight Club.” The second rule was, “Don’t talk about the Fight Club.” Sad to say but Sabah’s opposition are (again) running their antithetical version of this movie. Constantly and incessantly they are lobbing strident vocal grenades at…wait for it, each other. Griping, ranting, sniping, jockeying and accusing each other of being not worthy of the rakyat’s support. Makes you wonder why they are so excited about the coming PRU, and who they actually want to unseat. Hey! look over there, the unblinking lizards of the Incumbents.

And how they extolled themselves: how good they are, and how clever they are that they can decide for us, little people. There is a recent article in this news site where a party member was waxing lyrical about the prime credentials and high schooling of his three deputy party presidents. He disparaged the people who had the temerity to not support them and question their “intellectual integrity”, calling them the failures, the poor and the uneducated—the “nincompoops”. This is exactly the kind of discombobulated and tone-deaf, and elitist thinking that got us into this present deep morass. This is exactly the “We-are-so-intellectually-superior-so-we-know-what’s-good-for-you” attitude that voters hate. Well may he heed the words of a late eminent politician who said, “Everyone…is three individuals. What you think you are. What you are. And what others think you are.”

If there is one thing that we can learn from Donald Trump’s seemingly implausible ascendancy to the U.S. presidency, it is this: He knew how to talk about the people’s actual concerns. He talked for Middle America—”the forgotten men and women”—about economic atrophy, loss of jobs, security and illegal immigration, terrorism, and law and order. He railed against the influence-peddling moneyed lot, the entrenched but out-of-touch politicians, and about the self-serving and corruption of the ruling elites in the swamps of Washington DC.

Well, we are the many forgotten lot of Sabah. Too many, I regret to say. We do not want to be talked down to; we want our leaders instead, to talk (and act) for us. Instead of hearing about the uncomely warts and pimples of your fellow (but different party) opposition wannabes, we want to hear how you are going to intelligently allocate and maximise the impact of the annual RM4 billion or so state budget. How you are going to help our unemployed youngsters, many with degrees but who are jobless, work at McDonald or daily-paid. How government can help working mums and dads. How to keep us Malaysians safe from the hundreds of thousands of illegal immigrants in Sabah. How to improve KK City Hall and the town and districts councils which are frankly short-budgeted every year. How to leverage the resources of Sabah for the benefit of the majority of the Rakyat, and not for a few individuals or large corporations only. How to positively engage the Federal Government (Yes, in a federation, Putrajaya is not going to close shop anytime soon) for the betterment of Sabah in a healthy, mutually beneficial state-federal relationship. How to arc a 20-year trajectory of our state’s development to make everyone prosper and benefit. I know that is a lot to ask, but I rather hear about these than the endless internecine sniping.

Show us a path.

Someone said that during election time, the most dangerous place is between a politician and a microphone. Personally I would not hold it against a politician if he/she wants to grab that mic, but really, talk for us—for our rights, our future, and our prosperity.

Cats!

cats

Reading this. Too long-winded, I say. You know why we like cats (and dogs)? They are the most expressive when carefree, happy and playful. It reminds us—no, it forces us—to think that in life, basically that is just all what matters. No?

Come on, I dare you. There was a time when you did not have: screaming credit cards to pay, cranky spouse, mortgages, bosses-from-hell, kids-to-wake-up-early-to-send-to-school, bills to pay! leaky roofs, and tomorrow horrendous jams to negotiate to work…yeah, that cute kitten happily sleeping near the sofa doesn’t have to put with all that shit 🙂

Pink Chick Story (There is sex here but it’s not what you think)

coloredchicks
I once bought my son a coloured chick. You shouldn’t.

Many years ago when my 3 kids were young I brought them to the Farmers’ Day Celebration at Tanjung Lipat, Kota Kinabalu. My young son, Darrien, saw the coloured chicks for sale, and you know, those are irresistible to children. And so I bought one—a pink one.

It became his baby. It was kept in a box inside the house and which he diligently took care and fed. That was the start of the house woes of course. The endless cheeping drove everybody crazy. The chick would be quiet and happy when it could see one human being, but alone, it will soon start its infernal cheeping motor – cheep, cheeeep, CHEEEEEP! Of course the sales hook—the pretty pink fluff—disappeared in 2 weeks, leaving a regular chick. And an older chick that refused to be boxed in or go to sleep without hearing people around. And so it followed us around the house – cheep, cheeeep, CHEEEEEP!

In no time it grew to a size when crapping all over the floor all day long was even too much for the poor kid to go around the house cleaning after. And so it was exiled outside the house but still a well-taken-care-off pet. Still, it thought it was the youngest sibling in the house, always peering into the house trying to get in.

Well, the chick grew…and grew.

rooster

And it turned out to be a rooster. A strapping, beautiful white rooster. A strapping, beautiful white and HORNY rooster who thought human beings are its species, and who he have the hots for. This is no surprise as he grew up knowing only people I guess. And so every day he will chase us trying to hump our legs. Marion, my daugher, was always screaming, “Daddy! Darrien’s chicken is chasing me again!” My aunty who lived next door hollered every other day, “Manuk nu Rayn!” I would come out from the car after work, and there he was with obvious lust in his eyes, wings down and dancing sidesteps trying to telegraph sex to me.

My brother, who lives next door, had many chickens. I tried a few times to throw some hens in the rooster’s direction. He looked at them with disdain and disinterest, and then threw me a look with lascivious longing. I could almost see it put out it metaphorical tongue. Bleeeh!

My late mum (God bless her; she passed away last Dec, 16th) took the brunt of the rooster’s amorous, although misplaced, moves. At least we all, having to go to work and school, can escape its intentions for most of the day. Not my mum: she would be putting out laundry to dry on the outside clothesline and the rooster will come running from some bushes (Yeah, it had learned since to hide from my mum and ambushed her when she was not looking), pecked her legs and tried to hump. Then my mum would yell loudly, “Darrien! Manuk nu arat tomod! Mogu’rub!” (“Darrien! your chicken is so bad! It is pecking me!”). I had to explain to my mum that the chicken was not being nasty, and that it was just trying to mate with her.

This went on for weeks until mum, at the end of her long-suffering patience, put out a fatwa, “Kill the chicken. Look at my legs. They are black and blue!” All of us siblings looked at each other. Someone said, “Who dare to tell Darrien? Who dare to do the execution?” Of course no one had the guts!

Of course a fatwa is a fatwa. And so I took Darrien aside and asked him three times if it is ok to get rid of his chicken. Come on, this is serious stuff: you don’t kill a Primary 2 kid’s only pet chicken. He could be scarred for life and turn into a sociopath. Fortunately he said yes. Of course I chickened out (pardon the pun) from actually killing the rooster. And so I called my brother-in-law to get the chicken. Weeks later, he reported that it made for tasty curry chicken.

I did not dare to tell Darrien.

Curry Lamb

Last night in preparing the Coconut Battered Ikan Bakulan I saved the santan. Instead of throwing the perfectly good natural organic santan (yeah, much better than the supermarket package kind) I used it to cook lamb curry, yeah :-). Nothing special here – I mixed 2/3 kari daging with 1/3 curry ikan. Ignore the greens too; just to make me feel less guilty – ada makan sayur! Taste like ox tail curry ah 🙂

16463290_1728781187147350_5905871409516937127_o