Hope At Last! First Patient with Sickle Cell Disease Treated in Paris


Keith Haring Necker Chidren’s Hospital,Paris



Imagine Institute


News published on 2 March 2017, in New England Journal of Medicine states that remission of the signs of sickle cell disease of the first patient in the world was recorded after being treated with gene therapy. The treatment was undertaken for a French teenager with sickle cell disease which is a group of inherited blood disorders with abnormal hemoglobin in red blood cells that causes blood to clog in tiny vessels and organs.

The team from Necker Children’s Hospital, AP-HP and Imagine Institute (AP-HP/Inserm/Université Paris Descartes) led by Marina Cavazzana conducted the gene therapy from October 2014 with a phase I clinical trial/II in the 13 year old patient with severe sickle cell disease. This disease affects known segments in the world – Africa, Middle East, Caribbean and among Asian origin people. Worldwide 275,000 babies are born with it every year. In India tribal populations are largely afflicted with the disease.

The results of this life-long disease remain complex and varied. Patients suffer from bouts of severe anemia due to the inefficient transport of oxygen. They are also at higher risk of contracting major infections and can suffer from occasional, extremely painful spasms known as sickle cell crises. Stunted growth and strokes are also symptoms in some cases.

Conventional medicine does not cure sickle cell disease. Till date the only way to attempt to cure it has been to use bone marrow or stem cell transplants from a healthy donor to allow the patient’s body to produce healthy blood cells. Although this works but risks of dangerous immune response (transplanted cells begin attacking patient’s normal cells) is too high.

Now we see this case of a teenager in the Paris hospital since 2014 who underwent the unique new therapy. A specific gene that induces “anti-sickling” effect on red blood cells was inserted into the patient’s own stem cells using a modified virus. The cells were then allowed to transform into red blood cells and they began to proliferate in the patient’s body. Fifteen months later the boy, now 15, is not suffering from any of the effects of sickle cell disease.

It appears that at least 50 percent of his blood cells have normal hemoglobin and are normally shaped. Although this isn’t a cure for the disease, the treatment has proven effective as the symptoms have been quashed and he is off all other forms of medication and treatment. “Since therapy was applied, he hasn’t had any pain, any complications. He is free of any transfusions. He plays sports and goes to school,” said Dr. Philippe Leboulch, an author of the new research and a professor of medicine at the University of Paris. “So we are quite pleased with the results.”

The gene therapy was invented by scientists at Bluebird Bio based out of Cambridge, Massachusetts. They’ve treated six other patients with sickle cell disease in the same way, and although the results have yet to be published, it appears that it has not worked as successfully in all of them just yet. However, trials are still taking place, and the future is looking increasingly bright for those with this unfortunate condition.

And now there is hope for patients!


Stay Involved with Life and Living

Connect with Nature

Connect with Nature

The best among us are subject to depression. There are times you feel drained of all hope, drive and energy. Life appears difficult in the face of the storm you may be facing.  But while severe neurosis will have to be taken care of by experts, if you are simply feeling low or find yourself in the grips of fleeting melancholia then you can do things on your own to improve the situation. Some circumstances will of course take their time in resolving themselves but for bouts of depression that affect us all from time to time, you can work on snapping out of it.

With due diligence and persistence you can even wean yourself out of stubborn depression. Start by taking baby steps to feel better. And in no time you will be able to get yourself out of the woods. You can best do this by making positive changes around yourself, everyday. The road to recovery needs to be action oriented. It is but this fact of taking action is what you must be focused on. Set yourself in the frame of mind to do all the things you chalk out or yourself.

You should start by making small changes and adjustments for your psyche to regain its balance. If you like to schedule action points start with daily walks. Nothing is more invigorating than brisk walking. Besides the exercise doing you a world of good, it becomes the reason to step out of the confines of the indoors that seem to tie you down. Stay focused on your plan and do it with regularity. You should miss the activity if you happen to miss out any day. Getting out and enjoying the outdoors restores your sense of well being.

Next you could begin to look for likeminded people to befriend. Cultivate supportive relationships that will make a huge difference to your state of mind. Enjoy small exchanges and interactions – having tea, going for a movie or conversations. These will help you to maintain perspective over situations as well as keep you from being isolated. It is essential not to be lonely as being alone definitely increases depressive states. It is therefore extremely important to enjoy social relationships and activities that will keep you engaged.

Turn to loyal friends or trusted family members who want your betterment. You should share with them whatever is affecting you and help you to face up to things as they are. Sometimes all you need are people who will just listen to what you have to say. Having people you trust in your life keeps you from withdrawing into yourself. Also good company keeps you away from being down in the dumps. In extreme cases people have joined support groups, where often it is far easier to talk to relative strangers.

It helps to talk to people about your feelings. So never miss an opportunity to have coffee, lunch, movies, concerts, parties with friends. Seek out loved ones.  Be thankful they check on you with such regularity. Stay in touch, make calls, write letters, go back to old friends and meet new people too. Extend your social circle and make no bones about enjoying it all. Keep challenging all negativity around you. Negativity puts a spin on things so make sure you enhance positivity around yourself.

An important aspect of self help is taking good care of yourself. Do all the things that keep you happy. Have a healthy lifestyle. It is half the battle won. It automatically puts you on a get well mode and you manage stress better.  Eat well, sleep well and live well. Get good sleep. Stick to a tidy schedule. Enjoy your favorite show on television. Enjoy music. Enjoy sunshine daily. Sunlight offers a very positive impact. Enjoy eating out on the patio. All these small things will help you to feel better, naturally! Last but not the least – Pray. Enjoy your conversations with God – the most powerful therapist of all times.

Sunlight - Powerful Antidote

Sunlight – Powerful Antidote