French doctors offer hope to people with sickle cell disease with a successful DNA reversal treatment on a teenager – Ventures Africa

Thanks to the constant advancement of Science, a cure may have been discovered for sickle cell disease. Doctors in Paris have monitored and confirmed the success of a DNA reversal treatment carried out on a teenager 15 months ago. Since the disease is such that the bone marrow produces deformed red blood cells, scientists altered the genetic instructions in his bone marrow, so it produced healthy red blood cells.

James Gallagher for BBC reports that the teenager had his bone marrow taken out, stem cells harvested and genetically altered with a virus to infect it with correct instructions. Next, he underwent chemotherapy for four days to eliminate his diseased stem cells, before the corrected bone marrow was replanted. Since then, the teenager has received a clean bill of health. So far the patient has no sign of the disease, no pain, no hospitalisation We are quite pleased with that, Philippe Leboulch, a professor of medicine at the University of Paris, told the BBC.

Prior to the surgery, the condition of the teenager is said to have been critical. So much that he needed to have his spleen removed and his hips replaced. He also used to have a monthly blood transfusion to dilute his defective blood. But now, he no longer requires a transfusion.

Although Professor Leboulch is hesitant about asserting the treatment as a cure for sickle cell anaemia, the success of the pilot case is a significant milestone in the treatment of the disease. According to the professor, there needs to be more performance of this therapy on several patients to create a certainty that it is robust enough to propose it as a mainstream therapy.

However, just as the bone marrow transplant, the only known cure for sickle cell anaemia is expensive, this pioneering treatment is quite costly and can only be performed in highly advanced hospitals. This already poses a huge challenge to the African continent where the disease is predominant, as the cost implications are bound to widen between developed and developing countries, largely due to exchange rates.

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French doctors offer hope to people with sickle cell disease with a successful DNA reversal treatment on a teenager - Ventures Africa

This University of Nevada Student Proved Doctors Wrong About MS – Study Breaks

Multiple sclerosis left Alexa Silvers legally disabled at sixteen, but an unpublicized treatment option completely cured her.

By Tylah Silva, Emerson College

Alexa Silvers is a college student, survivor and an all-around amazing human being.

The biggest obstacles most of us had to face at sixteen were hormonal acne and a bad breakup. Silvers had to deal with multiple sclerosis. The autoimmune disease known as M.S. attacked her legs and put her in wheelchair. The doctors told Silvers that treatments could help, but that ultimately her condition was permanent.

They were wrong.

Through it all, Silvers kept a faithful mind, putting her efforts into her treatment and researching a cure. Then, she stumbled upon stem cell research. With the help of her mother, many charitable donators and a staff of skillful doctors, Silvers was cured after having M.S. for four years.

Now, this University of Nevada at Reno student is making an effort to get the word out on stem cell treatments.

Tylah Silva: What was it like being diagnosed with M.S. at 16?

Alexa Silvers: It was completely life changing. I suddenly felt like an outcast, and felt like I was dealing with an issue that even my closest friends didnt completely get. I kept it a secret as best I could, but as my condition worsened, it became harder and harder to hide.

UN student Alexa Silvers

TS: Had you ever heard about M.S. before you were diagnosed?

AS: I had never heard of it until I started experiencing weird symptoms. Every Google search led me to undeniable M.S. When I brought the possibility up to my physician, she actually laughed and told me it was impossible for someone my age to have a disease like M.S.

TS: Were you frightened by your diagnosis?

AS: I was terrified. The scariest part of multiple sclerosis is its unpredictability. I was already legally disabled at 16, and I had no idea if I would wake up one day not able to walk suddenly. It was a constant struggle, countless failed medications, neurologists telling me that patients with images like mine should already be in a wheelchair, and that every day I wasnt I should be cherishing.

TS: What methods did you use to cope with your ailment?

AS: I am very strong in my faith. I notice that some people, when faced with adversity, tend to adopt a victim mentality and distance themselves from church and all support, but I made a conscious choice to do the opposite. I surrounded myself with people who helped me stay hopeful even when things werent looking great.

TS: How did your family and friends react?

AS: My family and closest friends were amazing, I did not want to be treated differently, and they were really great about making sure things still felt normal. Although, I think a lot of people didnt really understand, and to this day still dont. It was a lot of but you look fine.

TS: How did you stumble upon the stem cell research?

AS: I was running out of options as far as common M.S. treatments go. I had just relapsed on my third therapy, and decided there had to be a better option than a medication that only slows disability progression down 40 percent of the time. I watched M.S. news articles closely, and one day came across an article on a stem cell treatment that was successfully halting M.S. in England.

I immediately began searching on clinicaltrials.gov for a similar study in the U.S. I found one that Dr. Richard Burt had been conducting in Chicago for over 10 years. It had almost a 90 percent success rate in stopping disease progression. I just remember thinking, this has been happening since before I was born, and none of the many neurologists I have seen have even thought to mention it. This is a government trial with results posted in JAMA, and theres no way they havent heard of it. It really is a disservice to autoimmune patients worldwide.

TS: Have you heard any other stem cell success stories that have inspired you?

AS: Hundreds! I met so many other patients, even in just the two months I was in Chicago for my transplant. It is the most effective treatment for autoimmune disease, and it is saving lives.

TS: Do you have any thoughts about the future of U.S. healthcare as someone who is affected so personally by it?

AS: This experience has been very eye opening to where this countrys healthcare priorities lay. The fact is, a cure for autoimmune disease takes money away from big pharma. With all the successful trials already conducted with HSCT, statistically there is no logical reason it is not yet approved for treatment of MS. Stem cells have so much potential for future use; I have even seen ALS being treated with a similar protocol in the news very recently.

TS: I saw your Go Fund Me page that hit over its $115,000 goal. What was it like to get that kind of support?

AS: I never expected that level of support. It just went viral overnight. I remember the amount was stagnant for weeks, and one morning I woke up and it was over eighty thousand. Every time I refreshed the page it went up even higher.

I was crying all day, I couldnt believe this was all happening so quickly.

For the first time in years, I felt without a doubt that everything was going to turn out okay.

TS: What is like being M.S. free after so long?

AS: Its very surreal. I dont think Ill even completely believe it until I get my repeat images. Its crazy trying to comprehend that this is it. I notice improvements all the time; for example, I used to have to hug the rail on stairs and slowly guide myself down, and now I fly down hands free most days, not even thinking about it. I feel like I have my life back, and its the biggest blessing I have ever received.

TS: What is your relationship with your body now?

AS: I feel much more confident. Looking back at what I was able to get through reminds me not to sweat the little stuff. Being diagnosed with a chronic debilitating illness at 16 and completing an intense stem cell transplant three years later is not a small feat. I try to remember all I was able to accomplish.

I have lingering fears of course. This treatment doesnt heal existing nerve damage immediately, so I still have days where my symptoms resurface. I have to remember not get discouraged by the bad days; recovery is a roller coaster.

TS: What are you hoping for by getting your story out?

AS: I am hoping that this story can get to as many people as possible. Millions are affected by autoimmune disease or know someone who is, and if one person pursues a stem cell transplant after hearing my story, that makes it all worthwhile.

TS: What do you want people to know about MS or stem cell research?

AS: I want to make sure people know that if you choose to pursue a stem cell transplant, it may well be one of the hardest things you ever have to go through. At times it will be scary; you may feel weaker than you ever have and you may question your ability to cross the finish line, but a lifetime with an unpredictable chronic illness is much more terrifying. This is the best treatment out there. It gives second chances and a newfound appreciation for what it means to be in good health.

TS: Whats your next step?

AS: I will be returning to Chicago periodically for the next 5 years to get follow-up imaging. These MRIs will confirm my M.S. is in permanent remission. In the meantime, I am back at school pursuing a degree in Nutrition and Dietetics. I plan to take my life back and live it to the fullest. Never take your good health for granted, it is one of the greatest gifts you could ever receive.

Alexa SilversM.S.multiple sclerosis

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This University of Nevada Student Proved Doctors Wrong About MS - Study Breaks

Spokane woman willing to fight rare disease to the end – MyNorthwest.com

Cat Davis' body is hardening on the inside and outside due to a disease called Scleroderma. (Cat Davis)

There are walks, runs, auctions and GoFundMes for every cause under the sun, and theyre all vying for your money. That, perhaps, makes it even more difficult to get people to pay attention to Scleroderma, a disease that hardens organs to the texture of leather and has no cure.

But there is Cat Davis.

I met Cat four years ago while working for KXLY TV in Spokane when the first attempt to save her life launched. The first thing you notice about Cat isnt her disease its her smile. A big, toothy smile that stretches from ear to ear. She beams despite her seven-year battle with Scleroderma.

Sclero means hard and derma means skin. Its a very literal translation.

If somebody touches their hand, for instance, the top of your skin, that skin moves. Mine doesnt. Thats how tight it is. My fingers are not straight, they are curled because the skin is pulling so tight. Basically, my body mass produces collagen so my skin is almost leather-like, Cat explained.

Her body is slowly hardening on the outside and on the inside.

My esophagus is completely paralyzed from it and also most of my digestive system is paralyzed, Cat said.

She was diagnosed when she was 22, and with no cure in sight, she isnt sure shell live much longer. That isnt because she hasnt tried.

Four years ago, Cat launched a successful campaign to raise enough money for a procedure doctors believed would save her life. The Spokane community together raised $150,000 for the stem-cell transplant of her own stem cells.

It worked, but only for the first two years. She relapsed.

My skin started tightening again, skin started curling, I was really devastated. After the first transplant, I saw positive things from it, but I never was super well if that makes sense. I would watch other people who had done the transplant and kind of been following their stories and they were back to work and that was never me, Cat said.

She was devastated but never deterred, despite losing her father to Alzheimers Disease just three months ago and her family losing their home of 30 years under crippling medical bills.

Cat has never lost her hope or her sense of mission. This time, she says, its not about saving just her life.

Ive had some of my closest friends die from Scleroderma over the last couple of years, and that is why I do what I do. I do it for them. I do it for myself. I do it for everyone out there with Scleroderma who doesnt have a voice. For the unheard. For the too sick to speak out. I am fighting for everyone, Cat said.

Her mission is why shes willing to be what she calls the guinea pig for another procedure a stem-cell transplant from a Scleroderma-free, perfect match. That would be her brother.

No one with her condition has ever had both stem-cell procedures. She had to convince a doctor to be the first.

Im pretty convincing. I know what I want. He was iffy at first and I just was like you have to meet me, you have to see that I am strong and I want this and I can fight. I just need you on my team. And we met and afterward he was like, Im on board, Cat said.

So, CureforCat.com has launched again with everyone she loves giving you every reason you need to support this one cause. a video on the homepage features her mother, her brothers, friends and community members who believe in her mission to fight for everyone suffering from this rare disease.

Every day I wake up and something is different. Ive lost another thing that I can do. and because we dont know whats going to happen with the transplant whats going to be reversed, whats not we need it now. We dont need it in six months or a year. I wont even be alive in a year. Thats how sick I am. So its an urgent thing and I need the money ASAP, Cat said.

She needs $500,000 for the stem-cell transplant alone. Like last time, in addition to her disease, Cat has also been battling Medicare. Scleroderma is not on their pre-approved list. Shes had six of her doctors write letters on her behalf, but still no insurance coverage.

If shes able to successfully raise the money for the procedure, shell stay down in Los Angeles for recovery and care. The risks include graft vs host disease which gives her only a 50 percent chance of survival.

Whether this transplant works or not at least I will have fought all the way until the end, Cat said.

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Spokane woman willing to fight rare disease to the end - MyNorthwest.com