There’s probably nothing worse for a parent to hear than that their two young children have a rare brain disease that will kill them within 5-10 years.
But that’s precisely what happened to Brooklyn couple Gary and Jennie Landsman.
It all started when Jennie realized that their 8-month old son Benny, was still unable to sit up, support his head or roll over.
The young parents took him to several neurologists back in 2017 until they discovered that both he – and his new baby brother, Josh, (who was born a month earlier) suffered from a rare neurological disease called ‘Canavan disease’.
This genetic neurological disorder is not only progressive, but also fatal. However before it kills, this cruel disorder ensures that the children who get it will be unable to walk, talk and even swallow, giving them a lifespan of just 5 to 10 years.
“It was the end of the world” Jennie recalls after hearing that both of her sons were diagnosed with the disease.
But Jennie wasn’t going to take the decree lying down. The Landsman’s are determined to find a cure even though there is none available.
Unfortunately, they can’t rely on pharmaceutical companies to find a cure. That’s because there isn’t much financial incentive for these corporations to develop drugs for rare diseases. “Pharmaceutical companies are not really interested in developing drugs for rare diseases because developing a drug is outrageously expensive and a gene therapy drug is even more expensive than any other drug” explains director of the Cell and Gene Therapy Center at Rowan University’s School of Osteopathic Medicine in New Jersey Dr. Paola Leone.
So the Landsmans are taking matters into their own hands. Instead of relying on pharmaceutical companies to fund a treatment for the disease, these desperate parents are raising the money themselves.
This treatment will cost at least $1.2 million – money that the Landsman’s just don’t have. But if they can raise the funds, it will allow Dr. Leone to finish developing the cutting-edge therapeutic. “The hope is that they will lead full, functional lives,” says Leone, whose gene therapy has already demonstrated success in mice.
So far, Jennie and Gary’s community has come together to help them raise the funds. But they need to go outside their friends and family to reach their goal.
That’s why the Landsmans are currently running a crowd-funding campaign to raise enough money to bring the treatment to market and save not just their children, but other children worldwide who suffer from Canavan.
In the meantime, there is some good news. Although many people have taken a financial hit with the corona-crisis, in the last two weeks of May alone, over 7,000 new donors stepped up and donated generously. “We needed $650k to make the May 31st payment deadline of the drug manufacturing company” Jennie said. “Over $550K has been raised and we have pledges for another $100K. These donations have allowed us to make the payment and gets us to the next step, the long awaited treatment. Thank you, thank you!”
And now the final stage…the money for “clinical” costs, or hospitalization for the children, to participate in the trial.
Each child will require approximately 3 weeks in the hospital (not covered by insurance companies). These three weeks consist of one week prior to the operation (to deliver the gene therapy) for monitoring, testing, and MRI’s. Then about two weeks after the surgery the kids will need to be monitored in the ICU, with follow ups over the subsequent weeks and months to monitor the effects. The estimated cost per child is $280K. We now need to come up with the final $560K to help save Benny and Josh.
This journey has lasted over 2.5 years and it comes down to this. Over $4 Million raised & spent on research, testing, FDA filings, consultants and the production of the drug. There is a bright light at the end of this long and difficult journey. Benny and Josh making medical history, their treatment may not only cure children of Canavan disease, but it can lead to a cure for Multiple Sclerosis, ALS or even Alzheimer’s disease. Imagine being a part of saving not just our two precious souls, but millions of people. Please donate generously.
Now is your chance to help them.
That’s because this is a progressive disease which means that the older the children get, the harder it will be to treat. Everyday that goes by creates irreversible damage. Time is not on their side. That’s why if you want to donate, you need to do it now.
This is your chance. Donate to the Chesed Fund and save a child’s life today.
Source: Israel in the News