Race against time for two-year-old with 'childhood dementia' as parents plead for funding
The parents of a two-year-old girl suffering from 'childhood dementia' have issued an impassioned plea for more research into the rare condition.
Listen to this article
Leni Forrester, two, has Sanfillippo disease - a rare genetic condition often described as childhood dementia.
For the first time, Leni's parents Gus and Emily have spoken about the need for more funding and support for the condition.
“All your dreams for your child's future are taken away,” Emily told the broadcaster.
“To be told that she has this condition, and there is no treatment and no cure and no support… it’s completely earth-shattering.
“It is every parent's worst nightmare.”
Read More: Mother with rare cancer calls for reform of ‘unfair’ school places policy
Without treatment, the disease will steadily damage their daughter’s brain cells.
“Every day that passes without treatment, this toxic waste is building up in our child’s body,” Emily said.
“If we can’t get treatment, she will suffer the most awful physical and mental decline you can imagine and then die in her early to mid-teens.”
A new clinical trial for a potential treatment is expected to begin in the United States later this year.
Leni's parents are now calling on the Government to help fund the research so that UK patients can also be included in the trial.
Emily added: “Early treatment is key for these children. The damage cannot be reversed once it’s done.
“If she has to wait six months, that could mean she can no longer talk. If she waits 12 months, that could mean she loses the ability to walk.
“It’s extremely frustrating because the science is there. The data is there. It’s proven to be effective, and yet we cannot access it.”
The Forresters have raised more than £200,000 to access treatments for Leni and to help put pressure on the Government to fund clinical trials.
They have said that if they are unsuccessful in accessing these treatments, the money will be donated to Great Ormond Street Hospital, and to helping improve Leni's quality of life.
Another option for Leni could be a gene therapy developed by Professor Brian Bigger at the University of Edinburgh.
The nascent treatment sees a missing gene into patients’ blood stem cells - but cannot reach clinical trials without significant funding.
Professor Bigger told ITV: “We’re racing against time. She will start to lose brain cells and when that happens we’re not going to get them back.”
“Charities typically can’t fund this kind of thing,” he said. “It would be really good if we could see more commitment from the Government towards these kinds of therapies.
“There are hundreds of kids like Leni out there who don’t have any therapy, and these treatments have the potential to be transformative.”