REVOLVING FUNDING FOR EARLY-STAGE PATIENT-FOCUSED MEDICINE DISCOVERY STARTUPS

CF is a life-limiting disease affecting over 70,000 people globally. People with CF experience a build-up of thick sticky mucus in their lungs and other organs, which leads to the development of frequent and persistent infections. Antimicrobial resistance (AMR) is frequently seen in people with CF, who die from antibiotic failures. The discovery and development of new antimicrobials to treat infections associated with CF is, therefore, an urgent unmet need. The economic cost of infections in CF is $7.5 billion globally per year.

Luke’s Story:

Luke, 49, is often asked what is it like having CF? He describes it as “spending every day of your life with a heavy head and chest cold, being constantly tired and as if someone is holding a damp flannel over your mouth and nose the entire time.” 

Some people with CF spend huge swathes of their days (and sometimes nights too) following strict drug regimens and treatment patterns that prevent them from following a ‘normal’ life; unable to continue with their education, preventing them from following their desired career paths or preventing them live a rewarding and ultimately fulfilling life. The hope for every CF patient is that as treatments improve and new medications are developed, an element of stability can be brought into their lives.”

Hearing loss and tinnitus have a big societal impact – One in six people in Europe have some form of hearing loss. Unaddressed hearing loss impedes communication, leads to social isolation, has negative impacts on employment prospects, mental health and wellbeing and is also a major risk factor for other conditions such as dementia. Tinnitus is another related condition that is often, although not always associated with the development of hearing loss and is estimated to affect 10% of adults. Tinnitus can lead to further social isolation, increased stress, anxiety and, in extreme cases, suicide. The economic cost of hearing loss is estimated to be over US $980 billion globally per year.

Now is the time to invest in medicines for hearing. Many scientific advances, for example in stem cell research, in the discovery of genes linked to hearing loss and in gene therapy, are being made and this coupled with increasing interest from the pharmaceutical sector means that with investment, it is possible that we could see the first treatments to prevent or restore hearing loss or silence tinnitus for some people with some conditions within the next 5-10 years.

Hugh’s Story:

Hugh has a form of hereditary deafness that is progressive over time and affects both ears. 

“If you aren’t deaf it’s hard to understand how much of communication is non-verbal. I also suffer from tinnitus, it’s a constant noise and in order to communicate I have to concentrate so hard that social interactions are exhausting for me.”

“I could have a cochlear implant in my left ear but I’m reluctant to do that until I absolutely have to because new medicines round the corner might just be able to regain enough hearing for me to be able to use a hearing aid again – that would be a major benefit for me”.

The scale and impact of mental illness across society is huge. One in ten children and one in four adults (approx. 15 million people) experience mental illness each year, affecting their wellbeing, relationships, and potential ability to work. The economic and social cost has been estimated as £105bn a year in England alone. 

There have been no new effective types of treatment for psychiatric disorders for over 30 years and current treatments are ineffective with detrimental side effects, and finding the right treatment is typically a process of trial and error. New treatments are needed urgently.

Deepanshi’s Story

Deepanshi has been living with OCD, anxiety and ADHD for 11 years. She is an MQ Ambassador, and has created an online voluntary support group ‘Rain on Me’, to give people space to be heard.

‘My panic attacks started around the age of 14. Even the slightest bit of tension triggered me. I would scream and cry uncontrollably if my parents tried to comfort me. And by 15, I was sleeping with loud music every night. That was the only way I knew how to fall asleep. I was terrified of the silence.’

‘It took me a decade to open up to someone about what I was going through, and I felt completely unheard. I was put in a box. If there was more mental health research, the psychiatrist might’ve been able to put me on a spectrum, not a box.’

Want to read Deepanshi’s story? Dive deeper here.